A gene therapy developer is making big changes to its four-year-old partnership with a large French drugmaker.
Cambridge, Massachusetts-based Voyager Therapeutics said Monday it would restructure a partnership it started with Sanofi Genzyme in February 2015 to develop gene therapies for central nervous system disorders.
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Shares of Voyager were up 14 percent on the Nasdaq late Monday morning following the news. Shares of Sanofi were down slightly on the Euronext Paris exchange.
Under the restructured relationship, Voyager takes worldwide rights to VY-HTT01, a gene therapy in preclinical, lead-selection development for Huntington’s disease, as well as ex-U.S. rights to VY-FXN01, also in preclinical development, for Friedreich’s ataxia, which it is then transferring to Neurocrine Biosciences. Meanwhile, Sanofi will have exclusive option rights to select adeno-associated viral vector-based capsids owned by Voyager for exclusive use in up to two non-central nervous system indications. Voyager plans to find a partner for VY-SOD102, its preclinical gene therapy candidate for amyotrophic lateral sclerosis, and as such no longer plans to file with the Food and Drug Administration later this year to start clinical development.
Voyager will pay $10 million upfront to Sanofi, which it said is partially offset by a $5 million payment from Neurocrine. It will also pay a $10 million milestone to Sanofi after it files with the FDA to begin clinical trials of VY-HTT01, which it plans to do later this year assuming preclinical studies are successful.
In a note to investors Monday, Cowen analyst Phil Nadeau wrote that Voyager’s announcement provides further clarity around its pipeline development and commercialization plans, and that it is focusing development on a wholly owned candidate with strong preclinical data. He noted that animal studies of VY-HTT01, including in primates, have not turned up safety concerns.
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The partnership was originally announced in February 2015, focusing on adeno-associated viral vector-based therapies for central nervous system disorders, in particularly Huntington’s disease, Friedreich’s ataxia, Parkinson’s disease and others. Sanofi paid $100 million upfront to Voyager, with the potential for up to $745 million in milestones and royalties on sales.
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