Bellerophon Reaches Agreement with FDA on Study Design of Phase 2b Trial of INOpulse® for Treatment of Pulmonary Hypertension Associated with Chronic Obstructive Pulmonary Disease

WARREN, N.J., Bellerophon Therapeutics, Inc. (“Bellerophon” or the “Company”), a clinical-stage biotherapeutics company, today announced that, following the receipt of minutes from a recent meeting with the U.S. Food and Drug Administration (FDA), the Company has reached agreement with FDA on all key aspects of its planned Phase 2b study of INOpulse® for the treatment of Pulmonary Hypertension Associated with Chronic Obstructive Pulmonary Disease (PH-COPD).

The U.S. based Phase 2b study will be a double-blind, placebo-controlled, clinical trial in  approximately 90 PH-COPD patients assessing the benefit of pulsed inhaled nitric oxide (iNO) delivered by the INOpulse system. The primary end point will be six-minute walking distance (6MWD) and the study will also assess multiple secondary endpoints including right ventricular function.

“Reaching agreement with the FDA on the Phase 2b study design in PH-COPD represents an important achievement for our INOpulse development program,” said Fabian Tenenbaum, Chief Executive Officer of Bellerophon. “Based on the data generated to date and INOpulse’s dual mechanism of action, to provide targeted vasodilation as well as improve ventilation-perfusion matching, we believe INOpulse has the potential to be the first treatment approved for PH-COPD and we look forward to advancing our INOpulse therapy in this serious and unmet medical condition.”

The FDA meeting followed positive Phase 2a data, reported in September 2017 that showed statistically significant and clinically meaningful increases in 6MWD after both 2 and 4 weeks of treatment on INOpulse (+50.7m; p=0.04), as compared to baseline.  In addition, the trial results demonstrated a statistically significant increase (average 4.2%; p=0.03) in blood vessel volume and a statistically significant correlation in Ventilation-Vasodilation (p=0.01), indicating targeted delivery to the well-ventilated alveoli, as well as clinically meaningful decrease of 19.9% (p=0.02) in systolic pulmonary arterial pressure.  The therapy was well-tolerated with no related safety concerns.

COPD is a common, but potentially life-altering disease with a diagnosed prevalence greater than 12 million in the U.S.  Approximately 25-30% of COPD patients have associated pulmonary hypertension.  Although there are multiple therapies indicated for the treatment of COPD, there are no approved therapies for the treatment of PH associated with COPD.