AU2017308865B2 - Therapeutic agents for neurodegenerative diseases - Google Patents

Therapeutic agents for neurodegenerative diseases Download PDF

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Publication number
AU2017308865B2
AU2017308865B2 AU2017308865A AU2017308865A AU2017308865B2 AU 2017308865 B2 AU2017308865 B2 AU 2017308865B2 AU 2017308865 A AU2017308865 A AU 2017308865A AU 2017308865 A AU2017308865 A AU 2017308865A AU 2017308865 B2 AU2017308865 B2 AU 2017308865B2
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Prior art keywords
disease
leucine
acetyl
neurodegenerative disease
patient
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AU2017308865A
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AU2017308865A1 (en
Inventor
Michael Strupp
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Intrabio Ltd
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Intrabio Ltd
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Priority claimed from GBGB1613829.9A external-priority patent/GB201613829D0/en
Priority claimed from GBGB1702551.1A external-priority patent/GB201702551D0/en
Priority claimed from GBGB1705766.2A external-priority patent/GB201705766D0/en
Priority claimed from GBGB1706867.7A external-priority patent/GB201706867D0/en
Application filed by Intrabio Ltd filed Critical Intrabio Ltd
Publication of AU2017308865A1 publication Critical patent/AU2017308865A1/en
Priority to AU2023202121A priority Critical patent/AU2023202121B2/en
Publication of AU2017308865B2 publication Critical patent/AU2017308865B2/en
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Priority to AU2025202077A priority patent/AU2025202077A1/en
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    • 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
    • A61K31/13—Amines
    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/20—Hypnotics; Sedatives
    • 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
    • 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
    • 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
    • A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
    • A61P25/32—Alcohol-abuse
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/45—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups
    • C07C233/46—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pain & Pain Management (AREA)
  • Obesity (AREA)
  • Addiction (AREA)
  • Transplantation (AREA)
  • Anesthesiology (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2017308865A 2016-08-11 2017-08-11 Therapeutic agents for neurodegenerative diseases Active AU2017308865B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2023202121A AU2023202121B2 (en) 2016-08-11 2023-04-05 Therapeutic agents for neurodegenerative diseases
AU2025202077A AU2025202077A1 (en) 2016-08-11 2025-03-21 Therapeutic agents for neurodegenerative diseases

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
GBGB1613829.9A GB201613829D0 (en) 2016-08-11 2016-08-11 Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage
GB1613829.9 2016-08-11
GB1702551.1 2017-02-16
GBGB1702551.1A GB201702551D0 (en) 2017-02-16 2017-02-16 Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage
GBGB1705766.2A GB201705766D0 (en) 2017-04-10 2017-04-10 Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage
GB1705766.2 2017-04-10
GB1706867.7 2017-04-28
GBGB1706867.7A GB201706867D0 (en) 2017-04-28 2017-04-28 Therapeutic agents for neurodegenerative diseases, lysosomal storage disorders and neurodegenerative diseases associated with defects in lysosomal storage
PCT/IB2017/054929 WO2018029658A1 (en) 2016-08-11 2017-08-11 Therapeutic agents for neurodegenerative diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023202121A Division AU2023202121B2 (en) 2016-08-11 2023-04-05 Therapeutic agents for neurodegenerative diseases

Publications (2)

Publication Number Publication Date
AU2017308865A1 AU2017308865A1 (en) 2019-02-21
AU2017308865B2 true AU2017308865B2 (en) 2023-04-13

Family

ID=59859424

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AU2017308865A Active AU2017308865B2 (en) 2016-08-11 2017-08-11 Therapeutic agents for neurodegenerative diseases
AU2023202121A Active AU2023202121B2 (en) 2016-08-11 2023-04-05 Therapeutic agents for neurodegenerative diseases
AU2025202077A Pending AU2025202077A1 (en) 2016-08-11 2025-03-21 Therapeutic agents for neurodegenerative diseases

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Application Number Title Priority Date Filing Date
AU2023202121A Active AU2023202121B2 (en) 2016-08-11 2023-04-05 Therapeutic agents for neurodegenerative diseases
AU2025202077A Pending AU2025202077A1 (en) 2016-08-11 2025-03-21 Therapeutic agents for neurodegenerative diseases

Country Status (28)

