Eloxx Pharmaceuticals Doses First Patients in Phase 2b Study of Exaluren for Alport Syndrome

Eloxx Pharmaceuticals has initiated dosing in its Phase 2b EXACT study, evaluating the use of exaluren in patients with nonsense mutation Alport syndrome. The company confirmed that the first two patients have received their initial doses. Alport syndrome is a rare genetic disorder that affects the kidneys and can lead to kidney failure.

The initial 16-week, placebo-controlled portion of the study is expected to yield topline data by mid-2027. The final readout for the entire study is anticipated by the end of 2027.

Exaluren is being investigated for its potential to correct the production of faulty proteins caused by nonsense mutations, which are a genetic basis for Alport syndrome. The therapy aims to restore the production of functional proteins.

This study seeks to demonstrate that exaluren can improve kidney function and slow the progression of the disease. Positive results could offer a new therapeutic option for patients with Alport syndrome, a condition with limited treatment alternatives.