Elamipretide hydrochloride is under clinical development by Stealth BioTherapeutics and currently in the Phase I and Phase II in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Elamipretide hydrochloride’s likelihood of approval (LoA) and phase transition for Geographic Atrophy took place on 01 Jul 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Elamipretide hydrochloride Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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.

Quick View Elamipretide hydrochloride LOA Data

Report Segments
  • Innovator
Drug Name
  • Elamipretide hydrochloride
Administration Pathway
  • Intravenous
  • Ophthalmic
  • Oral
  • Subcutaneous
Therapeutic Areas
  • Cardiovascular
  • Central Nervous System
  • Genetic Disorders
  • Genito Urinary System And Sex Hormones
  • Metabolic Disorders
  • Musculoskeletal Disorders
  • Ophthalmology
Key Developers
Highest Development Stage
  • Filing rejected/Withdrawn

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.