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Stealth BioTherapeutics intiates Phase I study with a novel compound targeting mitochondrial dysfunction in retinal disorders.

Stealth BioTherapeutics Inc., a biotechnology company based in Newtown, Massachusetts, have announced the initiation of a Phase I study in AMD to test the safety and tolerability of a water-soluble tetrapeptide, “elamipretide”, a compound designed to target mitochondrial dysfunction. The study, named “ReCLAIM”, is an open-label study evaluating 12 weeks of treatment with daily subcutaneous injections of 40mg elamipretide in patients aged 55 years or older with a diagnosis of intermediate AMD. The study represents a new line of attack focused on mitochondrial biology and the company states that their approach may have benefit for other ocular disorders including Leber’s Hereditary Optic Neuropathy (LHON), dry age related macular degeneration and Fuchs Corneal Endothelial Dystrophy.


According to research supported by the company, elampretide (previously referred to as MTP-131, SS-31 or Bendavia) is a water-soluble tetrapeptide that selectively targets mitochondrial cardiolipin, while simultaneously promoting efficient electron transfer and ATP recovery. The tetrapeptide is understood to selectively target the inner mitochondrial membrane (IMM) and inhibit mitochondrial permeability transition however, the exact mode of action is yet to be fully characterised. Stealth BioTherapeutics distinguishes itself through its exclusive focus on mitochondrial disease. The company states there are in excess of 200 mitochondrial disorders, including LHON, mitochondrial encephalomyopathy, lactic acidosis, stroke-like episodes (MELAS), Barth Syndrome, among many others. According to the company, there are no FDA-approved treatments for any rare mitochondrial disease and therefore a significant demand for effective therapies currently remains unmet by the pharmaceutical industry.


Topline data on the Phase I trial are due to be available by mid 2017 and the compound is currently under-going testing for other indications, including chronic heart failure. Commenting on the Phase I initiation milestone for intermediate AMD, Dr. Scott Cousins, the trial investigator, and Professor of Ophthalmology and Director of the Duke University Center for Macular Diseases stated, “there are currently no FDA-approved treatment options for dry AMD, so we are eager to better understand the effect that elamipretide may have in treating these roughly 13 million patients. Mitochondrial dysfunction that stems from various environmental toxins may be an important causative factor in dry AMD, and in laboratory models, elamipretide appears to prevent mitochondrial dysfunction in the retinal pigment epithelium.”