BioShares Biotechnology Clinical Trials (BBC): $19.05, -$0.07, -34.4% YTD

BioShares Biotechnology Products (BBP): $33.45, -$0.05, 8.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed to slightly lower and the dollar edged lower as markets cautiously awaited the Federal Reserve’s policy statement later in the day. Producer prices, retail sales, industrial production and capacity utilization are on the economic calendar. European shares retreated from an 11-month high, with Actelion’s stock slumping after Johnson & Johnson ended discussions over a potential deal. Asian markets closed mixed. Oil prices fell, following a reported rise in U.S. crude inventories and an estimate that OPEC may have produced more crude in November than previously thought. Gold edged higher.

 

 

MARKET HIGHLIGHTS

 

Genticel announced the final results (24-month time point) of the phase 2 trial of its HPV16/18 immunotherapeutic candidate, GTL001.  The final data at month 24 showed no statistical differences in viral clearance rates between the GTL001 and placebo groups. No consistent statistical differences between groups were demonstrated in any of the secondary endpoints (confirmed and sustained clearance) over the 2-year duration of the trial. The incidence of subjects progressing to high-grade lesions was identical in both groups. A significant increase in anti-CyaA1 antibodies following treatment was observed in the GTL001 group, but not in the placebo group. The significant increase in anti-CyaA titers in the treated group persisted during the entire course of the study. Genticel announced that it has concluded its HPV therapeutics development program.

 

Prothena Corporation announced the publication of previously disclosed clinical results from a Phase 1 single ascending dose study in healthy volunteers of PRX002, a monoclonal antibody for the potential treatment of Parkinson’s disease and other related synucleinopathies, in the peer-reviewed journal Movement Disorders. PRX002, also known as RG7935, is the focus of a worldwide collaboration between Prothena and Roche.

 

Achaogen announced the pricing of an underwritten public offering of 6,500,000 shares of its common stock at a price to the public of $13.50 per share, for gross proceeds of approximately $87,750,000 before deducting underwriting discounts and commissions and offering expenses payable by Achaogen. In addition, Achaogen has granted the underwriters of the offering a 30-day option to purchase up to an additional 975,000 shares of common stock at the public offering price, less underwriting discounts and commissions.  All of the shares are being offered by Achaogen.

 

Alcobra announced today that it recently held a productive face-to-face meeting with the Division of Psychiatry Products (DPP) of the U.S. Food and Drug Administration (FDA) to discuss their decision to place a full clinical hold on the development programs of Metadoxine Extended Release (MDX), and paths to resume human clinical trials with MDX. The Company expects to provide further details regarding the MDX regulatory path upon receipt of final meeting minutes from the DPP in the coming weeks.

 

DelMar Pharmaceuticals announced that it received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for United States Patent Application Serial No. 13/933,844 entitled, "Improved Analytical Methods for Analyzing and Determining Impurities in Dianhydrogalactitol."

 

Ohr Pharmaceutical announced that it has closed its previously announced public offering of common stock and warrants.  The Company sold 3,885,000 shares of common stock at a price to the public of $2.00 per share. Investors also received series A warrants to purchase up to an aggregate of 1,942,500 shares of common stock with an exercise price of $2.75 per share and series B warrants to purchase up to an aggregate of 3,885,000 shares of common stock with an exercise price of $3.00 per share. The series A warrants are immediately exercisable and have a term of five years and the series B warrants are immediately exercisable and have a term of six months.  Gross proceeds to the Company from the sale of the shares were approximately $7,770,000, excluding any proceeds from the exercise of warrants.

 

BrainStorm Cell Therapeutics announced that new data from the Company’s Phase II study of NurOwn in ALS were presented by lead investigator Dr. James Berry at the 27th International Symposium on ALS/MND in Dublin, Ireland. In the Phase II trial, levels of neurotrophic factors and inflammatory markers were measured in cerebral-spinal fluid (CSF) samples collected from patients. In the samples of those patients treated with NurOwn, a statistically significant increase in levels of neurotrophic factors VEGF, HGF and LIF was observed from pre- to post-transplantation. There was also a statistically significant reduction in inflammatory markers (MCP-1 and SDF-1) over this period, in patients treated with NurOwn(R) and this was not observed in the placebo group.

