Satellos Bioscience Inc. has designated SAT-3153 as a development candidate for Duchenne muscular dystrophy and will move the candidate into pre-IND-enabling studies and toward an IND filing. SAT-3153 is a small molecule designed to inhibit a particular kinase protein which the company believes controls Notch polarity within muscle stem cells.
Satellos Bioscience Inc. has created, prioritized and advanced novel small-molecule drug candidates into further preclinical studies. The company's compounds have been designed to be potent and selective inhibitors of a particular kinase protein in the Notch pathway.
Metriopharm AG has received a grant of €125,000 from charity Duchenne UK that will support development of the company's lead compound MP-1032 (luminol sodium salt) for the treatment of Duchenne muscular dystrophy (DMD).
Solid Biosciences Inc. found a way to continue its Duchenne muscular dystrophy push, inking a deal to acquire privately held Aavantibio Inc., at the same time nailing down $75 million by way of a private placement with institutional investors.
Sarepta Therapeutics Inc. said it plans to file a BLA for its gene therapy for Duchenne muscular dystrophy (DMD), SRP-9001, with the U.S. FDA, potentially setting up a decision in the first half of 2023 for the therapy developed in partnership with Switzerland’s Roche Holding AG. The Cambridge, Mass.-based biotech said the BLA will seek accelerated approval for the therapy, also known as delandistrogene moxeparvovec, for ambulant individuals with DMD.
Another biopharma consultant got on the wrong side of the U.S. SEC for insider trading. To resolve the allegations, Hugh Lee Sweeney, a longtime consultant for Catabasis Pharmaceutical Inc., agreed to a cease-and-desist order, disgorgement of $57,931 and prejudgment interest of $2,499.71, and civil penalties of $57,931.
Italfarmaco SpA will seek meetings with the U.S. FDA and the European Medicines Agency to discuss filing requirements for givinostat in Duchenne muscular dystrophy (DMD) on the strength of data from a phase III trial in which those on the drug exhibited a slower decline in their ability to climb four stairs than those on placebo.
Sarepta Therapeutics Inc. aims to resolve “very quickly” the clinical hold placed by the U.S. FDA on SRP-5051 (vesleteplirsen) for Duchenne muscular dystrophy (DMD), said Louise Rodino-Klapac, chief scientific officer.
After an up-and-down day – mostly up, toward the end – during which the phrase “totality of the data” got air time aplenty, shares of PTC Therapeutics Inc. (NASDAQ:PTCT) closed at $34.07, a rise of $5.66, or almost 20%, on word of top-line data from Study 041 with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (DMD).
