BioShares Biotechnology Clinical Trials (BBC): $23.19, -$.013, -20.2% YTD

BioShares Biotechnology Products (BBP): $33.79, -$0.40, +9.6% YTD





U.S. stock index futures inched lower, amidst a surprise output cut agreement by the Organization of the Petroleum Exporting Countries, as investors questioned whether this move will help rebalance the supply glut. Investors keenly await second-quarter growth, pending homes index and wholesale inventory data to gauge strength of the economy. European equities advanced and Asian stocks closed higher, following the OPEC’s decision, which boosted oil company shares and lifted the currencies of crude-producing countries. The yen tumbled against the dollar, while gold pared early gains.





Innate Pharma SA announced that safety data for two Phase I studies of lirilumab in combination with nivolumab or ipilimumab conducted by Bristol-Myers Squibb in patients with advanced refractory solid tumors will be presented by Dr. Neil H. Segal, Memorial Sloan-Kettering Cancer Center, in a poster at the ESMO 2016 congress (October 7 – 11, 2016) in Copenhagen, Denmark. Lirilumab is Innate Pharma’s anti-KIR antibody partnered with Bristol-Myers Squibb.


Innate Pharma SA announced that the Data and Safety Monitoring Board (“DSMB”) completed its seventh assessment of the EffiKIR study and recommended continuation of the trial without modification.  Enrollment in the EffiKIR study was completed in July 2014. The analysis on the primary endpoint, leukemia-free survival, is event driven and could occur by the end of 2016.  As specified in the study protocol, the DMSB meets every six months to examine the safety data accumulated during progress of the trial.


Pernix Therapeutics Holdings announced the distribution of TREXIMET® (sumatriptan and naproxen sodium) 10/60 mg to wholesalers has begun.  TREXIMET 10/60 mg is indicated for use in pediatric patients 12 years of age and older for the acute treatment of migraine with or without aura.


Alcobra announced that it has received verbal notice from the Division of Psychiatry Products of the U.S. Food and Drug Administration (FDA) that a full clinical hold has been placed on its Investigational New Drug (IND) applications for MDX in ADHD and Fragile X Syndrome. The clinical hold affects Alcobra’s ongoing Phase III clinical study of MDX in adult patients with ADHD, known as the “MEASURE” study.  Alcobra has not yet received written notice of the clinical hold from the FDA, however, based on verbal communications, the FDA indicated that the clinical hold is due to adverse neurological findings in a pre-clinical study. The FDA notification was not based on clinical safety data observed in the ongoing MEASURE study, which has enrolled nearly 500 subjects, or previous clinical studies involving MDX. Alcobra plans to work diligently with the FDA to seek the removal of the clinical hold.


ASPiRA LABs, a Vermillion company, today announced a major contract agreement with CareFirst BlueCross BlueShield for their U.S. FDA cleared ovarian cancer risk assessment test, OVA1. CareFirst serves over three million patients throughout Maryland and Washington, D.C., and Virginia and the area has the greatest population density in the US.


Intra-Cellular Therapies announced top-line results from the second Phase III clinical trial (Study ‘302) of ITI-007, an oral, first-in-class investigational medicine for the treatment of schizophrenia. In this trial, neither dose of ITI-007 separated from placebo on the primary endpoint, change from baseline on the Positive and Negative Syndrome Scale (PANSS) total score, in the pre-defined patient population. The active control, risperidone, did separate from placebo. In this trial, ITI-007 was statistically significantly better than risperidone on key safety and tolerability parameters and exhibited a safety profile similar to placebo. This replicates the safety and tolerability findings of a previous study (our Phase II Study ‘005) in which the efficacy of ITI-007 60 mg and risperidone, the active control, were similar. We believe ITI-007 did not separate from placebo on the pre-specified primary endpoint in Study ‘302 in part due to an unusually high placebo response at certain sites which disproportionately affected the trial results and contributed to the efficacy outcome of this study compared to our two previous positive efficacy studies. Following the news, SunTrust analyst Edward Nash downgraded the stock to “neutral” from “buy” and decreased his price target to $15 from $60; JMP analyst Jason Butler downgraded the stock to “market perform” from “market outperform.”


