GM6: Phase IIa data

A double-blind, U.S. Phase IIa trial in 12 ALS patients showed that 6 doses of 320 mg IV GM604 given over 2 weeks significantly reduced total microtubule-associated protein tau (MAPT; tau; FTDP-17) from baseline to week 7 vs. placebo (p=0.0369). GM604 also significantly reduced TAR DNA binding protein 43 (Tdp-43; Tardbp) from baseline to week 12 vs. placebo (p=0.0078). Additionally, GM604 reduced superoxide dismutase 1 (SOD1) from baseline to week 2 vs. placebo (p=0.055). Furthermore, GM604 significantly reduced the decline in ALSFRS-R score vs. historical controls (p=0.0047) and 7 of 8 patients treated with GM604 had their ALS disease progression slowed or stopped at week 12. At week 12, mean FVC was reduced by 4.7% in GM604-treated patients vs. a reduction of 11.5% in placebo-treated patients and 5 of 7 patients treated with GM604 had their FVC disease progression slowed or reversed at week 12. GM604 was well tolerated with no deaths or withdrawals due to adverse events and no serious adverse events reported. Genervon said it submitted the data to FDA for guidance on the development pathway for GM604. ...