Blueprint Medicines Corp. is making the jump from cancer to rare disease with an inhibitor for the rare pediatric bone disease FOP that sets the company up against at least two competitors further ahead in development.
Blueprint is betting high selectivity and precision targeting will give its IND-ready BLU-782 a better benefit-risk profile than others in the field. It plans to test the compound in healthy volunteers in 1Q19.
On Oct. 24, Clementia Pharmaceuticals Inc. announced it will submit an NDA in 2H19 for palovarotene to prevent fibrodysplasia ossificans progressiva (FOP) associated with flare-up symptoms.
Other competitors are Regeneron Pharmaceuticals Inc., which has garetosmab in Phase II testing, and BioCryst Pharmaceuticals Inc., which also has a preclinical program slated to enter the clinic next half.
Patients with FOP form abnormal bone deposits throughout the body that are preceded by painful, inflammatory flare-ups. Symptoms can start in infancy, and patients typically become immobilized in their 20s.
The disease involves a progressive transformation of soft tissues and skeletal muscle into bone. It is caused by mutations in ALK2, a protein in the BMP pathway that controls development of the skeletal system.
In FOP, ALK2 mutations turn the receptor’s inhibitory ligands, paradoxically called activins, into activating ligands. Drug development has focused on blocking the aberrant ALK2 signaling, but selective inhibition has been hard to achieve because the seven members