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BioShares Biotechnology Clinical Trials (BBC): $19.58, -$0.78, -32.6% YTD

BioShares Biotechnology Products (BBP): $33.45, -$1.20, +8.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were higher, continuing gains after the S&P 500 and Dow surged to fresh highs in the previous session. Encouraging data from China and expectations that the European Central Bank will extend its already generous stimulus program buoyed investment sentiment in Europe and Asia. In the forex markets, New Zealand’s dollar was the biggest gainer among major currencies after a speech by Reserve Bank Governor Graeme Wheeler added to speculation the bank would raise interest rates next year. Oil prices rose as the dollar weakened and gold crept higher.

 

 

MARKET HIGHLIGHTS

 

Neovacs announced that the U.S. Food and Drug Administration (FDA) has granted “Fast Track” status to IFNα Kinoid in Lupus, the most advanced therapeutic vaccine issued from Neovacs R&D.

 

Transgene announced the signature of a collaborative arrangement with UC Davis to support an investigator-initiated study led UC Davis Medical Center that will evaluate TG4010 in combination with Opdivo® (nivolumab) for the treatment of 2nd line metastatic non-small cell lung cancer (NSCLC). This trial is supported by Transgene through financial support and supply of TG4010 and by Bristol-Myers Squibb through supply of nivolumab for use in the clinical study.

 

Kitov Pharmaceuticals Holdings announced that its lead drug candidate KIT-302 met the U.S. Food and Drug Administration’s (FDA) standards for establishing six months stability.

 

Endo International announced that it has entered into an agreement with its partner, BioDelivery Sciences International, to return the BELBUCA  (buprenorphine) buccal film product to BDSI. Specific financial terms of the agreement have not been disclosed and are not material to Endo. Endo will not have any future royalty or milestone payments to BDSI and BDSI is not obligated for any future royalty payments to Endo. With the return of BELBUCA to BDSI, Endo has a portfolio of established pain products that the Company believes no longer requires field sales promotion. As a result, Endo also announced that it is eliminating its 375-member U.S. Branded pain sales field force, which consisted of both full-time employees and contract sales representatives, as well as internal support to the promoted pain business unit. This will allow the Company to focus efforts and resources more fully on its core U.S. Branded assets, including XIAFLEX in the approved indications and the cellulite development program. The Company’s legacy pain portfolio products C including OPANA ER and Percocet, among others will be managed as mature brands.

 

The MEDICREA Group will host a Key Opinion Leader breakfast in New York City for investors focused on advances in the personalized spine market at 8:00 am Eastern Time on Thursday, December 15, 2016.

 

Zealand Pharma has increased its share capital with nominal DKK 13,999 divided into 13,999 new shares with a nominal value of DKK 1 each. The increase is a consequence of the exercise of warrants granted under one of Zealand`s employee warrant programs. Employee warrant programs are part of Zealand`s incentive scheme, and each warrant gives the owner the right to subscribe for one new Zealand share at a pre-specified price, the exercise price, in specific pre-defined time periods before expiration.

 

Palatin Technologies has closed on a previously disclosed underwritten public offering of units with gross proceeds of $16,500,000.  Canaccord Genuity acted as sole book-running manager, Roth Capital Partners acted as lead manager and Chardan Capital Markets acted as co-manager for the offering.

 

CTD Holdings announced that its Senior V.P. for Medical Affairs, Dr. Sharon Hrynkow, will present "Use of Cyclodextrins in Compassionate Programs and Clinical Trials for Niemann Pick Disease Type C" as part of a panel discussion titled Pediatrics: Latest Advances in Diagnosis and Therapy at the BioFlorida Annual Conference. The presentation will cover the use of Trappsol® Cyclo™, CTD’s proprietary formulation of hydroxypropyl beta cyclodextrin, in the treatment of Niemann Pick Disease Type C.

                 

CymaBay Therapeutics announced the appointment of Robert Booth, Ph.D. and Caroline Loewy to the company’s Board of Directors, effective December 6, 2016.

 

Ohr Pharmaceutical has priced a registered public offering of an aggregate of 3,875,000 shares of common stock at a price to the public of $2.00 per share. Investors will also receive series A warrants to purchase up to an aggregate of 1,937,500 shares of common stock with an exercise price of $2.75 per share and series B warrants to purchase up to an aggregate of 3,875,000 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. The offering is expected to close on or about December 13, 2016, subject to satisfaction of customary closing conditions.

