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Real progress looming with RPL554

First person with cystic fibrosis (CF) joins new trial for potential lung treatment.

The first person with cystic fibrosis has been enrolled onto a trial of a new drug called RPL554, which is hoped will treat the symptoms of CF and other lung conditions. The trial is being funded by one of the Cystic Fibrosis Trust’s Venture and Innovation Awards (VIAs), awarded in October 2016. The Trust put £100,000 of funding towards the trial through this VIA, while £200,000 was contributed by Verona Pharma, a biopharmaceutical company focused on developing treatments for respiratory diseases. Verona Pharma received its first VIA in 2014, which helped to fund an exploratory pre-clinical study of RPL554 for the treatment of cystic fibrosis.

What is RPL554?

RPL554 is a new and unique inhaled drug that is currently in development for the treatment of CF and chronic obstructive pulmonary disease (COPD). The drug has already been through Phase 1 testing and is now in a Phase 2a trial. The aim of this trial is to analyse the effect that the drug has on lung function and the inflammation of the lungs, how it moves through the body and how it is tolerated, by trialling it on approximately 10 people with cystic fibrosis.

The RPL554 trial is being performed at the Cambridge Centre for Medical Research at Papworth Hospital in Cambridge, one of the largest specialist cardiothoracic hospitals in Europe, by Lead Investigator Professor Andres Floto. Professor Floto is also leading the Trust’s Mycobacterium abscessus Strategic Research Centre (SRC), which is investigating the infection.

Initial trials showed that RPL554 led to significant improvements in lung function when used on its own. The drug also improved lung function when it was used alongside two common bronchodilators (medications that make breathing easier by relaxing and widening the muscles in the lungs).

How could RPL554 help to treat cystic fibrosis?

Pre-clinical data suggests that RPL554 may help to reduce sticky mucus in the airways, minimising the symptoms of chronic inflammation and making breathing easier for people with cystic fibrosis.

Jan-Anders Karlsson, PhD, Chief Executive of Verona Pharma, said: “Recently approved therapies are only available for a small number of people with CF and there is no effective anti-inflammatory medication approved to treat the underlying inflammation caused by cystic fibrosis. Due to the unique effects of RPL554, we believe that the drug has the potential to be a novel and important treatment for this debilitating condition.”

Paula Sommer, Head of Research at the Trust, said: “The VIAs are designed to ensure that the Trust’s funds go as far as possible to support ground-breaking treatments and innovative research by bringing in vital external funding. The RPL554 trial has the potential to make an incredible difference to the lives of people with CF, and we are thrilled that the drug is moving into this important trial stage.”

To help ensure the Trust can continue to fund research like this and create a brighter future for everyone with cystic fibrosis, why not sign up to make a regular donation?