BioShares Biotechnology Clinical Trials (BBC): $20.36, +$0.43, -29.9% YTD    

BioShares Biotechnology Products (BBP): $34.65, +$0.71, +12.4% YTD

 

 

MARKET COMMENTARY

 

Tracking gains in global equities, major U.S. stock index futures pointed to a slightly higher start for Wall Street. Job openings and labor turnover, and consumer credit data occupy the day’s light economic calendar. The euro edged up against the dollar ahead to Thursday’s European Central Bank policy meeting. Oil prices fell as investors questioned whether a deal to cut output would be enough to end the global glut. Gold firmed after falling for eight out of the last 10 sessions. 

 

 

MARKET HIGHLIGHTS

 

Aeglea BioTherapeutics will participate on a panel and provide a corporate update at the upcoming BMO Capital Markets Prescription for Success Healthcare Conference on Wednesday, December 14, 2016 in New York.

              

CymaBay Therapeutics announced the initiation of a new Phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC).  In this open label study, patients who have had an inadequate response to, or are intolerant to, ursodiol will be enrolled to receive seladelpar, either 5 or 10 mg, for 8 weeks. Based on the review of the 8-week data, new patients will be enrolled to receive seladelpar 25 mg for 8 weeks. The study also incorporates an extension phase where patients will be able to continue treatment for a total of 26 weeks during which it will be possible to adjust the dose of seladelpar. The primary endpoint will be the change in alkaline phosphatase (ALP), a parameter that has been used in prior clinical studies with PBC and which is believed to reflect the status of the disease. A variety of secondary outcomes will also be studied. The study is designed to enroll approximately 36 patients in the U.S., Canada, Germany and the U.K.

 

Advaxis announced that preclinical data on the potential use of detoxified Listeriolysin O (dtLLO) as an effective adjuvant for the development of infectious disease vaccines will be presented at the 11th annual ImVacS-Immunization and Vaccine Summit in Boston on December 9.

 

Lysogene appointed David Schilansky to the board of directors, as a non-executive, independent member. Mr. Schilansky, currently Deputy CEO and Chief Operating Officer of DBV Technologies, has 20 years of leadership experience in the field of biotechnology and finance.

 

Moberg Pharma has entered into a definitive agreement to acquire DermoPlast for a purchase price of USD 47.6M plus stock value. The acquisition is financed through a directed new issue of shares in the amount of SEK 148M, an issue of corporate bonds in the amount of SEK 215M and by available cash resources.

 

GeNeuro and Servier, a leading independent international pharmaceutical company, announce the setting-up of ANGEL-MS, a long-term extension study for patients with multiple sclerosis (MS) and treated with GNbAC1.

 

Ocera Therapeutics announced it has completed enrollment in its Phase 2b STOP-HE Study evaluating the efficacy, safety and tolerability of Ornithine Phenylacetate (OCR-002) in hospitalized patients with Hepatic Encephalopathy (STOP-HE).  STOP-HE is a placebo-controlled, randomized, double-blind clinical trial designed to evaluate the safety, pharmacokinetics and efficacy of intravenously-administered OCR-002 in resolving neurocognitive symptoms of acute HE in hospitalized cirrhotic patients with elevated ammonia. Either OCR-002 or placebo was administered to patients intravenously for up to five days along with standard of care. The OCR-002 arm was dosed with 20, 15, or 10 grams over 24 hours based on the patient’s degree of liver impairment in order to normalize drug exposure. The primary efficacy endpoint is time to meaningful clinical improvement in HE symptoms as analyzed using a 0.05 level 2-sided log-rank test of equality of time to event curves with 80% power.

 

Cancer Genetics announced a collaboration with BeiGene. The two companies will work together on BeiGene’s immune marker and genomics initiatives to accelerate studies of tumor microenvironment in cancer patients, which will aid in drug target identification and biomarker discovery.

 

Anthera Pharmaceuticals announced positive trends from the Week 48 analysis of the Phase II BRIGHT-SC proof-of-concept study in 57 patients with IgA nephropathy.

