Combination of a bile acid and a phenyl butyrate compound for the treatment of neurodegenerative diseases
AssigneeAmylyx Pharmaceuticals Inc.
InventorsJoshua Cohen, Justin Klee
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Abstract
Provided herein are methods and compositions for treating a neurodegenerative disease (e.g., AD or PSP). The methods can include administering to the subject a bile acid or a pharmaceutically acceptable salt thereof and a phenylbutyrate compound.
Claims40
A method of treating at least one symptom of progressive supranuclear palsy (PSP), the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of treating at least one symptom of Alzheimer's disease (AD) in a human subject, the method comprising administering to the human subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate, wherein the human subject:
- (a) carries one or more copies of the APOEε4 allele;
- (b) has a cerebral spinal fluid (CSF) level of total tau of about 300 μg/mL or higher;
- or
- (c) has a CSF level of phospho-tau of about 70 μg/mL or higher.
A method of slowing Alzheimer's disease (AD) progression in a human subject having one or more symptoms of AD, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of increasing survival time of a human subject having one or more symptoms of Alzheimer's disease, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of decreasing the level of total CSF tau, decreasing the level of CSF phospho-tau, increasing CSF Aβ1-42/Aβ1-40, or increasing the level of CSF 8-OHDG in a human subject having one or more symptoms of Alzheimer's disease, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of treating and/or preventing a tauopathy in a human subject, the method comprising administering to the human subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of treating and/or preventing an amyloidosis related condition in a human subject, the method comprising administering to the human subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method comprising administering to a human subject at risk for developing Alzheimer's disease a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of decreasing the CSF levels of FABP3, neurogranin, YKL-40, or IL-15 in a human subject having one or more symptoms of Alzheimer's disease, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of treating at least one symptom of a neurodegenerative disease characterized by elevated total tau levels or phospho-tau levels, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of treating at least one symptom of a neurodegenerative disease characterized by elevated YKL-40 levels, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
A method of decreasing the level of CSF YKL-40, decreasing the level of Ptpn1, or increasing the CSF ratio of 33 kDa tau to 55 kDa tau in a human subject having one or more symptoms of PSP, the method comprising administering to the subject a pharmaceutically effective amount of a combination of TURSO and sodium phenylbutyrate.
The method of any one of the above claims, wherein the TURSO and the sodium phenylbutyrate are administered once a day or twice a day.
The method of any one of the above claims, wherein TURSO is administered to the subject at a dose of about 5 mg/kg to about 100 mg/kg.
The method of any one of the above claims, wherein sodium phenylbutyrate is administered to the subject at a dose of about 10 mg/kg to about 400 mg/kg.
The method of any one of the above claims, wherein the TURSO is administered at an amount of about 0.5 to about 5 grams per day.
The method of any one of the above claims, wherein the sodium phenylbutyrate is administered at an amount of about 0.5 grams to about 10 grams per day.
The method of any one of the above claims, comprising administering to the subject 1 gram of TURSO and 3 grams of sodium phenylbutyrate once a day or twice a day.
The method of any one of the above claims, comprising administering to the subject 1 gram of TURSO once a day and 3 grams of sodium phenylbutyrate once a day for about 14 days or more, followed by administering to the subject about 1 gram of TURSO twice a day and 3 grams of sodium phenylbutyrate twice a day.
The method of any one of the above claims, wherein the TURSO and the sodium phenylbutyrate are administered orally.
The method of any one of the above claims, wherein the TURSO and the sodium phenylbutyrate are formulated as a single powder formulation.
The method of any one of the above claims, further comprising administering one or more additional therapeutic agents to the subject.
The method of any one of the proceeding claims, further comprising administering to the human subject a plurality of food items comprising solid foods or liquid foods.
The method of any one of the proceeding claims, wherein the human subject is about 18 years or older.
Dependencies are inferred from the claim text.