Funding for new trials after promising results

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Trust announces second award to support Phase 2a clinical study of inhaled treatment as a therapy for cystic fibrosis.

A collaboration between the Cystic Fibrosis Trust and Verona Pharma exploring the impact of RPL554, a drug product candidate for the treatment of Chronic Obstructive Pulmonary Disease (COPD), for people with cystic fibrosis, will move to the next phase of clinical development after further pre-clinical data, which will be presented at North American Cystic Fibrosis Conference today, suggested the drug could activate CFTR.

Results from previously published studies in COPD patients suggested that the nebulised treatment is an effective bronchodilator – which makes breathing easier by relaxing the lung muscles and widening the airways – and pre-clinical data has suggested that RPL554 may also stimulate CFTR, the channel that does not function properly in cells in people with cystic fibrosis.

In November 2014 the Trust issued a Venture and Innovation Award to fund pre-clinical trials of RPL554 in patients with CF, and this second award will support a Phase 2a clinical study, expected to begin recruiting patients with CF in the first half of 2017. 

Find out more about how clinical trials work.

Jan-Anders Karlsson, Chief Executive Officer of Verona Pharma, said: “We are delighted to have received a second Venture and Innovation Award from the Cystic Fibrosis Trust, which will enable us to accelerate the clinical development of RPL554 in cystic fibrosis, an orphan disease where there is very high unmet medical need. The award recognises RPL554’s potential as a novel and important new treatment for this debilitating condition and we look forward to progressing the drug through clinical development.” 

Ed Owen, outgoing Chief Executive of the Cystic Fibrosis Trust, said: “We are delighted to continue to support the development of this compound and explore its potential to be an effective and complementary new treatment for cystic fibrosis, which remains an area of significant unmet medical need. 

"A vital part of our fight for a Life Unlimited is to accelerate the development of potential new therapies for people with cystic fibrosis. Building innovative collaborations with companies like Verona ensures that promising drug candidates are developed for use in cystic fibrosis and are given the best chance to get to those who need them as quickly as possible."

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