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 (FEV1) 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
FEV1 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 FibrosisCF 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 StatementsThis
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 plcJan-Anders Karlsson, Chief
Executive OfficerTel: +44 (0)20 3283 4200info@veronapharma.com
Stifel Nicolaus Europe Limited (Nominated
Adviser and UK Broker)Stewart Wallace / Jonathan Senior / Ben
MaddisonTel: +44 (0) 20 7710 7600SNELVeronaPharma@stifel.com
FTI Consulting (UK Media and Investor
enquiries)Simon Conway / Natalie Garland-CollinsTel: +44 (0)20 3727
1000veronapharma@fticonsulting.com
ICR, Inc. (US Media and Investor enquiries)James
HeinsTel: +1 203-682-8251James.Heins@icrinc.comStephanie
CarringtonTel. +1 646-277-1282Stephanie.Carrington@icrinc.com
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