BioShares Biotechnology Clinical Trials (BBC): $17.57, -$0.66

BioShares Biotechnology Products (BBP): $29.05, -$0.29

 

 

MARKET COMMENTARY

 

U.S. stock index futures were little changed and pointed to the longest losing streak for the S&P 500 since the 2008 financial crisis as election uncertainty lingered amidst a busy earnings and economic calendar. The volatility index rose for an eighth straight day for its longest steak in three years. Factory orders, ISM’s non-manufacturing PMI data, Markit’s services PMI, and challenger layoffs are due later in the day. Activision, Starbucks, Motorola Solutions and Consolidated Edison are expected to report results after the closing bell. European shares looked set to snap their longest streak of losses in more than two years as good corporate results, particularly from eurozone banks, helped support the indexes. Asian stocks closed mostly lower. The dollar edged lower and gold slipped. Oil rose after news of an attack on a Nigerian oil pipeline raised concerns about supply disruptions.

 

 

MARKET HIGHLIGHTS

 

Aurinia Pharmaceuticals announced its plans for a single Phase III clinical trial for voclosporin in the treatment of lupus nephritis (LN). Pursuant to its recent End of Phase II meeting with the U.S. Food & Drug Administration (FDA) Division of Pulmonary, Allergy and Rheumatology Products, Aurinia believes this Phase III clinical trial whose design is consistent with the ongoing AURA study, will support a New Drug Application (NDA) submission.  The Phase III AURORA trial will be a global 52-week double-blind, placebo controlled study of approximately 320 patients. The Company is finalizing the study protocol and regulatory submissions and in parallel is working on site selection with trial initiation anticipated in Q2 2017.

 

Ignyta announced the details of a poster presentation at the Connective Tissue Oncology Society (CTOS) 21st Annual Meeting being held in Lisbon, Portugal. The poster session will be held on Friday, November 11, 2016.

 

Catalyst Biosciences announced financial results for the third quarter ended September 30, 2016.  Net loss for the three months ended September 30, 2016 was $4.8 million, or ($0.40) per basic and diluted share, compared to $5.1 million, or ($0.93) per basic and diluted share for the prior year period.  Cash, cash equivalents and short-term investments as of September 30, 2016 were $19.5 million. The Company believes that its existing capital resources will be sufficient to meet its projected operating requirements for at least the next 12 months.

 

Innate Pharma SA announced its revenues and cash position for the first nine months of 2016.  Cash, cash equivalents and financial instruments of the Company amounted to €239.6 million at September 30, 2016, including current and non-current financial assets (€243.6 million at June 30, 2016). At the same date, its financial liabilities amounted to €5.6 million (€4.1 million at June 30, 2016).

 

Aeglea BioTherapeutics will present preclinical results from studies evaluating AEB3103, which targets a vulnerability of cancer metabolism for the treatment of hematological malignancies, at the 58th Annual American Society of Hematology (ASH) Meeting being held December 3-6, 2016 in San Diego, California.

 

Prothena Corporation plc announced that clinical data from its NEOD001 Phase 1/2 dose-escalation and expansion study will be highlighted in two oral presentations, and data highlighting quality of life measures in patients with AL amyloidosis will be featured in three posters at the 58th Annual American Society of Hematology (ASH) Meeting to be held December 3-6, 2016, in San Diego, CA.

 

Kamada will release financial results for the third quarter ended September 30, 2016 prior to the open of the U.S. financial markets on Thursday, November 10, 2016.

 

Foamix Pharmaceuticals will provide a corporate overview at the 25th Annual Credit Suisse Healthcare Conference, being held at the Phoenician Hotel in Scottsdale, Arizona, November 7-8, 2016.

 

BioLife Solutions announced that the Company’s third quarter 2016 financial results will be released on Thursday, November 10, 2016 after the market close.

 

TetraLogic Pharmaceuticals announced that it has entered into an asset purchase agreement with Medivir to sell its SMAC mimetic program, including its clinical stage asset birinapant, and its topical HDAC inhibitor, SHAPE, to Medivir.

