BioCryst Pharmaceuticals has initiated a Phase I clinical trial to treat fibrodysplasia ossificans progressiva (FOP) using its activin receptor-like kinase-2 (ALK-2) inhibitor, BCX9250.

FOP is a rare disease that leads to the abnormal formation of bone outside the normal skeleton, called heterotopic ossification (HO).

It limits movement and is characterised by fused joints. The disease currently lacks approved treatments.

The ALK-2 enzyme, which is involved in the signalling pathway for bone formation, works by inducing normal bone growth and renewal in healthy individuals. ALK-2 gene mutations are found in all FOP patients.

The inhibition of the ALK-2 enzyme is intended to slow or prevent the progressive formation of HO.

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BCX9250 was discovered by BioCryst and showed potency for the target kinase, selectivity, safety and suppression of HO in animal models.

During the Phase I trial, single and multiple ascending doses of oral BCX9250 will be assessed in healthy participants. Data from the trial is expected to be available in the second half of next year.

BioCryst Pharmaceuticals chief medical officer Dr William Sheridan said: “BCX9250 for FOP represents the third programme 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.

“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 programme.”

The company is also developing drug candidates for the treatment of conditions including hereditary angioedema, complement-mediated diseases, Marburg virus, and yellow fever.

BioCryst’s viral neuraminidase inhibitor, Rapivab, has regulatory approval in multiple markets for the treatment of influenza.