VRP.L Regulatory News (VRP)

VERONA PHARMA PLC - RPL554 Phase 2a trial started

PR Newswire

London, June 11

Verona Pharma plc

("Verona Pharma" or the "Company")

RPL554 Phase 2a trial started; headline data expected Q1 2016

First asthma patients dosed with RPL554 in new proprietary nebulised formulation

11 June 2015, Cardiff – Verona Pharma plc (AIM: VRP.L), the drug development company focused on first-in-class medicines to treat respiratory diseases, today announces that the first patients have been dosed with RPL554 in a Phase 2a dose-finding trial in asthma patients using the Company’s new proprietary nebulised formulation. RPL554 is a novel inhaled PDE3/PDE4 inhibitor, currently in development as a nebulised treatment for acute exacerbations in chronic obstructive pulmonary disorder (COPD) patients in a hospital or home-care setting. The nebulised bronchodilator market was worth approximately $1 billion in 2014 in the US.1 

The study is being carried out in asthmatics as typically a dose response relationship to bronchodilators can be more accurately established in this group of patients, compared to COPD patients. In this trial, up to 30 patients with mild to moderate chronic asthma will each receive single doses of nebulised RPL554, from the very low dose to the highest dose previously tested in the Phase 1b single ascending and multiple ascending dose (SAD and MAD) studies of the same drug in healthy subjects. In this double-blind, placebo-controlled, crossover study each patient will also receive two different doses of nebulised salbutamol, a commonly used bronchodilator in these patients, and placebo. The primary objective of the trial is to establish the bronchodilator effect and duration of action of RPL554 in asthma patients. The study is being performed at Celerion (Belfast, Ireland) and Skane University Hospital (Lund, Sweden).

As reported on 23 March 2015 and 8 June 2015, Phase 1b results in SAD and MAD studies of nebulised RPL554 with this new formulation in healthy volunteers, have demonstrated that the drug is well tolerated in doses up to 16 times larger than those previously demonstrated to produce bronchodilation using the prior formulation. Given that this new formulation is better tolerated and higher doses can be administered, this latest trial is likely to provide useful data for further development work on RPL554 and also its potential as a future treatment for asthma sufferers.

Dr Jan-Anders Karlsson, the CEO of Verona Pharma, said:

“We have demonstrated in our previous studies in healthy volunteers with RPL554 in its new formulation that the drug is well tolerated at significantly higher doses than have previously been shown to produce bronchodilation. We have now begun examining the safety and bronchodilator responses to RPL554 in COPD, in the ongoing Phase 1b study, and in asthma patients using our new formulation.

Given that constriction of the airways is a key feature of different lung diseases, including asthma, a novel drug that produces potent bronchodilation but has a novel mechanism of action, would also be of potential value in these patients, especially when presenting with an acute exacerbation whilst on the maximum tolerated doses of their current asthma medicine. The data from this latest study should inform our further development plans for RPL554 when it reports initial data in Q1 2016.” 

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Notes to Editors

About Verona Pharma plc

Verona Pharma plc is a UK-based clinical stage biopharmaceutical company focused on the development of innovative prescription medicines to treat respiratory diseases with significant unmet medical needs, such as chronic obstructive pulmonary disease (COPD), asthma and cystic fibrosis.

Verona Pharma's lead drug, RPL554, is a first-in-class drug currently in Phase 2 trials as a nebulised treatment for acute exacerbations of COPD in the hospital setting. The drug is a dual phosphodiesterase (PDE) 3/4 inhibitor and therefore has both bronchodilator and anti-inflammatory effects, which are essential to the improvement of patients with COPD and asthma.

Verona Pharma is also building a broader portfolio of RPL554-containing products to maximise its benefit to patients and its value. This includes the very significant markets for COPD and asthma maintenance therapy. The Company is also exploring the potential of the drug in different diseases, such as cystic fibrosis, where it is in pre-clinical testing and has recently received a Venture and Innovation Award from the UK Cystic Fibrosis Trust.

About Lung and Allergy, Clinical Trial Unit (CTU), Skane University Hospital, Lund, Sweden

The Lung and Allergy clinical trial unit (http://www.akcsyd.se/forskning/provningsenheten) has been working mainly with early phase II and III studies in Asthma and or COPD for 10 years. The CTU is a part of an academic respiratory research network group run by Professor Leif Bjermer a recognised expert in this field. The scientific work is focused on disease mechanisms in asthma and COPD identifying and validating potential new treatment targets by means of invasive and non-invasive techniques, advanced histology, cell and molecular biology. The CTU is equipped with a complete respiratory physiology lab with a special focus on techniques that can address small airway pathology. Biomarkers, biogenetics and functional imaging is also part of the explorative profile. We also perform invasive studies with bronchoscopy, biopsies, brushing and lavage.

About Celerion

Celerion, a leader in early clinical research, delivers Applied Translational Medicine. Celerion applies expertise and experience to translating information gained in research discoveries, to knowledge of drug action and effect in humans to support early drug development decisions and the clinical pharmacology labeling of new medicines.

With over 40 years of experience and 600 global clinic beds (including 24 in-hospital), Celerion conducts and analyses First-in-Human, clinical Proof-of-Concept, Cardiovascular Safety Services (TQT, robust QT), ADME and NDA-enabling clinical pharmacology studies. Celerion provides expertise on modeling and simulation, study design, medical writing (protocols and reports), clinical data sciences, biostatistics, and PK/PD analysis as well as small and large molecule bioanalytical assays through clinical drug development. Regulatory, drug development and program management complement Celerion’s service offerings. For more information please visit http://celerion.com/.

About Asthma

Asthma remains one of the most common chronic diseases in the world and is characterised by recurrent breathing problems and symptoms such as breathlessness, wheezing, chest tightness, and coughing. In the U.S. asthma accounts for approx. 1.9 million annual emergency room visits and approx. 500,000 annual hospitalisations.

About Chronic Obstructive Pulmonary Disease (COPD)

Sixty-five million people worldwide suffer from moderate to severe COPD and the World Health Organisation (WHO) expects COPD to be the third leading cause of death globally by 2020. It is the only major chronic disease with increasing mortality. Currently available drugs are aimed at long-term maintenance therapy, with the market dominated by large pharma. Despite the wide availability of these therapies, COPD patients suffer acute periods of worsening symptoms (exacerbations), which cause, in the US alone, some 1.5 million A&E visits, 726,000 hospitalisations and 120,000 deaths per annum.

Bronchodilating therapy is considered to be the standard of care, and agents can be administered via handheld devices such as metered dose inhaler (MDI), dry powder inhaler (DPI) and by nebulisers. The nebulised bronchodilator market was worth about $1 billion in 2014 in the US.1 RPL554 is being developed by Verona Pharma as an add-on therapy to the “Standard of Care” with the objectives of providing rapid and pronounced improvement in lung function, reduced symptoms and both shortened duration of hospital stays and reduced re-admission rates 30 days after discharge from hospital. Studies to date on RPL554 have demonstrated that it has a strongly differentiated 3-way mode of action, being: (1) bronchodilation (the relaxation of smooth muscle in the airway); (2) anti-inflammatory effects on cells and (3) ion channel (CFTR) activation in epithelial cells, and thus the potential to increase mucociliary clearance in the airway.

References

1 IMS Consulting Group market research 2014