Verona Pharma’s investigational agent RPL554 will be named ensifentrine, the company announced.
The “-fentrine” stem in the International Nonproprietary Names (INN) Programme indicates recognition from the World Health Organization (WHO) that RPL554 inhibits multiple phosphodiesterases.
Ensifentrine is being developed for the maintenance treatment of chronic obstructive pulmonary disease (COPD) that is not adequately managed with standard care therapies. It works as both a bronchodilator (to widen the airways) and an anti-inflammatory agent by inhibiting the phosphodiesterase 3 and 4 (PDE3 and PDE4) enzymes linked to COPD development.
“We are pleased to receive approval from the WHO for the use of ensifentrine, which to our knowledge is the only molecule with an International Nonproprietary Name assignment using the ‘–fentrine’ stem currently in clinical development,” Jan-Anders Karlsson, PhD, CEO of Verona Pharma, said in a press release. “We believe that this designation underlines our drug candidate’s position as a novel compound with a dual bronchodilator and anti-inflammatory mechanism of action.”
Results from Phase 2 clinical trials (NCT02542254 and NCT03028142) in patients with COPD demonstrated that ensifentrine can be an effective bronchodilator when used alone or as add-on treatment to standard COPD therapies.
Verona Pharma is recruiting COPD patients for two additional Phase 2 clinical trials. One (NCT03673670) will evaluate a nebulized formulation of ensifentrine as an add-on treatment with Stiolto Respimat (a combination therapy of tiotropium bromide and olodaterol, marketed by Boehringer Ingelheim) and inhaled corticosteroid. The second study will assess the safety and efficacy of a dry powder inhaler formulation of ensifentrine as maintenance treatment for COPD.
The company also plans to evaluate ensifentrine when used in a metered-dose inhaler formulation, part of a comprehensive clinical program to fully demonstrate the clinical utility of ensifentrine for COPD treatment.
Results of these studies will be used to support the progression of ensifentrine into a Phase 3 COPD clinical program.
“Ensifentrine is the only dual bronchodilator and anti-inflammatory drug candidate currently in development that I have come across in my research. This could be significant for patients and healthcare providers alike as there have been no new bronchodilator treatment classes over the last four decades,” said Gerard Criner, MD, chair and professor of Thoracic Medicine and Surgery at Lewis Katz School of Medicine, Temple University.
Ensifentrine is also being developed as a potential treatment for cystic fibrosis and asthma.