Verona Pharma plc Verona Pharma Reports Positive Efficacy And Safety Data With Single Dose Pmdi Formulation Of Ensifentrine I...

 
TIDMVRP 
 
   Statistically significant and clinically meaningful improvements in lung 
function 
 
   Ensifentrine has now demonstrated positive efficacy and safety in COPD 
patients via three widely used inhalation modes: nebulizer, DPI and pMDI 
 
 
   Initiation of multiple dose part of pMDI trial postponed due to the 
COVID-19 situation 
 
   LONDON, March 30, 2020 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM: VRP) 
(Nasdaq: VRNA) ("Verona Pharma"), a clinical-stage biopharmaceutical 
company focused on respiratory diseases, announces positive efficacy and 
safety data with a single dose of pressurized metered-dose inhaler 
("pMDI") formulation of ensifentrine in a Phase 2 clinical trial in 
patients with moderate to severe chronic obstructive pulmonary disease 
("COPD"). Results from the single dose part of the study (Part A) 
demonstrated a statistically significant and clinically meaningful 
increase in lung function as measured by forced expiratory volume in one 
second ("FEV(1) ")(1) compared to placebo. 
 
   In the first part of the trial, 40 patients with moderate to severe COPD 
were randomized to receive a single dose of one out of five dosage 
strengths of ensifentrine: 100 ug(2) , 300 ug, 1000 ug, 3000 ug, 6000 ug 
or placebo. In these patients, we observed the following: 
 
 
   -- Improvements in peak FEV1 corrected for placebo demonstrated a general 
      dose response (ranging from 47 mL to 391 mL, p<0.05 for doses 300 ug and 
      above). 
 
   -- Improvements in average FEV1 over 4 hours corrected for placebo also 
      showed a general dose response (average FEV1 AUC(0-4hr)3: ranging from 69 
      mL to 345 mL, p<0.05 for doses 300 ug and above). 
 
   -- Improvements in average FEV1 over 12 hours corrected for placebo also 
      showed a dose response and demonstrated durability of effect over the 
      dosing interval (average FEV1 AUC(0-12hr4): ranging from 48 mL to 222 mL, 
      p<0.05 for doses 3000 ug and above), supporting twice-daily dosing. 
 
   -- Ensifentrine pMDI formulation was well tolerated at each dose with an 
      adverse event profile similar to placebo. 
 
 
   The positive data support initiation of the second, multiple dose, part 
of the study (Part B), which will evaluate the pMDI formulation in this 
patient population over 7 days of twice daily treatment. Verona Pharma 
has decided to postpone the initiation of Part B due to concerns 
regarding the safety of trial subjects, caregivers and medical staff 
during the COVID-19 pandemic. We will continue to monitor this evolving 
situation and will provide an updated timeline for the start of Part B 
at a later date. 
 
   With these results and those observed in previous Phase 2 clinical 
trials, ensifentrine has demonstrated statistically significant and 
clinically meaningful improvements in lung function in COPD patients 
when delivered via any of the three widely used inhaled modes: nebulizer, 
dry powder inhaler ("DPI") and pMDI. 
 
   David Zaccardelli, Pharm. D., President and CEO of Verona Pharma, said: 
"Across all three inhaled formulations, ensifentrine has demonstrated 
statistically significant and clinically meaningful lung function 
improvements and duration of action, supporting twice-daily dosing and a 
safety profile similar to placebo. The results from the single dose part 
of this pMDI study are very encouraging and essentially consistent with 
data from Phase 2 clinical trials with nebulized and DPI formulations of 
ensifentrine." 
 
   "Following the public health advice associated with COVID-19, we have 
postponed enrollment of Part B of our pMDI Phase 2 trial in COPD. Our 
planned End-of-Phase 2 meeting with the FDA is scheduled in the second 
quarter of 2020, and the initiation of our Phase 3 trials of nebulized 
ensifentrine is planned for later this year." 
 
   An estimated 5.5 million people in the US use inhaled delivery, pMDI or 
DPI formulations delivered via handheld inhalers, for COPD maintenance 
treatment. Delivery of a pMDI formulation of ensifentrine may create new 
opportunities for using ensifentrine with existing inhaled medications. 
US sales of inhaled COPD maintenance medication were approximately $9 
billion in 2019. 
 
   (1) FEV(1) : Forced Expiratory Volume in one second, a standard measure 
of lung function 
 
   (2) ug: microgram, or mcg 
 
   (3) FEV(1) AUC(0-4hr) : Area Under the Curve 0-4 hours calculated using 
the trapezoidal rule, divided by the observation time (4 hours) to 
report in mL, a measure of the aggregate effect over 4 hours 
 
   (4) FEV(1) AUC(0-12hr) : Area Under the Curve 0-12 hours calculated 
using the trapezoidal rule, divided by the observation time (12 hours) 
to report in mL, a measure of the aggregate effect over 12 hours 
 
   THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF 
ARTICLE 7 OF REGULATION (EU) NO 596/2014. 
 
