PALO ALTO, Calif., Dec. 9, 2013 /PRNewswire/ -- Neuraltus Pharmaceuticals, Inc. announced today that promising efficacy results of the Company's Phase 2 clinical program of NP001 for the treatment of amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease) were highlighted in an oral presentation at the 24th International Symposium on ALS/MND, held in Milan, Italy. Study efficacy results demonstrated positive trends in the ability of NP001 to slow 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. 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. The high dose of NP001 (2mg/kg) halted progression in 27% of patients compared to 11% of patients on placebo. Further, NP001 was found to be safe and well-tolerated in the study.
In addition, the researchers discovered a trend in greater improvement in patients with higher baseline inflammation who received the high dose of NP001, achieving a 44% improvement compared to placebo. These findings provide further support for the hypothesis that NP001's primary mechanism of action is the restoration of normal function in inflammatory macrophages. The Phase 2 data were reported in an oral presentation, titled "Additional Follow-up and Biomarker Data from a Phase II Safety and Preliminary Efficacy Trial of NP001: A Novel Immune Regulator for Slowing Progression of ALS," delivered by Principal Investigator Robert G. Miller, MD, who serves as Director of the Forbes Norris MDA/ALS Research and Treatment Center of the California Pacific Medical Center.
NP001 is a small molecule regulator of inflammatory macrophage activity. Aberrant macrophage activity 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.
According to Dr. Miller, "We are pleased to report the current, most up-to-date findings from the Phase 2 study. Clinical effects of NP001 are consistent with what we've seen in earlier research, which demonstrated that NP001 is able to lower markers of abnormal inflammatory macrophages in in vitro and in vivo preclinical studies, as well as in a dose-dependent manner in earlier-stage clinical work. In the Phase 2 study, we saw a halting of disease progression in 27% of the high-dose treatment group patients, a finding we have never seen in prior ALS trials. In addition, we strongly believe the trial design confirmed the utility of using historical placebo controls for signal amplification and hypothesis generation and that the benefit-risk supports further development of NP001. We look forward to the continued progress of this program."
Neuraltus recently updated their development timeline for NP001, noting that the Company is expecting to initiate a Phase 3 study sometime in 2014.
More About the Phase 2 NP001 Study
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. Further, the benefit of NP001 related to the degree of baseline inflammation in patients. Those patients with greater baseline inflammation and who received the high dose of NP001 experienced a 44% greater slope improvement (positive trend did not reach significance, however).
About Neuraltus Pharmaceuticals, Inc.
Neuraltus Pharmaceuticals, Inc. is a privately-held biopharmaceutical company developing novel therapeutics to treat neurological disorders. Neuraltus' clinical-stage product candidate, NP001, has been shown to regulate the activation of select immune cells (macrophages), transforming them from an activated or inflammatory state to their normal quiescent state in order to normalize the cellular environment of critical nerve cells. Neuraltus has completed a Phase 2 clinical study of NP001 in patients with amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), results of which were reported in late 2012.
For more information on Neuraltus, please visit www.neuraltus.com.
Neuraltus Pharmaceuticals, Inc.
Rich Casey, President and CEO
Burns McClellan, on behalf of Neuraltus
Tel: (650) 424-1600 x. 300
Justin Jackson (media)
SOURCE Neuraltus Pharmaceuticals, Inc.