Treatment for amyotrophic lateral sclerosis (ALS)

JP2026519584APending Publication Date: 2026-06-16ZYDUS LIFESCIENCES LTD

Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
ZYDUS LIFESCIENCES LTD
Filing Date
2024-05-23
Publication Date
2026-06-16

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を有し得る式(I)の化合物の使用を開示する。

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Abstract

The present invention relates to the development of therapeutic compounds for the treatment of amyotrophic lateral sclerosis (ALS). More specifically, the present invention provides NLRP3 inhibitors or pharmaceutically acceptable salts thereof or suitable compositions useful in the treatment of amyotrophic lateral sclerosis (ALS) and other related disorders, including progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedy disease, and post-polio syndrome. JPEG2026519584000011.jpg22170
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Claims

1. Equation (I) 【Chemistry 1】 This includes administering a compound or a pharmaceutically acceptable salt thereof. Methods for the treatment of amyotrophic lateral sclerosis (ALS) and other related forms of disorder.

2. The method according to claim 1, wherein other related forms of disorder are selected from progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedy disease, and post-polio syndrome.

3. The method according to claim 1, wherein the therapeutically effective dose of the compound of formula (1) or a pharmaceutically acceptable salt thereof is selected from 1 mg to 500 mg, preferably from 1 mg to 250 mg, and more preferably from 1 mg to 150 mg.

4. The method according to claim 1, wherein a compound of formula (1) or a pharmaceutically acceptable salt thereof is administered by an oral, topical, or parenteral route, preferably by an oral route.

5. The method according to claim 1, wherein a compound of formula (1) or a pharmaceutically acceptable salt thereof is administered in combination with another suitable therapeutic agent.

6. The method according to claim 1, wherein a compound of formula (1) or a pharmaceutically acceptable salt thereof is administered in the form of a pharmaceutical composition.

7. Use of a compound of formula (1) according to any one of claims 1 to 6 for the preparation of a medicament for the treatment of amyotrophic lateral sclerosis (ALS) and other related forms of disorder.

8. Use of a compound of formula (1) as defined in any one of claims 1 to 7, wherein the compound is administered in a daily dose range selected from 1 mg to 500 mg, preferably selected from 1 mg to 250 mg, and more preferably selected from 1 mg to 150 mg.

9. The compound of formula (1) 【Chemistry 2】 A pharmaceutical composition comprising a compound of formula (1) or a pharmaceutically acceptable salt thereof for the treatment of amyotrophic lateral sclerosis (ALS) and other related forms of disorder.

10. The pharmaceutical composition according to claim 9, wherein the other related form of disorder is selected from progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedy disease, and post-polio syndrome.

11. The pharmaceutical composition according to claim 9, wherein the therapeutically effective amount of the compound of formula (1) or a pharmaceutically acceptable salt thereof is selected from 1 mg to 500 mg, preferably from 1 mg to 250 mg, and more preferably from 1 mg to 150 mg.

12. The method according to claim 9, wherein the compound of formula (1) or a pharmaceutically acceptable salt thereof is administered by an oral, topical, or parenteral route, preferably by an oral route.

13. A pharmaceutical composition according to claim 9, comprising a compound of formula (1) and other pharmaceutically acceptable excipients, for the treatment of amyotrophic lateral sclerosis (ALS) and other related forms of disorder.

14. The pharmaceutical composition according to claim 9, wherein a compound of formula (1) or a pharmaceutically acceptable salt thereof is administered in combination with another suitable therapeutic agent.

15. Use of the pharmaceutical composition according to claim 9 for the preparation of a pharmaceutical for the treatment of amyotrophic lateral sclerosis (ALS) and other related forms of disorder.

16. The pharmaceutical composition according to claim 13, wherein the other pharmaceutically acceptable excipient is selected from diluents, carriers, binders, disintegrants, lubricants, encapsulating agents, and the like.

17. The pharmaceutical composition according to any one of claims 9 to 16, wherein, as a diluent, but not limited to, microcrystalline cellulose, polymethacrylates selected from: Eudragit, potassium chloride, sulfobutyl ether β-cyclodextrin, sodium chloride, and spray-dried lactose, combinations thereof, and other such materials known to those skilled in the art.

18. A pharmaceutical composition according to any one of claims 1 to 17, wherein the carrier is selected from lactose, white sugar, sodium chloride, glucose, urea, starch, calcium carbonate and kaolin, crystalline cellulose, silicic acid and suitable combinations thereof.

19. The pharmaceutical composition according to any one of claims 9 to 18, wherein the binder is Carbopol, gellan, gum arabic, hydrogenated vegetable oil, polymethacrylate selected from: Eudragit, xanthan gum, lactose and zein, pregelatinized starch, combinations thereof, and other such materials known to those skilled in the art.

20. The pharmaceutical composition according to any one of claims 9 to 19, wherein the disintegrant is selected from pregelatinized starch, bicarbonate, chitin, gellan gum, potassium polariphosphate and sodium docusate, combinations thereof, and other such materials known to those skilled in the art.

21. The pharmaceutical composition according to any one of claims 9 to 20, wherein the lubricant is selected from magnesium stearate, glyceryl behenate, hydrogenated vegetable oil, sodium stearyl fumarate, and myristic acid, combinations thereof, and other such materials known to those skilled in the art.

22. The pharmaceutical composition according to any one of claims 9 to 21, wherein the encapsulating agent is selected from empty hard gelatin capsule shells.

23. The compound of formula (I) 【Transformation 3】 A method for treating amyotrophic lateral sclerosis (ALS) and other related forms of disorder using a compound of formula (I) or a pharmaceutically acceptable salt thereof.