Eloxx Pharmaceuticals (NASDAQ:ELOX) reported that the FDA cleared its IND for a Phase 2 trial of ELX-02 in patients with cystic fibrosis (CF).
ELX-02 is a small molecule designed to restore production of CF transmembrane conductance regulator proteins in the lungs.
“These patients have a high unmet medical need and few, if any, targeted treatment options,” Ahmet Uluer, director of Boston Children’s Hospital adult cystic fibrosis program, said in a statement.
“ELX-02 is the only therapy to have demonstrated positive results in organoids derived from CF patients across the majority of nonsense mutations and studies have shown the organoid model to be highly predictive of clinical benefit,” he added.
The company will launch a Phase 2 clinical trial in the U.S., Europe, Israel and Canada, with top line data expected later this year.
