CA2656643C - Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase - Google Patents

Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase Download PDF

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Publication number
CA2656643C
CA2656643C CA2656643A CA2656643A CA2656643C CA 2656643 C CA2656643 C CA 2656643C CA 2656643 A CA2656643 A CA 2656643A CA 2656643 A CA2656643 A CA 2656643A CA 2656643 C CA2656643 C CA 2656643C
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Prior art keywords
isofagomine
substituted
activity
unsubstituted
gcase
Prior art date
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Expired - Fee Related
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CA2656643A
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English (en)
French (fr)
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CA2656643A1 (en
Inventor
Brandon Wustman
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Amicus Therapeutics Inc
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Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CA2656643A1 publication Critical patent/CA2656643A1/en
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/194Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic 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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/45Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A90/00Technologies having an indirect contribution to adaptation to climate change
    • Y02A90/10Information and communication technologies [ICT] supporting adaptation to climate change, e.g. for weather forecasting or climate simulation

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
CA2656643A 2006-06-23 2007-06-25 Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase Expired - Fee Related CA2656643C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US81595206P 2006-06-23 2006-06-23
US60/815,952 2006-06-23
PCT/US2007/072016 WO2007150064A2 (en) 2006-06-23 2007-06-25 METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE

Publications (2)

Publication Number Publication Date
CA2656643A1 CA2656643A1 (en) 2007-12-27
CA2656643C true CA2656643C (en) 2015-08-25

Family

ID=38834440

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2656643A Expired - Fee Related CA2656643C (en) 2006-06-23 2007-06-25 Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase

Country Status (15)

Country Link
US (4) US7829579B2 (https=)
EP (1) EP2040548B1 (https=)
JP (1) JP5303458B2 (https=)
KR (1) KR101402554B1 (https=)
AT (1) ATE555788T1 (https=)
AU (1) AU2007260812B2 (https=)
BR (1) BRPI0713442A2 (https=)
CA (1) CA2656643C (https=)
DK (1) DK2040548T3 (https=)
ES (1) ES2387845T3 (https=)
IL (1) IL196151A (https=)
MX (1) MX2009000032A (https=)
PT (1) PT2040548E (https=)
WO (1) WO2007150064A2 (https=)
ZA (1) ZA200900270B (https=)

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GB0622702D0 (en) * 2006-11-15 2006-12-27 Univ Dundee Selective glycosidase inhibitors
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
AU2009262670B2 (en) * 2008-06-26 2013-06-20 Zevra Denmark A/S Use of Hsp70 as a regulator of enzymatic activity
WO2010056413A2 (en) * 2008-11-14 2010-05-20 Parkinson's Institute COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALTERED α-SYNUCLEIN FUNCTION
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
US8940766B2 (en) * 2009-04-09 2015-01-27 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
AU2010281403B2 (en) * 2009-07-28 2016-03-10 Takeda Pharmaceutical Company Limited Compositions and methods for treating Gaucher disease
WO2011049737A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
SI2490532T1 (sl) * 2009-10-19 2017-03-31 Amicus Therapeutics, Inc. Novi sestavki za preventivo in/ali zdravljenje degenerativnih motenj centralnega živčnega sistema
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
CA2817773A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
DK2707101T3 (da) 2011-05-12 2019-05-13 Proteostasis Therapeutics Inc Proteostaseregulatorer
EP2533051A1 (en) 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
WO2012177997A1 (en) * 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
CA2861464C (en) 2012-01-25 2018-03-06 Proteostasis Therapeutics, Inc. Proteasome activity modulating tricyclic compounds
JP2016503405A (ja) * 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
WO2014089449A1 (en) 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2015095963A1 (en) 2013-12-23 2015-07-02 Alectos Therapeutics Inc. Glucocerebrosidase modulators and uses thereof
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
HRP20240644T1 (hr) 2014-11-06 2024-08-02 Bial-R&D Investments, S.A. Supstituirani pirazolo(1,5-a)pirimidini i njihova uporaba u liječenju medicinskih poremećaja
EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
CA3020305A1 (en) 2016-04-06 2017-10-12 Lysosomal Therapeutics Inc. Imidazo [1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders
US11192892B2 (en) 2016-04-06 2021-12-07 Bial—R&D Investments, S.A. Substituted pyrazolo[1,5-a]pyrimidines for the treatment of medical disorders
EP3440081A4 (en) 2016-04-06 2019-09-18 Lysosomal Therapeutics Inc. PYRROLO [1,2-A] PYRIMIDINYL-CARBOXAMIDE COMPOUNDS AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDER
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
DK3782624T3 (da) 2016-04-29 2025-12-15 Zevra Denmark As Arimoclomol til behandling af glucocerebrosidase-associerede lidelser
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CN108428452B (zh) 2018-03-14 2019-12-13 百度在线网络技术(北京)有限公司 终端支架和远场语音交互系统
CN112585130A (zh) 2018-06-27 2021-03-30 蛋白质平衡治疗股份有限公司 蛋白酶体活性增强化合物
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JP7642561B2 (ja) * 2019-04-25 2025-03-10 武田薬品工業株式会社 イソファゴミン塩、その使用方法および製剤
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EP1682504B1 (en) 2003-11-12 2012-12-26 Amicus Therapeutics Inc. Hydroxy piperidine derivatives to treat gaucher disease
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JP5303458B2 (ja) 2006-06-23 2013-10-02 アミークス セラピューティックス インコーポレイテッド β−グルコセレブロシダーゼの活性増強による神経学的疾患の治療方法
KR101650726B1 (ko) * 2007-05-22 2016-08-24 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법

Also Published As

Publication number Publication date
US7829579B2 (en) 2010-11-09
DK2040548T3 (da) 2012-08-13
US9119845B2 (en) 2015-09-01
IL196151A (en) 2015-08-31
AU2007260812B2 (en) 2012-08-16
ES2387845T3 (es) 2012-10-02
US20150025109A1 (en) 2015-01-22
EP2040548A4 (en) 2009-12-23
EP2040548B1 (en) 2012-05-02
US20080009516A1 (en) 2008-01-10
KR101402554B1 (ko) 2014-06-19
US10064851B2 (en) 2018-09-04
IL196151A0 (en) 2009-11-18
CA2656643A1 (en) 2007-12-27
EP2040548A2 (en) 2009-04-01
JP5303458B2 (ja) 2013-10-02
ATE555788T1 (de) 2012-05-15
BRPI0713442A2 (pt) 2012-03-06
US20170027919A1 (en) 2017-02-02
US20110052613A1 (en) 2011-03-03
WO2007150064A3 (en) 2008-02-21
AU2007260812A1 (en) 2007-12-27
ZA200900270B (en) 2011-02-23
US9402837B2 (en) 2016-08-02
WO2007150064A2 (en) 2007-12-27
JP2009541489A (ja) 2009-11-26
MX2009000032A (es) 2009-01-23
PT2040548E (pt) 2012-08-02
KR20090021393A (ko) 2009-03-03

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