London, UK-based Verona Pharma’s experimental phosphodiesterase therapy has hit targets in a mid-stage trial in patients with cystic fibrosis.
Top-line data from a Phase IIa trial with nebulised RPL554 show that the investigational therapy has a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile, the firm said.
Single doses of the drug achieved statistically significant increases in average forced expiratory volume in one second (FEV1) - a key measure of lung function - in patients with the condition, paving the way for its continued development.
“Achieving a six percent sustained improvement from baseline in FEV1 after a single dose is very impressive in this group of CF patients,” said to the trial’s lead investigator, Professor Andres Floto, Wellcome Trust senior investigator, and research director of the Department of Medicine, University of Cambridge and Papworth Hospital, UK.
RPL554 is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 designed to have anti-inflammatory as well as bronchodilator properties.
Based on available data, the drug, which is also being developed for chronic obstructive pulmonary disorder (COPD), has the potential to reduce phlegm in the airways and airway obstruction, as well as inhibit inflammation, Verona noted.
“While we believe these results support further development of the drug in CF, we await the results of our ongoing Phase 2b trial for the maintenance treatment of COPD, for which we anticipate reporting top-line data early in the second quarter of 2018, to prioritise the future therapeutic focus of RPL554 development,” said Jan-Anders Karlsson, PhD, Verona’s chief executive.