CN114667139B - 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂 - Google Patents

用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂 Download PDF

Info

Publication number
CN114667139B
CN114667139B CN202080070704.XA CN202080070704A CN114667139B CN 114667139 B CN114667139 B CN 114667139B CN 202080070704 A CN202080070704 A CN 202080070704A CN 114667139 B CN114667139 B CN 114667139B
Authority
CN
China
Prior art keywords
certain embodiments
alkyl
hydrogen
pmm2
compound
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.)
Active
Application number
CN202080070704.XA
Other languages
English (en)
Chinese (zh)
Other versions
CN114667139A (zh
Inventor
R·佩尔费蒂
S·申德曼
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.)
Applied Therapy Co
Original Assignee
Applied Therapy Co
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 Applied Therapy Co filed Critical Applied Therapy Co
Priority to CN202510299373.1A priority Critical patent/CN120114457A/zh
Priority to CN202510299188.2A priority patent/CN120093757A/zh
Publication of CN114667139A publication Critical patent/CN114667139A/zh
Application granted granted Critical
Publication of CN114667139B publication Critical patent/CN114667139B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/5025—Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
CN202080070704.XA 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂 Active CN114667139B (zh)

Priority Applications (2)

Application Number Priority Date Filing Date Title
CN202510299373.1A CN120114457A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
CN202510299188.2A CN120093757A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962912441P 2019-10-08 2019-10-08
US62/912,441 2019-10-08
PCT/US2020/054607 WO2021071965A1 (en) 2019-10-08 2020-10-07 Aldose reductase inhibitors for treatment of phosphomannomutase 2 deficiency

Related Child Applications (2)

Application Number Title Priority Date Filing Date
CN202510299188.2A Division CN120093757A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
CN202510299373.1A Division CN120114457A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂

Publications (2)

Publication Number Publication Date
CN114667139A CN114667139A (zh) 2022-06-24
CN114667139B true CN114667139B (zh) 2025-03-28

Family

ID=75437528

Family Applications (3)

Application Number Title Priority Date Filing Date
CN202080070704.XA Active CN114667139B (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
CN202510299373.1A Pending CN120114457A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
CN202510299188.2A Pending CN120093757A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂

Family Applications After (2)

Application Number Title Priority Date Filing Date
CN202510299373.1A Pending CN120114457A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
CN202510299188.2A Pending CN120093757A (zh) 2019-10-08 2020-10-07 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂

Country Status (10)

Country Link
US (1) US20220226323A1 (https=)
EP (1) EP4041219A4 (https=)
JP (2) JP7778686B2 (https=)
CN (3) CN114667139B (https=)
AU (1) AU2020363699A1 (https=)
BR (1) BR112022005895A2 (https=)
CA (1) CA3153108A1 (https=)
IL (1) IL291946A (https=)
MX (1) MX2022004270A (https=)
WO (1) WO2021071965A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL272246B2 (en) 2017-07-28 2026-01-01 Applied Therapeutics Inc History of 2-(4-oxo/thioketone/azo-3-((substituted)benzo[d]thiazol-2-yl)methyl)- 3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid for use as an aldose reductase inhibitor for the treatment of galactosemia or prevention of galactosemia-related complications
CN113840825A (zh) 2019-04-01 2021-12-24 应用治疗公司 醛糖还原酶抑制剂
WO2020227430A1 (en) 2019-05-07 2020-11-12 University Of Miami Treatment and detection of inherited neuropathies and associated disorders
WO2024064147A1 (en) * 2022-09-20 2024-03-28 Mayo Foundation For Medical Education And Research Treating congenital disorders of glycosylation
WO2025171282A1 (en) * 2024-02-09 2025-08-14 Landry's Smile, Llc Methods for treating congenital disorders of glycosylation

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019023648A1 (en) * 2017-07-28 2019-01-31 Applied Therapeutics Inc. COMPOSITIONS AND METHODS FOR TREATING GALACTOSEMIA
WO2020040831A1 (en) * 2018-08-20 2020-02-27 Perlara Pbc Methods for treating congenital disorders of glycosylation
WO2020205846A1 (en) * 2019-04-01 2020-10-08 Applied Therapeutics Inc. Inhibitors of aldose reductase

