IP Library Granted Patent US 11,773,097
Granted Patent B2
US 11,773,097 · App. 17/342,910 · Granted Oct 3, 2023

Polymorphs of sepiapterin and salts thereof

Inventor: Daniel E. Levy (San Mateo, CA)
Assignee: PTC Therapeutics MP, Inc.
C07D475/04
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Quick Facts
Patent No.
US 11,773,097
App. No.
17/342,910
Granted
Oct 3, 2023
Kind
B2
Abstract

Disclosed are crystalline forms of sepiapterin free base selected from polymorphs A, B, C, D, E, F, and G, and combinations thereof, as well as crystalline polymorphs of salts of sepiapterin. Also disclosed are pharmaceutical compositions containing one or more such polymorphs and methods for preparing such polymorphs. Sepiapterin is useful in the treatment of a number diseases associated with low cellular levels of BH4, for example, phenylketonuria.

Claims (17)

1. A pharmaceutical composition comprising crystalline Form D of sepiapterin free base having peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, and about 26.0° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry and a pharmaceutically acceptable carrier.

2. The pharmaceutical composition of claim 1 , wherein the crystalline Form D is present in an amount of at least 90 percent by weight of the composition.

3. The pharmaceutical composition of claim 1 , wherein the crystalline Form D is formulated as particles less than 100 μm in size.

4. The pharmaceutical composition of claim 1 , wherein the crystalline Form D has peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, about 10.9°, about 17.8°, about 24.9°, about 26.0°, about 26.7°, about 26.8°, and about 28.3° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

5. A method of treating a tetrahydrobiopterin (BH4)-related disorder in a subject in need thereof, the method comprising administering an effective amount of the pharmaceutical composition of claim 1 to the subject, wherein the BH4-related disorder is selected from primary BH4 deficiency and phenylketonuria.

6. The method of claim 5 , wherein the pharmaceutical composition is a liquid suspension.

7. A method of treating a tetrahydrobiopterin (BH4)-related disorder in a subject in need thereof, the method comprising administering an effective amount of crystalline Form D of sepiapterin free base having peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, and about 26.0° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry to the subject, wherein the BH4-related disorder is selected from primary BH4 deficiency and phenylketonuria.

8. The method of claim 7 , wherein the crystalline Form D of sepiapterin free base is administered as a liquid suspension.

9. The method of claim 7 , wherein the crystalline Form D has peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, about 10.9°, about 17.8°, about 24.9°, about 26.0°, about 26.7°, about 26.8°, and about 28.3° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

10. A method for decreasing phenylalanine levels in a subject in need thereof, the method comprising administering to the patient an effective amount of the pharmaceutical composition of claim 1 to the subject.

11. The method of claim 10 , wherein the pharmaceutical composition is a liquid suspension.

12. A method for decreasing phenylalanine levels in a subject in need thereof, the method comprising administering an effective amount of crystalline Form D of sepiapterin free base having peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, and about 26.0° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry to the subject.

13. The method of claim 12 , wherein the crystalline Form D of sepiapterin free base is administered as a liquid suspension.

14. The method of claim 12 , wherein the crystalline Form D has peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, about 10.9°, about 17.8°, about 24.9°, about 26.0°, about 26.7°, about 26.8°, and about 28.3° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

15. A method of preparing a liquid formulation comprising dispersing crystalline Form D of sepiapterin free base having peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, and about 26.0° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry in a liquid.

16. The method of claim 15 , wherein the liquid formulation is a suspension.

17. The method of claim 15 , wherein the crystalline Form D has peaks at diffraction angle 2θ (°) of about 8.9°, about 10.3°, about 10.9°, about 17.8°, about 24.9°, about 26.0°, about 26.7°, about 26.8°, and about 28.3° as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

Assignments (4)
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 Nov 17, 2021
From: LEVY, DANIEL E.
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 058136/0668 →
CHANGE OF NAME Recorded Nov 17, 2021
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 058166/0875 →
Continuity (4)
Continuation 16464771
Provisional Application 62546390 · Aug 16, 2017
Provisional Application 62427686 · Nov 29, 2016
Related Publication 20210300930A1 · Sep 30, 2021
Cited By (7)
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