Verona Pharma PLC (LON:VRP) (NASDAQ:VRNA) said it has been given the regulatory green light for two phase III studies of the nebulised formulation of its drug for chronic obstructive pulmonary disease (COPD).
It followed what is called an End of Phase II meeting with the US Food & Drug Administration (FDA) at which the company was provided “clarity” by officials on how it should proceed with the trials.
Specifically, the FDA provided its input on the dosing, primary and secondary endpoints, patient population and study design.
The company said it was accelerating preparations for the phase III clinical assessment of its inhaled treatment, called ensifentrine, which will start later this year.
"We continue to be very encouraged by the phase II results that have demonstrated ensifentrine’s effects on lung function, COPD symptoms and quality of life as well as its favourable safety profile,” Verona chief executive, David Zaccardelli said in a statement
“We look forward to building on this positive data to support the potential submission of a new drug application for ensifentrine for the maintenance treatment of chronic obstructive pulmonary disease.”
The ENHANCE programme will consist of two randomised, double-blind, placebo-controlled studies.
They will evaluate the efficacy and safety of twice-daily nebulised ensifentrine as a stand-alone therapy and as an add-on to the standard of care treatment.
The two study designs are essentially identical over 24 weeks, but ENHANCE-1 will also evaluate longer-term safety.
The nuts and bolts are the phase III programme are as follows:
- Each study will enrol around 800 moderate to severe COPD sufferers in the US and Europe
- Patients will receive 3mg of ensifentrine or placebo twice daily via a nebuliser
- The primary endpoint will be a clinically significant improvement in lung function after 12 weeks
- Secondary goals for the treatment include health-related quality of life
- Safety will be assessed over 24 weeks in both studies and over 48 weeks in 400 patients in the ENHANCE-1 part of the phase III programme