Journal of Clinical Toxicology
Open Access
ISSN: 2161-0495
+44 1478 350008
ISSN: 2161-0495
+44 1478 350008
Mirum Pharmaceuticals, San Francisco Bay Area, California, United States
Research Article
Use of Nonclinical and Expanded Access Clinical Study data to Guide Development Programs Targeting Mitochondrial Diseases of Lipid Peroxidation
Author(s): Mark G. Midei*, Paldeep Atwal, Jayshree Krishnaswami, Frederic Heerinckx, Sarah Endemann, Peter Milner and Mikhail Shchepinov
Background: Many diseases of neurodegeneration associated with mitochondrial Lipid Peroxidation have poorly
predictive animal models and present unique challenges for clinical trial design. RT001 is a dideuterated isotopomer
of linoleic acid that inhibits Lipid Peroxidation. To correlate non clinical models with clinical response, we reviewed
the effects of RT001 in disease models and clinical indications associated with Lipid Peroxidation.
Methods: We identified 10 specific target diseases known to be associated with Lipid Peroxidation. We evaluated the
effects of RT001 in cellular models, animal models, and expanded access clinical study of these Lipid Peroxidation
associated diseases.
Results: Significant beneficial effects were seen in 7 disorders. Clinical efficacy was seen in 6, no benefits were se.. View More»
DOI:
10.35248/2161-0495.21.11.490