| Dimension | Scoring Notes | Score |
|---|---|---|
| Study Design | Multiple Phase II and one Phase III RCT completed. MMPOWER-3 was a rigorous 24-week, randomized, double-blind, placebo-controlled trial enrolling 218 participants across 27 international sites — gold-standard design. PROGRESS-HF and TAZPOWER are Phase II. The design quality is genuinely high; the outcomes are the limitation. Scored at Phase I/II tier to reflect that the best-powered trial (MMPOWER-3) failed its primary endpoints, and no completed Phase III has demonstrated efficacy. |
10 / 25 |
| Sample Size | MMPOWER-3 enrolled 218 participants — the largest SS-31 human trial to date. PROGRESS-HF enrolled ~47. TAZPOWER enrolled 12 (Barth syndrome is an ultra-rare disease). Cumulative human exposure across completed trials is in the low hundreds. Meaningful Phase II/III-level sample in MMPOWER-3; smaller in other indications. |
8 / 20 |
| Replication | Multiple trials across multiple independent sites and research groups — Harvard/MGH, Cleveland Clinic, Nationwide Children's, and international academic centers contributed to MMPOWER-3. TAZPOWER and PROGRESS-HF were conducted by separate principal investigator groups. Meaningful independent replication exists at the trial level, though all sponsored by Stealth BioTherapeutics. Separate academic-led investigator-initiated studies (e.g., FRDA trial at NCT05168774) add independent replication signal. |
10 / 20 |
| Journal Impact Factor | MMPOWER-3 results published in Neurology (IF ~9). TAZPOWER in Genetics in Medicine (IF ~8). Foundational cardiolipin/SS-31 mechanism papers in PNAS (IF ~11) and JASN (Journal of the American Society of Nephrology, IF ~13). Solid journal placement across the evidence base — this is one of the better-published compounds in the Longevity category. |
12 / 15 |
| Funding Independence | Stealth BioTherapeutics (formerly Stealth Peptides) funded the major clinical trials. The company filed for bankruptcy in 2022 after MMPOWER-3 failed — trials were pre-registered and conducted to pharmaceutical standards, but funding was entirely industry (no NIH or independent academic primary funding for Phase II/III trials). Foundational Szeto laboratory work at Weill Cornell was NIH-funded. Mixed picture — industry for clinical, academic for mechanistic. Scored to reflect industry dominance in the clinical evidence. |
4 / 10 |
| Population Diversity | MMPOWER-3 was 94% White, mean age 45.6, 64% women — conducted across 7 countries but ethnically narrow by modern trial standards. Barth syndrome trials by definition enroll a very narrow rare-disease pediatric/young adult population. Limited diversity score reflects this honestly. |
1 / 5 |
| Researcher h-Index | Hazel Szeto (Weill Cornell) is the discoverer of the SS-peptide class with h-Index ~50+ in mitochondrial pharmacology. Vincenzo Carelli (University of Bologna), lead MMPOWER-3 investigator, has h-Index ~50+ in mitochondrial disease research. Top-tier researchers across the program. |
5 / 5 |