Welcome to Oppenheimer's 34th Annual Healthcare Conference. I'm Jay Olson, one of the biotech analysts at Oppenheimer, and I want to thank you all for joining us here today. It's my pleasure to welcome Voyager Therapeutics to our conference, and it's an honor to introduce Peter Pfreundschuh, the Chief Financial Officer. If you have any questions during the discussion, please feel free to submit them using the Q&A function. With that, we'll get started. Thanks for joining us, Pete. Jay, thank you very much for the invitation to present at your conference this year. We always appreciate the invite, and we appreciate the partnership with Oppenheimer. So I'm looking forward to taking your participants through our corporate presentation. And look forward to some Q&A at the back end of it. So Voyager Therapeutics, what are we all about? We're actually at the cusp of defining neurogenetic medicines, and we're really excited about the state of the business and where we are today. We've made tremendous strides in progress as a company over the last couple of years, and we're more than happy to share some of that with you all. What the future looks like for the company as we move forward over the next couple of years. Before I get started, here's obviously a set of our forward-looking statements. I'm not going to get into any detail with regards to these forward-looking statements, but please familiarize yourself with regards to them. As we go through the materials. So Voyager is really predominantly fundamentally invested around four key pillars of value. Our pipeline of wholly owned programs that we're developing ourselves in combination with some of our partners. And we're really advancing very aggressively. We should have at least four IND filings expected during 2024 and 2025 between ourselves and our partners. And we believe those programs will generate clinical data in the 2025-2026 timeline. Our platform, which is our highly engineered capsid platform TRACER. Which really has been able to show preclinically that across multiple capsid families and various species of animals. That we really have some very unique capsids that can deliver CNS gene therapy. To the deep gray structures of the brain, also do targeting and detargeting. And we've been able to show very good transduction rates in multiple brain areas. A very low 2 × 10^12 vg/kg in marmosets. That was at ESGCT in 2023. Our partnerships, we've got a number of really high-class blue chip partnerships, most notably. Earlier this year, we actually executed a partnership with Novartis. It was our second partnership with them. We're really excited about that partnership. It is around our HD program. As well as an SMA program. All those partnerships, the 13 of them. Actually create a very strong cash position over the last 18 months. We've been able to raise over $300 million of cash. Including some various other things, and that provides the company with great runway into 2027. To be able to advance our wholly owned pipeline. As well as continue to advance our platform and other things that we're working on. Finally, the potential, the potential of being able to leverage and expand the platform, the TRACER platform, and the capsids that we've engineered. Because we've been able to actually identify the receptors associated with some of these families of capsids. And so first and foremost, Receptor X, which is in one of our families, we've been able to show that this is a humanized receptor. We believe that that potentially can allow for the ability of delivery of other molecules across the blood-brain barrier, and we're really excited about where we can take that as an organization. As we move forward, so we'll talk more about each one of these key pillars of value as we get into the presentation. But let's first talk about our wholly owned pipeline. So our wholly owned pipeline in combination with some of our partnerships really is largely focused in and around Alzheimer's disease. Most notably, our most advanced program is an anti-tau antibody program, which we plan on filing an IND in the first half of this year around. Followed by a SOD1 program for silencing. Specifically around SOD1. And that is, we picked a development candidate late last year with regards to that program. So that program is moving forward very nicely. We should file an IND in the middle of 2025. And we're very excited about where that's going. We have two earlier stage research pipeline programs. One specifically a tau knockdown program for silencing gene therapy. And then an Anti-Ab program for vectorized antibody here. We're really excited about the advancement of those, and we're going to talk in a little bit more detail as we get into the presentation around those. On the partnership side of things, there are two types of partnerships that we've been able to strike as a company. These are partnerships where we get reimbursed and we actually collaborate with our partners, both Neurocrine and Novartis, around the advancement of those programs. Those include both GBA1, Friedreich's ataxia, and then also some other additional programs with Neurocrine. There's five gene therapy programs around undisclosed targets. Most notably, and very recently, we struck a deal with Novartis that included both a program that we were advancing ourselves around Huntington's disease, and then also around SMA with regards to the