Country Link
US (3) US12144792B2 (enExample)
EP (3) EP3416631B1 (enExample)
JP (3) JP6957602B2 (enExample)
KR (4) KR20220093385A (enExample)
CN (4) CN121081445A (enExample)
AU (3) AU2017308865B2 (enExample)
BR (1) BR112019002730A2 (enExample)
CA (1) CA3033564A1 (enExample)
CY (1) CY1121930T1 (enExample)
DK (1) DK3416631T3 (enExample)
ES (1) ES2733677T3 (enExample)
HR (1) HRP20191055T1 (enExample)
HU (1) HUE045043T2 (enExample)
IL (3) IL264641B2 (enExample)
LT (1) LT3416631T (enExample)
MA (2) MA47521A (enExample)
MD (1) MD3416631T2 (enExample)
ME (1) ME03454B (enExample)
MX (3) MX383499B (enExample)
PL (1) PL3416631T3 (enExample)
PT (1) PT3416631T (enExample)
RS (1) RS59048B1 (enExample)
RU (1) RU2756519C2 (enExample)
SG (1) SG11201901063SA (enExample)
SI (1) SI3416631T1 (enExample)
SM (1) SMT201900370T1 (enExample)
TN (1) TN2019000033A1 (enExample)
WO (1) WO2018029658A1 (enExample)

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US10219815B2 (en) 2005-09-22 2019-03-05 The Regents Of The University Of Michigan Histotripsy for thrombolysis
US11135454B2 (en) 2015-06-24 2021-10-05 The Regents Of The University Of Michigan Histotripsy therapy systems and methods for the treatment of brain tissue
LT3416631T (lt) 2016-08-11 2019-08-26 Intrabio Ltd Terapiniai agentai, skirti neurodegeneracinių ligų gydymui
TN2019000032A1 (en) 2016-08-11 2020-07-15 Intrabio Ltd Pharmaceutical compositions and uses directed to lysosomal storage disorders
GB201709459D0 (en) 2017-06-14 2017-07-26 Intrabio Ltd Treatment for migraine
CN111542314B (zh) * 2017-10-18 2023-11-24 内在生物技术有限公司 神经退行性疾病的治疗剂
ES2983441T3 (es) 2018-02-15 2024-10-23 Intrabio Ltd Acetil-leucina para su uso en el tratamiento de síndrome de las piernas inquietas
AU2019389001B2 (en) 2018-11-28 2025-08-14 Histosonics, Inc. Histotripsy systems and methods
CN113348018B (zh) * 2018-12-06 2025-04-01 内在生物技术有限公司 乙酰基-亮氨酸的氘代类似物
IL321899A (en) * 2019-03-02 2025-09-01 Intrabio Ltd Leucine, acetyl leucine and related analogs for the treatment of disease
WO2021055443A1 (en) * 2019-09-17 2021-03-25 Hoffmann-La Roche Inc. Improvements in personalized healthcare for patients with movement disorders
WO2021144720A1 (en) * 2020-01-13 2021-07-22 Intrabio Ltd Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers
WO2022264037A1 (en) * 2021-06-14 2022-12-22 Intrabio Ltd. Branched-chain amino acid derivatives to treat disease
AU2023366591A1 (en) 2022-10-28 2025-04-24 Histosonics, Inc. Histotripsy systems and methods
WO2024221001A2 (en) 2023-04-20 2024-10-24 Histosonics, Inc. Histotripsy systems and associated methods including user interfaces and workflows for treatment planning and therapy
WO2025151578A1 (en) * 2024-01-12 2025-07-17 Intrabio Inc. N-acetyl-leucine for use in the treatment of prodromal alpha-synucleinopathies
WO2025163129A1 (en) * 2024-02-01 2025-08-07 Intrabio Ltd. Acetyl-leucine for treating parkinson´s disease
US12403111B2 (en) 2024-02-02 2025-09-02 SynGAP Research Fund Compositions and methods for treating neurodevelopmental disorders

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WO2011151685A1 (en) * 2010-06-03 2011-12-08 Raouf Rekik N-acetyl-dl-leucine, neuroprotective and retinoprotective medicament

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MX378668B (es) * 2013-10-28 2025-03-10 Naurex Inc Profarmacos y moduladores de receptores nmda, sales y usos de estos.
EP4501406A3 (en) 2016-04-19 2025-03-26 IntraBio Ltd Acetyl-leucine or a pharmaceutically acceptable salt thereof for improved mobility and cognitive function
LT3416631T (lt) 2016-08-11 2019-08-26 Intrabio Ltd Terapiniai agentai, skirti neurodegeneracinių ligų gydymui