 

Novocure announced that United Healthcare will cover Optune as an adjunctive therapy for the treatment of glioblastoma (GBM) when used according to FDA labeled indications and contraindications, effective Jan. 1, 2017. United Healthcare administers medical benefit plans that cover 44.7 million individuals.

 

After yesterday’s close, Alnylam Pharmaceuticals reported 3Q16 GAAP EPS loss of $1.21, which compares to a loss $0.91 for the same period a year ago. The company beat the First Call GAAP EPS mean estimate loss of $1.14. Total revenue for 3Q16 was 13.7 million, which compares to $6.3 million for the same period a year ago. This compares to a mean estimate of $9.1 million. Following Alnylam Pharmaceuticals’ earnings, JP Morgan analyst Anupam Rama decreased his price target to $51 from $49.

 

After yesterday’s close, Neurocrine BioSciences reported 3Q16 GAAP EPS loss of $0.43, which compares to a loss $0.40 for the same period a year ago. The company beat the First Call GAAP EPS mean estimate loss of $0.57. There was no revenue reported for 3Q16, which compares to no revenue for the same period a year ago. This compares to a mean estimate of no revenue. Following Neurocrine’s earnings, JP Morgan analyst Anupam Rama decreased his price target to $64 from $65.

 

After yesterday’s close, Qiagen reported 3Q16 non-GAAP EPS of $0.29, which compares to $0.27 for the same period a year ago. The company beat the First Call non-GAAP EPS mean estimate of $0.28. Total revenue for 3Q16 was $338.7 million, which compares to $314.6 million for the same period a year ago. This compares to a mean estimate of $336.0 million. In other company news, Qiagen announced a series of restructuring initiatives to improve efficiency and profitability through targeted actions planned to be implemented in the fourth quarter of 2016 and in 2017. Based on these initiatives, QIAGEN has revised its adjusted diluted EPS outlook for 2016 to approximately $0.87-0.88 per share at constant exchange rates (CER), but reaffirms its previous guidance for $1.10-1.11 CER per share without these measures. QIAGEN also reaffirms its full-year 2016 sales outlook for 6-7% CER growth. Following Qiagen’s earnings, Evercore analyst Vijay Kumar increased his price target to $28 from $26.50; Barclays analyst Jack Meehan increased his price target to $28 from $27; Goldman analyst Isaac Ro increased his price target to $23 from $21; Bank of America analyst Derik de Bruin upgraded the stock to “neutral” from “underperform” and increased his price target to $26 from $25.

 

After yesterday’s close, PRA Health Sciences reported 3Q16 non-GAAP EPS of $0.64, which compares to $0.52 for the same period a year ago. The company matched the First Call non-GAAP EPS mean estimate of $0.64. Total revenue for 3Q16 was $399.8million, which compares to $345.1 million for the same period a year ago. This compares to a mean estimate of $394.9 million. Following PRA Health Sciences’ earnings, Baird analyst Eric Coldwell increased his price target to $61 from $58.

 

Akorn Incorporated reported 3Q16 non-GAAP EPS of $0.56, which compares to $0.56 for the same period a year ago. The company beat the First Call non-GAAP EPS mean estimate of $0.54. Total revenue for 3Q16 was $284.1 million, which compares to $256.8 million for the same period a year ago. This compares to a mean estimate of $267.1 million.

 

China Biologic reported 3Q16 GAAP EPS of $1.01, which compares to $0.82 for the same period a year ago. The company beat the First Call GAAP EPS mean estimate of $0.86. Total revenue for 3Q16 was $86.5 million, which compares to $78.8 million for the same period a year ago. This compares to a mean estimate of $88.5 million.

 

Radius Health reported 3Q16 GAAP EPS loss of $1.07, which compares to $0.68 loss for the same period a year ago. The company missed the First Call GAAP EPS mean estimate loss of $1.00. There was no revenue reported for 3Q16, which compares to no revenue for the same period a year ago. This compares to a mean estimate of $100,000 of revenue.

