Enterprise Therapeutics publishes preclinical profile of ETD001, a novel inhaled ENaC blocker
June 12, 2024 – Biotechnology, Clinical Trials, Drug Delivery, Drug Discovery, Pharmaceutical – cystic fibrosis, drug development, drug discovery, drug profile, lungs, preclinical
Low doses of ETD001 demonstrate exceptionally long duration of action in sheep model of airway mucus clearance
Report in Journal of Cystic Fibrosis discloses best-in-class profile of ETD001
ETD001 scheduled to commence Phase 2 clinical study in people with cystic fibrosis in summer 2024
12 June 2024 – Brighton, UK – Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of people with respiratory diseases, today announced the publication of a peer reviewed study in the Journal of Cystic Fibrosis1. The paper describes low doses of the ENaC blocker ETD001, Enterprise’s lead asset, enhancing airway mucus clearance with an exceptionally long duration of action in a sheep model.
This study indicates that ETD001, at a dose level that was well tolerated in healthy volunteer studies, provides an opportunity to test whether a long-acting ENaC blocker can deliver benefit to people with cystic fibrosis (pwCF). ETD001 is scheduled to commence a Phase 2 clinical study in pwCF in summer 2024, to understand whether 28 days of treatment will improve lung function.
Blocking ENaC in the airways offers a novel approach to improve mucus clearance in pwCF, including in the more than 10% of individuals who are either intolerant of or genetically unsuited to CFTR modulators. Recently, several other ENaC blocking drugs (VX-371, AZD5634, BI 1265162, QBW276) failed to show any benefit in clinical trials. This study provides a potential explanation for these failures as data from the sheep model indicate that each may have been dosed in clinical trials at a dose too low to observe an extended duration of action on mucus clearance.
Dr Henry Danahay, head of biology, Enterprise Therapeutics, and lead author of the paper, said: “This data provides strong evidence that ETD001 has a superior profile compared to other inhaled ENaC blockers. Along with the results from the Phase 1 trials where ETD001 was well-tolerated, this study supports our high level of confidence going into the Phase 2 clinical trial. We remain passionate in our drive to discover novel therapies that have the potential to transform the lives of all people with cystic fibrosis.”
About Enterprise Therapeutics: Enterprise Therapeutics is discovering and developing new therapies that target the underlying mechanisms of mucus congestion in the lungs, one of the main causes of difficulty in breathing and increased risk of infection in respiratory diseases such as cystic fibrosis and COPD. Reducing mucus congestion will reduce the frequency of lung infections and improve patient quality of life.
The company’s approach is to increase the hydration and clearance of mucus. Enterprise’s lead asset, ETD001, is a novel inhaled blocker of the epithelial sodium channel (ENaC) with first in class potential. Enterprise has also identified novel targets and compounds that reduce mucus production, an approach that complements mucus hydration therapies. The Enterprise Therapeutics management team has significant expertise in drug discovery, drug development, respiratory biology and ion channel pharmacology. Visit: www.enterprisetherapeutics.com
About Cystic Fibrosis (CF): Cystic Fibrosis is a lethal genetic disease, with more than 100,000 patients worldwide living with the disease. The average life expectancy of a person with CF, although improving, is only ~50 years.
