BioShares Biotechnology Clinical Trials (BBC): $22.88, +$0.15

BioShares Biotechnology Products (BBP): $34.18, +$0.50

 

 

MARKET COMMENTARY

 

World shares, commodities and bonds rallied, while the dollar weakened after the U.S. Federal Reserve left interest rates unchanged and projected a less aggressive rise in interest rates next year and in 2018. A flurry of economic indicators expected later in the day include initial claims, existing home sales, home price indexes, leading index change and national activity index. The yen touched a four-week high against the greenback. Oil rose after a surprisingly large drop in U.S. crude inventories emboldened investors ahead of next week’s meeting between OPEC members and Russia to discuss supply.

 

 

MARKET HIGHLIGHTS

 

Ocera Therapeutics announced completion of enrollment in STOP-ALF,  a Phase 2a clinical trial to evaluate the Safety and Tolerability of Ornithine Phenylacetate in patients with Acute Liver Failure. The study was conducted by the Acute Liver Failure Study Group, an NIH-sponsored network of university tertiary care liver transplant sites, with support and supply of study medication from Ocera.

 

Rockwell Medical provided an update on the commercial launch of Calcitriol, stating that stability data for completed finished product for commercial sale continues to remain within specification. As a result, the Company anticipates Calcitriol will become commercially available in the U.S. in approximately 45 days. Calcitriol is Rockwell’s FDA approved active Vitamin D injection for the management of hypocalcemia in patients undergoing chronic renal dialysis.

 

Aradigm Corporation announced that the last patient has completed the final dosing visit in the ORBIT-3 and ORBIT-4 Phase 3 clinical studies of Pulmaquin®, Aradigm’s proprietary inhaled ciprofloxacin, an investigational new medication being studied for the treatment of patients with non-cystic fibrosis bronchiectasis (non-CF BE) who have chronic lung infections with Pseudomonas aeruginosa.

 

TiGenix NV announced that it has appointed June Almenoff, M.D., Ph.D., as a member of its Board of Directors in replacement of Dirk Reyn.  June S. Almenoff MD, PhD, is an accomplished pharmaceutical executive with close to 20 years of industry experience. She has extensive expertise in clinical development, translational medicine and business development.

 

Aurinia Pharmaceuticals will participate in a Fireside Chat at the Leerink Partners Roundtable Series: Rare Disease and Immuno-Oncology on September 28.

 

Interleukin Genetics announced their contribution to the key publication in The Lancet (September 21, 2016) of the article “Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomized, double blind, placebo-controlled, dose ranging trials.” The article provides strong evidence that the antisense drug IONIS-APO(a)-LRX is well tolerated and capable of achieving substantial reductions in Lp(a) concentrations in patients. The drug is intended, in part, to reduce the risk for secondary cardiovascular events in individuals with elevated Lp(a).

 

Gemphire Therapeutics will present a corporate overview at the Ladenburg Thalmann 2016 Healthcare Conference on Tuesday, September 27.

 

Mainstay Medical International plc announced its report for the Half Year ended June 30, 2016.  Cash on hand at 30 June was $42.8 million.

 

Gilead Sciences announced that the company is stopping its combined Phase II/III clinical study of GS-5745, an investigational anti-MMP9 antibody, among patients with moderately to severely active ulcerative colitis. This decision follows a planned interim analysis of unblinded efficacy and safety data by the Data Monitoring Committee (DMC) after the first 150 patients of a planned 1600-patient trial were treated for an 8-week induction duration. The DMC recommended that the study be terminated early due to meeting the pre-specified futility and efficacy criteria. No safety concerns were noted in this interim analysis. Gilead has also reviewed the data and determined that there is insufficient evidence of a treatment benefit in the group of patients randomized to receive either one of two doses of GS-5745.

 

Reuters reported that a new drug to treat a rare inherited bone disorder that was developed by Alexion Pharmaceuticals and could cost up to 1.5 million pounds ($2 million) a year per patient has been cleared for use by Britain’s health cost watchdog.

