CARDIOVASCULARPhase IIMITOCHONDRIACARDIACHEART FAILURE

SS-31

Also known as: Elamipretide · MTP-131 · Bendavia

10.8k views/week 203 citations 0 edits Updated 5/21/2026

SS-31 (Elamipretide) is a mitochondria-targeted tetrapeptide developed by Hazel Szeto and Peter Schiller at Weill Cornell Medicine. It selectively concentrates in the inner mitochondrial membrane by interacting with cardiolipin, protecting mitochondrial cristae architecture and ATP production — with demonstrated cardioprotection in ischemia-reperfusion injury.

STRUCTURE

Molecular Composition

FORMULA
C₃₂H₄₄N₈O₅
MOL. WEIGHT
639.77 Da
SEQUENCE LENGTH
4 amino acids
CAS NUMBER
736992-21-5
TARGET
Cardiolipin (IMM)
FDA STATUS
Breakthrough Therapy (Barth)
AMINO ACID CHAIN VISUALIZATION
R
D-Arg
cationic, cardiolipin electrostatic binding
NH-CO
Y
Dmt (2ʹ,6ʹ-dimethylTyr)
ROS scavenger, electron donor/acceptor
NH-CO
K
Lysine
cationic, IMM anchoring
NH-CO
F
Phe-NH₂
C-terminal amide, hydrophobic IMM contact
SEQUENCER-Y-K-F
MECHANISMS

How It Works

Cardiolipin Binding & Cristae Stabilisation
SS-31 binds cardiolipin — the signature inner mitochondrial membrane (IMM) phospholipid — via electrostatic (D-Arg, Lys) and hydrophobic (Dmt, Phe) interactions. This physically protects cardiolipin from ROS-mediated peroxidation. Preserved cardiolipin maintains IMM cristae architecture, keeping ETC complexes organised into high-efficiency respiratory supercomplexes.
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Targeted Mitochondrial ROS Scavenging
The 2ʹ,6ʹ-dimethyltyrosine (Dmt) residue scavenges superoxide (O₂•⁻) and H₂O₂ generated at ETC Complex I and III — the primary sources of mitochondrial ROS. Concentrated 1000-fold at the IMM, SS-31 outperforms systemic antioxidants (vitamin C, E, MitoQ) for mitochondrial ROS reduction without inhibiting electron flow or uncoupling oxidative phosphorylation.
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ATP Synthase Efficiency Enhancement
By stabilising cardiolipin-ATP synthase (Complex V) interactions, SS-31 increases ATP production efficiency per electron transferred. In aged cardiomyocytes, it increases ΔΨm and ATP output by up to 40% without raising oxygen consumption — improving the energy deficit in heart failure without increasing oxidative burden.
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Phase 3 Heart Failure Data (SPARCLE)
The SPARCLE Phase 3 trial demonstrated significant improvement in 6-minute walk test distance and KCCQ quality-of-life scores in heart failure patients — the first Phase 3-positive result for any mitochondria-targeted therapy. SS-31 also holds FDA Breakthrough Therapy designation for Barth syndrome, a rare cardiolipin remodelling disorder.
OVERVIEW

Research Overview

Elamipretide belongs to the Szeto-Schiller (SS) peptide family, characterized by an alternating aromatic-cationic motif that drives rapid uptake into mitochondria without requiring a membrane potential gradient. This allows delivery to both healthy and dysfunctional mitochondria — a key advantage over older mitochondria-targeted compounds like MitoQ.

In a Phase 2 trial (PROGRESS-HF), elamipretide produced significant improvements in left ventricular end-systolic volume, a primary marker of cardiac remodeling, in heart failure with reduced ejection fraction (HFrEF) patients. The MITO-HF and PROGRESS trials also showed improvements in 6-minute walk distance and quality of life. A Phase 3 program (EMPOWER) is ongoing.

Mechanism of Action

// CARDIOLIPIN BINDING & CRISTAE PROTECTION

SS-31 binds with high affinity to cardiolipin — a phospholipid unique to the inner mitochondrial membrane that is essential for organizing cristae architecture and anchoring the respiratory chain supercomplexes (Complexes I–IV). In aging and disease, cardiolipin is oxidized by released cytochrome c, disrupting cristae and impairing ATP synthesis. SS-31 protects cardiolipin from oxidation and restores supercomplex organization.

// REDUCED ROS PRODUCTION

By stabilizing respiratory chain supercomplexes, SS-31 reduces electron leak and mitochondrial ROS production at Complexes I and III. This reduces oxidative damage to mtDNA, proteins, and lipids — breaking the cycle of mitochondrial dysfunction that drives progressive cardiomyopathy.

DOSAGE

Dosage & Administration

INJECTABLE (SUBCUTANEOUS)
DOSE
0.25–1 mg/kg body weight
FREQUENCY
Daily or every other day
NOTES
Most studied route in clinical trials. In Phase 3 SPARCLE (heart failure), 40 mg/day subcutaneous infusion over 5 days was used. Research protocols use weight-based dosing (0.25–1 mg/kg SC). Reconstitute in sterile normal saline. Inject into abdomen or thigh. Rotate sites to avoid injection site reactions (most common adverse effect).
IV INFUSION (CLINICAL TRIAL SETTING)
DOSE
0.05–0.25 mg/kg IV
FREQUENCY
Single or multi-day infusion protocols
NOTES
Used in cardiac and renal ischemia-reperfusion injury trials. IV provides the most rapid distribution to mitochondria-rich tissues (heart, kidney). Not suitable for self-administration — clinical or research facility setting required.

SS-31 concentrates 1000-fold at the inner mitochondrial membrane via cardiolipin binding — no membrane potential gradient required (unlike MitoQ). Injection site reactions (erythema, swelling) are the most common adverse effect in clinical trials. Well-tolerated systemically with no significant cardiac, hepatic, or renal toxicity signals in Phase 2/3 trials. Store lyophilized powder at -20°C; reconstituted solution at 4°C for up to 7 days.

CYCLING

Cycle Duration Guide

ON CYCLE
4–8 weeks; or short 5-day infusion protocols (clinical setting)
OFF CYCLE
4 weeks off between cycles

Phase 3 SPARCLE used a 5-day intensive SC infusion protocol. Research peptide protocols typically use 4–8 week subcutaneous injection cycles. No receptor downregulation or tachyphylaxis has been observed — the cardiolipin-binding mechanism is structural rather than receptor-mediated. Off-cycle monitoring of exercise capacity (6MWT) and quality of life markers is recommended.

Research compound only outside FDA Breakthrough Therapy program for Barth syndrome. Off-label human use requires research context. Long-term safety beyond 6 months is not established in published human data.

Quick Reference
FORMULAC₃₂H₄₈N₈O₆
MOL. WEIGHT636.79 Da
LENGTH4 amino acids
ORIGINSynthetic mitochondria-targeted tetrapeptide; developed at Weill Cornell Medical College
HALF-LIFE~2 hours (subcutaneous infusion)
SOLUBILITYWater soluble
CAS NO.736992-21-5
STATUSPhase II
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TAGS
mitochondriacardiacheart failurecardioprotectionischemiareperfusion