| Size | Price | Stock | Qty |
|---|---|---|---|
| 5mg |
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| Targets |
Mitochondrial cardiolipin
Elamipretide TFA targets the inner mitochondrial membrane, specifically interacting with cardiolipin. Cardiolipin is a phospholipid unique to mitochondria that is essential for maintaining the structure and function of the mitochondrial membrane. By binding to cardiolipin, elamipretide stabilizes the mitochondrial membrane, reduces the production of reactive oxygen species (ROS), and improves mitochondrial bioenergetics. |
|---|---|
| ln Vitro |
Barth syndrome is a rare X-linked genetic disorder characterized by mitochondrial dysfunction. Elamipretide is a mitochondrial cardiolipin binder that penetrates cells and accumulates in the mitochondria: It localizes to the inner mitochondrial membrane, improving mitochondrial morphology and function. Elamipretide's mechanism of action involves electrostatic interactions with cardiolipin, a phospholipid critical for mitochondrial structure and electron transport chain function. By binding to cardiolipin, elamipretide stabilizes the mitochondrial membrane, prevents oxidative damage, and maintains membrane potential. This interaction preserves cristae integrity, reduces reactive oxygen species (ROS) production, and maintains ATP production. Elamipretide also enhances the activity of mitochondrial respiratory complexes, facilitating efficient electron transfer and ATP synthesis. Furthermore, elamipretide inhibits the opening of the mitochondrial permeability transition pore (mPTP), protecting against mitochondrial damage during reperfusion and reducing cell death in ischemic conditions.
In vitro, Elamipretide TFA protects cells from oxidative stress injury. It has been shown to reduce ROS production and improve mitochondrial function in various cell types. Its ability to stabilize the mitochondrial membrane and inhibit cardiolipin peroxidation is key to its cytoprotective effects. |
| ln Vivo |
Elamipretide is a mitochondrial cardiolipin binder that localizes to the inner mitochondrial membrane to improve mitochondrial morphology and function. Elamipretide was shown to attenuate neuronal oxidative stress and neuroinflammation, activate neural mitochondrial biogenesis, enhance mitochondrial respiration, and protect against neural apoptosis. Protective effects of elamipretide against neuronal loss and inflammation have been reported in traumatic brain injury, hind limb ischemia–reperfusion injury, type II diabetes, and spinal cord injury. Clinically significant QTc interval prolongation was not observed at three times the peak concentration of the maximum recommended dose.
Elamipretide is a mitochondrial cardiolipin binder being investigated for diseases involving mitochondrial dysfunction. Elamipretide is a synthetic tetrapeptide that selectively binds to cardiolipin, a phospholipid in the inner mitochondrial membrane. This interaction improves mitochondrial morphology and function. In September 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval for FORZINITY™ (elamipretide) to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. ELAMIPRETIDE is a Protein drug with a maximum clinical trial phase of III (across all indications) and has 10 investigational indications.
In vivo, Elamipretide TFA has demonstrated efficacy in animal models of various diseases. It ameliorates myocardial infarction, improves renal function, and protects neurons. Its mitochondria-targeting properties allow it to accumulate in tissues with high energy demands, such as the heart, kidney, and brain, where it exerts its protective effects. |
| Enzyme Assay |
In vitro assays for Elamipretide TFA typically involve measuring its effects on mitochondrial function. Cells are treated with the peptide, and parameters such as mitochondrial membrane potential, ROS production, and ATP levels are measured using fluorescent dyes and biochemical assays.
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| Cell Assay |
For cellular assays, cells are treated with Elamipretide TFA, and its protective effects are assessed following a stressor, such as oxidative stress or ischemia. Cell viability is measured using standard assays like MTT or CellTiter-Glo. The compound's effects on mitochondrial morphology and function are also evaluated.
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| Animal Protocol |
In vivo efficacy is evaluated in animal models of disease. For example, in a model of myocardial infarction, animals are treated with Elamipretide TFA, and infarct size and cardiac function are assessed. In models of kidney disease, renal function and histology are evaluated. The peptide is typically administered via injection.
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| ADME/Pharmacokinetics |
Absorption
After daily subcutaneous injection of 2 to 80 mg, ilapipritide exposure increases proportionally with increasing dose, with minimal accumulation. Maximum ilapipritide concentrations are reached 0.5 to 1 hour after subcutaneous injection. The absolute bioavailability after subcutaneous injection is approximately 92%. Exposure is comparable after subcutaneous injection into the thigh or abdomen. Elimination Route Ilamipretide and its metabolites M1 and M2 are excreted in the urine. In patients with normal renal function, approximately 100% of the ilapipritide dose is recovered in the urine 48 hours after administration, present as ilapipritide, M1, or M2. Volume of Distribution Ilamipretide is distributed in systemic water, with a volume of distribution of approximately 0.5 L/kg. Protein Binding Protein binding is approximately 39%. Metabolism/Metabolites Elamipretide is metabolized via C-terminal degradation into M1 tripeptide and M2 dipeptide metabolites, which are not pharmacologically active. Its metabolic pathway is not fully elucidated. Elamipretide TFA has a molecular weight of 753.81 g/mol and a chemical formula of C34H50F3N9O7. As a peptide, it is not orally bioavailable and must be administered parenterally. It is expected to have a short half-life in circulation but can accumulate in mitochondria-rich tissues. |
| Toxicity/Toxicokinetics |
Elamipretide TFA is generally well-tolerated in preclinical studies at therapeutic doses. As a mitochondria-targeting peptide, its toxicity is related to its mechanism of action. Its safety profile is evaluated in toxicology studies to determine the therapeutic window.