 

Omeros announced that it has filed an application for orphan drug designation with the FDA for OMS721 in the treatment of immunoglobulin A (IgA) nephropathy (also known as Berger’s disease). IgA nephropathy leads to end-stage renal disease in up to 40 percent of patients. Orphan drug designation is granted to treatments that target conditions affecting 200,000 or fewer U.S. patients per year. Orphan-designated drugs are eligible for financial incentives and regulatory advantages such as a faster approval process and additional market exclusivity. 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.

 

Bloomberg reported that Mallinckrodt and Japan’s Daiichi Sankyo are considering bids for Depomed, which develops long-term pain delivery technologies, people familiar with the matter said.

 

Horizon Pharma announced that RAVICTI (glycerol phenylbutyrate) Oral Liquid is now available in Canada. In March 2016, Horizon received a Notice of Compliance (NOC) from Health Canada for RAVICTI for use as an adjunctive therapy for chronic management of adult and pediatric patients two years of age and older with Urea Cycle Disorders (UCDs).

 

RedHill Biopharma provided an update on its ongoing Phase III and Phase II clinical studies with BEKINDA for the treatment of acute gastroenteritis and gastritis and for diarrhea-predominant irritable bowel syndrome (IBS-D), respectively.

 

Revance Therapeutics announced initiation of a Phase II placebo-controlled trial of its investigational drug candidate DaxibotulinumtoxinA for Injection (RT002) for the management of plantar fasciitis. This painful affliction, caused by inflammation of the ligament running along the bottom of the foot, is the most common cause of heel pain. Topline clinical results from the Phase II trial are expected in 2017.

 

Sanofi Genzyme, the specialty care global business unit of Sanofi, announced that the first adult patient in the UK has enrolled and been dosed in a pivotal Phase II/III clinical trial named ASCEND for the investigational therapy olipudase alfa. Olipudase alfa is an enzyme replacement therapy being studied for the treatment of non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type B (NPD B). The first patient was dosed at the National Hospital for Neurology and Neurosurgery at the University College of London Hospitals (UCLH).

 

Halozyme Therapeutics announced that the FDA has accepted Genentech‘s Biologics License Application for a subcutaneous formulation of rituximab in multiple blood cancer indications. This is a co-formulation with Halozyme’s proprietary recombinant human hyaluronidase enzyme (ENHANZE platform), approved and marketed under the MabThera SC brand in countries outside the U.S.

 

Argos Therapeutics announced the publication of a Case Report in the Kidney Cancer Journal which details the clinical outcomes of two patients with intermediate risk metastatic renal cell carcinoma who had participated in a Phase II clinical trial of the company’s investigational immunotherapy AGS-003 in combination with sunitinib. These patients have obtained long-term control of metastatic disease that is ongoing seven years after initiation of treatment.

 

ACADIA Pharmaceuticals announced the initiation of ENHANCE-1, a Phase III study to evaluate pimavanserin for adjunctive treatment of schizophrenia in patients with an inadequate response to current antipsychotic therapy. Current antipsychotics approved for schizophrenia primarily target the dopaminergic pathway. As a selective serotonin inverse agonist (SSIA), pimavanserin is a new class of antipsychotic medication with a distinct mechanism of action targeting serotonergic 5-HT(2A) receptors while avoiding activity at dopamine and other receptors commonly targeted by other antipsychotics.

 

BioLineRx disclosed positive Phase IIa correlative data, as well as detailed mechanism-of-action data, for BL-8040, the Company’s leading oncology platform, that will be presented at the 58th American Society of Hematology (ASH) Annual Meeting and Exhibition in San Diego, California, taking place December 3-6, 2016. In a poster titled, "The Selective Anti Leukemic Effect of BL-8040, a Peptidic CXCR4 Antagonist, is Mediated by Induction of Leukemic Blast Mobilization, Differentiation and Apoptosis: Results of Correlative Studies from a PhIIa Trial in Acute Myeloid Leukemia", BioLineRx reports the final correlative results from its Phase IIa trial in acute myeloid leukemia (AML). The trial consisted of 45 AML patients receiving BL-8040 monotherapy on days 1-2, followed by the same dose of BL-8040 plus chemotherapy (Cytarabine) on days 3-7. All patients had poor-risk disease and had been heavily pretreated, with 19% having relapsed after a short first remission (≤12 months), 17% having 2 or more relapses, while 45% were refractory to 1-2 induction treatments.

