Friedreich’s ataxia Phase 1b/2a trial achieves all goals with early signals of efficacy
LOS ALTOS, CA, April 21, 2017 – Dr. Theresa Zesiewicz, University of South Florida Movement Disorders Clinic, will give a podium presentation at the American Academy of Neurology annual meeting of clinical trial results of Retrotope’s RT001 in the neuromuscular disease, Friedreich’s ataxia (FA) on April 24, 2017 (Session S17, 3:30 pm). The study showed good safety and tolerability of this novel drug class, including early signals of efficacy with regard to disease progression. The trial, a randomized, double--blind, comparator-controlled study of RT001 in 18 FA patients for 28 days, 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 disease progression measures showed signals of drug effect in post hoc analysis, unexpected in such a short, small study.
Robert J Molinari, CEO and co--founder of Retrotope, commented: “We are extremely excited about these results in humans. Retrotope has observed robust signals of efficacy for its stabilized polyunsaturated fatty acids (PUFA) drugs in many animal models of neurodegeneration, including major diseases like Alzheimer’s and Parkinson’s all the way to ultra--rare genetic conditions such as neurodegeneration with brain iron accumulation (NBIA). Historically, many CNS drugs with good animal data eventually failed in human studies. Seeing the benefits of our stabilized lipid drug (RT001) actually transfer to human studies is very encouraging. As lipids are dosed and distributed quite differently from other types of drugs, we believe this novel drug approach to treat conditions behind the blood brain barrier may transfer well to other human diseases.”
Retrotope is in discussions with FDA about the design and endpoints surrounding studies that can support an NDA for Friedreich’s ataxia, and has recently started an Expanded Access trial in a second neurological disease. So far, the only severe adverse event in the highest dose cohort was uncontrolled diarrhea, experienced by a subject with very low body mass index (BMI). This is a common complication of high fatty acid oil doses in conditions such as hypercholesterolemia, and the problem self-resolved in this study. Other adverse events were either very mild or non-drug related. Fatty acid metabolites of RT001 were also detected and demonstrate that the drug participated in normal fatty acid processing into a brain penetrant form of the drug. For more information on the reported study, please visit: https://clinicaltrials.gov/ct2/show/NCT02445794
About FA
FA is a debilitating, life--shortening neuro--degenerative disorder that affects approximately 5,000 people in the United States, and over 20,000 people worldwide. A progressive loss of coordination and muscle strength leads to motor incapacitation, the full--time use of a wheelchair, and ultimately early death, typically from cardiomyopathy. There is currently no approved treatment for FA. Earlier this year, the FDA granted Retrotope Orphan Drug Designation for RT001 in FA. Retrotope is greatly indebted to The Friedreich’s Ataxia Research Alliance (FARA), for its support in all aspects of its clinical trial design and execution, including recruiting and financial support for patients’ travel costs to participate in the trial.
About RT001
RT001 is a patented, orally available, modified fatty--acid therapeutic that stabilizes (“fireproofs”) mitochondrial and cellular membranes against attack and restores cellular health. Retrotope and others have discovered that lipid peroxidation, the free--radical degradation of lipids in mitochondrial and cellular membranes, may be causative of a wide range of degenerative disease phenotypes. The degradation products of these fats involved in critical cell functions like mitochondrial ATP generation create toxic cascades that lead to cell death, disease onset and progression.
About Retrotope
Retrotope, a privately--held, clinical--stage pharmaceutical company, is creating a new category of drugs to treat degenerative diseases. Composed of proprietary compounds that are chemically stabilized forms of essential nutrients, these compounds are being studied as disease modifying therapies for many intractable diseases such as Parkinson’s, Alzheimer’s, mitochondrial myopathies, and retinopathies. RT001, Retrotope’s first lead candidate, is for the treatment of Friedreich’s ataxia, a fatal orphan disease. For more information about Retrotope, please visit www.retrotope.com.
About FARA: The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non--profit, tax exempt organization dedicated to curing Friedreich’s ataxia (FA), a rare neuromuscular disorder, through research. For more information about FA, visit the FARA website at www.curefa.org.
