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Retrotope announces Phase I/II Clinical Trial Results of RT001 in Treatment of Friedreich’s ataxia

First-in-human trial achieves safety, tolerability, and PK goals, with early signals of efficacy

LOS ALTOS, CA, September 15, 2016 - Dr. Theresa Zesiewicz, principal investigator in Retrotope’s first-in-human clinical trial of RT001 in Friedreich’s ataxia (FA), today presented early results from Retrotope’s Phase I/II trial conducted at the University of South Florida Ataxia Research Center and the Collaborative NeuroSciences Network in Long Beach, CA. The trial, a randomized, double-blind, comparator controlled, two-dose study of RT001 in 18 FA patients for 28 days, met all of its primary safety, tolerability and pharmacodynamics (PK) goals. While biological activity was not a primary goal of the study, a number of clinically important activity measures were tested, found to be highly correlated to well-studied disease severity scales and showed multiple, unexpected, robust signals of drug effect at one or more doses.

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