Good morning, and welcome to the last day of the 41st annual J.P. Morgan Healthcare Conference. My name is Dave Praharaj, and I'm an associate with the healthcare investment banking group. Today, I have the pleasure of introducing Voyager Therapeutics and their CEO Alfred Sandrock. In the audience, we also have CSO Todd Carter and CFO Peter Pfreundschuh. Please note there will be time for Q&A at the end of the session. A mic will be passed around. Thank you. Take it away, Al. Thank you, David. It's my pleasure to tell you how Voyager Therapeutics is breaking through barriers in neurology and in gene therapy. Today, I'll be making some forward-looking statements during this presentation, please refer to our SEC filings, our annual 10-K and our most recent 10-Q. Why invest in Voyager? This is our investment thesis. We have three pillars of value. It begins with our capsids. You know, this capsid platform, which we call TRACER, has the potential to address some of the key barriers holding back gene therapy by widening the therapeutic window. What do I mean by widening the therapeutic window? With IV-delivered gene therapy, we hope to increase the transduction efficiency across multiple regions in the central nervous system and hopefully therefore increase the efficacy. We hope to do so at lower doses than currently being used with systemic gene therapy. With that, we hope we provide better benefit to patients at a safer doses. Recently, we identified the receptor for a leading class of capsids, and we found that this receptor actually has a human homologue. We believe this greatly increases the likelihood that this capsid family will also work in humans when we go to clinical trials. Finally, this capsid platform has led to some very important alliances for us with Pfizer, Novartis, and Neurocrine as well. The second pillar of value is our pipeline. You know, we believe that this is we are starting the renaissance of a new era in neurotherapeutics, and we see evidence of that all around us. I think we've seen evidence of that at this particular meeting. The reason for that is that we now have validated targets, drug targets, due in large part to advancements in human genetics. We also have biomarkers that really help us with drug development. These, the biomarkers are tools that help us decrease the risk early during the course of development. By them, I mean, by biomarkers, I include imaging biomarkers such as MRI or PET scans, but also fluid-based biomarkers in the spinal fluid as well as blood-based biomarkers. No one doubts, I think, the high unmet need in this category of diseases. These are some of the worst diseases on the planet, and they're growing in incidence as we age. Diseases like ALS, Alzheimer's disease and Parkinson's disease, polygenic diseases like that, but also monogenic diseases like Friedreich's ataxia. Now we expect to nominate lead candidates, which should lead to IND filings in 2024 and 2025 timeframe. The third pillar of value are our partnerships. These have generated non-dilutive revenue for us. There are broadly two kinds of partnerships. One are capsid licenses. These capsid license structures basically are exclusive to target, but not the capsid. Several companies, including ourselves, could be using the same capsid for all we know. Pfizer, after spending a year kicking the tires in their own labs with our capsids, decided to opt in last October, and we expect a Novartis decision in Q1 of this year. Neurocrine, we've enjoyed a long-standing collaboration with them on Friedreich's ataxia and two undisclosed targets. We just signed... On Monday morning, we announced that we signed a new collaboration around GBA1 and three additional undisclosed targets. I'll talk more about that in a minute. I should emphasize that we're exploring additional partnerships, both types, capsid license as well as co-development partnerships. On Monday morning, we announced this collaboration with Neurocrine, and I just wanted to review this transaction. In this transaction, Voyager receives an upfront consideration of $175 million, of which $136 million is in cash and $39 million is in equity purchased at a 50% premium. This secures program funding for one of our high priority programs. Neurocrine will pay for the costs of the GBA1 program, at least until phase 1. At the end of phase 1, we have the option to opt in on a 50/50 cost and profit share. This has transformational downstream value. There are up to $4.2 billion in potential milestones, of which $1.5 billion are during development and $2.7 billion in commercial, potential commercial milestones, as well as royalties, as shown on this slide. In this transaction, Neurocrine receives worldwide rights to Voyager's GBA1 gene therapy program for Parkinson's disease and other GBA1-mediated diseases, as well as three additional programs for rare CNS targets. Each of these programs will be enabled by Voyager's next generation TRACER capsids. Moreover, we're very happy and very excited to announce that Jude Onyia, the CSO at Neurocrine, will join our board of directors. Let me explain to you why we're so excited about this collaboration. First, it recognizes the value of our pipeline. It underscores that when we combine novel capsids with cutting-edge payloads, we have a powerful combination that can really help patients and thus shareholders. We believe that this transaction increases the asset's overall value. Neurocrine