Eloxx Begins Phase 2b Trial of Exaluren for Alport Syndrome

Eloxx Pharmaceuticals has dosed the first two patients in its Phase 2b EXACT study. This global clinical trial evaluates exaluren as a treatment for nonsense mutation Alport syndrome (NMAS). The trial focuses on the underlying genetic causes of the condition, as there are currently no FDA-approved, genetically targeted therapies available.

"Dosing the first patients in the EXACT Study is an important milestone for Eloxx and for the Alport syndrome community, where no FDA-approved therapy currently addresses the underlying genetic cause of disease," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx, in a statement.

Study Design and Timeline

The EXACT study is a randomized, placebo-controlled trial designed to enroll 24 patients. According to a company press release, the study will provide an initial data readout from the 16-week placebo-controlled period by mid-2027. A final study readout at the 32-week mark is anticipated by the end of 2027.

"Dosing the first patients in the EXACT Study is a significant step for people living with nonsense mutation Alport syndrome, a disease with a clear genetic cause but no approved treatment that addresses it directly," said Professor Daniel Gale, Professor of Nephrology at University College London and Consultant Nephrologist at the Royal Free London NHS Foundation Trust.

Understanding Nonsense Mutation Alport Syndrome

Alport syndrome is a genetic disorder characterized by kidney disease, hearing loss, and eye abnormalities. The specific subset targeted by Eloxx, NMAS, affects approximately 7% of the broader Alport syndrome patient population. This form of the disease is aggressive, with a mean age of kidney failure occurring at approximately 20 years.

Financial Context

The initiation of the Phase 2b trial is a development phase for the company as it seeks to validate exaluren's efficacy in a rare disease space. As of September 14, 2026, Eloxx Pharmaceuticals (ELOX) was trading at $15.88.