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.
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).
Analysts have already started tagging Cogent Biosciences Inc.’s bezuclastinib as potentially best in class, after the company presented impressive, though early stage, data at the European Hematology Association Congress in Vienna demonstrating promising efficacy and a possibly differentiating safety profile for the selective KIT D816V inhibitor in advanced systemic mastocytosis.
Code Biotherapeutics Inc. has raised an upsized and oversubscribed series A financing to develop programs for treating rare and genetic diseases that include Duchenne muscular dystrophy and type 1 diabetes.
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