VRP.L Regulatory News (VRP)

THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF REGULATION (EU) NO 596/2014.

Verona Pharma Doses First Patient in Phase 2a Clinical Trial Evaluating RPL554 for Cystic Fibrosis

4 April 2017, London- Verona Pharma plc (AIM: VRP.L), a clinical-stage biopharmaceutical company focused on developing and commercialising innovative therapeutics for the treatment of respiratory diseases, announced today that the first patient has been enrolled and dosed in a Phase 2a clinical study to evaluate Verona Pharma's product candidate, RPL554, as a treatment for cystic fibrosis (CF).

The Phase 2a single-dose trial is being conducted in the United Kingdom. The primary objective of this double-blind, placebo-controlled study will evaluate the pharmacokinetic and pharmacodynamics profile and tolerability of RPL554 in up to 10 CF patients, as well as examine the effect on lung function. Verona Pharma received its second Venture and Innovation Award from the UK Cystic Fibrosis Trust in October 2016 to fund this clinical trial. Verona Pharma received its first Venture and Innovation Award to fund exploratory pre-clinical studies of RPL554 for the treatment of CF in 2014.

The trial is being performed at the Cambridge Centre for Medical Research at Papworth Hospital, Cambridge, UK, one of the largest specialist cardiothoracic hospitals in Europe, by lead investigator Professor Andres Floto.

RPL554 is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 (PDE3/PDE4) with anti-inflammatory as well as bronchodilatory properties, currently in development for the treatment of chronic obstructive pulmonary disease (COPD) and CF.

In pre-clinical studies, RPL554 has been observed to stimulate the CF transmembrane conductance regulator, a protein whose mutation results in dysfunctional ion channels in epithelial cells, leading to CF. Based on available data, RPL554 has the potential to enhance mucociliary clearance (reduce phlegm in the airways), reduce airway obstruction and inhibit inflammation.

In previous clinical trials in patients with COPD, RPL554 has been observed to result in statistically significant improvements in lung function as compared to placebo and has shown clinically meaningful and statistically significant improvements in lung function when added to two commonly used bronchodilators as compared to either bronchodilator administered as a single agent. RPL554 has also shown anti-inflammatory effects in a standard challenge study with COPD-like inflammation in human subjects. In these previous studies, RPL554 has been well tolerated.

"Cystic fibrosis is the most common fatal inherited disease in the United States and Europe. More than 30,000 people in the U.S. and more than 70,000 people worldwide are living with cystic fibrosis," stated Jan-Anders Karlsson, PhD, CEO of Verona Pharma.

"Two recently approved therapies are indicated only for a subset of CF patients, and there is a need for novel, effective anti-inflammatory medications to treat the underlying inflammation in cystic fibrosis. RPL554 has a differentiated mechanism of action and pre-clinical data in cystic fibrosis combined with our positive clinical data in COPD leads us to believe it has the potential to be an important new treatment for this debilitating condition. We look forward to progressing the drug through this study and expect to announce top-line data in the first half of 2018."

Paula Sommer, Head of Research of the UK Cystic Fibrosis Trust, said: "The Venture Innovation Awards are designed to ensure that the Trust's funds go as far as possible to supporting ground-breaking treatments and innovative research by bringing in vital external funding. The RPL554 trial has the potential to make a difference to the lives of people with CF, and we are thrilled that the drug is moving into this important trial stage."

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About Verona Pharma plc

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercialising innovative therapeutics for the treatment of respiratory diseases with significant unmet medical needs. 

Verona Pharma's product candidate, RPL554, is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 that acts as both a bronchodilator and an anti‑inflammatory agent in a single compound. Verona Pharma is developing RPL554 for the treatment of chronic obstructive pulmonary disease (COPD), cystic fibrosis, and potentially asthma.

About The Cystic Fibrosis Trust

The Cystic Fibrosis Trust is the only UK-wide charity making a daily difference to the lives of people with cystic fibrosis, and those who care for them. Since its start in 1964 it has dedicated itself to promoting excellence in research and clinical care, as well as providing practical support and advice to people with cystic fibrosis and their families. The Trust believes that everyone with cystic fibrosis deserves the best quality of life and real hope for the future, with access to high quality, specialist care. To achieve this goal, it funds research to better understand and treat cystic fibrosis, review standards of cystic fibrosis care, and provide information and advice to the CF community.

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. 

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 number of patients to be dosed in and endpoints of our Phase 2a clinical trial to evaluate RPL554 as a treatment for cystic fibrosis, the timing of top-line data from the Phase 2 clinical trial, and the potential of RPL554 to treat cystic fibrosis.

These and other important factors 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.