BioShares Biotechnology Clinical Trials (BBC): $22.84, +$0.39, -21.4% YTD
BioShares Biotechnology Products (BBP): $33.52, +$0.44, +8.8% YTD
A day after U.S. stocks closed higher as data showed the services sector rebounded, Wall Street looked set to open lower. Weekly jobless claims will be in focus on the economic calendar. European shares retreated, despite a rally amongst banking shares, after easyJet’s stock dropped to its lowest in more than three years. Asian stocks closed mostly higher. The dollar touched a one-month high versus the yen, on growing optimism that the U.S. Federal Reserve will raise interest rates in December. Oil prices were steady, underpinned by a surprisingly large drop in U.S. inventory levels. Gold edged up.
Transgene presented an original approach to improve the cytotoxic activity of oncolytic viruses at the 10th International Meeting on Replicating Oncolytic Virus Therapeutics, Vancouver, Canada October 1–4, 2016. This approach is based on the use of intrabodies, fragments of recombinant monoclonal antibodies designed to act intracellularly and to bypass a mechanism which prevents viral-induced cytolysis that is seen in some resistant tumor cell lines. These results open new possibilities for engineering the next generation of oncolytic viruses.
CoLucid Pharmaceuticals provided additional data regarding onset of action demonstrated as soon as 30 minutes after dosing for 100 mg and 200 mg of lasmiditan from its Phase 3 pivotal trial evaluating lasmiditan, the SAMURAI study. In addition, CoLucid had presented non-clinical data at the 5th European Headache and Migraine Trust International Congress (EHMTIC) in Glasgow, Scotland, U.K., supporting the mechanism of action of lasmiditan through central nervous system (CNS) penetration, distribution into areas of the brain relevant to migraine pathophysiology, expression of the 5-HTIF receptor mRNA in relevant regions of the brain, inhibition of trigeminal nociceptive responses, and lack of any vasoconstrictive activity in non-clinical models.
Aurinia Pharmaceuticals announced 24-week data in all 10 patients from the AURION study, an open-label exploratory study to assess the short-term predictors of response using voclosporin (23.7mg BID) in combination with mycophenolate mofetil (MMF) and oral corticosteroids in patients with active lupus nephritis (LN). The data are being presented by Robert Huizinga, Vice President of Clinical Affairs at Aurinia at the 10th Annual European Lupus Meeting in Venice, Italy. The primary objective of the study is to examine biomarkers of disease activity at eight weeks and their ability to predict response at 24 and 48 weeks. In this study, 70% (7/10) patients achieved complete remission (CR) at 24 weeks as measured by a urinary protein creatinine ratio (UPCR) of ≤ 0.5mg/mg, eGFR within 20% of baseline and concomitant steroid dose of <5mg/day. Of the 10 patients that achieved a reduction of UPCR of ≥ 25% at 8 weeks, 80% were responders (≥ 50% reduction in UPCR over baseline) at 24 weeks and 70% were in CR at 24 weeks. In addition, inflammatory markers such as C3, C4 and anti-dsDNA all continued to normalize to 24 weeks. Voclosporin was well-tolerated with no unexpected safety signals observed.
CymaBay Therapeutics announced that an abstract describing results from a study evaluating the activity of MBX-8025 in reversing non-alcoholic steatohepatitis (NASH) in diabetic obese mice has been accepted for an oral presentation at the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) in Boston, November 11-15, 2016. MBX-8025 is an orally administered potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist.
Advaxis announced that Robert Petit, Ph.D., executive vice president and chief scientific officer of Advaxis, was invited to speak at the Immune Profiling World Congress in Barcelona on October 10 – 12, 2016.
Kamada announced the extension of the strategic partnership with Shire plc for GLASSIA®. Minimum revenue for GLASSIA in the extended agreement for the years 2017 to 2020 will reach approximately $237 million and may be expanded to $288 million during that period. Kamada will now continue to produce GLASSIA through 2020 for Shire, after which Shire may produce the product at their facility and pay Kamada established royalty rates. This represents the fourth time the companies have extended the contract for manufacturing supply of GLASSIA since the start of the strategic relationship in 2010.
Gemphire Therapeutics announced the appointment of Lee Golden, M.D. as Chief Medical Officer, effective October 5, 2016. In this newly-created role, Dr. Golden will be responsible for advancing the global clinical development of gemcabene, Gemphire’s product candidate.
