BioCryst begins phase 1 trial with BCX9250 for treatment of fibrodysplasia ossificans progressiva

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.

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