Eloxx Shares Plummet As Mid-Stage Cystic Fibrosis Study Results Miss Efficacy Endpoints

Eloxx Pharmaceuticals ELOX announced topline results from the Phase 2 clinical trial of ELX-02 in combination with ivacaftor in Class 1 cystic fibrosis (CF) patients with at least one nonsense mutation.

The Phase 2 clinical trial of ELX-02 in combination with ivacaftor was designed to evaluate safety and assess biological activity in G542X nonsense mutation Class 1 cystic fibrosis patients as monotherapy.

The combination trial of ELX-02 with ivacaftor was well tolerated but did not achieve statistical significance for efficacy endpoints, including changes from baseline in sweat chloride concentration (SCC) and percent forced expiratory volume (FEV1).

Sumit Aggarwal, President and Chief Executive Officer, commented: "We are disappointed that ELX-02 failed to achieve statistical significance for its key efficacy endpoints in this Phase 2 trial in combination with ivacaftor for the treatment of Class 1 CF. Despite this setback, we were pleased to observe that ELX-02 was well tolerated and demonstrated additional evidence of activity in this underserved patient population. We will work closely with the CF Foundation, as it has generously supported this trial, to determine the next steps in the development of ELX-02 for CF.”

The company is planning to initiate a proof-of-concept trial for ELX-02 in Alport syndrome, a rare kidney disease, later this year.

Price Action : Eloxx shares are trading around 40 percent down at $0.23 on Wednesday during after-hours session.