ALS Pipeline Companies

Amyotrophic Lateral Sclerosis Pipeline Companies

This page provides an index of biotechnology and pharmaceutical companies actively developing treatments for amyotrophic lateral sclerosis (ALS).

Overview

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting motor [neurons](/entities/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: [@clinicaltrialsgov]

• Gene therapy: AAV-based gene silencing, gene replacement
• Antisense oligonucleotides (ASOs): Targeted mRNA degradation
• Small molecule neuroprotective agents: SOD1, [C9orf72](/entities/c9orf72) targets
• Anti-inflammatory approaches: Microglial modulation
• Cell therapy: Stem cell-based approaches

Major Pharmaceutical Companies

Large Pharma ALS Programs

| Company | Key Drugs | Phase |
|---------|-----------|-------|
| [Biogen Inc.](/companies/biogen) | Tofersen (approved), BIIB100 (Phase 1) | Approved/Phase 1 |
| [Eli Lilly and Company](/companies/eli-lilly) | Retevmo (approved for other indications), ALS programs | Various |
| [Novartis](/companies/novartis) | ALS programs | Research |
| [Roche](/companies/roche) | Genentech collaboration | Research |
| [Pfizer](/companies/pfizer) | ALS programs | Various |
| [Sanofi](/companies/sanofi) | ALS programs | Various |
| [AstraZeneca](/companies/astrazeneca) | ALS programs | Various |

Biotechnology Companies

Amyotrophic Lateral Sclerosis Pipeline Companies

This page provides an index of biotechnology and pharmaceutical companies actively developing treatments for amyotrophic lateral sclerosis (ALS).

Overview

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease affecting motor [neurons](/entities/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: [@clinicaltrialsgov]

• Gene therapy: AAV-based gene silencing, gene replacement
• Antisense oligonucleotides (ASOs): Targeted mRNA degradation
• Small molecule neuroprotective agents: SOD1, [C9orf72](/entities/c9orf72) targets
• Anti-inflammatory approaches: Microglial modulation
• Cell therapy: Stem cell-based approaches

Major Pharmaceutical Companies

Large Pharma ALS Programs

| Company | Key Drugs | Phase |
|---------|-----------|-------|
| [Biogen Inc.](/companies/biogen) | Tofersen (approved), BIIB100 (Phase 1) | Approved/Phase 1 |
| [Eli Lilly and Company](/companies/eli-lilly) | Retevmo (approved for other indications), ALS programs | Various |
| [Novartis](/companies/novartis) | ALS programs | Research |
| [Roche](/companies/roche) | Genentech collaboration | Research |
| [Pfizer](/companies/pfizer) | ALS programs | Various |
| [Sanofi](/companies/sanofi) | ALS programs | Various |
| [AstraZeneca](/companies/astrazeneca) | ALS programs | Various |

Biotechnology Companies

Approved ALS Therapies

| Company | Drug | Mechanism | Status |
|---------|------|-----------|--------|
| [Amylyx Pharmaceuticals](/companies/amylyx) | Relyvrio (AMX0035) | SOD1 inhibitor + energy | Approved (2022) |
| [Biogen](/companies/biogen) | Tofersen (Qalsody) | SOD1 ASO | Approved (2023) |

Late-Stage ALS Programs

| Company | Key Drug | Mechanism | Phase |
|---------|----------|-----------|-------|
| [CytoKinetics](/companies/cytokinetics) | Reldesomstat | Fast skeletal muscle troponin inhibitor | Phase 3 |
| [Biohaven](/companies/biohaven) | BHV-0223 | Glutamate modulator | Phase 2/3 |
| [Clene Nanomedicine](/companies/clene-nanomedicine) | CNM-Au8 | Catalytic gold nanocrystals | Phase 2 |
| [BrainStorm Cell Therapeutics](/companies/brainstorm-cell-therapeutics) | NurOwn | MSC-NTF cells | Phase 3 (completed) |
| [AB Science](/companies/ab-science) | Masitinib | Tyrosine kinase inhibitor | Phase 3 |

Early-Stage ALS Programs

| Company | Focus Area |
|---------|------------|
| [Acadia Pharmaceuticals](/companies/acadia-pharmaceuticals) | 5-HT2A agonist |
| [ALN-ALS](/companies/alnylam) | Gene silencing |
| [Ionis Pharmaceuticals](/companies/ionis) | ASO therapies |
| [Denali Therapeutics](/companies/denali-therapeutics) | DNL788 (SOD1) |
| [Alector](/companies/alector) | Progranulin (FTD/ALS) |
| [Voyager Therapeutics](/companies/voyager-therapeutics) | Gene therapy |
| [Prevail Therapeutics](/companies/prevail-therapeutics) | Gene therapy (C9orf72) |
| [Passage Bio](/companies/passage-bio) | Gene therapy |
| [Lexeo Therapeutics](/companies/lexeo-therapeutics) | Gene therapy |

Therapeutic Approaches

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 |

Neuroprotective Agents

| 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 |

Anti-inflammatory Approaches

| Company | Drug | Target | Phase |
|---------|------|--------|-------|
| AB Science | Masitinib | CSF1R/Tyro3/Axl | Phase 3 |
| [Annexon](/companies/annexon) | ANX005 | C1q | Phase 1/2 |
| [Alector](/companies/alector) | AL001/AL101 | Progranulin | Phase 2/3 |

Cell Therapy

| Company | Product | Approach | Phase |
|---------|---------|----------|-------|
| BrainStorm Cell Therapeutics | NurOwn | MSC-NTF cells | Phase 3 |
| [Cynata Therapeutics](/companies/cynata-therapeutics) | CYP-001 | iPSC-derived MSCs | Phase 1 |
| [BlueRock Therapeutics](/companies/blue-rock-therapeutics) | Cell therapy | DA neurons | Preclinical |

Clinical Trial Trends

Active Clinical Trials by Phase (2024)

| Phase | Number of ALS Trials |
|-------|---------------------|
| Phase 1 | ~30 |
| Phase 2 | ~60 |
| Phase 3 | ~25 |

Key Success Factors

Company Collaboration Map

Company Collaboration Map

Additional Resources

• [ClinicalTrials.gov - ALS](https://clinicaltrials.gov/search?cond=Amyotrophic+Lateral+Sclerosis)
• [ALS Association - Research](https://www.als.org/research)
• [ALS Therapy Development Institute](https://www.als.net/)

External Links

• [ClinicalTrials.gov ALS Search](https://clinicaltrials.gov/search?spons=Amyotrophic+Lateral+Sclerosis)
• [ALS Association](https://www.als.org/)
• [NEALS Consortium](https://www.nealsconsortium.org/)

References