Looks like there will soon be an alternative to Rilutek. I hope this drug does well in phases 2 & 3 of the trail.
A double-blinded, placebo-controlled, Phase 1, single ascending dose study was performed to assess the safety and tolerability of four single intravenous doses of NP001 in a total 32 individuals with ALS. The primary objective of this study was the establishment of safety with patients being assessed at select time intervals for seven days post dosing. The secondary objective was to investigate the effects of NP001 on an ALS disease-associated blood biomarker pre- and 24 hours post dosing.
Single dose safety and tolerability of NP001 at four different dose levels was established in ALS patients. Importantly, following a single dose, there was a dose-dependent, statistically significant improvement in blood levels of the biomarker thought to be involved in ALS disease progression; this is a first in ALS.
Robert G. Miller, M.D., the study’s principal investigator and Medical and Program Director of the Forbes Norris MDA/ALS Research Center, commented, “NP001 appears to have affected the level of a biomarker directly related to ALS pathogenesis in a dose-dependent manner. This is extremely promising and is virtually unprecedented in ALS research. In addition, the safety profile of NP001 is very reassuring and gives us confidence going into the Phase 2 clinical study.”
[From Neuraltus Pharmaceuticals]
