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First two patients dosed in Phase 2b trial of exaluren for rare kidney disease Alport syndrome

Company Fundamentals
11 Sep 2026
PRNewsWire
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Bullish
First two patients dosed in Phase 2b trial of exaluren for rare kidney disease Alport syndrome

Eloxx Pharmaceuticals has dosed the first two patients in its Phase 2b EXACT study testing exaluren for nonsense mutation Alport syndrome (NMAS), a rare genetic kidney disease with no FDA-approved treatments. The trial will evaluate the drug's safety and efficacy in 24 patients, with topline data from the initial 16-week placebo-controlled phase expected by mid-2027 and final results by the end of 2027. Exaluren aims to address the genetic cause by promoting production of functional protein, offering hope for patients who often face kidney failure by their twenties.

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