BioShares Biotechnology Clinical Trials (BBC): $20.88, +$0.68, -28.1% YTD

BioShares Biotechnology Products (BBP): $34.56, +$0.83, +12.1% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were lower, a day after the Dow Jones hit a record high and the S&P 500 financial sector surged to its highest since the 2008 financial crisis. University of Michigan’s surveys of consumers sentiment data is on the economic calendar. Major European shares fell on concerns over the risks involved in U.S. President-elect Donald Trump’s fiscal stimulus program and Asian stocks traded mixed. The dollar was little changed against a basket of currencies while gold lost ground. A lingering fuel glut overhang hurt oil prices.

 

 

MARKET HIGHLIGHTS

 

ADMA Biologics announced its financial results for the quarter ended September 30, 2016.  The consolidated net loss for the third quarter ended September 30, 2016 was $4.3 million, or $(0.34) per share, as compared to a consolidated net loss of $5.1 million, or ($0.48) per share, for the third quarter ended September 30, 2015.  At September 30, 2016, ADMA had cash, cash equivalents and short-term investments of $18.9 million, as compared to $16.8 million at December 31, 2015.

 

BioTime announced that Co-Chief Executive Officer Michael D. West, Ph.D., is participating at two conferences during the week of November 14. On Monday, November 14, Dr. West is serving as Chairperson for the Cell Therapy Track at Terrapinn’s World Precision Medicine Congress USA 2016.  On Thursday, November 17, Dr. West is delivering a talk on building a successful biotechnology company at the Young Jewish Professionals (YJP) Life Sciences & Pharmaceuticals CEO Symposium, November 16 and 17 in Midtown Manhattan.

 

BioLife Solutions reported financial results for the three and nine months ended September 30, 2016.  The net loss attributable to BioLife was $1.0 million and $1.1 million for the three month periods in 2016 and 2015, respectively.  The Company ended the third quarter with $1.4 million in cash.

 

Pernix Therapeutics Holdings announced financial results for the three and nine months ended September 30, 2016.  For the three months ended September 30, 2016, net revenues were $41.5 million.  Net loss was $26.4 million for the three months ended September 30, 2016.  As of September 30, 2016, the Company had total liquidity of $39.9 million, consisting of $28.5 million of cash and approximately $11.4 million available to draw under its $50.0 million revolving credit facility.

 

STRATA Skin Sciences reported financial results for the quarter ended September 30, 2016.  Third quarter revenues were $7.7 million.  Net loss for the third quarter of 2016 was $1.5 million or ($0.14) per diluted share, compared with a net loss for the third quarter of 2015 of $12.2 million or ($1.29) per diluted share.  As of September 30, 2016 the Company had cash, cash equivalents and short-term investments of $3.0 million, compared with $3.3 million as of December 31, 2015.

 

CymaBay Therapeutics will host a Key Opinion Leader breakfast on the treatment of Primary Biliary Cholangitis (PBC) at 8:00 am Eastern Time on Wednesday, November 16 in New York City.

 

Advaxis announced new data highlighting the potential therapeutic benefit of Advaxis’ lead immunotherapy candidate, axalimogene filolisbac (AXAL), both as a monotherapy and in combination with antibody-based immunotherapies in multiple patient populations with HPV+ cancers.  These data will be presented at the Society for Immunotherapy of Cancer (SITC) Annual Meeting & Associated Programs this week in National Harbor, MD.  Preliminary results from Part A dose escalation showed that there were no dose limiting toxicities observed, and the safety profile was consistent with previous findings for both AXAL and durvalumab.

 

TRACON Pharmaceuticals presented updated data from the Company’s Phase 1b/2 study of TRC105 and Votrient (pazopanib) at the Connective Tissue Oncology Society (CTOS) annual meeting taking place in Lisbon, Portugal.

 

Ignyta will make a presentation at the Jefferies 2016 London Healthcare Conference on November 17.

 

Aurinia Pharmaceuticals will present a corporate overview of the Company at the Stifel 2016 Healthcare Conference on Wednesday, November 16.

 

Achaogen will be presenting at the Stifel Healthcare Conference on Wednesday, November 16.

 

PTC Therapeutics announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has recommended the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.

 

Genocea Biosciences presented new findings supporting the potential of ATLAS, the Company’s proprietary rapid antigen identification screening system, to identify clinically meaningful personalized neoantigens that could guide development of neoantigen vaccines.

