Voyager Therapeutics Reports Third Quarter 2021 Financial and Operating Results
License option agreement with Pfizer provides Voyager
TRACER platform identifies AAV9 and AAV5 capsid variants with broad CNS tropism and neuronal and glial cell targeting in non-human primates
Preclinical data demonstrate GBA1 gene therapy achieved widespread CNS distribution and sustained correction of GCase activity in multiple brain regions in mice
“We’ve taken notable steps in recent months to execute on our new strategy, including our recent capsid licensing agreement with Pfizer, continued progress with our RNA-driven AAV TRACER screening platform, and initial data from our GBA1 gene replacement program,” said
“We’re pleased to also announce that in addition to our prior results with AAV9 variants, our TRACER screening campaigns have identified AAV5 capsid variants with enhanced brain and spinal cord tropism, compared to conventional AAV5 and AAV9, as well as a new class of AAV9 variants selective for glial cells, which may enable more precise targeting of CNS diseases affecting non-neuronal cells,” said
Pfizer License Option Agreement Supports TRACER Platform Potential for Multiple Therapeutic Areas
- In October,
Voyagerand Pfizer Inc. (NYSE: PFE) entered an agreement through which Pfizer may exercise options to license novel capsids generated from Voyager’s RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) screening technology. Under the agreement, Pfizer may evaluate novel TRACER capsids and exercise options to license up to two capsids for transgene-exclusive Pfizer gene therapy programs for neurologic and cardiovascular disease associated with those transgenes.
- The Pfizer transgenes are distinct from those planned for Voyager’s internal pipeline, and Voyager retains global rights to all TRACER capsids for use with other transgenes and to all other applications of its TRACER technology.
- Voyager received a
$30 millionupfront payment from Pfizer and is entitled to receive up to $20 millionin exercise fees in aggregate for two options, exercisable by Pfizer within 12 months of signing. Voyager is also eligible to earn up to $580 millionin total development, regulatory, and commercial milestones associated with licensed products incorporating the two undisclosed Pfizer transgenes together with a Voyager licensed capsid. Voyager is also eligible to receive mid- to high-single-digit tiered royalties based on net sales of Pfizer’s products incorporating the licensed capsids.
TRACER Identifies AAV9 and AAV5 Capsid Variants with Broad CNS Tropism, Neuronal and Glial Cell Targeting; Screening Campaigns Advancing
- Voyager is performing further screening with its TRACER platform to identify additional proprietary AAV9- and AAV5-derived capsids targeting multiple tissue and cell types for use in gene therapies to treat a broad range of diseases.
- Numerous promising capsids have been identified from these screens, which are undergoing rigorous testing across multiple non-human primate and rodent species to evaluate their clinical translatability.
- In addition to capsids with substantially enhanced CNS tropism for neurons in non-human primates, a new array of TRACER AAV9 and AAV5 variants show robust CNS targeting in both non-human primates and rodents and demonstrate a strong tropism for glial cells, which may enable more effective targeting of certain CNS diseases.
- Capsid candidates derived from AAV5 present significant potential advantages in terms of lower prevalence of preexisting neutralizing antibodies in the general population and manufacturing. In addition, these variants demonstrate cross-species translatability among primates and rodents. Initial testing on these AAV5-derived capsids has demonstrated approximately 20-fold improvements in brain transduction compared with AAV9 in non-human primates.
- As previously disclosed, initial data from AAV9-derived TRACER capsids demonstrated robust delivery across the blood-brain barrier and widespread CNS transduction in non-human primates compared to AAV9 delivery following intravenous (IV) administration. In addition, a proprietary capsid displayed strong cardiac transduction and significant dorsal root ganglia detargeting in non-human primates, which may avoid dose-related toxicities associated with AAV9 delivery.
Positive Preclinical Data from GBA1 Program
- Voyager continues to advance its early-stage programs including multiple programs utilizing proprietary AAV capsids derived from the TRACER platform. Voyager believes these capsids may enable new, best-in-class gene therapy programs with systemic IV delivery and lower risk of dose-limiting toxicities. Pipeline programs include those for Huntington’s disease, monogenic ALS (SOD1), spinal muscular atrophy, and diseases linked to GBA1 mutations, including Parkinson’s disease, Lewy body dementia, and Gaucher disease.
- Voyager presented initial preclinical data from its GBA1 gene replacement program at the virtual 2021 Annual Congress of the
European Society of Gene & Cell Therapy, Oct. 19-22, 2021. The results demonstrated that an IV-administered gene replacement therapy using a proprietary AAV capsid achieved widespread distribution in the CNS and peripheral tissues, and raised levels of the deficient GCase enzyme by 300% - 660% over endogenous levels in a mouse model. By comparison, increases of 30% - 50% are expected to be clinically impactful. These data support the potential of a novel therapeutic approach for diseases associated with GBA mutations, including Parkinson’s disease, Lewy body dementia, and Gaucher disease.
