SS-31 (Elamipretide) is a mitochondria-targeted peptide that concentrates in the inner mitochondrial membrane and protects cardiolipin from oxidative damage. Research focuses on cardiac function, heart failure, and mitochondrial disease.
Standard Research Dosage
0.05–0.25 mg/kg body weight, administered subcutaneously once daily. Clinical trials for heart failure (PROGRESS-HF) used 40 mg/day subcutaneous infusion. Research protocols typically use 5–20 mg/day.
Mechanism of Action
SS-31 binds cardiolipin in the inner mitochondrial membrane, stabilizing the electron transport chain and reducing reactive oxygen species (ROS) production. This mechanism is distinct from other antioxidants and is specific to mitochondrial membranes.
Clinical Trial Record
PROGRESS-HF Phase 2 trial showed encouraging signals in HFpEF. However, the pivotal Phase 3 MMPOWER-3 trial (primary mitochondrial myopathy; Neurology 2023, n≈218) did not meet its primary endpoint. A separate heart failure program (MMPOWER-HF) also did not demonstrate efficacy. Human efficacy is not established.