 

Voyager Therapeutics announced positive results from the ongoing Phase Ib trial of VY-AADC01 at six and twelve months of follow-up in patients with advanced Parkinson’s disease. The interim data from Cohorts 1 and 2 of this trial demonstrated that accurate MRI-guided delivery of escalating doses of VY-AADC01 were well tolerated and resulted in increased coverage of the putamen, increased AADC enzyme activity, enhanced response to levodopa, and dose-related, clinically meaningful improvements in various measures of patients’ motor function. This was especially evident at the higher dose in Cohort 2 with improved UPDRS off medication and on medication scores, and corresponding improvements in patient-reported diary hours, suggesting higher peak effects and a longer duration of action of levodopa. These effects were maintained and in some patients improved at 12 months of follow-up.

 

Tonix Pharmaceuticals presented new results from a sub-group analysis of the data from the AtEase study, a 12-week, randomized, double-blind, placebo-controlled Phase 2 study evaluating TNX-102 SL*, 5.6 mg, in military-related PTSD.

 

ContraVir Pharmaceuticals provided new insights into the mechanism of action of its hepatitis B (HBV)-optimized cyclophilin inhibitor CRV431, showing that it effectively blocks a critical interaction between HBV X protein (HBx) and host cyclophilin A, with nanomolar potency.  CRV431 is believed to be the first antiviral drug with the potential to selectively block the HBx pathway, which is known to play several significant roles in HBV infection including protecting infected liver cells from immune destruction, and promoting the development of liver cancer.

 

Horizon Pharma announced that the Phase III trial, STEADFAST (Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich’s Ataxia study), evaluating ACTIMMUNE (interferon gamma-1b) for the treatment of Friedreich’s ataxia (FA) did not meet its primary endpoint of a statistically significant change from baseline in the modified Friedreich’s Ataxia Rating Scale (FARS–mNeuro) at 26 weeks versus treatment with placebo. FARS–mNeuro is an exam-based rating scale that measures disease progression based on functional parameters such as speech, ability to swallow, upper and lower limb coordination, gait and posture. In addition, the secondary endpoints did not meet statistical significance. No new safety findings were identified on initial review of data other than those already noted in the ACTIMMUNE prescribing information for approved indications. The Company, in conjunction with the independent Data Safety Monitoring Board, the principal investigator and the Friedreich’s Ataxia Research Alliance (FARA) Collaborative Clinical Research Network (CCRN) in FA, has determined that, based on the trial results, the FA development program will be discontinued, including the 26-week extension study and the long-term safety study.

 

GTx announced positive initial data from its ongoing open-label enobosarm Phase II clinical trial in women with advanced, estrogen receptor positive (ER+), androgen receptor positive (AR+) breast cancer. The pre-specified threshold for success of the trial was met early in the 9 mg cohort with 9 patients achieving a clinical benefit response at 24 weeks among the first 22 evaluable patients in that cohort, as reported by the Company on November 28, 2016. Clinical Benefit Response (CBR) is defined as a complete response (CR), partial response (PR) or stable disease (SD), as measured by Response Evaluation Criteria in Solid Tumors (RECIST) at 24 weeks of treatment.

 

Oxford Immunotec Global announced that the New England Journal of Medicine has published an article on a three-year clinical trial completed by Oxford Immunotec’s Imugen division, and the American Red Cross to evaluate a serology and Nucleic acid Amplification Test testing regime for Babesia in blood screening. The review article "Screening for Babesia microti in the US blood supply" is authored by Susan Stramer of the American Red Cross, and will appear in the December 8, 2016 print issue.

 

Anavex Life Sciences announced a positive 57-week update from its Phase IIa study in mild-to-moderate Alzheimer’s disease (AD) patients for ANAVEX 2-73, which targets cellular homeostasis. The study met both primary and secondary endpoints.

 

Amicus Therapeutics announced positive preliminary data from a global Phase I/II study (ATB200-02) to investigate ATB200/AT2221. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

 

Soligenix announced the long-term follow-up data from its Phase II clinical trial with SGX942 (dusquetide), a first-in-class Innate Defense Regulator (IDR), in the treatment of oral mucositis (OM) in head and neck cancer patients undergoing chemoradiation therapy (CRT). The additional 12-month safety data remains consistent with the preliminary positive safety and efficacy findings from the Phase II study and provide further support for advancing SGX942 into a pivotal Phase IIb/III clinical trial. Following the positive results announced in December 2015, in which SGX942 at a dose of 1.5 mg/kg, successfully reduced the median duration of severe OM by 50% in all patients and by 67% in patients at highest risk of developing severe OM, long-term follow-up visits conducted throughout 2016 further demonstrated that SGX942 was safe, well-tolerated, and did not interfere with CRT as demonstrated by improved survival and tumor resolution at one and 12 months. Overall, there were no drug-related toxicities identified in this study.