Amgen and Arrowhead Pharmaceuticals announced two license and collaboration agreements to develop and commercialize RNA interference (RNAi) therapies for cardiovascular disease. These are the first programs to utilize Arrowhead’s proprietary subcutaneous RNAi delivery platform. RNAi molecules may be designed to target and shut down specific gene products that contribute to various diseases. Under one agreement, Amgen receives a worldwide, exclusive license to Arrowhead’s novel, RNAi ARC-LPA program. These RNAi molecules are designed to reduce elevated lipoprotein(a), which is a genetically validated, independent risk factor for atherosclerotic cardiovascular disease. Under the second agreement, Amgen receives an option to a worldwide, exclusive license for a RNAi therapy for an undisclosed genetically validated cardiovascular target. In both agreements, Amgen will be wholly responsible for clinical development and commercialization. Under the terms of the agreements taken together, Arrowhead will receive $35 million in upfront payments; $21.5 million in the form of an equity investment by Amgen in Arrowhead common stock; and up to $617 million in option payments, and development, regulatory and sales milestone payments. Arrowhead is further eligible to receive single digit royalties for sales of products against the undisclosed target and up to low double digit royalties for sales of products under the ARC-LPA agreement. Additional financial terms of the agreements are not disclosed.


Vertex Pharmaceuticals announced that the FDA approved ORKAMBI (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. ORKAMBI is the first and only medicine to treat the underlying cause of CF for people with this mutation. It was previously approved by the FDA for use in people ages 12 and older with two copies of the F508del mutation. With today’s approval, approximately 11,000 people with CF are eligible for treatment with ORKAMBI in the United States. ORKAMBI will be available for eligible children ages 6 through 11 in the United States as soon as possible. Vertex also today lowered its guidance for 2016 ORKAMBI revenues to a range of $950 million to $990 million.


AstraZeneca announced that the FDA has approved a blood-based companion diagnostic for TAGRISSO (osimertinib). The companion diagnostic for TAGRISSO is the only FDA-approved and clinically validated companion diagnostic test that uses either tissue or a blood sample to confirm the presence of a T790M mutation in patients with metastatic epidermal growth factor receptor (EGFR) mutation-positive non-small cell lung cancer (NSCLC), who have progressed on or after an EGFR tyrosine kinase inhibitor (TKI) medicine.


Genocea Biosciences announced positive results from its ongoing Phase IIb trial evaluating a new Phase III-ready formulation of GEN-003 for the treatment of genital herpes. The study achieved its primary endpoint, with GEN-003 demonstrating a statistically significant reduction of 40 percent in the rate of viral shedding in the 60 ug per protein / 50 ug of Matrix-M2 dose group compared to both baseline and placebo. The viral shedding rate reduction for this dose was consistent with its performance at the same time point in a prior Phase II trial.


Catabasis Pharmaceuticals and Sarepta Therapeutics announced a joint research collaboration to explore a combination drug treatment approach for Duchenne muscular dystrophy (DMD). The two companies will contribute their respective expertise to study an exon skipping treatment developed by Sarepta, together with an oral NF-kB inhibition treatment developed by Catabasis in a mouse model of DMD.


Alnylam Pharmaceuticals announced new clinical and non-clinical research results demonstrating continued RNAi therapeutics platform innovation and optimization, including improved potency, durability, metabolic stability and tolerability with its GalNAc platform, as well as clinical translation across multiple pipeline programs. The research was presented at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held in Montreal, Quebec.


Aegerion Pharmaceuticals announced that Japan’s Ministry of Health, Labor & Welfare (MHLW) has approved JUXTAPID for patients with homozygous familial hypercholesterolemia (HoFH).


Enanta Pharmaceuticals announced that it has initiated a Phase I clinical study and has begun dosing healthy adults with EDP-305, Enanta’s lead farnesoid X receptor (FXR) agonist under development to treat patients with NASH.


Amgen announced positive top-line results for erenumab (AMG 334) from A Phase III, RandomIzed, double-blind, placebo-controlled Study to Evaluate the efficacy and safety of erenumab in migraine prevention (ARISE). These data showed the ARISE study met the primary endpoint, demonstrating a statistically significant reduction from baseline in monthly migraine days in patients with episodic migraine treated with erenumab compared with placebo at 12 weeks. Erenumab is specifically designed to prevent migraine by blocking the Calcitonin Gene-Related Peptide (CGRP) receptor, which is believed to have a critical role in mediating the incapacitating pain of migraine.


