It’s been a year since Novartis ventured into orbit with Voyager Therapeutics to find new central nervous system targets. Now, the Big Pharma is ready to go deeper into space with the adeno-associated virus (AAV) capsid discovery biotech.
Novartis has pulled the trigger on the March 2022 deal to opt in on two out of three targets, which means a $25 million payment for Voyager and the potential to access up to $600 million later in milestones.
“For a company like Novartis to be kicking the tires with our capsids for a year in their own hands and then opting in after a year of evaluation, I think is pretty significant,” Voyager CEO Al Sandrock, M.D., Ph.D., said in an interview with Fierce Biotech. He highlighted Novartis’ work with spinal muscular atrophy gene therapy Zolgensma, calling the Swiss pharma a leader in the field.
The original deal included a $54 million upfront payment and $300 million in potential payments down the line for each program Novartis chose to move forward with. The company opted not to move forward with the third program that was part of the deal, which was an internal pipeline decision on Novartis’ part.
The opt-in is the latest validation of Voyager’s new direction. The biotech focused its attention on the central nervous system last year, putting three assets for Alzheimer’s, Parkinson’s and a type of amyotrophic lateral sclerosis (ALS) in the lead.
Voyager’s TRACER platform screens genetic data to build AAV capsids, creating gene therapies that have better specificity and less toxicity, all at lower doses than conventional alternatives. Capsids are the outer protein layer of the virus vectors where genetic material is held. Voyager’s platform aims to select capsids that can penetrate the blood-brain barrier.
Sandrock would not reveal what diseases the Novartis partnership will address, but said to look to the ALS, Alzheimer’s, Parkinson’s and Friedreich ataxia programs from Voyager’s internal pipeline for clues as to what the company’s wheelhouse is.
The Novartis deal is structured similarly to another signed with Pfizer. Voyager gives the pharma giants access to the capsids and lets them consider for a year whether to go all in. Like Novartis, Pfizer opted in on capsids for neurologic diseases but declined to continue on a program for cardiovascular diseases. That deal meant $10 million upfront and potentially up to $290 million in milestones for Voyager but a miss on $300 million tied to the cardiovascular program.
Sandrock said the Novartis and Pfizer opt-ins, plus an additional collaboration with Neurocrine signed in January, are external validation of Voyager’s capsid program and the new direction.