Last update 22 Mar 2025

PLX-300

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
PLX 300, PLX300
Target
PPARα
Action
agonists, inhibitors
Mechanism
PPARα agonists(Peroxisome proliferator-activated receptor α agonists), Apoptosis inhibitors
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Niemann-Pick Disease, Type ALeukodystrophy, Globoid Cell
Inactive Indication
Niemann-Pick Disease, Type BSandhoff DiseaseTay-Sachs Disease
Originator Organization
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
Active Organization
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
Inactive Organization-
Drug Highest PhasePhase 1
First Approval Date-
RegulationOrphan Drug (United States), Rare Pediatric Disease (United States)
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Related

100 Clinical Results associated with PLX-300
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100 Translational Medicine associated with PLX-300
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100 Patents (Medical) associated with PLX-300
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100 Deals associated with PLX-300
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Niemann-Pick Disease, Type APhase 1
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
18 Dec 2020
Leukodystrophy, Globoid CellPhase 1
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
14 Nov 2020
Niemann-Pick Disease, Type BPreclinical
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
10 Nov 2020
Sandhoff DiseasePreclinical
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
10 Nov 2020
Tay-Sachs DiseasePreclinical
United States
Polaryx Therapeutics, Inc.Polaryx Therapeutics, Inc.Startup
04 Nov 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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Approval

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Regulation

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