Release Details
Voyager Therapeutics Announces Second Quarter 2018 Financial Results and Corporate Highlights
Clinical data updates and regulatory feedback for VY-AADC during the quarter support planned pivotal program for Parkinson’s disease
Pipeline programs progressing with updated preclinical data from ALS and Huntington’s disease programs expected during the fourth quarter of this year
Conference call scheduled for today at
“The significant progress made during the second quarter with our lead program VY-AADC for Parkinson’s disease and our pipeline programs positions Voyager to potentially achieve important milestones for the remainder of this year and into 2019,” said
Recent Clinical and Preclinical Program Highlights
VY-AADC for Parkinson’s disease:
- VY-AADC granted Regenerative Medicine Advanced Therapy (RMAT) designation from the
U.S. Food and Drug Administration (FDA ) for Parkinson’s disease in patients with motor fluctuations that are refractory to medical management. RMAT designation is an expedited program for the advancement and approval of regenerative medicine products, including gene therapy products. The designation includes all the benefits of the FDA’s Fast Track and Breakthrough Therapy designation programs, with the ability for early interactions with the agency to discuss any surrogate or intermediate endpoints to support the potential acceleration of approval and satisfy post-approval requirements. RMAT designation for VY-AADC was granted based on clinical data from the ongoing Phase 1b trial with VY-AADC in patients with Parkinson’s disease.
- Based on written feedback from a Type C meeting with the
FDA , Voyager announced plans to submit for review a biologics license application (BLA) for VY-AADC based on the nonclinical and clinical safety and efficacy data from the pivotal program, including plans to submit a BLA based on data from the randomized, placebo-controlled Phase 2 trial alone, or if needed, from the randomized, placebo-controlled Phase 3 trial.
- Achieved positive interim results from a Phase 1 posterior trajectory trial that achieved enhanced coverage of the putamen, reduced surgical times, and improvements in patients’ motor function at six months that were consistent with improvements achieved from patients in Cohorts 2 and 3 at the same time point in Voyager’s Phase 1b trial with VY-AADC. The posterior approach will serve as the preferred surgical route of administration for the pivotal program. Voyager plans to present data from the full cohort of eight patients from the Phase 1 posterior trajectory trial at future scientific and medical conferences.
- Pivotal program progressing with twenty-four clinical trial sites (including neurosurgical and neurology patient referral sites) selected for participation in the Phase 2 randomized, placebo-controlled trial with institutional review board submission and site activation underway. During the remainder of the year, Voyager plans to provide updates on the enrollment status of this trial.
Preclinical programs for ALS SOD-1, Huntington’s disease, Friedreich’s ataxia and tau for Alzheimer’s and other neurodegenerative diseases:
- During the second quarter, Voyager continued to advance its multiple preclinical programs towards clinical trials through further vector optimization and exploration of optimal routes of administration. At the
American Society of Gene and Cell Therapy (ASGCT) May 16-19, 2018 , inChicago, Ill. , Voyager presented results for VY-SOD101, which targets the superoxide dismutase 1 gene (SOD1), the first mutant gene discovered to be causal for the development of amyotrophic lateral sclerosis (ALS). The results demonstrated that a one-time administration of VY-SOD101 lowered SOD1 mRNA levels by 78% in the spinal cord motor neurons of non-human primates. Additional new data at this year’s ASGCT meeting included tolerability data in non-human primates for VY-HTT01 for Huntington’s disease, along with previous data with VY-HTT01 that demonstrated a 54% suppression of huntingtin (HTT) mRNA in the non-human primate putamen after a single administration. Efforts to further optimize delivery for ALS and Huntington’s disease programs are underway, and Voyager plans to provide results from these efforts at scientific conferences during the fourth quarter of this year.
Management Updates
Voyager recently announced the appointment of
Voyager today announced the resignation of
“Since joining Voyager over four and a half years ago, Bernard played an integral role in advancing the Parkinson’s disease program and in leading our medical activities,” said
Second 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
Selected Financial Information | ||||||||||||||||
($-amounts in thousands, except per share data) | ||||||||||||||||
(Unaudited) | ||||||||||||||||
Three Months Ended | Six Months Ended | |||||||||||||||
June 30 | June 30 | |||||||||||||||
Statement of Operations Items: | 2018 | 2017 | 2018 | 2017 | ||||||||||||
Collaboration revenue | $ | 2,575 | $ | 1,177 | $ | 3,517 | $ | 2,642 | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 16,507 | 15,300 | 31,360 | 29,372 | ||||||||||||
General and administrative | 11,762 | 4,516 | 18,945 | 9,430 | ||||||||||||
Total operating expenses | 28,269 | 19,816 | 50,305 | 38,802 | ||||||||||||
Operating loss | (25,694 | ) | (18,639 | ) | (46,788 | ) | (36,160 | ) | ||||||||
Total other income (expense) | 153 | (42 | ) | 1,140 | 606 | |||||||||||
Loss before income taxes | (25,541 | ) | (18,681 | ) | (45,648 | ) | (35,554 | ) | ||||||||
Income tax (provision) benefit | — | (195 | ) | 180 | 31 | |||||||||||
Net loss | $ | (25,541 | ) | $ | (18,876 | ) | $ | (45,468 | ) | $ | (35,523 | ) | ||||
Net loss per share, basic and diluted | $ | (0.80 | ) | $ | (0.73 | ) | $ | (1.43 | ) | $ | (1.37 | ) | ||||
Weighted-average common shares outstanding, basic and diluted | 31,976,922 | 25,946,333 | 31,868,995 | 25,869,390 |
June 30, | December 31, | |||||
Selected Balance Sheet Items | 2018 | 2017 | ||||
Cash, cash equivalents, and marketable debt securities | $ | 196,983 | $ | 169,052 | ||
Total assets | $ | 214,663 | $ | 184,477 | ||
Accounts payable and accrued expenses | $ | 9,255 | $ | 12,517 | ||
Deferred revenue | $ | 117,148 | $ | 31,560 | ||
Total stockholders’ equity | $ | 81,972 | $ | 134,051 |
Investor Relations:Matt Osborne Vice President of Investor Relations & Corporate Communications 857-259-5353 mosborne@vygr.com Media:Josie Butler W2O/Pure Communications, Inc. 910-239-5099 jbutler@w2ogroup.com