Voyager Therapeutics at H.C. Wainwright: Genetic Innovation in Focus

On Tuesday, 17 June 2025, Voyager Therapeutics (NASDAQ:VYGR) presented its strategic vision at the H.C. Wainwright 6th Annual Neuro Perspectives Hybrid Conference. The company outlined its focus on leveraging genetics to treat neurological diseases, highlighting its multimodality approach. While the emphasis on partnerships and financial stability was evident, the challenges of crossing the blood-brain barrier and market undervaluation were also discussed.

Key Takeaways

• Voyager aims to develop differentiated medicines for CNS diseases using genetics.
• The company maintains a strong financial position with $295 million in cash.
• Partnerships with Novartis and Neurocrine are crucial for future milestones.
• Alzheimer’s programs target both tau and amyloid, with upcoming data readouts.
• Voyager’s unique capsid technology aims to improve drug delivery efficiency.

Financial Results

• $295 million in cash, providing financial runway into mid-2027.
• Expected near-term milestones of up to $35 million from Neurocrine partnerships.
• Potential $7.4 billion in milestones across 11 partnered programs.
• Partnered programs have generated approximately $500 million in non-dilutive revenue.

Operational Updates

• Alzheimer’s Programs: VY-7523 in Phase 1b trial; BY-1706 IND submission targeted for 2026.
• Neurocrine Collaboration: Two INDs planned for 2025 for Friedreich’s ataxia and GBA1 Parkinson’s disease.
• Capsid Platform: Continued development of AAV gene therapy capsid and non-viral delivery platforms.
• Delivery Technology: Focus on ALPL receptor to enhance delivery across the blood-brain barrier.

Future Outlook

• Advancement of Alzheimer’s programs with data expected in late 2026.
• Progress on partnered programs with Neurocrine IND filings in 2025.
• Exploration of new partnership opportunities for gene therapy platforms.
• Focus on the shuttle program as a high-priority initiative.

Q&A Highlights

• Emphasis on tau PET imaging as a critical readout for Alzheimer’s programs.
• Discussion on the optionality of combining anti-amyloid and anti-tau therapies.
• Highlighted the unique approach to gene therapy using neurotropic capsids for safety and efficacy.
• FDA endorsement of frataxin as a biomarker for accelerated approval in Neurocrine discussions.

For more detailed insights, readers are encouraged to refer to the full transcript below.

Full transcript - H.C. Wainwright 6th Annual Neuro Perspectives Hybrid Conference:

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Hello, everyone, and welcome to the sixth annual HC Wainwright NeuroPerspectives Conference. My name is Patrick Trucchio. I’m a senior health care analyst at HC Wainwright. We have a robust agenda at the conference this year with more than 30 companies presenting with their sessions available on demand through the conference portal. In addition, we’re expecting a full day of panels and fireside chats with world class KOLs on June 16 for the in person portion of the conference.

With a broad CNS drug drug development focus across multiple indications from depression and epilepsy to Alzheimer’s disease and ALS and featuring novel methods of drug delivery to the CNS, this is by far our strongest agenda ever. With that, it’s my pleasure to introduce our next presenters, Al Sandrock, CEO Toby Ferguson, CMO and Nate Jorgensen, CFO of Voyager Therapeutics, a clinical stage neurotherapeutics company advancing genetically driven medicines for serious CNS diseases. Voyager’s pipeline includes multiple programs targeting tau, gene therapy pipeline enabled by next generation capsids, and 11 partner programs with companies including Novartis and Neurocrine. So first, if we could, to start, maybe you can provide an overview of Voyager’s strategy, including how you remove its multiple modalities of neurotherapeutics, including antibody, gene therapy, and receptor mediated delivery sets you apart.

Al Sandrock, CEO, Voyager Therapeutics: Oh, thank you very much, Patrick, for inviting us, and very happy to be here with my colleagues, Nate and Toby. Yeah. So our strategy is to leverage genetics to treat neurological diseases. So we we go after validated targets, and we view human genetics as a high level of validation. And what we’re trying to do is to get better delivery into the brain.

