Verona Pharma plc Data From Clinical And Pre-Clinical Trials Evaluating Verona Pharma's Rpl554 In Cystic Fibrosis Presented A...
October 26 2018 - 4:12AM
UK Regulatory
TIDMVRP
Pre-clinical findings show RPL554 stimulates rare class III and IV CFTR
mutants
Phase 2a results demonstrate RPL554 has favorable pharmacokinetic and
pharmacodynamic profile in cystic fibrosis patients
LONDON, Oct. 26, 2018 (GLOBE NEWSWIRE) -- Verona Pharma plc (AIM: VRP)
(Nasdaq: VRNA) ("Verona Pharma" or the "Company"), a clinical-stage
biopharmaceutical company focused on developing and commercializing
innovative therapies for respiratory diseases, announce the presentation
of positive data from pre-clinical and Phase 2a trials evaluating RPL554
as a potential treatment for cystic fibrosis ("CF") at the 2018 North
American Cystic Fibrosis Conference in Denver, CO. Data from the trials
show that RPL554 stimulates rare Class III and Class IV cystic fibrosis
transmembrane conductance regulator ("CFTR") mutants and that RPL554 has
a favorable pharmacokinetic ("PK") profile and increased forced
expiratory volume in one second ("FEV1") among patients with CF,
respectively. Top-line data from this Phase 2a trial were previously
reported by Verona Pharma on March 2, 2018
https://www.globenewswire.com/Tracker?data=vhfDfMySvp6Jew3jB8yyKCRtogdF82p6Mgabwf8NLcjPvCly-xFcQHDzEMcfRT-57BXMxONw4ZFSjXeQqFfE233qiJoJSJMUr1pwsIpeFqWj9ffcEp9Gea3XhQyodBeshUTPh5hVrah_4dvQBG22WOZFd-oZ-tSWVIyphgISXzbo1oLWQRRCrZM6ZmGZ4qw7VCpBqylVI9ZBYC2-IJOuIbybj0r5xm-MP1_05lyoRMSSJp3MyGs7lTz7pyVG-SuDc8VzY6McWHqNZvgompeAmw==
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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 chronic obstructive pulmonary disease ("COPD")
and for the treatment of CF. In pre-clinical studies, RPL554 has been
observed to stimulate the 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.
Pre-clinical data presented at the meeting show that RPL554
significantly enhanced activity in cells that expressed T338I and R334W
CFTR mutants, demonstrating that RPL554 alone positively regulates these
CFTR mutants, which is consistent with previous findings in cells
expressing the R117H mutation. In addition, when cells expressing S549R
and G551D mutants were pretreated with VX809 (Orkambi(R) ) for 24 hours,
RPL554 further enhanced CFTR-dependent activity, indicating that RPL554
can stimulate rare Class III and Class IV mutants when administered
alone or in combination with Orkambi(R) and has the potential to benefit
patients who possess a wide range of different CFTR mutations. Results
from the pre-clinical trial were presented by Mark Turner, PhD,
Postdoctoral Research Fellow, Cystic Fibrosis Translational Research
Centre (CFTRc), McGill University.
As part of the Phase 2a trial results presented, a single dose of
nebulized RPL554 administered to 10 patients with CF demonstrated a PK
profile that was consistent with that observed in patients with COPD in
previous trials. Furthermore, RPL554 demonstrated a statistically
significant increase in average FEV(1) in patients treated with 1.5 mg
(all p<0.01) and 6 mg (all p<0.05) at four, six and eight hour time
points. Results from the Phase 2a trial were authored by Odiri Eneje, MD,
Clinical Fellow, Department of Thoracic Medicine, Papworth Hospital NHS
Foundation Trust. This 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.
Abstracts included as part of the 2018 NACFC program have been published
in the September 2018 issue of the supplement to Pediatric Pulmonology
(Volume 53, Issue S2), which can be found online
https://www.globenewswire.com/Tracker?data=Mj_jhi3izX5Ix-KTgWpA3WpKMQUAnh3qPnCbaQ3DbqKRTFaq90gpcS8r5B09-O-_yRl80almfbeltqgFtjbEzl7HkdojIcVO17IUleQntImr5N1GD9ljhf4XMVejvJx2
here.
"The potential for RPL554 to stimulate numerous rare class III and IV
CFTR mutations is an important advancement for CF patients as it
highlights RPL554 as a novel therapeutic. Current FDA-approved therapies
are limited in their ability to address this need for patients," said
Dr. Turner.
"We are encouraged by the positive data being accrued from pre-clinical
and clinical studies with RPL554 in both CF and COPD," said Jan-Anders
Karlsson, PhD, CEO of Verona Pharma. "RPL554 has been well-tolerated and
demonstrated bronchodilator and anti-inflammatory activity in our
extensive COPD clinical trial program and we are encouraged that this
dual effect also shows promise in CF. There remains a high unmet need
among CF patients to address inflammation which is known to cause
disease progression and can lead to worsening of symptoms as well as
pulmonary exacerbations."
Verona Pharma has demonstrated in previous Phase 2 trials in patients
with COPD that RPL554 significantly improves lung function, including
improved peak lung function, reduced lung hyperinflation, and faster
onset-of-action, when added to some of the most commonly used COPD
treatments, including tiotropium, ipratropium, and albuterol. Verona
Pharma is currently conducting a Phase 2 clinical trial to evaluate
RPL554 as an add-on treatment to dual LAMA/LABA therapy and triple
LAMA/LABA/ICS therapy, as part of a comprehensive clinical program to
fully demonstrate the clinical utility of RPL554 in improving the
standard of care for COPD. These data will also support the planning of
the RPL554 Phase 3 COPD program.
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 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 treatment
potential for RPL554, the results of the Phase 2a trial of RPL554
supporting the further development of RPL554 in CF, 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 CF, and the value of the data and insights that may be gathered
from the Phase 2 clinical trial, including for the purpose of designing
pivotal Phase 3 trials.
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 and the
completion of our Phase 2 trial; we may not be successful in developing
RPL554 for multiple indications; our ability to obtain regulatory
approvals necessary to conduct later stage trials and to 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 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: +44 (0)20 3283 4200
Jan-Anders Karlsson, Chief Executive Officer info@veronapharma.com
Stifel Nicolaus Europe Limited (Nominated Adviser Tel: +44 (0) 20
and UK Broker) 7710 7600
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)
James Heins Tel: +1 203-682-8251
James.Heins@icrinc.com
Stephanie Carrington Tel. +1 646-277-1282
Stephanie.Carrington@icrinc.com
(END) Dow Jones Newswires
October 26, 2018 05:12 ET (09:12 GMT)
Copyright (c) 2018 Dow Jones & Company, Inc.
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