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BioCryst Begins Phase 1 Trial With BCX9250, an Oral ALK-2 Inhibitor, for Treatment of Fibrodysplasia Ossificans Progressiva
—BCX9250 significantly suppressed heterotrophic ossification in preclinical studies— —Urgent medical need with no approved treatments for FOP — —Phase 1 data
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"—BCX9250 significantly suppressed heterotrophic ossification in preclinical studies—\n —Urgent medical need with no approved treatments for FOP — —Phase 1 data expected in 2H 2020— RESEARCH TRIANGLE PARK, N.C., Nov. 01, 2019 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the company has begun a Phase 1 clinical trial with BCX9250, an oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva (FOP). The Phase 1 trial will evaluate single and multiple ascending doses of oral BCX9250 in healthy volunteers. The company expects to report the results from the trial in the second half of 2020. “BCX9250 for FOP represents the third program for potent and selective oral compounds for rare diseases invented and developed by BioCryst to enter the clinic, joining our plasma kallikrein inhibitors for hereditary angioedema and Factor D inhibitor for complement-mediated diseases,” said Dr. William Sheridan, chief medical officer of BioCryst. “FOP is a devastating disease with no approved therapies, and we look forward to seeing clinical data with BCX9250 from this initial study in healthy volunteers to inform how we proceed strategically with the program,” Sheridan added. In preclinical studies, BCX9250 demonstrated potency for the target kinase, selectivity, safety and strong suppression of heterotopic ossification (HO) in animal models. About Fibrodysplasia Ossificans Progressiva Fibrodysplasia Ossificans Progressiva is a rare, severely disabling condition characterized by the irregular formation of bone outside the normal skeleton, also known as heterotopic ossification (HO). HO can occur in muscles, tendons and soft tissue. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities and premature mortality. There are currently no approved treatments for FOP. About BCX9250 Discovered by BioCryst, BCX9250 is a novel, oral, inhibitor of the ALK-2 enzyme. The ALK-2 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 ALK-2 gene ar...