BioShares Biotechnology Clinical Trials (BBC): $19.27, -$0.01, +4.7% YTD

BioShares Biotechnology Products (BBP): $34.32, +$0.10, +4.8% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures were lower after immigration curbs introduced by Donald Trump added an extra layer of uncertainty to the economic impact of the new U.S. president’s policies. Personal consumption, personal income, Core PCE price index and pending home sales data are on the economic calendar. European shares dipped, tracking weakness in Asian shares. Oil prices fell as news of another increase in drilling activity spread concern over rising output. In the currency markets, investors sought the traditional security of the yen and the greenback was little changed against the sterling and the euro. Gold inched up.

 

 

MARKET HIGHLIGHTS

 

Advaxis announced that an abstract on data from the company’s Phase 2 GOG-0265 trial evaluating axalimogene filolisbac for metastatic cervical cancer has been accepted as a late-breaking presentation at the Society of Gynecologic Oncology’s (SGO) 48th Annual Meeting on Women’s Cancer in March.

 

Eiger BioPharmaceuticals will present a corporate overview and business update at the 19th Annual BIO CEO & Investor Conference at 10:00 am ET on Monday, February 13, 2017, at the Waldorf Astoria Hotel in New York City.

 

Zogenix announced today the recent issuance of U.S. Patent No. 9,549,909 from the U.S. Patent & Trademark Office.  The patent, entitled “Method for the Treatment of Dravet Syndrome”, covers claims related to a method for the adjunctive treatment of seizures associated with Dravet syndrome with ZX008.  This patent is expected to provide protection of the associated claims through 2033.

 

Ocera Therapeutics announced top-line results from its exploratory STOP-HE Phase 2b Study evaluating the efficacy, safety and tolerability of intravenously-administered Ornithine Phenylacetate (OCR-002) in hospitalized patients with Hepatic Encephalopathy (HE). Although not statistically significant, OCR-002 demonstrated a 17-hour reduction over placebo (47 versus 64 hours, respectively) for the primary endpoint, which was time to improvement in HE symptoms, p=0.129, hazard ratio 1.25. In addition, OCR-002 demonstrated a 15-hour reduction over placebo (87 versus 102 hours, respectively) for the secondary endpoint, which was median time to complete response in HE symptoms, p=0.361, hazard ratio 1.16. Consistent with its mechanism of action, OCR-002 exhibited a statistically significant reduction over placebo for the study’s pre-specified exploratory endpoint which was time to achieve normal plasma ammonia levels, p=0.028, hazard ratio 1.69.

 

Gemphire Therapeutics announced interim data on the LDL-C primary endpoint from the ongoing open label COBALT-1 trial. COBALT-1 is investigating gemcabene in homozygous familial hypercholesterolemia (HoFH) patients diagnosed by genetic confirmation or a clinical diagnosis to assess the efficacy, safety, and tolerability of multiple rising doses of gemcabene in patients with HoFH who are on maximally tolerated lipid lowering therapies. Conventional therapies such as statins and ezetimibe are the most commonly used drugs for HoFH, reducing LDL-C 15% to 25% in this very high-risk patient population. Interim results on two genetically confirmed HoFH patients through 8 weeks of treatment (4 weeks on 300 mg of gemcabene followed by 4 weeks on 600 mg of gemcabene) are reported herein. Both subjects were on a background of high intensity statin therapy (atorvastatin 80 mg or rosuvastatin 40 mg), and one subject was also on ezetimibe before receiving gemcabene. Gemcabene lowered mean LDL-C by 23% and 28% at doses of 300 mg and 600 mg, respectively. Adverse events (AEs) have been mild to moderate in intensity across all doses of gemcabene; there have been no serious AEs or withdrawals due to AEs in the COBALT-1 study.

 

BrainStorm Cell Therapeutics has validated its cryopreservation process for NurOwn® in preparation for the upcoming Phase 3 clinical study in Amyotrophic Lateral Sclerosis (ALS).

