BioShares Biotechnology Clinical Trials (BBC): $19.97, +$0.23, +8.5% YTD

BioShares Biotechnology Products (BBP): $35.64, +$0.15, +8.8% YTD

 

 

MARKET COMMENTARY

 

Comments from U.S. President-elect Donald Trump that the dollar is too strong and hurting U.S. competitiveness pushed U.S. stock index futures and the greenback lower. European shares were down, following a speech by British Prime Minister Theresa May. Asian markets closed mixed. A weaker dollar and commitment from Saudi Arabia to cut output pushed oil higher, while gold hit its highest in more than seven weeks.

 

 

MARKET HIGHLIGHTS

 

Alcobra reported the top-line results from MEASURE, its second Phase 3 clinical trial for the investigational product Metadoxine Extended Release (MDX) for the treatment of ADHD in adult patients. In this trial, MDX did not meet the primary endpoint of demonstrating a statistically significant difference from placebo in the change from baseline of the investigator rating of the Conners’ Adult ADHD Rating Scales (CAARS).

 

BrainStorm Cell Therapeutics will give a presentation entitled "NurOwn® – ground breaking treatment for ALS and other neurodegenerative diseases", during the Biotech Showcase at the Phacilitate Cell & Gene Therapy World 2017 Conference, on January 18.

 

CTD Holdings announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Trappsol® Cyclo™ for the treatment of systemic Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease. Trappsol Cyclo is the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin in development for the treatment of NPC; it has previously been designated an Orphan Drug by the FDA and the European Medicines Agency.

 

Immune Pharmaceuticals will hold a Satellite Symposium on February 20th, 2017 at the International Symposium on Acute Leukemias (ISAL) XVI in Munich, Germany.

 

Biogen announced that it has agreed to enter into a settlement and license agreement with Forward Pharma, subject to the approval of Forward Pharma’s shareholders and other customary conditions. The license agreement will provide Biogen an irrevocable license to all intellectual property owned by Forward Pharma. Upon the effectiveness of the settlement and license agreement, Biogen will provide Forward Pharma a cash payment of $1.25 billion. Under certain circumstances outlined in the agreement, Biogen will pay Forward Pharma royalties on net sales of Biogen products for the treatment of multiple sclerosis that are covered by a Forward Pharma patent and have dimethyl fumarate ("DMF") as an active pharmaceutical ingredient.

 

Celsion announced data from the fourth cohort of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the Company’s IL-12 gene-mediated immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with Stage III and IV ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. In the first twelve patients dosed in the OVATION Study, GEN-1 plus standard chemotherapy produced impressive results, with no dose limiting toxicities and highly promising efficacy signals in this difficult to treat cancer.

 

BioLineRx announced the initiation of a second Phase IIa trial investigating BL-8040 in combination with KEYTRUDA (pembrolizumab), MSD’s anti-PD-1 therapy, in patients with metastatic pancreatic cancer. The study is part of a research collaboration between MSD and MD Anderson Cancer Center.

 

Eli Lilly and Incyte announced that the FDA has extended the review period for the NDA for investigational baricitinib, a once-daily oral medication for the treatment of moderate to severe rheumatoid arthritis (RA). The NDA for baricitinib was submitted to the FDA in January 2016. The FDA extended the action date to allow time to review additional data analyses recently submitted by Lilly in response to the FDA’s Information Requests. The submission of the additional information has been determined by the FDA to constitute a Major Amendment to the NDA, resulting in an extension of the PDUFA goal date by three months.

 

Concert Pharmaceuticals provided further details on the development plan in the U.S. and Europe for CTP-656, a next generation CFTR potentiator being developed for the treatment of cystic fibrosis. In the U.S., Concert initiated a placebo-controlled Phase II trial in December 2016 to evaluate CTP-656 in cystic fibrosis patients with gating mutations. Subsequent to the initiation of the study, the FDA informed Concert that, in order to support dose selection for Phase III, an adequate washout period, in which Kalydeco (ivacaftor) treatment is withheld, would be required in addition to a placebo-control. The Company intends to further discuss the additional feedback with FDA. The ongoing Phase II trial is being conducted at multiple U.S. study sites within the Cystic Fibrosis Foundation’s Therapeutic Development Network with topline data expected by year-end 2017. In Europe, Concert intends to initiate an open-label Phase II trial in the second quarter of 2017. The trial is expected to enroll 14 cystic fibrosis patients with gating mutations stable on Kalydeco. Patients will be switched to one dose of CTP-656 for two weeks followed by a switch to a higher dose of CTP-656 for an additional two weeks. Subsequently, patients will resume treatment with Kalydeco. The primary analysis will be for non-inferiority for sweat chloride compared to the Kalydeco baseline. Topline data from the European Phase II study of CTP-656 is expected by year-end 2017.

 

Corbus Pharmaceuticals Holdings announced that the European Commission has granted Orphan Designation in the European Union for the Company’s novel synthetic oral endocannabinoid-mimetic drug, JBT-101 ("Resunab") for the treatment of systemic sclerosis.

