Neuraltus Pharmaceuticals, Inc. has announced results from its phase II clinical study of NP001 in patients with ALS. Results demonstrated that NP001 was safe and well-tolerated.
Neuraltus Announces Encouraging Phase II Study Results
Results from a phase II clinical study of NP001 in patients with Amyotrophic Lateral Sclerosis were announced on October 30, 2012 by Neuraltus Pharmaceuticals, Inc. The study enrolled 136 patients with ALS in this multi-center, double-blind, placebo controlled study. Subjects received placebo, 1mg/kg or 2mg/kg intravenous infusions over six months. Results demonstrated that NP001 was safe and well-tolerated. Though not reaching pre-defined endpoints of statistical significance, results demonstrated positive trends in slowing the rate of disease progression by 13 to 19% using ALS Functional Rating Scale –Revised (ALSFRS-R). In addition, a post hoc analysis showed that 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.
Dr. Robert Miller of California Pacific Medical Center in San Francisco, the lead investigator of the study, enumerated four goals of this phase II study. First, to clarify whether there are any safety concerns - the study demonstrated that multiple dose over six months were generally safe and well tolerated. Second, to detect a sign of beneficial effect - the results indicate a promising signal. Third, to inform the decision of whether to go on to further study- these results justify going forward to next stage of testing. Fourth, the trial utilized a new trial design by receiving approval to use historical placebo controls to help the analysis. In summary, Dr. Miller stated, “all four goals were met and we are greatly encouraged”.
NP001 is a small molecule regulator of macrophage activation and is thought to restore the normal functioning of macrophages within the central nervous system. Aberrant macrophage activation is believed to be a significant contributor to the pathology underlying neurodegenerative diseases such as ALS.
Neuraltus Pharmaceuticals Inc. has met with the FDA and based on these results is planning a phase III clinical trial that is scheduled to begin in the second half of 2013. The company has set up a toll‐free number (1-888-347-7799) for patients and physicians to answer questions about the study. The toll free number will be available through December 31, 2012.