Verona Pharma plc Verona Pharma Initiates Phase 2 Clinical Trial To Evaluate Dry Powder Inhaler Formulation Of Rpl554 For Mai...

 
TIDMVRP 
 
 
   Delivery via inhalers could dramatically expand clinical utility and 
commercial opportunity for RPL554 
 
   LONDON, Dec. 17, 2018 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM:VRP) 
(NASDAQ: VRNA) ("Verona Pharma"), a clinical stage biopharmaceutical 
company focused on developing and commercializing innovative therapies 
for respiratory diseases, announces today the initiation of a Phase 2 
clinical trial to evaluate the pharmacokinetic ("PK") profile, efficacy 
and safety of a dry powder inhaler ("DPI") formulation of RPL554 in 
patients with moderate to severe chronic obstructive pulmonary disease 
("COPD"). 
 
   RPL554 is a first-in-class, inhaled, dual inhibitor of the enzymes 
phosphodiesterase 3 and 4 designed to have bronchodilator as well as 
anti-inflammatory properties, and is currently in development for the 
maintenance treatment of COPD, cystic fibrosis ("CF") and asthma. 
 
   This randomized, double-blind, placebo-controlled, two-part study with 
the DPI formulation, being conducted at one site in the US, will enrol 
approximately 36 patients with COPD. The primary objective of the first 
part of the study is to evaluate the PK profile following a single dose 
of RPL554 when administered by DPI. Secondary objectives include 
evaluating the safety, tolerability and the bronchodilator effect of a 
single dose of RPL554 administered by DPI. The second part of the study, 
conducted as a multiple dose crossover study, will evaluate the 
bronchodilator effect of repeat doses of RPL554 administered by DPI in 
terms of peak forced expiratory volume in one second ("FEV(1) "). 
Secondary objectives of this part of the study include evaluating the 
safety, tolerability and bronchodilator effect of repeat doses of RPL554 
administered by DPI, as well as the PK profile, onset of action, and the 
amount of rescue medication use during treatment periods. Data from the 
single dose part of the study are expected during the first half of 
2019. 
 
   RPL554 has been studied via the nebulized route of administration in 12 
completed clinical trials involving more than 730 subjects. Verona 
Pharma has demonstrated in previous Phase 2 trials in patients with COPD 
that nebulized RPL554 significantly improves lung function, including 
improved peak FEV(1) , reduced lung hyperinflation, and faster 
onset-of-action, when added to some of the most commonly used COPD 
treatments, including tiotropium, ipratropium, and albuterol. The 
studies also showed that RPL554 is well tolerated. 
 
   "Positive results using inhaler formulations could dramatically expand 
the clinical utility and commercial opportunity for RPL554 in the 
maintenance treatment of COPD and potentially broaden its use for other 
respiratory indications, such as asthma," said Jan-Anders Karlsson, PhD, 
CEO of Verona Pharma. "With an estimated 5.4 million people using 
inhalers for the maintenance treatment of COPD in the US alone, and 
another one million patients using nebulized formulations, we believe it 
is important to understand how RPL554 works via these different delivery 
platforms in order to fully realize and leverage its clinical potential 
for patients with respiratory diseases.(1) We look forward to building 
upon the positive results that have been garnered to date utilizing the 
nebulized formulation of RPL554." 
 
   In addition, Verona Pharma is currently conducting a Phase 2 clinical 
trial to evaluate nebulized RPL554 as an add-on treatment to dual 
LAMA/LABA therapy and triple LAMA/LABA/ICS therapy and also plans to 
evaluate RPL554 as a metered-dose inhaler ("MDI") formulation as part of 
a comprehensive clinical program to fully demonstrate the clinical 
utility of RPL554 in improving the standard of care for COPD. 
 
   About Chronic Obstructive Pulmonary Disease 
 
   Chronic obstructive pulmonary disease ("COPD") is a progressive and 
life-threatening respiratory disease for which there is no cure.(2) 
Although COPD is thought to be underdiagnosed, globally, around 384 
million people suffer from the disease.(3) This number, according to the 
World Health Organization ("WHO"), is likely to increase in coming years, 
with estimates that COPD will become the third leading cause of death 
worldwide by 2030.(2,[4]) The condition damages the airways and the 
lungs, leading to persistent symptoms of breathlessness, impacting a 
person's daily life and their ability to perform simple activities such 
as walking a short flight of stairs or carrying a suitcase.(2) Many 
experience acute periods of worsening symptoms called 'exacerbations', 
often leading to emergency department visits or hospital admissions and 
are also associated with high mortality.(5) In the United States alone, 
the 2010 total annual medical costs related to COPD were estimated to be 
$32 billion and are projected to rise to $49 billion in 2020.(6) About 
30-40% of moderate to severe COPD patients on triple inhaled therapy 
(ICS/LAMA/LABA) remain uncontrolled and continue to experience airway 
obstruction (breathing difficulties), COPD symptoms and 
exacerbations.(7,8) There is an urgent need for drugs with novel 
mechanisms of action that can be used by these patients in addition to 
current therapies. 
 