 

Incyte and Calithera Biosciences announced that the companies have entered into a global collaboration and license agreement for the research, development and commercialization of Calithera’s first-in-class, small molecule arginase inhibitor CB-1158 in hematology and oncology. CB-1158 is currently being studied in a monotherapy dose escalation trial and additional studies are expected to evaluate CB-1158 in combination with immuno-oncology agents, including anti-PD-1 therapy.

 

uniQure announced that AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B, has received Breakthrough Therapy designation by the FDA This designation is based on results from the ongoing, dose-ranging Phase I-II study that show sustained increases in Factor IX (FIX), reductions in FIX replacement usage and a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up.

 

Rigel Pharmaceuticals announced updates from the clinical program of fostamatinib in patients with chronic immune thrombocytopenic purpura (ITP). Previously released results from the FIT Phase III clinical studies (047, 048) of fostamatinib in chronic ITP demonstrated that patients who responded to fostamatinib have a timely, robust, and sustained response to treatment.  As of September 2016, the open-label, long-term extension study (049), was tracking the experience of 124 patients who opted to receive treatment with fostamatinib after their participation in either Study 047 or Study 048.  Seventeen patients who achieved a stable response to fostamatinib in the parent studies enrolled in Study 049. As of September 2016, responders who enrolled in the 049 study had maintained a median platelet count of 106,500/?L over this extended period of time.  These patients have been on fostamatinib treatment for a median of 16 months as of September 2016.  In addition, there were now 41 out of 44 former placebo patients who had been treated with fostamatinib for a minimum of 12 weeks. Of those, 22% (9/41, p=0.0078) achieved a prospectively defined stable platelet response, which Rigel believes further validates fostamatinib as a potential new treatment option for some patients with this serious disease. Rigel also announced its calculations of an overall response rate for Study 047 and Study 048 by combining stable and transient responders.  The overall response rate to fostamatinib is 29% (29/101) compared to 2% (1/49) for placebo (p=<0.0001). A stable response was defined as a patient achieving platelet counts of ≥ 50,000/?L on ≥ 4 of the 6 visits between weeks 14 and 24. In the post-study analysis performed by Rigel, a transient response was defined to include patients achieving at least 2 consecutive median platelet counts over 50,000/?L during the trial without rescue, but who did not otherwise meet the stable response criteria.

 

FibroGen reported positive topline results from the two Phase III clinical studies of roxadustat (FG-4592 or ([1]) ) designed to support new drug application submission in China. Roxadustat is a first-in-class, orally administered small molecule for treatment of anemia in dialysis-dependent CKD (DD-CKD) and non-dialysis-dependent CKD (NDD-CKD) patients. Both of the Phase III studies met their primary efficacy endpoints, which were evaluated in the comparator-controlled portions of the studies.

 

Regulus Therapeutics announced that it received written communication from the FDA that the clinical development program for RG-101 remains on clinical hold.  In June 2016, RG-101 was placed on clinical hold following the Company’s submission of a second serious adverse event (SAE) of jaundice.

 

Mallinckrodt announced it has entered into an agreement under which it will sell its Intrathecal Therapy business to Piramal Enterprises Limited’s subsidiary in the U.K., Piramal Critical Care, for approximately $203 million, including fixed and contingent consideration. Piramal Critical Care is an integral business unit of Piramal Enterprises Limited and a global leader in the field of anesthesia.

 

BioCryst Pharmaceuticals announced that the EMA has accepted the filing of its peramivir MAA for treatment of symptoms typical of influenza in adults 18 years and older. The acceptance of the MAA begins the review process by the EMA under the centralized licensing procedure for all 28 member states of the European Union, Norway and Iceland. Peramivir’s efficacy has been demonstrated when treatment is initiated within two days of first onset of symptoms.

 

Supernus Pharmaceuticals announced the issuance on January 24, 2017 of a seventh patent (number 9,549,940) by the USPTO covering Trokendi XR, its novel once-daily extended-release topiramate product. The patent provides protection for the product with expiration that is no earlier than 2027.

