IP Library Granted Patent US 10,899,734
Granted Patent B2
US 10,899,734 · App. 16/307,473 · Granted Jan 26, 2021

Small molecule modulators of pantothenate kinases

Inventors: Lalit Kumar Sharma (South San Francisco, CA); Richard E. Lee (Cordova, TN); Charles O. Rock (Bartlett, TN); Suzanne Jackowski (Bartlett, TN); Mi Kyung Yun (Collierville, TN); Chitra Subramanian (Cordova, TN); Jiuyu Liu (Collierville, TN)
Assignee: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
C07D401/04A61K31/501A61P3/10A61P25/00C07D213/85C07D237/24C07D241/04C07D401/12C07D401/14C07D403/04C07D403/12C07D409/12C07D409/14C07D413/14
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Quick Facts
Patent No.
US 10,899,734
App. No.
16/307,473
Granted
Jan 26, 2021
Kind
B2
Abstract

The present disclosure relates to chemical compounds that modulate pantothenate kinase (PanK) activity for the treatment of metabolic disorders (such as diabetes mellitus type II), neurologic disorders (such as pantothenate kinase-associated neurodegeneration), pharmaceutical compositions containing such compounds, and their use in treatment. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Claims (33)

1. A compound having a structure represented by a formula:

wherein A is CH 2 ;

wherein Q 1 is CH; and wherein R 2 is selected from —SCH 3 , C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, C1-C8 alkoxyhaloalkyl, cyclopropyl, cyclobutyl, and oxetane, wherein the cyclopropyl, cyclobutyl, and oxetane are optionally substituted with 1, 2, or 3 groups independently selected from —OH, C1-C4 alkyl, and C1-C4 alkoxy; or

wherein Q 1 is N; and R 2 is selected from halogen, —SCH 3 , C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, C1-C8 alkoxyhaloalkyl, cyclopropyl, cyclobutyl, and oxetane, wherein the cyclopropyl, cyclobutyl, and oxetane are optionally substituted with 1, 2, or 3 groups independently selected from —OH, C1-C4 alkyl, and C1-C4 alkoxy;

wherein Q 2 is a structure selected from:

wherein each of R 3a and R 3b is independently selected from hydrogen, halogen, —OH, C1-C4 alkoxy, and C1-C4 alkyl; and

wherein R 4 is halogen or CN,

or a pharmaceutically acceptable salt thereof.

2. The compound of claim 1 , wherein Q 1 is CH and R 2 is selected from C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, and cyclopropyl; or wherein Q 1 is N and R 2 is selected from halogen, C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, and cyclopropyl; and wherein Q 2 is a structure selected from:

3. The compound of claim 1 , wherein Q 2 is a structure selected from:

4. The compound of claim 1 , wherein Q 2 is a structure selected from:

5. The compound of claim 1 , wherein the compound has a structure represented by a formula:

or a pharmaceutically acceptable salt thereof.

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

or a pharmaceutically acceptable salt thereof.

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

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

9. A method of treating a disorder associated with pantothenate kinase activity in a subject, the method comprising administering to the subject an effective amount of at least one compound having a structure represented by a formula:

or a pharmaceutically acceptable salt thereof,

wherein A is CH 2 ;

wherein Q 1 is CH; and wherein R 2 is selected from —SCH 3 , C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, C1-C8 alkoxyhaloalkyl, cyclopropyl, cyclobutyl, and oxetane, wherein the cyclopropyl, cyclobutyl, and oxetane are optionally substituted with 1, 2, or 3 groups independently selected from —OH, C1-C4 alkyl, and C1-C4 alkoxy; or

wherein Q 1 is N; and R 2 is selected from halogen, —SCH 3 , C1-C8 acyclic alkyl, C2-C8 acyclic alkenyl, C1-C8 monohaloalkyl, C1-C8 polyhaloalkyl, C1-C8 alkoxyhaloalkyl, cyclopropyl, cyclobutyl, and oxetane, wherein the cyclopropyl, cyclobutyl, and oxetane are optionally substituted with 1, 2, or 3 groups independently selected from —OH, C1-C4 alkyl, and C1-C4 alkoxy;

wherein Q 2 is a structure selected from:

wherein each of R 3a and R 3b is independently selected from hydrogen, halogen, —OH, C1-C4 alkoxy, and C1-C4 alkyl;

wherein R 4 is halogen or CN; and

wherein the disorder is hyperglycemia, neurodegeneration, or diabetes, wherein hyperglycemia is caused by misregulated and/or elevated coenzyme A; and neurodegeneration is caused by a deficiency of pantothenate kinase or coenzyme A.

10. The method of claim 9 , wherein the neurodegeneration is pantothenate kinase-associated neurodegeneration (PKAN).

11. The compound of claim 1 , wherein Q 1 is CH.

12. The compound of claim 1 , wherein R 2 is cyclopropyl.

13. The compound of claim 1 , wherein each of R 3a and R 3b is hydrogen.

14. The compound of claim 1 , wherein Q 2 is a structure:

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

16. The method of claim 9 , wherein the compound is:

Assignments (4)
CORRECTIVE ASSIGNMENT TO CORRECT THE RECEIVING PARTY ASSIGNEE NAME TO READ AS ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. PREVIOUSLY RECORDED ON REEL 049663 FRAME 0753. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT. Recorded Sep 1, 2021
From: SHARMA, LALIT KUMAR; LEE, RICHARD E.; ROCK, CHARLES O.; JACKOWSKI, SUZANNE; YUN, MI KYUNG; SUBRAMANIAN, CHITRA
To: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Reel/Frame 057394/0711 →
CORRECTIVE ASSIGNMENT TO CORRECT THE RECEIVING PARTY ASSIGNEE NAME TO READ AS ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC. PREVIOUSLY RECORDED ON REEL 049664 FRAME 0058. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT. Recorded Sep 1, 2021
From: LIU, JIUYU
To: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Reel/Frame 057394/0741 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 3, 2019
From: SHARMA, LALIT KUMAR; LEE, RICHARD E.; ROCK, CHARLES O.; JACKOWSKI, SUZANNE; YUN, MI KYUNG; SUBRAMANIAN, CHITRA
To: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Reel/Frame 049663/0753 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 3, 2019
From: LIU, JIUYU
To: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Reel/Frame 049664/0058 →
Continuity (2)
Provisional Application 62354012 · Jun 23, 2016
Related Publication 20190300499A1 · Oct 3, 2019
Cited By (2)
US 12,545,663 US 12,552,765