BioShares Biotechnology Clinical Trials (BBC): $19.46, -$0.45

BioShares Biotechnology Products (BBP): $30.15, -$0.33

 

 

MARKET COMMENTARY

 

After the third and final presidential debate, U.S. stock index futures pointed to a higher open as investors shifted focus to a flurry of economic data releases scheduled later in the day. Initial claims, existing home sales, leading indicator and Philly Fed business index are on the economic calendar. Markets will also watch for shares of Microsoft, Schlumberger and Paypal as they report quarterly earnings later in the day. European shares were mixed and Asian stocks closely mostly higher. Oil slipped on profit-taking, while gold was little changed.

 

 

MARKET HIGHLIGHTS

 

CymaBay Therapeutics announced that MBX-8025 has been granted  the PRIority MEdicine (PRIME) designation by the European Medicines Agency (EMA) for the treatment of primary biliary cholangitis (PBC) in patients who do not tolerate or respond to ursodeoxycholic acid (UDCA) or do not respond to combination UDCA/obeticholic acid treatment. MBX-8025 is an orally administered potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist currently in Phase 2 clinical development.

 

Advaxis announced the commencement of Part B of the KEYNOTE-046 clinical trial evaluating Advaxis’ Lm immunotherapy candidate, ADXS-PSA, in combination with KEYTRUDA (pembrolizumab) in patients with previously treated, metastatic castration-resistant prostate cancer (mCRPC). In Part A of the Phase 1/2 study, 14 patients were treated with ADXS-PSA monotherapy across three dose levels, with no dose limiting toxicities, paving the way for initiating Part B of the study.

 

BioTime reported that data from the first patient cohort of the Phase I/IIa clinical trial of OpRegen® in the advanced form of dry age-related macular degeneration (dry-AMD) will be presented at the International Symposium on Ocular Pharmacology and Therapeutics (ISOPT) on Friday, December 2, 2016, in Rome, Italy.

 

Transgene announces the launch and terms of a share capital increase with shareholders’ preferential subscription rights for a total gross amount of circa €48 million, issue premium included.

 

Transgene published its quarterly financial results as of September 30, 2016.  Cash, cash equivalents, available-for-sale financial assets and other financial assets stood at €25.4 million as of September 30, 2016, compared to €31.7 million as of December 31, 2015.

 

Evogene Ltd. and Instituto Mato-grossense do Algodão announced today a collaboration for the discovery and validation of novel genomic promoters to support IMAmt’s product development of insect-resistant cotton varieties. The multi-year collaboration agreement between the companies provides for R&D fees and royalty payments from any future products developed as a result of the collaboration, to be paid to Evogene.

 

Rigel Pharmaceuticals announced results for the second of two double-blind studies in the FIT Phase III clinical program for fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in adult chronic/persistent immune thrombocytopenia (ITP).  The primary endpoint in the study was a stable platelet response, defined as platelet counts greater than 50,000/uL of blood on at least four of the last six scheduled clinic visits between weeks 14 and 24 of treatment. In the FIT 2 (Study 048) Phase III study, the fostamatinib response rate was 18%, consistent with the recently reported FIT 1 (Study 047) Phase III study. In Study 048, one patient in the placebo group (4%) achieved a stable platelet response; therefore the difference between those on treatment and those on placebo did not reach statistical significance (p=0.152) and the study did not meet its primary endpoint. When the data from both studies are combined, however, this difference is statistically significant (p=0.007).  Data from both FIT Phase III studies and the open-label extension study demonstrates the consistent benefit of fostamatinib in ITP.

 

Theravance Biopharma and Mylan announced positive results from two replicate Phase III efficacy studies of revefenacin (TD-4208), an investigational long-acting muscarinic antagonist (LAMA) and the first once-daily, nebulized bronchodilator in development for the treatment of chronic obstructive pulmonary disease (COPD). Top-line results across more than 1,250 moderate to very severe COPD patients confirmed that both Phase III studies met their primary efficacy endpoint, demonstrating statistically significant improvements over placebo in trough forced expiratory volume in one second (FEV1) after 12 weeks of dosing for each of the revefenacin doses studied (88 mcg once daily and 175 mcg once daily). 

