BioShares Biotechnology Clinical Trials (BBC): $23.50, +$0.53, +27.6% YTD

BioShares Biotechnology Products (BBP): $38.29, +$0.92, +16.9% YTD

MARKET COMMENTARY

As concerns over Donald Trump’s setback on healthcare reform waned, U.S. stock index futures inched higher. Wholesale inventory, CaseShiller index for house prices and consumer confidence data are on the radar. European shares rose, buoyed by strong earnings and deals across the region, and Asian stocks closed higher. The dollar steadied on talks of more U.S. interest rate hikes this year, while gold slipped. Oil prices rose on supply disruption in Libya and the latest comments from officials suggesting OPEC could extend its deal cutting global production.

MARKET HIGHLIGHTS

Rockwell Medical received notice from the State Intellectual Property Office of the People’s Republic of China (SIPO) that patent claims relating to ESA Sparing, Compositions and Use for Triferic have been accepted for grant in China. Triferic is the only FDA approved therapy indicated to replace iron and maintain hemoglobin in hemodialysis patients who suffer from anemia. The claims allowed in the patent application, entitled “Methods of Treating Iron Deficiency with Soluble Ferric Pyrophosphate” include composition of matter and use of soluble ferric pyrophosphate to reduce ESA usage using hemodialysis solution with the specific dose. The patent provides protection through February 2034.

DelMar Pharmaceuticals will present three abstracts at the American Association of Cancer Research (AACR) Annual Meeting. The abstracts are based on research conducted with DelMar’s lead anti-cancer product candidate, VAL-083 (dianhydrogalactitol), a "first-in-class" small-molecule DNA-targeting agent. The AACR Annual Meeting will be held April 1-5, 2017 in Washington, D.C.

Catalyst Biosciences announced that the Korean Ministry of Food and Drug Safety (MFDS) approved the Investigational New Drug Application (IND) for CB 2679d/ISU304. Hemophilia is a serious bleeding disorder that results in spontaneous bleeding episodes as well as substantially prolonged bleeding times upon injury. 

BioTime announced the successful defense of two key patents from challenge before the European Patent Office (EPO) Opposition division. A large pharmaceutical company and an anonymous filer challenged the EPO’s previous grant of two European patents (EP2554661 and EP2147094), which cover the proprietary directed differentiation methods to produce pluripotent stem cell-derived cell replacement therapies being developed to treat retinal degenerative diseases such as age-related macular degeneration (“AMD”). BioTime addressed the challenges at the EPO headquarters in Munich, Germany during public oral hearings, which took place March 16 -17, 2017.

Aradigm Corporation announced financial results for the fourth quarter and full year ended December 31, 2016. The net loss for the year ended December 31, 2016 was $32.9 million, or $2.23 per share, compared with a net loss of $17.2 million, or $1.17 per share, in 2015. As of December 31, 2016, the Company’s cash and cash equivalents totaled $22.6 million.

Marrone Bio Innovations and Evogene Ltd announced that MBI will advance certain novel bacteria and Evogene-identified related proteins into MBI’s bioinsecticide product development pipeline under their previously announced multi-year collaboration for the discovery and development of novel insect control solutions.

OncoSec Medical will present preclinical results from recent studies describing the latest developments of its gene delivery platform, at a poster session at the upcoming American Association of Cancer Research (AACR) Annual Meeting, in Washington, D.C., on April 1- 5, 2017.

Kiadis Pharma N.V. announced that the Pediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the Company’s Pediatric Investigation Plan (PIP) for ATIR101™ for the adjunctive treatment in hematopoietic stem cell transplantation (HSCT) for a malignant disease. In addition, the PDCO has agreed that the Company may defer conducting the studies defined in the PIP until after it files a Marketing Authorization Application (MAA) in Europe for the use of ATIR101™ for the treatment of blood cancers.

Lipocine will present a company overview at the 16th Annual Needham Healthcare Conference on Tuesday, April 4, 2017 at 9:20 a.m. ET.

Albireo Pharma will provide a corporate overview at the 1^st Annual HCWainwright & Co NASH Investor Conference on Monday, April 3^rd @ 11amET, and at the 16^th Annual Needham Healthcare Conference on Tuesday, April 4^th @ 3pmET.

VBL Therapeutics will provide a corporate overview at the H.C. Wainwright 1st Annual NASH Investor Conference on April 3 @ 8:20amET.

RXi Pharmaceuticals announced that it was granted a patent from the Japan Patent Office (JPO) for the composition of matter of sd-rxRNAs targeting connective tissue growth factor (CTGF) for the treatment or prevention of fibrotic disorders, including but not limited to skin fibrosis and proliferative retinopathy (Japanese Patent #: 6060071).  This patent includes the Company’s lead clinical candidate RXI-109, an sd-rxRNA therapeutic compound, which is currently being evaluated in Phase II clinical trials. The patent, once issued, will be set to expire in 2031.

Cara Therapeutics announced positive top-line results from Part A of its Phase II/III trial showing that I.V. CR845 met both primary and secondary endpoints for efficacy (reduced itching and improved quality of life, respectively) in patients with uremic pruritus (UP) with statistical significance. UP is an intractable and debilitating systemic itch condition with a high prevalence in patients with chronic kidney disease (CKD), for which there are no approved therapies in the United States.

Calithera Biosciences announced that it has achieved pharmacokinetic and pharmacodynamic goals for CB-1158 which, under its agreement with Incyte, entitles the Company to receive a $12 million payment from Incyte.

TESARO announced that the FDA has approved ZEJULA (niraparib), an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, for the maintenance treatment of women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response (CR or PR) to platinum-based chemotherapy. ZEJULA is the first PARP inhibitor to be approved by the FDA that does not require BRCA mutation or other biomarker testing. TESARO anticipates launching ZEJULA in the U.S. in late April. Following the news, SunTrust analyst Peter Lawson increased his price target to $235 from $219; Wedbush analyst David Nierengarten increased his price target to $174 from $164; Bank of America analyst Tazeen Ahmad increased her price target to $179 from $165.

TESARO announced a substantial expansion of its niraparib clinical development program. Following the landmark results of the Phase III NOVA trial of niraparib, a comprehensive portfolio review, and the FDA approval of ZEJULA (niraparib) for patients with recurrent ovarian cancer, TESARO is implementing its plans to initiate registration strategies in the settings of metastatic ovarian, breast and lung cancers.

Cempra announced that the company has withdrawn its MAA seeking EMA approval of oral capsule and intravenous formulations of solithromycin for the treatment of community-acquired pneumonia in adults.

AstraZeneca announced that the FDA has accepted the company’s NDA for LYNPARZA (olaparib) tablets (300mg twice daily) for use in platinum-sensitive, relapsed ovarian cancer patients in the maintenance setting. The FDA has also granted priority review status with a PDUFA set for third quarter 2017. The NDA submission includes the LYNPARZA Phase III SOLO-2 trial data, which showed a reduced risk of disease progression by 70 percent, compared with placebo in germline BRCA-mutated patients. SOLO-2 trial results were presented on March 14(th) at the Society of Gynecologic Oncology Annual Meeting on Women’s Cancer.

AcelRx Pharmaceuticals announced that the EMA has notified the company that the ARX-04 (sufentanil sublingual tablet, 30 mcg) MAA has passed validation, and that the scientific review of the MAA is underway. The MAA for ARX-04 (known as DSUVIA in the U.S.) was filed in early March 2017 for the treatment of patients with moderate-to-severe acute pain in a medically supervised setting. AcelRx expects an opinion on the MAA from the CHMP in the first half of 2018.

InVivo Therapeutics Holdings announced that the patient enrolled in January in the INSPIRE study of the Neuro-Spinal Scaffold has improved from a complete AIS A spinal cord injury (SCI) to an incomplete AIS B SCI in the time between the one-month and the two-month evaluations. This is the seventh out of the 11 patients (63.6% conversion rate) with at least one month of follow-up to have had an AIS grade improvement. Two additional patients are early in follow-up and have not yet had their one-month visit. The AIS conversion rate observed in the INSPIRE study to date is considerably higher than published rates observed in a range of SCI natural history databases that are all below 25%.

Kadmon Holdings announced preclinical data demonstrating the importance of the Rho-associated coiled-coil kinase (ROCK) signaling pathway in the pathogenesis of pulmonary fibrosis, supporting the therapeutic potential of ROCK inhibition to treat the disease. The data will be presented today as a poster at the 2017 Keystone Symposia on Injury, Inflammation and Fibrosis, taking place in Snowbird, UT.

AstraZeneca announced new results data which evaluated the cost-effectiveness of SYNAGIS (palivizumab) for respiratory syncytial virus (RSV) in preterm infants 29-34 weeks gestational age compared to those who did not receive SYNAGIS. These results, derived from age-specific information on the incidence and cost of RSV hospitalization and cost of SYNAGIS, demonstrated that SYNAGIS may reduce overall costs in infants born at 29-32 weeks gestational age who are <3 months of age over a one-year period. These data were presented as a poster at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting 2017 in Denver, CO.

Ligand Pharmaceuticals announced that CNA Development LLC, an affiliate of Janssen Pharmaceuticals, Inc. has filed an IND for an antibody discovered using Ligand’s OmniAb technology. This IND filing results in a $1 million milestone payment to Ligand.

Fibrocell Science announced that it received notice from The NASDAQ Stock Market LLC (NASDAQ) on March 27, 2017 indicating that the Company has regained compliance with the minimum bid price requirement under NASDAQ Listing Rule 5550(a)(2) for continued listing on The NASDAQ Capital Market. Accordingly, Fibrocell is in compliance with all applicable listing standards and its common stock will continue to be listed on The NASDAQ Capital Market and NASDAQ considers the matter closed.

CRISPR Therapeutics reported that the European Patent Office (EPO) has announced its intention to grant a patent broadly covering CRISPR’s in-licensed gene editing technology. The claims are directed to the CRISPR/Cas9 single-guide gene editing system for uses in both non-cellular and cellular settings, including in cells from vertebrate animals such as human or mammalian cells – as well as composition claims for use in any setting, including claims for use in a method of therapeutic treatment of a patient.

Icagen announced a collaboration with Bayer to develop new X-ray fluorescence assays using the proprietary XRpro platform.  These assays are designed to help Bayer to progress various early discovery programs in the area of transporter targets.

Ampio Pharmaceuticals announced that it entered into a Waiver and Consent Letter Agreement with CVI Investments, amending the terms of warrants previously issued to CVI on September 1, 2016.

AbbVie and M2Gen announced that AbbVie has joined the Oncology Research Information Exchange Network (ORIEN) Avatar Research Program. Launched in April of 2016, the ORIEN Avatar Research Program fosters collaboration among key stakeholders in cancer research with the shared goal of discovering and developing novel therapies in clinical trials.

Sofinnova Partners has appointed Antoine Papiernik as Chairman. Papiernik succeeds Denis Lucquin who remains a Managing Partner. The appointment starts a new chapter in Sofinnova Partners’ growth strategy.

ERS Genomics announced that the European Patent Office intends to grant Dr. Emmanuelle Charpentier, ERS Genomics’ co-founder, together with the University of California and University of Vienna, a patent with very broad claims covering the use of CRISPR-Cas9 technology for gene editing. The claims intended for grant by the EPO are directed to the widely-used single-guide CRISPR-Cas9 gene editing system and cover uses in both cellular and non-cellular settings, including use in bacteria, plants, animals, and cells from vertebrate animals such as humans.

epliCel Life Sciences reported compelling safety and clinical data from its phase I/II tendon repair study investigating the use of RepliCel’s type 1 collagen-expressing, hair follicle-derived fibroblasts (RCT-01) as a treatment for Achilles tendinosis.

Omeros announced presentation of a case report describing resolution of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) in a dialysis- and transfusion-dependent adolescent girl who was treated with OMS721 under a compassionate-use protocol. The presentation “Resolution of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal antibody OMS721 in a pediatric HSCT recipient” occurred at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation in Marseille, France on Monday, March 27, 2017. Marco Zecca, M.D., Director of Pediatric Oncology at the Fondazione IRCCS Policlinico San Matteo, presented the data. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

AVEO Pharmaceuticals announced that it has priced an underwritten public offering of 30,000,000 shares of common stock at a price to the public of $0.50 per share, before underwriting discounts, which would result in aggregate gross proceeds of approximately $15 million. All of the shares in the offering are to be sold by AVEO. AVEO has also granted the underwriter a 30-day option to purchase up to an additional 4,500,000 shares of common stock on the same terms and conditions. Closing of the offering is expected to occur on or about March 31, 2017, subject to customary closing conditions. Piper Jaffray & Co. is acting as the sole bookrunner for the offering.

The Parker Institute for Cancer Immunotherapy, Bristol-Myers Squibb and the Cancer Research Institute announced a multi-year clinical research collaboration to coordinate and rapidly initiate clinical Immuno-Oncology (I-O) studies across the Parker Institute and CRI networks. Bristol-Myers Squibb will work closely with leading Parker Institute and CRI scientists and researchers, soliciting clinical research proposals from their networks and coordinating multi-site collaboration clinical studies to pursue some of the most difficult questions in cancer research. It will provide scientists with an ecosystem of advanced translational tools, precision immunotherapy and cutting-edge bioanalytical expertise to maximize learning and ensure the generation of high quality data to inform future development.

Millendo Therapeutics announced the initiation of a Phase II clinical trial evaluating the safety and efficacy of a novel oral drug candidate, ATR-101, in patients with endogenous Cushing’s syndrome (CS), a rare endocrine disease characterized by increased cortisol production from the adrenal glands. ATR-101, an adrenal-selective small molecule inhibitor of ACAT1, is also being studied in an ongoing Phase II clinical trial in patients with classic congenital adrenal hyperplasia (CAH) and a Phase I clinical trial in patients with adrenocortical carcinoma (ACC).

Akebia Therapeutics announced the publication of positive results from a Phase IIa study of vadadustat, a once-daily oral HIF stabilizer in development for the treatment of anemia related to chronic kidney disease (CKD). The study demonstrated that vadadustat increased hemoglobin levels in a dose-dependent manner and improved iron mobilization in non-dialysis CKD patients when compared to placebo. The peer-reviewed paper, titled “Clinical Trial of Vadadustat in Patients with Anemia Secondary to Stage 3 or 4 Chronic Kidney Disease,” was published online by the American Journal of Nephrology. Vadadustat is currently being evaluated in a global Phase III clinical program in non-dialysis and dialysis patients with anemia related to CKD.

CytoSorbents announced the issuance today of U.S. Patent No. 9,604,196 entitled, "Size Selective Hemocompatible Polymer System".

Compugen announced the selection of COM902 as the lead clinical antibody candidate for its CGEN-15137/TIGIT T cell checkpoint inhibitor program in immuno-oncology. COM902 follows COM701 into the Company’s preclinical development pipeline. COM701 is the Company’s lead therapeutic antibody targeting PVRIG, for which IND is anticipated later this year. As previously disclosed, PVRIG and TIGIT represent two distinct but complementary arms of the same biological pathway, and inhibition of the two results in increased activation of tumor infiltrating lymphocytes (TILs). This provides a strong clinical rationale for the combination of COM701 and COM902, in addition to monotherapy use, as immunotherapies to treat various cancer types.

Nuvo Pharmaceuticals announced that it has entered into an exclusive license agreement with Sayre Therapeutics PVT to distribute, market and sell Pennsaid 2% in India, Sri Lanka, Bangladesh and Nepal (the Territory).  Nuvo has received an upfront payment and is eligible to receive milestone payments and a double-digit royalty on net sales.  Nuvo will supply Pennsaid 2% to Sayre on an exclusive basis from its manufacturing facility in Varennes, Québec.

Orexigen Therapeutics announced that its wholly owned subsidiary, Orexigen Therapeutics Ireland, and Bruno Farmaceutici S.p.A. have executed a distributorship agreement for Mysimba (naltrexone HCl / bupropion HCl prolonged release) in Italy. Mysimba is approved by the EMA for the management of weight in adult patients (≥18 years) with an initial Body Mass Index (BMI) of ≥ 30 kg/m2 (obese), or ≥ 27 kg/m2 to < 30 kg/m2 (overweight) in the presence of one or more weight-related co-morbidities (e.g., type 2 diabetes, dyslipidaemia, or controlled hypertension).

Therapix Biosciences announced the closing of its public offering in the U.S of 2,000,000 ADSs, each ADS representing 40 ordinary shares of the Company, at a price of $6.00 per ADS. In addition, Therapix has granted the underwriters a 45-day over-allotment option to purchase up to 300,000 additional ADSs at the public offering price. The gross proceeds to Therapix from this offering were $12,000,000, prior to deducting underwriting discounts and offering expenses. Therapix plans to use the net proceeds from this offering to advance the formulation and clinical development efforts for its two lead product candidates, including Phase II clinical trials, and for working capital and other general corporate purposes. Laidlaw & Company is acting as sole book running manager for the offering.

Calithera Biosciences announced the closing of its previously announced public offering of 7,854,500 shares of common stock, including 1,024,500 shares sold pursuant to the underwriters’ exercise in full of their option to purchase additional shares. Gross proceeds from the offering at a public offering price of $10.25 per share, before underwriting discounts and commissions and offering expenses, were approximately $80.5 million. Leerink Partners acted as sole book-running manager for the offering. Wells Fargo Securities acted as lead manager and JMP Securities acted as co-manager for the offering.

Perrigo Company announced it has completed the divestiture of its rights to the royalty stream from global net sales of the multiple sclerosis drug Tysabri (natalizumab) to RPI Finance Trust, an affiliate of Royalty Pharma ("RPI"). The transaction comprises a total consideration of $2.2 billion in cash and up to $650 million in royalties earned if global net sales of Tysabri meet specific thresholds in 2018 and 2020.

Mylan N.V. announced the appointment of Daniel M. Gallagher as chief legal officer. He will be joining the company on April 17, 2017.

ANALYST RECOMMENDATIONS

HC Wainwright analyst Shaunak Deepak increased his price target of Calithera to $14 from $10, citing the addition of CB-1158 to the valuation on the strength of the partnership.

Aegis analyst Difei Yang initiated coverage of Novocure with a “buy” rating and $14 price target, citing Novocure has a ground breaking technology named Tumor Treating Fields (TTFields) which has been approved for use in treating glioblastoma (GBM) patients based on well controlled clinical trials that an impressive 4-5 months in overall survival benefits was demonstrated.

Maxim analyst Lauren Chung initiated coverage of VistaGen Therapeutics with a “buy” rating and $4 price target, citing VistaGen’s AV-101, is part of an emerging class of antidepressant drugs targeting the NMDA receptor for adjunctive treatment of MDD and has the potential for rapid relief of depressive symptoms within days (vs. months).

Chardan analyst Madhu Kumar initiated coverage of the following companies: CRISPR Therapeutics with a “buy” rating and $23.50 price target; Editas with a “buy” rating and $65 price target; Intellia Therapeutics with a “buy” rating and $19 price target; ToolGen with a “buy” rating and KRW162,500 price target.

BioShares Biotechnology Clinical Trials (BBC): $23.22, -$0.28, +26.1% YTD

BioShares Biotechnology Products (BBP): $38.05, -$0.24, +16.2% YTD

MARKET COMMENTARY

U.S. stock index futures were little changed ahead of the formal triggering of Britain’s exit process from the European Union. Pending home sales is the on the economic calendar. European shares slipped on concerns over the formal Brexit process, while Asian stocks ended higher mostly. The dollar edged higher as Federal Reserve Vice Chairman Stanley Fischer talked of more rate hikes to come, while gold fell. Oil prices gained after a severe disruption to Libyan oil supplies.

MARKET HIGHLIGHTS

Achaogen announced an upcoming data presentation at the Society for Healthcare Epidemiology in America (SHEA) Spring 2017 Conference. The presentation highlights the use of the Cerner Health Facts electronic health record dataset to estimate the prevalence of carbapenem-resistant Enterobacteriaceae (CRE).

CymaBay Therapeutics announced that Harold Van Wart, Ph.D., will retire as President and Chief Executive Officer. The Board of Directors has promoted Sujal Shah, to interim President and Chief Executive Officer. During this transition Robert Wills, Ph.D., will assume the role of Executive Chairman to provide additional continuity and support to the Company.

Vermillion announced the acceptance for publication of the original research titled, "Evaluation of a Validated Biomarker Test in Combination With a Symptom Index to Predict Ovarian Malignancy," by Renata R. Urban, MD, Alan Smith, MS, Kathy Agnew, Vinicius Bonato, PhD, and Barbara A. Goff, MD, in the International Journal of Gynecological Cancer.

Pernix Therapeutics Holdings announced financial results for the three and twelve months ended December 31, 2016.  Net loss for full-year 2016 was $169.6 million, compared to net loss of $148.3 million for the full-year 2015.  As of December 31, 2016, the Company had total liquidity of approximately $53 million, consisting of approximately $36 million of cash and approximately $17 million available to draw under our $50 million revolving credit facility.

Ignyta announced three data presentations at the 2017 Annual Meeting of the American Association for Cancer Research (AACR) in Washington, D.C.  Two poster presentations will include new preclinical data on the immuno-oncology therapeutic capabilities of RXDX-106, which represents a novel class of immunomodulatory agents that appears to restore innate immunity in preclinical models via potent inhibition of the TYRO3, AXL and MER (or TAM) family of receptors.  Additionally, the company will present its first ever data from molecularly targeting hematological malignancies with entrectinib – an orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions – in previously unexplored, molecularly defined acute myeloid leukemia (AML). Entrectinib is currently being studied in a registration-enabling Phase 2 clinical trial known as STARTRK-2.

OSE Immunotherapeutics SA reports its financial results for the year ended December 31, 2016.  As of December 31, 2016, available cash amounted to €17.8 million following the license agreement for FR104 signed with Janssen Biotech in July 2016.  As of December 2016, the Company recorded a net annual profit of €21 million as a result of the license agreement for FR104 with Janssen Biotech in July 2016 which triggered a payment of €10 million, and of the merger of Effimune and OSE Pharma to create OSE Immunotherapeutics in May 2016.

Heron Therapeutics will present at the 16th Annual Needham Healthcare Conference on Tuesday, April 4, 2017, at 8:00 a.m. ET.

Aeglea BioTherapeutics announced that it will present a corporate update at the 16th Annual Needham Healthcare Conference in New York on Tuesday, April 4 at 2:20 p.m. ET.

Vertex Pharmaceuticals announced results from two Phase III studies of the tezacaftor (VX-661) / ivacaftor combination treatment that showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV(1)) in people with cystic fibrosis (CF) ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The 24-week EVOLVE study evaluated the combination treatment in people who have two copies of the F508del mutation. This study met its primary endpoint with a mean absolute improvement in ppFEV(1) through 24 weeks of 4.0 percentage points from baseline compared to placebo (p<0.0001). The second study, EXPAND, was an 8-week crossover study that evaluated the combination treatment in people who have one mutation that results in residual CFTR function and one F508del mutation. This study met the primary endpoints of absolute change in ppFEV(1) from baseline to the average of the Week 4 and Week 8 measurements, with the tezacaftor/ivacaftor combination treatment demonstrating a mean absolute improvement of 6.8 percentage points compared to placebo (p<0.0001) and the ivacaftor monotherapy group demonstrating a mean absolute improvement of 4.7 percentage points compared to placebo (p<0.0001). Based on these results, Vertex plans to submit a NDA to the U.S. Following the news, Janney analyst Debjit Chattopadhyay increased his price target to $102 from $98; Stifel analyst Adam Walsh increased his price target to $129 from $100; Credit Suisse analyst Alethia Young increased her price target to $108 from $100; BMO analyst Do Kim upgraded the company to “outperform” from “market perform” and increased his price target to $128 from $78; JMP analyst Liisa Bayko increased her price target to $121 from $115; UBS analyst Carter Gould increased his price target to $112 from $106; Citi analyst Robyn Karnauskas increased her price target to $124 from $105. 

Sangamo Therapeutics announced the presentation of new human in vitro and animal model data demonstrating significant reduction of tau mRNA and tau protein expression using the Company’s zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation technology. These results are the first evidence of a tau lowering agent demonstrating efficacy on neuritic dystrophy in an amyloid mouse model of Alzheimer’s disease. The data were presented by Sangamo’s collaborators from Massachusetts General Hospital Alzheimer’s Disease Research Center and Harvard Medical School at the 13(th) International Conference on Alzheimer’s & Parkinson’s Diseases.

Tonix Pharmaceuticals Holding announced that it will present at The MicroCap Conference on April 4, 2017 in New York, NY. Seth Lederman, M.D., president and chief executive officer of Tonix, will provide a corporate update and an overview of Tonix’s posttraumatic stress disorder (PTSD) clinical program.

Ritter Pharmaceuticals announced topline findings from its Phase IIb/III study designed to evaluate RP-G28 in subjects with lactose intolerance. Results from the 377-subject trial show a clinically meaningful benefit to subjects in the reduction of lactose intolerance symptoms across a variety of outcome measures. The majority of analyses showed positive outcome measures and the robustness of the data point to a clear drug effect. Based on these trial results, the Company believes that the successful completion of a confirmatory Phase III program could be adequate to support a NDA submission and therefore has requested an end-of-Phase II meeting with the FDA.

Egalet announced the FDA does not object to Egalet’s distribution of promotional materials and communications to healthcare professionals regarding the abuse-deterrent properties of ARYMO ER (morphine sulfate) extended-release tablets for oral use only –CII via the intranasal route. The materials will be based on data, submitted to FDA as part of its original NDA, from in vitro laboratory studies which characterized the physicochemical abuse-deterrent properties of ARYMO ER and from the Category 2/3 intranasal human abuse liability study.

AstraZeneca and its global biologics research and development arm, MedImmune, announced that the EMA has granted orphan designation to inebilizumab (formerly MEDI-551) for the treatment of neuromyelitis optica spectrum disorder (NMOSD). Developed by MedImmune, inebilizumab is currently in Phase IIb clinical development for NMOSD.

Genentech, a member of the Roche Group, announced that the FDA approved OCREVUS (ocrelizumab) as the first and only medicine for both relapsing and primary progressive forms of multiple sclerosis. The majority of people with MS have a relapsing form or primary progressive MS at diagnosis.

Athenex announced that it has received FDA allowance to proceed into human clinical trials with the combination of oral topotecan and its proprietary P-glycoprotein (P-gp) inhibitor HM30181A. This represents Athenex’s seventh IND allowance by the FDA (6 oral anticancer drugs, one topical ointment) and is the fourth oncology clinical drug candidate in Athenex’s oral absorption platform.

Depomed announced that Arthur J. Higgins has joined the Company as President and Chief Executive Officer and member of the Board of Directors, following the resignation of James Schoeneck as President and Chief Executive Officer and member of the Board of Directors. Following the news, Mizuho analyst Irina Koffler decreased her price target to $13 from $16; JMP analyst Jason Butler decreased his price target to $16 from $33; RBC analyst Randall Stanicky decreased his price target to $19 from $22; Leerink analyst Jason Gerberry decreased his price target to $16 from $20; Roth analyst Scott Henry decreased his price target to $18 from $23; Morgan Stanley analyst David Risinger decreased his price target to $16 from $19.

Acorda Therapeutics announced results from two ongoing, long-term safety studies of CVT-301 in people with Parkinson’s that showed no differences in pulmonary function between the group receiving CVT-301 and an observational control group. These results are consistent with previously reported data from Phase IIb and Phase III clinical trials.

ARMO BioSciences announced that the first patient has been dosed in the Company’s international Phase III pivotal clinical trial to evaluate its lead investigational immuno-oncology drug AM0010 (PEGylated Interleukin-10) in combination with FOLFOX as second-line treatment for patients with advanced pancreatic cancer.

GW Pharmaceuticals announced that the EMA has granted Orphan Drug Designation to GW’s investigational product Epidiolex (cannabidiol or CBD) in the treatment of Lennox-Gastaut Syndrome (LGS), a treatment-resistant, debilitating childhood-onset epilepsy.

Axsome Therapeutics announced that the underwriter of the Company’s previously announced public offering has exercised in full its option to purchase an additional 561,497 shares. With the exercise of the underwriter’s option, total gross proceeds from the offering of an aggregate 4,304,813 shares at a price to the public of $3.74 per share are expected to be approximately $16.1 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Axsome. All shares in the offering were sold by Axsome.

Cytokinetics announced that the second cohort of the Phase II clinical trial of CK-2127107 in patients with spinal muscular atrophy (SMA), is open to enrollment. This clinical trial is designed to assess the effect of CK-2127107, a next-generation fast skeletal troponin activator (FSTA), on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. SMA is a severe, genetic neuromuscular disease that leads to debilitating muscle function and progressive, often fatal, muscle weakness. The decision to proceed to Cohort 2 follows a review of safety and pharmacokinetics data from Cohort 1 by the Data Monitoring Committee (DMC). In collaboration with Astellas, Cytokinetics is developing CK-2127107 as a potential treatment for people living with SMA and certain other debilitating diseases and conditions associated with skeletal muscle weakness and/or fatigue.

Blueprint Medicines announced that it has commenced an underwritten public offering of $150,000,000 in shares of its common stock. In addition, Blueprint Medicines expects to grant the underwriters a 30-day option to purchase up to an additional of $22,500,000 in shares of its common stock in connection with the public offering. All shares of common stock will be offered by Blueprint Medicines.

Intec Pharma provided an update on the ACCORDANCE study, the Company’s global Phase III clinical trial of the Accordion Pill Carbidopa/Levodopa (AP-CD/LD) as a treatment for Parkinson’s disease (PD) symptoms in advanced PD patients.

InVivo Therapeutics Holdings announced the appointment of Christopher McNulty as CFO, effective March 29, 2017. He will be taking over for the interim CFO, Melanie Morel-Ferris, CPA, who will remain as the company’s Senior Director, Finance and Controller.

Genomic Health announced the presentation of new results from a large multi-center validation study, which confirmed that the Oncotype DX Genomic Prostate Score (GPS) is a strong independent predictor of metastases at 10 years in prostate cancer patients across all National Comprehensive Cancer Network (NCCN) clinical risk groups.

Generex Biotechnology announced that it has entered into a securities purchase agreement with an institutional investor pursuant to which such investor has agreed to purchase in a private placement (the “Private Placement”) in multiple tranches 109,000 shares of the Company’s newly designed Series H Convertible Preferred Stock (the “Series H CPS”) and 6,000 shares of the Company’s newly designed Series I Convertible Preferred Stock (the “Series I CPS”) (together, the “Preferred Stock”) for an aggregate purchase price of $115,000,000 ($1,000 per share of Preferred Stock). The Preferred Stock is convertible into shares of the Company’s common stock at a conversion price of $2.50 per share (subject to adjustment under certain circumstances).

ANALYST RECOMMENDATIONS

JMP analyst Michael King increased his price target of Calithera to $17 from $12, citing the updated financial model to better reflect recent company developments, especially with respect to the company’s two lead clinical candidates, CB-839 and CB-1158.

William Blair analyst Katherine Xu initiated coverage of Atherys with an “outperform” rating, citing Athersys, a leader in the development of regenerative medicines, is initiating Phase III studies with its cell therapy candidate MultiStem for the treatment of acute ischemic stroke; if successful, this could herald a revolution in stroke treatment.

Cowen analyst Doug Schenkel increased his price target of Illumina to $200 from $175, citing increased confidence in the forecast for ILMN’s NovaSeq with potential for upside to 2018 EPS.

BioShares Biotechnology Clinical Trials (BBC): $23.45, +$0.23

BioShares Biotechnology Products (BBP): $38.40, +$0.35

MARKET COMMENTARY

U.S. stock index futures slipped ahead of growth data which will shed light on the health of the economy. Jobless claims data is on the radar. European shares seesawed as investors mulled implications of Britain formally beginning the process of leaving the European Union, while Asian stocks ended lower. The dollar rose against a basket of currencies, while gold dipped. Oil prices fell as ballooning U.S. inventories foreshadowed the impact of supply disruptions in Libya.

MARKET HIGHLIGHTS

Mateon Therapeutics announced 2016 financial results.  For the year ended December 31, 2016, Mateon reported a net loss of $13.7 million, similar to the net loss for the year ended December 31, 2015.  At December 31, 2016, Mateon had cash and short-term investments of $12.0 million.

VBI Vaccines announced the appointment of Avi Mazaltov as the Global Head of Manufacturing and General Manager of the SciVac manufacturing site in Rehovot, Israel.  In this new role, Mr. Mazaltov will define and manage VBI’s manufacturing activities, including SciVac’s manufacturing and commercialization operations in Israel.

Vermillion reported fourth quarter and full year ended December 31, 2016 financial results.  For the full year of 2016, net loss was $15.0 million or $(0.29) per share as compared to a net loss of $19.1 million or $(0.41) per share in 2015.  As of December 31, 2016, cash and equivalents totaled $5.2 million.

ASPiRA Labs, a Vermillion company announced it has signed an in-network, contracted agreement with TriCare South for ASPiRA’s U.S. FDA cleared, Centers for Medicare and Medicaid Services (CMS) covered, American College of Obstetricians and Gynecologists (ACOG) Level B recommended ovarian cancer risk assessment test, OVA1® (Multivariate Index Assay or MIA). TriCare South serves about 2.5 million beneficiaries in the states of Alabama, Arkansas, Florida, Georgia, Louisiana, Mississippi, Oklahoma, South Carolina, Tennessee, Texas (excluding El Paso) and Fort Campbell, Kentucky.

