Good afternoon, everyone. I'm Sean Laaman, Head of U.S. MidCap Biotech Equity Research here at Morgan Stanley, and welcome to our Global Healthcare Conference. Before we begin, make you aware of some important disclosures, and for those disclosures, visit our website at www.morganstanley.com/researchdisclosures. If you do have any questions, please reach out to your Morgan Stanley sales representative. For this session, we have the pleasure to host from Denali Therapeutics, their CEO, Ryan Watts. Thank you and pleasure to host you.
Great to be here.
Yep. A couple of macro questions to begin with. Ryan, we were just debating it then. How is the rise of China-originated innovation changing, if at all, your competitive positioning and your R&D and BD playbook?
Yeah, I think that it's a great question, and it's a relevant question, and it's a question that we ask in really two levels. One is a biotech community, and specifically here in the U.S., how do we view China from a competitive perspective? Then in our case, talking about it around invention and the blood-brain barrier specifically. I think there's varying views, and I often refrain from talking too much about my general feelings about either politics or regional considerations. But I will say that my general impression is that competition is almost always considered, in my opinion, a good thing, because it will ultimately lead to the very best medicines for patients. The more competition there is, the more invention there is in order to either stay ahead or to find the next best medicine.
That's ultimately who wins in that category are the patients. As a company, you view it a little differently. You'd love to be on an island and have a technology that no one else in the world has, and that technology is essentially needed, and no one else can access it. That's not how biotech works. We all live in this biotech world, and when something shows promise, almost everyone starts pursuing it, from Big Pharma to mid-size biotech, even academic labs will start going into it. When I look back 20 years ago, when we started working on blood-brain barrier, there was essentially very few or no one working on it. There were a couple academic labs and some obscure companies that were working on blood-brain barrier technologies.
We picked this up 20 years ago, this is prior to the founding of Denali, and just made everything that was in the patent literature and that had been published and asked, "Do any of these technologies get medicines into the brain?" Usually what happens is, if there's not a lot of people working on it, what was first published doesn't really work. People tried the same thing. They made it, it didn't work. That's not where we are today. With blood-brain barrier technologies, you're now seeing the first FDA-approved medicine in AVLAYAH, which we launched earlier this year. You're seeing exciting data in Alzheimer's disease, where you can take a standard antibody and put a blood-brain barrier technology on it and get 3x faster plaque reduction. As a reality to that, everyone's going to start pursuing it, including in China.
The question is how to stay ahead, how to continue to invent. I think importantly, when you're pioneering a field, it's important to really lay that solid foundation with great science. Recognize you should fear the competition, but if you don't have competition, you're probably going in the wrong direction. My expectation is that China's competition is just going to heat up, including in the blood-brain barrier space, and I think ultimately it should be great for patients.
Sure. Thank you, Ryan. Moving on to another macro consideration, AI adoption. How are you adopting or implementing AI in your business? Is there an example you can give us where it's changed a decision, a cost, a POS on a program?
Yeah.
Yeah.
It's a timely question as AI starts to take over the world, even in an unmonitored way. We, I think like many, have started to experience the absolute value in efficiency, and in some cases accuracy, but really efficiency of AI. I think the most important thing is to take repetitive tasks or tasks that may be prone to bias and implement AI in that context. You can do that in the preclinical setting, you can do it in the clinical setting. I know for me personally, I use it all the time in terms of my analysis. I actually sometimes like to compare or even compete with AI to see do I have the ability to still make those independent decisions that maybe my project and Claude couldn't make. The reality is, it's extraordinary. It really accelerates the mundane.
I don't know yet if it's going to accelerate the actual fundamental discovery. Part of that is because you need the data to train it, and biology's complexity is such that I don't know if we have enough data to really train—
Sure.
—to have that independent thought. But that doesn't matter. In our world, especially now, we have a proven platform, and that platform we need to apply. So application with example is a great place to use AI. We use it in all aspects of our business. In fact, we reward it, we accelerate it, we find any way we can use it. But I think there are areas where it's more advantageous than others, and I think it's really those repetitive tasks where you're looking for consistency.
