Funds further studies exploring potential for RPL554 as a novel treatment for CF
Verona Pharma first drug development company to receive Award
3 November 2014, Cardiff – Verona Pharma plc (AIM: VRP), the drug development company focused on first-in-class medicines to treat respiratory diseases, today announces it has received a Venture and Innovation Award from the Cystic Fibrosis Trust. The Award will be used to fund further exploratory studies to investigate the potential of the Company’s lead pipeline drug, RPL554, as a novel treatment for cystic fibrosis (CF). Verona Pharma is the first drug development company to receive a Venture and Innovation Award from the Trust.
The planned studies, in well-recognised and validated translational models of CF disease, entail examining further the effect of RPL554 on airway cells obtained from CF patients. They follow on from encouraging preliminary data of such effects that were recently notified on 29 September 2014 and presented at The 28th Annual North American Cystic Fibrosis Conference (NACFC), Atlanta, Georgia, USA, 9-11 October, 20141. The studies will be performed in collaboration with Professors John Hanrahan and David Thomas of the CF Translational Research Centre at McGill University (who also collaborated on the initial studies) and Professor Scott Randell, Dept of Cell Biology and Physiology and the Dept of Medicine, The University of North Carolina at Chapel Hill, all of whom are recognised authorities in CF translational research.
RPL554, a first-in-class dual PDE3/PDE4 inhibitor, is currently in phase 2 clinical development as a nebulized treatment for acute Chronic Obstructive Pulmonary Disease (COPD).
Dr Jan-Anders Karlsson, CEO of Verona Pharma, commented:
“We are delighted to have been awarded this Venture and Innovation Award from the Cystic Fibrosis Trust. It supports our view that RPL554’s novel mode of action could be very important in this disease and will enable us to work with our collaborators to build on the encouraging preliminary findings that we reported at the recent North American Cystic Fibrosis conference. This data suggests our lead pipeline drug RPL554, a PDE3/PDE4 inhibitor, currently in phase 2 clinical trials for acute COPD, might also have utility as a novel treatment for patients with cystic fibrosis, an under-treated and orphan disease.”
Dr Janet Allen, Director of Research & Care at the Cystic Fibrosis Trust, said:
“Cystic Fibrosis afflicts over 10,000 people in the UK alone and over 70,000 people worldwide. While it is one of the most common life-threatening genetic diseases, it is still under-treated and there is great need for effective, new treatments. The initial data on the impact of RPL554 on a central target in CF drug discovery are very encouraging and makes it one of the most promising drugs currently in early testing. As a charity that supports research to better understand and treat cystic fibrosis, we are delighted to further fund Verona Pharma and their collaborators in this work, through a Venture and Innovation Award, which will enable them to further expand on these preliminary findings.
“This research is a prime example of what Venture and Innovation Awards were set up to achieve, helping leverage financial support from other agencies to support transformational research projects.”
Prof John Hanrahan, Director, CF Translational Research centre, McGill University commented: “In our experiments, RPL554 increased the activity of CFTR, anion channels on the surface of well-differentiated cells from the lining of the airway. In CF patients it is the dysfunction of these channels, due to genetic mutations, that is responsible for the symptoms of the disease. This award will enable us to continue to examine this effect of RPL554 in further studies. Ultimately, if found effective and safe in cystic fibrosis patients, RPL554 could emerge as a new medicine for this debilitating disease.”
Assoc Prof Scott Randell, Dept of Cell Biology and Physiology and the Dept of Medicine, The University of North Carolina at Chapel Hill, commented:
“The preliminary data of the effect of RPL554 on a well-recognised target in cystic fibrosis drug discovery is intriguing and clearly warrants further study. We are excited to be part of a collaboration to do this and thank The Cystic Fibrosis Trust for the Venture and Innovation Award that will help fund these next steps.”