 

Viking Therapeutics announced that the FDA has granted orphan drug designation to VK0214 for the treatment of X-linked adrenoleukodystrophy (X-ALD).  VK0214 is a novel, orally available thyroid receptor beta (TRβ) agonist that selectively regulates the expression of genes believed to be relevant to the manifestation of X-ALD.

 

Novartis announced that the EC has granted an additional indication for Lucentis (ranibizumab) to treat patients with visual impairment due to choroidal neovascularization (CNV) associated with causes other than neovascular age-related macular degeneration (nAMD), or secondary to pathologic myopia (PM). With this approval, Lucentis is the first retinal treatment approved for these conditions, addressing an important unmet medical need.

 

Merck announced positive health-related quality of life (HRQoL) findings from an exploratory analysis from the Phase III KEYNOTE-024 study investigating the use of KEYTRUDA (pembrolizumab), the company’s anti-PD-1 therapy, compared to standard of care (SOC) platinum-containing chemotherapy for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express high levels of PD-L1 (tumor proportion score [TPS] of 50 percent or more). Specifically, patient-reported outcomes showed clinically meaningful improvement with KEYTRUDA compared to chemotherapy. Findings will be presented today at the 17(th) World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer.

 

Reuters reported that Bristol-Myers Squibb’s immunotherapy drug Opdivo has been approved in Europe for the treatment of classical Hodgkin lymphoma, a rare blood cancer, the company said. Opdivo is already approved for melanoma, lung cancer and kidney cancer.

 

Celgene announced that the results of its randomized Phase II tnAcity trial of ABRAXANE for injectable suspension (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) will be presented at the 2016 San Antonio Breast Cancer Symposium (SABCS) December 6-10, 2016. The trial found that an investigational weekly combination regimen of ABRAXANE + carboplatin had significantly longer progression-free survival (PFS) (7.4 months) compared to weekly regimens of either ABRAXANE + gemcitabine (5.4 months) or of carboplatin + gemcitabine (6.0 months) as first-line treatment of patients with metastatic triple-negative breast cancer (mTNBC).

 

ARIAD Pharmaceuticals announced clinical data on brigatinib, its investigational anaplastic lymphoma kinase (ALK) inhibitor, from the pivotal ALTA trial in ALK-positive (ALK+) non-small cell lung cancer (NSCLC) patients who had experienced disease progression on crizotinib therapy. As of May 31, 2016, the data show that of patients on the 180-mg regimen with a median follow-up of 11 months, 55 percent achieved confirmed objective response as assessed by the investigator. In this arm, the median progression-free survival (PFS) was 15.6 months in this post-crizotinib setting, by both investigator and independent review committee (IRC) assessment. Additionally, in this arm, 67 percent of patients with measurable brain metastases achieved a confirmed intracranial objective response, and intracranial PFS was 18.4 months among patients with any brain metastases at baseline. These data will be presented today at the International Association for the Study of Lung Cancer (IASLC) 17th World Conference on Lung Cancer (WCLC) being held in Vienna.

 

Cascadian Therapeutics announced that following a recent meeting with the FDA and discussions with the Company’s external Steering Committee, it has amended the HER2CLIMB Phase II clinical trial of tucatinib (also known as ONT-380) by increasing the sample size so that, if successful, the trial could serve as a single pivotal study to support registration. This decision is supported by the most recent data from the Company’s ongoing Phase Ib study evaluating the same "triplet combination" therapy being investigated in the amended Phase II trial, which is tucatinib in combination with capecitabine and trastuzumab for patients with metastatic or locally advanced HER2-positive breast cancer, including patients with and without brain metastases. Tucatinib is an oral, small molecule kinase inhibitor that is highly selective for HER2 without significant inhibition of EGFR.

 

Omeros announced that an abstract highlighting positive results from the ongoing OMS721 Phase II clinical trial in patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) has been accepted for presentation at the 2017 Tandem Meeting of the American Society of Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research in Orlando, Florida. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2). In addition to its Phase II clinical program in TMAs, OMS721 is currently in a Phase III program for patients suffering from atypical hemolytic uremic syndrome and in a Phase II program for renal diseases, including immunoglobulin A (IgA) nephropathy and membranous nephropathy.