 

Spectrum Pharmaceuticals announced the oral presentation of data from a preclinical study evaluating poziotinib in lung cancer by scientists from MD Anderson Cancer Center at the 17th International Association for the Study of Lung Cancer (IASLC) World Conference on Lung Cancer which took place in Vienna, Austria, December 4-7, 2016.

 

Daiichi Sankyo Europe GmbH announced that the first patient has been enrolled in the EMIT-AF/VTE (Edoxaban Management In diagnostic and Therapeutic procedures) study. This registry will collect real-world clinical data on the use of once-daily LIXIANA (edoxaban) with regard to diagnostic and interventional procedures in patients with non-valvular atrial fibrillation (NVAF) or venous thromboembolism (VTE).

 

Puma Biotechnology announced that interim results from a Phase II clinical trial of Puma’s investigational drug PB272 (neratinib) were presented at the 2016 CTRC-AACR San Antonio Breast Cancer Symposium (SABCS) that is currently taking place in San Antonio, Texas. The presentation entitled, "Incidence and severity of diarrhea with neratinib plus intensive loperamide prophylaxis in patients with HER2-positive early-stage breast cancer (EBC): Interim analysis from the multicenter, open-label, phase II CONTROL trial" was presented as a poster presentation.

 

Puma Biotechnology announced that updated interim results from an ongoing Phase II clinical trial of Puma’s investigational drug PB272 (neratinib), given as monotherapy and in combination with the anticancer drug fulvestrant, were presented at the 2016 CTRC-AACR San Antonio Breast Cancer Symposium (SABCS) that is currently taking place in San Antonio, Texas. The presentation entitled “Neratinib plus fulvestrant for ERBB2 mutant, HER2 non-amplified, estrogen receptor-positive, metastatic breast cancer: Preliminary analysis from the Phase II SUMMIT trial” was presented as a poster discussion by Dr. David Hyman, Director, Developmental Therapeutics at Memorial Sloan Kettering Cancer Center.

 

Vanda Pharmaceuticals announced it has entered into a License Agreement with Apotex Inc. and Apotex Corp. to resolve Vanda’s patent litigation against Apotex regarding Apotex’s ANDA seeking approval of its generic version of Vanda’s Fanapt (iloperidone). Under the License Agreement, Vanda granted Apotex a non-exclusive license to manufacture and commercialize Apotex’s version of Fanapt in the U.S. effective November 2, 2027, unless prior to that date Vanda obtains pediatric exclusivity for Fanapt, in which case, the license will be effective May 2, 2028.  Apotex may enter the market earlier under certain limited circumstances.

 

Regeneron Pharmaceuticals and Sanofi announced that the EMA has accepted for review the MAA for Dupixent (dupilumab) for the treatment of adults with moderate-to-severe atopic dermatitis (AD) who are candidates for systemic therapy. The investigational biologic therapy Dupixent inhibits signaling of IL-4 and IL-13, two key cytokines required for the type 2 (including Th2) immune response, which is believed to be a major driver in the pathogenesis of the disease.

 

AbbVie announced the EC has granted conditional marketing authorization for VENCLYXTO (venetoclax) monotherapy for the treatment of chronic lymphocytic leukaemia (CLL) in the presence of 17p deletion or TP53 mutation in adult patients who are unsuitable for or have failed a B-cell receptor pathway inhibitor; and for the treatment of CLL in the absence of 17p deletion or TP53 mutation in adult patients who have failed both chemoimmunotherapy and a B-cell receptor pathway inhibitor. The EC approved VENCLYXTO as a first-in-class, oral, once-daily medicine that selectively inhibits the function of the BCL-2 protein. BCL-2 prevents the natural death of cells, including CLL cells. VENCLYXTO is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the U.S. and by AbbVie outside of the U.S.

 

Pharming Group announced that it has completed a definitive agreement to acquire all North American commercialization rights for its own product, RUCONEST (recombinant human C1 esterase inhibitor), including all rights in the U.S, Mexico and Canada, from certain subsidiaries of Valeant Pharmaceuticals. RUCONEST is an orphan drug designated therapy developed by Pharming, already approved for the treatment of acute Hereditary Angioedema ("HAE") attacks in patients in the U.S and EU. This transaction will accelerate Pharming’s development into a profitable specialty pharmaceutical company with its own independent commercial infrastructure, which will form the foundation for future growth.

