Voyager Therapeutics, Inc.

Good afternoon and welcome to Voyager's Therapeutics First Quarter 2024 Financial Results Conference call. At this time, all participants are on listen-only mode. There will be a question-and-answer session at the end of this call. Please note that today's conference is being recorded. Your play of today's call will be available on the Investors section of the company's website approximately two hours after completion of this call. I would like to turn the call over to Trista Morrison, Chief Corporate Affairs Officer.

Thank you, and good afternoon. We issued our first quarter 2024 financial results press release this afternoon. The press release and 10Q are available on our website. Joining me on today's call are Dr. Al Sandrock, our Chief Executive Officer, Dr. Toby Ferguson, our Chief Medical Officer, and Dr. Todd Carter, our Chief Scientific Officer. We will also be joined for the Q&A portion of the call by our Chief Operating Officer and Principal Financial Officer, Robin Swartz. Before we get started, I would like to remind everyone that during this call, Voyager representatives may make forward-looking statements, as noted in slide two of today's deck. These statements are based on our current expectations and beliefs. They are subject to risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings, which are available on our website. And now, I will turn the call over to Al.

Thank you, Trista, and good afternoon, everyone. Please turn to slide three. I'd like to start by thanking the Voyager team for their dedication to creating transformative genetic medicines. We made tremendous progress advancing these medicines in the first quarter. We just announced that we have obtained IND clearance for our anti-Tau antibody VY-Tau-01 for Alzheimer's disease, and we expect to announce the dosing of the first subject in a single ascending dose trial in healthy volunteers in the coming week or so. Our gene therapy pipeline also advanced during the quarter, with development candidates selected in the GBA1 and Friedreich's ataxia programs partnered with Neurocrin. These programs, along with our wholly owned SOD1 ALS program, are advancing toward IND filings in 2025. In March, we appointed Dr. Toby Ferguson as our chief medical officer. Toby is an exceptional biotech executive with deep experience advancing novel therapies for CNS diseases. This includes Tofersen, the first genetically targeted therapy to be FDA approved for SOD1 ALS, and the first treatment to receive accelerated approval based on plasma neurofilament light chain response. Toby has hit the ground running. and we look forward to his leadership of our emerging clinical portfolio. In January, we announced an expansion of our relationship with Novartis through a new strategic collaboration and capsid license agreement to advance potential gene therapies for Huntington's disease and spinal muscular atrophy. This agreement, together with the public offering we completed in January, brought $200 million of total consideration to Voyager in the first quarter. This bolstered our balance sheet and extended our runway into 2027, and we expect that it will enable us to achieve multiple clinical data readouts. Finally, we presented a robust set of data at the recent ADPD and ASGCT meetings, including data on our multiple targeting programs in our second-generation capsules. Given this significant progress, we believe Voyager is emerging as a leader in neurogenetic medicine. Our pillars of value are summarized on slide four. First, we have a strong pipeline of four wholly owned and 13 partnered programs with the first expected to enter clinical trials in the coming weeks and the potential for three more to follow next year. Second, we have an industry-leading platform designed to overcome the delivery challenges inherent to CNS gene therapies. Our tracer platform enables us to create novel capsids that, following IV delivery, harness the extensive cerebrovasculature to enable widespread payload distribution across multiple brain regions and cell types. These capsids have demonstrated translatability in multiple species, and have enabled the selection of multiple development candidates in our wholly owned and partnered gene therapy programs. Third, we have blue chip partnerships anchored by Tracer's potential to transform the treatment of CNS diseases. In addition to Neurocrine, our partners include Novartis and Alexion. In total, our partnered programs could generate up to $8.2 billion in longer-term milestone payments. Finally, we continue to explore the potential to leverage receptors we have identified to shuttle non-viral genetic medicines into the brain. Ultimately, we aim to expand from gene therapy and antibodies into other modalities of neurogenetic medicine, broadening our impact. With that, I'll turn the call over to Toby.