BioShares Biotechnology Clinical Trials (BBC): $22.73, +$0.48, -21.8% YTD

BioShares Biotechnology Products (BBP): $33.68, +$0.64, +9.3% YTD

 

 

MARKET COMMENTARY

 

Global stocks advanced, led by a surge in bank shares, after the Bank of Japan surprised markets by adopting a target for long-term interest rates and as focus shifted towards the U.S. Federal Reserve policy decision due later in the day. Red Hat, Bed Bath and Beyond and Jabil Circuit are scheduled to report their results after the closing bell. The Japanese yen recovered from lows as markets turned skeptical about whether the BoJ’s latest measures will be enough to generate inflation and the dollar edged lower. Gold ticked higher and is poised for further gains if the Fed keeps rates unchanged in accordance with market expectations. Oil prices jumped after a surprisingly large drop in U.S. crude inventories.

 

 

MARKET HIGHLIGHTS

 

Aimmune Therapeutics announced early completion of North American target enrollment in its ongoing Phase 3 PALISADE trial of AR101. The company also announced that RAMSES (Real-World AR101 Market-Supporting Experience Study), a new clinical study designed to bolster real-world experience with AR101, will begin enrolling patients in 1Q 2017. AR101 is Aimmune’s biologic oral immunotherapy for desensitization of patients with peanut allergy.  This enrollment milestone was achieved approximately three months ahead of schedule. PALISADE enrollment in Europe is ongoing and is expected to complete by the end of the year. Accordingly, the company is reaffirming its expectations that topline data from PALISADE will be available in 4Q 2017, followed by regulatory submissions for marketing approval of AR101 in both the United States and Europe in 2018.

 

ABIVAX announced its half-year financial results as of June 30 2016.  The company reported a net loss 8.3 million as of June 30, 2016, compared with7.2 million as of June 30, 2015.  As of June 30, 2016, the company had 1.1 million available cash plus 25.0 million of fixed-term investments and €2.0 million of unit trust/UCITS funds. ABIVAX continues to expect that its financial resources will support the company’s operations through the end of 2017.

 

Aurinia Pharmaceuticals will host a Lupus Nephritis expert breakfast on Friday, September 30th in New York.

 

Aradigm Corporation will host an Analyst Lunch on the topic of Non-Cystic Fibrosis Bronchiectasis on Monday, September 26. The event will feature presentations by two key opinion leaders: David E. Griffith, MD and Gregory Tino, MD.

 

BioDelivery Sciences International will present at the Ladenburg Thalmann 2016 Healthcare Conference on Tuesday, September 27.

 

Mateon Therapeutics will present an overview of the company at the Ladenburg Thalmann 2016 Healthcare Conference on Tuesday, September 27.

 

Aimmune Therapeutics announced early completion of North American target

Akarna Therapeutics announced that it has been acquired by Allergan. Allergan obtains rights to AKN-083, Akarna’s lead product candidate for the potential treatment of non-alcoholic steatohepatitis (NASH) and other liver diseases. Under the terms of the agreement, Allergan acquired Akarna for an upfront cash payment of $50 million and success-based development, regulatory and sales milestones.

 

Cerecor announced that it has completed patient enrollment in its Phase II clinical trial for CERC-301, Clin301-203, as an oral, rapidly acting adjunctive treatment of major depressive disorder (“MDD”). The Company now expects to report top-line data from this trial in November 2016.

 

Adamas Pharmaceuticals announced the results of the EASE LID 3 study, the second Phase III trial of ADS-5102 (amantadine hydrochloride) extended-release capsules for the treatment of levodopa-induced dyskinesia (LID) in patients with Parkinson’s disease (PD). The trial met its primary endpoint, the reduction of the UDysRS (Unified Dyskinesia Rating Scale) total score at week 12 (p<0.0001), reflecting a decrease in the duration, intensity and disability associated with LID. In addition, a statistically significant reduction in OFF time (a key secondary endpoint) was observed, as measured by patient reported home diaries. A separate post hoc analysis of data from the EASE LID trial was also announced, reporting the impact LID has on activities of daily living (ADLs) of PD patients as well as the favorable effects of ADS-5102 on these ADLs.

