JP2024502304A - Ｅｒ型ミスセンス変異を有する患者におけるニーマンピック病ｃ型の治療用アリモクロモル - Google Patents

Ｅｒ型ミスセンス変異を有する患者におけるニーマンピック病ｃ型の治療用アリモクロモル Download PDF

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JP2024502304A
JP2024502304A JP2023539080A JP2023539080A JP2024502304A JP 2024502304 A JP2024502304 A JP 2024502304A JP 2023539080 A JP2023539080 A JP 2023539080A JP 2023539080 A JP2023539080 A JP 2023539080A JP 2024502304 A JP2024502304 A JP 2024502304A
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キェルケゴール ジェンセン，トーマス
ダリ，クリスティーン イ
ハブンソー トープ ピーターセン，ニコライ
ブラエットラー，トーマス
クレメント フライベルク，ラルケ
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ゼブラ デンマーク エー／エス
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/444—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring heteroatom, e.g. amrinone
    • A—HUMAN NECESSITIES
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
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    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4412—Non condensed pyridines; Hydrogenated derivatives thereof having oxo groups directly attached to the heterocyclic ring
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
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    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • 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
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A61K31/4523—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545—Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers

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  • Obesity (AREA)
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  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
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JP2023539080A 2020-12-24 2021-12-23 Ｅｒ型ミスセンス変異を有する患者におけるニーマンピック病ｃ型の治療用アリモクロモル Pending JP2024502304A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063130438P 2020-12-24 2020-12-24
US63/130,438 2020-12-24
EP21155579 2021-02-05
EP21155579.2 2021-02-05
US202163185111P 2021-05-06 2021-05-06
US63/185,111 2021-05-06
PCT/EP2021/087483 WO2022136640A1 (en) 2020-12-24 2021-12-23 Arimoclomol for the treatment of niemann pick disease, type c, in patients with er type missense mutations

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JP2024502304A true JP2024502304A (ja) 2024-01-18
JP2024502304A5 JP2024502304A5 (https=) 2025-01-06

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US (1) US20240115558A1 (https=)
EP (1) EP4267139A1 (https=)
JP (1) JP2024502304A (https=)
KR (1) KR20230128037A (https=)
AU (1) AU2021405780A1 (https=)
CA (1) CA3205873A1 (https=)
IL (1) IL303968A (https=)
MX (1) MX2023007551A (https=)
WO (1) WO2022136640A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023366474A1 (en) * 2022-10-27 2025-05-15 Zevra Denmark A/S Arimoclomol compositions for use in treating niemann pick disease type c (npc)
WO2026010894A1 (en) * 2024-06-30 2026-01-08 Zevra Therapeutics, Inc. Arimoclomol in combination with miglustat for use in the treatment of lysosomal storage disorders
US20260000661A1 (en) * 2024-07-01 2026-01-01 Zevra Therapeutics, Inc. Use of arimoclomol in activating clear gene expression as treatment for lysosomal storage disorders
WO2026010893A1 (en) * 2024-07-01 2026-01-08 Zevra Therapeutics, Inc. Arimoclomol in combination with miglustat for use in the treatment of lysosomal storage disorders such as niemann-pick disease, type-c

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013544263A (ja) * 2010-11-30 2013-12-12 オルファザイム エーピーエス Ｈｓｐ７０の細胞内活性を上昇させる方法

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HU222994B1 (hu) 1995-11-02 2004-01-28 BIOREX Kutató és Fejlesztő Rt. Hidroxilaminszármazékok és azok alkalmazása sejtek molekuláris chaperon-termelésének fokozására alkalmas gyógyszerkészítmények előállítására
HUP9900475D0 (en) 1999-02-26 1999-04-28 Biorex Kutato Fejlesztoe Kft O-(3-piperidino-2-hydroxy-1-propyl)-hiyroximic acid-halogenid derivative, it's use for treating insulin resistance, and pharmaceutical compositions containing them as active component
HUP0001583A2 (hu) 2000-04-18 2002-11-28 BIOREX Kutató és Fejlesztő Rt. Egy piridin-1-oxid-származék és eljárás annak átalakítására gyógyászati hatású vegyületekké

