Release Details
Voyager Therapeutics Provides First Quarter 2016 Investor Update
Enrollment Completed in 2nd Cohort of Ongoing Phase 1b Clinical Trial of VY-AADC01 in Advanced Parkinson’s Disease
VY-AADC01 Interim Phase 1b Results to be Presented at 20th International
“Voyager is off to a great start in 2016. Today, we announced the completion of enrollment of the second patient cohort in the ongoing Phase 1b trial of VY-AADC01. We will present interim results, specifically surgical coverage data for the putamen from cohorts 1 and 2, as well as safety data, at a major medical conference next month. Importantly, we remain on track to deliver top-line, proof-of-concept clinical results for VY-AADC01 in the fourth quarter of this year,” said
VY-AADC01 Updates
- Voyager completed enrollment of the second cohort (five patients) in the ongoing Phase 1b dose-ranging safety clinical trial of VY-AADC01 in patients with advanced Parkinson’s disease. The first patient was dosed at the second clinical trial site at the University of
Pittsburgh, Pennsylvania . The initial clinical trial site at theUniversity of California, San Francisco, California , continues to enroll patients. - In
June 2016 , the Company will present surgical coverage and safety data from the first 10 patients (cohorts 1 and 2) enrolled in this trial at the 20th InternationalCongress of Parkinson’s Disease & Movement Disorders being held inBerlin, Germany June 19-23, 2016 . - Voyager remains on track to report top-line, human proof-of-concept data in the fourth quarter of 2016. These results will include six-month follow up clinical efficacy, biomarker and safety data from cohorts 1 and 2.
Recent Pipeline & Corporate Highlights
- In
April 2016 , Voyager announced two new preclinical pipeline programs, VY-TAU01 and VY-NAV01, which are focused on the molecular targets tau and Nav1.7, respectively.- Voyager is developing an AAV vectorized version of an anti-tau monoclonal antibody, VY-TAU01, 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 is targeting the knockdown of Nav1.7 in the sensory neurons of the spinal cord 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.
- Voyager remains on track to file an Investigational New Drug (IND) application in late 2017 for its next most advanced pipeline program, VY-SOD101 for a monogenic form of ALS.
- Voyager expanded its leadership team with the appointment of
Jim Olesen , Ph.D., as vice president of intellectual property and legal affairs, andMatt Osborne as head of investor relations and corporate communications.
Upcoming Events and Presentations
- Piper Jaffray GenomeRx Symposium,
New York City ,May 17 th UBS Global Healthcare Conference ,New York City ,May 23 rdJefferies Healthcare Conference ,New York City ,June 7 th- 20th International
Congress of Parkinson’s Disease & Movement Disorders,Berlin, Germany ,June 22 nd
First Quarter 2016 Financial Results and Guidance
For the first quarter ended
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Cash, cash equivalents and marketable securities as of
Based on the Company’s current operating plan, Voyager continues to expect to end 2016 with cash, cash equivalents and marketable securities of approximately
Conference Call
Voyager will host a conference call and webcast today at
About
Forward-Looking Statements
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities law. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress and results of its preclinical programs and clinical trials and its research and development programs, its ability to advance its AAV-based gene therapies into, and successfully complete, clinical trials, its ability to continue to develop its product engine, its ability to add new programs to its pipeline, its expected cash, cash equivalents and marketable securities at the end of the fiscal year and anticipation for how long expected cash, cash equivalents and marketable securities will last, and the timing or likelihood of its regulatory filings and approvals, are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the
Selected Financial Information | |||||||||||
(amounts in thousands) | |||||||||||
(Unaudited) | |||||||||||
Three Months Ended | |||||||||||
March 31, | |||||||||||
Statement of Operations Items: | 2016 | 2015 | |||||||||
Collaboration revenue | $ | 4,830 | $ | 2,576 | |||||||
Operating expenses: | |||||||||||
Research and development | 8,732 | 5,523 | |||||||||
General and administrative | 3,565 | 1,881 | |||||||||
Total operating expenses | 12,297 | 7,404 | |||||||||
Operating loss | (7,467 | ) | (4,828 | ) | |||||||
Total other income (expense) | 279 | (9,749 | ) | ||||||||
Net loss | (7,188 | ) | (14,577 | ) | |||||||
GAAP charges related to pre-IPO preferred stock | - | (1,236 | ) | ||||||||
Net loss attributable to common stockholders | $ | (7,188 | ) | $ | (15,813 | ) | |||||
Selected Balance Sheet Items: | March 31, | December 31, | |||
2016 | 2015 | ||||
Cash, cash equivalents and marketable securities | $ | 213,981 | $ | 224,345 | |
Total assets | $ | 219,980 | $ | 229,457 | |
Accounts payable and accrued expenses | $ | 4,358 | $ | 4,042 | |
Deferred revenue | $ | 50,633 | $ | 54,982 | |
Total stockholders' equity | $ | 163,597 | $ | 169,074 |
Investor Relations:Matt Osborne Head of Investor Relations & Corporate Communications 857-259-5353 mosborne@vygr.com Media:Katie Engleman Pure Communications, Inc. 910-509-3977 Katie@purecommunicationsinc.com