 

Dimension Therapeutics announced a poster presentation that highlights Dimension’s leadership in the development of improved AAV-based vectors for diverse therapeutic applications. Preclinical in vivo research conducted with Dimension’s collaborators at the University of Pennsylvania (Penn) demonstrates that the choice of capsid, a key component of AAV vectors, has a significant impact on both product efficiency and immunogenicity in preclinical studies relating to liver gene therapy. The poster presentation will take place during the 58th ASH Annual Meeting, which is being held in San Diego, California, December 3-6, 2016.

 

Spark Therapeutics and Pfizer announced that Lindsey A. George, M.D., a hematologist and attending physician at the Children’s Hospital of Philadelphia and lead investigator in the Phase I/II clinical trial of SPK-9001 for treatment of hemophilia B, will present an updated dataset from the ongoing Phase I/II clinical trial of SPK-9001 at the 58(th) American Society of Hematology (ASH) Annual Meeting, to be held Dec. 3-6, 2016, in San Diego. The conference abstract, including a plot of factor IX activity levels (as a % of normal) expressed over time by the first seven participants as of Aug. 4, 2016, was made available today: ASH Abstract. The results are from the first seven participants who received a single administration of investigational SPK-9001 at a dose of 5 x 10(11) vector genomes (vg)/kg body weight. Four of the seven participants had reached greater than 12 weeks post-vector administration at the time of abstract submission; those four participants experienced consistent and sustained factor IX activity levels, with a mean greater than 30% of normal, with no sustained elevation in liver enzyme levels. Spark Therapeutics and Pfizer report that one participant, who had not reached 12 weeks post-vector administration, manifested an immune response to the adeno-associated virus (AAV) capsid, accompanied by a drop in factor IX activity level, and was put on a tapering course of corticosteroids. Despite the immune response and decline in factor IX activity level, this participant has not had any bleeds or required replacement factor. No other participant has required the use of corticosteroids. The companies expect to announce updated trial results at the upcoming ASH Annual Meeting in December. None of the participants received infusions of factor IX concentrates to prevent bleeding events. One precautionary infusion took place in one participant two days after administration of SPK-9001 due to a suspected ankle bleed.

 

Global Blood Therapeutics announced that new data supporting its GBT440 program in sickle cell disease (SCD) will be presented at the 58(th) American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego. The poster presentations will include additional data from the ongoing Phase I/II GBT440-001 trial of GBT440 including dosing of 900 mg per day in SCD patients for up to 6 months, and the metabolism of GBT440 in healthy subjects. The poster presentations will also include the methodology utilized to develop the Patient Reported Outcome (PRO) tool that will be used in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study. Patient enrollment of variant SCD genotypes in the GBT440-001 trial is ongoing, and will support the inclusion of patients with variant genotypes in the HOPE Study.

 

Syros Pharmaceuticals announced that new data on its lead candidate, SY-1425, a selective retinoic acid receptor alpha (RARα) agonist currently in Phase II clinical development in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), will be highlighted in two presentations at the 58th American Society of Hematology (ASH) Annual Meeting and Exhibition taking place December 3-6, 2016, in San Diego. The new preclinical data will demonstrate that SY-1425 produces a biologic response in in vitro models of AML with high levels of RARA gene expression similar to that seen in models of acute promyelocytic leukemia (APL), while having little effect on AML cells with low levels of RARA gene expression. APL is a form of AML with a distinct genetic alteration in the RARA gene. SY-1425 is approved in Japan for the treatment of APL, with a well-established efficacy and safety profile. The consistent biological responses suggest that SY-1425 may provide a clinical benefit for subsets of AML patients whose tumors are driven by high levels of RARA as it does in APL patients. In both diseases, RARα is a key oncogenic driver directly targeted by SY-1425.

 

Pacific Biosciences of California announced that it has filed a complaint with the U.S. International Trade Commission (the “ITC”) against Oxford Nanopore Technologies and its affiliates. The complaint asserts that Oxford Nanopore’s MinION and PromethION devices and associated consumables, reagents, and software, as well as related commercialization activities by Oxford Nanopore and its affiliates, infringe one or more claims of U.S. Patent No. 9,404,146 (the “146 Patent”), owned by Pacific Biosciences.

