Verona Pharma PLC Positive headline data from RPL554 study

 
TIDMVRP 
 
Verona Pharma plc 
 
("Verona Pharma" or the "Company") 
 
       Positive headline data from RPL554 clinical study in COPD patients 
 
New suspension formulation is well tolerated; results signal marked improvement 
                               in lung function 
 
  RPL554 remains on track for entry into Phase IIb clinical trials in 2H 2016 
 
 
29 September 2015, Cardiff - Verona Pharma plc (AIM: VRP.L), the drug 
development company focused on first-in-class medicines to treat respiratory 
diseases, today announces encouraging positive headline data of the third and 
final part (part C) of a randomised, double blind, placebo controlled Single 
Ascending Dose (SAD) / Multiple Ascending Dose (MAD) clinical study in stable 
chronic obstructive pulmonary disease (COPD) patients. This study uses a new 
proprietary and commercially scalable nebulised suspension formulation of the 
Company's lead pipeline drug, RPL554.1,2 The primary objective of this part of 
the study was to show the safety and tolerability of the new drug formulation 
in stable COPD patients with moderate severity of disease.3 Measurement of lung 
function using FEV14 was included. Evaluation of the full data set is ongoing 
and will be presented in an appropriate scientific, peer-reviewed forum at a 
later date. 
 
Highlights: Part C of study in 32 stable COPD patients with moderate disease 
severity3 
 
  * Primary objective of study met: Drug formulation well tolerated at all dose 
    levels; No serious adverse events reported 
      + Adverse event profile similar to that seen with placebo 
      + Absence of gastro-intestinal or cardiovascular adverse events 
 
  * RPL554 caused pronounced improvement in lung function 
      + Mean peak FEV1 increase ranged from 199ml to 257ml versus placebo 
      + Extent of bronchodilation exceeds that seen in earlier studies with 
        original proof of concept formulation 
 
  * Data continue to support twice daily dosing regimen with RPL554 
 
  * Data consistent with that from earlier part A and B of study in healthy 
    volunteers 
      + Evaluation of the full data set from this trial is ongoing 
 
In the third part of the trial, nebulised RPL554, a novel dual PDE3/PDE4 
inhibitor, was administered twice daily using a new proprietary, commercially 
scalable, suspension formulation to stable COPD patients with moderate disease 
severity for up to five-and-a-half consecutive days at doses significantly in 
excess of the previously used active dose. Patients withheld their regular 
bronchodilator therapy for the duration of the treatment phase of the study. 
 
The study met its primary objective and all doses of RPL554 were found to be 
well tolerated. As with earlier parts of the trial, which were conducted in 
healthy volunteers, there were no reports of serious adverse events and the 
adverse event profile was similar to that seen with placebo. In particular, 
there was an absence of gastro-intestinal or cardiovascular adverse events. 
 
Lung function, as measured by peak FEV1, was increased in all dose groups and 
ranged between 199-257ml over placebo suggesting a clinically meaningful 
bronchodilator effect which will be confirmed in Phase IIb studies. In the 
highest dose there was a small increase in heart rate as might be expected from 
the pharmacology of the product. 
 
The new commercially scalable formulation of RPL554 results in a uniform 
suspension of RPL554 and was designed to provide improved tolerability, 
allowing higher doses of RPL554 to be inhaled, yielding an optimised 
bronchodilator effect than with the previous prototype, solution formulation. 
Additionally the new formulation offers potential for improvements in 
convenience and compliance. Data to date supports this profile, suggesting a 
twice daily dosing regimen and is consistent with a longer residence time of 
the drug in lung tissue and slower release into the blood than with the 
original formulation. In addition, the commercial viability of the new 
formulation is underlined by significantly improved stability compared to the 
previous formulation. 
 
Professor Dave Singh of the Medicines Evaluation Unit, University of 
Manchester, lead investigator on this study, commented:  "I am very encouraged 
by the headline results of this study using the new suspension formulation of 
RPL554. The marked improvement in lung function seen in this initial small 
study shows that this product has potential to be a meaningful addition to 
existing treatment options for COPD." 
 
