Eloxx Pharmaceuticals Adds Additional Treatment Arm to Ongoing Phase 2 Clinical Studies for Cystic Fibrosis

WALTHAM, Mass. – Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) today announced the addition of a new study arm in 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. The Phase 2 clinical program now includes a fifth treatment arm to evaluate safety of ELX-02 in combination with Kalydeco (ivacaftor), an FDA-approved CFTR (CF transmembrane conductance regulator) potentiator for the treatment of cystic fibrosis in patients who have at least one mutation in their CF gene amenable to ivacaftor. The Phase 2 trials are designed to evaluate the safety of ELX-02 and assess its biological activity.

“We are extremely pleased to include this additional treatment arm in the ongoing Phase 2 clinical trial to further explore the potential of ELX-02 to treat cystic fibrosis,” said Sumit Aggarwal, President and Chief Executive Officer. “Given ELX-02’s potential to treat CFTR nonsense mutations and the synergistic effects of ELX-02 and ivacaftor seen to date in pre-clinical models, we believe that combination treatment with a CFTR potentiator, such as ivacaftor, provides the opportunity to improve clinical efficacy in the treatment of CF. We remain on track to present data from the first four treatment arms of the study in the second half of this year.”

ELX-02 is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR gene. The trial currently has sites in the U.S., Europe, Israel, Australia and Canada. Several planned Safety Review Committee meetings have occurred and allowed dose escalation up to the top dose level, and data have shown no drug-related serious adverse events reported to date. Multiple patients have progressed through the four-dose escalation treatment range. 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.

For more information, please visit www.eloxxpharma.com.


John Woolford
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Laureen Cassidy
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