Sure. I'm asking these questions across all our companies, but this last one's probably very interesting for you, particularly because Denali's just crossed that threshold from being clinical to commercial stage. Which policy variable, is it FDA, Medicare negotiation, MFN, tariffs, or global pricing, matters most to your economics, and what have you changed, if anything, because of it?
Yeah, I think in some ways, I want to say that we had the foresight to do this, but maybe we did, maybe we didn't. But we decided to onshore manufacturing in the U.S. Part of it is, if you look at the cycle of invention and application across biotech, it was the case that everyone externalized manufacturing. I mean, why build your own manufacturing? It's expensive. It takes time. What ended up happening is what used to be inexpensive and super efficient to outsource manufacturing has become extraordinarily expensive because when those are made available, what happens, those companies are trying to really maximize their margins in the way that they interact with biotech. So we made the decision now four years ago to onshore manufacturing.
I think as a result, something like tariffs really matters because you're buying raw material, so you're looking for obviously a paradigm in which you can reduce cost. It's been great for us. I think the flexibility, the speed, and what we manufacture, which are biologics which there are many CDMOs that do this. Ours are actually fusion proteins that are unique. We end up having to build our own parallel teams for process development for AVLAYAH, our first product launch. It was just much easier for us to bring that in-house. I think we're at a point where it's an advantage to do your own manufacturing, I think, here in the U.S.
Sure. Thank you. On to Denali-specific questions.
Yeah.
AVLAYAH a year ago was pre-commercial. Now it's been launched. What can you tell investors about, it's the first Transport Vehicle therapy for neurological symptoms in Hunter, but what can you tell us about the differentiation to the standard of care and why you think there'll be ultimately a displacement of the standard of care with AVLAYAH?
Yeah. I think it is important first to describe briefly Hunter syndrome and what the standard of care is.
Hunter syndrome is one of many mucopolysaccharidoses or MPS diseases. It is a monogenic disease, a loss of enzyme function. What happens is lysosomes become dysfunctional. It is a lysosomal storage disease. I think 30+ years ago, it was discovered that you can take these lysosomal enzymes, you can manufacture them, and you can inject them systemically, and a fraction of what you inject gets taken up into cells and starts to restore lysosomal function. These are the enzyme replacement therapies. It has been decades since that was discovered, and this was a really important class of medicines. The challenge with all of those enzyme replacement therapies is that they cannot access the brain. Of the totality of MPS patients, 70% of them have some type of neurologic dysfunction. It is probably greater if you include hearing.
It might be as high as 95% have some neurologic manifestation. The standard of care does not cross the blood-brain barrier. These enzymes do not get into the brain, and hence there is this big unmet need. I will just give one example. In Hunter syndrome, about 70% of patients upon diagnosis have neuronopathic disease, so they have complete enzyme loss of function. Those patients on standard of care will live on average to about age 15. So they will initially learn how to walk and talk, and then by age five, six, seven, eight, they will lose the ability to walk or sorry, lose the ability to talk. Some will be able to walk and have other dysfunction, toileting, and that is on the standard of care. The reason is that the nervous system is degenerating.
In the case of Sanfilippo, which is another disease we are working on, that degeneration is super rapid. It is like Alzheimer's of childhood. It is a very rapid neurodegenerative disease. There, there is actually no standard of care because basically it is a neurologic disease, and the standard enzymes do not get into the brain. Ultimately what AVLAYAH is engineered to do, it is hitching a ride with an iron transporter. It can treat the whole body. That is important. So we replace the standard of care, but it crosses the blood-brain barrier, addressing the neurologic manifestations of disease. I think the data that supports that was published earlier this year in The New England Journal of Medicine, and obviously that went into the broader context of the filing with the approval in March and now the launch of AVLAYAH.
Sure. Thank you. Maybe talk about when the neurological deficits start associated with Hunter and the optimal time for intervention. You mentioned patients living to [15], but I imagine that the earlier the intervention, the better the long-term outcomes, and that data will play out over time.