Upcoming Events and Presentations
Society for Neuro-Oncology26th Annual Meeting, Nov. 18-21, 2021
Third Quarter 2021 Financial Results
- Collaboration Revenues: Collaboration revenue was
$1.5 millionfor the third quarter of 2021, compared to collaboration revenue of $117.8 millionfor the same period of 2020. The decrease in collaboration revenue was largely due to a reduction of revenue related to research services and cost reimbursements from the collaborations with Neurocrine and AbbVie. In February 2021, Neurocrine provided notice that effective August 2, 2021it was terminating its participation in the VY-AADC program for Parkinson’s disease under the collaboration agreement between Voyager and Neurocrine, and that wind-down activities, including the termination by Neurocrine of its support for ongoing development activities for the VY-AADC program, would commence immediately. Additionally, the collaborations with AbbVie were terminated in August 2020and resulted in recognition of $47.2 millionof revenue related to the collaboration targeting tauopathies and $59.4 millionof revenue related to the collaboration targeting synucleinopathies during the quarter ended September 30, 2020, as Voyager had no further service obligations at that time.
- Net Loss: Net loss was
$25.1 millionfor the third quarter of 2021, compared to net income of $85.6 millionfor the same period of 2020.
- R&D Expenses: Research and development expenses were
$17.9 millionfor the third quarter of 2021, compared to $25.0 millionfor the same period in 2020. The decrease in R&D expenses was primarily related to lower research and development employee-related expenses as well as manufacturing and clinical expenses for the VY-AADC program for Parkinson’s disease.
- G&A Expenses: General and administrative expenses were
$8.7 millionfor the third quarter of 2021, compared to $8.3 millionfor the same period in 2020. The increase in G&A expenses was primarily related to increased employee-related expenses in connection with a reduction in force and facility costs.
- Cash Position: Cash, cash equivalents, and marketable debt securities as of
September 30, 2021were $121.5 million.
- Based on Voyager’s current operating plan and excluding any potential financing or business development activities in 2021, Voyager anticipates cash, cash equivalents, and marketable debt securities will be greater than
$130 millionat the end of 2021.
About the TRACER™ AAV Capsid Discovery Platform
Voyager’s TRACER™ system is a broadly applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV capsids with enhanced tropisms and cell- and tissue-specific transduction properties in multiple species, including non-human primates (NHPs). Initial data from the first of many libraries screened in NHPs demonstrated the proprietary capsid variants effectively penetrated the blood-brain barrier and achieved widespread biodistribution and transduction of multiple regions of the brain. Separate results have demonstrated the ability of certain capsids to transduce cardiac muscle and to de-target the dorsal root ganglia. Voyager is proceeding with additional capsid campaigns derived from unique capsid serotypes to identify novel AAV vectors optimized for specific therapeutic applications.
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 Voyager’s ability to continue to identify and develop proprietary capsids from its TRACER AAV screening platform; Voyager’s ability to identify and develop proprietary capsids from its TRACER AAV screening platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV5 and AAV9 capsids; Voyager’s ability to utilize its novel proprietary capsids in its own product development programs; Voyager’s ability to attract parties to license its novel proprietary capsids or to participate with Voyager in research and development collaborations utilizing its novel proprietary capsid; Voyager’s ability to advance its AAV-based gene therapy programs; Voyager’s ability to perform its obligations under its license option agreement with Pfizer; Voyager’s entitlement to receive upfront, milestone and royalty based fees from Pfizer under the license option agreement; Voyager’s ability to maintain its current partnerships and collaborations and to enter into new partnerships or collaborations; and Voyager’s ability to generate sufficient cash resources to enable it to continue to identify and develop proprietary capsids from its TRACER AAV screening platform are forward looking.
All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements 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. Such risks and uncertainties include, among others, the severity and length of the COVID-19 health crisis, the imposition of governmental controls and guidance addressing the COVID health crisis, and the financial and human resources available to Voyager to manage the COVID-19 health crisis; the continued development of various technology platforms, including Voyager’s TRACER platform; Voyager’s scientific approach and general development progress; the ability to attract and retain talented contractors and employees, including key scientists and business leaders; the ability to create and protect intellectual property; the sufficiency of cash resources; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer license option agreement and other collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements on terms acceptable to Voyager and third parties; and the availability or commercial potential of Voyager’s product candidates.
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, except per share data)
|Three Months Ended||Nine Months Ended|
|Statement of Operations
|Research and development||17,914||25,039||59,767||86,757|
|General and administrative||8,714||8,277||28,895||26,721|
|Total operating expenses||26,628||33,316||88,662||113,478|
|Operating (loss) income||(25,146||)||84,527||(79,320||)||51,113|
|Total other income||9||1,084||2,414||1,554|
|Net (loss) income||$||(25,137||)||$||85,611||$||(76,906||)||$||52,667|
|Net (loss) income per share,
|Net (loss) income per share,
shares outstanding, basic
|Weighted-average common shares
|Selected Balance Sheet Items||2021||2020|
|Cash, cash equivalents, and marketable debt securities||$||121,499||$||174,782|
|Accounts payable and accrued expenses||$||11,916||$||14,839|
|Total stockholders’ equity||$||87,422||$||154,320|
Source: Voyager Therapeutics, Inc.