NP001 is an investigational immunotherapy for the treatment of ALS that has not been approved by the US Food and Drug Administration.

The safety and efficacy of NP001 in this indication have not been established. Statements and contents on this website have not been evaluated by the FDA or other health authorities.

Proprietary Platform with Transformational Potential

What is NP001?

NP001 is an immunotherapy under investigation for ALS engineered for appropriate administration as an infusion into the bloodstream.

This formulation is VERY important for two critical reasons:

  1. The product is pure enough to eliminate the risk of life-threatening contaminants
  2. Ability to penetrate deep within innate immune cells to facilitate the formation of an essential regulator of inflammation
Proposed Mechanism of Action (MOA)

NP001 is a small molecule prodrug that is intracellularly converted to taurine chloramine (TauCl) – an established regulator needed to balance pro- and anti-inflammatory processes of the innate immune system. In ALS, TauCl is depleted due to a constant call for inflammatory mediators that results in an imbalance of the innate immune response and destructive inflammation to motor neurons.

Aligned Mechanistic Function

By refueling the body with an essential regulator of inflammation, NP001's goal is to transform tissue-damaging inflammatory macrophages to a non-inflammatory, wound-healing state.

poster
Key Takeaways:
  • NP001 is a prodrug to taurine chloramine (TauCl) – an established regulator of innate inflammation
  • Taurine is converted to TauCl and released from activated neutrophils to regulate production of inflammatory mediators
  • TauCl triggers the resolution of inflammatory processes within macrophages that transform from damaging to wound-healing thus slowing destructive disease processes in patients with ALS
Publications
Quantitative Measures of Time to Loss of 15% Vital Capacity and Survival Extension in Slowly Progressive Amyotrophic Lateral Sclerosis (ALS) Patients Treated with the Immune Regulator NP001 Suggests an Immunopathogenic Subset of ALS, Biomedicines, Dec 12, 2025
Respiratory Function Improvement and Lifespan Extension Following Immunotherapy with NP001 Support the Concept That Amyotrophic Lateral Sclerosis (ALS) Is an Immuno-Neurologic Disease, International Journal of Molecular Sciences, May 3, 2025
The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis, Biomedicines, Oct 16, 2024
Systemic Innate Immune System Restoration as a Therapeutic Approach for Neurodegenerative Disease: Effects of NP001 on Amyotrophic Lateral Sclerosis (ALS) Progression, Biomedicines, Oct 16, 2024
Regulation of the Innate Immune System as a Therapeutic Approach to Supporting Respiratory Function in ALS, Cells, March 28, 2023
Macrophage-Targeted Sodium Chlorite (NP001) Slows Progression of Amyotrophic Lateral Sclerosis (ALS) through Regulation of Microbial Translocation, Biomedicines, Nov 12, 2022
Phase 2B Randomized Controlled Trial of NP001 in Amyotrophic Lateral Sclerosis: Pre-Specified and Post-Hoc Analysis, Muscle & Nerve, Jan 31, 2022
Randomized Phase 2 Trial of NP001 – a Novel Immune Regulator: Safety and Early Efficacy in ALS, Neurology Neuroimmunology & Neuroinflammation, April 9, 2015
NP001 Regulation of Macrophage Activation Markers in ALS: A Phase I Clinical and Biomarker Study, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Dec 15, 2014