Eloxx Pharmaceuticals Inc（ELOX）資訊與事件ELOXEloxx Pharmaceuticals Inc

Eloxx Finalizes ADPKD Clinical Trial Design

Eloxx Pharmaceuticals announced the finalized design of its EXPRESS Phase 2 global study in nonsense-mutation autosomal dominant polycystic kidney disease. 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, 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. In written pre-IND feedback, FDA feedback noted that a substantial and durable height-adjusted Total Kidney Volume treatment effect, with supporting eGFR and safety data could serve as a potential basis for Breakthrough Therapy Designation. Separately, FDA identifies change in htTKV as a potential surrogate endpoint for accelerated approval in ADPKD. Any expedited pathway or potential designation would depend on clinical results and further FDA discussions. Exaluren has also now received orphan drug designations for ADPKD in the United States in addition to the previously announced Orphan Drug Designation in and Europe. Peer-reviewed preclinical data show exaluren restores full-length polycystin-1 in cells carrying PKD1 and PKD2 nonsense mutations, with readthrough accompanied by reduced signaling through cyst-promoting pathways.

Eloxx Pharmaceuticals Appoints T.R. Srinivas as Chief Medical Officer

Eloxx Pharmaceuticals announced the appointment of T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer. Dr. Srinivas will provide renal medical leadership across the Company's Alport and ADPKD programs, including medical strategy, interpretation of clinical evidence and investigator engagement. Dr. Srinivas brings extensive experience in nephrology, clinical research and medical leadership. At Novartis, his responsibilities included U.S. Medical Affairs leadership for ADPKD product development and contributions to the Phase 3 development program for farabursen.

Selling Stockholders Plan to Sell Up to 600,000 Shares

This filing relates to the offer and sale from time to time by the selling stockholders of up to 600,000 shares of common stock. The company is not selling any shares under this prospectus and will not receive any of the proceeds from the sale of shares by the selling stockholders.