On October 6, 2021 Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) platform technologies, reported an agreement through which Pfizer Inc (NYSE: PFE) may exercise options to license novel capsids generated from Voyager’s RNA-driven TRACERTM (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 (Press release, Voyager Therapeutics, OCT 6, 2021, View Source [SID1234590861]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"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 24th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 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.