BioShares Biotechnology Clinical Trials (BBC): $21.71, +$0.21, -25.3% YTD

BioShares Biotechnology Products (BBP): $35.60, +$0.41, +15.5% YTD

 

 

MARKET COMMENTARY

 

U.S. stock index futures signaled a lower start for Wall Street on the first trading day of the week, after closing at record highs in the previous session. European shares dragged lower by oil majors and Italian banks, while Asian shares ended mixed. Oil prices were little changed as the market struggled over the shaky prospect of an OPEC output cut. As the dollar wilted in the currency markets, gold bounced back.

 

 

MARKET HIGHLIGHTS

 

Foamix Pharmaceuticals has completed patient enrollment according to plan in the Company’s two Phase 3 clinical trials to evaluate the efficacy and safety of FMX101, a topical 4% minocycline foam, in the treatment of moderate-to-severe acne. Foamix is conducting the two Phase 3 pivotal trials simultaneously, and expects to report top-line results in the first half of 2017.

 

Aimmune Therapeutics has completed global enrollment of its Phase 3 PALISADE trial of AR101 for the treatment of peanut allergy, with the total randomized to the trial expected to be approximately 540 patients. AR101 is Aimmune’s investigational biologic oral immunotherapy for desensitization of patients with peanut allergy.  Aimmune continues to expect topline data from PALISADE in the fourth quarter of 2017, followed by regulatory submissions for marketing approval of AR101 in 2018 in both the United States and Europe. The company announced completion of North American enrollment in PALISADE in September, ahead of schedule and above target enrollment.  Aimmune also announced ARTEMIS (AR101 Trial in Europe Measuring oral Immunotherapy Success), a new dedicated European clinical trial of AR101 in peanut-allergic children and adolescents. ARTEMIS is designed to expand the data available on the efficacy profile of AR101 by exploring a higher level of protection after a shorter treatment period in a broader group of patients (in terms of baseline reaction) than in PALISADE. The primary efficacy endpoint in this new trial will be tolerating a cumulative amount of 2,043 mg of peanut protein in an exit double-blind, placebo-controlled food challenge (DBPCFC) after approximately nine months of treatment with AR101.

 

Aimmune Therapeutics announced the closing of the $145 million equity investment by Nestlé Health Science. Nestlé Health Science purchased 7,552,084 shares of Aimmune’s common stock at $19.20 per share on November 23, 2016, in a private placement transaction. The sale of shares was made pursuant to the terms of the Securities Purchase Agreement entered into on November 3, 2016, by Aimmune and Nestlé Health Science, as previously reported by Aimmune. In connection with the closing of the equity investment, Greg Behar, Chief Executive Officer of Nestlé Health Science, was appointed to Aimmune’s board of directors on November 23, 2016. Aimmune has a total of 50,044,727 shares of common stock outstanding after taking into account the shares issued to Nestlé Health Science.

 

Cellect Biotechnology announced financial results for the third quarter ended September 30, 2016.  Net loss for the third quarter of 2016 was $1.5 million, or $0.015 per share, compared to $0.8 million, or $0.01 per share, in the second quarter of 2016, and $0.7 million, or $0.008 per share, in the third quarter of 2015.  Cash and cash equivalents (including marketable securities and short terms deposits) totaled $9.4 million as of September 30, 2016, compared to $3.1 million on December 31, 2015, and $3.8 million on September 30, 2015.

 

CymaBay Therapeutics announced that management will participate in a fireside chat at the 28th Annual Piper Jaffray Healthcare Conference on November 30, 2016.

 

Gemphire Therapeutics announced enrollment of its first patients in ROYAL-1, a Phase 2b trial designed to investigate gemcabene in the treatment of patients with hypercholesterolemia not adequately controlled on high-intensity or moderate-intensity stable statin therapy.  The open-label Phase 2b trial, “A 12-Week, Phase 2 Study of Gemcabene in Hypercholesterolemia Patients on Stable Moderate and High-Intensity Statins (ROYAL-1)” will enroll up to 104 adult patients at clinical sites in the United States.  Patients meeting eligibility requirements are being randomized in a 1:1 ratio to 600mg of gemcabene or placebo.  The primary endpoint is the percent change from baseline of LDL-C at 12 weeks.  Secondary endpoints include the change from baseline in non-HDL-C, total cholesterol, triglycerides, ApoB, and hsCRP at the 12-week time point.

