Treatments in Phase 1 Trials for FA
CTI-1601 is an investigational therapy designed to deliver the frataxin protein to the mitochondria of Friedreich’s ataxia patients. Dosing was completed in a Phase 1 trial in December 2020, and top-line data are expected in the second quarter of 2021.
Methylprednisolone is a steroid used to reduce inflammation, and may be able to help ease the symptoms of Friedreich’s ataxia. A Phase 1 trial was completed in 2019, but it did not meet its primary objective, with results showing no clear benefit for FA patients. No information is available yet on further studies.