 

Akcea Therapeutics, a wholly-owned subsidiary of Ionis Pharmaceuticals, announced the publication in The Lancet of key clinical results of two randomized, controlled studies of IONIS-APO(a)(Rx) and IONIS-APO(a)-L(Rx) , the company’s Lp(a)-lowering drugs designed to treat cardiovascular disease and aortic valve stenosis. Lipoprotein(a), or Lp(a), is an independent, causal, genetic risk factor for cardiovascular disease and aortic valve narrowing (stenosis). In these studies, substantial Lp(a) reductions of up to 99% were noted, regardless of starting Lp(a) levels. In addition, reductions in low-density lipoprotein-cholesterol – (LDL-C) and pro-inflammatory oxidized phospholipids were observed, as well as a decrease in the inflammatory effects of white blood cells, which can initiate and accelerate cardiovascular disease.

 

Teva Pharmaceutical Industries announced SD-809 (deutetrabenazine) showed statistically significant results in the second Phase III registration trial studying the potential of SD-809 for the treatment of tardive dyskinesia (TD). These new results for the AIM-TD trial follow positive results from the ARM-TD trial announced in June 2015. Both ARM-TD and AIM-TD were 12 week treatment studies. The FDA granted Breakthrough Therapy Designation for SD-809 for the treatment of TD in November 2015. Teva expects to make a regulatory submission to the FDA by the end of 2016.

 

Allergan and Adamas Pharmaceuticals announced that all four dosage strengths of NAMZARIC (memantine and donepezil hydrochlorides) extended-release capsules that allow patients on donepezil 10 mg to start directly on NAMZARIC are now available by prescription in pharmacies throughout the U.S. NAMZARIC is a once-daily, fixed-dose combination of memantine hydrochloride (a NMDA receptor antagonist) and donepezil hydrochloride (an acetylcholinesterase inhibitor, AChEI) for the treatment of moderate to severe Alzheimer’s Disease in patients stabilized on 10 mg of donepezil hydrochloride once daily. NAMZARIC’S new indication and dosage strengths were approved by the FDA in July 2016.

 

PharmaMar announced the submission to the EMA of the MAA for Aplidin (plitidepsin) in combination with dexamethasone for the treatment of relapsed/refractory multiple myeloma (MM). This is a type of blood cancer which represents 10% of all hematological malignancies.

 

Sanaria announced it has received FDA Fast Track designation for its preventative vaccine for malaria, Sanaria PfSPZ Vaccine.  Sanaria believes its PfSPZ Vaccine is the only malaria vaccine to have ever received this distinction.

 

Syros Pharmaceuticals announced that the first patient has been dosed in the Phase II clinical trial of its lead drug candidate, SY-1425, a first-in-class selective retinoic acid receptor alpha agonist, in genomically defined subsets of patients with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) identified using a novel biomarker discovered by its gene control platform.

 

Ligand Pharmaceuticals announced the licensing of rights to four programs to Seelos Therapeutics, a newly formed biopharmaceutical company focused on central nervous system , respiratory and other disorders.

 

VistaGen Therapeutics provided a business outlook including the clinical status of its flagship CNS product candidate, AV-101 (L-4-chlorokyurenine or 4-CI-KYN), currently in Phase II development for the treatment of major depressive disorder (MDD), and an overview of anticipated events and near-term corporate, business, clinical and regulatory milestones expected through the first half of 2017.

 

Kadmon Holdings announced that the first patient has been dosed in a Phase II clinical trial of KD025, the Company’s rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of chronic graft-versus-host disease (cGVHD). The randomized, open-label, 24-week study examines the safety, tolerability and activity of KD025 dosed at 200 mg once daily (QD), 200 mg twice daily (BID) or 400 mg QD in 48 cGVHD patients in the United States.

