Eloxx Pharmaceuticals Finalizes Design For Its EXPRESS Phase 2 Global Trial In Nonsense-Mutation Autosomal Dominant Polycystic Kidney Disease To Enrolol 60 Adults With Genetically Confirmed PKD1 Or PKD2 Nonsense Mutations

Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, today announced the finalized design of its EXPRESS Phase 2 global study in nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD) and the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer.

Following significant interactions between regulatory authorities and key opinion leaders, Eloxx has now finalized the design of its planned global ADPKD clinical trial. EXPRESS, a global Phase 2 clinical trial, is designed to enroll 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations, randomized 1:1 to receive exaluren or placebo for 52 weeks. The primary efficacy endpoint is the annualized rate of change in height-adjusted total kidney volume (htTKV), assessed by MRI at baseline and Weeks 28, 36 and 52. The design is intended to better characterize treatment effects over time and generate a full year of kidney-function and safety data. First patient dosing is expected in 3Q 2027, with 52-week topline results expected in 1H 2029.