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US 12,582,652 B2Granted
3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine as an ion channel modulator
AssigneePraxis Precision Medicines, Inc.
InventorsMichael Kristopher Mathieu Kahlig, Marion Wittmann, Zoe A. Hughes, Bernard Ravina
Life of this patent
Estimating the term…
Abstract
The present disclosure is generally directed to methods of treating a disease, disorder, or condition, e.g., a neurological disorder, a disorder associated with excessive neuronal excitability, or a disorder associated with de novo gain-of-function or loss-of-function mutations in major central nervous system sodium channel genes, such as for example, SCN1A, SCN2A, and SCN8A, using a Compound 1, or a pharmaceutically acceptable salt thereof, of the following formula:
Claims28
A method for treating a genetic epilepsy or a genetic epilepsy syndrome in a human in need thereof, wherein the method comprises administering to the human a therapeutically effective amount of Compound 1 of the following formula:
- or a pharmaceutically acceptable salt thereof;
- wherein Compound 1, or a pharmaceutically acceptable salt thereof, is administered to the human in need thereof in a therapeutically effective dosage amount in the range of 0.1 mg/kg/day to 10 mg/kg/day;
- and
- wherein the administration of the therapeutically effective dosage amount of Compound 1, or a pharmaceutically acceptable salt thereof, to the human in need thereof results in any one of (a), (b), (c), (d), (e), (f), or (g);
- (a) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (b) a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (c) a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (d) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (e) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (f) a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (g) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof.
23 more elements
A method for effecting any one of (a), (b), (c), (d), (e), (f), or (g) in a human having a genetic epilepsy or a genetic epilepsy syndrome, wherein the method comprises administering to the human in need thereof a therapeutically effective amount of Compound 1 of the following formula:
- or a pharmaceutically acceptable salt thereof;
- wherein (a), (b), (c), (d), (e), (f), or (g) is:
- (a) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (b) a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (c) a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (d) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (e) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (f) a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- or
- (g) a reduction in the severity of seizures experienced by the human as compared to the severity of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- a reduction in the number of seizures experienced by the human as compared to the number of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- a reduction in the frequency of seizures experienced by the human as compared to the frequency of seizures experienced by the human prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof;
- and
- wherein Compound 1, or a pharmaceutically acceptable salt thereof, is administered to the human in need thereof in a therapeutically effective dosage amount in the range of 0.1 mg/kg/day to 10 mg/kg/day.
23 more elements
Dependencies are inferred from the claim text.