 

Sarepta Therapeutics announced that it is offering to sell, subject to market and other conditions, up to $225 million of its common stock in an underwritten public offering. Sarepta also intends to grant the underwriters a 30-day option to purchase from it up to an additional 15% of the shares of common stock offered in the public offering.  J.P. Morgan and Goldman Sachs are acting as joint book-running managers of the proposed offering. Credit Suisse is also acting as a joint book-runner. Sarepta intends to use the net proceeds from the offering principally for the continuation and initiation of further clinical trials, commercialization, manufacturing, business development activities including the potential licensing or acquisition of complementary products and technologies and other general corporate purposes.

 

Regen BioPharma has submitted additional data and other responses to the FDA supporting its application requesting Orphan Drug status for the use of HemaXellerate in aplastic anemia.

 

Kleo Pharmaceuticals announced the appointment of Roy Prieb as Chief Operating Officer and Chief Financial Officer effective immediately. Mr. Prieb will also serve on the company’s Board of Directors. He is a co-founder of Kleo Pharmaceuticals and the entrepreneur who secured its technology license from Yale University and organized the company’s series A financing, which was led by Biohaven Pharmaceuticals.

 

PharmaMar announced the submission to the EMA of the MAA for Aplidin (plitidepsin) in combination with dexamethasone for the treatment of relapsed/refractory multiple myeloma (MM). This is a type of blood cancer which represents 10% of all hematological malignancies.

 

Neuraltus Pharmaceuticals announced the initiation of a second Phase II study of its investigational treatment, NP001, in patients with amyotrophic lateral sclerosis in North America. This Phase II study (NCT02794857) is being conducted to confirm previous Phase II clinical study findings in ALS patients with elevated levels of baseline inflammation.

 

Tocagen announced the first patient has been enrolled in Toca 6, a Phase Ib study evaluating intravenously delivered Toca 511 (vocimagene amiretrorepvec) in combination with Toca FC (extended-release 5-fluorocytosine) in patients with metastatic cancers. The study is planned to include patients with colorectal cancer with liver metastases; metastatic renal cell carcinoma; locally advanced or metastatic pancreatic cancer or breast cancer; brain metastases from non-small cell lung cancer or breast cancers; and locally advanced or recurrent melanoma.

 

Charles River Laboratories International announced that it has entered into a strategic partnership agreement with The Tri-Institutional Therapeutics Discovery Institute (Tri-I TDI), intended to accelerate the development of therapeutics that arise from discoveries made in Tri-I TDI’s academic labs.

 

Transgenomic signed a data sharing agreement with Ventana Medical Systems, a subsidiary of Roche Holdings. The agreement allows Ventana to access DNA test results from an existing research agreement between Transgenomic and the University of Melbourne in Australia. As part of this research agreement, the University of Melbourne is conducting additional clinical validation studies of Transgenomic’s Multiplexed ICE COLD-PCR(TM) (MX-ICP) technology.

 

SYGNIS AG announced that Navicyte Scientific, part of SYGNIS Group in the US, and the European Collection of Authenticated Cell Cultures (ECACC), part of Public Health England, an agency of the United Kingdom Department of Health have entered into a collaboration agreement for licensing and distributiing the Caco-2 cell line to commercial companies. Under the collaboration, ECACC will organize that companies obtain the license for the use of the Caco-2 cell line from SYGNIS in the US  and afterwards will provide the Caco-2 materials directly.

 

OrganiGram Holdings announced that it has successfully amended its Health Canada licensed sales capacity of dried marijuana to 1,200kg per annum. Additionally, Health Canada also increased OrganiGram’s licensed production and sales capacity for cannabis oils to 500kg per annum.

 

Egalet announced that results from a Category III oral human abuse potential (HAP) study and a Category 3 intranasal HAP study of product candidate ARYMO ER (morphine sulfate) extended-release tablets have been published in Pain Medicine, the official journal of the American Academy of Pain Medicine. These two primary publications of the oral and intranasal HAP studies report that, after manipulation and administration via the oral and intranasal routes, respectively, ARYMO ER had a significant reduction in maximum drug liking compared to crushed MS Contin (morphine sulfate extended-release tablets) CII and placebo.