 

BioTime announced that it has achieved an important patient enrollment milestone in its Renevia® pivotal trial in Europe. The company now has more than 50 patients in the trial, keeping it on track with the anticipated forecast for top-line data by the middle of 2017.

 

BioDelivery Sciences International announced that Peter Ginsberg has joined the company as Vice President of Business Development.  Mr. Ginsberg joins BDSI with over 25 years of business development and other closely related experience.

 

MediciNova announced that the Company will be added to the NASDAQ Biotechnology Index (NASDAQ: NBI) effective prior to the market open on Monday, December 19, 2016. 

 

Intercept Pharmaceuticals announced that the European Commission has granted conditional approval for Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. Ocaliva is a potent and selective agonist of the farnesoid X receptor (FXR), which is expressed at high levels in the liver and intestine and thought to be a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

 

Vericel announced that the FDA has approved MACI (autologous cultured chondrocytes on porcine collagen membrane) for the repair of symptomatic single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults.

 

Syndax Pharmaceuticals entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health, for intramural preclinical and clinical research. As part of the CRADA, Syndax will collaborate with the Laboratory of Tumor Immunology and Biology at the NCI to evaluate the therapeutic benefit of entinostat, an oral, Class 1 selective small molecule histone deacetylase (HDAC) inhibitor in development for the treatment for multiple cancers, and SNDX-6352, a humanized IgG4 monoclonal antibody with high affinity against colony-stimulating factor 1 receptor (CSF-1R) in development as a treatment of neoplastic diseases. This CRADA complements the existing CRADA in place with the NCI’s Cancer Therapy Evaluation Program to evaluate entinostat in clinical trials.

 

Cidara Therapeutics announced that data from preclinical and clinical studies evaluating the company’s novel echinocandin antifungal CD101 appear in the December 5 online issue of the journal Antimicrobial Agents and Chemotherapy (AAC). These latest findings add to the growing body of published scientific evidence that demonstrate the unique attributes of Cidara’s lead antifungal drug candidate and showcase the compound’s strategic discovery and rapid development.

 

Cellectar Biosciences announced that the USPTO has issued patent allowances covering method of use for radiotherapy with the company’s lead compound, CLR 131, as well as CLR 125, another of the company’s pipeline products using its proprietary phospholipid drug conjugate ("PDC") to deliver a radiotherapeutic.

 

KemPharm announced the results of its Phase I proof-of-concept clinical trial of KP415, KemPharm’s prodrug, extended release d-threo-methylphenidate (d-MPH) product candidate for the treatment of ADHD. Data from the study indicated that KP415 demonstrated pharmacokinetic (PK) properties that produced earlier d-MPH exposure followed by a slower extended release of d-MPH relative to the comparator, Concerta.

 

bluebird bio announced treatment of the first patient in Northstar-2, the Phase III study of its LentiGlobin drug product in patients with transfusion-dependent β- thalassemia (TDT) and non-β0/β0 genotypes. This study will use LentiGlobin drug product manufactured with the addition of transduction enhancers intended to increase the drug product vector copy number and percent of cells transduced. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study.

 

Johnson & Johnson confirmed it has ended discussions with Actelion Pharmaceuticals regarding a potential transaction. Johnson & Johnson was not able to reach an agreement that it believed would create adequate value for its shareholders.

 

IntelGenx and RedHill Biopharma announced the signing of an exclusive license agreement with Pharmatronic Co. for the commercialization of RIZAPORT in the Republic of Korea. RIZAPORT is a proprietary oral thin film formulation of rizatriptan for the treatment of acute migraines.

 

Concert Pharmaceuticals announced the Phase I single and multiple ascending dose trial evaluating CTP-543 in healthy volunteers has been completed. Concert is developing CTP-543 for the treatment of alopecia areata, an autoimmune disorder in which the immune system attacks hair follicles, resulting in patchy or complete hair loss. CTP-543 is a deuterium-modified analog of ruxolitinib, a Janus Kinase (JAK) inhibitor that is commercially available under the brand name Jakafi(R) for the treatment of myelofibrosis and for polycythemia vera. The Phase 1 program was designed to determine the safety, tolerability and pharmacokinetics of CTP-543 as well as determine doses for the planned Phase IIa clinical trial. Given the overall exposure and safety parameters observed, the Company has selected four doses (4, 8, 12 and 16 mg BID) to explore in its Phase 2a trial. The Phase IIa clinical trial is expected to begin in 1Q17.

