Bellerophon Announces Agreement With FDA on Regulatory Approval Pathway for INOpulse


Bellerophon Therapeutics, Inc. recently announced it has reached agreement with the US FDA on the regulatory approval pathway for INOpulse in patients with Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD).

In January 2019, the company reported positive results from Cohort 1 of its ongoing Phase 2b randomized, double-blind, placebo-controlled clinical study (iNO-PF) of INOpulse for the treatment of PH-ILD. Subjects on active treatment demonstrated a statistically significant improvement of 34% in moderate to vigorous physical activity (MVPA) as compared to subjects on placebo, as well as improvements in overall activity, oxygen saturation and additional functional measures. Based on these data and INOpulse’s safety profile, the FDA agreed with Bellerophon on the use of MVPA as the primary endpoint in the pivotal Phase 3 study, as measured by a medical wearable continuous activity monitor (actigraphy). In addition, the agency agreed with the company’s proposal that the Phase 2b study be amended to a Phase 2/3 trial. This agreement allows a seamless transition into Cohort 3 of iNO-PF, which will serve as the pivotal Phase 3 trial.

“Reaching this critical agreement with the FDA for INOpulse in PH-ILD represents a significant milestone for Bellerophon as it confirms the validity of MVPA as a clinically meaningful endpoint, enables us to build upon the robust data generated to date from iNO-PF and substantially shortens the regulatory pathway for our therapy in a disease with a serious unmet need,” said Fabian Tenenbaum, Chief Executive Officer of Bellerophon. “We appreciate the FDA’s support and their agreement on both the primary endpoint and the seamless pivotal Phase 2/3 design, creating the opportunity for INOpulse to become the first approved therapy in PH-ILD. We expect to complete Cohort 2 and initiate the pivotal Phase 3 trial in the second half of this year.”

“Compared to surrogate endpoints, such as 6-minute walk distance or patient reported outcomes, change in MVPA provides a direct and continuous measure of physical activity in PH-ILD patients, who have limited ability to perform even the most basic daily tasks,” said Steven D. Nathan, MD, FCCP, Medical Director of the Advanced Lung Disease and Lung Transplant Program at Inova Fairfax Hospital and Chair of Bellerophon’s Steering Committee. “INOpulse is a selective vasodilator that can improve both cardiopulmonary circulation and oxygenation, increasing the ability to perform physical activities. The improvements in MVPA seen to date in iNO-PF support INOpulse’s potential to address this unmet medical need. I am excited by the acceleration of this program into Phase 3 and look forward to the further evaluation of this promising therapy in the clinic.”

PH-ILD is a life threatening and debilitating disease that places a significant burden on daily life. Patients describe severe limits on their mobility and difficulty with moderate daily activities, such as walking and housework, with serious negative implications on their physical and emotional well-being. Actigraphy continuously measures real-world physical activity, allowing for an objective and clinically meaningful assessment of the therapeutic benefit. This is a well-established methodology utilized in a range of clinical applications, including in late-stage trials in cardiopulmonary indications, such as heart failure and Chronic Obstructive Pulmonary Disease.

Bellerophon Therapeutics is a clinical-stage biotherapeutics company focused on developing innovative therapies that address significant unmet medical needs in the treatment of cardiopulmonary diseases. The company is currently developing multiple product candidates under its INOpulse program, a proprietary pulsatile nitric oxide delivery system. For more information, visit www.bellerophon.com.

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