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.