Drug Profile

AMX 0114

Alternative Names: AMX-0114

Latest Information Update: 12 Jun 2025

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At a glance

Originator Amylyx Pharmaceuticals
Class Antisense oligonucleotides
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

03 Jun 2025 AMX 0114 receives Fast Track designation for Amyotrophic lateral sclerosis [Intrathecal,Injection] in USA
03 Jun 2025 Adverse events data from a preclinical trial in Amyotrophic lateral sclerosis was released by Amylyx Pharmaceuticals
07 Feb 2025 Phase-I clinical trials in Amyotrophic lateral sclerosis in Canada (Intrathecal) (NCT06665165)

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