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AMX 0114

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Drug Profile

AMX 0114

Alternative Names: AMX-0114

Latest Information Update: 26 Jun 2026

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At a glance

  • Originator Amylyx Pharmaceuticals
  • Class Antisense oligonucleotides; Neuroprotectants
  • Mechanism of Action Calpain expression inhibitors; Gene silencing
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes
  • Available For Licensing Yes

Highest Development Phases

  • Phase I Amyotrophic lateral sclerosis
  • Preclinical Neurodegenerative disorders

Most Recent Events

  • 23 Jun 2026 Pharmacodynamics and adverse events data from the phase I LUMINA trial in Amyotrophic lateral sclerosis released by Amylyx Pharmaceuticals
  • 07 Dec 2025 Adverse events data from the phase I LUMINA trial in Amyotrophic lateral sclerosis released by Amylyx Pharmaceuticals
  • 03 Jun 2025 AMX 0114 receives Fast Track designation for Amyotrophic lateral sclerosis [Intrathecal,Injection] in USA

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