This page provides an index of biotechnology and pharmaceutical companies actively developing treatments for amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting motor neurons. The ALS drug development landscape includes major pharmaceutical companies with approved therapies and innovative biotechnology companies developing novel mechanisms of action. Key therapeutic approaches include:
- Gene therapy: AAV-based gene silencing, gene replacement
- Antisense oligonucleotides (ASOs): Targeted mRNA degradation
- Small molecule neuroprotective agents: SOD1, C9orf72 targets
- Anti-inflammatory approaches: Microglial modulation
- Cell therapy: Stem cell-based approaches
| Company |
Drug |
Mechanism |
Status |
| Amylyx Pharmaceuticals |
Relyvrio (AMX0035) |
SOD1 inhibitor + energy |
Approved (2022) |
| Biogen |
Tofersen (Qalsody) |
SOD1 ASO |
Approved (2023) |
¶ Gene Therapy and ASO
| Company |
Drug |
Target |
Approach |
Phase |
| Biogen/Ionis |
Tofersen |
SOD1 |
ASO |
Approved |
| Biogen |
BIIB100 |
C9orf72 |
ASO |
Phase 1 |
| Ionis |
ION363 |
C9orf72 |
ASO |
Phase 1/2 |
| Wave Life Sciences |
WVE-004 |
C9orf72 |
ASO |
Phase 1 |
| Denali |
DNL788 |
SOD1 |
Small molecule |
Preclinical |
| Prevail Therapeutics |
AAV.C9orf72 |
C9orf72 |
Gene therapy |
Preclinical |
| Company |
Drug |
Mechanism |
Phase |
| Amylyx |
Relyvrio |
Mitochondrial protection |
Approved |
| Clene Nanomedicine |
CNM-Au8 |
Catalytic nanomedicine |
Phase 2 |
| Biohaven |
BHV-0223 |
Glutamate modulation |
Phase 2/3 |
| Prilenia |
Pridopidine |
Sigma-1 agonist |
Phase 2 |
| Araim Pharmaceuticals |
ARA-290 |
Innate repair receptor agonist |
Phase 2 |
| Company |
Drug |
Target |
Phase |
| AB Science |
Masitinib |
CSF1R/Tyro3/Axl |
Phase 3 |
| Annexon |
ANX005 |
C1q |
Phase 1/2 |
| Alector |
AL001/AL101 |
Progranulin |
Phase 2/3 |
| Company |
Product |
Approach |
Phase |
| BrainStorm Cell Therapeutics |
NurOwn |
MSC-NTF cells |
Phase 3 |
| Cynata Therapeutics |
CYP-001 |
iPSC-derived MSCs |
Phase 1 |
| BlueRock Therapeutics |
Cell therapy |
DA neurons |
Preclinical |
| Phase |
Number of ALS Trials |
| Phase 1 |
~30 |
| Phase 2 |
~60 |
| Phase 3 |
~25 |
- Genetic targeting: Success with SOD1 and C9orf72 has validated genetic approaches
- Biomarker confirmation: Neurofilament light chain (NfL) as efficacy marker
- Patient selection: Genetic stratification (SOD1, C9orf72, FUS)
- Endpoint selection: ALSFRS-R, survival, forced vital capacity
graph TD
Biogen --- Ionis
Biogen --- Roche
BrainStorm --- Academic
Clene --- Academic
Alector --- Academic
Ionis --- Roche
Prevail --- Eli_Lilly
Biohaven --- Academic