Ignyta announced data demonstrating the successful treatment with entrectinib – Ignyta’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions – of a patient with a primary brain tumor harboring an NTRK1 fusion. The study, exploring genetic alterations associated with glioneuronal tumors, was led by researchers at Massachusetts General Hospital and was published in Precision Oncology. Researchers discovered novel oncogenic fusions involving members of the NTRK gene family in three out of 26 patients evaluated, and reported that in a patient with a BCAN-NTRK1 fusion, treatment with entrectinib resulted in a 60 percent regression in tumor size and the resolution of clinical symptoms that was maintained for 11 months on treatment. Entrectinib is currently being studied in a registration-enabling Phase 2 clinical trial known as STARTRK-2.

Aeglea BioTherapeutics announced that it will deliver a poster presentation, “Reducing systemic arginine with arginase (AEB1102) therapy does not suppress the immune response induced by anti-PD-1 and anti-PD-L1, and exerts an additive anti-tumor and synergistic survival benefit”at the 2017 American Association for Cancer Research (AACR) Annual Meeting taking place April 1 – 5 in Washington, DC.

BrainStorm Cell Therapeutics announced financial results for the year ended December 31, 2016.  Net loss for the year ended December 31, 2016, was $5.0 million, or ($0.27) per share, compared with a net loss of $8.5 million, or ($0.46) per share, for the year ended December 31, 2015.  As of December 31, 2016, BrainStorm had approximately $10.0 million in cash, cash equivalents and short-term deposits, compared with approximately $16.0 million at December 31, 2015.

Achaogen will be presenting at Needham & Company’s 16th Annual Healthcare Conference at 10:40 a.m. Eastern Time on Tuesday, April 4^th.

CymaBay Therapeutics announced that management will provide a corporate overview at the H.C. Wainwright & Co. 1st Annual NASH Investor Conference on Monday, April 3^rd @ 11:40amET, and the BioCentury Future Leaders in the Biotech Industry Conference on Friday, April 7^th @ 1:30pmET.

TRACON Pharmaceuticals will present at the 16th Annual Needham Healthcare Conference at 2:20 pm EDT on Wednesday, April 5.

Moberg Pharma AB announced today that, following a successful test launch in Japan, Moberg Pharma’s local partner CMIC Group will initiate a national roll-out of Zanmira® Nail (Kerasal Nail®) in several major cities during the second quarter.

Progenics Pharmaceuticals announced that the Company’s registrational Phase IIb trial of its novel radiotherapeutic candidate, AZEDRA (iobenguane I 131) Injection, has achieved its primary endpoint. The open-label, multi-center study was conducted under a SPA agreement with the FDA. The trial was designed to evaluate the efficacy and safety of AZEDRA in patients with malignant and/or recurrent pheochromocytoma or paraganglioma, which are rare neuroendocrine tumors. There are currently no approved therapeutics in the U.S. for the treatment of malignant and/or recurrent pheochromocytoma or paraganglioma. AZEDRA has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA.

Omeros announced additional positive data from the company’s Phase II clinical trial of OMS721 for the treatment of serious kidney disorders, which frequently lead to end-stage renal disease and dialysis. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the complement system’s lectin pathway. The clinical trial data include results from additional study patients treated with OMS721 as well as longer-term follow-up on earlier enrolled patients. The new data corroborate and expand on trial results reported in the fourth quarter of 2016. Based on the uniformly positive data in the fully enrolled cohort of OMS721-treated patients with immunoglobulin A nephropathy (IgAN), Omeros recently met with the FDA to discuss the Phase III development program.

Karyopharm Therapeutics announced that the FDA Division of Hematology Products has lifted the partial clinical hold placed on the clinical trials for selinexor (KPT-330), enabling patient enrollment and dosing of new patients in the Company’s clinical trials of selinexor in hematological malignancies. The partial clinical hold was not the result of any patient death or any change in the safety profile of selinexor.  Enrollment may now resume in all selinexor studies in hematologic malignancies, including the STORM study in refractory multiple myeloma, the SADAL study in relapsed/refractory diffuse large B-cell lymphoma (DLBCL), and the STOMP study of selinexor and backbone therapies in multiple myeloma.  In addition, Investigator Sponsored Trials in hematologic malignancies with selinexor may resume accruing patients.

Amgen announced that the FDA has accepted for priority review the sBLA for BLINCYTO (blinatumomab) to include overall survival (OS) data from the Phase III TOWER study. The application also includes new data supporting the treatment of patients with Philadelphia chromosome-positive (Ph+) relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The application aims to expand BLINCYTO’s indication for the treatment of all patients with relapsed or refractory B-cell precursor ALL and supports the conversion of BLINCYTO’s accelerated approval to full approval.

Novartis announced that the FDA has accepted the company’s BLA filing and granted priority review for CTL019 (tisagenlecleucel-T), an investigational chimeric antigen receptor T cell (CAR-T) therapy, in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). This is the first BLA submission by Novartis for a CAR-T. The priority review designation is expected to shorten the anticipated review time by the FDA.

Upsher-Smith Laboratories announced that it has received FDA final approval of two sNDAs for Qudexy XR (topiramate) Extended-Release Capsules for use as prophylaxis of migraine headache in adults and adolescents 12 years of age and older. Topiramate is the molecule that neurologists prescribe most frequently for the prevention of migraines, and the American Academy of Neurology and the American Headache Society have given topiramate a "top tier" rating for migraine prevention in adults. A study designed to address post-marketing requirements in the United States is planned to evaluate the efficacy and safety of Qudexy XR for the prophylaxis of migraine in pediatric patients ages 6-11. Upsher-Smith was granted tentative approval of its sNDA in April 2016, but was not eligible to receive final approval until after the innovator drug’s exclusivity for the adolescent population expired in March 2017.

Agenus announced that it is reorganizing its business and operations to sharpen its focus on clinical development of its two checkpoint inhibitor antibodies and vaccine program. Agenus plans to close its Basel site and consolidate key functions to its Cambridge, UK and Lexington, MA facilities, and phase out approximately 50 positions across the organization. Additionally, Robert Stein, M.D., Ph.D., President of R&D, will retire to become a senior R&D advisor exclusive to Agenus.

Bristol-Myers Squibb and Foundation Medicine announced a collaboration that leverages Foundation Medicine’s comprehensive genomic profiling and molecular information solutions to identify predictive biomarkers such as Tumor Mutational Burden (TMB) and Microsatellite Instability (MSI) in patients enrolled across clinical trials investigating Bristol-Myers Squibb’s cancer immunotherapies. Biomarkers can be used to characterize a tumor and the tumor microenvironment, which may reveal immune-related mechanisms predictive of how a patient may respond to immunotherapy.

Anthera Pharmaceuticals announced the findings from the Extension Period of the Phase III SOLUTION, non-inferiority clinical study in cystic fibrosis patients with exocrine pancreatic insufficiency (EPI). The Extension Period was designed as an observational analysis of the long-term effects of Sollpura and Pancreaze as it pertains to weight, height, BMI, and safety.

Advanced Accelerator Applications S.A. announced a 10-year exclusive supply agreement for lutetium 177 with the University of Missouri Research Reactor (MURR). Through this agreement, MURR will supply AAA with GMP-quality lutetium 177 Chloride, the precursor for production of investigational product lutetium Lu 177 dotatate (Lutathera and other Lu 177-based therapeutics in development.

Invitae announced a major expansion of its genetic testing services with the introduction of exome sequencing and interpretation services, bringing the company’s available test menu to more than 20,000 genes.

Valeant Pharmaceuticals International‘s wholly owned subsidiary, Bausch Lomb,announced the launch of Bausch + Lomb ULTRA for Astigmatism contact lenses, an innovative monthly silicone hydrogel lens that combines MoistureSeal technology, which helps to maintain 95% of lens moisture for a full 16 hours, and OpticAlign design, engineered for stability to promote a successful first fit.

Halozyme Therapeutics announced it has been informed by SWOG, an independent network of researchers that design and conduct cancer clinical trials, that the SWOG Phase Ib/II trial evaluating PEGPH20 plus modified FOLFIRINOX chemotherapy versus modified FOLFIRINOX alone in patients with previously untreated metastatic pancreas cancer has been temporarily closed to enrollment.

Shire announced that its partner in Japan, Shionogi & Co., has received the approval of the Japanese Ministry of Health, Labor and Welfare to manufacture and market INTUNIV. INTUNIV is a new, once-daily non-stimulant indicated for the treatment of ADHD.

Mundipharma announced that mundesine, a Purine-nucleoside phosphorylase (PNP) inhibitor, has been approved for the treatment of relapsed/refractory PTCL by the Ministry of Health, Labor and Welfare in Japan. The Minister’s decision follows successful clinical trials and makes Japan the first in the world to make mundesine available for treatment.

Egalet announced that one of the company’s specialty sales forces has begun promotion of ARYMO ER (morphine sulfate) extended-release tablets for oral use only –CII. Egalet executives will commemorate the product’s official launch by ringing the Nasdaq Stock Market closing bell.

Jazz Pharmaceuticals announced that it has entered into license agreements withNippon Shinyaku for Defitelio (defibrotide sodium) and Vyxeos (cytarabine and daunorubicin liposome injection), or CPX-351, in Japan. Under the terms of the agreements, Nippon Shinyaku will receive exclusive rights to develop and commercialize Defitelio and Vyxeos in Japan in return for an upfront payment to Jazz Pharmaceuticals and subsequent payments based on the successful achievement of certain regulatory and commercial milestones. Jazz Pharmaceuticals will manufacture and supply Defitelio and Vyxeos to Nippon Shinyaku, and will receive revenue based on a percentage of product sales in Japan. Financial terms of the agreement have not been disclosed.

Lannett announced that the company voluntarily made a $25 million payment against its existing revolving credit facility.

Spectrum Pharmaceuticals announced that the University of Texas MD Anderson Cancer Center initiated a Phase II trial of poziotinib in NSCLC patients with EGFR exon 20 insertion mutations. This Phase II trial will evaluate Objective Response Rate (ORR) as the primary endpoint and is expected to yield preliminary results before year-end.

FibroGen announced that clinical results from two Phase II studies in China of roxadustat for the treatment of anemia in patients with chronic kidney disease (CKD) were published in the journal Nephrology Dialysis Transplantation. In these studies, in dialysis-dependent, and in non-dialysis-dependent CKD patients, roxadustat corrected and maintained hemoglobin levels regardless of the patients’ baseline iron repletion status, and levels of C-reactive protein (CRP), a marker of inflammation, consistent with what was seen in previously published results from the roxadustat global program. The publication, entitled “Phase 2 studies of oral hypoxia-inducible factor prolyl hydroxylase inhibitor FG-4592 for treatment of anemia in China,” is now available online at Nephrol Dial Transplant 2017 gfx011.

Takeda Pharmaceutical Company announced that data from a 6-month interim analysis of the ongoing DEN-204 trial of its live-attenuated tetravalent dengue vaccine candidate, TAK-003 (also referred to as TDV), have been published in The Lancet Infectious Diseases.

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals announced the initiation of a Phase IIb dose-ranging study of AKCEA-APO(a)-L(Rx) in patients with hyperlipoproteinemia(a) and established cardiovascular disease. The goal of the study is to determine the dose level and frequency for use of AKCEA-APO(a)-L(Rx) in a planned Phase III cardiovascular outcome study.

AbbVie announced that the FDA approved the inclusion of moderate to severe fingernail psoriasis data in the HUMIRA (adalimumab) prescribing information for patients with moderate to severe chronic plaque psoriasis. HUMIRA is now the first-and-only biologic treatment with data on fingernail psoriasis in its U.S. prescribing information.

Vanda Pharmaceuticals announced that it has entered into a license agreement with UC San Francisco (UCSF), under which Vanda will acquire an exclusive worldwide license from UCSF to develop and commercialize a portfolio of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) activators and inhibitors.

Alzheon announced the results from studies of ALZ-801 and its active molecule, tramiprosate. The new clinical data and analyses support Alzheon’s study design for the pivotal clinical program for ALZ-801, a first-in-class, oral amyloid-targeted medicine, in a genetically-defined population of patients at the Mild stage of Alzheimer’s disease, with ongoing amyloid accumulation. In addition, the company presented clinical data with pharmacokinetic and pharmacodynamics analyses that support the dose selection of ALZ-801 for the pivotal study expected to initiate in 2017.

Aura Biosciences announced that it has enrolled and dosed the first patient in its Phase Ib clinical trial of light-activated AU-011, an investigational, first-in-class targeted therapy in development for the treatment of ocular melanoma. Aura additionally announced that the FDA has granted AU-011 fast track designation for the treatment of primary ocular melanoma, also known as uveal or choroidal melanoma. This designation enables Aura to have more frequent interactions with the FDA throughout AU-011’s drug development process, as well as priority review of the NDA. 

InVivo Therapeutics Holdings announced that it has received approval from the Toronto Western Hospital’s Research Ethics Board to enroll patients as part of its cervical spinal cord injury study.

CBT Pharmaceuticals announced that the first patient has been enrolled in the genolimzumab (CBT-501) Phase I study, the company’s first clinical trial. The trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of genolimzumab in patients with advanced solid tumors, recurrent or refractory to standard of care therapies. The trial also aims to determine any dose limiting toxicities or biologically relevant dose and establish a recommended Phase II dose for future clinical studies. Once the dose and schedule is established, two or more select tumors will be evaluated for preliminary efficacy, and secondarily, progression free survival.

Cellectar Biosciences announced the initiation of a Phase II clinical study of its lead phospholipid drug conjugate (PDC) CLR 131 in patients with multiple myeloma and other hematologic malignancies.

Sun BioPharma, provided top-line, interim data from the Phase I dose-escalation phase of its clinical study and financial results for the year ended December 31, 2016.

Anavex Life Sciences announced the filing of an order of dismissal of the appeal in the Cortina v. Anavex Life Sciences Corp. et. al. lawsuit, without payment of any fees or costs by the Company.  The withdrawal represents a full and final conclusion of the lawsuit.

Exelixis announced that it has repaid all amounts outstanding under its term loan with Silicon Valley Bank initiated in 2010 and which was due for repayment on May 31, 2017. The $80.1 million payment included $80.0 million in principal and approximately $60,000 in interest outstanding. Exelixis also plans to eliminate another source of indebtedness later this year by retiring the Deerfield Notes, a series of Convertible Secured Notes issued to entities associated with Deerfield Management Company due July 1, 2018.

Tonix Pharmaceuticals Holding announced the pricing of an underwritten public offering of 1,800,000 shares of its common stock at a public offering price of $4.45 per share. The gross proceeds to Tonix from this offering are expected to be $8,010,000. Tonix intends to use the net proceeds from this offering to support the continued development of TNX-102 SL for the treatment of PTSD, including the HONOR study in military-related PTSD, to further develop other pipeline programs, for working capital and other general corporate purposes, and possibly acquisitions of other companies, products or technologies, though no such acquisitions are currently contemplated. Tonix has granted the underwriters a 45-day (or as otherwise specified in the underwriting agreement) option to purchase up to 270,000 additional shares of common stock at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about April 4, 2017. Aegis Capitalis acting as the sole book-running manager for the offering.

Bellicum Pharmaceuticals announced the closing, on March 29, 2017, of its previously announced underwritten public offering of 5,750,000 shares of its common stock, including 750,000 shares sold pursuant to the underwriters’ full exercise of their option to purchase additional shares, at a public offering price of $12.00 per share. The aggregate offering size was approximately $69.0 million, before deducting the underwriting discounts and commissions and other offering expenses. Citigroup and Jefferies acted as lead book-running managers for the offering. Guggenheim Securities, Ladenburg Thalmann, Raymond James and SunTrust Robinson Humphrey acted as co-managers.

Cara Therapeutics announced that it has commenced an underwritten public offering of $80 million of shares of its common stock. In addition, Cara Therapeutics expects to grant the underwriters a 30-day option to purchase up to $12 million of additional shares of its common stock on the same terms and conditions. The proceeds of the offering are expected to be used to fund the company’s clinical and research development activities, including the completion of the Phase III program for I.V. CR845 in uremic pruritus, two Phase III trials of I.V. CR845 in acute pain and a Phase IIb trial of oral CR845 in osteoarthritis pain, as well as for working capital and general corporate purposes. Piper Jaffray & Co. and Stifel are acting as joint book-running managers for the proposed offering.

Blueprint Medicines announced the pricing of an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $40.00 per share, before underwriting discounts and commissions. In addition, Blueprint Medicines has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price, less underwriting discounts and commissions. Goldman Sachs, Morgan Stanley and Cowen and Company are acting as joint book-running managers for the offering. Raymond James is acting as co-manager for the offering.

Inovio Pharmaceuticals announced that its academic and industry collaborators received a multi-year $6.95 million grant from the NIH’s National Institute of Allergy and Infectious Diseases to develop a single or combination therapy using Inovio’s PENNVAX-GP with the goal of attaining long-term HIV remission in the absence of antiviral drugs.

Cellular Biomedicine Group announced the completion of its newly expanded 30,000 square foot facility in Huishan High Tech Park in Wuxi, China. 20,000 square feet of the Wuxi GMP facility will be dedicated to advanced stem cell culturing, centralized plasmid and viral vector production, cell banking and development of reagents.  By the end of 2017, the Company anticipates that the combination of this Wuxi site, the new Zhangjiang Shanghai and the Beijing GMP facilities will be capable of supporting simultaneous clinical trials for five different CAR-T and stem cell products, or capacity to treat up to 10,000 cancer and 10,000 Knee Osteoarthritis (KOA) patients per year.

Promentis Pharmaceuticals announced it has completed a third round of equity investment totaling $26,000,000. The Series C investment is led by OrbiMed, F-PrimeCapital Partners (formerly Fidelity Biosciences) and Aisling Capital. Existing investors, including Black Pearl GmbH, the Golden Angel Network and individual investors, also participated in the Series C financing.

ACADIA Pharmaceuticals announced Michael J. Yang has joined ACADIA as Executive Vice President and Chief Commercial Officer. Mr. Yang succeeds Terrence O. Moore, who has served as ACADIA’s Chief Commercial Officer since 2013, and is now retiring.

Sarepta Therapeutics announced the addition of Kenneth Fischbeck, M.D., and Matthew Wood, M.D., Ph.D., to the Company’s Strategic and Scientific Advisory Board. Dr. Fischbeck and Dr. Wood join Dr. Beverly Davidson and Dr. Louis Kunkel, who were appointed to the board at the time of its formation in 2015.

ANALYST RECOMMENDATIONS

Cantor analyst Chiara Russo increased her price target of Paratek Pharmaceuticals to $32 from $28, citing a renewed confidence that PRTK’s omadacycline is correctly dosed for success in the IV/Oral Phase 3 in ABSSSI, that the heritage of tetracyclines should not present any safety concerns and that PK/PD and in vitro data all point to a positive read-out for pneumonia.

UBS analyst Martin Auster initiated coverage of the following companies: Alexion with a “buy” rating and $138 price target; Avexis with a “buy” rating and $95 price target; Spark Therapeutics with a “buy” rating and $70 price target; Alnylam with a “neutral” rating and $55 price target; BioMarin with a “neutral” rating and $92 price target; United Therapeutics with a “sell” rating and $112 price target; the analyst was also transitioned coverage of Esperion which he upgraded to “buy” from “neutral” and increased his price target to $52 from $23; Selecta the analyst maintained a “buy” rating and decreased his price target to $26 from $29.

SunTrust analyst Peter Lawson upgraded NewLink Genetics to “buy” from “hold” and increased his price target to $30 from $12, citing favorable risk/reward ahead of upcoming data.

HC Wainwright analyst Ed Arce upgraded Galectin Therapeutics to “buy” from “neutral” and increased his price target to $3.50 from $0.60, citing a more positive view of the therapeutic potential of the mechanism of galectin-3 inhibition.

Cowen analyst Tyler Van Buren increased his price target of Innoviva to $16 from $15, citing increased Breo estimates after Mylan announced that it received a CRL for its generic Advair program, which was first in line.

BioShares Biotechnology Clinical Trials (BBC): $23.15, -$0.30, +25.7% YTD

BioShares Biotechnology Products (BBP): $37.99, -$0.41, +16.0% YTD

MARKET COMMENTARY

U.S. stock index futures fell as investors booked profits on the last trading day of the month and quarter. Consumer spending and University of Michigan’s sentiment data are on the radar. European shares traded lower, as losses among miners and a slump in insurer Old Mutual weighed in. Asian stocks dipped. The dollar rose, benefitting from a weaker euro, while gold slipped. Oil prices fell as growing U.S. crude stockpiles posed a threat to OPEC-led production cuts.

MARKET HIGHLIGHTS

Transgene will present a poster entitled “Local and abscopal effects in oncolytic virotherapy are boosted by immune checkpoint blockade, immunogenic chemotherapy, or IFNAR blockade” with new and encouraging preclinical data of a next generation armed engineered oncolytic virus at the American Association for Cancer Research annual meeting in Washington, DC.

Catalyst Biosciences announced receipt of notice from the European Patent Office that the opposition period for a patent granted to Catalyst has expired and no opposition has been filed.

NEOVACS announced its full-year 2016 financial results.  The Company’s net loss amounted to €13.9 million in 2016, compared with €4.7 million recorded in the previous year.  At December 31, 2016, Neovacs had €3.9 million in cash. 

Windtree Therapeutics today reported financial results for the fourth quarter ended December 31, 2016.  The Company reported a net loss of $6.6 million ($0.77 per basic share) on 8.5 million weighted-average common shares outstanding for the quarter ended December 31, 2016, compared to a net loss of $10.1 million ($1.26 per basic share) on 8.1 million weighted average common shares outstanding for the comparable period in 2015.  As of December 31, 2016, the Company had cash and cash equivalents of $5.6 million.

BioDelivery Sciences International announced that two important new patents were granted extending patent protection around all three of its FDA approved products, BELBUCA® (buprenorphine) buccal film, BUNAVAIL® (buprenorphine and naloxone) buccal film, and ONSOLIS® (fentanyl buccal soluble film), further strengthening BDSI’s overall intellectual property position.

Kiadis Pharma N.V. announces its audited annual results for the year ended December 31, 2016.  The net loss for the year decreased to €14.8 million in 2016 from €16.5 million in 2015.  The cash position decreased to €14.6 million at year-end 2016 compared to €28.7 million at the end of 2015. This is mainly due to the cash used in operating activities in 2016.

Mateon Therapeutics announced that updates on several of its preclinical programs are being presented at the American Association for Cancer Research (AACR) annual meeting in Washington, D.C.

Valneva Chief Executive Thomas Lingelbach was featured in an article in New Scientist entitled Lyme Disease is Set to Explode and We Still Don’t Have a Vaccine.

The Medicrea Group published its 2016 IFRS annual results.  The Company reported a net loss of €7.6M for the year ended December 31, 2016, compared to a net loss of €1.5M in prior year period.  Available cash amounted to €8 million at December 31,2016.

Akari Therapeutics announced that the FDA has granted Fast Track designation for Coversin for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in patients who have polymorphisms conferring eculizumab resistance. Coversin is a second-generation complement inhibitor that acts on complement component-C5, preventing the release of C5a and the formation of C5b–9 (also known as the membrane attack complex or MAC), and independently also inhibits LTB4 activity.

Incyte and Merck announced additional details of their clinical development program investigating the combination of epacadostat, Incyte’s investigational oral selective IDO1 enzyme inhibitor, with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, in patients across five tumor types: metastatic melanoma, non-small cell lung cancer (NSCLC), bladder cancer, renal cell carcinoma (RCC), and squamous cell carcinoma of the head and neck (SCCHN).

Mesoblast announced that the Independent Data Monitoring Committee (IDMC) for the ongoing Phase III trial in chronic heart failure (CHF) has initiated the process for the pre-specified interim futility analysis of the trial’s efficacy endpoint. The interim analysis dataset has been locked and will be analyzed and reviewed by the trial’s independent statisticians. Throughout this review process, Mesoblast will remain blinded to individual treatment allocation as well as grouped safety and efficacy data.

Novocure announced that the last patient has been enrolled in the STELLAR trial, a Phase II pilot trial testing Tumor Treating Fields (TTFields) in combination with either pemetrexed and cisplatin or carboplatin in patients with newly diagnosed, unresectable malignant mesothelioma. The final data collection date will be 12 months after the last patient in.

AstraZeneca announced that the FDA has granted full approval for TAGRISSO (osimertinib) 80mg once-daily tablets, for the treatment of patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test, whose disease has progressed on or after an EGFR tyrosine kinase inhibitor (TKI) therapy. TAGRISSO is the first and only approved medicine in the US indicated for NSCLC patients who have tested positive for the EGFR T790M mutation, and efficacy data suggest it may be a new standard of care for these patients.

China Cord Blood Corporation announced that Ms. Yue Deng, Chief Executive Officer of the Company’s Beijing subsidiary, has resigned from the Company, effective April 1, 2017. Ms. Deng’s departure is for personal reasons and does not reflect any disagreements between Ms. Deng and the Company. The Board of Directors has named Ms. Ting Zheng, Chief Executive Officer of CCBC, to serve as interim Chief Executive Officer of the Beijing subsidiary upon Ms. Deng’s departure.

miRagen Therapeutics announced that the FDA has granted orphan-drug designation to miRagen’s product candidate, MRG-106, for the treatment of mycosis fungoides. Mycosis fungoides is the most common form of a type of blood cancer called cutaneous T-cell lymphoma (“CTCL”). CTCL occurs when certain types of T-cells become cancerous and cause debilitating tumors in the skin and in other parts of the body.

Emergent BioSolutions announced that it has signed a modification to its contract with the Biomedical Advanced Research and Development Authority (BARDA) to manufacture and store bulk drug substance for its botulism antitoxin, BAT [Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) – (Equine)], valued at approximately $53 million with a five-year period of performance. This modification to the contract will enable future filling and deliveries of final drug product to the Strategic National Stockpile (SNS). BAT is indicated for the treatment of symptomatic botulism following documented or suspected exposure to botulinum neurotoxin serotypes A, B, C, D, E, F, or G in adults and pediatric patients.

FibroGen announced the approval by the CFDA of the Company’s clinical trial application (CTA) in China for a Phase II/III pivotal trial of roxadustat in anemia associated with lower risk myelodysplastic syndromes (MDS). FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat in China, the U.S., and other major markets. FibroGen is conducting all clinical trials and regulatory submissions in both the U.S. and China, and will retain all regulatory licenses and manufacturing permits in China.

Impax Laboratories announced that Judge Richard G. Andrews of the U.S. District Court, District of Delaware, has found that the U.S. patents 6,750,237 and 7,220,767 protecting Zomig (zolmitriptan) Nasal Spray are not invalid and are infringed by the Lannett Holdings and Lannett Company ANDA . 

Johnson & Johnson announced that its Swiss subsidiary, Janssen Holding GmbH, published the provisional notice of the interim result of its all-cash public tender offer in Switzerland to acquire all publicly held shares of Actelion for $280 per share, payable in U.S. dollars, per the offer prospectus of February 16, 2017.

argenx announced the dosing of the first patient in a Phase II proof-of-concept study of ARGX-113 in patients with primary immune thrombocytopenia (ITP).

Cara Therapeutics announced the pricing of its underwritten public offering of 4,450,000 shares of its common stock at a public offering price of $18.00 per share. The gross proceeds from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by Cara Therapeutics, are expected to be approximately $80 million. In addition, Cara Therapeutics granted the underwriters a 30-day option to purchase up to 667,500 additional shares of its common stock on the same terms and conditions. The proceeds of the offering are expected to be used to fund the company’s clinical and research development activities, including the completion of the Phase III program for I.V. CR845 in uremic pruritus, two Phase III trials of I.V. CR845 in acute pain and a Phase IIb trial of oral CR845 in osteoarthritis pain, as well as for working capital and general corporate purposes.  Piper Jaffray & Co. and Stifel are acting as joint book-running managers for the offering. Canaccord Genuity, Needham & Company, Janney Montgomery Scott and H.C. Wainwright & Co. are acting as co-managers.

BioLineRx announced that it has priced an underwritten public offering of approximately 29.4 million American Depositary Shares ("ADSs"), each representing one (1) of its ordinary shares, at a public offering price of $0.85 per ADS for gross proceeds of $25 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by BioLineRx. All of the ADSs in the offering are to be sold by BioLineRx.  Delivery of the ADSs is expected to occur on April 5, 2017, subject to customary closing conditions.  BioLineRx has granted the underwriters a 30-day option to purchase up to an additional approximately 4.4 million ADSs at the public offering price. BioLineRx anticipates using the net proceeds from the offering to fund a number of clinical trials, including for BL-8040 and AGI-134, and for working capital and general corporate purposes. JMP Securities acted as sole book-running manager for the offering. H.C. Wainwright & Co., LLC acted as lead manager. Maxim Group acted as co-manager.

CytoSorbents announced the pricing of an underwritten public offering of 2,222,222 shares of its common stock at a public offering price of $4.50 per share, representing gross proceeds of approximately $10.0 million. In addition, CytoSorbents has granted the underwriters a 30-day over-allotment option to purchase up to 333,333 additional shares of common stock at the public offering price.  The offering is expected to close on or about April 5, 2017, subject to customary closing conditions. The company intends to use the net proceeds from this offering for general corporate and working capital purposes, including advancing its U.S. pivotal trial for treatment of inflammation in conjunction with cardiac surgery. Cowen and Company is acting as the sole book-running manager and representative of the underwriters for the offering.  Aegis Capital, H.C. Wainwright & Co., B. Riley & Co., Maxim Group LLC and Northland Capital Markets are acting as co-managers for the offering.

Dicerna Pharmaceuticals announced that it has signed a stock purchase agreement with a syndicate of current and new investors, led by Bain Capital Life Sciences, for the sale of redeemable convertible preferred stock for gross proceeds of $70.0 million. Other participants in the financing include EcoR1 Capital, Cormorant Asset Management, RA Capital, Domain Associates and Skyline Ventures, among others. Under the terms of the stock purchase agreement, upon closing of the Preferred Stock transaction, Adam M. Koppel, M.D., Ph.D., a managing director of Bain Capital Life Sciences, will be named to the Company’s Board of Directors, increasing the membership to nine. The Company intends to use the proceeds from the offering to further develop its GalXC pipeline programs, including both pre-clinical and clinical work, as well as for general corporate purposes. The transaction is expected to close on or before April 11, 2017, subject to the satisfaction of customary closing conditions.

ANALYST RECOMMENDATIONS

Maxim analyst Jason Kolbert increased his price target on BrainStorm Cell Therapeutics to $8 from $6, citing the company has taken strategic steps to begin Phase 3 clinical trials of NurOwn in ALS.

Raymond James analyst Reni Benjamin initiated coverage of the following companies:BioTime with an “outperform” rating and $6 price target; MacroGenics with an “outperform” rating and $26 price target.

Needham analyst Chad Messer initiated coverage of Exelixis with a “buy” rating and $28 price target, citing with two approvals now under its belt, and several more to come, Exelixis looks poised to turn profitable.

Credit Suisse analyst Erin Wilson Wright initiated coverage of Quintiles with an “outperform” rating and $89 price target, citing Q is the world’s largest contract research organization (CRO) and healthcare information & technology services company with solutions that uniquely span the clinical development and commercial drug lifecycle.

Mizuho analyst Irina Koffler initiated coverage of Dermira with a “buy” rating and $42 price target, citing an innovative dermatology "biotech" that may serve as a 2018 takeout target in a sector that has recently become more exciting with the entry of multiple large-cap pharma companies.

Maxim analyst Jason McCarthy upgraded Vertex to “buy” from “hold” and established a $143 price target, citing positive data from two pivotal studies of tezacaftor/ ivacaftor (tez/iva) in F508del homozygotes (EVOLVE) and in the first of two studies in heterozygotes (EXPAND, F508del/residual CFTR).

Credit Suisse analyst Vamil Divan increased his price target of Alkermes to $78 from $72, citing higher Vivitrol forecasts following meetings with management.

Chardan analyst Gbola Amusa initiated coverage of GenSight Biologics with a “buy” rating and €35 price target based on the opportunity likely addressed by GS010, a Phase III AAV-based gene therapy (GT) for a rapidly-progressing form of blindness, LHON.

Chardan analyst Gbola Amusa decreased his price target of Adverum Biotechnologies to $3.50 from $5, citing an updated model. 

Jefferies analyst Anthony Petrone increased his price target of Antares Pharma to $6 from $3, citing expectations of positive catalysts over next 24 months.

BioShares Biotechnology Clinical Trials (BBC): $23.19, +$0.04, +26.0% YTD

BioShares Biotechnology Products (BBP): $37.75, -$0.24, +15.3% YTD

MARKET COMMENTARY

U.S. stock index futures were trading modestly higher as focus shifted towards a meeting between the U.S. and Chinese presidents scheduled later in the week. Investors will keep an eye on total vehicle sales data along with latest reading on manufacturing activity from Markit and ISM. The dollar index edged up and was holding above four-month lows hit last week, while gold prices slipped. European shares ticked higher helped by stronger oil stocks as investors turned more confident in a sector that has been a worst performer in Europe this year. Asian stocks ended higher after upbeat economic data from China and Japan. A rebound in Libyan oil production weighed on oil prices.

MARKET HIGHLIGHTS

OncoSec Medical Incorporated presented a poster titled "Intratumoral Delivery of a P2A-linked Bicistronic IL-12 Construct Leads to High Intratumoral Expression and Systemic Anti-tumor Response" (Abstract ID # 1614) at the American Association of Cancer Research (AACR) Annual Meeting, in Washington, D.C. The poster included preclinical data demonstrating the latest developments of OncoSec’s gene delivery platform in a murine melanoma model.