brings expertise in neuroscience R&D, and they've demonstrated that. They also have expertise in commercialization of products, especially in the movement disorders. This, we believe, improves the likelihood that this program will reach patients and thus benefit shareholders as well. This demonstrates partnership momentum. We have had a good track record of success in capsid licensing. We now have an example of a co-development partnership, and we're exploring and open to other types of partnerships as well. This collaboration provides attractive financials, which I just discussed in the previous slide. Moreover, the resources derived from this transaction enables our future growth. We hope to use the resources to advance our platform, advance our prioritized pipeline programs, and also add cutting-edge research. You know, enabling genetic medicines is the key feature of what we wanna do at Voyager. Let me show you how we combine novel capsids with diverse payloads. Our capsids, as I said, are gonna be IV delivered. They get into the brain, cross the blood-brain barrier at unprecedented levels. We have seen capsids that are 100-1,000 fold better than the standard AAV9 capsid. AAV9, by the way, is considered the king of neurotrophic capsids. not only do we improve cell tropisms across a wide variety of cells, so we can transduce neurons, we can transduce glial cells, and some capsids transduce both. At the same time, many of these capsids also de-target the cells that lead to toxicity, such as the liver cell or the dorsal root ganglion cell. As I said, identification of a receptor for a leading class helps us greatly, not only in understanding the potential for human translation, but also we can use the receptor identification to reverse engineer novel capsids. You know, the diversity of payloads that capabilities that Voyager has may not have been appreciated, but I'll tell you, I feel like a kid in a candy store sometimes because we have incredible capabilities on the payload side. I think the GBA1 collaboration demonstrates that. For gain-of-function mutations, we can vectorize siRNAs. In fact, our SOD1 ALS program is a vectorized siRNA. For loss-of-function mutations, we can replace the gene that's missing. That's what we're doing in GBA1 as well as Friedreich's ataxia. We can also vectorize antibodies, so we can have cells within the central nervous system make antibodies in a durable manner that can target well-validated drug targets on the cell surface. This is an amazing array of tools for a drug developer like me to look at, and we hope this will enable neurogenetic medicines, many more medicines in the future. Just an update on our financials. As of September 30th, we reported $131.6 million in cash equivalents, and marketable securities. This did not include the $10 million option payment from Pfizer that we will that we received in October. At the same time, in our Q3 earnings call, we communicated that this balance sheet plus expected reimbursements were sufficient to meet planned expenses and Capital Expenditures requirements into 2024. Now, with this collaboration with Neurocrine that we just announced on Monday, we add $175 million of upfront consideration. We expect to close this, the last year preliminarily in an unaudited manner. We expect to close with $119.2 million. This leads us to $294 million in pro forma cash following the Neurocrine collaboration. We expect a Novartis decision in Q1 of this year around three options to license capsids on select CNS targets at $12.5 million each, and this could be upsized by two additional targets of $18 million each. You can see, we are continuing to strengthen our cash, our balance sheet and our runway, while enhancing long-term value. We expect to provide further financial guidance as part of our 10-K financial results in March of this year. I'd like to take a look now at our pipeline. I wanna begin with the Neurocrine collaboration programs. With the deal announced on Monday, we now have two programs. Parkinson's and other diseases that are GBA1 mediated, as well as the Friedreich's ataxia program which we had before. These are our co-development collaborations. In addition to that, we have three wholly owned programs. A passive antibody against tau for Alzheimer's disease. This is a differentiated C-terminal antibody that blocks the spread of tau in animals, highly differentiated from the N-terminal antibodies that don't do that. We also have, as I said earlier, an SOD1 gene silencing program using a vectorized siRNA against SOD. We have multiple early stage programs. On Monday, we added some color into one of those. We have a unique, we believe a relatively unique approach to Huntington's disease, where we're taking into account the latest clinical trial data, as well as data coming from biological studies on the pathophysiology of disease. This new approach will employ a allele-specific vectorized siRNA in combination with an MSH3 knockdown. MSH3, we believe, will be important in limiting or reducing the somatic expansions that occurs in cells that are destined to die, and the allele specificity of the mHTT knockdown, mutant mHTT knockdown will help us increase the therapeutic window and be able to provide benefit more safely. We also, as I mentioned earlier, have a number of license, capsid license structures around five gene therapy programs for undisclosed disease, undisclosed diseases with Neurocrine, a