KR20220078644A - 미토콘드리아 질환 치료를 위한 바티퀴논의 퀴논-, 히드로퀴논- 및 나프토퀴논-유사체 - Google Patents

미토콘드리아 질환 치료를 위한 바티퀴논의 퀴논-, 히드로퀴논- 및 나프토퀴논-유사체 Download PDF

Info

Publication number
KR20220078644A
KR20220078644A KR1020227014887A KR20227014887A KR20220078644A KR 20220078644 A KR20220078644 A KR 20220078644A KR 1020227014887 A KR1020227014887 A KR 1020227014887A KR 20227014887 A KR20227014887 A KR 20227014887A KR 20220078644 A KR20220078644 A KR 20220078644A
Authority
KR
South Korea
Prior art keywords
och
ocf
compound
independently
ocd
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
KR1020227014887A
Other languages
English (en)
Korean (ko)
Inventor
데니스 키프
궈주 쳉
파벨스 아르센잔스
Original Assignee
스텔스 바이오테라퓨틱스 인코포레이티드
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 스텔스 바이오테라퓨틱스 인코포레이티드 filed Critical 스텔스 바이오테라퓨틱스 인코포레이티드
Publication of KR20220078644A publication Critical patent/KR20220078644A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C50/00—Quinones
    • C07C50/26—Quinones containing groups having oxygen atoms singly bound to carbon atoms
    • C07C50/28—Quinones containing groups having oxygen atoms singly bound to carbon atoms with monocyclic quinoid structure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D305/00—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms
    • C07D305/02—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings
    • C07D305/10—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having one or more double bonds between ring members or between ring members and non-ring members
    • 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
    • A61K31/12—Ketones
    • A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C07C39/00—Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring
    • C07C39/18—Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring monocyclic with unsaturation outside the aromatic ring
    • C07C39/19—Compounds having at least one hydroxy or O-metal group bound to a carbon atom of a six-membered aromatic ring monocyclic with unsaturation outside the aromatic ring containing carbon-to-carbon double bonds but no carbon-to-carbon triple bonds
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C49/00—Ketones; Ketenes; Dimeric ketenes; Ketonic chelates
    • C07C49/76—Ketones containing a keto group bound to a six-membered aromatic ring
    • C07C49/82—Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups
    • C07C49/835—Ketones containing a keto group bound to a six-membered aromatic ring containing hydroxy groups having unsaturation outside an aromatic ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D305/00—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms
    • C07D305/02—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings
    • C07D305/04—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C07D305/06—Heterocyclic compounds containing four-membered rings having one oxygen atom as the only ring hetero atoms not condensed with other rings having no double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to the ring atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
    • C07F7/02—Silicon compounds
    • C07F7/08—Compounds having one or more C—Si linkages
    • C07F7/0803—Compounds with Si-C or Si-Si linkages
    • C07F7/081—Compounds with Si-C or Si-Si linkages comprising at least one atom selected from the elements N, O, halogen, S, Se or Te
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/04—Systems containing only non-condensed rings with a four-membered ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/06—Systems containing only non-condensed rings with a five-membered ring
    • C07C2601/08—Systems containing only non-condensed rings with a five-membered ring the ring being saturated
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/12—Systems containing only non-condensed rings with a six-membered ring
    • C07C2601/14—The ring being saturated
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/12—Systems containing only non-condensed rings with a six-membered ring
    • C07C2601/16—Systems containing only non-condensed rings with a six-membered ring the ring being unsaturated

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
KR1020227014887A 2019-10-04 2020-10-02 미토콘드리아 질환 치료를 위한 바티퀴논의 퀴논-, 히드로퀴논- 및 나프토퀴논-유사체 Pending KR20220078644A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962911069P 2019-10-04 2019-10-04
US62/911,069 2019-10-04
US202062991525P 2020-03-18 2020-03-18
US62/991,525 2020-03-18
PCT/US2020/054107 WO2021067836A1 (en) 2019-10-04 2020-10-02 Chinone-, hydrochinome- and naphthochinone-analogues of vatiquione for treatment of mitochondrial disorder diseases

Publications (1)

Publication Number Publication Date
KR20220078644A true KR20220078644A (ko) 2022-06-10

Family

ID=72964859

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227014887A Pending KR20220078644A (ko) 2019-10-04 2020-10-02 미토콘드리아 질환 치료를 위한 바티퀴논의 퀴논-, 히드로퀴논- 및 나프토퀴논-유사체

Country Status (9)

