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Voyager Demonstrates ALPL Receptor-Mediated Blood-Brain Barrier Transport of Novel AAV Capsids in Molecular Therapy Publication
Globenewswire· 2025-05-15 11:00
Core Insights - Voyager Therapeutics has published data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB) [1] - The research indicates that the novel AAV capsid VCAP-102 shows a 20- to 400-fold increase in gene transfer across multiple brain regions compared to AAV9 in both rodents and non-human primates [2] - The company is advancing two gene therapy programs towards IND filings this year with a partner, leveraging the findings from the ALPL research [2] Group 1: Research and Development - The publication in Molecular Therapy outlines the generation of VCAP-102 and identifies ALPL as the primary receptor for crossing the BBB [2] - Voyager's next-generation capsids have demonstrated significant transduction rates, with up to 98% of dopaminergic neurons in the substantia nigra being transduced [2] - The TRACER™ capsid discovery platform enables rapid discovery of novel AAV capsids, facilitating gene therapy for neurological diseases [3] Group 2: Strategic Partnerships and Pipeline - Voyager's pipeline includes programs targeting Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and ALS, among others [4] - The company collaborates with partners such as Alexion, AstraZeneca Rare Disease, Novartis Pharma AG, and Neurocrine Biosciences to advance its gene therapy programs [4] - The multi-modality approach of combining viral and non-viral delivery methods is seen as critical for addressing unmet needs in neurological diseases [3]
Voyager Therapeutics (VYGR) Reports Q1 Loss, Lags Revenue Estimates
ZACKS· 2025-05-06 22:10
分组1 - Voyager Therapeutics reported a quarterly loss of $0.53 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.35, and compared to a loss of $0.20 per share a year ago, indicating an earnings surprise of -51.43% [1] - The company posted revenues of $6.47 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 66.26%, and down from $19.52 million in the same quarter last year [2] - Voyager Therapeutics shares have declined approximately 38.5% since the beginning of the year, contrasting with the S&P 500's decline of -3.9% [3] 分组2 - The current consensus EPS estimate for the upcoming quarter is -$0.31 on revenues of $19.19 million, and for the current fiscal year, it is -$1.41 on revenues of $90.91 million [7] - The Medical - Biomedical and Genetics industry, to which Voyager Therapeutics belongs, is currently ranked in the top 31% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]
Voyager Therapeutics(VYGR) - 2025 Q1 - Quarterly Report
2025-05-06 20:02
Pipeline Development - The company is advancing a proprietary pipeline focused on neurological diseases, particularly Alzheimer's disease (AD), with two key programs targeting tau: VY7523 and VY1706[74] - VY7523, an anti-tau antibody, reduced tau spread by approximately 70% in preclinical studies and is currently in a Phase 1 multiple ascending dose clinical trial, with initial data expected in the second half of 2026[74] - VY1706, a gene therapy targeting tau mRNA, demonstrated a 50% to 73% reduction in tau mRNA levels in non-human primates and an investigational new drug application is anticipated in 2026[74] - The GBA1 program targets both Gaucher and Parkinson's diseases, with clinical trials expected to begin in 2026[76] - The TRACER platform is utilized across all gene therapies in the pipeline, enabling rapid discovery of AAV capsids with enhanced CNS tropism[77] Collaborations and Partnerships - The company has partnered with Neurocrine on five gene therapy programs, with milestone payments expected to total up to $35 million related to clinical trials for the GBA1 and FXN programs in 2025-2026[75] - The collaboration with Novartis includes an upfront payment of $80 million and potential milestone payments totaling $625 million across two programs, with royalties in the high single-digit to low double-digit percentages[81] - Total partnerships have provided over $500 million in non-dilutive funding, with the potential to earn up to $7.4 billion in milestone