Verona Pharma plc Verona Pharma Reports Positive Interim Data With Ensifentrine Dry Powder Inhaler Formulation In First Of Tw...

 
TIDMVRP 
 
   Single dose of ensifentrine DPI formulation produced statistically 
significant and clinically meaningful dose-dependent bronchodilator 
response and was well tolerated at all doses 
 
   Data support initiation of part 2 of study to evaluate effect of 
ensifentrine DPI formulation over one week of treatment 
 
   First study to show efficacy of DPI formulation; Delivery via DPI could 
dramatically expand the clinical utility and commercial opportunity for 
ensifentrine in COPD and asthma 
 
   LONDON, March 04, 2019 (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 positive interim efficacy and safety 
data from part one of a two-part Phase 2 clinical trial of a dry powder 
inhaler ("DPI") formulation of ensifentrine in patients with 
moderate-to-severe chronic obstructive pulmonary disease ("COPD"). The 
positive data support initiation of the second part of the Phase 2 trial 
to evaluate the ensifentrine DPI formulation in patients with 
moderate-to-severe COPD over one week of twice-daily treatment. 
 
   In the first part of the trial, 37 patients with moderate-to-severe COPD 
received a single dose of one (out of five) dosage strengths of 
ensifentrine (150 ug, 500 ug, 1500 ug, 3000 ug, or 6000 ug) or placebo. 
Interim efficacy and safety data from this single dose study showed a 
statistically significant and clinically meaningful increase in lung 
function as measured by forced expiratory volume in one second ("FEV(1) 
"), compared to placebo. 
 
 
   -- Peak FEV1 increased from baseline in a dose-dependent manner (ranging 
      from 68 mL to 333 mL, p<0.05 for doses 1500 ug and above). 
 
   -- Average FEV1 0-4 hours and 0-12 hours also showed a dose response and 
      demonstrated durability of effect over the dosing interval (average FEV1 
      0-4h: ranging from 68 mL to 296 mL, p<0.05 for doses 500 ug and above; 
      average FEV1 0-12h: ranging from 54 mL to 254 mL, p<0.05 for doses 1500 
      ug and above, supporting twice-daily dosing). 
 
   -- Ensifentrine DPI formulation has been observed to be well tolerated at 
      each dose with an adverse event profile similar to placebo. 
 
 
   "The large bronchodilator response, 12-hour duration of action and good 
tolerability observed with the DPI ensifentrine formulation in the first 
portion of this study are very encouraging and we look forward to 
proceeding with the second part to evaluate treatment over a one-week 
period," said Jan-Anders Karlsson, PhD, CEO of Verona Pharma. "Positive 
data from this and future studies with inhaler formulations could 
dramatically expand the clinical utility and commercial opportunity for 
ensifentrine not only in the treatment of COPD, but potentially in other 
respiratory diseases such as asthma." 
 
   An estimated 5.5 million people in the US alone use inhalers for the 
maintenance treatment of COPD.(1) Delivery of a DPI formulation of 
ensifentrine creates exciting new opportunities for combining 
ensifentrine with existing therapeutics delivered by handheld inhalers. 
The value of the COPD maintenance market delivered via inhaler devices 
in the US alone was approximately $6 billion in 2017.(2) 
 
   The second part of this Phase 2 trial evaluating DPI ensifentrine is a 
randomized, double-blind, placebo-controlled, multiple dose crossover 
study, conducted at one site in the US. Patients will be randomized to 
one of four dose levels (150 ug, 500 ug, 1500 ug, or 3000 ug) or placebo, 
administered twice daily over one week. All patients will receive each 
dose level and placebo over five seven-day treatment periods. The 
primary endpoint of bronchodilator effect of repeat doses of 
ensifentrine delivered via DPI will be assessed in terms of peak FEV(1) 
. Secondary objectives of this part of the study include evaluating the 
safety, tolerability and bronchodilator profile of repeat doses of 
ensifentrine administered by DPI, as well as the pharmacokinetic 
profile. The full data from parts one and two are expected to be 
reported in the second half of 2019. 
 
   Ensifentrine, also known as RPL554, is an investigational first-in-class, 
inhaled, dual inhibitor of the enzymes phosphodiesterase 3 and 4 
designed to have bronchodilator as well as anti-inflammatory properties. 
Verona Pharma continues to advance the nebulized formulation of 
ensifentrine through Phase 2b clinical development and plans to initiate 
a Phase 2 clinical trial of a pressurised metered-dose inhaler ("pMDI") 
formulation of ensifentrine in patients with COPD in the second quarter 
of 2019. Ensifentrine is also in development for cystic fibrosis and 
asthma. 
 
   About Verona Pharma plc and ensifentrine 
 
   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, ensifentrine, is an investigational 
first-in-class, inhaled, dual inhibitor of the enzymes phosphodiesterase 
3 and 4 that is designed to act as both a bronchodilator and an 
anti-inflammatory agent in a single compound. Ensifentrine has been 
studied via the nebulized route of administration in 13 completed 
clinical trials involving more than 800 subjects. The nebulized 
formulation of ensifentrine has been observed to result in significantly 
improved lung function, including improved peak FEV(1) , reduced lung 
hyperinflation and faster onset-of-action when used alone or as an add 
on treatment to some of the most commonly used COPD treatments, 
including tiotropium (Spiriva(R) ), tiotropium/olodaterol fixed-dose 
combination, ipratropium, and albuterol. In addition, ensifentrine has 
shown anti-inflammatory effects in a standard challenge study with 
COPD-like inflammation in human subjects. Ensifentrine has been observed 
to be well tolerated in these trials. Verona Pharma is developing 
ensifentrine for the treatment of COPD, CF, and asthma. 
 
