Imbria Pharmaceuticals has achieved positive results in a phase 2 trial for its heart failure medication. The Boston-based biotech, in a statement issued on Tuesday, announced that their drug, ninerafaxstat, not only met the safety and tolerability objectives but also showed an improvement in a functional measurement relevant to symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM), an inherited heart condition.
The IMPROVE-HCM phase 2 trial conducted by Imbria was primarily focused on assessing the safety and tolerability of ninerafaxstat, a partial fatty acid oxidation inhibitor designed to shift the heart’s metabolic preference from fatty acids to glucose. This shift in metabolism is expected to lead to more efficient energy generation and enhanced cardiac function. nHCM results in the abnormal thickening of the heart muscle, resulting in complications, including deficiencies in cardiac energy and inefficient energy utilization.
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The trial revealed that after 12 weeks of treatment, patients administered with ninerafaxstat experienced a statistically significant improvement in a functional cardiopulmonary exercise test, which was associated with clinically relevant enhancements in patient-reported outcomes, according to Imbria. The drug was well-tolerated, with no safety concerns identified. Further data from the IMPROVE-HCM trial will be presented at an upcoming medical conference.
“Ninerafaxstat is the first investigational drug to demonstrate an improvement in a functional measure assessed by cardiopulmonary exercise test in nHCM, which combined with a clinically meaningful improvement in patient symptoms measured by the KCCQ heart failure questionnaire, bodes well for patients suffering from nHCM.”
– Martin Maron, M.D., Ph.D., Director of the Hypertrophic Cardiomyopathy Center at Lahey Hospital and Medical Center in Burlington, Massachusetts
“Based on the topline results from IMPROVE-HCM, we are focused on advancing ninerafaxstat into a Phase 3 clinical trial of patients with nHCM and plan to meet with regulatory agencies within the coming months to confirm trial design. As cardiac energy deficiency is a hallmark feature of a range of cardiac diseases, the results of the IMPROVE-HCM clinical trial support the potential of ninerafaxstat to improve symptoms and function, not only in patients with nHCM, but also in patients with HFpEF.”
– Anne Prener, M.D., Ph.D., president and chief executive officer of Imbria Pharmaceuticals
Ninerafaxstat is the sole pipeline medication in Imbria’s portfolio. The biotech is pursuing a three-pronged clinical approach to test the medication in nHCM, HFpEF, and obstructive coronary arterial disease.