IP Library Granted Patent US 9,675,627
Granted Patent B2
US 9,675,627 · App. 14/684,967 · Granted Jun 13, 2017

Dosing regimens for treating and/or preventing cerebral amyloidoses

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Quick Facts
Patent No.
US 9,675,627
App. No.
14/684,967
Granted
Jun 13, 2017
Kind
B2
Abstract

Described herein are dosing regimens and kits for the treatment and/or prevention of cerebral amyloidoses such as Alzheimer's disease (AD) and/or cerebral amyloid angiopathy (CAA).

Claims (34)

1. A method for treating Alzheimer's disease or cerebral amyloid angiopathy in a patient diagnosed with the same, the method comprising:

administering daily to the patient an effective amount of 2-acetamido-1,2-dideoxynojirimycin (AdDNJ) for a first enzyme enhancement period of about 1 day to about 8 days;

not administering the AdDNJ for a substrate turnover period of about 48 hours to about 96 hours; and then

administering to the patient an effective amount of AdDNJ for a second enzyme enhancement period of about 1 day to about 8 days.

2. The method of claim 1 , wherein the first enzyme enhancement period and the second enzyme enhancement period have the same duration.

3. The method of claim 2 , wherein one or more of the first enzyme enhancement period and the second enzyme enhancement period is a period of about 4 days to about 6 days.

4. The method of claim 1 , wherein the AdDNJ is administered orally.

5. The method of claim 1 , wherein the AdDNJ is administered at a dose in the range of about 3 mg/kg/day to about 300 mg/kg/day.

6. The method of claim 5 , wherein the AdDNJ is administered at a dose of about 100 mg/kg/day.

7. The method of claim 6 , wherein not administering the AdDNJ for the substrate turnover period comprises not administering AdDNJ for a period of about 72 hours.

8. A method for treating Alzheimer's disease or cerebral amyloid angiopathy in a patient diagnosed with the same, the method comprising:

administering daily to the patient an effective amount of 2-acetamido-1,2-dideoxynojirimycin (AdDNJ) for a first enzyme enhancement period of about 5 days;

not administering the AdDNJ for a substrate turnover period of about 72 hours; and then

administering to the patient an effective amount of AdDNJ for a second enzyme enhancement period of about 1 day to about 8 days.

9. A method for treating Alzheimer's disease or cerebral amyloid angiopathy in a patient diagnosed with the same wherein the method comprises:

administering to the patient an effective amount of AdDNJ on a first day;

not administering the AdDNJ on a second day;

administering to the patient an effective amount of AdDNJ on a third day;

not administering the AdDNJ on a fourth day;

administering to the patient an effective amount of AdDNJ on a fifth day; and then

not administering the AdDNJ for a period of about 72 hours.

10. The method of claim 1 , wherein not administering the AdDNJ for a substrate turnover period comprises not administering AdDNJ for a period of about 24 hours.

11. The method of claim 1 , wherein not administering the AdDNJ for a substrate turnover period comprises not administering AdDNJ for a period of about 48 hours.

12. The method of claim 1 , wherein the first enzyme enhancement period is about 3 days and not administering the AdDNJ for the substrate turnover period comprises not administering AdDNJ for a period of about 120 hours.

13. The method of claim 1 , wherein the method comprises alternating between enzyme enhancement periods and substrate turnover periods for a total treatment time of at least 1 month.

14. The method of claim 13 , wherein the total treatment time is at least 3 months.

15. The method of claim 13 , wherein at least one substrate turnover period has a different duration than at least one other substrate turnover period.

16. A kit comprising:

one or more active dosage forms comprising an effective amount of 2-acetamido-1,2-dideoxynojirimycin (AdDNJ);

one or more inactive dosage forms that do not comprise an effective amount of AdDNJ; and

instructions for:

administering the dosage forms for a first enzyme enhancement period of about 1 day to about 8 days;

not administering the dosage forms for a substrate turnover period of about 48 hours to about 96 hours; and then

administering the dosage forms for a second enzyme enhancement period of about 1 day to about 8 days.

Assignments (5)
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 4, 2015
From: WUSTMAN, BRANDON ALAN
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 036494/0768 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 31, 2015
From: WUSTMAN, BRANDON ALAN
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 036456/0484 →