- Verona Pharma plc reported on Friday positive results from a Phase 2a study testing a formulation of its main pipeline asset in cystic fibrosis patients.
- Nebulized RPL554 showed a "favorable" pharmacokinetic and pharmacodynamic profile in the study, which evaluated 1.5 mg and 6 mg doses of the drug versus placebo. Across both doses, patients demonstrated a significant improvement in average forced expiratory volume over one second (FEV1), a measure of lung function and health.
- RPL554 inhibits two enzymes, phosphodiesterase 3 and 4, that can promote diseases like cystic fibrosis and chronic obtrusive pulmonary disorder (COPD). Verona is also investigating its drug in a Phase 2b trial of COPD patients, and will wait for results from that trial before deciding the next development steps for RPL554.
The fresh data boosted Verona's stock to its highest mark on the NASDAQ since the company conducted an initial public offering last April. Shares opened at $15.30 apiece Friday, and shortly after ticked up to $17.45 apiece — reflecting a 26% increase from the prior day's per-share closing price.
And the climb may be just starting.
Jefferies analyst Peter Welford reissued a "Buy" rating for the company in a March 2 note, estimating its stock could hit $28 per share. He also acknowledged RPL554's novel mechanism of action, which allows it to reduce inflammation as well as open up a patient's airway, gives it a differentiated product profile.
"Detailed data and patient baseline characteristics are required to put these data in context, but we believe the outcome is encouraging and suggests further development is justified," Welford wrote, referring to the Phase 2a cystic fibrosis results.
Late-stage development may be tricky, however. That's because more than half of the 75,000 cystic fibrosis patients worldwide are able to take at least one of the three approved drugs from Vertex Pharmaceuticals Inc. Analysts have noted that any drugmaker looking to challenge Vertex will have a tough time convincing patients to go off biotech's proven treatments in lieu of an investigational medication.
Yet Verona CEO Jan-Anders Karlsson doesn't see that as much of barrier. In fact, he told BioPharma Dive that Phase 3 studies of RPL554 would likely enroll patients who couldn't go off their current therapy.
"Patients that are on a steady dose of steroid, they're allowed to continue it in our COPD trials. So it will be the same thing in CF. If they are on a steady dose of Kalydeco and they need Kalydeco, they would need to stay on that," he told BioPharma Dive.
"It's not replacing any of the CF active compounds, it's addressing what these compounds do not affect — and that's the inflammatory compound. And as far as we know today, there's no negative interactions,", Karlsson added.
The Phase 2a trial operated in that way. Investigators couldn't enroll patients who received an experimental drug within the last 3 months or five half-lives of the drug, yet there was no exclusion criteria related to taking an approved treatment. Notably, the trial enrolled just 10 patients.
Welford predicts $230 million in peak global sales of RPL554 in cystic fibrosis and a 20% probability of success. He acknowledged, though, that the nebulized form of the drug could tap into three market's with blockbuster potential: cystic fibrosis, COPD maintenance and COPD acute exacerbations.
Additional results showed patients receiving RPL554 demonstrated a sustained improvement in average FEV1 after four hours, six hours and eight hours. The 1.5 mg and 6 mg regimens also showed a dose-dependent half-life of 7.5 and 10.1 hours, respectively.