Bellerophon Announces FDA Agreement on Phase 2b Study Design

Bellerophon Therapeutics, Inc. recently announced agreement with the US FDA on the Phase 2 study design for INOpulse in pulmonary hypertension associated with Interstitial Lung Disease (ILD).

The company met with the FDA in June 2017 to present positive results from its recently completed Phase 2a study in idiopathic pulmonary fibrosis (IPF), and to review clinical plans for its Phase 2b trial, entitled iNO-PF, in IPF as well as other pulmonary fibrosing diseases within ILD. Subsequently, the agency has accepted the company’s proposed Phase 2b study design as well as an Investigational New Drug (IND) application to assess the effect of INOpulse on patients at both low- and high-risk for pulmonary hypertension associated with pulmonary fibrosis.

The FDA recognized the dual mode of action of vasodilation and ventilation/perfusion matching of pulsed iNO therapy, which the company believes can provide a clinically important benefit to a wide range of patients, including those that may not exhibit signs of pulmonary hypertension at rest.

“We are very pleased to have concordance with the FDA on our iNO-PF Phase 2b trial for INOpulse in ILD and to have a finalized plan to move forward with this important trial,” said Fabian Tenenbaum, Chief Executive Officer of Bellerophon Therapeutics. “The proprietary targeted delivery and the dual mode of action of INOpulse may allow it to be used in pulmonary fibrosing diseases where systemic vasodilators have proven to be ineffective. The lack of approved therapies for pulmonary hypertension associated with interstitial lung diseases represents a unique opportunity to develop a new therapy in this serious and significant unmet medical need.”

The iNO-PF Phase 2b study design is based on the results of the prior Phase 2a study for INOpulse in the treatment of Pulmonary Hypertension associated with Idiopathic Pulmonary Fibrosis (PH-IPF), presented at the American Thoracic Society (ATS) International Conference on May 21, 2017. This Phase 2a study met its primary endpoint, showing an average of 15.3% increase in blood vessel volume (p<0.001). There was a significant association between ventilation and vasodilation, demonstrating the ability of INOpulse to provide targeted selective delivery to well ventilated sections of the lung. The study also showed consistent benefit in hemodynamics and exercise capacity, with a clinically meaningful reduction of 14% in systolic pulmonary arterial pressure (sPAP) and an average improvement of 75 meters in 6-Minute Walk Distance.

The iNO-PF trial is planned for 2018 and is designed to recruit 40 subjects diagnosed with pulmonary fibrosis, half of which are at intermediate to high risk of pulmonary hypertension as determined by echocardiography. Importantly, the FDA has agreed to a Phase 2b design that eliminates the need for right heart catheterization, an invasive procedure that can present significant challenges for potential study participants.

To support the progress of its program for INOpulse in the treatment of ILDs, Bellerophon has formed a Scientific Advisory Committee chaired by Dr. Steven D. Nathan (Inova Fairfax Hospital) as well as Dr. Ganesh Raghu (University of Washington), Dr. Kevin Flaherty (University of Michigan), Dr. Marilyn K. Glassberg Ceste (University of Miami), Dr. Jeffrey Swigris (National Jewish Health – Denver) and Dr. Lisa Lancaster (Vanderbilt University Medical Center).

Bellerophon Therapeutics is a clinical-stage biotherapeutics company focused on developing innovative therapies at the intersection of drugs and devices that address significant unmet medical needs in the treatment of cardiopulmonary diseases. The company is currently developing three product candidates under its INOpulse program, a proprietary pulsatile nitric oxide delivery system. The first is for the treatment of pulmonary arterial hypertension (PAH), for which the company has commenced Phase 3 clinical trials in 2016. The second is for the treatment of pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD) and the third candidate is for the treatment of pulmonary hypertension associated with Interstitial Lung Disease (PH-ILD), both of which are in Phase 2 development. For more information, visit www.bellerophon.com.