payments across the partnered portfolio[76] - The company has initiated a collaboration agreement with Neurocrine for four programs, with Neurocrine covering all preclinical development costs[86] - The 2023 Neurocrine Collaboration Agreement includes potential development milestone payments of up to $985.0 million for the GBA1 Program and up to $175.0 million for each of the three 2023 Discovery Programs[87] - The company received a $3.0 million milestone payment in October 2024 following the selection of a development candidate for a gene therapy program under the 2023 Neurocrine Collaboration Agreement[87] Financial Performance - The company reported a net loss of $31.0 million for the three months ended March 31, 2025, compared to a net loss of $11.3 million for the same period in 2024[108] - Research and development expenses increased to $31.5 million for the three months ended March 31, 2025, up from $27.1 million in the same period in 2024[108] - The accumulated deficit as of March 31, 2025, was $357.2 million, indicating ongoing significant losses[95] - The company anticipates continued significant expenses and operating losses due to ongoing clinical trials and research activities[95] - Collaboration revenue decreased to $6.5 million for the three months ended March 31, 2025, down from $19.5 million in the same period of 2024[109] Cash Flow and Funding - Net cash used in operating activities was $37.9 million in Q1 2025, compared to a net cash provided of $58.8 million in Q1 2024[119] - Net cash provided by investing activities was $41.2 million in Q1 2025, a significant improvement from $96.1 million used in Q1 2024[121] - Net cash provided by financing activities decreased to $0.1 million in Q1 2025, down from $112.9 million in Q1 2024[122] - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $295.1 million[124] - The company expects to need substantial additional funding to support ongoing research and development and operational expenses[123] Market Risks and Economic Factors - The company is exposed to market risk related to interest rate changes, with policies in place to manage this risk[134] - The company is not currently exposed to market risk related to changes in foreign currency exchange rates[135] - Future contracts with vendors located in Asia and Europe may subject the company to fluctuations in foreign currency rates[135] - Inflation has generally increased the costs of labor, goods, and services for the company[135] - The company does not believe that inflation had a material effect on its business during the three months ended March 31, 2025[135]
Voyager Therapeutics(VYGR) - 2025 Q1 - Quarterly Results
2025-05-06 20:01
Financial Performance - Voyager ended Q1 2025 with a cash position of $295 million, expected to provide runway into mid-2027[2] - Net loss for Q1 2025 was $31.0 million, compared to a net loss of $11.3 million in Q1 2024, representing an increase of 174.0%[8] - Voyager's total operating expenses for Q1 2025 were $41.2 million, compared to $35.7 million in Q1 2024, an increase of 15.2%[8] - Voyager's total assets decreased to $353.2 million as of March 31, 2025, down from $393.1 million as of December 31, 2024[19] Revenue - Collaboration revenue for Q1 2025 was $6.5 million, down from $19.5 million in Q1 2024, a decrease of approximately 66.8%[8] - GAAP collaboration revenue for Q1 2025 was $6.473 million, a decrease from $19.516 million in Q1 2024[23] - Net collaboration revenue for Q1 2025 was $4.845 million, down from $16.338 million in Q1 2024[23] Expenses - Research and development expenses increased to $31.5 million in Q1 2025 from $27.1 million in Q1 2024, a rise of 16.3%[8] - General and administrative expenses rose to $9.6 million in Q1 2025 from $8.6 million in Q1 2024, an increase of 11.6%[8] - Total research and development expenses for Q1 2025 increased to $31.526 million, compared to $27.092 million in Q1 2024[23] - Net research and development expenses for Q1 2025 were $29.898 million, up from $23.914 million in Q1 2024[23] - Reimbursable research and development services incurred in Q1 2025 amounted to $1.628 million, down from $3.178 million in Q1 2024[23] Clinical Development - The company anticipates IND submissions for the Neurocrine-partnered FA and GBA1 programs in 2025[3] - Initial tau PET imaging data from the MAD clinical trial of VY7523 is expected in the second half of 2026[3] - A single IV dose of VY1706 achieved up to 73% knockdown of tau mRNA in NHPs[5]