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| References | |
| Additional Infomation |
Barth syndrome primarily affects men, typically presenting in infancy, manifesting as severe heart failure, and leading to premature death. Patients who survive into adolescence and adulthood often experience fatigue, decreased physical strength, and exercise intolerance. Quality of life and daily functioning are significantly impacted throughout a person's life due to Barth syndrome. Forzinity's mechanism of action involves binding to the mitochondrial interior, improving mitochondrial structure and function. The FDA has granted Forzinity accelerated approval. This pathway allows for earlier approval of drugs that treat serious diseases and address unmet medical needs, based on metrics that reasonably predict patient benefit but do not directly assess that benefit. Forzinity's accelerated approval is based on its ability to improve muscle strength used to straighten the knee. The FDA considers this improvement to reasonably predict patient benefit, such as easier standing or walking further. As a condition of accelerated approval, the FDA required the manufacturer of Forzinity to conduct a post-marketing randomized, double-blind, placebo-controlled trial to confirm that the observed improvement in knee muscle strength translates into patient benefit. Forzinity is administered subcutaneously once daily. The most common side effect observed in clinical trials is mild to moderate injection site reaction. There have also been reports of serious adverse reactions to Forzinity.
Elamipretide TFA is a mitochondria-targeting peptide that ameliorates myocardial infarction, improves renal function, and protects neurons from inflammatory and oxidative stress injury. It is a promising therapeutic candidate for diseases involving mitochondrial dysfunction. |
| Molecular Formula |
C34H50F3N9O7
|
|---|---|
| Molecular Weight |
753.812118053436
|
| Exact Mass |
753.378
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| CAS # |
1606994-55-1
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| Related CAS # |
736992-21-5;1334953-95-5 (acetate);2244098-12-0 (HCl); 1606994-55-1
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| PubChem CID |
137795331
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| Sequence |
D-arginyl-2,6-dimethyl-D-tyrosyl-D-lysyl-L-phenylalaninamide trifluoroacetic acid; H-D-Arg-D-Tyr(2,6-diMe)-D-Lys-Phe-NH2.2TFA
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| SequenceShortening |
RXKF
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| Appearance |
White to off-white solid powder
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| Hydrogen Bond Donor Count |
10
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| Hydrogen Bond Acceptor Count |
13
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| Rotatable Bond Count |
19
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| Heavy Atom Count |
53
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| Complexity |
1070
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| Defined Atom Stereocenter Count |
0
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| SMILES |
FC(C(=O)O)(F)F.O=C(C(CC1C(C)=CC(=CC=1C)O)NC(C(CCC/N=C(\N)/N)N)=O)NC(C(NC(C(N)=O)CC1C=CC=CC=1)=O)CCCCN
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| InChi Key |
WLZHRKGRZBGUJU-UHFFFAOYSA-N
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| InChi Code |
InChI=1S/C32H49N9O5.C2HF3O2/c1-19-15-22(42)16-20(2)23(19)18-27(41-29(44)24(34)11-8-14-38-32(36)37)31(46)39-25(12-6-7-13-33)30(45)40-26(28(35)43)17-21-9-4-3-5-10-21;3-2(4,5)1(6)7/h3-5,9-10,15-16,24-27,42H,6-8,11-14,17-18,33-34H2,1-2H3,(H2,35,43)(H,39,46)(H,40,45)(H,41,44)(H4,36,37,38);(H,6,7)
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| Chemical Name |
6-amino-2-[[2-[[2-amino-5-(diaminomethylideneamino)pentanoyl]amino]-3-(4-hydroxy-2,6-dimethylphenyl)propanoyl]amino]-N-(1-amino-1-oxo-3-phenylpropan-2-yl)hexanamide;2,2,2-trifluoroacetic acid
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| Synonyms |
MTP-131 TFA; 1334953-96-6; Elamipretide 2TFA; Elamipretide ditrifluoroacetate; MTP131 trfluoroacetic acid; RX31; SS31
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
May dissolve in DMSO (in most cases), if not, try other solvents such as H2O, Ethanol, or DMF with a minute amount of products to avoid loss of samples
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|---|---|
| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 1.3266 mL | 6.6330 mL | 13.2659 mL | |
| 5 mM | 0.2653 mL | 1.3266 mL | 2.6532 mL | |
| 10 mM | 0.1327 mL | 0.6633 mL | 1.3266 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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