Verona says FDA backs plan to take COPD drug into phase 3

More than two years after first saying it would take its lead drug into a pivotal trial, Verona Pharma looks like it may finally follow through on that promise.

The UK biopharma says it has received a positive response from the FDA on its end-of-phase 2 data package for ensifentrine, intended to treat chronic obstructive pulmonary disease (COPD), and so will start a pair of phase 3 trials later this year.

Shares in Verona yo-yoed after the announcement but were in positive territory by the end of the day as investors digested the news, delivered alongside its quarterly business update.

Verona has been talking about advancing ensifentrine into late-stage development since reporting a successful phase 2b trial in 2018, but suffered a setback last year when another mid-stage trial missed its primary endpoint.

The dual PDE3/PDE4 inhibitor – formerly known as RPL554 and designed to dampen down inflammation in the lungs – is designed to be administered via a nebuliser to COPD patients as an add-on therapy to other drugs for the progressive lung condition.

In a statement, Verona said it now had “clarity from the FDA” on the key features of the planned phase 3 programme, including the dose that should be tested in the trials, the main outcome measures, and the patient population and study design.

The two ENHANCE trials have the same design, but the first (ENHANCE-1) will include longer safety follow-up than the second.

Each will test twice-daily nebulised ensifentrine 3mg as monotherapy and as an add-on to standard of care treatment with a single drug design to dilate the airways, such as a long-acting beta agonist (LABA) or long-acting muscarinic antagonist (LAMA), in around 800 moderate to severe COPD patients.

The primary endpoint will be improvement in lung function, measured by forced expiratory volume in one second (FEV1) over 12 hours, after 12 weeks of treatment, while secondary analyses will look at peak and trough FEV1 and COPD symptoms like shortness of breath, cough and chest infections.

COPD is a serious lung disease that affects millions of people, and trends to progress over time with patients frequently becoming highly debilitated.

It’s also proved a lucrative disease for drugmakers to target, with combination therapies delivered by inhaler based on beta agonists and inhaled corticosteroids (ICS) – such as GlaxoSmithKline’s Advair – making billions of dollars a year in sales.

Latterly treatment has started to shift towards triple therapy with LAMA, LABA and ICS drugs given together in products like GSK’s Trelegy and while there has been a slowdown in the respiratory market overall COPD was still a near $10 billion market in the US alone last year.

Verona reckons that there are more than a million COPD patients who can’t control COPD symptoms using current dual or triple therapies in the US, and ensifentrine could have a role to play in this sizeable group.