Voyager Therapeutics signs landmark RNA deal with Pfizer

Massachusetts-based gene therapy company Voyager Therapeutics announced an agreement through which Pfizer could licence novel capsids generated from Voyager’s RNA-driven ‘TRACER’, or ‘Tropism Redirection of AAV by Cell-type-specific Expression of RNA’ screening technology on Wednesday.

The Nasdaq-traded firm said the deal was part of Pfizer’s efforts to develop, manufacture, and commercialise gene therapies using two undisclosed transgenes to treat certain neurologic and cardiovascular diseases.

It explained that proprietary AAV capsids derived from the TRACER platform had 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.

Voyager presented data at the 24th annual meeting of the American Society of Gene and Cell Therapy for one capsid candidate, demonstrating a more-than-1,000-fold increased transgene expression compared to conventional AAV9, across a “wide array” of brain regions when dosed intravenously in non-human primates.

Results presented also included another capsid candidate showing “significantly-enhanced” cardiac muscle transduction and dorsal root ganglia de-targeting compared to conventional AAV9.

Voyager said it was 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 agreement, Pfizer would 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 licence capsids for exclusive use in its development of AAV gene therapies incorporating two undisclosed transgenes.

Those transgenes would be distinct from the ones planned for Voyager’s internal pipeline.

Voyager said it would retain global rights to all licensed capsids for use with other transgenes, and to all other applications of its TRACER technology.

“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 interim chief executive officer Michael Higgins.

“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.”

The company said it would receive $30m upfront, and would be entitled to receive up to $20m in fees for the two options exercisable by Pfizer within 12 months of signing.

In addition, Voyager would be eligible to earn up to $580m in total development, regulatory, and commercial milestones associated with licensed products incorporating the two undisclosed Pfizer transgenes together with a Voyager-licensed capsid.

Voyager said it would also be eligible to receive mid- to high-single-digit tiered royalties based on net sales of Pfizer’s products incorporating the licensed capsids.

“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, 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 utilise these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases.”

At 0754 EDT, Voyager Therapeutics shares were up 40.49% in premarket trading.