Verona Pharma Initiates Additional Phase 2b Clinical Trial with Nebulized Ensifentrine to Inform Dose Selection for Phase 3 Trials

LONDON, May 07, 2019 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM: VRP) ( VRNA) (“Verona Pharma”), a clinical-stage biopharmaceutical company focused on respiratory diseases, announces the initiation of a Phase 2b dose-ranging study evaluating nebulized ensifentrine (RPL554) added on to a long acting bronchodilator in patients with moderate-to-severe chronic obstructive pulmonary disease (“COPD”). The Company anticipates reporting data around year end and commencing Phase 3 clinical trials for this indication in 2020.

Ensifentrine is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 that has been shown to act both as a bronchodilator and anti-inflammatory agent in a single compound.

Jan-Anders Karlsson, PhD, CEO of Verona Pharma, said: “In addition to further evaluating the potential of nebulized ensifentrine as a first-in-class therapy in symptomatic COPD patients already receiving a long-acting bronchodilator, this study is an important step towards optimizing the clinical profile of ensifentrine for the planned End-of-Phase 2 meeting with the FDA in the first half of 2020. The study is also expected to further inform the Phase 3 clinical development program for ensifentrine in terms of dose selection, patient population, background therapy and clinical endpoints.”

The four-week, randomized, double-blind, dose-ranging Phase 2b trial plans to enroll about 400 patients with moderate-to-severe COPD to evaluate the safety and efficacy of nebulized ensifentrine when added on to inhaled tiotropium, a long-acting anti-muscarinic (“LAMA”) commonly used to treat COPD. Patients will receive twice-a-day dosing for 28 days of nebulized ensifentrine at four dosage levels: 0.375 mg, 0.75 mg, 1.5 mg and 3.0 mg. The trial is being performed at approximately 50 sites in the US.

The primary endpoint of the trial is improvement in lung function with ensifentrine after 4 weeks of treatment, as measured by peak forced expiratory volume in one second (“FEV₁”), a standard measure of lung function. FEV₁ will be measured from 0 to 3 hours after dosing. Key additional endpoints include measurements of respiratory symptoms and quality of life measures via different patient-reported outcome tools.

In prior Phase 2 clinical studies in patients with moderate to severe COPD, ensifentrine has shown significant and clinically meaningful improvements in both lung function and COPD symptoms, including breathlessness. 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.

In addition to advancing the nebulized formulation of ensifentrine through Phase 2b clinical development, Verona Pharma has also developed a dry powder inhaler (“DPI”) and a pressurised metered dose inhaler (“pMDI”) formulation of ensifentrine. In an ongoing Phase 2 clinical trial, the DPI formulation demonstrated dose-dependent, significant bronchodilation following a single dose, and is currently being evaluated over one week of twice-daily treatment, with top-line data expected in the third quarter this year. The pMDI formulation is planned to enter clinical evaluation in a Phase 2 study in COPD patients later this quarter.

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. 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 may also develop ensifentrine for the treatment of cystic fibrosis and asthma.

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 2b clinical trial of ensifentrine, the timing of availability of data from the Phase 2b clinical trial, the importance of the Phase 2b clinical trial to our planned end-of-phase 2 meeting with the FDA and the development plans for ensifentrine, the timing of the end-of-phase 2 meeting with the FDA and the planned Phase 3 trials, the value of the data and insights that may be gathered from the Phase 2b clinical trial, including for the purpose of designing pivotal Phase 3 trials, and the potential of ensifentrine as a promising first-in-class treatment option for COPD.

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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