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Compound Evidence InstituteEvidence synthesis · established 2023Graded assessments of compounds, trials, methods and supply
Document set current to 30 July 2026
Compound monograph · §2

SS-31 (elamipretide) — pharmacology

Molecular targets, mechanism of action, pharmacokinetics and interactions as characterised in humans.

Document identifier
CEI-MN-044/2
Series
Compound monograph
Version
4.2
Published
17 May 2024
Last reviewed
17 Apr 2025
Next review
17 Apr 2027
Identifier
10.71829/cei.mono.44
Certainty
Moderate
Cycle
2024 Q2

§2Pharmacology

§2.1Molecular targets

Table 2. Molecular targets recorded for SS-31 (elamipretide), with the character of the interaction and the potency where the Institute holds it.

TargetInteractionNote
Cardiolipin (inner mitochondrial membrane)Direct bindingNot a receptor interaction; the peptide associates with the lipid and stabilises cristae architecture and electron-transport-chain supercomplexes

§2.2Mechanism of action

Elamipretide binds cardiolipin in the inner mitochondrial membrane, stabilising cristae curvature and the assembly of electron-transport-chain supercomplexes, and reducing cytochrome-c peroxidase activity. The result in preclinical models is improved oxidative phosphorylation efficiency and reduced reactive oxygen species generation. The mechanism is unusual among the compounds in this series in being a specific, well-characterised lipid interaction rather than a receptor hypothesis.[1,2]

§2.3Pharmacokinetics

Terminal half-life
≈1–2 h in plasma; tissue mitochondrial accumulation persists longer
Time to maximum concentration
≈1 h after subcutaneous administration
Volume of distribution
large apparent volume reflecting mitochondrial partitioning
Plasma protein binding
moderate
Clearance
not published
Bioavailability
well absorbed subcutaneously

Proteolysis and renal excretion. Plasma pharmacokinetics understate the pharmacodynamic duration because the site of action is an intracellular membrane compartment.

Reconstructed plasma concentration–time profilePlasma concentration plotted against time after dosing, reconstructed from published pharmacokinetic parameters.0.00.20.40.602468Time after first dose (hours)Relative concentrationt max ≈ 1 ht½ ≈ 2 h
Modelled profileSimulated observationsDose administration
Figure 2. Illustrative. Plasma concentration–time profile reconstructed by the Institute from the published half-life and time-to-maximum-concentration parameters using a one-compartment model with first-order absorption. The curve is not digitised from a published figure and its vertical scale is relative. It is published to convey the shape of the profile and the approach to steady state, not to supply a concentration at any time point.

§2.4Interactions

  • Not characterised as clinically significant

References cited on this page

References are numbered in order of first citation in this document. Each superscript in the text links to its entry below.

  1. Szeto HH. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. British Journal of Pharmacology 2014;171(8):2029–2050. doi:10.1111/bph.12461 · PMID 24117165
  2. D’Hondt M, Bracke N, Taevernier L, Gevaert B, Verbeke F, Wynendaele E, De Spiegeleer B. Related impurities in peptide medicines. Journal of Pharmaceutical and Biomedical Analysis 2014;101:2–30. doi:10.1016/j.jpba.2014.06.012 · PMID 25044089

Identifiers are reproduced only where the Institute holds them. Where a digital object identifier or PubMed identifier is not shown, the Institute has recorded the journal and year and has not constructed an identifier.

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