Retrotope Announces Peer-Reviewed Publication of Positive Phase 1b/2a Findings for RT001 in Friedreich’s Ataxia

Retrotope, April 18, 2018.
Protocol for Pivotal Clinical Trial Has Been Submitted to the US FDA
LOS ALTOS, Calif., April 18, 2018 (GLOBE NEWSWIRE) -- Retrotope announced today peer-reviewed publication of positive Phase 1b/2a trial results for the company's lead candidate, RT001, in patients with Friedreich's ataxia (FA). RT001 is the first in class of a new category of drugs called D-PUFAs (deuterated polyunsaturated fatty acids), which are designed to protect against free radical damage resulting in cell death that is a hallmark of several degenerative diseases, including FA. In an article titled, "Randomized, Clinical Trial of RT001: Early Signals of Efficacy in Friedreich's Ataxia" (DOI:10.1002/mds.27353), appearing online in Movement Disorders, results of the randomized, double-blind, comparator-controlled Phase 1b/2a trial demonstrated early signals of drug effect (including statistically significant improvements in peak exercise workload compared to placebo) and positive safety and tolerability.

 Retrotope Announces Peer-Reviewed Publication of Positive Phase 1b/2a Findings for RT001 in Friedreich’s Ataxia