Stealth to develop mitochondrial therapeutic for Friedreich's ataxia

Stealth BioTherapeutics Inc., a biotech company in clinical stage, and Friedreich’s Ataxia Research Alliance (FARA) have announced that Stealth has received the Kyle Bryant Translational Research Award from FARA to study SBT-589 in Friedreich’s ataxia (FA), a disease related to mitochondrial dysfunction.

The preclinical research will help guide the progress of SBT-589 for FA neuropathy. Dysfunction of the mitochondria is key to the disease process in FA and results in deficiencies in iron control, energy production, and responses to oxidative stress. These disturbances may decrease the level at which iron-induced cell death occurs, resulting in early loss of neuronal cells during the disease.

Preclinical study has demonstrated that SBT-589 specifically focuses on addressing mitochondrial dysfunction in order to enhance bioenergetics, alleviate issues related to iron dysregulation, and decrease the production of reactive oxygen species (ROS). In the research, SBT-589 effects on neurological and biochemical aspects of disease will be examined in a transgenic mouse model of FA neuropathy.

“We are grateful for our continued partnership with FARA to determine the therapeutic potential of SBT-589 for individuals living with Friedrich’s ataxia,” said David A. Brown, Senior Vice President of Discovery at Stealth. “This collaboration will enable further development of SBT-589 as a potential new treatment that directly targets mitochondrial pathology in FA.  Stealth has a diverse pipeline of molecules designed to improve cellular energy production, and this award is expected to bolster the progression of one of our lead candidates, SBT-589, into clinical testing”