Drug Name: NP001
Company: Neuraltus Pharmaceuticals
Location: US-California
Drug Type: Small Molecule
Conditions: ALS, Parkinson’s disease, Alzheimer’s disease, frontotemporal dementia, ataxis-telangiectasia
Mechanism Type: Immunomodulation
Mechanism: NP001 is a small molecule regulator of macrophage activation. It is thought to restore macrophages to their neuroprotective state and reduce inflammation seen in ALS. Treatment of ALS mouse models with NP001 extended survival. In the Phase IIa safety study, administration of a high dose of NP001 (2mg/kg) over a 6 month period was associated with a slowing of disease progression in 27% of patients, approximately 2.5 times greater than the percentage in patients on placebo (10%).
U.S. Status for ALS: Inactive

[1] Serum C-Reactive Protein as a Prognostic Biomarker in Amyotrophic Lateral Sclerosis. Lunetta, C. et al. JAMA Neurol. 2017 Jun 1;74(6):660-667.
[2] Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS. Miller, RG et al. Neurol Neuroimmunol Neuroinflamm. 2015 Apr 9;2(3):e100.
[3] Differential effects on innate versus adaptive immune responses by WF10. Giese, T. et al. Cell Immunol. 2004 Jun;229(2):149-58.

Last updated June 21st, 2017

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