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SBT-272, given the international nonproprietary name bevemipretide, is a mitochondria-targeted peptidomimetic from Stealth BioTherapeutics that binds and stabilises cardiolipin, the signature phospholipid of the inner mitochondrial membrane [1]. In a hTDP-43 mouse model of ALS it restored mitochondrial structure, motility and respiratory function in diseased upper motor neurons, and sixty days of chronic dosing from an early symptomatic stage reduced astrogliosis, microgliosis and TDP-43 pathology in the motor cortex [1]. Reviews of the cardiolipin field describe it as a second-generation follow-on to elamipretide, redesigned for higher mitochondrial uptake and higher brain concentrations so that the same membrane mechanism can be tested in the central nervous system [2][6]. One primary peer-reviewed paper carries the entire preclinical case.
- Stabilizes cardiolipin and restores mitochondrial structure, respiration, and motility in neurons
- Reduced TDP-43 pathology and glial inflammation in a preclinical ALS model, with brain penetration
- No adverse-event profile has been published for SBT-272 in humans or animals; the source study reports only that the compound was well tolerated in mice [1]
Overview
Mitochondrial dysfunction is a recurring theme across neurodegenerative diseases, and disruption of the inner mitochondrial membrane has been observed in the upper motor neurons of ALS patients with TDP-43 proteinopathy. SBT-272 was designed as a cardiolipin-stabilizing peptide, structurally related to the mitochondria-targeting chemistry that Stealth BioTherapeutics also explored with elamipretide [1].
In the hTDP-43 mouse ALS model, SBT-272 significantly improved mitochondrial structural integrity and restored respiratory function and organelle motility, translating to improved health of diseased upper motor neurons in vitro. In a head-to-head comparison it appeared more effective than edaravone or AMX0035 in that assay. Sixty days of chronic treatment beginning at an early symptomatic stage reduced astrogliosis, microgliosis, and TDP-43 pathology in the motor cortex [1].
SBT-272 remains a preclinical, experimental compound available for laboratory research only. The published evidence, while mechanistically encouraging, rests on a single primary study in animal and cellular models and has not been validated in humans.
- SBT-272 works by stabilizing cardiolipin, the signature lipid that gives inner mitochondrial membranes their folded cristae shape.
- In an ALS mouse model it outperformed both edaravone and AMX0035 at restoring diseased motor-neuron health in vitro.
- It is brain-penetrant, allowing it to reach the central nervous system where many mitochondrial peptides cannot.
Mechanism
Cardiolipin is the phospholipid unique to the inner membrane; it shapes the cristae folds and holds the electron transport chain complexes together as supercomplexes, so when cardiolipin is lost or oxidised the membrane flattens, respiration falls and the organelle leaks electrons. SBT-272 concentrates in mitochondria and binds cardiolipin, which preserves that inner-membrane architecture and restores respiratory function [1][7]. Work on its parent SS-31 showed the family does this by inserting into the lipid bilayer and altering the membrane surface electrostatics rather than by hitting a classical receptor or enzyme, which is why the effect is structural rather than catalytic [10]; that paper is about SS-31, not about SBT-272.
Structurally SBT-272 is the SS-31 sequence, D-arginine then 2,6-dimethyltyrosine then lysine, with the C-terminal phenylalanine amide replaced by a 3-benzyl-1,2,4-oxadiazole, which is the change credited with its higher uptake and higher brain exposure [2][6]. Downstream of the membrane effect, treated neurons in the ALS model recovered ATP output, motility along the axon and overall cell health, and the motor showed less TDP-43 pathology and less glial activation [1][8][4]. Nothing in this chain is a claim about lowering a specific disease marker in a human being.
receptor fingerprint
Cardiolipin (inner membrane)Binds and stabilises
electron transport chainRestores function
axonal transportRestores motility
TDP-43 proteinopathy and glial activationReduces
Dosingtypical ranges, not medical advice
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Safetyrisks and cautions, not medical advice
SBT-272 is an investigational drug candidate sold to laboratories for research use only; it is not approved, not a supplement, and not intended for people. It is further along than most compounds on this shelf: a phase 1 study in healthy volunteers has been conducted, and two PubMed-indexed reviews list the programme at phase 1 [2][3]. Stealth BioTherapeutics stated in a November 2022 announcement that the dose levels selected for further clinical evaluation appeared generally safe and well tolerated.
