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Retrotope Expands its Drug Pipeline with the First Dosing of RT001 in patients with Friedreich's ataxia (FA)LOS ALTOS, Calif., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Retrotope announced today that it has dosed its first patient in a Phase 2/3 clinical trial of RT001 in Friedreich’s ataxia, the most common of the inherited ataxias. RT001, a stabilized fatty acid drug, has been shown to reduce lipid peroxidation leading to cell death in patients across a wide swath of degenerative diseases, including FA. More information and a list of participating clinical sites for the current trial in FA can be obtained at ClinicalTrials.gov http://bit.ly/2T2FWA5. With this trial, the company expands its pipeline of indications for RT001 which is currently being studied in a pivotal trial in another fatal, neurodegenerative disease, Infantile Neuroaxonal Dystrophy (INAD), which is fully enrolled. Retrotope previously demonstrated safety and an early signal of efficacy in a small, randomized, placebo-controlled trial in FA http://bit.ly/2QQk8Fi. In that study, subjects on drug outperformed those on placebo in generating peak workload in a controlled cardiopulmonary exercise test (CPET). After only a month of dosing, other measures of disease progression showed trends towards improvement in the Friedreich’s Ataxia Rating Scale, Neurological exam (FARS-Neuro), in an electronically-timed walk, and in maximum oxygen consumption during CPET. RT001 has been granted U.S. orphan drug designation for the treatment of Friedreich’s ataxia by the FDA. “This Phase 2/3 clinical trial is an important milestone in the development pathway of RT001” commented Peter G Milner, MD of Retrotope. “We believe the CPET is a very sensitive probe of the mitochondrial function and stamina of patients with this disease who suffer from profound fatigue due to mitochondrial neuropaty, myopathy and cardiomyopathy. FDA has agreed with us that CPET may be a primary endpoint in a marketing approval study, and will be evaluated for approval with other secondary, supportive efficacy measures and validated scales important to patient function.” Jennifer Farmer, Chief Executive Officer, Friedreich’s Ataxia Research Alliance (FARA) commented, “Friedreich’s ataxia is a progressive and debilitating disease for which there are no approved treatments, thus there is high unmet need for individuals and families diagnosed with FA. We are excited to see RT001 advance to a phase 2/3 efficacy study, and we are actively working to assist in patient recruitment efforts to ensure the study enrolls efficiently and meets its enrollment goals. FARA has been committed to creating and sharing the research community resources such as patient registries, natural history data, and validated clinical outcome measures along with fostering patient engagement in research in order to assist Retrotope and other drug development partners in advancing clinical development programs.” About Friedreich’s ataxia About RT001 About Retrotope SOURCE: Retrotope, Inc. 4300 El Camino Real, Suite 201 Los Altos, CA 94022 650-575-7551
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