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Voyager Therapeutics Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Highlights
Initiated RESTORE-1 Phase 2 trial for VY-AADC for Parkinson’s disease
Began IND-enabling preclinical studies for ALS-SOD1 and Huntington’s disease programs
Recently announced strategic collaborations with funding for certain programs and significant upfront and potential near-term payments while retaining commercial optionality and allowing further investment in fully-retained pipeline
“Since the beginning of 2018, we advanced our lead and pipeline programs, further developed our vectorized antibody platform, and entered into key enabling collaborations,” said
2018 and Recent Corporate Highlights
- Entered into a strategic development and commercialization collaboration with
Neurocrine Biosciencesfor VY-AADC for Parkinson’s disease, for VY-FXN01 for Friedreich’s ataxia and two additional programs to be determined.
- Formed two separate, exclusive, global strategic collaborations with
AbbVieto develop and commercialize vectorized antibodies. The first collaboration is directed against tau for the potential treatment of Alzheimer’s disease and other tauopathies. The second collaboration targets pathological species of alpha-synuclein for the potential treatment of Parkinson’s disease and other diseases characterized by the abnormal accumulation of misfolded alpha-synuclein protein (synucleinopathies).
- Expect to strengthen the balance sheet with approximately
$230 millionof additional capital from the upfront cash payments from the recent AbbVieand Neurocrine collaborations, including $50 millionfrom the sale of shares of Voyager’s common stock to Neurocrine. In addition to the upfront payments, the AbbViecollaborations potentially include up to $460 millionin preclinical and Phase 1 option payments as well as potential development, regulatory, and commercial milestone payments. For the Neurocrine collaboration for VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, Voyager will receive reimbursement of all development costs until the programs reach the point of Voyager’s option to co-commercialize in the U.S. under a cost- and profit-sharing arrangement or grant Neurocrine full U.S. commercial rights in exchange for milestone payments and royalties.
- Appointed new members to the executive management team including
Andre Turenneas Chief Executive Officer, Omar Khwaja, M.D., Ph.D., as Chief Medical Officer, Robert Hessleinas General Counsel, and Allison Dorvalas Chief Financial Officer.
- Enhanced the engagement with
The Michael J. Fox Foundationfor Parkinson’s Research (MJFF) to include participation in the Parkinson’s Progression Markers Initiative (PPMI) and the Parkinson’s Disease Education Consortium(PDEC), expanding Voyager’s commitment to the Parkinson’s disease community by supporting high quality educational programs and ongoing research.
- Entered into strategic manufacturing collaborations with
Brammer Bioand Fujifilm Diosynth Biotechnologies to provide Voyager the ability and flexibility to transfer its manufacturing expertise across multiple high-quality vendors to support the development of its pipeline programs and increase capacity and scale of its gene therapies.
2018 and Recent Program Highlights
- Initiated RESTORE-1, a Phase 2, randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of VY-AADC for the treatment of Parkinson’s disease in patients with motor fluctuations that are refractory to medical management. RESTORE-1 and a planned Phase 3 trial (RESTORE-2) of similar size and design to RESTORE-1 incorporate guidance from the
FDAfrom the Type B meeting to conduct two adequate and well-controlled clinical trials for a large patient population such as Parkinson’s disease.
- Announced positive longer-term results from the Phase 1b open-label, dose-escalating trial of VY-AADC. The combined seven of 10 patients in Cohorts 2 and 3 who would have met the eligibility criteria for the RESTORE-1 Phase 2 trial demonstrated a 2.8-hour and 2.5-hour (mean) improvement in good ON time at 12 months and 18 months, respectively. These results were achieved with clinically meaningful and sustained reductions in daily oral levodopa and related medications.
- Presented preclinical data for VY-HTT01 for Huntington’s disease and VY-SOD102 for Amyotrophic Lateral Sclerosis (ALS) programs at the
Congressof the European Society of Gene and Cell Therapy. One-time delivery of VY-HTT01 generated significant reduction of HTT gene expression in deeper tissues and outer layers of the brain of non-human primates. One-time delivery of VY-SOD102 generated significant reduction of SOD1 gene expression in multiple locations throughout a large animal spinal cord, including an 82% reduction in cervical motor neurons near the injection site.
