Neurocrine Biosciences and Voyager Therapeutics inked a CNS-targeted collaboration potentially worth a potential $4.4 billion.
Voyager CEO Al Sandrock/courtesy of Voyager Therapeutics
After taking a second strike with a $50 million epilepsy drug acquired from Idorsia, Neurocrine Biosciences is targeting Parkinson’s disease and other GBA1-mediated diseases in a multi-target deal with Voyager Therapeutics.
The CNS-targeted collaboration, which will yield $175 million up front for Voyager, grants Neurocrine worldwide rights to the Cambridge, Mass.-based company’s preclinical GBA1 program.
The gene therapy combines a GBA1 gene replacement payload with novel capsids from Voyager’s TRACER platform.
The companies will seek to correct the underlying cause of Parkinson’s for some patients. GBA1 mutation is the most common genetic risk factor for the disease, increasing risk by 20-fold.
Voyager’s capsid technology has the ability to cross the blood-brain barrier to deliver gene replacement therapies and potentially achieve lasting correction of GBA1 mutations.
Solving the delivery issue opens up a lot of opportunities in neuroscience, Voyager CEO Al Sandrock told BioSpace in an interview.
“We’ve known for a while what the validated drug targets are... but it’s very hard to get them delivered into the brain,” he said. “Our gene therapy, our capsids, allow us to deliver these genes broadly across the CNS.”
In an investor relations call Monday, Sandrock said interest in the program was high, but Neurocrine emerged the winner due to its position as an “expert in the field of movement disorders” and track record of success.
Neurocrine has a catechol-O-methyltransferase (COMT) inhibitor for Parkinson’s already on the market to increase dopamine in the brain. In 2017, the company brought its first drug to market for tardive dyskinesia, a movement disorder resulting from the use of antipsychotic drugs.
The $175 million upfront payment includes a $39 million investment in Voyager stock. Voyager will have the right to co-develop and co-commercialize the GBA1 program under a 50/50 cost and profit-sharing arrangement with Neurocrine after a Phase I trial is completed.
If Voyager declines this option, it will be eligible for $1.5 billion in development milestones and $2.7 billion in sales milestones, bringing the total deal potential to $4.4 billion.
The agreement also includes another $175 million in potential milestones for the three undisclosed programs. Neurocrine will foot the bill for all costs incurred under the collab.
Voyager Chief Business Officer Allen Nunnally told BioSpace the company will focus this cash infusion on its ALS gene therapy program, passive anti-Tau antibody for Alzheimer’s disease and other early research initiatives.
Voyager’s stock soared 36% Monday morning following news of this second collaboration with Neurocrine. The two tied up in 2019 on gene therapy programs for Parkinson’s and Friedreich’s ataxia.
The deal is a big move for Sandrock, who took the helm at Voyager after a 23-year career at Biogen. One of his first projects was the reprioritization of assets based on potential value for patients and shareholders. The GBA1 program was one of three chosen.
“This collaboration will leverage extensive R&D and commercial capabilities,” Sandrock said during the investor call. “We believe it increases the overall value of the asset and improves the likelihood that this gene therapy can help patients.”
Neurocrine CSO Jude Onyia, Ph.D. will join Voyager’s Board of Directors once the deal is sealed.