Eloxx Pharmaceuticals Announces FDA Clearance of Investigational New Drug Application for Exaluren in Nonsense-Mutation ADPKD
Key Highlights
- ➤FDA clears Eloxx (ELOX) IND, enabling U.S. sites for exaluren’s EXPRESS Phase 2 study
- ➤EXPRESS will randomize approximately 60 patients 1:1 to exaluren or placebo for 52 weeks
- ➤First patient dosing expected in 3Q 2027; topline 52-week data expected in 1H 2029
- ➤FDA says substantial, durable htTKV effects could support Breakthrough Therapy Designation
- ➤FDA identifies htTKV change as a potential surrogate endpoint for accelerated ADPKD approval
Expert Statements
Sumit Aggarwal, President and Chief Executive Officer of Eloxx Pharmaceuticals
“IND clearance is an important milestone for people living with nonsense-mutation ADPKD, who have no therapy that addresses the genetic cause of their disease.”
Sumit Aggarwal, President and Chief Executive Officer of Eloxx Pharmaceuticals
“With the trial design finalized, our focus now is execution: activating sites and preparing to dose the first patient in the third quarter of 2027.”
Eloxx Pharmaceuticals Announces FDA Clearance of Investigational New Drug Application for Exaluren in Nonsense-Mutation ADPKD PR Newswire
* FDA clearance of the IND enables Eloxx to open U.S. clinical sites for EXPRESS, its global Phase 2 study of exaluren in nonsense-mutation ADPKD * EXPRESS will randomize approximately 60 patients 1:1 to exaluren or placebo for 52 weeks, with annualized htTKV growth as the primary endpoint; first patient dosing expected in 3Q 2027 and topline data in 1H 2029 * FDA pre-IND feedback indicated that a substantial and durable htTKV effect could support Breakthrough Therapy Designation; FDA also lists htTKV as a potential surrogate endpoint for accelerated approval in ADPKD ARLINGTON, Mass., Oct. 29, 2026 /PRNewswire/ -- Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application for exaluren for the treatment of nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD). The IND clearance enables Eloxx to initiate EXPRESS, its global Phase 2 clinical trial, at clinical sites in the United States.
Get started
Create a free account to read the full story.