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Neuraltus Pharmaceuticals Announces Phase 2 Clinical Results for NP001 in Patients with Amyotrophic Lateral Sclerosis (ALS)

PALO ALTO, Calif., Oct. 30, 2012 /PRNewswire/ -- Neuraltus Pharmaceuticals, a private biopharmaceutical company developing novel therapies for rare neurological disorders, announced today top-line results from the Company's Phase 2 clinical study of NP001 in patients with Amyotrophic Lateral Sclerosis (ALS, or Lou Gehrig's disease).  Study efficacy results demonstrated positive trends in slowing the rate of disease progression, ranging from 13 to 19% in multiple parameters of clinical benefit, although these pre-defined endpoints did not reach statistical significance.  Importantly, according to a post hoc analysis, a greater percentage of patients receiving NP001 experienced a halt in disease progression which reached statistical significance when compared to the combination of concurrent and matched historical (placebo) controls.  Further, NP001 was found to be safe and well-tolerated.  Based on these results, and after a meeting with the FDA, Neuraltus is planning a Phase 3 clinical program that is expected to begin in the second half of 2013.

"ALS is a rare neurodegenerative disease that typically leads to respiratory failure and death within five years of diagnosis.  The results from this study with NP001 are most encouraging, as halting or slowing the rate of disease progression, in a subset of patients, as this study suggests, would translate into a clear clinical benefit for these patients," commented Robert G. Miller, M.D., of California Pacific Medical Center, the Principal Investigator of the study.

NP001 is a small molecule regulator of macrophage activation.  Aberrant macrophage activation is believed to be a significant 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.

The multi-center, double-blind, placebo-controlled, Phase 2 study enrolled 136 patients with ALS.  Patients were randomized to receive either placebo, or 1mg/kg or 2mg/kg intravenous infusion of NP001 over a period of six months, followed by a six-month follow-up period.  The study was designed to evaluate the change in slope of the ALS Functional Rating Score Revised (ALSFRS-R) and the safety and tolerability of NP001, as well as change from baseline in ALSFRS-R through six months, a joint rank analysis of change of ALSFRS-R adjusted for mortality and changes in readily measured peripheral inflammation biomarkers.  An additional secondary endpoint agreed upon with the FDA was the inclusion of matched historical placebo control ALS patients to increase signal detection and power.

Post hoc analysis of the Phase 2 study showed a dose response to NP001 with 27% of the patients who received 2mg/kg NP001 having no progression of their disease during the six-month dosing period.  This is approximately 2.5 times as many as were seen in the concurrent placebo group.  When historical placebo controls were included with concurrent controls, this difference versus placebo became statistically significant.  Although post hoc analysis must be considered cautiously and can be subject to bias, the magnitude of the benefit identified underscores a potentially clinically relevant and meaningful result.  In addition, results from the study demonstrated trends of clinical benefit for the 2 mg/kg cohort in the primary endpoint of change in slope of the ALSFRS-R, and in the secondary endpoints of change from baseline in ALSFRS-R through six months and a joint rank analysis of change of ALSFRS-R adjusted for mortality.

Neuraltus has established a toll-free number for patients and physicians to answer questions about the study.  That number, 1-888-347-7799, will be available through December 31, 2012.

About Neuraltus Pharmaceuticals, Inc.
Neuraltus Pharmaceuticals, Inc. is a privately held biopharmaceutical company developing novel, first-in-class therapeutics that address critical unmet needs in the treatment of rare neurological disorders.  In addition to NP001, Neuraltus also has a pre-clinical program, NP003, for the treatment of lysosomal storage disorders such as Fabry's disease and Gaucher's disease.  For more information on Neuraltus, please visit

 CONTACT:Neuraltus Pharmaceuticals, Inc.Burns McClellanJohn WalkerJustin Jackson (media)Chairman and Interim CEOjjackson@burnsmc.com650-424-orMichelle Szwarcberg (media) 212-213-0006

SOURCE Neuraltus Pharmaceuticals
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