CF Trust Venture and Innovation Award

Verona Pharma plc ("Verona Pharma" or the "Company") Verona Pharma announces Venture and Innovation Award from the Cystic Fibrosis Trust 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 developmentcompany focused on first-in-class medicines to treat respiratory diseases,today announces it has received a Venture and Innovation Award from the CysticFibrosis Trust. The Award will be used to fund further exploratory studies toinvestigate the potential of the Company's lead pipeline drug, RPL554, as anovel treatment for cystic fibrosis (CF). Verona Pharma is the first drugdevelopment company to receive a Venture and Innovation Award from the Trust. The planned studies, in well-recognised and validated translational models ofCF disease, entail examining further the effect of RPL554 on airway cellsobtained from CF patients. They follow on from encouraging preliminary data ofsuch effects that were recently notified on 29 September 2014 and presented atThe 28th Annual North American Cystic Fibrosis Conference (NACFC), Atlanta,Georgia, USA, 9-11 October, 20141. The studies will be performed incollaboration with Professors John Hanrahan and David Thomas of the CFTranslational Research Centre at McGill University (who also collaborated onthe initial studies) and Professor Scott Randell, Dept of Cell Biology andPhysiology and the Dept of Medicine, The University of North Carolina at ChapelHill, all of whom are recognised authorities in CF translational research. RPL554, a first-in-class dual PDE3/PDE4 inhibitor, is currently in phase 2clinical development as a nebulized treatment for acute Chronic ObstructivePulmonary Disease (COPD). Dr Jan-Anders Karlsson, CEO of Verona Pharma, commented: "We are delighted to have been awarded this Venture and Innovation Award fromthe Cystic Fibrosis Trust. It supports our view that RPL554's novel mode ofaction could be very important in this disease and will enable us to work withour collaborators to build on the encouraging preliminary findings that wereported at the recent North American Cystic Fibrosis conference. This datasuggests our lead pipeline drug RPL554, a PDE3/PDE4 inhibitor, currently inphase 2 clinical trials for acute COPD, might also have utility as a noveltreatment for patients with cystic fibrosis, an under-treated and orphandisease." 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 70,000people worldwide. While it is one of the most common life-threatening geneticdiseases, it is still under-treated and there is great need for effective, newtreatments. The initial data on the impact of RPL554 on a central target in CFdrug discovery are very encouraging and makes it one of the most promisingdrugs currently in early testing. As a charity that supports research to betterunderstand and treat cystic fibrosis, we are delighted to further fund VeronaPharma and their collaborators in this work, through a Venture and InnovationAward, which will enable them to further expand on these preliminary findings. "This research is a prime example of what Venture and Innovation Awards wereset up to achieve, helping leverage financial support from other agencies tosupport transformational research projects." Prof John Hanrahan, Director, CF Translational Research centre, McGillUniversity commented: "In our experiments, RPL554 increased the activity of CFTR, anion channels onthe surface of well-differentiated cells from the lining of the airway. In CFpatients it is the dysfunction of these channels, due to genetic mutations,that is responsible for the symptoms of the disease. This award will enable usto continue to examine this effect of RPL554 in further studies. Ultimately, iffound effective and safe in cystic fibrosis patients, RPL554 could emerge as anew medicine for this debilitating disease." Assoc Prof Scott Randell, Dept of Cell Biology and Physiology and the Dept ofMedicine, The University of North Carolina at Chapel Hill, commented: "The preliminary data of the effect of RPL554 on a well-recognised target incystic fibrosis drug discovery is intriguing and clearly warrants furtherstudy. We are excited to be part of a collaboration to do this and thank TheCystic Fibrosis Trust for the Venture and Innovation Award that will help fundthese 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 ofwhich is in Phase 2 trials for two diseases. The lead programme, RPL554, is aninnovative dual phosphodiesterase (PDE) 3 and 4 inhibitor with bothbronchodilator 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 inhibitorbeing developed as a novel treatment for chronic obstructive airways diseasesuch as COPD (chronic obstructive pulmonary disorder) and asthma, withbronchodilator and anti-inflammatory effects. Both effects are essential toimprove symptoms in patients with COPD or asthma. RPL554 is currently in Phase2 for both diseases. COPD is a chronic lung disease with significant unmet need for which currenttreatment is far from optimal, as it often has unwanted side-effects and/orlimited effectiveness. COPD is most commonly characterised by fixed airflowobstruction and chronic airways inflammation resulting from exposure toirritants like tobacco smoke. Asthma, which remains one of the most commonchronic diseases in the world, is characterised by recurrent breathing problemsand symptoms such as breathlessness, wheezing, chest tightness, and coughing.The combined market for COPD and asthma drugs is currently estimated to beGBP20 billion (source: visiongain). About Cystic Fibrosis Cystic fibrosis (CF) is an orphan disease that afflicts approximately 70,000people worldwide. The disease affects mostly the lungs, and also the pancreas,liver, and intestine. Difficulty breathing is the most serious symptom andresults from frequent lung infections. CF is caused by one of many differentmutations in the gene for the protein cystic fibrosis transmembrane conductanceregulator (CFTR). This protein is required to regulate the components of sweat,digestive fluids, and mucus. Healthy people have two working copies of the CFTRgene. Carriers have one working copy. People with CF have no working copy. CFtherefore has autosomal recessive inheritance. The underlying mechanism isabnormal transport of chloride and sodium across the epithelium, which is thecell layer that covers membranes over organs. This leads to thick, viscoussecretions. Individuals with cystic fibrosis can be diagnosed before birth bygenetic testing or by a sweat test in early childhood. The name cystic fibrosisrefers to the characteristic scarring (fibrosis) and cyst formation within thepancreas, first recognised in the 1930s. About The Cystic Fibrosis Trust The Cystic Fibrosis Trust is the only UK-wide charity making a daily differenceto the lives of people with cystic fibrosis, and those who care for them. Sinceits start in 1964 it has dedicated itself to promoting excellence in researchand clinical care, as well as providing practical support and advice to peoplewith cystic fibrosis and their families. The Trust believes that everyonewith cystic fibrosis deserves the best quality of life and real hope for thefuture, with access to high quality, specialist care. To achieve this goal itfunds research to better understand and treat cystic fibrosis, review standardsof cystic fibrosis care, and provide information and advice to the CFcommunity. About the Cystic Fibrosis Translational Research centre (CFTRc), McGillUniversity The CFTRc was established in the Faculty of Medicine at McGill University in2011 with $5.5M of infrastructure funding from the Canada Foundation forInnovation. It comprises 28 researchers at McGill and other institutions fromQuebec to British Columbia. It provides core facilities and other resources forCF research at the level of cells, tissues, and animal models, integratingphysiological studies with chemical and structural biology, and withbiochemical and cell biological studies of the mutant protein. It fostersinteractions with industry, advises members concerning technology transfer andcommercial agreements, and promotes preclinical and clinical studies ofpotential CF therapies. About Assoc Prof Scott Randell, Dept of Cell Biology and Physiology and theDept of Medicine, The University of North Carolina at Chapel Hill (UNC) Research in Dr. Randell's lab is focused on studies of lung cell biologyapplied towards overcoming clinical lung disease problems. Since 2001 he hasalso directed the UNC Cystic Fibrosis (CF) Center Tissue Procurement and CellCulture Core. This facility is a nationally and internationally recognizedresource, whose services are sought for collaboration, contract research, andtraining by academics, non-profit organizations, biotech and the pharmaceuticalindustry.