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007008548A2 (en) * 2005-07-07 2007-01-18 Sirtris Pharmaceuticals, Inc. Methods and related compositions for treating or preventing obesity, insulin resistance disorders, and mitochondrial-associated disorders
US20110257233A1 (en) * 2010-03-19 2011-10-20 Sanford-Burnham Medical Research Institute Benzoisothiazolones as inhibitors of phosphomannose isomerase
US8916563B2 (en) 2010-07-16 2014-12-23 The Trustees Of Columbia University In The City Of New York Aldose reductase inhibitors and uses thereof
WO2013123403A1 (en) * 2012-02-15 2013-08-22 Sanford-Burnham Medical Research Institute Theranostics platform and methods of use
DK3352754T3 (da) 2016-06-21 2020-12-07 Univ Columbia Aldosereduktaseinhibitorer og fremgangsmåder til anvendelse deraf
EP3275863A1 (en) * 2016-07-29 2018-01-31 Universidad Autónoma de Madrid Compounds for treating congenital disorders of glycosylation

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019023648A1 (en) * 2017-07-28 2019-01-31 Applied Therapeutics Inc. COMPOSITIONS AND METHODS FOR TREATING GALACTOSEMIA
WO2020040831A1 (en) * 2018-08-20 2020-02-27 Perlara Pbc Methods for treating congenital disorders of glycosylation
WO2020205846A1 (en) * 2019-04-01 2020-10-08 Applied Therapeutics Inc. Inhibitors of aldose reductase

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Maria Monticelli 等.β-Glucose-1,6-Bisphosphate Stabilizes Pathological Phophomannomutase2 Mutants In Vitro and Represents a Lead Compound to Develop Pharmacological Chaperones for the Most Common Disorder of Glycosylation, PMM2-CDG.International Journal o f Molecular Sciences.2019,第1-15页. *
Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG;Sangeetha Iyer 等;bioRxiv;第1-47页 *
Sangeetha Iyer 等.Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG.bioRxiv.2019,第1-47页. *
β-Glucose-1,6-Bisphosphate Stabilizes Pathological Phophomannomutase2 Mutants In Vitro and Represents a Lead Compound to Develop Pharmacological Chaperones for the Most Common Disorder of Glycosylation, PMM2-CDG;Maria Monticelli 等;International Journal o f Molecular Sciences;第1-15页 *

Also Published As

Publication number Publication date
US20220226323A1 (en) 2022-07-21
MX2022004270A (es) 2022-05-03
IL291946A (en) 2022-06-01
JP7778686B2 (ja) 2025-12-02
JP2022552831A (ja) 2022-12-20
WO2021071965A1 (en) 2021-04-15
JP2025100631A (ja) 2025-07-03
CN120114457A (zh) 2025-06-10
CA3153108A1 (en) 2021-04-15
BR112022005895A2 (pt) 2022-06-28
CN120093757A (zh) 2025-06-06
AU2020363699A1 (en) 2022-05-26
EP4041219A1 (en) 2022-08-17
CN114667139A (zh) 2022-06-24
EP4041219A4 (en) 2023-11-01

Similar Documents

Publication Publication Date Title
CN114667139B (zh) 用于治疗磷酸甘露变位酶2缺乏的醛糖还原酶抑制剂
US20230121312A1 (en) Aldose reductase inhibitors for treating sorbitol dehydrogenase deficiency
JP7541064B2 (ja) ガラクトース血症を処置するための組成物および方法
US9707219B2 (en) Losmapimod for use in treating glomerular disease
RU2858535C2 (ru) Ингибиторы альдозоредуктазы для лечения дефицита сорбитолдегидрогеназы
HK40113967A (en) Compositions and methods for treating galactosemia
RU2830187C2 (ru) Композиции и способы лечения галактоземии
HK40030466B (en) Compositions and methods for treating galactosemia
HK40030466A (en) Compositions and methods for treating galactosemia
BR112020001755B1 (pt) Usos de um inibidor da aldose redutase para o tratamento de galactosemia
US20120302636A1 (en) Method of treating a disorder associated with mtp

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant