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