CF Foundation Awards Eloxx $1.61M for Phase 2 Trials in People with Nonsense Mutations

The Cystic Fibrosis Foundation (CFF) is giving Eloxx Pharmaceuticals up to $1.61 million to support its planned Phase 2 clinical trial program assessing the safety, tolerability, and chemical properties of ELX-02, Eloxx’s lead investigational compound to treat cystic fibrosis (CF) caused by nonsense, or stop, mutations.

The program is set to include two open-label, dose escalation, Phase 2 trials. One, called EL-012, will enroll up to eight patients in the U.S.; the other, EL-004, will enroll up to 16 patients in Europe.

Both studies will focus on the effects of multiple doses of ELX-02 in patients with at least one G542X allele.

The G542X allele is an abnormal variant of the CFTR gene (the gene defective in CF patients) that is included in Class I, which comprises a group of nonsense mutations that insert a stop signal in the coding sequence of CFTR. Because of this premature stop signal, the production of the CFTR protein halts, leading to the production of a shorter, non-functional protein.

ELX-012 is an experimental treatment that targets ribosomes — the small structures that are responsible for the production of proteins in cells — to tell them to bypass the stop signal in the mutated CFTR coding sequence. In this way, ELX-012 increases the amount of full-length CFTR protein that is being produced, potentially minimizing the effects of CF.

The financial support provided by the CFF will help to bring EL-012 in testing in patients. The U.S. study will take place at clinical sites that are part of CFF’s Therapeutics Development Network, the largest global network of CF clinical trials.

The company anticipates that early trial data will be available before the end of the year.

“We are very gratified by the CF Foundation’s support as we advance our development program for ELX-02 in cystic fibrosis. The CF Foundation’s funding will accelerate the program in the U.S.,” Robert E. Ward, chairman and chief executive officer of Eloxx Pharmaceuticals, said in a press release.

“We are also pleased that Dr. Ahmet Uluer, director of the Adult Cystic Fibrosis Program at the Boston Children’s Hospital has agreed to be the lead investigator in the U.S., and we look forward to reporting top line data from this trial later this year,” Ward added.

If Eloxx’s clinical trial program succeeds, ELX-02 may become the first treatment option for CF patients carrying at least one nonsense mutation. This would fulfill one of the top priorities of CFF: identifying and implementing therapies that tackle the root cause of CF.

“Despite extraordinary progress in helping people with CF live longer and healthier lives, there is still critical work to be done to help all people living with this disease,” the CFF stated in a news release.