DURECT Corporation will be presenting at the H.C. Wainwright NASH Investor Conference on Monday, April 3  at 4:40 p.m. Eastern Time.

VBL Therapeutics  announced that in a post-hoc, hypothesis-driven analysis of data from completed Phase 2 studies,  VB-201 appears to reduce certain liver enzymes. The analysis was conducted following the effect seen with VB-201 in pre-clinical models for non-alcoholic steatohepatitis (NASH) and renal fibrosis, in which VB-201 and VB-703, a next-generation Lecinoxoid drug candidate, reduced inflammation and fibrosis without affecting the lipid profile or steatosis.  The data will be presented today at the H.C. Wainwright 1st Annual NASH Investor Conference, at the St. Regis Hotel in New York City.

Cellect Biotechnology has received a formal notice of Intention to Grant for a patent (Application No. 11751949.6-1466) covering a key method of treatment from the European Patent Office. The allowed claims relate to the engineering of regulatory immune cells with enhanced apoptotic activity to be used for immunomodulation in treating or preventing immune-related disorders.

Kitov Pharmaceuticals Holdings announced today that the U.S. Food and Drug Administration has granted Kitov a waiver related to the $2,038,100 New Drug Application (NDA 210045) filing fee for KIT-302. KIT-302 is Kitov’s patented combination of Amlodipine Besylate-Celecoxib tablets, intended to treat osteoarthritis pain and hypertension simultaneously.  The fee waiver, which Kitov requested in accordance with sections 736(d)(1)(D) of the Federal Food, Drug and Cosmetic Act, is granted to a small business for its first human drug application submitted to the FDA for review. The Company will not be required to remit the NDA filing fee, provided that the marketing application for KIT-302 is filed prior to March 27, 2018.

Kiadis Pharma N.V. has obtained regulatory approval from the national authority in Belgium (the FAGG, the Federal Agency for Medicines and Health Products) to start its randomized, controlled, transatlantic Phase III clinical trial with ATIR101™ for acute leukemia (CR-AIR-009) in Belgium. In addition, the Company has received regulatory approval from the national authority in Germany (the PEI, the Paul-Ehrlich-Institute) to start its Phase I/II clinical trial with ATIR201™ for thalassemia (CR-BD-001).

Prothena presented clinical results from its Phase Ib multiple ascending dose study of PRX002/RG7935 in patients with Parkinson’s disease. PRX002, also known as RG7935, is an antibody under investigation as a potentially disease-modifying treatment for Parkinson’s disease and is the focus of a worldwide collaboration between Prothena and Roche. The study results were presented as part of a late-breaking oral session at the 13th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in Vienna, Austria, by Joseph Jankovic, MD, Professor of Neurology, Distinguished Chair in Movement Disorders at Baylor College of Medicine, Houston, Texas. As highlighted in the November 2016 topline press release, PRX002/RG7935 was found to have an acceptable safety and tolerability profile in patients with Parkinson’s disease, meeting the primary objective of this study. Robust CNS penetration was demonstrated by a dose-dependent increase in PRX002/RG7935 levels in CSF, and a mean concentration of PRX002/RG7935 in CSF of 0.3 percent relative to serum across all dose levels. Additional results showed a rapid, dose- and time dependent mean reduction of free serum alpha-synuclein levels of up to 97 percent after a single dose, which were statistically significant (p<0.0001), and maintained following two additional monthly doses.

Novocure announced final results from its Phase III pivotal EF-14 trial adding Optune to standard temozolomide chemotherapy for the treatment of newly diagnosed glioblastoma (GBM). Landmark analyses show a consistent and maintained improvement in overall survival at two, three, four and five years. The final results include data from all 695 patients included in the EF-14 trial with a median follow-up of 40 months. The two-year survival rate increased from 30 percent to 43 percent for patients treated with Optune together with temozolomide versus patients treated with temozolomide alone. The five-year survival rate increased from five percent to 13 percent for patients treated with Optune together with temozolomide versus patients treated with temozolomide alone. These are the best results reported for newly diagnosed GBM patients in a Phase III trial to date and represent clinically meaningful increases in landmark survival rates (hazard ratio, 0.63; p <0. 00006).

AVEO Oncology announced receipt of a $500,000 milestone payment from CANbridge Life Sciences related to a technology transfer milestone for AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate. AV-203 has demonstrated preclinical activity in a number of different tumor models including breast, head and neck, lung, ovarian and pancreatic cancers. CANbridge is planning clinical development of AV-203 in squamous cell esophageal cancer as its initial indication.

Dynavax Technologies announced today that the FDA has informed the company that the Vaccines and Related Biological Products Advisory Committee (VRBPAC) will review HEPLISAV-B [Hepatitis B Vaccine, Recombinant (Adjuvanted)] at its meeting scheduled for July 28, 2017. The scheduled VRBPAC meeting is close to the HEPLISAV-B Prescription Drug User Fee Act (PDUFA) date of August 10, 2017 solely as a function of meeting logistics. The PDUFA date remains unchanged. The FDA will communicate specific questions for the VRBPAC to address closer to the meeting date, and will post a draft agenda and draft questions on its website 48 hours prior to the meeting. HEPLISAV-B is the company’s vaccine candidate for immunization against hepatitis B infection in adults ages 18 years of age and older.

BioCryst Pharmaceuticals announced that Mundipharma has obtained regulatory approval of Mundesine (Forodesine hydrochloride) for the treatment of relapsed/refractory PTCL (Peripheral T-Cell Lymphoma) by the Ministry of Health, Labor and Welfare in Japan. Mundesine is a Purine-nucleoside phosphorylase (PNP) inhibitor developed by BioCryst, under an exclusive license with Albert Einstein College of Medicine and Victoria Link Limited. The Ministry’s decision follows successful clinical trials and makes Japan the first country in the world to make Mundesine available for treatment of PTCL.

Kite Pharma announced two plenary presentations of positive data from the primary analysis of ZUMA-1 for its lead CAR-T candidate, axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research Annual Meeting in Washington, D.C. Both presentations were given by Frederick L. Locke, M.D., the ZUMA-1 Co-Lead Investigator, and Director of Research for the Immune Cell Therapy Program at Moffitt Cancer Center in Tampa, Florida. The study met the primary endpoint of objective response rate (ORR) recorded after a single infusion of axicabtagene ciloleucel, with 82 percent (p <0.0001). These results demonstrate the treatment effect of axicabtagene ciloleucel in diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL) and transformed follicular lymphoma (TFL), which are types of aggressive NHL.

Infinity Pharmaceuticals reported updated Phase I clinical data for IPI-549, an orally administered immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma). These Phase I clinical dose-escalation data demonstrated that IPI-549 was well tolerated both as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor. Additionally, data from the more advanced monotherapy module of the study showed IPI-549 has a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile that supports once daily (QD) dosing. The data included 15 evaluable patients who received IPI-549 as a monotherapy and six evaluable patients who received IPI-549 in combination with Opdivo. The data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2017 taking place in Washington, D.C., April 1 – 5. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

Antares Pharma announced that data from the 52-week pharmacokinetics and safety Phase III study of subcutaneous testosterone enanthate delivered through the QuickShot auto injector was selected for a moderated poster presentation at the Endocrine Society Annual Meeting (ENDO 2017).  The poster will be presented today, April 3, 2017.

Cyclacel Pharmaceuticals announced the presentation by independent investigators of preclinical data demonstrating therapeutic potential of CYC065, the Company’s second-generation, cyclin-dependent kinase (CDK) 2/9 inhibitor, as a targeted anti-cancer agent. The data show that CYC065 substantially inhibited growth, triggered apoptosis, and induced anaphase catastrophe in murine and human lung cancer cells with known high metastatic potential. This was in marked contrast to effects in immortalized pulmonary epithelial murine and human cells. CYC065 markedly inhibited migration and invasion of lung cancer cells and affected distinctive pathways involved in DNA damage response, apoptosis, cell cycle regulation and cell migration. The data were presented at the American Association for Cancer Research (AACR) Annual Meeting 2017, April 1 – 5, 2017, in Washington, D.C.

Seattle Genetics highlighted multiple data presentations that support the company’s advancing antibody-drug conjugate (ADC) and immuno-oncology programs at the upcoming 108(th) Annual Meeting of the American Association for Cancer Research (AACR) being held April 1-5, 2017, in Washington, D.C. The presentations describe the ability of ADCETRIS (brentuximab vedotin) to activate antitumor immune responses, supporting continued clinical evaluation in combination with checkpoint inhibitors. Additionally, preclinical data feature two immuno-oncology agents, SEA-CD40 and SGN-2FF, both of which are in Phase I trials. Seattle Genetics and Unum Therapeutics are presenting preclinical data evaluating combination treatment with Antibody-Coupled T cell Receptor (ACTR) engineered autologous T cells and an antibody targeting B-cell maturation antigen (BCMA), SEA-BCMA, for multiple myeloma. Further data highlight clinical biomarker analyses for vadastuximab talirine (SGN-CD33A; 33A), an ADC under evaluation in the global Phase III CASCADE trial for acute myeloid leukemia (AML).

Sunovion Pharmaceuticals announced that Utibron Neohaler (indacaterol/glycopyrrolate) inhalation powder is now available at pharmacies in the United States for the long-term maintenance treatment of airflow obstruction in people with chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema. UTIBRON NEOHALER is not indicated to treat asthma or for the relief of sudden symptoms of COPD.

Five Prime Therapeutics announced that a poster featuring preclinical data related to Five Prime’s CSF-1R antibody, cabiralizumab (FPA008), was presented at the 2017 American Association for Cancer Research (AACR) Annual Meeting in Washington, D.C. 

Janssen Pharmaceuticals announced it has collaborated with Premier on the first and largest study of its kind to address an unmet medical need for hospitalized patients with atrial fibrillation (AF) who are at risk for ischemic stroke. Named QUANTUM AF (Quantify Use of ANTicoagUlation to improve Management of AF), the study will evaluate the effect of a structured hospital quality improvement (QI) program on oral anticoagulant (OAC) use in these patients.

Sunesis Pharmaceuticals announced results from an Ohio State University-sponsored preclinical study evaluating the efficacy of non-covalent BTK inhibitor SNS-062 in chronic lymphocytic leukemia (CLL) proprietary cell lines and patient samples. The study demonstrated that, unlike ibrutinib, SNS-062 inhibition of BTK signaling is unaffected by the presence of the C481S mutation and may address acquired resistance to covalent BTK inhibitors. The results are being presented in a poster session titled “Reversal of Drug Resistance” on Monday, April 3, 2017 from 8:00 AM to 12:00 PM ET at the American Association for Cancer Research Annual Meeting in Washington, D.C.

Tetraphase Pharmaceuticals announced completion of enrollment in IGNITE4, its ongoing Phase III clinical trial evaluating the efficacy and safety of intravenous (IV) eravacycline compared to meropenem in complicated intra-abdominal infections (cIAI).  The Company expects to report top-line data from this trial in the third quarter of 2017.

XBiotech announced top-line results today from its double-blind, placebo-controlled, Phase I-II study evaluating the safety and efficacy of its FDA Fast Tracked true human antibody (514G3) for the treatment of Staphylococcus aureus bloodstream infections.

MEI Pharma announced the appointment of Brian Drazba as Chief Financial Officer.

Halozyme Therapeutics demonstrated in preclinical models that its investigational drug PEGPH20 increases the number of cancer-fighting white blood cells accumulating in the tumor and the effectiveness of immunotherapies. The research was presented at the 108(th) annual meeting of the American Association of Cancer Research (AACR) and builds upon prior preclinical findings.

Audentes Therapeutics announced that the FDA has cleared the IND application for AT132, the Company’s gene therapy product candidate to treat X-Linked Myotubular Myopathy (XLMTM). The IND is now active and Audentes plans to initiate ASPIRO, the multicenter, multinational, open-label, ascending dose Phase I/II clinical study of AT132. Preliminary data from ASPIRO is expected to be available in the fourth quarter of 2017.

Syros Pharmaceuticals announced that SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, shows significant anti-proliferative activity in multiple in vitro and in vivo models of difficult-to-treat solid tumors, including triple negative breast, small cell lung and ovarian cancers. Leveraging its expertise in transcriptional biology and chemistry, Syros also showcased its work to further elucidate the biology of cyclin-dependent kinase 12 (CDK12) and cyclin-dependent kinase 13 (CDK13), advancing its aim of designing the first highly selective CDK12 and CDK13 inhibitors suitable for clinical development. These data were presented at the American Association of Cancer Research (AACR) Annual Meeting in Washington, D.C.

Peregrine Pharmaceuticals announced the presentation of positive new data from the company’s ongoing collaboration with researchers from Memorial Sloan Kettering Cancer Center (MSK).  Presented preclinical study results highlighted the potential of the company’s  phosphatidylserine (PS)-targeting antibodies to enhance the anti-tumor activity of adoptive T cell transfer therapy without triggering any off-target toxicities.  Data were presented this morning by MSK researchers at the 2017 Annual Meeting of the American Association for Cancer Research (AACR), which is being held April 1-5, 2017 in Washington, D.C.

Aytu BioScience announced the divestiture of Primsol, an oral antibiotic solution for urinary tract infections, to Allegis Holdings. The sale price of $1.75 million was paid in cash upon closing on March 31, 2017. Primsol will remain on the market during the transition. Aytu and Allegis will work collectively over the coming weeks to effectively transition Primsol to Allegis.

Biocept announced that it has entered into a Preferred Provider Collaboration and Services Agreement with Oregon Health & Sciences University on behalf of the OHSU Knight Cancer Institute (collectively "OHSU"). The multiphase agreement grants OHSU the rights to commercially offer Biocept’s Target Selector liquid biopsy testing services exclusively throughout the state of Oregon. Additionally, Biocept and OHSU plan to engage in technology transfer, whereby OHSU will have the ability to use Target Selector assays in-house, and act as a secondary laboratory for Biocept’s research and testing activities. Biocept and OHSU also plan to co-develop additional liquid biopsy assay technologies and platform capabilities including highly sensitive, multiplexed assay panels for molecular biomarker detection and assessment.

Bristol-Myers Squibb announced the first report of five-year overall survival (OS) data from the Phase I dose-ranging study CA209-003 evaluating Opdivo in patients with previously treated advanced non-small cell lung cancer (NSCLC; n=129). Overall survival was an exploratory endpoint in this study. The estimated OS rate at five years was 16% in heavily pre-treated NSCLC patients; survival was observed across PD-L1 expression levels and tumor histologies. The safety profile of Opdivo from this study was previously reported; no new safety signals were identified in this analysis. These data were featured today during the official press program at the American Association for Cancer Research (AACR) Annual Meeting 2017 in Washington, D.C.

Bristol-Myers Squibb announced the first overall survival (OS) data from the Phase III CheckMate -067 clinical trial. With a minimum follow-up of 28 months, the median OS had not yet been reached in either of the two Opdivo treatment groups and was 20 months for the Yervoy monotherapy group (95% CI: 17.1-24.6). Opdivo in combination with Yervoy and as a monotherapy reduced the risk of death 45% [hazard ratio (HR) 0.55; 95% CI: 0.42-0.72; P <0.0001] and 37% (HR 0.63; 95% CI: 0.48-0.81; P <0.0001), respectively, compared with Yervoy alone. The two-year OS rates were 64% for the Opdivo plus Yervoy combination, 59% for Opdivo alone and 45% for Yervoy alone. Results will be presented in the press program and an oral presentation during the Update, Novel Indication, and New Immuno-oncology Clinical Trials session from 3:35 to 3:50 p.m. ET (Late-Breaking Abstract CT075) at the American Association for Cancer Research Meeting 2017 in Washington, D.C.

AVEO Pharmaceuticals announced the closing of its previously announced underwritten public offering of 34,500,000 shares of common stock, including the exercise in full by the underwriter of its option to purchase 4,500,000 shares at the public offering price of $0.50 per share. The exercise of the option increased the amount of net proceeds raised in the offering, after underwriting discounts and estimated offering expenses, to approximately $15.5 million.  The proceeds of the offering are expected to be used for working capital and general corporate purposes, including development and pre-commercial expenses incurred in connection with the TIVO-3 trial, AVEO’s ongoing Phase III clinical trial of tivozanib in the third-line treatment of patients with refractory renal cell carcinoma (RCC), for which top line data is anticipated in the first quarter of 2018, and the TiNivo trial, AVEO’s ongoing Phase I/II clinical trial of tivozanib in combination with Opdivo (nivolumab). Piper Jaffray & Co.acted as sole manager for the offering. Joseph Gunnar & Co. acted as financial advisor to AVEO in connection with the offering.

Bayer announced positive data on its investigational compound copanlisib, an intravenous pan-Class I phosphatidylinositol-3-kinase (PI3K) inhibitor with predominant inhibitory activity against PI3K-α and PI3K-δ isoforms. The Phase II CHRONOS-1 trial, an open-label, single-arm study evaluating patients with relapsed or refractory indolent non-Hodgkin’s lymphoma (iNHL), met its primary endpoint of a pre-specified objective response rate (ORR). The results across all patient groups show an ORR of 59.2%, with a 12% complete response (CR) rate and a median

Mylan announced that Meridian Medical Technologies, a Pfizer company and Mylan’smanufacturing partner for EpiPen Auto-Injector, has expanded a voluntary recall of select lots of EpiPen (epinephrine injection, USP) and EpiPen Jr (epinephrine injection, USP) Auto-Injectors to now include additional lots distributed in the U.S. and other markets in consultation with the FDA.

Amgen and UCB announced results from the fourth year of a Phase II study showing the efficacy and safety of a second course of treatment with EVENITY (romosozumab), an investigational agent for postmenopausal women with osteoporosis. The results were presented in an oral session (OR08-1) at ENDO 2017, the Endocrine Society’s Annual Meeting in Orlando, Fla.

Bristol-Myers Squibb announced the companies have agreed to advance their clinical development program evaluating the combination of epacadostat, Incyte’s investigational oral selective IDO1 enzyme inhibitor, with Opdivo, Bristol-Myers Squibb’s PD-1 immune checkpoint inhibitor, into Phase III registrational studies in first-line non-small cell lung cancer across the spectrum of PD-L1 expression and first-line head and neck cancer. Additionally, the companies are expanding the ECHO-204 Phase I/II study, established under a collaboration between the companies in 2014, to include anti-PD-1/PD-L1 relapsed/refractory melanoma cohorts. The expanded clinical development program, including the Phase III registrational studies, will be co-funded by the two companies.

Puma Biotechnology announced that interim results from the Phase Ib/II FB-10 clinical trial of Puma’s investigational drug PB272 (neratinib) given in combination with the antibody drug conjugate T-DM1 (Kadcyla, ado-trastuzumab emtansine) were presented at the 2017 American Association for Cancer Research Annual Meeting (AACR) that is currently taking place in Washington, D.C. The presentation entitled, “NSABP FB-10: Phase Ib dose-escalation study evaluating trastuzumab emtansine (T-DM1) with neratinib in women with metastatic HER2-positive breast cancer” was selected for an oral presentation.

United Therapeutics announced that regulatory delays will result in postponement of the planned U.S. launch of the RemoSynch Implantable System for Remodulin until 2018.

Piramal Imaging and AC Immune presented the pre-clinical profile and first clinical data of the investigational next generation tau PET-imaging tracer PI-2620 at the International conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD) , in Vienna, Austria. These results are an important step in Piramal Imaging’s mission to develop molecular imaging innovations for an earlier and more accurate diagnosis of Alzheimer’s disease and other neurodegenerative disorders. PI-2620 is the result of a research collaboration between the two companies.

Sucampo Pharmaceuticals and Vtesse announced that Sucampo has acquired Vtesse for upfront consideration of $200 million.  Sucampo funded the acquisition through the issuance of 2,782,678 shares of Sucampo Class A common stock and $170 million of cash on hand; no external financing was utilized.

Ascendis Pharma announced data supporting its product pipeline presented at ENDO 2017, the annual meeting of the Endocrine Society in Orlando, Florida.

Radius Health presented results from new analyses of the Phase III ACTIVE clinical trial of abaloparatide-SC, one during a late-breaker oral session at ENDO 2017, in Orlando, Fla., and the other in a late-breaker poster presentation.

Intrexon, ZIOPHARM Oncology, and Merck KGaA, announced an update on the development of next-generation chimeric antigen receptor T cell (CAR-T) therapy for cancer as part of their strategic collaboration and license agreement.

Tonix Pharmaceuticals announced that it has regained compliance with the minimum bid price requirement for continued listing on the NASDAQ Global Market. On March 17, 2017, Tonix effected a 1-for-10 reverse stock split of its outstanding common stock intended to increase the per share trading price of Tonix’s common stock to satisfy the $1.00 minimum bid price requirement of $1.00 per share for continued listing on the NASDAQ Global Market, as set forth in NASDAQ Listing Rule 5450(a)(1) (the “Bid Price Rule”).

Anavex Life Sciences announced the presentation of new mechanism of action data related to ANAVEX compounds targeting the sigma-1 receptor at the AD/PD(TM) 2017 Meeting.

ProQR Therapeutics announced the grant of two key patents protecting QR-010 in the US and EU. These patents provide the Company exclusive rights for QR-010 for the treatment of cystic fibrosis (CF) until at least July 2033.

Sierra Oncology reported that preclinical results for its Chk1 inhibitor, SRA737, were presented on April 2 in a poster at the American Association of Cancer Research (AACR) Annual Meeting being held in Washington D.C. This research was conducted in the laboratory of Professor Paul Workman, Chief Executive and President of The Institute of Cancer Research (ICR), London, UK, and funded by Wellcome.

Zymeworks announced that it has filed a registration statement on Form F-1 (the “Registration Statement”) with the U.S. Securities and Exchange Commission (the “SEC”) and a preliminary prospectus with the securities regulatory authorities in each of the provinces and territories of Canada for a proposed initial public offering of its common shares. The number of common shares to be sold and the price range for the proposed offering have not yet been determined. Zymeworks has applied to list its common shares on the New York Stock Exchange and intends to apply to list its common shares on the Toronto Stock Exchange, under the ticker symbol “ZYME” for both exchanges.

Amicus Therapeutics has completed enrollment in the ongoing Phase III clinical study (ESSENCE) of the novel topical medicine SD-101 for patients with all 3 major types of epidermolysis bullosa (EB) (Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB). With the achievement of full enrollment, top-line data from this study are expected in the third quarter of 2017.

Invitae announced a program in partnership with Alnylam Pharmaceuticals to provide genetic testing for hereditary ATTR (hATTR) amyloidosis, a rare, progressive and life-threatening disease caused by a mutation in the TTR gene which causes accumulation of misfolded proteins in nerves and cells of other organs.

Versartis announced that new data on somavaratan in pediatric and adult GHD were presented during the Endocrine Society’s 99th Annual Meeting & Expo (ENDO 2017), being held April 1-4, 2017 in Orlando, Florida. Somavaratan was featured in four posters and an oral session over the weekend and three-year safety and efficacy results from the ongoing VISTA pediatric long-term safety study are scheduled to be presented during an oral session today from 11:15-12:45 ET.

Novartis announced the EC has approved Tafinlar (dabrafenib) in combination with Mekinist (trametinib) for the treatment of patients with BRAF V600-positive advanced or metastatic non-small cell lung cancer (NSCLC). The approval marks the first targeted treatment approved for the patient population, who previously had few treatment options, in all 28 member states of the EU, plus Iceland and Norway.

Diffusion Pharmaceuticals announced that it entered into subscription agreements for the sale of an aggregate of 4,558,030 shares of its Series A convertible preferred stock in a private offering for gross proceeds of approximately $9.2 million, prior to deducting placement agent fees and estimated expenses payable by Diffusion.  The closing is the second and final closing of Diffusion’s previously announced private placement. The private placement was oversubscribed as the original raise targeted $15.0 million. Together with the shares issued at and gross proceeds from the initial closing, Diffusion issued an aggregate of 12,395,053 shares of Series A convertible preferred stock for aggregate gross proceeds of approximately $25.0 million in the private placement. The Series A convertible preferred stock is initially convertible into one share of the Diffusion’s common stock at a conversion price equal to the purchase price of $2.02 per share.  In addition, each investor received a 5-year warrant to purchase one share of common stock for each share of Series A preferred stock purchased by such investor at an exercise price equal to $2.22, subject to adjustment thereunder. Maxim Merchant Capital acted as the Company’s placement agent in the Private Placement.

Sarepta Therapeutics announced the appointment of Catherine Stehman-Breen, M.D., M.S., as chief medical officer.

ESSA Pharma provided an update on the status of its Phase I clinical study of EPI-506 for patients with metastatic castration-resistant prostate cancer ("mCRPC"). EPI-506 targets the N-terminal domain ("NTD") of the androgen receptor (AR) – a novel approach to AR inhibition. Research into NTD AR biology is also being highlighted in three posters at the American Association for Cancer Research Annual Meeting ("AACR") in Washington, DC, April 1-5, 2017.

Kadmon Holdings announced that the Company, together with its lending syndicate, led by Perceptive Credit Opportunities Fund, entered into an amendment to its 2015 Credit Agreement and related warrants.

Morphotek, a subsidiary of Eisai, announced the launch of its Antibody Drug Conjugate (ADC) Services business based on its proprietary REsidue-SPEcific Conjugation Technology (RESPECT) and eribulin-linker toxin platforms. RESPECT is a site-specific conjugation technology that targets select amino acid residues as a way of producing investigational homogeneous ADCs with defined drug-to-antibody ratios.  The platform allows for site-specific conjugation of a single cytotoxic payload or two payloads with different mechanisms of action.  The platform employs eribulin as one of the cytotoxic payloads along with a proprietary high-throughput screening system that can evaluate multiple ADC products simultaneously for client-desired biophysical properties and target-specific binding.  As part of our services, the eribulin-linker payload is offered as an option to develop investigational ADCs using traditional bioconjugation for companies interested in developing next-generation formats of their own antibodies.

Aptevo Therapeutics announced the presentation of new preclinical data on the Company’s next generation bispecific antibody candidates, APVO436 and APVO437, at the American Association for Cancer Research 2017 annual meeting.

Jazz Pharmaceuticals announced the completion on March 31, 2017 of a rolling submission of a NDA to the FDA for the approval of Vyxeos (cytarabine and daunorubicin) liposome for injection, an investigational treatment for acute myeloid leukemia (AML), a rapidly progressing and life-threatening blood cancer. The company has requested a priority review for the Vyxeos NDA, which, if granted, would accelerate the expected timing of the FDA’s review.

ANALYST RECOMMENDATIONS

Morgan Stanley analyst Matthew Harrison initiated coverage of Loxo Oncology with an “overweight” rating and $59 price target, citing Loxo is developing larotrectinib (LOXO-101), a cancer therapy that is designed to specifically target ultra rare tropomyosin-related kinase (TRK) fusions.

Canaccord analyst Arlinda Lee increased her price target of Blueprint Medicines to $42 from $40, citing at AACR, Blueprint highlighted key features of BLU-285, and importantly, showed steadystate plasma concentration of BLU-285 at the selected expansion cohort dose of 400mg QD is in therapeutic range to inhibit both ATP binding site and activation loop KIT mutations.

Raymond James analyst Chris Raymond increased his price target of Blueprint Medicines to $50 from $41, citing ~$450M on the balance sheet after last week’s follow-on, and a recent publication suggesting that PDGFRa D842V mutations may be more prevalent in adjuvant GIST.

Janney analyst Ken Trbovich downgraded Acorda Therapeutics to “neutral” from “buy” and decreased his price target to $19 from $38, citing firm sees a generic Ampyra as likely in Q3/18.

Piper Jaffray analyst Edward Tenthoff decreased his price target of AVEO Pharmaceuticals to $1.60 from $1.75 based on dilution. 

Citi analyst Robyn Karnauskas decreased her price target of Juno Therapeutics to $30 from $34, citing while JCAR015 has been discontinued for strategic reasons, the company expects to initiate a trial in adult ALL with a defined cell product next year.

Following Forward Pharma’s announcement the court decided that all FWP’s ‘187 patent claims are invalid, making Biogen the winner of the interference, JMP analyst Jason Butler downgraded Forward Pharma to “market perform” to “market outperform;” Jefferies analyst Biren Amin downgraded the stock to “hold” from “buy” and decreased his price target to $24 from $41; Leerink analyst Jason Gerberry decreased his price target to $28 from $37.

Leerink analyst Jonathan Chang resumed coverage of Calithera with an “outperform” rating and $16 price target, citing CALA is developing oncology drugs in the promising tumor and immune cell metabolism space.

Citi analyst Yigal Nochomovitz increased his price target of Portola Pharmaceuticals to $51 from $39, citing recent Ph3 successes are poised to expand the indication set for approved novel oral anticoagulants (NOAC) and should drive broader need for Portola’s bleeding reversal agent AndexXa.

Leerink analyst Michael Schmidt increased his price target of Incyte to $141 from $130, citing epacadostat is better positioned in the emerging IO combination landscape.

BioShares Biotechnology Clinical Trials (BBC): $23.05, -$0.14, +25.2% YTD

BioShares Biotechnology Products (BBP): $37.17, -$0.58, +13.5% YTD

MARKET COMMENTARY

U.S. stock index futures fell as investors appeared concerned over President Donald Trump’s ability to deliver on his policy plans. Data on international trade and factory orders is due later in the day. Weakness in the autos sector weighed on the European shares even as gains in oil-related stocks and miners provided some support. Asian stock markets recorded a mixed trading day. The dollar edged up against a basket of major currencies but lost ground against the safe-haven Japanese yen. Oil prices recovered from recent losses to trade higher and gold rose.

MARKET HIGHLIGHTS

Onconova Therapeutics announced promising pre-clinical data for first-in-class dual inhibitor of CDK4/6 + ARK5, as well as a Type 1 novel inhibitor of FLT3 and Src pathways as a novel strategy for Acute Myeloid Leukemia (AML) therapy at the 2017 AACR Annual Meeting. The Company presented its findings in two poster presentations on April 3, 2017.

BioTime Co-Chief Executive Officer Michael D. West, Ph.D., is delivering a keynote presentation at the GTC Bio Stem Cell Summit, on April 6 at the Hyatt Regency in Boston, Massachusetts.

BIOPHYTIS announced that it has gathered funding to conduct its phase 2b SARA-OBS/SARA-INT clinical study aimed at testing its leading product, Sarconeos, for the treatment of sarcopenia. First, a capital increase subscribed by several private investors among which Bracknor Fund, and the management, of an amount of €3.7 million was completed by issuing 1,310,431 new shares at a price of €2.85 per share. Secondly, a line of funding that could reach €15 million was set up with Bracknor Fund in the form of 1,500 note warrants for Bonds Redeemable in Cash or New or Existing Shares (ORNANE), at a par value of €10,000 each, combined with Share Subscription Warrants (BSAs), together referred to as ORNANEBSA.

Sigmoid Pharma Limited has entered into an agreement with Dr. Falk Pharma GmbH to advance Sigmoid’s SmPill® cyclosporine franchise for ulcerative colitis, graft versus host disease and other gastrointestinal diseases.  The terms of the agreement include an upfront equity investment in Sigmoid, development funding, milestone payments as well as tiered double-digit royalties for the future development and commercialization of CyCol®, AlloCol® and related products in Europe. Sigmoid retains the rights to these programs outside of Europe and Canada. This collaboration with Dr. Falk in Europe will serve as an important catalyst to Sigmoid advancing its pipeline to patients in territories including the United States and Japan.

Paratek Pharmaceuticals announced positive top-line results from a global, pivotal Phase 3 clinical study comparing its once-daily oral and IV, broad spectrum investigational antibiotic, omadacycline, to moxifloxacin in the treatment of patients with community-acquired bacterial pneumonia (CABP).  This study represents the second positive Phase 3 registration study of omadacycline, which will be used to support marketing applications to the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).  The global, pivotal Phase 3 clinical study known as OPTIC (Omadacycline for Pneumonia Treatment in the Community), compared the safety and efficacy of once-daily, IV-to-oral omadacycline to IV-to-oral moxifloxacin for treating adults with CABP.  In the study, 774 patients were randomized.  Omadacycline met the FDA-specified primary endpoint of statistical non-inferiority (NI) in the intent-to-treat (ITT) population (10% NI margin, 95% confidence interval) compared to moxifloxacin at the early clinical response (ECR) 72-120 hours after initiation of therapy. The ECR rates for the omadacycline and moxifloxacin treatment arms were 81.1 % and 82.7%, respectively.

VBL Therapeutics announced today the presentation of new data on MOSPD2, a novel potential target in oncology. VBL’s study, entitled "MOSPD2, a Newly Characterized Protein, Promotes Breast Cancer Metastasis" by Mendel et al., will be presented today at the American Association of Cancer research (AACR) conference in Washington, DC. The study observed from clinical biopsies that MOSPD2 is prevalent in invasive human breast cancer tissue and that levels of MOSPD2 correlate to breast cancer invasiveness. It was further observed that a knockdown of MOSPD2 in a human breast cancer cell line using CRISPR technology led to blockade of EGF signaling and significant reduction of breast cancer cell migration in vitro and metastasis in a mouse model.

Santhera Pharmaceuticals announced that a large majority of shareholders approved all proposals by the Board of Directors at the Annual Shareholders` Meeting (ASM) today. Participating shareholders represented a total of 3,097,267 shares or 49.3% of the share capital with voting power.

Rockwell Medical filed a preliminary proxy statement with the Securities and Exchange Commission for the Company’s 2017 Annual Meeting of Shareholders, highlighting the nomination of David Domzalski to the Company’s Board of Directors, disclosing the appointment of a Lead Independent Director to the Company’s Board of Directors and describing corporate governance enhancements.