Reuters reported that a U.S. health agency said it had "expressly advised" Mylan that the company had misclassified the EpiPen in a way that led to the company paying a lower rebate to states. The Centers for Medicare and Medicaid Services, part of the U.S. Department of Health and Human Services, said in a statement it has "on multiple occasions, provided guidance to the industry and Mylan on the proper classification of drugs and has expressly advised Mylan that their classification of EpiPen for purposes of the Medicaid Drug Rebate program was incorrect."     Mylan classified the EpiPen with the Medicaid Drug Rebate Program as a non-innovator multiple source drug, which pays a rebate of 13 percent to states, rather than innovator, which pays a rebate of a minimum of 23.1 percent.


Sarepta Therapeutics announced the first patient dosed in the Phase III clinical trial of SRP-4045 and SRP-4053 in patients with Duchenne muscular dystrophy amenable to exon 45 or 53 skipping.


Roche announced that the FDA has approved a label extension of the cobas EGFR Mutation Test v2 for use with plasma samples as a companion diagnostic for Astra Zeneca’s non-small cell lung cancer (NSCLC) therapy TAGRISSO (osimertinib).


MorphoSys AG and Galapagos announced that the first patient with atopic dermatitis was dosed in an ongoing clinical Phase I study with their jointly discovered and developed human monoclonal antibody MOR106 against IL-17C.


Cellectar Biosciences announced positive data from the second cohort of patients enrolled in its orphan-drug designated Phase I study of CLR 131 in patients with relapsed or refractory multiple myeloma. Following these outcomes, the study’s Data Monitoring Committee approved patient enrollment to the third cohort, which will include a 33 percent dose increase from 18.75 to 25mCi/m(2) of CLR 131 from the previous cohort.


Eisai announced the initiation of a multicenter, global, randomized Phase III study that will evaluate the efficacy and safety of two regimens containing Eisai’s multiple receptor tyrosine kinase inhibitor lenvatinib, marketed in the U.S. as LENVIMA – lenvatinib in combination with the anti-PD-1 immunotherapy pembrolizumab, marketed under the brand name KEYTRUDA by Merck & Co., known as MSD outside of the U.S. and Canada, and lenvatinib in combination with everolimus (LEN+EVE) – versus sunitinib in patients with advanced renal cell carcinoma (RCC) in the first-line setting. The CLEAR (Comparison of the efficacy and safety of Lenvatinib in combination with Everolimus or pembrolizumab versus sunitinib alone in first-line treatment of subjects with Advanced Renal cell carcinoma) study is designed to assess the potential of these combinations to extend PFS in these patients.


TESARO and Zai Lab (Shanghai) Co., Ltd. announced a collaboration to support the development and commercialization of niraparib for patients in China and the potential to advance two immuno-oncology programs outside of China. This partnership reflects the commitment of both companies to develop novel therapeutic approaches for people living with cancer around the world.


NeuroDerm announced that the Company requires additional time to activate clinical sites in certain countries that are participating in its long-term safety trial (trial 012, the "BeyoND" trial) of ND0612, the Company’s continuous, subcutaneously delivered levodopa/carbidopa (LD/CD) liquid formulation. As previously disclosed, upon the completion of the BeyoND trial, as well as the other parallel trials outlined in the Company’s clinical development plan, NeuroDerm intends to submit marketing applications in the U.S. and the EU for ND0612.


Marinus Pharmaceuticals announced top-line results from a Phase II exploratory open-label clinical trial evaluating ganaxolone, its CNS-selective GABA(A) modulator, in females with PCDH19 pediatric epilepsy. In the trial, ganaxolone reduced seizure frequency from baseline in the majority of patients enrolled in the study and was generally safe and well tolerated. PCDH19 pediatric epilepsy is a rare, serious epilepsy characterized by early-onset cluster seizures, cognitive and sensory impairment, and behavioral disturbances, with no approved treatments. In 2015, the FDA granted Orphan Drug Designation to ganaxolone for the treatment of PCDH19.


