Oral brain-penetrant therapy for ALS that may slow or reverse progression
Official title: Development of a novel oral brain penetrant therapeutic for ALS disease
Funded at Aquinnah Pharmaceuticals, INC. · NIH-11354018 · Data updated 2026-11-12
A promising new pill designed to reach the brain and slow or reverse ALS progression might help people living with ALS.
Quick facts
| Grant type | Sbir 2 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Aquinnah Pharmaceuticals, INC. NIH-funded |
| Lab location | 1 site (Newton, United States) |
| Project start | June 15, 2025 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $1,499,529 |
| NIH project number | 5R44NS141531-02 |
| Project ID | NIH-11354018 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults diagnosed with ALS who fit the study’s inclusion criteria and are eligible for early-phase drug development.
✕Less likely to help
Patients with ALS who do not meet the trial criteria or who do not respond to this mechanism may not experience benefit.
What this research studies
This grant funds the development of a groundbreaking oral drug intended to cross into the brain and reduce the buildup of a toxic protein in ALS patients. The approach focuses on a common ALS feature called TDP-43 clumping, with preclinical and early clinical exploration to see if symptoms and disease progression can improve. If successful, it could lead to a treatment that complements existing supportive care and extends quality of life for people with ALS.
Why it matters
Potential benefit: If successful, patients could experience slower disease progression and better daily functioning.
How similar studies have gone: Early-stage approaches targeting ALS biology have shown potential in slowing progression in some studies, but this remains an area with high unmet need.
Where this research is happening
-
Aquinnah Pharmaceuticals, INC.
Newton, United States Active
Researchers
- Principal investigator: Larsen, Glenn — Aquinnah Pharmaceuticals, INC.
- Study coordinator: Larsen, Glenn
Related research
Other NIH-funded projects on the same topics.
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A new Alzheimer’s treatment targeting tau proteins
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A new approach to treating Alzheimer's disease by targeting tau protein
This research is developing new medicines to help people with Alzheimer's disease by focusing on a specific protein called tau.
-
Creating a new oral treatment for ALS
This study is testing a new oral medication for people with ALS that aims to help slow down the disease by targeting a harmful protein, which could improve muscle control and make daily life a bit easier.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.