 

Antibe Therapeutics continued to demonstrate the anti-cancer potential of its lead drug, ATB-346. In a separate study earlier this year, ATB-346 was shown to be very effective in reversing colon and intestinal tumour growth in mice. Further to this study, a new publication has shown promising results in the chemoprevention and treatment of melanoma in mice. Melanoma is one of the most drug-resistant and invasive types of cancer, and both the incidence and mortality rates are increasing.

 

Impax Laboratories announced the launch of a generic version of Metadate CDR) (methylphenidate hydrochloride extended-release capsules), 10 mg, 20 mg, 30 mg, 40 mg, 50 mg, and 60 mg.

 

Lannett Company announced the resignation of its chief of scientific affairs, Mahendra Dedhiya, Ph.D., effective October 21, 2016. 

 

Navidea Biopharmaceuticals announced it will receive payments totaling $1 million from two recently achieved Lymphoseek commercial milestones under its distribution agreements with U.S. partner Cardinal Health and European partner SpePharm AG, an affiliate of Norgine B.V. (Norgine). Navidea will collect a $500,000 milestone payment from Cardinal based on the sale of a 100,000th patient dose of Lymphoseek (technetium Tc 99m tilmanocept) injection since launch. The Company will also receive a $500,000 payment from Norgine resulting from the EMA CHMP positive opinion for the Lymphoseek 50 microgram kit for radiopharmaceutical preparation, a reduced-mass, single-dose vial appropriate for the radiopharmaceutical distribution model in Europe.

 

CTI BioPharma announced that on September 20, 2016, the NASDAQ Listing Qualifications staff granted the Company an additional 180 calendar day period, or until March 20, 2017, to regain compliance with the Minimum Bid Price Rule. To do so, the bid price of the Company’s common stock must close at or above $1.00 per share for a minimum of 10 consecutive trading days prior to that date.

 

 

ANALYST RECOMMENDATIONS

 

LifeSci Capital analyst Jerry Isaacson, Ph.D., initiated coverage of Kitov Pharmaceuticals.

 

Credit Suisse analyst Kennen MacKay increased his price target of Puma Biotechnology to $111 from $54, citing increased sales expectations for neratinib in the HER2+ extended adjuvant setting.

 

Maxim analyst Gabrielle Zhou initiated coverage of the following companies: Cellular Biomedicine with a “buy” rating and $16 price target; Beigene with a “buy” and $41 price target; Casi Pharmaceuticals with a “buy” and $4 price target.

 

Roth analyst Mark Breidenbach initiated coverage of Kiadis Pharm with a “buy” rating and €17.00 price target, citing Kiadis has zeroed in on an area of high unmet medical need and is planning to take its lead program, ATIR-101 into a well-designed Phase 3 trial later this year.

 

Roth analyst Mark Breidenbach assumed coverage of bluebird bio with a “buy” rating and $87 price target, citing the company can no longer be considered a fledgling given the clinical success seen in its Lenti-D and Lentiglobin programs, both hatched from bluebird’s versatile core lentiviral technology platform.

 

Roth analyst Mark Breidenbach initiated coverage of Fate Therapeutics with a “buy” rating and $8 price target, citing the company’s lead asset, ProTmune, could address a serious unmet medical need in patients requiring hematopoietic transplants and expect to see early clinical data by the end of 2016.

 

HC Wainwright analyst Swayampakula Ramakanth initiated coverage of Proteon Therapeutics with a “buy” and $18 price target, citing the company is developing a novel therapy to help prolong vascular access patency for dialysis patients.

 

HC Wainwright analyst Shaunak Deepak initiated coverage of Oncomed with a “buy” rating and $20 price target, citing upcoming opt-in decisions for its lead drug candidates expected from its three partners Celgene, Bayer, and GlaxoSmithKline.