Eloxx Pharmaceuticals Announces First Patients Dosed in Phase 2b EXACT Study of Exaluren in Nonsense Mutation Alport Syndrome
PRNewsWire
Published: Sep 11, 2026, 11:30 AM
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First two patients dosed in the EXACT study Topline data from the initial 16-week placebo-controlled portion of the study expected mid-2027; final readout expected by the end of 2027 ARLINGTON, Mass. , Sept. 11, 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 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).
"Dosing the first patients in the EXACT Study is an important milestone for Eloxx and, more importantly, 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. "This trial reflects our continued commitment to patients with nonsense mutation Alport syndrome, and we look forward to advancing exaluren through this Phase 2b study with topline data expected in mid-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. "Patients with this form of Alport syndrome can progress to kidney failure while still in their twenties, so there is real urgency behind bringing genetically targeted therapies like exaluren into clinical testing. Advancing this trial is an important step forward for a patient population that has been underserved for too long."
About the EXACT Study The EXACT study is a randomized, placebo-controlled, delayed start, global Phase 2b clinical trial 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-U.S. pediatric 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 biopsiesTopline data from this portion is expected mid-2027 and a final readout at 32 weeks is expected by the end of 2027.
About Nonsense Mutation Alport Syndrome (NMAS) Nonsense mutation Alport syndrome is a rare, inherited kidney disease caused by nonsense mutations in the COL4A3, COL4A4, or COL4A5 genes, which prevent normal production of type IV collagen, a key structural protein of the kidney's filtration barrier. Approximately 7% of Alport syndrome patients carry these nonsense mutations, which are associated with a more severe prognosis than missense mutations and a mean age of kidney failure of approximately 20 years. The disease is characterized by progressive kidney damage, including podocyte injury, hematuria, proteinuria, and kidney failure. There is currently no FDA-approved therapy that addresses the underlying genetic cause of NMAS.
About Exaluren 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 nonsense mutation Alport syndrome, 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.
About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. (Nasdaq: E...
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