Data from Clinical and Pre-Clinical Trials Evaluating Verona Pharma’s RPL554 in Cystic Fibrosis Presented at North American Cystic Fibrosis Conference
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
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®) 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® 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
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 FEV1 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,
Abstracts included as part of the 2018 NACFC program have been published in the
“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
“We are encouraged by the positive data being accrued from pre-clinical and clinical studies with RPL554 in both CF and COPD,” said
About Cystic Fibrosis
CF is the most common fatal inherited disease in
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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
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Verona Pharma plc | Tel: +44 (0)20 3283 4200 |
Jan-Anders Karlsson, Chief Executive Officer | info@veronapharma.com |
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Stewart Wallace / Jonathan Senior / Ben Maddison | |
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Simon Conway / Natalie Garland-Collins | veronapharma@fticonsulting.com |
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James Heins | Tel: +1 203-682-8251 James.Heins@icrinc.com |
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Source: Verona Pharma plc