 

Moberg Pharma AB obtained a waiver from its bondholders to increase flexibility in acquisition financing. The waiver provides better possibility to optimize financing of acquisitions of profitable assets.

 

Addex Therapeutics announced today the appointment of Roger G. Mills, M.D., to the newly created position of Chief Medical Officer.  Dr. Mills brings more than 25 years of biopharmaceutical industry experience at both global pharmaceutical companies and biotechnology companies, including Acadia Pharmaceuticals, Pfizer, Gilead Sciences, Abbott Laboratories and Wellcome, across a spectrum of disease areas.

 

Idera Pharmaceuticals and Vivelix Pharmaceuticals announced that they have entered into an exclusive license and collaboration agreement granting Vivelix worldwide rights to develop and market IMO-9200, an antagonist of TLR 7,8 and 9, for non-malignant gastrointestinal disorders. As part of the agreement, Idera has agreed to create and characterize potential back-up compounds for Vivelix. Under the terms of the agreement, Idera will receive an upfront fee of $15 million. In addition, Idera will be eligible for future IMO-9200 related development, regulatory and sales milestone payments totaling up to $140 million, and escalating royalties ranging from the mid single-digits to low double-digits of global net sales. Additionally, under the terms of the agreement and if requested by and at Vivelix’s expense, Idera is responsible for developing potential back-up compounds to IMO-9200. As it relates to back-up compounds Idera will be eligible for related development, regulatory sales and milestone payments totaling up to $52.5 million and escalating royalties ranging from the mid single-digits to low double-digits of global net sales.

 

Merck announced that the FDA accepted for review the sBLA for KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, for the treatment of previously treated patients with advanced microsatellite instability-high (MSI-H) cancer. The FDA granted Priority Review with a PDUFA, or target action date, of March 8, 2017; the sBLA will be reviewed under the FDA’s Accelerated Approval program based on tumor response rate and durability of response. The FDA recently granted Breakthrough Therapy Designation to KEYTRUDA for unresectable or metastatic MSI-H non-colorectal cancer, and previously granted it for the treatment of patients with unresectable or metastatic MSI-H colorectal cancer.

 

Recro Pharma announced positive results from its second of two Phase III clinical trials evaluating intravenous (IV) meloxicam for the treatment of acute postoperative pain. In this trial, IV meloxicam achieved the primary endpoint of a statistically significant difference in Summed Pain Intensity Difference (SPID) over the first 24 hours (SPID24), compared to placebo, in patients following abdominoplasty surgery. With the positive data from this study, the Company believes this completes the efficacy program for the IV meloxicam NDA.

 

GTx announced that enobosarm achieved the pre-specified primary efficacy endpoint in the 9 mg dose cohort from patients in both stage 1 and the ongoing stage 2 of its Phase II clinical trial in women with advanced, estrogen receptor positive (ER+), androgen receptor positive (AR+) breast cancer. The primary efficacy endpoint requires at least nine patients (out of a total of 44 evaluable patients) to achieve clinical benefit, defined as either a complete response, partial response or stable disease, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) at 24 weeks of treatment. In this ongoing trial, the efficacy endpoint was achieved in the first 22 confirmed evaluable patients, and the trial will continue enrolling and treating eligible patients with enobosarm until 44 evaluable patients have completed the trial. Enobosarm has been well tolerated among patients treated to date in the 9 mg dose cohort with the majority of adverse events being either grade 1 or 2. The Company plans to report top-line clinical results from these 22 evaluable patients from the 9 mg dose cohort in December 2016, and expects to report top-line clinical results from the full study by the middle of 2017, following completion of the clinical trial.

 

AVEO Oncology announced that its development partner, EUSA Pharma, a specialty pharmaceutical company with a focus on oncology and oncology supportive care, has submitted its responses to the EMA Day 120 List of Questions. The Day 120 List of Questions were issued by the CHMP as part of the centralized review process of the MAA for tivozanib for the first-line treatment of advanced renal cell carcinoma (RCC). The next step in the filing process, the EMA Day 180 List of Outstanding Issues, is expected in the first quarter of 2017.

