Aires Pharmaceuticals inks deal with Duke University for Phase 2 study of AIR001 in HFpEF patients

This 100-patient study, known as the Inorganic Nitrite Delivery to Improve Exercise Capacity in HFpEF (INDIE-HFpEF) study, will be sponsored by Duke Clinical Research Institute (DCRI) as the Coordinating Center for the Heart Failure Clinical Research Network (HFN) and conducted at premier clinical centers in the United States that are part of the HFN.

HFpEF is a common form of heart failure that has not responded as well to previously tested treatments as heart failure with reduced ejection fraction.

AIR001, a sodium nitrite solution that is administered by way of intermittent inhalation via nebulization, was obtained by Mast Therapeutics in 2014 through the acquisition of then privately-held Aires Pharmaceuticals. The Company recently reported positive top-line results from a blinded and randomized Phase 2a clinical study of AIR001 in HFpEF patients conducted at Mayo Clinic.

The HFN was established by the NHLBI to expedite clinical research on treatments and strategies to improve the management of acute and chronic heart failure. The HFN’s work is supported by a grant awarded by the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH).

Under the agreement with the HFN Coordinating Center, as previously anticipated, the Company will provide test materials, nebulizers, and regulatory, technical and additional financial support.

The Chair of the HFN Executive Committee is Dr. Eugene Braunwald, Distinguished Hersey Professor of Medicine at Harvard Medical School. The collaborative scientific leadership for the study will include Dr. Barry Borlaug, the Principal Investigator, and other investigators from the HFN Regional Coordinating Centers. The HFN Coordinating Center will be led by Dr. Adrian Hernandez and Dr. Kevin Anstrom of the DCRI.

"We appreciate the NIH’s, NHLBI’s, and HFN’s recognition of the study of AIR001 in heart failure as an appropriate area of investigation," stated Brian M. Culley, Chief Executive Officer of Mast Therapeutics, Inc.

"This study will accelerate efforts to define the potential efficacy of AIR001 for patients who have heart failure with preserved ejection fraction. This is an area of significant unmet need. There are more than 1 million heart failure hospitalizations each year in the U.S., about half are patients with HFpEF, and, currently, there are no proven effective therapeutic agents available for this large patient population."

"The investigator initiated Investigational New Drug Application (IND) has been submitted and DCRI anticipates that the study will begin recruiting patients in the third quarter of this year," continued Mr. Culley. "We look forward to supporting DCRI and the HFN on this study."

About the INDIE-HFpEF Study

The Inorganic Nitrite Delivery to Improve Exercise Capacity in HFpEF (INDIE-HFpEF) study is a randomized, double-blind, placebo-controlled crossover study to assess the effect of AIR001 on peak exercise capacity as assessed by cardiopulmonary exercise testing (CPET). Approximately 100 patients with a diagnosis of HFpEF will be enrolled across approximately 20 clinical centers in the United States.

The primary endpoint will be the peak oxygen consumption (VO2) after four weeks of treatment with nebulized inhaled AIR001 or placebo as assessed by CPET performed at peak drug levels. Secondary objectives include evaluation of whether AIR001 improves (i) submaximal activity tolerance chronically, (ii) quality of life, (iii) chronic filling pressures as assessed by echocardiography and natriuretic peptide levels, and/or (iv) ventilator efficiency or submaximal exercise capacity at peak drug levels, and evaluation of the safety and tolerability of AIR001.