Clinical-stage gene therapy company Voyager Therapeutics Inc. (VYGR:NASDAQ), which is focused on the development of treatments for central nervous system (CNS) diseases and next-generation adeno-associated virus (AAV) platform technologies, today announced "an agreement through which Pfizer Inc. (PFE:NYSE) 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."
"Pfizer's global portfolio includes dozens of brand prescription medications including Celebrex, Eliquis, Lipitor, Prevnar 13, Pristiq, Viagra, and Xeljanz."
Voyager Therapeutics' Interim CEO Michael Higgins commented, "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…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."
"[P]roprietary 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 tissue."
Seng Cheng, Ph.D., SVP and Chief Scientific Officer of Pfizer's Rare Disease Research Unit remarked, "Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio…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."
Voyager claimed that "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)."
Execution of the agreement provides Pfizer with the rights to select and evaluate novel capsids for CNS and cardiac tropisms from Voyager's TRACER platform. In addition, Pfizer will be able to exercise options to exclusively license those capsids for its AAV gene therapies development efforts that incorporate two specific undisclosed transgenes.
Under the terms of the agreement, Voyager will retain the full global rights to all other licensed capsids for use with other transgenes in its development pipeline as well as all other applications of its TRACER technology.
The agreement stipulates that Pfizer is to pay Voyager $30 million upfront and an additional $20 million in exercise fees for two options, should Pfizer elect to exercise those options which are valid for a period of 12 months from signing. The report indicated that, "Voyager will also 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." On top of the develop and milestone payments, the firm may be entitled to receive mid- to high-single-digit tiered royalties from net sales of Pfizer's products that contain the licensed capsids.
The company explained that its TRACERᵀᴹ AAV capsid discovery 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)."
Voyager Therapeutics is based in Cambridge, Mass., and is actively engaged in the development of next generation adeno-associated virus (AAV) gene therapies to treat devastating diseases. The company stated that, "proprietary capsids born from its 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." The firm at present is largely focused on several severe neurological diseases including Huntington's disease, spinal muscular atrophy and SOD1, which is a monogenic form of amyotrophic lateral sclerosis.
Pfizer is headquartered in New York, N.Y., and is a research-based global biopharma company involved in discovery, development and manufacturing of pharmaceuticals and other various healthcare products. The firm's global portfolio includes dozens of brand prescription medications including Celebrex, Eliquis, Lipitor, Prevnar 13, Pristiq, Viagra, and Xeljanz. Pfizer has a market cap exceeding $237 billion.
Voyager Therapeutics began the day with a market cap of around $93.7 million with approximately 37.94 million shares outstanding and a short interest of about 2.3%. VYGR shares opened more than 60% higher today at $3.99 (+$1.52, +61.54%) over yesterday's $2.47 closing price. The stock has traded today between $3.49 and $4.12 per share and is currently trading at $3.74 (+$1.27, +51.42%).
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