Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment

LEXINGTON, Mass., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced that the joint steering committee with its collaborator Neurocrine Biosciences has selected a lead development candidate in the Friedreich’s ataxia (FA) program. The candidate combines a frataxin (FXN) gene replacement payload with an intravenously administered, blood-brain barrier penetrant, novel capsid derived from Voyager’s TRACER capsid discovery platform. The companies expect the program to advance into first-in-human clinical trials in 2025.

Selection of the development candidate triggered a $5 million milestone payment to Voyager, which the Company expects to receive in the first quarter of 2024. Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.

“The nomination of this development candidate in FA marks an important step in our strategic collaboration with Neurocrine, reflecting the power of combining Voyager’s TRACER AAV capsids and payload design capabilities with Neurocrine’s expertise in neuroscience and clinical development,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “While there has been encouraging recent progress in the treatment of FA, it remains a very challenging and eventually fatal disease for which new therapeutic approaches are needed. We believe our strategy to replace the defective frataxin gene could address the underlying disease etiology of FA. We look forward to progressing this and our other gene therapy programs, including our wholly-owned SOD1 ALS program and our Neurocrine-partnered GBA1 Parkinson’s program, towards clinical studies.”

The FA program is being developed under the 2019 strategic collaboration agreement between Voyager and Neurocrine Biosciences for research, development, and commercialization of certain AAV gene therapy products for programs targeting Friedreich’s ataxia and two other undisclosed targets. Under the terms of the 2019 collaboration agreement, Voyager is eligible to receive up to $1.3 billion in potential development and commercial milestone payments, tiered royalties on net sales, and program funding, and Voyager could exercise an option for 60/40 cost- and profit-sharing (Neurocrine/Voyager) in the U.S. for the FA program following the determination by the joint steering committee of proof of mechanism based on established milestones and metrics.