Release Details
Voyager Therapeutics Announces Third Quarter 2018 Financial Results and Corporate Highlights
One-time treatment with VY-AADC demonstrates durable improvements in motor function at 18 months and beyond from ongoing Phase 1b trial
Recent preclinical results for Huntington’s disease and Amyotrophic Lateral Sclerosis programs support progress towards IND filings
“We made important progress advancing our innovative pipeline in the third quarter as we presented exciting new preclinical data with our Huntington’s disease and SOD1 ALS programs and today announced durable improvements in motor function at 18 months and beyond from the ongoing Phase 1b trial with VY-AADC in patients with Parkinson’s disease,” said
Recent highlights for VY-AADC for Parkinson’s disease:
- Today, we announced in a separate press release (link here) positive longer-term results from the open-label, dose-escalating Phase 1b trial of VY-AADC demonstrating sustained improvements in patients’ motor function. Patients in the two highest dose cohorts (Cohorts 3 and 2) experienced mean improvements in good ON time per day of 1.7 hours at 18 months and 2.7 hours at two years, respectively. Having selected a dose for the Phase 2 trial between the two highest dose cohorts from the Phase 1b trial, results from the combined ten patients in Cohorts 2 and 3 demonstrated an increase from baseline in good ON time of 2.4 hours per day at 12 months, the timepoint for the primary endpoint in the Phase 2 trial, and 2.6 hours per day at 18 months, the latest timepoint measured for both cohorts. These results were achieved with clinically meaningful and sustained reductions in daily oral levodopa and related medications at the two highest cohorts.
- As previously announced, in
July 2018 , Voyager received written Type C meeting feedback from theFood and Drug Administration (FDA ) indicating that the results from the Phase 2 randomized, placebo-controlled trial, if it were to meet its primary endpoint and in the absence of major safety concerns, likely may be considered sufficient for acceptance of submission for review of a BLA. In lateOctober 2018 , Voyager received an addendum from theFDA to theJuly 2018 written responses in which theFDA informed the Company that although the data from the Phase 2 trial may support the safety and efficacy of VY-AADC and could be considered in the BLA review, theFDA currently considers the Phase 2 trial as an early phase exploratory study. Voyager plans to engage with theFDA to gain further clarity on their most recent responses, including through a planned Type B meeting later this year and through the additional mechanisms available to the Company under the RMAT designation. Voyager plans to continue to seek and incorporateFDA guidance into the ongoing development plans for VY-AADC.
- 24 clinical trial sites (including neurosurgical and neurology patient referral and management sites) have been selected for participation in the Phase 2 randomized, placebo-controlled trial. Institutional review board approvals, site activation, and patient screening efforts are underway. Voyager expects to announce when the first patient has been dosed.
Recent highlights for preclinical programs:
At the
- For VY-HTT01, the use of a novel delivery paradigm targeting both the putamen and thalamus significantly reduced huntingtin, or HTT, gene expression in both deeper tissues (caudate, putamen, and thalamus) and outer layers (cortex) of the brain of adult, non-human primates. Targeting both the putamen and thalamus leverages more extensive and preserved neuronal pathways to the cortex than delivery to the putamen alone, offering the potential of a one-time treatment with VY-HTT01 to address motor, cognitive and behavioral disabilities associated with Huntington’s disease. Robust analyses including quantitative measurement in multiple tissue punches and in neurons captured by laser microdissection revealed that VY-HTT01 reduced HTT messenger RNA (mRNA) on average by 68% in the caudate, 67% in the putamen, 73% in the thalamus, and 32% in cortical neurons.
- For VY-SOD102 for amyotrophic lateral sclerosis (ALS) targeting SOD1, a novel delivery paradigm comprising a one-time, intraparenchymal infusion to the cervical region of the spinal cord significantly reduced SOD1 throughout the spinal cord of the mini-pig, which has a spinal cord similar in length and diameter to the human spinal cord. This novel delivery approach with VY-SOD102 reduced SOD1 mRNA in the spinal cord on average by 70% and 50% in the cervical and thoracic regions, respectively, both regions critical for respiratory function, and 82% near the site of cervical injection. In addition, VY-SOD102 reduced SOD1 mRNA by 22% in the lumbar region.
Voyager’s V-TAG™ Neuro-Navigational Surgical Device for Gene Therapy Delivery
Voyager has implemented the use of real-time, intra-operative magnetic resonance imaging (MRI) compatible devices and techniques allowing neurosurgeons more precise delivery of its gene therapy vectors. For the Phase 1b trial of VY-AADC for Parkinson’s disease, investigators used an MRI-compatible device called the ClearPoint® System from
Management Update
Voyager promoted
Third Quarter 2018 Financial Results
Voyager reported a GAAP net loss of
Collaboration revenues of
Research and development (R&D) expenses of
General and administrative (G&A) expenses of
Cash, cash equivalents, and marketable debt securities as of
Conference Call Information
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 laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “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 reporting of 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 initiate, enroll and complete, clinical trials, the potential clinical utility of its product candidates, its ability to continue to develop its gene therapy platform, its ability to develop manufacturing capability for its products and successfully transition its manufacturing process, its ability to perform under existing collaborations with, among others, Sanofi Genzyme and
Investor Relations:
Vice President of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
949-903-4750
sseapy@w2ogroup.com
Selected Financial Information ($-amounts in thousands, except per share data) (Unaudited) |
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Three Months Ended | Nine Months Ended | ||||||||||||||||
September 30, | September 30, | ||||||||||||||||
Statement of Operations Items: | 2018 | 2017 | 2018 | 2017 | |||||||||||||
Collaboration revenue | $ | 2,094 | $ | 1,148 | $ | 5,611 | $ | 3,790 | |||||||||
Operating expenses: | |||||||||||||||||
Research and development | 16,632 | 19,561 | 47,991 | 48,933 | |||||||||||||
General and administrative | 6,609 | 4,942 | 25,554 | 14,372 | |||||||||||||
Total operating expenses | 23,241 | 24,503 | 73,545 | 63,305 | |||||||||||||
Operating loss | (21,147 | ) | (23,355 | ) | (67,934 | ) | (59,515 | ) | |||||||||
Total other income | 858 | 9 | 1,997 | 615 | |||||||||||||
Loss before income taxes | (20,289 | ) | (23,346 | ) | (65,937 | ) | (58,900 | ) | |||||||||
Income tax benefit | — | — | 180 | 31 | |||||||||||||
Net loss | $ | (20,289 | ) | $ | (23,346 | ) | $ | (65,757 | ) | $ | (58,869 | ) | |||||
Net loss per share, basic and diluted | $ | (0.63 | ) | $ | (0.89 | ) | $ | (2.06 | ) | $ | (2.27 | ) | |||||
Weighted-average common shares outstanding, basic and diluted | 32,191,475 | 26,164,527 | 31,977,670 | 25,968,849 | |||||||||||||
September 30, | December 31, | ||||||
Selected Balance Sheet Items | 2018 | 2017 | |||||
Cash, cash equivalents, and marketable debt securities | $ | 179,601 | $ | 169,052 | |||
Total assets | $ | 197,064 | $ | 184,477 | |||
Accounts payable and accrued expenses | $ | 9,777 | $ | 12,517 | |||
Deferred revenue | $ | 115,054 | $ | 31,560 | |||
Total stockholders’ equity | $ | 65,588 | $ | 134,051 |