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PLX 300

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Drug Profile

PLX 300

Alternative Names: PLX-300

Latest Information Update: 03 Jan 2024

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At a glance

  • Originator Polaryx Therapeutics
  • Class Anti-inflammatories; Small molecules
  • Mechanism of Action Apoptosis inhibitors; Peroxisome proliferator-activated receptor alpha agonists
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Niemann-Pick disease type A; Niemann-Pick disease type B; GM2 gangliosidoses; Globoid cell leukodystrophy
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase II Globoid cell leukodystrophy; Niemann-Pick disease type A; Niemann-Pick disease type B; Sandhoff disease; Tay-Sachs disease
  • Preclinical Neuronal ceroid lipofuscinosis

Most Recent Events

  • 03 Jan 2024 Phase-II clinical trials in Globoid cell leukodystrophy (PO) (Polaryx Therapeutics pipeline, January 2024)
  • 03 Jan 2024 Phase-II clinical trials in Niemann-Pick disease type A (PO) (Polaryx Therapeutics pipeline, January 2024)
  • 03 Jan 2024 Phase-II clinical trials in Niemann-Pick disease type B (PO) (Polaryx Therapeutics pipeline, January 2024)

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