Six years ago, Third Rock Ventures launched Voyager Therapeutics with $45 million and a goal of bringing gene therapy into some of the most notorious neurological diseases, most notably Parkinson’s. Now that program, long their lead and only clinical asset, is in jeopardy.
Voyager announced Tuesday afternoon that Neurocrine Biosciences has terminated their agreement to co-develop the Parkinson’s gene therapy after safety issues put the program on hold last year. Voyager said they are still evaluating whether or not to continue developing the gene therapy internally. They pinned Neurocrine’s decision on a “portfolio review and prioritization of its current pipeline assets.”
The Parkinson’s program was the centerpiece not only of the Cambridge biotech but also of the $1.8 billion collaboration they signed with Neurocrine 2 years ago, along with earlier stage collaborations on Friedreich’s ataxia and two undisclosed diseases. Neurocrine has now become the second company to back out of a deal with Voyager on the Parkinson’s program, after Sanofi opted in 2017 to decline an option they had under a larger $845 million partnership. They ditched the rest of the partnership two years later, although Voyager is still led by Sanofi alum Andre Turenne.
Voyager and Neurocrine will continue to work together on Friedreich’s ataxia and the undisclosed diseases, Voyager said.
The company’s stock $VYGR, already eroded by the Sanofi exit and last year’s safety delays, was not majorly impacted, sliding 5% after the bell from $8.00 to $7.55.
The Parkinson’s program was already in the clinic when Voyager launched in 2014 and three years later it offered them an early glimpse of success, reducing the need for Parkinson’s medications and increasing motor function in a 10-patient cohort. They also showed increased expression of AADC, an enzyme needed to turn levodopa into dopamine, the key molecule Parkinson’s patients need.
After releasing similar tentative results in a 15-patient group a year later, they moved into a pivotal trial, where the setbacks began. In November, the trial’s data safety and monitoring board paused the study after finding worrying signs in “certain patient imaging data.” The FDA followed up a month later with a formal clinical hold, citing “MRI abnormalities.”
Voyager has still not disclosed details on what triggered the pause, but concern around the industry has been over the route of administration. Neurosurgeons inject the therapy directly into the brain.
Without the Parkinson’s program, Voyager would become a pre-clinical biotech. The FDA has prevented the No. 2 project in the pipeline, a gene therapy for Huntington’s disease, from going into the clinic over what Voyager said were CMC issues.
