Voyager Therapeutics, Inc.

Good day. Thank you for standing by. Welcome to the Q4 2023 Voyager Therapeutics Earnings Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star 1-1 on your telephone. You'll then hear an automated message advising your hand is raised. To respond to your question, please press star 1-1 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Peter Frenchew, Chief Financial Officer. Please go ahead.

Thank you, and good afternoon. Joining me on the call today is Dr. Al Sandrock, our CEO, and Dr. Todd Carter, our Chief Scientific Officer. We issued our Q4 and year-end 2023 financial results press release this afternoon. The press release and 10-K are available on our website. In a moment, I will turn the call over to Al. Before I do this, I want to remind everyone that during this call, VOYTRA 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, Form 10-K, filed with the FCC this afternoon. All FCC filings are available on the company's website. Now it is my pleasure to turn the call over to Al.

Thank you, Pete, and good afternoon, everyone. Please turn to slide three. I'd like to start by defining Voyager's position as an emerging leader in neurogenetic medicine. First, our pipeline. We anticipate having at least four wholly owned and partnered CNS programs in the clinic by the end of 2025, with the potential to generate clinical data in 2025 and 2026. Our most advanced programs are our anti-tau antibody for Alzheimer's disease, and our SOD1 gene therapy program for amyotrophic lateral sclerosis, or ALS. I will talk more about both programs in a few minutes. Second, our platform. Voyager is working to solve the delivery challenges inherent to CNS gene therapies with our tracer capsid discovery platform. We have demonstrated high transduction in multiple brain areas at relatively low doses, with detargeting of the liver and dorsal root ganglia across multiple species. We have also shown blood-brain barrier penetrance across multiple animal species, and we have identified a receptor that is expressed in humans. Third, partnerships. In January of 2024, we received $100 million from Novartis in a combination of upfront payment and equity investment to develop gene therapies for Huntington's disease and spinal muscular atrophy. This brings our total of partnered programs to 13, with the potential to generate $8.2 billion in longer-term milestone payments. Whereas this is a, quote, biobucks, unquote, number, it is not factored into our cash runway guidance. I will note that some of it is becoming real. Earlier this week, we triggered a $5 million milestone payment upon selection of a lead development candidate for our Nuroquin-partnered Friedreich's ataxia program. All this has given us a strong balance sheet, which we expect to provide runway into 2027, removing our financial overhang and enabling us to potentially generate value-creating clinical data in 2025 and 2026. Finally, potential. We have already demonstrated our strengths as a leader in CNS Capsid technology. We now aim to expand from gene therapy and antibodies into other modalities of neurogenetic medicine, potentially broadening our impact. We continue to explore the potential to leverage Receptor X to shuttle non-viral genetic medicines across the blood-brain barrier and look forward to sharing data on this in the future. On slide four, I want to take a moment to acknowledge just how much Borger has achieved recently. Following the Novartis collaboration, we closed a $100 million public offering. We closed 2023 with approximately $231 million in cash. When you add the $100 million from Novartis and the $100 million from the offering, that brings us to a pro forma cash number of approximately $431 million as of December 31st, 2023. We're also progressing our GLP toxicology work with our anti-Tau antibody, VY-Tau-01. for Alzheimer's disease and remain on track for an IND filing in the first half of this year. We achieved two development candidate selections with gene therapy programs, one our wholly owned SOD1-ALS gene therapy, and one with our NeuroQuint-partnered Friedreich's ataxia program. We also generated data with our wholly owned tau silencing gene therapy program, showing robust reductions in human tau mRNA and protein in a mouse model, which Todd will share more on later. All of these milestones are helping us build a robust pipeline, as you can see on slide five. I do want to note that the wholly owned programs at the top of this slide, denoted in orange, are the only programs we fund. The rest of our pipeline is funded by our partners. While I won't go into detail on all of these today, I do want to dig into some of our wholly owned programs, particularly our anti-tau antibody, our tau knockdown gene therapy, and our SOD1 ALS gene therapy. Moving to slide six. When I look at the Alzheimer's space, I'm encouraged by the progress, particularly the approval of two anti-amyloid antibodies. I view tau as the next exciting target in this field. Why? We've long known that the spread of pathological tau correlates to the progression of Alzheimer's disease. In fact, Alzheimer's disease progression is characterized by Brock staging, which is based on the spread of pathological tau. Our anti-tau antibody, VY tau 01, is differentiated from other approaches based on the epitope it targets. which is located in the C terminal rather than the N terminal or mid domain, and which has been shown to inhibit the spread of pathological tau by more than 70% preclinical study. We are currently progressing through IND enabling studies and remain on track to file an IND in the first half of this year. We plan to initiate a single ascending dose study this year in healthy volunteers. And we plan to initiate a multiple ascending dose study next year in patients with early stages of Alzheimer's disease. We hope to generate proof of concept data for slowing the spread of pathological tau via PET imaging in 2026. I'll now turn it over to Todd to talk about another approach we are developing to target tau.