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WO2011151685A1 (en) * 2010-06-03 2011-12-08 Raouf Rekik N-acetyl-dl-leucine, neuroprotective and retinoprotective medicament

Non-Patent Citations (2)

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Title
ROMAN SCHNIEPP, STRUPP MICHAEL, WUEHR MAX, JAHN KLAUS, DIETERICH MARIANNE, BRANDT THOMAS, FEIL KATHARINA: "Acetyl-DL-leucine improves gait variability in patients with cerebellar ataxia—a case series", CEREBELLUM & ATAXIAS, vol. 3, 1 December 2016 (2016-12-01), pages 8, XP055390569, DOI: 10.1186/s40673-016-0046-2 *
TATIANA BREMOVA, MALINOVÁ VĚRA, AMRAOUI YASMINA, MENGEL EUGEN, REINKE JÖRG, KOLNÍKOVÁ MIRIAM, STRUPP MICHAEL: "Acetyl-DL-leucine in Niemann-Pick type C: A case series", NEUROLOGY, LIPPINCOTT WILLIAMS & WILKINS , PHILADELPHIA, US, vol. 85, no. 16, 20 October 2015 (2015-10-20), US , pages 1368 - 1375, XP055390576, ISSN: 0028-3878, DOI: 10.1212/WNL.0000000000002041 *

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Publication number Publication date
AU2025202077A1 (en) 2025-04-10
CY1121930T1 (el) 2020-10-14
CN121081445A (zh) 2025-12-09
IL264641B2 (en) 2024-07-01
MA47521A (fr) 2021-04-21
PT3416631T (pt) 2019-07-11
US12144792B2 (en) 2024-11-19
IL310799A (en) 2024-04-01
RU2019106506A3 (enExample) 2020-10-29
EP3583940A1 (en) 2019-12-25
CN109789114A (zh) 2019-05-21
JP6957602B2 (ja) 2021-11-02
MD3416631T2 (ro) 2019-09-30
US20200179320A1 (en) 2020-06-11
HRP20191055T1 (hr) 2019-09-06
KR102413756B1 (ko) 2022-06-27
SMT201900370T1 (it) 2019-09-09
EP3416631A1 (en) 2018-12-26
MA43828B1 (fr) 2019-09-30
CN116459244A (zh) 2023-07-21
SG11201901063SA (en) 2019-03-28
RU2756519C2 (ru) 2021-10-01
HUE045043T2 (hu) 2019-12-30
WO2018029658A1 (en) 2018-02-15
AU2017308865A1 (en) 2019-02-21
JP2022003085A (ja) 2022-01-11
IL264641A (en) 2019-04-30
EP4467195A2 (en) 2024-11-27
MX2024004439A (es) 2025-11-03
MX383499B (es) 2025-03-14
DK3416631T3 (da) 2019-07-15
MX2021006901A (es) 2021-07-07
CN116492328A (zh) 2023-07-28
MA43828A (fr) 2018-12-26
CA3033564A1 (en) 2018-02-15
JP2023175762A (ja) 2023-12-12
MX2019001575A (es) 2019-08-01
AU2023202121B2 (en) 2025-01-23
IL310801A (en) 2024-04-01
KR102785448B1 (ko) 2025-03-21
SI3416631T1 (sl) 2019-09-30
US20240189267A1 (en) 2024-06-13
EP3416631B1 (en) 2019-05-15
ES2733677T3 (es) 2019-12-02
KR20240068761A (ko) 2024-05-17
KR20190039227A (ko) 2019-04-10
IL264641B1 (en) 2024-03-01
LT3416631T (lt) 2019-08-26
RU2021128045A (ru) 2021-11-16
EP4467195A3 (en) 2025-03-19
BR112019002730A2 (pt) 2019-05-14
KR20220093385A (ko) 2022-07-05
JP2019524822A (ja) 2019-09-05
AU2023202121A1 (en) 2023-05-04
KR20250042197A (ko) 2025-03-26
RS59048B1 (sr) 2019-08-30
RU2019106506A (ru) 2020-09-17
TN2019000033A1 (fr) 2020-07-15
ME03454B (me) 2020-01-20
US20230210799A1 (en) 2023-07-06
PL3416631T3 (pl) 2019-11-29

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