- Received 510(k) regulatory clearance of V-TAG™ from the
Center for Devices and Radiological Healthof the FDA. Voyager developed the Variable Trajectory Array Guide, or V-TAG™, a real-time, intra-operative, MRI-compatible neuro-navigational device, as a choice for neurosurgeons in addition to the ClearPoint® System from MRI Interventions, Inc.Beyond serving as an additional choice for neurosurgeons in the Parkinson’s disease program, V-TAG™ could also be used for Voyager’s Huntington’s disease and other programs.
Corporate Goals and 2019 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 diseases. During 2019, Voyager plans to achieve the following corporate and financial goals towards fulfilling this commitment:
- During the second quarter of 2019, provide 12-month safety, biomarker and motor function data from the open-label Phase 1 trial of VY-AADC in patients with Parkinson’s disease exploring the posterior (i.e., back of the head) infusion trajectory. The posterior trajectory better aligns the infusion of VY-AADC with the anatomical structure of the putamen and is the recommended infusion trajectory for the RESTORE-1, Phase 2 trial. During 2019, provide longer-term safety, biomarker, motor function and quality of life data from Cohorts 1-3 from the ongoing, open-label, Phase 1b trial of VY-AADC for Parkinson’s disease.
- Complete activation of neurosurgical and neurology patient referral and management trial sites and continue to enroll patients in the RESTORE-1 Phase 2, randomized, placebo-controlled trial of VY-AADC for Parkinson’s disease.
- Advance VY-HTT01 for Huntington’s disease and VY-SOD102 for ALS-SOD1 towards clinical trials. Preclinical pharmacology and toxicology studies are underway to support potential filings of IND applications for both programs later this year.
- Advance Friedreich’s ataxia program VY-FXN01 towards identifying a lead candidate and IND-enabling preclinical pharmacology and toxicology studies.
- Continue to identify, evaluate and progress business development opportunities and continue to invest in the discovery programs, vectorized antibody platform and new capsid development.
- Based on the Company’s current operating plan, Voyager expects to end 2019 with cash, cash equivalents and marketable debt securities of approximately
$280 million to $290 million. This includes the anticipated upfront payment of $165 millionand expected reimbursement of development costs from the Neurocrine collaboration, as well as the $65 millionupfront payment from the recently announced AbbViecollaboration. Operating expenses in 2019 are expected to range from $130 million to $140 million, including amounts reimbursable under the collaborations. Voyager projects that its cash, cash equivalents and marketable debt securities, including amounts to be received under the recently announced Neurocrine and AbbViecollaborations, will be sufficient to fund operating expenses and capital expenditure requirements into mid-2022.
Fourth Quarter and Full Year 2018 Financial Results
For the fourth quarter and full year of 2018, Voyager reported:
A GAAP net loss of
Collaboration revenues of
Research and development (R&D) expenses of
General and administrative (G&A) expenses of
Cash, cash equivalents, and marketable debt securities as of
Conference Call Information
Voyager will host a conference call and webcast today at
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding the initiation, timing, progress, activities, goals and reporting of results of its preclinical programs and clinical trials and its research and development programs, the potential benefits and future operation of the collaboration agreements with
Selected Financial Information
($-amounts in thousands, except per share data)
|Three Months Ended||Year Ended|
|December 31,||December 31,|
|Statement of Operations Items:||2018||2017||2018||2017|
|Research and development||16,914||13,327||64,905||62,260|
|General and administrative||8,255||5,366||33,809||19,738|
|Total operating expenses||25,169||18,693||98,714||81,998|
|Total other income||629||549||2,627||1,165|
|Loss before income taxes||(22,532||)||(11,798||)||(88,468||)||(70,698||)|
|Income tax provision (benefit)||—||30||(180||)||—|
|Net loss per share, basic and diluted||$||(0.70||)||$||(0.40||)||$||(2.75||)||$||(2.64||)|
|Weighted-average common shares outstanding, basic and diluted||32,327,241||29,281,071||32,065,781||26,803,711|
|Selected Balance Sheet Items||2018||2017|
|Cash, cash equivalents, and marketable debt securities||$||155,806||$||169,052|
|Accounts payable and accrued expenses||$||10,826||$||12,517|
|Total stockholders’ equity||$||46,446||$||134,051|
Matt OsborneVice President of Corporate Affairs, Communications and Investor Relations 857-259-5353 email@example.com Voyager Media: Sheryl SeapyW2Opure 949-903-4750 firstname.lastname@example.org