Voyager Therapeutics Reports Fourth Quarter and Full Year 2021 Financial and Operating Results
License option agreement with Novartis for target-specific access to TRACER™ next-generation AAV capsids for potential use with three CNS targets plus options to access capsids for two additional targets
Novartis agreement marks second recent major transaction to leverage Voyager’s TRACER technology following
Strengthened leadership with appointment of
Cash runway extended into 2024 with
Conference call and webcast today at
“We’re extremely pleased with the value created by our TRACER™ capsid discovery platform, evidenced by the two major license option agreements with Novartis and
Target-Specific License Option Agreement with Novartis Expands Applications of TRACER Capsids
- Voyager today announced a license option agreement with
Novartis AG(NYSE: NVS) through which Novartis may exercise options to license novel AAV capsids generated from Voyager’s RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform for potential use with three undisclosed CNS targets and options to access capsids for two additional targets to be agreed on in the future, subject to availability. These targets are distinct from those being explored in Voyager’s internal pipeline and other licensing agreements. Voyager retains global rights to its TRACER discovery platform and all capsids arising from it, subject to certain obligations under its agreement with Pfizer, as detailed below.
- Voyager will receive
$54 millionup front and is entitled to receive up to $37.5 millionin exercise fees for three initial CNS targets, exercisable by Novartis within 12 months of signing. In addition, Novartis may elect to evaluate capsids for up to two additional targets, to be agreed upon in the future, subject to availability, for $18 millionupon selection of each target, and a $12.5 millionexercise fee for selection of a capsid for each target. Voyager is also eligible to earn up to $1.5 billionin total development, regulatory, and commercial milestones for products utilizing Voyager-licensed capsids, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.
Transgene-Specific License Option Agreement with Pfizer Supports CNS and Cardiac Applications for TRACER
- In October,
Voyagerand Pfizer Inc.(NYSE: PFE) announced a license option agreement through which Pfizermay exercise options to license AAV capsids generated from the TRACER platform. Under the terms of the agreement, Pfizerreceived target-specific access to Voyager’s TRACER capsids for one CNS and one cardiovascular target and the right to exercise options to license capsids for exclusive use in Pfizer’s development of AAV gene therapies for each target. Voyager retains global rights to all TRACER capsids for use with other targets and to all other applications of its TRACER technology.
- Voyager received
$30 millionupfront and is entitled to receive up to $20 millionin exercise fees in aggregate for two options exercisable by Pfizerwithin 12 months of signing, and is eligible to earn up to $580 millionin total development, regulatory, and commercial milestones, as well as mid- to high-single-digit tiered royalties based on net sales of Pfizerproducts incorporating the licensed capsids.
Additional Data from Evolving TRACER AAV9 and AAV5 Capsid Portfolio Planned for
- Voyager continues to perform screening campaigns with its TRACER capsid discovery platform to identify additional proprietary AAV9- and AAV5-derived capsids and to refine capsids already identified to target multiple tissue and cell types for use in gene therapies to treat a broad range of diseases. Numerous promising capsids have been identified and developed from these screens, which are undergoing testing across multiple non-human primate (NHP) and rodent species to evaluate their clinical translatability.
- Voyager previously presented data on a set of AAV9-derived TRACER capsids from its first campaign that demonstrated robust delivery across the blood-brain barrier and widespread CNS transduction in NHPs, compared to conventional AAV9 delivery, when dosed intravenously. In addition, another AAV9-derived TRACER capsid displayed strong cardiac transduction and significant dorsal root ganglia detargeting in NHPs, which may help avoid dose-related toxicities associated with AAV9 delivery.
- Voyager has identified a new array of TRACER AAV9 and AAV5 variants that have demonstrated improved CNS targeting in both NHPs and rodents with certain AAV TRACER capsids showing a preferential tropism for glial cells, which may enable more effective targeting of relevant cell types for certain CNS diseases. AAV5 capsids have been shown to have a low prevalence of preexisting neutralizing antibodies in the general population and significant manufacturing advantages over conventional AAV9. In addition, some of these variants have demonstrated cross-species translatability across primates and rodents.
- Voyager plans to present preclinical data on the performance of these TRACER capsids in NHP and mouse models at a spring medical conference.
Positive Preclinical Data from Vectorized Anti-HER2 Antibody Program Highlights Advancement of Rich Early-Stage Pipeline
- Voyager continues to advance its preclinical programs powered by its TRACER capsids. The Company believes these capsids may enable new, best-in-class gene therapy programs with systemic IV delivery with lower risk of dose-limiting toxicities. Our pipeline includes gene replacement programs for spinal muscular atrophy, diseases linked to GBA1 mutations (including Parkinson’s disease, Lewy body dementia, and Gaucher disease), gene knockdown or silencing programs for SOD1 ALS and Huntington’s disease, and vectorized antibody programs for HER2+ brain metastases and diseases associated with pathological tau (including Alzheimer’s disease, frontal-temporal dementia, and progressive supranuclear palsy). Voyager plans to present further preclinical data from select programs at a spring medical conference.
- In November, the Company presented positive preclinical data demonstrating that IV dosing of a novel TRACER AAV vector encoding an anti-HER2 antibody payload prevented tumor growth in mouse models of HER2+ brain metastases. This innovative, intravenously delivered vectorized antibody approach is designed to activate the innate immune system, destroy metastatic breast cancer tumors, and inhibit tumor progression in the CNS. In preclinical studies, widespread transduction of brain tissue was achieved, effectuating expression of HER2-directed antibodies for extended periods, resulting in substantially reduced tumor growth in multiple orthotopic xenograft models.
