Promising New Inhaled Therapy Shows Safety in Early Trials for Cystic Fibrosis
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A novel inhaled therapy, ETD001, has demonstrated promising safety and tolerability in a Phase 1 clinical trial, offering potential hope for individuals with cystic fibrosis (CF), particularly those who do not respond to existing treatments. The findings, published in The Journal of Cystic Fibrosis, signal a significant step forward in the development of new treatments for this debilitating genetic disease.
Addressing an Urgent Need in cystic Fibrosis Treatment
Cystic fibrosis affects over 100,000 people globally, with an average life expectancy of approximately 60 years. The disease is characterized by the buildup of thick mucus in the lungs, leading to chronic infections and progressive lung damage.While recent advances in CFTR modulators have revolutionized treatment for many, a substantial portion of the CF population does not benefit from these therapies due to their genetic makeup. This unmet need underscores the importance of exploring alternative treatment strategies.
ETD001: A Novel Approach to mucus Clearance
ETD001 is a long-acting inhaled epithelial sodium channel (ENaC) blocker developed by Enterprise Therapeutics Ltd. The therapy aims to restore airway mucus hydration by inhibiting enac, a key regulator of salt and water transport in the lungs. This process is expected to improve mucociliary clearance – the natural mechanism by which the lungs remove mucus and debris – and ultimately enhance lung function.
The Phase 1 trial evaluated the safety, tolerability, and how the body processes ETD001 in healthy volunteers. Participants received single and multiple inhaled doses of the therapy,at levels predicted to be effective in humans. According to a company release, ETD001 was well-tolerated at all doses tested, even at levels higher than those anticipated to deliver therapeutic benefits.
Key Findings from the Phase 1 Trial
Unlike previous inhaled ENaC blockers, ETD001 exhibited a unique pharmacokinetic profile. The trial results indicated slow absorption of the drug from the lung into the bloodstream, suggesting prolonged retention within the lungs and the potential for extended duration of action. This is a critical advantage, as it could reduce the frequency of dosing required for effective treatment.
A potential concern with ENaC blockade is the possibility of altering blood potassium levels due to target-mediated effects in the kidney. However, the Phase 1 trial demonstrated that ETD001 did not significantly impact potassium levels at any dose evaluated. These findings align with pre-clinical data, which also showed a favorable safety profile and extended duration of action – exceeding 16 hours after a single inhaled dose.
Looking Ahead: Phase 2 Trial Underway
Enterprise Therapeutics is currently conducting a Phase 2 trial (NCT06478706) to assess the efficacy of ETD001 in individuals with cystic fibrosis. The trial,involving 28 days of treatment,will investigate whether the therapy improves lung function. Headline data from the Phase 2 trial are anticipated in early 2026.
“Ther is an urgent need for new therapies to treat mucus obstruction in the lungs of people with CF, and especially those who are genetically unsuited to CFTR modulators,” stated Dr. Henry Danahay, Lead Author of the Paper and Head of Biology at Enterprise Therapeutics. “We are passionate about working towards treatments that will benefit all people with cystic fibrosis and are excited to publish these promising results from our phase 1 trial.”
the development of ETD001 represents a significant advancement in the pursuit of more effective therapies for cystic fibrosis, offering renewed hope for patients and their families.