(1) Matthes, E., Billet, A., Darling, A., Goepp, J., Robert, R., Thomas, D.Y., Banner, K.H., Hanrahan, J.W.; CFTR activation by the dual phosphodiesterase 3/4 inhibitor RPL554 and the MRP4 inhibitor MK571, Abstract 277
For further information please contact:
Verona Pharma plc Tel: +44 (0) 20 7863 3300 Jan-Anders Karlsson, CEO
N+1 Singer Tel: +44 (0)20 7496 3000 Aubrey Powell / Jen Boorer
FTI Consulting Tel: +44 (0)20 3727 1000 Julia Phillips / Simon Conway
Notes to Editors
About Verona Pharma plc
Verona Pharma is developing first-in-class drugs to treat respiratory disease, such as COPD and asthma. The Company currently has two drug programmes, one of which is in Phase 2 trials for two diseases. The lead programme, RPL554, is an innovative dual phosphodiesterase (PDE) 3 and 4 inhibitor with both bronchodilator and anti-inflammatory properties. In its second programme, Verona Pharma is investigating novel anti-inflammatory molecules, called NAIPs, for a wide range of respiratory and inflammatory diseases.
About RPL554 for the treatment of COPD and Asthma
Verona’s lead drug, RPL554, is a dual phosphodiesterase (PDE) 3 and 4 inhibitor being developed as a novel treatment for chronic obstructive airways disease such as COPD (chronic obstructive pulmonary disorder) and asthma, with bronchodilator and anti-inflammatory effects. Both effects are essential to improve symptoms in patients with COPD or asthma. RPL554 is currently in Phase 2 for both diseases.
COPD is a chronic lung disease with significant unmet need for which current treatment is far from optimal, as it often has unwanted side-effects and/or limited effectiveness. COPD is most commonly characterised by fixed airflow obstruction and chronic airways inflammation resulting from exposure to irritants like tobacco smoke. Asthma, which remains one of the most common chronic diseases in the world, is characterised by recurrent breathing problems and symptoms such as breathlessness, wheezing, chest tightness, and coughing. The combined market for COPD and asthma drugs is currently estimated to be GBP20 billion (source: visiongain).
About Cystic Fibrosis
Cystic fibrosis (CF) is an orphan disease that afflicts approximately 70,000 people worldwide. The disease affects mostly the lungs, and also the pancreas, liver, and intestine. Difficulty breathing is the most serious symptom and results from frequent lung infections. CF is caused by one of many different mutations in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). This protein is required to regulate the components of sweat, digestive fluids, and mucus. Healthy people have two working copies of the CFTR gene. Carriers have one working copy. People with CF have no working copy. CF therefore has autosomal recessive inheritance. The underlying mechanism is abnormal transport of chloride and sodium across the epithelium, which is the cell layer that covers membranes over organs. This leads to thick, viscous secretions. Individuals with cystic fibrosis can be diagnosed before birth by genetic testing or by a sweat test in early childhood. The name cystic fibrosis refers to the characteristic scarring (fibrosis) and cyst formation within the pancreas, first recognised in the 1930s.
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.
About the Cystic Fibrosis Translational Research centre (CFTRc), McGill University
The CFTRc was established in the Faculty of Medicine at McGill University in 2011 with $5.5M of infrastructure funding from the Canada Foundation for Innovation. It comprises 28 researchers at McGill and other institutions from Quebec to British Columbia. It provides core facilities and other resources for CF research at the level of cells, tissues, and animal models, integrating physiological studies with chemical and structural biology, and with biochemical and cell biological studies of the mutant protein. It fosters interactions with industry, advises members concerning technology transfer and commercial agreements, and promotes preclinical and clinical studies of potential CF therapies.
About Assoc Prof Scott Randell, Dept of Cell Biology and Physiology and the Dept of Medicine, The University of North Carolina at Chapel Hill (UNC)
Research in Dr. Randell’s lab is focused on studies of lung cell biology applied towards overcoming clinical lung disease problems. Since 2001 he has also directed the UNC Cystic Fibrosis (CF) Center Tissue Procurement and Cell Culture Core. This facility is a nationally and internationally recognized resource, whose services are sought for collaboration, contract research, and training by academics, non-profit organizations, biotech and the pharmaceutical industry.
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