Sept. 6 Quick Takes: FDA lifts clinical hold on Sarepta DMD trial
Plus Arsenal raises $220M and updates from Roche, Phoremost, Synlogic and more
Sarepta Therapeutics Inc. (NASDAQ:SRPT) said that FDA has removed the clinical hold on the 5051-201 trial evaluating vesleteplirsen (SRP-5051) to treat patients with Duchenne muscular dystrophy amenable to exon 51 skipping. Vesleteplirsen is a peptide-conjugated morpholino that binds to exon 51 of dystrophin pre-mRNA. FDA had placed the hold in June after a serious adverse event of hypomagnesemia. As part of the lift, Sarepta said it will adjust the global trial protocol to include expanded monitoring of urine biomarkers. Sarepta, which said it is on track to complete enrollment by year-end, fell $0.70 to $108.68 on Tuesday.
Arsenal Biosciences Inc. will take its cell therapy programs into clinical development with an oversubscribed, $220 million series B. New investors included Softbank Vision Fund 2, Bristol Myers Squibb Co. (NYSE:BMY), Byers Capital, Emerson Collective Investments, Green Sands, Hitachi Ventures, and Sixth Street. Also participating were existing investors Parker Institute for Cancer Immunotherapy (PICI), Westlake Village BioPartners, the University of California San Francisco Foundation Investment Company, Euclidean Capital, Waycross Ventures and Kleiner Perkins. Arsenal was founded by CEO Ken Drazan, former president of Grail Inc., and is developing CAR T therapies to treat solid tumors. ...