Voyager Therapeutics, Inc.

Good morning and welcome to Voyager Therapeutics first quarter 2023 conference call. All participants on a listen-only mode. There will be a question and answer session at the end of this call. Please be advised that this call is being recorded at the company's request. A replay of today's call will be available on the investor section of the company's website approximately two hours after completion of this call. I would now like to turn the call over to Pete Frong Chu, Chief Financial Officer. Please go ahead.

Thank you, and good morning. Joining me on the call today are Drs. Al Sandrock, CEO, and Dr. Todd Carter, our Chief Scientific Officer. We issued our Q1 2023 financial results press release this morning. The press release and 10Q are available on our website. We plan to provide a brief summary of key highlights from the quarter and reserve the majority of time for Q&A. In a moment, I will turn the call over to Al. Before I do this, I want to remind everyone that during this call, Voyager representatives may make forward-looking statements, as noted in slide two of today's deck. These forward-looking statements include future expectations, plans, and prospects. All forward-looking statements are inherently uncertain and are subject to risks and uncertainties that may cause actual results to differ materially from those indicated by these forward-looking statements. You are encouraged to review and understand the various material risks and uncertainties facing the company as described in the company's most recent annual report on Form 10-K filed with the SEC. As of the filing of today's quarterly report on Form 10-Q, there have been no material changes to the risk factors described in our annual report. All SEC filings are available on the company's website. Now, it is my pleasure to turn the call over to Alex.

Thank you, Pete, and good morning, everyone. Please turn to slide three. I'd like to take a moment to recognize the incredible innovation happening right now in neurotherapeutics and in gene therapy. We believe we are witnessing a renaissance in neurotherapeutics. Just this year, the second disease-modifying therapy for Alzheimer's disease received accelerated approval, and the first drug was approved for Friedreich's ataxia. We've seen breakthroughs in treating negative symptoms of schizophrenia, something for which there are no approved therapies. Just two weeks ago, the FDA granted accelerated approval to an antisense oligonucleotide for SOD1 amyotrophic lateral sclerosis. Importantly, the FDA based the approval on the finding that treatment-driven reductions in neurofilament are reasonably likely to predict clinical benefit in SOD1 ALS patients. establishing a precedent for a biomarker-based path to accelerated approval. I hope this will drive further new treatments for patients suffering from this devastating disease. At the same time, the gene therapy field is coming of age. We have recently seen the FDA approval of the first gene therapy for hemophilia B. Gene therapies offering important potential advances in treating Duchenne's muscular dystrophy and hemophilia A are approaching sedupa dates. And through that, we may see the accelerated approval path utilized for a gene therapy. Importantly, long-term data on Zolgensma, one of the first gene therapies approved, recently demonstrated durability of effect after 7.5 years, which physicians have called transformational. Voyager sits at the intersection of neurotherapeutics and gene therapy, and we believe we are uniquely positioned to leverage the advancements in both fields. To date, the delivery of gene therapies into the central nervous system has proven challenging. Approaches to inject these therapies into the brain parenchyma or various CSF spaces have not been very successful. Voyager's tracer capsid discovery platform is the foundation of our approach to solving this delivery challenge. Voyager scientists have engineered multiple capsid libraries, each with more than 20 million novel variants of AAV9 and AAV5 capsids to select those novel capsids that display greatly increased transduction in the central nervous system following intravenous delivery. We have leveraged these capsids to advance our own and our partners' CNS gene therapy programs, several of which are now advancing towards clinical trials. This is how Voyager is enabling the future of neurogenetic medicine, and from where I sit, it's an incredibly exciting place to be. Moving to slide four, I will briefly review our investment rationale. Our first pillar of value is our tracer capsule discovery platform, which I just discussed. In the preclinical study, our novel capsids delivered intravenously have demonstrated more than 100-fold higher transgene expression in the brain compared to conventional AAV9 capsids. We have shown high levels of CNS gene expression at low doses, and we have demonstrated the ability to target specific cells such as neurons or glia while detargeting the liver and dorsal root ganglion cells. We look forward to sharing more data on our capsids at ASGCT later this month. Our second pillar of value is our CNS pipeline. We are advancing four programs through late research and towards IMD. Two of these programs are wholly owned, our humanized anti-Tau antibody for Alzheimer's disease and SOD1 gene therapy program for ALS. The other two, our GBA-1 gene therapy program for Parkinson's disease, and our frataxin gene therapy program for free dry phataxia, are being co-developed with NeuroPrint. Our third pillar of value is partnerships. We completed TAPS at option and license agreements with Pfizer and Novartis. We've executed strategic collaborations around our pipeline programs with NeuroPrint. and we are exploring more such transactions. Turning to slide five, we continue to make progress advancing our CNS pipeline. I'll note a few recent highlights. During the first quarter, we selected a lead humanized anti-Tau antibody candidate, VY-Tau01, for the treatment of Alzheimer's disease. In March, we presented new data at the ADPD meeting highlighting the differentiating characteristics of this lead candidate. Last month, we received pre-IND written feedback from the FDA for BY-PAL01. Borger continues to expect to initiate GLP toxicology studies this year to enable an IND filing in the first half of 2024. Another change this quarter was to the timeline for our SOV1 ALS gene therapy program. Voyager previously said we expected to identify a lead development candidate for this program in the first half of this year. That has moved out to the second half of this year as we continue to evaluate data from this program to identify the optimal development candidate. We intend to provide updated guidance on the IND timeline once we select the development candidate Given where we are today, we expect the IMD to occur in mid-2025. Our frataxin gene therapy program for plebryx ataxia and our GBA1 gene therapy program for Parkinson's disease and other GBA1-mediated diseases both continue to advance in collaboration with Nurocrine. I'm pleased with the progress we're making here. Additionally, during the first quarter, We launched two new early-stage gene therapy programs combining vectorized siRNAs with our novel intravenous tracer capsids. One combines two siRNAs to enable specific knockdown of mutant HCT and MSH3 for the treatment of Huntington's disease. The other uses siRNA to reduce tau expression in the brain for the treatment of Alzheimer's disease. I'd like to now turn the call over to Pete Moinshew to discuss our financial results for the quarter.