CA3102070A1 - Pharmaceutically acceptable salts of sepiapterin - Google Patents

Pharmaceutically acceptable salts of sepiapterin Download PDF

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Publication number
CA3102070A1
CA3102070A1 CA3102070A CA3102070A CA3102070A1 CA 3102070 A1 CA3102070 A1 CA 3102070A1 CA 3102070 A CA3102070 A CA 3102070A CA 3102070 A CA3102070 A CA 3102070A CA 3102070 A1 CA3102070 A1 CA 3102070A1
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CA
Canada
Prior art keywords
crystal
salt
pharmaceutically acceptable
acceptable salt
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3102070A
Other languages
English (en)
French (fr)
Inventor
Daniel E. Levy
Neil Smith
Jonathan Reis
Hiroshi Yoshino
Taichi KOMODA
Yuichi Shiro
Shunichi MURATA
Takayoshi Matsumoto
Kaito KISHIMOTO
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
PTC Therapeutics MP Inc
Original Assignee
PTC Therapeutics MP Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by PTC Therapeutics MP Inc filed Critical PTC Therapeutics MP Inc
Publication of CA3102070A1 publication Critical patent/CA3102070A1/en
Pending legal-status Critical Current

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Classifications

    • 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
    • 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
    • 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (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)
CA3102070A 2018-05-30 2019-05-30 Pharmaceutically acceptable salts of sepiapterin Pending CA3102070A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3102070A1 true CA3102070A1 (en) 2019-12-05

Family

ID=68699017

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3102070A Pending CA3102070A1 (en) 2018-05-30 2019-05-30 Pharmaceutically acceptable salts of sepiapterin

Country Status (13)

Country Link
US (1) US20210269443A1 (https=)
EP (1) EP3807279B1 (https=)
JP (2) JP7778477B2 (https=)
KR (1) KR20210038848A (https=)
CN (1) CN112543758A (https=)
AU (2) AU2019277372B2 (https=)
BR (1) BR112020024332A2 (https=)
CA (1) CA3102070A1 (https=)
CL (1) CL2020003101A1 (https=)
CO (1) CO2020016582A2 (https=)
IL (1) IL279101A (https=)
MX (1) MX2020012978A (https=)
WO (1) WO2019232120A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11072614B2 (en) 2016-11-29 2021-07-27 Ptc Therapeutics Mp, Inc. Polymorphs of sepiapterin and salts thereof
US11130760B2 (en) 2016-11-29 2021-09-28 Ptc Therapeutics Mp, Inc. Polymorphic form of sepiapterin
JP7811085B2 (ja) 2017-09-01 2026-02-04 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 セピアプテリンを含む医薬組成物及びその使用
EP3801536B1 (en) 2018-05-30 2024-07-17 PTC Therapeutics MP, Inc. Administration of sepiapterin without food for use in a method for increasing sepiapterin plasma exposure
AU2019277372B2 (en) * 2018-05-30 2024-12-19 Ptc Therapeutics Mp, Inc. Pharmaceutically acceptable salts of sepiapterin
CN112654356A (zh) * 2018-05-30 2021-04-13 Ptc医疗Mp公司 增加四氢生物蝶呤血浆暴露的组合物和方法
IL290321B1 (en) 2019-08-05 2026-04-01 Ptc Therapeutics Inc Use of spiapterin and its metabolites for radiation exposure treatment
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
JP2024506336A (ja) 2021-02-09 2024-02-13 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 膠芽腫の治療方法
EP4291196A1 (en) 2021-02-09 2023-12-20 PTC Therapeutics MP, Inc. Methods for treating covid-19 with sepiapterin

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5910304A (en) 1982-12-13 1999-06-08 Texas A&M University System Low-dose oral administration of interferons
CA1320905C (en) 1986-11-06 1993-08-03 Joseph M. Cummins Treatment of immuno-resistant disease
ATE218345T1 (de) * 1994-08-05 2002-06-15 Suntory Ltd Arzneimittel gegen spinocerebellare degeneration
US6036949A (en) 1998-03-05 2000-03-14 Amarillo Biosciences, Inc. Treatment of fibromyalgia with low doses of interferon
WO2008128049A2 (en) * 2007-04-11 2008-10-23 Biomarin Pharmaceutical Inc. Methods of administering tetrahydrobiopterin, associated compositions, and methods of measuring
JP2012512848A (ja) 2008-12-23 2012-06-07 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング 有機化合物の塩の形態
EP2562176B1 (en) 2010-04-22 2018-08-15 Nihon University Medicinal agent and beverage/food for preventing cerebral dysfunction and improving same
IN2014DN09053A (https=) 2012-05-07 2015-05-22 Shiratori Pharm
WO2017218421A1 (en) * 2016-06-13 2017-12-21 Meharry Medical College Modulation of the nitric oxide synthase pathway for oral health
US11130760B2 (en) * 2016-11-29 2021-09-28 Ptc Therapeutics Mp, Inc. Polymorphic form of sepiapterin
US11072614B2 (en) * 2016-11-29 2021-07-27 Ptc Therapeutics Mp, Inc. Polymorphs of sepiapterin and salts thereof
JP7811085B2 (ja) 2017-09-01 2026-02-04 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 セピアプテリンを含む医薬組成物及びその使用
AU2019277372B2 (en) * 2018-05-30 2024-12-19 Ptc Therapeutics Mp, Inc. Pharmaceutically acceptable salts of sepiapterin

Also Published As

Publication number Publication date
AU2019277372A1 (en) 2021-01-21
EP3807279A4 (en) 2022-04-06
CL2020003101A1 (es) 2021-05-14
IL279101A (en) 2021-01-31
AU2025201002A1 (en) 2025-03-06
CN112543758A (zh) 2021-03-23
WO2019232120A1 (en) 2019-12-05
JP7841008B2 (ja) 2026-04-06
EP3807279B1 (en) 2025-12-31
JP2021527031A (ja) 2021-10-11
US20210269443A1 (en) 2021-09-02
KR20210038848A (ko) 2021-04-08
MX2020012978A (es) 2021-04-28
CO2020016582A2 (es) 2021-04-08
BR112020024332A2 (pt) 2021-02-23
AU2019277372B2 (en) 2024-12-19
EP3807279A1 (en) 2021-04-21
JP7778477B2 (ja) 2025-12-02
JP2024063143A (ja) 2024-05-10

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