Galena Biopharma announced a poster was presented on the Company’s NeuVax (nelipepimut-S) investigator-sponsored Phase II clinical trial (IST) in high-risk, HER2 3+ patients at the American Association for Cancer Research (AACR) Annual Meeting 2017 in Washington, DC.  The Phase II trial is a multi-center, prospective, randomized, single-blinded, placebo-controlled trial combining NeuVax and trastuzumab in the adjuvant setting to prevent recurrence in HER2-positive (HER2 3+) breast cancer patients.

ContraVir Pharmaceuticals provided new insights into the mechanism of action (MOA) of its hepatitis B virus (HBV)-optimized cyclophilin inhibitor CRV431. New studies show that CRV431 potently blocks the interaction between hepatitis B surface antigen (HBsAg), a key HBV protein, and cyclophilin A, an important cellular protein. This new information could explain previous findings that CRV431 reduces HBsAg in many experimental systems. High levels of HBsAg in HBV-infected patients is a predictor for progression of disease, including liver fibrosis, cirrhosis, and cancer.

TherapeuticsMD announced that data on TX-001HR, an investigational bio-identical hormone therapy combination of 17ß-estradiol and progesterone in a single, oral softgel, for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause, was presented at ENDO 2017, the annual meeting of the Endocrine Society in Orlando, Florida. The positive results of the pivotal Phase III Replenish Trial data were featured in an oral and a poster presentation.

RedHill Biopharma announced that the FDA has granted YELIVA (ABC294640) Orphan Drug designation for the treatment of cholangiocarcinoma.

Teva announced that the FDA has approved AUSTEDO (deutetrabenazine) tablets for the treatment of chorea associated with Huntington’s disease (HD). Previously referred to by the developmental name SD-809, AUSTEDO is the first deuterated product approved by the FDA and only the second product approved in HD. The product was previously granted Orphan Drug Designation by the FDA.

uniQure N.V. announced the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure’s AAV5 vector in a large animal model. uniQure has exclusive, worldwide rights to AAV5 for use in therapeutic products delivered to the brain or liver.

Boston Children’s Hospital, the pediatric teaching hospital of Harvard University, and Mesoblast announced that the FDA has cleared the commencement of a 24-patient trial combining Mesoblast’s proprietary allogeneic mesenchymal precursor cells (MPCs) with corrective heart surgery in children under the age of 5 with hypoplastic left heart syndrome (HLHS). The trial is sponsored and funded by the Boston Children’s Hospital with support from Bulens and Capozzi Foundation and the Ethan Lindberg Foundation.

Aclaris Therapeutics announced that Columbia University has received a Notice of Allowance from the USPTO for two patent applications covering methods related to the use and administration of baricitinib (LY3009104) and decernotinib (VX-509), respectively, for the treatment of hair loss disorders and for inducing hair growth. These newly allowed patent applications are owned by The Trustees of Columbia University in the City of New York and exclusively licensed to Aclaris Therapeutics, Inc. These patent applications are the latest U.S. applications to be allowed in connection with Aclaris’ janus kinase inhibitor (JAK) drug development program for hair loss disorders. A separate Columbia patent covering the use of ruxolitinib for the treatment of hair loss disorders and for inducing hair growth was issued in the U.S. in December 2015.

Immunomedics announced that its lead antibody-drug conjugate (ADC), sacituzumab govitecan (IMMU-132), with a proprietary SN-38-delivery platform, has the potential to provide clinical benefit both to chemosensitive solid tumors with low Trop-2 expression, as well as to chemoresistant tumors with high Trop-2 expression.

Benitec Biopharma announced that the initial pre-clinical efficacy results of the OPMD program have been published in Nature Communications, an open access scientific journal published by the Nature Publishing Group.  OPMD, a rare progressive muscle-wasting disease caused by mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, is characterised by eyelid drooping, swallowing difficulties, and proximal limb weakness.

Bellicum Pharmaceuticals announced two presentations of preclinical results on the Company’s proprietary dual-switch technology for use in CAR-T and TCR product candidates at the 2017 American Association for Cancer Research (AACR) Annual Meeting in Washington, D.C.

Amgen announced the submission of a sBLA to the FDA and an application for a variation to the marketing authorization to the EMA for XGEVA (denosumab). The submissions to regulatory authorities seek to expand the currently approved XGEVA indication for the prevention of skeletal-related events (SREs) in solid tumors to include patients with multiple myeloma. The applications include new data from the pivotal Phase III head-to-head ‘482 study, the largest international multiple myeloma trial ever conducted.

Nektar Therapeutics announced five preclinical data presentations for its immuno-oncology programs made at the American Association for Cancer Research (AACR) Annual Meeting 2017.  The presentations featured new preclinical data on NKTR-214, the Company’s immuno-stimulatory CD122-biased agonist, as well as on NKTR-255, the Company’s IL-15 therapeutic candidate.

NANOBIOTIX announced the expansion and acceleration of its clinical development activities. These include: Acceleration of the head and neck cancer program. Phase I/II data will be presented at ASCO in June; Expansion of Nanobiotix’s Immuno-Oncology program into patients focused on the objective of turning cold tumors into hot tumors. Nanobiotix will present the first clinical data from this program mid-year.

Galapagos announced three new Phase II Proof-of-Concept studies investigating filgotinib in Sjögren’s syndrome, ankylosing spondylitis, and psoriatic arthritis. The Sjögren’s study is being led by filgotinib collaboration partner Gilead Sciences; the ankylosing spondylitis and psoriatic arthritis studies by Galapagos.

Karyopharm Therapeutics announced that Justin Renz, Executive Vice President, Chief Financial Officer and Treasurer, has stepped down in order to pursue other opportunities, effective. Mr. Renz will continue to serve the Company in an advisory capacity in order to ensure a smooth transition.

NantKwest and Viracta Therapeutics announced the initial closing of a Series B Preferred financing round of up to $18.4 million with NantKwest as the lead investor. Joining NantKwest as a new investor in Viracta is Wicklow Capital. Viracta’s founding investors, Latterell Venture Partners and Forward Ventures also participated in the round.

AMAG Pharmaceuticals announced the closing of the licensing agreement withEndoceutics for the U.S. commercial rights to Intrarosa (prasterone). Intrarosa is the only FDA-approved, locally administered, non-estrogen steroid hormone for the treatment of moderate-to-severe dyspareunia (pain during intercourse), a common symptom of vulvar and vaginal atrophy (VVA), due to menopause. Unlike all other FDA-approved medications for the treatment of VVA symptoms, Intrarosa does not carry a boxed safety warning in its label. The agreement was announced on February 14, 2017, and its closing broadens AMAG’s presence in women’s health.

Syros Pharmaceuticals announced that new preclinical data on SY-1425, its first-in-class selective retinoic acid receptor alpha (RARα) agonist currently in a Phase II clinical trial in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), were presented at the American Association of Cancer Research (AACR) Annual Meeting in Washington, D.C.

Peregrine Pharmaceuticals announced the presentation of results of a new analysis of the Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC).  Data demonstrated that for patients in the study’s bavituximab plus docetaxel treatment arm who received subsequent immunotherapy, the median overall survival (mOS) was not reached, while mOS was 13.0 months for patients in the study’s placebo plus docetaxel arm who received subsequent immunotherapy [HR = 0.43; p=0.005].  These are the first clinical results reported supporting the hypothesis that bavituximab may modulate the tumor microenvironment to enhance the anti-tumor activity of immunotherapy agents. Data were presented by Peregrine scientists at the 2017 Annual Meeting of the American Association for Cancer Research (AACR), which is being held April 1-5, 2017 in Washington, D.C.

RepliCel Life Sciences reported statistically and clinically significant positive data from the interim analysis of its phase I study evaluating RCS-01 for the treatment of aging and sun-damaged skin.

Provectus Biopharmaceuticals announced the entry into definitive agreements related to the previously announced Definitive Financing Commitment Term Sheet (the "Definitive Financing") it entered into on March 19 with a group of the Company’s stockholders referred in the Definitive Financing as the "PRH Group."

AbbVie announced a sNDA was accepted for review by the FDA for ibrutinib (IMBRUVICA) in chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy. cGVHD is a severe, potentially life-threatening consequence of stem cell or bone marrow transplant. If approved by the U.S. FDA, ibrutinib will be the first therapy specifically approved to treat this condition. IMBRUVICA is jointly developed and commercialized by Pharmacyclics, an AbbVie company, and Janssen Biotech.

Xenetic Biosciences announced that it has appointed James F. Parslow, MBA, CPA as Chief Financial Officer, effective April 3, 2017

Flexion Therapeutics announced the U.S. Patent and Trademark Office has issued two new patents covering the company’s lead investigational product candidate Zilretta™(FX006). The new patents further strengthen the existing patent estate surrounding Zilretta. The patents cover the injectable formulation comprised of controlled or sustained-release microparticles that contain triamcinolone acetonide in a poly lactic-co-glycolic acid co-polymer (PLGA) matrix.

Aytu BioScience announced that it presented two posters demonstrating the safety advantages of Natesto testosterone nasal gel, including the one-year hematologic safety and tolerability among men with seasonal allergies, in men with hypogonadism.

Medidata and Karyopharm Therapeutics announced an expanded partnership to bring novel machine-learning technology and targeted risk-based monitoring (RBM) techniques to clinical oncology trials.

Kite Pharma highlighted a publication in the latest issue of Nature Methods that describes pre-clinical findings demonstrating that a novel serum-free, 3-dimensional cell culture technology, known as the Artificial Thymic Organoid (ATO) cell culture system, recapitulates T-cell differentiation. The findings support the potential of ATO technology to generate off-the-shelf engineered T cells to treat cancer and other diseases. The research was led by Gay M. Crooks, M.D. at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles (UCLA). Kite holds an exclusive license to the ATO cell culture technology from UCLA.

Evoke Pharma announced that the Company recently completed a positive Type A meeting with the FDA to finalize the design of the pivotal comparative exposure pharmacokinetic (PK) trial and to reach agreement on additional aspects of the Chemistry, Manufacturing & Controls (CMC) section of the NDA for Gimoti, the Company’s patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women.

NewLink Genetics reported interim results from NLG2103, a Phase II study evaluating its IDO pathway inhibitor, indoximod, in combination with checkpoint inhibitors for the treatment of patients with advanced melanoma.

Diffusion Pharmaceuticals announced that a key patent has been issued by the USPTO on March 28, 2017 for its lead drug candidate trans sodium crocetinate (TSC).

Chembio Diagnostics is collaborating with FIND to develop a POC test that can identify multiple life-threatening acute febrile illnesses common in the Asia Pacific region. Over the next twelve months, Chembio and FIND will endeavor to develop a simple, rapid, and cost-effective test based on Chembio’s proprietary DPP technology platform to detect multiple diseases simultaneously.

Kadmon Holdings announced that on March 31, 2017, it submitted its second ANDA to the FDA for KD034, the Company’s trientine hydrochloride formulation for the treatment of Wilson’s disease, a rare genetic liver disorder, for patients who are intolerant of penicillamine. The Company also submitted an ANDA for a bottled form of trientine hydrochloride in December 2016.

Depomed announced that, in accordance with its stated plans and the terms of the secured debt facility with Deerfield and Pharmakon Advisors, LP, the company prepaid $100 million of principal amount of its $475 million secured indebtedness. In addition, Depomed paid a prepayment fee of $4 million. Under the terms of the 7-year loan agreement, which matures in 2022, Depomed intends to refinance the remainder of the $375 million secured indebtedness in 2017.

ANALYST RECOMMENDATIONS

Jefferies analyst Biren Amin assumed coverage of Progenics with a “buy” rating and increased his price target to $14 from $13, citing Azedra’s recent positive Phase III in rare PGG/PCC indications positions Azedra to be approved by FDA in early 2018.

Chardan analyst Madhu Kumar upgraded Arbutus to “buy” from “neutral” and increased his price target to $6 from $3, citing HBV represents a significant market in infectious diseases, multiple RNAi drugs (including ARB-1467) have demonstrated clinical proof of concept for hepatitis B surface antigen (HBsAg) suppression, and preclinical data from HBV combination therapies has looked promising.

Rodman & Renshaw analyst Joseph Pantginis initiated coverage of BeyondSpring with a “buy” rating and $43 price target, citing BeyondSpring’s lead asset, Plinabulin, is in pivotal studies for both the treatment of neutropenia (abnormally low neutrophils) associated with chemotherapy and non-small cell lung cancer (NSCLC).

Aegis analyst Difei Yang increased her price target of Nektar Therapeutics to $27 from $24, citing a valuation methodology update for NKTR-181 and NKTR-214.

FBR suspended coverage of the following companies, due to a reallocation of resources: Achillion; Aduro; Alnylam; Arena; Asterias; BioCryst; Curis; Genocea; Trevena; and Zafgen.

BioShares Biotechnology Clinical Trials (BBC): $21.85, -$0.69, +18.8% YTD

BioShares Biotechnology Products (BBP): $36.55, -$0.57, +11.6% YTD

MARKET COMMENTARY

U.S. stocks index futures were little changed as markets turned cautious by signs the Federal Reserve might start paring asset holdings later this year just as the chance of early U.S. fiscal stimulus seems to be evaporating. Weekly jobless claims data is on the economic radar. The euro declined and hit a three-week low on dovish comments from the head of the European Central Bank and gold edged up. Banking stocks dragged European bourses lower, and Asian shares declined. Oil prices rose after recovering from losses triggered by record high U.S. crude inventories.

MARKET HIGHLIGHTS

Eiger BioPharmaceuticals announced that abstracts from its LOWR HDV (LOnafarnib With Ritonavir in Hepatitis Delta Virus) Program will be presented at the European Association for the Study of the Liver (EASL) meeting in Amsterdam, Denmark, April 19 to 23, 2017.  Forty-eight week data from the Phase 2 LOWR HDV Program will be presented.

iCAD announced the Company will highlight PowerLook® Tomo Detection, an innovative, concurrent-read computer aided detection solution for digital breast tomosynthesis that can save radiologists valuable reading time without compromising clinical performance, at its booth #300 at the SBI/ACR Breast Imaging Symposium from April 6-9, 2017 in Los Angeles, California. PowerLook Tomo Detection received Premarket Approval (PMA) from the U.S. Food and Drug Administration (FDA) on March 24, 2017 and is the latest innovation available on the PowerLook® Breast Health Solutions platform.

BioTime announced the formation of AgeX Therapeutics, which will consolidate certain BioTime subsidiaries and programs in the field of interventional gerontology. The formation of AgeX continues the implementation of BioTime’s strategy to simplify its corporate structure and operations as well as focus resources on the continued clinical development of its two lead programs; Renevia, a proprietary cell delivery matrix designed to facilitate the stable engraftment of transplanted cells that is currently in a pivotal clinical trial, and OpRegen, a pluripotent cell-derived cell therapy that is in a Phase I/IIa clinical trial for the dry form of age related macular degeneration (dry-AMD). The formation of AgeX provides the new BioTime subsidiary greater flexibility to explore external financing alternatives as well as strategic options to grow its technology platform.

TiGenix NV reported its financial highlights for 2016.  The profit for the year 2016 amounted to €3.8 million compared to a loss of €35.1 million in 2015.  Cash and cash equivalents amounted to €78.0 million on December 31, 2016.

Regeneron Pharmaceuticals announced that the FDA has granted Breakthrough Therapy Designation status to evinacumab for the treatment of hypercholesterolemia in patients with Homozygous Familial Hypercholesterolemia (HoFH), an inherited disorder that can lead to premature cardiovascular disease due to very high levels of LDL cholesterol. Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.

Ultragenyx Pharmaceutical and Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin Co., Ltd., announced positive 64-week data from a pediatric Phase II study of burosumab (KRN23) for the treatment of X-linked hypophosphatemia (XLH) in children aged five to 12 years of age. The data demonstrated that serum phosphorus levels, rickets, growth rates, and other functional outcomes improved with burosumab, and these treatment effects were sustained through 64 weeks of treatment. In addition, interim 24-week data from the separate pediatric Phase II study in patients aged one to five years demonstrated that burosumab increased serum phosphorus levels into the low normal range. Adverse events were consistent with what has been previously observed for burosumab for the treatment of XLH. Ultragenyx is conducting the studies under a collaboration and license agreement with Kyowa Hakko Kirin to develop and commercialize burosumab.

Supernus Pharmaceuticals announced that the FDA has granted final approval to the Company’s sNDAs requesting a label expansion for Trokendi XR to include prophylaxis of migraine headache in adults and adolescents 12 years and older. Supernus was granted tentative approval of one of the two sNDAs in August 2016, with final approval subject to the pediatric exclusivity of the innovator’s drug in the adolescent population, which expired March 28, 2017.

Allergan announced topline data from a Phase II study with Major Depressive Disorder (MDD). The study evaluated the efficacy, safety and tolerability of a single administration of 2 different doses of BOTOX (30 units or 50 units) relative to placebo in adult females with MDD over duration of up to 24 weeks.   The BOTOX 30 U dose demonstrated numerically superior efficacy in MADRS total score compared to placebo. The treatment (LS mean) difference for 30 U was -4.2 at 3 weeks (p- value 0.005); -3.7 at week 6 (p-value 0.053) and -3.6 at week 9 (p-value 0.049). The primary end point was at week 6. The 50 U did not demonstrate superior efficacy over placebo (LS mean difference was 1.3). Both secondary efficacy variables (CGI-S and HAMD-17) showed numerically superior efficacy over placebo and trended in the same direction as the primary efficacy variable for 30 U, but not for 50 U. Both 30 U and 50 U were well tolerated.

Jazz Pharmaceuticals announced that certain of its subsidiaries have entered into agreements with Hikma Pharmaceuticals and related entities resolving patent litigation related to Xyrem (sodium oxybate) oral solution. The litigation, which has been pending in the U.S. District Court for the District of New Jersey since 2010, resulted from the submission by Roxane Laboratories (which was subsequently acquired by Hikma) of an ANDA to the FDA seeking approval to market a generic version of Xyrem. Following the news, Mizuho analyst Irina Koffler downgraded the stock to “neutral” from “buy” and decreased her price target to $150 from $162; Leerink analyst Jason Gerberry increased his price target to $170 from $169; Cantor analyst William Tanner increased his price target to $197 from $189; Janney analyst Ken Trbovich upgraded to “buy” from “neutral” and increased his fair value estimate to $175 from $138; Deutsche Bank analyst Gregg Gilbert increased to $176 from $148.

Tenax Therapeutics provided an update related to its business strategy, the clinical development of its product candidates and its management team. The Company’s Board of Directors is conducting a comprehensive review of strategic alternatives focused on maximizing stockholder value and has formed a strategic committee of three independent board members to supervise management in this review. In addition, the Company has engaged Ladenburg Thalmann & Co. Inc., a subsidiary of Ladenburg Thalmann Financial Services, as its financial advisor to assist in the strategic review process; including, but not limited to a merger, a business combination, a strategic investment into the Company, or a purchase, license or other acquisition of assets. This process may not result in any transaction and the Company does not intend to disclose additional details unless and until it has entered into a specific transaction. Effective April 3, 2017, John Kelley resigned as CEO of the Company and from the Company’s board of directors, but has agreed to provide consulting services to the Company for a period of time following his resignation. The Company’s Board of Directors appointed Michael Jebsen, the Company’s current President and CFO, as Interim CEO, effective immediately upon Mr. Kelley’s resignation. Mr. Jebsen previously served as Interim CEO for the Company from 2011 to 2013.

AnaptysBio announced first-in-human dosing of its wholly-owned proprietary anti-Interleukin-36 receptor (IL-36R) antibody, ANB019, in a healthy volunteer Phase I clinical trial. The double-blind, placebo-controlled, single and multiple ascending dose trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ANB019 through subcutaneous and intravenous routes of administration.

Lµbris BioPharma announced that Novartis has exercised an option to in-license ECF843, Lµbris’ proprietary recombinant human lubricin for ophthalmic indications worldwide (outside Europe) including the treatment of dry eye. The closing of the deal is subject to customary closing conditions including regulatory approvals. Lµbris retains rights to commercialize products outside of ophthalmology. Financial terms were not disclosed.

Achillion Pharmaceuticals announced initiation of patient dosing in a Phase II open-label study of ACH-4471, Achillion’s first orally-administered, small molecule factor D inhibitor, for patients with paroxysmal nocturnal hemoglobinuria (PNH). This proof of concept study will assess the efficacy, safety, and pharmacokinetics of ACH-4471 in untreated patients with PNH.

Galapagos announces dosing of the first patient with psoriatic arthritis in the EQUATOR Phase II study. This achievement triggers a $10 million milestone payment from Gileadto Galapagos. The EQUATOR Phase II study will be a multi-center, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of the selective JAK1 inhibitor filgotinib in adult patients with moderately to severely active psoriatic arthritis.

Cardiome Pharma updated that its partner, SteadyMed Therapeutics, announced the successful completion of a clinical study of Trevyent with a press release discussing the study on April 5, 2017. Cardiome licensed the commercial rights to Trevyent from SteadyMed for many international markets in 2015.

Spark Therapeutics announced updated preliminary data today from 10 infused participants in the ongoing Phase I/II clinical trial of investigational SPK-9001 for hemophilia B. All participants have experienced consistent and sustained increases in factor IX activity following administration of the investigational therapy. These data will be presented at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium in Scottsdale, Arizona on Friday, April 7, by Adam Cuker, M.D., assistant professor of medicine at the Perelman School of Medicine of the University of Pennsylvania and a clinical investigator at Children’s Hospital of Philadelphia.

Innocrin Pharmaceuticals announced the appointment of Fred Eshelman, PharmD, of Eshelman Ventures as CEO, effective March 15, 2017. Innocrin also announced that the FDA granted a second Fast Track Designation for seviteronel (VT-464). The new designation is for the treatment of women with advanced AR+ triple-negative breast cancer (TNBC) and women or men with advanced estrogen receptor-positive (ER+) breast cancer.

Immune-Onc Therapeutics announced today it has entered into an exclusive license and collaboration agreement with The University of Texas Health Science Center at Houston and The University of Texas Southwestern Medical Center, two member institutions of The University of Texas System.

BlackThorn Therapeutics announced preclinical study results demonstrating that its investigational novel kappa opioid receptor (KOR) antagonist BTRX-335140 is selective and short-acting. Results of the study, which was conducted by BlackThorn and researchers at the University of California, San Francisco (UCSF), were presented this week at the Fourth Conference on the Therapeutic Potential of Kappa Opioids in Philadelphia. BlackThorn is building its proprietary KOR antagonist program through a partnership with The Scripps Research Institute.

Imprimis Pharmaceuticals announced that it has entered into an licensing agreement for the exclusive worldwide rights to Klarity, an innovative and patented ophthalmic topical solution and gel technology for patients with dry eye disease.

Allergan and ZELTIQ Aesthetics announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 has expired with respect to Allergan’s proposed acquisition of ZELTIQ.

Myriad Genetics announced that it has entered into a companion diagnostic development collaboration with BeiGene to accelerate precision medicine in oncology.

Biostage presented preclinical data of its Cellspan Esophageal Implant at the Society for Biomaterials 2017 Annual Meeting and Exposition, being held April 5-8, 2017 in Minneapolis, MN.

Valeant Pharmaceuticals International‘s wholly owned subsidiary, Bausch Lomb, announced it has received 510(k) clearance from the FDA for the Stellaris Elite Vision Enhancement System, the company’s next generation phacoemulsification cataract platform.

FibroGen announced that the company has priced an underwritten follow-on offering, which is expected to raise gross proceeds of approximately $120 million. FibroGen is offering 5,228,750 shares of its common stock at an offering price of $22.95 per share, before underwriting discounts and commissions. All of the shares are being offered by FibroGen. The offering is expected to close on April 11, 2017, subject to customary closing conditions.

Cara Therapeutics announced the closing of its underwritten public offering of 5,117,500 shares of its common stock at a public offering price of $18.00 per share, which included 667,500 shares sold pursuant to the full exercise by the underwriters of their option to purchase additional shares. The gross proceeds from the offering, before deducting the underwriting discounts and commissions and offering expenses payable by Cara Therapeutics, are expected to be approximately $92.1 million. The proceeds of the offering are expected to be used to fund the company’s clinical and research development activities, including the completion of the Phase III program for I.V. CR845 in uremic pruritus, two Phase III trials of I.V. CR845 in acute pain and a Phase IIb trial of oral CR845 in osteoarthritis pain, as well as for working capital and general corporate purposes. Piper Jaffray & Co. and Stifel acted as joint book-running managers for the offering. Canaccord Genuity, Needham & Company, Janney Montgomery Scott and H.C. Wainwright & Co. acted as co-managers.

Merrimack Pharmaceuticals announced that its Board of Directors has authorized and declared a special cash dividend of $140 million on the company’s common stock. This special dividend will be paid from the proceeds of the company’s asset sale to Ipsen S.A., which was completed on April 3, 2017.

China Cord Blood Corporation announced that the Company received notification of exercise of all 7% senior convertible notes ("Notes") held by Golden Meditech Stem Cells (BVI) Company Limited ("GMSC", the "Holder"), a subsidiary of Golden Meditech Holdings Limited beneficially owning 65.4% of the Company on a fully diluted basis.

Ocular Therapeutix announced the appointment of George Migausky as interim CFO. 

Ironwood Pharmaceuticals announced the appointment of Christopher Wright, M.D., MMSc., Ph.D., as senior vice president of global development and chief development officer.

ANALYST RECOMMENDATIONS

Rodman & Renshaw analyst Joseph Pantginis assumed coverage of Rexahn Pharmaceuticals with a “buy” rating and $2 price target, citing the company is on the cusp of the transition to meaningful clinical data readouts this year with well thought out clinical studies.

Jefferies analyst Brian Abrahams assumed coverage of Ultragenyx, downgraded the stock to “hold” from “buy,” and decreased his price target to $66 from $109, citing potential headwinds they may face building into small/less well-defined markets– which alongside split economics for some programs could impact potential LT revenue opportunities.

William Blair analyst Tim Lugo initiated coverage of the following companies: Retrophin with an “outperform” rating; Akari Therapeutics with an “outperform” rating.

Citi analyst Liav Abraham downgraded Synergy to “sell” from “neutral” and decreased her price target to $3.70 from $6.50, citing updated operating expense assumptions to more adequately reflect the promotional investment required in the launch of Trulance.

Jefferies analyst Brian Abrahams made price target changes to the following companies: Incyte to $165 from $140; Biogen to $281 from $289; Gilead to $82 from $83. Jefferies analyst Matthew Andrews increased his price target of Spectrum Pharmaceuticals to $8.50 from $7.50.

Leerink analyst Michael Schmidt increased his price target of Seattle Genetics to $74 from $70, citing an adjusted forecast for Adcetris.

BioShares Biotechnology Clinical Trials (BBC): $21.66, -$0.19, +17.7% YTD

BioShares Biotechnology Products (BBP): $36.49, -$0.06, +11.4% YTD

MARKET COMMENTARY

U.S. stock index futures were little changed as focus shifted to monthly payrolls report due later in the day for further cues on the health of the economy. European markets fell as mining and financial stocks slipped, while Asian stocks ended on a mixed note. Gold prices rose as bids for safe-haven assets picked up after the United States fired dozens of cruise missiles at a Syrian airbase, escalating tensions with Russia and Iran. Oil prices hit a one-month high on concerns that the conflict could spread in the oil-rich region.

MARKET HIGHLIGHTS

Bellerophon Therapeutics released clinical data from a poster presented at the 37th Annual Meeting for the International Society for Heart and Lung Transplantation (ISHLT) in San Diego, CA on April 6th. The poster Effects of Ambulatory Inhaled Nitric Oxide on Exercise Induced Increases in Pulmonary Pressures featured data from an investigator led study of INOpulse® in patients with pulmonary arterial hypertension (PAH).

Catalyst Biosciences announced the pricing of an underwritten public offering of units for gross proceeds of $18 million, prior to deducting underwriting discounts and commissions and offering expenses payable by Catalyst. Ladenburg Thalmann & Co., a subsidiary of Ladenburg Thalmann Financial Services, is sole book-running manager in connection with the offering.

Aurinia Pharmaceuticals announced the outcome of discussions with both the European Medicines Agency (EMA) and the Pharmaceutical and Medical Devices Agency (PMDA) in Japan regarding the development of voclosporin for the treatment of active lupus nephritis (LN). Pursuant to these discussions, the Company believes that the confirmatory data that can be generated from the single Phase III clinical trial (AURORA) and the recently completed AURA-LV (AURA) Phase IIb study should support regulatory submissions in the US, Europe and Japan.

Bioblast Pharma announced the cancellation of its previously announced underwritten public offering of the Company’s securities due to adverse market conditions. H.C. Wainwright had been serving as the underwriters for the proposed offering. The offering was conducted pursuant to a registration statement Form F-1 filed with the SEC. The Company will provide a business update in the near future that deals with its financing plans and clinical development program for patients with Ocular Pharyngeal Muscular Dystrophy (OPMD) using its investigational proprietary intravenous (IV) form of trehalose 90 mg/mL solution.

STRATA Skin Sciences announced a reverse stock split of its shares of common stock at a ratio of 1-for-5.  At the opening of trading on April 7, 2017, STRATA’s common stock will begin trading on a split-adjusted basis and the number of shares of STRATA’s common stock outstanding will decrease from approximately 10.9 million pre-split shares to approximately 2.2 million post-split shares.  STRATA’s new CUSIP number will be 86272A 206, and its common stock will continue to trade on The Nasdaq Capital Market under the symbol “SSKN.”

Merck announced that the FDA has issued a CRL regarding Merck’s sNDA for JANUVIA (sitagliptin), JANUMET (sitagliptin and metformin HCl) and JANUMET XR (sitagliptin and metformin HCl extended-release). With these applications, Merck is seeking to include data from TECOS (Trial Evaluating Cardiovascular Outcomes with Sitagliptin) in the prescribing information of sitagliptin-containing medicines. Merck is reviewing the letter and will discuss next steps with the FDA.

Kura Oncology a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, today announced that the first patient has been dosed in its Phase I clinical trial of  KO-947, a potent and selective small molecule inhibitor of extracellular-signal-regulated kinases 1 and 2 (ERK1/2).

AbbVie, in cooperation with Neurocrine Biosciences, announced detailed results from a Phase IIb clinical trial evaluating the efficacy and safety of elagolix alone or in combination with add-back therapy (estradiol/norethindrone acetate) compared to placebo. The data demonstrated that elagolix, with and without add-back therapy, met the primary efficacy endpoint of reduced heavy menstrual bleeding as compared to placebo.

Medivir AB announced the completion of the Phase II clinical study of the topical, skin-directed histone deacetylase (HDAC) inhibitor, remetinostat, in patients with early stage CTCL. The trial included 60 patients with the mycosis fungoides (MF) variant of CTCL, who were randomized to receive either 0.5% remetinostat gel BID, 1% remetinostat gel QD or 1% remetinostat gel BID for between 6 and 12 months. The primary end-point of the study was the proportion of patients with either a complete or partial confirmed response to therapy, assessed using the Composite Assessment of Index Lesion Severity (CAILS). Based on an intent-to-treat analysis, patients in the 1% remetinostat gel BID arm had highest proportion of confirmed responses (8/20, 40%), including 1 complete response. The response rates in the other two arms were 5/20 (25%) and 4/20 (20%) in the 0.5% BID arm and the 1% QD arm respectively, and did not include any complete responses. Across all the dose groups, remetinostat was well-tolerated without signs of systemic adverse effects, including those associated with systemic HDAC inhibitors.

Galapagos announced a share capital increase arising from warrant exercises. Galapagos issued 247,070 new ordinary shares on 6 April 2017, for a total capital increase (including issuance premium) of €4,033,858.30. Pursuant to the warrant exercise program of Galapagos’ executive committee, executive committee members automatically are committed to exercise a minimum number of warrants, subject to certain conditions. In accordance with the rules of this program, CEO Onno van de Stolpe exercised 15,000 warrants. Two other executive committee members exercised an aggregate number of 10,000 warrants. In addition to Onno van de Stolpe, one other board member exercised 5,400 warrants.

PeptiDream announced that it has entered into a multi-target discovery and optimization collaboration with US-based Janssen Pharmaceuticals, one of the JanssenPharmaceutical Companies of Johnson & Johnson. Under the agreement, facilitated by Johnson & Johnson Innovation, PeptiDream will use its proprietary Peptide Discovery Platform System (PDPS) technology to identify macrocyclic/constrained peptides against multiple metabolic and cardiovascular targets of interest selected by Janssen, and to optimize hit peptides into therapeutic peptides or small molecule products. Janssen also holds an exercisable option to peptide-drug conjugate (PDC) use and applications. Janssen will have the right to develop and commercialize all compounds resulting from the collaboration. Under the terms of the agreement, PeptiDream would receive an undisclosed upfront payment and research funding and is eligible to receive preclinical, clinical, and commercialization milestone payments potentially totaling up to $1.15 billion. In addition, PeptiDream is eligible to receive royalties on sales of any products that arise from the collaboration.

VWR announced that it has acquired MESM Ltd., a provider of laboratory and medical equipment and ancillary supplies to clinical trials worldwide.

Flexion Therapeutics announced that Yamo Deniz, MD, has been named Chief Medical Officer. This appointment strengthens the company’s leadership with an industry veteran who brings extensive experience leading medical and clinical development teams at major biotechnology and pharmaceutical companies.

