Verona’s COPD drug shines in PhIII study, potentially clearing path to FDA — shares jump
UK-based Verona Pharma’s COPD drug, ensifentrine, has succeeded in its Phase III trial, paving the way for a possible FDA approval.
In Verona’s Phase III ENHANCE-2 study, roughly 800 patients with moderate to severe COPD received ensifentrine or placebo through a nebulizer twice a day for 24 weeks. At 12 hours post-treatment on week 12, the placebo-corrected change in forced expiratory volume (FEV1) — a standard measure of lung function that tests how much breath one can forcefully exhale in one second — was 94 mL, leading the trial to meet its primary endpoint.
The data took investors’ breath away, as Verona shares $VRNA jumped more than 70% in early Tuesday trading.
Approximately 52% of patients had received background COPD therapy, either a long-acting muscarinic antagonist (LAMA) or a long-acting beta-agonist (LABA). Additionally, approximately 15% of all subjects received inhaled corticosteroids (ICS) with concomitant LAMA or LABA. However, the results of these specific subgroups were not shared.
“These data, along with results from our ongoing Phase 3 trial, ENHANCE-1, which is on track to be reported around the end of 2022, if similarly positive, are expected to support the submission of a New Drug Application to the US Food and Drug Administration in the first half of 2023,” said Verona’s CEO David Zaccardelli in a press statement.
The study also met its secondary endpoint of improving lung function. At week 12, the peak FEV1 in the 0 to 4 hours after dosing with ensifentrine was higher than the placebo by 146 mL. The placebo-corrected morning trough FEV1 at week 12 was 49 mL.
While the study achieved its primary and secondary endpoints, the drug failed to beat placebo on measures of COPD symptoms and quality of life.
Antonio Anzueto, a professor of medicine and chief of pulmonary at South Texas Veterans Healthcare System, said in a release:
Ensifentrine has demonstrated clear improvements in lung function in addition to favorable safety results. I am extremely excited by the clinically meaningful 42% reduction in the rate of exacerbations observed over 24 weeks in these symptomatic patients, many receiving background therapy.
Verona has touted ensifentrine, or RPL554, as the first potential therapy for respiratory diseases that acts as both a bronchodilator and anti-inflammatory agent in a solitary compound. The biotech, founded in 2005 and built solely around ensifentrine, says the molecule is the first that can both widen patients’ airways and reduce inflammation.
But it hasn’t always been smooth sailing. In 2019, the drug failed to significantly improve lung function in a three-day study when given on top of current COPD drugs. Last February, though, a longer study found the nebulized form did improve lung function when given on top of existing drugs, and Verona was able to raise nearly $200 million to launch a pair of pivotal Phase III studies, testing the drug both alone and in combination.
Meanwhile, other recent drugs for COPD include AstraZeneca’s FDA-approved three-in-one inhaler Breztri Aerosphere, which helps in the maintenance of COPD.