Pfizer, Voyager Therapeutics ink USD 630 million gene therapy deal

With this agreement Pfizer may exercise options to license novel capsids generated from Voyager's RNA-driven TRACER TM (Tropism Redirection of 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.

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.

"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 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.