Enterprise Therapeutics has reported that its phase 2 trial of ETD001, an investigational inhaled epithelial sodium channel blocker, met its primary efficacy outcome in people with cystic fibrosis who do not benefit from CFTR modulators.
The study showed clinically meaningful and statistically significant improvements in lung function over 28 days compared with placebo.
The trial evaluated efficacy, safety, tolerability and pharmacokinetics in the 10% of people with cystic fibrosis with the greatest unmet medical need. According to the company, data demonstrated a 3.4% point difference in ppFEV1 between ETD001 and placebo, with a p value of 0.0053. Exploratory analysis indicated that participants receiving ETD001 were three times more likely to show ppFEV1 improvement.
ETD001 was overall well tolerated, with adverse events consistent with expectations for people with cystic fibrosis receiving inhaled therapies.
The study was conducted across sites in the UK, Germany, France and Italy. Part A assessed safety and tolerability over 7 days, while part B used a double-blind, placebo-controlled cross-over design to measure the effect of twice daily dosing over 28 days.
Dr Renu Gupta, CMO at Enterprise Therapeutics, said: “We are delighted to report positive results from this Phase 2 trial of ETD001. These data demonstrate that this novel, inhaled ENaC blocker is well tolerated in pwCF, and has shown improvement in lung function over a 28 day period compared to placebo.”
Dr Martin Gosling, CSO, explained: “The data from this trial represents a critical milestone as it is the first time an ENaC blocker has demonstrated efficacy in pwCF.”
Dr John Ford, CEO, added: “I would like to thank everyone involved in developing the ETD001 programme to this stage, it’s really heartening to see the results from this Phase 2 trial.”
Enterprise now plans longer duration phase 2b studies and will also explore ETD001 in non-CF bronchiectasis.
