Enterprise Therapeutics Begins Phase 2 Trial of ETD001 for Cystic Fibrosis

BRIGHTON, UK I July 23, 2024 I Enterprise Therapeutics Ltd, a biopharmaceutical company focused on creating new therapies for respiratory diseases, has begun dosing the first patient with cystic fibrosis (CF) in its Phase 2a trial of ETD001. This marks a significant step forward in developing treatments aimed at improving the lives of those with respiratory conditions.

ETD001 is a promising new compound designed to target the epithelial sodium channel (ENaC) in the airway epithelium. By inhibiting this channel, ETD001 aims to increase mucus hydration and clearance in the lungs. The primary goal of the Phase 2a trial is to validate the clinical feasibility of this approach and evaluate the safety of ETD001 in the 10% of CF patients with the greatest unmet medical need. This trial, conducted across multiple sites in the UK, Germany, France, and Italy, will focus on assessing lung function (FEV1) in CF patients who are either ineligible for or not currently receiving CFTR modulators.

Cystic fibrosis affects over 100,000 individuals worldwide, with an average life expectancy of just 50 years. The disease is characterized by poor mucociliary clearance and mucus buildup in the lungs, leading to recurrent infections and inflammation, which progressively damage lung function. ETD001 aims to counteract these issues by increasing lung fluid volume through ENaC inhibition, thereby hydrating mucus, improving its clearance, and reducing congestion. Previous trials have shown ETD001 to be safe in healthy participants and indicated that it has long-lasting effects in pre-clinical studies.

Dr. John Ford, CEO of Enterprise Therapeutics, expressed his excitement about this milestone, emphasizing the company’s commitment to advancing ETD001 as a novel treatment for those with severe unmet medical needs in the CF community. He noted that the compound has already shown a favorable safety profile and prolonged lung residency in earlier studies, and he looks forward to further progress in the Phase 2 trials and beyond.

Paul Russell, Head of Development at Enterprise Therapeutics, highlighted the transformative potential of ETD001. By addressing the root causes of mucus congestion in the lungs through ENaC inhibition, ETD001 could become a groundbreaking treatment not only for the approximately 10% of CF patients who do not benefit from CFTR modulators but also for individuals with other muco-obstructive lung diseases such as COPD and asthma. Russell emphasized that the initiation of the Phase 2a trial is a crucial step towards realizing this potential.

Dr. Renu Gupta, Chief Medical Officer of Enterprise Therapeutics, thanked the patients participating in the Phase 2 study and the clinical investigators for their efforts in achieving this important milestone. She expressed hope that the company’s dedicated work on this innovative ENaC-targeting molecule, in collaboration with the CF community, will result in a treatment that significantly enhances the quality of life for people living with cystic fibrosis.