Alnylam Pharmaceuticals announced that upon the recommendation of the ENDEAVOUR Phase III study Data Monitoring Committee (DMC) to suspend dosing, the Company has decided to discontinue development of revusiran, an investigational RNA interference (RNAi) therapeutic that was being developed for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM). This decision was made yesterday evening and has been communicated to investigators, study sites, and regulatory authorities. Following the news, Stifel analyst Stephen Willey decreased his price target to $36 from $68; Piper Jaffray analyst Edward Tenthoff decreased his price target to $106 from $118; Leerink analyst Michael Schmidt downgraded the stock to “market perform” from “outperform” and decreased his price target to $40 from $107; Barclays analyst Geoff Meacham downgraded the stock to “equal weight” from “overweight” and decreased his price target to $50 from $85; Chardan analyst Madhu Kumar decreased his price target to $77 from $115; JMP analyst Michael King decreased his price target to $69 from $127; Credit Suisse analyst Alethia Young decreased her price target to $50 from $145; JP Morgan analyst Anupam Rama decreased his price target to $51 from $83; Cowen analyst Ritu Baral decreased her price target to $100 from $150; Jefferies analyst Gena Wang decreased her price target to $58 from $86; Needham analyst Alan Carr decreased his price target to $137 from $152; Goldman analyst Terence Flynn decreased his price target to $34 from $65; Janney analyst Roy Buchanan decreased his fair value estimate to $39 from $74.
RedHill Biopharma provided an update on the RHB-104 Phase III Crohn’s disease development program, planned enhancements to the MAP US first Phase III study and expected milestones, including an option for early stop for success for overwhelming efficacy.
PTC Therapeutics announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory nonsense mutation Duchenne muscular dystrophy patients (nmDMD). The results, which are being presented as part of a company-sponsored symposium, are based on PTC’s analyses of lung function data from one of PTC’s ongoing open-label extension studies (Study 019) versus natural history data from a comparable non-ambulatory cohort.
SCYNEXIS announced the complete results of its two recently completed Phase II studies as well as the closing of a $15 million term loan with Solar Capital. In the first study, treatment with oral SCY-078 in patients with vulvovaginal candidiasis (VVC), resulted in significantly better clinical cure rates and fewer recurrences of VVC at the four-month follow-up when compared to the standard of care (oral fluconazole). In the second study, which evaluated oral SCY-078 as a step down therapy in patients with invasive candidiasis, oral SCY-078 achieved the target exposure for efficacy and was well-tolerated.
The Medicines Company provided a progress update on the ongoing ORION-1 study of PCSK9si, its investigational first-in-class PCSK9 synthesis inhibitor.
Flamel Technologies announced that its Irish subsidiary, Flamel Ireland Holdings, has reached agreement with the FDA for the design and planned analysis of a Phase III clinical trial of FT218, a once nightly formulation of sodium oxybate utilizing the company’s proprietary drug delivery platform, Micropump. The agreement was reached through the Special Protocol Assessment process.
Arrowhead Pharmaceuticals and Spring Bank Pharmaceuticals announced an agreement to perform collaborative studies on Arrowhead’s ARC-520 and Spring Bank’s SB 9200, for the treatment of chronic Hepatitis B (HBV). The companies plan to first conduct preclinical models with both agents together and then study the agents clinically in a cohort to be added to Arrowhead’s ongoing MONARCH Phase IIb study, in which patients will receive a dosing regimen that includes ARC-520, SB 9200, and an oral direct-acting antiviral.
Sun BioPharma announced that the Data Safety Monitoring Board (DSMB), an independent group of medical experts closely monitoring the clinical trial, has completed its safety review of the data from the dosing of the third cohort of patients. As a result of this positive review by the DSMB, Sun BioPharma has initiated the fourth patient cohort in the dose escalation phase of the study. The Company currently expects to begin dosing patients in the fourth cohort as early as October 10, 2016, which is approximately 60 days after the third patient cohort commenced dosing.
Teva Pharmaceutical Industries, Celltrion, Inc. and Celltrion Healthcare announced that the companies have entered into an exclusive partnership to commercialize two of Celltrion’s mAb biosimilar candidates in the U.S. and Canada. CT-P10 is a proposed mAb biosimilar to Rituxan (rituximab), which is used to treat patients with Non-Hodgkin’s Lymphoma (NHL), Chronic Lymphocytic Leukemia (CLL), Rheumatoid Arthritis (RA), Wegener’s Granulomatosis and Microscopic Polyangiitis (MPA). CT-P6 is a proposed mAb biosimilar to Herceptin (trastuzumab), which is used for the treatment of HER2-overexpressing breast cancer and for the treatment of HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.
