Benzinga | Jan 13, 2021 09:02AM EST

Eloxx Pharmaceuticals Announces Cystic Fibrosis Foundation (CF Foundation) To Provide Increased Funding And Support For ELX-02 Global Phase 2 Clinical Trial Program

The CF Foundation's partial funding of the U.S. trial has been extended to partial funding of the global clinical trial program which includes Europe and Israel

Expect to report top line data from our Phase 2 cystic fibrosis clinical trial program for ELX-02 in the first half of 2021

Eloxx had previously reported the formation of a joint program advisory group with the CF Foundation focused on the development of ELX-02 in cystic fibrosis

WALTHAM, Mass., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc., (NASDAQ:ELOX) a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced an expansion of its agreement with the CF Foundation beyond the U.S. portion of the trial to include additional funding for the global clinical trial program which includes Europe and Israel.

"We are gratified by the CF Foundation's expanded support for our Phase 2 proof of concept global clinical trial program for ELX-02 for the treatment of patients with nonsense mediated cystic fibrosis, for whom there remains a high unmet medical need," said Dr. Gregory Williams, Chief Executive Officer of Eloxx Pharmaceuticals. "We Look forward to reporting top line data from this program in the first half of this year and are pleased that ELX-02 has been granted an orphan drug designation by the FDA and orphan medicinal product designation by the EMA."

Cystic Fibrosis Phase 2 Program

* Our Phase 2 program consists of two trials, one currently enrolling patients at sites in Europe and Israel and the second in the U.S. In the U.S., in addition to the partial funding being provided by the CF Foundation, our protocol has been sanctioned by the Cystic Fibrosis Therapeutics Development Network (TDN). In Europe, the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) has given our trial a "high priority" ranking.

* Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program at the Boston Children's Hospital/Brigham and Women's Hospital CF Center, is the lead study investigator in the U.S.

* We expect to report top line data from our proof of concept Phase 2 program in the first half of 2021, which is contingent on no further disruptions due to COVID-19.

* The Safety Review Committee has held several planned meetings and approved dose escalation up to the highest dose level. To date, we have had no reported serious adverse events in the clinical trial.

* We are participating in the European HIT-CF consortium to support the collection of cystic fibrosis patient-derived organoids and the initiative to conduct a prospective clinical trial to confirm the translational potential of the organoid model. The intent of the program is to use these positive results to enroll patients with responsive organoids in a prospective trial with ELX-02. We believe this program will continue to expand the application of organoid technology from drug discovery through drug approval, and also offers possible label expansion opportunities.

ELX-02 is an investigational agent not approved by any regulatory authority for therapeutic use, which is currently in Phase 2 clinical trials in cystic fibrosis.