IP Library › Granted Patent US 12,697,316
Granted Patent B2
US 12,697,316 · App. 17/623,313 · Granted Aug 4, 2026

Combination therapy with acetyl-leucine and miglustat

Inventors: Mallory Factor (Mayfair, GB); Michael Strupp (Munich, DE)
Assignee: INTRABIO LTD.
A61K31/197A61K9/0053A61K31/445A61P3/00
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Quick Facts
Patent No.
US 12,697,316
App. No.
17/623,313
Filed
Dec 28, 2021
Granted
Aug 4, 2026
Kind
B2
Art Unit
1654
USPC
514/1.1
Abstract

The present disclosure provides methods of treating lysosomal storage disorders (LSDs) in a subject in need thereof by administering a combination of acetyl-leucine and miglustat to the subject, wherein subject is naive to treatment with miglustat.

Claims (24)

1 . A method of reducing, or ameliorating Niemann-Pick Disease Type C, or a symptom thereof, in a subject in need thereof, the method comprising administering a combination of:

(i) a therapeutically effective amount of acetyl-leucine; and

(ii) a therapeutically effective amount of miglustat,

to the subject, wherein the subject is naive to treatment with miglustat, and the combination of acetyl-leucine and miglustat reduces weight loss associated with the administration of miglustat.

2 . The method of claim 1 , wherein acetyl-leucine and miglustat are administered simultaneously.

3 . The method of claim 2 , wherein acetyl-leucine and miglustat are administered as a single pharmaceutical formulation.

4 . The method of claim 2 , wherein acetyl-leucine and miglustat are administered as two separate pharmaceutical formulations.

5 . The method of claim 1 , wherein acetyl-leucine and miglustat are administered sequentially.

6 . The method of claim 5 , wherein acetyl-leucine is administered before miglustat.

7 . The method of claim 5 , wherein acetyl-leucine is administered after miglustat.

8 . The method of claim 5 , wherein acetyl-leucine and miglustat are administered about 1 minute to about 6 hours apart.

9 . The method of claim 8 , wherein acetyl-leucine and miglustat are administered about 1 minute to 3 hours apart.

10 . The method of claim 9 , wherein acetyl-leucine and miglustat are administered about 1 minute to 1 hour apart.

11 . The method of claim 1 , wherein acetyl-leucine and miglustat are administered orally.

12 . The method of claim 1 , wherein acetyl-leucine is administered once, twice, or three times per day.

13 . The method of claim 1 , wherein miglustat is administered once, twice, or three times per day.

14 . The method of claim 1 , wherein about 3 g to about 15 g of acetyl-leucine is administered per day.

15 . The method of claim 1 , wherein about 0.05 g to about 2 g of miglustat is administered per day.

16 . The method of claim 1 , wherein acetyl-leucine and miglustat are administered as a first-line therapy to treat Niemann-Pick Disease Type C, or a symptom thereof.

17 . The method of claim 1 , wherein the acetyl-leucine is N-acetyl-DL-leucine.

18 . The method of claim 1 , wherein the acetyl-leucine is N-acetyl-L-leucine.

19 . The method of claim 1 , wherein the combination of acetyl-leucine and miglustat reduces appetite suppression associated with the administration of miglustat.

20 . A kit for carrying out the method of claim 1 , the kit comprising acetyl-leucine and miglustat for treating Niemann-Pick Disease Type C, or a symptom thereof, in a subject.

21 . The kit of claim 20 further comprising instructions for administering the acetyl-leucine and miglustat to the subject.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 27, 2022
From: STRUPP, MICHAEL; FACTOR, MALLORY
To: INTRABIO LTD.
Reel/Frame 061227/0639 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 27, 2022
From: STRUPP, MICHAEL; FACTOR, MALLORY
To: INTRABIO LTD.
Reel/Frame 061227/0722 →
Continuity (3)
Provisional Application 62931003 · Nov 5, 2019
Provisional Application 62868383 · Jun 28, 2019
Related Publication 20220362189A1 · Nov 17, 2022
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