Biogen Inc. has enrolled the first patient in a global phase III study of BIIB-093 (I.V. glibenclamide) for the prevention and treatment of severe cerebral edema in large hemispheric infarction, one of the most severe types of ischemic stroke. The trial, governed by an FDA special protocol assessment, will measure the fast-tracked drug's effect on a rating of functional outcomes at 90 days following treatment.
Pfizer Inc. is quitting a pair of phase II studies testing its experimental Duchenne muscular dystrophy (DMD) drug domagrozumab after finding the anti-myostatin candidate offered boys with DMD no more benefit than a placebo during a stair-climbing test. The candidate was one of the 15 programs the company had identified as potential blockbuster medicines. The company's early stage gene therapy candidate for DMD remains in development.
An FDA complete response letter (CRL) for the experimental familial chylomicronemia syndrome (FCS) drug Waylivra (volanesorsen) left the team at Ionis Pharmaceuticals Inc. subsidiary Akcea Therapeutics Inc. disappointed and possibly surprised following a qualified endorsement of the candidate by an agency advisory committee last spring. Akcea relayed nothing about the agency's reasons for the response, but concerns over the drug's safety, clinical benefit and trial population arose in the adcom meeting last May.
New data from a phase III study of Pfizer Inc.'s tafamidis in treating the rare and fatal disorder transthyretin amyloid cardiomyopathy (ATTR-CM) showed a 30 percent reduction in the risk of mortality and 32 percent reduction in the rate of cardiovascular-related hospitalizations vs. placebo. The data, presented at the European Society of Cardiology Congress in Munich and simultaneously published online in The New England Journal of Medicine, are likely to lay the groundwork for an upcoming U.S. regulatory filing for the drug.
A phase III test combining Novartis AG's phosphoinositide 3-kinase (PI3K) inhibitor, BYL-719 (alpelisib), with fulvestrant to treat a genetically-defined subset of advanced breast cancer patients found that it improved progression-free survival in those individuals, as compared to treatment with fulvestrant alone. The top-line success set the stage for the company to move closer to filing global regulatory applications for the drug, which Novartis oncology chief Samit Hirawat called the first of its kind "to show potential increased benefit and acceptable tolerability for patients."
Psilocybin, the psychoactive core of magic mushrooms, could be headed toward late-stage trials in treatment-resistant depression (TRD), pending the results of a soon-to-start phase IIb study. London-based Compass Pathways Ltd. has received permission from the FDA and additional regulators to run a dose-ranging study in up to 216 patients across Europe and North America. The trial, which seeks to enroll 216 patients at 12 to 15 research sites, will begin in the U.K. later this month and is expected to read out toward the end of 2019.
An NIH workshop aimed at promoting the efficient development and regulation of gene therapies highlighted a crucial bottleneck for the field Tuesday, as policy members of the rare disease community raised substantial questions about how to value and pay for the therapies. With three gene therapies already approved in the U.S. and a growing queue of new ones in development, panelists painted a picture of financial and institutional innovation struggling to keep pace with medical innovation.
The world-saving antics of Sandra Boynton's Amazing Cow appear to have a real-life equivalent in the transgenic cattle of SAB Biotherapeutics Inc. An anti-Ebola immunotherapy produced by company cows has proved fully protective against a lethal dose of the killer virus in a recent animal study run by the U.S. National Interagency Confederation for Biological Research.
Cleveland Biolabs Inc. (CLBI), a company that has long seen the potential for additional applications for its acute radiation exposure drug, entolimod, is putting the TLR5 agonist at the center of Genome Protection Inc. (GPI), a new joint venture formed with Everon Biosciences Inc. to develop and commercialize drugs for anti-aging applications, including frailty. Initially funded through a $10.5 million equity financing with venture capital fund Norma Investments Ltd., GPI's portfolio also includes small-molecule and vaccine candidates from Everon intended to protect organisms from the accumulation of cells with damaged DNA that drives aging and multiple age-related diseases, including cancer.
Magnolia Neurosciences Corp., a startup developing a drug with the mission of making neurons harder to kill, launched with a $31 million series A financing backed by a roster heavy with corporate and regional venture funds. Though headquartered in New York, where it will be managed by Accelerator Life Science Partners, Magnolia is expected to develop medicines based primarily on discoveries made by researchers in Houston-based MD Anderson's Therapeutics Discovery division, a unit that has already sparked the formation of at least two new cancer-focused companies, Navire Pharma Inc. and Vescor LLC.
