Eloxx finalizes Phase 2 ADPKD trial design, names new CMO

Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX) has finalized the design of its EXPRESS Phase 2 global clinical trial evaluating exaluren in patients with nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD), and appointed T.R. Srinivas, M.D., as Senior Vice President and Chief Medical Officer.

The EXPRESS trial will enroll approximately 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations, randomized 1:1 to receive exaluren or placebo for 52 weeks. The primary endpoint is the annualized rate of change in height-adjusted total kidney volume (htTKV), measured by MRI at baseline and at weeks 28, 36, and 52. First patient dosing is expected in the third quarter of 2027, with 52-week topline results anticipated in the first half of 2029.

In written pre-IND feedback, the FDA noted that a substantial and durable htTKV treatment effect, supported by eGFR and safety data, could serve as a potential basis for Breakthrough Therapy Designation. The FDA also identifies change in htTKV as a potential surrogate endpoint for accelerated approval in ADPKD. Any expedited pathway would depend on clinical results and further FDA discussions.

Exaluren has received orphan drug designation for ADPKD in both the United States and Europe.

Dr. Srinivas joins Eloxx from Novartis, where he held U.S. Medical Affairs leadership responsibilities for ADPKD product development. He previously served as Chief Medical Officer at CareDx and as Chief of the Division of Nephrology and Hypertension at University Hospitals in Cleveland. He holds a medical degree from the All India Institute of Medical Sciences and completed training at Montefiore Medical Center and Columbia University.

Peer-reviewed preclinical data published separately show that exaluren restores full-length polycystin-1 in cells carrying PKD1 and PKD2 nonsense mutations, with readthrough accompanied by reduced signaling through cyst-promoting pathways.

ADPKD is estimated to affect 160,000 to 200,000 people in the United States, with approximately 26% of PKD1 and PKD2 mutations classified as nonsense mutations, according to the company's statement.

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