 

Allergan announced the FDA has approved the company’s sNDA to update the label for AVYCAZ (ceftazidime and avibactam) with clinical data from two Phase III trials supporting the indication to treat patients with complicated urinary tract infections (cUTI), including pyelonephritis, caused by designated susceptible Gram-negative microorganisms.

 

Benitec Biopharma announced that it has initiated work on two new oncology pipeline programs after executing a Research Collaboration Agreement with Nant Capital. This transaction represents a further step in establishing a strategic alliance with Nant around the development and funding of the head and neck cancer squamous cell carcinoma (HNSCC) programs.

 

Teva Pharmaceutical Industries announced that the FDA approved two products for adolescent and adult patients with asthma. These products, AirDuo RespiClick (fluticasone propionate and salmeterol inhalation powder) and ArmonAir RespiClick (fluticasone propionate inhalation powder), include medication delivered via Teva’s RespiClick breath-activated, multi-dose dry powder inhaler (MDPI) which is used with other approved medicines in Teva’s respiratory product portfolio.

 

Pulmatrix announced that it has entered into a definitive agreement with several institutional investors to purchase an aggregate of approximately $5.0 million of shares of common stock in a registered direct offering. The Company agreed to sell an aggregate of approximately 2,000,000 shares of common stock, at a price of $2.50 per share, in the registered direct offering. The closing of this offering is expected to take place on or about February 2, 2017, subject to the satisfaction of customary closing conditions. Rodman & Renshaw, a unit of H.C. Wainwright & Co., is acting as exclusive placement agent in connection with this offering.

 

DURECT provided an update on the DUR-928 development program. DUR-928, our Epigenetic Regulator Program’s lead product candidate, is an endogenous, small molecule, new chemical entity (NCE), which may have broad applicability in several metabolic diseases such as nonalcoholic steatohepatitis (NASH) and other liver conditions, and in acute organ injuries such as acute kidney injury (AKI).

 

Joseph M. Limber, in partnership with Ampersand Capital Partners and 1315 Capital, entered into a definitive agreement to acquire the commercial laboratory of Genoptix, a Novartis company that specializes in oncology diagnostics and informatics services. This transaction is expected to close in the first quarter of 2017 subject to certain closing conditions. The financial terms of the transaction are not being disclosed.

 

ESSA Pharma announced the receipt of a $4.0 million payment from the Cancer Prevention Research Institute of Texas ("CPRIT"). The payment is part of a total non-dilutive grant of $12.0 million, repayable out of potential product revenues, which was originally awarded in February 29, 2014.

 

Cumberland Pharmaceuticals announced that on January 26, 2017, the U.S. Court of Appeals for the Federal Circuit ruled in favor of Cumberland in a patent case associated with its Acetadote product.

 

EyeGate Pharmaceuticals announced topline results from the first-in-human pilot trial of its EyeGate Ocular Bandage Gel for the acceleration of re-epithelialization of large corneal epithelial defects in patients having undergone photorefractive keratectomy ("PRK").

 

Neurotrope announced that it has signed a licensing agreement with Stanford University for an accelerated synthesis of bryostatin-1. Prior to this synthetic route, bryostastin-1 had to be isolated from tons of natural sources to obtain grams of the drug.

 

Cerecor announced that it has entered into an equity distribution agreement with Maxim Group, as sales agent, pursuant to which Cerecor may from time to time offer and sell shares of its common stock having an aggregate gross sales price of up to $12,075,338. Cerecor intends to use the net proceeds from the at-the-market offering program, if any, to partially fund the research and development of CERC-501 and CERC-611 in Cerecor’s pipeline, pursue potential partnerships, collaborations or out-licenses, and for general working capital.

 

Sunovion Pharmaceuticals announced that the FDA approved the sNDA for Latuda (lurasidone HCI) for the treatment of schizophrenia in adolescents aged 13 to 17 years. LATUDA is also approved in the U.S. for the treatment of adults with schizophrenia and for the treatment of adults with major depressive episodes associated with bipolar I disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate.