Dr. Jan-Anders Karlsson, Chief Executive of Verona Pharma, said:  "We are 
excited by the robust and consistent results arising from our SAD/MAD study of 
RPL554 in both healthy volunteers and now, stable COPD patients with moderate 
disease severity. The data demonstrates that, as designed, the new commercially 
scalable, suspension formulation is well tolerated and has allowed us to extend 
the dose range and the duration of bronchodilation effect that can be produced 
in COPD patients. We will now fully analyse the data from this trial. While we 
need to discuss these results and confirm our further development plans for the 
drug with the appropriate regulatory authorities, we currently expect to begin 
Phase IIb studies in the second half of 2016." 
 
RPL554 is currently in development as a nebulised treatment for acute 
exacerbations in COPD patients in a hospital or home-care setting. The 
nebuliser bronchodilator market was worth about $1 billion in 2014 in the US.5 
RPL554 also has potential as a novel drug for the maintenance therapy of COPD, 
for patients with asthma and cystic fibrosis. 
 
Phase I and Phase II studies with RPL554 in the previous nebulised solution 
formulation were successfully conducted in over 100 subjects.6 Results 
collectively showed that the drug is a very potent bronchodilator with the 
ability to elicit a unique anti-inflammatory response. In these initial 
studies, patients treated with RPL554 had an adverse event profile which was 
similar to that in patients treated with placebo. The original nebulised 
formulation of the drug used in these studies was devised to provide 
proof-of-concept data, before the development of a new formulation suitable for 
commercial scale-up. 
 
                                    -Ends- 
 
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 
 
Julia Phillips / Simon Conway 
 
Notes to Editors 
 
About Verona Pharma plc 
 
Verona Pharma plc is a UK-based clinical stage biopharmaceutical company 
focused on the development of innovative prescription medicines to treat 
respiratory diseases with significant unmet medical needs, such as chronic 
obstructive pulmonary disease (COPD), asthma and cystic fibrosis. 
 
Verona Pharma's lead drug, RPL554, is a first-in-class drug currently in Phase 
II trials as a nebulised treatment for acute exacerbations of COPD in the 
hospital setting.  The drug is a dual phosphodiesterase (PDE) 3/4 inhibitor and 
therefore has both bronchodilator and anti-inflammatory effects, which are 
essential to the improvement of patients with COPD and asthma. 
 
Verona Pharma is also building a broader portfolio of RPL554-containing 
products to maximise its benefit to patients and its value.  This includes the 
very significant markets for COPD and asthma maintenance therapy.  The Company 
is also exploring the potential of the drug in different diseases, such as 
cystic fibrosis, where it is in pre-clinical testing and has recently received 
a Venture and Innovation Award from the Cystic Fibrosis Trust. 
 
About The Medicines Evaluation Unit ("MEU") 
 
The Medicines Evaluation Unit is one of the UK's leading contract research 
organisations, working in collaboration with the University Hospital of South 
Manchester. The MEU specialises in performing clinical trials (from Phase I 
through to IV) in respiratory/inflammatory medicine and related areas. The MEU 
has an outstanding reputation for performing high quality clinical research 
complying with UK Clinical Trials legislation and EU Directives and holds MHRA 
Phase I accreditation. 
 
About Chronic Obstructive Pulmonary Disease (COPD) 
 
Sixty-five million people worldwide suffer from moderate to severe COPD and the 
World Health Organisation (WHO) expects COPD to be the third leading cause of 
death globally by 2020.  It is the only major chronic disease with increasing 
mortality.  Currently available drugs are aimed at long-term maintenance 
therapy, with the market dominated by large pharma.  Despite the wide 
availability of these therapies, COPD patients suffer acute periods of 
worsening symptoms (exacerbations), which cause, in the US alone, some 1.5 
million A&E visits, 726,000 hospitalisations and 120,000 deaths per annum. 
 
References 
 
1 See www.clinicaltrials.gov - ClinicalTrials.gov Identifier:NCT02307162 
 
2 Initial data from Part A and B for this trial were reported in Company press 
releases dated 23 March 2015 and 8 June 2015 
 
3 GOLD Stage 2 with no requirement for bronchodilator reversibility: http:// 
www.goldcopd.org/uploads/users/files/GOLD_Report_2015_Sept2.pdf 
 
4 FEV1: forced expiratory volume in one second 
 
5 IMS Consulting Group market research 2014 
 
6 Franciosi, L.G., et al., Efficacy and safety of RPL554, a dual PDE3 and PDE4 
inhibitor, in healthy volunteers and in patients with asthma or chronic 
obstructive pulmonary disease: findings from four clinical trials. Lancet 
Respir Med, 2013. 1(9): p. 714-27 
 
 
 
END
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