rare neurological disease with Pfizer, and three gene therapy programs for rare CNS diseases with Novartis. I wanna close with this slide. You know, I'm continuing to build my team, and you'll meet a couple of members of that team in a minute during the Q&A. We're building the board of directors, and I'm very excited about what we're doing here at Voyager in terms of the team. We presented data in the second half of last year at AAIC and ESGCT, where we showed data on the differentiation of our anti-tau passive antibody program at the AAIC meeting. At the ESGCT meeting, we showed that we can deliver low doses intravenously, much lower than currently being used, on the order of 10 to the 12 VGs per kg, in effect, a very efficient CNS cell transduction. We also presented our data without identifying the actual receptor. The data that led us to believe that we actually have the receptor for one of our leading capsid families. In Q3 of 2022, Pfizer exercised its option on a rare neurology target. For that we received a $10 million payment. In Q1, on Monday actually, we announced the Neurocrine strategic collaboration that extends the collaboration that we already enjoyed with them. As I said earlier, potential for $4.4 billion for GBA1 and three discovery stage programs. As we look forward, you know, we look forward to the Novartis decision in Q1 of this year. Potential for up to $37.5 million payment. We expect to identify lead candidates for all three priority pipeline programs, for the tau program for Alzheimer's, GBA1 Parkinson's program, gene therapy program, in collaboration with Neurocrine, and the SOD1 ALS gene therapy program. As always, we continue to talk to multiple parties, and we're open to additional value-adding, value-creating partnerships, and these discussions are ongoing. Thank you very much for your attention. We'd like to now open it up for Q&A, and I'd like to invite Peter Pfreundschuh, our CFO, and Todd Carter, our CSO, to join me up here. Any questions in the audience? Okay, I'll kick it off. First off, congratulations on this week's announcement. Given the size of the collaboration agreement, You know, you mentioned how this agreement affects your balance sheet, but what does this allow you to really focus on as you continue to advance Voyager? Yeah. It's, it's really about continuing to think about how we can add diverse payloads to our novel capsids and tackle some of the worst diseases on the planet with the highest unmet need. I talked about our ability to vectorize siRNAs to knock down toxic gain-of-function gene expression. We can also replace, missing, essentially, due to loss-of-function mutations, missing genes, and we can vectorize antibodies. You know, not only does this collaboration take sort of the expenses of one of our three priority pipeline programs off of our expenses, but also we get resources to reinvest in early research. You can expect us to announce more early research programs as the, as time goes on. Todd, Pete, do you wanna add anything? No, I don't think I would add much more, Al. I think, you know, the benefit of course, inking the Neurocrine collaboration earlier this week is allowing us to not only secure the advancement of the GBA program and these new programs that Neurocrine wants to advance in collaboration with us, but also for us to reinvest in our business and continue to grow as Al described. I think, you know, that is our incentive as we move forward to further the advancement of our science and technology in the business. It's a, it's a great situation. It sets up, us up for 2023 and beyond. As Al said, I think you're gonna hear more from the company with regards to some further early-stage programs as we move into the year. Okay. Moving, advancing into 2023, I guess, what would be your number one priority? Well, I mean, you know, all three of our priority programs are sort of equal, if you will. They're all exciting in the sense that they address high unmet need, but also, we believe that they will provide proof of biology very efficiently. We chose these programs so that we can, do clinical trials and with, very efficiently provide the evidence that we've got adequate gene expression to provide the therapeutic benefit that we will need. All three are high priority. Okay. Actually, go ahead. Well, well done on creating some excitement here for shareholders after a period of difficulty. I really admire the fact that you hung in there and persevered and brought some real strong talent added to the talent that you obviously had. When you see numbers like this, you know, from a big pharma outlook, they're looking at the next five, 10, and 15 years. $4.4 billion, whoever, well, Neurocrine made that investment, they gotta be looking at least five or 10x their money to do that. Where are the commercial points of excitement? Where does this go commercial, and what are the total available marketplaces as you would be successful in commercial? Pete, you wanna take that? Yeah. I think, first and foremost, from the perspective of the GBA1 target, you know, the advancement of that particular program, from a PD perspective, you know, we always envision that, quite frankly, we must probably need a partner in order to be really successful. I think the benefits of Neurocrine is that they're already playing in this space to some degree, and they really do have a calling sales force already in this space. I think, quite