Voyager Reports First Quarter 2025 Financial and Operating Results
GlobeNewswire News Room· 2025-05-06 20:01
Core Insights - Voyager Therapeutics reported a strong cash position of $295 million, expected to last until mid-2027, excluding potential milestone payments from partnerships [2][8] - The company is advancing its pipeline, particularly focusing on tau-targeting programs for Alzheimer's disease, with IND submissions anticipated in 2026 [2][5] Financial Results - Collaboration revenue for Q1 2025 was $6.5 million, down from $19.5 million in Q1 2024, primarily due to decreased revenue from Neurocrine collaboration [14] - Net loss for Q1 2025 was $31.0 million, compared to $11.3 million in Q1 2024, attributed to lower collaboration revenue and increased operating expenses [14][19] - Research and development expenses increased to $31.5 million in Q1 2025 from $27.1 million in Q1 2024, driven by higher program-related spending [14][19] Pipeline and Development Updates - The tau silencing gene therapy VY1706 demonstrated up to 73% knockdown of tau mRNA in non-human primates after a single intravenous dose [5] - The company is preparing for IND submissions for VY1706 and the Neurocrine-partnered Friedreich's ataxia and GBA1 programs in 2026 [6][14] - Voyager's anti-tau antibody VY7523 is currently in a multiple ascending dose clinical trial for Alzheimer's Disease, with initial tau PET data expected in the second half of 2026 [6][14] Upcoming Milestones - Multiple presentations at the ASGCT 2025 annual meeting are planned, including data on VY1706 and VY7523 [5][6] - The initiation of clinical trials for the FA and GBA1 programs is expected in 2026, with potential milestone payments totaling $35 million [6][14]
Voyager Next-Generation CNS Capsids Featured at ASGCT 28th Annual Meeting
Globenewswire· 2025-04-28 20:30
Core Insights - Voyager Therapeutics announced eight presentations at the ASGCT annual meeting, focusing on advancements in gene therapies for neurological diseases, particularly Alzheimer's disease [1][2] Group 1: Gene Therapy Developments - The company highlighted its TRACER capsids, which have shown the ability to transduce 43%-98% of neurons and 87%-99% of astrocytes in non-human primates after a single intravenous dose [2] - The tau silencing gene therapy VY1706 demonstrated up to 73% knockdown of tau mRNA in non-human primates following a single intravenous dose of 1.3e13 vg/kg [2][6] - Presentations will include data on anti-amyloid gene therapy and enhancements to capsids aimed at immune evasion to broaden patient eligibility [2][6] Group 2: Presentation Details - Oral presentations will cover various topics, including the intravenous delivery of VY1706 and its effects on tau levels in the central nervous system [7] - Other presentations will focus on the discovery of AAV9-derived capsids that evade neutralizing antibodies and machine-learning applications for capsid screening [7][8] Group 3: Company Overview - Voyager Therapeutics is dedicated to leveraging human genetics to treat neurological diseases, with a pipeline that includes programs for Alzheimer's disease, Friedreich's ataxia, and Parkinson's disease [9] - The TRACER platform has enabled the development of novel capsids for gene therapy, enhancing the potential for high brain penetration following intravenous dosing [9][8]
Voyager Presents Robust Preclinical Data from Tau Targeting Gene Therapy and Antibody Programs at AD/PD™ 2025
Newsfilter· 2025-03-31 11:00
- Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution and liver de-targeting, in NHP study - - Preclinical murine data strengthen case for specifically targeting pathologic forms of tau with the clinical-stage anti-tau antibody VY7523 - - Voyager to host live webcast on April 7 recapping key AD/PD™ 2025 data - Dr. Ferguson will be speaking on two panels at AD/PD™ 2025: Challenges and Opportunities for Anti-Tau Therapi ...
