Systemic Delivery

Targeted Agent for Treating Mitochondrial Disease (Subcutaneous and IV Delivery Methods)

The systemic delivery of elamipretide targets mitochondrial dysfunction, a key element believed to be at the root of a variety of serious and debilitating diseases. Elamipretide has been developed to treat dysfunctional mitochondria by restoring energy production and reducing harmful oxidative stress, thereby increasing energy supply to affected cells and organs.8

Elamipretide penetrates the cellular and outer mitochondrial membranes, and targets cardiolipin, which is found exclusively in the inner mitochondrial membrane.6 Elamipretide has been shown in preclinical studies to positively affect dysfunctional mitochondria, with no effect on healthy mitochondria. Elamipretide may address diseases that are primarily caused by mitochondrial dysfunction as well as diseases in which mitochondrial dysfunction is a contributor to the disease process.6,39,42

Reaching the Inner Mitochondrial Membrane

Developing drugs that target mitochondria is challenging because it is difficult to penetrate cell and outer mitochondrial membranes. In multiple preclinical studies, the systemic delivery of elamipretide has been shown to reach the inner mitochondrial membrane and target cardiolipin, a lipid critical to maintaining mitochondrial function and cellular energy supply. In these studies, elamipretide has been shown to promote energy production and reduce the production of excess ROS.6,39,42

Elamipretide (Systemic Delivery)
Elamipretide uniquely reaches the inner membrane of damaged mitochondria by penetrating first the cell membrane, and then the mitochondrial outer membrane. In preclinical and clinical studies, elamipretide has shown no effect on healthy mitochondrial or cellular function.6,39,42


Elamipretide: Pioneering the Development of Mitochondrial Drugs

Elamipretide has been studied in multiple phase 1 and 2 clinical studies for a variety of diseases, both rare and common. Data from these clinical trials suggest that elamipretide is well-tolerated in those patients and conditions studied.19,22

Therapeutic Potential

In common diseases, dysfunctional mitochondria are believed to be a principal source of oxidative stress, which may lead to harmful effects, particularly in organs that require the greatest energy to function. Elamipretide may restore bioenergetics to dysfunctional mitochondria, thus providing the therapeutic potential to modify disease and improve patient outcomes in diseases including:37

  • Genetic (inherited) mitochondrial diseases
  • Ophthalmic disorders
  • Neurodegenerative diseases
  • Skeletal muscle disorders
  • Metabolic and diabetic diseases
  • Heart failure
  • Renal disease

Charting New Territory in Patient Outcomes and Clinical Practice

Currently, there are no FDA-approved treatments for patients with inherited orphan mitochondrial diseases or addressing mitochondrial dysfunction associated with certain common diseases. Stealth BioTherapeutics is developing investigational drugs to address mitochondrial dysfunction. One of these drugs, elamipretide, has been designed to treat the underlying defect in mitochondria with the intention of reducing oxidative stress and increasing the quantity of energy supplied to affected cells and organs.

Studies in Primary Mitochondrial Diseases

One common characteristic of rare mitochondrial disease is mitochondrial myopathy (myo means muscle, and pathos means disease). Mitochondrial myopathy is characterized by muscle weakness, exercise intolerance and impaired coordination. Mitochondrial myopathies are caused by mutations, or changes, in genes. They are inheritable, although they can also occur with no family history and can affect members of the same family in different ways.37 Like mitochondrial myopathy, Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene, and characterized by muscle weakness, recurrent infections and delayed growth. The systemic delivery of elamipretide is being studied in patients with mitochondrial myopathy and Barth syndrome, given that muscle cells have particularly high energy needs.

Elamipretide in Clinical Trials

For more information about clinical trials, visit and search “Stealth BioTherapeutics.”