Stealth BioTherapeutics ADRs Up After FDA Orphan-Drug Designation

 Stealth BioTherapeutics Corp.'s American depositary receipts were up 7.2% to 76 cents in premarket trade after it said the U.S. Food and Drug Administration granted orphan-drug designation to elamipretide for the treatment of patients with Friedreich's ataxia.

The company said a Phase 2a trial evaluating elamipretide for Friedreich's ataxia has been initiated at Children's Hospital of Philadelphia. The trial is evaluating two doses of elamipretide in patients with Friedreich's ataxia to assess safety, visual function and cardiac function.

Friedreich's ataxia is a rare genetic disease affecting an estimated 4,000 in the U.S.

The disease is caused by a defect in the frataxin gene resulting in a relative deficiency of frataxin, leading to mitochondrial iron accumulation and oxidative stress. Most patients with Friedreich's ataxia experience a progressive decline in visual function. Progressive cardiomyopathy, which affects more than 90% of individuals with this disease, usually becomes fatal by early adulthood and is the leading cause of early mortality.

Elamipretide has been shown to improve frataxin levels in Friedreich's ataxia patient-derived cells and to improve frataxin levels and motor and cardiac function in an animal model of Friedreich's ataxia, the company said.

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