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BioCryst Announces Positive Phase 1 Results with BCX9250, an Oral ALK-2 Inhibitor for Treatment of Fibrodysplasia Ossificans Progressiva
RESEARCH TRIANGLE PARK, N.C., Dec. 21, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that in a Phase 1 clinical trial
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"RESEARCH TRIANGLE PARK, N.C., Dec. 21, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that in 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), BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting once-daily dosing. FOP is an ultra-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. The randomized, double-blind, placebo-controlled dose-ranging trial evaluated safety, tolerability and pharmacokinetics of single ascending doses (SAD) and multiple ascending doses (MAD) of BCX9250 in healthy subjects. The SAD study was designed to randomize four cohorts of eight subjects each to receive oral BCX9250 (n=6) or placebo (n=2) at dose levels of 5 mg, 10 mg, 15 mg and 25 mg. Subjects in the 15 mg cohort also received a second single dose to evaluate food effect on absorption of BCX9250. The MAD study was designed to randomize four cohorts of 12 subjects each to receive oral BCX9250 (n=10) or placebo (n=2) at dose levels of 5 mg, 10 mg, 15 mg and 20 mg once daily (QD) for seven days. Drug exposure increased with dose in an approximately linear and dose-proportional manner. Drug levels after a high fat meal were similar to those after dosing on an empty stomach. Drug exposure (area under the curve) at 20 mg QD in the MAD was similar to that achieved with doses that suppressed HO in a nonclinical model of activity of orally dosed BCX9250. Additional data can be found in slides in the investors section of the company’s website at https://ir.biocryst.com/. In both the SAD and the MAD studies, oral BCX9250 was safe and well tolerated, with no serious adverse events, no study discontinuations due to adverse events, no grade 3 or 4 adverse events and no clinically significant changes in vital signs, electrocardiograms or safety laborato...