Voyager Therapeutics (NasdaqGS:VYGR) FY Conference Transcript

Summary of Voyager Therapeutics FY Conference Call Company Overview - Company: Voyager Therapeutics (NasdaqGS: VYGR) - Industry: Biotechnology, specifically focusing on gene therapy and treatments for neurodegenerative diseases, particularly Alzheimer's disease Key Points 1. Strategic Focus Areas - Three Pillars of Value: - Tau Target: Focus on tau as a target for Alzheimer's with two programs: VY7523 (anti-tau antibody) and VY-1706 (gene therapy) [2][3] - Gene Therapy: Plans to advance two gene therapy assets into clinical trials, one in partnership with Neurocrine and one wholly owned by Voyager [2] - NeuroShuttle Platform: Development of a platform to deliver various drugs across the blood-brain barrier [3] 2. Tau as a Target for Alzheimer's - Importance of Tau: The spread of tau in the brain correlates more closely with clinical decline in Alzheimer's than amyloid accumulation [6] - Recent Developments: Upcoming data from J&J and Biogen on tau-targeting therapies will provide further validation for tau as a target [5][6] - Mixed Results from Competitors: UCB's bepranemab showed some effect on tau spread but failed to meet primary clinical endpoints, indicating the complexity of targeting tau [8][9] 3. Clinical Development Insights - VY7523: Preliminary safety data shows a favorable profile, with a brain-to-plasma ratio of 0.3% and a half-life supporting monthly dosing [20] - MAD Study Design: The multiple ascending dose (MAD) study is designed to assess the effect on tau PET imaging, which is the key biomarker for evaluating the spread of pathological tau [21] - Future Studies: Plans to include tau PET imaging data in future studies and emphasize its importance over fluid-based biomarkers [24][25] 4. Gene Therapy Developments - VY-1706: Aiming for a 50%-70% reduction in tau mRNA/protein, with a focus on lower doses to enhance safety and reduce costs [31] - FDA Interactions: Productive discussions with the FDA regarding trial designs and plans to file an IND in Q2 2026 [33] 5. NeuroShuttle Platform - Differentiation: The ALPL shuttle shows longer half-life and no adverse effects on reticulocyte counts compared to transferrin receptor shuttles, making it a promising delivery method for various therapies [46] - Therapeutic Modalities: Exploring antibodies, peptides, and oligonucleotides for use with the NeuroShuttle platform [47] 6. Partnerships and Collaborations - Neurocrine Partnership: Progress on gene therapy programs, including FA and GBA1, with plans to enter the clinic this year [48] 7. Market Opportunities - Alzheimer's Disease: The potential for anti-tau therapies to be used in combination with anti-amyloid treatments, especially for patients who do not respond to the latter [41] - Broader Applications: Potential to address other tauopathies beyond Alzheimer's, expanding the market opportunities for Voyager's therapies [42] Additional Insights - Clinical Predictions: Anticipation that BIIB080 will show significant effects in clinical measures, which could influence Voyager's approach to VY-1706 [36] - Patient Population: Targeting early Alzheimer's patients for clinical trials, aligning with trends in the amyloid treatment landscape [26][27] This summary encapsulates the key discussions and insights from the Voyager Therapeutics FY Conference, highlighting the company's strategic focus, clinical developments, and market opportunities in the biotechnology sector.