Ninerafaxstat
NINERAFAXSTAT: A UNIQUE MODULATOR OF CARDIAC ENERGETICS
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 contractile function, the heart requires substantial amounts of energy, which is produced primarily by the mitochondria in the form of ATP. The heart normally uses two main fuels 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.
Ninerafaxstat Development
Ninerafaxstat has broad potential utility for treating cardiovascular diseases by improving cardiac efficiency without negative effects on heart rate, rhythm, ejection fraction, or blood pressure.
Investigational therapy for nHCM
We are conducting FORTITUDE-HCM (NCT07023614), a Phase 2b global, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of ninerafaxstat compared to placebo on top of standard of care in patients with symptomatic nHCM.
Potential in HFpEF
Plans are also underway to initiate a Phase 2b clinical trial in HFpEF, a large and growing unmet need.
Encouraging safety profile in trials to date
Phase 2a clinical trials have been completed in nHCM and HFpEF. In Phase 1 and 2 clinical trials, ninerafaxstat was shown to be safe and well tolerated.