Phase 3 Study of INOpulse Device as a PAH Treatment Enrolls First Patient

Bellerophon Therapeutics, Inc., announced the enrollment of the first patient with pulmonary arterial hypertension (PAH) in its Phase 3 INOvation-1 clinical study, assessing the efficacy and safety of the next-generation INOpulse delivery system.

The portable device is designed to continuously administer inhaled nitric oxide (iNO) to ambulatory patients using a carefully controlled, fixed microgram per kilogram of body weight per hour (mcg/kg IBW/hr) dose that is independent of changes in respiration rate during exercise or sleep.

In total, the company plans to enroll 188 PAH patients in 17 countries across North America and Europe. The first patient is being treated in Arizona.

Bellerophon also announced the enrollment of a first patient in a Phase 2 trial assessing the efficacy of the system in people with pulmonary hypertension associated with idiopathic pulmonary fibrosis (PH-IPF).

“The initiation of enrollment in INOvation-1, Bellerophon’s first Phase 3 trial in PAH, marks an important milestone for the company and offers a new potential treatment to patients who continue to suffer poor outcomes,” Jonathan Peacock, chairman and chief executive officer of Bellerophon Therapeutics, said in a press release. “Additionally, with the initiation of the clinical trial in IPF and a planned clinical trial in pulmonary hypertension associated with COPD [chronic obstructive pulmonary disease], we are positioned to gain additional insight in 2016 for our future development programs for INOpulse.”

PAH is the lead indication for the Phase 3 INOpulse program, for which the U.S. Food and Drug Administration (FDA) has issued a Special Protocol Assessment, meaning that the trial’s application was accepted even though its design and endpoints are not fully defined. A similar agreement was reached with European Medicines Agency (EMA) through its Scientific Advice Working Party (SAWP) process.

The planned clinical program will be composed of two confirmatory studies in about 450 patients, with one trial having two treatment arms (iNO 75 and placebo) and the other three treatment arms (iNO 75, iNO 50, and placebo). The primary outcome for both is changes in the 6 minute walk distance (MWD) test (an exercise/function capacity test) after 16 weeks of treatment. Time to Clinical Worsening (TTCW) is the secondary endpoint.

These Phase 3 trials will use the second-generation INOpulse device, which is lighter and smaller, with improved user interface and better breath detection technology than the device used in earlier trials, the company said.

In February, Bellerophon reported positive data from the Phase 2 extension study of a combination INOpulse and iNO treatment for PAH, establishing clinical benefit for patients on long-term oxygen and whose disease was worsening despite taking at least one PAH therapy.

The company will continue its Phase 2 evaluation of patients with PH-IPF, and PH associated with chronic obstructive pulmonary disease (PH-COPD). Data from these studies are expected by the end of the year.