BRIEF—CF Foundation funds expansion of program for potential nonsense mutations therapy

29 March 2022

The Cystic Fibrosis Foundation announced an award for up to $15.9 million in additional funding to USA-based Eloxx Pharmaceuticals to expand clinical studies of ELX-02, a potential therapy for people with CF who have nonsense mutations, with the news lifting the firm’s shares by 8.5% to $0.52 by mid-day trading.

The Foundation previously provided up to  $3.6 million in funding for Eloxx’s initial ELX-02 clinical studies.

The new funding will enable the company to expand the studies to determine whether ELX-02 with a cystic fibrosis transmembrane conductance regulator modulator results in increased biological activity over ELX-02 alone.

The award, which provides financial and scientific support to Eloxx, is part of the CF Foundation's $500 million Path to a Cure initiative to accelerate treatments for the underlying cause of CF and develop a cure.

Current therapies have significantly improved the lives of many people living with CF. However, there are still people - including those with nonsense mutations - who do not have an effective treatment for the underlying cause of their disease.