SS-31 (elamipretide) — clinical evidence
Assessed outcomes with certainty ratings, contributing trials and the reasoning for each rating.
§3Clinical evidence
§3.1Atherosclerotic cardiovascular disease and cardiovascular risk reduction
Anchor outcome. Three-point major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction, non-fatal stroke).
Effect as recorded. A phase 2 trial in ST-elevation myocardial infarction did not reduce infarct size; negative result.[1,2]
Certainty. Moderate certainty A clear null result on an objective imaging endpoint.
Contributing trials. EMBRACE-STEMI. Full structured abstracts are published for each.
Full evidence extract for atherosclerotic cardiovascular disease and cardiovascular risk reduction · Indication assessment
§3.2Primary mitochondrial myopathy and bioenergetic disorders
Anchor outcome. Six-minute walk distance.
Effect as recorded. 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; negative result on both primary endpoints.[2,3]
Certainty. Moderate certainty The Institute rates the negative finding moderate certainty. A well-conducted phase 3 trial with a clear null result is stronger evidence about this compound than the entire preclinical literature, and is reported first for that reason.
Contributing trials. MMPOWER-3 · MMPOWER-2. Full structured abstracts are published for each.
Full evidence extract for primary mitochondrial myopathy and bioenergetic disorders · Indication assessment
§3.3Heart failure with preserved ejection fraction and obesity
Anchor outcome. Change in Kansas City Cardiomyopathy Questionnaire clinical summary score.
Effect as recorded. A phase 2 trial in heart failure with preserved ejection fraction did not demonstrate benefit on the primary endpoint; negative result.[3,4]
Certainty. Low certainty Small sample.
Contributing trials. ELAM-PH2-HFPEF. Full structured abstracts are published for each.
Full evidence extract for heart failure with preserved ejection fraction and obesity · Indication assessment
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.
- 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
- 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
- Guyatt GH, Oxman AD, Kunz R, Brozek J, Alonso-Coello P, Rind D, Devereaux PJ, Montori VM, Freyschuss B, Vist G, Jaeschke R, Williams JW, Murad MH, Sinclair D, Falck-Ytter Y, Meerpohl J, Whittington C, Thorlund K, Andrews J, Schünemann HJ. GRADE guidelines: 6. Rating the quality of evidence — imprecision. Journal of Clinical Epidemiology 2011;64(12):1283–1293. doi:10.1016/j.jclinepi.2011.01.012 · PMID 21839614
- Sterne JAC, Savović J, Page MJ, Elbers RG, Blencowe NS, Boutron I, Cates CJ, Cheng HY, Corbett MS, Eldridge SM, Emberson JR, Hernán MA, Hopewell S, Hróbjartsson A, Junqueira DR, Jüni P, Kirkham JJ, Lasserson T, Li T, McAleenan A. RoB 2: a revised tool for assessing risk of bias in randomised trials. BMJ 2019;366:l4898. doi:10.1136/bmj.l4898 · PMID 31462531
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