Eloxx announces first patients enrolled in Phase 2 study of ELX-02

Eloxx Pharmaceuticals announced that the first patients have now been enrolled in its Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations. "With enrollment of the first patients in our Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations, we remain on track to deliver topline clinical results from this trial in the first half of 2023," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. "ELX-02 treatment has demonstrated restoration of full-length protein in multiple preclinical models, including collagen IV and collagen VII in cells, and also clinical activity in our Phase 2 cystic fibrosis trial. We believe our results, previously conducted in vivo studies showing improvement of kidney function with collagen IV restoration and the ability to achieve high kidney drug levels at tolerable doses support the clinical development of ELX-02 in Alport syndrome." This Phase 2 trial is targeting dosing of up to eight Alport syndrome patients with nonsense mutations in the COL4 gene. In addition to the primary endpoint of safety, the key secondary efficacy endpoint of proteinuria will be measured every two weeks. Topline results are expected in the first half of 2023.