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. 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 orally administered kinase inhibitor drug candidates that slow or prevent HO. BioCryst is pursuing two ALK2 inhibitor molecules, BCX9250 and BCX9499, that have dramatically reduced excess bone formation in an experimental model of ALK2-driven HO in laboratory rats. Our plan is to move to Phase 1 clinical trials in the first half of 2019.