 

CytoSorbents announced the achievement of a permanent, dedicated reimbursement procedure code for the CytoSorb therapy in Germany, providing for specific and enhanced reimbursement in the largest medical device market in Europe.

 

Compugen disclosed CGEN-15137, its cancer immunotherapy program for TIGIT. TIGIT is an immune checkpoint in the B7/CD28 family which has recently gained broad industry interest in the field of immuno-oncology.

 

Regulus Therapeutics announced two new drug development candidates at its R&D day, held in New York. The first candidate, RGLS5040, is an anti-miR targeting microRNA-27 (miR-27) for the treatment of cholestatic diseases. The second candidate, RGLS4326, is an anti-miR targeting microRNA-17 (miR-17) for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

 

Juno Therapeutics provided an update of key data from studies of its investigational CAR-T cell product candidates, presented at the 58(th) ASH Annual Meeting.

 

Celgene announced results from two studies (Abstracts #1143, LBA-1) evaluating the investigational use of REVLIMID (lenalidomide) maintenance therapy in patients with multiple myeloma. These studies were presented at the 58(th) ASH Annual Meeting.

 

Puma Biotechnology has initiated a Managed Access Program for PB272 (neratinib). Managed access programs provide physicians and patients access to medicines when there are limited or no other therapeutic options available.

 

Xencor announced the completion of an underwritten public offering of 5,272,750 shares of its common stock, including 687,750 shares sold pursuant to the full exercise of an option previously granted to the underwriters to purchase additional shares of common stock. The gross proceeds to Xencor from this offering were approximately $126.5 million. Xencor anticipates using the net proceeds from the offering for working capital and general corporate purposes, including additional investments in advancing its lead compound XmAb5871 through later stages of clinical development and additional investment in its immune-oncology pipeline and technology.  Leerink Partners acted as sole book-running manager in the offering. Canaccord Genuity and Wedbush PacGrow acted as co-managers in the offering.

 

Repros Therapeutics participated in the industry presentation at the Bone, Reproductive and Urologic Drugs Advisory Committee Meeting. The panel provided the FDA with advice regarding a clinical and regulatory path to approval for products, such as enclomiphene, in subjects with obesity-related hypogonadism who wish to maintain spermatogenesis. The panel voted 16 to 5 that the achievement of testosterone improvement while maintaining evidence of spermatogenesis was not sufficient, in and of itself, to provide evidence of clinical benefit.

 

Neovacs announced that the FDA has granted “Fast Track” status to IFNα Kinoid in Lupus, the most advanced therapeutic vaccine issued from Neovacs R&D.

 

Reuters reported that Britain’s competition watchdog said it had fined Pfizer a record 84.2 million pounds ($106.54 million) for ramping up the cost of an epilepsy drug by as much as 2,600 percent. The Competition and Markets Authority also fined Flynn Pharma 5.2 million pounds for its role in hiking prices of phenytoin sodium capsules in 2012. Pfizer used to market the medicine itself, under the brand name Epanutin, but sold the rights to Flynn in September 2012, after which the product was debranded and the price soared.

 

Cellceutix announced initiation of the second cohort of patients treated with Brilacidin for Ulcerative Proctitis/Ulcerative Proctosigmoiditis (UP/UPS), two types of Inflammatory Bowel Disease (IBD).

 

Origent Data Sciences and Cytokinetics announced results from the first part of a research collaboration to refine and prospectively validate an Origent computer model to predict the course of ALS disease progression leveraging data from Cytokinetics’ clinical trials of tirasemtiv. The poster, presented by David L. Ennist, Ph.D., MBA, Chief Science Officer, Origent Data Sciences and Jinsy A. Andrews, M.D., Director of Neuromuscular Clinical Trials, Columbia University, at the 27th International Symposium on ALS/MND in Dublin, Ireland, showed that the Gradient Boosting Machine (GBM) algorithm was the optimal model to predict slow vital capacity (SVC) at times subsequent to baseline and that forced vital capacity (FVC) records could be used to predict slow vital capacity (SVC) scores of ALS patients using this machine learning technique.

