Enterprise Therapeutics Reveals Preclinical Data on ETD001, Novel Inhaled ENaC Blocker

Enterprise Therapeutics Ltd, a biopharmaceutical company focusing on respiratory diseases, has announced the publication of a significant study in the Journal of Cystic Fibrosis. The study highlights that low doses of ETD001, an ENaC blocker and Enterprise's lead asset, show a remarkably long duration of action in a sheep model of airway mucus clearance.

ETD001, tested at doses that were well tolerated in healthy volunteers, holds promise for benefiting individuals with cystic fibrosis (pwCF). The study paves the way for a Phase 2 clinical study of ETD001 in pwCF, scheduled to begin in summer 2024. This trial aims to determine whether a 28-day treatment regimen can improve lung function in these patients.

The novel approach of blocking ENaC in the airways aims to enhance mucus clearance in pwCF, notably for the more than 10% of patients who either cannot tolerate CFTR modulators or are genetically unsuited to them. Previous clinical trials for other ENaC blockers, such as VX-371, AZD5634, BI 1265162, and QBW276, did not show benefits, potentially because the doses used were too low to achieve a prolonged action on mucus clearance. This study with ETD001 provides a possible explanation for these past failures and suggests a new dosing strategy.

Dr. Henry Danahay, Head of Biology at Enterprise Therapeutics and the lead author of the paper, emphasized the significance of the findings. He noted that the data demonstrate ETD001's superiority compared to other inhaled ENaC blockers. Coupled with the Phase 1 trial results where ETD001 was well-tolerated, these findings bolster confidence as they move into Phase 2 trials. Dr. Danahay reaffirmed the company's commitment to discovering new therapies that could significantly improve the lives of those with cystic fibrosis.

The promising results from the sheep model study and the upcoming Phase 2 clinical trial could mark a significant advancement in treating cystic fibrosis, particularly for patients who have limited options due to intolerance or genetic incompatibility with existing CFTR modulators.