Tail variants of redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomarkers
AssigneeEdison Pharmaceuticals, Inc.
InventorsGuy M. Miller, Sidney M. Hecht
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Abstract
Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, stroke (MELAS), or Kearns-Sayre Syndrome (KSS) are disclosed, as well as compounds useful in the methods of the invention. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed.
Claims21
A method of treating a mitochondrial disorder, modulating one or more energy biomarkers, normalizing one or more energy biomarkers, or enhancing one or more energy biomarkers, comprising administering to a subject a therapeutically effective amount or effective amount of one or more compounds of the formulas:
- where R1 and R2 are independently selected from —C1-C4 alkyl, —C1-C4 haloalkyl, —CN, —F, —Cl, —Br, and —I;
- R3 is selected from —C1-C4 alkyl, —C1-C4 haloalkyl, —CN, —F, —Cl, and —I;
- R20 is independently selected from —C1-C20 alkyl, —C1-C20 alkenyl, —C1-C20 alkynyl, and —C1-C20 containing at least one double bond and at least one triple bond;
- and all salts, stereoisomers, mixtures of stereoisomers, and prodrugs thereof.
A compound of the formula:
- where R1 is selected from —C1-C4 alkyl, —C1-C4 perhaloalkyl, —CN, —F, -and —Br;
- R2 is selected from —C1-C4 alkyl, —C1-C4 perhaloalkyl, —CN, —F, —Cl,—Br, and —I;
- R3 is selected from —C1-C4 alkyl, —C1-C4 perhaloalkyl, —CN, —F, —Cl, and —I;
- R20 is independently selected from —C1-C20 alkyl, —C1-C20 alkenyl, —C1-C20 alkynyl, and —C1-C20 containing at least one double bond and at least one triple bond;
- with the proviso that R20 excludes C6 n-alkyl, C7 n-alkyl, and C11 n-alkyl when R1, R2, and R3 are all methyl;
- and all salts, stereoisomers, mixtures, of stereosoisomers, and prodrugs thereof.
Dependencies are inferred from the claim text.