BioShares Biotechnology Clinical Trials (BBC): $23.05, -$0.07, -20.7% YTD
BioShares Biotechnology Products (BBP): $34.38, -$0.24, +11.6% YTD
U.S. stock index futures retreated as investors waited to see how Republican Donald Trump and Democratic Hillary Clinton would fare in the first U.S. presidential debate. New home sales and the Dallas Fed’s manufacturing business index are expected on the economic calendar. European stocks fell, weighed down by a pullback in the shares of major banking companies, with Deutsche Bank hitting a record low. Asian companies were lower. Safe-haven yen strengthened and the greenback edged down. Oil rebounded after Algeria’s energy minister Noureddine Bouterfa said on Sunday that all options were possible for an output cut or freeze at this week’s informal meeting of OPEC producers. Gold was little changed.
Mateon Therapeutics announced a collaboration under which sites affiliated with US Oncology Research will participate in the FOCUS Study, Mateon’s recently initiated phase 2/3 clinical trial in platinum-resistant ovarian cancer.
Bioblast Pharma announced that Zohar Argov, MD, Special Medical Advisor to the Executive Chairman of Bioblast Pharma, will present an invited lecture on “Trehalose in Oculopharyngeal Muscular Dystrophy (OPMD)” at the annual scientific meeting of the Muscle Study Group Society (MSGS) to be held today in Snowbird, Utah. The MSGS is an interdisciplinary US-based group devoted to the development of treatments for neuromuscular disorders.
Onconova Therapeutics announced receipt of the End-of-Phase 2 meeting minutes from the U.S. Food and Drug Administration (FDA) for the combination of oral rigosertib with azacitidine for the treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS). Based on these discussions, Onconova will design a randomized, controlled Phase 3 clinical trial comparing the combination of oral rigosertib plus azacitidine to azacitidine plus placebo in 1st-line HR-MDS patients. The primary endpoint of this pivotal trial will be overall response rate.
Gemphire Therapeutics announced enrollment of its first patient in COBALT-1, a Phase 2b trial designed to investigate gemcabene in the treatment of homozygous familial hypercholesterolemia (HoFH). The purpose of this study is to assess the efficacy, safety, and tolerability of multiple rising doses of gemcabene in patients with HoFH who are on stable, lipid-lowering therapy, including statins, ezetimibe and Repatha.
SciClone Pharmaceuticals announced two important achievements in advancing the Company’s clinical pipeline and development portfolio in Greater China. The first patient has been treated in the Phase 3 trial of ZADAXIN® in sepsis, a life-threatening infectious disease in which the drug has previously demonstrated therapeutic benefit in a Phase 2 clinical trial. In addition, the first patient has been treated in the Phase 1 trial of PT-112, a multi-targeted platinum-pyrophosphate anticancer agent being developed for patients with advanced solid tumors.
MediciNova announced that the Chicago Board Options Exchange (CBOE) has listed options (calls and puts) for MediciNova common stock.
VBI Vaccines will present a corporate overview at the Ladenburg Thalmann Healthcare Conference on Tuesday, September 27th.
CoLucid Pharmaceuticals will provide a corporate overview at the Ladenburg Thalmann 2016 Healthcare Conference on September 27, 2016.
Pain Therapeutics announced that it has received a Complete Response Letter from the FDA on the resubmission of its NDA for REMOXY ER (oxycodone capsules CII). The CRL informs that REMOXY ER cannot be approved in its present form and specifies additional actions and data that are needed for drug approval.
Array BioPharma and Pierre Fabre jointly announced top-line results from Part 1 of the Phase III COLUMBUS (Combined LGX818 Used with MEK162 in BRAF Mutant Unresectable Skin Cancer) study evaluating LGX818 (encorafenib), a BRAF inhibitor, and MEK162 (binimetinib), a MEK inhibitor, in patients with BRAF-mutant advanced, unresectable or metastatic melanoma. The study met its primary endpoint, significantly improving progression free survival (PFS) compared with vemurafenib, a BRAF inhibitor, alone.
