Enterprise Therapeutics publishes preclinical profile of ETD001, a novel inhaled ENaC blocker

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 Fibrosis1. 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 other inhaled ENaC blockers. Along with the results from the Phase 1 trials where ETD001 was well-tolerated, this study supports our high level of confidence going into the Phase 2 clinical trial. We remain passionate in our drive to discover novel therapies that have the potential to transform the lives of all people with cystic fibrosis.”

Dr Henry Danahay, Head of Biology, Enterprise Therapeutics, and lead author of the paper


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
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