Sialic acid analogs

Patent metadata

Field	Value
Publication number	US-10889607-B2
Application number	US-201916571589-A
Country	US
Kind code	B2
Filing date	Sep 16, 2019
Priority date	Oct 24, 2011
Publication date	Jan 12, 2021
Grant date	Jan 12, 2021

Abstract

The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.

First claim

What is claimed is:

1. A compound selected from the group consisting of:

2. The compound of claim 1 , wherein the compound is:

3. The compound of claim 1 , wherein the compound is:

4. The compound of claim 1 , wherein the compound is:

5. The compound of claim 1 , wherein the compound is:

6. The compound of claim 1 , wherein the compound is:

7. The compound of claim 1 , wherein the compound is:

8. A pharmaceutical composition comprising a compound selected from the group consisting of:

or a pharmaceutically acceptable salt or solvate thereof, and a pharmaceutically acceptable carrier.

9. The pharmaceutical composition of claim 8 , comprising the compound:

10. The pharmaceutical composition of claim 8 , comprising the compound:

11. The pharmaceutical composition of claim 8 , comprising the compound:

12. The pharmaceutical composition of claim 8 , comprising the compound:

13. The pharmaceutical composition of claim 8 , comprising the compound:

14. The pharmaceutical composition of claim 8 , comprising the compound:

15. A sustained release pharmaceutical composition comprising a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof, wherein:
a) the release of the compound is over a period of about 4 hours to about 24 hours; and/or
b) the pharmacological effect from the compound lasts about four hours to about 24 hours upon administration of the composition.

16. A sustained release pharmaceutical composition comprising a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof,
wherein the composition, upon administration, provides a therapeutically effective amount of the compound for about 4 hours to about 24 hours.

17. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering an effective amount of a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof.

18. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering a compound of claim 1 , or a pharmaceutically acceptable salt or solvate thereof wherein upon administration, the compound, or a pharmaceutically acceptable salt or solvate thereof, continuously provides a therapeutically effective amount of sialic acid for about 4 hours to about 24 hours.

19. The method of claim 17 , wherein the sialic acid deficiency is myopathy caused by sialic acid deficiency.

20. The method of claim 19 , wherein the myopathy caused by sialic acid deficiency is Hereditary Inclusion Body Myopathy (HIBM), Nonaka myopathy, or Distal Myopathy with Rimmed Vacuoles (DMRV).

Assignees

• Ultragenyx Pharmaceutical Inc

Inventors

• Kakkis Emil
• Jungles Steven
• Zhao He

Classifications

• C07D493/08 Primary

  Bridged systems · CPC title

• C07H7/027

  Keto-aldonic acids · CPC title

• C07H13/10

  having the esterifying carboxyl radicals directly attached to heterocyclic rings · CPC title

• C07H13/04

  having the esterifying carboxyl radicals attached to acyclic carbon atoms · CPC title

• C07H7/02 Primary

  Acyclic radicals · CPC title