DISCONTINUED: Stages of Development for Retrotope

The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.

2012: FARA awarded a grant for testing Retrotope compounds in established cell models. Results of this work have been published and can be accessed in the Related Publications section below.

October 2012: Retrotope had an introductory (pre-IND) meeting with the FDA. At that meeting, the FDA provided clear information and guidance on the requirements for Retrotope’s Investigational New Drug (IND) Application for a Phase Ib trial of Retrotope’s drug for FA.

2013: FARA awarded a grant to Retrotope to assist with the manufacturing of drug product and toxicology studies required for the IND filing.

2015: Retrotope filed their IND with FDA and announced enrollment of a Phase Ib/IIa study of RT001.

June 2016: Retrotope announced that FDA granted Orphan Drug Designation for RT001 in FA.

2015: The Phase Ib/IIa study design was a 28-day, first-in-human, randomized, double-blind, placebo controlled, ascending dose study of orally dosed RT001 with the goal of evaluating the safety, tolerability, pharmacokinetics (PK), disease state, and exploratory endpoints in patients with FA.

FARA provided a grant to Retrotope to support participant travel for the study.

September 2016: Retrotope, along with Dr. Theresa Zesiewicz, study Principal Investigator, presented results at the annual USF symposium of the trial of  RT001. The trial 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.

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

February 2022: FARA was notified by Retrotope that the recently completed Phase II/III trial of RT001 in FA did not successfully meet its endpoints.