OREANDA-NEWS. Voyager Therapeutics, Inc., a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS), today reported its second quarter 2016 financial results and highlighted recent pipeline and corporate achievements, as well as expected upcoming milestones.

“Voyager’s mission to become the leading gene therapy company focused on severe diseases of the CNS further advanced this quarter with the progress of our lead program, VY-AADC01 for advanced Parkinson’s disease, and the progress of our additional programs in earlier stages of development,” said Steven Paul, MD, president and chief executive officer of Voyager Therapeutics.  “During the second quarter, VY-AADC01 was successfully administered at a second clinical trial site and we are in the process of opening more clinical trial sites.  In addition, our preclinical pipeline programs continue to advance with the selection of lead clinical candidates underway.”

Second Quarter Pipeline and Corporate Highlights

  • Completed enrollment in Cohort 2 in the Phase 1b clinical trial of VY-AADC01 in patients with advanced Parkinson’s disease and recently announced dosing of the first patient in the third cohort in this ongoing trial of up to 20 patients.  In Cohorts 1 and 2, patients received a single administration of VY-AADC01 at a total dose of up to 7.5?1011 vector genomes (vg) and 1.5?1012 vg, respectively.  Patients recently began enrolling in Cohort 3 and will receive up to a three-fold higher total dose (4.5?1012 vg) than Cohort 2.  A final, additional cohort (Cohort 4) could increase the total dose to approximately 6-fold higher than Cohort 2.  The Company remains on track to provide six-month data on safety, motor function, and biomarkers from patients in Cohort 1 and 2, as well as preliminary data from some patients in Cohort 3, by the end of this year and will report longer-term safety and clinical data from this trial next year as the program advances.
     
  • Presented interim surgical results from the Phase 1b study of VY-AADC01 in patients with advanced Parkinson’s disease on June 22 at the 20th International Congress of Parkinson’s Disease and Movement Disorders in Berlin, Germany.  The interim surgical data presented at the conference demonstrated the use of real-time, intra-operative MRI-guided delivery to allow the surgical teams to visualize the delivery of VY-AADC01, administer higher infusion volumes, and achieve greater coverage of the putamen, the brain region that the Company is targeting with its gene therapy program.  Increased infusion volumes of VY-AADC01 in Cohort 2 resulted in a higher average coverage of the overall putamen compared to Cohort 1 and substantially greater coverage of the putamen than has been achieved in previous gene therapy trials using a similar vector. 
     
  • Advanced multiple preclinical programs towards selection of lead clinical candidates by late 2016 or early 2017, with the goal of filing an Investigational New Drug (IND) application for VY-SOD101 for a monogenic form of ALS in the fourth quarter of 2017.
     
  • Announced two new preclinical programs at the Company’s R&D day in April; VY-TAU01 and VY-NAV01, which are focused on the molecular targets tau and Nav1.7, respectively.  Voyager owns worldwide rights to both programs.
     
    • VY-TAU01 is an adeno-associated virus vectorized version of an anti-tau monoclonal antibody for direct one-time delivery to the CNS.  VY-TAU01 could be a potential treatment for severe neurological disorders, such as frontotemporal dementia and Alzheimer’s disease.  Based on preclinical data, Voyager believes that this approach could achieve significantly higher levels of the therapeutic anti-tau antibody in the CNS when compared to the systemic administration of an antibody.
       
    • VY-NAV01 targets the knockdown of Nav1.7 in sensory neurons of the dorsal root ganglia as a potential one-time treatment of certain forms of severe, chronic pain. Such an approach may avoid the addictive potential associated with many current treatments for severe, chronic pain.

Second Quarter 2016 Financial Results and Guidance

For the second quarter ended June 30, 2016, Voyager reported a GAAP net loss of $9.3 million, or $0.37 per share, compared to a GAAP net loss of $6.7 million, or $5.94 per share, for the same period in 2015.

Research and development (R&D) expenses increased to $10.5 million for the second quarter ended June 30, 2016 compared to $6.5 million for the same period in 2015 primarily due to R&D, manufacturing, and personnel costs associated with Voyager’s advancing pipeline.

General and administrative (G&A) expenses increased to $2.9 million for the second quarter ended June 30, 2016 compared to $2.4 million for the same period in 2015 primarily due to G&A personnel costs to support Voyager’s growth, and expenses related to operating as a public company.

Voyager reported cash, cash equivalents and marketable securities of $204.0 million as of June 30, 2016.  Voyager continues to expect to end 2016 with cash, cash equivalents and marketable securities of $160.0 million and that its existing cash, cash equivalents and marketable securities will be sufficient to fund operating expenses and capital expenditure requirements into 2019.

About Voyager Therapeutics

Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the CNS.  Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector engineering and optimization, manufacturing and dosing and delivery techniques.  The Company’s pipeline is focused on severe CNS diseases in need of effective new therapies, including advanced Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, Huntington’s disease, spinal muscular atrophy (SMA), frontotemporal dementia, Alzheimer’s disease and severe, chronic pain.  Voyager has broad strategic collaborations with Sanofi Genzyme, the specialty care global business unit of Sanofi, and the University of Massachusetts Medical School.  Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager Therapeutics is headquartered in Cambridge, Massachusetts.