BioCentury
ARTICLE | Clinical News

Blueprint debuts selective ALK2 inhibitor for rare musculoskeletal disease

October 5, 2018 7:27 PM UTC

Blueprint Medicines Corp. (NASDAQ:BPMC) provided the first glimpse of its preclinical ALK2 inhibitor program, BLU-782, when it presented data at the American Society for Bone and Mineral Research (ASBMR) meeting in Montreal on Sept. 30. Blueprint said the compound is headed for Phase I testing in 1Q19 for fibrodysplasia ossificans progressiva (FOP), following a planned IND submission by year end.

Data showed BLU-782 targeted with high specificity the R206H driving mutation in activin receptor-like kinase 2 (ALK2; ACVR1), which is present in nearly all FOP patients. Via an unknown mechanism, the mutation changes the kinase's response to its activin ligands such that the ligands activate, rather than inhibit the receptor. The switch thus spurs spontaneous and recurrent heterotopic bone formation that characterizes the disease...