Enterprise Therapeutics (Enterprise) has announced that its Phase II clinical trial of ETD001, an investigational inhaled epithelial sodium channel (ENaC) blocker in people with cystic fibrosis (pwCF), has met its primary efficacy outcome.
The study is designed to assess the efficacy, pharmacokinetics, safety and tolerability of inhaled ETD001.
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It evaluated the therapy in the 10% of pwCF with the highest unmet need, who do not derive benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators, and showed improved lung function over 28 days compared to placebo.
ETD001 is designed to enhance lung function by supporting mucus clearance and reducing airway obstruction.
The trial data indicated a 3.4% point difference in per cent predicted forced expiratory volume in one second (ppFEV1) among individuals treated with ETD001 against placebo.
Exploratory analyses revealed that participants using ETD001 had a three times greater likelihood of ppFEV1 improvement.
The trial found ETD001 to be generally well tolerated, with adverse events consistent with the expected profile for pwCF using inhaled therapies.
It was conducted at sites in France, Germany, Italy, and the UK in pwCF who are ineligible for, or not receiving, CFTR modulators.
Part A of the trial assessed the safety and tolerability of repeat doses over seven days.
Part B used a double-blind, placebo-controlled, cross-over design, with participants randomised to treatment with either ETD001 or placebo, on a repeat twice-daily 4.5mg dosing schedule for 28 days, separated by a 28-day washout.
Enterprise is planning to move forward to longer-duration Phase IIb dose-ranging trials in pwCF and will evaluate ETD001’s efficacy when combined with CFTR modulators.
Based on the evidence that people with non-CF bronchiectasis have similar mucus burden, Enterprise also aims to begin trials in this group.
Enterprise Therapeutics chief medical officer Dr Renu Gupta said: “We are encouraged by the potential of ETD001 to be a novel therapeutic option for improving the lives of all pwCF, particularly those currently without effective therapies, and we are grateful to everyone who took part in this trial.”
