Imbria Pharmaceuticals Announces First Patient Randomized in FORTITUDE-HCM Phase 2b Clinical Trial Evaluating Ninerafaxstat for Non-Obstructive Hypertrophic Cardiomyopathy
Advances development of ninerafaxstat, a potential first-in-class therapy designed to improve cardiac energetics in patients with serious cardiovascular diseases
Enrollment underway across U.S. sites with U.K. and EU expansion anticipated by end of 2025 and early 2026, respectively
BOSTON, Nov. 06, 2025 (GLOBE NEWSWIRE) -- Imbria Pharmaceuticals, Inc. (“Imbria”), a clinical-stage company transforming cardiovascular disease therapeutics by targeting cardiac energy metabolism, today announced that the first patient has been randomized in FORTITUDE-HCM, a Phase 2b clinical trial evaluating ninerafaxstat in symptomatic non-obstructive hypertrophic cardiomyopathy (“nHCM”), a debilitating disease with no currently approved treatments.
“Randomizing the first patient in FORTITUDE-HCM represents a significant step forward in our mission to bring innovative therapies to patients with nHCM,” said Albert Kim, MD, PhD, FACC, FHRS, Chief Medical Officer of Imbria. “These patients face a substantial disease burden with limited treatment options. Building on ninerafaxstat’s strong mechanistic rationale and prior clinical data, this trial is designed to evaluate whether improving cardiac energetics can deliver meaningful benefits for patients.”
FORTITUDE-HCM (NCT07023614) is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b clinical trial enrolling approximately 165 patients with symptomatic nHCM. Participants will receive ninerafaxstat 200 mg BID or placebo. The primary endpoint is change from baseline in the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (“KCCQ-CSS”). Secondary endpoints include changes in KCCQ-CSS component and Overall Summary scores, as well as ventilatory efficiency during standardized cardiopulmonary exercise testing.
“Patients with nHCM have long lacked effective, disease-specific treatment options,” said Martin S. Maron, MD, Director of the Hypertrophic Cardiomyopathy Center at Lahey Hospital and Medical Center and Principal Investigator for the FORTITUDE-HCM trial. “By targeting the underlying energetic inefficiency of the heart muscle, ninerafaxstat offers a promising and differentiated approach to improving symptoms and quality of life for this patient population.”
Enrollment in FORTITUDE-HCM is underway with several clinical sites activated across the U.S., and additional U.S. and U.K. sites expected to open in the coming months. EU site activations are anticipated to commence in early 2026.
About FORTITUDE-HCM
Initiated in 2025, FORTITUDE-HCM (NCT07023614) is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b clinical trial enrolling approximately 165 patients with symptomatic nHCM. Participants will receive ninerafaxstat 200 mg BID or placebo. The primary endpoint is change from baseline in the Kansas City Cardiomyopathy Questionnaire-Clinical Summary Score (“KCCQ-CSS”). Secondary endpoints include changes in KCCQ-CSS component and Overall Summary scores, as well as ventilatory efficiency during standardized cardiopulmonary exercise testing (CPET). Enrollment for FORTITUDE-HCM is underway in the U.S. Additional sites in the U.K. and EU are anticipated to begin enrolling by the end of 2025 and early 2026, respectively.
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 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 various complications. HCM is most often diagnosed in middle age, and symptoms can vary widely. In non-obstructive HCM (nHCM), the heart muscle becomes abnormally thickened, impairing the blood flow out of the heart, leading to the heart muscle working harder to pump blood to the body. Patients with nHCM experience a high burden of symptoms of heart failure and are at risk for adverse disease complications, including irregular heart rhythms, yet there are no approved treatments available.
About Imbria
Imbria Pharmaceuticals is a clinical-stage company developing transformational cardiovascular disease therapeutics by targeting cardiac energy metabolism. The company’s focus is ninerafaxstat, a differentiated, first-in-class cardiovascular therapy. A pipeline-in-a-pill, it has broad potential utility by improving cardiac energetics without adverse effects on heart rate, rhythm, left ventricular ejection fraction, or blood pressure. The recently initiated FORTITUDE-HCM (NCT07023614), Phase 2b clinical trial is addressing non-obstructive hypertrophic cardiomyopathy (nHCM), a debilitating disease with no currently approved treatments. 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, AN Ventures, and Cytokinetics. For more, visit www.imbria.com.
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