Country Link
US (4) US11584728B2 (https=)
EP (1) EP4038076A1 (https=)
JP (2) JP2022551270A (https=)
KR (1) KR20220078644A (https=)
CN (2) CN121949079A (https=)
AU (1) AU2020358970B2 (https=)
CA (1) CA3156499A1 (https=)
IL (1) IL291898A (https=)
WO (1) WO2021067836A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121949079A (zh) * 2019-10-04 2026-05-01 康德生物医疗有限公司 治疗线粒体异常疾病的伐替苯醌的醌、氢醌和萘醌类似物
CA3222132A1 (en) * 2021-06-10 2022-12-15 Stealth Biotherapeutics Inc. Compounds, compositions and methods for the prevention and/or treatment of various mitochondrial diseases or disorders, including friedreich's ataxia
US11541116B1 (en) 2022-01-07 2023-01-03 Kojin Therapeutics, Inc. Methods and compositions for inducing ferroptosis in vivo
WO2025024247A1 (en) * 2023-07-21 2025-01-30 Stealth Biotherapeutics Inc. Methods, compositions and uses related to the treatment and prevention of leigh's syndrome
WO2025101748A1 (en) * 2023-11-09 2025-05-15 Stealth Biotherapeutics Inc. Cyclo-hexa-2,5-diene-1,4-diones for use in the treatment of cardiomyopathy
WO2025231245A1 (en) * 2024-05-02 2025-11-06 Stealth Biotherapeutics Inc. Use of substituted 1,4 benzoquinones to treat alpha-synucleinopathies
WO2026020091A1 (en) * 2024-07-19 2026-01-22 Stealth Biotherapeutics Inc. Methods, compounds, compositions, formulations and uses for addressing mitochondrial disease resulting from nuclear dna mutations

Family Cites Families (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
JP3507486B2 (ja) 1991-03-15 2004-03-15 アムジエン・インコーポレーテツド 顆粒球コロニー刺激因子の肺内投与
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5674534A (en) 1992-06-11 1997-10-07 Alkermes, Inc. Composition for sustained release of non-aggregated erythropoietin
US5716644A (en) 1992-06-11 1998-02-10 Alkermes, Inc. Composition for sustained release of non-aggregated erythropoietin
US5451569A (en) 1994-04-19 1995-09-19 Hong Kong University Of Science And Technology R & D Corporation Limited Pulmonary drug delivery system
US5989463A (en) 1997-09-24 1999-11-23 Alkermes Controlled Therapeutics, Inc. Methods for fabricating polymer-based controlled release devices
CA2272971C (en) 1998-10-29 2008-01-15 Institut National De La Sante Et De La Recherche Medicale (Inserm) Quinone derivatives for treating or preventing diseases associated with iron overload
US6245740B1 (en) 1998-12-23 2001-06-12 Amgen Inc. Polyol:oil suspensions for the sustained release of proteins
WO2005033093A1 (en) * 2003-09-19 2005-04-14 Galileo Pharmaceuticals, Inc. 7,8-bicycloalkyl-chroman derivatives
ES2619303T3 (es) * 2005-06-01 2017-06-26 Edison Pharmaceuticals, Inc. Productos terapéuticos redox activos para el tratamiento de enfermedades mitocondriales y otras afecciones y modulación de biomarcadores energéticos
SI1933821T1 (sl) 2005-09-15 2020-11-30 Ptc Therapeutics, Inc. Repne variante redoks-aktivnih terapevtikov za zdravljenje mitohondrijskih bolezni in drugih bolezni ter modulacijo energijskih biooznačevalcev
WO2007100652A2 (en) 2006-02-22 2007-09-07 Edison Pharmaceuticals, Inc. Side chain variants of redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomarkers
EP2332534B1 (en) * 2006-02-22 2018-11-14 Bioelectron Technology Corporation Phenol and benzoquinone derivatives for use in the treatment of mitochondrial diseases and modulation of energy biomarkers
WO2009111576A2 (en) 2008-03-05 2009-09-11 Edison Pharmaceuticals, Inc. 2-SUBSTITUTED-p-QUINONE DERIVATIVES FOR TREATMENT OF OXIDATIVE STRESS DISEASES
JP5798481B2 (ja) 2008-06-25 2015-10-21 エジソン ファーマシューティカルズ， インコーポレイテッド 酸化ストレス疾患を治療するための２−ヘテロシクリルアミノアルキル−（ｐ−キノン）誘導体
MX363223B (es) 2008-09-10 2019-03-15 Bioelectron Tech Corp Tratamiento de trastornos generalizados del desarrollo con terapeuticos con actividad redox.
ES2553557T3 (es) 2008-10-28 2015-12-10 Edison Pharmaceuticals, Inc. Proceso para la producción de alfa-tocotrienol y derivados
EP2246048A1 (en) 2009-04-30 2010-11-03 Santhera Pharmaceuticals (Schweiz) AG Quinone derivative 2,3-dimethoxy-5-methyl-6-(10-hydroxydecyl)-1,4-benzoquinone for the treatment of primary progressive multiple sclerosis
NO2470168T3 (https=) 2009-08-26 2018-06-30
SG187744A1 (en) 2010-08-06 2013-03-28 Edison Pharmaceuticals Inc Treatment of mitochondrial diseases with naphthoquinones
EP2606022A2 (en) * 2010-08-16 2013-06-26 Santhera Pharmaceuticals (Schweiz) AG Novel benzoquinone derivatives and use thereof as modulators of mitochondrial function
DK2605769T3 (en) 2010-08-16 2016-10-24 Santhera Pharmaceuticals (Schweiz) Ag Benzoquinonderivater for the treatment of mitochondrial diseases of the eye
US20120295985A1 (en) 2010-11-19 2012-11-22 Miller Guy M Methods for improving blood glucose control
WO2012170773A1 (en) 2011-06-08 2012-12-13 Edison Pharmaceuticals, Inc. Adjunctive therapy for the treatment of mitochondrial disorders with quinones and naphthoquinones
KR102137450B1 (ko) * 2012-07-12 2020-07-27 콘드리온 아이피 비.브이. 미토콘드리아 질환을 치료하기 위한 크로마닐 유도체
CA2883882A1 (en) 2012-09-07 2014-03-13 Ptc Therapeutics, Inc. Benzoquinone derivatives for treating oxidative stress disorders
CN104870432B (zh) 2012-11-16 2017-05-03 美国健康及人类服务部 作为溶酶体贮积症的修复剂的生育酚和生育醌衍生物
US10189830B2 (en) 2013-03-15 2019-01-29 Bioelectron Technology Corporation Alkyl-heteroaryl substituted quinone derivatives for treatment of oxidative stress disorders
WO2015183963A2 (en) 2014-05-28 2015-12-03 Stealth Peptides International, Inc. Therapeutic compositions including redox-active parabenzoquinones and uses thereof
EP3377179B1 (en) * 2015-11-19 2021-04-07 Concert Pharmaceuticals Inc. Deuterated epi-743
US10703701B2 (en) * 2015-12-17 2020-07-07 Ptc Therapeutics, Inc. Fluoroalkyl, fluoroalkoxy, phenoxy, heteroaryloxy, alkoxy, and amine 1,4-benzoquinone derivatives for treatment of oxidative stress disorders
CA3011293A1 (en) 2016-01-12 2017-07-20 Ptc Therapeutics, Inc. Tocopherol and tocotrienol quinone derivatives for increasing thiosulfate levels or decreasing hydrogen sulfide levels
WO2018191732A1 (en) * 2017-04-14 2018-10-18 Bioelectron Technology Corporation Methods and compositions for treatment of inflammation and oxidative stress
JP7266304B2 (ja) 2017-04-21 2023-04-28 ユニヴァーシティー オブ タスマニア 治療化合物および方法
ES2942631T3 (es) 2017-12-15 2023-06-05 Stealth Biotherapeutics Inc Péptidos dirigidos a mitocondrias
CN121949079A (zh) * 2019-10-04 2026-05-01 康德生物医疗有限公司 治疗线粒体异常疾病的伐替苯醌的醌、氢醌和萘醌类似物

