Professor Eitan Kerem, head of the Division of Pediatrics at the Hadassah Medical Organization, will serve as the Global Lead Investigator for this clinical trial.

Eloxx Pharmaceuticals, Inc. (ELOX), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis, inherited retinal disorders, and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that an IND for ELX-02 in cystic fibrosis is now open in the U.S., and the Phase 2 clinical trial has been endorsed by the Cystic Fibrosis Foundation (CFF).

Robert E. Ward, Chairman and Chief Executive Officer of Eloxx Pharmaceuticals stated, “We are very pleased that our IND is open in the U.S. and the protocol for our Phase 2 clinical trial in cystic fibrosis has been endorsed by the Cystic Fibrosis Foundation (CFF) in the U.S.”

He continued, “Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program at Boston Children’s Hospital/Brigham and Women’s Hospital Cystic Fibrosis Center, has agreed to be the lead study investigator in the U.S., and Prof. Eitan Kerem, M.D., head of the Division of Pediatrics at the Hadassah Medical Center, will serve as the Global Lead Investigator.

“While important progress has been made in the development of disease modifying treatments for patients with cystic fibrosis,” Prof. Kerem says, “patients with nonsense mutations represent the most severe phenotypes and often do not respond to currently available therapies. I am excited to lead this clinical trial of ELX-02 which may provide a new therapeutic approach for these patients.”

We are gratified by the participation of these two leading experts and we look forward to reporting top-line data later this year. We believe that the positive data we have generated for ELX-02 in cystic fibrosis patient-derived organoids substantially de-risk our Phase 2 program.”

Dr. Uluer adds, “I am very pleased to be leading the Phase 2 clinical trial of ELX-02 in cystic fibrosis patients with the G542X mutation on one or both alleles in the U.S. These patients have a high unmet medical need and few, if any, targeted treatment options. ELX-02 is the only therapy to have demonstrated positive results in organoids derived from cystic fibrosis patients across the majority of nonsense mutations, and studies have shown the organoid model to be highly predictive of clinical benefit.” 

Prof. Kerem relates, “While important progress has been made in the development of disease modifying treatments for patients with cystic fibrosis, patients with nonsense mutations represent the most severe phenotypes and often do not respond to currently available therapies. I am excited to lead this clinical trial of ELX-02 which may provide a new therapeutic approach for these patients.”