Drugmakers juggle isotopes in hopes of achieving novelty, stability, and success. Heavier than hydrogen by a single neutron, deuterium might not seem to have much chemical heft. But the small matter of that subatomic particle makes a massive difference in the reactivity of hydrogen versus its isotope deuterium.
Currently in Friedreich ataxia's Research Pipeline: RT001 (RETROTOPE).
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. Retrotope filed their IND with FDA in 2015 and announced enrollment of a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of orally dosed RT001 to evaluate the safety, tolerability, pharmacokinetics (PK), disease state, and exploratory endpoints in patients with Friedreich’s ataxia (FA) in August 2015. This study is taking place at University of South Florida and University of California Los Angeles.
