26 October 2016

VERONA PHARMA PLC - Second Venture and Innovation Award from the CF Trust

PR Newswire

Verona Pharma plc

("Verona Pharma" or the "Company")

Verona Pharma receives second Venture and Innovation Award from the Cystic Fibrosis Trust

Supports first clinical study in patients to explore potential for RPL554 as a novel treatment for cystic fibrosis

New data from pre-clinical studies to be presented at Annual North American Cystic Fibrosis Conference, 26-28 October 2016

26 October 2016, Cardiff – Verona Pharma plc (AIM: VRP.L), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for the treatment of respiratory diseases, announces that it has received a second Venture and Innovation Award from the UK Cystic Fibrosis Trust. The award will be used to help fund a Phase 2a clinical study to investigate the potential of the Company’s drug candidate, RPL554, as a novel treatment for cystic fibrosis (CF).

The study is expected to start recruiting patients with CF during the first half of 2017. The primary objective of the study will be to determine the pharmacokinetic and pharmacodynamics profile and assess the tolerability and safety of RPL554 in patients with this disease.

The first Venture and Innovation Award the Company received from the UK Cystic Fibrosis Trust was granted in November 2014 and was used to help fund exploratory pre-clinical studies, which delivered encouraging data presented last year at the North American Cystic Fibrosis Conference and published in the American Journal of Physiology that has shown the potential of RPL554 as a treatment of CF.

New pre-clinical data of RPL554 in CF will be available from 26 October 2016 and presented during two poster sessions on 27 and 28 October 2016 at the Annual North American Cystic Fibrosis Conference in Orlando, Florida.

CF is an orphan disease that afflicts approximately 70,000 people worldwide. The disease is caused by mutations in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). CF affects several organs in the body but pulmonary disease is the primary cause of mortality. Current therapies are directed at the impaired mucociliary clearance, the chronic lung infections and the chronic inflammation of the lungs, but with limited efficacy. Recently, drugs targeting the mutant CFTR function have become available. While these latter therapies are effective in small sub-sets of patients, most patients are still without adequate therapy for this life-shortening, chronic progressive disease.

RPL554, a first-in-class, inhaled, dual PDE3/PDE4 inhibitor, is currently in Phase 2 clinical development as a nebulized treatment for Chronic Obstructive Pulmonary Disease (COPD). In clinical trials, RPL554 has been observed to be an effective bronchodilator and to have anti-inflammatory properties in vitro as well as in a standard inhaled lipopolysaccharide challenge study in healthy volunteers. In these studies, the drug candidate has been well tolerated. Pre-clinical data to date has suggested that RPL554 is also a stimulator of the CFTR, which is dysfunctional in cells of cystic fibrosis patients. Based on these observed favorable properties, the Company believes RPL554 may improve mucociliary clearance (reduce phlegm in the airways), reduce symptoms of chronic inflammation and ease breathing.  

Commenting on the award, Verona Pharma’s CEO, Jan-Anders Karlsson, said:

“We are delighted to have received a second Venture and Innovation Award from the Cystic Fibrosis Trust, which will enable us to accelerate the clinical development of RPL554 for the treatment of cystic fibrosis, an orphan disease where there is very high unmet medical need. The award recognises RPL554’s potential to be a novel and important treatment for this debilitating condition and we look forward to progressing the drug candidate through clinical development.”

Ed Owen, outgoing Chief Executive of the Cystic Fibrosis Trust, said: “We are delighted to continue to support the development of this compound and explore its potential to be an effective and complementary new treatment for cystic fibrosis, which remains an area of significant unmet medical need.

A vital part of our fight for a Life Unlimited is to accelerate the development of potential new therapies for people with cystic fibrosis. Building innovative collaborations with companies like Verona ensures that promising drug candidates are developed for use in cystic fibrosis and are given the best chance to get to those who need them as quickly as possible."

The title, timing and location of the abstract presentations at the Annual North American Cystic Fibrosis Conference are as follows:

Abstract Number 40
Authors: M.Turner, K.H. Abbott-Banner, S.H Randell, J.W Hanrahan
Day/Date: October 26-28, 2016
Location: Hall C
Time: Thursday, October 27: 11:15 a.m. – 1:45 p.m.
Friday, October 28: 4:00 p.m. – 6:00 p.m.
Session Type: Poster

This announcement contains price sensitive information.


For further information please contact:

Verona Pharma plc Tel: +44 (0)20 3283 4200
Jan-Anders Karlsson, Chief Executive Officer  
N+1 Singer Tel: +44 (0)20 7496 3000
Aubrey Powell / Jen Boorer  
FTI Consulting Tel: +44 (0)20 3727 1000
Simon Conway / Stephanie Cuthbert /
Natalie Garland-Collins

Notes to Editors

About Verona Pharma plc and RPL554

Verona Pharma plc is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for the treatment of respiratory diseases with significant unmet medical needs, such as chronic obstructive pulmonary disease (COPD), cystic fibrosis (CF) and asthma. 

Verona Pharma's drug candidate, RPL554, is a first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4, or PDE3 and PDE4, that acts as both a bronchodilator and an anti-inflammatory agent in a single molecule. The Company has successfully completed eight Phase 1 and 2a clinical trials with RPL554 to date, with 299 subjects enrolled. RPL554 has been observed to provide clinically meaningful and statistically significant improvements in lung function as both a single agent and as an add-on therapy with currently marketed bronchodilator drugs. In addition, RPL554 has been observed to shorten the time of onset of bronchodilation when administered as an add-on therapy with currently marketed bronchodilators, and has also shown anti-inflammatory effects. RPL554 has been well tolerated in each of its clinical trials. Verona Pharma also intends to pursue development of RPL554 to address multiple other respiratory diseases, including 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.

For further information please contact:


Verona Pharma plc
Jan-Anders Karlsson, CEO
Victoria Stewart, Director of Communications
Tel: +44 (0)20 3283 4200

N+1 Singer (Nominated Adviser and UK Broker)
Aubrey Powell /Jen Boorer /Iqra Amin (Corporate Finance)
Brough Ransom /Mia Gardner (Corporate Broking)
Tel: +44 (0)20 3283 4200



Optimum Strategic Communications
(European Media and Investor enquiries)
Mary Clark, Annemarieke Ezendam
Hollie Vile
Tel: +44 (0) 203 950 9144

Argot Partners (Investor enquiries)
Stephanie Marks
Tel. +1 646 644 9590


Verona Pharma plc
3 More London Riverside, London SE1 2RE
T: +44 (0)203 283 4200

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