BOSTON, June 24, 2026 (GLOBE NEWSWIRE) — Imbria Pharmaceuticals, Inc. (Imbria), a clinical-stage company developing innovative therapeutics for cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ninerafaxstat for the treatment of symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
Ninerafaxstat, Imbria’s first-in-class investigational oral therapy designed to improve the heart’s ability to produce energy efficiently, is currently being evaluated in the Phase 2b FORTITUDE-HCM clinical trial (NCT07023614) in patients with symptomatic nHCM.
“Receiving Orphan Drug Designation for ninerafaxstat is an important milestone as we advance its development for patients living with symptomatic nHCM, a population with significant unmet medical need,” said Alvin Shih, MD, Chief Executive Officer of Imbria. “Despite recent progress in HCM, there are still no approved therapies for nHCM, and patients continue to experience debilitating symptoms, including shortness of breath, fatigue, and exercise intolerance. By improving cardiac energetics, ninerafaxstat represents a differentiated approach to addressing nHCM, with the potential to be used as a standalone therapy or in combination with other treatments. We look forward to advancing ninerafaxstat through the ongoing FORTITUDE-HCM trial and anticipate reporting topline data in the first half of 2027.”
The FDA grants Orphan Drug Designation to therapies that are being developed to treat rare diseases and conditions that affect fewer than 200,000 people in the U.S. The designation provides certain incentives, such as assistance in the drug development process, tax credits toward the cost of clinical trials, and prescription drug user fee waivers. Upon approval, products with Orphan Drug Designation may be eligible for seven years of market exclusivity for the designated indication, independent of patent protection. This exclusivity complements Imbria’s existing intellectual property portfolio for ninerafaxstat, which includes multiple granted patents extending to 2038 and beyond.
About Ninerafaxstat
Ninerafaxstat is an innovative treatment under development for cardiac diseases characterized by an imbalance of energy supply and demand in the heart. To maintain normal function, the heart requires substantial amounts of energy, which is produced primarily by the mitochondria in the form of ATP. The heart uses two main sources of fuel for energy generation: fatty acids and glucose. Ninerafaxstat, a partial fatty acid oxidation (pFOX) inhibitor, acts to shift the heart’s fuel preference from fatty acids towards glucose. This shift in metabolism leads to more efficient mitochondrial energy generation with the potential for improved cardiac function both at rest and during exercise.
About Non-Obstructive Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is the most common inherited cardiac disease, characterized by the abnormal thickening of the heart muscle, which can lead to a range of complications. HCM is most often diagnosed in adulthood, and symptoms can vary widely.
In non-obstructive HCM (nHCM), the thickened heart muscle does not block blood flow out of the heart but can impair the heart’s ability to relax and fill properly, leading to reduced cardiac efficiency. Patients with nHCM experience a significant burden of heart failure symptoms, including shortness of breath, fatigue, and exercise intolerance, and remain at risk for serious complications such as irregular heart rhythms. Despite this, there are currently no approved therapies for nHCM.
About Imbria
Imbria Pharmaceuticals is a clinical-stage company developing innovative cardiovascular disease therapeutics by targeting cardiac energy metabolism. The company’s focus is ninerafaxstat, a differentiated, first-in-class cardiovascular therapy that acts by enhancing energy production in the heart. The FORTITUDE-HCM (NCT07023614) Phase 2b clinical trial is evaluating ninerafaxstat’s efficacy and safety in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM), a debilitating disease with no currently approved treatments. For more information, visit www.FORTITUDEHCM.COM.
Based in the Boston area, Imbria is backed by a leading syndicate of investors, including RA Capital, SV Health Investors, Deep Track Capital, Catalio Capital Management, BNP Paribas, AN Ventures, and Cytokinetics. For more, visit www.Imbria.com.
Media Contact:
Gina Mangiaracina
6 Degrees
(917) 797-7904
gmangiaracina@6degreespr.com
