
A study evaluating the safety and efficacy of Ninerafaxstat, a novel cardiac mitotrope targeting impaired myocardial energetics in nonobstructive hypertrophic cardiomyopathy (nHCM), has concluded. Conducted over 12 weeks with 67 patients, the trial observed 11.8% of patients on Ninerafaxstat experiencing serious adverse events compared to 6.1% on placebo. Ninerafaxstat demonstrated significantly improved ventilatory efficiency (VE/Vco2 slope) compared to placebo, though no significant difference was noted in peak VO2. While the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score showed non-significant improvement overall, it was significant in patients with baseline scores ≤80. These findings support further evaluation of Ninerafaxstat in phase 3 trials for symptomatic nHCM patients.
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