Elamipretide hydrochloride is under clinical development by Stealth BioTherapeutics and currently in Phase I for Duchenne Muscular Dystrophy. According to GlobalData, Phase I drugs for Duchenne Muscular Dystrophy have an 84% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how Elamipretide hydrochloride’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Elamipretide hydrochloride overview

Elamipretide hydrochloride (Bendavia) is under development for the treatment of primary mitochondrial diseases including mitochondrial myopathy, Senger syndrome, Barth syndrome, Leber’s hereditary optic neuropathy, dry age-related macular degeneration with geographic atrophy, Duchenne muscular dystrophy, Friedreich ataxia, Becker muscular dystrophy and other cardiomyopathies. It is administered through intravenous, oral, ophthalmic and subcutaneous routes. It is a new chemical entity that targets mitochondria and binds to mitochondrial membrane permeability transition pore (mPTP). It was also under development for diseases associated with aging including acute heart failure with reduced ejection fraction (HFrEF) and preserved rejection failure (HFpEF), endothelial dysfunction induced by a single cigarette, Fuchs endothelial corneal dystrophy, acute kidney injury, ischemic reperfusion injury, diabetic macular edema, and acute coronary syndrome.

Stealth BioTherapeutics overview

Stealth BioTherapeutics, formerly Stealth Peptides International, a subsidiary of Morningside Venture Investments Ltd, is an early stage biotechnology company. It discovers, develops, and commercializes novel therapies for diseases and disorders associated with mitochondrial dysfunction. The company is investigating elamipretide, a peptide compound that penetrates into cell membranes, for the treatment of primary mitochondrial myopathy, barth syndrome, leber’s hereditary optic neuropathy, duchenne cardiomyopathy, and dry age-related macular degeneration. It is also evaluating SBT-20 for the treatment of neurodegenerative diseases related to mitochondrial dysfunction; and SBT-272, a phase I candidate, for the treatment of rare neurodegenerative diseases. Stealth BioTherapeutics is headquartered in Grand Cayman, Cayman Islands.

For a complete picture of Elamipretide hydrochloride’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.