The blood brain barrier is still, an issue for everything except small molecules. The problem is that if you’re limited to small molecules, there are lot of targets that are not accessible accessible to small molecule drug development or that’s very, very difficult. So we we like to leverage other modalities, proteins such as antibodies or enzymes, gene therapy, of course, with AAV, and oligonucleotides. The problem with those last three modalities is that they don’t get into the brain very well with systemic delivery, and that that limits It may have may create safety issues.

And, of course, it’s a huge, inconvenience and, burden on the health care system, inconvenience to patients. So so, so what we’re so we’re experts in delivery, and, we wanna we wanna, treat these diseases in a better way with regardless of modality.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. Voyager has recently emphasized capital efficiency and biomarker driven derisking. How do you define clinical and platform success across your pipeline?

Al Sandrock, CEO, Voyager Therapeutics: So we define success as having as developing truly differentiated medicines with a transformative benefit. And and, we don’t choose programs unless we see a path, forward where we can efficiently derisk the programs in the clinic. And so we will we require that not only that the targets are validated, but that the that there are biomarkers available that can help us derisk the programs.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And, your partner, Novartis, has had tremendous commercial success with Zolgensma. What do you think made that program so successful, particularly as others in gene therapy have struggled? And what lessons from that experience are most relevant as you scale Voyager’s pipeline?

Al Sandrock, CEO, Voyager Therapeutics: Yeah. Well, so ZOLGENSMA is I would note that it’s an IV delivered gene therapy. And so, but it only works it’s

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Hello, everyone, and welcome to the sixth annual H. C. Wainwright Neuroperspectives Conference. My name is Patrick Trucchio. I’m a senior health care analyst at HC Wainwright.

We have a robust agenda at the conference this year with more than 30 companies presenting with their sessions available on demand through the conference portal. In addition, we’re expecting a full day of panels and fireside chats with world class KOLs on June 16 for the in person portion of the conference. With a broad CNS drug drug development focus across multiple indications from depression and epilepsy to Alzheimer’s disease and ALS and featuring novel methods of drug delivery to the CNS, This is by far our strongest agenda ever. With that, it’s my pleasure to introduce our next presenters, Al Sandrock, CEO Toby Ferguson, CMO and Nate Jorgensen, CFO of Voyager Therapeutics, a clinical stage neurotherapeutics company advancing genetically driven medicines for serious CNS diseases. Voyager’s pipeline includes multiple programs targeting tau, gene therapy pipeline enabled by next generation capsids, and 11 partner programs with companies including Novartis and Neurocrine.

So first, if we could to start, maybe you can provide an overview of Voyager’s strategy, including how you remove its multiple modalities of neurotherapeutics, including antibody, gene therapy, and receptor mediated delivery sets you apart.

Al Sandrock, CEO, Voyager Therapeutics: Oh, thank you very much, Patrick, for inviting us, and very happy to be here with my colleagues, Nate and Toby. Yeah. So our strategy is to leverage genetics to treat neurological diseases. So we we go after validated targets, and we view human genetics as a high level of validation. And what we’re trying to do is to get better delivery into the brain.

The blood brain barrier is still, an issue for everything except small molecules. The problem is that if you’re limited to small molecules, there are a lot of targets that are not accessible to small molecule, drug development or that’s very, very difficult. So we we like to leverage, other modalities, proteins, such as antibodies or enzymes, gene therapy, of course, with AAV, and oligonucleotides. The problem with those last three modalities is that they don’t get into the brain very well with systemic delivery, and that that limits its efficacy. It may have may create safety issues.

And, of course, it’s a huge inconvenience and burden on the health care system, inconvenience to patients. So so so what we’re so we’re experts in delivery, and we wanna we wanna treat these diseases in a better way with regardless of modality.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. Voyager has recently emphasized capital efficiency and biomarker driven derisking. How do you define clinical and platform success across your pipeline?