 

Puma Biotechnology announced the pricing of an underwritten public offering of 3,750,000 shares of its common stock at a public offering price of $40.00 per share. The offering is expected to close on or about October 25, 2016, subject to customary closing conditions. The Company has granted the underwriters a 30-day option to purchase up to 562,500 additional shares of its common stock at the public offering price, less underwriting discounts and commissions. Citigroup and J.P. Morgan are acting as lead book-running managers, Credit Suisse is acting as joint book-running manager and BofA Merrill Lynch is acting as lead manager for the offering. Stifel is acting as co-manager for the offering.

 

BioMarin Pharmaceutical provided an update on its Phase II study of vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from 8 children in cohort 4, who completed six months of daily dosing at 30 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). These data are comparable to those observed at the lower dose of 15 µg/kg/day in cohort 3. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 µg/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.01). Vosoritide was generally well tolerated at all doses. The majority of adverse events (AEs) were mild and no serious AEs were reported as study drug-related. Across all doses, injection site reactions and hypotension were the most common drug-related AEs.  All injection site reaction events were mild and transient.

 

Celgene announced that adult patients in England and Wales with chronic plaque psoriasis will now have access to oral OTEZLA(apremilast) following a positive final appraisal determination from the National Institute for Health and Care Excellence (NICE). The decision is the conclusion of a NICE Rapid Review and ensures patients in England and Wales will join those in Scotland, who have been benefitting from access to OTEZLA since it was recommended by the Scottish Medicines Consortium (SMC) in June 2015.

 

TG Therapeutics announced the launch of a Phase I/II study to evaluate the safety and efficacy of TGR-1202, the Company’s oral PI3K delta inhibitor in combination with carfilzomib, the FDA-approved proteasome inhibitor, in patients with relapsed or refractory lymphoma.

 

Teva Pharmaceutical Industries announced that the FDA has accepted the resubmission of the NDA for SD-809 (deutetrabenazine) for the treatment of chorea associated with Huntington disease (HD). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of April 3, 2017.

 

Capricor Therapeutics announced that it has been awarded a grant of up to $4.2 million from the National Institutes of Health to study CAP-2003 (cardiosphere-derived cell exosomes) for hypoplastic left heart syndrome (HLHS).

 

Inspyr Therapeutics announced that the USPTO recently issued patent 9,446,141 B2 "Methods of Producing Cancer Compounds." Inspyr’s patent portfolio includes the commercial form and the proprietary methods for manufacturing the Company’s lead therapy Mipsagargin, a novel prodrug for the treatment of cancer. This new patent covers the methods for producing Mipsagargin (G-202) and is a key component to protecting the Company’s exclusive right to manufacture Mipsagargin. This patent expands Mipsagargin’s coverage through 2034.

 

Sage Therapeutics announced the publication in the journal Neuropharmacology of pre-clinical data demonstrating new findings relevant to Sage’s GABA product candidate portfolio. The paper, titled "Endogenous and synthetic neuroactive steroids evoke sustained increases in the efficacy of GABAergic inhibition via a protein kinase C-dependent mechanism" (Modgil et al., doi: 10.1016/j.neuropharm.2016.10.010), documents in vitro data on a novel mechanism of action that enables certain neuroactive steroids to have sustained effects on extrasynaptic GABA(A) receptors, in particular by increasing the number of extrasynaptic receptors on the membrane surface by means of receptor trafficking.

 

Horizon Pharma announced that Horizon Pharma, Inc. and Horizon Pharma USA, Inc., its wholly owned subsidiaries, have priced their offering of $300 million aggregate principal amount of 8.750% senior notes due 2024. The note offering is expected to close on or about October 25, 2016, subject to customary closing conditions.

 

Dipexium Pharmaceuticals provided an update on the status of results from the two pivotal Phase III clinical trials (OneStep-1 and OneStep-2). Data tables and listings from both of these trials are currently being produced and evaluated by the Company’s scientific advisors according to our pre-specified data review procedure. 

