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Enterprise Publish Novel Therapeutic Approach for Cystic Fibrosis Patients

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Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company aimed towards the discovery and development of novel therapies to improve the lives of patients suffering with respiratory disease, has announced the publication of its first peer-reviewed paper.

The open access paper, published in the American Journal of Respiratory and Critical Care Medicine, 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.

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.”