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Enterprise Therapeutics' ETD001 Shows Promise in Cystic Fibrosis Phase 2 Trial

• Enterprise Therapeutics' ETD001, an inhaled ENaC blocker, is currently in Phase 2 clinical trials to assess its impact on lung function in cystic fibrosis patients. • ETD001 has received 'rare pediatric disease designation' from the FDA, highlighting its potential to address unmet needs in CF treatment. • Preclinical studies demonstrate ETD001's potent effect and low permeability through the airway epithelium, making it an ideal inhaled therapy. • The Phase 2 study is expected to be completed in 2025, with results anticipated to demonstrate the drug's efficacy and safety.

Enterprise Therapeutics is advancing ETD001, a novel inhaled ENaC blocker, through Phase 2 clinical trials, offering new hope for improving cystic fibrosis (CF) treatment. The study, initiated in July 2024, aims to evaluate the impact of 28 days of ETD001 treatment on lung function in individuals with CF.
The development of ETD001 is detailed in a peer-reviewed study published in the European Journal of Medicinal Chemistry, highlighting the medicinal chemistry process that led to its creation. The research, conducted in collaboration with Evotec, emphasizes the optimization of the compound to achieve ETD001's unique properties.

ETD001: A Potential Game-Changer for CF Treatment

ETD001 has demonstrated a potent effect in preclinical models, coupled with low permeability through the airway epithelium. This profile positions it as an ideal inhaled therapy for CF patients. The FDA has granted ETD001 'rare pediatric disease designation' in 2024, underscoring its potential to address unmet needs in this patient population.
Dr. Steve Collingwood, head of chemistry at Enterprise Therapeutics and lead author of the study, stated, "We are proud of our inhaled medicinal chemistry strategy, the success of which is evidenced by the superior profile of ETD001 compared to other inhaled ENaC blockers..."

Mechanism of Action and Clinical Potential

By inhibiting the epithelial sodium channel (ENaC), ETD001 helps hydrate mucus, improve clearance, and reduce congestion in the lungs, potentially leading to improved lung function. Cystic fibrosis affects over 100,000 people globally, and ETD001 could represent a significant advancement in CF therapy, particularly for those unable to benefit from CFTR modulators.
Prof. Martin Gosling, Enterprise’s Chief Scientific Officer, emphasized the broader potential of ETD001, stating, "We firmly believe that the unique properties of ETD001, giving rise to class-leading lung retention, will deliver an effective therapy to people with CF currently unable to benefit from CFTR modulators."

Ongoing Phase 2 Trial

The Phase 2 clinical trial is currently recruiting participants and is expected to be completed in 2025. The study will assess the effect of ETD001 on lung function over a 28-day treatment period. ETD001 has already demonstrated a favorable safety profile in Phase 1 trials and shown long-acting potential in preclinical studies.
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Reference News

[1]
Breakthrough trial offers new hope for cystic fibrosis patients - Drug Target Review
drugtargetreview.com · Nov 28, 2024

Enterprise Therapeutics' ETD001, an inhaled ENaC blocker, shows promise in Phase 2 trials for cystic fibrosis treatment,...

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