 

UCB and Dermira announced topline results from CIMPASI-1, a Phase III, multi-center, placebo-controlled clinical trial evaluating the efficacy and safety of CIMZIA (certolizumab pegol) in adult patients with moderate-to-severe chronic plaque psoriasis. In the CIMPASI-1 trial, CIMZIA demonstrated statistically significant improvements for both co-primary endpoints compared to placebo at both treatment doses. This is the second of three Phase III clinical trials evaluating CIMZIA in this patient population. UCB and Dermira announced topline results from CIMPASI-2 in October 2016.

 

Advanced Accelerator Applications announced that the Centers for Medicare & Medicaid Services (CMS) has granted NETSPOT Transitional Pass-Through status under an "A-code" (A9587) for drug reimbursement, effective January 1, 2017. Additionally, the same Healthcare Common Procedure Coding System ("HCPCS") "A Code" will be used on claims to private payers. NETSPOT was approved by the FDA in June 2016 for the preparation of gallium Ga 68 dotatate for injection, for the localization of somatostatin receptor positive neuroendocrine tumors ("NETs") in adult and pediatric patients using Positron Emission Tomography ("PET’).

 

Matinas BioPharma Holdings announced that it has received a research contract award from Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, to study its lead antibiotic product candidate MAT2501, for the treatment of pre-clinical nontuberculous mycobacterium infection (NTM) in models of cystic fibrosis (CF). The award provided by CFFT will support a collaborative research program between Matinas BioPharma and Colorado State University (CSU) to study the efficacy of MAT2501 in the treatment of NTM infection by a range of mycobacterium species, including mycobacterium abscessus, in a CF lung infection model developed by CSU.

 

Orexigen Therapeutics announced that its wholly owned subsidiary Orexigen Therapeutics Ireland Ltd. and Consilient Health Ltd. have executed a commercialization and distributorship agreement for Mysimba (naltrexone HCl / bupropion HCl prolonged release) in the UK and Ireland. Mysimba is approved by the EMA, as an adjunct to a reduced-calorie diet and increased physical activity, for the management of weight in adult patients (≥18 years) with an initial Body Mass Index (BMI) of ≥ 30 kg/m2 (obese), or ≥ 27 kg/m2 to < 30 kg/m2 (overweight) in the presence of one or more weight-related co-morbidities (e.g., Type II diabetes, dyslipidaemia, or controlled hypertension). Founded in 2005, Consilient is one of the fastest growing pharmaceutical companies in the UK with sales and marketing teams dedicated to working with primary care prescribers and payers across the UK and Ireland.

 

InVivo Therapeutics Holdings announced the resignation of Steven McAllister from the position of CFO effective December 31, 2016.

 

Catalyst Pharmaceuticals provided an update on its clinical trial with amifampridine phosphate in patients with congenital myasthenic syndromes (CMS). After discussions with the FDA the study has been expanded beyond pediatric patients to include adult CMS patients and the enrollment size has been increased to approximately 20 patients.

 

Gilead Sciences announced that it has submitted a NDA to the FDA for an investigational, once-daily single tablet regimen containing sofosbuvir 400 mg, velpatasvir 100 mg, and voxilaprevir 100 mg (SOF/VEL/VOX) for the treatment of direct-acting antiviral (DAA)-experienced chronic hepatitis C virus (HCV)-infected patients. The data submitted in the NDA support the use of the regimen for 12 weeks in DAA-experienced patients with genotype 1 to 6 HCV infection without cirrhosis or with compensated cirrhosis.

 

PTC Therapeutics announced that the company received France’s 2016 Prix Galien for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in the Medicines "Rare Disease" category. The award recognizes the scientific innovation represented by Translarna’s ability to target the underlying cause of nmDMD and the impact the drug is having on patients. The award was presented at the annual gala and ceremony in Paris, France.

 

AstraZeneca announced results from a pre-specified subgroup analysis of the positive Phase III FALCON trial suggesting that treatment effects in terms of progression-free survival (PFS) were largely consistent across the subgroups analyzed with some possible exceptions, including patients with non-visceral disease. The FALCON trial assessed FASLODEX (fulvestrant) 500 mg compared to ARIMIDEX (anastrozole) 1 mg in the 1st line endocrine treatment of postmenopausal women who were initially diagnosed with locally-advanced or metastatic breast cancer who had not had prior hormonal treatment for hormone receptor positive (HR+) breast cancer.