 

Kura Oncology announced that the first patient has been dosed in its Phase II clinical trial of tipifarnib in patients with chronic myelomonocytic leukemia (CMML).

 

XBiotech announced the publication of results from its pivotal Phase III trial of the Company’s lead monoclonal antibody therapy. The results were published in The Lancet Oncology in an article titled, "MABp1 as a Novel Antibody Treatment for Advanced Colorectal Cancer: A Randomised, Double-Blind, Placebo-Controlled, Phase III Study." The article is now available online.

 

Tetraphase Pharmaceuticals announced dosing of the first patient in IGNITE3, the Company’s Phase III clinical trial evaluating the efficacy and safety of once-daily intravenous (IV) eravacycline compared to ertapenem in complicated urinary tract infections (cUTI). Eravacycline is a novel antibiotic candidate with potent activity against multidrug-resistant (MDR) pathogens, including carbapenem-resistant Enterobacteriaceae (CRE), Acinetobacter baumannii, and colistin-resistant bacteria carrying the mcr-1 gene, that is being developed for the treatment of serious and life-threatening bacterial infections.

 

Elite Pharmaceuticals reported positive topline results from a pivotal bioequivalence fed study initiated in November 2016. The topline results indicate that Elite’s generic product is bioequivalent to the branded product, OxyContin (extended release Oxycodone Hydrochloride). A fasted study was previously conducted in September 2016, also with positive results.

 

Protagonist Therapeutics announced that it has initiated a global Phase IIb induction study in ulcerative colitis with PTG-100, an oral peptide that targets alpha4beta7 integrin. The aim of this randomized, double-blind, placebo-controlled, adaptive design study is to evaluate the safety/tolerability and efficacy of PTG-100 in approximately 240 adult ulcerative colitis patients with moderate to severe active disease.

 

InVivo Therapeutics Holdings announced that the University of New Mexico Hospital (UNMH) in Albuquerque, NM 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. The University of New Mexico Hospital (UNMH) is the state’s only Level I trauma center.

 

TESARO announced the opening of an expanded access program (EAP) in the United States for the investigational PARP inhibitor, niraparib. Through this program, niraparib is being made available for eligible women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer following a complete or partial response to platinum-based chemotherapy. An EAP for niraparib in Europe is planned to open in the first half of 2017, and will be initiated on a country-by-country basis.

 

Pharming Group announced that, following the positive opinion of the CHMP, the EC has adopted the Commission Implementing Decision to amend the marketing authorization for RUCONEST to include self-administration using the RUCONEST Administration Kit. This decision allows for self-administration of RUCONEST for acute hereditary angioedema (HAE) attacks by adolescents and adults with a new custom-designed RUCONEST Administration Kit in the comfort and privacy of their own homes or at any other place they choose, without the necessity of a healthcare professional (HCP) being present.  The Administration Kit will become available for use in the various EU markets, following approval of the Educational Materials by the local authorities, expected over the coming one to three months.

 

Hutchison China MediTech announced that it has initiated a Phase II study of a combination therapy using fruquintinib and Iressa in the first-line setting for patients with advanced or metastatic non-small cell lung cancer in China. Fruquintinib is a highly selective and potent oral inhibitor of vascular endothelial growth factor receptors. The first drug dose was administered on January 9, 2017.

 

Allergan and Gedeon Richter announced positive results from Venus II, the second of two pivotal Phase III clinical trials evaluating the efficacy and safety of ulipristal acetate in women with abnormal bleeding due to uterine fibroids. A NDA filing for ulipristal acetate is planned for the second half of 2017.

 

AstraZeneca provided an update on its Immuno-Oncology (IO) late -stage clinical development program in 1st-line non-small cell lung cancer (NSCLC), including a refinement of the Phase III MYSTIC trial. The MYSTIC trial was initially designed to assess the benefit of durvalumab monotherapy and durvalumab and tremelimumab (durva + treme) combination therapy versus standard-of-care (SoC) chemotherapy, focused on progression-free survival (PFS).  The MYSTIC trial will now assess PFS and overall survival (OS) endpoints in patients with PDL1-expressing tumours for both durvalumab monotherapy and the combination of durva + treme, as well as in `all comers’ for the combination of durva + treme, versus SoC chemotherapy.  While the focus remains on exploring the benefit of durva + treme as combination therapy, the Company has updated the endpoints of the MYSTIC trial to include OS and PFS in durvalumab monotherapy. This is based on recent internal and external data, including durvalumab’s strong efficacy in monotherapy presented at recent medical meetings, as well as significant opportunities in the competitive landscape. The estimated primary completion date has been updated to reflect both an increase in patient recruitment (as reported in February 2016 with the inclusion of OS as a co-primary endpoint) and the event-based nature of the trial. As a result, the Company anticipates MYSTIC PFS data in mid-2017 and final OS data at the latest in 2018. MYSTIC also includes several undisclosed interim analyses for OS.

