SS-31 (elamipretide) — compound monograph
Mitochondria-targeted cardiolipin-binding tetrapeptide. Discontinued. The Institute assesses 3 outcomes for this compound and grades the strongest at moderate certainty.
§1Identification and status
§1.1Nomenclature
- Preferred name
- SS-31 (elamipretide)
- Compound class
- Mitochondria-targeted cardiolipin-binding tetrapeptide
- Assessment series
- Mitochondrial and metabolic cofactors
- Synonyms and codes
- elamipretide · MTP-131 · Bendavia · SS-31 tetrapeptide
- Route as evaluated
- Subcutaneous or intravenous in clinical studies
§1.2Chemistry
Chemical identifiers are reproduced where they are public. Where a structure has not been published the monograph records not disclosed rather than constructing one.[1]
- CAS registry number
- 736992-21-5
- Molecular formula
- C32H49N9O5
- Average mass
- 639.79
- Monoisotopic mass
- 639.38
- ATC classification
- not assigned
§1.3Sequence and structural notes
An aromatic-cationic tetrapeptide containing D-arginine and the unnatural residue 2,6-dimethyltyrosine. Its alternating aromatic-cationic motif drives concentration-independent accumulation in the inner mitochondrial membrane, where it binds cardiolipin. The unnatural residue and the D-configuration make this one of the more demanding short peptides to synthesise and to characterise.
§1.4Assessment status
The Institute assesses SS-31 (elamipretide) across 3 indications and grades the strongest of them at moderate certainty. Clinical development was terminated. The Institute treats a discontinued programme as evidence.
Table 1. Assessed outcomes for SS-31 (elamipretide), ordered by certainty. Each row links to the per-indication evidence extract.
| Indication | Effect as recorded | Certainty | Trials |
|---|---|---|---|
| Atherosclerotic cardiovascular disease and cardiovascular risk reduction | A phase 2 trial in ST-elevation myocardial infarction did not reduce infarct size | Moderate | 1 |
| Primary mitochondrial myopathy and bioenergetic disorders | A phase 3 trial in primary mitochondrial myopathy did not meet its primary endpoints of six-minute walk distance and symptom score at 24 weeks | Moderate | 2 |
| Heart failure with preserved ejection fraction and obesity | A phase 2 trial in heart failure with preserved ejection fraction did not demonstrate benefit on the primary endpoint | Low | 1 |
| Effects are reproduced as the contributing trials reported them. Where a confidence interval is held it appears on the per-indication extract rather than in this summary table. | |||
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.
- International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. ICH Q6B Specifications: Test Procedures and Acceptance Criteria for Biotechnological/Biological Products. ICH Harmonised Tripartite Guideline 1999;Step 4 version. identifier not held by the Institute
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.