 

BiondVax Pharmaceuticals announced that the last participant in a European Phase IIb clinical trial of M-001, its universal flu vaccine candidate, has completed their final visit. Conducted in collaboration with the EU-sponsored UNISEC consortium, the trial is designed to evaluate the safety and immunogenicity of M-001 when used ahead of a pandemic influenza vaccine.

 

BioMarin Pharmaceutical announced that it intends to seek a review of the Patent Trial and Appeal Board (PTAB) of the United States ruling in Interference No. 106,008, related to composition of matter (COM) claims related to exon 51 skipping antisense oligonucleotides.  BioMarin is completing its review of the decision and the specific means it may use to seek a further review.

 

Mirna Therapeutics announced its decision to close the ongoing Phase I study of MRX34, its investigational microRNA therapy for multiple cancers. The Company voluntarily halted enrollment and dosing in the clinical study following multiple immune-related severe adverse events (SAE) observed in patients dosed with MRX34 over the course of the trial. Following the news, Mirna Therapeutics’ announcement the company is closing its Phase 1 study of MRX34, Oppenheimer analyst Michelle Gilson downgraded the stock to “perform” from “outperform;” Leerink analyst Michael Schmidt decreased his price target to $1.50 from $4.00; Cantor analyst Mara Goldstein downgraded the stock to “hold” from “buy” and decreased her price target to $2 from $14.

 

 

Kite Pharma announced that the Company has entered into an exclusive, worldwide license with the National Institutes of Health (NIH) for intellectual property related to multiple T-cell receptor (TCR) based product candidates for the treatment of tumors expressing mutated KRAS antigens.  Kite expects the first of the licensed KRAS product candidates to enter clinical study in 2016 as part of the Company’s Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) under the direction of Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI.

 

Janssen Pharmaceuticals announced the FDA has approved INVOKAMET XR—a once-daily, fixed-dose combination therapy of canagliflozin and metformin hydrochloride extended-release (XR)—for first-line use as an adjunct to diet and exercise to improve blood glucose control in adults with type 2 diabetes when treatment with the two medications is appropriate.1 INVOKAMET XR combines INVOKANA (canagliflozin), the most prescribed sodium glucose co-transporter 2 (SGLT2) inhibitor, with more than 9 million U.S. prescriptions since launch,2 and an XR formulation of metformin. Metformin is commonly prescribed as an initial therapy for the treatment of type 2 diabetes.

 

bluebird bio announced that the EMA has granted access to its Priority Medicines (PRIME) scheme for LentiGlobin drug product in the treatment of patients with transfusion-dependent beta-thalassemia (TDT).

 

Cidara Therapeutics announced that the company has selected the first development candidate from its proprietary immunotherapy discovery platform, Cloudbreak. Cloudbreak is the first platform of its kind designed specifically to create compounds that direct a patient’s immune cells to attack and eliminate bacterial, fungal or viral microbial pathogens. The selected candidate, CD201, is a novel, bispecific antimicrobial immunotherapy being developed for the treatment of multi-drug resistant (MDR) bacterial infections, including those caused by MCR-1 resistant pathogens.

 

Neuralstem announced that it has reached over 50% enrollment in its Phase II clinical trial evaluating NSI-189, a novel neurogenic small molecule, for the treatment of major depressive disorder (MDD).

 

Dermira announced it has entered into an exclusive license agreement with Maruho Co., a Japanese company specializing in dermatology. Pursuant to the terms of the agreement, Dermira has granted Maruho an exclusive license to develop and commercialize DRM04, a topical anticholinergic product candidate currently being developed by Dermira for the treatment of primary axillary hyperhidrosis (excessive underarm sweating), in Japan.

 

Five Prime Therapeutics announced the promotions of Aron Knickerbocker to Chief Operating Officer, Francis Sarena to Chief Strategy Officer and Luis Borges to Senior Vice President, Research.

 

Mast Therapeutics reported top-line results from EPIC, a Phase III clinical study of its investigational new drug vepoloxamer (also known as MST-188) for the treatment of individuals with sickle cell disease experiencing vaso-occlusive crisis (VOC). The study did not meet its primary efficacy endpoint of demonstrating a statistically significant reduction in the mean duration of VOC (82 hours in the vepoloxamer group compared to 78 hours in the placebo group in the intent-to-treat population (p=0.09)). There were no statistically significant differences between treatment groups in the intent-to-treat population across the two secondary efficacy endpoints, rate of re-hospitalization for VOC and the occurrence of acute chest syndrome. Consistent with previously conducted studies, vepoloxamer was generally well tolerated with no statistically significant differences in treatment-related serious adverse events in the vepoloxamer group compared to the placebo group. No deaths occurred on the study.