Key Leadership Appointments Add to Deep R&D and Operational Experience
- In February, Voyager announced the appointment of
Alfred W. Sandrock, Jr., M.D., Ph.D., to its Board of Directors and a newly-formed Executive Committee of the Board consisting of Dr. Sandrock, Mr. Higgins, and Glenn Pierce, M.D., Ph.D., Voyager’s interim chief scientific officer. Dr. Sandrockis working work with Voyager leadership to help shape future strategies leveraging the Company’s TRACER™ platform and therapeutic programs, and to expand its external collaborations with scientific leaders and industry partners. Dr. Sandrockjoined Voyager following a prolific career in biopharmaceutical drug development.
- The Company also recently announced the promotion of
Robin Swartzto chief operating officer. Ms. Swartzjoined Voyager in 2021 following a 25-year career with Genzymeand Sanofi Genzyme where she held a number of leadership positions, including Senior Vice President of U.S.and Global Business Operations, Senior Vice President U.S.Rare Disease Patient and Product Services, and Senior Director Finance.
Fourth Quarter and Full Year 2021 Financial Results
- Collaboration Revenues: Voyager had collaboration revenue of
$28.1 millionfor the fourth quarter of 2021, compared to $6.5 millionfor the same period in 2020. The increase in collaboration revenue was largely due to a change in estimate related to our collaboration with Neurocrine Biosciences. For the year ended December 31, 2021, collaboration revenue was $37.4 million, compared to $171.1 millionfor the same period of 2020. The decrease in collaboration revenue is largely due to $105.2million of revenue related to the AbbVie collaborations that was recognized upon the termination of the collaborations during 2020.
- Net Loss: Net income was
$5.7 millionfor the fourth quarter of 2021, compared to a net loss of $15.9 millionfor the same period of 2020. Net income was higher driven by the recognition of revenue related to our collaboration with Neurocrine Biosciences. Net loss was $71.2 millionfor the year ended December 31, 2021, compared to net income of $36.7 millionfor the same period of 2020. Voyager had net income for 2020 driven primarily by revenue related to the AbbVie collaborations which were terminated during 2020.
- R&D Expenses: Research and development expenses were
$14.0 millionfor the fourth quarter of 2021, compared to $22.0 millionfor the same period in 2020. For the year ended December 31, 2021, R&D expenses were $73.8 million, compared to $108.8 millionfor the same period of 2020. The decrease in R&D expenses was related to employee-related expenses driven by a reduction in headcount as well as lower external costs primarily for clinical development and manufacturing.
- G&A Expenses: General and administrative expenses were
$8.4 millionfor the fourth quarter of 2021, compared to $8.3 millionfor the same period in 2020. For the year ended December 31, 2021, G&A expenses were $37.2 million, compared to $35.0 millionfor the same period of 2020. The increase in G&A expenses was primarily related to employee-related severance expenses in connection with a reduction-in-force commenced in the third quarter of 2021 and facility costs.
- Cash Position: Cash, cash equivalents and marketable securities as of
December 31, 2021were $132.5 million. This cash position does not include the $54 millionupfront payment Voyager is entitled to receive from Novartis as part of the Novartis license option agreement announced on March 8, 2022.
- Voyager expects that its cash, cash equivalents, and marketable securities inclusive of upfront payments anticipated to be received under the Novartis license option agreement will be sufficient to meet Voyager’s planned operating expenses and capital expenditure requirements into 2024.
Participation in Upcoming Investor Event
- Cowen 42nd Annual
Health Care Conference, corporate panel discussion, Mar. 9, 2022, 12:50 p.m. ET.
About the TRACER™ AAV Capsid Discovery Platform
Voyager’s TRACER™ capsid discovery platform is a broadly applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV9- and AAV5-derived 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 AAV9, AAV5, and other 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 capsid discovery platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV9 and AAV5 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 respective license option agreements with Novartis and
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; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s scientific approach and general development progress; Voyager’s ability to attract and retain talented contractors and employees to continue the development of the TRACER capsid discovery platform and the identification of proprietary capsids; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform and the capsids identified by the platform; the response of the FDA and other regulators to Voyager’s regulatory submissions and communications; the ability to attract and retain talented contractors and employees, including key scientists and business leaders; the ability to create and protect intellectual property; Voyager’s ability to perform its obligations under its license option agreements and its counterparties’ respective abilities to perform their obligations under such agreements; the sufficiency of cash resources; the possibility or the timing of the exercise of development, commercialization, license and other options under the
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||Year Ended|
|Statement of Operations Items:||2021||2020||2021||2020|
|Research and development||14,020||21,996||73,787||108,753|
|General and administrative||8,351||8,270||37,246||34,991|
|Total operating expenses||22,371||30,266||111,033||143,744|
|Operating income (loss)||5,702||(23,729||)||(73,618||)||27,384|
|Total other income||6||7,802||2,421||9,357|
|Net income (loss)||$||5,708||$||(15,927||)||$||(71,197||)||$||36,741|
|Net income (loss) per share, basic||$||0.15||$||(0.43||)||$||(1.89||)||$||0.99|
|Net income (loss) per share, diluted||0.15||(0.43||)||(1.89||)||0.98|
|Weighted-average common shares outstanding, basic||37,804,372||37,290,259||37,668,947||37,132,447|
|Weighted-average common shares outstanding, diluted||37,804,372||37,290,259||37,668,947||37,348,514|
|Selected Balance Sheet Items||2021||2020|
|Cash, cash equivalents, and marketable securities||$||132,539||$||174,782|
|Accounts payable and accrued expenses||$||11,524||$||14,839|
|Total stockholders’ equity||$||95,055||$||154,320|
Contacts Investors Investors@voyagertherapeutics.com
Andrew Funderburkafunderburk@kendallir.com Media Scott Santiamossantiamo@vygr.com
Source: Voyager Therapeutics, Inc.