Catabasis Pharmaceuticals announced that it is presenting positive data from Part A of the MoveDMD trial of edasalonexent for the treatment of Duchenne muscular dystrophy (DMD) at the World Muscle Society Congress.
Kite Pharma announced the first patient was enrolled in ZUMA-6, a Phase Ib/II clinical study of KTE-C19 in combination with atezolizumab, Genentech’s anti-PD-L1 cancer immunotherapy. The trial is designed to evaluate the safety and efficacy of the combination in patients with refractory diffuse large B-cell lymphoma (DLBCL).
Cellectar Biosciences announced the enrollment of the first patient into Cohort 3 of the company’s Phase I clinical study of CLR 131 in patients with relapsed or refractory multiple myeloma.
ARCA biopharma announced the randomization of the first European patients in the GENETIC-AF Phase IIB/III clinical trial. GENETIC-AF, which has been enrolling patients in the United States and Canada, is evaluating Gencaro as a potential treatment for atrial fibrillation (AF). Last week, ARCA hosted a GENETIC-AF European Investigator Meeting which brought together European physicians and research coordinators for study-specific training and information sharing. It is estimated that approximately six million patients in Europe have atrial fibrillation.
Orexo AB announced that it has received approval from the FDA of a new unique low dose, 0.7mg/0.18 mg, tablet of Zubsolv (buprenorphine/naloxone) sublingual tablet (CIII) for the treatment of opioid dependence. The new dosage is expected to be available in US pharmacies in early 2017.
Shire announced that the USPTO’s Patent Trial and Appeal Board (PTAB) has issued their decision upholding the validity of U.S. Patent No. 6,773,720, related to Shire’s LIALDA (mesalamine) product. The petition seeking to institute inter partes review (IPR) was filed with the USPTO’s Patent Trial and Appeal Board (PTAB) in April of 2015 by the Coalition For Affordable Drugs (CFAD) II L.L.C. Fund. The PTAB found that the patent was valid in light of the challenges put forward by the petitioner.
Metabrain Research announced the signature of an agreement with Medicxi, for the inception of Kymo Therapeutics, incorporated in Cambridge (UK).
Asceneuron SA announced it has been awarded a research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) for the further development of positive allosteric modulators of the M1 muscarinic acetylcholine receptor (M1 PAMs). M1 PAMs induce a change in the shape of the receptor, enhancing binding to the neurotransmitter acetylcholine. As a result, receptor activity is potentiated so that it can fulfil its signaling functions, critical for cognition, even in situations where acetylcholine levels are reduced, as observed in dementia.
AroCell AB announced that a distribution agreement has been signed with the New England-based company Eagle Biosciences, for distribution of the AroCell TK 210 ELISA test in North America.
aTyr Pharma announced that additional clinical data from aTyr’s Phase Ib/II Trial (002) in adult patients with FSHD were presented at the 21st International Congress of the World Muscle Society in Granada, Spain. The poster presentation is titled "A Randomized, Double-blinded, Placebo-controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Biological Activity of ATYR1940 (Resolaris) in Adult Patients with Facioscapulohumeral Muscular Dystrophy."
Jaguar Animal Health announced that it has signed a non-binding letter of intent (“LOI”) with Napo Pharmaceuticals, potentially to merge the two companies. Napo focuses on human product development and commercialization from plants used traditionally in rainforest areas, and has provided Jaguar with exclusive worldwide rights for veterinary applications to crofelemer and corresponding rights to all related Napo technology.
CytoDyn announced that the FDA has accepted protocol modifications for the Company’s Phase III pivotal trial with PRO 140 in combination with current standard-of-care antiretroviral therapy (ART) for highly treatment-experienced patients with HIV. The new protocol reduces the number of patients to be enrolled in the trial from 150 to 30 patients and lowers the primary endpoint for viral load reduction from viral load of 0.7log to viral load of 0.5log. The Company intends to provide data from its monotherapy trial (conducted in parallel to this study) to satisfy the safety portion of its pivotal trial and will meet with the FDA when the trial’s primary end point data becomes available to further discuss the safety portion requirement.
