Friedreich’s Ataxia Clinical Study of Oral Drug, RT001, Enrolling New Cohort of Patients

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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neuronal loss in FA

Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its ongoing, 28-day clinical study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. The treatment to date has been well-tolerated, with no serious adverse effects or dose limiting toxicities, the pharmaceutical company reported.

“We are very pleased that RT001 appears to be safe and well tolerated in this ongoing first-in-human study in FA patients. Primary endpoints are safety, tolerability, and the pharmacokinetic profile of orally dosed RT001. Secondary endpoints are the disease-related Friedreich’s Ataxia Rating Scale neurological score, the timed 25-foot walk and various exploratory measures and biomarkers,” Robert De Jager, MD, Retrotope’s CMO, said in a press release.

RT001 is Retrotope’s lead candidate for the treatment of FA. According to the company, lipid peroxidation — the degradation of lipids in mitochondrial and cellular membranes due to free-radical action — may cause a broad range of degenerative diseases. Free-radicals target polyunsaturated fats (PUFAs), which are vital membrane components. Previous research has revealed that as these fats degrade they generate toxic cascades that have been linked to many diseases, including FA. The compound RT001 is a chemically stabilized natural fatty acid that resists lipid peroxidation in mitochondrial and cellular membranes.

Jennifer Farmer, MS, CGC, executive director of the Friedreich’s Ataxia Research Alliance (FARA), said, “FARA is pleased to continue our support of Retrotope and this clinical study of a new approach to treat FA. We are excited by the potential of RT001 and we are grateful to the individuals who have volunteered for this study.”

The Phase 1-2 trial, “A First in Human Study of RT001 in Patients with Friedreich’s Ataxia,” is being conducted at two sites: the Collaborative Neuroscience Network in Long Beach, California, and the USF Ataxia Research Center in Tampa, Florida.

Anyone interested can find more information on this study, including enrollment contact information, through its clinical site.