LEXINGTON, Mass. - Voyager Therapeutics, Inc. (NASDAQ:VYGR), a biotechnology firm focused on neurogenetic medicine development, today announced the selection of a lead candidate for their gene therapy program targeting Parkinson’s disease and other diseases mediated by the GBA1 gene.
The candidate is a result of the company's collaboration with Neurocrine (NASDAQ:NBIX) Biosciences and utilizes a novel capsid from Voyager’s proprietary TRACER™ capsid discovery platform.
This advancement has triggered a $3 million milestone payment to Voyager, expected to be received in the second quarter of 2024. The company also stands to gain from future development and commercialization milestones. The Investigational New Drug (IND) application for this program is anticipated to be filed with the FDA in 2025.
Voyager’s CEO, Alfred W. Sandrock, Jr., M.D., Ph.D., expressed optimism about the progress, highlighting the potential for three gene therapies using their novel capsids to enter clinical trials in 2025. These include the GBA1 and Friedreich’s ataxia programs partnered with Neurocrine and Voyager’s own SOD1 ALS program.
The GBA1 program is part of a strategic collaboration agreement from 2023 between Voyager and Neurocrine Biosciences, which encompasses the research, development, manufacture, and commercialization of AAV gene therapy products for Parkinson’s disease and other GBA1-mediated diseases, along with three other undisclosed programs targeting central nervous system diseases or conditions associated with rare genetic targets.
Under the terms of the collaboration, Voyager could receive up to $1.5 billion in potential development milestone payments, along with additional commercial milestone payments, tiered royalties on net sales, and program funding. The company also retains an option for 50/50 cost- and profit-sharing in the U.S. for the GBA1 program following top-line data from the first Parkinson’s disease clinical trial.
The TRACER™ Capsid Discovery (NASDAQ:WBD) Platform is an RNA-based screening platform designed for the rapid discovery of AAV capsids that can penetrate the blood-brain barrier and deliver gene therapies directly to the central nervous system.
The platform has generated capsids that show superior and widespread gene expression in the CNS compared to conventional AAV capsids and has the potential to target specific areas of the brain that are traditionally difficult to reach.
This announcement is based on a press release statement by Voyager Therapeutics, Inc. and reflects the company's ongoing efforts to advance treatments for neurological diseases using their gene therapy technologies.
InvestingPro Insights
Voyager Therapeutics, Inc. (NASDAQ:VYGR) has recently made significant strides in their gene therapy program, which is reflected in their financial and market metrics. With an impressive market capitalization of $447.44 million, the company's commitment to innovation in neurogenetic medicine is clear.
A standout figure from their recent financial performance is the remarkable revenue growth of over 511% in the last twelve months as of Q4 2023, demonstrating the potential of their proprietary TRACER™ capsid discovery platform and the value of their strategic collaborations.
Two InvestingPro Tips that are particularly relevant to Voyager Therapeutics at this juncture are the company's strong cash position compared to its debt, and the positive revisions by analysts regarding the company's earnings for the upcoming period. These insights suggest a solid financial foundation and growing confidence in the company's future performance.
Moreover, the company's valuation implies a strong free cash flow yield, which may be attractive to investors looking for companies with the potential for high returns on their investments.
Investors interested in a deeper dive into Voyager's financial health and future prospects can explore more InvestingPro Tips, with a total of 9 additional tips available at https://www.investing.com/pro/VYGR. For those seeking to make the most informed investment decisions, using the coupon code PRONEWS24 can provide an additional 10% off a yearly or biyearly Pro and Pro+ subscription at InvestingPro.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.