   About Verona Pharma plc and RPL554 
 
   Verona Pharma is a clinical-stage biopharmaceutical company focused on 
developing and commercializing innovative therapies 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. In 
previous clinical trials, RPL554 has been observed to result in 
bronchodilator effects when used alone or as an add-on treatment to 
other COPD bronchodilators. It has shown clinically meaningful and 
statistically significant improvements in lung function when 
administered in addition to frequently used short- and long-acting 
bronchodilators, such as tiotropium (Spiriva(R) ), compared with such 
bronchodilators administered as a single agent. RPL554 improved FEV(1) 
over four weeks in patients with moderate-to-severe COPD when compared 
to placebo and improved COPD symptoms and Quality of Life in a Phase 2b 
multicenter European study performed in 403 patients. In addition, 
RPL554 has shown anti-inflammatory effects in a standard challenge study 
with COPD-like inflammation in human subjects. RPL554 has been well 
tolerated in these studies and has a favorable safety and tolerability 
profile, having been administered to more than 730 subjects in 12 
clinical trials. Verona Pharma is developing RPL554 for the treatment of 
chronic obstructive pulmonary disease ("COPD"), cystic fibrosis ("CF"), 
and potentially asthma. 
 
   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, 
including, but not limited to, statements regarding the design of the 
Phase 2 clinical trial of RPL554, the timing of availability of top-line 
data for the Phase 2 clinical trial, the importance of the Phase 2 
clinical trial to our development plans for RPL554, the potential of 
RPL554 as a promising first-in-class treatment option for COPD, and the 
value of the data and insights that may be gathered from the Phase 2b 
clinical trial. 
 
   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 
following: our limited operating history; our need for additional 
funding to complete development and commercialization of RPL554, which 
may not be available and which may force us to delay, reduce or 
eliminate our development or commercialization efforts; the reliance of 
our business on the success of RPL554, our only product candidate under 
development; economic, political, regulatory and other risks involved 
with international operations; the lengthy and expensive process of 
clinical drug development, which has an uncertain outcome; serious 
adverse, undesirable or unacceptable side effects associated with 
RPL554, which could adversely affect our ability to develop or 
commercialize RPL554; potential delays in enrolling patients, which 
could adversely affect our research and development efforts; we may not 
be successful in developing RPL554 for multiple indications; our ability 
to obtain approval for and commercialize RPL554 in multiple major 
pharmaceutical markets; misconduct or other improper activities by our 
employees, consultants, principal investigators, and third-party service 
providers; material differences between our "top-line" data and final 
data; our reliance on third parties, including clinical investigators, 
manufacturers and suppliers, and the risks related to these parties' 
ability to successfully develop and commercialize RPL554; and lawsuits 
related to patents covering RPL554 and the potential for our patents to 
be found invalid or unenforceable. These and other important factors 
under the caption "Risk Factors" in our final prospectus filed with the 
Securities and Exchange Commission ("SEC") on April 28, 2017 relating to 
our Registration Statement on Form F-1, and our other reports filed with 
the SEC, 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. 
 
   For further information, please contact: 
 
 
 
 
Verona Pharma plc                                  Tel: [phone] 
Jan-Anders Karlsson, Chief Executive Officer       [e-mail] 
 
Stifel Nicolaus Europe Limited (Nominated Adviser  Tel: [phone] 
 and UK Broker) 
Stewart Wallace / Jonathan Senior / Ben Maddison 
 
FTI Consulting (UK Media and Investor enquiries)   Tel: [phone] 
Simon Conway / Natalie Garland-Collins             [e-mail] 
 
ICR, Inc. (US Media and Investor enquiries) 
Darcie Robinson                                    Tel: [phone] 
                                                    [e-mail] 
Stephanie Carrington                               Tel. [phone] 
                                                    [e-mail] 
 
