BioShares Biotechnology Clinical Trials (BBC): $22.45, -$0.06, -22.7% YTD
BioShares Biotechnology Products (BBP): $33.08, -$0.08, +7.3% YTD
U.S. stock index futures edged down, taking cues from European markets on speculation that the European Central Bank would eventually be winding down its bond-buying stimulus program. Investors await a flurry of economic indicators, including ADP’s employment data, trade figures and Markit’s PMI data amongst others. Asian stocks closed mixed. Britain’s pound traded below $1.27 for the first time since 1985, and a five-year low versus the euro. Gold held steady. Oil advanced, hitting its highest since June, supported by American Petroleum Institute’s report that U.S. inventories likely fell for a fifth straight week and OPEC’s deal to cut supply.
VBI Vaccines is scheduled to present at the 17th Annual World Vaccine Congress Europe on Monday, October 10, 2016 at 9:45am Eastern Time. The event is being held at the Fairmont Rey Juan Carlos I in Barcelona, Spain.
CymaBay Therapeutics announced that after receiving input from the FDA, it has determined the next step in the development of MBX-8025 in subjects with primary biliary cholangitis (PBC). MBX-8025 is an orally administered potent and selective peroxisome proliferator-activated receptor delta agonist. An initial Phase II study of MBX-8025 in subjects with primary biliary cholangitis was terminated earlier this year when proof-of-concept was demonstrated by marked improvements in biochemical markers of cholestasis, including alkaline phosphatase (ALP), a surrogate biomarker that has been linked to clinical outcomes in PBC. The study also identified a treatment emergent signal of transaminase elevations. The magnitude of the ALP reductions in the study suggested that lower doses may retain ALP reductions while avoiding transaminase elevations. After discussions with regulatory authorities, the company will be initiating a second Phase II study that will investigate lower doses of MBX-8025 in subjects with PBC to optimize the response. Initiation of the study is expected by year end 2016.
BioDelivery Sciences International announced additions to its Board of Directors. Timothy C. Tyson will join the BDSI Board of Directors as an independent member, and Dr. Mark A. Sirgo, BDSI’s President and Chief Executive Officer and board member, will, in addition to his current positions, assume the role of Vice Chairman. Both changes are effective immediately and help to further strengthen BDSI’s existing board-level leadership.
DelMar Pharmaceuticals published a new blog post on The Chairman’s Blog, written by the Company’s Chairman and Chief Executive Officer, Jeffrey Bacha. TheChairmansBlog.com is an exclusive online media publication that enables key executive officers a unique platform to share insights about their company and industry trends. In his new blog, Mr. Bacha highlights DelMar’s focus on targeting DNA repair mechanisms to treat cancers that show resistance to currently available types of chemotherapy.
Anavex Life Sciences announced a collaboration with Ariana Pharma to use Ariana’s proprietary KEM (Knowledge, Extraction, Management) patient stratification technology to potentially accelerate ANAVEX 2-73′s Phase II/III Alzheimer’s clinical development timelines. KEM is a comprehensive and FDA-tested clinical data analysis system that enables full exploitation of complex datasets including of smaller numbers of patients.
Genentech, a member of the Roche Group, announced that the FDA has granted Breakthrough Therapy Designation status to Actemra (tocilizumab) for giant cell arteritis (GCA), a chronic, potentially life-threatening autoimmune condition. The disease is caused by inflammation of large and medium-sized arteries, most often in the head, but also in the aorta and its branches.
RedHill Biopharma announced that a Phase II clinical study evaluating YELIVA (ABC294640) in patients with advanced hepatocellular carcinoma (HCC) has been initiated, with enrollment expected to commence shortly, pending final regulatory clearance.
Collegium Pharmaceutical announced that it has submitted a sNDA to the FDA to enhance the label for Xtampza ER (oxycodone extended-release), an abuse-deterrent, extended-release opioid, for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate.
Fate Therapeutics announced that the USPTO issued U.S. Patent No. 9,452,186 covering enhanced hematopoietic stem cell compositions. This newly-issued patent expands the Company’s significant intellectual property position covering ex vivo small molecule modulation of hematopoietic stem cells, which includes compositions and methods for enhanced lentiviral transduction and engraftment in the field of hematopoietic cell transplantation. The Company also has filed corresponding patent applications to seek similar patent protection for its hematopoietic stem cell compositions in key markets throughout the world, including Europe and Japan.
