Neuraltus Pharmaceuticals, a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet medical needs, primarily for the treatment of neurodegenerative diseases, announced  top-line results from the company's phase 1 clinical study of NP001 for the treatment of Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).

NP001 is a small molecule regulator of macrophage activation. Aberrant macrophage activation is believed to be a primary contributor to the pathology underlying ALS and other neurodegenerative diseases. Neuraltus' NP001 is designed to restore the normal functioning of macrophages within the central nervous system. In addition to ALS, NP001 is being developed for the treatment of Parkinson's disease, Alzheimer's disease and Multiple Sclerosis.

A double-blinded, placebo-controlled, phase 1, single ascending dose study was performed to assess the safety and tolerability of four single intravenous doses of NP001 in a total 32 individuals with ALS. The primary objective of this study was the establishment of safety with patients being assessed at select time intervals for seven days post dosing. The secondary objective was to investigate the effects of NP001 on an ALS disease-associated blood biomarker pre- and 24 hours post dosing.

Single dose safety and tolerability of NP001 at four different dose levels was established in ALS patients. Importantly, 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; this is a first in ALS.

Robert G. Miller, MD, the study's principal investigator and Medical and Program Director of the Forbes Norris MDA/ALS Research Centre, commented, “NP001 appears to have affected the level of a biomarker directly related to ALS pathogenesis in a dose-dependent manner. This is extremely promising and is virtually unprecedented in ALS research. In addition, the safety profile of NP001 is very reassuring and gives us confidence going into the Phase 2 clinical study.”

Stanley H. Appel, MD, Chair of the Department of Neurology at The Methodist Hospital, commented, “The goal of the NP001 programme is to restore macrophages to their neuro-protective state, reducing inflammation and normalizing the cellular environment of critical nerve cells. The results of the Phase 1 trial are encouraging, providing the first clinical evidence that a therapeutic agent may be able to modify the activation state of macrophages in patients with ALS.”

Andrew Gengos, Neuraltus' President and CEO, stated, “Neuraltus efficiencies in clinical development have enabled the Company to successfully complete initial clinical studies in two of our three lead programs within 20 months of the Company's Series A funding. In addition to reporting the first clinical results for NP001, we will announce later this week the first phase 1/2 clinical results for our second lead program, NP002, in Parkinson's disease. These programs exemplify the Company's commitment to addressing serious unmet needs in treating neurodegenerative diseases, and we are pleased with their rapid progress.”

Neuraltus Pharmaceuticals, Inc. is a privately held biopharmaceutical company dedicated to developing and commercializing high-impact therapeutics that address critical unmet needs, primarily in the treatment of neurodegenerative diseases.