Verona Pharma plc Verona Pharma Reports Positive Top-Line Data From Phase 2a Trial With Rpl554 In Cystic Fibrosis Patients

 
TIDMVRP TIDMVRP 
 
 
   RPL554 demonstrates a favorable pharmacokinetic and pharmacodynamic 
profile in cystic fibrosis patients 
 
 
 
   LONDON, March 02, 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 that top-line data from its 
Phase 2a trial with nebulized RPL554 demonstrate that the 
investigational therapy has a favorable pharmacokinetic (PK) and 
pharmacodynamic (PD) profile, with single doses achieving statistically 
significant (P<0.05) increases in average forced expiratory volume in 
one second (FEV(1) ) in patients with cystic fibrosis (CF). These data 
provide a solid foundation for further development of RPL554 for the 
treatment of CF. 
 
   RPL554 is a first-in-class, inhaled, dual inhibitor of the enzymes 
phosphodiesterase 3 and 4 designed to have anti-inflammatory as well as 
bronchodilator properties, and is currently in development for the 
maintenance treatment of COPD and for the treatment of CF. RPL554 has 
anti-inflammatory effects, and in pre-clinical studies, has been 
observed to stimulate the CF transmembrane conductance regulator (CFTR), 
a protein whose mutation results in dysfunctional ion channels in 
epithelial cells, leading to CF. Based on available data, RPL554 has the 
potential to enhance mucociliary clearance (reduce phlegm in the 
airways), reduce airway obstruction and inhibit inflammation. 
 
   "Achieving a six percent sustained improvement from baseline in FEV(1) 
after a single dose is very impressive in this group of CF patients," 
according to the trial's lead investigator, Professor Andres Floto, 
Wellcome Trust Senior Investigator, and Research Director of the 
Department of Medicine, University of Cambridge and Papworth Hospital, 
UK. 
 
   "These top-line findings demonstrate a favorable pharmacokinetic profile 
of inhaled RPL554 in CF patients, which is consistent with our earlier 
findings in COPD," said Jan-Anders Karlsson, PhD, CEO of Verona Pharma. 
"While we believe these results support further development of the drug 
in CF, we await the results of our ongoing Phase 2b trial for the 
maintenance treatment of COPD, for which we anticipate reporting 
top-line data early in the second quarter of 2018, to prioritize the 
future therapeutic focus of RPL554 development. 
 
   Trial details 
 
   This Phase 2a trial evaluated the PK and PD profile, and tolerability of 
RPL554 in 10 patients with CF, at 1.5 mg and 6.0 mg doses as compared to 
placebo. Patients displayed a range of CF genotype mutations in the 
CFTR. 
 
   Primary endpoint measure: 
 
 
   -- Pharmacokinetics of single nebulized doses of RPL554 in 10 patients with 
      CF:   -  The PK profile was consistent with that observed in patients 
      with COPD, although with lower peak serum levels of RPL554 in CF 
      patients; and   -  Serum half-life was dose-dependent; 7.5 to 10.1 hours 
      for 1.5 mg and 6 mg, respectively. 
 
 
   Secondary endpoint measures: 
 
 
   -- The drug also elicited a statistically significant increase in average 
      FEV1 in treated patients for 1.5 mg (all P<0.01) and 6 mg (all P<0.05) at 
      4, 6 and 8 hour time points; 
 
   -- The drug was well-tolerated in this patient group with an adverse event 
      profile consistent with other studies with RPL554. 
 
 
   The trial was conducted at Papworth Hospital, UK, one of the largest 
specialist cardiothoracic hospitals in Europe, and was supported by the 
UK Cystic Fibrosis Trust pursuant to the second Venture and Innovation 
Award received by Verona Pharma in October 2016. 
 
   "We're pleased to see these interesting results come through, and that 
our investment in this area continues to show promise. We look forward 
to seeing the next steps of developing RPL554 for the treatment of 
cystic fibrosis" said Janet Allen, Director of Strategy and Innovation 
at the Cystic Fibrosis Trust. 
 
   About Cystic Fibrosis 
 
   CF is the most common fatal inherited disease in the United States and 
Europe. CF causes impaired lung function and is commonly associated with 
repeat and persistent lung infections due to the inability to clear 
thickened mucus from the lung. This condition often results in frequent 
exacerbations and hospitalizations. There is no cure for CF and the 
median age of death for CF patients is around 40 years. CF is considered 
a rare, or orphan, disease by both the U.S. Food and Drug Administration 
and the European Medicines Agency. According to the Cystic Fibrosis 
Foundation, more than 30,000 people in the United States and more than 
70,000 people worldwide are living with CF and approximately 1,000 new 
cases of CF are diagnosed each year. CF patients require lifelong 
treatment with multiple daily medications, frequent hospitalizations and, 
ultimately, lung transplants in some end-stage patients. The quality of 
life for CF patients is compromised as a result of spending significant 
time on self-care every day and frequent outpatient doctor visits and 
hospitalizations. CF patients take an average of seven medications 
daily. 
 
   About Verona Pharma plc 
 
   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 
clinical trials, treatment with RPL554 has been observed to result in 
statistically significant improvements in lung function as compared to 
placebo, and has shown clinically meaningful and statistically 
significant improvements in lung function when administered in addition 
to frequently used short- and long-acting bronchodilators as compared to 
such bronchodilators administered as a single agent. 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 treatment 
potential for RPL554, the results from the Phase 2a trial of RPL554 
supporting further development of RPL554 in CF, the timing of data for 
the Phase 2b trial of RPL554 in COPD, the ability of RPL554 to address 
the urgent unmet medical need for therapeutics that treat in CF. 
 
   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 annual report on Form 20-F filed 
with the Securities and Exchange Commission ("SEC") on February 27, 
2018, 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 
 
   Jan-Anders Karlsson, Chief Executive Officer 
 
   Tel: +44 (0)20 3283 4200 
 
   info@veronapharma.com 
 
   Stifel Nicolaus Europe Limited (Nominated Adviser and UK Broker) 
 
   Stewart Wallace / Jonathan Senior / Ben Maddison 
 
   Tel: +44 (0) 20 7710 7600 
 
   SNELVeronaPharma@stifel.com 
 
   FTI Consulting (UK Media and Investor enquiries) 
 
   Simon Conway / Natalie Garland-Collins 
 
   Tel: +44 (0)20 3727 1000 
 
   veronapharma@fticonsulting.com 
 
   ICR, Inc. (US Media and Investor enquiries)

   James Heins 
 
   Tel: +1 203-682-8251 
 
   James.Heins@icrinc.com 
 
   Stephanie Carrington 
 
   Tel. +1 646-277-1282 
 
   Stephanie.Carrington@icrinc.com 
 
   This announcement is distributed by Nasdaq Corporate Solutions on behalf 
of Nasdaq Corporate Solutions clients. 
 
   The issuer of this announcement warrants that they are solely 
responsible for the content, accuracy and originality of the information 
contained therein. 
 
   Source: Verona Pharma plc via Globenewswire 
 
 
  http://www.veronapharma.com/

(END) Dow Jones Newswires

March 02, 2018 02:00 ET (07:00 GMT)

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