Al Sandrock, CEO, Voyager Therapeutics: So we define success as having as developing truly differentiated medicines with a transformative benefit. And and, we don’t choose programs unless we see a path, forward where we can efficiently derisk the programs in the clinic. And so we we require that not only that the targets are validated, but that the that there are biomarkers available that can help us derisk the programs.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And, your partner, Novartis, has had tremendous commercial success with Zolgensma. What do you think made that program so successful, particularly as others in gene therapy have struggled? And what lessons from that experience are most relevant as you scale Voyager’s pipeline?

Al Sandrock, CEO, Voyager Therapeutics: Yeah. Well, so Zolgensma is I would note that it’s an IV delivered gene therapy. And so but it only works it’s only approved for infants up to age two years old. And so the blood brain barrier changes and precludes its its use with with IV delivery in older people. That’s probably why Novartis did a deal with us on SMA to improve delivery in older people.

But yeah. I mean but Zolgensma, you know, it goes after a very well genetically validated target. It’s a single gene disease. Zolgensma has high is a transformative treatment. It’s I mean, it’s transformative for the patients and and the families.

And there was a it was a pretty rapid path to approval, so a pretty efficient path to approval. And and in fact, for SMA now, there is a a well laid out regulatory, and development pathway. So so, hopefully, we we will reproduce, what we what we saw with Zolgensma, with other targets of similar types.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And then just moving on to the Alzheimer’s field, Voyager has three Alzheimer’s programs in its pipeline. Do you view the Alzheimer’s field today? What do you see as the key unmet needs, and how is Voyager addressing them?

Al Sandrock, CEO, Voyager Therapeutics: Yeah. So, you know, I think what the way I look at Alzheimer’s is we just had we we just started getting the first disease modifying treatments. I mean, it was a breakthrough to get the first disease modifying treatments to patients, I believe. It reminds me a lot of MS, you know. In the old days with MS, the the first treatments were only about thirty percent effective.

But we’ve got better and better, and now we have truly transformative treatments for MS and multiple types of treatments. I hope we get there with Alzheimer’s disease as well. And we believe that the next target, to go after, and we think it’s well validated, is tau. And so for that reason, we have two programs against tau, an antibody, a regular antibody, that has not been optimized for delivery, to be honest with you. We may we have the option to do that later, of course, if we get proof of concept, and we have a gene therapy knockdown approach.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. So and you have programs targeting amyloid and tau. How do you see these two approaches fitting into the treatment landscape?

Al Sandrock, CEO, Voyager Therapeutics: Well, I believe that, just like oncology uses multiple drugs either in sequence or in combination, we may we may get to that point with, Alzheimer’s disease as well. And that’s why I think it’s it’s, it’s useful to have more than one target and more than one treatment paradigm. And and then, ultimately, it’ll be personalized depending on how patients respond to the first treatment, and whether they’re complete responders or partial responders.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And then just moving on then to the anti tau antibody program, VY seventy five twenty three, now in a phase one b multiple ascending dose trial in early Alzheimer’s disease. What are the goals of this study, and what data should we expect in the second half of twenty twenty six?

Toby Ferguson, CMO, Voyager Therapeutics: Toby? Thanks, Al. Thank you, Patrick. So to remind seventy five twenty three is our our anti tau antibody program. It’s in a multiple setting dose program.

And, really, I think what we’ve learned, particularly from some recent discussions, is that the most relevant readout is TauPet, and we’ll see that in the second half of twenty two 2026. I I think the point I make there is is there’s been some exploration of fluid biomarkers in the field, but some recent discussions at at ADP, for example, highlighted potential, discordance between fluid and fluid biomarkers and TauPet. So we really are focused on TauPet as the key readout. Of course, we’ll explore, clinical scores like CDS, some of boxes and fluid biomarkers, but but the study formally is is really looking at tau PET as the key readout. I’d also highlight that the bopanemab data and really the idea that you can move tau PET with an antibody was an important learning.

And so these both the ADPD discussions and the panamab discussions really highlight the importance of tau PET.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And in your preclinical data, BY seventy five twenty three showed potent inhibition of tau spread and superior target engagement in a tau seating model. Can you walk us through that model, and what makes it translationally relevant?

