The 50 percent enrollment mark is a milestone because it will trigger an interim analysis of the nitric oxide therapy’s results. Under an agreement between the U.S. Food and Drug Administration and Bellerophon, an independent review board will conduct the analysis after half the trial participants complete 16 weeks of treatment. The company expects the analysis to be wrapped up in mid-2018.
Bellerophon said the review board, known as the Data Monitoring Committee, will look at preliminary findings on INOpulse’s safety and effectiveness to decide whether the trial should continue or be stopped. If the board decides it should continue, it will also consider whether the number of participants should be increased.
The company expects to announce the key results of the trial by the end of 2018.
“We are pleased with the progress achieved to date in this Phase 3 study,” Fabian Tenenbaum, the president and CEO of Bellerophon, said in press release. “We believe this speaks to the desire for PAH [pulmonary arterial hypertension] patients to seek more effective and tolerable treatment alternatives, as well as the enthusiasm and dedication of our investigators and clinical development team.
“We look forward to the interim analysis in mid-2018, and the continued advancement of our INOvation-1 trial over the remainder of the year,” Tenenbaum added.
INOpulse is an inhaled nitric oxide treatment. Bellerophon uses a portable device to deliver the gas in pulses through a tube in the nose.
Nitric oxide helps reduce blood pressure in the lungs by relaxing blood vessels. Bellerophon said its inhaled formulation should reduce the risk of side effects that can occur with other delivery methods.
INOpulse’s delivery system, which is about the size of a paperback book, is programmed to automatically adjust nitric oxide doses to a patient’s breathing pattern.
The INOvation-1 trial (NCT02725372) is evaluating INOpulse’s safety and effetiveness in PAH patients who are on long-term oxygen treatment. Researchers are conducting it at more than 100 sites in 17 countries. They are randomizing patients to receive either inhaled nitric oxide or a placebo.
Two of the trial’s objectives are to see whether the INOpulse system can prevent patients’ PAH from progressing and whether it can improve their physical capacity. Researchers are using a measure of exercise capability known as the six-minute walk distance test to assess patients’ physical capacity.
“I am excited to be participating in the INOvation-1 study, and pleased to see strong momentum in recruitment,” said Marion Delcroix, a professor at the KU Leuven university in Belgium. “INOpulse’s unique mechanism of action delivers a precise dose of pulsatile nitric oxide, a potent and well validated vasodilator [blood-vessel-widening agent], and has the potential to become an important new therapy in the management of PAH patients,” said Delcroix, a trial investigator.
Bellerophon expects the INOvation-1 trial and another study covering about 40 patients to generate findings that support it filing a New Drug Application for INOpulse with the FDA. The application would cover PAH patients on long-term oxygen.