Reviewed by Peptide Treatments Medical Advisory Board (Medical Advisory Board)

SS-31: Evidence Summary

Evidence summary for SS-31 across studied indications. Each indication is graded using our evidence tier system based on the quality and quantity of available clinical data.

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Indication Evidence Tier Trial Count Summary
Barth syndrome Tier A 1 FDA approved September 2025 via accelerated approval. TAZPOWER trial showed +95.9m 6MWT, +45% knee extensor strength.
Heart failure (HFpEF) Tier C 4 EMBRACE-HF FAILED primary endpoint (KCCQ score). 6MWT improvement (+42m vs +10m) was a secondary endpoint only. Phase 3 SPRING-HF recruiting.
Primary mitochondrial myopathy Tier C 3 MMPOWER-3 FAILED primary endpoint. Genotype-specific post-hoc benefit observed. Mixed evidence overall.
Age-related decline Tier C 1 Improved exercise tolerance in elderly subjects via cardiolipin stabilization mechanism; preclinical/early clinical

References (11)

  1. EMBRACE-HF Phase 2 trial of elamipretide in HFpEF — Various . JACC: Heart Failure (2021) PMID: 32068002
  2. Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential . International journal of molecular sciences (2025) PMID: 39940712
  3. The Mitochondria-Targeted Peptide Therapeutic Elamipretide Improves Cardiac and Skeletal Muscle Function During Aging Without Detectable Changes in Tissue Epigenetic or Transcriptomic Age . Aging cell (2025) PMID: 40080911
  4. Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial . Orphanet journal of rare diseases (2024) PMID: 39574155
  5. Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER . Genetics in Medicine (2024) PMID: 38602181
  6. Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial . Neurology (2023) PMID: 37268435
  7. New insight for SS-31 in treating diabetic cardiomyopathy: Activation of mitoGPX4 and alleviation of mitochondria-dependent ferroptosis . International journal of molecular medicine (2024) PMID: 39364755
  8. SS-31 alleviated nociceptive responses and restored mitochondrial function in a headache mouse model via Sirt3/Pgc-1alpha positive feedback loop . The journal of headache and pain (2023) PMID: 37271805
  9. Elamipretide (SS-31) Attenuates Idiopathic Pulmonary Fibrosis by Inhibiting the Nrf2-Dependent NLRP3 Inflammasome in Macrophages . Antioxidants (2023) PMID: 38136142
  10. The mitochondrially targeted peptide elamipretide (SS-31) improves ADP sensitivity in aged mitochondria by increasing uptake through the adenine nucleotide translocator (ANT). — Pharaoh G, Kamat V, Kannan S, et al. . GeroScience (2023) PMID: 37462785
  11. Application research of novel peptide mitochondrial-targeted antioxidant SS-31 in mitigating mitochondrial dysfunction. — Du X, Zeng Q, Luo Y, et al. . Mitochondrion (2024) PMID: 38237649