 

Curis announced that the EMA’s CHMP has granted full approval to Erivedge (vismodegib) for the treatment of adult patients with symptomatic metastatic basal cell carcinoma (BCC) or locally advanced BCC inappropriate for surgery or radiotherapy. Erivedge is also approved in the U.S. for the treatment of adults with metastatic basal cell carcinoma, or with locally advanced basal cell carcinoma that has recurred following surgery or who are not candidates for surgery, and who are not candidates for radiation. Erivedge was developed and is marketed by Roche and Genentech, a member of the Roche Group, under a collaboration agreement between Curis and Genentech.

 

Takeda Pharmaceutical announced that the European Commission has granted conditional marketing authorization for NINLAROTM (ixazomib) capsules, indicated in combination with lenalidomide and dexamethasone for adult patients with multiple myeloma who have received at least one prior therapy. The decision to approve NINLARO as the first and only oral proteasome inhibitor to treat multiple myeloma follows a positive opinion by the EMA CHMP for Human Use in September 2016.

 

MorphoSys announced that its licensee Janssen Research & Development, through one of its affiliates, has submitted a MAA to the EMA seeking approval of guselkumab for the treatment of adults living with moderate to severe plaque psoriasis. Guselkumab is a fully human anti-IL-23 monoclonal antibody discovered by Janssen utilizing the HuCAL antibody library technology licensed from MorphoSys.

 

Epizyme announced advancements in the Company’s clinical programs evaluating tazemetostat, its first-in-class EZH2 inhibitor. The FDA has granted tazemetostat Fast Track designation in patients with diffuse large B-cell lymphoma (DLBCL) with EZH2 activating mutations. Additionally, Epizyme is focusing its Phase II registration-enabling study in adult patients with genetically-defined solid tumors on those cancers marked by loss of INI1, and has expanded enrollment in the epithelioid sarcoma cohort of the study based on encouraging early activity in this patient population.

 

Lannett Company announced that it received approval from the FDA of its ANDA for Metaxalone Tablets USP, 800 mg, the therapeutic equivalent to the reference listed drug, Skelaxin of King Pharmaceuticals.  According to IMS, total U.S. sales for the 12 months ended September 2016 of Metaxalone Tablets USP, 800 mg, at Average Wholesale Price (AWP) were approximately $173 million.

 

Mylan announced that the company has signed an agreement with the Medicines Patent Pool (MPP) to expand access to chronic hepatitis C medicines in developing countries. The agreement licenses Mylan to produce and market a generic version of Bristol-Myers Squibb‘s DAKLINZA (daclatasvir) Tablets, 30 mg and 60 mg, for distribution in 112 low and middle income countries.

 

Catalent announced that it has agreed to acquire Accucaps Industries Limited (Accucaps), the Canada-based developer and manufacturer of Over-the-Counter (OTC), high potency and conventional pharmaceutical softgels. Financial details of the transaction have not been disclosed.

 

Intec Pharma announced it has entered into an agreement with the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support patient recruitment and retention support for the Intec Pharma’s pivotal Phase III clinical trial of its lead product candidate, the Accordion Pill Carbidopa/Levodopa, or AP-CD/LD, for the treatment of Parkinson’s disease symptoms in advanced Parkinson’s disease patients, which began enrolling participants in April 2016.

 

Boehringer Ingelheim and China Southeast University Institute of Life Sciences announced the start of a joint research project to develop new treatment approaches for hearing loss through regeneration of hair cells from inner ear stem cells.

 

Global Blood Therapeutics announced enrollment of the first patient in ZEPHYR, a Phase IIa clinical trial evaluating the safety and efficacy of GBT440 for the treatment of hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest.

 

MabVax Therapeutics Holdings reported that the first patients have been treated with MVT-5873, the company’s therapeutic antibody, in combination with a standard of care chemotherapy in a previously untreated pancreatic cancer patient population. The company recently reported that it had established sufficient safety and tolerability utilizing MVT-5873 as a monotherapy in relapsed or refractory locally advanced or metastatic pancreatic cancer patents to initiate the expansion into first line treatment.

 

Lion Biotechnologies announced that it has entered into a new three-year Manufacturing Services Agreement (MSA) and related statements of work with WuXi AppTec to manufacture and perform services related to the manufacture of the Company’s autologous cell therapy products.

