Verona Pharma Reports Positive Efficacy and Safety Data with Single Dose pMDI Formulation of Ensifen
Statistically significant and clinically meaningful improvements in lung function
Ensifentrine has now demonstrated positive efficacy and safety in COPD patients via three widely used inhalation modes: nebulizer, DPI and pMDI
Initiation of multiple dose part of pMDI trial postponed due to the COVID-19 situation
In the first part of the trial, 40 patients with moderate to severe COPD were randomized to receive a single dose of one out of five dosage strengths of ensifentrine: 100 µg2, 300 µg, 1000 µg, 3000 µg, 6000 µg or placebo. In these patients, we observed the following:
- Improvements in peak FEV1 corrected for placebo demonstrated a general dose response (ranging from 47 mL to 391 mL, p<0.05 for doses 300 µg and above).
- Improvements in average FEV1 over 4 hours corrected for placebo also showed a general dose response (average FEV1 AUC(0-4hr)3: ranging from 69 mL to 345 mL, p<0.05 for doses 300 µg and above).
- Improvements in average FEV1 over 12 hours corrected for placebo also showed a dose response and demonstrated durability of effect over the dosing interval (average FEV1 AUC(0-12hr4): ranging from 48 mL to 222 mL, p<0.05 for doses 3000 µg and above), supporting twice-daily dosing.
- Ensifentrine pMDI formulation was well tolerated at each dose with an adverse event profile similar to placebo.
The positive data support initiation of the second, multiple dose, part of the study (Part B), which will evaluate the pMDI formulation in this patient population over 7 days of twice daily treatment.
With these results and those observed in previous Phase 2 clinical trials, ensifentrine has demonstrated statistically significant and clinically meaningful improvements in lung function in COPD patients when delivered via any of the three widely used inhaled modes: nebulizer, dry powder inhaler (“DPI”) and pMDI.
“Following the public health advice associated with COVID-19, we have postponed enrollment of Part B of our pMDI Phase 2 trial in COPD. Our planned End-of-Phase 2 meeting with the FDA is scheduled in the second quarter of 2020, and the initiation of our Phase 3 trials of nebulized ensifentrine is planned for later this year.”
An estimated 5.5 million people in the US use inhaled delivery, pMDI or DPI formulations delivered via handheld inhalers, for COPD maintenance treatment. Delivery of a pMDI formulation of ensifentrine may create new opportunities for using ensifentrine with existing inhaled medications. US sales of inhaled COPD maintenance medication were approximately
1 FEV1: Forced Expiratory Volume in one second, a standard measure of lung function
2 µg: microgram, or mcg
3 FEV1 AUC(0-4hr): Area Under the Curve 0-4 hours calculated using the trapezoidal rule, divided by the observation time (4 hours) to report in mL, a measure of the aggregate effect over 4 hours
4 FEV1 AUC(0-12hr): Area Under the Curve 0-12 hours calculated using the trapezoidal rule, divided by the observation time (12 hours) to report in mL, a measure of the aggregate effect over 12 hours
THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF REGULATION (EU) NO 596/2014.
COPD is a progressive and life-threatening respiratory disease without a cure. The
Nebulized ensifentrine (RPL554) has shown statistically significant and clinically meaningful improvements in both lung function and COPD symptoms, including breathlessness, in Verona Pharma’s prior Phase 2 clinical studies in patients with moderate to severe COPD. In addition, nebulized ensifentrine showed further improved lung function and reduced lung volumes in patients taking standard short- and long-acting bronchodilator therapy, including maximum bronchodilator treatment with dual/triple therapy. Ensifentrine has been well tolerated in clinical trials involving more than 1300 people to date.
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, the development of ensifentrine, the progress and timing of clinical trials, data and meetings with the FDA, the potential for ensifentrine to become the first therapy approved for the treatment of respiratory diseases to combine bronchodilator and anti-inflammatory activities in one compound, the potential for ensifentrine, if approved, to have a significant impact on the treatment of COPD, estimates of market size for COPD, and the potential application of ensifentrine for the treatment of cystic fibrosis, asthma and other respiratory diseases.
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 and the completion of our clinical trials; 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; our ability to retain our key personnel and recruit additional qualified personnel, as well as the impact of our management team transition; material differences between our “top-line” data and final data; our reliance on third parties, including clinical research organizations, clinical investigators, manufacturers and suppliers, and the risks related to these parties’ ability to successfully develop and commercialize ensifentrine; lawsuits related to patents covering ensifentrine and the potential for our patents to be found invalid or unenforceable; and our vulnerability to natural disasters, global economic factors and other unexpected events, including health epidemics or pandemics like the novel coronavirus (COVID-19). These and other important factors under the caption “Risk Factors” in our Annual Report on Form 20-F filed with the
For further information, please contact:
|Tel: +44 (0)20 3283 4200|
|N+1 Singer |
(Nominated Adviser and
|Tel: +44 (0)20 3283 4200|
(European Media and Investor Enquiries)
|Tel: +44 (0)20 950 9144 |
|Mary Clark / |
(US Investor Enquiries)
|Tel: +1 212-600-1902|
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