After missing the primary and secondary endpoints in its phase III study of losmapimod in treating the rare disease facioscapulohumeral muscular dystrophy (FHSD), Fulcrum Therapeutics Inc. is yanking the program’s plug. The selective p38α/β mitogen activated protein kinase inhibitor had a lot of money behind it. It was originally in-licensed from GSK plc and then, in May, Sanofi SA signed on to help Fulcrum develop and commercialize losmapimod for FHSD worldwide, excluding the U.S., in a deal worth $1.06 billion.
Fulcrum Therapeutics Inc.’s deal with Sanofi SA to develop and commercialize oral losmapimod shone more light on facioscapulohumeral muscular dystrophy (FSHD), a rare genetic disease where Avidity Biosciences Inc. also has an earlier-stage but high-profile program.
Shares of Fulcrum Therapeutics Inc. shot up 38.5% on Aug. 22 following news that the U.S. FDA had lifted the clinical hold on the company’s phase Ib sickle cell disease candidate, FTX-6058.
Fulcrum Therapeutics Inc. has entered into a worldwide, exclusive license agreement with Camp4 Therapeutics Corp. to advance the discovery, development and commercialization of new therapeutic agents against an undisclosed target for the potential treatment of Diamond-Blackfan anemia (DBA).
At the European Hematology Association's annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
At the European Hematology Association’s annual meeting in Vienna last week, companies reported impressive progress for the treatment of sickle cell disease.
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.
Fulcrum Therapeutics Inc. shares (NASDAQ:FULC) closed at $18.77, up $10.44 or 125%, on word of positive interim results from a phase I trial in healthy adult volunteers with oral FTX-6058 for sickle cell disease (SCD). The firm has “already achieved maximal target engagement [MTE] at all three doses,” said Christopher Morabito, the company’s chief medical officer. “I don’t think we’ll exceed that.”
Fulcrum Therapeutics Inc.’s phase IIb data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) brought renewed hope for patients in what historically has proved a challenging therapeutic space. Though the firm’s oral p38 mitogen-activated protein kinase inhibitor missed its primary biomarker endpoint – changes in DUX4-driven gene expression – other indicators of benefit in the study called ReDUX4 painted a bright picture.
An interim analysis from Fulcrum Therapeutics Inc.’s phase II study of losmapimod for treating facioscapulohumeral muscular dystrophy (FSHD) produced data that pleased the CEO and displeased investors.
