AU2019277372B2 - Pharmaceutically acceptable salts of sepiapterin - Google Patents

Pharmaceutically acceptable salts of sepiapterin Download PDF

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Publication number
AU2019277372B2
AU2019277372B2 AU2019277372A AU2019277372A AU2019277372B2 AU 2019277372 B2 AU2019277372 B2 AU 2019277372B2 AU 2019277372 A AU2019277372 A AU 2019277372A AU 2019277372 A AU2019277372 A AU 2019277372A AU 2019277372 B2 AU2019277372 B2 AU 2019277372B2
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salt
crystal
subject
sepiapterin
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AU2019277372A1 (en
Inventor
Kaito KISHIMOTO
Taichi KOMODA
Daniel E. Levy
Takayoshi Matsumoto
Shunichi MURATA
Jonathan Reis
Yuichi Shiro
Neil Smith
Hiroshi Yoshino
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PTC Therapeutics MP Inc
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PTC Therapeutics MP Inc
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Priority to AU2025201002A priority Critical patent/AU2025201002A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C209/00—Preparation of compounds containing amino groups bound to a carbon skeleton
    • C07C209/04—Preparation of compounds containing amino groups bound to a carbon skeleton by substitution of functional groups by amino groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C209/00—Preparation of compounds containing amino groups bound to a carbon skeleton
    • C07C209/30—Preparation of compounds containing amino groups bound to a carbon skeleton by reduction of nitrogen-to-oxygen or nitrogen-to-nitrogen bonds
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C309/00—Sulfonic acids; Halides, esters, or anhydrides thereof
    • C07C309/01—Sulfonic acids
    • C07C309/02—Sulfonic acids having sulfo groups bound to acyclic carbon atoms
    • C07C309/03—Sulfonic acids having sulfo groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C07C309/04—Sulfonic acids having sulfo groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton containing only one sulfo group
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C309/00—Sulfonic acids; Halides, esters, or anhydrides thereof
    • C07C309/01—Sulfonic acids
    • C07C309/28—Sulfonic acids having sulfo groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton
    • C07C309/29—Sulfonic acids having sulfo groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton of non-condensed six-membered aromatic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C309/00—Sulfonic acids; Halides, esters, or anhydrides thereof
    • C07C309/01—Sulfonic acids
    • C07C309/28—Sulfonic acids having sulfo groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton
    • C07C309/29—Sulfonic acids having sulfo groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton of non-condensed six-membered aromatic rings
    • C07C309/30—Sulfonic acids having sulfo groups bound to carbon atoms of six-membered aromatic rings of a carbon skeleton of non-condensed six-membered aromatic rings of six-membered aromatic rings substituted by alkyl groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C55/00—Saturated compounds having more than one carboxyl group bound to acyclic carbon atoms
    • C07C55/02—Dicarboxylic acids
    • C07C55/08—Malonic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C57/00—Unsaturated compounds having carboxyl groups bound to acyclic carbon atoms
    • C07C57/02—Unsaturated compounds having carboxyl groups bound to acyclic carbon atoms with only carbon-to-carbon double bonds as unsaturation
    • C07C57/13—Dicarboxylic acids
    • C07C57/15—Fumaric acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C59/00—Compounds having carboxyl groups bound to acyclic carbon atoms and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups
    • C07C59/01—Saturated compounds having only one carboxyl group and containing hydroxy or O-metal groups
    • C07C59/06—Glycolic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C65/00—Compounds having carboxyl groups bound to carbon atoms of six—membered aromatic rings and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups
    • C07C65/01—Compounds having carboxyl groups bound to carbon atoms of six—membered aromatic rings and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups containing hydroxy or O-metal groups
    • C07C65/03—Compounds having carboxyl groups bound to carbon atoms of six—membered aromatic rings and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups containing hydroxy or O-metal groups monocyclic and having all hydroxy or O-metal groups bound to the ring
    • C07C65/05—Compounds having carboxyl groups bound to carbon atoms of six—membered aromatic rings and containing any of the groups OH, O—metal, —CHO, keto, ether, groups, groups, or groups containing hydroxy or O-metal groups monocyclic and having all hydroxy or O-metal groups bound to the ring o-Hydroxy carboxylic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/60—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D213/78—Carbon atoms having three bonds to hetero atoms, with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D213/79—Acids; Esters
    • C07D213/80—Acids; Esters in position 3
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D475/00—Heterocyclic compounds containing pteridine ring systems
    • C07D475/02—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4
    • C07D475/04—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07B—GENERAL METHODS OF ORGANIC CHEMISTRY; APPARATUS THEREFOR
    • C07B2200/00—Indexing scheme relating to specific properties of organic compounds
    • C07B2200/13—Crystalline forms, e.g. polymorphs

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AU2019277372A 2018-05-30 2019-05-30 Pharmaceutically acceptable salts of sepiapterin Active AU2019277372B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025201002A AU2025201002A1 (en) 2018-05-30 2025-02-13 Pharmaceutically acceptable salts of sepiapterin

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862678025P 2018-05-30 2018-05-30
US62/678,025 2018-05-30
US201862726612P 2018-09-04 2018-09-04
US62/726,612 2018-09-04
US201962822336P 2019-03-22 2019-03-22
US62/822,336 2019-03-22
GC201937661 2019-05-28
GC2019/37661 2019-05-28
PCT/US2019/034505 WO2019232120A1 (en) 2018-05-30 2019-05-30 Pharmaceutically acceptable salts of sepiapterin