That is a company disclosure and not a published dataset; no results paper exists, and a ClinicalTrials.gov search for SBT-272 and for bevemipretide returns no registered trial, so the safety claim cannot be independently checked. The animal work reports the compound as well tolerated [1], which is a statement about mice over sixty days and not about chronic human exposure. Because the compound works by binding a lipid that every mitochondrion in the body contains, effects on healthy tissue cannot be ruled out from the published record. No human half-life, no published pharmacokinetics, no drug interaction data and no reproductive or long-term toxicology are available.
History
SBT-272 came out of Stealth BioTherapeutics, the company built around the Szeto-Schiller mitochondria-targeting peptides. Its predecessor elamipretide (SS-31) reached late-stage trials but its phase 3 in primary mitochondrial myopathy missed its primary endpoints, and one reading of that failure was that the peptide binds cardiolipin relatively weakly and reaches some tissues poorly [2].
SBT-272 was the redesign: keep the cardiolipin mechanism, swap the C-terminal residue for an oxadiazole, and gain mitochondrial uptake and brain exposure so the idea could be tested in neurodegeneration [2][6]. By 2021 it was being tracked in mitochondrial therapy reviews as an ALS candidate [9], and Nature Reviews Drug Discovery listed it in the 2023 ALS pipeline as a phase 1 mitochondria-targeted peptide and peptidomimetic [3].
The FDA granted it orphan drug designation for ALS, announced by the company on 1 November 2022. The primary preclinical paper, a collaboration between Stealth and the Ozdinler laboratory at Northwestern, appeared in Neurobiology of Disease in 2023 [1]. The parent compound elamipretide went on to receive FDA accelerated approval as Forzinity for Barth syndrome in September 2025, the first approved mitochondria-targeted therapeutic, and Stealth now describes bevemipretide as its second-generation clinical-stage candidate for ophthalmic and neurological indications, including a topical formulation being evaluated in dry age-related macular degeneration.
Reputation
Inside the neurodegeneration and mitochondrial-medicine literature SBT-272 has a genuinely good name; it turns up by name in ALS pipeline reviews, cardiolipin reviews and brain mitochondria reviews as the CNS-capable member of the cardiolipin-stabiliser class [3][2][7][4]. The enthusiasm rests on a narrow base. One primary peer-reviewed paper carries the preclinical case, its most-quoted headline is an in-vitro comparison in which SBT-272 restored diseased upper motor neuron health more effectively than edaravone or AMX0035 [1], and the Parkinson's findings that reviews repeat have never been published as a primary study. As a consumer compound it has almost no presence; the research-chemical listings that do exist are recent and thin, and the family resemblance to elamipretide makes mislabelled material a real concern for anyone buying an unverified vial.
Subjective profileweighing the evidence above
The mechanism is real chemistry, not a story. This is the elamipretide scaffold with the tail swapped for an oxadiazole, and elamipretide itself is now an approved drug, so the family is not speculative. What SBT-272 does not have is published human evidence: one primary peer-reviewed animal paper, a phase 1 in healthy volunteers whose only public description is a company press release, and no efficacy result in any patient. Buying a vial does not put a person in that phase 1. There is no established human dose, no published half-life, and nothing that tells you what chronic exposure does to healthy mitochondria. Interesting to follow, not something to take.
Resources
This entry is here for reference.
Research
- 2020first citedThe mitochondria-targeted peptide SS-31 binds lipid bilayers and modulates surface electrostati…
- 2023most active year3 papers
- 2025most recentGlycerophospholipids in ALS: insights into disease mechanisms and clinical implication.
- 1.SBT-272 improves TDP-43 pathology in ALS upper motor neurons by modulating mitochondrial integrity, motility, and function.
- 2.The Role of Cardiolipin in Mitochondrial Function and Neurodegenerative Diseases.
- 3.Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation.
- 4.Glycerophospholipids in ALS: insights into disease mechanisms and clinical implication.
- 5.Mitochondrial therapeutics and mitochondrial transfer for neurodegenerative diseases and aging.
- 6.Cardiolipin and mitochondrial membrane integrity in neurodegeneration: insights from α-synuclein-driven Parkinson's disease.
- 7.Focusing on mitochondria in the brain: from biology to therapeutics.
- 8.Boosting Mitochondrial Potential: An Imperative Therapeutic Intervention in Amyotrophic Lateral Sclerosis.
