CF Trust Award to Support Clinical Study of Possible Respiratory Therapy, RPL554

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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CARSTAR CFF recognition

Verona Pharma recently announced that it has received its second Venture and Innovation Award from the U.K. Cystic Fibrosis Trust. The award will help fund a Phase 2 clinical trial to investigate RPL554 as a possible treatment of cystic fibrosis (CF).

The trial, which expects to start recruiting CF patients during the first half of 2017, will primarily evaluate the pharmacokinetic and pharmacodynamics profiles of RPL554, as well as its tolerability and safety.

RPL554 is a first-in-class, inhaled dual inhibitor of the enzymes phosphodiesterase 3 and 4 (PDE3 and PDE4), and has been seen to act as an effective bronchodilator and to have anti-inflammatory properties.

“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 for the treatment of cystic fibrosis,” Jan-Anders Karlsson, chief executive officer of Verona Pharma, said in a press release. The award’s total was not announced.

RPL554 has successfully completed eight Phase 1 and 2a clinical studies, involving 229 people, most recently in patients with chronic obstructive pulmonary disease (COPD). Verona researchers reported that the drug candidate provides clinically meaningful and statistically significant improvements in lung function, as both a single agent and as an add-on therapy with bronchodilators.

In previous studies, RPL554 was also observed to shorten the time of onset of bronchodilation when administered as an add-on therapy, and to have anti-inflammatory effects. The treatment was well-tolerated by patients.

RPL554 is of interest to CF because preclinical data has suggested it is a stimulator of CFTR (cystic fibrosis transmembrane conductance regulator), a protein that fails to work as needed in CF patients. Verona believes RPL554 may improve mucociliary clearance (reduce phlegm in the airways) to ease symptoms of chronic inflammation and improve breathing.

The company’s first Venture and Innovation Award was granted in November 2014 and used to partly fund those exploratory studies of RPL554 in CF. The preclinical results were published in the American Journal of Physiology, in the 2015 study, “The dual phosphodiesterase 3 and 4 inhibitor RPL554 stimulates CFTR and ciliary beating in primary cultures of bronchial epithelia.”

Updated preclinical data of RPL554 in CF was also presented at this year’s Annual North American Cystic Fibrosis Conference (NACFC), which took place in Orlando, Florida.

“A vital part of our fight for a Life Unlimited is to accelerate the development of potential new therapies for people with cystic fibrosis,” said  Ed Owen, outgoing chief executive of the CF Trust. “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.”