Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences, Triggering Milestone Payment
- None.
- None.
Insights
The announcement by Voyager Therapeutics regarding the selection of a lead development candidate for Friedreich’s ataxia (FA) represents a significant milestone in the company's pipeline development. The financial implications for Voyager include an immediate $5 million milestone payment, which enhances the company's cash flow and could positively influence investor sentiment. Additionally, the potential for up to $1.3 billion in future milestone payments and tiered royalties provides a substantial upside for the company's revenue prospects.
Furthermore, the option for a 60/40 cost- and profit-sharing agreement with Neurocrine Biosciences in the U.S. market could lead to a favorable financial position for Voyager if the FA program demonstrates proof of mechanism. This strategic collaboration with Neurocrine Biosciences could also mitigate some of the financial risks associated with drug development by sharing costs and leveraging Neurocrine's expertise in clinical development.
The progression of Voyager's gene therapy program targeting Friedreich’s ataxia (FA) into clinical trials is a noteworthy advancement in the field of neurogenetics. FA is a rare genetic disease that currently has limited treatment options, making this program a potential game-changer for patients. The use of a novel capsid that can penetrate the blood-brain barrier is a key innovation, potentially increasing the efficacy of the gene replacement therapy.
The TRACER™ capsid discovery platform represents a cutting-edge approach in the development of AAV (adeno-associated virus) vectors, which are commonly used in gene therapy. The ability to address the underlying disease etiology of FA by replacing the defective frataxin gene could set a new standard in treatment if proven successful in clinical trials.
The strategic collaboration between Voyager Therapeutics and Neurocrine Biosciences underscores the trend of partnerships within the biotech industry aimed at pooling resources and expertise to accelerate drug development. The focus on rare diseases like Friedreich’s ataxia (FA) aligns with the industry's move towards precision medicine and treatments for niche markets. Such markets, while smaller in patient numbers, often allow for premium pricing strategies due to the lack of effective therapies.
The advanced stage of this program and the planned progression to clinical trials in 2025 could position Voyager as a leader in the FA market and enhance its competitive edge. The market's response to this news will likely be monitored closely, as it could influence the valuation of similar biotech companies engaged in gene therapy research and development.
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
“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
About the TRACER™ Capsid Discovery Platform
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 AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). TRACER™ generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach, while de-targeting the liver and dorsal root ganglia. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER™ capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.
About Voyager Therapeutics
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.
Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc.
Forward-Looking Statements
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “expect,” “believe,” “could,” “future,” or “potential,” and other similar expressions are intended to identify forward-looking statements.
For example, all statements Voyager makes regarding Voyager’s ability to advance its AAV-based gene therapy programs into first-in-human clinical trials, including the FA program in 2025; the potential of the FA program to address the underlying disease etiology of FA; and Voyager’s eligibility to receive development and commercial milestone payments, tiered royalties on net sales, and program funding under the 2019 Neurocrine collaboration agreement are forward-looking.
All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued development of Voyager’s technology platforms, including Voyager’s TRACER™ platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in animal models; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER™ capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER™ capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; the possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s current licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives.
These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.
Contacts
Trista Morrison, NACD.DC, tmorrison@vygr.com
Investors: Adam Bero, Ph.D., abero@kendallir.com
Media: Brooke Shenkin, brooke@scientpr.com
FAQ
What is the lead development candidate selected in the Friedreich’s ataxia program by Voyager Therapeutics and Neurocrine Biosciences?
When is the program expected to enter first-in-human clinical trials?
How much milestone payment is Voyager set to receive for the selection of the development candidate?
What is the potential total amount of milestone payments Voyager could receive under the collaboration agreement?
What is the strategic focus of the collaboration between Voyager and Neurocrine Biosciences?
