Verona jumps as midphase cystic fibrosis data dial up hopes

The trial linked a single dose of RPL554 to a sustained 6% improvement in FEV1 over baseline. (AstraZeneca)

Shares in little Verona Pharma have jumped 20% on the back of data from a phase 2a cystic fibrosis trial. The limited look at data from a small, short trial contains tidbits of encouragement for Verona but ultimately paints an inconclusive picture of the prospects of PDE3/PDE4 inhibitor RPL554.

Investigators enrolled 10 cystic fibrosis patients in the crossover trial and gave each patient one of two doses of RPL554 and placebo in a random sequence. Verona ran the trial to assess the pharmacokinetics of RPL554, which was in line with previous studies of the drug in COPD. But it is the effect on the FEV1 measure of lung function that has dialed up expectations.

The trial linked a single dose of RPL554 to a sustained 6% improvement in FEV1 over baseline in the eight hours following treatment. That suggests the mix of bronchodilation and anti-inflammatory effects provided by nebulized RPL554 may improve outcomes in patients with cystic fibrosis.


Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

Verona is a long way from showing that conclusively, though. The size and duration of the trial mean limited conclusions can be drawn from the data. And the lack of details about baseline characteristics and the performance of the placebo cohort in Verona’s release means the public only has a partial view of the limited data generated in the study.

RELATED: Verona secures cash for mid-phase cystic fibrosis trial

A larger study is needed to address doubts about the prospects of RPL554 in cystic fibrosis. But while Verona is upbeat about the phase 2a, it is hanging fire on advancing in cystic fibrosis until it gets a look at data from an ongoing COPD trial.

“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 prioritize the future therapeutic focus of RPL554 development,” Verona CEO Jan-Anders Karlsson, Ph.D., said in a statement.

Suggested Articles

By employing heart rate signals, physical activity and sleep quality, common Fitbit trackers may be able to predict the spread of the flu.

Nanox has raised $26 million to help fuel the development and commercialization of its "Star Trek"-inspired digital X-ray bed.

Oncology is clearly a major medical and societal issue, but one that sees too much focus from biopharmas at the expense of other killers.