Elamipretide Peptide CAS 736992-21-5

SS-31 Introduction

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Elamipretide is a small molecule peptide composed of four amino acids, targeting the mitochondrial inner membrane of SS-31 powder in vivo, which can prevent oxidative damage to nerve cells and other cell types; Pure MTP 131 powder prevents mitochondrial depolarization, reduces pancreatic cell apoptosis, increases pancreatic cell production, and improves post transplant function. Additionally, Elamipretide is also undergoing clinical research for other diseases such as dry age-related macular degeneration and primary mitochondrial myopathy.Elamipretide peptide powder is a white to off white hygroscopic powder, and special attention should be paid during storage.Its CAS number is 736992-21-5; molecular formula is C32H49N9O5; molecular weight is 639.79; EINECS number is 205-858-1.

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What are the unique features of the mechanism of action of Elamipretide?

Elamipretide Peptide is a synthetic peptide targeting mitochondria. It is also one of the first active peptides approved by the FDA that specifically intervene in mitochondrial dysfunction. It has a fixed amino acid sequence and its active core targets CARDIOLIPIN FROM BOVINE HEART SODIUM, which is located in the inner mitochondrial membrane and is a key lipid that ensures the structural integrity of mitochondria and the normal production of energy. Disorders related to mitochondria, such as Barth syndrome, often result from damaged cardiolipin, directly causing a decline in mitochondrial energy production efficiency.
SS-31 functions as a stabilizing regulator of mitochondria, binding to damaged cardiolipin, stabilizing the respiratory chain complex, restoring electron transfer efficiency, and reducing free radical generation. Its multiple effects work together to repair mitochondrial morphology and optimize energy metabolism. Unlike many substances that only alleviate external manifestations, it directly acts on the core cellular organelles involved in energy metabolism, improving mitochondrial dysfunction problems at the source.

Which disease has MTP-131 been approved for? What is the actual clinical effect like?

Based on its unique mechanism of action, Elamipretide Peptide is mainly used in the research of various mitochondrial dysfunction-related diseases. This peptide was granted accelerated approval by the US FDA on September 19, 2025, for improving the muscle strength of patients with Bart syndrome. Bart syndrome is a rare X-linked genetic mitochondrial disease. Genetic mutations cause abnormal remodeling of cardiolipin, and the typical symptoms include cardiomyopathy, muscle weakness, and persistent fatigue.
The core basis of this approval is data from an open-label extension trial lasting 168 weeks. The results showed that the six-minute walking distance of the subjects who continued to take the medication showed a stable increase, with a cumulative average increase of 96.1 meters. Fatigue perception and heart-related indicators were also improved. The most common adverse reaction of this component is mild injection site discomfort, and the overall tolerance is good.
It should be noted that this is an accelerated approval model. The regulatory agency allows early market launch based on alternative endpoints that can predict clinical benefits. The pharmaceutical company still needs to conduct confirmatory trials to fully confirm the long-term clinical benefits. In addition, a Phase III clinical trial for dry age-related macular degeneration is also underway.

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FAQ:

Q1:What is the fundamental difference between Elamipretide and ordinary antioxidant components?
Most common antioxidants merely indiscriminately eliminate free radicals in the cytoplasm, merely addressing the symptoms rather than the root cause. Elamipretide, on the other hand, directly targets the source of mitochondrial damage, combines with damaged cardiolipin, repairs the inner membrane structure, and stabilizes the respiratory chain. It reduces oxidative stress at the root and restores energy metabolism, representing a structural repair rather than a simple free radical clearance.
Q2:How does the safety and side effects of Elamipretide manifest?

The overall tolerance is excellent, and there are very few adverse reactions in the clinical trials. The most common ones are only mild transient reactions at the injection site, without serious liver or kidney toxicity, no obvious systemic side effects. Its safety is far superior to that of traditional hormone-based and metabolite-based products.

Q3:Apart from Barth syndrome, what other research indications does it have?

At present, several phase III clinical trials are underway. The most representative one is dry age-related macular degeneration. At the same time, there is a large amount of scientific research data supporting it in the fields of heart failure, mitochondrial myopathy, neurodegenerative injury, and ischemia-reperfusion injury.

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