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AMX0114

Oligonucleotide (Antisense oligonucleotide) · Gene silencing: calpain-2 (CAPN2) · developed by Amylyx Pharmaceuticals

Phase 1 Most advanced trial

AMLX Developer

1 Registered trials

What is AMX0114?

AMX0114 is an antisense oligonucleotide that targets the mRNA encoding calpain-2, reducing its production. It is being studied in amyotrophic lateral sclerosis (ALS), where calpain-2 has been linked to neuron degeneration and death.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Oligonucleotide (Antisense oligonucleotide)
Targets: calpain-2 (CAPN2)
Mechanism: Gene silencing: calpain-2 (CAPN2)
Pathways: neuron degeneration, neuron death
Therapy area: Neurology
Developer: Amylyx Pharmaceuticals Inc. (AMLX)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT06665165	Phase 1	ALS	Recruiting	AMLX

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

ALS

More from Amylyx Pharmaceuticals

AMX0035 Phase 2/3 Avexitide Phase 3

Sources

ClinicalTrials.gov NCT06665165

Updated Oct 11, 2026. Something wrong or missing? Report it on the AMLX page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z