Eloxx Pharmaceuticals Provides Enrollment Update for Ongoing Phase 2 Clinical Studies for Cystic Fibrosis

Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today provided an update on enrollment for the ongoing global Phase 2 clinical program for ELX-02 for the treatment of cystic fibrosis (CF) in patients with at least one G542X allele. Based on enrollment to date, Eloxx expects to present data from the first four treatment arms of the study in the fourth quarter of this year. As of the end of June 2021, Eloxx has enrolled a sufficient number of patients to assess biological activity of ELX-02. Eloxx will continue to enroll additional patients to support Phase 3 clinical trial planning. The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity.

"Given the substantial efforts of our clinical team, we have made considerable progress in enrolling patients in our ongoing Phase 2 clinical program to explore the potential of ELX-02 to treat cystic fibrosis," said Sumit Aggarwal, President and Chief Executive Officer. "Given this progress, we are positioned to present data from the first four treatment arms of the study in the fourth quarter of this year."

ELX-02 is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene. The trial currently has sites in the U.S., Europe, Israel, Australia and Canada. Following several planned Safety Review Committee meetings, dose escalation has proceeded up to the top dose level, and data have shown no drug-related serious adverse events reported to date. Recently, a fifth treatment arm was added to the program to evaluate the safety of ELX-02 in combination with Kalydeco (ivacaftor), an FDA-approved CFTR potentiator for the treatment of cystic fibrosis in patients who have at least one mutation in their CF gene amenable to ivacaftor.

The program is partially funded by the Cystic Fibrosis Foundation (CFF).

The U.S. Food and Drug Administration has granted orphan drug designation for ELX-02 for the treatment of CF.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging both its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx's lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in clinical development focusing on cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx also has preclinical programs focused on select rare diseases including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.