Eloxx Pharmaceuticals, Inc. announced the opening of clinical trial sites for its Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations. This proof-of-concept clinical trial will include up to eight Alport syndrome patients with nonsense mutations in the second half of 2022. Patients will be dosed for two months with a three month follow-up.

Trial primary endpoints will include safety, while secondary endpoints will include reduction in proteinuria and induction of COL4A5 protein expression in the kidney. Topline results are expected in the first half of 2023. Eloxx believes there is a strong rationale to pursue clinical development of ELX-02 in Alport syndrome, based on encouraging preclinical results demonstrating potentially therapeutic levels of readthrough, ability to deliver high drug concentrations in the kidney relative to plasma at clinically tolerable dose levels, and the clinical readthrough results seen in the Phase 2 cystic fibrosis trial.