Neurocrine Returns Two Gene Therapy Programs to Voyager Amidst Ongoing CNS Collaboration

Neurocrine Biosciences has opted to hand back two discovery-stage gene therapy programs to Voyager Therapeutics, even as the two companies continue to progress their broader collaboration focused on central nervous system (CNS) disorders.

The two biopharmaceutical firms have been engaged in a substantial CNS-focused partnership since a potential $1.7 billion agreement in 2019. This relationship was further solidified in 2023 with an increased emphasis on Voyager’s gene therapy program targeting Parkinson’s disease and other conditions mediated by the GBA1 gene. The GBA1 program leverages a gene replacement payload in conjunction with novel capsid delivery vehicles derived from Voyager’s proprietary TRACER platform.

The updated 2023 collaboration also encompassed work on three new gene therapy programs aimed at rare CNS targets, each expected to utilize Voyager’s innovative TRACER capsids. However, in Voyager’s first-quarter earnings report, released on Tuesday, May 6th, the company disclosed that Neurocrine had “deprioritized” two of these discovery-stage programs against undisclosed targets, resulting in their return to Voyager. Voyager emphasized in the release that these discontinuations were not related to any safety concerns.

Despite this adjustment, Voyager remains optimistic about advancing other key programs under the collaboration. The biotech anticipates submitting regulatory requests this year to initiate clinical trials for the lead candidates, specifically the GBA1 gene therapy program for both Gaucher and Parkinson’s diseases, as well as a program targeting Friedreich’s ataxia. Voyager projects that these studies will commence in 2026, triggering potential milestone payments of $35 million.

Voyager reported a “strong cash position” of $295 million at the end of March, which is projected to sustain operations into mid-2027. The company’s clinical pipeline includes VY7523, an anti-tau antibody for Alzheimer’s disease currently in clinical trials, and VY1706, a preclinical tau silencing gene therapy. “We continue to thoughtfully and strategically advance our pipeline, including our two wholly-owned tau targeting programs VY7523 and VY1706 for Alzheimer’s disease, as well as the FA and GBA1 programs, which are advancing towards INDs this year,” stated Voyager CEO Alfred Sandrock Jr., M.D., Ph.D., in the May 6th release.

Under the 2023 agreement, Neurocrine made an upfront payment of $175 million and retained the option to either share the costs when the GBA1 program reaches Phase 2 trials or allow Voyager to retain eligibility for up to $985 million in U.S.-based milestone payments. Regardless of the chosen path for the U.S., Voyager will still be eligible for milestone payments from international territories. For the three additional gene therapy collaborations, Voyager stood to receive a more modest $175 million per program in potential milestone payments.

Voyager reported collaboration revenue of $6.5 million for the first quarter of 2025, a decrease from the $19.5 million reported for the same period in 2024. The company attributed this decline to “decreased revenue recognized under our Neurocrine collaboration agreements.”

The partnership between Neurocrine and Voyager has not been without its challenges. In 2021, Neurocrine withdrew from the Parkinson’s disease portion of their original deal due to safety issues that led to an FDA clinical hold.