The U.S. Food and Drug Administration has signed off on the design of a Phase 2b trial of INOpulse, a device that delivers nitric oxide, as a treatment for patients with pulmonary hypertension associated with lung disease.
INOpulse’s developer, Bellerophon Therapeutics, said the sign-off came after a meeting with the FDA in June. In addition to the Phase 2b design, the meeting covered positive results of the completed Phase 2a iNO-PF trial (NCT01457781). That study involved INOpulse’s use in patients with PH associated with idiopathic pulmonary fibrosis.
INOpulse widens narrowed blood vessels, allowing more blood flow to the heart. The Phase 2a trial achieved its primary objective of widening vessels by increasing blood vessel volume 15.3 percent.
The trial also showed a significant association between the blood-vessel widening — or vasodilation — that INOpulse provides and improved ventilation, or movement of air in and out of the lungs.
In addition, INOpulse’s use led to a 14 percent reduction in systolic pulmonary arterial pressure — the pressure in the heart’s main artery. And it improved patients’ scores on a lung-function measure known as the six-minute walk distance.
The FDA also accepted Bellerophon’s investigational new drug application for INOpulse as a treatment for people at both low and high risk of developing PH associated with pulmonary fibrosis. This green light will accelerate the device’s regulatory approval process for that condition.
“We are very pleased to have concordance with the FDA on our iNO-PF Phase 2b trial for INOpulse in ILD (lung diseases) and to have a finalized plan to move forward with this important trial,” Fabian Tenenbaum, Bellerophon’s CEO, said in a press release.
Because INOpulse is designed to improve both vasodilation and ventilation, it may help those with “pulmonary fibrosing diseases where systemic vasodilators have proven to be ineffective,” Tenanbaum said. “The lack of approved therapies for PH associated with ILD represents a unique opportunity to develop a new therapy in this serious and significant unmet medical need.”
Bellerophon said NOpulse may even help those who do not show signs of PH while at rest.
The Phase 2b trial, planned for 2018, will involve 40 pulmonary fibrosis patients, half of whom will be at intermediate to high risk of developing PH.
Bellerophon said the trial design does not require right heart catheterization, an invasive procedure with potential challenges for patients.
Bellerophon has formed a Scientific Advisory Committee to support its INOpulse program. Its chair is Dr. Steven D. Nathan of Inova Fairfax Hospital, and members include Dr. Ganesh Raghu of the University of Washington, Dr. Kevin Flaherty of the University of Michigan, Dr. Marilyn K. Glassberg Ceste of the University of Miami, Dr. Jeffrey Swigris of National Jewish Health in Denver, and Dr. Lisa Lancaster of Vanderbilt University Medical Center.