ANALYST RECOMMENDATIONS

Barclays analyst Geoff Meacham increased his price target of Incyte to $185 from $135, citing the IDO landscape is increasingly tilting in favor of Incyte’s epacadostat and increased confidence in the upcoming FDA approval and differentiation of Olumiant (baricitinib) following checks with rheumatologists.

Mizuho analyst Irina Koffler increased her price target of Ironwood Pharmaceuticals to $22 from $21, citing a more bullish outlook on the IW-3718 refractory GERD program.

Leerink analyst Geoffrey Porges decreased his price target of Biogen to $290 from $300, citing the FDA granted approval to Roche’s Ocrevus (Ocrelizumab) for the treatment of both relapsing remitting multiple sclerosis (RRMS) and primary progressive multiple sclerosis (PPMS).

BioShares Biotechnology Clinical Trials (BBC): $21.77, +$0.11, +18.3% YTD

BioShares Biotechnology Products (BBP): $36.71, +$0.22, +12.1% YTD

MARKET COMMENTARY

U.S. stock index futures were little changed as investors remained on the sidelines before the earnings season starts. Stock markets in Asia and Europe trended lower as investors remained cautious because of geopolitical tensions in the Middle East and the Korean peninsula. The dollar inched towards three-week highs drawing support from U.S. rate hike expectations, while gold prices slipped. Oil prices rose as another shutdown at Libya’s largest oilfield and heightened tension over Syria following U.S. missile attacks raised supply concerns.

MARKET HIGHLIGHTS

Rockwell Medical announced today a poster presentation at the National Kidney Foundation Spring Clinical Meeting, Wednesday, April 19th at 6:00PM – 7:30PM EDT during the opening session in the exhibit hall at the Swan and Dolphin Exhibit Hall in Orlando, FL.

Albireo Pharma announced that it will host a Key Opinion Leader breakfast meeting focused on rare pediatric liver diseases at the Lotte New York Palace in New York City on Wednesday, April 12, 2017, from 8:00 a.m. to 9:30 a.m. ET.

TherapeuticsMD announced that, on April 7, 2017, the Company received a letter from the U.S. Food and Drug Administration (FDA) stating that, as part of the FDA’s ongoing review of the Company’s new drug application (NDA) for TX-004HR, the Company’s applicator-free vaginal estradiol softgel drug candidate for the treatment of moderate to severe dyspareunia (vaginal pain during sexual intercourse), a symptom of vulvar and vaginal atrophy (VVA), in post-menopausal women, the FDA has identified deficiencies that preclude discussion of labeling and postmarketing requirements/commitments at this time. The letter states that the notification does not reflect a final decision on the information under review.  The letter does not specify the deficiencies identified by the FDA and at this time the Company is not aware of the nature of the deficiencies. The Company intends to work with the FDA to understand the nature of the deficiencies and resolve them as quickly as possible.

Valneva SE announced today that it will be holding two presentations on Lyme disease and mosquito-transmitted diseases on April 11, 2017 at the 17th World Vaccine Congress in Washington DC. Over 600 vaccine specialists will converge to the event.

MediciNova announced that an abstract regarding a mouse model study that examined the potential clinical efficacy of MN-166 for glioblastoma (GBM) has been accepted for presentation at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting to be held June 2-6, 2017 in Chicago, Illinois.

Cytori Therapeutics announced that the FDA has approved an Investigational Device Exemption (IDE) for a pilot clinical trial to evaluate Cytori Cell Therapy in patients with thermal burn injury. This trial, named the RELIEF trial, is a continuation of Cytori’s ongoing research and development efforts under its contract with the Biomedical Advanced Research and Development Authority (BARDA), a division of the U.S. Department of Health and Human Services.

Geron announced that Janssen Research & Development has completed the second internal data reviews of IMerge and IMbark, the clinical trials of the telomerase inhibitor imetelstat in lower risk myelodysplastic syndromes (MDS) and relapsed or refractory myelofibrosis (MF), respectively. For IMerge, the benefit/risk profile of imetelstat in the treated patients supports continued development in lower risk myelodysplastic syndromes. A data package and proposed trial design refinements are planned to be provided to the FDA. For IMbark, the current results suggest clinical benefit and a potential overall survival benefit associated with imetelstat treatment in relapsed or refractory myelofibrosis; the trial will continue unchanged to evaluate maturing efficacy and safety data, including an assessment of overall survival.

Intellipharmaceutics International announced that Purdue Pharma L.P., Purdue Pharmaceuticals L.P., The P.F. Laboratories, Inc., Rhodes Technologies, and Grünenthal GmbH have commenced patent infringement proceedings against the Company in the United States District Court for the District of Delaware in respect of the Company’s NDA filing for its Rexista product candidate (abuse-deterrent oxycodone hydrochloride extended release tablets) with the FDA.

Akorn confirmed that Akorn is currently in discussions with Fresenius Kabi, a subsidiary of Fresenius SE & Co. KGaA concerning a potential acquisition of Akorn.

Mesoblast announced that the Phase III trial of its allogeneic mesenchymal precursor cell (MPC) product candidate MPC-150-IM in patients with moderate to advanced chronic heart failure (CHF) was successful in the pre-specified interim futility analysis of the efficacy endpoint in the trial’s first 270 patients. It is expected that the trial will enroll in total approximately 600 patients. After notifying the Company of the interim analysis results, the trial’s Independent Data Monitoring Committee (IDMC) additionally stated that they had no safety concerns relating to MPC-150-IM and formally recommended that the trial should continue as planned.

NantCell, part of the NantWorks ecosystem of companies, announced that the FDA has granted Orphan Drug Designation to the company’s Ganitumab, Insulin Growth Factor-1R (IGF-1R) monoclonal antibody therapy for the treatment of patients with Ewing sarcoma.

InVivo Therapeutics announced that the Brigham and Women’s Hospital (BWH) in Boston, MA has been added as a clinical site for The INSPIRE Study: InVivo Study of Probable Benefit of the Neuro-Spinal Scaffold for Safety and Neurologic Recovery in Subjects with Complete Thoracic AIS A Spinal Cord Injury. BWH is a teaching hospital of Harvard Medical School and hosts one of the largest neurosurgical intensive care units in the country.

OncoMed Pharmaceuticals reported top-line results from the company’s Phase II YOSEMITE clinical trial of demcizumab (anti-DLL4, OMP-21M18) in combination with Abraxane (paclitaxel protein-bound particles for injectable suspension) (albumin bound) plus gemcitabine in previously untreated patients with metastatic pancreatic cancer.  The randomized Phase II “YOSEMITE” trial was designed to assess the efficacy and safety of demcizumab plus standard-of-care chemotherapy in first-line metastatic pancreatic cancer with the primary endpoint of progression-free survival and a secondary endpoint of overall survival.  The trial did not meet the primary endpoint of progression-free survival.  Additionally, the interim median overall survival analysis did not show a benefit for demcizumab in combination with Abraxane plus gemcitabine compared to the Abraxane, gemcitabine plus placebo arm in patients with first-line metastatic pancreatic cancer.

Anthera Pharmaceuticals announced the completion of dosing in the randomized, double-blind, placebo controlled, Phase II BRIGHT-SC study of blisibimod in patients with IgA nephropathy (IgAN).  After Week 24, patients were given the opportunity to continue blinded treatment for up to 104 weeks, discontinue treatment but continue to be followed, or discontinue from the study.  Most patients, 42 of 57, completed at least 60 weeks of evaluation and 21 completed assessments through at least 104 weeks.  Anthera anticipates reporting top-line data in Q3.

Mast Therapeutics and Savara announced that, pending completion of their proposed merger, the combined company’s common stock has been approved for listing on the Nasdaq Capital Market under the symbol "SVRA."  Trading on the Nasdaq Capital Market is expected to commence on the first business day after completion of the merger.  Mast’s common stock is expected to continue to trade on the NYSE MKT until completion of the merger.  Mast has provided notification to the NYSE MKT of its intent to voluntarily withdraw its common stock from listing and trading on NYSE MKT in connection with the completion of the merger and the move to Nasdaq.

Syros Pharmaceuticals announced that the FDA accepted the Company’s IND application to advance SY-1365, its first-in-class selective cyclin-dependent kinase 7 (CDK7) inhibitor, into a Phase I clinical trial in patients with advanced solid tumor malignancies, including transcriptionally dependent cancers such as triple negative breast, small cell lung and ovarian cancers. Syros is on track to initiate the Phase I trial in the second quarter of 2017.

OncoMed Pharmaceuticals announced that Bayer Pharma has notified OncoMed of its decision not to exercise its option to license the first-in-class Wnt pathway inhibitors vantictumab (anti-Fzd, OMP-18R5) and ipafricept (Fzd8-Fc, OMP-54F28) for strategic reasons.  Effective June 2017, OncoMed will retain worldwide development and commercialization rights to vantictumab, ipafricept and all other Wnt pathway biologics under the collaboration.  The small molecule program under the companies’ collaboration continues without change.

Cellular Biomedicine Group announced that they have established a strategic research collaboration to co-develop certain high-quality industrial control processes in Chimeric Antigen Receptor T-cell (CAR-T) and stem cell manufacturing.  In connection with the collaboration, a joint laboratory within CBMG’s new Shanghai Zhangjiang GMP-facility will be established and dedicated to the joint research and development of a functionally integrated and automated immunotherapy cell preparation system.

Axovant Sciences announced that David Hung, MD, has been named CEO of Axovant Sciences, effective April 7, 2017. Dr. Hung, who also joined Axovant’s Board of Directors, succeeds founding CEO Vivek Ramaswamy. Mr. Ramaswamy will continue to serve on Axovant’s Board of Directors and will lead parent company and majority owner Roivant Sciences as its full-time CEO.

Aptevo Therapeutics announced the presentation of new clinical data evaluating the safety and efficacy of IXINITY [Coagulation Factor IX (Recombinant)] in previously treated patients under 12 years of age with Hemophilia B.  The data suggest that IXINITY appears to be safe and well tolerated in this subject population, and is comparable to the results from the overall patient population studied in the pivotal clinical trial of IXINITY.

Chugai Pharmaceutical announced that Alecensa as an initial (first-line) treatment showed that patients lived significantly longer without disease worsening (progression-free survival, PFS) compared to crizotinib in the ALEX Study, a global phase III study targeting ALK fusion gene positive non-small cell lung cancer (NSCLC), conducted by F. Hoffmann-La Roche Ltd. The safety profile of Alecensa was consistent with that observed in previous studies, with no new or unexpected adverse events.

Ferring Pharmaceuticals and Alrise Biosystems announced that the companies have entered into a development agreement with exclusive option rights for Ferring to leverage Alrise’s ImSus Technology Platform for the development of an injectable, controlled-release formulation of a peptide therapeutic.

Innoviva announced that on May 15, 2017, the next interest payment date under its non-recourse royalty notes due 2029 (the “Royalty Notes”), Innoviva will prepay $50 million in outstanding principal, representing a substantial portion of the Company’s $150 million capital return plan for 2017.

Aerie Pharmaceuticals announced the appointments of new employees Gary Menichini as Vice President of Sales, Dale Seibt as Vice President of Market Access, and Gerry McKenzie as Vice President of Commercial Operations. In addition, Michael McCleerey has changed positions within the Company and is now Vice President of Portfolio Development. All four positions are newly established and will report to Judith Robertson, Chief Commercial Officer.

Ovid Therapeutics announced that it has initiated a Phase I clinical trial to evaluate the pharmacokinetics (PK), safety and tolerability of OV101 in adolescents diagnosed with Angelman syndrome or Fragile X syndrome. OV101 (gaboxadol), a delta (?)-selective GABA(A) receptor agonist, is believed to be the first investigational drug to target the disruption of tonic inhibition, a key mechanism that allows a healthy human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. Tonic inhibition is believed to play a significant role in the neurodevelopmental symptoms characteristic of disorders such as Angelman syndrome and Fragile X syndrome.

Portage Biotech announced that Biohaven Pharmaceutical Holding Company, in which Portage holds an equity interest of approximately 28.3%, issued a press release announcing that it filed a registration statement on Form S-1 with the SEC relating to a proposed offering of its common shares.

Reuters reported that Stada said it has decided to support an offer from Bain Capital and Cinven for 66 euros per share, valuing the company at about 5.32 billion euros. The private equity consortium is offering 65.28 euros per share and a dividend of 0.72 euro per Stada share, the company said in a statement. Stada, which had received offers from two consortia, said it has signed an investor agreement which would include protection provisions for employees.

Genentech, a member of the Roche Group, announced that the global, randomized Phase III ALEX study met its primary endpoint and showed that Alecensa (alectinib) as an initial (first-line) treatment significantly reduced the risk of disease worsening or death (progression-free survival, PFS) compared to crizotinib in people with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC). This is the second Phase III trial to show that Alecensa was superior as an initial treatment compared to crizotinib in this type of lung cancer. The safety profile of Alecensa was consistent with that observed in previous studies, with no new or unexpected adverse events.

Alexo Therapeutics announced that it has initiated dosing in its Phase I clinical program evaluating the safety of ALX148 in patients with advanced solid tumors and lymphoma.

ANALYST RECOMMENDATIONS

Piper Jaffray analyst Joshua Schimmer initiated coverage of Gemphire with an “overweight” rating and $30 price target, citing a minimal ~ $100M mkt cap and a clinically validated and differentiated asset.

Janney Capital analyst Debjit Chattopadhyay initiated coverage of TESARO with a “neutral” rating and $141 price target, citing TSRO is relatively well-positioned after Zejula’s broad label unencumbered by a companion diagnostic, although transient myelo-suppression is a concern for some physicians.

RBC suspended coverage of the following companies due to the departure of both Adnan Butt and Michael Yee: Acorda Therapeutics, Aerie Pharmaceuticals, Amgen, Arbutus Biopharma, ArQule, Axovant Sciences, Biogen, BioMarin Pharmaceutical, Celgene Corporation, Curis, Endocyte, Esperion Therapeutics, FibroGen, Forward Pharma, Gemphire Therapeutics, Gilead, Intercept Pharmaceuticals, Intra-Cellular Therapies, Kite Pharma, Nabriva Therapeutics, Ocular Therapeutix, PDL BioPharma, Proteostasis, Prothena, Regeneron Pharmaceuticals, SCYNEXIS, Inc. Seattle Genetics, Spark Therapeutics, Spectrum Pharmaceuticals, Synthetic Biologics, TESARO, The Medicines Company, United Therapeutics, and Vertex Pharmaceuticals. 

Ladenburg Thalman analyst Kevin DeGeeter increased his price target of Mesoblast to $13.25 from $7.35, citing a positive interim futility analysis from the company’s Phase III program of MPC-150-IM in patients with moderate to advanced chronic heart failure.

HC Wainwright analyst Shaunak Deepak increased his price target of Rigel to $6 from $5, citing a reflection of BerGenBio value following Olso IPO.

Rodman & Renshaw analyst Joseph Pantginis assumed coverage of Celsion with a “buy” rating and $1.50 price target, citing Celsion’s lead asset is ThermoDox, which is a thermally sensitive liposome containing doxorubicin.

BioShares Biotechnology Clinical Trials (BBC): $21.77, $0.00, +18.3% YTD

BioShares Biotechnology Products (BBP): $36.45, -$0.26, +11.3% YTD

MARKET COMMENTARY

U.S. stock index futures were little changed as investors abstained from betting on riskier assets amid rising global geopolitical tensions. Safe-haven assets benefited, with the yen recording broad gains against major currencies and gold hit its highest level since November. On the economic front, the U.S. Labor Department is expected to issue its job openings and labor turnover survey for February. Weakness in shares of banking and tech companies weighed on European bourses, while Asian markets ended on mixed. Oil eased as rising U.S. shale oil production reduced concerns over geopolitical tensions in the Middle East.

MARKET HIGHLIGHTS

Windtree Therapeutics announced that the AEROSURF® (lucinactant for inhalation) phase 2b independent Data Safety Monitoring Board (DSMB) has completed its second and final interim safety review and has recommended continuing the trial without modification. The final DSMB interim review was convened following achievement in mid-March 2017 of a pre-specified patient enrollment milestone.  In addition, the Company reaffirms its plan to announce top-line results from the AEROSURF phase 2b clinical trial in mid-year 2017.

Catalyst Biosciences has achieved a key milestone under its collaboration with ISU Abxis to advance the Factor IX program towards its first human clinical trial. Completion of the CB 2679d/ISU304 toxicology studies supported the recent Investigational New Drug (IND) approval by the Korean Ministry of Food and Drug Safety (MFDS) and triggered a milestone payment to Catalyst.

AzurRx BioPharma provided an update on the ongoing open-label Phase IIa trial of lead candidate MS1819 for the treatment of exocrine pancreatic insufficiency (EPI) associated with chronic pancreatitis (CP). The trial is being conducted in partnership with Mayoly Spindler, a European pharmaceutical company.  Early results indicate that MS1819 exhibits a favorable safety profile, with no moderate or severe adverse events having been reported to date.  Additionally, no patients have dropped out of the study once they have qualified to take the active drug.  Regarding efficacy, results to date indicate that MS1819 is active, as measured by coefficient of fat absorption (CFA), which is the parameter being measured for the primary efficacy endpoint.  There was a favorable dose response of greater than 20% improvement in CFA seen in all patients at either the second or third of the four escalating dosage levels of MS 1819. 

Ohr Pharmaceutical announced that it plans to amend the ongoing clinical trial investigating Squalamine in wet-AMD (the MAKO Study) to enable efficacy analyses by the end of calendar 2017 or early 2018.  The study remains a multi-center, randomized, double-masked, placebo controlled clinical trial.  The subjects enrolled in the study, over 200 in total, will continue to receive their assigned study treatment of monthly Lucentis® and either Squalamine or placebo drops twice daily, and undergo scheduled visits and assessments through nine months.  The primary endpoint will be an assessment of visual acuity at nine months.  Separately, Ohr announced announced that it has closed its previously announced public offering of common stock and warrants. The Company sold an aggregate 20,250,032 shares of common stock at a price to the public of $0.70 per share. Investors also received warrants to purchase up to an aggregate of 14,175,059 shares of common stock with an exercise price of $1.00. The warrants are immediately exercisable and have a term of five years. Gross proceeds to the Company from the sale of the shares were approximately $14,175,000, excluding any proceeds from the exercise of warrants. The transaction was led by existing investors and included the participation of management and the board of directors.

TRACON Pharmaceuticals announced that the Company’s Phase 3 TAPPAS clinical trial of TRC105 in angiosarcoma was awarded Most Innovative Clinical Trial Design at the 2017 Clinical and Research Excellence (CARE) Awards, which are presented by Informa’s Pharma Intelligence, a leading provider of drug, device, company, clinical trial, and market intelligence.

CTD Holdings issued an Open Letter to address false and misleading statements, recently made by another public company, regarding CTD’s hydroxypropyl beta cyclodextrin (HPBCD) product, Trappsol® Cyclo™, and its clinical use to treat Niemann-Pick Disease Type C (NPC). The false and misleading statements were made as part of another public company’s announcement of their acquisition of a hydroxypropyl beta cyclodextrin product.

Aurinia Pharmaceuticals announced that it will host an Investor Event during the National Kidney Foundation Spring Clinical Meeting on April 20, 2017 at 6:00pm Eastern Time in Orlando, FL.

Transgene announced that management will participate in the Kempen 10th Life Sciences Conference on April 19^th in Amsterdam, Netherlands, and the Kepler Biotech Days on June 9^th in Paris, France.  Transgene will host a “R&D Day”, on June 22, 2017 in Paris, France.

Tonix Pharmaceuticals announced the receipt of official minutes from its Initial Cross-Disciplinary Breakthrough Meeting held with the FDA on March 9, 2017. Upon being awarded Breakthrough Therapy designation in December 2016, Tonix was invited to meet with the FDA to evaluate the feasibility of accelerating the development and registration of TNX-102 SL for the treatment of posttraumatic stress disorder (PTSD).

Idera Pharmaceuticals a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare diseases, today announces successful completion of the Phase I portion of the ongoing Phase I/II clinical trial of intratumoral IMO-2125. Intratumoral IMO-2125 is an agonist of TLR9, in combination with ipilimumab for the treatment of anti-PD-1 refractory metastatic melanoma. Enrollment has begun for the Phase II portion of the trial with the 8mg dose of intratumoral IMO-2125.  The Phase I dose escalation of IMO-2125 in combination with pembrolizumab is ongoing.

Intellia Therapeutics announced that the European Patent Office (EPO) has decided to grant a patent broadly covering the CRISPR/Cas9 genome editing technology. The patent includes claims covering compositions of the widely adopted CRISPR single guide RNA technology for use in any non-cellular and cellular setting, including eukaryotic cells such as human or mammalian cells, as well as for use in human therapeutics.

Inotek Pharmaceuticals announced the completion of the active recruitment phase of the Phase II dose-ranging trial of a fixed-dose combination (FDC) of trabodenoson and latanoprost for the treatment of glaucoma. Trabodenoson is a highly selective adenosine mimetic that lowers intraocular pressure (IOP) by augmenting the natural function of the trabecular meshwork, the primary outflow pathway in the eye. Latanoprost, a prostaglandin analog, targets the secondary uveoscleral pathway and is the most commonly used drug for lowering IOP. Top-line data from the FDC study are expected in July.

Inovio Pharmaceuticals announced preliminary results from the expanded stage of its Phase I study, EBOV-001. The expanded study examined different regimens of its Ebola DNA vaccine INO-4201 using intradermal (skin) administration. The results across both stages of the trial, including both intramuscular and intradermal delivery, demonstrated that 95% (170/179) of evaluable subjects generated an Ebola-specific antibody immune response, with the mean antibody titer comparable or superior to those reported from viral vector-based Ebola vaccines.

Bristol-Myers Squibb and Apexigen announced a clinical trial collaboration to evaluate Bristol-Myers Squibb’s Opdivo  (nivolumab) in combination with Apexigen’s APX005M in patients with advanced solid tumors. APX005M is an investigational compound that is designed to activate CD40, a key immune co-stimulatory receptor essential to regulating the activation of both innate and adaptive immune responses against cancer.

ArQule announced that it has received clearance from the FDA for the IND application to conduct a phase I clinical trial with ARQ 531 in patients with B-cell malignancies who are refractory to other therapeutic options. ARQ 531 is an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK).

Agile Therapeutics announced it has received the final meeting minutes from its recent NDA pre-submission meeting with the FDA for its lead product candidate, Twirla, an investigational low-dose combined hormonal contraceptive patch. Based on the feedback from the FDA, Agile believes it has the necessary information to complete the resubmission of its NDA, which is expected to be submitted by the end of the second quarter of 2017.

Nabriva Therapeutics announced that it has completed enrollment in its first lefamulin evaluation against pneumonia (LEAP 1) Phase III clinical trial. LEAP 1, which is designed to assess the efficacy and safety of lefamulin (IV/oral) compared to moxifloxacin (IV/oral) in patients with moderate to severe community-acquired bacterial pneumonia (CABP), met its enrollment target of 550 patients. The company anticipates availability of top-line clinical data from LEAP 1 in the third quarter of 2017.

Five Prime Therapeutics announced that it has completed enrollment in the initially planned 30-patient cohort of the Phase II part of the ongoing clinical trial evaluating cabiralizumab in patients with tenosynovial giant cell tumor, diffuse type, also known as pigmented villonodular synovitis (PVNS), an aggressive tumor confined to the synovium.

PTC Therapeutics announced that Shane Kovacs, Chief Financial Officer, will be leaving the company in mid-May 2017 to return to the finance sector, joining RBC Capital Markets as Managing Director where he will help lead the firm’s biotech investment banking efforts.

Axovant Sciences announced the pricing of its underwritten public offering of 6,742,179 of its common shares at a price to the public of $18.54 per share. Gross proceeds to Axovant from the offering are expected to be approximately $125.0 million, before deducting underwriting discounts and commissions and estimated offering expenses.  All of the common shares are being offered by Axovant. In connection with this offering, Axovant has granted the underwriters a 30-day option to purchase up to an additional 1,011,326 of its common shares on the same terms and conditions.  The offering is expected to close on April 17, 2017, subject to customary closing conditions.

Cytori Therapeutics announced that it has priced an underwritten public offering of 8,600,000 shares of its common stock at a price to the public of $1.10 per share. Gross proceeds, before underwriting discounts and commissions and estimated offering costs, are expected to be approximately $9.5 million. Cytori currently intends to use the net proceeds of the offering for working capital and general corporate purposes, including funding of the HABEO and ATI-0918 development programs. Cytori has granted the underwriter a 45-day option to purchase up to 15% of additional shares of its common stock. The offering is expected to close on or about April 17, 2017, subject to satisfaction of customary closing conditions. Maxim Group is acting as sole book-running manager for the offering.

ANALYST RECOMMENDATIONS

HC Wainwright analyst Ed Arce increased his price target of Paratek to $36 from $33, citing top-line results from its pivotal Phase 3 trial of omadacycline for the treatment of community-acquired bacterial pneumonia (CABP) that demonstrates the drug’s strong efficacy across the board, and better overall safety than active comparator, moxifloxacin.

Cantor analyst Elemer Piros initiated coverage of Aurinia Pharmaceuticals with an “overweight” rating and $14 price target, citing voclosporin, a drug analog of cyclosporine, has demonstrated compelling efficacy in its Phase 2b AURA-LV study for patients with active lupus nephritis (LN) and is well-positioned in late-stage development to succeed.

FBR analyst Vernon Bernardino increased his price target of Aurinia Pharmaceuticals to $11 from $9, citing AUPH is now in a strong position to independently advance voclosporin (VCS), its investigational therapy for patients with active lupus nephritis (LN), into Phase III testing.

Wedbush analyst Robert Driscoll assumed coverage of Cidara Therapeutics with an “outperform” rating and $13 price target, citing the upbeat tone at the analyst day, and see the re-focused development strategy on targeting life-threatening infections with IV therapies as risk-reduced.

Chardan analyst Gbola Amusa increased his price target of Regenxbio to $50 from $35, citing conviction on the landmark Danos appointment.

Following OncoMed’s announcement that demcizumab had missed its primary endpoint in the Phase 2 YOSEMITE trial in pancreatic cancer and its partner Bayer had decided not to opt-in to develop Wnt inhibitors vantictumab and ipafricept; HC Wainwright analyst Shaunak Deepak decreased his price target to $9 from $20; BMO analyst Ian Somaiya downgraded the stock to “market perform” from “outperform” and decreased his price target to $6 from $18; SunTrust analyst Peter Lawson decreased his price target to $9 from $16; Jefferies analyst Brian Abrahams decreased his price target to $10 from $15. 

Chardan analyst Gbola Amusa increased his price target of Avexis to $102.50 from $100, citing model updates.

BioShares Biotechnology Clinical Trials (BBC): $21.64, -$0.13, +17.5% YTD

BioShares Biotechnology Products (BBP): $36.47, +$0.02, +11.4% YTD

MARKET COMMENTARY

Futures pointed to a modestly lower start for Wall Street as focus shifted towards earnings season which kick starts on Thursday. Data on import and export prices occupies the day’s economic calendar. Federal budget from the U.S. Treasury Department is also on the economic radar. European equities trended higher buoyed by gains in the shares of financials and carmakers, while stock markets in Asia recorded a mixed trading day. The yen recorded broad gains against major currencies and gold steadied after hitting a five-month peak. Oil extended gains after Saudi Arabia was said to be pushing its fellow OPEC members and some rivals to prolong supply cuts beyond June.

MARKET HIGHLIGHTS

On April 5, 2017, Prothena Therapeutics Limited and Prothena Biosciences Limited, wholly owned subsidiaries of Prothena Corporation, entered into a Letter Agreement with Boehringer Ingelheim Biopharmaceuticals GmbH ("BI"), effective as of March 31, 2017, under which BI reserved for Prothena capacity to manufacture and supply bulk drug substance of NEOD001 (Prothena’s investigational monoclonal antibody for the potential treatment of AL amyloidosis), PRX003 (Prothena’s investigational monoclonal antibody for the potential treatment of inflammatory diseases, including psoriasis and psoriatic arthritis) and possibly additional biologics, and Prothena made commitments for the purchase of Products from BI.

Immune Pharmaceuticals announced a reverse stock split of its shares of common stock at a ratio of 1-for-20. Beginning with the opening of trading on April 13, 2017, the Company’s common stock will continue to trade The Nasdaq Capital Market ("Nasdaq") under the symbol "IMNP," but will trade on a split-adjusted basis under a new CUSIP number 45254C 200.  The reverse stock split was approved by the Company’s stockholders at the Company’s 2016 Annual Meeting on December 20, 2016.

Novan announced positive topline results from the Company’s Phase II clinical trial with SB208, a topical, silicone based-gel under development for the treatment of infections caused by dermatophytes such as Trichophyton rubrum, or T. rubrum. Novan is developing SB208 as a broad-spectrum antifungal gel for the treatment of superficial cutaneous fungal infections of the skin and nails, including tinea pedis and onychomycosis. Novan’s SB208 Gel, at both the 4% and 16% concentrations, demonstrated a statistically significant effect (p <0.05) compared to vehicle in a clinical trial in patients with tinea pedis, or athlete’s foot. Clinical activity against dermatophytes was measured by incidence of a negative fungal culture after two weeks of treatment.

Neurocrine Biosciences announced that the FDA has approved INGREZZA (valbenazine) capsules for the treatment of adults with tardive dyskinesia (TD). INGREZZA, a novel, selective vesicular monoamine transporter 2 (VMAT2) inhibitor, is the first and only FDA-approved product indicated for the treatment of adults with TD. Following the news, Leerink analyst Paul Matteis increased his price target to $68 from $65; Jefferies analyst Biren Amin increased his price target to $65 from $58.

Synergy Pharmaceuticals announced that the USPTO has issued three new patents covering TRULANCE (plecanatide). The first patent relates to the method for manufacturing TRULANCE and will expire March 1, 2032. The two other patents relate to formulations and methods of using TRULANCE for treating chronic idiopathic constipation (CIC) and irritable bowel syndrome with constipation (IBS-C) at 3mg or 6 mg dose; both of these patents will expire September 15, 2031.

Protalix BioTherapeutics announced positive results from the Company’s Phase II clinical trial of alidornase alfa for the treatment of Cystic Fibrosis (CF).  Sixteen patients were enrolled in the study, all of whom completed the study.  alidornase alfa is a plant cell-expressed, chemically-modified recombinant DNase enzyme resistant to inhibition by actin, which the Company has specifically designed to enhance the enzyme’s efficacy in CF patients.

BioCryst Pharmaceuticals announced today plans to explore a new oral liquid formulation of BCX7353 for the treatment of acute attacks in patients with hereditary angioedema (HAE).  The Company has received initial regulatory approvals in Europe to initiate the ZENITH-1 exploratory clinical trial this summer.

Oncolytics Biotech announced its initial registration pathway and clinical development plan for REOLYSIN, its proprietary immuno-oncology viral agent.  The Company’s clinical development plan has two main objectives. The primary objective is to obtain regulatory approval for REOLYSIN as quickly as possible and is based on the compelling metastatic breast cancer survival data recently presented at the American Academy of Cancer Research (AACR) Annual Meeting, in Washington, D.C.  The second objective is to expand REOLYSIN into commercially valuable new treatment areas that include immunotherapy and immunomodulatory (IMiD) agents in collaboration with pharmaceutical partners.

Dicerna Pharmaceuticals announced that it has closed its previously announced stock purchase transaction for the sale of redeemable convertible preferred stock to a syndicate of current and new investors led by Bain Capital Life Sciences, under which the Company received gross proceeds of $70.0 million. Under terms of the Preferred Stock purchase agreement, Adam M. Koppel, M.D., Ph.D., a managing director of Bain Capital Life Sciences, has joined Dicerna’s Board of Directors, which has been expanded to nine seats.

Neuralstem announced that a new cohort of four patients will be added to its ongoing Phase I human clinical trial evaluating the safety and feasibility of using NSI-566 spinal cord-derived neural stem cells to repair chronic spinal cord injury (cSCI). The amended protocol was approved by the FDA and the Institutional Review Board at the study site, University of California San Diego (UCSD).  NSI-566 is Neuralstem’s lead stem cell therapy candidate.

Five Prime Therapeutics announced that it has completed enrollment in the initially planned 30-patient cohort of the Phase II part of the ongoing clinical trial evaluating cabiralizumab in patients with tenosynovial giant cell tumor, diffuse type, also known as pigmented villonodular synovitis (PVNS), an aggressive tumor confined to the synovium.

Kadmon Holdings announced the publication of clinical data from its completed Phase II open-label clinical trial of KD025, its oral Rho-associated coiled-coil kinase 2 (“ROCK2”) inhibitor, in patients with moderate to severe psoriasis. In the study, KD025 treatment improved clinical scores and skin pathology in psoriasis patients via concurrent modulation of the pro- and anti-inflammatory immune cell response. The results were published this week in the Cutting Edge section of the Journal of Immunology.

Cytori Therapeutics announced that it intends to offer and sell shares of its common stock in an underwritten public offering. Cytori currently intends to use the net proceeds of the offering for working capital and general corporate purposes, including funding of the HABEO and ATI-0918 development programs. Maxim Group is acting as sole book-running manager for the offering. 

Axovant Sciences announced that it has commenced an underwritten public offering of $100 million of its common shares. In connection with this offering, Axovant expects to grant the underwriters a 30-day option to purchase up to an additional $15 million of its common shares in the offering on the same terms and conditions. J.P. Morgan Securities and Morgan Stanley are acting as joint book-running managers for the offering.

Cellectar Biosciences announced it has appointed John Friend, II, M.D. as vice president and chief medical officer effective April 17, 2017.

