Verona jumps as midphase cystic fibrosis data dial up hopes

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.

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.

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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.