Use of pyrrolopyrimidine compound for treating hemophagocytic syndrome

Patent metadata

Field	Value
Publication number	US-12447154-B2
Application number	US-202117910331-A
Country	US
Kind code	B2
Filing date	Mar 9, 2021
Priority date	Mar 9, 2020
Publication date	Oct 21, 2025
Grant date	Oct 21, 2025

Abstract

The present invention relates to use of a pyrrolopyrimidine compound for treating hemophagocytic syndrome, and particularly relates to use of a compound of formula (I), a stereoisomer thereof, or a pharmaceutically acceptable salt thereof, and a pharmaceutical composition thereof for treating hemophagocytic syndrome.

First claim

The invention claimed is:

1. A method for treating hemophagocytic syndrome in a patient comprising administering to the patient an effective amount of a compound of formula I, or a stereoisomer or pharmaceutically acceptable salt thereof, or a pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof,

2. The method according to claim 1 , wherein the hemophagocytic syndrome is selected from the group consisting of primary hemophagocytic syndrome and secondary hemophagocytic syndrome.

3. The method according to claim 2 , wherein the primary hemophagocytic syndrome is selected from the group consisting of familial HLH, immunodeficiency syndrome-related HLH, EB virus-driven HLH and HLH caused by abnormalities of a gene selected from the group consisting of JAK2/STAT, PRF1, UNC13D, STX11, STXBP2, Rab27a, LYST, SH2D1A, BIRC4, ITK, AP3β1, MAGT1 and CD27.

4. The method according to claim 2 , wherein the secondary hemophagocytic syndrome is selected from the group consisting of infection-related HLH, malignancy-related HLH, macrophage activation syndrome and HLH induced by pregnancy, a drug, organ and hematopoietic stem cell transplantation or a rare metabolic disease.

5. The method according to claim 1 , wherein the hemophagocytic syndrome is a hemophagocytic syndrome with a mutated gene selected from the group consisting of JAK2/STAT, PRF1, UNC13D, STX11, STXBP2, Rab27a, LYST, SH2D1A, BIRC4, ITK, AP3β1, MAGT1 and CD27.

6. The method according to claim 1 , wherein the compound of formula I is:

7. The method according to claim 1 , wherein the method comprises administering to the patient a daily dose of 1 mg to 100 mg of the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof.

8. The method according to claim 7 , wherein the daily dose 1 mg, 5 mg, 10 mg, 15 mg, 20 mg, 25 mg, 30 mg, 35 mg, 40 mg, 45 mg, 50 mg, 55 mg, 60 mg, 65 mg, 70 mg, 75 mg, 80 mg, 85 mg, 90 mg, 95 mg, or 100 mg, or a range defined by any two of the foregoing values as endpoints.

9. The method according to claim 7 , wherein the daily dose is 1 mg to 90 mg, 5 mg to 80 mg, 10 mg to 70 mg, 15 mg to 60 mg, 20 mg to 50 mg, or 30 mg to 40 mg.

10. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof once or multiple times a day.

11. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof once or twice a day.

12. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in a single-dose formulation once or twice a day.

13. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in a single-dose oral solid formulation once or twice a day.

14. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in a multiple-dose formulation once or twice a day.

15. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in a multiple-dose oral solid formulation once or twice a day.

16. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in twice-daily doses, with each dose being a multiple-dose formulation consisting of single doses of 5 mg, 10 mg, 15 mg and/or 20 mg of the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof.

17. The method according to claim 1 , wherein the method comprises administering to the patient the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof, or the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof in twice-daily dose, with each dose being a single dose of 5 mg, 10 mg, 15 mg and/or 20 mg of the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof.

18. The method according to claim 1 , wherein the method comprises treating the patient over one treatment cycle of 28 days, and administering to the patient the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof consecutively from day 1 to day 28.

19. The method according to claim 1 , wherein the method comprises treating the patient over one treatment cycle of 28 days, and administering to the patient a total dose of 140-840 mg of the pharmaceutical composition comprising the compound of formula I, or the stereoisomer or pharmaceutically acceptable salt thereof.

20. The method according to claim 19 , wherein the total dose is selected from the group consisting of 140 mg, 280 mg, 420 mg, 560 mg, 700 mg and 840 mg or a range defined by any two of the aforementioned values.

Assignees

• Chia Tai Tianqing Pharmaceutical Group Co Ltd
• Lianyungang Runzhong Pharmaceutical Co Ltd
• Shouyao Holdings Beijing Co Ltd

Inventors

• Liang Hongming
• Zhang Xiquan
• Wang Xunqiang
• Yu Ding
• Wang Dong
• Ge Qi
• Wang Zhao
• Wang Yini

Classifications

• A61K9/2054

  Cellulose; Cellulose derivatives, e.g. hydroxypropyl methylcellulose · CPC title

• A61K9/2018

  Sugars, or sugar alcohols, e.g. lactose, mannitol; Derivatives thereof, e.g. polysorbates · CPC title

• A61K9/2013

  Organic compounds, e.g. phospholipids, fats · CPC title

• A61P37/06

  Immunosuppressants, e.g. drugs for graft rejection · CPC title

• A61P7/00 Primary

  Drugs for disorders of the blood or the extracellular fluid · CPC title