Verona Pharma plc (AIM: VRP) (NASDAQ: VRNA) announces that it has received written comments from the U.S. Food and Drug Administration (FDA) in response to its End-of-Phase 2 briefing package for nebulized ensifentrine as a maintenance treatment for chronic obstructive pulmonary disease ("COPD"). The response supports Verona Pharma progressing with its planned Phase 3 clinical program, which is expected to start later in 2020. It is planned to be called the ENHANCE (Ensifentrine as a Novel inHAled Nebulized COPD thErapy) program.
The FDA's comments follow its review of the End-of-Phase 2 briefing package that included data from 16 clinical trials involving over 1,300 subjects as well as supportive nonclinical and product development data. Verona Pharma has obtained clarity from the FDA on important features of the pivotal Phase 3 clinical program to support a New Drug Application (NDA) including: dose, primary and secondary endpoints, patient population and program design. Based on the FDA response, Verona Pharma is accelerating preparations for the Phase 3 clinical program to start later in 2020.
The two randomized, double-blind, placebo-controlled studies (ENHANCE-1 and ENHANCE-2) will evaluate the efficacy and safety of twice daily nebulized ensifentrine as monotherapy and as an add-on to standard of care treatment with a single bronchodilator, either a LAMA, long acting muscarinic antagonist, or a LABA, long acting beta-agonist. The two study designs are essentially identical over 24 weeks but ENHANCE-1 will also evaluate longer-term safety.
Patient Population: Each of the studies will be expected to enroll approximately 800 moderate to severe, symptomatic COPD patients at sites primarily in the US and Europe.
Dose/Duration: Patients will receive 3 mg nebulized ensifentrine or nebulized placebo twice daily.
Primary endpoint: Improvement in lung function as measured by forced expiratory volume in one second (FEV1) over 12 hours with ensifentrine after 12 weeks of treatment.
Key secondary endpoints: Peak and trough FEV1 as well as COPD symptoms and health-related quality of life will be assessed through 24 weeks via the validated patient reported outcome tools, SGRQ and E-RS: COPD.
Safety: Assessed over 24 weeks in both studies and over 48 weeks in approximately 400 patients in ENHANCE-1.
"We are very pleased with the FDA's response to our End-of-Phase 2 briefing package. Subject to securing additional funding, we look forward to starting our pivotal ENHANCE program later in 2020," said David Zaccardelli, Pharm. D., President and Chief Executive Officer. "We continue to be very encouraged by the Phase 2 results that have demonstrated ensifentrine's effects on lung function, COPD symptoms and quality of life as well as its favorable safety profile. We look forward to building on this positive data to support the potential submission of a NDA for ensifentrine for the maintenance treatment of COPD."