ANALYST RECOMMENDATIONS

Piper Jaffray analyst Steven Breazzano initiated coverage of Prothena with an “overweight” rating and $69 price target, citing a favorable view of the company’s lead drug, NEOD001 for AL amyloidosis.

Aegis analyst Robert LeBoyer initiated coverage of Neurotrope with a “buy” rating and $31 price target, citing the company is developing Bryostatin-1 as a stimulator of PKCε, an early regulator of brain cell growth and repair.

Ladenburg Thalman analyst Kevin DeGeeter initiated coverage of Cidara Therapeutics with a “buy” rating and $11.25 price target, citing CDTX has a balanced portfolio of infectious disease therapeutics led by an experienced management team with a track record of successful clinical development and returning value for shareholders.

Piper Jaffray analyst initiated coverage of Tesaro with an “overweight” rating and $180 price target, citing a LT bullish view of the PARPi class, and TSRO’s recently approved Zejula (niraparib) in ovarian cancer is poised to become a major blockbuster driven by broad use in multiple solid tumors.

BioShares Biotechnology Clinical Trials (BBC): $21.57, -$0.07, +17.2% YTD

BioShares Biotechnology Products (BBP): $36.44, -$0.03, +11.3% YTD

MARKET COMMENTARY

The dollar slid against a basket of currencies, while gold rose, after U.S. President Donald Trump’s comments that he would like to see interest rates stay low and that the greenback was too strong. U.S. stock index futures indicated a lower start on Wall Street as focus shifted to quarterly results of major banks such as Wells Fargo and Citigroup. Weekly jobless claims and producer prices data occupies the day’s economic calendar. The University of Michigan will also release its consumer sentiment data for April. Asian bourses fell, and financial shares dragged European markets lower. Oil was little changed on concerns about rising U.S. output.

MARKET HIGHLIGHTS

Transgene announced that the first patient with soft tissue sarcoma (STS) has been treated in the Phase 2 part of the METROmaJX clinical trial at Institut Bergonié (Bordeaux, France). METROmaJX is a Phase 1/2 clinical trial evaluating the tolerability and efficacy of the co-administration of Pexa-Vec with metronomic cyclophosphamide (low doses given with high frequency) in patients with advanced solid tumors such as breast cancer and STS (NCT02630368).

Rexahn Pharmaceuticals announced that it will implement a 1-for-10 reverse stock split of outstanding shares of the Company’s common stock, together with a corresponding proportional reduction in the number of authorized shares of the Company’s capital stock. The Company expects that the reverse stock split will be effective on May 5, 2017 upon the filing and effectiveness of a Certificate of Amendment to the Company’s Certificate of Incorporation, and that trading of the Company’s common stock on the NYSE MKT will begin on a split-adjusted basis at the opening of trading on May 5, 2017. The authorized share reduction will be effective concurrently with the reverse stock split. As a result of the reverse stock split, each 10 shares of issued common stock will be converted into one share of common stock. Shareholders will receive cash in lieu of any fraction of a share that they would otherwise be entitled to receive as a result of the reverse stock split.

DelMar Pharmaceuticals announced that it has priced a registered public offering of an aggregate of 2,769,232 shares of common stock and warrants to purchase an aggregate of 2,076,924 shares of common stock at a price to the public of $3.25 per share and related warrant. The warrants have an exercise price of $3.50 per share, are immediately exercisable and have a term of exercise of five years. The offering is expected to close on or about April 18, 2017, subject to the satisfaction of customary closing conditions.

CymaBay Therapeutics announced that data from its Phase 2 proof-of-concept study of seladelpar in patients with primary biliary cholangitis (PBC) will be presented at the International Liver Congress™  2017 sponsored by the European Association for the Study of Liver Diseases (EASL), in Amsterdam, The Netherlands, April 19-23 2017. Seladelpar is an orally administered potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist.

Achaogen announced seven upcoming presentations on plazomicin at the 27th European Congress of Clinical Microbiology and Infectious Disease (ECCMID). The company and its collaborators will deliver two oral presentations and five poster presentations at the event, which will be held in Vienna, Austria from April 22 to 25, 2017. Achaogen is developing plazomicin, its lead product candidate, to treat serious bacterial infections due to MDR Enterobacteriaceae, including carbapenem-resistant Enterobacteriaceae (CRE).

Aimmune Therapeutics announced the appointment of Eric Bjerkholt as Chief Financial Officer.  Mr. Bjerkholt joins Aimmune from Sunesis Pharmaceuticals, Inc., where, over 13 years with the company, his role as CFO expanded to oversee multiple aspects of governance, corporate relations, and other functions.  Mr. Bjerkholt holds a Cand. Oecon. (master’s) degree in economics from the University of Oslo and an M.B.A. from Harvard Business School. He has served on the boards of directors of several publicly traded companies and is currently a member of the board of directors and chair of the audit committee for Corium International, Inc.

Ocera Therapeutics announced that data from an investigator-sponsored preclinical study of Ocera’s validated ammonia scavenger, ornithine phenylacetate (OCR-002), will be presented in a poster session at The International Liver Congress™ of the European Association for the Study of the Liver (EASL 2017) in Amsterdam, The Netherlands, on Thursday, April 20, 2017.

Catalyst Biosciences announced the closing of an underwritten public offering of units for gross proceeds of $20.7 million, which includes the full exercise of the underwriters’ over-allotment option to purchase additional shares and warrants, prior to deducting underwriting discounts and commissions and offering expenses payable by Catalyst.

Aerie Pharmaceuticals reported the successful six-month topline safety and efficacy results of its Rocket 4 Phase III clinical trial for product candidate Rhopressa, a novel once-daily eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. The Rocket 4 trial was designed to provide six-month safety data adequate for European regulatory filing purposes. Rocket 4 was not necessary for U.S. NDA filing purposes, although data from the 90-day efficacy component of the trial was included in the February 2017 Rhopressa NDA submission as supportive.   Rocket 4 enrollment totaled approximately 700 patients and was a two-arm six-month trial, which included a 90-day efficacy readout evaluating once-daily Rhopressa for non-inferiority to twice-daily timolol. The 90-day efficacy readout, which took place in October 2016, demonstrated successful achievement of the 90-day primary efficacy endpoint. This final readout from Rocket 4 includes six-month safety data and diurnal efficacy measurements at months four, five and six, which do not constitute a primary efficacy endpoint.

AVEO Oncology announced that its European licensee for tivozanib, EUSA Pharma, has submitted responses to the EMA Day 180 List of Outstanding Issues (LOI) related to the MAA for tivozanib as a first-line treatment for renal cell carcinoma. With submission of the response complete, EUSA remains tentatively scheduled to provide an oral explanation to the EMA’s CHMP at its May 2017 meeting.

Relmada Therapeutics announced that the FDA has granted Fast Track designation for d-Methadone (REL-1017 dextromethadone), the company’s novel N-methyl-D-aspartate (NMDA) receptor antagonist in development for the adjunctive treatment of major depressive disorder.

RedHill Biopharma together with IntelGenx announced that the Ministry of Health of Luxembourg has granted national marketing authorization for RIZAPORT(5 mg and 10 mg), a proprietary oral thin film formulation of rizatriptan benzoate for the treatment of acute migraines.

Bristol-Myers Squibb announced that it has entered into two separate agreements to license BMS-986168, an anti-eTau compound in development for Progressive Supranuclear Palsy (PSP), to Biogen, and BMS-986089, an anti-myostatin adnectin in development for Duchenne Muscular Dystrophy (DMD), to Roche. Under the agreement to license BMS-986168, Biogen will pay to Bristol-Myers Squibb an upfront payment of $300 million with potential milestone payments of up to $410 million. Biogen also will assume all remaining obligations to the former stockholders of iPierianrelated to Bristol-Myers Squibb’s acquisition of the company in 2014. Under the agreement to license BMS-986089, Roche will pay to Bristol-Myers Squibb an upfront payment of $170 million with potential milestone payments of up to $205 million. Bristol-Myers Squibb will receive tiered double-digit royalties if either asset is approved and commercialized. These agreements are subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act, and are expected to close in the second quarter of 2017.

Arbutus Biopharma announced that a justice of the Court of Appeal for British Columbia denied an attempt by Acuitas Therapeutics to appeal the injunction issued by the Supreme Court of British Columbia, which prevents Acuitas from any further licensing of Arbutus’ lipid nanoparticle (LNP) technology.

Celsion announced publication of the article, “Lyso-Thermosensitive Liposomal Doxorubicin for Treatment of Bladder Cancer,” in the International Journal of Hyperthermia. The article describes the results of porcine in vivo studies to evaluate ThermoDox in combination with loco-regional mild hyperthermia for targeted drug delivery to the bladder wall as a potential treatment for bladder cancer.  Swine bladder walls are similar in proportion and composition to human bladders.  Doxorubicin accumulation and distribution within the bladder wall with ThermoDox plus mild bladder hyperthermia was achieved at concentrations nearly ten times higher than with free intravenous doxorubicin combined with mild bladder hyperthermia. The study was conducted under a Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health (NIH) to evaluate whether ThermoDox combined with mild heating of the bladder can target drug delivery in the bladder.

BioMarin Pharmaceutical announced that it has entered into a settlement agreement with Par Pharmaceutical that resolves patent litigation in the U.S. related to BioMarin’s Kuvan (sapropterin dihydrochloride) 100mg oral tablets and powder for oral solution in 100mg packets.

Nuvox Pharma announced that the FDA has allowed an IND application to initiate a Phase II clinical trial for its oxygen therapeutic, NVX-108, in patients with the hypoxic solid brain tumor, glioblastoma multiforme (GBM). NVX-108 is an injectable drug that travels through the bloodstream arriving first at the lungs to pick up oxygen and finally to hypoxic tissue where it passively delivers the oxygen. It is designed to reduce tumor hypoxia in order to make tumors more sensitive to radiation therapy and chemotherapy.

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomicsannounced that The Regents of the University of California, the University of Vienna, and Dr. Emmanuelle Charpentier (collectively “UC”), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome engineering, have appealed to the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”) the decision by the Patent Trial and Appeal Board (“PTAB”) to terminate the interference between certain CRISPR/Cas9 patent claims owned by UC and patents and patent applications owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology.

resTORbio, a subsidiary of PureTech Health announced the appointment of Joan Mannick, M.D., as Chief Medical Officer.

Medicenna BioPharma, a wholly owned subsidiary of Medicenna Therapeutics, announced that it has treated the first patient in its Phase IIb clinical trial of MDNA55, the Company’s lead candidate. MDNA55 is being developed for the treatment of recurrent glioblastoma (rGB), the most common and uniformly fatal form of brain cancer.

Cellectis announced that it is exploring the possibility of an IPO of a minority interest in its plant sciences business, Calyxt. No decisions have been taken at this point on the structure or timing of any IPO, and no assurance can be given that an IPO will be pursued.

Tocagen announced the pricing of its initial public offering of 8,500,000 shares of common stock at a public offering price of $10.00 per share. The shares are expected to begin trading on the NASDAQ Global Select Market on April 13, 2017 under the ticker symbol "TOCA." In addition, Tocagen has granted the underwriters a 30-day option to purchase up to an additional 1,275,000 shares of common stock at the initial public offering price. Leerink Partners LLC, Evercore Group L.L.C. and Stifel, Nicolaus & Company, Incorporated are acting as joint bookrunning managers for the offering.

Sorrento Therapeutics announced that it intends to offer and sell, subject to market and other conditions, shares of its common stock in an underwritten public offering. Sorrento will grant the underwriter an option exercisable, in whole or in part, in the sole discretion of the underwriter, to purchase additional shares, for a period of up to 30 days. Cantor Fitzgerald & Co. is acting as the lead book-running manager for the offering. FBR & Co. is also acting as a joint book-running manager. 

Endo International announced that Endo Designated Activity Company, Endo Finance LLC and Endo Finco Inc., its wholly-owned subsidiaries, priced $300 million aggregate principal amount of 5.875% senior secured notes due 2024 at an issue price of $1000 per $1000 principal amount in connection with their previously announced private offering. The notes will be senior obligations of the Issuers and will be guaranteed by Endo and certain of Endo’s subsidiaries and will be secured by first priority liens on the same collateral that secures Endo’s obligations under its previously announced newly proposed senior secured credit facilities. Endo intends to use the net proceeds from the offering, together with approximately $3.4 billion of borrowings under its new term loan facility and cash on hand, to repay all outstanding loans and all other obligations under its existing credit facilities and to pay related fees and expenses.

ANALYST RECOMMENDATIONS

Ladenburg Thalmann analyst Matthew Kaplan initiated coverage of Reata with a “buy” rating and $55 price target, citing the development of novel small molecule drugs for the treatment of orphan diseases driven by mitochondrial dysfunction and inflammation.

RBC Capital analyst Randall Stanicky initiated coverage of Pacira Pharmaceuticals with a “buy” rating and $58 price target, citing the combination of a focused selling strategy on the back of the DPS deal and accelerating data reads will set EXPAREL sales up for an inflection that could come as early as 2H2017.

Oppenheimer analyst Leah Rush Cann initiated coverage of the following companies:Epizyme with an “outperform” rating and $26 price target; Verastem with an “outperform” rating and $6 price target.

BioShares Biotechnology Clinical Trials (BBC): $21.94, +$0.37, +19.2% YTD

BioShares Biotechnology Products (BBP): $36.84, +$0.40, +12.5% YTD

MARKET COMMENTARY

Following a long holiday weekend, U.S. stock index futures pointed to a lower open as geopolitical tensions dampened investor sentiment. Rising tensions over North Korea pushed gold higher, and kept the safe-haven Japanese currency in demand, nudging the dollar to a five-month low. Shares of Netflix and United Continental Holding will be watched as they are due to report quarterly results after the closing bell. NAHB housing market index, net capital inflows and foreign buying are on the economic calendar. European markets were closed for Easter holidays, while Asian shares closed mixed. Crude oil slid lower on signs that the United States is continuing to add output, largely counteracting strong economic growth in China and OPEC efforts to cut production.

MARKET HIGHLIGHTS

Arena Pharmaceuticals announced three data presentations on etrasimod (APD334) at ASPET 2017 at Experimental Biology (EB).  The conference is taking place April 22-26 at McCormick Place Convention Center in Chicago, IL.  The presentations encompass preclinical data highlighting the potent and selective immunomodulatory effects of etrasimod in inflammatory bowel disease (IBD), including ulcerative colitis (UC).

DURECT Corporation announced today that it will present clinical data on DUR-928 at the International Liver Congress 2017 (the 52nd annual meeting of the European Association for the Study of the Liver (EASL)), which will be held April 19-23 in Amsterdam.  The poster presentation will report safety, pharmacokinetics and biomarker data from both cohorts of a Phase 1b study utilizing DUR-928 in patients with nonalcoholic steatohepatitis (NASH).

Eli Lilly and Incyte announced that the FDA has issued a CRL for the NDA of the investigational medicine baricitinib, a once-daily oral medication for the treatment of moderate-to-severe rheumatoid arthritis (RA). The letter indicates that the FDA is unable to approve the application in its current form. Specifically, the FDA indicated that additional clinical data are needed to determine the most appropriate doses. The FDA also stated that additional data are necessary to further characterize safety concerns across treatment arms. The companies disagree with the agency’s conclusions. The timing of a resubmission will be based on further discussions with the FDA. Following the news, JP Morgan analyst Cory Kasimov decreased his price target of Incyte to $149 from $155; Piper Jaffray analyst Joshua Schimmer downgraded Incyte to “neutral” from “overweight” and decreased his price target to $124 from $140;Jefferies analyst Brian Abrahams decreased his price target to $148 from $165;SunTrust analyst Peter Lawson decreased his price target to $145 from $160; Barclaysanalyst Geoff Meacham decreased his price target to $180 from $185; BMO analyst Ian Somaiya decreased his price target to $144 from $170; Nomura analyst Christopher Marai decreased his price target to $144 from $148; Morgan Stanley analyst Andrew Berens decreased his price target to $142 from $150; William Blair analyst Katherine Xu decreased her fair value estimate to $169 from $170; Goldman Sachs analyst Salveen Richter decreased her price target to $136 from $149.

Cleveland BioLabs announced that the EMA has accepted the company’s pediatric investigation plan (PIP), paving the way for submission of a MAA for entolimod as a medical radiation countermeasure.

OncoMed Pharmaceuticals reported top-line results from the company’s randomized 145-patient Phase II PINNACLE clinical trial of tarextumab (anti-Notch2/3, OMP-59R5) in combination with etoposide plus either cisplatin or carboplatin chemotherapy (“chemotherapy”) in previously untreated patients with extensive-stage small cell lung cancer.  Results for the combination of tarextumab plus chemotherapy were undifferentiated from those of chemotherapy plus placebo, and therefore the trial did not meet its primary endpoint of progression-free survival or secondary endpoints of overall survival and biomarkers reflective of Notch pathway gene activation.

Genentech, a member of the Roche Group, announced interim results from the Phase III HAVEN 2 study evaluating emicizumab prophylaxis in children less than 12 years of age with hemophilia A and inhibitors to factor VIII. At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time. These findings are consistent with results from the Phase III HAVEN 1 study in adults and adolescents (12 years of age or older) with hemophilia A and inhibitors to factor VIII, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time compared to no prophylaxis, as well as compared to prior prophylaxis with bypassing agents. The most common adverse events with emicizumab in the HAVEN 2 study were injection site reactions and nasopharyngitis (common cold symptoms).

Abbott and Alere announced that the companies have agreed to amend the existing terms of their agreement for Abbott’s acquisition of Alere. Under the amended terms, Abbott will pay $51 per common share to acquire Alere, for a new expected equity value of approximately $5.3 billion, reduced from the originally expected equity value of approximately $5.8 billion.

CytoDyn announced that its application for Orphan Drug Designation (ODD) was not granted by the Office of Orphan Products Development of the FDA because PRO 140 appears to have the potential to treat more than just the subset of multi-drug resistant HIV patients for which the designation was requested.

Bristol-Myers Squibb and Nordic Bioscience announced a collaboration agreement to develop biomarker technology to potentially aid in the diagnosis and monitoring of fibrotic diseases including Non-alcoholic steatohepatitis (NASH).

Novocure announced that data from a post hoc analysis of the EF-14 pivotal Phase III clinical trial of Optune in combination with temozolomide for the treatment of newly diagnosed glioblastoma (GBM) have been published in CNS Oncology. The objective of the pre-specified post hoc analysis was to evaluate the efficacy and safety of Optune when added to physician’s best choice second-line treatment after first disease recurrence among patients enrolled in the EF-14 trial. The analysis shows that the median overall survival of patients treated with Optune in combination with physician’s best choice second line chemotherapy increased by 28 percent compared to patients treated with physician’s best choice second line chemotherapy alone from 9.2 months to 11.8 months (HR= 0.70, p= 0.049). Bevacizumab, alone or in combination with chemotherapy, was the most frequently used second-line treatment. The analysis also shows that the median overall survival of patients treated with Optune in combination with bevacizumab increased by 31 percent compared to patients treated with bevacizumab alone from 9.0 months to 11.8 months (HR=0.61, p= 0.043).

Nabriva Therapeutics announced that its supervisory board and management board approved the relocation of the holding company of Nabriva AG and its subsidiaries (“Nabriva Group”) from Austria to Ireland. The Redomiciliation Transaction will be effected by the exchange of American depositary shares (“Nabriva AG ADSs”) and shares (the “Exchange Offer”) of Nabriva AG for shares of Nabriva Therapeutics Plc (“Nabriva Ireland”), a newly-formed Irish public limited company, with Nabriva Ireland becoming the publicly-traded parent entity of Nabriva AG. Once the Exchange Offer is completed, the current Austrian publicly-traded parent company, will become a subsidiary of the newly-formed Irish company, and it is expected that Nabriva Ireland will then become the publicly-traded parent company of the Nabriva Group and its subsidiaries with its tax residency in Ireland.

Myriad Genetics announced that new data comparing BRCA1 and BRCA2 variant classifications between Myriad Genetics and a commonly used public genetic database was published in the journal The Oncologist.  A key finding was that the public database provided discrepant variant classifications more than 26 percent of the time, which can introduce uncertainty and diminish patient care.

Synergy Pharmaceuticals announced that Gary G. Gemignani has been appointed as Executive Vice President and CFO, effective April 17, 2017. Mr. Gemignani replaces Senior Vice President, Finance, Bernard Denoyer, who will assist in the transition process until his retirement on July 1, 2017.

CRISPR Therapeutics and Casebia Therapeutics, a joint-venture established by CRISPR Therapeutics and Bayer AG for developing CRISPR-based therapeutics in select disease areas, announced they have signed a collaboration agreement with StrideBio, a US-based company developing novel AAV vectors for in vivo gene delivery applications. Under the terms of the agreement, StrideBio will use its proprietary platform to develop AAV vectors with improved properties such as tissue specificity and reduced susceptibility to immune responses.  CRISPR Therapeutics and Casebia will have an option to exclusively license AAV vectors with desired properties for use in their in vivo gene-editing programs.  StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

AMAG Pharmaceuticals announced that it has submitted a sNDA to the FDA for the Makena subcutaneous auto-injector, a drug-device combination product. The current Makena intramuscular (IM) injection is the only FDA-approved treatment indicated to reduce the risk of preterm birth in women who are pregnant with one baby and who spontaneously delivered one preterm baby in the past.

Puma Biotechnology announced that the FDA has scheduled the NDA for neratinib for discussion by the Oncologic Drugs Advisory Committee (ODAC) on May 24, 2017. Neratinib is an investigational therapy for the extended adjuvant treatment of early stage HER2-positive breast cancer that has previously been treated with a trastuzumab containing regimen.

AmpliPhi Biosciences announced that the FDA provided positive feedback on the Company’s previously submitted detailed development proposal to commence a Phase II trial with its proprietary bacteriophage cocktail AB-SA01 for the treatment of antibiotic-resistant Staphylococcus aureus (S. aureus) infections in patients with chronic rhinosinusitis (CRS). The FDA’s feedback followed a Type B telephonic meeting held with AmpliPhi on February 21, 2017. In the official minutes from the meeting, the FDA acknowledged that phage therapy is an exciting approach to treatment of multidrug-resistant organisms and expressed a commitment to addressing the unique regulatory challenges that might arise during product development. AmpliPhi also announced that following a review of the status of its internal programs, resources and capabilities, the Company has begun to explore a wide range of strategic alternatives to maximize value for its shareholders. The Company has retained H.C. Wainwright & Co., LLC to advise the Company and its board of directors in this effort.

Oncobiologics announced that it has amended its Note and Warrant Purchase Agreement dated December 22, 2016 (the “NWPA”) and issued additional senior secured promissory notes that bear interest at a rate of 5.0% per year and mature on the one-year anniversary of the NWPA (the “Notes”) and 5-year warrants to purchase shares of its common stock at an exercise price of $3.00 per share (the “Warrants”) in connection therewith. Under the amended NWPA, on April 13, 2017 Oncobiologics issued an additional $3.5 million aggregate principal amount of Notes and an additional 1,165,500 Warrants.  Oncobiologics may issue up to $1.5 million of additional Notes and 499,500 Warrants in additional closings over 180 days from the original date of NWPA without approval of holders of the Notes.

AmpliPhi Biosciences announced that its board of directors has approved a one-for-ten reverse split of its outstanding common stock and a corresponding, proportional reduction in the number of its authorized shares of common stock, each to become effective pursuant to the filing of articles of amendment to AmpliPhi’s articles of incorporation.

ANALYST RECOMMENDATIONS

Ladenburg Thalmann analyst Christopher James initiated coverage of Bellerophon Therapeutics with a “buy” rating and $4.50 price target, citing Bellerophon is developing and optimizing INOpulse, a second generation, drug-device combination product for the outpatient management of chronic pulmonary diseases.

Leerink analyst Geoffrey Porges increased his price target of Vertex to $128 from $115, citing the recent positive phase III tezacaftor results in CF patients with F508del and residual function mutations.

HC Wainwright analyst Ed Arce decreased his price target of Cidara Therapeutics to $14 from $18, citing higher guided operating expenses in 2017.

BMO analyst Do Kim initiated coverage of Alnylam with an “outperform” rating and $73 price target, citing Alnylam has three late-stage drugs that are significantly de-risked and an additional two with potentially accelerated path to approval.

BMO analyst Do Kim initiated coverage of Halozyme with a “market perform” rating and $14 price target, citing valuation support from Halozyme’s ENHANZE platform, with visibility into $7bn of partnered drug sales.

BMO analyst Alex Arfaei downgraded Eli Lilly to “underperform” from “market perform” and decreased his price target to $71 from $73, citing the major setback with Bari in the U.S., coupled with ongoing headwinds for the mature franchises (e.g. Humalog), and cautious views on Abemaciclib, make the risk/reward in LLY shares unfavorable.

Cantor Fitzgerald analyst William Tanner decreased his price target of TherapeuticsMD to $33 from $34, citing model changes and anticipation the FDA will not approve TX-004 on the May 7 PDUFA date.

BioShares Biotechnology Clinical Trials (BBC): $21.96, +$0.02, +19.3% YTD

BioShares Biotechnology Products (BBP): $36.86, +$0.02, +12.5% YTD

MARKET COMMENTARY

Wall Street looked set to follow European markets lower, after Britain’s Prime minister called for an early election on June 8 and as investors remained cautious ahead of Sunday’s French election. Asian stock markets closed mostly lower. On the economic front, investors will look forward to data on building permits, housing starts and manufacturing output. IBM, Yahoo and Intuitive Surgical are some of the major companies scheduled to report results after the closing bell. Oil prices slipped on news that U.S. shale oil output in May is expected to post the biggest monthly increase in more than two years, undermining efforts to cut oversupply. Gold steadied after touching five month highs in the previous session, bolstered by the dollar softening versus the yen and tensions in the Korean peninsula.

MARKET HIGHLIGHTS

Eiger BioPharmaceuticals announced the appointment of Lisa Porter, M.D. to lead the development of exendin 9-39 for the treatment of post-bariatric hypoglycemia (PBH).

Mateon Therapeutics announced interim data from its first scheduled analysis of the ongoing Phase 2/3 study (FOCUS) in patients with platinum resistant ovarian cancer (prOC). The interim analysis was conducted after the first 20 patients enrolled into the trial had been treated for at least two months or had discontinued from the trial. The analysis was conducted primarily to evaluate the safety of the triplet drug combination as well as to evaluate early signs of efficacy.  No significant safety issues were identified in the interim analysis, and the overall safety profile of CA4P was similar to or better than results seen in previous randomized trials of CA4P. Most adverse events that occurred more frequently in the CA4P-treated arm were mild to moderate in severity.

Valneva SE announced that it has entered into a commercial agreement with Danish biotech company Bavarian Nordic (OMX:  BAVA, OTC:BVNRY), for the production of poxvirus-based vaccines using Valneva`s EB66® cell-line technology.  The agreement grants Bavarian Nordic the rights to develop and commercialize multiple poxvirus-based vaccines on the EB66® cell-line.  Bavarian Nordic`s vaccine candidates are currently produced on chicken embryonic fibroblast (CEF) and the company will explore the potential of switching to Valneva`s EB66® technology.  Valneva will support Bavarian Nordic in establishing and optimizing the manufacturing process on EB66® for future large scale industrialization under a dedicated service agreement.

Arena Pharmaceuticals announced the pricing of an underwritten public offering of 60,000,000 shares of its common stock, offered at a price to the public of $1.15 per share.  The gross proceeds from the offering are expected to be $69.0 million, before deducting the underwriting discounts and commissions and offering expenses payable by Arena.  Arena has granted the underwriters a 30‑day option to purchase up to 9,000,000 additional shares of common stock. All of the shares are being offered by Arena.  The offering is expected to close on April 21, 2017, subject to customary closing conditions.  Arena anticipates using the net proceeds from the offering for clinical and preclinical development of drug candidates, for general corporate purposes, including working capital and costs associated with manufacturing services, and for capital expenditures.

Bioblast Pharma announced that in response to a written notification from the Listing Qualifications Department of The NASDAQ Stock Market LLC that the Company does not satisfy the Nasdaq Global Market continued listing requirement set forth in Nasdaq Stock Market Rule 5450(b)(1)(A) which requires that the company maintain a minimum of $10,000,000 in stockholders’ equity, Bioblast will transfer the listing of its ordinary shares to the Nasdaq Capital Market, which requires a minimum of $2,500,000 in stockholders’ equity for continued listing.

Aurinia Pharmaceuticals announced that it has signed a Definitive Agreement granting Merck Animal Health, known as MSD Animal Health outside of the United States and Canada, worldwide rights to develop and commercialize Aurinia’s patented nanomicellar voclosporin ophthalmic solution (VOS) for the treatment of dry eye syndrome in dogs.  Under the terms of this agreement Aurinia will receive an upfront payment and is eligible to receive further payments based on certain development and sales milestones.  Furthermore, Aurinia will receive royalties based on global product sales. Merck Animal Health will be responsible for the remaining clinical development and commercialization of VOS for use in the animal health field, while Aurinia retains all human health rights. The companies will share in the final work product and any technical knowledge that may be generated during the collaboration.

GeNeuro announced today the initiation of a Phase 2a clinical study in Australia with GNbAC1 in patients with Type 1 diabetes (T1D). GNbAC1 is a monoclonal antibody designed to neutralise MSRV-Env, a pathogenic protein that has been detected in the pancreas of T1D patients. GeNeuro is already evaluating GNbAC1 in Phase 2 clinical studies in patients with multiple sclerosis (MS), a disease in which evidence shows MSRV-Env to be a potential causal factor.

Reuters reported that Johnson & Johnson reported quarterly revenue that missed estimates due to slowing pharmaceutical sales, but the company, which is in the process of closing its $30 billion acquisition of Actelion, raised its 2017 forecast. The company raised its 2017 forecast to reflect the Actelion acquisition, which is expected to close in the second quarter. J&J raised its sales expectations to $75.40 billion-$76.10 billion and adjusted profit to $7.00-$7.15 per share. The diversified healthcare company said sales rose to $17.77 billion in the first quarter from $17.48 billion a year earlier, but came in below analysts’ average estimate of $18.04 billion, according to Thomson Reuters I/B/E/S. Net earnings in the first quarter were $4.42 billion, or $1.61 per share, compared with $4.46 billion, or $1.59 per share, in the year-earlier period. Excluding items, J&J earned $1.83 per share, beating Street expectations of $1.77, helped by lower operating expenses and taxes.

XOMA announced the presentation of positive data from pre-clinical studies investigating the activity of its anti-PTH1R antagonist monoclonal antibody (mAb). The antibody is a potential first-in-class therapeutic agent for the treatment of hyperparathyroidism (HPT) and humoral hypercalcemia of malignancy (HHM). These presentations were made at the American Association for Cancer Research (AACR) and the Endocrine Society (ENDO) annual meetings.

Rigel Pharmaceuticals announced that it has submitted a NDA to the FDA for fostamatinib in patients with chronic and persistent immune thrombocytopenia (ITP).

Biocept announced that it has been awarded Japanese Patent No. 6081434 entitled, DEVICES AND METHODS OF CELL CAPTURE AND ANALYSIS. The issued patent covers the use of antibodies for the capture of any target of interest from any sample type on a device surface. This includes CTCs, as well as other targets of interest such as sub-cellular vesicles and exosomes that are shed by solid tumors into the blood stream. This patent is broader than previously issued patents owned by the Company, and is the nineteenth patent issued to Biocept related to its core liquid biopsy technology.

Protalix BioTherapeutics announced new, promising results from a preclinical trial conducted in collaboration with Prof. Raphael Schiffmann, Director, Institute of Metabolic Disease at the Baylor Research Institute, Dallas, Texas.  It was demonstrated that treatment of Fabry mice with pegunigalsidase alfa (PRX-102) slows the progression of small fiber neuropathy when compared to treatment of Fabry mice with currently approved enzyme replacement therapies, and when compared to untreated Fabry mice.

RedHill Biopharma announced that it has received notices of allowance from the USPTO for two new patents covering BEKINDA (RHB-102)(1). Once granted, the patents are expected to be valid until at least 2034.

Lion Biotechnologies highlighted that a publication in the journal Science provided new translational data from a clinical trial of TIL therapy for the treatment of advanced metastatic cervical cancer conducted at the Surgery Branch of the National Cancer Institute (NCI). This trial has been supported in part by Lion under a Cooperative Research and Development Agreement (CRADA) with Dr. Steven Rosenberg, Chief of the Surgery Branch, National Cancer Institute (NCI), National Institutes of Health.

Allergan announced that it has entered into a clinical trial agreement with Novartis to conduct a Phase IIb study, using Allergan’s cenicriviroc (CVC) and Novartis’ lead FXR agonist for the treatment of non-alcoholic steatohepatitis (NASH). The Phase IIb study will assess the safety, efficacy and tolerability of this multi-therapy treatment approach for NASH.  The financial details of this transaction are not disclosed.

Novartis announced that the FDA has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies. This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

HitGen announced that the company has entered into a multi-year research collaboration and license agreement with Pfizer to build and screen novel DNA-encoded libraries (DELs) in order to potentially discover unique small molecule leads to be used in drug development. Through the collaboration, HitGen and Pfizer scientists will apply HitGen’s advanced technology platform and research capabilities in the design, synthesis, and screening of multiple proprietary DELs for Pfizer’s drug discovery efforts. In addition, HitGen will screen their own DELs, consisting of billions of compounds, against a selected number of Pfizer’s therapeutic targets. Novel lead compounds from the HitGen DELs will be licensed exclusively to Pfizer for further research and development. Pfizer will fund the research at HitGen.

