Last update 08 May 2025

GHF-201

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide
Target
LAMP-1
Action
inhibitors
Mechanism
LAMP-1 inhibitors(Lysosome-associated membrane glycoprotein 1 inhibitors)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Polyglucosan Body Disease, Adult FormGlycogen Storage Disease Type IV
Inactive Indication-
Originator Organization
Golden Heart Flower Ltd.
Active Organization
Golden Heart Flower Ltd.
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (European Union)

Structure/Sequence

Molecular FormulaC16H20N2O3S
InChIKeyBBEZNLRXWCUHAJ-UHFFFAOYSA-N
CAS Registry1257155-76-2

Related

1
Clinical Trials associated with GHF-201
CTIS2024-519813-74-00
/ Not yet recruitingPhase 1
- CGH24001
100 Clinical Results associated with GHF-201
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100 Translational Medicine associated with GHF-201
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100 Patents (Medical) associated with GHF-201
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1
Literatures (Medical) associated with GHF-201
Biomolecules
The Autophagic Activator GHF-201 Can Alleviate Pathology in a Mouse Model and in Patient Fibroblasts of Type III Glycogenosis
100 Deals associated with GHF-201
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Polyglucosan Body Disease, Adult FormPreclinical
Israel
Golden Heart Flower Ltd.
14 Aug 2023
Glycogen Storage Disease Type IVPreclinical
United States
Golden Heart Flower Ltd.
09 Aug 2023
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Clinical Result

Indication
Phase
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Approval

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Regulation

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