AU2023205441A1 - Treatment of gm2 gangliosidosis - Google Patents

Treatment of gm2 gangliosidosis Download PDF

Info

Publication number
AU2023205441A1
AU2023205441A1 AU2023205441A AU2023205441A AU2023205441A1 AU 2023205441 A1 AU2023205441 A1 AU 2023205441A1 AU 2023205441 A AU2023205441 A AU 2023205441A AU 2023205441 A AU2023205441 A AU 2023205441A AU 2023205441 A1 AU2023205441 A1 AU 2023205441A1
Authority
AU
Australia
Prior art keywords
compound
subject
formula
disease
crystalline form
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.)
Pending
Application number
AU2023205441A
Other languages
English (en)
Inventor
Fritz Blatter
Robert Hett
Kyle LANDSKRONER
Jennifer ROBIN
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.)
Azafaros BV
Original Assignee
Azafaros BV
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
Priority claimed from EP22150783.3A external-priority patent/EP4209215A1/en
Application filed by Azafaros BV filed Critical Azafaros BV
Publication of AU2023205441A1 publication Critical patent/AU2023205441A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P25/28Drugs 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

Landscapes

  • 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)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pyridine Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2023205441A 2022-01-10 2023-01-10 Treatment of gm2 gangliosidosis Pending AU2023205441A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP22150783.3 2022-01-10
EP22150783.3A EP4209215A1 (en) 2022-01-10 2022-01-10 Treatment of gm2 gangliosidosis
EP22193136 2022-08-31
EP22193136.3 2022-08-31
PCT/EP2023/050459 WO2023131720A1 (en) 2022-01-10 2023-01-10 Treatment of gm2 gangliosidosis

Publications (1)

Publication Number Publication Date
AU2023205441A1 true AU2023205441A1 (en) 2024-07-25

Family

ID=84982296

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2023205441A Pending AU2023205441A1 (en) 2022-01-10 2023-01-10 Treatment of gm2 gangliosidosis

Country Status (9)

Country Link
US (1) US20250120958A1 (https=)
EP (2) EP4714497A2 (https=)
JP (1) JP2025501646A (https=)
AU (1) AU2023205441A1 (https=)
CA (1) CA3242701A1 (https=)
IL (1) IL314212A (https=)
MX (1) MX2024008583A (https=)
TW (1) TW202337456A (https=)
WO (1) WO2023131720A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202539666A (zh) * 2024-02-01 2025-10-16 荷蘭商雅捷法羅斯公司 Gm1神經節苷脂儲積症之治療
EP4595964A1 (en) * 2024-02-01 2025-08-06 Azafaros B.V. Treatment of gm1 gangliosidosis
WO2026013261A1 (en) 2024-07-11 2026-01-15 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders
EP4678174A1 (en) 2024-07-11 2026-01-14 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3461812B1 (en) 2014-03-27 2021-10-13 Academisch Medisch Centrum N-(5-(biphen-4-ylmethyloxy)pentyl)-substituted iminosugars as inhibitors of glucosylceramide synthase
EP3978474A1 (en) * 2020-10-02 2022-04-06 Azafaros B.V. Crystalline forms of a pharmaceutical compound

Also Published As

Publication number Publication date
WO2023131720A1 (en) 2023-07-13
TW202337456A (zh) 2023-10-01
EP4714497A2 (en) 2026-03-25
US20250120958A1 (en) 2025-04-17
JP2025501646A (ja) 2025-01-22
EP4463157A1 (en) 2024-11-20
IL314212A (en) 2024-09-01
CA3242701A1 (en) 2023-07-13
MX2024008583A (es) 2024-07-23
EP4463157C0 (en) 2026-02-25
EP4463157B1 (en) 2026-02-25

Similar Documents

Publication Publication Date Title
EP4463157B1 (en) Treatment of gm2 gangliosidosis
EP3171871B1 (en) Solid dosage form composition for buccal or sublingual administration of cannabinoids
BRPI0612921A2 (pt) composições farmacêuticas de um esteróide neuroativo e métodos de uso do mesmo
TWI694069B (zh) 治療精神分裂症之醫藥調配物
JP7809655B2 (ja) 医薬化合物の結晶形態
KR20180051561A (ko) 특정 환자 집단에서 신경퇴행성 장애를 치료하는 방법
EP4209215A1 (en) Treatment of gm2 gangliosidosis
KR20250007527A (ko) 메셈브린의 염 형태
US9717718B2 (en) Anti-inflammatory compounds
CN118632693A (zh) Gm2神经节苷脂贮积症的治疗
TW202539666A (zh) Gm1神經節苷脂儲積症之治療
EP4595964A1 (en) Treatment of gm1 gangliosidosis
JP2020511455A (ja) 医薬組成物およびその使用
CN112691102A (zh) 黄芩素在防治帕金森病/帕金森综合征抑郁症状中的应用
EP4678174A1 (en) Nizubaglustat for use in the treatment of lysosomal storage disorders
WO2026013261A1 (en) Nizubaglustat for use in the treatment of lysosomal storage disorders
JP2006290772A (ja) 多発性硬化症治療剤
JP2025542194A (ja) (r)-1-(1-アクリロイルピペリジン-3-イル)-4-アミノ-3-(4-フェノキシフェニル)-1h-イミダゾ[4,5-c]ピリジン-2(3h)-オンの製剤
CN121401316A (zh) 一种神经疾病类药物组合物及其制备方法和用途