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SS-31 (Elamipretide): The Mitochondrial Peptide That Reached Real Trials

SS-31 is elamipretide, a mitochondria-targeting peptide studied in Barth syndrome and primary mitochondrial myopathy. What the trials found, including where they missed.

Not approvedStudied in rare disease

Caroline S · Published 2026-09-07

Length

Tetrapeptide

Sequence

Cardiolipin-targeting peptide

Origin

Designed to concentrate in the inner mitochondrial membrane

Last reviewed

2026-09-07

What is it good for?

Mitochondrial function. It concentrates in the inner mitochondrial membrane and interacts with cardiolipin, a lipid essential to how mitochondria generate energy — which is why it was tested in genetic diseases of that machinery.

Illustration: A clear glass ampule with faint green liquid on a white lab bench.
Illustration

What it is

SS-31 is elamipretide, a short peptide designed to do something specific: concentrate in the inner mitochondrial membrane, where it interacts with cardiolipin — a lipid essential to how mitochondria generate energy.

That targeting is the whole point of the molecule. Rather than acting broadly, it accumulates where the machinery it is meant to affect actually sits. MOTS-c, the other mitochondrial entry in this library, arrives at the same organelle from the opposite direction. The cell's own general-purpose antioxidant, glutathione, is the broad-acting comparison this molecule was designed not to be.

What makes this entry different

Most compounds in this library were never taken into registered clinical trials by anyone. This one was, by Stealth BioTherapeutics, in two rare genetic diseases of mitochondrial function: Barth syndrome and primary mitochondrial myopathy.

That means there is a real evidence trail — with a real, published miss in it.

What the trials found

Primary mitochondrial myopathy: participants on high-dose subcutaneous elamipretide walked further in a six-minute walk test than those on placebo, and the increase was dose-dependent. A dose-response relationship is one of the stronger signals a trial can produce, because it is hard to explain by chance.

Barth syndrome: in a randomised, double-blind, placebo-controlled crossover trial, neither primary endpoint was met in part 1. In the later open-label phase, 40 mg subcutaneously improved six-minute walk scores with no serious adverse events, and benefit began to accrue after about six months.

Both halves belong in the summary. A trial that misses its primary endpoints and then shows something in an open-label extension is genuinely weaker evidence than one that hits — open-label means everyone knew what they were getting, and a walking test is sensitive to that.

Who was studied

This is the part that does not survive the trip into a vendor listing.

The participants had rare genetic diseases in which mitochondrial function is measurably impaired from birth. Improving a system that is failing is a different proposition from improving one that is working normally.

No trial has examined SS-31 for energy, performance or longevity in healthy people. The compound is marketed for exactly those things, which is what places it among the compounds sold for longevity.

Safety, in context

In the trials, the most common adverse events were headache and dizziness, and the Barth syndrome work reported no serious adverse events.

That is a reassuring profile — for a manufactured pharmaceutical product, at defined doses, in a monitored trial population. It carries over to a research-chemical vial only as far as one is prepared to assume the contents match.

Where it stands

Approved as a medicine: not approved.

Sold as: a research chemical, for uses no trial has studied.

Within the mitochondrial group, the 24-residue peptide encoded in mitochondrial DNA itself is the opposite case: a large literature, 312 human-tagged papers, and no study anywhere in which it was given to a person.

Last checked

2026-09-07.

What the research shows

Primary mitochondrial myopathy

Human trial: dose-dependent gain in six-minute walk distance

Barth syndrome

Human trial: neither primary endpoint met in part 1; walk scores improved later in the open-label phase

Energy or performance in healthy people

Not what any of the trials studied

Bars show how much of the evidence is in humans, not how well anything works.

Where it stands

Approved as a medicine

Not approved.

Studied in

Barth syndrome and primary mitochondrial myopathy — rare genetic diseases of mitochondrial function.

Reported side effects

Most commonly headache and dizziness; no serious adverse events reported in the Barth syndrome work.

Sold as

A research chemical, marketed for energy and longevity — neither of which the trials examined.

Frequently asked questions

What is SS-31?

SS-31 is elamipretide, a short peptide designed to concentrate in the inner membrane of mitochondria, where it interacts with cardiolipin — a lipid that is essential to how mitochondria produce energy. Unlike most compounds in this library it was developed by a pharmaceutical company and taken into registered clinical trials.

What was it tested for?

Rare genetic diseases of mitochondrial function: Barth syndrome and primary mitochondrial myopathy. Both are serious conditions in which the mitochondrial machinery is impaired from birth. That is the population the trials studied, and it is a long way from the audience the compound is marketed to.

Did the trials work?

Partly, and the honest answer includes the miss. In primary mitochondrial myopathy, people on high-dose subcutaneous elamipretide walked further in a six-minute walk test than those on placebo, with a dose-dependent increase. In Barth syndrome, a randomised double-blind placebo-controlled crossover trial did not meet either primary endpoint in part 1; in the later open-label phase, 40 mg improved six-minute walk scores, with benefit accruing after about six months. A trial that misses its primary endpoint and shows something in an open-label extension is a weaker result than one that hits, and it should be described that way.

Does SS-31 boost energy in healthy people?

No trial has examined that. The research was conducted in people with rare genetic mitochondrial disease, where the machinery is measurably broken. Improving function in a system that is failing is a different proposition from improving one that is working normally, and nothing in the published record addresses the second.

Is it safe?

In the trials, the most common adverse events were headache and dizziness, and the Barth syndrome work reported no serious adverse events. That is a reassuring profile from a supervised trial with a manufactured product, given at defined doses to a monitored population. It says nothing about material bought as a research chemical.