Verona builds case for COPD drug ensifentrine as filing nears

Verona Pharma is looking odds on for a regulatory approval for its chronic obstructive pulmonary disease (COPD) therapy ensifentrine, after chalking up a second positive phase 3 trial.

The Anglo-US company reported the results of the ENHANCE-1 study earlier today, with the first-in-class phosphodiesterase 3 and 4 inhibitor achieving significant improvements in lung function, as well as a 36% reduction in the moderate or severe COPD exacerbation rate over 24 weeks.

On a conference call, chief executive David Zaccardelli said the readout marks a "momentous day for ensifentrine Verona Pharma and, most importantly, the millions of patients suffering from COPD", adding that the data confirms the results of the ENHANCE-2 study reported in August and sets up regulatory filings for the drug in the first half of 2023.

He said ensifentrine will "change the treatment paradigm" for COPD by providing bronchodilation and anti-inflammatory properties in one compound.

In both studies, it was administered on top of therapy with either a long-acting beta agonist (LABA) or long-acting muscarinic antagonist (LAMA), which are standard therapies for COPD, with the pooled data from them finding a 40% reduction in the exacerbation rate.

Around one in five subjects were also taking corticosteroids, so were already receiving therapy targeting both bronchodilation and inflammation.

The drug – which is administered twice-daily using a nebuliser as a chronic maintenance therapy for people with COPD – achieved significant improvements on the primary endpoint of forced expiratory volume (FEV1) at 12 weeks, as well as in COPD symptom scores, with tolerability said to be similar to placebo over a 24-week follow-up period.

Shares in Verona popped on the news, rising more than 40% to approach $19.50 and drive the company's market cap to almost $1.5 billion.

Wedbush Securities recently forecast more than $1 billion in 2029 sales for ensifentrine in the US alone, assuming a launch in the first quarter of 2024, and said today the stock could easily reach $30.

COPD is the third leading cause of death worldwide, with 3.23 million deaths in 2019 alone, and ensifentrine's top-line exacerbation reduction rate of 40% compares favourably with established therapies like GSK's Breo (fluticasone furoate/vilanterol) and Trelegy (fluticasone furoate/umeclidinium/vilanterol), as well as Boehringer Ingelheim's Spiriva (tiotropium).

Zaccardelli also said that he anticipates that partnering activity will step up quickly now that the combined dataset is available, with the company expected to seek further commercial arrangements for ensifentrine in the US and Europe.

It has already licensed Chinese rights to the drug to Nuance Pharma for $40 million upfront, plus up to $179 million in milestones and double-digit royalties.