Enterprise Therapeutics announced that a Phase 2 trial of ETD001, an inhaled epithelial sodium channel blocker, met its primary endpoint, demonstrating a statistically significant and clinically relevant improvement in lung function versus placebo after 28 days. According to the company, patients who received ETD001 had a 3.4 percentage point improvement in percent predicted FEV1 compared to patients who received placebo. Enterprise says that it next intends to perform a longer-term Phase 2a trial of ETD001 plus CFTR modulators.
The Phase 2 study enrolled 57 CF patients who were not receiving treatment with CFTR modulators. The first arm of the study, which was designed to enroll 8 subjects, evaluated twice-daily 4.5 mg doses of ETD001 delivered via nebulization versus placebo over 7 days. The second arm was a crossover with the same dosage level over 28 days and a 28-day washout between the treatment periods.
Enterprise Therapeutics Chief Medical Officer Renu Gupta commented, “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. 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.”
Chief Scientific Officer Martin Gosling said, “The data from this trial represents a critical milestone as it is the first time an ENaC blocker has demonstrated efficacy in pwCF. We are excited by the potential that these data offer for enhancing treatment options for all pwCF, and to expand into other muco-obstructive lung diseases including bronchiectasis. To observe lung function improvement within 28 days of treatment with ETD001 is a significant milestone and we are now looking forward to progressing into longer duration clinical trials, where we expect to see further clinical benefit.”
Read the Enterprise Therapeutics press release
