NP001: Phase II data

Neuraltus plans to start Phase III testing of NP001 to treat ALS in 2H13 despite reporting top-line data from a double-blind, U.S. Phase II trial in 136 patients with ALS showing that NP001 missed the primary and secondary endpoints vs. placebo. Neuraltus said high-dose NP001 non-significantly slowed the rate of disease progression as measured by the change in slope of ALSFRS-R, the primary endpoint, by 13% vs. the concurrent placebo group and by 19% when matched historical placebo control data were included with concurrent placebo control data. Neuraltus also said there were "trends of clinical benefit" with high-dose NP001 vs. placebo on the secondary endpoints of change in ALSFRS-R score from baseline to 6 months and a joint rank analysis of change of ALSFRS-R score adjusted for mortality. NP001 was well tolerated. Patients received placebo or 1 or 2 mg/kg IV NP001 for 5 consecutive days in month 1 and then for 4 consecutive days each month for 5 months with a 6-month follow-up period. ...