 

Galena Biopharma announced that Dr. Doreen Jackson delivered a podium presentation on Galena’s GALE-301 and GALE-302 clinical program at the American College of Surgeons Clinical Congress 2016 in Washington, D.C. GALE-301 (E39) and GALE-302 (E39′ — variant of E39) are cancer immunotherapies that consist of a peptide derived from Folate Binding Protein (FBP) combined with the immune adjuvant, granulocyte macrophage-colony stimulating factor (GM-CSF) for the prevention of cancer recurrence in the adjuvant setting. The Phase Ib is a single-center, randomized, single-blinded, three-arm study in patients with breast or ovarian cancer diagnosis who were treated with standard of care and were without evidence of disease. This trial augments the Phase I/IIa trial with single-agent GALE-301 in ovarian and endometrial cancers.

 

Alexion Pharmaceuticals announced that the FDA has granted orphan drug designation (ODD) to ALXN1007, a novel anti-inflammatory monoclonal antibody targeting complement protein C5a, for the treatment of acute graft-versus-host disease (GVHD). Alexion is currently investigating ALXN1007 in patients with acute GVHD of the lower gastrointestinal tract (GI-GVHD), a severe and life-threatening rare autoimmune disease that can occur as a complication of stem cell or bone marrow transplantation.

 

Eli Lilly announced that the FDA has granted approval of LARTRUVO (olaratumab injection, 10 mg/mL), in combination with doxorubicin, for the treatment of adults with soft tissue sarcoma (STS) with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery. LARTRUVO’s indication is approved under Accelerated Approval, and is based on data from the Phase II portion of the pivotal JGDG trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. LARTRUVO, in combination with doxorubicin, is the first FDA-approved front-line therapy for STS in four decades. The confirmatory Phase III trial, ANNOUNCE, is fully enrolled.

 

Reuters reported that the benefits associated with Allergan’s experimental drug to treat frequent nightly urination outweigh the risks for certain patients, an advisory panel to the FDA concluded. The panel voted 14-4 to recommend that the drug, SER120, be approved for certain patients with nocturia. The FDA is not obliged to follow its advisory panel’s recommendations but typically does so.

 

PureTech Health announced the appointment of Joseph Bolen, Ph.D., as the Company’s Chief Scientific Officer. In this new role, Dr. Bolen will lead the scientific development and advancement of PureTech’s pipeline and will work closely with the team and Scientific Advisory Board as the Company identifies and pursues new modalities to address significant medical needs.

 

MorphoSys AG announced that it has received a milestone payment from Novartis in connection with the initiation of a Phase I clinical trial with a novel HuCAL antibody. The antibody will be tested in the field of cancer. Financial details were not disclosed.

 

Spark Therapeutics announced a multi-year research agreement with Guangping Gao, Ph.D., the Penelope Booth Rockwell Chair in Biomedical Research, director of the Horae Gene Therapy Center, and professor of microbiology and physiological systems at the University of Massachusetts Medical School. Dr. Gao will collaborate broadly with Spark Therapeutics to identify adeno-associated virus (AAV) vectors from a proprietary library of AAV capsids and evaluate their efficacy, with the goal of enhancing the efficiency of gene delivery to cells in the retina, liver and central nervous system.

 

Laboratoris Sanifit announced successful initiation of the first clinical trial of its lead candidate, SNF472, for the treatment of the orphan disease calciphylaxis (calcific uraemic arteriolopathy, CUA).

 

NBE Therapeutics and SOTIO announced that the companies have entered into a collaboration for the development of next-generation antibody-drug conjugates (ADCs) for improved cancer therapy. Under the agreement, NBE and SOTIO will collaborate on the discovery, non-clinical development and manufacturing of novel ADC products against undisclosed targets. The ADC products will be based on NBE’s proprietary antibody discovery and conjugation platforms, including NBE’s Transpo-mAb antibody platform, its site-specific SMAC conjugation technology and its novel ultra-potent toxin platform. SOTIO will have global responsibility for clinical development, registration and commercialization of the ADC products.

 

Propeller Health announced the closing of its $21.5 million Series C financing. The funding round includes new investors 3M Ventures, S.R. One, Limited and Hikma Ventures, and existing investors Safeguard Scientifics and Social Capital. Proceeds will be used to expand Propeller’s digitally-guided therapy platform and modernize the management of respiratory disease.

