CFF Awards Up to $2M to Advance Eloxx’s Therapy ELX-02

The Cystic Fibrosis Foundation (CFF) is awarding Eloxx Pharmaceuticals up to $2 million in additional funding to support the clinical program of ELX-02, a potential therapy for cystic fibrosis (CF) caused by nonsense mutations.

“The Cystic Fibrosis Foundation is committed to accelerating treatments for the underlying cause of disease for all people with CF,” JP Clancy, MD, vice president of clinical research at the Cystic Fibrosis Foundation, said in a press release.

CF is caused by mutations in the CFTR gene. One type of CF-causing mutation is known as a nonsense mutation. This type of mutation creates an early stop signal in the gene sequence, resulting in the production of a shorter, incomplete, and non-functional CFTR protein. Without this protein, the transport of water and ions in cells of the lungs and other organs is impaired, resulting in the production of a thick and sticky mucus that affects their function and promotes infections.

Almost 13% of CF patients in the U.S. have at least one nonsense mutation, according to the CFF Patient Registry. Of those, about 3% do not benefit from any currently available therapy.

“We are excited to support the clinical development of a therapy with the potential to benefit individuals who currently have no approved treatment options,” Clancy said.

Eloxx’s investigational therapy, ELX-02, is designed to restore the production of a functional CFTR protein. The therapy helps the ribosome — the cells’ protein-building machinery — ignore the early stop signal (a process called read-through) to generate a fully working CFTR protein.

In 2019, the CFF awarded Eloxx with up to $1.6 million to support the development of ELX-02 in the U.S. Results from Phase 1 clinical trials conducted in healthy volunteers showed that ELX-02 was generally well-tolerated, and preclinical studies have supported its efficacy.

Additional funding now will provide continued support to the ongoing Phase 2 clinical trials — EL-012 (NCT04135495) and EL-004 (NCT04126473).

EL-012 is a proof-of-concept clinical study recruiting up to eight adult patients at clinical sites across the U.S.; more information and contacts can be found here. EL-004 is a related international study currently enrolling up to 16 eligible adults in Europe and Israel; more information it can be found here.

These two studies are evaluating the safety, tolerability, pharmacokinetics (the therapy’s movement into, through, and out of the body) and pharmacodynamics (the therapy’s effects on the body) of ELX-02 in the treatment of CF patients with at least one G542X mutation in CFTR, the most common CF-causing nonsense mutation.

Preliminary results from these clinical trials are expected in the first half of this year.

Besides ELX-02, the CFF also is supporting three other programs focused on nonsense mutations and four clinical development programs that could help CF patients regardless of their CFTR mutations. This support is part of the Foundation’s initiative Path to a Cure, of which the goal is to address the root cause of CF.