Masitinib

Masitinib

AB Science · ALS
Late-stage active recruiting CT.gov grounded

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

Program overview

Mechanism
tyrosine kinase / mast cell modulation
Modality
small molecule
Phase
Late-stage
Status
active
Recruitment
recruiting
Confidence
medium
Watch priority
2
Last verified
20 Apr 2026

Sponsor and trial identifiers

Sponsor
AB Science
Trial IDs
NCT03127267, NCT07174492
ClinicalTrials.gov exact matches: NCT03127267, NCT07174492

Regions and recruitment

Active in 17 regions
United States Belgium Denmark France Germany Greece Israel Italy Norway Poland Portugal Russia Serbia Slovenia Spain Sweden Ukraine

Indication tags

ALS

Milestones and catalysts

Last milestone
One of the few still-material late-stage ALS programs outside the genetic precision therapy track.
Next expected catalyst
Phase 3 progression and whether the long-running controversy resolves into a usable signal.

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.

Mechanism
SOD1 antisense reduction
Modality
ASO
Next catalyst
Longer-term follow-up and real-world use data.
Last verified
20 Apr 2026

DNL343

Denali Therapeutics · ALS
Priority 2
Mid-stage completed completed

ALS program aimed at stress-response biology rather than gene-specific targeting.

Mechanism
integrated stress response modulation
Modality
small molecule
Next catalyst
Readouts on clinical progression and tolerability.
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.

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

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.

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

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