 

ProMetic Life Sciences announced that its Phase II clinical trial in patients with metabolic syndrome and type 2 diabetes has been completed and has met its primary and secondary endpoints. In addition to safety and tolerability, the study was designed to evaluate the effect of PBI-4050 on metabolic syndrome parameters as well as on pro-inflammatory/fibrotic and diabetic biomarkers in blood and urine. In this open label Phase II clinical trial, PBI-4050 (800 mg) was administered once daily to 24 patients for a period of 12 weeks.  Ten of these patients were enrolled in an additional 12 weeks extension. PBI-4050 has been well tolerated with no serious drug related adverse events.

 

RedHill Biopharma announced that it has received from the Japan Patent Office a Notice of Allowance for a new patent covering RHB-104 for the treatment of multiple sclerosis (MS), which is expected to be valid until 2032, once granted. This notice follows RedHill’s recent announcement that the counterpart European patent application was approved by the European Patent Office.

 

Spring Bank Pharmaceuticals announced the presentation of scientific data relating to its novel, proprietary STING (STimulator of INterferon Genes) agonist compound, SB 11285, at the American Association for Cancer Research (AACR) Special Conference on Tumor Immunology and Immunotherapy taking place on October 20-23, 2016 at the Boston Marriott Copley Place in Boston, MA.

 

Madrigal Pharmaceuticals announced that the first patient has been dosed in its Phase II study of MGL-3196 for the treatment of non-alcoholic steatohepatitis (NASH). MGL-3196 is a first-in-class, oral, once-daily, liver-directed, thyroid hormone receptor (THR) ß-selective agonist medication.

 

Abeona Therapeutics provided at the Orphan Drugs & Rare Disease Conference (London, UK), an update on clinical results through 30 days post-injection for the completed low-dose cohort (n=3) in the ongoing Phase I/II trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes a single intravenous injection of AAV gene therapy for subjects with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

 

Neuralstem announced the presentation of the poster entitled, "Reversal of radiation-induced cognitive impairment by oral administration of a neurogenic small molecule compound NSI-189," at the Radiation Research Society Annual Meeting on October 19(th) . The preclinical study data concluded that NSI-189 provided treatment benefits in the reversal of radiation-induced cognitive deficits.

 

Debiopharm International announced that it has entered into a collaboration agreement with Merck KGaA and Pfizer to evaluate Debio 1143, an oral, small molecule inhibitor of IAPs (Inhibitor of Apoptosis Proteins), in combination with avelumab, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in patients with advanced or metastatic Non-Small Cell Lung Cancer (NSCLC). Debio 1143 is currently in Phase II development for Head & Neck and Ovarian Cancer. Avelumab is under clinical investigation across a broad range of tumor types by Merck KGaA, Darmstadt, Germany, and Pfizer. Under the terms of the agreement, Debiopharm will be responsible for conducting the Phase I/Ib clinical trial in NSCLC.

 

HedgePath Pharmaceuticals announced further positive interim data from its ongoing, open-label Phase II(b) clinical trial studying the effect of SUBA-Itraconazole (SUBA-Cap) oral capsules in patients with Basal Cell Carcinoma Nevus Syndrome (BCCNS), also known as Gorlin Syndrome. Initial positive interim data were first reported by HPPI in early August 2016.

 

Selecta Biosciences announced that encouraging results from preclinical studies conducted in collaboration with Federico Mingozzi, Ph.D., Head of Immunology and Liver Gene Therapy at Genethon in Evry, France were presented at the Annual Congress of the European Society of Gene and Cell Therapy in Florence, Italy in a poster titled, "Antigen-specific modulation of capsid immunogenicity with tolerogenic nanoparticles results in successful AAV vector re-administration". The new preclinical data elucidate the mechanism by which SVP-Rapamycin mitigates undesired immune responses. Further, the data demonstrate therapeutic benefits of co-administration of SVP-Rapamycin with an AAV8 gene therapy vector expressing Factor IX, a coagulation protein deficient in patients with Hemophilia B.