   About COPD 
 
   COPD is a progressive and life-threatening respiratory disease without a 
cure. The World Health Organization estimates that it will become the 
third leading cause of death worldwide by 2030. The condition damages 
the airways and the lungs, leading to debilitating breathlessness that 
has a devastating impact on performing basic daily activities such as 
getting out of bed, showering, eating and walking. In the United States 
alone, the total annual medical costs related to COPD are projected to 
rise to $49 billion in 2020. About 1.2 million US COPD patients on 
dual/triple inhaled therapy, long-acting beta-agonist (LABA)/long-acting 
muscarinic antagonist (LAMA) +/- inhaled corticosteroid (ICS) remain 
uncontrolled, experiencing symptoms that impair quality of life. These 
patients urgently need better treatments. 
 
   About Ensifentrine 
 
   Nebulized ensifentrine (RPL554) has shown statistically significant and 
clinically meaningful improvements in both lung function and COPD 
symptoms, including breathlessness, in Verona Pharma's prior Phase 2 
clinical studies in patients with moderate to severe COPD. In addition, 
nebulized ensifentrine showed further improved lung function and reduced 
lung volumes in patients taking standard short- and long-acting 
bronchodilator therapy, including maximum bronchodilator treatment with 
dual/triple therapy. Ensifentrine has been well tolerated in clinical 
trials involving more than 1300 people to date. 
 
   About Verona Pharma 
 
   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. If 
successfully developed and approved, Verona Pharma's product candidate, 
ensifentrine, has the potential to become the first therapy approved for 
the treatment of respiratory diseases that combines bronchodilator and 
anti-inflammatory activities in one compound. Verona Pharma is currently 
evaluating three formulations of ensifentrine for the treatment of COPD 
in Phase 2 clinical trials: nebulized, dry powder inhaler, and 
pressurized metered-dose inhaler. Ensifentrine also has potential 
applications in cystic fibrosis, asthma and other respiratory diseases. 
For more information, please visit www.veronapharma.com 
 
   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, the development of ensifentrine, the 
progress and timing of clinical trials, data and meetings with the FDA, 
the potential for ensifentrine to become the first therapy approved for 
the treatment of respiratory diseases to combine bronchodilator and 
anti-inflammatory activities in one compound, the potential for 
ensifentrine, if approved, to have a significant impact on the treatment 
of COPD, estimates of market size for COPD, and the potential 
application of ensifentrine for the treatment of cystic fibrosis, asthma 
and other respiratory diseases. 
 
   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 ensifentrine, 
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 ensifentrine, 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 ensifentrine, which could adversely affect our ability to develop 
or commercialize ensifentrine; potential delays in enrolling patients, 
which could adversely affect our research and development efforts and 
the completion of our clinical trials; we may not be successful in 
developing ensifentrine for multiple indications; our ability to obtain 
approval for and commercialize ensifentrine in multiple major 
pharmaceutical markets; misconduct or other improper activities by our 
employees, consultants, principal investigators, and third-party service 
providers; our ability to retain our key personnel and recruit 
additional qualified personnel, as well as the impact of our management 
team transition; material differences between our "top-line" data and 
final data; our reliance on third parties, including clinical research 
organizations, clinical investigators, manufacturers and suppliers, and 
the risks related to these parties' ability to successfully develop and 

commercialize ensifentrine; lawsuits related to patents covering 
ensifentrine and the potential for our patents to be found invalid or 
unenforceable; and our vulnerability to natural disasters, global 
economic factors and other unexpected events, including health epidemics 
or pandemics like the novel coronavirus (COVID-19). 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, 2020, 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] 
David Zaccardelli, Chief Executive Officer            [e-mail] 
Victoria Stewart, Director of Communications 
 
N+1 Singer                                            Tel: [phone] 
 (Nominated Adviser and UK Broker) 
Aubrey Powell / George Tzimas / Iqra Amin (Corporate 
 Finance) 
Tom Salvesen (Corporate Broking) 
 
Optimum Strategic Communications                      Tel: [phone] 
 (European Media and Investor Enquiries)               [e-mail] 
Mary Clark / Eva Haas / Hollie Vile 
 
Argot Partners                                        Tel: [phone] 
 (US Investor Enquiries)                               [e-mail] 
Stephanie Marks / Kimberly Minarovich / Michael 
Barron 
 
 
 
 
 
 

(END) Dow Jones Newswires

March 31, 2020 02:00 ET (06:00 GMT)

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