 

Ultragenyx Pharmaceutical announced that is has withdrawn its conditional MAA from the EMA for Aceneuramic Acid Prolonged Release (Ace-ER) for the treatment of adult patients with GNE Myopathy.

 

Alexion Pharmaceuticals announced that researchers presented new longer-term data from an ongoing, open-label extension of the pivotal Phase III ARISE trial of Kanuma (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease. Two-thirds of patients treated with Kanuma for 52 weeks had a reduction in liver fibrosis stage from baseline, as measured by Ishak score. Moreover, half of patients achieved at least a 2-stage reduction, including five patients who had fibrosis at baseline and one who had cirrhosis at baseline. Reduction of liver fibrosis stage was accompanied by sustained improvements in alanine aminotransferase (ALT), LDL cholesterol (LDL-C), and liver fat content through 52 weeks of Kanuma treatment.

 

Corvus Pharmaceuticals announced preliminary clinical safety and efficacy data from the dose-selection phase of its ongoing Phase I/Ib study of CPI-444 as a single agent and in combination with Genentech’s Tecentriq (atezolizumab), a fully humanized monoclonal antibody targeting protein programmed cell death ligand 1 (PD-L1). CPI-444 is a selective and potent inhibitor of the adenosine A2A receptor.

 

Amicus Therapeutics announced that data from the pivotal Phase III Study 012 (ATTRACT) evaluating the efficacy and safety of the oral pharmacological chaperone migalastat compared with Enzyme Replacement Therapy (ERT) in individuals with Fabry disease were published online in the Journal of Medical Genetics.

 

AbbVie announced high SVR(12) rates with 8 weeks of treatment with its investigational, pan-genotypic regimen of glecaprevir (ABT-493)/pibrentasvir (ABT-530) (G/P) across all major chronic HCV genotypes. In more than 700 genotype 1-6 (GT1-6) chronic HCV infected patients without cirrhosis and who are new to treatment, 97.5 percent achieved sustained virologic response at 12 weeks post treatment (SVR(12) ), regardless of baseline viral load. The rate of virologic failure was 1%.

 

Flexion Therapeutics announced that its lead investigational product candidate Zilretta demonstrated clinically and statistically significant improvements in an analysis of Phase III trial data conducted in patients with osteoarthritis (OA) of the knee.

 

CytRx presented a poster with updated interim results from its on-going Phase Ib/II trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas at the 2016 Annual Meeting of the Connective Tissue Oncology Society (CTOS) being held in Lisbon, Portugal.

 

Intercept Pharmaceuticals announced results from three new exploratory analyses of the Phase III POISE trial of Ocaliva (obeticholic acid) in patients with primary biliary cholangitis.

 

MyoKardia announced an exploratory digital health substudy that is under way as part of the Company’s PIONEER-HCM trial of MYK-461 in symptomatic, obstructive hypertrophic cardiomyopathy (oHCM) patients.

 

AstraZeneca announced that results from the international, multicenter ChildHood Asthma Safety and Efficacy (CHASE) 3 Phase III study showed that SYMBICORT (budesonide/formoterol fumarate dihydrate) Inhalation Aerosol 80/4.5 micrograms significantly improved lung function in pediatric patients between 6 to 12 years of age with asthma versus budesonide 80 micrograms, demonstrating its appropriateness as step-up therapy in this patient population.

 

Gilead Sciences announced that the CHMP, the scientific committee of the EMA, has adopted a positive opinion on the company’s MAA for Vemlidy (tenofovir alafenamide, TAF) 25 mg, an investigational, once-daily tablet for the treatment of chronic HBV infection in adults and adolescents. The data included in the application support the use of TAF in treatment-naïve and treatment-experienced adults and adolescents with HBeAg-negative and HBeAg-positive HBV infection.

 

Sanofi announced that the CHMP of the EMA has adopted a positive opinion for the marketing authorization of Suliqua, the once-daily titratable fixed-ratio combination of basal insulin glargine 100 Units/mL and GLP-1 receptor agonist lixisenatide. CHMP recommended the use of Suliqua in combination with metformin for the treatment of adults with Type II diabetes mellitus to improve glycemic control when this has not been provided by metformin alone or metformin combined with another oral glucose lowering medicinal product or with basal insulin.