 

Sarepta Therapeutics announced that the Patent Trial and Appeal Board (PTAB) of the USPTO has issued two favorable decisions for Sarepta in the composition of matter patent interferences for exon 51 (Interference No. 106,008) and exon 53 (Interference No. 106,007). These decisions, subject to appeal, finally refused all of BioMarin’s claims in the exon 51 and exon 53 composition of matter interferences that, if granted, could have formed a basis for a claim of infringement against eteplirsen and SRP-4053. The PTAB decision for exon 53 allows BioMarin to obtain a narrow composition of matter claim, however, SRP-4053 does not infringe this claim. The PTAB ended the exon 51 interference in our favor based on a statute of limitations bar.

 

Takeda Pharmaceutical and Affilogic jointly announced that the companies have entered into a research collaboration to explore using Affilogic’s proprietary Nanofitins platform in therapies targeting the central nervous system. Specifically, Affilogic and Takeda, through its research center in San Diego, California, will leverage their respective competencies to validate and optimize Nanofitins that enable Takeda to deliver biotherapeutic candidates into the brain to address neurological disorders.

 

Cancer Genetics announced that it has entered into a partnership with the Bio Analytical Research Corporation.

 

AstraZeneca announced its decision to withdraw the MAA submitted to the EMA’s CHMP in June 2015 for cediranib in combination with platinum-based chemotherapy followed by maintenance monotherapy for the treatment of adult patients with platinum-sensitive relapsed ovarian cancer (including fallopian tube or primary peritoneal sub types).

 

Epizyme announced that the Company has entered into a collaboration agreement with Foundation Medicine to support patient identification and enrollment for Epizyme’s ongoing Phase II clinical trial of tazemetostat in patients with non-Hodgkin lymphoma (NHL). Foundation Medicine’s SmartTrials Precision Enrollment Program and FoundationOne Heme panel will assist in identifying a population of individuals with NHL who harbor EZH2 mutations and constitute specific cohorts in the Epizyme trial.

 

Eleven Biotherapeutics and Viventia Bio announced that the two companies and the shareholders of Viventia entered into a definitive share purchase agreement under which Eleven Biotherapeutics agreed to, and simultaneously completed, the acquisition of Viventia. Under the agreement, Eleven purchased all of the outstanding capital stock of Viventia in exchange for the issuance of 4,013,431 newly issued shares of Eleven common stock, which represented approximately 19.9% of the voting power of Eleven as of immediately prior to the issuance of such shares, and the agreement by Eleven to pay to the selling shareholders certain post-closing contingent cash payments upon the achievement of specified milestones and based upon net sales related to Viventia’s lead product candidate, Vicinium.

 

Apricus Biosciences announced the launch of Vitaros, Apricus’ novel on-demand topical cream for the treatment of erectile dysfunction (ED), in the Czech Republic and Slovakia by Recordati.

 

MyoKardia will highlight the expected path to registration for its lead product candidate MYK-461 in the initial indication of symptomatic oHCM and its plans to progress MYK-491 into the clinic for DCM. The Company’s scientific leadership will also provide further insights into MyoKardia’s precision medicine and pipeline strategy enabled by its product engine. The updates will be presented at MyoKardia’s inaugural R&D Day in New York.

 

Abeona Therapeutics announced the exclusive worldwide license of a next generation gene therapy AAV capsid portfolio from University of North Carolina at Chapel Hill. The AIM vector system is a next generation platform of AAV capsids capable of widespread central nervous system gene transfer and can be used to confer high transduction efficiency for various therapeutic indications. Studies indicate that AIM vectors can efficiently and broadly target CNS tissue, and may provide a treatment for patients that have inhibitory antibodies to natural AAV serotypes. Importantly, the AIM vector system may provide second-generation treatment approaches for patients that have received a previous AAV injection.

 

The Medicines Company announced that it has entered into a new strategic partnership with the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response within the U.S. Department of Health and Human Services (HHS), that will provide the Company with up to $132 million to support the development of new antibiotics to fight drug-resistant, gram-negative infections. The partnership was established under HHS’s Other Transactional Authority (OTA) and is a distinctive, flexible, portfolio-based approach to funding drug development. It is the first portfolio-based partnership between BARDA and a U.S. headquartered private-sector company.

