AU2020376801A1 - Methods and compositions for treatment of Rett syndrome - Google Patents

Methods and compositions for treatment of Rett syndrome Download PDF

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Publication number
AU2020376801A1
AU2020376801A1 AU2020376801A AU2020376801A AU2020376801A1 AU 2020376801 A1 AU2020376801 A1 AU 2020376801A1 AU 2020376801 A AU2020376801 A AU 2020376801A AU 2020376801 A AU2020376801 A AU 2020376801A AU 2020376801 A1 AU2020376801 A1 AU 2020376801A1
Authority
AU
Australia
Prior art keywords
subject
trofinetide
administered
rett syndrome
daily amount
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
AU2020376801A
Other languages
English (en)
Inventor
Mona Darwish
Lawrence Irwin Glass
Nancy Elizabeth Jones
Sean Paul Oosterholt
Oscar Della Pasqua
James M. Youakim
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.)
Neuren Pharmaceuticals Ltd New Zealand
Acadia Pharmaceuticals Inc
Original Assignee
NEUREN PHARMACEUTICALS Ltd
Acadia Pharmaceuticals 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 NEUREN PHARMACEUTICALS Ltd, Acadia Pharmaceuticals Inc filed Critical NEUREN PHARMACEUTICALS Ltd
Publication of AU2020376801A1 publication Critical patent/AU2020376801A1/en
Pending legal-status Critical Current

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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
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/06—Tripeptides
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/401—Proline; Derivatives thereof, e.g. captopril
    • 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
    • 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
    • A61P25/28—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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/08—Tripeptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Detergent Compositions (AREA)
  • Chemical Or Physical Treatment Of Fibers (AREA)
  • Peptides Or Proteins (AREA)
AU2020376801A 2019-10-28 2020-10-28 Methods and compositions for treatment of Rett syndrome Pending AU2020376801A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962927008P 2019-10-28 2019-10-28
US62/927,008 2019-10-28
US202063031201P 2020-05-28 2020-05-28
US63/031,201 2020-05-28
PCT/US2020/057627 WO2021086892A1 (en) 2019-10-28 2020-10-28 Methods and compositions for treatment of rett syndrome

Publications (1)

Publication Number Publication Date
AU2020376801A1 true AU2020376801A1 (en) 2022-06-09

Family

ID=75715568

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020376801A Pending AU2020376801A1 (en) 2019-10-28 2020-10-28 Methods and compositions for treatment of Rett syndrome

Country Status (14)

Country Link
US (1) US20220339138A1 (https=)
EP (1) EP4051308A4 (https=)
JP (2) JP2022553888A (https=)
KR (1) KR20220106982A (https=)
CN (1) CN115335071A (https=)
AU (1) AU2020376801A1 (https=)
BR (1) BR112022008095A2 (https=)
CA (1) CA3156680A1 (https=)
CL (1) CL2022001079A1 (https=)
CO (1) CO2022007501A2 (https=)
IL (1) IL292617B2 (https=)
MX (1) MX2022004785A (https=)
TW (1) TW202116300A (https=)
WO (1) WO2021086892A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL310045A (en) 2021-07-12 2024-03-01 Acadia Pharm Inc Crystalline forms of tropintide
WO2023242844A1 (en) * 2022-06-15 2023-12-21 Ramot At Tel-Aviv University Ltd. Method and therapeutic agent for treatment of disease or disorder associated with impaired firing rate and/or mitochondrial calcium homeostasis
WO2025262646A1 (en) * 2024-06-22 2025-12-26 Biophore India Pharmaceuticals Pvt. Ltd Dosage forms of trofinetide and its pharmaceutically acceptable salt thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6709817B1 (en) * 1999-09-07 2004-03-23 Baylor College Of Medicine Method of screening Rett syndrome by detecting a mutation in MECP2
US9708366B2 (en) * 2011-01-27 2017-07-18 Neuren Pharmaceuticals Ltd. Treatment of fragile X syndrome using glycyl-L-2-methylprolyl-L-glutamate
WO2014130691A2 (en) * 2013-02-20 2014-08-28 Theravasc Inc. Pharmaceutical formulations of nitrite and uses thereof
WO2015013397A2 (en) * 2013-07-25 2015-01-29 Neuren Pharmaceuticals Limited Neuroprotective bicyclic compounds and methods for their use in treating autism spectrum disorders and neurodevelopmental disorders
US20150224164A1 (en) * 2013-11-26 2015-08-13 Neuren Pharmaceuticals Limited Treatment of autism spectrum disorders using glycyl-l-2-methylprolyl-l-glumatic acid
GB2554064B (en) * 2016-09-01 2019-11-20 Jit Singh Shailinder An enteral feeding tube

Also Published As

Publication number Publication date
CN115335071A (zh) 2022-11-11
TW202116300A (zh) 2021-05-01
CA3156680A1 (en) 2021-05-06
CL2022001079A1 (es) 2023-04-21
EP4051308A4 (en) 2023-08-23
MX2022004785A (es) 2022-05-16
CO2022007501A2 (es) 2022-08-30
KR20220106982A (ko) 2022-08-01
US20220339138A1 (en) 2022-10-27
IL292617B1 (en) 2025-11-01
IL292617B2 (en) 2026-03-01
EP4051308A1 (en) 2022-09-07
WO2021086892A1 (en) 2021-05-06
BR112022008095A2 (pt) 2022-07-12
IL292617A (en) 2022-07-01
JP2025148461A (ja) 2025-10-07
JP2022553888A (ja) 2022-12-26

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