- 9.Pharmacological advances in mitochondrial therapy.
- 10.The mitochondria-targeted peptide SS-31 binds lipid bilayers and modulates surface electrostatics as a key component of its mechanism of action.
10 listed here; entry last updated August 2026
Reviews
My notesprivate to this device
FAQ
What is SBT-272 designed to do?
It targets mitochondria and stabilizes cardiolipin, a key inner-membrane lipid, to restore energy production and structural integrity in stressed neurons. This is being explored for ALS and other neurodegenerative diseases.
Can SBT-272 be used to treat ALS in people?
No. The evidence is preclinical, from mouse and cell models only. It is an experimental research compound, not an approved therapy.
Is SBT-272 the same thing as elamipretide or SS-31?
No, but it is close family. Elamipretide is D-Arg-Dmt-Lys-Phe-NH2; SBT-272 keeps the first three residues and replaces the terminal phenylalanine amide with a benzyl-oxadiazole. The mechanism is the same cardiolipin stabilisation, and the point of the change was higher mitochondrial uptake and higher brain concentrations [2][6]. Findings reported for elamipretide or SS-31 are not findings for SBT-272 and are labelled separately here.
Has SBT-272 ever been given to a human?
Yes. A phase 1 study in healthy volunteers was conducted, and reviews list the programme at phase 1 [2][3]. The only public account of the result is a November 2022 company announcement saying the selected dose levels appeared generally safe and well tolerated. No results paper has been published and no registration for the trial appears on ClinicalTrials.gov, so there is no independent record of what happened.
Does it work for ALS?
That is unknown. In a hTDP-43 mouse model it improved mitochondrial integrity, motility and function, improved the health of diseased upper motor neurons in vitro, and after sixty days of chronic dosing reduced astrogliosis, microgliosis and TDP-43 pathology in the motor cortex [1]. No trial has tested whether any of that changes the course of the disease in a patient. It holds FDA orphan drug designation for ALS, which is a regulatory incentive and not a statement that the drug works.
What about the Parkinson's results people mention?
Treat them as unpublished. A 2025 review states that SBT-272 reduced dopaminergic neuron loss, alpha-synuclein aggregation and the glial markers Iba1 and GFAP in A53T alpha-synuclein mice [5], but it gives no primary citation for that statement, and the claim traces back to Stealth conference posters. Another 2025 review notes only that whether the compound's pharmacokinetic advantages translate into benefit in Parkinson's remains to be determined [6]. No primary peer-reviewed Parkinson's study for SBT-272 is indexed in PubMed.
Why do PubMed searches only return one paper?
Because PubMed matches the string SBT-272 mainly in titles, abstracts and keywords, and only the 2023 primary paper puts it there. Full-text search across the same literature finds it discussed by name in a further eight PubMed-indexed reviews. One paper being the only primary study is true; one paper being the only literature is a search artefact.
Limitations of the evidence
- No human safety data; effects on healthy mitochondria not fully characterized
- Evidence limited to a single primary preclinical study
- One primary peer-reviewed study carries the entire preclinical case [1]
- The phase 1 result exists only as a company announcement; no results paper and no ClinicalTrials.gov registration
- No published half-life, pharmacokinetics or human dose
- The Parkinson's findings repeated in reviews have no primary peer-reviewed source [5]
- A 2025 review states the mouse study improved survival [4]; the primary abstract makes no survival claim, so no survival benefit should be assumed
- Effects of long-term cardiolipin binding on healthy mitochondria are not characterised
Adverse effects
- No adverse-event profile has been published for SBT-272 in humans or animals; the source study reports only that the compound was well tolerated in mice [1]
Notes and cautions
- International nonproprietary name: bevemipretide. CAS 2356106-71-1, C31H45N9O4, molecular weight 607.7, PubChem CID 138688997, IUPHAR/GtoPdb ligand 13258.
- The compound is the SS-31 scaffold with a 3-benzyl-1,2,4-oxadiazole in place of the C-terminal phenylalanine amide.
- Its parent elamipretide was approved by the FDA as Forzinity for Barth syndrome in September 2025, the first approved mitochondria-targeted therapeutic.
- Stealth currently describes bevemipretide as a second-generation candidate for ophthalmic and neurological indications, including a topical formulation in dry age-related macular degeneration.
- Sold by Umbrella Labs as a 25 mg lyophilised vial for laboratory research use only.