Also Published As

Publication number Publication date
CA3156499A1 (en) 2021-04-08
US12049455B2 (en) 2024-07-30
US20210206736A1 (en) 2021-07-08
AU2020358970B2 (en) 2026-02-05
US11584728B2 (en) 2023-02-21
AU2020358970A1 (en) 2022-04-14
US20240368098A1 (en) 2024-11-07
EP4038076A1 (en) 2022-08-10
IL291898A (en) 2022-06-01
WO2021067836A1 (en) 2021-04-08
US20240400529A1 (en) 2024-12-05
JP2025164962A (ja) 2025-10-31
CN121949079A (zh) 2026-05-01
CN114746430B (zh) 2026-01-30
JP2022551270A (ja) 2022-12-08
CN114746430A (zh) 2022-07-12
US20230303506A1 (en) 2023-09-28

Similar Documents

Publication Publication Date Title
KR20220078644A (ko) 미토콘드리아 질환 치료를 위한 바티퀴논의 퀴논-, 히드로퀴논- 및 나프토퀴논-유사체
US20230303501A1 (en) C-abl tyrosine kinase inhibitory compound embodiments and methods of making and using the same
US12037350B2 (en) Compositions and methods for the prevention and/or treatment of mitochondrial disease, including Friedreich's ataxia
US20160108035A1 (en) Cycloalkyl acid derivative, preparation method thereof, and pharmaceutical application thereof
US20240293339A1 (en) Compounds, compositions and methods for the prevention and/or treatment of various mitochondrial diseases or disorders, including friedreich's ataxia
EP4735422A1 (en) Substituted cyclic compounds and methods of treating phenylketonuria and other amino acidurias
US20190177336A1 (en) THIENO[2,3-b]PYRIDINE DERIVATIVE AND QUINOLINE DERIVATIVE, AND USE THEREOF
HK40088188A (zh) 用於预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法
HK40088188B (zh) 用於预防和/或治疗包括弗里德赖希共济失调的线粒体疾病的组合物和方法
WO2018130207A1 (zh) 4,4-二苯基哌啶类化合物或其可药用盐、药物组合物及用途

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902