Arlington, Mass. – – September 16, 2026 -- Eloxx Pharmaceuticals, Inc. (Nasdaq: ELOX) has dosed the first two patients in the EXACT Study, a Phase 2b trial evaluating its investigational drug exaluren in patients with nonsense mutation Alport syndrome (NMAS), a rare kidney disease with no FDA-approved therapy targeting its genetic cause.
Trial targets 24 patients with a 16-week placebo-controlled start
The global, randomized, delayed-start Phase 2b trial will enroll 24 patients carrying nonsense mutations in the COL4A3, COL4A4, or COL4A5 genes. The initial 16-week placebo-controlled portion measures the drug's effect on structural podocyte changes via foot process effacement, assessed through kidney biopsies in non-U.S. pediatric patients and all adult participants.
Topline data expected mid-2027, final readout by year-end
Eloxx expects topline results from the 16-week placebo-controlled segment in mid-2027, with a final readout at 32 weeks anticipated by the end of 2027. CEO Sumit Aggarwal called the dosing milestone central to the company's commitment to the Alport syndrome community.
Disease strikes young, with kidney failure averaging age 20
NMAS accounts for roughly 7% of Alport syndrome cases and carries a more severe prognosis than missense mutations, with a mean age of kidney failure of approximately 20 years. Professor Daniel Gale of University College London noted that affected patients can progress to kidney failure while still in their twenties, underscoring the urgency for genetically targeted therapies.
Exaluren already holds orphan drug status in two markets
The FDA and European Commission have granted orphan drug designation to exaluren for Alport syndrome, while the European Commission has separately granted orphan medicinal product designation for autosomal dominant polycystic kidney disease (ADPKD), where a Phase 2 trial is planned. Exaluren works as a small-molecule ribosomal modulator designed to enable readthrough of premature stop codons caused by nonsense mutations, restoring production of full-length functional protein.