Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of people with respiratory diseases, today announced the publication of a peer reviewed study in the Journal of Cystic Fibrosis 1 . The paper describes low doses of the ENaC blocker ETD001, Enterprise’s lead asset, enhancing airway mucus clearance with an exceptionally long duration of action in a sheep model. This study indicates that ETD001, at a dose level that was well tolerated in healthy volunteer studies, provides an opportunity to test whether a long-acting ENaC blocker can deliver benefit to people with cystic fibrosis (pwCF).
ETD001 is scheduled to commence a Phase 2 clinical study in pwCF in summer 2024, to understand whether 28 days of treatment will improve lung function. Blocking ENaC in the airways offers a novel approach to improve mucus clearance in pwCF, including in the more than 10% of individuals who are either intolerant of or genetically unsuited to CFTR modulators. Recently, several other ENaC blocking drugs (VX-371, AZD5634, BI 1265162, QBW276) failed to show any benefit in clinical trials.
This study provides a potential explanation for these failures as data from the sheep model indicate that each may have been dosed in clinical trials at a dose too low to observe an extended duration of action on mucus clearance. This data provides strong evidence that ETD001 has a superior profile compared to ot.
