McGowan Institute for Regenerative Medicine affiliated faculty member Marc Simon, MD, MS, FACC, Associate Professor of Medicine, Bioengineering, and Clinical Translational Science, and Director, Heart Failure Research / Clinical Hemodynamics Core Facility, at the Pittsburgh Heart, Lung, and Blood Vascular Medicine Institute at the University of Pittsburgh, is the lead investigator on a 50-patient, open-label Phase 2 study in patients with pulmonary hypertension (PH) from multiple different etiologies. Dr. Simon recently presented the study’s results in an invited lecture at the 4th Annual Drug Discovery and Development Symposium for Pulmonary Hypertension. The study’s product, Aironite, is in development by Savara, Inc., a clinical-stage specialty pharmaceutical company focused on the development and commercialization of novel therapies for the treatment of serious or life-threatening rare respiratory diseases.
In the 41 patients enrolled to date in the study, administration of Aironite significantly improved multiple hemodynamic measures, with most pronounced improvements in patients with pulmonary hypertension due to heart failure with preserved ejection fraction (PH-HFpEF). In the 10 PH-HFpEF patients analyzed, Aironite administration resulted in significant overall decreases in right atrial pressure, systolic and diastolic right ventricular pressure, systolic and diastolic pulmonary artery pressure, and pulmonary capillary wedge pressure. Of note, pulmonary capillary wedge pressure decreased by 7.5 mmHg (95% CI –9.0 to –6.0; baseline median value 18mmHg; p < 0.05) and mean pulmonary artery pressure decreased by 7.9 mmHg (95% CI –9.4 to –6.3; baseline median value 34 mmHg; p < 0.05). Pulmonary artery compliance (Cpa) was improved by 35% (+0.97 ml/mmHg, 95% CI +0.25 to +1.68; p < 0.01). Aironite was generally well-tolerated and no significant safety concerns were identified, supporting the primary safety outcome of the study.
“These additional results build upon prior interim data published last year in the Journal of Clinical Investigation, and demonstrate that Aironite can significantly improve cardiopulmonary hemodynamics in HFpEF as well as Group 3 PH patients, both clinical conditions which are inadequately treated by currently approved medicinal treatments,” stated Taneli Jouhikainen, MD, PhD, Chief Operating Officer of Savara. “If the observed short-term improvements are translated into clinically meaningful functional improvements in our ongoing placebo-controlled studies in HFpEF patients, we believe the product will have exciting potential to be advanced towards Phase 3 studies and hopefully an eventual NDA filing.”
Abstract (Acute hemodynamic effects of inhaled sodium nitrite in pulmonary hypertension associated with heart failure with preserved ejection fraction. Marc A. Simon, Rebecca R. Vanderpool, Mehdi Nouraie, Timothy N. Bachman, Pamela M. White, Masataka Sugahara, John Gorcsan III, Ed L. Parsley, and Mark T. Gladwin. JCI Insight; 2016,1(18):e89620.