Regeneron Pharmaceuticals and Sanofi announced that the FDA has accepted for priority review the BLA for dupilumab for the treatment of adult patients with inadequately controlled moderate-to-severe atopic dermatitis (AD), a serious, chronic inflammatory skin disease. The application has been given a PDUFA target action date of March 29, 2017. The investigational antibody therapy dupilumab inhibits signaling of IL-4 and IL-13, two key cytokines required for the Type II (including Th2) immune response, which is believed to be a major driver in the pathogenesis of the disease.
GW Pharmaceuticals announced positive results of the second randomized, double-blind, placebo-controlled Phase III clinical trial of its investigational medicine Epidiolex (cannabidiol or CBD) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. In this trial, Epidiolex, when added to the patient’s current treatment, achieved the primary endpoint for both dose levels with high statistical significance. During the treatment period, patients taking Epidiolex 20mg/kg/day achieved a median reduction in monthly drop seizures of 42 percent compared with a reduction of 17 percent in patients taking placebo (p=0.0047), and patients taking Epidiolex 10mg/kg/day achieved a median reduction in monthly drop seizures of 37 percent compared with a reduction of 17 percent in patients taking placebo (p=0.0016).
Summit Therapeutics announced it has received Fast Track designation from the FDA for ezutromid in the treatment of DMD. Ezutromid is a utrophin modulator and represents a potential disease modifying treatment for all patients with the fatal muscle wasting disease DMD.
Biogen and Ionis announced that Biogen has completed the rolling submission of a NDA to the FDA for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Biogen has also applied for Priority Review which, if granted, would shorten the review period of nusinersen following the Agency’s acceptance of the NDA.
Axovant Sciences announced that the company has received Fast Track designation for its IND application for intepirdine in the treatment of dementia with Lewy bodies from the FDA.
Amgen announced that the FDA has approved AMJEVITA (adalimumab-atto) across all eligible indications of the reference product, Humira (adalimumab). AMJEVITA is the first adalimumab biosimilar approved by the FDA and has been approved for the treatment of seven inflammatory diseases, including moderate-to-severe rheumatoid arthritis, moderate-to-severe polyarticular juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, moderate-to-severe chronic plaque psoriasis, adult moderate-to-severe Crohn’s disease and moderate-to-severe ulcerative colitis. AMJEVITA is Amgen’s first biosimilar to receive regulatory approval.
Alnylam Pharmaceuticals announced initial data from its ongoing Phase I/II study with ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of Primary Hyperoxaluria Type 1 (PH1). Initial clinical results were presented during an oral presentation at the 17th Congress of the International Pediatric Nephrology Association (IPNA), being held September 20 – 24, 2016 in Iguaçu, Brazil. These data were from Part A of the ongoing Phase I/II study, which is being conducted in healthy adult volunteers. Results showed that single, subcutaneous doses of ALN-GO1 achieved dose-dependent increases in plasma and urine glycolate. Glycolate is the substrate used by the GO enzyme to produce excessive oxalate in patients with PH1; thus, increases in plasma and urine glycolate in normal volunteers confirm effective GO knockdown and provide preliminary human proof of concept for ALN-GO1. Further, ALN-GO1 was found to be generally well tolerated, with no serious adverse events (SAEs) reported through the safety data transfer date. The Company plans to soon transition to Part B of the Phase I/II study, which will evaluate multiple doses of ALN-GO1 in patients with PH1.
Corvus Pharmaceuticals announced preclinical data as well as preliminary biomarker data from its ongoing Phase I/Ib study of CPI-444 as a single agent and in combination with Genentech’s TECENTRIQ (atezolizumab), a fully humanized monoclonal antibody targeting protein programmed cell death ligand 1 (PD-L1). CPI-444 is a selective and potent inhibitor of the adenosine A2A receptor.
ERYTECH Pharma announced the final patient has been enrolled in its Phase II trial of eryaspase, also known as ERY-ASP or GRASPA, for the treatment of pancreatic cancer.
Exelixis announced that its partner Daiichi Sankyo Company has initiated a Phase III pivotal trial to evaluate CS-3150 (esaxerenone (r-INN)), an oral, non-steroidal, selective mineralocorticoid receptor antagonist, as a treatment for essential hypertension in Japanese patients. As a result of Daiichi Sankyo enrolling the first patient in the program’s Phase III pivotal trial, Exelixis is eligible for a $15 million milestone payment, which it expects to receive in the fourth quarter of 2016.
