Verona cues up filings for COPD drug after it aces phase 3 test

UK-based Verona Pharma has scored a key win in a phase 3 trial of its chronic obstructive pulmonary disease (COPD) drug ensifentrine, setting up regulatory filings in the first half of next year.

The drug was administered twice-daily using a nebuliser as a chronic maintenance therapy for people with COPD in the ENHANCE-2 study, cutting the rate of moderate to severe exacerbations – sudden worsening of COPD symptoms – by 42% over 24 weeks.

Ensifentrine also achieved significant improvements in key measures of lung function in the trial, including the primary endpoint of forced expiratory volume (FEV1) at 12 weeks, as well as in COPD symptom scores, with safety results "similar to placebo."

There was a trend towards improved and quality-of-life scores that did not reach statistical significance, the only miss in the dataset, which is remarkable given that ensifentrine failed a phase 2 trial in 2019.

Ensifentrine is billed as a first-in-class dual inhibitor of the enzymes phosphodiesterase 3 and 4 (PDE3 and PDE4), and is thought to combine bronchodilator and anti-inflammatory properties in one compound.

The ENHANCE-2 results are particularly impressive as more than half (52%) of the 789 patients enrolled into the study were receiving background therapy, mainly with either long-acting muscarinic antagonist (LAMA) or long-acting beta agonist (LABA) drugs.

Moreover, 15% of them were taking LAMA or LABA therapies in combination with inhaled corticosteroids (ICS), so were already receiving therapy targeting both bronchodilation and inflammation.

Shares in Verona listed on the Nasdaq shot up more than 28% ahead of the market opening as investors reacted to the positive news, and the prospect of a potential approval and launch of the company's lead drug candidate into the large COPD market in late 2023 or 2024.

There's another hurdle to overcome before a filing can take place – the results of the companion ENHANCE-1 phase 3 trial which are due towards the end of the year – but the positive results across the board in ENHANCE-2 bode well for that readout.

Analyst Gil Aharon of Rosalind Advisors said on a discussion of the trial ahead of the readout that positive results in a blockbuster indication – for a company with a market cap of just $420 million even with the latest leap – would make for an "extraordinary" risk:reward scenario.

Wedbush Securities meanwhile recently forecast more than $1 billion in 2029 sales for ensifentrine in the US alone, assuming a launch in the first quarter of 2024.

Verona also reported its second-quarter results this morning, ending the period with cash reserves of just under $112 million, enough to cover its operations through to the end of 2023.

With its phase 3 programme for ensifentrine halfway complete and biopharma M&A activity picking up, the company must surely now be on the hit-list of potential takeover targets.

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