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013544263A (ja) * 2010-11-30 2013-12-12 オルファザイム エーピーエス Ｈｓｐ７０の細胞内活性を上昇させる方法

Non-Patent Citations (11)

* Cited by examiner, † Cited by third party
Title
"Orphazyme reports positive results from full data set of Phase II/III arimoclomol trial in Niemann-P", COMPANY ANNOUNCEMENT, JPN6025049929, 2019, pages 1 - 4, ISSN: 0005749219 *
ATKINSON, B. N. ET AL.: "Regioselective and enantiospecifc synthesis of the HSP co-inducer arimoclomol from chiral glycidyl d", ORG. BIOMOL. CHEM., vol. 15, JPN6025049935, 2017, pages 9794 - 9799, ISSN: 0005749225 *
CUISSET, JEAN-MARIE ET AL.: "Impact of miglustat on evolution of atypical presentation of late-infantile-onset Niemann-Pick disea", JOURNAL OF MEDICAL CASE REPORTS, vol. 10, no. 1, JPN6025049934, 2016, pages 1 - 6, ISSN: 0005749224 *
EUROPEAN MEDICINES AGENCY DECISION P/0213/2018, JPN6025049927, 2018, pages 1 - 8, ISSN: 0005749217 *
INGEMANN, LINDA: "Rescue of NPC1 protein and effect on biomarkers by arimoclomol treatment in Niemann-Pick disease typ", MOLECULAR GENETICS AND METABOLISM, vol. 129, no. 2, JPN6025049925, 2019, pages 78, ISSN: 0005749215 *
KIRKEGAARD, T. ET AL.: "Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses", SCIENCE TRANSLATIONAL MEDICINE, vol. 8, no. 355, JPN6025049926, 2016, pages 1 - 16, ISSN: 0005749216 *
MILLAT, G. ET AL.: "Niemann-Pick C1 disease: correlations between NPC1 mutations, levels of NPC1 protein, and phenotypes", AM. J. HUM. GENET., vol. 68, no. 6, JPN6025049933, 2001, pages 1373 - 85, ISSN: 0005749223 *
PATTERSON, MARC: "Efficacy and safety of arimoclomol in patients with Niemann-Pick disease type C: Results from a doub", MOLECULAR GENETICS AND METABOLISM, vol. 129, no. 2, JPN6025049928, 2019, pages 127, ISSN: 0005749218 *
SCHULTZ, M. L. ET AL.: "Coordinate regulation of mutant NPC1 degradation by selective ER autophagy and MARCH6-dependent ERAD", NATURE COMMUNICATION, vol. 9, no. 1, JPN6025049930, 2018, pages 1 - 13, ISSN: 0005749220 *
SHAMMAS, H. ET AL.: "Different Niemann-Pick C1 Genotypes Generate Protein Phenotypes that Vary in their Intracellular Pro", SCIENTIFIC REPORTS, vol. 9, JPN6025049932, 2019, pages 1 - 12, ISSN: 0005749222 *
WANG, C. ET AL.: "Individualized management of genetic diversity in Niemann-Pick C1 through modulation of the Hsp70 ch", HUMAN MOLECULAR GENETICS, vol. 29, no. 1, JPN6025049931, 2019, pages 1 - 19, ISSN: 0005749221 *

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KR20230128037A (ko) 2023-09-01
IL303968A (en) 2023-08-01
WO2022136640A1 (en) 2022-06-30
US20240115558A1 (en) 2024-04-11
CA3205873A1 (en) 2022-06-30
MX2023007551A (es) 2023-09-04
EP4267139A1 (en) 2023-11-01
AU2021405780A1 (en) 2023-07-06

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