Fibrodysplasia Ossificans Progressiva (FOP)
Pursuing activin receptor-like kinase-2 (ALK2) inhibition in the race to stop Fibrodysplasia Ossificans Progressiva (FOP).
BioCryst is developing activin receptor-like kinase-2 (ALK2) inhibitors for treatment of FOP. The ALK2 enzyme is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK2 gene are seen in all cases of FOP. An activating mutation in ALK2 is necessary for the disease to occur, making the ALK2 kinase an ideal drug target for treatment of FOP with an ALK2 kinase inhibitor.
The goal of BioCryst’s ALK2 inhibitor project is to discover and develop an orally administered kinase inhibitor that slows or prevents heterotopic ossification (HO). BioCryst is pursuing an ALK2 inhibitor molecule, BCX9250, that has dramatically reduced excess bone formation in an experimental model of ALK2-driven HO in laboratory rats. A Phase 1 clinical trial evaluating single and multiple ascending doses of oral BCX9250 in healthy volunteers has been completed.
BCX9250 is investigational and has not been deemed safe and effective by the FDA