 

Astellas Pharma and Ironwood Pharmaceuticals announced top-line results indicating that the Phase III clinical trial of linaclotide conducted in Japan in adults with chronic constipation (CC) met its primary endpoint. Linaclotide is approved in Japan as the first prescription treatment for adults with irritable bowel syndrome with constipation (IBS-C). Linaclotide is currently approved in the United States for the treatment of adults with IBS-C or chronic idiopathic constipation (CIC). It is also approved for adults with IBS-C or CIC in more than 30 other countries.

 

Illumina announced that it has declared conformity with the requirements of the IVD Directive and has applied the CE mark to an expanded VeriSeq NIPT Analysis Software for clinical laboratories in the European Union (EU). This updated VeriSeq NIPT Analysis Software includes an innovative method for processing samples and is designed for larger batches of 48 samples, versus the current 16 samples, which can scale to meet the future demands of the growing noninvasive prenatal testing (NIPT) market. Using this software, clinical labs in the EU have access to fast and reliable software for analysis of sequencing data for NIPT.

 

Immune Therapeutics issued a Special Letter to Shareholders from its CEO, Noreen Griffin, which summarizes some of the Company’s achievements in 2016 and discusses its growth outlook for 2017 and beyond.

 

CytoDyn announced the appointment of Anthony D. Caracciolo as Executive Chairman of CytoDyn effective immediately. In this new capacity, Mr. Caracciolo will assume an active leadership role in the Company’s strategic planning, business development and operations. Nader Pourhassan, Ph.D., will continue to serve as President and Chief Executive Officer, and Mr. Caracciolo will retain his position as Chairman of the Board.

 

AmpliPhi Biosciences announced the appointment of Igor P. Bilinsky, Ph.D., as its Senior Vice President and Chief Operating Officer, effective January 30, 2017. He succeeds Wendy S. Johnson, who served as AmpliPhi’s interim COO since September 2014 and continues to serve on AmpliPhi’s board of directors.

 

Agenus announced that Jean-Marie Cuillerot, M.D. was appointed CMO. Dr. Cuillerot initially joined Agenus in 2016 as the Vice President and Global Head of Clinical Development.

 

ANALYST RECOMMENDATIONS

 

Oppenheimer analyst Jay Olson decreased his price target of Prothena to $70 from $77, citing new methodology ahead of 4Q16 earnings.

 

HC Wainwright analyst Ed Arce upgraded CymaBay Therapeutics to “buy” from “neutral” and increased his price target to $6 from $2.50, citing potential aggregate milestone payments totaling up to $200M from Kowa (Japan), CymaBay’s new U.S. partner for arhalofenate (gout), and a more positive view of the potential of MBX-8025 to treat PBC in the ongoing open-label, dose ranging Phase 2 study.


William Blair analyst Katherine Xu initiated coverage of Albireo, Ltd with an “Outperform” rating.

 

RBC analyst Randall Stanicky decreased his price target of Endo International to $15 from $18, citing recent corporate actions, ongoing strategic review, and an increasingly challenging 2017 operating environment.

 

First Analysis analyst Tracy Marshbanks decreased her price target of NantHealth to $13 from $14, citing lower revenue estimates through 2018.

 

Following Regulus Therapeutics’ announcement that the FDA has extended RG-101 clinical hold and requested additional long-term clinical data from ongoing trials, Needham analyst Alan Carr downgraded the stock to “hold” from “buy;” BMO analyst Matthew Luchini decreased his price target to $2 from $5; Wells Fargo analyst Jim Birchenough downgraded the stock to “market perform” from “outperform” and decreased his valuation range to $1-$2 from $6.75-$8.00.

 

Leerink analyst Michael Schmidt downgraded Array Biopharma to “market perform” from “outperform” and increased his price target to $11 from $9, citing limited near-term upside drivers while the company’s pipeline now is fairly valued following the recent run.