Toby Ferguson, CMO, Voyager Therapeutics: So, fundamentally, I think there are there are lots of ways you can you can try to choose an antibody, to see if it impedes the biology of tau. And I think at Voyager, we think the fundamental issue is is in humans, we know that tau starts in a localized region of of the temporal cortex, the interlateral cortex. And then with confluence of beta amyloid, you get this spread of tau, and we know that’s what correlates with critical signs and symptoms and disease progression. So, really, we think it’s that biologic process of tau spreading that is critical to disease. Why we like the p three zero one s model is what’s done in that model is you on the background of the of the tau three zero one s mutation, you inject purified human pathologic tau into the mouse, and you and you watch it spread.

Fundamentally, what we show with our antibody is that in this model that we could inhibit substantially that tau spread. I think also quite importantly, and really what got us quite interested in this approach, was that there have been two prior failures of internal antibodies. And in those cases, both of the those antibodies also failed in our model. So we we really like this model because we think it it emphasized that process of biologic spread, which we think has potential translatability in humans. I’d also highlight that the bripenumab data, the their their antibody from UCB worked in this model as well.

So some potential partial positive read through as well.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And you noted that the field has struggled with the n terminal tau antibodies. How is your c terminal approach differentiated, and what are the advantages of targeting that epitope?

Toby Ferguson, CMO, Voyager Therapeutics: So I think fundamentally, the the our epitope think the key point that we make is, one, the epitopes are different. So n terminal versus c terminal, and just highlighted our preclinical data in the three zero one s models. But I also think a key point is that our our antibody is pathologic tau specific. That’s really key point of differentiation. I think the other is based on what we’ve seen in the field holistically, we think we can explore based on the safety quite high doses.

I think there’s potential differentiation there as well.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. You’ve mentioned, learnings from beprenumab’s readout. What are your takeaways from that program, and what other programs do you see as potentially having read through?

Toby Ferguson, CMO, Voyager Therapeutics: So I think I think the the dopanorop data, I think, was certainly a a positive step forward for the field. It showed for the first time that an antibody to tau in a human being can impede the accumulation of of of of tau pathologically as measured by, tau PET. So important first step. And they also showed, they missed their primary end point on C. Difficile boxes, but they showed in the whole population that you could potentially have an effect on ADAS Cog and a measure of of cognitive performance.

I think missing the primary did engender some skepticism in the field. I think, fundamentally, the question that the field is struggling with is what is the appropriate exposure you need of a tau antibody to get a a full clinical effect on on measures like CVS and the boxes? I think there’s there’s some coming readouts that will certainly help with that. I’d highlight there’s there’s a pathologic antibody from Merck. We’ll have some fluid biomarkers readout of that in July, hopefully.

But, really, I’d highlight the the J and J readout and the Viveni readout in in 2026 that will move the tau four field four holistic. The J and J study is an antibody in a large phase two study that will have appropriate power to open critical endpoints. And so really understanding that exposure response is our key next step for the field. And we really think our antibody is nicely differentiated from pathologic tau. It really has a good chance of of impacting, this in in human expansion.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Great. So maybe then moving on to the tau silencing gene therapy. This is the BY 17 o six program. It’s a vectorized microRNA therapy delivered intravenously with a tracer capsid. Can you, just maybe provide some background on this program and then walk us through your most recent nonhuman primate data?

Toby Ferguson, CMO, Voyager Therapeutics: Certainly. So I’d I’d refer the group. We had a nice oral presentation at ASGCT in 2025, so that’s that’s available. I think fundamentally, the the concept here is different than the antibody. Really, the goal here is is to sidestep the question of what are the appropriate pathologic species of tau and and take a more general approach and knock down tau, in in cells in the nervous system to to to decrease overall tau burden.

