VY-AADC01: Additional Ph Ib data

Additional data from 10 patients with advanced PD in cohorts 1 and 2 of an open-label, dose-escalation, U.S. Phase Ib trial showed that a single neurosurgical infusion of 7.5x10¹¹ vector genomes (vg) and 1.5x10¹² vg VY-AADC01 led to increases in putaminal AADC activity of 13% and 56%, respectively, from baseline to 6 months as measured by 18F-DOPA PET scans. Low- and high-dose VY-AADC01 also reduced patient-intake of daily PD medications, including levodopa, by 14% and 34%, respectively, at 6 months. Additionally, low- and high-dose VY-AADC01 reduced UPDRS Part III off medication scores by 42% and 50%, respectively, at 6 months and by 44% for both doses at 12 months. Furthermore, high-dose VY-AADC01 led to a 56% reduction in UPDRS Part III on medication score at 6 months vs. a 21% worsening in the low-dose cohort. High-dose VY-AADC01 also increased diary on-time with no dyskinesias or non-troublesome dyskinesias by 20% at 6 months and by 43% at 12 months, while low-dose VY-AADC01 reduced the endpoint by 3% at 6 months and increased the endpoint by 16% at 12 months. High-dose VY-AADC01 reduced diary off-time by 27% at 6 months and by 48% at 12 months, while low-dose VY-AADC01 reduced the endpoint by 16% and 27% at 6 and 12 months, respectively. VY-AADC01 was well tolerated with no vector-related serious adverse events reported. The trial is enrolling up to 20 patients. Voyager previously reported interim data from the trial (see BioCentury, July 18). The company plans to report additional data in mid-2017 from cohorts 1 and 2, plus cohort 3 in which up to 5 patients are receiving 4.5x10¹² vg VY-AADC01. In 4Q17, Voyager plans to start a placebo-controlled trial of VY-AADC01...