BCX17725

BCX17725 is a potent and selective investigational fusion protein therapeutic developed as a kallikrein 5 (KLK5) inhibitor for the treatment of Netherton syndrome, an ultra-rare, lifelong genetic disorder characterized by severe skin manifestations and caused by deficiency of the natural KLK5 inhibitor SPINK5. By inhibiting KLK5, a serine protease unregulated in Netherton syndrome, BCX17725 aims to address the underlying protein deficiency that drives disease pathology. It is BioCryst Pharmaceuticals' first protein therapeutic to enter clinical trials and is designed to provide best-in-class, potentially disease-modifying therapy for patients with significant unmet medical needs. The drug is currently being evaluated in Phase 1 clinical studies assessing safety, tolerability, pharmacokinetics, and immunogenicity in both healthy volunteers and patients with Netherton syndrome.