Enterprise Therapeutics publishes open access paper on novel approach to treat cystic fibrosis

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 the publication of its first peer-reviewed paper. The open access paper, published in the American Journal of Respiratory and Critical Care Medicine1, describes TMEM16A potentiation via ETX001 as a novel approach for the treatment of cystic fibrosis (CF). The research was conducted in collaboration with University of Sussex, University of North Carolina and University of Miami.

Dr. Henry Danahay, Head of Biology, Enterprise Therapeutics

The paper, entitled “TMEM16A Potentiation: A Novel Therapeutic Approach for the Treatment of Cystic Fibrosis”, demonstrates the ability of Enterprise’s proprietary compound, ETX001, to enhance the activity of TMEM16A in human bronchial epithelial cells from CF patients, increasing epithelial fluid secretion and mucus clearance providing the first pre-clinical proof of principle for this approach.

CF is estimated to affect 75,000 patients globally and is caused by loss of function mutations in the anion channel, cystic fibrosis transmembrane conductance regulator (CFTR). Increasing anion conductance via CFTR modulation is a clinically validated approach for treating CF, however it does not treat =10% of patients with a combination of nonsense and other rare mutations. In addition, many patients eligible for CFTR repair therapy do not benefit from these therapies. TMEM16A potentiation offers a non-CFTR mediated approach for the treatment of CF and can be delivered as a monotherapy or in combination with other therapies such as CFTR repair.

Dr. Henry Danahay, Head of Biology, Enterprise Therapeutics, and lead author of the paper, said:

We have successfully demonstrated the positive effects of ETX001 on both airway fluid secretion and mucus clearance in CF patients. Given the percentage of the population of CF patients who are not genetically matched to existing CFTR repair therapies, this paper builds a strong case for testing TMEM16A potentiation in the clinic.”

Authors on the paper include Henry L Danahay (Enterprise Therapeutics), Sarah Lilley (Sussex Drug Discovery Centre, University of Sussex), Roy Fox (Sussex Drug Discovery Centre, University of Sussex), Holly Charlton (Sussex Drug Discovery Centre, University of Sussex), Juan Sabater (Mount Sinai Medical Centre, University of Miami), Brian Button (Department of Biochemistry & Biophysics, UNC Chapel Hill, North Carolina), Clive McCarthy (Enterprise Therapeutics), Stephen P Collingwood (Enterprise Therapeutics), and Martin Gosling (Enterprise Therapeutics, and Sussex Drug Discovery Centre, University of Sussex).

This work was in part funded by a Therapeutics Development Award from the Cystic Fibrosis Foundation to Enterprise Therapeutics.