Voyager Therapeutics Announces Updates from Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease
January 20, 2017 at 12:31 pm
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Voyager Therapeutics, Inc. announced updates regarding the Phase 1b trial of VY-AADC01 for patients with advanced Parkinson’s disease. The five patients enrolled in Cohort 3 received similar infusion volumes of VY-AADC01 compared to Cohort 2 (up to 900 µL per putamen), but three-fold higher vector genome concentrations, representing up to a three-fold higher total dose of up to 4.5×1012 vector genomes (vg) of VY-AADC01 compared to patients in Cohort 2 (1.5 × 1012 vg). Patients enrolled in Cohort 3 were similar in baseline characteristics to Cohort 1 and 2. The use of real-time, intra-operative MRI-guided delivery allowed the surgical teams to visualize the delivery of VY-AADC01 and continue to achieve greater average coverage of the putamen in Cohort 3 (42%) compared to Cohort 2 (34%) with similar infusion volumes and Cohort 1 (21%) with a lower infusion volume. The surgical procedure was successfully completed in all five patients. Infusions of VY-AADC01 have been well-tolerated with no vector-related serious adverse events (SAEs) or surgical complications in Cohort 3, and all five patients were discharged from the hospital within two days following surgery. The Phase 1b trial remains on track to deliver six-month safety, motor function, and biomarker data from Cohort 3, as well as longer-term safety and motor function data from Cohorts 1 and 2, in mid-2017. Interim positive results from the Phase 1b trial from Cohorts 1 and 2 reported in early December 2016 demonstrated that VY-AADC01 dose-dependently improved measures of motor function and enhanced response to levodopa at six and twelve months and that administration of VY-AADC01 was well-tolerated. Since then, the Company has further analyzed the relationship between surgical coverage of the putamen with VY-AADC01 and enzyme activity of aromatic L-amino acid decarboxylase (AADC) that is responsible for converting levodopa to dopamine in the putamen using [18F] fluorodopa (or F-Dopa) positron emission tomography (PET). The results demonstrate that surgical coverage of the putamen was highly correlated with change in AADC enzyme activity measured by F-Dopa PET (r=0.81, p<0.05).
Voyager Therapeutics, Inc. is a biotechnology company focused on advancing neurogenetic medicines. The Companyâs pipeline includes programs for Alzheimerâs disease, amyotrophic lateral sclerosis (ALS), Parkinsonâs disease, and multiple other diseases of the central nervous system. Many of its programs are derived from its TRACER AAV capsid discovery platform, which is used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Its pipeline of programs, all of which are in preclinical development, include Anti-Tau Antibody (VY-TAU01), SOD1 Silencing Gene Therapy Program, Tau Silencing Gene Therapy Program, Vectorized Anti-Amyloid Antibody Early Research Program, Friedreichâs Ataxia Program: VY-FXN01, GBA1 Gene Replacement Program, HD Program, and others. VY-TAU01 is for the treatment of Alzheimerâs disease. SOD1 Silencing Gene Therapy Program is for the treatment of ALS.