Eloxx Pharmaceuticals, Inc. announced the final data assessment from the Phase 2 clinical trial of ELX-02 in combination with ivacaftor in Class 1 CF 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. Final Assessment of ELX-02 Phase 2 Combination Trial in Class 1 CF Patients: The Phase 2 combination clinical trial of ELX-02 was designed to evaluate safety and assess biological activity in G542X nonsense mutation Class 1 CF patients as monotherapy and in combination with ivacaftor after 5 weeks of treatment. Results from the final analysis of 13 patients evaluable, versus 11 at time of initial assessment are summarized below: 6 of 13 patients entered trial from monotherapy arm (after average 463 days) and had a decrease in lung function (annualized -4.26% reduction in ppFEV1) due to disease progression.

Treatment with ELX-02 stabilized disease overall and resulted in a clinically relevant increase in ppFEV1 in six of thirteen patients based on change in ppFEV1 at the end of treatment at Day 35 compared to the start of treatment at Day 1. Topline data previously confirmed biological activity. Patients with higher baseline sweat chloride levels had increased responses to treatment as indicated by sweat chloride concentration (p=0.00013 at Day 35). ELX-02 was generally well tolerated in the trial, with no treatment-related serious adverse events noted.