Retrotope recently announced a second clinical trial site, the Collaborative Neuroscience Network, LLC (CNS), has opened in Long Beach, California, as part of the company’s ongoing 28-day, first-in-human randomized, double-blind, controlled, ascending dose study of orally dosed experimental therapy RT001. The study will evaluate the safety, tolerability, pharmacokinetics (PK), disease state and exploratory endpoints in Friedreich’s ataxia (FA) patients.
Retrotope has discovered that the free radical degradation of lipids in mitochondrial and cellular membranes, a common mechanism to degenerative conditions, may cause the disease. Free radicals damage the polyunsaturated fats (PUFAs) that are essential to cellular membranes’ composition, and the company’s researchers have demonstrated that the degradation products of these fats are associated with neurodegeneration and aging, leading to further toxic cellular damage. RT001, Retrotope’s lead compound is a patented, stabilized fatty-acid that shuts down this degradation process and stabilizes cellular membranes against further aggressions.
Friedreich’s ataxia is a degenerative neuro-muscular disorder whose symptoms can begin to show from five years old into adulthood. The disease may decrease motor coordination levels and lead to loss of muscle strength, resulting in incapacitation, full-time use of a wheel chair in some cases, and cardiac complications. There are no approved treatments for FA at the moment, and the disease affects nearly 6,000 people in the U.S. alone, according to the Friedreich’s Ataxia Research Alliance (FARA).
Retrotope’s CEO, Robert Molinari, Ph.D., commented in a press release, “Retrotope is excited to have the second clinical trial site open and participating in the ongoing study in FA. This second center is located in Southern California and is working closely with the University of South Florida (USF) and the Friedreich’s Ataxia Research Alliance (FARA) as part of the overall study team working to develop a treatment for this devastating disease.”
The RT001-002 protocol is a dual-centered study designed for 18 ambulatory (regardless of having an assistive device) Friedreich’s ataxia patients. Its main objectives include the evaluation of safety and tolerability of two dose levels of RT001 when orally administered for 28 consecutive days; to determine the PK profile of RT001 at both dose levels, following a single and multiple oral intake; and to determine a dosage for future research. In the future, the company expects to evaluate RT001’s effects on disease state endpoints using the FA Rating Scale neurological sub-score and the Timed 25-Foot Walk (T25FW) performance measurement relevant to ataxia.
The trial has been open since August 2015 and is still enrolling patients at the University of South of Florida.