Vical Incorporated announced the initiation of a Phase II trial of the company’s Vaxfectin-formulated, bivalent, therapeutic DNA vaccine for herpes simplex virus type 2 (HSV-2) infection. The randomized, double-blind, placebo-controlled trial will evaluate the efficacy and safety of the vaccine in approximately 225 healthy adults aged 18 to 50 years with symptomatic genital HSV-2 infection at up to 15 U.S. clinical sites.
Pfizer announced that, after an extensive evaluation, the company’s Board of Directors and Executive Leadership Team have determined the company is best positioned to maximize future shareholder value creation in its current structure and will not pursue splitting Pfizer Innovative Health and Pfizer Essential Health into two, separate publicly traded companies at this time.
Fate Therapeutics announced that the FDA has granted Orphan Drug Designation for ProTmune, the Company’s lead product candidate that is currently undergoing Phase I/II clinical investigation. The FDA designation is for "prevention of graft-versus-host disease in patients undergoing allogeneic hematopoietic cell transplantation" and broadly covers diseases, including blood cancers and genetic disorders, for which the procedure is performed.
Lipocine reported positive top-line results from a Phase IIb clinical study of LPCN 1111, a novel oral testosterone replacement therapy ("TRT") product candidate. The primary objectives of the study were to determine the Phase III dose of LPCN 1111 along with the safety and tolerability of LPCN 1111 and its metabolites following oral administration of single and multiple doses in hypogonadal males.
Minerva Neurosciences announced recent progress in its clinical programs with MIN-101 for schizophrenia and with MIN-117 for major depressive disorder (MDD).
Cumberland Pharmaceuticals announced that it has launched the U.S. promotion of Ethyol (amifostine) for Injection, a hospital-based product used to treat oncology patients. Ethyol is an FDA approved cytoprotective drug indicated to reduce the incidence of xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer. It also reduces the cumulative renal toxicity associated with the repeated administration of cisplatin in patients with advanced ovarian cancer. Cumberland entered into an exclusive agreement with Clinigen Group to commercialize Ethyol in the United States earlier this year. Cumberland is responsible for all distribution, promotion, and medical support of Ethyol in the United States.
Aldeyra Therapeutics provided updates on its plans for late-stage clinical trials at the Aldeyra 2016 Research and Development Day. Aldeyra announced plans for the first-ever vehicle-controlled Phase III clinical trial in noninfectious anterior uveitis, as well as a first-ever Phase III clinical trial in Sjögren-Larsson Syndrome. Aldeyra also announced the expected advancement of ADX-102 (formerly NS2) to a Phase IIb clinical trial in allergic conjunctivitis, and the addition of a clinical program in dry eye syndrome.
Idera Pharmaceuticals reported initial clinical data from its ongoing Phase I/II clinical trial of intra-tumoral IMO-2125, a Toll-like receptor (TLR) 9 agonist. In this arm of the Phase I portion of the trial, IMO-2125 is being evaluated in combination with ipilimumab for treatment of patients with metastatic melanoma who have failed prior PD-1 therapy. These early results indicate that IMO-2125 is demonstrating promising clinical activity and is being well tolerated in a patient population with minimal options and low expectation of clinical response with ipilimumab treatment alone. Further clinical information from the ongoing dose escalation portion of the trial, as well as detailed information on the translational results, will be presented during an oral session at the 2016 Society for Immunotherapy of Cancer Annual Meeting beginning November 9th in Maryland.
Cytori Therapeutics announced that its licensee Kerastem Technologies, has completed enrollment of STYLE, a US phase II clinical trial investigating early stage female and male pattern hair loss.
AbbVie announced that it submitted a sNDA to the FDA for ibrutinib (IMBRUVICA) to treat patients with marginal zone lymphoma (MZL). MZL is a slow-growing form of non-Hodgkin’s lymphoma. The Company’s sNDA submission is based on data from a multi-center, open-label Phase II PCYC-1121-CA trial assessing ibrutinib as a single-agent treatment for MZL. If approved, MZL will be the fifth unique type of blood cancer indication for IMBRUVICA. IMBRUVICA is jointly developed and commercialized by Pharmacyclics, an AbbVie company and Janssen Biotech.
