Eloxx Pharmaceuticals, Inc. provided an update on the continued advancement of ELX-02 for the treatment of Alport syndrome with nonsense mutations, including additional positive results from its Phase 2 clinical study evaluating ELX-02, as well as an update on ZKN-013. Alport syndrome is a rare genetic kidney disorder caused by mutations in COL4A3/4/5 genes, characterized by podocyte injury and impaired kidney filter function leading to proteinuria. Based on the encouraging clinical results to date, Eloxx has submitted an IND application with the U.S. FDA for Alport Syndrome.

All three patients (100% response rate) treated with ELX-02 in its proof-of-concept Phase 2 open-label clinical trial (NCT05448755) showed an improvement in podocyte foot process effacement, a hallmark of proteinuric kidney diseases like Alport syndrome.