Topline Phase 2 Trial Data on ELX-02 Expected in First Half of 2020, Eloxx Says

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by Iqra Mumal MSc |

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Splicing Defects, oligonucleotides

Eloxx Pharmaceuticals anticipates reporting topline data from the Phase 2 clinical trial program for ELX-02 — its lead therapeutic candidate to treat cystic fibrosis caused by nonsense mutations — during the first half of 2020, the company has announced.

“We are committed to ensuring full enrollment of our Phase 2 cystic fibrosis clinical trials and reporting topline data in the first half of this year,” Gregory Williams, PhD, Eloxx’s newly appointed CEO, said in a press release.

Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTR gene, which causes the CFTR protein to be made incorrectly or not at all. Some patients with CF have a mutation known as a nonsense mutation, which results in a premature stop signal in the CFTR gene, causing the cellular machinery to stop making the CFTR protein midway.

ELX-02 is a small molecule designed to restore the production of full-length functional CFTR protein in patients with a nonsense mutation. It is currently in the early stages of clinical development for CF.

Eloxx believes that data from both pre-clinical and clinical studies that have been collected to date support the continued development of its program.

The company’s board of directors have approved a re-structuring of the its leadership and organization to decrease the managerial layers, reduce operating expenses, and combine roles across the company.

Additionally, changes have been made to top positions, including the CEO and chief operating officer (COO) positions.

“We are confident that the succession of Dr. Gregory Williams to the position of CEO and Neil Belloff, Esq. to COO provides the right leadership to deliver our novel therapeutics to patients bearing nonsense mutations in rare genetic disorders in a timely and cost efficient manner,” said Tomer Kariv, who was recently chosen as chairman of the board.

This re-structuring will allow Eloxx to carry out its development plans, and reach Phase 2 proof-of-concept with ELX-02.

In the release, Eloxx expressed its gratitude to Bob Ward, the departing chairman and CEO of the company, as well as to other former employees, for their contributions that allowed Eloxx to progress to this critical stage of development by launching operations in the United States, listing Eloxx on Nasdaq, and progressing the company’s research and development programs.

“I am confident that our streamlined organization has the capabilities and resources necessary to achieve our clinical and portfolio objectives,” Williams said. “We are also extremely gratified by the level of engagement for our Phase 2 CF clinical trials for ELX-02 by leading global investigators and patient advocacy groups.”

Eloxx announced earlier this year that enrollment was nearly complete for two Phase 2 trials (NCT04135495, NCT04126473) testing ELX-02 in CF patients.


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