Voyager Therapeutics to Receive $25 Million Payment for License of Next-Generation AAV Capsids for Multiple Neurologic Disease Targets

Rare Daily Staff

Voyager Therapeutics said that Novartis exercised its options to license novel capsids generated from Voyager’s TRACER capsid discovery platform for use in gene therapy programs against two undisclosed neurologic disease targets, triggering a $25 million payment to the company.

Voyager said its TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs).

TRACER generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach. Separate results have demonstrated the enhanced ability of certain capsids to target cardiac muscle and to de-target the dorsal root ganglia. Voyager is expanding its library of AAV capsids optimized to deliver diverse therapeutic payloads to address a broad range of CNS and other diseases.

Under the terms of the license option agreement announced in March 2022, Voyager previously received a $54 million upfront payment from Novartis for the option to license capsids for up to three central nervous system targets. With Novartis’ option exercise on two targets, Voyager is eligible to receive up to $600 million in associated potential development, regulatory, and commercial milestone payments, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.

In addition, over the next 18 months, Novartis retains the right to expand the agreement to include options to license capsids for up to two additional rare CNS targets, subject to their availability, for a fee of $18 million per target. Under such an expansion, Voyager would be eligible to receive a $12.5 million license option exercise fee for each target exercised, as well as future potential milestone payments of $300 million per target and tiered mid- to high-single digit royalties on products incorporating the licensed capsids.

The two Novartis targets licensed under the agreement are distinct from targets in Voyager’s internal and partnered pipeline. Novartis elected not to license a capsid for one CNS target under the original agreement, and all capsid rights with respect to that target are returned to Voyager. Voyager retains global rights to all licensed capsids for use with other targets and to all other applications of its TRACER technology.

“We believe intravenously-delivered, brain-penetrant capsids, such as those generated through Voyager’s TRACER capsid discovery platform are critical to enable utilization of gene therapy for neurologic diseases,” said Alfred Sandrock, Jr., CEO of Voyager. “We view Novartis as a leader in the gene therapy field, so we greatly appreciate their decision to leverage our technology in their programs. Additionally, the non-dilutive funding provided through this capsid license, as well as recent agreements with Pfizer and Neurocrine Biosciences, further strengthens Voyager’s balance sheet and supports the advancement of our platform and pipeline.”

Photo: Alfred Sandrock, Jr., CEO of Voyager