Release Details
Voyager Therapeutics Reports Fourth Quarter and Full Year 2017 Financial Results
VY-AADC for advanced Parkinson’s disease on track for start of planned global, pivotal Phase 2-3 program during the middle of this year
Two IND filings expected from the ALS, Huntington’s disease and Friedreich’s ataxia programs for 2019
Recent
“Our strong performance in 2017 was underscored by advancing our lead program VY-AADC for advanced Parkinson’s disease including successfully manufacturing our gene therapy product candidate using our baculovirus/Sf9 cell process and commercial-scale GMP runs, completing our dose-ranging Phase 1b clinical trial as well as our posterior trajectory trial to select both an optimal dose and delivery protocol for the planned pivotal trial, and reporting longer-term Phase 1b data with clinically meaningful and durable responses with this one-time treatment,” said
2017 and Recent Key Pipeline and Corporate Highlights
- Announced updated results to the ongoing, dose-ranging Phase 1b program of VY-AADC for advanced Parkinson’s disease. At our mid-dose studied in Cohort 2 at 18 months, patients had a mean increase of approximately five hours a day of on-time without any dyskinesia and experienced 65% less off-time, making this the likely dose for the pivotal Phase 2-3 program.
- Investigational New Drug (IND) application cleared by the
Food and Drug Administration (FDA ) for VY-AADC inJanuary 2018 , allowing the Company to formally initiate clinical trial sites and screen and begin dosing patients for its pivotal Phase 2-3 program for advanced Parkinson’s disease. As part of this IND, the chemistry, manufacturing, and controls (CMC) section included data demonstrating comparability between VY-AADC produced under good manufacturing process (GMP) using Voyager’s baculovirus/Sf9 manufacturing process and VY-AADC produced using a mammalian cell system consisting of triple-transfection of human embryonic kidney (HEK293) cells similar to that used in Voyager’s Phase 1b clinical trial.
- Initiated a separate Phase 1 trial and successfully dosed seven patients with advanced Parkinson’s disease with a posterior (i.e., back of the head) infusion trajectory of VY-AADC. A posterior trajectory better aligns the infusion of VY-AADC with the anatomical structure of the putamen and resulted in higher total volume of coverage of the putamen and shorter total procedure time compared to Cohorts 1 through 3 from the ongoing Phase 1b trial that employed a transfrontal, or top of the head, delivery approach into the putamen.
- In
February 2018 ,FDA granted VY-AADC Fast Track designation for advanced Parkinson’s disease. Fast Track designation is granted for programs that demonstrate the potential to address an unmet medical need for a serious or life-threatening disease and is intended to facilitate development and expedited regulatory review, including the ability to submit completed sections of a Biologics License Application for a “rolling” review or submission.
- Gained worldwide development and commercial rights to VY-AADC for the treatment of advanced Parkinson’s disease. Voyager is on track to dose the first patient in its planned global, pivotal Phase 2-3 program during mid-2018.
- Entered into an exclusive strategic collaboration and option agreement with
AbbVie to develop and commercialize vectorized antibodies directed against tau for the treatment of Alzheimer’s disease and other neurodegenerative diseases, combining Voyager’s gene therapy platform with AbbVie’s monoclonal antibody expertise, global clinical development and commercial capabilities. The research period is underway for each company to identify up to five antibodies for inclusion in the collaboration. Voyager received$69 million upfront cash payment and is eligible to receive potentially up to$155 million in preclinical and Phase 1 option payments as well as development and regulatory milestone payments and royalties.
- Further strengthened the balance sheet with approximately
$127 million of additional capital raised from the upfront cash payment from theAbbVie collaboration and a follow-on public equity offering in November, extending Voyager’s expected ability to fund its operating expenses and capital expenditure requirements into early 2020.
- Announced plans during 2018 for
Steven Paul , M.D., to transition from president and chief executive officer to executive science advisor where he will focus on preclinical discovery research and portfolio development including progressing theAbbVie collaboration. In addition to the executive science advisor position, Dr. Paul will continue to serve on Voyager’s Board of Directors, and as a member of Voyager’s Science & Technology Committee.
- Strengthened the management team with the additions of
Matthew P. Ottmer as Chief Operating Officer andLuis Maranga , Ph.D. as Chief Technical Operations Officer. Mr. Ottmer brings to Voyager more than 18 years of biotechnology industry experience, including executive leadership of business operations, product development, and commercialization across multiple therapeutic areas and all stages of development. Dr. Maranga joins Voyager with over 20 years of biotechnology industry experience, focused on bioprocess development, CMC, GMP, process validation and regulatory submissions, and facilities management including extensive work with the baculovirus/Sf9 expression system.
