Eloxx Wins FDA Clearance for Exaluren Phase 2 Trial in Genetic Kidney Disease

Eloxx Pharmaceuticals (ELOX) said the FDA has cleared its investigational new drug application for exaluren in nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD), allowing U.S. sites to join the global Phase 2 EXPRESS study. The trial is expected to enroll about 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations, randomized to exaluren or placebo for 52 weeks. The primary endpoint is the annualized change in height-adjusted total kidney volume (htTKV), measured by MRI at baseline and Weeks 28, 36 and 52. First patient enrollment is expected in the third quarter of 2027, with topline results anticipated in the first half of 2029. The FDA indicated that a substantial, durable effect on htTKV supported by eGFR and safety data could potentially support Breakthrough Therapy Designation. Exaluren holds orphan drug designation for ADPKD in the U.S. and Europe. ELOX traded at $12.84, down 1.80%.