BioCryst begins enrollment for Phase 1 trial evaluating BCX17725

BioCryst Pharmaceuticals announced the enrollment of the first participant in a Phase 1 trial evaluating BCX17725, a potent and selective investigational kallikrein 5, or KLK5, inhibitor designed to provide best-in-class disease-modifying treatment for people with Netherton syndrome. BCX17725 is a protein therapeutic that is designed to treat the underlying protein deficiency that causes Netherton syndrome by inhibiting KLK5, a serine protease in the skin that is unregulated in people with Netherton syndrome. The trial will evaluate the safety, tolerability, pharmacokinetics and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult participants, and multiple doses in participants with Netherton syndrome in an open-label design. The company expects to report initial results from the trial by the end of 2025. Netherton syndrome is a serious, rare, lifelong genetic disorder affecting the skin, hair, and immune system, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have red, scaly, inflamed skin, fragile hair, and are more likely to develop skin infections, allergies, asthma and eczema. Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments for Netherton syndrome.