Eloxx Pharmaceuticals Inc (Nasdaq:ELOX), a clinical-stage biopharmaceutical company developing exaluren for the treatment of rare kidney diseases, announced on Friday that the first two patients have been dosed in the EXACT Study, a Phase 2b clinical trial evaluating exaluren in patients with nonsense mutation Alport syndrome (NMAS).

The randomised, placebo-controlled, delayed start, global Phase 2b clinical trial is designed to evaluate the safety and efficacy of exaluren in patients with NMAS caused by nonsense mutations in COL4A3, COL4A4, or COL4A5 in 24 patients. The trial's initial portion is placebo-controlled over 16 weeks, with a primary efficacy endpoint for non-US paediatric patients and all adult patients is to evaluate the effect of exaluren on structural changes in podocyte FPE as measured by change in FSD in kidney biopsies. Topline data from this portion is expected mid-2027 and a final readout at 32 weeks is expected by the end of 2027.

Exaluren is Eloxx's investigational, small-molecule ribosomal modulator designed to promote readthrough of premature stop codons caused by nonsense mutations, enabling production of full-length, functional protein. Exaluren is being developed for the treatment of rare kidney diseases, including NMAS, for which it is being evaluated in the Phase 2b EXACT Study, and autosomal dominant polycystic kidney disease (ADPKD), for which a Phase 2 clinical trial is planned.

The FDA and European Commission have granted orphan drug designation to exaluren for the treatment of Alport syndrome, and the European Commission has granted orphan medicinal product designation to exaluren for the treatment of ADPKD.