VY-AADC01 program for advanced Parkinson's disease on track for interim Phase 1b results in early DecemberPreclinical pipeline advances along with prioritization Recent licensing deal increases access to more novel AAV capsids CAMBRIDGE, Mass., Nov. 10, 2016 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today reported its third quarter 2016 financial results and highlighted recent pipeline and corporate achievements, as well as expected upcoming milestones. "Voyager has made significant progress this quarter with its lead and pipeline programs and business development activities," said Steven Paul, M.D., president and chief executive officer of Voyager Therapeutics. "During the third quarter, we continued to enroll patients in our ongoing, dose-ranging Phase 1b trial of VY-AADC01 in advanced Parkinson's disease. We remain on track to report top-line, interim safety and efficacy data from Cohorts 1 and 2 in early December. Our pipeline of AAV gene therapy programs continued to advance during the quarter, and we have expanded our portfolio of novel AAV capsids through a recent licensing agreement with the California Institute of Technology for capsids which have demonstrated in preclinical models markedly enhanced blood-brain barrier penetration for the potential treatment of CNS diseases." Recent Highlights and UpdatesVY-AADC01 for Advanced Parkinson's Disease In August, investigators dosed the first patient in the third cohort of the ongoing Phase 1b open-label trial of VY-AADC01 in advanced Parkinson's disease. Patients enrolling in Cohort 3 (up to five patients) will receive a single administration of VY-AADC01 at a total dose of up to 4.5×10 12 vector genomes (vg), representing a three-fold higher total dose than patients in Cohort 2 (1.5 × 10 12 vg). Voyager plans to complete Cohort 3 enrollment by early 2017 and remains on track to report six-month data from this cohort in mid-2017. Voyager also remains on track to provide six-month safety, motor function, and biomarker data from patients in Cohort 1 and 2 in early December of this year. Clinical data from Cohorts 1-3 will help inform the design of the placebo-controlled (sham surgery) trial of VY-AADC01 in advanced Parkinson's disease planned to begin in the fourth quarter of 2017. The goals of the current Phase 1b trial are to assess the safety, biomarker activity, and relevant clinical measures across ascending doses of VY-AADC01 as well as to optimize vector delivery. Cohorts 1-3 employ a transfrontal (i.e., top of the head) trajectory of VY-AADC01 into the putamen. To further optimize delivery, a study exploring a posterior (i.e., back of the head) trajectory is planned. A posterior trajectory aligns the infusion of VY-AADC01 with the anatomical structure of the putamen. Voyager believes this will result in a higher total volume of coverage of the putamen and a higher total dose of VY-AADC01, up to 9.4×10 12 vg, representing a two-fold higher total dose than patients in Cohort 3 and a six-fold higher total dose than patients in Cohort 2. Voyager is activating additional clinical trial sites this quarter and plans to dose the first patient with this trajectory in the first quarter of 2017. Data from this trial will also help inform the design of the placebo-controlled trial planned to begin in the fourth quarter of 2017. Preclinical Pipeline Update During the quarter, Voyager continued to advance its multiple preclinical programs towards selection of lead clinical candidates with the goal of filing an Investigational New Drug (IND) application for VY-SOD101 for a monogenic form of amyotrophic lateral sclerosis (ALS) in the fourth quarter of 2017. Given the progress of VY-SOD101, VY-FXN01 for Friedreich's ataxia, and VY-HTT01 for Huntington's disease, Voyager is prioritizing and allocating its resources towards these and its other pipeline programs and deprioritizing VY-SMN101 for the treatment of spinal muscular atrophy (SMA).