 

Mucodel Pharma announced the successful completion of a pilot clinical study involving buccal naloxone (Exonal), a Mucodel product under development for the reversal of opioid overdose.

 

IntelGenx and RedHill Biopharma announced that they have entered into a binding term sheet agreement with Pharmatronic, granting Pharmatronic the exclusive license to commercialize RIZAPORT in the republic of Korea (South Korea). RIZAPORT is a proprietary oral thin film formulation of rizatriptan for the treatment of acute migraines.

 

SIGA Technologies announced that the United States Bankruptcy Court for the Southern District of New York entered an order approving SIGA’s proposed transaction under Option 1 of the Debtor’s Third Amended Chapter 11 Plan, dated April 7, 2016, under which SIGA will pay PharmAthene the entire balance of the outstanding judgment on or before November 30, 2016.

 

Agios Pharmaceuticals announced the closing of its previously announced underwritten public offering of common stock, including the exercise in full by the underwriters of their option to purchase an additional 505,617 shares at the public offering price of $44.50 per share. The exercise of the option to purchase additional shares brought the total number of shares of common stock sold by Agios to 3,876,403 shares and increased the amount of gross proceeds raised in the offering, before underwriting discounts  and estimated expenses of the offering, to approximately $173 million. J.P. Morgan Securities and Goldman Sachs acted as joint book-running managers for the offering. Cowen served as lead manager.

 

CareDx announced the pricing of its public offering of 2.25 million shares of its common stock at a price to the public of $4.00 per share. In addition, the Company has granted the underwriters a 30-day option to purchase up to 337,500 additional shares of its common stock. All of the shares will be offered and sold by CareDx.

 

Insys Therapeutics announced that the Company’s Board of Directors has initiated a search to identify a candidate to succeed Dr. John N. Kapoor as President and Chief Executive Officer. Dr. Kapoor will continue to serve as President and CEO until his replacement is appointed. The Board has appointed a special committee, comprised of independent Board members, Patrick P. Fourteau, Steven Meyer and Pierre Lapalme, to conduct the search for the Company’s next CEO.  These board members are, respectively, the chairs of the compensation committee, audit committee and nominating and corporate governance committee of the Board.

 

The Board of Directors of Sarepta Therapeutics appointed Edward M. Kaye, M.D. as President and Chief Executive Officer of the Company and to serve as a member of the Board. Dr. Kaye will continue in his role as Chief Medical Officer, a role which he has held since June 2011. Prior to his new appointment Dr. Kaye, 67, served as the Company’s Interim Chief Executive Officer since March 31, 2015. Dr. Kaye serves as a member of the Board of Directors of Cytokinetics, Inc. since May 2016 and has held various executive and officer positions including being Group Vice President of Clinical Development at Genzyme Corporation, a biotechnology company, from April 2007 to June 2011.

 

 

ANALYST RECOMMENDATIONS

 

Guggenheim suspended coverage of the following Biotechnology companies due to the departure of William Tanner: Acorda, Alkermes, Alexion, AMAG, Aquinox, Biogen, Corium, Intra-Cellular, Seres Therapeutics, Novavax, Regulus, Supernus, TherapeuticsMD, and Acceleron. The firm terminated coverage of Aviragen.

 

Stifel analyst Thomas Shrader upgraded Puma Biotechnology to “buy” from “hold” and increased his price target to $88 from $61, citing the FDA has accepted for review the neratinib NDA in the extended adjuvant treatment.

 

The following analysts revised their price targets of Clovis Oncology, citing potential for acquisition: Credit Suisse analyst Kennen MacKay upgraded the stock to “outperform” from “neutral” and increased his price target to $41 from $19; Stifel analyst Thomas Shrader increased his price target to $45 from $30.

 

Ladenburg Thalman analyst Kevin DeGeeter initiated coverage of Aviragen Therapeutics with a “buy” rating and $2.50 price target, citing AVIR is an emerging small molecule antiviral story with important proof-of-concept data from 2 Phase II programs – RSV and HRV – expected in the next six months.

 

Cantor analyst Elemer Prios downgraded Tobira Therapeutics to “hold” from “buy” and increased his price target to $42 from $19, citing purchase by Allergan.