capsid. So we'll talk a little bit more in detail later in the presentation around some of those. But our partnerships really are either programs that we're partnering with our partner to advance. And then at some point along the way, we'll hand those over. Or they're strictly capsid type partnerships where we actually license our capsid technology against a specific target. I do want to highlight before I get off this page that two of the programs where we do get reimbursed in combination with Neurocrine, we do retain co-promotion co-development rights relative to those programs, and we retain value associated with the U.S. marketplace. Those are around GBA1 and Friedreich's ataxia. Go to the next slide. Our big focus is really in and around kind of Alzheimer's disease as an organization. There are 6 million people in the United States alone who are obviously living with some form of Alzheimer's disease. We're a big believer as an organization that multiple approaches are needed. Similar to like in the spaces of oncology to treat these patients. So we really focused in around kind of amyloid and tau relative to the validation of those targets and where we believe we can actually take therapeutic care. For us personally, you know, we're very big believers in a potential efficient path to proof of biology for both amyloid as well as tau. There are good ways to track clinical biomarkers associated with this, either through amyloid or tau pet tracers. So we're big believers in, as we advance these programs, being able to measure that clinically speaking and be able to kind of show that we really do have some assets and programs that are very unique and novel here. Why is our tau antibody program so unique and differentiated? I think that's largely, and we'll get into that a little bit further on the next slide. It targets the C-terminus domain. It has a unique epitope to it. Multiple programs, as we know, have really kind of advanced but failed largely in the tau antibody space. But most of those programs really have been focused in around kind of the N-terminus. We believe through some of the preclinical work that we did in mouse seeding models that we've actually been able to show the inhibition of the spread of pathological tau associated with our C-terminus antibody approach. As I mentioned earlier, this program is in GLP tox and that's underway. We plan to file an IND in the first half of 2024, and we're very excited about that program. As I mentioned earlier as well, there's two earlier programs, one in tau silencing. Then an anti-amyloid program that we're also advancing along here. We plan to hopefully have some further data and information as we advance these programs for our investors and for patients out there. This slide very simply summarizes why we believe our antibody is actually differentiated both by a terminal perspective as well as epitope. On the right hand side of the page, you'll see the data from AAIC in 2022 really showing the inhibition of the spread of pathological tau in the mouse seeding model. This is this P301 study that we did. Where we actually showed it both in the hippocampus as well as the contralateral, about a 70% reduction overall. On the left hand side of the. You know, through various studies that we've seen both preclinically as well as others, mouse seeding model. As well as the clinical results around the internal antibodies. We don't believe these are as effective, and so this is really a key differentiating feature associated with our antibody program. Shifting gears a little bit and talking a bit about ALS. So from an ALS perspective, this is an area of high unmet medical need. There's about 20,000 people in the United States with ALS. Obviously, SOD1 is a mutation that is very prevalent. We believe that through the approval of tofersen, which was the first ASO targeting SOD1, that that really has demonstrated very good clinical efficacy. But that our gene therapy approach could really follow with a much more durable solution for these patients. And in treating SOD1 patients here. SOD1, obviously from a path of proof of biology, there is very measurable elements to it in the CSF. We believe NfL obviously is a measurable element within the plasma, and so this is a way that we can actually validate this program as we move forward into the clinic and start showing some data and information here. I mentioned earlier that we selected a development candidate actually at the end of last year in December. And we do plan to file an IND in mid-2025. And really lay the foundation towards potentially generating further data and information around this program. So what's unique about our kind of overall organization is fundamentally it really kind of. We repivoted the organization back in 2021 really around kind of our tracer platform in our novel capsids, and why that really has been so revolutionary is because I think it starts to really set the foundation for the way we should think about delivering. You know, gene therapy agents, but specifically neuro CNS gene therapy agents. And you can see from some of the quotes from various parties on this page that we truly do believe that we're revolutionizing the delivery of those gene therapy opportunities. We do have these class leading blood-brain barrier penetrating capsids, and you know, we're very excited about where this could go. On the right hand side of