Bragar Eagel & Squire, P.C. Is Investigating Dave, Red Cat, Perpetua, and Voyager and Encourages Investors to Contact the Firm
Globenewswire· 2025-03-21 01:00
Core Viewpoint - Bragar Eagel & Squire, P.C. is investigating potential claims against four companies: Dave, Inc., Red Cat Holdings, Inc., Perpetua Resources Corp., and Voyager Therapeutics, Inc. for possible violations of federal securities laws and other unlawful business practices [1] Company Summaries Dave, Inc. (NASDAQ: DAVE) - The Justice Department and FTC announced a civil enforcement action against Dave, Inc. and its CEO Jason Wilk for alleged violations of the FTC Act and ROSCA, claiming misleading advertising regarding cash advances, hidden fees, and recurring monthly fees without a cancellation mechanism [2] - Following the announcement, Dave shares opened at $84.00 on December 31, 2024, reflecting a drop of over 10% from the previous day [3] Red Cat Holdings, Inc. (NASDAQ: RCAT) - Kerrisdale Capital published a report alleging that Red Cat overstated the revenue potential of its U.S. Army SRR drone contract and lacks the production capacity to fulfill its promises, raising concerns about executive departures and insider transactions [4] - As a result, Red Cat's stock price fell by $2.36 per share, or 21.63%, closing at $8.55 per share on January 17, 2025 [4] Perpetua Resources Corp. (NASDAQ: PPTA) - The investigation focuses on whether Perpetua issued false or misleading statements and failed to disclose pertinent information. The company released an updated cash flow model for the Stibnite Gold Project, indicating increased capital expenditures and costs, but improved economic metrics due to rising commodity prices [5] - Following this news, shares of Perpetua fell by 22.3% on February 14, 2025 [5] Voyager Therapeutics, Inc. (NASDAQ: VYGR) - Voyager announced it would assess alternate payloads for its gene therapy program for ALS, stating that emerging preclinical data indicated issues with the siRNA payload component of VY9323, which does not meet their standards [6] - Consequently, Voyager's stock price dropped by $1.11 per share, or 20.86%, closing at $4.21 per share on February 11, 2025 [6]
Voyager Therapeutics(VYGR) - 2024 Q4 - Earnings Call Transcript
2025-03-11 23:35
Financial Data and Key Metrics Changes - The company reported $332 million in cash as of the end of 2024, with $8.2 billion in potential future milestone payments expected from partnerships, indicating a strong financial position [9][10]. - Developmental milestones amount to $2.9 billion, with total potential milestone payments exceeding $8 billion, providing significant upside to the company's cash runway guidance extending to mid-2027 [28][30]. Business Line Data and Key Metrics Changes - The SOD1 silencing gene therapy program has moved back into the research stage due to the payload not meeting target profiles, while the VY1706 tau silencing gene therapy has advanced into IND-enabling studies [14][15]. - The company is seeing promising results from non-human primate studies, with 50% to 73% knockdown of tau messenger RNA across the brain [16][111]. Market Data and Key Metrics Changes - The anti-tau antibody, VY7523, has shown favorable results in a single ascending dose study, with no serious adverse events reported and a CSF to serum ratio of 0.3%, consistent with other approved monoclonal antibodies for Alzheimer's treatment [17][126]. - The company is closely monitoring third-party data from competitors in the anti-tau space, which could influence its own programs and strategies [101][105]. Company Strategy and Development Direction - Voyager Therapeutics is focused on building a multimodal neuro-therapeutics company, emphasizing gene therapy as a key component of its pipeline, particularly targeting tau in Alzheimer's disease [8][11]. - The company is open to additional partnerships to enhance its pipeline and revenue streams, indicating a proactive approach to business development [10][134]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of its tau-targeting programs, highlighting the importance of genetically validated targets in severe diseases with high unmet needs [12][18]. - The company acknowledges the competitive landscape in the tau space and is prepared to adapt its strategies based on emerging data from both its own studies and those of competitors [144]. Other Important Information - The company is exploring the applicability of its ALPL-based non-viral shuttle for various diseases, with ongoing assessments of its potential to transport proteins, antibodies, and oligonucleotides across the blood-brain barrier [37][81]. - The management emphasized the importance of understanding the mechanisms of action for both antibody and knockdown approaches in treating Alzheimer's disease, indicating a comprehensive strategy to address the complexities of the disease [140][144]. Q&A Session Summary Question: Insights on upcoming data at ADPD - Management anticipates data from bepranemab regarding exposure PD relationships and the necessary decrease in tau spreading for clinical relevance [22][24]. Question: Upcoming milestones from partnered programs - The company confirmed that developmental milestones are not included in the current cash runway guidance, representing additional upside potential [30]. Question: Details on tau gene silencing IND - The main requirement before filing the IND is completing the GLP tox study and ensuring a therapeutic window [35]. Question: Applications of the ALPL shuttle - The ALPL shuttle is being evaluated for its ability to transport various modalities across the blood-brain barrier, with ongoing assessments of its effectiveness [37][81]. Question: Differences between tau silencing and antibody approaches - The knockdown approach is believed to provide broader tau knockdown and may be more effective in certain patient populations compared to antibody therapies [48][141]. Question: Cash runway and investment strategy - The company plans to balance investments in internal programs with potential licensing opportunities, remaining open to various partnership structures [132][134].