Dicerna Pharmaceuticals announced that the Company will focus its resources on its proprietary GalXC technology platform to advance development of product candidates in its core therapeutic areas of rare diseases, chronic liver diseases, cardiovascular disease and viral infectious diseases. Under this plan, Dicerna will transition its primary hyperoxaluria (PH) development program to focus on DCR-PHXC, a subcutaneously delivered GalXC clinical candidate, which was announced earlier this year. The Company also announced that it will discontinue clinical development of DCR-MYC, a DsiRNA-based therapeutic formulated as an EnCore(TM) lipid nanoparticle (LNP) for delivery to solid tumors, because preliminary results do not meet the Company’s expectations for further development.
Cancer Genetics announced that it has entered into a strategic alliance and partnership with ApoCell, the developer of ApoStream, a breakthrough, high-performance technology to detect, isolate and capture circulating tumor cells (CTC) and other rare cells without the need of labels or antibodies. ApoCell is a spin-out of globally recognized, University of Texas MD Anderson Cancer Center, and licenses key technology used in ApoStream from MD Anderson Cancer Center.
Adaptimmune Therapeutics and The University of Texas MD Anderson Cancer Center announced that they have entered into a multi-year strategic alliance designed to expedite the development of novel adoptive T-cell therapies for multiple types of cancer.
Beleave announced that it has received notification from Health Canada that, upon the Company’s confirmation that the Company’s proposed site and storage security measures are in place, functional and comply with the requirements of the Access to Cannabis for Medical Purposes Regulations (the "ACMPR") and the Security Directive, Health Canada will prepare to request a pre-licensing inspection of the Company’s proposed site. The issuance of a license under the ACMPR is in part dependent upon the completion of a satisfactory pre-licensing inspection by Health Canada of the Company’s proposed site.
PhytoPain Pharma, a subsidiary of GrowPros Cannabis Ventures, received an Acknowledgement Letter from the FDA after submitting a Request for Designation ("RFD"). PPP had filed a RFD for PPP001 to be classified as a drug and assigned to the Center for Drug Evaluation and Research ("CDER"). The RFD enables the FDA to determine the product type and appropriate lead center. If the FDA has not issued a designation letter within 60 calendar days of the filing of the RFD, PPP’s recommendation will become the designated classification and assignment.
Viking Therapeutics announced the presentation of positive data from a proof-of-concept study of VK0214 in a mouse model of X-linked adrenoleukodystrophy (X-ALD). The study successfully achieved its primary objective, which was to demonstrate the ability of VK0214 to lower plasma very long chain fatty acid (VLCFA) levels after six weeks of treatment.
Abeona Therapeutics announced that the first patient was enrolled in the Phase II portion of the clinical trial for EB-101 (gene-corrected skin grafts). EB-101 is the Company’s lead gene therapy program for patients suffering with recessive dystrophic epidermolysis bullosa (RDEB), a severe form of epidermolysis bullosa (EB).
Akebia Therapeutics announced the publication of a study which concluded that higher altitude is associated with higher hemoglobin levels and lower mortality, despite lower utilization of erythropoiesis-stimulating agent (ESA) and intravenous iron. Treatments that mimic the effect of higher altitude, such as HIF therapies, may provide beneficial effects for renal anemia patients, including improved iron mobilization and erythropoietin synthesis. The article, titled "The Effect of Altitude on Erythropoiesis-Stimulating Agent Dose, Hemoglobin Level, and Mortality in Hemodialysis Patients," was published online in the Journal of Nephrology.
Vela Diagnostics announced that the Sentosa SA ZIKV RT-PCR Test has received Emergency Use Authorization (EUA) from the FDA. The test, which is developed and manufactured in Singapore, also received the European CE mark for in-vitro diagnostic use in August 2016.
Capricor Therapeutics announced that the U.S. Department of Defense has awarded the company a grant in the amount of approximately $2.4 million to be used toward establishing a scalable, commercially-ready process to manufacture CAP-2003 (Cardiosphere-Derived Cell Exosomes). Capricor has identified ocular graft-versus-host disease (oGVHD) as the first clinical development opportunity for CAP-2003, and expects to submit an IND for this indication in the first half of 2017.
Versartis announced the initiation of the Phase III portion of its J14VR5 trial in Japanese children with GHD following the completion of the Phase II portion of the trial.
