FARA has been notified by Retrotope that the recently completed Phase 2/3 trial of RT001 in FA did not successfully meet its endpoints.

In October 2019, Retrotope launched a Phase 2/3 trial in FA, A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with FA. This was a double-blind, placebo-controlled trial to study the impact of RT001 on neurological and cardiac symptoms and evaluate safety over 11 months of treatment. The trial enrolled 65 individuals who are ages 12-50 yrs and the primary outcome measure was peak workload change from baseline to 11 months using cardiopulmonary exercise testing (CPET). There were also secondary outcome measures to further assess neurological outcomes and fatigue.

The sponsor, Retrotope, has informed FARA and the trial investigators that the drug failed to reach its primary and key secondary endpoints in this study. Unfortunately, they report that there was no improvement in exercise measures or neurological outcomes assessed in the study. Even though the drug failed to show positive effects, we may learn important information from the trial. Therefore, the site investigators, along with Retrotope, are in the process of reviewing the detailed results over the next few weeks to see if there are insights from the trial that will be important to share with the FA community. We are disappointed in the results of the trial, however negative results are valuable and inform our future progress for other treatments in our pipeline. We are appreciative to Retrotope for bringing a potential therapy for FA to clinical trials. We want to thank and acknowledge all of the individuals who volunteered for the trial of RT001 along with the site investigators and coordinators who performed the trial.

What is RT001?

RT001 is a polyunsaturated fatty acid, linoleic acid, that has been synthetically made with isotopic stabilization using deuterium. Polyunsaturated fatty acids (PUFAs) are known as good fats and are essential to cells in the nervous system, specifically the mitochondrial membrane. It has been shown the PUFAs are susceptible to oxidative damage, lipid peroxidation. In models of FA, protecting against lipid peroxidation has been shown to be beneficial. The strategy here is to stabilize the PUFAs and protect the cells from this oxidative damage. One approach to stabilizing the PUFAs is to create mimetics (very similar chemical substitutes) of PUFAs. Deuterated compounds are those that have hydrogen molecules replaced by deuterium. Deuterium is a stable isotope of hydrogen. RT001 is clinical-stage, isotopically stabilized linoleic acid.

For more background and information on RT001 - https://www.curefa.org/pipeline#rt001