IP Library Granted Patent US 11,130,760
Granted Patent B2
US 11,130,760 · App. 16/464,916 · Granted Sep 28, 2021

Polymorphic form of sepiapterin

Inventors: Hiroshi Yoshino (Narashino, JP); Taichi Komoda (Narashino, JP); Yuichi Shiro (Narashino, JP); Shunichi Murata (Narashino, JP); Takayoshi Matsumoto (Narashino, JP); Kaito Kishimoto (Narashino, JP); Daniel E. Levy (San Mateo, CA)
Assignee: PTC Therapeutics MP, Inc.
C07D475/04C07B2200/13
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Quick Facts
Patent No.
US 11,130,760
App. No.
16/464,916
Granted
Sep 28, 2021
Kind
B2
Abstract

Disclosed is a crystalline form of sepiapterin, a method of preparing the crystalline form, pharmaceutical compositions containing the crystalline form, and a method for treating patients with a disease associated with low intracellular BH4 levels or with dysfunction of various BH4 dependent metabolic pathways, which involves administering to the patient an effective amount of the crystalline form.

Claims (21)

1. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising:

(i) combining sepiapterin free base and hydrochloric acid;

(ii) isolating the hydrochloride salt of sepiapterin formed in step (i); and

(iii) neutralizing the hydrochloride salt of sepiapterin obtained in step (ii) with a base to obtain crystalline Form A of sepiapterin free base.

2. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising dissolving sepiapterin free base in dimethyl acetamide, adding to the solution acetone, ethyl acetate, or THF, and isolating the solids to obtain the crystalline Form A of sepiapterin free base.

3. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising dissolving sepiapterin free base in dimethyl sulfoxide, adding to the solution isopropyl alcohol, and cooling the solution to obtain the crystalline Form A of sepiapterin free base.

4. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising preparing a slurry of sepiapterin free base in methyl tert-butyl ether, n-heptane, toluene, a mixture of chloroform and n-heptane, or a mixture of acetone and methyl tert-butyl ether, stirring the resulting suspension, and isolating the solids to obtain the crystalline Form A of sepiapterin free base.

5. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising exposing sepiapterin free base to a vapor of water, methyl t-butyl ether, n-heptane, or toluene, and obtaining the crystalline Form A of sepiapterin free base.

6. A method of preparing crystalline Form A of sepiapterin free base having peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 12.4°±0.5, 26.2°±0.5, and 28.9°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays, comprising dissolving sepiapterin free base in dimethyl acetamide and exposing the solution to a vapor of dichloromethane and obtaining the crystalline Form A of sepiapterin free base.

7. The method of claim 1 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

8. The method of claim 1 , wherein Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, or an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

9. The method of claim 2 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

10. The method of claim 2 , wherein Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, or an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

11. The method of claim 3 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

12. The method of claim 3 , wherein Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, or an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

13. The method of claim 4 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

14. The method of claim 4 , wherein the Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

15. The method of claim 5 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

16. The method of claim 5 , wherein the Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, or an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

17. The method of claim 6 , wherein the Form A of sepiapterin free base has peaks at diffraction angle 2θ (°) of 4.7°±0.5, 7.4°±0.5, 9.5°±0.5, 11.3°±0.5, 12.4°±0.5, 15.6°±0.5, 16.4°±0.5, 26.2°±0.5, 27.2°±0.5, and 28.9°±0.5, as measured by X-ray diffractometry by irradiation with Cu Kα X-rays.

18. The method of claim 6 , wherein the Form A of sepiapterin free base has one or more of the following: the X-ray powder diffraction spectrum essentially as shown in FIG. 1 , a loss of weight from 30° C. to 150° C. of less than 15% as measured by thermal gravimetric analysis, an endothermic onset at about 84° C. or about 180° C. in differential scanning calorimetry (DSC) profile, or an endothermic onset at about 84° C. and about 180° C. in differential scanning calorimetry (DSC) profile.

Assignments (6)
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 Jun 28, 2021
From: YOSHINO, HIROSHI; KOMODA, TAICHI; SHIRO, YUICHI; MURATA, SHUNICHI; MATSUMOTO, TAKAYOSHI; KISHIMOTO, KAITO
To: SHIRATORI PHARMACEUTICALS CO., LTD.
Reel/Frame 056686/0235 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 28, 2021
From: SHIRATORI PHARMACEUTICALS CO., LTD.
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 056686/0271 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 28, 2021
From: LEVY, DANIEL E.
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 056686/0287 →
CHANGE OF NAME Recorded Jul 23, 2020
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 053300/0057 →
Continuity (2)
Provisional Application 62427686 · Nov 29, 2016
Related Publication 20200010469A1 · Jan 9, 2020
Cited By (8)
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