Retrotope Opens Patient Enrolment in Florida to Test Friedreich’s Ataxia Drug

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Retrope, Inc., a privately held clinical-stage pharmaceutical company leading the advance of a revolutionary new unifying theory of aging and degeneration, has just opened patient enrolment for a 28-day, first-in-human, randomized, double-blind, controlled, ascending dose study of oral drug RT001. The study aims to determine the drug’s safety, tolerability and pharmacokinetic profile, disease state, and exploratory endpoints in patients suffering from Friedreich’s ataxia (FA), a debilitating, life-shortening degenerative neuro-muscular disorder that affects approximately 6,000 people in the United States.

“Retrotope is excited to begin human trials working with FARA and the Friedreich’s ataxia community to develop a treatment for this devastating disease,” said Robert Molinari, Ph.D., CEO of Retrotope. “Retrotope and others have discovered that free radical lipid peroxidation cascades in the mitochondrial membrane represent a common mechanism by which many degenerative diseases, and especially FA, trigger and amplify damage that kills cells.  In FA, free iron associated with the disease is a catalyst for lipid peroxidation of exactly the type we can mitigate with our drug, RT001, a chemically stabilized form of a natural membrane fatty acid that is resistant to lipid peroxidation.”

This study will be conducted in two centers and will involve 18 ambulatory FA patients, primarily evaluating RT001’s drug profile in two dose levels over a course of 28 days. Its PK profile at both dosages will also be assessed following single and multiple oral administrations. The study’s second endpoints include the drug’s effects on disease state according to the FA Rating Scale neurological sub-score and the Timed 25-Foot Walk (T25FW) performance measurement relevant to ataxia.

“FARA has been working with Retrotope for the past few years as RT001 has gone through pre-clinical development and we believe this provides an excellent example of the power of patient engagement early in the drug development process. FARA provided assistance in developing partnerships with academic collaborators who have tested RT001 in FA cellular models, grant support for manufacturing, attendance at regulatory meetings and engagement of the patient community in focus group discussions on diet modifications recommended for this early phase study. We are excited to continue our partnership and support as we approach this new and important milestone,” said Jennifer Farmer, MS, CGC, Executive Director at FARA.

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax exempt organization dedicated to curing the disease. Two of its Collaborative Clinical Research Network sites, the University of South Florida and the University of California Los Angeles, will be home to the RT001 study. Enrolment has already begun in the University of South Florida, while enrolment in UCLA is expected to begin early in the Fall of 2015.