Egalet announced that the submission of a prior approval supplement (PAS) for OXAYDO (oxycodone HCl, USP) tablets C-II, seeking approval of 10 mg and 15 mg dosage strengths, has been  accepted for filing by the FDA.  The submission is based on a pharmacokinetic study demonstrating bioequivalence (BE) of OXAYDO to its reference drug, Roxicodone (oxycodone hydrochloride tablets USP) at the 15 mg dosage strength.

Enzyvant announced that the FDA has granted RVT-802, Enzyvant’s investigational tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), Breakthrough Therapy designation as well as Regenerative Medicine Advanced Therapy (RMAT) designation. Both designations confer special access to the Office of Tissues and Advanced Therapies for development guidance as well as expedited review pathways.

Sucampo Pharmaceuticals announced the publication of a new study in an independent scientific journal that confirms the unique composition of VTS-270, a 2-Hydroxypropyl-beta-cyclodextrin (HPβCD) product under investigation as a novel treatment for Niemann-Pick Disease Type C1 (NPC-1). In this study, researchers demonstrated that VTS-270 has a low degree of consistently specific hydroxypropylation, or degree of substitution (DS), as well as a low level of doubly charged hetero-dimers as seen through an ion mobility false color representation, or heat map. The analysis was based on ion distribution and abundance profiles using mass spectrometry methodology as a means for assessing key molecular activity.

Ionis Pharmaceuticals announced a collaboration and license agreement with Suzhou Ribo Life Science Co., Ltd. (Ribo) to develop and commercialize RNA-targeted therapeutics in China. Ionis granted Ribo a license for the right to commercialize in China two Ionis Generation 2+ antisense drugs in metabolic disease and cancer and an option to license a third pre-specified Generation 2+ antisense drug. In addition, Ribo will be responsible for conducting a multi-year research and drug discovery program to identify drugs that utilize Ionis’ ssRNAi technology. Ionis will receive an undisclosed up-front payment and equity in Ribo. Ionis retains the rights to develop and commercialize ssRNAi technology and all drugs under the collaboration outside of China.

Prometic Life Sciences announced that it has received concurrence from the FDA on the design of the first of its PBI-4050’s planned Phase II/III clinical trials for IPF based on the efficacy data generated in the recently completed 40 patient Phase II open-label study.

RXi Pharmaceuticals announced that Gerrit Dispersyn, Dr. Med Sc. has been appointed as RXi’s Chief Development Officer effective April 24, 2017.   Dr. Dispersyn will bring a wealth of experience to RXi in both clinical and product development.

Antibe Therapeutics provided an update on its clinical development program for ATB-346, its lead drug that targets the global need for a safer drug for chronic pain and inflammation.

Regen BioPharma, in conjunction with its medicinal chemistry partner, ChemDiv, has identified three new series of compounds that activate NR2F6. These compounds are small molecules and were identified using Regen’s patented screening methodology and unique chemical libraries.

BioSpecifics Technologies announced that it has initiated an open-label, dose escalation Phase I clinical trial of XIAFLEX for the treatment of uterine fibroids.

Evoke Pharma announced that the Company has reached an agreement to work in partnership with Spaulding Clinical Research for its planned comparative exposure pharmacokinetic (PK) trial for its lead product candidate, Gimoti, a patented nasal delivery formulation of metoclopramide for the relief of symptoms associated with acute and recurrent diabetic gastroparesis in adult women. The pivotal PK trial conducted in healthy volunteers is designed to establish bioequivalence with the listed drug, Reglan Tablets, and may serve as a basis for the 505(b)(2) NDA planned for submission to the FDA.

Imprimis Pharmaceuticals and Cameron Ehlen Group, d/b/a Precision Lens ("Precision Lens"), announced the signing of a three-year exclusive sales representation agreement.  Under the agreement, Precision Lens will deploy a dedicated sales team to introduce Imprimis’ ophthalmic portfolio into select geographies in the U.S., primarily focused in 13 states in the U.S. Midwest.

Cellectar Biosciences announced the Japanese Patent Office has granted a method of use patent for two of the company’s phospholipid drug conjugates (PDCs), CLR 131, the company’s lead compound, and CLR 125, each in combination with radiation and/or other therapies to treat cancer stem cells. CLR 125 is a radiotherapeutic isotope conjugated to the company’s proprietary PDC delivery platform, which may be uniquely suited to treat select cancer and micro-metastatic disease.

Galapagos announced the pricing of its U.S. public offering totaling $338 million gross proceeds of 3,750,000 new ordinary shares in the form of ADSs at $90 per ADS, before underwriting discounts. In addition, Galapagos has granted the underwriter an option to purchase up to an additional 562,500 ADSs, representing 15% of the ADSs placed in the offering. This option can be exercised during the 30 day period commencing 17 April 2017. The closing of the offering is expected to occur on 21 April 2017, subject to customary closing conditions. Each of the ADSs offered in the offering represents the right to receive one ordinary share. Morgan Stanley is acting as sole book-running manager for the proposed offering.

Agios Pharmaceuticals announced that it is offering to sell, subject to market and other conditions, up to 4.5 million shares of its common stock in an underwritten public offering. Agios also intends to grant the underwriters a 30-day option to purchase from it up to an additional 15% of the shares of common stock offered in the public offering. The proceeds of the offering are expected to be used to fund the company’s research, clinical development and pre-commercial activities. J.P. Morgan Securities and Goldman Sachs & Co. are acting as joint book-running managers for the offering.Cowen and Company is serving as lead manager.

Audentes Therapeutics announced that it intends to offer and sell 4,750,000 shares of its common stock in an underwritten public offering. In addition, Audentes intends to grant the underwriters a 30-day option to purchase up to an additional 712,500 shares of common stock. BofA Merrill Lynch, Cowen and Company and Piper Jaffray are acting as joint book-running managers for the offering. Wedbush PacGrow is acting as a co-manager.

Axovant Sciences announced the closing of its underwritten public offering of 7,753,505 of its common shares at a price to the public of $18.54 per common share, including 1,011,326 common shares sold pursuant to the underwriters’ exercise in full of their option to purchase additional common shares. Gross proceeds to Axovant from the offering were approximately $143.7 million, before deducting underwriting discounts and commissions and estimated offering expenses. J.P. Morgan, Morgan Stanley and Jefferies acted as joint book-running managers for the offering. JMP Securities, Baird and H.C. Wainwright & Co. acted as co-managers.

ANALYST RECOMMENDATIONS

Following the news of the FDA’s Complete Response Letter to the baricitinib NDA, UBS analyst Carter Gould decreased his price target of Incyte to $136 from $140.

Canaccord analyst Mark Massaro increased his price target of Quidel to $24 from $22, citing Quidel likely just wrapped up its strongest February and March in the company’s history.

Deutsche Bank analyst Gregg Gilbert revised his price target for the following companies, citing lower 1Q17EPS estimates: Mylan to $51 from $52; Perrigo to $86 from $87; Valeant to $18 from $19.

SunTrust analyst Yatin Suneja initiated coverage of Global Blood Therapeutics with a “buy” rating and $56 price target, citing shares should outperform on clinical and regulatory newsflow.

Credit Suisse analyst Alethia Young assumed coverage of the following companies from Kennen MacKay: Acceleron with an “outperform” rating and $46 price target; Agios with an “outperform” rating and $61 price target; AnaptysBio with an “outperform” rating and $34 price target; BioMarin with an “outperform” rating and $112 price target; Clovis with an “outperform” rating and $74 price target; Incyte with an “outperform” rating and $174 price target; ObsEva with an “outperform” rating and $27 price target; Puma Biotechnology with an “outperform” rating and $58 price target; Ra Pharmaceuticals with an “outperform” rating and $19 price target; OvaScience with an “underperform” rating and $1 price target. 

BioShares Biotechnology Clinical Trials (BBC): $21.70, -$0.26, +17.9% YTD

BioShares Biotechnology Products (BBP): $36.51, -$0.35, +11.5% YTD

MARKET COMMENTARY

Wall Street was set for a modestly higher start, recovering from previous session’s decline, as focus shifted towards fresh batch of corporate earnings. Markets will watch for quarterly results of major companies scheduled to report results later in the day, including Qualcomm, American Express and eBay. Some positive first-quarter results coupled with a bounce in banking shares helped European bourses to trade higher, while Asian stocks closed in lower. The dollar managed to recoup its broader losses and was trading marginally higher against a basket of currencies, while gold futures slipped. Oil prices rose after OPEC said it was committed to eroding a global supply overhang that has dogged markets since 2014.

MARKET HIGHLIGHTS

Advaxis announced that Anthony A. Lombardo, an industry veteran with nearly 30 years’ experience in the field of life sciences, has joined the company as Chief Business Officer. Lombardo is a senior leader with extensive experience in helping global companies achieve significant growth.  Previously, Lombardo was President and CEO of E-Z-EM Inc., a company focusing on oral imaging contrast agents.

TherapeuticsMD announced that, based on continued correspondence with the U.S. Food and Drug Administration (FDA), the Company expects that the FDA will finalize an action on the New Drug Application (NDA) for TX-004HR, an investigational bio-identical 17?-estradiol vaginal softgel capsule for the treatment of moderate-to-severe vaginal pain during sexual intercourse (dyspareunia), a symptom of vulvar vaginal atrophy (VVA) in postmenopausal women, including informing the Company of the deficiencies in the NDA identified by the FDA, on or before the originally scheduled Prescription Drug User Fee Act (PDUFA) target action date of May 7, 2017.  At this time, the Company is not aware of the nature of the deficiencies in the NDA identified by the FDA. The Company continues to communicate with the FDA to understand the nature of the deficiencies and intends to resolve them as quickly as possible.

BrainStorm Cell Therapeutics announced that a preclinical study evaluating the use of NurOwn® mesenchymal stem cells in a mouse model of autism was published in the April issue of the journal Behavioural Brain Research [Perets N. et al. Epub ahead of print, PMID: 28392323]. The publication, entitled "Long Term Beneficial Effect of Neurotrophic Factors-Secreting Mesenchymal Stem Cells Transplantation in the BTBR Mouse Model of Autism, was authored by Prof. Daniel Offen, Head of Tel Aviv University’s Translational Neuroscience Laboratory, Sackler School of Medicine, and Brainstorm’s Chief Scientific Advisor.  The publication reported that transplantation of mesenchymal stem cells (MSC) induced to secrete neurotrophic factors (MSC-NTF cells, NurOwn®) in the BTBR mouse model of autism demonstrated significant long-term improvements in autistic behavior in the BTBR mice compared to MSC treated and to untreated BTBR mice.

Argos Therapeutics reported interim results from its randomized, active controlled, open-label, multi-center Phase III ADAPT trial of Rocapuldencel-T in combination with sunitinib/standard-of-care for the treatment of newly diagnosed metastatic renal cell carcinoma (mRCC). The Company also provided perspective on its decision to continue the trial.

Soligenix announced that results from its ricin toxin vaccine (RiVax) development program will be presented at the 20(th) Annual Conference on Vaccine Research, being held April 24-26 in Bethesda, Md. RiVax is the Company’s proprietary vaccine candidate for the prevention of exposure to ricin toxin that utilizes a unique antigen that is completely devoid of the toxic activity of ricin. When formulated with ThermoVax, Soligenix’s proprietary vaccine heat stabilization technology, RiVax has demonstrated significantly enhanced thermostability and 100% protection in preclinical ricin aerosol challenge models. In collaboration with the New York State Department of Health and the laboratory of Nicholas Mantis, Ph.D., Soligenix has been investigating immune correlates of protection in sera of animals vaccinated with RiVax. The findings demonstrate that: 1) the ThermoVax thermostabilization process significantly enhances the stability of the RiVax antigen; 2) degradation in the antigen can be measured with specific monoclonal antibodies; and 3) these same monoclonal antibodies can be used to probe the immune profile of vaccinated mice and primates and predict their survival to subsequent ricin exposure challenge. These findings are expected to facilitate the potential approval of the RiVax product under the FDA "Animal Rule" and represent a significant step forward in the understanding of ricin toxin immunology.

CytRx announced the FDA has reached an agreement with CytRx on preparations for a NDA submission for aldoxorubicin in soft tissue sarcomas (STS). STS remains a high unmet medical need.

Flex Pharma announced that human efficacy data from its study in nocturnal leg cramps (NLC) will be presented at the upcoming American Academy of Neurology (AAN) 69(th) Annual Meeting in Boston, MA. When a neurologist evaluated, in a blinded manner, subjects likely to have NLC based upon a questionnaire administered after the study was completed, the data from first treatment exposure of these 26 subjects showed a statistically significant effect in the reduction in cramp frequency when compared to placebo (p=0.03).

Ultragenyx Pharmaceutical, Kyowa Hakko Kirin Co. and Kyowa Kirin International, a wholly owned subsidiary of Kyowa Hakko Kirin, announced positive 24-week data from the randomized, double-blind, placebo-controlled Phase III study of burosumab (KRN23) in adults with X-linked hypophosphatemia (XLH). Patients treated with burosumab demonstrated a statistically significant improvement in serum phosphorus levels, with 94% of patients achieving normal levels compared to 8% on placebo (p <0.0001). Patients treated with burosumab also achieved a statistically significant improvement in stiffness and strong trends in improvements in physical function and pain. Adverse events were consistent with what has been previously observed in open label studies in adults and children. Ultragenyx is conducting the study under a collaboration and license agreement with Kyowa Hakko Kirin.  Burosumab is being developed by Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin International. Following the news, Wedbush analyst David Nierengarten downgraded Ultragenyx to “neutral” from “outperform” and decreased his price target to $75 from $80; HC Wainwright analyst Carol Ann Werther downgraded the stock to “neutral” from “buy” and decreased her price target to $72 from $88; SunTrust analyst Ed Nash increased his price target to $105 from $100; Leerink analyst Joseph Schwartz increased his price target to $93 from $90.

Nivalis Therapeutics and Alpine Immune Sciences jointly announced they have entered into a definitive merger agreement under which Alpine will merge with a wholly-owned subsidiary of Nivalis in an all-stock transaction. The merger will result in a combined company with a novel protein-based discovery platform focused on inflammation and immuno-oncology.

Enanta Pharmaceuticals announced new data from its lead FXR agonist candidate EDP-305 for NASH and PBC. This new data is being presented during The International Liver Congress (ILC) 2017, April 19-23, in Amsterdam. Data from three poster presentations being presented at the Congress will demonstrate that EDP-305 is a potent Farnesoid X Receptor (FXR) agonist that has been shown to reduce expression of fibrogenic genes, reduce fibrosis progression and improve non-alcoholic fatty liver disease (NAFLD) activity scores (NAS) in a variety of preclinical models.

Vical announced that it has completed the target enrollment of 225 subjects in a Phase II trial of its therapeutic bivalent vaccine for herpes simplex virus type 2 (HSV-2), the leading cause of recurrent genital herpes.

TherapeuticsMD announced that, based on continued correspondence with the FDA, the Company expects that the FDA will finalize an action on the NDA for TX-004HR, an investigational bio-identical 17?-estradiol vaginal softgel capsule for the treatment of moderate-to-severe vaginal pain during sexual intercourse (dyspareunia), a symptom of vulvar vaginal atrophy (VVA) in postmenopausal women, including informing the Company of the deficiencies in the NDA identified by the FDA, on or before the originally scheduled PDUFA target action date of May 7, 2017.

Kura Oncology announced issuance of a U.S. patent for KO-947, its drug candidate targeting extracellular-signal-regulated kinases (ERK). The new patent, U.S. 9,624,228, entitled “Inhibitors of ERK and Methods of Use,” covers KO-947 and structurally-related compounds as well as methods of using the compounds for the treatment of diseases including cancer.

Lion Biotechnologies and The University of Texas MD Anderson Cancer Centerannounced a multi-year strategic alliance agreement involving multi-arm clinical trials to evaluate the safety and efficacy of TIL therapy in ovarian cancer, various sarcomas, and pancreatic cancer. In addition, preclinical research will explore the expansion of TIL in other rare tumor types.

Synthetic Biologics announced positive data for SYN-005, the Company’s orphan drug program for the prevention and treatment of pertussis (whooping cough). SYN-005 combines two highly synergistic humanized monoclonal antibodies (hu1B7 and hu11E6) to target and potentially inactivate the life-threatening pertussis toxin that causes disease symptoms.

Argentum Pharmaceuticals announced that it has reached a settlement and license agreement with Allergan related to Argentum’s generic version of RESTASIS (0.05% cyclosporine ophthalmic emulsion). The agreement generally provides that Argentum may commence marketing its generic equivalent product sometime prior to the expiration of the patents covering RESTASIS. The specific date on which Argentum may launch its generic product and other details concerning the settlement have not been disclosed. RESTASIS is a registered trademark of Allergan.

Prima BioMed announced that approval has been granted for the third cohort of its Phase I clinical trial for IMP321 in combination with KEYTRUDA being conducted in Australia. The third cohort will recruit six patients with unresectable or metastatic melanoma. Interim data results from the first patient cohort released in December 2016 indicate IMP321 at the 1mg dose level is safe and well tolerated. Out of the six patients in the first cohort (all with suboptimal response to KEYTRUDA monotherapy) two patients had a partial or complete radiological tumour response according to immune related response criteria (irRC).

Boehringer Ingelheim Pharmaceuticals announced that the first patient has been enrolled in the PF-ILD (progressive fibrosing interstitial lung disease) trial. This study investigates the efficacy and safety of nintedanib in a range of progressive fibrosing lung conditions other than idiopathic pulmonary fibrosis (IPF).

Pluristem Therapeutics announced that it has been issued its 100(th) patent, an important milestone attesting to the significant accomplishments of its scientists and engineers, and its enduring commitment to innovative research and development.

ANI Pharmaceuticals announced launch of Indapamide Tablets, 1.25mg and 2.5mg, which is used in the treatment of hypertension and swelling associated with congestive heart failure.

Advaxis announced that Anthony A. Lombardo, an industry veteran with nearly 30 years’ experience in the field of life sciences, has joined the company as Chief Business Officer.

Audentes Therapeutics announced the pricing of its underwritten public offering of 5,200,000 shares of its common stock at a price to the public of $14.50 per share before underwriting discounts and commission. The gross proceeds to Audentes from the offering, before deducting the underwriting discounts and commissions and other offering expenses payable by Audentes, are expected to be approximately $75.4 million. In addition, Audentes has granted the underwriters a 30-day option to purchase up to an additional 780,000 shares of common stock. BofA Merrill Lynch, Cowen and Company and Piper Jaffray are acting as joint book-running managers for the offering.Wedbush PacGrow is acting as a co-manager.

Agios Pharmaceuticals announced that it has priced an underwritten a public offering of 5,050,505 shares of common stock at a price to the public of $49.50 per share, before underwriting discounts, which would result in aggregate gross proceeds of approximately $250 million. Agios has also granted the underwriters a 30-day option to purchase from it up to an additional 15% of the shares of common stock offered in the public offering on the same terms and conditions. J.P. Morgan Securities and Goldman Sachs & Co. are acting as joint book-running managers for the offering. Cowen and Company is serving as lead manager.

Edge Therapeutics announced that Edge has entered into a subscription agreement for the sale of shares of its common stock in a registered direct offering with Satter Medical Technology Partners and certain of its affiliated parties. Pursuant to the agreement, Edge has agreed to sell to the investors 1,800,000 shares at a purchase price of $10.00 per share with gross proceeds of $18 million.

ANALYST RECOMMENDATIONS

LifeSci Capital analyst David Sherman initiated research coverage of Minerva Neurosciences, citing the unmet need in the treatment of central nervous system disorders.

Oddo BHF analyst Pierre Corby upgraded Transgene to a Buy from Reduce, citing the company’s restructuring is now complete and the company is focused on therapeutic vaccines and oncolytic viruses with checkpoint inhibitors.

Credit Suisse analyst Alethia Young decreased her price target of Incyte to $167 from $174, citing baricitinib’s U.S. launch moves to 2019 and a lower probability of success on U.S. revenues to 80%.

Goldman Sachs analyst Robert Jones made price target changes to the following companies: Icon to $90 from $93; Quintiles to $84 from $83; Parexel to $57 from $59.

Leerink analyst Michael Schmidt upgraded Immunogen to “outperform” from “market perform,” citing a higher level of conviction on IMGN’s lead asset mirvetuximab soravtansine.

Cantor analyst Bryan Brokmeier downgraded PerkinElmer to “neutral” from “overweight,” citing there may be risk from a moderation of newborn screening growth in China, the uncertain geopolitical environment, and an over-reliance on a contribution from new products.

BioShares Biotechnology Clinical Trials (BBC): $21.91, +$0.21, +19.0% YTD

BioShares Biotechnology Products (BBP): $36.45, -$0.06, +11.3% YTD

MARKET COMMENTARY

U.S. stock index futures pointed to a higher start for Wall Street with investors focused on first quarter results from some of the major corporate bellwethers. Visa and Mattel are scheduled to report quarterly results after markets close. The day’s economic calendar carries initial jobless claims and the Conference Board’s leading economic index. Upbeat results from Unilever boosted consumer stocks and helped European markets to trade higher. Asian stocks rose as commodity prices steadied and prompted some bargain hunting by investors. In currency markets, the New Zealand dollar was the biggest mover after the country’s inflation surged past 2 percent to its highest level in five years. The euro strengthened against the dollar despite looming uncertainty around French elections over the weekend and gold steadied. Oil prices recovered from losses in the previous session as leading Gulf oil producers signaled a likely extension of OPEC-led supply cuts beyond the middle of the year.

MARKET HIGHLIGHTS

Transgene announced the evolution and reinforcement of the Company’s Board of Directors. In the Annual General Meeting notice published today, Transgene’s Board of Directors has proposed a resolution to shareholders to appoint two new independent board members, Marie Landel and Maya Said. The notice also proposes resolutions on the renewal of the mandates of current Board members Philippe Archinard, Benoît Habert, Alain Mérieux, Dominique Takizawa.  Following the Shareholders General meeting on June 8, 2017, assuming shareholder approval of these proposals, Transgene’s Board of Directors will comprise ten members, with a majority of independent members.

Mateon Therapeutics announced that the Markey Cancer Center at the University of Kentucky has enrolled the first patient into a new phase 1 study of CA4P in combination with everolimus for the treatment of neuroendocrine tumors.

iCAD announced that updated clinical data on the use of intraoperative radiation therapy (IORT) using the Xoft® Axxent® Electronic Brachytherapy (eBx®) System will be presented during the American Brachytherapy Society (ABS) Annual Meeting in Boston, MA. The Xoft System and its suite of products will also be showcased at the Xoft booth (#T1) from April 21-22, 2017.

Immune Pharmaceuticals announced that it has entered into a letter of intent with Pint Pharma GmnH, a pharmaceutical company focused on Latin America and other markets (“Pint”), which binds the parties to seek agreement regarding an exclusive license by Pint of the rights to commercialize Ceplene throughout Latin America, including Argentina, Brazil, Chile, Colombia and Mexico. Immune and Pint target closing a final agreement within 30 days. Pursuant to the anticipated final agreement, Pint will be responsible for registration of Ceplene in Latin American countries based on the existing European marketing authorization and will carry out the full commercialization of the licensed product in the territory, including Ceplene registration, pricing and reimbursement, and sales and marketing activities.

CTD Holdings today corrected false and misleading statements published on April 18 in the on-line open access journal PLOS One.

BrainStorm Cell Therapeutics announced today that Chaim Lebovits, Chief Executive Officer, will present at the Alliance for Regenerative Medicine’s (ARM) 5th Annual Cell & Gene Therapy Investor Day on Thursday, April 27, 2017 at 9:40 EDT in Boston.

Oramed Pharmaceuticals announced that the European Patent Office has granted the Company a patent titled, "Methods and Compositions for Treating Diabetes: Capsules containing oil-based liquid compositions of combined therapeutic agents for treating diabetes." The patent covers Oramed’s invention, a combination therapy including both insulin and a GLP-1 analog in a single oral capsule.

ContraVir Pharmaceuticals announced new data demonstrating clinical antiviral activity, as well as safety and pharmacokinetic (PK) data of tenofovir exalidex (TXL). TXL is the Company’s proprietary liver targeting prodrug of the antiviral agent tenofovir for treating chronic hepatitis B virus (HBV), designed to offer equal or better HBV viral load reductions at doses lower than Viread (TDF), a commercially available tenofovir prodrug. ContraVir is also focusing on optimizing drug delivery of TXL to improve bioavailability and enhance its pharmacological activity.

Aldeyra Therapeutics announced that the FDA has granted Aldeyra’s novel compound ADX-102 orphan drug designation for the treatment of congenital ichthyosis, a severe skin disease characteristic of Sjögren-Larsson Syndrome (SLS). There are no FDA-approved therapies specifically indicated for the treatment of SLS, and ADX-102 is believed to be the only potential SLS therapy in clinical development.

AbbVie announced that 99 percent (n=145/146) of chronic hepatitis C virus (HCV) infected patients with genotype 1, 2, 4, 5 or 6 and compensated cirrhosis (Child-Pugh A) achieved sustained virologic response at 12 weeks post-treatment (SVR(12)) with its investigational, pan-genotypic regimen of glecaprevir/pibrentasvir (G/P). These high SVR(12) rates were seen following 12 weeks of G/P treatment without ribavirin. Patients with specific virus strains associated with resistance or with a high quantity of the virus in their bloodstream before treatment initiation were not excluded from the study. These new data, from the Phase III EXPEDITION-1 study, will be featured as an oral presentation today at The International Liver Congress (ILC) 2017 in Amsterdam, The Netherlands.  Glecaprevir (GLE) was discovered during the ongoing collaboration between AbbVie and Enanta Pharmaceuticals for HCV protease inhibitors and regimens that include protease inhibitors.

AbbVie announced that two Phase III studies evaluating veliparib, an investigational, oral poly (adenosine diphosphate ADP-ribose) polymerase (PARP) inhibitor did not meet their primary endpoints. The studies evaluated veliparib in combination with the chemotherapy regimen carboplatin and paclitaxel in patients with squamous non-small cell lung cancer (NSCLC) and triple negative breast cancer (TNBC). Full results will be presented at upcoming medical meetings or published in a peer-reviewed journal.

Targovax ASA announced that it has recruited the first patient in an exploratory Phase Ib clinical trial of TG02 in patients with locally recurrent RAS-mutated rectal cancer scheduled to have surgery. The trial is being conducted at clinical sites in Australia and New Zealand.

TESARO announced that ZEJULA (niraparib), an oral, once-daily poly(ADP-ribose) polymerase (PARP) inhibitor, is now available by prescription in the U.S. The FDA approved ZEJULA on March 27, 2017 for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response (CR or PR) to platinum-based chemotherapy. Following the news, Leerink analyst Seamus Fernandez decreased his price target to $158 from $186; Citi analyst Robyn Karnauskas decreased her price target to $216 from $232.

Novartis announced the publication of a study conducted by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) demonstrating that 58% of patients with treatment-naïve severe aplastic anemia (SAA) achieved complete response at six months when treated with eltrombopag at the initiation of and concurrently with standard immunosuppressive treatment. The study evaluated three sequential treatment groups, or cohorts. Cohort 3 added eltrombopag at the initiation of immunosuppressive therapy and showed a higher complete response rate than cohorts 1 and 2, where eltrombopag was initiated on day 14. The data is published in the latest issue of The New England Journal of Medicine.

Clearside Biomedical announced completion of enrollment of an exploratory clinical trial (the “HULK” trial) of CLS-TA for suprachoroidal administration, its proprietary suspension formulation of the corticosteroid triamcinolone acetonide, with or without intravitreal Eylea (aflibercept), for the treatment of diabetic macular edema (“DME”).

Celsion provided an update on its OVATION Study, a Phase Ib dose escalating clinical trial combining GEN-1, the Company’s DNA-based immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with advanced (stage III/IV) ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery.  GEN-1 is an IL-12 DNA plasmid vector formulated as a nanoparticle in a non-viral delivery system to cause the sustained local production and secretion of the Interleukin-12 (IL-12) protein loco-regionally to the tumor site.

Teva Pharmaceutical Industries announced the simultaneous launch of AirDuo RespiClick (fluticasone propionate and salmeterol) inhalation powder and its authorized generic for the treatment of asthma in patients aged 12 years and older who are uncontrolled on an inhaled corticosteroid (ICS) or whose disease severity clearly warrants the use of an ICS/long-acting beta(2)-adrenergic agonist (LABA) combination.

Agenus announced that the first patient has been dosed in a Phase I/II clinical trial of its anti-PD-1 antibody, AGEN2034.

Bicycle Therapeutics announced the receipt of a preclinical milestone in connection with the advancement of a Bicycle into preclinical development for the treatment of diabetic macular edema, under its ophthalmology alliance with ThromboGenics.

RedHill Biopharma announced the publication of an article describing the positive results from the Phase I clinical study with YELIVA (ABC294640) in advanced solid tumors. The article, entitled “A Phase I Study of ABC294640, a First-in-Class Sphingosine Kinase-2 Inhibitor, in Patients with Advanced Solid Tumors”, was authored by scientists from the Medical University of South Carolina (MUSC) Hollings Cancer Center and Apogee Biotechnology and was published in Clinical Cancer Research. The article is available online on the journal’s website.

uniQure announced that it will not pursue the renewal of the Glybera (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.

Prometic Life Sciences presented new results at the International Liver Congress ILC 2017 of the European Association for the Study of the Liver EASL in Amsterdam on the positive effects of PBI-4050 on reduction of non-alcoholic steatohepatitis (NASH) in a mouse model of obesity and metabolic syndrome.

H3 Biomedicine, a member of Eisai’s global Oncology Business Group, announced that is has extended its multi-year collaboration with Foundation Medicine for the discovery and development of precision medicines in oncology, which was signed in February 2015.

Allergan announced that it will collaborate with TARGET PharmaSolutions, a clinical data company focused on real world evidence, on its TARGET-NASH study. TARGET-NASH is a five-year longitudinal observational study that looks at patients with nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH). 

SIGA Technologies announced the completion of enrollment and dosing in the final cohort of healthy subjects in a Phase I clinical study of an intravenous (IV) formulation of its lead drug candidate, TPOXX (tecovirimat).  TPOXX is being developed for the treatment of smallpox, as well as other orthopoxvirus infections. There were no drug-related Serious Adverse Events (“SAEs”) reported.

Tocagen announced the closing of its initial public offering of common stock, including the underwriters’ exercise of their option to purchase an additional 1,275,000 shares at the public offering price of $10.00 per share. The underwriters’ option brought the total number of shares of common stock sold by Tocagen to 9,775,000 shares and increased the amount of gross proceeds raised in the offering to approximately $97.8 million, prior to deducting the underwriting discount and estimated expenses of the offering. Leerink Partners, Evercore Group and Stifel, Nicolaus & Company acted as joint bookrunning managers for the offering.

Sorrento Therapeutics announced the closing of its previously announced underwritten public offering of 23,625,084 shares of its common stock at a public offering price of $2.00 per share, before deducting underwriting discounts and commissions and estimated offering expenses payable by Sorrento. The net proceeds to Sorrento from this offering were approximately $43.5 million, after deducting underwriting discounts and commissions and other estimated offering expenses. Cantor Fitzgerald & Co. acted as the lead book-running manager for the offering. FBR Capital Markets & Co. acted as a joint book-running manager. Oppenheimer & Co. and Aegis Capital Corp. acted as co-lead managers and Joseph Gunnar & Co., Rodman & Renshaw and Roth Capital Partners acted as co-managers.

ANALYST RECOMMENDATIONS

Roth analyst Mark Breidenbach initiated coverage of Mateon Therapeutics with a “buy” rating and $2 price target, citing positive results from a randomized Phase 2 study conducted by the Gynecologic Oncology Group (GOG), Mateon is conducting a pivotal trial (FOCUS) of its lead drug, CA4P, in a group with high unmet need—patients with platinum-resistant ovarian cancer.

Cantor Fitzgerald analyst Mara Goldstein increased her price target of NewLink Genetics to $32 from $20, citing increased conviction about indoximod and due to the IDO program overall, based on data presented at AACR.

Roth analyst Sa’ar Yaniv initiated coverage of the following companies: Zafgen with a “buy” rating and $9 price target; Poxel with a “buy” rating and €11 price target.  

Goldman analyst Terence Flynn revised his price target of the following companies: Amgen increased to $204 from $202; Biogen increased to $285 from $282.

JP Morgan analyst Chris Schott decreased his price target of Valeant to $10 from $15, citing a number of uncertainties around core business trends that are unlikely to be resolved in the near term.

BioShares Biotechnology Clinical Trials (BBC): $22.08, +$0.17, +19.9% YTD

BioShares Biotechnology Products (BBP): $36.79, +$0.34, +12.3% YTD

MARKET COMMENTARY

U.S. stock index futures were marginally higher on rising expectations for first-quarter corporate profits. On the economic front, investors will look forward to existing homes sales and flash reading of Markit’s composite PMI. European markets were broadly lower and the euro steadied below three-week high as investors retreated from risky bets ahead of the first round of France’s Presidential election that is seen as too close to call. Asian stocks recorded a mixed trading day. Oil prices edged lower over doubts that an OPEC-led production cut will restore balance to an oversupplied market. Gold was little changed as risk-averse investors sought refuge in the safe-haven asset.