And so, really, the what we’ve the team has done is generated this microRNA to do just that. I think what we’re really excited about is we’ve shown in primates with an IV dose of about one point three e thirteen, which is that we have knockout of up to seventy three percent in the primate tissue. And so we get just emphasize, we get substantial knockdown throughout the CNS up to seventy three percent at a low dose of one point three e thirteen, and then that’s IV. And so, really, we feel that we can move this is a key program to see, can IV approach really broadly reduce tau in in the human nervous system? So quite excited by it.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. What are your expectations for durability, and how do you benchmark this program against ASO approaches like BIV eighty?

Toby Ferguson, CMO, Voyager Therapeutics: So I think, fundamentally, this this program is meant to be a onetime IV administration. So the expectation is that you’d have continuous production of of this microRNA, therefore, long term knockdown of tau. Of course, we’ll need to do the studies, and the studies will be long term in terms of this, but our expectation is this will be a one time one time treatment. I I think, fundamentally, the the benchmark will be the knockdown and responses seen in the ASO approach of 80. What they’ve shown is that you can get about 60% reduction in CSF compared to baseline, and so I think that that’s sort of the the biomarker based benchmark.

They’ve also shown in their in their studies, phase one studies, that you can see you have baseline pathologic tau as evidenced by tau PET, and some of that can be removed. So I think those two things are really the biomarker based benchmark we’ll be aspiring to. That being said, I think with our single IV administration based on preclinical data, where we see quite substantial reductions of both, protein and message that we think can meet this benchmark.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And what’s the timeline for IND submission? What studies remain before filing?

Toby Ferguson, CMO, Voyager Therapeutics: So what we’ve communicated is that the IND will be in in 2026. And, really, we’re in the process of working through the the IND enabling work, particularly toxicology to support that, IND or CTA submissions in The US and Canada in 2026.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Great. And then BY 17 o six and BY seventy five twenty three target different forms of tau, one extracellular, one intracellular. How are you thinking about the potential for sequential or combination use?

Al Sandrock, CEO, Voyager Therapeutics: Toby, do wanna take that?

Toby Ferguson, CMO, Voyager Therapeutics: I’m sorry, Al?

Al Sandrock, CEO, Voyager Therapeutics: I said, Toby, do you wanna take that?

Toby Ferguson, CMO, Voyager Therapeutics: Sure. I’ll take that. So I think I think fundamentally, I think back to Al’s prior point, both with amyloids, anti amyloid based therapies, and potential very different tau based approaches, antibody or knockdown, I think they’re gonna be you’re gonna need multiple options. I think at the highest level, you’re gonna understand some patients who respond differentially to antibody, tau antibodies, as much as they could to, beta amyloid therapies. For example, in someone who you’ve kept relatively early in progression, who has the presence of tau but more limited spread as evidenced by tau PET, may be an excellent candidate for an antibody approach.

Someone who has, more widespread house spread may be may be appropriate for knockdown, or you may need, a combination of of antibody and and knockdown simultaneously. You also probably wanna sequence the beta m word somewhere before these. So I think, fundamentally, over time, we’re gonna learn about different sets of responders to each of these therapies. I also highlight that the the safety of of these approaches will be taken in consideration. So I think, really, you’re gonna need this optionality for full treatment of Alzheimer’s disease as as as a entity.

I I really think it’s gonna be important to have both options available for patients.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And just earlier in the discussion talking about importance of, crossing blood brain barriers, I’m wondering if you can speak to the ALPL as a shuttle and what data we might expect to see this year to further characterize this platform.

Al Sandrock, CEO, Voyager Therapeutics: Well, so, the significance of ALPL or identifying the receptor is that the history in the field of AAV, delivery is that you can find viruses that are very species specific. So we we didn’t wanna get a virus that only works in mouse or only works in nonhuman primates. So typically, what we’ve done is to make sure that our capsids cross multiple species, but there’s nothing like having, identified the receptor. Then you know whether or not there’s a human homolog, and and for ALPL, there is a human homolog. So that has derisked the translatability as much as you possibly can before actually going into the clinic.

And now that we’ve identified the receptor, we know that that receptor is leveraged to bring in a very large AAV particle, essentially, the virus, into the brain. So then the is, can we leverage that receptor by making other kinds of ligands, conjugating them to protein therapeutics or oligonucleotides, and get them in into the brain as well? And that’s what we’re working on.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And on the gene therapy side, the gene therapy field has suffered several setbacks recently. What makes Voyager different from the companies who have struggled?

