Clinical Data At 12 months post-dosing, patients in the first dose cohort (4.2 x 106 TU) demonstrated an average 22-point change from baseline in motor function as assessed by the UPDRS Part III “OFF” score, which represents a 37% improvement. Individual patient improvements were 24-points and 20-points, respectively. Previously, at 6 months post-dosing, these patients demonstrated an average 17-point change from baseline, or 29% improvement, on the same scale. The 12-month timepoint is considered an important timeframe for assessment of therapeutic response, differentiation from sham/placebo effect, and durability of gene therapy in Parkinson’s disease.
• AXO-Lenti-PD was observed to be well-tolerated with no serious adverse events attributable to the gene therapy.
• Improvement in the UPDRS Part III “OFF” score in the first cohort exhibited evidence of dose response when compared to the low (n=3), medium (n=6), and high (n=6) dose cohorts of ProSavin that were previously evaluated in a separate Phase 1/2 study at 12 months