frankly, they bring great development capabilities, but also wonderful commercial capabilities. I think for us as an organization, you've gotta kinda know your limitations as to what you can do as a small biotech company. Partnerships like this really reinforce creating value for the long term, but also de-risking these programs, both in the development as well as in the commercialization phases of the product. In the direction you've done a great job, got a great partner. There may not be a better one than Neurocrine, and they're commercial ready. To address the commercial opportunity that they had to be looking at I'm a Voyager shareholder, you all had to be looking at to put this in place five and 10 years out. This is gonna take a while, but you get through into the late 2020s and early 2030s, what kind of revenue opportunities are out there if they deliver, if you deliver, and the team delivers the product, what can they do with it? Yeah. Well, I mean, well, we could look at the patient numbers. Yeah. You know, 10%, up to 10% of Parkinson's patients are GBA1 carriers. Parkinson's is the second most common neurodegenerative disease. There are about 1 million patients just in the U.S. with Parkinson's. Ten percent is about 100,000 patients that are addressable. That's. You know, obviously the price will determine the ultimate commercial potential. I think we're still a ways off from that, but. The, I mean, the label will drive the price. Yes. The price will drive the revenues. If you look at any of the big pharmas, they're all talking 25 to 30, and they roll out what their numbers are gonna be. My, my view is that those of us in the small pharma ought to do the same thing 'cause it really impacts our cost of capital. Mm-hmm. and impacts your ability to attract talented people when they see this is gonna be a powerful machine. That's what you're building. It's clearly what you have in mind. Expressing that as a potential, I think makes a lot of sense. It's helpful what you just said on Parkinson's. Thank you. Switching gears a little bit, you know, you mentioned your early development program in HD. You deprioritized HD programs in the past. Why advance this one? The previous HD program was actually using a sort of a standard capsid delivered intraparenchymally. You know, Voyager was one of the very first gene therapy companies, certainly one of the very first in CNS. We were one of the first to learn that localized delivery can get you very good transduction efficiencies in certain regions. But there are issues associated with that. In fact, the lead program, which was in Parkinson's for AADC, was stopped after the safety monitoring committee recommended that the trial be stopped due to MRI abnormalities. Localized delivery is not gonna get you there for most of the diseases we're talking about. Even though they may start in the deep gray structure, such as the striatum or the midbrain, eventually the disease affects the entire brain, certainly the entire cerebral cortex. You need broader distribution. That's why I'm so excited about our IV-delivered novel capsids, because we get broad brain distribution, and much higher transduction efficiencies. That explains why we stopped the previous program in HD, and why we're coming back to it with our novel capsids. We also are taking lessons from the ongoing clinical studies that have occurred already. We believe that it's important to have an allele-specific approach. We also think that we have to address the somatic expansion that occurs in cells. We used to think that Huntington's is caused by either a toxic protein or a toxic RNA. Now with these somatic expansions, these massive expansions in the cells that are destined to die, we think that the DNA itself might be toxic. It may affect chromatin structure. We don't actually know whether it's the DNA, the RNA, or the protein that's causing a toxicity. Our approach would tackle all three, so it wouldn't matter. I think the point I wanna emphasize is that we're taking learnings as the field progresses in HD and applying our novel capsids with diverse payloads to address all these learnings. Todd, you wanna add? Yeah. Maybe I can add a couple of. Yeah. Comments. We've been in HD, as Al points out, for quite some time, and we had our first generation program that he described. That particular program, we were targeting all of Huntington's disease mRNA to result in knockdown. We have deep expertise in vectorizing siRNAs to do that. We came up with a potent vectorized siRNA. It did what we needed it to do in terms of knocking down both the protein and the mRNAs. As we moved down the road, as the field has progressed, these other concerns about leaving residual wild type functional Huntington's disease protein to do its normal job have arisen. Our next generation program really takes advantage of our broad BBB penetrating capsids, where we can deliver broadly throughout the brain without trying to inject a large amount of material in a very focal region. We can get that delivery with a relatively benign intravenous delivery and pairing that with kind of a state-of-the-art payload, where we are harnessing both the vectorized siRNA technology that we have to do allele-specific targeting to knock down that mutant, along with the knockdown of this MSH3, which will impact both the DNA that Al described and the mRNA and protein. We're pretty excited by the program. Can you talk a little