 

Argos Therapeutics announced that it has entered into a strategic research agreement with Personalis, a precision medicine company, focused on genomics solutions for immuno-oncology, cancer, and genetic disease.

 

CytomX Therapeutics announced that the FDA has cleared the IND application for its lead program, CX-072, a wholly-owned PD-L1-targeting Probody therapeutic for the treatment of cancer. The company plans to immediately initiate the study and open clinical sites to support patient enrollment.

 

Protalix BioTherapeutics received a letter from Fundação Oswaldo Cruz (Fiocruz), an arm of the Brazilian Ministry of Health, detailing intended purchases by the Brazilian Ministry of alfataliglicerase to treat Gaucher patients in Brazil. The letter requests three shipments of alfataliglicerase; the first shipment to be made in the middle of 2017, and the last at the end of 2017. The Company estimates total revenues from these shipments to be approximately $24 million in aggregate.

 

Galena Biopharma announced that the NeuVax (nelipepimut-S) Phase II clinical trial entitled VADIS: Phase II trial of the Nelipepimut-S Peptide VAccine in Women with DCIS of the Breast is now open for enrollment and screening patients. The trial is being run in collaboration with the National Cancer Institute (NCI) and The University of Texas MD Anderson Cancer Center Phase I and II Chemoprevention Consortium.

 

Genocea Biosciences announced a research collaboration with Checkmate Pharmaceuticals. The goal of the collaboration is to characterize the T cell responses of patients in the ongoing Checkmate Phase 1b clinical trial and to identify antigens associated with protective T cell responses.

 

VistaGen Therapeutics announced it has signed an exclusive sublicense agreement with BlueRock Therapeutics, a stem cell research company established by Bayer AG and Versant Ventures, for VistaGen’s rights to certain proprietary technologies relating to the production of cardiac stem cells for the treatment of heart disease. VistaGen licensed exclusive rights of the cardiac stem cell technologies from University Health Network (UHN), Canada’s largest research hospital, pursuant to a strategic research agreement with UHN and distinguished UHN researcher, Dr. Gordon Keller, Director of UHN’s McEwen Centre for Regenerative Medicine (McEwen Centre). Under the sublicense agreement, VistaGen will receive an upfront cash payment of $1.25 million, as well as potential future milestone payments and royalties.

 

Aurora Cannabis and Radient Technologies signed a memorandum of understanding ("MOU") to evaluate an exclusive partnership for the Canadian market with regard to the joint development and commercialization of superior and standardized cannabinoid extracts. As part of the MOU, Aurora will, subject to certain conditions precedent being satisfied, invest up to $2 million into Radient by means of a convertible debenture (the "Debenture").

 

Natera announced a collaboration with Dr. Laura van ‘t Veer and Dr. Laura Esserman of UCSF and QuantumLeap Healthcare Collaborative to participate in the I-SPY 2 TRIAL (Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging And moLecular Analysis 2), a multi-center study evaluating the safety and efficacy of investigational therapies combined with neoadjuvant treatment in women with newly diagnosed, locally advanced breast cancer. In this trial, Natera will be analyzing blood samples at various points throughout patient treatment to evaluate the effectiveness of liquid biopsy in monitoring tumor burden, treatment response, and residual disease, as compared to traditional imaging methods.

 

Medivir AB announced that MIV-323 has been selected as a candidate drug (CD) from its fusion inhibitor project for the treatment of respiratory syncytial virus (RSV) infection, and has now entered non-clinical development.

 

Abide Therapeutics announced initiation of dosing in a Phase Ib study to evaluate the effects of ABX-1431, a first-in-class investigational monoacylglycerol lipase (MGLL) inhibitor, on gastric accommodation in patients with functional dyspepsia (FD), a gastrointestinal disorder characterized by altered gut motility and pain.

