AMX0035

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AMX0035

Amylyx · ALS
Late-stage / Withdrawn active active, not recruiting CT.gov grounded

Important failed or withdrawn ALS program that still matters as pipeline context.

Open ALS hub Filter main pipeline

Program overview

Mechanism
cell stress and mitochondrial support combination
Modality
small molecule combination
Phase
Late-stage / Withdrawn
Status
active
Recruitment
active, not recruiting
Confidence
medium
Watch priority
3
Last verified
20 Apr 2026

Sponsor and trial identifiers

Sponsor
Amylyx
Trial IDs
NCT05021536, NCT05619783
ClinicalTrials.gov exact matches: NCT05021536, NCT05619783

Regions and recruitment

Active in 12 regions
United States Belgium France Germany Ireland Italy Netherlands Poland Portugal Spain Sweden United Kingdom

Indication tags

ALS

Milestones and catalysts

Last milestone
The withdrawal story makes this a valuable negative reference point for the monitor.
Next expected catalyst
No active development catalyst currently tracked here.

Related pages

Disease hub ALS hub Disease-specific views with programs, mechanisms, and related context.
Research Research agenda Research notes and background around ALS.
Key Targets Key targets The mechanisms and intervention levers behind these programs.
Discover Discover Explainers and visual introductions to the science behind neurodegeneration.
Join Get involved Contribute research, writing, design, engineering, or review.

Related programs

Each card explains why it is here so you can read the overlap at a glance.

Tofersen

Biogen · ALS
Priority 1
Approved / Post-approval completed completed

Gene-targeted ALS therapy with major strategic importance for precision neurodegeneration treatment.

Shown because it shares same disease bench and shared biology: als with AMX0035.
Mechanism
SOD1 antisense reduction
Modality
ASO
Next catalyst
Longer-term follow-up and real-world use data.
Last verified
20 Apr 2026

MND-SMART

University of Edinburgh · ALS
Priority 2
Mid-stage / Late-stage active recruiting

A UK-centered ALS platform trial that matters as infrastructure as much as any single drug inside it.

Shown because it shares same disease bench and shared biology: als with AMX0035.
Mechanism
multi-arm adaptive platform trial
Modality
platform / multi-intervention trial
Next catalyst
Ongoing arm-level readouts and additions within the adaptive platform framework.
Last verified
20 Apr 2026

Masitinib

AB Science · ALS
Priority 2
Late-stage active recruiting

Late-stage ALS program that stays relevant because the field still lacks enough credible shots on goal.

Shown because it shares same disease bench and shared biology: als with AMX0035.
Mechanism
tyrosine kinase / mast cell modulation
Modality
small molecule
Next catalyst
Phase 3 progression and whether the long-running controversy resolves into a usable signal.
Last verified
20 Apr 2026

NurOwn

BrainStorm Cell Therapeutics · ALS
Priority 2
Late-stage active not yet recruiting

A controversial but undeniably field-relevant ALS cell-therapy program that should be represented in a serious pipeline monitor.

Shown because it shares same disease bench and shared biology: als with AMX0035.
Mechanism
autologous MSC-NTF cell therapy
Modality
cell therapy
Next catalyst
Whether the renewed Phase 3 path can produce a cleaner efficacy narrative than prior attempts.
Last verified
20 Apr 2026

Source links

ClinicalTrials.gov NCT05021536 ClinicalTrials.gov NCT05619783 ClinicalTrials.gov search Amylyx news
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