this page, as we talk a little bit why they have such potential to really kind of be game changing. You can clearly see both when you actually study this in mouse brain as well as non-human primates. Where we've been able to take these engineered capsids from the parental capsid and really be able to kick that up a notch, so to speak. From 1st-gen capsids to 2nd-gen capsids and really get greater than 100-fold improvement in the CNS across species. So we're really excited about how we've been able to prove this, and we've done that through a number of different ways. Through the receptor identification, which I mentioned earlier in the conversation. Through validation of cross species from mice to marmosets to monkeys. Through the ability of really showing improved broad CNS transduction rates. I mentioned that at the start of the presentation. To being able to show that we can actually manufacture these novel capsids and really kind of take them down the road from an engineering and scalability perspective. Then really the capsids' ability to be customizable around specific cell tropisms. So really focusing largely in around kind of neurons or glial cells, both targeting as well as detargeting. I think this is really the novel element of the capsids that we've been able to develop and engineer here. And overall, the thing that also makes them very unique is that they're minimally invasive and IV delivered, and so you can get very broad brain distribution when you deliver, you know, kind of a tracer capsid that way. And so this is, we believe this is the future of really delivering genetic medicines and most notably really gene therapy agents for CNS neuro. I won't get into a lot of the details on this slide, but obviously from that work that we did in and around TRACER and with our capsids, we've been able to identify the receptors associated with some of our capsids, and we're really excited about that work and where that potentially could take us as well as our organization. Ultimately speaking, you know, the ability to do that. You can then start thinking about different modalities that you could deliver across the blood-brain barrier and really kind of thinking about how you therapeutically treat patients from a multimodality perspective. And I think the real future of being able to treat some human diseases that are challenging, especially from a CNS perspective, is in thinking about this a little bit more broadly. And so we're really excited about the work that we've done here. From a business perspective, the company is well situated, and I think I alluded to at the start of this presentation that we struck a deal with our second deal with Novartis earlier this year. Tied to that deal, we also did a subsequent public offering on the heels of that. And so, you know, that brought us tremendous consideration. Obviously, the validation of the Novartis deal is kind of in some of the pre-work that we did to that. We actually did our original option to license agreement with Novartis back in 2022. Where they took an option on three targets exclusively. Ultimately, they triggered two. And you know, that was to our capsids, which are not exclusive on our side of the equation. They really were able to, you know, do a lot of work scientifically in their own laboratories with regards to our capsids and be able to validate kind of what we were seeing ourselves. And I think ultimately that what that did was that led to this second agreement around both HD as well as SMA, and we're really excited about this second agreement because as many of you know, you know, Novartis is a world leader in gene therapy and the development of gene therapy programs and commercial products. We believe that they're actually a great partner with regards to both HD as a target. As well as SMA, of course, on the SMA side of the equation, they already have a commercially viable product in Zolgensma, which is doing quite well. And so we're really excited about what this can bring in terms of both a partnership with them and what we can learn together as we move down the road in terms of the advancement of both the HD as well as SMA programs. We did complete a public offering, a secondary on the heels of this transaction. When you look at that in total, that really brought us a tremendous amount of money into our coffers, and we'll talk about that in a second relative to the runway of the organization. So that being said, you know, the Novartis deal that we most recently did builds on a series of other blue chip portfolio collaboration and licensing agreements that we've done. Most notably really over the last 18+ months, but there's some older ones here. This in total really has potential milestones across all 13 of these partnered programs. In excess of $8 billion, and none of this is really currently factored into our runway analysis. Where the company's ability to advance fund and develop further science both around our wholly owned programs, but also around our platform and other things that we would like to invest in. And so we're really excited about both the partnerships, what they can bring for the organization, but where we can take take the organization as we move forward in the future. So from a financial perspective, post Novartis and post the secondary offering, the company is in great shape. If you focus in the middle of this page here, you know, we pretty