IP Library Granted Patent US 10,829,493
Granted Patent B2
US 10,829,493 · App. 16/842,969 · Granted Nov 10, 2020

Compositions and methods for treating KIT- and PDGFRA-mediated diseases

Inventor: Thomas A. Dineen (Cambridge, MA)
Assignee: Blueprint Medicines Corporation
C07D487/04A61P43/00
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Quick Facts
Patent No.
US 10,829,493
App. No.
16/842,969
Granted
Nov 10, 2020
Kind
B2
Abstract

The present disclosure provides compounds of Formula I, pharmaceutical salts thereof, and/or solvates of any of the foregoing, which are useful for treating diseases and conditions related to mutant KIT and PDGFRα and present an advantageously non-brain penetrant profile for treating diseases and conditions related to mutant KIT and PDGFRα. The present disclosure also provides methods for treating gastrointestinal stromal tumors and systemic mastocytosis.

Claims (78)

1. A compound of Formula I:

a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein:

A is

R 3 is chosen from hydrogen and methyl;

R 4 is chosen from hydrogen and methyl, or

R 3 and R 4 taken together form a cyclopropyl;

R 5 is chosen from hydrogen and methyl;

R 6 is chosen from hydrogen and methyl, or

R 5 and R 6 taken together form a cyclopropyl, or

R 4 taken together with R 6 forms a cyclobutyl; and

R 7 is hydrogen.

2. The compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein:

A is:

R 3 is chosen from hydrogen and methyl;

R 4 is chosen from hydrogen and methyl, or R 3 and R 4 taken together form a cyclopropyl;

R 5 is chosen from hydrogen and methyl;

R 6 is chosen from hydrogen and methyl; or

R 5 and R 6 taken together form a cyclopropyl, and

R 7 is hydrogen.

3. The compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein A is chosen from

4. The compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein A is chosen from

5. The compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein A is chosen from

6. The compound of claim 5 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein A is chosen from

7. The compound of claim 6 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein A is chosen from

8. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

9. The compound of claim 8 ,

10. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

11. The compound of claim 10

12. A pharmaceutical composition comprising:

a compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

13. A pharmaceutical composition comprising:

a compound of claim 8 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

14. A pharmaceutical composition comprising:

a compound of claim 10 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

15. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 1 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

16. The method of claim 15 , wherein the disease or condition is systemic mastocytosis.

17. The method of claim 16 , wherein the systemic mastocytosis is chosen from indolent systemic mastocytosis and smoldering systemic mastocytosis.

18. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

19. The compound of claim 18 ,

20. A pharmaceutical composition comprising:

a compound of claim 18 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

21. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 18 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

22. The method of claim 21 , wherein the disease or condition is indolent systemic mastocytosis.

23. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

24. The compound of claim 23 ,

25. A pharmaceutical composition comprising:

a compound of claim 23 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

26. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 23 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

27. The method of claim 26 , wherein the disease or condition is indolent systemic mastocytosis.

28. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

29. The compound of claim 28 ,

30. A pharmaceutical composition comprising:

a compound of claim 28 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

31. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 28 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

32. The method of claim 31 , wherein the disease or condition is indolent systemic mastocytosis.

33. A compound

or a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing.

34. The compound of claim 33 ,

35. A pharmaceutical composition comprising:

a compound of claim 33 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing; and

a pharmaceutically acceptable excipient.

36. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 33 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

37. The method of claim 36 , wherein the disease or condition is indolent systemic mastocytosis.

38. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 8 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

39. The method of claim 38 , wherein the disease or condition is indolent systemic mastocytosis.

40. A method of treating a disease or condition in a patient in need thereof, wherein the method comprises administering to the patient a compound of claim 10 , a pharmaceutically acceptable salt thereof, and/or a solvate of any of the foregoing, wherein the disease or condition is chosen from systemic mastocytosis and gastrointestinal stromal tumors.

41. The method of claim 40 , wherein the disease or condition is indolent systemic mastocytosis.

Assignments (5)
RELEASE OF SECURITY INTEREST (REEL/FRAME NUMBER 060616/0923) Recorded Jul 23, 2025
From: TAO TALENTS, LLC
To: BLUEPRINT MEDICINES CORPORATION
Reel/Frame 072193/0847 →
RELEASE OF SECURITY INTEREST Recorded Jul 21, 2025
From: PROTOZOA INVESTMENTS, L.P.
To: BLUEPRINT MEDICINES CORPORATION
Reel/Frame 071777/0840 →
SECURITY INTEREST Recorded Jul 8, 2022
From: BLUEPRINT MEDICINES CORPORATION
To: GARNICH ADJACENT INVESTMENTS S.A.R.L.
Reel/Frame 060465/0357 →
SECURITY INTEREST Recorded Jul 8, 2022
From: BLUEPRINT MEDICINES CORPORATION
To: TAO TALENTS, LLC
Reel/Frame 060616/0923 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 20, 2020
From: KIM, JOSEPH L.; GUZI, TIMOTHY; DINEEN, THOMAS A.
To: BLUEPRINT MEDICINES CORPORATION
Reel/Frame 052439/0466 →
Continuity (4)
Provisional Application 62930240 · Nov 4, 2019
Provisional Application 62911016 · Oct 4, 2019
Provisional Application 62833529 · Apr 12, 2019
Related Publication 20200325141A1 · Oct 15, 2020
Cited By (2)
US 12,552,795 US 12,698,290