Corporate Goals and 2018 Financial Guidance
Voyager is committed to becoming the leading gene therapy company focused on severe neurological diseases with expertise in discovery, development, manufacturing and commercialization of gene therapy products for people living with these devastating diseases. During 2018, Voyager plans to achieve the following corporate goals towards fulfilling this commitment:
- During the second quarter of this year, complete a Type C meeting with the
Office of Tissues and Advanced Therapies division of the FDA’sCenter for Biologics Evaluation and Research and incorporate feedback from this meeting into the Phase 2-3 pivotal program for VY-AADC for advanced Parkinson’s disease.
- During the second quarter of this year, provide six-month safety and motor function data from the Phase 1 trial of VY-AADC using the posterior infusion trajectory. Seven patients have been dosed using this trajectory as the likely preferred surgical approach for the Phase 2-3 pivotal program.
- During mid-2018, dose the first patient in the planned Phase 2-3 pivotal program for advanced Parkinson’s disease. Neurosurgical and neurology clinical trial sites have been identified and are being activated through institutional review board processes, after which, patient referral, screening and dosing can proceed.
- During the second half of 2018, provide longer-term safety, biomarker, motor function and quality of life data from Cohorts 1-3 and from patients in the posterior trajectory trial of VY-AADC for advanced Parkinson’s disease.
- Advance multiple preclinical programs towards clinical trials through further vector optimization and exploration of additional routes of administration, to support filing two IND applications from the ALS SOD1, Huntington’s disease, and Friedreich’s ataxia programs during 2019.
- Continue to identify, evaluate and progress collaborative business development opportunities for certain Voyager programs, technology platform capabilities, or both.
- Based on the Company’s current operating plan, Voyager expects to end 2018 with cash, cash equivalents and marketable debt securities of approximately
$125 million to $135 million , which includes the$69 million upfront payment fromAbbVie , and projects that its existing cash, cash equivalents and marketable debt securities will be sufficient to fund operating expenses and capital expenditure requirements into early 2020.
Fourth Quarter and Full Year 2017 Financial Results
For the fourth quarter and full year of 2017, 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 its CEO transition plans, 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 product engine, 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:
Pure Communications, Inc.
910-509-3977
Katie@purecommunicationsinc.com
Selected Financial Information ($-amounts in thousands, except per share data) (Unaudited) |
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Three Months Ended | Year Ended | ||||||||||||||||
December 31, | December 31, | ||||||||||||||||
Statement of Operations Items: | 2017 | 2016 | 2017 | 2016 | |||||||||||||
Collaboration revenue | $ | 6,345 | $ | 2,362 | $ | 10,135 | 14,220 | ||||||||||
Operating expenses: | |||||||||||||||||
Research and development | 13,327 | 12,723 | 62,260 | 42,249 | |||||||||||||
General and administrative | 5,366 | 3,481 | 19,738 | 13,270 | |||||||||||||
Total operating expenses | 18,693 | 16,204 | 81,998 | 55,519 | |||||||||||||
Operating loss | (12,348 | ) | (13,842 | ) | (71,863 | ) | (41,299 | ) | |||||||||
Total other income (loss) | 550 | (477 | ) | 1,165 | 1,158 | ||||||||||||
Loss before income taxes | (11,798 | ) | (14,319 | ) | (70,698 | ) | (40,141 | ) | |||||||||
Income tax (benefit) provision | (31 | ) | 355 | — | 52 | ||||||||||||
Net loss | $ | (11,767 | ) | $ | (14,674 | ) | $ | (70,698 | ) | $ | (40,193 | ) | |||||
Net loss per share, basic and diluted | $ | (0.40 | ) | $ | (0.57 | ) | $ | (2.64 | ) | $ | (1.59 | ) | |||||
Weighted-average common shares outstanding, basic and diluted | 29,281,071 | 25,526,843 | 26,803,711 | 25,302,414 | |||||||||||||
December 31, | |||||||
Selected Balance Sheet Items | 2017 | 2016 | |||||
Cash, cash equivalents, and marketable debt securities | $ | 169,052 | $ | 174,418 | |||
Total assets | $ | 184,477 | $ | 189,566 | |||
Accounts payable and accrued expenses | $ | 12,517 | $ | 7,038 | |||
Deferred revenue | $ | 31,560 | $ | 41,582 | |||
Total stockholders’ equity | $ | 134,051 | $ | 135,922 |