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

Rhea-AI Summary

Voyager Therapeutics (Nasdaq: VYGR) has announced the selection of a third gene therapy development candidate in collaboration with Neurocrine Biosciences. This candidate, targeting an undisclosed neurological disease, utilizes Voyager's TRACER™ capsid discovery platform for intravenous administration and blood-brain barrier penetration.

The selection triggers a $3 million milestone payment to Voyager, expected in Q4 2024. This follows previous nominations for Friedreich's ataxia and GBA1 Parkinson's disease. Voyager is eligible for additional development and commercialization milestone payments, plus royalties on potential future sales.

The collaboration, established in 2023, covers multiple neurological programs. For each undisclosed program, Voyager can receive up to $175 million in development milestones, commercial milestone payments, and tiered royalties on global net sales.

Positive

• Selection of third gene therapy development candidate, expanding potential treatment pipeline
• $3 million milestone payment to be received in Q4 2024
• Eligibility for up to $175 million in development milestone payments per undisclosed program
• Potential for substantial commercial milestone payments and tiered royalties on future sales
• Advancement of SOD1 ALS gene therapy program towards expected IND in mid-2025

Negative

• Undisclosed target disease for the new development candidate, limiting investor assessment
• Dependence on Neurocrine Biosciences for program funding and development

Insights

Biotechnology Analyst

The selection of a third gene therapy development candidate in Voyager's collaboration with Neurocrine is a positive milestone, albeit with immediate financial impact. The $3 million payment, while modest, validates the progress of their partnership and the potential of Voyager's TRACER™ platform. This advancement strengthens Voyager's pipeline and financial position.

The undisclosed neurological target expands their potential market reach, complementing existing candidates for Friedreich's ataxia and GBA1-related disorders. The intravenous administration and blood-brain barrier penetration capabilities are significant technical advantages, potentially improving treatment efficacy and patient convenience.

Investors should note the substantial future milestone potential, with up to $175 million per program, plus royalties. This risk-sharing model allows Voyager to leverage Neurocrine's resources while retaining significant upside. However, the long development timelines and inherent risks in gene therapy should temper near-term expectations.

Financial Analyst

The $3 million milestone payment, while positive, is not materially significant to Voyager's immediate financial position. However, it's important to view this in the context of the broader collaboration agreement. With potential milestone payments of up to $175 million per program and tiered royalties, the long-term financial implications could be substantial.

Voyager's strategy of partnering while maintaining a wholly-owned pipeline balances risk and potential reward. The collaboration with Neurocrine offloads development costs, preserving Voyager's capital for its proprietary programs like the SOD1 ALS therapy. This approach could extend Voyager's cash runway and reduce the need for dilutive financing.

Investors should focus on the progress of all partnered programs and Voyager's ability to hit future milestones, as these will be key drivers of value and potential revenue streams in the coming years.

LEXINGTON, Mass., Sept. 16, 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, 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., M.D., Ph.D., 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.

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 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.

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,” “enable,” “future,” or “potential,” and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding the potential to advance Voyager’s SOD1 ALS gene therapy program towards an IND filing in mid-2025, and Voyager’s ability to receive development and commercial milestone payments, tiered royalties on net sales, and program funding under the 2023 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 expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyager’s preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; Voyager’s scientific approach and program development progress, and the restricted supply of critical research components; 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 possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s existing 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 success of programs controlled by third party collaboration partners in which Voyager retains a financial interest, and the success of Voyager’s product candidates; 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: Adam Silverstein, adam@scientpr.com

FAQ

What milestone did Voyager Therapeutics (VYGR) achieve in its collaboration with Neurocrine Biosciences?

Voyager Therapeutics and Neurocrine Biosciences selected a third gene therapy development candidate for an undisclosed neurological disease, triggering a $3 million milestone payment to Voyager.

How much is Voyager Therapeutics (VYGR) eligible to receive in milestone payments for each undisclosed program?

Voyager is eligible to receive up to $175 million in potential development milestone payments, plus substantial commercial milestone payments for each of the three undisclosed programs in the collaboration.

What technology is Voyager Therapeutics (VYGR) using for its new gene therapy candidate?

The new gene therapy candidate leverages an intravenously administered, blood-brain barrier-penetrant, novel capsid derived from Voyager's TRACER™ capsid discovery platform.

When does Voyager Therapeutics (VYGR) expect to file an IND for its SOD1 ALS gene therapy program?

Voyager expects to file an Investigational New Drug (IND) application for its SOD1 ALS gene therapy program in mid-2025.