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Sanofi Pulls Back from Voyager Gene Therapy Pact in Parkinson’s

Xconomy Boston — 

A key study for Voyager Therapeutics and its experimental gene therapy for Parkinson’s disease will have to move ahead without help from pharmaceutical giant Sanofi, which has walked away from the deal.

Global rights to Parkinson’s treatment VY-AADC01 are now squarely in the hands of Cambridge, MA-based Voyager (NASDAQ: VYGR). Two years ago, the company began a broad collaboration with Sanofi’s (NYSE: SNY) Genzyme subsidiary on central nervous system drugs. Voyager earned $100 million up front and kept U.S. rights to its drugs. Sanofi gained the option to license those drugs for development and commercialization in the rest of the world.

As the Parkinson’s therapy progressed in clinical studies, Sanofi used an “option review period” included in the original deal to ask for a share of U.S. rights to the Parkinson’s program, Voyager CEO Steven Paul told analysts on a conference call late Monday. Voyager was not interested in changing the deal’s original terms, Paul said. He declined to comment on Sanofi’s strategy.

“All we know is we’re pleased to retain worldwide rights to this program,” Paul said. “We believe this is a winner, and we believe this will be a launched product for us.”

Voyager shares opened at $20.50 Tuesday, down more than 10 percent, and then recovered slightly to close at $21.06.

Failing to grab a share of U.S. rights, Sanofi decided to give up its remaining rights to the program. Further testing of the therapy and its commercialization, if successful, now lies entirely with Voyager. The company says it has enough cash to last until 2019.

The 2015 deal with Sanofi included other gene therapies, such as experimental treatments for Friedreich’s ataxia and Huntington’s disease. The agreement remains in place for those therapies, meaning that Sanofi still has the right to license global rights to those drugs. Paul said those programs are ongoing and “progressing nicely.” Sanofi retains an equity stake in Voyager of less than 10 percent.

Voyager’s Parkinson’s therapy is meant to be a one-time treatment that restores the brain’s ability to produce dopamine, a neurotransmitter. This approach could improve motor function in Parkinson’s patients and reduce the need for levodopa, an older treatment for the disease that comes with side effects, Paul said. So far, Voyager has tested its gene therapy in a Phase 1 study. Paul now says the company expects to start a Phase 2/3 trial in the first half of 2018. That trial is expected to provide enough data to support an application for FDA approval.

The Phase 2/3 trial will be global, including a site outside of the United States. Despite losing Sanofi as a partner, Paul said that plans for the study are on track: “We think Sanofi could have been helpful [outside of the U.S.] but we actually feel that we’re in good shape.”

Paul said Voyager will discuss additional details during the company’s R&D day Nov. 16. Voyager will release its third quarter financial results on Thursday.

Image by Flickr user _DJ  via a Creative Commons license.