IP Library Patent Application 19021475
Patent Application
App. No. 19/021,475

POLYMORPHS OF SEPIAPTERIN AND SALTS THEREOF

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Patent No.
US None
App. No.
19/021,475
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 - 58 . (canceled)

59 . A pharmaceutical composition comprising crystalline Form B of sepiapterin free base having peaks at diffraction angle 2θ (°) of 8.4°±0.5, 16.9°±0.5, and 25.4°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry and a pharmaceutically acceptable carrier.

60 . The pharmaceutical composition of claim 59 , wherein the crystalline Form B of sepiapterin free base has peaks at diffraction angle 2θ (°) of 8.4°±0.5, 14.9°±0.5, 16.9°±0.5, 25.4°±0.5, and 34.1°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

61 . The pharmaceutical composition of claim 59 , wherein the crystalline Form B has the X-ray powder diffraction spectrum as shown in FIG. 1 .

62 . The pharmaceutical composition of claim 59 , wherein the crystalline Form B has an endotherm at about 195° C. in differential scanning calorimetry (DSC) profile.

63 . The pharmaceutical composition of claim 59 , wherein the crystalline Form B is present in an amount of at least 90 percent by weight of the composition.

64 . The pharmaceutical composition of claim 59 , wherein the crystalline Form B is formulated as particles less than 100 μm in size.

65 . A method of treating a tetrahydrobiopterin (BH4)-related disorder selected from primary BH4 deficiency and phenylketonuria in a subject in need thereof, the method comprising administering an effective amount of the pharmaceutical composition of claim 59 to the subject.

66 . 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 59 to the subject.

67 . A pharmaceutical composition comprising crystalline Form C of sepiapterin free base having peaks at diffraction angle 2θ (°) of 5.7°±0.5, 7.8°±0.5, and 25.4°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry and a pharmaceutically acceptable carrier.

68 . The pharmaceutical composition of claim 67 , wherein the crystalline Form C of sepiapterin free base has peaks at diffraction angle 2θ (θ) of 5.7°±0.5, 7.8°±0.5, 9.1°±0.5, 11.5°±0.5, 15.3°±0.5, 16.0°±0.5, 20.1°±0.5, 25.4°±0.5, and 26.6°±0.5 as measured by X-ray diffractometry by irradiation with Cu Kα X-rays or calculated from X-ray diffractometry.

69 . The pharmaceutical composition of claim 67 , wherein the crystalline Form C of sepiapterin free base has the X-ray powder diffraction spectrum as shown in FIG. 2 .

70 . The pharmaceutical composition of claim 67 , wherein the crystalline Form C of sepiapterin free base has endotherms at about 58° C., 102° C., 130° C., 156.5° C., and 168° C. in differential scanning calorimetry (DSC) profile.

71 . The pharmaceutical composition of claim 67 , wherein the crystalline Form C is present in an amount of at least 90 percent by weight of the composition.

72 . The pharmaceutical composition of claim 67 , wherein the crystalline Form C is formulated as particles less than 100 μm in size.

73 . A method of treating a tetrahydrobiopterin (BH4)-related disorder selected from primary BH4 deficiency and phenylketonuria in a subject in need thereof, the method comprising administering an effective amount of the pharmaceutical composition of claim 67 to the subject.

74 . 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 67 to the subject.

Assignments (2)
CHANGE OF NAME Recorded May 13, 2025
From: CENSA PHARMACEUTICALS INC.
To: PTC THERAPEUTICS MP, INC.
Reel/Frame 071279/0476 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 13, 2025
From: LEVY, DANIEL E.
To: CENSA PHARMACEUTICALS INC.
Reel/Frame 071279/0496 →