Voyager Therapeutics unlocked a potential $600 million plus $25 million upfront as Novartis exercised its option to leverage the company’s capsids against two undisclosed neurologic disease targets.
Monday's announcement comes nearly one year after Novartis paid Voyager $54 million to use adeno associated virus (AAV) capsids developed on its RNA-driven TRACER platform for up to three CNS targets. Voyager was eligible for a potential $1.7 billion overall.
All rights for the unselected target will be returned to Voyager.
In a previous interview, Al Sandrock, CEO of Voyager, told BioSpace that with modalities like protein therapeutics, antisense oligonucleotides and siRNAs, “there’s virtually no undruggable target” in neurotherapeutics. The problem, he said, is delivery. “The blood-brain barrier still poses an issue.”
Voyager’s capsids have shown widespread transgene expression superior to conventional adeno-associated virus serotypes.
“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,” Sandrock said in a prepared statement Monday.
Novartis and Voyager retained an air of mystery as to the exact targets, as they did in the March 2022 partnership announcement.
With the option exercise comes the potential for Novartis to expand the partnership to include two more CNS targets over the next 18 months. Voyager could receive $18 million per target, along with $12.5 million to select a capsid for each target.
After spending over 20 years at Biogen, including as head of R&D, Sandrock was named CEO of Mass.-based Voyager in May 2022. The move appears to be paying off.
In January, the company inked a multi-target deal with Neurocrine Biosciences worth a potential $1.5 billion. The deal centers on Voyager’s preclinical GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, for which San Diego-based Neurocrine received rights.
The companies will also collaborate on three new gene therapy programs for CNS targets, each leveraging Voyager’s TRACER platform.
And in October 2022, Pfizer exercised an option to use a novel capsid developed on the TRACER platform against an undisclosed rare neurologic disease target. Pfizer’s decision brought Voyager $10 million upfront and the potential for up to $290 million in development, regulatory, commercial milestones and tiered royalties.
Voyager will announce Q4 2022 earnings on March 7.
https://www.biospace.com/article/voyager-rolls-on-as-novartis-selects-two-of-three-cns-targets-/
