In vivo study of PLL001 in ALS model SODG93A mice model (Charles River study C0270120)

The safety of PLL001 was evaluated in vivo in SOD1 mice treated or. This study was carried out in 60 mice split 4 groups (WT vehicle group1, SOD1 vehicle group 2, SOD1 Low Dose group 3 and SOD1 High Dose group 4). The low dose was estimated to be equivalent of the foreseen clinical dose and the high dose was 10 folds higher.

PLL001 DP01R and DP02R was administered subcutaneously to G93A-SOD1 mice twice daily (BID) for 28-30 days at the high dose or for 74-106 days at the low dose in vehicle (sterile water). Control wild type and transgenic G93A-SOD1 mice were administered the vehicle BID for 105-106 days or 73-98 days, respectively.

The major conclusions of the Charles rivers studies revealed no relevant safety risk difference (behavioral, clinical, biochemical, ophthalmoscopy and histopathologic analyses) between the SOD1 non treated and the SOD1 treated mice regardless of their sex except for local tolerance with the high dose.