much closed last year with preliminary unaudited cash of $230.9 million. That was a good strong balance sheet to begin with. It gave us plenty of runway as we told the public into the middle of 2025. But now with the additional financing funding associated with Novartis and the offering, the company really has an excess of $400 million on its balance sheet. So this drives us really into 2027, and it allows us to advance our programs, our wholly owned programs, without any type of financial challenges or overhang. We should be able to drive to a number of considerable milestones over the next couple of years on both our wholly owned programs, tau, as well as the SOD1 program for ALS, as well as some of our partnered programs. We're really excited about that because we believe those are real material value inflection points for the company and will help the company drive north from here. So just a little bit of a recap with regards to, you know, some most recent milestones that we achieved as well as some milestones to come up over the near-term future. You know, recent milestones that we achieved obviously were the Novartis collaboration around HD and SMA with a $100 million investment. It was $80 million for the programs and a $20 million equity component. And then on top of that, we did the public offering $400 million. So that brought us over $400 million. You on the previous page you see almost $431 million pro forma as of the close of the offering, not including financing fees. And you know that then allows us to really drive the milestones that we've got for the company as we move forward. We're really excited about some of those that will be the IND filing on the VY-TAU01 program, obviously in the first half of 2024, as I mentioned earlier. Both, you know, a single ascending dose of phase I dose in 2024 on that program and then moving into a multiple ascending dose trial in 2025 on that program. The thing I would stress is there's other milestones with some of our partners during this time period as well. We believe, you know, some of our partners are gonna file INDs, hence the reason why we say we're gonna have between our wholly owned programs and our partners four INDs, you know, between now and kind of 2025. And we're very excited about where some of those programs could go, not including some of the other partner programs where we have not received any type of work, have not disclosed any additional public information with regards to the further advancement. And so I think there's further data and information to come beyond just the milestones that are on this page around some of these programs, both named and unnamed, which we're really excited about and potentially could also drive not only material inflection points for the business in terms of milestones and data and information, but also monetary rewards for our company as we get some of those milestone payments. So with that, I'm gonna say thank you. I appreciate the opportunity to give you a presentation today on Voyager Therapeutics. I think we're gonna spend a little bit of time on the backside here now taking some Q&A from Jay. So Jay, I'll stop sharing and I will turn it back to you. Great. Thank you so much, Pete. Really appreciate that comprehensive overview and congrats on all the impressive progress you and your team are making at Voyager. And appreciate that, Jay. Yeah, really, really exciting work going on there and amazing deals you've done, especially the most recent deal with Novartis. And since you mentioned that that's actually your second deal with Novartis, can you maybe compare the two deals and describe what was the motivation behind the second one? Yeah, Jay, that's an outstanding question. So, so again, I think, you know, first and foremost, the original deal that we struck with them back in 2022 was an option to license agreement around three undisclosed targets. So we've never disclosed what those targets were or are. They, with the option, they had a one-year option to basically study our capsids in their laboratories against those targets. And, you know, replicate a lot of experiments that we had done ourselves as well as a number of experiments that they were doing. And I think they walked away from that option period, very excited about what they saw. And ultimately it led to the triggering of two of those three options to a full license or those targets to a full license exclusive to to Novartis. Since then, you know, since March of 2023 when they triggered that to a full license, we've been in close contact with them and we've shared further updates and information with regards to our capsids, the advancement of that. And some of the other work that we're doing associated with the capsids. I think ultimately at the end of the day, they were very excited about what we were doing both internally on our HD program that was a named program in our pipeline. As well as, you know, I think they're very interested in SMA as you know, they have a commercial product there. So, you know, their belief in our technology and where that potentially can take both the HD as well as SMA program. I think were big elements for them wanting to trigger another license here. I do want to stress HD and SMA were kind of outside the scope of potential targets that they could go after as part of the first original option to license agreement. The reason why they