Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of those suffering from respiratory disease, today announced dosing of the first person with cystic fibrosis (pwCF) in its Phase 2a trial of ETD001. ETD001, a low molecular weight compound with first-in-class potential, targets the epithelial sodium channel (ENaC) in the airway epithelium to increase the hydration and clearance of mucus. The Phase 2a trial aims to deliver clinical proof-of-concept and to assess the safety profile of ETD001 in the 10% of pwCF with the highest unmet medical need.

The study will be performed at sites located in UK, Germany, France and Italy and will assess lung function (FEV1) in pwCF who are either ineligible for or are not receiving CFTR modulators. CF is estimated to affect over 100,000 people worldwide, with an average life expectancy of only 50 years. Failed mucociliary clearance and mucus congestion in the lungs of pwCF leads to cycles of infection and inflammation and an ongoing decline in lung function.

Increasing fluid volume in the lung by inhibiting ENaC with ETD001 will hydrate mucus, improve clearance, reduce mucus congestion, and is expected to drive substantial improvements in lung function. ETD001 has previously demonstrated a strong safety profile in healthy participants in a Phase 1 trial and has been shown to be long-acting in pre-clinical studies. Dr John Ford, CEO, Enterpris.