 

Ocular Therapeutix announced the appointment of Andy Hurley to the newly created position of chief commercial officer, effective immediately. In this role, Mr. Hurley will be responsible for leading Ocular’s commercial organization, focusing on effective execution of potential future product launches, brand development, and ongoing commercialization strategies.

 

Adverum Biotechnologies announced the presentation of preclinical data on ADVM-022 and ADVM-032, the Company’s novel gene therapy candidates for ophthalmology. The Company is presenting data during an oral presentation and two poster sessions at the European Society of Gene and Cell Therapy (ESGCT) meeting, held October 18 — 21, 2016 in Florence, Italy.

 

InVivo Therapeutics Holdings announced that Beth Israel Deaconess Medical Center (BIDMC) 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. A teaching hospital of Harvard Medical School, BIDMC consistently ranks as a national leader among independent hospitals in National Institutes of Health funding.

 

Seattle Genetics highlighted multiple data presentations at the 10th International Symposium on Hodgkin Lymphoma (ISHL) taking place in Cologne, Germany, October 22-25, 2016, evaluating ADCETRIS (brentuximab vedotin) across a broad range of Hodgkin lymphoma (HL) disease settings. ADCETRIS is an antibody-drug conjugate (ADC) directed to CD30, a defining marker of classical HL that plays a role in tumor pathogenesis. ADCETRIS is being evaluated globally as the foundation of therapy for HL in more than 45 ongoing corporate- and investigator-sponsored clinical trials. Data presented at ISHL include an update of the progression-free survival and safety from the Phase III AETHERA trial approximately four years since the last patient was enrolled, demonstrating sustained progression-free survival benefit after extended observation. In addition, updates will be presented from trials evaluating ADCETRIS as both mono- and combination therapy in frontline HL patients age 60 and older, and as second-line therapy for relapsed or refractory HL. Lastly, preclinical data will be presented indicating additional potential mechanisms of action for ADCETRIS, including immunogenic cell death, supporting evaluation of combination therapy with immuno-oncology agents.

 

Biocept announced that clinical results from a study evaluating 40 patients with advanced non-small cell lung cancer (NSCLC) demonstrated up to 90% concordance between the Company’s Target-Selector platform and tissue biopsy for the detection of actionable oncogene mutations and other validated cancer biomarkers. Analysis of a patient subset in the study showed that changes in both circulating tumor DNA (ctDNA) and circulating tumor cells (CTCs), detected using Biocept’s proprietary liquid biopsy assays, correlated significantly to response to systemic drug therapy in a majority of cases.  The clinical data were presented at the 2016 European Society for Medical Oncology (ESMO) Annual Congress in Copenhagen, Denmark.

 

Rosetta Genomics announced the appointment of Mark R. Willig as Chief Commercial Officer. In this newly created position, Mr. Willig will be responsible for sales, marketing and market access for Rosetta Genomics’ entire portfolio of high-value diagnostic assays. He will report to Kenneth A. Berlin, President and Chief Executive Officer of Rosetta Genomics.

 

ICON plc announced that, as part of its planned succession of executive leadership, Mr. Ciaran Murray will transition to the role of Chairman of the Board of Directors and Dr. Steve Cutler will be appointed as Chief Executive Officer, with both changes effective as of 1st March 2017.

 

 

ANALYST RECOMMENDATIONS

 

Keybanc analyst Matthew Mishan initiated coverage of Catalent with an “overweight” rating and $29 price target, citing it is differentiated by its scale and expertise in advanced delivery technologies; with Beinheim recovering and Blackstone exiting its final stake, key overhangs are lifting and it could meet/exceed conservative FY17 guidance; there are multiple drivers of LT revenue growth; EBITDA margin appears sustainable LT; and M&A pace and size potentially accelerate.

 

Barclays analyst Geoff Meachem revised his price target of the following companies: United Therapeutics decreased to $115 from $120; Vertex Pharmaceuticals decreased to $100 from $110.

 

HC Wainwright analyst Corey Davis initiated coverage of Scynexis with a “buy” rating and $14 price target, citing Scynexis has a novel anti-fungal, SCY-078, with very little competition, in an area that desperately needs innovation.

 

Credit Suisse analyst Erin Wilson increased her price target of ICON to $84 from $80, citing more consistent and respectable bookings and profit trends.