CF Foundation Awards Up to $2M to Eloxx Pharmaceuticals for Additional Support of Phase 2 Clinical Studies of Potential Therapy for Nonsense Mutations

January 13, 2021 |

3 min read

Today, the Cystic Fibrosis Foundation announced a new award of up to $2 million to Eloxx Pharmaceuticals Inc. to support the global Phase 2 clinical program of ELX-02, a potential therapy to treat people with cystic fibrosis who have nonsense mutations.

BETHESDA, Md.--(BUSINESS WIRE)-- Today, the Cystic Fibrosis Foundation announced a new award of up to $2 million to Eloxx Pharmaceuticals Inc. to support the global Phase 2 clinical program of ELX-02, a potential therapy to treat people with cystic fibrosis who have nonsense mutations. In 2019, the Foundation provided Eloxx with an award of up to $1.6 million to support the development of ELX-02 in the U.S. This additional funding will provide continued support for a Phase 2 proof-of-concept clinical study currently being conducted in the U.S, and a related international Phase 2 study being conducted in Europe and Israel in people with CF who have at least one G542X mutation.

Preliminary results from these trials are expected in the first half of this year, which is contingent on no further disruptions due to COVID-19.

“The Cystic Fibrosis Foundation is committed to accelerating treatments for the underlying cause of disease for all people with CF,” said JP Clancy, MD, vice president of clinical research at the Cystic Fibrosis Foundation. “We are excited to support the clinical development of a therapy with the potential to benefit individuals who currently have no approved treatment options.”

Nonsense mutations (also known as “x” or “stop” mutations) cause cells to stop the production of the CFTR protein midway through the process, resulting in shortened, non-functional protein that the cell recognizes as defective and destroys. The investigational drug ELX-02 could potentially “read through” or bypass these premature stop signals in people with CF, resulting in the production of full-length functioning protein.

According to the Cystic Fibrosis Foundation Patient Registry, nearly 13% of people with CF in the U.S. have at least one nonsense mutation. Of that number, about 3% cannot benefit from any currently available therapy. In addition to ELX-02, the Foundation is supporting three other programs specifically focused on nonsense mutations, as well as four clinical development programs that could help people regardless of their CFTR mutations.

About the Path to a Cure

The CF Foundation launched its $500 million Path to a Cure initiative in October 2019. This initiative centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF. Each approach requires a different set of scientific tools and knowledge, leading the Foundation to bring together researchers and industry leaders from a range of disciplines to advance multiple areas of research in parallel. Innovators who are interested in pursuing programs in cystic fibrosis can learn more on our Path to a Cure page, which includes links to our specific funding opportunities.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.