Enterprise Therapeutics, a biopharma company specialising in respiratory disease, has dosed the first patient with cystic fibrosis (pwCF) in its Phase IIa trial of ETD001.
Targeting the lung's ENaCs to reduce cystic fibrosis symptoms
ETD001 is a lung-specific epithelial sodium channel (ENaC) inhibitor which can assist in improving hydration in the lungs of patients with cystic fibrosis, as well as allowing for better mucus clearance.
The phase IIa trial aims to deliver clinical proof-of-concept, while also assessing the safety profile of ETD001 in the 10% of patients with cystic fibrosis with a high clinical unmet need.
The study is running at multiple sites across Europe, including the UK, Germany, France and Italy — where it will look into how lung function is impacted by ETD001 administration in patients who are ineligible or are not receiving CFTR modulators.
Unmet medical needs in the cystic fibrosis patient population
Cystic fibrosis affects more than 100,000 globally, with an average life expectancy of 50 years for those that suffer with the condition.
Failed mucociliary clearance and mucus congestion in the lungs of pwCF leads to cycles of infection and inflammation, which leads to the overall decline in lung function and health.
Enhancing the fluid volume in the lungs by inhibiting the ENaC channels present in the region can hydrate mucus, improve clearance, reduce mucus congestion and drive improvements in lung function.
ETD001 has exhibited a strong safety profile in health participants and has been demonstrated to be long-acting in pre-clinical studies.
Head of Development, Enterprise Therapeutics, Paul Russell, commented: “By targeting the underlying mechanisms of mucus congestion in the lungs through ENaC inhibition, ETD001 has the potential to be a transformative respiratory therapeutic. This is not only for the ~10% of pwCF who are not genetically suited to, or do not benefit from CFTR modulators, but also those suffering from other muco-obstructive lung diseases such as COPD and asthma. The commencement of our Phase IIa trial brings us an exciting step closer to realising that potential.”
