RT001 (11,11-di-deutero-linoleic acid ethyl ester) is an experimental fatty-acid from Retrotope that stabilizes the mitochondrial and cellular membranes against attacks and restores cellular health. Retrotope is assessing its product’s clinical potential in treating Friedreich’s ataxia (FA), a condition that, at the cellular level, is characterized by the oxidative degradation of lipids, which shuts down energy production in the mitochondria, leading to cell death.

How RT001 works

RT001 is a highly stabilized version of linoleic acid, also known as Omega-6 fatty acid. Linoleic acid is an essential polyunsaturated fatty acid (PUFA) meaning that it cannot be synthesized by the body and must be obtained from food.

Retrotope researchers discovered that the oxidative degradation of lipids in mitochondrial and cellular membranes may cause degenerative diseases, such as FA.

Free radicals are molecular species that have an unpaired electron. The presence of this unpaired electron makes them unstable and highly reactive, attacking important molecules in the human cells, such as lipids, nucleic acids, and proteins. In FA, free radicals attack and degrade the polyunsaturated fats (PUFAs) that are essential for the maintenance of cellular membranes.

RT001 blocks the chemical reactions that damage these lipids. It is incorporated into the membranes and restores membrane functions and cellular health.

RT001 clinical trial

A phase 1b/2a study (NCT02445794) tested the safety and effectiveness of RT001 in 18 people with FA.

The primary objective of the study was to assess the number of participants who developed adverse reactions at the end of 28 days of treatment. Other objectives included the study of the pharmacokinetics of RT001, which is a measure of the drug and its metabolites in the body at different intervals of the study. Measures of changes in the timed 25-foot walk and in the FA rating scale for neurologic score also were made at the end of the treatment.

This study met all its primary safety, tolerability, and pharmacodynamics objectives. Although biological activity was not a primary objective, various clinically important activity measures also were tested and were shown to be correlated to well-known disease severity scales, with multiple, unexpected and strong signals of drug effect at one or more doses.

Next steps

In 2016, following the announcement of the first results of the Phase 1b/2a study, the U.S. Food and Drug Administration (FDA) granted RT001 orphan drug designation for the treatment of FA. Orphan drugs are products intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 people, but are not expected to recover the costs of developing and marketing a treatment drug.

If Retrotope realizes its clinical plan for RT001 for FA treatment in 2017, a new drug application (NDA) will be submitted to the FDA in 2018.

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