   About Chronic Obstructive Pulmonary Disease 
 
   Chronic obstructive pulmonary disease ("COPD") is a progressive and 
life-threatening respiratory disease for which there is no cure.(3) 
Although COPD is thought to be underdiagnosed, globally, around 384 
million people suffer from the disease.(4) 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.(4,5) 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.(4) 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.6 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 2020.7 
Approximately 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), worsening 
symptoms and exacerbations.(8,9) There is an urgent need for new 
treatment options with novel mechanisms of action that can be used by 
these patients in addition to current therapies. 
 
   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 that the interim data support 
selection of doses for the second part of the Phase 2 clinical trial, 
inhaler formulations of ensifentrine could expand the clinical utility 
and commercial opportunity for ensifentrine, the design of clinical 
trials, the timing of initiating clinical trials and receipt of results 
from clinical trials, the need for new treatment options for COPD, 
ensifentrine as a first-in-class inhibitor, and projections regarding 
the mortality rate of, and medical costs related to, COPD. 
 
   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; 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; 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 ensifentrine; and lawsuits related to patents covering
ensifentrine 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 Tel: +44 (0)20 3283 4200
Jan-Anders Karlsson, Chief Executive Officer info@veronapharma.com
Victoria Stewart, Director of Communications

Stifel Nicolaus Europe Limited (Nominated Adviser Tel: +44 (0) 20 7710 7600
and UK Broker)
Stewart Wallace / Jonathan Senior / Ben Maddison

FTI Consulting (UK Media and Investor enquiries) Tel: +44 (0)20 3727 1000
Simon Conway / Natalie Garland-Collins veronapharma@fticonsulting.com

ICR, Inc. (US Media and Investor enquiries)
Darcie Robinson Tel: +1 203-919-7905
Darcie.Robinson@icrinc.com
Stephanie Carrington Tel. +1 646-277-1282
Stephanie.Carrington@icrinc.com

_______

(1) Verona Pharma COPD Market Survey May 2018; Trends in COPD:
Morbidity and Mortality, American Lung Association, 2013, Make et al,
Intl. Journal of COPD, 2012

(2) IQVIA MIDAS Sales '17, excluding SABA, SAMA

(3) World Health Organization. Chronic Obstructive Pulmonary Disease.
https://www.globenewswire.com/Tracker?data=iDjKU6sxmJuRZbEfDfD_xlefyUtB85BM16A3QUdlvvBXoiyEj4OKK3bbaot1_Y-Z11q8eIj1VGzIPZdId6QNygvGlRjdVg4txeam2RqfIw8UW9pukviVQxtJg7nOweo8QkaTJeijn3xlDO6BmJzALwnW361l-jlZXd_PLmaS-P0=
http://www.who.int/mediacentre/factsheets/fs315/en/. Accessed September
2017.

(4) 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.

(5) World Health Organization. Burden of COPD.
https://www.globenewswire.com/Tracker?data=iDjKU6sxmJuRZbEfDfD_xpsIgXpB2aoas8nh87uSRGyuGuWkb5NE0jtQRZj1Tk0oYS_9G4qPghIpTGz4qrLfd9_QflcbcyK1B9A_1n792kSwqDtbfJ9AMI0RvDKf_i3r_rFdDxQeDSvvh0wFl46475X9zUXd8uGd9_gvq5wOSAI=
http://www.who.int/respiratory/copd/burden/en/. Accessed September 2017.

(6) COPD Foundations. Characteristics of COPD Patients Using United
States Emergency Care or Hospitalization.
https://www.globenewswire.com/Tracker?data=03PF_UqSrAa53-BivdSuvcebnnqEON3O4Asa3bZL1el5Phyi8sylXVB5W0vHrKGfilItIyuQNL2NiK1ll9BGQzDAUmWYVcwBliqSC8VlRck2Mx5sd-JIHqaJeWCvcgrCJEN1Kk5FnRW267DHgXANmBnsokg_AoDyHLIBnnS7pG8pEeIk2oBE9ux1seTtr4BN_3jAUMYlZMs89VnqCEM6Ta6gsgIDKlkReg1R9mM-Zr9ErnOD0C-mLrMH026cudopNfs1V1_fNG55Ul9X5cJltZ8iVT_ZEdOJxySLJCnXVbGBDJYHqYahrAFfNN6Odi3rKIB1AE4xz4WuQHBaeabhcoQkYpXHKfJWTZyEEnfhSu9dV8apcOUz6J74CE9Xtl3yWvd7LZEyBtZh5gSwBSyJueHHgEE5RkGGdIgxTfN1BYvMKHTi-agNUnOflcL1WtZudHuP568jgcdMX-9zsrsdLg==
https://journal.copdfoundation.org/jcopdf/id/1103/Characteristics-of-COPD-Patients-Using-United-States-Emergency-Care-or-Hospitalization.
Accessed September 2017.

(7) Centers for Disease Control. Increase Expected in Medical Costs for
COPD.
https://www.globenewswire.com/Tracker?data=03PF_UqSrAa53-BivdSuvW4BlG9V_feCyXJec2AWyMjppHdC9rzSNLRfAxoxNO7VXBVcGf4jK8gZZ1tcyPV9kltImOERcXJo3nTM2NlTxHCfAv4-4ZeZ8Wx5jZBBP-qIgPUY6KEZ4gy5pGv0n2HcPA==
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(8) 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.

(9) Vestbo J, et al., Single inhaler extrafine triple therapy versus
long-acting muscarinic antagonist therapy for chronic obstructive
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controlled trial, The Lancet, Vol 389, p. 1919-1929; May 13, 2017.

(END) Dow Jones Newswires

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