AU2020336984B2 - Methods for treating CLN2 disease in pediatric subjects - Google Patents

Methods for treating CLN2 disease in pediatric subjects

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Publication number
AU2020336984B2
AU2020336984B2 AU2020336984A AU2020336984A AU2020336984B2 AU 2020336984 B2 AU2020336984 B2 AU 2020336984B2 AU 2020336984 A AU2020336984 A AU 2020336984A AU 2020336984 A AU2020336984 A AU 2020336984A AU 2020336984 B2 AU2020336984 B2 AU 2020336984B2
Authority
AU
Australia
Prior art keywords
subject
rhtpp1
cln2
patients
study
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
AU2020336984A
Other languages
English (en)
Other versions
AU2020336984A1 (en
Inventor
Joshua Henshaw
David JACOBY
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.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical Inc
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 Biomarin Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Publication of AU2020336984A1 publication Critical patent/AU2020336984A1/en
Application granted granted Critical
Publication of AU2020336984B2 publication Critical patent/AU2020336984B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts or implants
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • 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
    • A61K9/08—Solutions
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Inorganic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2020336984A 2019-08-29 2020-08-31 Methods for treating CLN2 disease in pediatric subjects Active AU2020336984B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962893535P 2019-08-29 2019-08-29
US62/893,535 2019-08-29
PCT/US2020/048704 WO2021042020A1 (en) 2019-08-29 2020-08-31 Methods for treating cln2 disease in pediatric subjects

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2026202611 Division 2020-08-31

Publications (2)

Publication Number Publication Date
AU2020336984A1 AU2020336984A1 (en) 2022-03-10
AU2020336984B2 true AU2020336984B2 (en) 2026-04-23

Family

ID=72470612

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020336984A Active AU2020336984B2 (en) 2019-08-29 2020-08-31 Methods for treating CLN2 disease in pediatric subjects

Country Status (25)

Country Link
US (1) US20220273775A1 (https=)
EP (2) EP4021575B1 (https=)
JP (2) JP2022546042A (https=)
KR (1) KR20220054375A (https=)
CN (1) CN114828960A (https=)
AR (1) AR119871A1 (https=)
AU (1) AU2020336984B2 (https=)
BR (1) BR112022003598A2 (https=)
CA (1) CA3152521A1 (https=)
CL (1) CL2022000496A1 (https=)
DK (1) DK4021575T3 (https=)
ES (1) ES2987156T3 (https=)
FI (1) FI4021575T3 (https=)
HR (2) HRP20240871T1 (https=)
HU (1) HUE067135T2 (https=)
IL (1) IL290763A (https=)
LT (1) LT4021575T (https=)
MX (1) MX2022002451A (https=)
PL (1) PL4021575T3 (https=)
PT (1) PT4021575T (https=)
RS (1) RS65763B1 (https=)
SI (1) SI4021575T1 (https=)
SM (1) SMT202400315T1 (https=)
TW (1) TW202122106A (https=)
WO (1) WO2021042020A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220323019A1 (en) * 2021-04-02 2022-10-13 Neuropace, Inc. Systems and methods for obtaining a clinical response estimate biomarker using machine-learned models trained on implanted neurostimulator data

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190216905A1 (en) * 2015-05-08 2019-07-18 Biomarin Pharmaceutical Inc. TPP-1 Formulations and Methods for Treating CLN2 Disease

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6302685B1 (en) 1997-09-16 2001-10-16 University Of Medicine And Dentistry Of New Jersey Human lysosomal protein and methods of its use
US20020006400A1 (en) * 2000-05-11 2002-01-17 Peter Lobel Recombinant human CLN2 protein and methods of its production and use
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
LT2588130T (lt) * 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR102738860B1 (ko) * 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
BR112021009370A2 (pt) * 2018-11-14 2021-08-17 Regenxbio Inc. método de tratamento da doença de batten cln2, composição farmacêutica e kit

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190216905A1 (en) * 2015-05-08 2019-07-18 Biomarin Pharmaceutical Inc. TPP-1 Formulations and Methods for Treating CLN2 Disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ALFRIED KOHLSCHÜTTER ET AL: "Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses", CNS DRUGS, 2019, vol. 33, no. 4, pgs. 315 - 325, DOI: 10.1007/s40263-019-00620-8 *
BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura (cerliponase alfa) for 3 Years Reduced Rate of Decline Maintained in Children with CLN2 Disease, a Form of Batten Disease *

Also Published As

Publication number Publication date
WO2021042020A1 (en) 2021-03-04
RS65763B1 (sr) 2024-08-30
SI4021575T1 (sl) 2024-10-30
PT4021575T (pt) 2024-07-30
PL4021575T3 (pl) 2024-09-23
EP4438120A3 (en) 2024-12-11
BR112022003598A2 (pt) 2022-05-24
EP4021575B1 (en) 2024-05-22
HRP20220370A1 (hr) 2022-09-16
HRP20240871T1 (hr) 2024-10-11
TW202122106A (zh) 2021-06-16
ES2987156T3 (es) 2024-11-14
EP4438120A2 (en) 2024-10-02
LT4021575T (lt) 2024-08-12
IL290763A (en) 2022-04-01
AU2020336984A1 (en) 2022-03-10
AR119871A1 (es) 2022-01-19
CL2022000496A1 (es) 2022-10-21
MX2022002451A (es) 2022-06-02
JP2022546042A (ja) 2022-11-02
CA3152521A1 (en) 2021-03-04
FI4021575T3 (fi) 2024-07-24
HUE067135T2 (hu) 2024-10-28
SMT202400315T1 (it) 2024-09-16
DK4021575T3 (da) 2024-08-05
CN114828960A (zh) 2022-07-29
EP4021575A1 (en) 2022-07-06
KR20220054375A (ko) 2022-05-02
JP2025165414A (ja) 2025-11-04
US20220273775A1 (en) 2022-09-01

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