Voyager Advances Collaboration With Neurocrine; Third Gene Therapy Development Candidate Selected

Voyager Therapeutics, Inc. recently announced that the joint steering committee with its collaborator Neurocrine Biosciences, Inc. has selected a development candidate in a gene therapy program for the potential treatment of an undisclosed neurological disease. The candidate leverages an intravenously administered, blood-brain barrier-penetrant, novel capsid derived from Voyager’s TRACER capsid discovery platform.

Selection of the development candidate triggered a $3 million milestone payment to Voyager, which the Company expects to receive in the fourth quarter of 2024. Voyager is eligible to receive additional future development and commercialization milestone payments based on the further advancement of this program.

“This is the third development candidate nominated under Voyager’s collaborations with Neurocrine, following nominations for Friedreich’s ataxia and for GBA1 Parkinson’s disease and other GBA1-mediated diseases,” said Alfred W. Sandrock, Jr, MD, PhD, Chief Executive Officer of Voyager. “These partnered programs provide multiple opportunities for success and complement our wholly-owned pipeline, including our SOD1 ALS gene therapy program, which is advancing towards an expected IND in mid-2025.”

This program is being developed under the 2023 strategic collaboration agreement between Voyager and Neurocrine Biosciences for research, development, manufacture, and commercialization of certain AAV gene therapy products for programs targeting Parkinson’s disease and other GBA1-mediated diseases and three other undisclosed programs. Under the terms of the 2023 collaboration agreement, for each of the three undisclosed programs, Voyager is eligible to receive up to $175 million in potential development milestone payments plus substantial potential commercial milestone payments, as well as tiered high single-digit to mid-teens royalties on U.S. net sales and mid-single-digit to low double-digit royalties on ex-U.S. net sales. Neurocrine Biosciences will fund the development of each program.

Voyager’s TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager’s wholly owned and partnered gene therapy programs for neurologic diseases.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit www.voyagertherapeutics.com.