   (1) Q2 2017 Verona US COPD physician survey research, Trends in COPD: 
Morbidity and Mortality, American Lung Association, 2013, Make et al, 
Intl. Journal of COPD, 2012 
 
   (2) World Health Organization. Chronic Obstructive Pulmonary Disease. 
https://www.globenewswire.com/Tracker?data=vq1FtPYtscLYW1G7wJC9E_xbaHNrgGr9CefPfAFZ3qxFbAgOawCi-3hjpJJ1htWH7Ksufu1lZOtdOCmbGkLTj7NBsMGXvAgol9-rR-mUhEa9D3ABpWxSUCvNkDMHRpf-gk-HOrHctXuY-snelrzj6GuHUAo_9xDby-C2KyXpyoo= 
http://www.who.int/mediacentre/factsheets/fs315/en/. Accessed September 
2017. 
 
   (3) Adeloye D, Chua S, et al. Global and regional estimates of COPD 
prevalence: Systematic review and meta--analysis. J Glob Health 2015; 
5(2): 020415. 
 
   (4) World Health Organization. Burden of COPD. 
https://www.globenewswire.com/Tracker?data=vq1FtPYtscLYW1G7wJC9E3LYvvQ6eX_IgUqKliuB9HJfscRa87uN8m7jpX-PmLkeFWAmQIAZZ7SfErLQ9lHEsOi6lIwNRMLIigIx6_q1QoA_QzejXpMA7vGOhw9SC2EM_-G-hsBKb3b0Yjck1-d-CuNjA4OZ8J1Mb6N7M8c2bmk= 
http://www.who.int/respiratory/copd/burden/en/. Accessed September 2017. 
 
   (5) COPD Foundations. Characteristics of COPD Patients Using United 
States Emergency Care or Hospitalization. 
https://www.globenewswire.com/Tracker?data=DXemKVOFpCLvJo-RZH9GS2_RjUAEoVMNUecBQ6ibtKUWrkdKBFJiFbECx9K3UPnR7MuNYgPoba_sFjKMpfkACKoIUMzuT_ca-d4cW5BPjekbu4VXppKF6D-6NjwKX7Ens9HoQj8wwxMscGIDTwvetH7iWa0cuif6nN6NwqXBdm2EiN_WVITUxBw7-5WEi0SLt174WyTeYgRBAakDOIl1cAaihJDv5QOGfzwr6elwo9gKs2EcbGtbz05OuNspEFyUrmptHpLZQ1PCb1telg0CHVj6at96wq8XNRHJBaq83-oMg6TMK-TBQzOvCQs4YyQg1dygBmmmiu8eUS60kA852zbqjvnLQdQ-ntLzmhXIdvMJLeiRvquXQ8qf1_WQnxfxMckRj3TePNWnuVj5u7l5Gw== 
https://journal.copdfoundation.org/jcopdf/id/1103/Characteristics-of-COPD-Patients-Using-United-States-Emergency-Care-or-Hospitalization. 
Accessed September 2017. 
 
   (6) Centers for Disease Control. Increase Expected in Medical Costs for 
COPD. 
https://www.globenewswire.com/Tracker?data=DXemKVOFpCLvJo-RZH9GS52eqiC9InG4gZEFR0JXV_RW1qGpxi6jdqV4MXsZp1kXV5GDQQT31sDjs4ttSHUbdRTLgV-k2Xv-44oDXnSKd9mmJozF3-ExZ5fk73ssyVSgClUWIomHYGLvfC0N-z9UJg== 
https://www.cdc.gov/features/ds-copd-costs/. Accessed September 2017. 
 
   7 Mullerova H., et al., Characterization of COPD Patients Treated With 
Inhaled Triple Therapy Containing Inhaled Corticosteroid [ICS], 
Long-Acting Beta2-Agonists [LABA], and Long-Acting Muscarinic 
Antagonists [LAMA] in the UK, American Journal of Respiratory and 
Critical Care Medicine 2017;195:A4986 
 
   8 Vestbo J, et al., Single inhaler extrafine triple therapy versus 
long-acting muscarinic antagonist therapy for chronic obstructive 
pulmonary disease (TRINTY); a double-blind, parallel group, randomised 
controlled trial, The Lancet, Vol 389, p. 1919-1929; May 13, 2017. 
 
 
 
 
 
 

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December 17, 2018 02:00 ET (07:00 GMT)

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