ArticleJBMR plus2026
BCX9250 is a potent small-molecule inhibitor of Activin A Receptor Type 1 (ACVR1/ALK2) for fibrodysplasia ossificans progressiva.
Article in JBMR plus, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disease of heterotopic ossification (HO) caused by mutations in the activin receptor-like kinase-2 (ALK2) protein, a BMP type I receptor of the TGF-β superfamily. These mutations in the ALK2 receptor alter receptor function, causing activin A, a protein that normally inhibits ALK2 signaling, to aberrantly trigger suppressor of mothers against decapentaplegic 1/5/8 (SMAD1/5/8) signaling, thereby activating the chondroosteogenic pathway and leading to HO. Here we report the initial characterization of BCX9250, an orally available small-molecule inhibitor of ALK2 developed by BioCryst with the potential to treat FOP. Inhibition of ALK2-mediated signaling by BCX9250 was assessed using biochemical binding assays and in vitro activity assays with the mouse myoblast C2C12 cell line. The impact of BCX9250 on HO was investigated using 2 mouse models and an HO recurrence mouse model. BCX9250 has potent inhibitory activity against wild-type ALK2 protein and mutant ALK2 protein associated with FOP. In the ALK2
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