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, such as alpha-tocopherol quinone. Methods and compounds useful in treating other disorders are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.
Claims53
A method of treating a mitochondrial disorder, comprising administering to a subject suffering from the mitochondrial disorder a composition comprising a therapeutically effective amount of one or more compounds of the formula:
- where R11, R12, and R13 are independently selected from the group consisting of H, —C1-C4 alkyl, —C1-C4 haloalkyl, —CN, —F, —Cl, —Br, and —I, with the proviso that if any of R11, R12, or R13 is H, then at least one of the other two substituents is neither H nor methyl;
- and all stereoisomers, and mixtures of stereoisomers thereof;
- wherein said one or more compounds are the sole active pharmaceutical agent or agents present in a therapeutically effective amount in the composition;
- and wherein the mitochondrial disorder is selected from the group consisting of Leber's Hereditary Optic Neuropathy (LHON);
- Friedreich's Ataxia (FA);
- Parkinson's disease;
- amyotrophic lateral sclerosis (ALS);
- and Huntington's Disease.
A method of treating a mitochondrial disorder, comprising administering to a subject suffering from the mitochondrial disorder a composition comprising a therapeutically effective amount of one or more compounds of the formula:
- where R11, R12, and R13 are independently selected from the group consisting of H, —C1-C4 alkyl, —C1-C4 haloalkyl, —CN, —F, —Cl, —Br, and —I, with the proviso that if any of R11, R12, and R13 is H, then at least one of the other two substituents is neither H nor methyl;
- and all stereoisomers, and mixtures of stereoisomers thereof;
- wherein the one or more compounds are the sole active pharmaceutical agent or agents used to treat the mitochondrial disorder;
- and wherein the mitochondrial disorder is selected from the group consisting of Leber's Hereditary Optic Neuropathy (LHON);
- Friedreich's Ataxia (FA);
- Parkinson's disease;
- amyotrophic lateral sclerosis (ALS);
- and Huntington's Disease.
A method of treating a mitochondrial disorder, comprising administering to a subject suffering from the mitochondrial disorder a composition comprising a therapeutically effective amount of one or more compounds of the formula:
- where R11, R12, and R13 are independently selected from the group consisting of H, —C1-C4 alkyl, —C1-C4 haloalkyl, —CN, —F, —Cl, —Br, and —I, with the proviso that if any of R11, R12, or R13 is H, then at least one of the other two substituents is neither H nor methyl;
- and all stereoisomers, and mixtures of stereoisomers thereof;
- and wherein the mitochondrial disorder is selected from the group consisting of Leber's Hereditary Optic Neuropathy (LHON);
- Friedreich's Ataxia (FA);
- and amyotrophic lateral sclerosis (ALS).
Dependencies are inferred from the claim text.