Al Sandrock, CEO, Voyager Therapeutics: Well, I mean, I think that one of the issues was that the capsids were not optimally designed. I thought I think delivery, has hampered the field. And if you do go to systemic delivery, we had to use very high doses, e fourteen VGs per kg range, which cause safety problems. So by having neurotropic capsids, we hope to avoid the the safety issues associated with with, localized delivery such as intraparenchymal delivery. I I remind you that Borger was doing that years ago.

Intraparenchymal delivered AAV and stopped those programs. So, so going to IV delivery that crossed the BBB, and because they’re so good at crossing, we hope to be able to use doses that are much lower than the e fourteen b g’s per kg, at least an order of magnitude lower. And that should be very helpful on the safety front. And also, I would say that with these localized deliveries, you get spotty, delivery to certain parts of the brain, and we we wanna optimize the efficacy as well. And many brain diseases, affect large region regions of the central nervous system, both the brain and spinal cord sometimes.

And so the the IV delivered AAV promises to help on the efficacy side as well.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. How close are you to using your capsids in non gene therapy modalities like oligonucleotide or enzyme delivery?

Al Sandrock, CEO, Voyager Therapeutics: Well, we’re we’re we’re probably not gonna use gene therapy to deliver oligonucleotides, but we we wanna be able to leverage the receptors that we discover through our tracer platform to find AAVs across the BBB, discover the receptors, and see if we can make ligands against those receptors, conjugate those ligands to protein or or or oligonucleotides, and deliver those IV and get them into into various tissues, into the brain, for example. This this, of course, has been done very well with transferrin receptor as you know. People like Denali have pioneered this. Other companies like Abidity and Dine are working on muscle delivery. So the TFR conjugation of of these oligonucleotides and proteins seems to work.

We we think that our receptors may offer some differences and that they’re gonna be differentiated perhaps on on distribution, on kinetics, and maybe on safety as well. And so I think it’s worth pursuing these other receptors to optimize brain delivery.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. Great. So just on the Neurocrine partner programs, you have two INDs planned for 2025 with Neurocrine for Friedreich’s ataxia and GBA one Parkinson’s. What’s the latest on those programs?

Al Sandrock, CEO, Voyager Therapeutics: Well, yeah. So there it’s a great partnership with Neurocrine. We have, five programs, that we’re working closely with them. It’s a great interaction scientist to scientist. And, look, as far as we know, both programs are on track for IND filings that are expected this year and that the clinical first in human studies, are expected, next year.

And I should remind you that Voyager gets up to $35,000,000 in milestones for these products in this period. Very important to us.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And what biomarker clinical endpoints might be used to support early proof of concept, particularly for FXM?

Toby Ferguson, CMO, Voyager Therapeutics: I’d I’d highlight, really, for for both programs, the clinical development is really owned by by Neurocrine. But I can comment generally on on the set of biomarkers for for both programs. For frataxin, I mean, so for FA, frataxin is is really gonna be the key biomarker. I’d highlight that there have been some discussions with the FDA in the context of other programs where they’ve endorsed using frataxin as a biomarker for accelerated approval. So I think measurement of frataxin is gonna be key, particularly in the in the in the CNS and in this context of a a neurotropic capsid.

I also think for GBA, there are a series of biomarkers you could consider using as well, that will be quite important. So, really, both programs fit into that concept of of development programs that have a path to biomarker based POC quite quickly.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And with 11 partner programs and up to 7,400,000,000 in potential milestones, how are you balancing internal innovation and external monetization?

Al Sandrock, CEO, Voyager Therapeutics: Well, we’re, you know, we’re always looking for business. We’re, for new partnerships, and, expanding partnerships that already exist. This this, partnership, has brought in a lot of our partnerships that we’ve already made have raised about $500,000,000 in non dilutive revenue. So so we so and and the partnerships, as I said, the the science scientist to scientist interactions have been tremendous. But we also intend to retain some wholly owned programs that we can advance in a very capital efficient manner, and we’ve built a world class clinical development team under Toby so we can do so.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. So what can we expect in terms of new partnership activity in the second half of twenty twenty five and 2026?