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025201002A Division AU2025201002A1 (en) 2018-05-30 2025-02-13 Pharmaceutically acceptable salts of sepiapterin

Publications (2)

Publication Number Publication Date
AU2019277372A1 AU2019277372A1 (en) 2021-01-21
AU2019277372B2 true AU2019277372B2 (en) 2024-12-19

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AU2019277372A Active AU2019277372B2 (en) 2018-05-30 2019-05-30 Pharmaceutically acceptable salts of sepiapterin
AU2025201002A Pending AU2025201002A1 (en) 2018-05-30 2025-02-13 Pharmaceutically acceptable salts of sepiapterin

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Country Status (13)

Country Link
US (1) US12637465B2 (enExample)
EP (1) EP3807279B1 (enExample)
JP (2) JP7778477B2 (enExample)
KR (1) KR20210038848A (enExample)
CN (1) CN112543758A (enExample)
AU (2) AU2019277372B2 (enExample)
BR (1) BR112020024332A2 (enExample)
CA (1) CA3102070A1 (enExample)
CL (1) CL2020003101A1 (enExample)
CO (1) CO2020016582A2 (enExample)
IL (1) IL279101A (enExample)
MX (1) MX2020012978A (enExample)
WO (1) WO2019232120A1 (enExample)

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CN110312721A (zh) 2016-11-29 2019-10-08 显莎制药公司 墨蝶呤的多晶型物
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MX2020002271A (es) 2017-09-01 2020-10-05 Ptc Therapeutics Mp Inc Composiciones farmaceuticas que comprenden sepiapterina y sus usos de las mismas.
CA3102105A1 (en) * 2018-05-30 2019-12-05 Ptc Therapeutics Mp, Inc. Compositions and methods for increasing tetrahydrobiopterin plasma exposure
JP7778477B2 (ja) * 2018-05-30 2025-12-02 ピーティーシー セラピューティクス エムピー，インコーポレイテッド セピアプテリンの薬学的に許容される塩
JP7502197B2 (ja) 2018-05-30 2024-06-18 ピーティーシー セラピューティクス エムピー，インコーポレイテッド セピアプテリン血漿曝露を増加させるための方法
LT4009978T (lt) 2019-08-05 2024-11-25 Ptc Therapeutics Mp, Inc. Sepiapterino ir jo metabolitų panaudojimas spinduliuotės poveikio gydymui
IL291565B1 (en) 2019-09-25 2026-04-01 Ptc Therapeutics Inc Method for treating phenylalanine irregularity
CA3165636A1 (en) 2020-01-24 2021-07-29 Neil Smith Methods for treating parkinson's disease with sepiapterin
CN114621198B (zh) * 2020-12-11 2025-01-21 北京夏禾科技有限公司 有机电致发光材料及其器件
US12691119B2 (en) 2021-02-09 2026-07-28 Ptc Therapeutics Mp, Inc. Methods for treating covid-19 with sepiapterin
IL305037A (en) 2021-02-09 2023-10-01 Ptc Therapeutics Mp Inc Methods of treating glioblastomas with spiafatrin

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WO2018102315A1 (en) * 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymoprhs of sepiapterin and salts thereof
WO2018102314A1 (en) * 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymorphic form of sepiapterin
WO2019046849A1 (en) * 2017-09-01 2019-03-07 Censa Pharmaceuticals Inc. PHARMACEUTICAL COMPOSITION COMPRISING SEPAPTERINE AND USES THEREOF

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JP7502197B2 (ja) 2018-05-30 2024-06-18 ピーティーシー セラピューティクス エムピー，インコーポレイテッド セピアプテリン血漿曝露を増加させるための方法
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IL291565B1 (en) 2019-09-25 2026-04-01 Ptc Therapeutics Inc Method for treating phenylalanine irregularity

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US20180078557A1 (en) * 2010-04-22 2018-03-22 Hiroyuki Hasegawa Medicinal agent and beverage/food for preventing cerebral dysfunction and improvidng same
US9181254B2 (en) * 2012-05-07 2015-11-10 Shiratori Pharmaceutical Co., Ltd. Method for producing sepiapterin and tetrahydrolactoylpterin
WO2018102315A1 (en) * 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymoprhs of sepiapterin and salts thereof
WO2018102314A1 (en) * 2016-11-29 2018-06-07 Censa Pharmaceuticals Inc. Polymorphic form of sepiapterin
WO2019046849A1 (en) * 2017-09-01 2019-03-07 Censa Pharmaceuticals Inc. PHARMACEUTICAL COMPOSITION COMPRISING SEPAPTERINE AND USES THEREOF

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CL2020003101A1 (es) 2021-05-14
MX2020012978A (es) 2021-04-28
JP2024063143A (ja) 2024-05-10
JP7841008B2 (ja) 2026-04-06
JP2021527031A (ja) 2021-10-11
EP3807279A1 (en) 2021-04-21
EP3807279B1 (en) 2025-12-31
US20210269443A1 (en) 2021-09-02
WO2019232120A1 (en) 2019-12-05
KR20210038848A (ko) 2021-04-08
JP7778477B2 (ja) 2025-12-02
CO2020016582A2 (es) 2021-04-08
AU2025201002A1 (en) 2025-03-06
EP3807279A4 (en) 2022-04-06
CN112543758A (zh) 2021-03-23
IL279101A (en) 2021-01-31
CA3102070A1 (en) 2019-12-05
BR112020024332A2 (pt) 2021-02-23
US12637465B2 (en) 2026-05-26
AU2019277372A1 (en) 2021-01-21

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