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. … 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. … 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.