 

Swedish Orphan Biovitrum AB and Horizon Pharma announced that the companies have entered into a five-year distribution agreement for Ravicti (glycerol phenylbutyrate) in European countries, including United Kingdom, Germany, France, Italy and Spain and for Ammonaps (sodium phenylbutryate) in the same European countries and certain Middle Eastern countries.  Under the agreement, Sobi will have exclusive marketing, sales and distribution rights for the two medicines in the territory until 31 December 2021. Horizon has the ability to terminate the agreement after two years subject to certain pre-defined termination fees.  Sobi currently distributes Ravicti in certain Middle Eastern countries and Ammonaps in certain European and Middle Eastern countries.

 

Regen BioPharma in a recent study published in the International Journal of Molecular Sciences a team of scientists in Guangzhou, China have demonstrated that expression of NR2F6 in patients with early cervical cancer correlates with poor prognoses.  These scientists tested for NR2F6 in patient’s cervical cancer tissue as well as in the normal tissue surrounding each of their tumors.

 

Boehringer Ingelheim announced that the LUME-Meso Phase II trial in patients with unresectable malignant pleural mesothelioma (MPM) met its primary endpoint of progression-free survival (PFS). The data presented at the 17th IASLC World Conference on Lung Cancer in Vienna, showed nintedanib* plus pemetrexed/cisplatin demonstrated a meaningful clinical benefit compared to placebo plus pemetrexed/cisplatin, with a significantly improved PFS (9.4 vs 5.7 months, HR = 0.56 and p value =0.0174). Preliminary overall survival (OS) data also favoured nintedanib (18.3 vs 14.5 months, HR = 0.78 and p=0.4132).

 

Progenics Pharmaceuticals announced that the first patient has been dosed in the Company’s Phase II/III clinical trial evaluating the diagnostic accuracy of its PSMA-targeted PET/CT imaging agent, 18F-DCFPyL (PyL(TM) ). PyL was discovered by a team led by Martin G. Pomper, M.D., Ph.D., William R. Brody Professor of Radiology at the Center for Translational Molecular Imaging at the Johns Hopkins University School of Medicine.

 

International Stem Cell Corporation announced the treatment of the second patient in the Australian trial for Parkinson’s Disease. The patient was injected with 30,000,000 ISC-hpNSC cells and is currently recovering.

 

Arena Pharmaceuticals announced that it has completed enrollment in the ralinepag Phase II trial. Ralinepag is an oral, selective IP receptor agonist targeting the prostacyclin pathway for the treatment of pulmonary arterial hypertension (PAH). The study enrolled approximately 60 patients at sites globally.

 

Panther Biotechnology announced that it has received positive feedback from the submission presented to the FDA pursuant to a Pre-IND  meeting with the Division of Oncology Products 1 (DOP1) of the Center for Drug Evaluation and Research (CDER) of the FDA. The purpose of the requested meeting was to obtain FDA’s input regarding Panther’s plans for the development of TRF-DOX, Panther’s novel transferrin-doxorubicin conjugate initially planned for the treatment of platinum-resistant ovarian cancer. In preparation for the meeting, Panther submitted a Pre-IND Package to FDA that described the information Panther intends on submitting in the TRF-DOX IND submission planned in 2017. The IND is the regulatory vehicle that will allow for the initiation of clinical trials with TRF-DOX initially for the treatment of ovarian cancer.

 

Aerie Pharmaceuticals announced the completion of patient enrollment in the Company’s second Phase III registration trial (“Mercury 2”) of Roclatan (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, a novel once-daily eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. Roclatan ophthalmic solution is a fixed dose combination of Aerie’s drug Rhopressa (netarsudil ophthalmic solution) 0.02% and latanoprost, the most widely prescribed PGA (prostaglandin analogue). Total patient enrollment has exceeded the 690 patients needed for this three-arm study, designed to demonstrate superiority of Roclatan solution to each of its two components, all dosed once-daily in the evening. Enrolled patients had maximum baseline IOPs ranging from above 20 to below 36 mmHg (millimeters of mercury). Mercury 2, which commenced in March 2016, is a 90-day registration trial with a topline efficacy readout that is expected in the second quarter of 2017.