Voyager Therapeutics(VYGR) - 2024 Q4 - Earnings Call Transcript
2025-03-12 06:57
Financial Data and Key Metrics Changes - The company reported $332 million in cash as of the end of 2024, with $8.2 billion in potential future milestone payments expected to contribute significantly to the bottom line [9][10]. - Developmental milestones amount to $2.9 billion, with total potential milestones exceeding $8 billion, providing a significant upside to the mid-2027 cash runway guidance [28][30]. Business Line Data and Key Metrics Changes - The SOD1 silencing gene therapy program has moved back into the research stage due to the payload not meeting target profiles, while the VY1706 tau silencing gene therapy has advanced into IND-enabling studies [14][15]. - The anti-tau antibody, VY7523, showed promising results in a single ascending dose study, with no serious adverse events reported and a CSF to serum ratio of 0.3% [16][17]. Market Data and Key Metrics Changes - The company is focusing on the tau target in Alzheimer's disease, which is viewed as critical, with multiple upcoming data readouts expected to generate excitement in the market [8][18]. - The recent data from UCB's bepranemab indicated that an anti-tau antibody can impact tau accumulation in the human brain, although it did not meet its primary endpoint [17][18]. Company Strategy and Development Direction - The company aims to build a multimodal neuro-therapeutics platform, emphasizing gene therapy as a key component of its pipeline [11][12]. - Partnerships have been a significant source of non-dilutive revenue, and the company remains open to additional business opportunities [10][11]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential of their IV-delivered AAV capsids to efficiently deliver therapies across the blood-brain barrier, which is critical for treating CNS diseases [13][18]. - The management acknowledged the challenges in the gene therapy field but emphasized the importance of focusing on genetically validated targets in severe diseases with high unmet needs [11][12]. Other Important Information - The company is preparing to share data from its non-human primate studies, which showed a 50% to 73% knockdown of tau mRNA across the brain [16][17]. - The company is also exploring the applicability of its ALPL-based non-viral shuttle for various diseases, including spinal cord and cerebral cortex conditions [37][39]. Q&A Session Summary Question: Insights on upcoming data at ADPD - Management anticipates data from bepranemab regarding exposure PD relationships and the necessary decrease in tau spreading for clinical relevance [22][24]. Question: Upcoming milestones from partnered programs - The company has $2.9 billion in developmental milestones, with additional upside to the mid-2027 cash runway guidance [28][30]. Question: Details on tau gene silencing IND - The main requirement is completing the GLP tox study and ensuring a therapeutic window [35]. Question: Applications of the ALPL shuttle - The ALPL shuttle is being assessed for various diseases, with potential for transporting proteins, antibodies, and oligonucleotides [37][39]. Question: Differences between tau silencing and antibody approaches - The knockdown approach may allow for broader tau knockdown and is administered via IV, which could be more beneficial for patients [45][46]. Question: Proof of concept for SOD1 gene silencing - The next opportunities for capsid proof of concept lie with the Friedreich's ataxia and GBA programs, which are partnered with Neurocrine [52][53]. Question: Comparison of ALPL shuttle to other shuttles - The ALPL shuttle may have advantages over TfR-based shuttles, particularly regarding safety and distribution [149][150]. Question: Future indications for tau silencing program - The company intends to focus on Alzheimer's disease first, given the extensive knowledge of its natural history and measurement techniques [114][118].