Voyager Therapeutics’ bet on its capsid technology continues to pay off as the company banks $175 million from a strengthened collaboration with long-term partner Neurocrine Biosciences.
The collaboration centers around Voyager’s GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases. The GBA1 program involves combining a gene replacement payload with novel capsids from Voyager’s TRACER platform. In addition, the two companies will work on three new gene therapy programs directed to rare central nervous system targets, each of which will also leverage Voyager’s TRACER capsids.
In return, Neurocrine is paying $175 million upfront, of which $136 million will be cash and the remainder an equity investment. When the GBA1 program reaches phase 2 trials, Voyager can either go all in on a 50-50 cost- and profit-sharing arrangement or retain its eligibility to up to $985 million in U.S.-based milestone payments on top of regional royalties. Regardless of which route Voyager picks for the U.S., it will still receive milestone payments from abroad.
For the three additional gene therapy collabs, Voyager is in line to a more modest $175 million payday per program. To seal the deal, Neurocrine Chief Scientific Officer Jude Onyia, Ph.D., will join Voyager’s board of directors.
The hefty upfront payment will come in handy for Voyager as the company continues to rebuild in the wake of a rocky few years marked by clinical holds, crumbling collaborations and falling value, culminating in the departure of Voyager's CEO and R&D chief in May 2021.
Key to the company’s current strategy is the TRACER platform. Its objective is to better screen for genetic data that Voyager can then use to build adeno-associated virus 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 has previously touted the platform as having the ability to select capsids that have "robust penetration of the blood-brain barrier."
The latest agreement with Neurocrine builds off an existing CNS-focused relationship with Voyager looking at Friedreich’s ataxia and related conditions, the two companies pointed out.
“This collaboration illustrates the value-creation opportunity presented by combining Voyager’s novel TRACER capsid platform with our deep knowledge of neuropharmacology and payloads to advance next-generation gene therapies for CNS diseases,” said Voyager CEO Alfred Sandrock Jr., M.D., Ph.D., in the release.
“We anticipate that the opportunities enabled by this collaboration will allow us to continue to invest in our platform and pipeline programs, as well as to advance cutting-edge research initiatives.”
Neurocrine isn’t the only company that wants in on Voyager’s capsids. Voyager granted Pfizer an option on capsids for use in the treatment of neurologic and cardiovascular diseases in 2020, securing $30 million upfront and early external validation of its decision to pivot to the TRACER platform.
Last October, Pfizer gave its verdict, paying out $10 million to take up its option to use the gene therapy technology against a rare neurological disease target but backing out of the cardiovascular part of the deal.