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HOPE for FATIGUE

Raptor Pharmaceuticals' RP103: Treatment Hope for Leigh Syndrome?

Raptor Pharmaceuticals is dedicated to developing medicines that alter cellular metabolic pathways for patients with rare, debilitating, and potentially fatal diseases, including inherited mitochondrial disorders. Raptor has two FDA-approved medications: RP103 (PROCYSBI) and MP376 (QUINSAIR). RP103 was the first brought to market for the treatment of Nephropathic Cystinosis, a rare metabolic disease. It is now being studied for an additional indication in the inherited mitochondrial disease, Leigh Syndrome.

About Leigh Syndrome

Leigh syndrome is a severe neurological disorder caused by genetic defects in mitochondrial or nuclear DNA affecting respiratory function that typically results in death within the first decade of life. The condition causes increased production of free radicals, which disrupts mitochondrial electron transport and affects cellular function in a variety of tissues. Typically observed during the first year of life, Leigh syndrome is characterized by a failure to thrive, lack of coordination, involuntary and sustained muscle contraction, muscle wasting, and multiple organ failure. Currently, there are no approved treatments for Leigh syndrome.

RP103 for the Treatment of Mitochondrial Diseases

Raptor is currently conducting a Phase 2/3 clinical trial to test the safety and efficacy of RP103 in children with Leigh Syndrome and other mitochondrial disorders.

RP103 is believed to increase mitochondrial glutathione, which acts as a scavenger for free radicals in the body. Free radicals are a byproduct of mitochondrial energy production and place stress on cells. In patients with mitochondrial dysfunction, these free radicals are produced at dangerously high levels. By increasing levels of the antioxidant glutathione in the mitochondria, RP103 may reduce the toxic stress typically associated with mitochondrial disorders.

Raptor Pharmaceutical’s Clinical Development Pipeline

Raptor has two active clinical development programs based on its proprietary formulations of cysteamine bitartrate.

RP103 for the potential treatment of:

  • Huntington’s disease, an inherited neurodegenerative disease
  • Mitochondrial diseases, including Leigh syndrome

Raptor also has two active clinical development programs in expanded indications for MP-376.

  • Bronchiectasis (BE) lung infections
  • Mycobacteria (NTM) lung Infections

Raptor’s has one further ongoing clinical development program:

  • Convivia® (oral 4-methylpyrazole) for the potential management of acetaldehyde toxicity due to ALDH2 deficiency, an inherited metabolic disorder (Phase 2)

With no currently available treatment for Leigh Syndrome, hopes are running high for a successful outcome of Raptor’s RP103-MITO-001 trial. "Leigh syndrome is one of the most devastating of mitochondrial disorders and the RP103-MITO-001 trial will explore a new mechanism of action against this deadly illness," said the trial's lead investigator, Bruce H. Cohen, M.D., Professor of Pediatrics and Director of Neurology at Akron Children's Hospital.

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