RPL554 Emerging As Viable Cystic Fibrosis Therapy

Data supporting a promising, new treatment for Cystic Fibrosis (CF) developed by Verona Pharma will be presented for the first time at the 28th Annual North American Cystic Fibrosis Conference (NACFC) in Atlanta, Georgia on October 9-11, 2014.

Verona Pharma’s key molecule, RPL554, is currently being evaluated in a phase two clinical trial for the treatment of COPD (Chronic obstructive pulmonary disease), but the company also believes that the therapy can treat CF as well. The company’s findings will be summarized in a presentation entitled “CFTR activation by the dual phosphodiesterase ¾ inhibitor RPL554 and the MRP4 inhibitor MK571.”

Cystic fibrosis is caused by mutations in the Cftr gene that results in the defective transport of ions across the membrane of epithelial cells, particularly in the lungs. This leads to thick mucus and recurrent infections that culminate in difficulty in breathing, which can in turn escalate to life-threatening symptoms.

RPL554 can activate CFTR (short for cystic fibrosis transmembrane conductance regulator) in CF patients. Currently without a cure, the demand for new treatments for CF is high, and Verona Pharma will further evaluate their findings in pre-clinical model systems, working together with academic scientists.

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Professor John Hanrahan, Director, CF Translational Research centre at McGill University, commented, “In our experiments, RPL554 increased the activity of CFTR, ion channels on the surface of cells obtained from the lining of the airway. In cystic fibrosis patients it is the dysfunction of these ion channels, as a result of genetic mutations, that is responsible for the symptoms of the disease. We will continue to examine this effect of RPL554 in further studies. Ultimately, if found effective and safe in cystic fibrosis patients, RPL554 could emerge as a new medicine for this debilitating disease.”

Dr. Jan-Anders Karlsson, CEO of Verona Pharma, noted, “Due to its mechanism of action, we expected that RPL554 might have utility in other respiratory diseases in addition to COPD and asthma, such as bronchiectasis and CF. These results support that hypothesis at least for CF. We will now seek to build on these findings by testing the activity of RPL554 in patients with this orphan disease. We are currently focused on progressing RPL554 in phase 2 clinical trials for COPD, initially positioning it as a novel treatment for acute exacerbations of the disease. We are also building a broader franchise around this drug to maximize its value, both to patients and to investors. We are therefore exploring the potential of the drug in different diseases as well as in the multi-blockbuster markets for COPD and asthma maintenance therapy. The results outlined in this NACFC presentation suggest another tangible opportunity for us to explore.”