Ridgeback Biotherapeutics LP said the FDA has granted orphan drug status to mAb-114, an experimental treatment for Ebola. It’s a step forward for the 4-year-old biotech focused on pediatric orphan and infectious diseases with limited or no treatment options for patients in the developing world.
Struggling Mereo Biopharma Group plc disclosed six-month data from the open-label arm of its phase IIb dose-ranging study in adults with type I, III or IV osteogenesis imperfecta treated with BPS-804 (setrusumab). Denise Scots-Knight, Mereo's CEO, said results from the Asteroid study were in line with company expectations and she expects 12-month headline data from blinded arms of the study in the second half of this year.
Amicus Therapeutics Inc. has upped the ante on its seven-month old collaboration with the University of Pennsylvania to research and develop gene therapies for lysosomal disorders and 12 additional rare diseases, many of them affecting extremely young children.
Eli Lilly and Co. acquired worldwide rights for CNTX-0290, a somatostatin receptor type 4 agonist as a non-opioid treatment for chronic pain, from Centrexion Therapeutics Corp.
Stoke Therapeutics Inc., fresh from its $90 million series B last October, has filed a prospectus for an IPO to raise up to $86.25 million. It is one of several companies in the past week that have filed for IPOs or are prepping for one.
Immunovant Inc., one of 14 'Vants in the Roivant Sciences Ltd. family, is initiating dosing in its phase IIa trial evaluating IMVT-1401 in patients with moderate to severe active Graves' ophthalmopathy.
It was time for Locana Inc., a San Diego-based biopharma, to grow itself, its science and its profile, according to newly installed director and CEO Jeffrey Ostrove, so the company sought out and just closed on an oversubscribed $55 million series A deal.
Ocular Therapeutix Inc.'s phase III trial of OTX-TP (travoprost), a sustained-release punctal plug for glaucoma and ocular hypertension, failed to meet its primary endpoint, but it also reached what it called a "statistically significant" reduction of intraocular pressure vs. placebo at eight of nine prespecified time points.
Abbvie Inc. is shuttering all studies of depatuxizumab mafodotin (ABT-414; Depatux-M) because a phase III trial of the drug demonstrated no survival benefit in newly diagnosed glioblastoma (GBM) patients.
Parvus Therapeutics Inc. and Genentech Inc. have agreed to develop, manufacture and commercialize Parvus' therapeutics to treat inflammatory bowel, autoimmune liver and celiac diseases. The platform gets the job done without immunosuppression, a singular approach and technology.