Boehringer Ingelheim Vetmedica announced an agreement with Elanco U.S., a subsidiary of Eli Lilly, to sell a portfolio of U.S. canine, feline and rabies vaccines, as well as a fully integrated manufacturing and R&D site, to Elanco. The sale price of $885 million includes the estimated cost of acquired inventory. The deal is conditioned on antitrust approval and closing of the Boehringer Ingelheim (BI) asset swap transaction with Sanofi that was signed in June 2016.
Lexicon Pharmaceuticals announced that it has entered into an agreement to buy out its remaining obligations under its clinical development financing arrangement with Symphony Icon Holdings upon regulatory approval in the United States for the marketing and sale of telotristat ethyl.
Capricor Therapeutics announced that its Phase II ALLSTAR clinical trial has completed patient enrollment. ALLSTAR (ALLogeneic Heart STem Cells to Achieve Myocardial Regeneration) is evaluating CAP-1002 (allogeneic cardiosphere-derived cells, or CDCs) in adults with cardiac dysfunction following a large heart attack (myocardial infarction, or MI).
AstraZeneca announced new data demonstrating that respiratory syncytial virus hospitalization (RSVH) rates increased significantly during the 2014-2015 RSV season among US infants <3 months of age born at 29-34 weeks gestational age (wGA), as compared to the 2013-2014 season. RSV is a contagious, seasonal respiratory virus that can lead to a serious lung infection and hospitalization in high-risk babies, particularly preterm infants. These data are based on data from over 2.2 million US infants and represent the first and only analysis conducted using national US Medicaid and Commercial insurance claims databases to further understand the changes in both immunoprophylaxis (IP) utilization and RSVH rates. This research was presented as a poster (poster number J14) at the annual Academy of Managed Care Pharmacy (AMCP) Nexus meeting in National Harbor, MD.
Abeona Therapeutics announced that the Data Safety Monitoring Board (DSMB) has reviewed the initial safety data from the low dose cohort (n=3) in the Phase I/II clinical trial of ABO-102 (AAV-SGSH) enrolling at Nationwide Children’s Hospital in Columbus, Ohio. Following review of the safety data, the DSMB authorized that the clinical trial proceed with enrollment and dose escalation for the second cohort. The high-dose cohort will enroll up to six additional patients dosed at 1.0 X 10(13) vg/kg, which is twice the amount of ABO-102 received by patients in the low-dose cohort.
Teligent announced that its Estonian subsidiary, Teligent OÜ, has received approval from Health Canada’s Therapeutic Products Directorate for Lidocaine Ointment USP 5%. This is the sixth approval for Teligent OÜ this calendar year and will be distributed by Teligent’s business in Canada.
Repligen announced the commercial launch of its single-use version of XCell ATF, a proprietary cell retention system used by biologic drug manufacturers to intensify cell culture and improve the efficiency and productivity of upstream processes. This innovative new single-use format, which will be offered alongside the existing stainless steel line, significantly reduces implementation time and improves overall ease of use for Repligen’s biopharmaceutical manufacturing customers. The new XCell ATF format aligns with single-use and continuous processing workflows that biomanufacturers are increasingly adopting for their flexibility, convenience and cost-saving advantages.
DiaMedica announced that the first patient has been dosed in the Phase Ib clinical trial of DM199, a novel recombinant tissue kallikrein ("rKLK1") protein for the development of neurological and kidney diseases.
Opiant Pharmaceuticals announced that NARCAN Nasal Spray was granted approval by Health Canada.
International Stem Cell Corporation announced that it has published the results of its preclinical safety studies. The data from a series of in vitro and in vivo preclinical studies show that the company’s proprietary ISC-hpNSC readily expandable neural stem cells are devoid of undifferentiated pluripotent stem cells and are safe for transplantation. The full manuscript titled, "Neural Stem Cell Tumorigenicity and Biodistribution Assessment for Phase I Clinical Trial in Parkinson’s Disease," was published in the September 30 issue of Scientific Reports, a multidisciplinary journal from the publishers of Nature.