 

Alder BioPharmaceuticals announced the initiation of PROMISE 2 (PRevention Of Migraine via Intravenous ALD403 Safety and Efficacy 2), the second pivotal Phase III clinical trial of eptinezumab (formerly known as ALD403), its monoclonal antibody candidate inhibiting calcitonin gene-related peptide (CGRP), for the prevention of migraine. Alder also announced that eptinezumab was selected as the International Nonproprietary Name for ALD403.

 

Cellectar Biosciences announced it has selected Hamilton, Ontario-based Centre for Probe Development and Commercialization (CPDC), a well-respected GMP manufacturing organization specializing in radiopharmaceuticals, as a supplier of the company’s lead phospholipid drug conjugate (PDC), CLR 131.

 

PhaseRx announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the FDA.

 

Araim Pharmaceuticals announced that the EC has granted Orphan Medicinal Product designation in the EU to the Company’s lead product candidate, Innate Repair Receptor activator ARA 290, for prevention of graft loss in pancreatic islet transplantation. The EC’s approval follows a positive opinion in July from the EMA’s Committee for Orphan Medicinal Products (COMP). The FDA has previously granted Orphan Drug Designation to ARA 290 for treatment to increase survival and improve functioning of pancreatic islets following transplantation.

 

Galenica Group announced that Relypsa, a Vifor Pharma Company, has received approval from the FDA for a sNDA with important updates to the U.S label of Veltassa (patiromer) for oral suspension. The U.S label for Veltassa no longer includes a Boxed Warning regarding the separation of Veltassa and other oral medications.

 

Medivir AB announced that a Phase IIb open-label study of the combination of simeprevir, odalasvir and AL-335, also known as JNJ-4178, has been initiated by Janssen Research & Development, part of the Janssen Pharmaceutical Companies of Johnson & Johnson, in treatment-naive and treatment-experienced subjects with chronic hepatitis C virus infection without cirrhosis. This global, multi-center study includes clinical trial sites in North America, Europe and Asia and forms part of Janssen’s global development program for JNJ-4178. The objectives of the Phase IIb study are to investigate the efficacy, safety and pharmacokinetics of JNJ-4178/ AL-335 (800mg QD), odalasvir (25mg QD), and simeprevir (75mg QD) in treatment-naive and treatment -experienced non-cirrhotic subjects with chronic hepatitis C virus genotype 1, 2, 4, 5, and 6 infection.

 

Acasti Pharma announced that Linda O’Keefe has been named CFO.

 

Catalent announced that its wholly owned subsidiary, Catalent Pharma Solutions, intends to offer, subject to market and other conditions, $400 million (U.S. dollar equivalent) aggregate principal amount of euro-denominated senior unsecured notes due 2024 in a private offering that is exempt from the registration requirements of the Securities Act of 1933, as amended.

 

 

ANALYST RECOMMENDATIONS

 

Cowen analyst Ritu Baral upgraded Achaogen to “outperform” from “market perform,” citing clarity to value-generating Phase III data.

 

Citi analyst Liav Abraham upgraded Horizon Pharma to “buy” from “neutral” and increased her price target to $25 from $21, citing upcoming pivotal data for Actimmune in FA are very important but difficult to predict.

 

Aegis analyst Jason Wittes initiated coverage of Mediwound with a “buy” rating and a $11 price target, citing the company has developed a next-generation proteolytic enzyme platform for wound debridement, the critical first step in the treatment of chronic wounds, by providing a significantly better result in terms of speed, selectivity, safety, and cost relative to the current standards of care.

 

Rodman &Renshaw analyst Yi Chen initiated coverage Imprimis Pharmaceuticals with a “buy” rating and $5 price target, citing its risk-mitigated approach to product development, based on optimizing formulations of existing approved drug substances as well as developing combination products that meld multiple known active pharmaceutical ingredients and its potential to deliver lower cost versions of existing approved therapies.

 

Canaccord analyst Arlinda Lee increased her price target of Xenocor to $32 from $27, citing the mechanistic rationale for treating IgG4-RD with XmAb5871 as sound, and the company’s XmAb pipeline and bispecifics capabilities as attractive.

 

Leerink analyst Michael Schmidt decreased his price target of Juno to $34 from $45, citing news last week that the pivotal ROCKET trial of JUNO’s JCAR015 product candidate in adult acute lymphocytic leukemia has again been placed on a clinical hold due to safety concerns.