 

Benitec Biopharma announced that the European Commission, based on a favorable recommendation from the EMA Committee for Orphan Medicinal Products (COMP), has granted Orphan Drug Designation to BB-301 as an orphan medicinal product for the treatment of patients with oculopharyngeal muscular dystrophy (OPMD).

 

Pluristem Therapeutics announced that Germany’s health regulatory agency, the Paul Ehrlich Institute (PEI), has cleared Pluristem to begin enrollment in Germany for its pivotal Phase III trial of PLX-PAD cells to treat Critical Limb Ischemia (CLI) in patients who are unsuitable for revascularization. The trial was recently cleared by the FDA and the United Kingdom’s Medicines & Healthcare products Regulatory Agency (MHRA).

 

Agenus announced a clinical trial collaboration with the National Cancer Institute (NCI). The double-blind, randomized controlled Phase II trial will evaluate the effect of Agenus’ personalized autologous vaccine candidate, Prophage (HSPPC-96), in conjunction with Merck‘s pembrolizumab on the overall survival rate of patients with newly diagnosed glioblastoma (ndGBM). The trial will be conducted by the Brain Tumor Trials Collaborative (BTTC), a consortium of top academic centers led by Dr. Mark Gilbert, Chief of the Neuro-Oncology Branch at the NCI Center for Cancer Research.

 

Novan announced that the company has entered into an exclusive license agreement with Sato Pharmaceutical, a Japanese company with a prescription pharmaceutical business specializing in dermatology. Upon execution of the agreement, Sato will pay to Novan an initial payment of 1.25 billion JPY (approximately $11.0 million) for the exclusive rights to develop and commercialize in Japan Novan’s topical nitric oxide-releasing product candidate SB204 and related dosage forms for the treatment of acne vulgaris. In addition, Novan will receive from Sato certain development and commercialization milestone payments and, subject to product approval by Japan’s Ministry of Health, Labor and Welfare, certain sales-based milestone payments and a royalty on net sales of such products in Japan.

 

Ophthotech announced that on December 16, 2016, the company announced that it had determined to implement a reduction in personnel to focus on an updated business plan. This reduction in personnel is expected to involve approximately 80% of the company’s workforce and is expected to be substantially complete during the first and second quarters of 2017. In connection with such reduction in personnel, the company estimates that it will incur approximately $14.4 million of pre-tax charges during the first and second quarters of 2017, of which approximately $13.8 million is expected to result in future cash expenditures. These pre-tax charges relate to (a) expected severance and other employee costs of approximately $12.3 million and (b) expected lease termination costs of approximately $2.1 million. The company expects to realize estimated annualized cost savings from the reduction in personnel in the range of $25 million to $30 million starting in the third quarter of 2017.

 

Brien Ragle, CFO of IsoRay, informed the Board of his resignation from all positions held with the Company and its subsidiaries, effective immediately. Even though not obligated to do so, the Company is in negotiations with Mr. Ragle to enter into a Separation Agreement for a to-bedetermined amount of severance pay as well as mutual releases. There is no assurance as to when or if an agreement will be executed with Mr. Ragle. Pending locating an ultimate successor CFO, the Company’s Board of Directors has appointed Matthew Branson as its Principal Financial Officer effective January 17, 2017, to serve at the discretion of the Board. Mr. Branson currently serves as the Controller for the Company’s operating subsidiary, IsoRay Medical, Inc.

 

 

ANALYST RECOMMENDATIONS

 

Kempen analyst Anastasia Karpova increased her valuation of DBV Technologies to €90 from €80, citing adjusted market estimates for more detailed epidemiology data and higher price in the US.

 

Oppenheimer analyst Leah Rush Cann upgraded Agios Pharmaceuticals to “outperform” from “perform,” citing anticipation that AG-120 will launch in 2018, two years earlier than its previous forecasts.

 

Morgan Stanley analyst Matthew Harrison revised his price target of the following companies: Biogen decreased to $353 from $359; DBV Technologies increased to $66 from $64; Gilead decreased to $85 from $91; Galapagos increased to $90 from $89; Alexion’s price target was established at $141.

 

BMO analyst Joanne Wuensch revised her price target of the following companies: Endologix decreased to $7 from $8.50; Dexcom increased to $105 from $90; Nevro decreased to $106 from $110; NuVasive increased to $82 from $70; ResMed decreased to $59 from $61; Wright Medical increased to $27 from $26.

 

Barclays analyst Douglas Tsao downgraded Chiasma to “underweight” from “equal-weight” and decreased his price target to $2.50 from $5.50, citing problems getting Mycapssa to market in US.

 

BMO analyst Do Kim decreased his price target of Ionis Pharmaceuticals to $63 from $68, citing royalties owed to Spinraza patent holders.

 

Stephens analyst Drew Jones increased his price target of Exact Sciences to $18 from $16, citing the Company’s pre-release of solid 4Q results.