to stopping ALS progression
Working to transform inflammatory macrophages
to control the progression of
motor neuron damage

A potential new treatment for ALS that could help patients who are non-responsive to current therapy options

Neuvivo is dedicated to creating therapeutics for the treatment of Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. We are investigating a patented macrophage-targeted technology, NP001, designed to address chronic inflammation, a key factor in the progressive loss of motor function in ALS.

To date, the treatment has received Orphan Drug and Fast Track designation by the FDA as it addresses a clear unmet medical need. We are diligently working to move this compound toward regulatory approval, with the goal of delivering an effective new treatment to reduce the suffering caused by ALS.

Targeting inflammatory macrophages with the goal of returning them to a non-inflammatory state

ALS is a neurological disorder that causes the loss of motor neurons and results in progressively debilitating muscle weakness. There is an emerging consensus that ALS disease progression is associated with systemic inflammation and abnormal inflammatory macrophage/microglia activity within affected spinal cord and brain regions. Macrophages are white blood cells, critical for wound healing, clearance of bacteria, and resolution of inflammation. In one state (M1), macrophages are toxic, producing inflammatory cytokines (TNF-α) and other proinflammatory factors. In a second state (M2), macrophages are neuroprotective and associated with the reversal of inflammation and the suppression of uncontrolled immune responses. In ALS, the appearance of new M1 macrophages in the spinal cord indicates disease progression.
Microglia are a type of macrophage located throughout the brain and spinal cord, accounting for 10-15% of all cells found within the brain.
They are a subset of macrophage cells and act as the first and main form of active immune defense in the central nervous system (CNS). Microglial inflammatory activation has been observed consistently in association with degenerating upper and lower motor neurons.
Neuvivo’s investigational therapeutic, NP001, is a small molecule which, through a series of biochemical interactions in the body, is converted to a compound that can transform inflammatory macrophages (M1) to the non-inflammatory, wound healing state (M2), with the goal of stopping damage to motor neurons.

Current research points to compromised macrophage function as a critical factor in many other CNS diseases, such as Parkinson’s Disease and Alzheimer’s Disease, suggesting that NP001 may potentially have a role in addressing a range of CNS diseases.

Implications for patients living with ALS

ALS is a neurological disorder that causes the loss of motor neurons with grave consequences for the patient. There are approximately 40,000-50,000 people living with ALS in the US and Europe. There are just two active ingredients currently approved to treat the disease, but both have limitations.

Neuvivo has developed NP001, a proprietary small molecule investigational therapeutic that in clinical trials has shown an ability to slow the progression of the disease in patients age 40-65. Clinical trials* of NP001 to date have not suggested any drug-related serious adverse events.

Neuvivo is working closely with leading ALS clinicians, researchers, and regulatory authorities to develop an effective and safe treatment for people living with ALS – as efficiently as possible.

* Miller RG, Zhang R, Block G, et al. NP001 regulation of macrophage activation markers in ALS: a Phase I clinical and biomarker study. Amyotroph Lateral Scler Frontotemporal Degener 2014;15:601–609.

Miller RG, Block G, Katz JS, Barohn RJ, Gopalakrishnan V, Cudkowicz M, et al. Randomized Phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS. Neurol Neuroimmunol Neuroinflamm. 2015;2(3):e100.

NP001 has gained Orphan Drug and Fast Track Status from the FDA

Neuvivo has completed two Phase 2 trials, providing data demonstrating NP001’s potential to slow the progression of ALS and to protect motor neurons. To date, the trials have not suggested any drug-related serious adverse events.

Neuvivo is now in a position to initiate Phase 2 studies in Parkinson’s Disease, Alzheimer’s Disease and Vascular Dementia.

NP001 has strong IP protection. 8 patents have been issued in the US and ROW with protection extending through 2042. Additional patents have been filed.

Early patents relating to the technology are under license from the University of California, San Francisco.

Pipeline for NP001


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The team at Neuvivo is committed to developing groundbreaking treatments for ALS and other motor neuron diseases.

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