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Trial Shows Drug Effect on ALS Blood Biomarker

Neuron illustration (NIH)

(National Institute on Aging, NIH)

Neuraltus Pharmaceuticals in Palo Alto, California said today an early stage clinical trial of its drug NP001 for the treatment of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) showed improvement in a blood biomarker associated with the disease with patients taking the drug. ALS is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement.

Aberrant macrophage activation is believed to be a primary contributor to ALS and other neurodegenerative diseases. Macrophages are a type of white blood cell that surrounds and kills microorganisms, removes dead cells, and stimulates the action of other immune system cells. Neuraltus says NP001 is designed to restore the normal functioning of macrophages in the central nervous system.

This phase 1 trial involved a double-blinded, placebo-controlled single ascending dose study to assess the safety and tolerability of four single intravenous doses of NP001 in a total 32 patients with ALS. The study aimed primarily to establish the drug’s safety with patients, which was assessed at select time intervals for seven days after dosing. The secondary objective was to test the effects of NP001 on an ALS disease-associated blood biomarker before and 24 hours after dosing.

Neuraltus says the trial established single dose safety and tolerability of NP001 at four different dose levels in ALS patients. Following a single dose, there was a dose-dependent, statistically significant improvement in blood levels of the biomarker thought to be involved in ALS disease progression. The company says this result is a first in ALS.

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