 

ACETO announced that ACETO, through its wholly-owned subsidiary Rising Pharmaceuticals, signed a definitive product purchase agreement to acquire generic products and related assets of Citron Pharma and Lucid Pharma for a total consideration, prior to a potential earn-out payment, of approximately $412 million, or $332 million net of expected tax benefits.

 

LifeNet Health has acquired the assets of Vivo Biosciences, as part of the organization’s growing emphasis on applying cell-based innovations to the process of pharmaceutical discovery and personalized medicine.

 

Merck announced the publication of research conducted by Merck scientists on the discovery and development of verubecestat, an investigational small molecule inhibitor of the enzyme beta-site amyloid precursor protein cleaving enzyme 1 (BACE1), for the potential treatment of Alzheimer’s disease (AD). The research was published online in the latest edition of the peer-reviewed journal Science Translational Medicine and includes results from the Phase I clinical trials in healthy volunteers and people with AD. The efficacy and safety of verubecestat is currently being evaluated in two pivotal Phase III clinical trials, EPOCH and APECS, for the treatment of mild-to-moderate AD and prodromal AD, respectively.

 

Eisai announced that The National Institute of Health and Care Excellence (NICE) has recommended Halaven (eribulin) for the treatment of locally advanced or metastatic breast cancer in a Final Appraisal Determination published. Eribulin is the first breast cancer treatment to be recommended by NICE in nearly a decade.

 

EnBiotix announced the completion of an asset acquisition of AMP Therapeutics GmbH (“AMPT”), an anti-microbial peptide portfolio company of Boehringer Ingelheim Venture Fund and Novartis Venture Fund. In connection with the acquisition, EnBiotix has established EnBiotix GmbH. EnBiotix has also initiated a collaboration with Leipzig University and AMPT’s scientific co-founder, Professor Ralf Hoffmann, Ph.D.

 

VWR announced the acquisition of two companies, Reliable Biopharmaceutical and BioArra.

 

Santalis Pharmaceuticals announced it has obtained allowance from the FDA to initiate a multi-center, placebo controlled, double blinded, Phase II efficacy and tolerability study for the treatment of mild to moderate atopic dermatitis (AD), also known as eczema. Patients must be 17 years of age or older with a clinically stable diagnosis of atopic dermatitis with a total body surface area (BSA) involvement of not more than 15%. Up to 72 patients will be enrolled to determine preliminary efficacy after 28 days of a twice-a-day treatment using a unique 5% East Indian Sandalwood Oil (EISO) cream formulation.

 

Delivra announced the company has successfully completed its first phase pre-clinical research into transdermal berberine for the prevention and treatment of cardiovascular disease related to high cholesterol and triglyceride levels.

 

Alkermes announced the company has awarded $1 million in funds to non-profit patient and community organizations as part of its newly launched ALKERMES Inspiration Grants program. The competitive grant program was designed to fund innovative and high-impact programs to support people affected by mental health and substance use disorders.

 

Galena Biopharma announced that Stephen F. Ghiglieri has been appointed as the Company’s Executive Vice President and Chief Financial Officer, effective immediately.

 

Immunovaccine announced positive results from preclinical studies completed in collaboration with UConn Health for Immunovaccine’s DPX-NEO program, which is designed to develop patient-specific neoepitope immunotherapies.

 

Conatus Pharmaceuticals announced the initiation of ENCORE-PH (PH for Portal Hypertension), a randomized, double-blind, placebo-controlled, Phase IIb clinical trial evaluating emricasan, the company’s first-in-class, orally-active pan-caspase inhibitor, in approximately 240 patients with compensated or early decompensated liver cirrhosis caused by nonalcoholic steatohepatitis (NASH), and severe portal hypertension confirmed by hepatic venous pressure gradient (HVPG) of ≥12 mmHg at baseline. This trial is designed to evaluate dosing, efficacy and safety of emricasan in NASH cirrhosis as an integral part of the company’s initial registration strategy. Top-line results from the ENCORE-PH clinical trial are expected in 2018.

