Mitoprotective Agent for Treating Mitochondrial Disease

Bendavia targets mitochondrial dysfunction, a key element believed to be at the root of a variety of serious, debilitating diseases. Bendavia has been developed to treat an underlying defect in mitochondria to reduce oxidative stress and increase energy supply to affected cells and organs.

Bendavia penetrates the cellular and outer mitochondrial membranes, and targets cardiolipin, which is found exclusively in the inner mitochondrial membrane. Bendavia has been shown to positively impact dysfunctional mitochondria in nonclinical studies, with no effect in healthy mitochondria. In this way, Bendavia may address mitochondrial disease and dysfunction, where no FDA-approved treatments currently exist.

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 nonclinical studies, Bendavia 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, Bendavia has been shown to promote energy production and reduce the production of excess ROS.
mitochondrial myopathy treatment


Bendavia:  Pioneering the Development of Mitochondrial Drugs

Bendavia is currently in multiple Phase 1 and 2 clinical studies for a variety of diseases, both common and rare.  Data from these clinical trials, enrolling more than 400 subjects, suggest that Bendavia is safe and well-tolerated in those patients and conditions studied.

Therapeutic Potential

In common diseases, dysfunctional mitochondria are believed to be a principal source of oxidative stress, which may lead to inflammation1 and other harmful effects, such as reduced mitochondrial bioenergetics. Bendavia may benefit dysfunctional mitochondria with the therapeutic potential to modify disease and improve patient outcomes in diseases including:

  • Heart failure2
  • Kidney disease3
  • Cardiovascular and metabolic diseases4
  • Neurodegeneration5
  • Certain skeletal muscle disorders6

Charting New Territory in Patient Outcomes and Clinical Practice

Currently, there are no FDA-approved treatments for 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, Bendavia, has been designed to treat the underlying defect in mitochondria with the intention of reducing oxidative stress and increasing energy supplies to affected cells and organs.

Studies in Orphan Mitochondrial Diseases

In nonclinical studies to evaluate its effect on genetic mitochondrial diseases, Bendavia restored mitochondrial function in patient fibroblasts.  Clinically, Bendavia is being studied in patients with mitochondrial myopathy (myo means muscle, and pathos means disease), which includes multiple identified mitochondrial diseases.  Mitochondrial myopathies are caused by mutations, or changes, in genes. They are inheritable, although they can occur with no family history, and they often affect members of the same family in different ways.  Muscle weakness, exercise intolerance, trouble with balance, and impaired coordination are common features of mitochondrial myopathy (MM) given that muscle cells have particularly high energy needs.6


  1. The mitochondrion — a Trojan horse that kicks off inflammation? New England Journal of Medicine. 2010 Jun 3;362(22):2132-4. doi: 10.1056/NEJMcibr1003521.
  2. The Failing Heart an Engine Out of Fuel. New England Journal of Medicine. March 15, 2007 356:1140-1151.
  3. Mitochondrial Biogenesis in Kidney Disease. JASN. 2011 Mar;22(3):431-6. doi: 10.1681/ASN.2010060643. Epub 2011 Feb 25.
  4. Normalizing mitochondrial superoxide production blocks three pathways of hyperglycaemic damage. Nature. 2000 Apr 13;404(6779):787-90.
  5. Reeve, A.K., Krishnan, K.J., Duchen, M.R., Turnbull, D.M. Mitochondrial Dysfunction in Neurodegenerative Disorders. 2012.
  6. Mitochondrial Myopathies.

Bendavia™ in Clinical Trials

For more information about clinical trials, visit