Karyopharm Therapeutics announced that preclinical data describing XPO1 inhibition with selinexor (KPT-330), the Company’s lead, oral Selective Inhibitor of Nuclear Export / SINE(TM) compound, in a KRAS-mutant NSCLC model, were published online in Nature. The paper, titled, "XPO1 Dependent Nuclear Export is a Druggable Vulnerability in KRAS-mutant Lung Cancer," discusses preclinical results supporting selinexor’s potential as a new therapeutic strategy for patients with highly aggressive and difficult to treat KRAS-mutant NSCLC.


Collegium Pharmaceutical announced the publication of "Oral Human Abuse Potential of Oxycodone DETERx (Xtampza ER)," in the peer-reviewed medical journal, The Journal of Clinical Pharmacology.


Cytokinetics announced that the Federal Trade Commission has granted early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR Act) in connection with the 2016 amendment to the License and Collaboration Agreement initially executed between Cytokinetics and Astellas Pharma Inc., in 2013 and amended in 2014. In July 2016, the companies expanded the collaboration related to the research, development and commercialization of skeletal muscle activators. With the termination of the applicable waiting period under the HSR Act, the 2016 amendment is deemed effective as of Sept. 26, 2016 and the upfront payment of $65 million from Astellas to Cytokinetics is due and payable within 30 days.


Abide Therapeutics announced that Celgene has exercised its option to obtain ex-US rights to Abide’s first-in-class endocannabinoid system modulator, ABX-1431, for the potential treatment of neurological diseases. Celgene will be responsible for development costs for all indications from Phase II clinical trials and beyond. Abide retains US rights and will conduct a number of Phase Ib studies. With the exercise of this option, Abide will receive a $20 million exercise fee.


Roche announced that the Harmony Prenatal Test for assessing the probability of Down syndrome (Trisomy 21) and other chromosomal disorders has received the CE mark. This enables laboratories to perform local in vitro diagnostic (IVD) testing in countries that accept the mandatory conformity marking.  Local testing availability permits laboratories to comply with reimbursement requirements and expedite test results for healthcare providers and expectant parents in their regions.


Elekta announced that its high-field MR-linac was the focus of multiple presentations at the American Society for Radiation Oncology (ASTRO) 2016 Annual Meeting, held September 25 – 28 in Boston. Additional abstracts presented by members of Elekta’s MR-linac Consortium also highlight the need for adaptation of radiation therapy to address moving tumors and nearby organs during treatment sessions. Naturally occurring physiological movements limit our ability to conform the treatment to the target and increase exposure of radiation to healthy tissues.


Actinium Pharmaceuticals announced the pricing of an underwritten public offering of 8,000,000 shares of its common stock at a price to the public of $1.25. In addition, Actinium has granted the underwriters a 30-day option to purchase up to an additional 1,200,000 shares of common stock solely to cover over-allotments, if any. The offering is expected to close on or about October 4, 2016. The gross proceeds to Actinium from this offering is expected to be $10,000,000. Actinium currently intends to use the net proceeds from the sale of securities for general corporate purposes, including capital expenditures, the advancement of its drug candidates in clinical trials, such as Iomab-B and Actimab- A, preclinical trials, and to meet working capital needs. H.C. Wainwright & Co. is acting as sole book-running manager for the offering. JonesTrading Institutional Services is acting as co-manager for the offering. ROTH Capital is acting as a financial advisor for the offering.


Hanmi Pharmaceutical Co. announced that it has entered into an exclusive development and license agreement with Genentech, a member of the Roche Group, for the development and commercialization of Hanmi’s pan-RAF inhibitor, HM95573 which is currently in Phase I clinical development.


Rennova Health announced the signing of a definitive agreement to acquire the remaining ownership (approximately 85%) of Genomas for a total of $1.75 million in newly-created Series F convertible preference shares, plus the assumption of approximately $800,000 of existing debt. The conversion price will be the higher of the average of the previous 10 days closing share price of Rennova shares prior to conversion or $1.95. The maximum number of common shares issuable upon conversion of these preference shares is 897,436. Rennova previously announced the acquisition of approximately 15% ownership in July and is pleased to complete the acquisition of 100% of Genomas, Inc. The acquisition brings to Rennova revolutionary PhyzioType Systems for DNA-guided management and prescription of drugs used to treat mental illness, pain, heart disease and diabetes.