Ocular Therapeutix announced the enrollment of the first patient in the first of two planned Phase III clinical trials with OTX-TP (sustained release travoprost) for the treatment of glaucoma and ocular hypertension. OTX-TP is a preservative-free drug product candidate that resides within the canaliculus and is designed to deliver the prostaglandin analog travoprost to the ocular surface for up to 90 days. The U.S.-based, prospective, multicenter, randomized, parallel-arm, placebo-controlled study is expected to enroll approximately 550 patients with open angle glaucoma or ocular hypertension at 50 clinical sites. Importantly, the Phase III study design will not include a timolol comparator or validation arm, and will not have active or placebo eye drops administered in either arm. The comparator arm will utilize a non-drug eluting hydrogel-based intracanalicular insert. The primary efficacy endpoint will be statistically superior reduction of intraocular pressure (IOP) from baseline with OTX-TP compared to placebo at 2, 6 and 12 weeks following insertion.
Cascadian Therapeutics announced that its board of directors has approved a plan for a reverse split of the Company’s common stock to increase its share price and reduce the number of authorized and outstanding shares.
Cellectis announced the issuance of U.S. patent 9,458,439 – which claims gene inactivation by use of chimeric restriction endonucleases. This patent granted by the USPTO to the Institut Pasteur and Boston Children’s Hospital naming Dr. André Choulika and Pr. Richard C. Mulligan as co-inventors, is exclusively licensed to Cellectis. This issued U.S. patent 9,458,439 claims the method of introducing chromosomal modifications at a locus by induction of double-stranded DNA cleavage using a chimeric restriction endonuclease and non-homologous end joining recombination (NHEJ). This pivotal invention is at the basis of current nuclease-based precise gene inactivation techniques using chimeric restriction endonuclease such as Cas9/CRISPR (and related families), Zinc finger Nucleases, TAL-Effector Nucleases, Mega-TALEs, some Meganucleases i.e. endonucleases generated by the juxtaposition of specific DNA binding sequences and DNA cleavage domains with a recognition site of at least 12 base pairs. This technology is universal as it can be applied to any types of cells, including human, animal, plant cells or microorganisms.
Karus Therapeutics announced that the first patients have been dosed in a Phase I study for its lead candidate, KA2237.
SastoMed GmbH has announced that the British National Health Service (NHS) has taken the wound spray Granulox licensed by Sangui to SastoMed GmbH on the list of reimbursable products under a discrete category (NHS Drug Tariff Part IX) effective October 01, 2016. The NHS provides to each resident of the UK medical care in the primary (general practitioner GP) and secondary level (hospitals) free of charge. Furthermore, in a declaration recently published the Scottish Health Technologies Group (SHTG – a division of NHSScottland) has clearly recommended the application of Granulox to all doctors working for NHSScottland because of Granulox’ efficacy and marked cost savings of several thousand British pounds proved by several studies.
Innovus Pharmaceuticals announced the initiation of a pre-clinical and clinical program intended to evaluate the safety and efficacy of the combination of its supplement Vesele for promoting sexual health with sildenafil indicated for treating erectile dysfunction. Sildenafil in the U.S. is sold under the name Viagra by Pfizer.
Karyopharm Therapeutics announced that an oral presentation highlighting updated clinical data from the Company’s ongoing Phase II study of selinexor (KPT-330), its lead, novel, oral Selective Inhibitor of Nuclear Export / SINE compound that inhibits exportin 1 (XPO1), for the treatment of gynecological cancers (the SIGN study) will be presented at the European Society of Medical Oncology (ESMO) 2016 annual meeting being held October 7-11, 2016 in Copenhagen, Denmark. Two posters featuring predictive biomarker data supporting selinexor’s activity in gynecological and colorectal cancers will also be presented. In the oral presentation, titled “Results of a Phase II Trial of Selinexor, an Oral Selective Inhibitor of Nuclear Export (SINE) in 114 Patients with Gynecological Cancers,” Ignace B. Vergote, MD, PhD, Head of the Department of Gynecologic Oncology, Catholic University of Leuven, Belgium, and lead investigator of the SIGN study, will describe clinical data from the study, which includes durable anti-cancer activity in gynecological malignancies with disease control rates up to 49% and good tolerability.
