Treatments in Phase 2 Trials for FA

Epicatechin is a naturally occurring compound that has been identified as a potential treatment for Friedreich’s ataxia. The flavonols (+)-epicatechin and (-)-epicatechin are found in very small amounts in wine, dark chocolate, and green tea. Results of a small Phase 2 trial were published in September 2020.

Etravirine is an anti-viral medication approved by the FDA to treat human immunodeficiency virus (HIV) infections. A screening study showed that it may have benefits for the treatment of Friedreich’s ataxia as well. A Phase 2 trial, launched in March 2020, is currently recruiting.

Nicotinamide, or vitamin B3, is being studied as a potential treatment for Friedreich’s ataxia. Initial study results show that it may improve frataxin production but that this may not translate into easing symptoms of the disease. A Phase 2 trial testing nicotinamide was recruiting FA patients in Europe as of December 2019.

Leriglitazone, or MIN-102, is an oral medication being developed for treating several diseases of the brain and spinal cord, including Friedreich’s ataxia. It has shown potential to treat the disease in recent animal and cell studies, and positive top-line results from a Phase 2 trial were released in December 2020.

Resveratrol is a natural compound found in the skin of red grapes, peanuts, blueberries, and some other berries. It is a very powerful antioxidant that helps prevent cell damage caused by free radicals. A Phase 2 trial studying micronized resveratrol is currently recruiting Friedreich’s ataxia patients.