Verona Pharma’s nebulized RPL554 can significantly improve lung function in patients with cystic fibrosis, according to top-line data from a Phase 2a clinical trial.
The latest results revealed that single administration of both high and low doses of the investigative drug significantly increased patients’ average forced expiratory volume in one second, or FEV1, a measure of lung function. This significant positive effect was sustained for at least eight hours.
In addition, RPL554 displayed favorable stability and distribution profiles, and was well tolerated by the patients. Its safety profile was similar to that previously reported in other clinical trials, with no additional concerns being reported.
“Achieving a six percent sustained improvement from baseline in FEV1 after a single dose is very impressive in this group of CF patients,” Andres Floto, Wellcome Trust senior investigator and professor at the University of Cambridge, and lead investigator of the trial, said in a press release.
The positive results demonstrating the efficacy of Verona Pharma’s lead drug candidate further support its development as a treatment for CF, researchers said.
RPL554 is an inhaled dual inhibitor of two enzymes, phosphodiesterase 3 and 4, which Verona designed to act as an anti-inflammatory as well as bronchodilator. It is under development for the treatment of chronic obstructive pulmonary disease (COPD) and CF.
This Phase 2a trial (NCT02919995) was planned to evaluate the tolerability, as well as stability and response, of RPL554 when administrated to CF patients. Ten patients were enrolled in the study, which was conducted at Papworth Hospital in the United Kingdom.
Participants were randomized to receive a placebo or RPL554, at either 1.5 mg or 6.0 mg single doses, administrated with a nebulizer.
“We’re pleased to see these interesting results come through, and that our investment in this area continues to show promise. We look forward to seeing the next steps of developing RPL554 for the treatment of cystic fibrosis,” said Janet Allen, director of strategy and innovation at the Cystic Fibrosis Trust.
The trial was supported by the trust in the form of Verona Pharma being awarded the second Venture and Innovation Award in October 2016.
“While we believe these results support further development of the drug in CF, we await the results of our ongoing Phase 2b trial [2016-005205-40] for the maintenance treatment of COPD, for which we anticipate reporting top-line data early in the second quarter of 2018, to prioritize the future therapeutic focus of RPL554 development,” said Jan-Anders Karlsson, the CEO of Verona Pharma.