Bellerophon, FDA Agree on Design of Phase 3 INOPulse Clinical Trial

Bellerophon Therapeutics has reached an agreement with the U.S. Food and Drug Administration (FDA) setting the design of an upcoming, pivotal Phase 3 clinical trial testing INOpulse, an investigational therapy, in people with pulmonary fibrosis (PF) who are at risk of pulmonary hypertension (PH).

PH-PF is characterized by a narrowing of the pulmonary arteries, the blood vessels that carry blood from the heart to the lungs. This raises the pressure within these arteries, which ultimately results in a decrease in cardiac output, or the amount of blood the heart is able to pump.

INOpulse is a device that delivers inhaled nitric oxide (iNO), which acts as a vasodilator — meaning it causes blood vessels to widen, reducing blood pressure.

This investigational treatment was previously tested in the Phase 2/3 iNO-PF clinical trial (NCT03267108). That trial assessed the effects of different dosages of INOpulse in three groups of people diagnosed with PF and at risk of developing PH.

Group 2 in that trial enrolled 44 participants who were randomly selected to receive 45 mcg/kg ideal body weight/per hour (mcg/kg IBW/hr) of INOpulse or a placebo for four months.

The results showed that patients treated with INOpulse had a significant increase in moderate-to-vigorous physical activity (MVPA), by 14 minutes a day. That corresponded to an approximately 20% improvement compared with the placebo group. MVPA is a parameter that include various tasks such as walking, climbing stairs, and doing yard work.

Significant improvements also were seen in self-reported quality of life and shortness of breath, as assessed through validated questionnaires.

Now, in consultation with the FDA, Bellerophon has finalized key elements of its upcoming Phase 3 clinical trial — including defining the population of people with PF who are at risk of PH. As in the previous trial, MVPA will be used as the primary endpoint, and 45 mcg/kg IBW/hr will be used as the therapeutic dose.

“We are very pleased with the alignment reached with the FDA on the design of our pivotal Phase 3 trial, which allows us to move confidently towards the initiation of this important study,” Fabian Tenenbaum, the CEO of Bellerophon, said in a press release.

“There is a pressing need to develop a safe and effective treatment for patients with PH-PF,” Tenenbaum added. “INOpulse, with its targeted pulmonary vasodilation, would potentially become the first therapy to treat a broad PH-PF population that includes patients at low, intermediate and high risk of pulmonary hypertension. We look forward to initiating our pivotal Phase 3 study in PH-PF shortly.”