 

Zenith Capital Corp. announced that its wholly owned subsidiary, Zenith Epigenetics Ltd. has initiated a Phase Ib clinical trial in metastatic Castration Resistant Prostate Cancer ("mCRPC") patients, dosing ZEN-3694 in combination with enzalutamide.  In this Phase Ib trial, Zenith Epigenetics will be evaluating the safety, pharmacokinetics, and efficacy of ZEN-3694 in combination with, enzalutamide (Xtandi) in mCRPC patients that have progressed on first-line standard-of-care anti-androgen therapy. The trial will encompass a dose escalation phase and an expansion phase, designed to potentially show early proof-of-concept. The trial also includes a broad translational program, measuring target modulation as well as evaluating potential pharmacodynamic biomarkers for identifying patients who may benefit the most from the combination treatment.

 

Invitae has seen a significant increase in genetic testing for prostate cancer since the publication of landmark data showing testing may be underutilized when screening for and treating the second most common cancer in men. The company’s panel is the most comprehensive and affordable genetic test available to urologists and oncologists who treat men with prostate cancer, establishing the company as a new leader in prostate cancer testing.

 

Blueprint Medicines announced the closing of its previously announced underwritten public offering of 5,750,000 shares of its common stock at a public offering price of $25.00 per share, including the exercise in full by the underwriters of their option to purchase additional shares of common stock. Blueprint Medicines estimates net proceeds from the offering will be approximately $134.5 million, after deducting underwriting discounts and commissions and estimated offering expenses. Goldman Sachs, Morgan Stanley and Cowen acted as joint book-running managers for the offering. JMP Securities and Wedbush PacGrow acted as co-managers for the offering.

 

Organovo Holdings announced an exclusive distributor agreement with Cosmo Bio Co., Ltd. for its NovoView Preclinical Services in Japan. CosmoBio and Organovo will work with CosmoBio’s Drug Discovery Services (DDS) team to gain direct access to customers and support projects throughout the Japanese biopharmaceutical industry.

 

RTI Surgical announced that Robert P. Jordheim, EVP and CFO of RTI, has been named interim CEO. He succeeds Brian K. Hutchison, who informed the RTI board of directors last August of his decision to retire from his position as president and CEO and step down from the company’s board of directors. The transition will be completed December 17, 2016.

 

Neurovance announced the appointment of Brian Goff to the newly created position of COO and to the Neurovance Board of Directors. Additionally, Executive Board Chairman, Jeff Bailey assumes the role of CEO, and Anthony McKinney is named Founder and Chief Development Officer, to further leverage his experience with CTN. Mr. McKinney will continue in his role as CEO of the Neurovance sister company, Euthymics.

 

 

ANALYST RECOMMENDATIONS

 

Cantor analyst Mara Goldstein adjusted her rating of Sunesis to “neutral” from “speculative buy,” citing the firm’s new rating system; in the new rating system, neutral is consistent with speculative buy.

 

Raymond James analyst Christopher Raymond revised his rating of the following companies: Sage Therapeutics upgraded to “strong buy” from “outperform;” Proteon downgraded to “market perform” from “strong buy” and removed his price target.

 

Jefferies analyst Carmen Augustine initiated coverage of Blueprint Medicines with a “buy” rating and $40 price target, citing BPMC is well positioned to build a balanced pipeline of proprietary & partnered products for cancer/rare disease.

 

Leerink analyst Puneet Souda decreased his price target of Illumina to $114 from $115, citing lower expected instrument sales and continued weak HiSeq utilization heading into 2017.

 

Jefferies analyst Gena Wang decreased her price target of Sarepta to $32 from $46, citing stronger payer pushback and slower uptake/launch for Exondys 51.

 

Following Proteon Therapeutics’ announcement of the top-line results from the PATENCY-1 pivotal Phase III trial of vonipanitase in CKD which did not meet its primary endpoint of improved primary unassisted patency compared to placebo, Maxim analyst Jason McCarthy decreased the price target to $6 from $19; Stifel analyst Stephen Willey downgraded the stock to “hold” from “buy” and decreased his price target to $3 from $19; Cowen analyst Boris Peaker downgraded the stock to “market perform” from “outperform;” JMP analyst Michael King downgraded the stock to “market perform” from “market outperform.”

 

JP Morgan analyst Michael Weinstein decreased his price target of Johnson & Johnson to $122 from $130, citing pharmaceutical script trends and contracting competitor multiples.

 

Following aTyr Pharma’s announcement that data from three studies in FSHD and LGMD 2B released on Tuesday demonstrated some efficacy signal, but not quite sufficient yet to inform the design of the next study, William Blair analyst Katherine Xu decreased her price target to $4 from $8.