Shineco announced the pricing of its IPO of 1,713,190 common shares at a price of $4.50 per share. Shineco’s common shares are expected to begin trading on the NASDAQ Capital Market on September 28, 2016 under the symbol "TYHT".
Oryzon Genomics announced that it has closed a second tranche of Debt Funding round of 5.3 Million Euros (5.9 Million USD). These proceeds come on top of the 10.5 Million Euros (11.6 Million USD) raised in May.In total, Oryzon have raised more than 32 Million Euros (35.5 Million USD) since July 2015.
PAREXEL International announced that the Company has entered into a definitive agreement to acquire ExecuPharm. The acquisition is expected to close in the next three weeks. Terms of the transaction were not disclosed.
Ironwood Pharmaceuticals announced the closing of a $150 million debt refinancing. Ironwood will use the net proceeds from this transaction to redeem the remaining principal balance of its existing Linaclotide PhaRMA 11% Notes due 2024.
Imprimis Pharmaceuticals announced that it has implemented an expense reduction program to better leverage the company’s state-of-the-art drug production infrastructure, eliminate redundant expenses and enable near term profitability without hindering growth plans.
BioPharmX announced that it has entered into a definitive agreement with institutional investors to purchase shares of common stock for aggregate gross proceeds of approximately $930,000 in a registered direct offering. The closing of the offering is expected to take place on or about September 29, 2016, subject to the satisfaction of customary closing conditions. In connection with the offering, the Company will issue approximately 1,550,000 registered shares of common stock at a purchase price of $0.60 per share. Concurrently in a private placement, for each share of common stock purchased by an investor, such investor will receive from the Company an unregistered warrant to purchase $0.80 shares of common stock. The warrants have an exercise price of $0.75 per share, and shall be initially exercisable six months following issuance and terminate five years following the initial exercise date. Rodman & Renshaw, a unit of H.C. Wainwright & Co., LLC, acted as the exclusive placement agent in connection with this offering.
JMP analyst Liisa Bayko initiated coverage of Aimmune Therapeutics with a “market outperform” rating and $30 price target, citing the potential for significant value creation as data unfold and new initiatives are started in 2017 and beyond. The analyst also initiated coverage of DBV Technologies with a “market outperform” rating and $47 price target, citing the stock could migrate higher with an exciting catalyst calendar over the next 12 months highlighted in the near term by long-term 36-month data from a Phase II trial expected soon, followed by an exploration of sustained unresponsiveness, followed by Phase III data in 2017.
Guggenheim analyst Louise Chen initiated coverage of Xenon Pharmaceuticals with a “buy” rating and $20 price target, citing earnings upside could be driven by XENE’s pipeline and XENE is an interesting asset in a consolidating industry.
Janney analyst Debjit Chattopadhyay upgraded Applied Genetic Technologies to “neutral” from “sell,” citing limited to no-competition, anticipated expansion into multiple other indications, balance sheet to support operations for at least two years, and patient screening/identification nearing completion.
Wedbush analyst Liana Moussatos initiated coverage of Ascendis Pharma with a “outperform” and $34 price target, citing TransCon technology platform creates unique sustained-release prodrug candidates that reduce treatment burden and regulatory hurdles while retaining composition-of-matter IP.
Leerink analyst Michael Schmidt initiated coverage of Ariad Pharmaceuticals with an “outperform” rating and $20 price target, citing expect new management team has positioned a leaner, more focused company for commercial execution in the US while also maintaining future strategic flexibility.
HC Wainwright analyst Corey Davis initiated coverage of the following companies: Aldeyra Therapeutics with a “buy” rating and $18 price target; VtV Therapeutics with a “buy” rating and $13 price target.
Rodman & Renshaw analyst Raghuram Selvaraju initiated coverage of Cancer Genetics with a “buy” rating and $6 price target, citing CGI is an emerging molecular diagnostics company focusing primarily on the development and commercialization of high-content precision diagnostics in the oncology sector.
Rodman & Renshaw analyst Raghuram Selvaraju initiated coverage of Aytu Bioscience with a “buy” rating and $9 price target, citing AYTU is a commercial-stage specialty pharmaceutical company focused on urology indications.
JP Morgan analyst Anupam Rama assumed coverage of OvaScience due to a reallocation of resources. The stock was previously covered by Tycho Peterson.