Synthetic Biologics announced it has been awarded a contract by the Centers for Disease Control and Prevention (CDC). The award will support research conducted during the Company’s ongoing randomized, placebo-controlled Phase IIb proof-of-concept clinical study of SYN-004 (ribaxamase), designed to protect the gut microbiome from the unintended effects of certain commonly used intravenous (IV) beta-lactam antibiotics for the prevention of C. difficile infection (CDI), antibiotic-associated diarrhea (AAD) and the emergence of antibiotic-resistant organisms.
Idera Pharmaceuticals announced that it intends to offer and sell up to $50,000,000 of shares of its common stock in an underwritten public offering. In connection with this offering, Idera expects to grant the underwriters a 30-day option to purchase additional shares of common stock, equal to up to 15% of the number of shares of common stock sold in the offering. Idera intends to use the net proceeds from this offering, together with existing cash, cash equivalents and investments, to advance clinical development of certain of its programs. J.P. Morgan and Goldman Sachs are acting as joint bookrunning managers for the offering.
Goldman analyst Salveen Richter increased her price target of Avexis to $71 from $46, citing potential AVXS-101 gene therapy data in SMA Type I at the World Muscle Society and clarity on the AVXS-101 pivotal trial design in late-Oct/early-Nov.
Following Aerie Pharmaceuticals’ investor day, Stifel analyst Annabel Samimiy increased her price target to $55 from $50; Brean analyst Difei Yang increased her price target to $63 from $48.
Jefferies analyst Matthew Andrews increased his price target of Vanda Pharmaceuticals to $23 from $15, citing continuing improvement in U.S. Fanapt sales/scrips, steady growth of Hetlioz in the U.S./EU-5, and pipeline contributions from Hetlioz in Jet Lag Disorder and tradipitant in gastroparesis starting in 2019/2021
Rodman &Renshaw analyst Raghuram Selvaraju initiated coverage of CytoDyn with a “buy” rating and $1.60 price target, citing next-generation anti-retroviral antibody-based therapy for HIV.
Leerink analyst Seamus Fernandez revised his price target of the following companies prior to 3Q16 earnings, Bristol-Myers decreased to $69 from $75; Merck increased to $65 from $63; Eli Lilly decreased to $103 from $104; Novo Nordisk decreased to DKK330 from DKK375; Sanofi decreased to €85 from €87.
Morgan Stanley analyst Matthew Harrison revised his price target of the following companies, Biogen decreased to $367 from $368; bluebird bio increased to $57 from $53; Celgene increased to $121 from $120; DBV increased to $64 from $63; Global Blood Therapeutics decreased to $58 from $62; Gilead decreased to $97 from $100; Juno decrease to $45 from $46; Ophthotech decreased to $99 from $101; Portola increased to $29 from $28; Ultragenyx decreased to $83 from $89; Regeneron increased to $421 from $409; Vertex increased to $140 from $137.
BMO analyst Do Kim upgraded IONIS to “outperform” from “market perform” and increased his price target to $48 from $42, citing Revusiran discontinuation could benefit the company.
Oddo Securities analyst Sebastien Malafosse upgraded DBV Technologies to “buy” from “neutral” and increased his price target to €92 from €80, citing the potential in the short and medium term as newsflow is set to be more abundant in the coming months.
Jefferies analyst assumed coverage of Immunomedics with a “buy” rating and increased his price target to $5.50 from $5, citing IMMU-132 has generated promising results in Triple Negative breast cancer and clin/reg/CMC progress in 2016 sets the stage for a deal in the coming months ahead of Ph. III start by late 2016/early 2017.
Chardan analyst Gbola Amusa decreased his price target of The Medicines Company to $57.50 from $65, citing RNAi platform risk assessment.
RBC analyst Adnan Butt decreased his price target of Regeneron to $633 from $648, citing an estimate reduction in early launch years of Eylea.
Goldman analyst Terence Flynn decreased his price target of Ophthotech to $23 from $45, citing competitive landscape for OPHT’s Fovista in wet AMD and a lack of meaningful catalysts.
William Blair analyst Y. Katherine Xu decreased her price target of Vertex to $110 from $116, citing revised estimates following preannounced earnings.
Following the news that Tenax Therapeutics does not plan to undertake further development of levosimendan in septic shock, WallachBeth analyst Brian Jeep decreased his price target to $8 from $12; Zacks analyst John Vandermosten decreased his price target to $9 from $10.