Horizon Pharma announced that it has entered into a rebate agreement with Prime Therapeutics, which secures formulary status for its primary care medicines. The rebate agreement begins on October 1, 2016.


Aequus Pharmaceuticals announced that it has successfully obtained provincial formulary coverage from the Ontario Drug Benefit Program for (Pr) Vistitan (bimatoprost 0.03% w/v, ophthalmic solution). According to IMS data, over 80% of Ontario’s glaucoma patient population rely on public medication coverage, who will now have improved access to this product.


Transgenomic announced that it has signed agreements with two additional distributors in China and India for its ICEme(TM) Mutation Enrichment Kits for cancer genomic testing. The kits incorporate Transgenomic’s Multiplexed ICE COLD-PCR (MX-ICP) technology and are designed to enable virtually any laboratory to conduct high quality DNA mutation detection in cancer patients using plasma, blood or tissue samples and existing sequencing platforms. The new distributors, JoyingBio in China and Biotron Healthcare in India, are important suppliers of advanced life science products in their respective markets.


ConsumerMedical announced Kyle Bray as the organization’s new Chief Operating Officer. Bray will be responsible for creating functional alignment among the ConsumerMedical team and establishing priorities to meet market demands. He will have sole oversight for ConsumerMedical’s operations and call center, research, technology, and finance.


Galmed Pharmaceuticals announced that, to date, it has raised approximately $5.0 million under the Company’s At-The-Market financing executed by Cantor Fitzgerald (the "ATM"). In total, to date the Company has issued 933,160 ordinary shares under the ATM at an average sale price of $5.11 per share.





Northland analyst David Buck upgraded Endo International to “outperform” from “market perform”, citing a positive risk/reward after the recent pullback in shares; JP Morgan analyst Chris Schott decreased his price target of Endo International to $38 from $40, citing an increased discount rate across the Specialty Pharma sector.


Janney analyst Roy Buchanan initiated coverage of the following companies: Ionis Pharmaceuticals with a “buy” rating and $47 fair value estimate; Intellia Therapeutics with a “buy” rating and $29 fair value estimate; Alnylam Pharmaceuticals with a neutral” rating and $74 fair value estimate; ProQR Therapeutics with a “neutral” rating and €7.00 fair value estimate.


Maxim analyst Jason McCarthy increased his price target of Abeona Therapeutics to $14 from $8, citing the "risk" in gene therapy and the "risk" in CAR-T is declining as clinical success emerges.


Following Incyte’s release of positive long-term follow up results for epacadostat in melanoma, Raymond James analyst Reni Benjamin upgraded the stock to “outperform” from “market perform” and established $115 price target; Morgan Stanley analyst Andrew Berens increased his price target to $98 from $90.


Mizuho analyst Irina Koffler decreased her price target of Mallinckrodt to $89 from $91, citing management guided to higher dilution associated with the nuclear imaging divestiture.


HC Wainwright analyst Ed Arce downgraded Galectin Therapeutics to “neutral” from “buy” and decreased his price target to $1.50 from $8, citing Galectin reported that the Phase II NASH-FX trial missed its primary endpoint, improvement in liver fibrosis measured by cT1, as well as both secondary endpoints, liver stiffness as measured by magnetic resonance elastography and FibroScan.


Citi analyst Robyn Karnauskas increased her price target of Celgene to $127 from $124, citing increased Beta-Thal estimates.


Citi analyst Robyn Karnauskas initiated coverage of Acceleron Pharma with a “neutral” rating and $40 price target, citing pivotal programs already in stock, multiple Phase II catalysts could provide upside.


Canaccord analyst Mark Massaro decreased his price target of Alere to $53 from $56, citing some possibility that Abbott chooses to sue Alere.  


Baird analyst Brian Skorney decreased his price target of Vertex Pharmaceuticals to $115 from $128, citing on slower-than-expected Orkambi uptake.


Brean analyst Difei Yang assumed coverage of Akebia Therapeutics with a “buy” rating and decreased her price target to $18 from $26, citing not including revenues from the MTPC deal, Akebia held cash and cash equivalents of about $189 mil, which should fund the company’s operations through 2Q17.


The following companies dropped coverage of Medivation now that the acquisition by Pfizer is complete: Canaccord Genuity, Credit Suisse, Cowen, Barclays, William Blair, and Leerink.