• ETD002 is a novel TMEM16A chloride channel potentiator
• Therapy applicable to all cystic fibrosis patients, independent of genotype
Brighton, UK, 17 August 2020: Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, today announced it has successfully dosed the first subjects in a Phase 1 trial for its novel inhaled cystic fibrosis therapy, ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).
CF is estimated to affect 75,000 people globally. One of the main causes of difficulty in breathing and increased risk of infection in CF is mucus congestion in the lungs. Enterprise’s compound ETD002 targets the underlying mechanisms of mucus congestion, and is expected to restore lung function, reduce the frequency of lung infections and improve patient quality of life. CF is caused by loss of function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), an anion channel highly expressed by the airway epithelium. Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it is not currently available or effective for all people with CF.
In pre-clinical models, Enterprise has demonstrated that ETD002 enhances the activity of TMEM16A, an alternative anion channel present in airway epithelial cells, and by doing so increases anion and fluid flow into the airways, thinning the mucus and increasing its clearance. As TMEM16A potentiation is independent of the mutational status of CFTR, this makes the approach applicable to all people with CF, and potentially patients with non-CF lung disease. Additionally, ETD002 is expected to deliver benefit as a monotherapy and in combination with other therapies, including CFTR repair.
Dr John Ford, CEO, Enterprise Therapeutics, said: “Considerable efforts to identify and develop this innovative compound have resulted in a TMEM16A potentiator that has the potential to significantly increase the quality of life for people living with CF, for many of whom existing therapies are not effective. We are excited to have begun the clinical stage of development for ETD002.”
Dr David Morris, CMO, Enterprise Therapeutics, said: “Although CFTR modulators have successfully demonstrated improved clinical outcomes in those genetically suited to these therapies, we are hopeful that TMEM16A potentiation via ETD002 will provide clinical benefit to the many people with CF who do not share these CFTR mutations. We look forward to generating our first data in human volunteers over the next few months and are grateful to the subjects and investigators who are helping us to advance this novel treatment for individuals with CF.”
This work is in part funded by a Therapeutics Development Award from the Cystic Fibrosis Foundation to Enterprise Therapeutics.
Notes for Editors
Dr John Ford
CEO, Enterprise Therapeutics
Dr David Morris
CMO, Enterprise Therapeutics
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About Enterprise Therapeutics www.enterprisetherapeutics.com
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 quality of life.
The Company’s novel muco-regulatory therapies target ion channels TMEM16A and ENaC to increase the hydration and clearance of mucus. 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. In April 2018 the Company closed an oversubscribed Series B funding round co-led by Versant Ventures and Novartis Venture Fund, with Forbion Capital Partners, Epidarex Capital and IP Group. In October 2019 the company received funding from the Cystic Fibrosis Foundation to advance TMEM16A through to clinical proof of concept in CF.
Cystic Fibrosis is the most common lethal genetic disease of Caucasians with more than 75,000 patients worldwide living with the disease. The average life expectancy of a CF patient, although improving, is approximately 40 years.