Voyager Provides Update on SOD1 ALS Gene Therapy Program

Rhea-AI Summary

Voyager Therapeutics (VYGR) announced a setback in its SOD1 ALS gene therapy program, as three-month non-human primate data revealed the need to assess alternate payloads for VY9323. The siRNA payload component showed off-target effects, resulting in a narrowed therapeutic window. While the novel capsid component remains unchanged, the company will no longer file its planned IND application in mid-2025.

This development extends the company's cash runway into mid-2027, excluding potential milestone payments from partnerships. Despite this setback, Voyager maintains its timeline for other programs, including expected IND filings in 2025 for GBA1 Parkinson's and Friedreich's ataxia programs, and in 2026 for VY1706 (tau silencing) gene therapy program.

Positive

• Cash runway extended into mid-2027
• Novel capsid component performing well across multiple programs
• Other gene therapy programs remain on track with planned IND filings

Negative

• SOD1 ALS program VY9323 development halted due to off-target effects
• IND application for VY9323 cancelled for mid-2025
• Timeline delay for SOD1 ALS program with uncertain new completion date

Insights

Biotechnology Research Analyst

The announcement reveals significant technical challenges in Voyager's SOD1 ALS program, with the siRNA payload component of VY9323 showing concerning off-target effects that narrow the therapeutic window. However, this setback offers several key insights:

Technical Analysis: The distinction between payload and capsid performance is important - while the payload requires redesign, the TRACER capsid platform continues to demonstrate consistent efficacy across multiple programs. This validates Voyager's core delivery technology, maintaining its value proposition in CNS-targeted gene therapies.

Pipeline Impact: The company maintains its timeline for three other significant programs:

• GBA1 Parkinson's program (2025 IND filing)
• Friedreich's ataxia program (2025 IND filing)
• VY1706 tau silencing program (2026 IND filing)

Financial Implications: The extended cash runway into mid-2027 suggests efficient resource reallocation from the delayed SOD1 program. This timeline extension, excluding potential milestone payments from partnerships, provides ample runway for advancing the remaining pipeline.

The market's reaction should consider that while the SOD1 setback is disappointing, it represents responsible development practices - identifying safety concerns early in preclinical stages rather than in human trials. The maintained strength of other programs and validated delivery platform technology suggests this is a tactical setback rather than a strategic failure.

- Company to assess alternate payloads; no changes planned to novel capsid component -

- Resulting shift of timeline and costs extends cash runway into mid-2027 –

- All other programs remain on track; IND filings expected in 2025 for GBA1 and FA programs -

LEXINGTON, Mass., Feb. 11, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced it has decided to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging three-month non-human primate (NHP) data suggest that an alternate payload would be necessary to achieve the desired product profile. No changes are planned to the novel capsid component. The same capsid in the VY1706 (tau silencing) gene therapy program achieved desired activity levels and was well-tolerated in three-month NHP studies. Voyager no longer anticipates filing an investigational new drug (IND) application for VY9323 in mid-2025. Voyager’s cash runway is now expected to extend into mid-2027; this does not include any potential milestone payments from existing partnerships.

“Emerging preclinical data indicate the siRNA payload component of VY9323 does not meet our high standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window. While we are disappointed that the development candidate VY9323 will not advance, we hope that we may be able to identify an alternate payload and find a path forward for this program, given the unmet need in ALS,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “We are encouraged that our novel TRACER capsids continue to perform consistently across multiple programs, and we believe they have the potential to transform gene therapy for CNS diseases. We continue to expect IND filings in 2025 for our gene therapy candidates for GBA1 and FA, and in 2026 for VY1706.”

The VY9323 U.S. IND application and Canadian clinical trial application (CTA) filings had previously been expected to occur in mid-2025. Given the need to assess potential alternate payloads, Voyager will provide an update on expected timing for its SOD1 ALS program when appropriate. The decision on the SOD1 ALS program does not impact Voyager’s other gene therapy programs; the Company continues to expect IND filings in 2025 from Neurocrine Biosciences for the program in GBA1 Parkinson’s and other GBA1-mediated diseases, as well as the program in Friedreich’s ataxia. Voyager also continues to expect to file an IND in 2026 for VY1706. The Company is presenting additional data at the Oppenheimer 25^th Annual Healthcare Life Sciences Conference today at 2:40 p.m. ET. A copy of the presentation can be accessed by visiting https://ir.voyagertherapeutics.com/events-presentations and will be available on the Company’s website for at least 90 days from the date of the presentation.

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; and Neurocrine Biosciences, 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,” “anticipate,” “potential,” “may,” “plan,” “future,” “suggest,” “would,” “believe,” “will,” or “continue,” and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding Voyager’s plans for the future of its SOD1 ALS gene therapy program, including its plan to assess alternate payloads for the program and expectations with respect to providing further updates on the program; Voyager’s beliefs regarding the potential cause of development candidate VY9323’s failure to achieve the desired product profile, including Voyager’s belief that such failure is not related to the capsid; Voyager’s cash runway; and Voyager’s ability to advance its AAV-based gene therapy programs and tau antibody program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates, such as the identification of lead development candidates, IND and CTA filings, the initiation of clinical trials, clinical trial enrollment, and the generation of clinical data and proof-of-concept, 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 and 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 success of Voyager’s product candidates; 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; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager’s 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: Michael Hencke, mhencke@kendallir.com
Media: Brooke Shenkin, brooke@scientpr.com

FAQ

What caused Voyager (VYGR) to halt the development of VY9323 for SOD1 ALS?

Three-month non-human primate data showed the siRNA payload component had off-target effects, resulting in a narrowed therapeutic window that didn't meet the company's standards.

How does the SOD1 ALS program delay affect VYGR's cash runway?

The timeline shift and cost adjustments have extended Voyager's cash runway into mid-2027, not including potential milestone payments from existing partnerships.

What are the expected IND filing dates for VYGR's other gene therapy programs?

Voyager expects IND filings in 2025 for GBA1 and Friedreich's ataxia programs, and in 2026 for VY1706.

Will VYGR continue development of the SOD1 ALS program?

Voyager plans to assess alternate payloads for the SOD1 ALS program and will provide updated timing when appropriate, while maintaining the current novel capsid component.