 

Novo Nordisk announced that the CHMP, under the EMA, adopted a positive opinion for the use of Fiasp(fast-acting insulin aspart), recommending marketing authorisation for the treatment of adults with Type I and Type II diabetes.

 

Retrophin announced it has reached an agreement with the FDA under the SPA process for a Phase III clinical trial evaluating RE-024, the Company’s novel investigational replacement therapy, for the treatment of pantothenate kinase-associated neurodegeneration (PKAN). The SPA indicates concurrence by the FDA that the design of the pivotal trial can adequately support a NDA seeking U.S. approval of RE-024 for the treatment of PKAN. The Company plans to initiate this trial before year-end 2016.

 

Capricor Therapeutics announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications. Based on preclinical data in DMD models that show significant improvement in skeletal, including diaphragmatic, muscle function with CAP-1002 (allogeneic cardiosphere-derived cells), Capricor is designing a clinical trial to evaluate the potential ability of CAP-1002 to benefit skeletal muscle function in boys and young men with DMD. In the planned study, the medication will be given by systemic intra-vascular administration. This trial is expected to begin in 2017 subject to regulatory approval, and is intended to enroll people with DMD irrespective of their mutation or ambulatory status.

 

Bristol-Myers Squibb entered into a five-year research collaboration with the Johns Hopkins University. The collaboration is designed to identify mechanisms of response and resistance in patients whose cancer is being treated with checkpoint inhibitor-based immunotherapies, including Opdivo (nivolumab) monotherapy, or Opdivo in combination with Yervoy (ipilimumab) or other investigational immunotherapies. Under the collaboration, Bristol-Myers Squibb and Johns Hopkins’ scientists will launch an interdisciplinary research program that will study patient tumor samples in four primary research areas: characterization of tumor antigens and tumor antigen-specific T-cells, multifaceted profiling of the tumor microenvironment, assessment of microbiome components that modulate systemic anti-tumor immunity, and elucidation of novel tumor and immuno-metabolism factors that modify responsiveness to immunotherapy.

 

Bristol-Myers Squibb announced that the FDA has approved Opdivo (nivolumab) injection, for intravenous use, for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy. Opdivo is the first and only Immuno-Oncology treatment proven in a Phase III trial to significantly extend overall survival (OS) for these patients. In oncology clinical trials, OS is considered the gold standard primary endpoint to evaluate the outcome of any therapy.

 

Reuters reported that Valeant Pharmaceuticals may change its name to help boost its reputation, board member and top investor William Ackman told CNBC. A name change would be the latest effort by the company to break from its past, after hiring a new chief executive, overhauling the board and exploring billions of dollars in asset sales to pay down a $30 billion debt load.

 

Cognition Therapeutics announced that the first Alzheimer’s patient has been dosed in a Phase Ib clinical trial of CT1812 in mild to moderate Alzheimer’s disease.

 

BeiGene presented updated clinical data from an ongoing Phase I study of anti-PD-1 antibody BGB-A317 in patients with advanced solid tumors in a poster presentation at the Society for Immunotherapy of Cancer (SITC) 31st Annual Meeting held in National Harbor, Maryland. BGB-A317 is an investigational humanized monoclonal antibody against the immune checkpoint inhibitor PD-1. The updated clinical data suggest that BGB-A317 is well-tolerated with anti-tumor activity observed across multiple tumor types.

 

Eagle Pharmaceuticals has initiated the rolling submission of its NDA and filed the first part of the application to the FDA for Ryanodex for injectable suspension for the treatment of exertional heat stroke (EHS), an investigational new indication for the product. The rolling submission allows completed portions of an NDA to be submitted, which may then be reviewed by the FDA on an ongoing basis. The FDA had previously granted Fast Track Designation for the development of Ryanodex for the treatment of EHS, which allows for submission of parts of an NDA application.

 

Eagle Pharmaceuticals signed a definitive agreement to acquire Arsia Therapeutics, an early-stage biotechnology firm with proprietary viscosity-reducing technology and formulation know-how. The acquisition will mark Eagle’s entry into biologics, the fastest growing sector of the pharmaceuticals market, and will allow the Company to apply its proven market strategy to offer "biobetter" formulations, and to aid in the rapid development of novel biologics. The closing of the acquisition is expected to occur within the next week, subject to the satisfaction of various customary closing conditions.

