Sanofi has left Parkinson’s gene therapy due to its $845 million alliance with Voyager Therapeutics. The decision will give Voyager the full global rights to VY-AADC but leaves it without a partner as it prepares for an important global phase ⅔ clinical trial. Voyager pinned Sanofi’s decision as a result of the Big Pharma’s interest to own the U.S right to the gene therapy. The 2015 deal gave Sanofi an option to pick up the ex-U.S rights to the Parkinson’s asset. At the same time, Sanofi was happy enough to spend $100 million and commit to $745 million in milestones for the option and rights to other programs.
However, there has been a shift resulting in discussions about sharing U.S rights, but Voyager was unwilling to loosen its grasp on the territory. Voyager CEO Steven Paul, M.D said, “We were not interested in sharing U.S rights to the program. We see tremendous value in retaining rights to our Parkinson’s disease program in the U.S”
If Voyager’s decision was in fact motivated by the U.S rights, rather than doubts about the program, it raises questions of what has changed since it struck the original deal. A possibility is that the failure of uniQure’s Glybera and a slow start of GlaxoSmithKline’s Strimvelis in Europe have put Sanofi off the prospect of commercializing the Parkinson’s gene therapy in the region.
Glybera and Strimvelis were destined to be niche products, however Voyager is pitching its candidate at a much bigger market, namely the estimated 10,000, Parkinson’s patients a year that undergo deep-brain stimulators (DBS). Europe played a central role in the rise of DBS remains a major market. Japan, with its elderly population, is another place where VY-AADC may succeed. Paul discussed the prospects of VY-AADC in those former U.S territories to investors, but acknowledged Europe poses specific challenges. Paul stated, “There are considerably more pricing pressures in Europe than there are in the U.S.” Voyager believes that it can sell the gene therapy itself in the U.S and use it as a launchpad for its evolution into a commercial-stage organization. However, Paul sounds open to offloading regional rights if a deal comes along that allows Voyager to retain the control it has prioritized.
Currently, Voyager is working to keep development program on track. While accepting Sanofi’s involvement could have benefited the ex-U.S part of the phase ⅔ trial, Paul said Voyager is in good shape as it approaches the study. Paul sees no other aspects of Sanofi’s exit stopping Voyager from starting dosing in the phase ⅔ in the first half of 2018.