Verona's shares jump as COPD drug hits goal in phase 3, teeing up FDA filing

Verona Pharma has hit the mark in its first of two phase 3 chronic obstructive pulmonary disease (COPD) clinical trials, keeping it on course to file for FDA approval of the inhaled PDE3 and PDE4 inhibitor in the first half of next year. Shares in Verona rose around 30% to the region of $9 in premarket trading. 

The candidate, ensifentrine, is designed to have bronchodilator and anti-inflammatory effects, enabling it to drive improvements in lung function and symptoms. To test that hypothesis, Verona randomized almost 800 patients with moderate-to-severe chronic COPD to receive ensifentrine or placebo via a nebulizer twice a day for 24 weeks. 

After 12 weeks of treatment, the placebo-corrected change from baseline lung function was 94 mL, causing the trial to hit its primary endpoint. Verona said statistically significant improvements were seen across all subgroups.

The successful subgroups include the 52% of subjects who received background COPD therapy, namely a long-acting muscarinic antagonist or beta-agonist (LAMA/LABA), during the study, as well as the 15% of participants who received inhaled corticosteroids with concomitant LAMA or LABA. Verona is yet to post data on the subgroups but reported statistically significant and clinically meaningful improvements. A small phase 2 of patients on Boehringer Ingelheim’s LAMA/LABA agonist Stiolto Respimat failed in 2019.

Verona reported the subgroup success alongside details of secondary endpoints. At week 12, the peak FEV1 in the 0 to 4 hours after dosing with ensifentrine beat placebo by 146 mL. The placebo-corrected morning trough FEV1 at week 12 was 49 mL. The study also tracked a 42% fall in the rate of moderate-to-severe COPD exacerbations over 24 weeks. Verona said ensifentrine was well tolerated. 

The main negative in the results shared so far is the failure of ensifentrine to beat placebo on measures of COPD symptoms and quality of life. Ensifentrine numerically outperformed placebo at week 6, 12 and 24 but improvements in the control group caused the difference to fall short of statistical significance. 

Attention now turns to the other phase 3 trial, Enhance-1, that is scheduled to report results by the end of the year. If Enhance-1 is successful, Verona will file for FDA approval of ensifentrine in the first half of 2023 and begin preparing for a launch opportunity centered on the more than 1 million patients who are still symptomatic after receiving therapies such as LAMA and LABA agonists.

If Verona seizes that opportunity, it will represent a win for investors including RA Capital Management, Access Biotechnology, Perceptive Advisors, OrbiMed and New Enterprise Associates that pumped $200 million into the company two years ago. After receiving the investment, Verona shifted its financial focus from the U.K. to the U.S., delisting from London’s AIM market while retaining its Nasdaq listing.