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Last Updated: December 23, 2024

Claims for Patent: 11,234,976


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Summary for Patent: 11,234,976
Title:Methods of using pyruvate kinase activators
Abstract: Described herein are methods for using compounds that activate pyruvate kinase.
Inventor(s): Agresta; Samuel V. (Lexington, MA), Chen; Yue (Quincy, MA), Cohen; Marvin B. (Newtown, PA), Dang; Lenny (Boston, MA), Kung; Charles (Arlington, MA), Merica; Elizabeth A. (Boston, MA), Silver; Bruce A. (Dunkirk, MD), Yang; Hua (Acton, MA)
Assignee: Agios Pharmaceuticals, Inc. (Cambridge, MA)
Application Number:15/735,036
Patent Claims: 1. A method for treating pyruvate kinase deficiency (PKD) in a subject in need thereof, comprising orally administering to the subject once or twice daily a dose of about 10 mg to about 60 mg or about 60 mg to about 200 mg of (1) Compound 1 or a pharmaceutically acceptable salt or hydrate thereof; or (2) a pharmaceutical composition comprising Compound 1 or a pharmaceutically acceptable salt or hydrate thereof, and a pharmaceutically acceptable carrier, to thereby treat PKD in the subject, wherein Compound 1 is N-(4-(4-(cyclopropylmethyl)piperazine-1-carbonyl)phenyl)quinoline-8-sulfo- namide.

2. The method of claim 1, wherein the method comprises orally administering to the subject once or twice daily a dose of about 10 mg to about 60 mg of Compound 1.

3. The method of claim 1, wherein the method comprises orally administering to the subject once or twice daily a dose of about 30 mg or about 120 mg of Compound 1.

4. The method of claim 1, wherein the method comprises orally administering to the subject once or twice daily a dose of about 60 mg to about 200 mg of Compound 1.

5. The method of claim 1, wherein the method comprises orally administering to the subject once or twice daily a dose of about 50 mg, about 75 mg, about 100 mg, about 125 mg, about 150 mg, about 175 mg or about 200 mg of Compound 1.

6. The method of claim 1, wherein the method comprises administering Compound 1 once daily.

7. A method for treating pyruvate kinase deficiency (PKD) in a subject in need thereof, comprising orally administering to the subject once or twice daily a dose of about 50 mg to about 300 mg of (1) Compound 1 or a pharmaceutically acceptable salt or hydrate thereof; or (2) a pharmaceutical composition comprising Compound 1 or a pharmaceutically acceptable salt or hydrate thereof, and a pharmaceutically acceptable carrier, to thereby treat PKD in the subject, wherein Compound 1 is N-(4-(4-(cyclopropylmethyl)piperazine-1-carbonyl)phenyl)quinoline-8-sulfo- namide.

8. The method of claim 7 wherein the method comprises orally administering to the subject once or twice daily a dose of about 225 mg, about 250 mg, about 275 mg or about 300 mg of Compound 1.

9. The method of claim 1 wherein the method comprises administering Compound 1 twice daily.

10. The method of claim 1 wherein the method comprises administering twice daily a dose of about 50 mg of Compound 1.

11. The method of claim 1 wherein the method comprises administering twice daily a dose of about 100 mg of Compound 1.

12. The method of claim 1 wherein the method comprises administering Compound 1 at a dose of about 15 mg about every 12 hours, about 60 mg about every 12 hours, about 120 mg about every 12 hours.

13. The method of claim 1 wherein the method comprises administering Compound 1 at a dose of about 10 mg to about 60 mg about every 12 hours or about 60 mg to about 200 mg about every 12 hours.

14. The method of claim 1 wherein the method comprises administering Compound 1 at a dose of about 10 mg to about 60 mg about every 12 hours or about 60 mg to about 200 mg about every 12 hours.

15. The method of claim 1 wherein the method comprises administering Compound 1 at a dose of about 60 mg to about 200 mg about every 24 hours.

16. The method of claim 1 wherein the method comprises administering Compound 1 at a dose of about 90 mg about every 24 hours, about 120 mg about every 24 hours, about 150 mg about every 24 hours, about 180 mg about every 24 hours, or about 200 mg about every 24 hours.

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