 

Eli Lilly announced that the neoMONARCH study of abemaciclib, a cyclin-dependent kinase (CDK) 4 and CDK 6 inhibitor, met its primary endpoint of reducing expression of Ki67, a biomarker of cell proliferation, after two weeks of treatment. Final data from the Phase II trial presented during the official press program at the 2016 San Antonio Breast Cancer Symposium (SABCS) evaluated abemaciclib, both alone or in combination with the non-steroidal aromatase-inhibitor anastrozole, in postmenopausal women with previously untreated early stage hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer.

 

Radius Health announced data from two ongoing Phase I studies of RAD1901, an oral selective estrogen receptor degrader (SERD), in patients with estrogen receptor positive (ER+) breast cancer, which were presented this morning at the San Antonio Breast Cancer Symposium 2016.

 

Biohaven Pharmaceutical Holding Company announced that the FDA has granted the Company’s orphan drug designation request covering its drug candidate BHV-0223, an orally dissolving tablet being developed for the treatment of Amyotrophic Lateral Sclerosis ("ALS"), also referred to as Lou Gehrig’s disease. This is the Company’s third orphan drug designation request granted by the FDA.

 

Provectus Biopharmaceuticals and POETIC, The Pediatric Oncology Experimental Therapeutics Investigators Consortium, a group of 10 top-tier academic medical centers developing new pediatric cancer therapies, announced a joint research agreement focused on pediatric applications of PV-10, an investigational ablative immunotherapy, as a potential treatment for childhood cancers.

 

Perrigo Company announced its intention to restructure the Branded Consumer Healthcare’s Omega Pharma Belgium business (which includes Omega Pharma Belgium NV, Etixx NV and Biover NV), located in Nazareth, Venecoweg 26 (Belgium) to improve the financial profile and enhance focus of the business on branded consumer OTC products.

 

Recro Pharma announced that it intends to offer and sell, subject to market conditions, shares of its common stock in an underwritten public offering. All of the shares to be sold in the offering will be offered by Recro. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. Recro intends to use the net proceeds of the proposed offering to fund the NDA filing and regulatory approval process and preparatory commercial activities for IV meloxicam, its planned IV meloxicam Phase IIIB program, and for general corporate purposes. Piper Jaffray is the sole underwriter for the offering.

 

Clearside Biomedical announced that it has commenced an underwritten public offering of $75 million of shares of its common stock. In addition, Clearside expects to grant the underwriters a 30-day option to purchase up to an additional $11.25 million of shares of common stock at the public offering price, less the underwriting discount. Clearside intends to use the net proceeds from the offering to complete its pivotal Phase III clinical trial of suprachoroidal Zuprata for the treatment of macular edema associated with non-infectious uveitis and to initiate its Phase III clinical program of suprachoroidal Zuprata with the intravitreal anti-VEGF agent, Eylea, for the treatment of macular edema associated with RVO. In addition, proceeds will also be used to complete its ongoing Phase I/II clinical trial of Zuprata alone or with Eylea for the treatment of DME and to initiate and complete a planned Phase I/II clinical trial of axitinib for the treatment of wet AMD as well as for continued research and development of its earlier-stage programs, working capital and general corporate purposes. J.P. Morgan Securities and Cowen are acting as joint active book-running managers for the proposed offering. Stifel, Nicolaus & Company is acting as a passive book-running manager and Wedbush Securities is acting as a co-manager.

  

Blueprint Medicines announced the pricing of an underwritten public offering of 5,000,000 shares of its common stock at a public offering price of $25.00 per share, before underwriting discounts and commissions. In addition, Blueprint Medicines has granted the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock at the public offering price. Goldman Sachs, Morgan Stanley and Cowen are acting as joint book-running managers for the offering. JMP Securities and Wedbush PacGrow are acting as co-managers for the offering.

 

 

Analyst Recommendations

 

Stifel analyst Thomas Shrader increased his price target of Selecta Biosciences to $32 from $23, citing SEL-212 has demonstrated initial proof of concept in humans with an acceptable safety profile for more serious disease states.

 

BMO analyst Ian Somaiya revised his price target of the following companies: Gilead increased to $88 from $84; BioMarin decreased to $119 from $123.

 

Raymond James analyst Christopher Raymond decreased his price target of Blueprint Medicines to $34 from $35, citing dilution following offering.