Voyager Therapeutics Announces License Option Agreement with Pfizer for Next-Generation TRACERᵀᴹ AAV Capsids to Enable Neurologic and Cardiovascular Gene Therapy Programs

Voyager receives $30 million up front with potential option exercise fees and milestone payments of up to $600 million plus product sales-based royalties

Pfizer receives transgene-specific access to Voyager’s novel AAV capsids with enhanced blood-brain-barrier penetration and cardiac muscle tropism for potential use with two transgenes

CAMBRIDGE, Mass., Oct. 06, 2021 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) platform technologies, today announced an agreement through which Pfizer Inc (NYSE: PFE) may exercise options to license novel capsids generated from Voyager’s RNA-driven TRACER^TM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) screening technology as part of Pfizer’s efforts to develop, manufacture, and commercialize gene therapies, utilizing two undisclosed transgenes to treat certain neurologic and cardiovascular diseases.

“This transaction highlights the potential of our TRACER platform to identify novel AAV capsids that target desired cells and tissues with greater specificity at lower doses and with fewer off-target risks than conventional AAV serotypes,” said Michael Higgins, Interim CEO of Voyager. “We believe that our TRACER platform has the ability to produce not only enhanced blood-brain-barrier penetrant capsids, but also novel capsids with enhanced tropisms across a diversity of tissues and cell types, offering promise to unlock the fullest potential of gene therapies for a wide array of diseases with unmet medical need.”

"Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio,” said Seng Cheng, Ph.D., Senior Vice President and Chief Scientific Officer of Pfizer's Rare Disease Research Unit. “We are impressed with Voyager’s results to date and are enthusiastic about the potential to utilize these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases.”

Proprietary AAV capsids derived from Voyager’s TRACER platform have demonstrated superior blood-brain-barrier penetration, enhanced cardiac muscle tropism, and increased transgene expression in target tissues compared to conventional AAV capsids as measured in non-human primates (NHPs). Voyager presented data at the 24^th Annual Meeting of the American Society of Gene and Cell Therapy for one capsid candidate demonstrating more than 1,000-fold increased transgene expression compared to conventional AAV9 across a wide array of brain regions when dosed intravenously in NHPs. Results presented also included another capsid candidate showing significantly enhanced cardiac muscle transduction and dorsal root ganglia de-targeting compared to conventional AAV9. Voyager is performing further screening with its TRACER platform to identify additional proprietary AAV capsids targeting multiple tissue and cell types for use in gene therapies to treat a broad range of diseases.

Under the terms of the agreement, Pfizer will have the right to evaluate novel capsids selected for central nervous system and cardiac tropisms from Voyager’s TRACER platform and to exercise options to license capsids for exclusive use in Pfizer’s development of AAV gene therapies incorporating two undisclosed transgenes. These transgenes will be distinct from those planned for Voyager’s internal pipeline. Voyager will retain global rights to all licensed capsids for use with other transgenes and to all other applications of its TRACER technology.

Voyager will receive $30 million upfront and is entitled to receive up to $20 million in exercise fees for two options, exercisable by Pfizer within 12 months of signing. In addition, Voyager will be eligible to earn up to $580 million in 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.

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 AAV9 and other capsid serotypes to identify novel AAV vectors optimized for specific therapeutic applications.

About Voyager Therapeutics
Voyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the technology to treat devastating diseases. Proprietary capsids born from the Company’s TRACER screening platform are powering a rich early-stage pipeline of new and second-generation programs and may elevate the field to overcome the limitations of conventional gene therapy vectors across neurologic disorders and other therapeutic areas. voyagertherapeutics.com LinkedIn Twitter

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 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 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 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; 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 Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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