 

Illumina announced the appointment of Sam A. Samad, Senior Vice President and Chief Financial Officer. He will join the company on January 6th, 2017.

 

ITUS Corporation announced preliminary results from its Cchek cancer patient efficacy study. Using its most recent protocols and methods for measuring a patient’s immunological response to a malignancy, the Company achieved Sensitivity of 92% and Specificity of 92% for 88 patient samples, including 54 samples from patients with multiple types and severities of cancer, and 34 healthy patients. During the initial phase of the study, which involved multiple experimental protocols and techniques for measuring immunological responses, the Company reviewed and analyzed data from a total of 315 patient samples, including 228 patients with varying stages of cancer, as well as blood samples from 87 healthy donors.

 

Otsuka Pharmaceutical Development & Commercialization and Lundbeck presented data on aripiprazole for extended-release injectable suspension for intramuscular use (hereinafter ABILIFY MAINTENA) in the maintenance treatment of bipolar I disorder (BP-I) at the 2016 Annual Meeting of the American College of Neuropsychopharmacology (ACNP) in Hollywood, Florida.

 

BioPharmX will share research at the Antibacterial Discovery and Development Summit in Boston that suggests topical delivery of minocycline may be effective for the treatment of acne because, in topical form, minocycline may fight P. acnes-induced local inflammation.

 

NeuroDerm announced the pricing of a follow-on offering of 4,000,000 shares of its ordinary shares at a price to the public of $18.75 per share. All of the ordinary shares are being offered by NeuroDerm. In addition, NeuroDerm has granted the underwriters a 30-day option to purchase up to an additional 600,000 shares at the public offering price. NeuroDerm anticipates the total gross proceeds from the offering before underwriting discounts and commissions and other offering expenses will be approximately $75.0 million. The offering is expected to close on or about December 12, 2016, subject to customary closing conditions. The company expects to use the net proceeds of the offering to fund ongoing and planned clinical trials for its product candidates and the development of related devices and for research and development of other product candidates and general corporate purposes. Jefferies and Cowen are acting as joint book-running managers for the offering, and Raymond James and Roth Capital Partners are acting as co-managers.

 

bluebird bio announced the pricing of an underwritten public offering of 3,289,473 shares of its common stock at a public offering price of $76.00 per share, before underwriting discounts. In addition, bluebird bio has granted the underwriters a 30-day option to purchase up to an additional 493,420 shares of common stock. All of the shares in the offering are to be sold by bluebird bio. Goldman Sachs, BofA Merrill Lynch and Cowen are acting as joint book-running managers of the offering. Wells Fargo Securities and Wedbush PacGrow are acting as co-managers.

 

 

Analyst Recommendations

 

Oppenheimer analyst Jay Olsen initiated coverage of Axovant Sciences with an “outperform” rating and $20 price target, citing the potential for intepirdine to succeed in the symptomatic treatment of Alzheimer’s Disease and Dementia with Lewy Bodies.

 

Oppenheimer analyst Derek Archila assumed coverage of the following companies: KemPharm with a $13 price target (up from $11), Teva Pharmaceutical with a $50 price target (down from $66), Mallinckrodt with a $72 price target (down from $115), Lannett Company, Endo International, and OvaScience

 

Chardan analyst Madhu Kumar decreased his price target of Regulus to $5 from $12, citing concerns for RG-101 and RG-012 and early-stage nature of the new preclinical assets.

 

Morgan Stanley analyst Steve Beuchaw decreased his price target of Illumina to $100 from $115, citing instrument revenue will see pressure continue into ’17, Pharma spend is slowing and Academic funding is likely to remain challenged in the US and Europe for at least 3 more quarters, diligence finds barriers to an inflection in clinical growth remain, diligence on TMO/QGEN competition finds users increasingly optimistic about alternatives to ILMN in clinical, the recent decline of the HiSeq installed base and flat HiSeq X placements present risk to consumables growth and are evidence of the impact of customer efforts to shift projects to instruments that reduce consumables spend, and hospital spending may emerge as a headwind for the clinical channel in ’17 pending uncertainty around the future of the ACA in the US.