were outside the scope was those were programs that we actually had as part of our internal group of programs. So that's the reason why ultimately we structured as a separate standalone kind of licensing agreement separate from the original option to license. The one last thing I would also stress is under the original option to license. They actually still have the ability to upsize that by two additional targets. And that option still exists until later this year. So, you know, we continue to remain in very close contact with them and we'll see where that goes. But, hey, listen, a great partner, a great partnership. Good validation of our science and we're very excited to be working with them on both fronts. So it's good for the company. Alright, that makes perfect sense and that's super helpful. Thank you for the additional color. Since you mentioned Huntington's and SMA and we haven't seen a breakdown of the deal value between Huntington's and SMA. Can you just talk about how you're evaluating those two programs internally? Yeah, so we have not really provided a breakdown as of yet with regards to the value of those programs, but I would suggest you kind of think about it as such. On the SMA side, it's really a capsid licensing type agreement. Similar to maybe the previous capsid licensing agreement that we had that option to license structure. So if you remember back to that, they paid originally $18 million upfront and another $12.5 million when they struck the full license on each of those capsid licenses for the targets. So, you know, you can kind of think about that $30.5 million as kind of the value for kind of a capsid element for SMA. On the HD side, you know, it's that was a full program. Inside of Voyager, we were advancing that on our own. So, you know, from our perspective, that obviously commanded a bit more value and, you know, even though it's maybe a bit smaller market opportunity, it's of great interest, I know, to Novartis and it's of great interest to us and we're very pleased to have them as a partner relative to both sides of the coin, both HD and SMA. I think they bring unique skills and capabilities that will really help advance both of those programs as we move forward here. So thanks. Okay, excellent. That's super helpful. And since you mentioned that Novartis does have two additional options they could exercise later this year. Are there any other opt-in decisions from other partners or near-term milestones for other collaborations that you have? That's a good question, Jay. As you know, we don't really publicly disclose kind of the breakdown of programs or milestones or when things will potentially be met. That being said, I did allude to earlier that we have a little over $8 billion in potential milestones that could come due to the company across those 13 partnered programs. You can imagine, Jay, that there are a number of those programs that are gonna advance hopefully as we move forward over the next 12, 24, 36 months. We believe they will. And so, yeah, from our perspective, there's not only validation of the advancement of those programs, but there's also monetary monies that would come due to the company. And And so I would say hopefully stay tuned and we'll be able to kind of announce some news to you guys as we move forward here. Okay, great. Well, that's super exciting, especially with, as you indicated, extending your cash runway out to 2027. Can you maybe talk about how you're thinking with regards to other programs in terms of partnering versus internally developing and anything that's currently wholly owned? Yeah, that's a great question, Jay. I think, you know, we've been very thoughtful and mindful as we've gone to partner programs. You know, our partnerships or licensing collaborations come in kind of two forms as I alluded to earlier. One area kind of program deals, kind of like GBA is an example or now HD. And then on the other side of the equation, you've kind of had these capsid licenses. I think we're very thoughtful as we go to partner, whether it's capsid licenses and/or full programs. We want to retain a certain number of programs ourselves because we believe that's a big key to building value for Voyager for the long haul. And but we're also mindful that we can't do it all because, you know, hey, listen, there's even though we've got a great balance sheet in excess of $400 million and runway into 2027, you gotta make choices in life. And so I think we weigh each situation as well as the targets. And we think very thoroughly about whether or not it makes sense to continue to retain or partner. I think we're always going through a very thoughtful process. Hey, listen, as Al says, we're always open for business and with the right price and the right partnership where we know that we have a partner who can really advance the science. We would consider doing further deals. So it's a very thoughtful process is the answer to your question. Alright, super. Well, that brings us to the end of our time. So we'll wrap things up there. Thank you so much, Pete, for bringing us up to speed on all the amazing work that you and your teammates at Voyager are doing. It's been great catching up with you here today and we'll look forward to future updates. Appreciate the opportunity to present at the conference and thank you again, Jay. Our pleasure. Thanks, everybody. Have a great day. Thanks. You too.