MARKET HIGHLIGHTS

DelMar Pharmaceuticals announced that it has closed its previously announced registered public offering of an aggregate of 2,769,232 shares of common stock and warrants to purchase an aggregate of 2,076,924 shares of common stock at a price to the public of $3.25 per share and related warrant. The warrants have an exercise price of $3.50 per share, are immediately exercisable and have a term of exercise of five years.  Rodman & Renshaw, a unit of H.C. Wainwright & Co., acted as exclusive placement agent for the offering.

Rockwell Medical announced that it has filed definitive proxy materials with the U.S. Securities and Exchange Commission (“SEC”) in connection with the company’s upcoming 2017 Annual Meeting of Shareholders (“Annual Meeting”) to be held on June 1, 2017. The proxy statement and other important information related to the Annual Meeting can be found on the company’s website at www.rockwellmed.com.

Eiger BioPharmaceuticals announced additional supportive and encouraging lonafarnib (LNF) data from the LOWR HDV (LOnafarnib With Ritonavir in Hepatitis Delta Virus) Program presented at The International Liver Congress™ 2017, in Amsterdam, Netherlands.  After 24 weeks of treatment, all-oral lonafarnib-based regimens (LNF 25 mg or 50 mg BID + Ritonavir, or RTV) suppressed HDV-RNA below the limit of quantitation in 36% of patients, and 60% achieved ALT normalization.  The addition of PEG IFN to LNF 25 mg BID + RTV (triple therapy) suppressed HDV-RNA below the limit of quantitation in 80% of patients, and 78% achieved ALT normalization.  In patients treated for 48 weeks with triple therapy of PEG IFN + LNF 25 mg BID + RTV, PCR-negativity was achieved in 67% of patients at the end of treatment.

Onconova Therapeutics announced that it intends to offer and sell shares of its common stock in an underwritten public offering. Onconova also expects to grant to the underwriter for the offering a 45-day option to purchase an additional 15% of the shares of common stock offered in the public offering. All of the shares to be sold in the offering are to be sold by Onconova. Onconova intends to use the net proceeds from the offering to support development of rigosertib for treatment in higher risk MDS.

Aurinia Pharmaceuticals announced additional 48-week results from its global Phase IIb AURA-LV (AURA) study in lupus nephritis (LN) during the National Kidney Foundation 2017 Spring Clinical Meetings in Orlando, FL. In addition to the trial meeting its complete and partial remission (“CR”/”PR”) endpoints at 48 weeks, all pre-specified secondary endpoints that have been analyzed to date were also met at 48 weeks. These pre-specified endpoints include: time to CR and PR (speed of remission); reduction in Systemic Lupus Erythematosus Disease Activity Index or SLEDAI score; and reduction in urine protein creatinine ratio (UPCR) over the 48-week treatment period. The data were presented during the late-breaking session by lead author Dr. Samir Parikh, a clinical investigator for the study and Assistant Professor of Clinical Nephrology at the Ohio State University.

VBL Therapeutics announced that the Independent Data Safety Monitoring Committee (DSMC) met to conduct its second safety review of the Phase 3 GLOBE Study investigating ofranergene obadenovec (VB-111) in recurrent glioblastoma (rGBM).  The DSMC is an independent multidisciplinary group that conducts detailed reviews of un-blinded study data, discusses potential safety concerns and provides recommendations regarding trial continuation. The committee reviewed the GLOBE safety data collected through a cutoff date in March 2017 and unanimously recommended that the study continue as planned.

Onxeo S.A. has received the EnterNext Tech 40 Label and is thus admitted to the “Tech 40” index.  The “Tech 40” label is an initiative launched two years ago by Euronext to give more prominence to outstanding Tech companies listed on the various European markets covered by EnterNext (Amsterdam, Brussels, Lisbon and Paris).  The 40 companies receiving this label were chosen by a committee of independent European experts based on their business, financial and stock market performances.

Aeglea BioTherapeutics names Anthony Quinn, M.B Ch.B, Ph.D., FRCP as interim chief medical officer, effective immediately.  Dr. Quinn also serves on the Company’s Board of Directors and, most recently, was executive vice president, chief medical officer and head of research & development at Synageva BioPharma Corp. until its acquisition by Alexion Pharmaceuticals in 2015.  F. Andrew Dorr, M.D., the Company’s previous interim chief medical officer, will transition his work to focus on the clinical development of the Company’s oncology programs.

XBiotech announced that the EMA rendered a negative “trend” vote after meeting with the Company to discuss the “Day 180 List of Outstanding Issues” related to the Company’s MAA for its candidate antibody for the treatment of colorectal cancer. A negative trend vote means it is unlikely that a positive CHMP opinion related to the Company’s MAA will be attained at the formal decision vote scheduled in May, and that additional steps would need to be taken to potentially gain marketing approval.

Aevi Genomic Medicine announced updated responder data from a subset of genomically identified patients in the SAGA trial of AEVI-001 in adolescent Attention Deficit Hyperactivity Disorder (ADHD). The new data will be presented at the 6(th) World Congress on ADHD in Vancouver, Canada on April 21, 2017. Key results of the trial include: Identification of nine genes that are predictive of clinically meaningful and statistically significant response [ADHD-Rating Scale (ADHD-RS) reduction of 17.6, p < .005] in the SAGA trial. These genes include certain glutamate metabotropic receptors (GRM) and neurodevelopmental genes and are found in approximately 10% of pediatric ADHD patients. One of the neurodevelopmental genes, contactin-4 (CNTN4) previously identified as important in Autism Spectrum Disorder (ASD), was highly enriched in the responder population (43%) and represents approximately 5% of pediatric ADHD patients. CNTN4 mutation phenotype is more severe with a higher prevalence of emotional dysregulation, including: anger control, risk taking, inappropriate movements and sounds.  All CNTN4 mutation positive patients on treatment (n=6) had clinically meaningful and statistically significant response to AEVI-001 (ADHD-RS reduction of 20.8, p=.03). 

Neurocrine Biosciences announced that long-term safety and efficacy data from the KINECT 3 Phase III extension study of INGREZZA (valbenazine) capsules for the treatment of adults with tardive dyskinesia will be presented as a platform presentation at the American Academy of Neurology (AAN) Annual Meeting April 22-28, 2017 in Boston. Additionally, two posters representing additional data from several clinical trials of INGREZZA will be presented, including an analysis of its pharmacologic characteristics.

Atara Biotherapeutics announced that it has entered into a clinical trial collaboration agreement with Merck, to evaluate Atara Bio’s allogeneic Epstein-Barr virus (EBV)-specific cytotoxic T lymphocytes (CTL), or ATA129, in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with platinum resistant or recurrent EBV-associated NPC. The Phase I/II trial will evaluate the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of the combination and is planned for initiation in 2018.

AbbVie announced high SVR(12 )rates were achieved with 8 weeks of treatment with its investigational, once-daily, ribavirin-free, pan-genotypic regimen of glecaprevir/pibrentasvir (G/P) in patients with challenging to treat genotype 3 (GT3) chronic hepatitis C virus (HCV) infection. In results from the Phase III ENDURANCE-3 study, 95 percent (n=149/157) of GT3 chronic HCV infected patients without cirrhosis and who are new to treatment achieved sustained virologic response at 12 weeks post-treatment (SVR(12)) following 8 weeks of treatment with G/P. These new data will be featured as an oral presentation today at The International Liver Congress (ILC) 2017 in Amsterdam, The Netherlands. Glecaprevir (GLE) was discovered during the ongoing collaboration between AbbVie and Enanta Pharmaceuticals for HCV protease inhibitors and regimens that include protease inhibitors.

Gilead Sciences announced results from an open-label, proof-of-concept study evaluating GS-0976, an investigational inhibitor of Acetyl-CoA carboxylase (ACC), in patients with nonalcoholic steatohepatitis (NASH). The data, from ten patients treated with GS-0976 20 mg taken orally once daily for 12 weeks, indicated that treatment was associated with statistically significant improvements in liver fat content and noninvasive markers of fibrosis, via inhibition of hepatic de novo lipogenesis (DNL). The late-breaking data were presented today during a general session at The International Liver Congress 2017 in Amsterdam (#GS-009).

PTC Therapeutics announced it has completed its acquisition of all rights to Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy (DMD) in the U.S. Execution of the asset purchase agreement setting forth the terms of the acquisition was announced on March 16, 2017. Financial terms of the acquisition include a total upfront consideration of $140 million paid to Marathon Pharmaceuticals. Marathon is also entitled to receive payments from PTC based on annual net sales of Emflaza beginning in 2018, which PTC expects will range as a percentage of net sales between the low to mid-20s on a blended average basis. In addition, Marathon has the opportunity to receive a single $50 million sales-based milestone.

Atara Biotherapeutics announced that its collaborating investigators at the Queensland Institute of Medical Research (QIMR Berghofer) and The University of Queensland are reporting interim Phase I trial results from the autologous version of ATA188, or autologous ATA188, in patients with primary or secondary progressive MS (PPMS and SPMS, respectively), at the 69(th) AAN Annual Meeting in Boston, Massachusetts.

Jounce Therapeutics announced enrollment of patients into the Phase II portion of the company’s Phase I/II ICONIC study of JTX-2011. JTX-2011 is a monoclonal antibody targeting ICOS, a protein found on the surface of certain T cells within many solid tumors.

Cempra announced the company is presenting eight abstracts highlighting solithromycin and the need for new treatments for community-acquired bacterial pneumonia (CABP) at the 27(th) European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) in Vienna, Austria.

BeiGene announced that the first patient was dosed in a pivotal clinical trial of BGB-A317, an investigational anti-PD-1 antibody, in Chinese patients with relapsed or refractory classical Hodgkin lymphoma (cHL).

Valeant Pharmaceuticals International announced that following the evaluation and approval of its Patient Access and Pricing Committee (PAPC), the company has decided to list SILIQ (brodalumab) injection, at $3,500 per month, which is the lowest injectable biologic psoriasis treatment currently on the market. SILIQ will also be included in the company’s patient access program to further offer financial support and access to patients. SILIQ, a monoclonal antibody that targets the IL-17 receptor for patients with moderate-to-severe plaque psoriasis, is indicated for the treatment of moderate to severe plaque psoriasis in adult patients who are candidates for systemic therapy or phototherapy and have failed to respond or have lost response to other systemic therapies. SILIQ is the only product that included the psoriasis area and severity index (PASI 100) during clinical trials as a primary endpoint. The sales and marketing of SILIQ are expected to commence in the U.S. during the second half of 2017.

Apricus Biosciences announced the pricing of an underwritten public offering of an aggregate of 5,030,000 units, with each unit consisting of one share of Apricus common stock and one warrant to purchase 0.75 of a share of common stock, at a public offering price of $1.40 per unit.  The shares of common stock and warrants are immediately separable and will be issued separately in this offering. Apricus’ gross proceeds from this offering are expected to be approximately $7.0 million, before deducting underwriting discounts and commissions and other estimated offering expenses, and excluding any proceeds Apricus may receive upon exercise of the warrants to be issued in this offering.  The offering is expected to close on or about April 26, 2017, subject to the satisfaction of customary closing conditions.

Syros Pharmaceuticals announced that it has entered into a stock purchase agreement with several institutional accredited investors for the private placement of 2,592,591 shares of common stock at a purchase price of $13.50 per share, yielding expected gross proceeds of $35 million. The purchase price for the shares represents an 8.8% discount to the average closing price of the Company’s common stock over the 30 trading days prior to the entry into the stock purchase agreement. The private placement is expected to close on or about April 26, 2017, subject to the satisfaction of customary closing conditions. The private placement was anchored by Samsara BioCapital, a new biotech investment firm founded by Srini Akkaraju, M.D., Ph.D., alongside a number of existing and new institutional investors. J.P. Morgan Securitiesand Cowen and Company, LLC acted as placement agents to the Company in connection with the offering.

ANALYST RECOMMENDATIONS

Citi analyst Liav Abraham removed Allergan from the “US Focus List” and increased her price target to $270 from $245, citing the company’s higher quality assets and growth profile, pipeline optionality, and stronger balance sheet vs. the peers.

BTIG analyst Robert Hazlett initiated coverage of Cascadian Therapeutics with a “buy” rating and $13 price target, citing Cascadian is focused on the development of Tucatinib, a novel, oral highly selective HER2 inhibitor for breast cancer.

Maxim analyst Lauren Chung initiated coverage of GW Pharmaceuticals with a “buy” rating and $135, citing GW’s lead product, Epidiolex, an orphan drug designated oral CBD (cannabidiol), showed positive results in three P3 trials for Dravet (DS) and Lennox-Gastaut syndromes (LGS).

Barclays analyst Geoff Meacham revised his price target of the following companies: Biogen to $360 from $380; Celgene to $125 from $120; Gilead to $85 from $90; Vertex to $100 from $90.

BioShares Biotechnology Clinical Trials (BBC): $21.70, -$0.38, +17.9% YTD

BioShares Biotechnology Products (BBP): $36.47, -$0.32, +11.4% YTD

MARKET COMMENTARY

Wall Street looked set to follow global stocks higher, after the market-favored candidate, Emmanuel Macron, won the first round of the French election, reducing the risk of another Brexit-like shock. The euro surged, while safe-haven assets including gold and the yen fell. Oil prices rose driven by expectations that OPEC will extend output cuts till the end of 2017. Alcoa, T-Mobile and Whirlpool are scheduled to report quarterly results after the closing bell.

MARKET HIGHLIGHTS

Immune Pharmaceuticals announced a major corporate restructuring with the objective of prioritizing and segregating its research and development efforts on a focused set of products in inflammatory disease and dermatology and strengthening its financial position. In line with this prioritization, the Company’s Board of Directors has authorized Dr. Daniel Teper to lead the Company’s oncology business within the Company’s Cytovia subsidiary and to pursue a possible spin-off of Cytovia into a separate, stand-alone company independent from Immune. Cytovia will focus on the development and commercialization of novel immuno-oncology and hematology therapeutics, led by Ceplene, an immunotherapy treatment in late stage development in combination with low dose interleukin 2 (IL-2) for the remission maintenance of patients with Acute Myeloid Leukemia; Azixa and crolibulin, two Phase II drug candidates with synergistic potential with immuno-oncology drugs; and a bispecific antibody platform to be supported by collaborative partnerships. Under the leadership of Dr. Teper, Cytovia will aim to grow into a global specialty biopharmaceutical company through these product candidates and the acquisition of additional late stage or commercial stage oncology products. Cytovia intends to raise sufficient capital to support R&D investment through product licensing and partnership transactions, government grants and issuance of debt and equity.

Immune Pharmaceuticals announced that on April 18, 2017 the Company has received written notice from the Listing Qualifications Department of The NASDAQ Stock Market LLC notifying that the Company does not comply with Nasdaq’s filing requirements for continued inclusion set forth in Listing Rule 5250(c)(1) because it had not yet filed its Form 10-K for the year ended December 31, 2016.  The Company is working to complete the 2016 Form 10-K and will file it as soon as practicable.

DURECT Corporation announced that clinical data on DUR-928 were presented at The International Liver Congress^TM 2017 (the 52nd annual meeting of the European Association for the Study of the Liver (EASL)) on April 22 in Amsterdam. 

Lipocine announced the completion of enrollment for both its dosing validation ("DV") study and its dosing flexibility ("DF") study for LPCN 1021.  LPCN 1021 is an oral testosterone product candidate for testosterone replacement therapy in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.

iCAD will release financial results for the three months ended March 31, 2017 after the close of the market, and host a conference call at 4:30pm Eastern Time, on Tuesday, May 2, 2017.

Eiger BioPharmaceuticals announced that an abstract describing prevalence of hepatitis delta virus (HDV) in the U.S. will be presented at Digestive Disease Week (DDW) in Chicago, Illinois, May 6 to 9, 2017.

Arrowhead Pharmaceuticals presented clinical data from a Phase 1a/1b study of ARC-AAT, the company’s prior generation investigational medicine that was being studied for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD), at The International Liver Congress™ 2017 (ILC), the annual meeting of the European Association for the Study of the Liver (EASL).  The data demonstrated that an RNA interference (RNAi) therapeutic can achieve deep durable, and dose dependent reductions of alpha-1 antitrypsin (AAT), with the highest dose studied in healthy volunteers achieving near full suppression of the liver production of the AAT protein. In addition, at doses of 2 and 4 mg/kg in AATD patients, ARC-AAT produced similar levels of knockdown as seen in healthy volunteers. These data support the continued advancement of ARO-AAT, Arrowhead’s follow-on product candidate that utilizes the company’s next generation, proprietary subcutaneously administered delivery vehicle.

BioTime announced that an abstract related to the Company’s retinal tissue implant has been accepted for a paper presentation at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, Maryland, May 7-11.

Paratek Pharmaceuticals announced today that an analysis of microbiology data from its Phase 3 study of omadacycline in acute skin infections found that once-daily treatment with IV-to-oral omadacycline is effective in treating the most frequently isolated bacterial pathogens associated with skin infections, including methicillin-resistant Staphylococcus aureus (MRSA). These findings were presented for the first time at the annual meeting of the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2017) in Vienna, Austria.

Paratek Pharmaceuticals announced today that two cost benefit analyses presented at the annual meeting of the European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2017) in Vienna, Austria, highlight the potential of IV-to-oral omadacycline to confer cost savings relative to the current standard of care for the treatment of patients with acute bacterial skin and skin structure infection (ABSSSI).

Paratek Pharmaceuticals announced that it has entered into a collaboration with Zai Lab (Shanghai) Co., Ltd., a biopharmaceutical company based in China, to support the development and commercialization of omadacycline for patients in China.  Under the agreement, Paratek has granted to Zai Lab an exclusive license to develop, manufacture and commercialize omadacycline for the greater China territory, specifically the People’s Republic of China, Hong Kong, Macau, and Taiwan markets.  The companies will establish a joint steering committee to review and oversee all development, manufacturing, and commercialization plans. Paratek will receive a $7.5 million upfront payment in connection with the signing of the agreement and is eligible for additional milestone payments related to development, regulatory, and commercial milestones.  In addition, Paratek will be eligible to receive royalty payments on sales of omadacycline in the territory.

Alcobra today mailed proxy materials for the Extraordinary General Meeting of Shareholders, called by Brosh Capital L.P. and certain of its affiliates ("the Brosh Group"), to describe the Board’s opposition to the Brosh Group’s calling of the meeting and to provide shareholders with the ability to vote on the Company’s proxy card to defend Alcobra against an opportunistic takeover by a new investor with no track record of managing pharmaceutical companies.  As previously announced, Alcobra believes that the Brosh Group’s proposals violate the organizational documents of the Company and applicable law, and therefore Alcobra respectfully rejected the Brosh Group’s request to convene an extraordinary general meeting of shareholders. Alcobra believes the Extraordinary Meeting of Shareholders should not be held in light of applicable law, but is nevertheless committed to defending shareholders against an opportunistic takeover should the meeting be deemed legally valid.

Achaogen announced multiple presentations that highlight the effectiveness of plazomicin against MDR gram-negative bacteria in multiple settings. Results were presented at the European Congress of Clinical Microbiology and Infectious Disease (ECCMID) which is being held in Vienna, Austria from April 22-25, 2017.

Albireo Pharma announced that the embargo on its late-breaker abstract discussing preliminary data from a Phase 2 clinical trial of its lead product candidate A4250 in children with cholestatic liver disease has been lifted and the data was presented on Saturday at The International Liver Congress™ 2017 in Amsterdam. The data demonstrated improvement in pruritus and reduction in serum bile acids (sBA) in most patients, particularly patients with progressive familial intrahepatic cholestasis (PFIC), after four weeks of treatment with A4250.

Arena Pharmaceuticals announced the completion of its previously announced underwritten public offering of 69,000,000 shares of its common stock, including 9,000,000 shares sold pursuant to the full exercise of an option previously granted to the underwriters. All of the shares were sold at a price to the public of $1.15 per share. Including the option exercise, the aggregate gross proceeds from the offering were approximately $79.4 million, before deducting the underwriting discounts and commissions and offering expenses payable by Arena. Arena anticipates using the net proceeds from the offering for clinical and preclinical development of drug candidates, for general corporate purposes, including working capital and costs associated with manufacturing services, and for capital expenditures. Citigroup and Leerink Partners acted as joint book-running managers for the offering.

Cara Therapeutics announced summary results from its Phase I safety trial showing that I.V. CR845 did not significantly differ from placebo across three quantitative measures of respiratory drive in healthy individuals. Respiratory depression remains the most life-threatening side effect of traditional, centrally acting, opioid analgesics, the most commonly used drug class for current treatment of postoperative pain in the United States.

Sanofi announced that the FDA approved Thymoglobulin [anti-thymocyte globulin (rabbit)], for use in conjunction with concomitant immunosuppression in the prophylaxis, or prevention, of acute rejection in patients receiving a kidney transplant.

Akari Therapeutics announced that it will present data from an interim analysis of its ongoing Phase II trial of Coversin in paroxysmal nocturnal hemoglobinuria (PNH), as well as preclinical data for additional indications and other opportunities, at their Research and Development Day.

PDL BioPharma announced that on April 21, 2017, the Company entered into a settlement agreement with certain subsidiaries of Merck  to resolve the patent infringement lawsuit between the parties pending in the U.S. District Court for the District of New Jersey related to Merck’s Keytruda humanized antibody product. Under the terms of the agreement, Merck will pay the Company a one time, lump-sum payment of $19.5 million, and the Company will grant Merck a fully paid-up, royalty free, non-exclusive license to certain of the Company’s Queen et al. patent rights for use in connection with Keytruda as well as a covenant not to sue Merck for any royalties regarding Keytruda.  In addition, the parties agreed to dismiss all claims in the relevant legal proceedings.

Auris Medical Holding announced key results from AMPACT2 (AM-101 in the Post-Acute Treatment of Peripheral Tinnitus 2), the open-label extension study of the Phase III TACTT3 clinical trial. The study confirms the long-term safety of Keyzilen and further supports early treatment from onset of inner ear tinnitus.

XOMA announced that it has achieved positive Phase II proof-of-concept results for X213 in physiological hyperprolactinemia (HPRL). X213 is a monoclonal antibody that neutralizes prolactin action.

Inovio Pharmaceuticals announced that it has commenced a Phase II trial to evaluate the efficacy of VGX-3100 in patients with pre-cancerous lesions of the vulva, or vulvar intraepithelial neoplasia (VIN). VGX-3100 is an immunotherapy that targets human papillomavirus (HPV) 16 and 18 and is being studied for the treatment of HPV-related pre-cancerous lesions and persistent HPV infection that causes these lesions.

Medivir communicated an update on the status of the development of JNJ-4178, the triple combination of simeprevir, odalasvir and AL-335, following The International Liver Congress 2017 of the European Association for the Study of the Liver (EASL), which was held in Amsterdam, on 19-23 April.

MOLOGEN AG announced the key results of the exploratory phase II IMPULSE study. The randomized study evaluated the efficacy and safety of lefitolimod in patients with extensive-disease small-cell lung cancer (SCLC). IMPULSE shows positive results regarding overall survival (OS) in two subgroups of patients in comparison to the control group (standard therapy). Additional, potentially promising subgroups will be identified. The results of this SCLC study provide significant guidance for defining patient populations that, even beyond this study, are most likely to benefit from the immune surveillance reactivator lefitolimod, even though in this highly challenging indication the primary endpoint OS was not met in the overall study population.

Pfizer announced positive results of the REPROVE1 Study that showed patients diagnosed with hospital-acquired pneumonia (HAP), treated with Zavicefta, a novel combination antibiotic for the treatment of certain known or suspected Gram-negative bacterial infections, or Meropenem (meropenem for injection), a broad spectrum carbapenem antibiotic currently considered the standard of care, experienced comparable rates of clinical cure at test-of-cure 21-25 days after randomization. Clinical cure was the primary endpoint of the study and defined as a complete resolution of all signs and symptoms of infection.

Eli Lilly announced that following a pre-planned interim analysis for MONARCH 3, the trial met its primary endpoint of demonstrating statistically significant improvement in progression-free survival (PFS). In addition, improvement was shown in a key secondary endpoint of objective response rate (ORR). The Phase III study evaluated abemaciclib, a cyclin-dependent kinase (CDK)4 and CDK6 inhibitor, in combination with an aromatase inhibitor (letrozole or anastrozole) compared to treatment with an aromatase inhibitor alone in women with hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. Detailed efficacy and safety results will be presented at a medical meeting in the second half of the year.

Sage Therapeutics announced the appointment of Michael Cloonan as Chief Business Officer. Additional executive appointments include Christopher Silber, M.D., as Senior Vice President, Clinical Development, Paul Hodgkins, Ph.D., as Vice President, Health Economics and Outcomes Research and Value Demonstration and Frank Sanders as Vice President, Sales & Marketing. In addition, Amy Schacterle, Ph.D., has been promoted to Senior Vice President, Regulatory Affairs and Quality Assurance.

Matinas BioPharma Holdings announced that it presented positive preclinical data at the 27(th) Annual European Congress of Clinical Microbiology and Infectious Diseases, being held April 22-25, 2017 in Vienna, Austria.

American Regent, a member of the Daiichi Sankyo Group, announced that the first patient has been enrolled into the phase III clinical trial, HEART-FID. This double-blind, multicenter, prospective, randomized, placebo-controlled study will assess the efficacy and safety of iron therapy using intravenous (IV) ferric carboxymaltose (FCM), relative to placebo, in the treatment of patients with heart failure, iron deficiency and a reduced ejection fraction.

RedHill Biopharma announced enrollment of the last patient in the Phase II study with BEKINDA (RHB-102)(1) 12 mg for the treatment of diarrhea-predominant irritable bowel syndrome (IBS-D).

Denovo Biopharma announced that it has obtained an exclusive license to liafensine (DB104), a serotonin-norepinephrine-dopamine reuptake inhibitor (SNDRI), a late-stage CNS drug, from Albany Molecular Research.  The agreement between Denovo Biopharma and AMRI concludes AMRI’s divesture of all its legacy intellectual property assets.  Under the terms of the definitive agreement, Denovo Biopharma gains an exclusive license to all rights to develop, manufacture and commercialize liafensine globally. Liafensine represents the third late stage product in Denovo Biopharma’s pipeline.

Biogen will present Phase III end of study SPINRAZA (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. The SPINRAZA development program represents the largest body of clinical data of its kind in SMA. SPINRAZA data will be presented at the American Academy of Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.

Biogen announced new real-world data that show treatment with its leading multiple sclerosis (MS) therapies, TECFIDERA (dimethyl fumarate) and TYSABRI (natalizumab), early in the course of the disease may improve outcomes for people living with relapsing MS. These data were presented at the 69th annual meeting of the American Academy of Neurology (AAN) in Boston.

aTyr Pharma announced promising clinical results from its Phase Ib/II 003 trial assessing the safety and potential activity of Resolaris in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).

Mustang Bio, a Fortress Biotech company, announced that Manuel Litchman, M.D., has been appointed President and CEO. Dr. Litchman has also joined Mustang’s Board of Directors. Michael S. Weiss, who oversaw Mustang’s corporate operations on an interim basis, will continue to serve as Chairman of the Board of Directors.

CytoSorbents announced that it was awarded a two-year $999,996 Small Business Innovation Research (SBIR) Phase II contract to continue development of CytoSorb for fungal mycotoxin blood purification. This follows the successful completion of its previously announced $150,000 Phase I SBIR contract for mycotoxin removal.  This contract is funded through the Chemical and Biological Defense (CBD) SBIR Program which represents a component of the Joint Chemical and Biological Defense Science & Technology Program.  This SBIR project is being managed by the Joint Program Executive Office for Chemical and Biological Defense (JPEO-CBD), the U.S. Department of Defense focal point for research, development, acquisition, fielding, & life-cycle support of Chemical, Biological, Radiological, Nuclear (CBRN) equipment and medical countermeasures.

Acasti Pharma announced the granting of additional patents by the Taiwanese and Australian patent offices, further expanding the intellectual property position of CaPre. The granted patents are valid until 2030 and relate to concentrated therapeutic krill oil-based phospholipid omega-3 compositions covering methods for treating or preventing disorders associated with cardiovascular diseases. These patents add to Acasti’s growing portfolio of issued patents in the United States, China, Mexico, Saudi Arabia, Panama, and South Africa. Patent applications with similar claim sets are being pursued in many other jurisdictions worldwide.

Alder BioPharmaceuticals presented additional data for eptinezumab (formerly ALD403), which is in Phase III clinical development for the prevention of migraine, at the 69(th) Annual American Academy of Neurology (AAN) meeting in Boston.

Oryzon Genomics announced that the Board of Directors of the Alzheimer’s Drug Discovery Foundation (ADDF) has approved a grant of $300,000 to Oryzon Genomics S.A. in support of the project "Clinical development of a companion marker for treatment with the dual LSD1/MAO-B inhibitor ORY-2001."

Theravance Biopharma announced that new preliminary data from the ongoing Telavancin Observational Use Registry (TOUR) study are the focus of three poster presentations at the 27th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID). TOUR, which has enrolled its target of 1,000 patients, is designed to report how VIBATIV (telavancin) is being used by healthcare practitioners to treat patients in real-world clinical settings. The presented findings, which focus on data from registry patients with diagnoses of complicated skin & skin structure infections (cSSSIs), bone and joint infections, or lower respiratory tract infections (LRTIs), report positive clinical responses for VIBATIV treatment ranging from 58.3% to 75.3% in these infection types. Positive clinical response was defined as cure or improvement leading to step-down oral therapy. The Company plans to present additional collections of data from the ongoing TOUR study at appropriate upcoming scientific conferences. The 27(th) ECCMID is being held in Vienna, Austria, April 22-25, 2017.

Adamas Pharmaceuticals announced the presentation of two separate analyses of pooled data from the placebo-controlled Phase III clinical trials of ADS-5102 (amantadine) extended-release capsules in oral platform and poster presentation sessions at the 69(th) American Academy of Neurology (AAN) Annual Meeting in Boston. The pooled data results, which are consistent with the original findings from EASE LID and EASE LID 3, demonstrated that people with Parkinson’s disease treated with ADS-5102 had a significant reduction in levodopa-induced dyskinesia (LID), as measured by the Unified Dyskinesia Ratings Scale (UDysRS), and showed statistically significant reduction in OFF time, as reported by Parkinson’s disease home diary data.

Galapagos announced the closing of its underwritten public offering of 4,312,500 ADSs, at a price of $90.00 per ADS, before underwriting discounts, for gross proceeds of €363.9 million. This includes the full exercise of the underwriters’ option to purchase additional ADSs. All of the ADSs were offered by Galapagos.

Bristol-Myers Squibb announced data from a Phase II study of BMS-986036, an investigational pegylated analogue of human fibroblast growth factor 21 (FGF21), a key regulator of metabolism, in patients with biopsy-confirmed nonalcoholic steatohepatitis (NASH) (F1-F3). The study achieved its primary endpoint of significant reduction in liver fat versus placebo. Statistically significant improvements were also seen in prespecified exploratory endpoints including biomarkers of fibrosis, metabolic parameters and markers of liver injury. These data were presented at a late-breaking oral presentation at EASL: The International Liver Congress in Amsterdam.

Merck announced the first sustained virologic response(1) (SVR) results 12 weeks after completion of therapy (SVR12, considered virologic cure) from C-SURGE, an ongoing, open label Phase II clinical trial evaluating MK-3682B [uprifosbuvir (MK-3682)(2)/grazoprevir(3)/rusazvir(4)], the company’s investigational triple-combination therapy in treatment-experienced patients with hepatitis C virus (HCV) genotype (GT) 1 infection for whom treatment with approved direct-acting antiviral regimens had failed. The study showed that 100 percent (43/43) of patients who completed 16 weeks of treatment plus ribavirin (RBV) achieved SVR12 and 100 percent (49/49) of patients who completed 24 weeks of treatment achieved SVR12 (abstract PS-159). These results were presented at The International Liver Congress 2017.

ContraVir Pharmaceuticals presented data demonstrating the synergistic antiviral activity from the combination of its two investigational drugs for the treatment of hepatitis B viral (HBV) infection, tenofovir exalidex (TXL, formerly CMX157) a nucleotide reverse transcriptase inhibitor and CRV431, a cyclophilin inhibitor. In addition, the mode of action (MOA) of CRV431 was further defined.

Achillion Pharmaceuticals announced the presentation of updated results from the ongoing Phase II ‘604 Study’ being conducted by Alios BioPharma, part of the Janssen Pharmaceutical Companies. These results were presented as an oral presentation during the European Association for the Study of the Liver (EASL) 2017 International Liver Congress in Amsterdam. These results demonstrate that the triple combination of simeprevir, odalasvir and AL-335 has the ability to shorten treatment duration, offer high efficacy and be generally well tolerated in those whose disease is caused by hepatitis C virus (HCV) genotype 1 (GT1), one of the most prevalent causes of hepatitis C globally.

Arbutus Biopharma presented results of the first three cohorts of a Phase II study of its RNAi agent, ARB-1467, at the European Association for the Study of the Liver (EASL) in Amsterdam, The Netherlands.

ANALYST RECOMMENDATIONS

Gabelli analyst Jing He downgraded Bioverativ to “hold” from “buy,” citing valuation.

Rodman & Renshaw analyst Raghuram Selvaraju increased his price target of Motif Bio to $25 from $16, citing Motif Bio Plc reported positive top-line data from the REVIVE-1 pivotal Phase III trial of its lead drug candidate, the next-generation dihydrofolate reductase inhibitor iclaprim.

CL King analyst David Westenberg increased his price target of Illumina to $220 from $175, citing ILMN has five to ten years of unobstructed growth in production-scale sequencing, and that investors underappreciate both Illumina’s ability to scale the SBS chemistry and Grail, Firefly and Helix.