Verona Pharma Initiates Phase 2 Clinical Trial with Metered Dose Inhaler Formulation of Ensifentrine for Maintenance Treatment of COPD

June 04 2019, LONDON – Verona Pharma plc (AIM: VRP) (Nasdaq: VRNA) (“Verona Pharma”), a biopharmaceutical company focused on respiratory diseases, announces the initiation of a Phase 2 dose-ranging trial to evaluate the pharmacokinetic (“PK”) profile, efficacy and safety of a pressurized metered-dose inhaler (“MDI”) formulation of ensifentrine (RPL554) in patients with moderate-to-severe chronic obstructive pulmonary disease (“COPD”). Verona Pharma is also developing a dry powder inhaler (“DPI”) formulation of ensifentrine.

In the US, DPI/MDI handheld inhalers are the most widely used option for medication in COPD, where an estimated 5.5 million people use inhalers for COPD maintenance therapy.[1] This market was valued at approximately $6 billion in 2017.[2]

The Phase 2 trial using the MDI formulation has a randomized, double-blind, placebo-controlled, two-part design. The Company anticipates reporting data from the first part of the trial in the second half of 2019, with final data expected in the first quarter of 2020.

The first part of the trial will evaluate the PK profile following a single dose of ensifentrine over 5 dose cohorts (100 µg, 300 µg, 1000 µg, 3000 µg, 6000 µg) or placebo. Verona Pharma is aiming to enroll 36 patients with moderate-to-severe COPD at two sites in the UK. The safety, tolerability and the bronchodilation effect of ensifentrine will also be assessed.

The second part of the trial will be conducted as a multiple dose crossover trial. Patients will be randomized to up to 4 dose levels or placebo, with the ensifentrine MDI formulation dosed twice-daily over one week. All patients will receive each of the dose levels and placebo, over the seven-day treatment period. The primary endpoint is based on bronchodilation and will be assessed in terms of peak forced expiratory volume in one second (FEV₁), a standard measure of lung function. Secondary objectives of this part of the trial include evaluating the safety, tolerability and the bronchodilator profile as well as the PK profile.

“We are excited to expand the ensifentrine portfolio with the MDI formulation for the treatment of patients with COPD. DPI and MDI inhaler formulations have the potential to expand dramatically the clinical utility and commercial opportunity of ensifentrine in the treatment of COPD,” said Jan-Anders Karlsson, PhD, CEO of Verona Pharma. “We plan to seek a strategic partner to develop and commercialize inhalers of ensifentrine to maximize the significant potential value from this opportunity.”

In addition to advancing the MDI and DPI formulations of ensifentrine into Phase 2 clinical development, Verona Pharma has also developed a nebulized formulation of ensifentrine, which is currently being evaluated in a Phase 2b clinical trial in moderate-to-severe COPD patients designed to inform dose selection for Phase 3 trials planned for 2020.

About COPD

COPD is a progressive and life-threatening respiratory disease without a cure. The World Health Organization estimates that it will become the third leading cause of death worldwide by 2030. The condition damages the airways and the lungs, leading to debilitating breathlessness that has a devastating impact on performing basic daily activities such as getting out of bed, showering, eating and walking. In the United States alone, the 2010 total annual medical costs related to COPD were estimated to be $32 billion and are projected to rise to $49 billion in 2020. About 800,000 US COPD patients on dual/triple inhaled therapy (LAMA/LABA +/- ICS) remain uncontrolled, experiencing symptoms that impair quality of life. These patients urgently need better treatments.

About Verona Pharma plc

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs. Verona Pharma’s product candidate, ensifentrine (RPL554), is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 that has been shown to act as both a bronchodilator and an anti-inflammatory agent in a single compound. Nebulized ensifentrine is currently in Phase 2b clinical development for the maintenance treatment of COPD and is planned to enter Phase 3 trials for this indication in 2020. Verona Pharma plans a targeted US launch of the nebulized formulation, which is expected to benefit from a simplified Medicare Part B reimbursement process in the US. Verona Pharma may also develop ensifentrine for the treatment of cystic fibrosis and asthma.

Ensifentrine has shown significant and clinically meaningful improvements in both lung function and COPD symptoms, including breathlessness in prior Phase 2 clinical studies in patients with moderate-to-severe COPD. In addition, ensifentrine has further improved lung function and reduced lung volumes in patients taking standard short- and long-acting bronchodilator therapy, including maximum bronchodilator treatment with dual/triple therapy. Ensifentrine has been well tolerated in clinical trials involving more than 800 people to date.

Forward-Looking Statements

This press release contains forward-looking statements. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to, statements regarding the design of the Phase 2 clinical trial of the MDI formulation of ensifentrine, the timing of availability of data from the Phase 2 clinical trial, the development plans for ensifentrine, the timing of the end-of-phase 2 meeting with the FDA and the planned Phase 3 trials of nebulized ensifentrine, the value of the COPD market, the potential of ensifentrine as a promising first-in-class treatment option for COPD and the availability of Medicare reimbursement.

These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from our expectations expressed or implied by the forward-looking statements, including, but not limited to, the following: our limited operating history; our need for additional funding to complete development and commercialization of ensifentrine, which may not be available and which may force us to delay, reduce or eliminate our development or commercialization efforts; the reliance of our business on the success of ensifentrine, our only product candidate under development; economic, political, regulatory and other risks involved with international operations; the lengthy and expensive process of clinical drug development, which has an uncertain outcome; serious adverse, undesirable or unacceptable side effects associated with ensifentrine, which could adversely affect our ability to develop or commercialize ensifentrine; potential delays in enrolling patients, which could adversely affect our research and development efforts and the completion of our Phase 2b trial; we may not be successful in developing ensifentrine for multiple indications; our ability to obtain approval for and commercialize ensifentrine in multiple major pharmaceutical markets; misconduct or other improper activities by our employees, consultants, principal investigators, and third-party service providers; material differences between our “top-line” data and final data; our reliance on third parties, including clinical investigators, manufacturers and suppliers, and the risks related to these parties’ ability to successfully develop and commercialize ensifentrine; and lawsuits related to patents covering ensifentrine and the potential for our patents to be found invalid or unenforceable. These and other important factors under the caption “Risk Factors” in our Annual Report on Form 20-F filed with the Securities and Exchange Commission (“SEC”) on March 19, 2019, and our other reports filed with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

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