Pyruvate kinase activators for use in therapy

Patent metadata

Field	Value
Publication number	US-10632114-B2
Application number	US-201815965088-A
Country	US
Kind code	B2
Filing date	Apr 27, 2018
Priority date	May 3, 2011
Publication date	Apr 28, 2020
Grant date	Apr 28, 2020

Abstract

Described herein are methods for using compounds that activate pyruvate kinase.

First claim

What is claimed is:

1. A method for treating sickle cell anemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is:

2. A method for treating thalassemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is:

3. The method of claim 2 wherein the thalassemia is beta-thalassemia.

4. A method for increasing the lifetime of red blood cells (RBCs) in need thereof comprising contacting blood with a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is:

5. The method of claim 4 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is administered to a subject in need thereof.

6. The method of claim 4 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is added directly to whole blood or packed red blood cells extracorporeally.

7. A method for regulating the levels of 2,3-diphosphoglycerate (2,3-DPG) levels in blood in need thereof comprising contacting blood with an effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the compound is:

8. The method of claim 7 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is administered to a subject in need thereof.

9. The method of claim 7 wherein the compound, pharmaceutically acceptable salt thereof or pharmaceutically acceptable composition is added directly to whole blood or packed red blood cells extracorporeally.

10. A method for treating hemolytic anemia in a subject comprising administering to the subject a therapeutically effective amount of (1) a compound or pharmaceutically acceptable salt thereof or (2) a pharmaceutically acceptable composition comprising a compound or pharmaceutically acceptable salt thereof and a pharmaceutically acceptable carrier wherein the hemolytic anemia is selected from congenital anemia and hereditary spherocytosis and wherein the compound is:

11. The method of claim 10 wherein the hemolytic anemia is congenital anemia.

12. The method of claim 10 wherein the hemolytic anemia is hereditary spherocytosis.

Assignees

• Agios Pharmaceuticals Inc

Inventors

• Su Shin-San Michael
• Dang Lenny

Classifications

• C07C307/10

  having nitrogen atoms of the sulfamide groups bound to carbon atoms of six-membered aromatic rings · CPC title

• A61K31/4406

  only substituted in position 3, e.g. zimeldine (nicotinic acid A61K31/455) · CPC title

• C07D333/24

  Radicals substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals · CPC title

• C07D241/04

  having no double bonds between ring members or between ring members and non-ring members · CPC title

• C07D307/14

  Radicals substituted by nitrogen atoms not forming part of a nitro radical · CPC title