Voyager reports additional Phase Ib data for PD gene therapy

Voyager Therapeutics Inc. (NASDAQ:VYGR) reported updated data from a Phase Ib trial showing that Parkinson’s disease candidate VY-AADC01 led to durable, dose-dependent improvements across multiple measures of motor function. The trial enrolled 15 advanced PD patients with disabling motor fluctuations to receive 1 of 3 doses of VY-AADC01 in a single neurosurgical infusion delivered transfrontally into the putamen. VY-AADC01 was dosed at 7.5x10¹¹, 1.5x10¹² or 4.5x10¹² vector genomes (vg).

The trial's secondary endpoints evaluated multiple measures of motor function. At 12 months, mid-dose VY-AADC01 led to an increase in diary on-time without dyskinesia of 4 hours from baseline, and a reduction in diary off-time of 2.3 hours from baseline. Additionally, VY-AADC01 improved patients' activities of daily living as shown by mean reductions from baseline in Unified PD Rating Scale (UPDRS) Part II off medication scores of 3.4 points at 12 months in the mid-dose arm, and of 3.6 points at 6 months in the high-dose arm. Voyager also said VY-AADC01 led to dose-dependent improvements in UPDRS Part III motor function scores while patients were on their medication...