Stealth Biotherapeutics: New Treatments for Genetic Mitochondrial Diseases

Stealth Biotherapeutics is one of a new group of pharmaceutical companies developing drug treatments for genetic mitochondrial disease and acquired mitochondrial dysfunction, including heart failure, diabetic macular edema, certain skeletal muscle disorders, and rare inherited diseases such as Leber’s Hereditary Optic Neuropathy (LHON).

Treating Mitochondrial Dysfunction

Mitochondrial disorders are believed to arise either as a result of inherited genetic defects or through damage caused by a variety of common diseases. In either case, the result is the creation of oxidative stress and reduced energy production. Dysfunctional mitochondria are a key element in a variety of serious, debilitating diseases, both rare and common, such as:  

  • Heart failure
  • Age-related macular degeneration
  • Kidney disease
  • Cardiovascular and metabolic diseases
  • Diabetic macular edema and retinopathies
  • Neurodegeneration
  • Certain skeletal muscle disorders

Stealth’s Drug Pipeline

Bendavia – As of January 2016, Stealth received Fast Track designation by the FDA for its leading drug candidate, Bendavia, for the treatment of primary mitochondrial myopathy. Stealth markets Bendavia as an investigational drug with the potential to modify disease by preserving energetics and restoring normal energy production in mitochondria, while decreasing oxidative stress. According to Stealth, 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.

Ocuvia – In nonclinical studies, Ocuvia has demonstrated the potential to be the first investigational topical ophthalmic formulation to treat back-of-eye diseases, offering a an alternative to treatments that require physician-administered ocular injections. Ocuvia is designed to treat the underlying defects in mitochondria associated with diseases such as Diabetic Macular Edema, genetic Mitochondrial Optic Neuropathies and Age-related Macular Degeneration. These defects include excessive oxidative stress and decreased energy supply to affected cells of the eye, particularly in the retina. Ocuvia is currently in clinical studies for patients with Diabetic Macular Edema. Plans are also in development for Ocuvia to be studied in Mitochondrial Optic Neuropathies including Leber’s Hereditary Optic Neuropathy (LHON).

Clinical Studies


  • ReVIEW Diabetic Macular Edema A dose-escalation study to evaluate the safety, tolerability and efficacy of Ocuvia to treat patients with diabetic macular edema (DME) and dry age-related macular degeneration (AMD)
  • ReSIGHT Mitochondrial Optic Neuropathies – Leber’s Hereditary Optic Neuropathy A study to evaluate the safety, tolerability and efficacy of Ocuvia to treat patients with Leber’s Hereditary Optic Neuropathy (LHON)

Skeletal Muscle Diseases

  • MOTION for Skeletal Muscle Disorders – A randomized, double-blind, placebo-controlled study to evaluate Bendavia on skeletal muscle function in the elderly
  • MMPOWER for Mitochondrial Myopathy – A double-blind, placebo-controlled study to evaluate the safety, tolerability and efficacy of Bendavia to treat myopathy in patients with genetically-confirmed mitochondrial disease

Cardio-renal Diseases

  • PREVIEW for Heart Failure – A double-blind, placebo-controlled study to evaluate safety, tolerability and efficacy of Bendavia to treat heart failure patients
  • EVOLVE for Acute Kidney Injury – A double-blind, placebo-controlled study to evaluate safety, tolerability and efficacy of Bendavia in chronic kidney disease (CKD) patients at risk of acute kidney injury (AKI)

Currently, there are no FDA-approved treatments for any orphan mitochondrial disease. This represents a significant unmet need for medicines that can restore mitochondrial function and work by improving energy supply at the cellular level. Stealth Biotherapeutics is focused on developing innovative treatments that can meet these needs.

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