 

KemPharm announced it has appealed the FDA’s CRL for Apadaz (benzhydrocodone and acetaminophen) through the initiation of the Formal Dispute Resolution Request (FDRR) process. Apadaz is KemPharm’s investigational product candidate that is intended to provide short-term management of acute pain in a product with abuse-deterrent attributes. The CRL was received in response to the KemPharm submission of a NDA for Apadaz last year. KemPharm completed an end-of-review meeting in August 2016 in which the FDA provided the Company with a more complete understanding of its assessment of the NDA.

 

Bio-Path Holdings announced that the EMA granted orphan drug designation to BP1001 for the treatment of acute myeloid leukemia (AML).

 

InVivo Therapeutics Holdings announced that Allegheny General Hospital (AGH) in Pittsburgh, PA, part of Allegheny Health Network, 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. Surgeries will be performed at AGH, which treats more than 24,000 inpatients each year. AGH was the first hospital in the region to receive designation as a Level I Shock Trauma Center, which is the highest designation available.

 

Merus N.V. announced the appointment of John Crowley as CFO. Former CFO, Ms. Shelley Margetson has been elevated to COO.

 

Oncolytics Biotech announced that Brad Thompson Ph.D. has stepped down as President and Chief Executive Officer of Oncolytics effective immediately. The Company’s Chief Operating Officer, Matt Coffey Ph.D., M.B.A., has been appointed Interim President and Chief Executive Officer while the Company conducts a search for Dr. Thompson’s replacement.

 

 

ANALYST RECOMMENDATIONS

 

Deutsche Bank analyst Andrew Peters initiated coverage of the following companies: Prothena with a “buy” rating and $73 price target; Halozyme with a “buy” rating and $12 price target; Alexion with a “buy” rating and $164 price target; Exelixis with a “buy” rating and $17 price target; BioMarin with a “buy” rating and $106 price target; Neurocrine Biosciences with a “buy” rating and $65 price target; Ariad with a “hold” rating and $9.50 price target.

 

Following Alkermes’ earnings, Leerink analyst Paul Matteis decreased his price target to $67 from $70; Cowen analyst Chris Shibutani increased his price target to $65 from $60.

 

Mizuho analyst Irina Koffler upgraded Eagle Pharmaceuticals to “buy” from “neutral” and increased his price target to $78 from $62, citing decision from CMS that will likely raise company’s Bendeka royalty for years.

 

Following Pacira Pharmaceuticals’ earnings, Mizuho analyst Irina Koffler decreased her price target to $46 from $64;Wedbush analyst Liana Moussatos decreased her price target to $89 from $93; Jefferies analyst David Steinberg decreased his price target to $53 from $60.

 

Piper Jaffray analyst Joshua Schimmer initiated coverage of Theravance Biopharma with an “overweight” rating and $42 price target, citing company’s medicinal chemistry expertise, particularly for developing topical therapies against validated targets, is an underappreciated long-term value driver.

 

Following Allergan’s earnings, Leerink analyst Jason Gerberry decreased his price target to $266 from $294; Wells Fargo analyst David Maris decreased his valuation range to $250-$255 from $280-$285; JP Morgan analyst Chris Schott decreased his price target to $300 from $325; Piper Jaffray analyst David Amsellem decreased his price target to $200 from $211; Bernstein analyst Aaron Gal decreased his price target to $275 from $335; Deutsche Bank analyst Gregory Gilbert decreased his price target to $250 from $278; Raymond James analyst Elliot Wilbur decreased his price target to $232 from $267; Barclays analyst Douglas Tsao decreased his price target to $220 from $250; Goldman analyst Jami Rubin decreased her price target to $260 from $300.

 

Following Novocure’s earnings, Deutsche Bank analyst Gregg Gilbert decreased his price target to $15 from $16; Wells Fargo analyst Larry Biegelsen increased his valuation range to $12-$14 from $11-$12.

 

Piper Jaffray analyst Joshua Schimmer decreased his price target of Flex Pharma to $15 from $25, citing recent challenges.