NP001 Phase 2 Data
The first randomized, placebo controlled Phase 2 study of NP001 in 136 patients with for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) demonstrated positive trends in slowing the rate of disease progression, although these pre-defined endpoints did not reach statistical significance. The study results suggest a clinically meaningful slowing of progression in patients with greater baseline inflammation. In a post hoc analysis of the Phase 2 study, 25 percent of patients who received 2 mg/kg NP001 had no progression of disease during the six-month dosing period, compared to 11 percent in the concurrent placebo group, although it did not reach statistical significance (p=0.22). When matched historical placebo controls were included, the difference versus placebo became statistically significant (p=0.02). In addition, results from the study demonstrated trends of clinical benefit for the 2 mg/kg patient cohort in the change in slope of the ALS Functional Rating Score Revised (ALSFRS-R), and in change from baseline in ALSFRS-R through six months and a joint rank analysis of change of ALSFRS-R adjusted for mortality. These results suggest that NP001 may have a dose-dependent ability to halt ALS progression in a subset of patients exhibiting elevated levels of select inflammation biomarkers. Further, data from the Phase 2 study suggest that NP001 was generally safe and well-tolerated.
Secondary NP001 Analyses
Secondary analyses of the Phase 2 clinical trial of NP001 in ALS suggest that a biomarker for systemic inflammation, C-reactive protein (CRP), may be an indicator of positive treatment response to NP001. As previously disclosed in the primary analysis of the study (published in the April 2015 issue of Neurology: Neuroimmunology & Neuroinflammation), NP001 showed clinically meaningful slowing of progression in patients with greater baseline inflammation. Further to these results, these secondary analyses demonstrated that patients with baseline CRP levels greater than the median had a statistically significant benefit (p< 0.02) from treatment with the high dose (2 mg/kg) of NP001 as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) [Figure 1].
Additionally, ALS patients with greater baseline inflammation also maintained better vital respiratory capacity, or the amount of air exhaled after the deepest possible inhalation, in the study [Figure 2].
Although the subset of patients with higher levels of CRP in the Phase 2 study is small, the data suggest NP001 may have an effect in ALS patients with greater than average systemic inflammation and warrants further study of NP001 in a second Phase 2 study.