PALO ALTO, Calif., Sept. 17, 2014 /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) are being highlighted today in an oral presentation at the inaugural Summit of the ALS Research Group, being held in Bloomington, Minnesota, September 17-19, 2014. According to a post hoc analysis, administration of a high dose of NP001 (2mg/kg) was associated with a halt in disease progression in 27% of patients, approximately 2.5 times greater than the percentage in patients on placebo (10%). The researchers determined that two major plasma factors – Interleukin-18 (IL-18) and lipopolysaccharide (LPS), both markers of inflammation – may differentiate NP001 responders from non-responders.
In the analysis, the responder population was shown to have had significantly higher levels of IL-18, a cytokine involved in inflammation-driven cell death, than the non-responders at baseline (p=0.02). Additionally, all NP001 responders had detectable levels of LPS in their plasma, signifying abnormal macrophage function, whereas none of the placebo non-progressors had detectable LPS at baseline. 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. Neuraltus' NP001 is designed to restore the normal functioning of macrophages within the central nervous system.
Neuraltus Founder and Chief Scientific Officer, Dr. Mike McGrath, will present the new data from the Summit later today. Dr. McGrath commented, "We are excited by these results, which to our knowledge provide the first evidence of a more favorable response to an investigational ALS clinical candidate based on the patient's own immune status. As will be discussed in today's presentation, we believe that elevated IL-18 and the presence of LPS, both of which indicate an ongoing neuroinflammatory process, may help identify patients more likely to benefit from NP001. Blood markers such as these may become an important tool in helping to identify potential treatments for different subpopulations in this heterogeneous disorder." Based on the current clinical evidence, Neuraltus expects to initiate dosing in its next clinical trial of NP001 in the second quarter of 2015.
About Neuraltus' 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 on a monthly basis 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. 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.
Overall, 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. 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). Further, NP001 was found to be generally safe and well-tolerated in the study.
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).
For more information on Neuraltus, please visit www.neuraltus.com.
SOURCE Neuraltus Pharmaceuticals, Inc.