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Voyager selects lead candidate for Friedreich's ataxia trial

EditorIsmeta Mujdragic
Published 02/26/2024, 07:15 AM
© Reuters.
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LEXINGTON, Mass. - Voyager Therapeutics, Inc. (NASDAQ:VYGR), a biotechnology firm focused on neurogenetic treatments, has announced the selection of a lead development candidate for its Friedreich’s ataxia (FA) program. This marks a significant milestone in the company's strategic partnership with Neurocrine (NASDAQ:NBIX) Biosciences, as the candidate is expected to enter first-in-human clinical trials in 2025.

The chosen candidate leverages a gene replacement approach targeting the frataxin (FXN) gene, linked to FA, and utilizes a novel capsid from Voyager’s proprietary TRACER™ capsid discovery platform. This capsid is designed to cross the blood-brain barrier via intravenous administration, a critical feature for delivering gene therapies to the central nervous system.

Voyager's announcement triggered a $5 million milestone payment as part of their collaboration with Neurocrine Biosciences, which the company anticipates receiving within the first quarter of 2024. Additional development and commercialization milestone payments may follow as the program progresses.

Dr. Alfred W. Sandrock, Jr., CEO of Voyager, expressed optimism about the potential of this gene therapy approach to address the root cause of FA, a debilitating and life-shortening neurodegenerative disorder. The company also has other gene therapy programs in the pipeline, targeting diseases like ALS and Parkinson’s, which are moving towards clinical study phases.

The partnership between Voyager and Neurocrine Biosciences began in 2019, focusing on the development and commercialization of AAV gene therapy products for FA and two other undisclosed targets. Voyager stands to gain up to $1.3 billion in potential development and commercial milestone payments, as well as tiered royalties on net sales and program funding. Additionally, Voyager may opt for a 60/40 cost- and profit-sharing arrangement in the U.S. for the FA program, subject to certain conditions.

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Voyager's TRACER™ capsid discovery platform is a key component of their research, allowing for the rapid development of capsids that can efficiently target the central nervous system and demonstrate improved gene expression compared to conventional AAV capsids.

This news is based on a press release statement from Voyager Therapeutics, Inc.

InvestingPro Insights

As Voyager Therapeutics, Inc. (NASDAQ:VYGR) continues to make strides in the field of gene therapy for neurogenetic disorders, the company's financial health and market performance offer additional insights. According to the latest data from InvestingPro, Voyager Therapeutics has a market capitalization of $504.72 million, reflecting its current valuation in the market. The company's P/E ratio stands at an attractive 6.24, suggesting that investors may find the stock's earnings relative to its price compelling.

Investors and analysts are showing optimism about Voyager's future. The InvestingPro Tips highlight that the company holds more cash than debt on its balance sheet, which provides a strong foundation for future investments and development. Additionally, analysts are expecting net income and sales growth this year, which could be indicative of the company's potential to capitalize on its advancements in gene therapy. Notably, two analysts have revised their earnings expectations upwards for the upcoming period, further underscoring the positive outlook on the company's financial performance.

These financial indicators are particularly relevant as Voyager Therapeutics prepares for the clinical trials of its FA program. The company's strong balance sheet and anticipated growth could provide the necessary resources to navigate the costly and complex process of bringing new therapies to market. For readers interested in a deeper analysis, there are 10 additional InvestingPro Tips available, which can be accessed through the InvestingPro platform at https://www.investing.com/pro/VYGR. Don't forget to use the coupon code PRONEWS24 to receive an additional 10% off a yearly or biyearly Pro and Pro+ subscription.

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This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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