 

Pfizer announced a major expansion of its humanitarian assistance program enabling broader access to its vaccine, Prevenar 13, in humanitarian emergency settings by offering its new multi-dose vial (MDV) at what will be the lowest prevailing global price, currently $3.10 per dose. In addition, given the acute need for aid on the ground, Pfizer will donate all sales proceeds for the first year of this program to humanitarian groups undertaking the difficult work of reaching vulnerable populations in emergency settings. Today’s announcement of a new and specific pricing tier for civil society organizations (CSOs) working in emergency settings builds on Pfizer’s longstanding support for humanitarian emergency relief around the world.

 

Five Prime Therapeutics announced that a poster featuring preclinical data related to its tetravalent anti-GITR agonist antibody, FPA154, was presented today at the Society for Immunotherapy of Cancer (SITC) Annual Meeting in National Harbor, Maryland.

 

Aphria entered into an agreement with Clarus Securities, on behalf of a syndicate of underwriters, pursuant to which the Underwriters have agreed to purchase, on a "bought deal" basis, 8,750,000 Common Shares of the Company at a price of C$4.00 per Common Share for aggregate gross proceeds to the Company of C$35,000,000.

 

Pacira Pharmaceuticals introduced a revolutionary new virtual reality simulation intended to provide clinicians with an immersive, hands-on training experience to reinforce the recommended technique for administering EXPAREL (bupivacaine liposome injectable suspension) in total knee arthroplasty (TKA) based on the infiltration protocol developed as part of the company’s ongoing Phase IV TKA study. This cutting-edge training tool utilizes virtual reality and real-time haptic feedback technology to create a realistic surgical experience in a risk-free computer-generated environment.

 

Cipher Pharmaceuticals announced that Stephen L. Lemieux, CFO and Secretary of the Company, has been named Interim CEO, replacing Shawn O’Brien, who is pursuing other opportunities. Cipher has commenced a formal search process for a new CEO.

 

 

ANALYST RECOMMENDATIONS

 

Following Blueprint Medicines’ earnings, Raymond James analyst Christopher Raymond increased his price target to $35 from $32; Canaccord analyst Arlinda Lee increased her price target to $35 from $26; Goldman analyst Terence Flynn increased his price target to $35 from $19.

 

Following Avexis’ earnings, BMO analyst Ian Somaiya increased his price target to $85 from $80.

 

Following Seres Therapeutics’ earnings, Goldman analyst Terence Flynn decreased his price target to $8 from $9.

 

Maxim analyst Jason McCarthy downgraded ImmunoCellular to “hold” from “buy” and removed his price target, citing the delayed timeline for ICT-107 Ph3 trial due to slower than expected enrollment.

 

Following Endo International’s earnings, BMO analyst Gary Nachman decreased his price target to $20 from $22; Mizuho analyst Irina Koffler decreased her price target to $25 from $29.

 

Needham analyst Alan Carr downgraded Cempra to “hold” from “buy,” citing minimal stock movement up from current levels until either turnover in management and/ or regulatory approval.

 

Goldman analyst Salveen Richter initiated coverage of Acadia Pharmaceuticals with a “neutral” and a $28 price target, citing their sole drug Nuplazid is the first approved drug for Parkinson’s disease (PD) psychosis in the US and KOLs anticipate it will emerge as standard-of-care given the favorable efficacy/safety profile.

 

Jefferies analyst Jeffrey Holford revised his price target of the following companies: Abbott decreased to $48 from $49.50; AstraZeneca decreased to 5,600p from 6,000p; Bayer AG increased to €102 from €99; Bristol-Myers increased to $60 from $58; GlaxoSmithKline decreased to 1,850p from 1,950p; Johnson & Johnson increased to $119 from $115; Merck decreased to $59 from $65; Pfizer decreased to $34 from $36; Roche decreased to CHF270 from CHF275.  

 

BMO analyst Matthew Luchini initiated coverage of bluebird bio with a “market perform” rating and $61 price target, citing recently presented preclinical Process 2 manufacturing data are encouraging, upside in BLUE is dependent on confirmation that recently implemented protocol amendments (including Process 2) will translate into better clinical outcomes in sickle cell disease and β0/ β0 TDT, which is not expected until mid-2017 (EHA meeting: June 22-25) at the earliest.

 

BMO analyst Matthew Luchini initiated coverage of Alder Biopharmaceuticals with an “outperform” rating and $42 price target, citing Alder’s lead drug ALD403 is well positioned to become a blockbuster treatment for migraine prevention.