Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients

Eloxx Pharmaceuticals, a leader in ribosomal RNA-targeted genetic therapies for rare diseases, have announced the final data assessment from the Phase 2 clinical trial of ELX-02 in combination with ivacaftor in Class 1 Cystic Fibrosis patients with at least one nonsense mutation. In the final assessment, ELX-02 demonstrated clinically relevant improvement in ppFEV1. The final data assessment includes a reanalysis using change in ppFEV1 from Day 1 instead of baseline, as multiple patients experienced disease progression between screening and treatment. Initial topline results from this trial were reported in September 2022.

“We believe that the clinical improvements with ELX-02 observed in a trial of CF patients with severe illness, coupled with remission observed in one patient in the ongoing Phase 2 trial of ELX-02 in Alport syndrome patients with nonsense mutations, reinforces our belief in the disease-modifying potential of ELX-02 and warrants further development. Similar to the patients in this Cystic Fibrosis trial, participants in our Phase 2 Alport syndrome trial also had highly progressive autosomally recessive disease, making a remission in even one patient highly clinically significant. The body of data strongly validate our decision to advance ELX-02 into a pivotal study in Alport syndrome,” said Sumit Aggarwal, President and CEO of Eloxx.

Mr. Aggarwal continued, “The high concentrations of ELX-02 observed in the kidney in those with Alport Syndrome make the disease an ideal target for further study, as the ELX-02 exposure in the Alport study is at least 25-fold higher than in the CF trial.”