ALS Pipeline Companies

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ALS Pipeline Companies

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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

Genetic targeting: Success with SOD1 and C9orf72 has validated genetic approaches

Biomarker confirmation: [Neurofilament light](/biomarkers/neurofilament-light-chain-nfl) chain (NfL) as efficacy marker

Patient selection: Genetic stratification (SOD1, C9orf72, FUS)

Endpoint selection: ALSFRS-R, survival, forced vital capacity

Company Collaboration Map

Mermaid diagram (expand to render)

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

Unknown, ALS Association - Clinical Trials (n.d.)

Unknown, ClinicalTrials.gov ALS Search (n.d.)

[Unknown, Nature Reviews Neurology - ALS Pipeline 2024 (2024)](https://doi.org/10.1038/s41582-024-00870-1)

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Related Entities

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📊 Evidence Profile Foundational

Evidence Balance

+0%

Certainty

100%

Debates

0

Incoming

44

Outgoing

68

0 supporting 0 contradicting 0 neutral

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📗 Cite This Artifact

ALS Pipeline Companies

Amyotrophic Lateral Sclerosis Pipeline Companies

Overview

Major Pharmaceutical Companies

Large Pharma ALS Programs

Biotechnology Companies

Amyotrophic Lateral Sclerosis Pipeline Companies

Overview

Major Pharmaceutical Companies

Large Pharma ALS Programs

Biotechnology Companies

Approved ALS Therapies

Late-Stage ALS Programs

Early-Stage ALS Programs

Therapeutic Approaches

Gene Therapy and ASO

Neuroprotective Agents

Anti-inflammatory Approaches

Cell Therapy

Clinical Trial Trends

Active Clinical Trials by Phase (2024)

Key Success Factors

Company Collaboration Map

Company Collaboration Map

Additional Resources

External Links

References

💬 Discussion