Sanofi quits Voyager gene therapy deal once worth up to $845M

Two years after it walked away from a Voyager Therapeutics-partnered Parkinson’s disease gene therapy, Sanofi Genzyme is at it again. This time, it’s quitting two more programs it picked up in a 2015 pact with Voyager—a gene therapy for Huntington’s disease and one for Friedreich’s ataxia. Voyager will hand over $10 million to regain the rights to those programs. 

Under the original deal, Sanofi Genzyme ponied up $100 million—and promised up to $745 million in milestones—in exchange for the option to license several programs from Voyager. At the time, Voyager held onto the U.S. rights for its programs in Parkinson’s and Friedreich’s ataxia and planned to split the U.S. profits for its Huntington’s product with Genzyme.

Sanofi ditched the Parkinson’s treatment, VY-AADC, in October 2017, because, as Voyager understood it, its option to take the program forward didn’t include the U.S. rights. Voyager saw “tremendous value” in holding on to the U.S. rights for VY-AADC, said Voyager CEO Steven Paul, M.D., on a conference call at the time—even if it meant losing a partner as the program approached a pivotal phase 2/3 study. 

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Sanofi is returning to Voyager the ex-U.S. rights to the Huntington’s program, VY-HTT01, as well as those to the Friedreich’s ataxia program, VYFXN01, Voyager said in a statement. The ataxia rights aren’t staying put, though; they will be transferred to Neurocrine Biosciences under an agreement it signed with Voyager in January. Voyager picked up $165 million upfront but could rake in up to $1.7 billion if it meets developmental, regulatory and sales milestones.

The Big Pharma will also end its work with Voyager on spinal muscular atrophy and lose its option to develop a future orphan central nervous system (CNS) program from Voyager.

Voyager will follow its $10 million payment to Sanofi with a second $10 million milestone when it files an IND for the Huntington’s program, which it expects to do by the end of this year.

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Now that its pipeline includes two partnerless programs, Voyager is putting its gene therapy for amyotrophic lateral sclerosis (ALS) on the back burner. It won’t file an IND for that program in 2019, the company said in the statement. Voyager kept the ALS program to itself in 2015, but is now looking for a partner to take it forward.

Sanofi and Voyager are completely retooling their partnership to focus on adeno-associated virus capsids—the protein shells of the virus—for the delivery of gene therapies. Sanofi will have the exclusive option to use these capsids to deliver treatments for up to two non-CNS diseases.

“Our alliance with Sanofi Genzyme has been foundational for Voyager as we optimized the development of capsids, transgenes and gene therapy delivery,” said Andre Turenne, who became Voyager’s CEO last year after a 12-year tenure at Genzyme and Sanofi, in the statement. “We look forward to this new stage of the relationship and to advancing our programs and research efforts for patients with severe neurological diseases.”