SS-31 (Elamipretide)

Mitochondria-Targeted Peptideinvestigational

Also known as: Elamipretide, Bendavia, MTP-131, SS-31 Peptide

A mitochondria-targeted tetrapeptide that concentrates in the inner mitochondrial membrane, protecting cardiolipin and restoring bioenergetics in aging and disease.

Overview

SS-31 (Elamipretide) is a synthetic cell-permeable tetrapeptide specifically designed to target and accumulate in the inner mitochondrial membrane. Developed by Hazel Szeto at Weill Cornell Medicine, SS-31 selectively binds to cardiolipin — a phospholipid essential for cristae structure, electron transport chain efficiency, and ATP production. By stabilizing cardiolipin and preventing its oxidation, SS-31 restores mitochondrial function, reduces reactive oxygen species (ROS) production at the source, and improves cellular bioenergetics. SS-31 has been investigated in clinical trials for Barth syndrome (a cardiolipin-deficiency disorder), heart failure, age-related mitochondrial dysfunction, and primary mitochondrial myopathy. Although it received FDA Fast Track designation for Barth syndrome, clinical trial results have been mixed, and it has not yet achieved regulatory approval. It remains one of the most scientifically rigorous approaches to targeting mitochondrial dysfunction in aging.

Mechanism of Action

SS-31 operates through direct mitochondrial membrane targeting: (1) Its alternating aromatic-cationic motif (D-Arg-dimethylTyr-Lys-Phe) allows rapid cell penetration and 1000-5000x concentration in the inner mitochondrial membrane; (2) Binds specifically to cardiolipin, stabilizing its interaction with cytochrome c and the electron transport chain complexes; (3) Prevents cardiolipin peroxidation, which is a primary driver of mitochondrial dysfunction in aging; (4) Optimizes electron transport chain efficiency, reducing electron leak and ROS generation at complexes I and III; (5) Restores mitochondrial membrane potential and ATP production; (6) Prevents mitochondrial permeability transition pore opening, protecting against apoptosis; (7) Reduces age-related mitochondrial fragmentation and promotes healthy mitochondrial dynamics.

Molecular Formula

C32H49N5O5

Molecular Weight

639.8 g/mol

Sequence

D-Arg-Dmt-Lys-Phe-NH2 (Dmt = 2',6'-dimethyltyrosine)

Dosage Protocols

Dose Range

10mg40mg

Frequency

Once daily

Route

subcutaneous

Cycle Length

4-12 weeks (experimental)

Clinical trials used 4mg-40mg SC or IV daily. The 40mg SC dose was most commonly tested. Research peptide users typically use lower doses. No approved human dosing exists.

Source: Clinical trial protocols

Side Effects

EffectSeverity
Injection site reactionmild
Headachemild
Nauseamild
Fatiguemild

Pros & Cons

Scientifically elegant mechanism — directly targets the inner mitochondrial membrane and cardiolipin

Addresses the root cause of mitochondrial dysfunction rather than downstream symptoms

Extensive clinical trial program with human safety data from multiple Phase 2/3 studies

FDA Fast Track designation demonstrates regulatory recognition of the therapeutic potential

Broad potential applications: heart failure, aging, neurodegenerative disease, mitochondrial myopathies

Clinical trial results have been mixed — primary endpoints not always met despite promising biomarker data

Not yet approved by any regulatory agency despite years of clinical development

Expensive to synthesize due to non-natural amino acid (Dmt) requirement

Short half-life requiring daily injection; no oral formulation available

Research Studies

Legal Status

Investigational drug — FDA Fast Track designation for Barth syndrome. Not yet approved by FDA or other agencies. Phase 2/3 clinical trials completed for multiple indications with mixed results. Available from select research peptide suppliers.

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