Voyager Therapeutics(VYGR)2025-03-12 06:57Financial Data and Key Metrics Changes - The company reported 8.2 billion in potential future milestone payments expected to contribute significantly to the bottom line [9][10]. - Developmental milestones amount to 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].