Retrotope to present first human data on the safety and early efficacy of its novel stabilized lipid drug platform against neurodegeneration.
On April 24, 2017 Dr. Theresa Zesiewicz from the University of South Florida Movement Disorders Clinic, will give a podium presentation at the American Academy of Neurology, Boston meeting, of clinical trial results of RT001 in the neuromuscular disease, Friedreich’s ataxia (FA). The study showed excellent safety and tolerability of the novel drug class, including early signals of efficacy (and even reversal) of disease progression. The trial, a randomized, double-blind, comparator controlled, 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 disease progression measures showed signals of drug effect unexpected in such a short, small study.