BRIGHTON, UK – ETD001, an investigational inhaled therapy for cystic fibrosis (CF), has demonstrated encouraging results in a small clinical trial, improving lung function more effectively than a placebo in patients who were not taking CFTR modulators. The findings were announced by Enterprise Therapeutics, the company developing the treatment.
The therapy is being developed specifically for the estimated 10% of people with CF who have limited or no treatment options because they cannot benefit from currently approved CFTR modulators.
A Different Approach to Treating CF
Cystic fibrosis is caused by mutations in the CFTR gene, which produces a protein responsible for regulating the movement of water and salt in and out of cells. When CFTR does not function properly, thick, sticky mucus accumulates in the lungs and other organs, leading to chronic respiratory and digestive complications.
Although CFTR modulators have transformed treatment for many patients, these therapies only work for individuals with specific genetic mutations. Some eligible patients are also unable to tolerate them because of side effects.
ETD001 takes a different approach. Rather than targeting the CFTR protein itself, the therapy blocks ENaC (epithelial sodium channel), a protein involved in sodium transport across cells. By inhibiting ENaC activity, ETD001 is designed to restore proper hydration in airway mucus, regardless of a patient’s underlying CF mutation.
According to Enterprise Therapeutics, this mechanism could make ETD001 suitable for all people living with CF.
Positive Results From Phase 2 Trial
The newly announced findings come from a Phase 2 clinical trial (NCT06478706) involving 57 people with CF who were not receiving CFTR modulators.
In the first stage of the study, participants received ETD001 for one week to evaluate safety. The company reported that the therapy was generally well tolerated, with a safety profile consistent with expectations for inhaled CF treatments.
The second phase compared ETD001 directly with a placebo using a crossover design. Participants received either twice-daily ETD001 or placebo treatment for 28 days, followed by a four-week washout period before switching to the opposite treatment.
Researchers evaluated lung function using forced expiratory volume in one second (FEV1), a standard measure of respiratory performance in CF. Results showed that ETD001 improved FEV1 by an average of 3.4% compared with placebo — a statistically significant improvement.
Enterprise Therapeutics also stated that the treatment remained safe and well tolerated throughout the study.
Company Leaders Highlight Potential Impact
“We are delighted to report positive results from this Phase 2 trial of ETD001,” said Renu Gupta, the company’s chief medical officer. “These data demonstrate that this novel therapy is well tolerated in people with CF and has shown improvement in lung function over a 28-day period compared to placebo.”
Gupta added that the company is particularly encouraged by the therapy’s potential to help patients who currently lack effective treatment options.
Martin Gosling noted that the study represents the first positive clinical results for an ENaC-targeting therapy in cystic fibrosis.
“To observe lung function improvement within 28 days of treatment with ETD001 is a significant milestone,” Gosling said. He added that the company is preparing for longer-term studies, where researchers hope to see even greater clinical benefits.
Next Steps for ETD001
Enterprise Therapeutics plans to present detailed data from the trial at the upcoming European Cystic Fibrosis Society Conference in Lisbon next month.
The company is also preparing additional Phase 2 studies to evaluate different dosing strategies and longer treatment durations. Future trials are expected to examine ETD001 both as a standalone therapy and in combination with existing CFTR modulators.
“Our focus now is on advancing ETD001 further and moving one step closer to improving outcomes for people living with respiratory disease,” said John Ford.
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