bit more about your capsid's brain penetrance versus your competitors? Go ahead, Todd. The, I mean, the competitors, I think gene therapy, we all wish everyone well, and so I'm hoping for success. I think success for just about any of us is success for the whole field. What we've seen with our capsids, we got in relatively early. A little bit of history, Caltech identified some capsids that worked quite well to cross the BBB in mice, but only in mice, and in fact, only particular strains of mice. That demonstrated that it was possible to do so. What the team at Voyager has done, what we've done is to take the technology which worked in mice and to change it so that it could be done in non-human primates. That's important because ultimately, we wanna work in primates. We want it to function in human beings. We've developed that technology. It was focused around, not only primates, but also the ability to identify, capsids that delivered not only the DNA, the genome itself, but functional forms of that DNA, so that it expressed. Those two have been key to seeing the kinds of success that we've had so far. On top of that, what we found is that you can identify something that works in mice or works in one species of primate, but it's really important to identify those that work in multiple species, multiple primate species or perhaps even rodents and primates. That's what we've been able to do. Those are the kinds of data we're finding. Fundamentally, we even have a capsid, a family where we've identified the receptor, and I think Al mentioned this as part of his talk. The receptor responsible for delivery across the blood-brain barrier. That allows us to do a couple of truly important things. One, it increases our chance of success in the clinic, we believe, because we have found that that receptor is expressed in monkeys, it's expressed in rodents, and it's also expressed in human beings. The capsids that traverse the BBB in monkeys bind the human form of the receptor, just like they do for the rodents and the primates, the non-human primates. That gives us visibility and the opportunity we might have in human beings for that to be able to translate. It also gives us the ability to really target that particular receptor so that we can begin doing receptor-driven evolution to identify and perhaps even improve even more using that particular route of delivery. Yeah, I just wanna emphasize that what the Voyager scientists did was nothing short of remarkable. To be able to test more than 20 million variants in non-human primates and pick out the ones that actually get into across into the brain, it's pretty remarkable technology. I was astounded when I first heard about it. You know. This team continues to amaze me because they identified the receptor, which is also not easy for a leading class of capsids. You add to that the payload capabilities that I mentioned. It really is an amazing period right now, and I think it adds to the excitement for neurotherapeutics. The better the patent picture, the longer the patent protection you have, obviously is the better. You've gotta move quickly, and it looks like you now have an edge that you've demonstrated that is able to be understood by others knowledgeable in the science. Your investor base, myself and others are not so insightful as to what you have and the meaningfulness of it other than it really seems to look pretty good. The more you can point out the what if this occurs and flourishes, the consequences of that commercially, the lesser cost to capital is. The lesser cost to capital is, the more you can add to the team and move at a quicker pace, which continues to separate you from your competition. At the right time, it is worthwhile. No one's looking for hyperbole that has a one in a 100 chance, but, you know, somebody ran some numbers here and came up with some serious dollars. Yes. Somebody's looking at those numbers. When you feel it's appropriate to share that on a what if basis with shareholders, I think you'll find that it adds value to the company by reducing cost of capital. Thank you very much for that advice. I'm glad you're sitting right in front of our CFO. We appreciate the feedback and I fully agree. I think if you look at our corporate deck, which was recently posted as part of, you know, the actual announcement of the deal earlier this week, we talked about size of the markets to Al's comment with regards to GBA1, and should have picked up on that when you asked that question. It's a very good point with regards to how does that translate into, you know, dollars and cents and opportunities. Rose on the deal and now rose higher, whereas, you know, a while back it rose up and came all the way back down because nobody... What? This is now. This is pretty real. The more help you can be on that, the better. We believe that's the case as well with the other programs that were chosen in the middle of this past year. When, you know, Al came on board earlier in the year, really looking at the full pipeline and the opportunities, and then prioritizing. We feel the same way with the other opportunities that are part of core programs in our pipeline, that there really is significant value there. That was one of the elements of why we chose those. I think we have time for one more question. If not, if there are no other questions, I'm happy to give everyone back some time. Thank you so much. Thank you, David. Thank you.