In Novartis’ perennial hunt for innovative therapies to bring under its wing, improving on gene therapy has always been kept top of mind. And the Swiss pharma continues to do just that thanks to a new biobucks deal with Voyager Therapeutics worth up to $1.75 billion.
The companies announced a new licensing deal this morning, which will see $54 million upfront for Voyager, a Cambridge, Massachusetts-based biotech developing improved adeno-associated virus capsids. Down the line, Novartis could pay up to $1.7 billion in various milestones and royalties. The deal covers three initial central nervous system targets, which Novartis can opt-in on within 12 months, plus the potential for two more later on.
Novartis will have access to Voyager’s proprietary RNA-driven Tracer capsid discovery program, an RNA-based screening platform that quickly develops in vivo AAV9- and AAV5-derived capsids. These are used to deliver the payload in gene therapy-based medicines.
Through the partnership, Novartis wants to build on previous learning in neurological diseases, particularly those that occur deep in the brain, where the most common gene therapy vectors have been unable to penetrate effectively up to now.
Voyager's shares were boosted 24% as the markets opened Tuesday, landing at $4.88 apiece—a nearly $1 gain compared to the prior close of $3.93.
Despite being used in the small number of gene therapies approved to date—one of which is Novartis’ Zolgensma—the modality has been dogged by safety concerns. The FDA last year held an advisory meeting to examine the issue and ways to improve safety for patients.
Glenn Pierce, M.D., Ph.D., interim chief scientific officer of Voyager, said that a critical challenge to scaling up gene therapies continues to be toxicity issues related to higher doses. The company has found evidence that its capsids are more precise and targeted in preclinical research. This could mean the technology has fewer off-target effects and “superior delivery,” he said.
This is precisely the kind of gene therapy improvement that Jay Bradner, M.D., president of the Novartis Institutes for BioMedical Research, alluded to when he boldly made a call for biotechs to reach out to him for dealmaking in January.
“The challenge of biodistribution of AAV gene therapies to target cell types in the brain is longstanding, but we refuse to believe it is insurmountable,” Bradner said in a statement.
The new capsid deal will be outside of Voyager’s existing pipeline and comes as Voyager is embarking on something of a comeback. The biotech signed a $630 million gene therapy pact with Pfizer in October 2021 for two targets in neurologic and cardiovascular disease, after weathering a series of development mishaps over the years.
This included AbbVie cancelling a $134 million deal for work in Alzheimer's and Parkinson’s disease, the FDA slapping a clinical hold on a therapy before it could enter clinical trials—a year after Sanofi’s Genzyme unit pulled out of a collab on the program and CEO Andre Turenne and R&D chief Omar Khwaja, M.D., Ph.D., leaving unexpectedly.
Editor's Note: This story was updated at 9:53 a.m. ET on March 8, 2022 to add details on Voyager's shares.