Al Sandrock, CEO, Voyager Therapeutics: Well, as I said, we’re always looking to to to talk to anybody about anything. I would just comment that, you know, we have two platforms now. One, a a g the AAV gene therapy capsid platform. But we are also, we have an emerging platform, the the nonviral delivery leveraging these receptors. And and, you know, we’re open to talking to to partners about, about anything, either either, capsid or other license, this is or even asset partnerships as we’ve done with both Neurocrine and and Novartis.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And you’ve disclosed royalty terms for some assets, including SMA. What’s the economic value of your partnered pipeline, and what milestones are, expected next?

Nate Jorgensen, CFO, Voyager Therapeutics: So let me take that one. So this is this is a good question, but it’s a very painful question because the the real answer is it’s massive, the value of those. But as you look at our current market cap, it doesn’t reflect that. So why would I say it’s massive? So there’s 11 partnered programs.

With those partnered programs, there’s $7,400,000,000 of potential milestones. Of those, 2,600,000,000.0 are development. So it’s not a complete bio box. Also in there, there’s high value opportunities. So think about GBA for PD, SMA, Huntington’s disease.

These are high value targets that we get significant royalties. So what else I won’t put a number on it, but I’ll just say it’s definitely multiples of our current market cap if some fundamental investor sat down and tried to value this out. Of course, this market, it just gets it’s difficult to get a deep and fundamental investor to do that.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And with $295,000,000 in cash and runway into mid-twenty seven, how are you prioritizing spend across the antibody gene therapy and platform initiatives?

Nate Jorgensen, CFO, Voyager Therapeutics: Yeah. And the the first comment I’ll I’ll make on that, that $295,000,000 gets us into mid-twenty twenty seven. That is enough to execute on Al’s visions for the next couple of years, but it doesn’t include our milestones payments. So those are on top of it. The milestone payments are on top of that runway guidance.

But in terms of, how we’re looking to prioritize, we don’t actually what I would say is we like all our babies and and, you know, we like the gene therapy that Al talked about. And the shuttle is very high priority for us internally. That doesn’t mean it’s a lot of spend. We just see there’s a lot of opportunity there as we continue to execute on Al’s multimodality vision. And then Al mentioned already that there’s 35,000,000 of milestones that we’ve guided to externally for the GBA and FA programs that we expect in in the near term.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. And finally, what do you think the street is still missing about Voyager’s story, and what are the key catalyst investors should be focused on in the next six to twelve months?

Nate Jorgensen, CFO, Voyager Therapeutics: Yeah. So I think what they miss is we’re not a gene therapy company. We’re a multimodality company, and that’s with Al’s vision from the beginning. And if you want to invest in tau, you should put your chips on Voyager because we have the antibody program, we have the gene therapy program. And in terms of the readouts, near term, we have the shuttle program that we expect to have some data against transfer on this year.

And then next year, we’ll have our tau antibody data. That’s pretty interesting data that’s going to, with the tau PET data that Toby described. And then also this year, Neurocrine is going to file a couple INDs for our partner programs, and that data isn’t too far off. So when I started at the company, you know, a few years ago, we were talking about data a couple of years in the future. But, basically, the future is now.

It’s in the next twelve months we’re gonna have these transformative readouts. So it’s it’s a very exciting time to be here at Voyager.

Patrick Trucchio, Senior Healthcare Analyst, HC Wainwright: Right. Terrific. Well, thank you so much. It’s always a pleasure to catch up with you all and the team and to see how this, incredible pipeline is advancing. So thank you to Al, Nate, and Toby, and thank you to Voyager for attending the conference.

Thank you for all of our attendees for being with us. Have a great rest of your day and a great rest of your conference. Thank you, Patrick.

Nate Jorgensen, CFO, Voyager Therapeutics: Thank you.

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