Insmed announced a licensing agreement with AstraZeneca for global exclusive rights to AZD7986, a novel oral inhibitor of dipeptidyl peptidase I (DPP1, also known as cathepsin C). DPP1 is an enzyme that catalyzes the activation of neutrophil serine proteases (NSPs), which play a key role in pulmonary diseases such as non-cystic fibrosis bronchiectasis (non-CF bronchiectasis).
Reuters reported that Novartis said it was shuttering research centers in Switzerland and China, part of the Swiss drug maker’s bid to trim some costly locations and centralize control over its drug discovery programs.
Catabasis Pharmaceuticals announced the promotion of Andrew Nichols, Ph.D. to Chief Scientific Officer and Angelika Fretzen, Ph.D., to Senior Vice President of Product Development.
Regulus Therapeutics announced the appointment of Timothy Wright, M.D. as Chief Research and Development Officer reporting to Paul Grint, M.D., President and CEO.
Immunomedics announced that it has priced an underwritten offering of 10,000,000 shares of common stock and warrants to purchase up to 10,000,000 shares of common stock with anticipated gross proceeds of $30 million. Each share of common stock and accompanying warrant will be priced at $3.00. The warrants will be exercisable six months following the date of issuance, will expire on the second anniversary of the date of issuance and have an exercise price of $3.75. Immunomedics intends to use the proceeds from the offering primarily in continuing support of clinical and regulatory activities for IMMU-132, including submission of a NDA to FDA for accelerated approval in metastatic triple-negative breast cancer, and for working capital and general corporate purposes. Wells Fargo Securities is acting as sole book-running manager. The closing of the offering is subject to certain customary conditions, and is expected to occur on or about October 11, 2016.
Actinium Pharmaceuticals announced the closing of its previously announced underwritten public offering of 8,000,000 shares of the Company’s common stock. The gross proceeds to Actinium from this offering is $10,000,000. Actinium currently intends to use the net proceeds from the sale of securities for general corporate purposes, including capital expenditures, the advancement of its drug candidates in clinical trials, such as Iomab-B and Actimab- A, preclinical trials, and to meet working capital needs. H.C. Wainwright & Co. acted as sole book-running manager for the offering. JonesTrading Institutional Services acted as co-manager for the offering. Roth Capital Partners acted as a financial advisor for the offering.
SunTrust analyst Yatin Suneja initiated coverage of ARIAD Pharmaceuticals with a “buy” rating and $22 price target, citing shares should outperform on clinical and/or regulatory newsflow.
Cantor Fitzgerald analyst Elemer Piros initiated coverage of Corbus Pharmaceuticals with a “buy” rating and $17 price target, citing anticipation that Corbus will achieve three clinical inflection points during the next 12 months, beginning with the first in November around the American College of Rheumatology (ACR) meeting.
Cantor Fitzgerald analyst Chiara Russo initiated coverage of Amarin with a “buy” rating and $6 price target, citing a potential positive read-out from the REDUCE-IT outcomes study expected in late 2017 could not just expand the indicated patient population of ~4M to ~74M, but also for the first time have an outcomes study that draws a straight line between triglyceride levels and cardiovascular risk, potentially changing how cardiovascular risk is managed and treated and creating a multi-billion dollar market opportunity.
HC Wainwright analyst Carol Ann Werther increased her price target of Ligand Pharmaceuticals to $160 from $146, citing partner Retrophin announced the Sparsentan’s Phase 2 DUET study met the primary efficacy endpoint for the overall treatment group in focal segmental glomerulosclerosis, LGND began a Phase 2 clinical trial with LGD-6972, a glucagon receptor antagonist, for the treatment of type 2 diabetes, and LGND licensed several therapeutic programs to a startup company Seelos.
Jefferies analyst Brian Abrahams made price target changes to the following companies ahead of 3Q earnings: Biogen to $316 from $323; Celgene to $134 from $137; Gilead to $93 from $91; Incyte to $109 from $98; Vertex to $105 from $104.
Piper Jaffray analyst Charles Duncan upgraded Catalyst Pharmaceuticals to “overweight” from “neutral” and increased his price target to $4 from $1, citing improved visibility on Firdapse’s timeline to NDA re-submission.
Goldman analyst Robert Jones resumed coverage of Quintiles and added the company to the “Buy List” with a $94 price target, citing modest improvements in win and conversion rates will result in upside to estimates/guidance.