Granted Patent
B2
US 12,257,252 · App. 17/059,632 · Granted Mar 25, 2025
Compositions and methods for increasing tetrahydrobiopterin plasma exposure
Assignee:
PTC Therapeutics MP, Inc.
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Abstract
The present invention features compositions including sepiapterin, or a pharmaceutically acceptable salt thereof, and methods for the treatment of BH4-related disorders. In some embodiments, these compositions and methods result in an increase in plasma exposure of BH4.
Claims (14)
1. A method of therapeutic treatment of a BH4-related disorder in a subject in need thereof, the method comprising administering to the subject an effective amount of sepiapterin, or a pharmaceutically acceptable salt thereof, with food,
wherein the BH4-related disorder is a BH4 deficiency, high plasma phenylalanine, or phenylketonuria.
2. The method of claim 1 , wherein the effective amount is an amount sufficient to produce a concentration of at least 50 ng/ml in the plasma of the subject within 10 hours of administration.
3. The method of claim 2 , wherein the effective amount comprises a dose that is at least 20% lower than the dose sufficient to produce a maximum BH4 plasma concentration (Cmax) of at least 50 ng/mL in the plasma of the subject within 10 hours of administration of sepiapterin, or a pharmaceutically acceptable salt thereof, without food.
4. The method of claim 1 , wherein the effective amount is 2.5 mg/kg to 100 mg/kg per dose.
5. The method of claim 1 , wherein the administration to the subject occurs less than 30 minutes prior to consuming food.
6. The method of claim 1 , wherein the administration to the subject is substantially at the same time as food.
7. The method of claim 1 , wherein the administration is immediately after the consumption of food up to 2 hours after the consumption.
8. The method of claim 1 , wherein the effective amount results in an increase in the maximum plasma concentration (Cmax) of BH4 compared to administration without food.
9. The method of claim 1 , wherein the effective amount results in an increase in the area under the concentration time curve from time zero to last concentration (AUC 0-last ) of BH4 compared to administration without food.
10. The method of claim 1 , wherein the BH4-related disorder is a BH4 deficiency.
11. The method of claim 10 , wherein the BH4 deficiency is primary BH4 deficiency.
12. The method of claim 1 , wherein the BH4-related disorder is phenylketonuria.
13. The method of claim 1 , wherein the BH4-related disorder is high plasma phenylalanine.
Assignments (6)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded May 14, 2025
From: SMITH, NEIL; REIS, JONATHAN
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 071108/0896 →
CHANGE OF NAME
Recorded May 14, 2025
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 071275/0555 →
TERMINATION AND RELEASE OF PATENT SECURITY AGREEMENT @ REEL 061584 AND FRAME 0377
Recorded Oct 23, 2023
From: WILMINGTON TRUST, NATIONAL ASSOCIATION, AS ADMINISTRATIVE AGENT
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 065321/0025 →
SECURITY INTEREST
Recorded Oct 28, 2022
From: PTC THERAPEUTICS MP, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 061584/0377 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Apr 28, 2021
From: SMITH, NEIL; REIS, JONATHAN
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 056069/0973 →
CHANGE OF NAME
Recorded Apr 28, 2021
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 056078/0408 →
Priority Claims (1)
GC 2019/37662
· May 28, 2019
· regional
Continuity (4)
Provisional Application
62822365
· Mar 22, 2019
Provisional Application
62771397
· Nov 26, 2018
Provisional Application
62677926
· May 30, 2018
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Cited By (1)