Yeah. I think you nailed it, which is earlier is better. I think our data shows that. In fact, we presented data recently that shows neurofilament, which is a marker of neurodegeneration. If you intervene early, the NfL goes down more rapidly. If you intervene later, you still get normalization, but there's a delay in the time before you actually halt the neurodegeneration, probably because there's a lot of neurodegeneration that's happening. I think, again, without newborn screening, which is really where we have to go as a country, newborn screening for all of these diseases especially if you have medicines that can really impact. If you're getting diagnosed at age two, usually it's ear infections. There's changes in skin features and facial features. But the newborn screening is the way to go.
When we think about the AVLAYAH launch, most of our patients right now are switching from standard of care to AVLAYAH, but there are a percentage of patients that are newly diagnosed, and even better if they come out of newborn screen and can start taking this medicine in their really early life and hopefully have a normal life.
Sure. As the, I guess, newborn screening proliferates, do you think estimates of the epidemiology may go up, or do you think that it's a fairly well-observed disease?
I think it's well- observed. The reason for that is that eventually patients get diagnosed. Hunter is a very severe disease, and so you'll miss it, but by age two, three, and sometimes even four, and maybe in the attenuated patients a little bit later, but it's diagnosed. Even on standard of care, just restate that at 15 years of age is the average lifespan of someone with severe disease. So that's happening. I don't think you're going to see the numbers go up, but we hope that the prevalence goes up because this medicine will have a big impact on life and other aspects, not just neurologic, which is a big piece of that.
Sure. Thank you. So far, we've seen the Q2 revenue, I think, was about $3.6 million. You've guided to Q3 at $10 million- $12 million. Of that $10 million- $12 million, are you able to tell us how many patients are new starts versus reimbursement conversions, if that question makes sense?
Yeah.
What particular, a rare disease, I imagine you have a pretty good hold on how the revenue's going to look for quarter-to-quarter as you get to the end of the quarter, if that makes sense.
Yeah.
Yeah.
I'll ask a question just to make sure I get it exactly right. New starts meaning also including switches from ELAPRASE versus—
Correct.
—those that convert from the phase I/II trial.
Correct.
Yeah. Okay.
Yeah.
Right now, the vast majority of our patients are all new starts.
Our goal is by end of year to switch patients on the phase I/II trial to commercial, so that is going to happen over time. Really out of the gate, it is new starts. I do not really have a percentage on that. We did something a little bit unusual. We guided to revenue on a quarter basis. We thought of all the different metrics that are useful, and it is a little complicated in Hunter syndrome because it is weight-based dosing. Obviously, we have a dose escalation. The simplest thing is to take away that mystery, we guided towards what we think will be revenue in Q3. We will likely do the same in Q4, and then we will reassess what we are going to do for 2027. Obviously, this is a great starting point for us. I think really importantly here is this is not a single product story.
It's really the platform that has been validated by this product, AVLAYAH, and its now application across many disease areas. We think that the commercial execution is critically important, and we can build an enzyme franchise that will create a lot of value for patients and obviously for Denali and hopefully for shareholders. But in reality, these numbers don't really matter today. It's can we execute. I think for us, what's been really exciting is to see that commercial team execute. It doesn't have to be a big team. It's an ultra-rare disease. Certainly, with the label the way that it is, we're in, I think, a really strong place in the initial launch of this medicine.
Sure. Thank you. How should we think about the shape of the launch into 2027 without drawing on specific numbers, but do you think it's going to be kind of a rapid conversion of a prevalent pool or a slower ramp over four to six quarters?
Yeah. It's actually early innings, so we don't know how that's going to look. I'll tell you what our dream is. Our dream is to try to hit a plateau as fast as possible.
Sure.
We want to get as many patients in the U.S. onto medicine. We then want to work on the next third of patients outside the U.S. that can benefit from, I think, this transformative medicine. The final third is probably going to be unlocked with our COMPASS trial as we think about Europe in particular. I think there, that's as much about reimbursement as anything. Our goal, the S-shaped launch, in part, is not the patient starts as much as it is dose escalation and growth and as you see a broader range of weights on drug.
Sure. Thank you. Thinking about some of the prescribing dynamics behind AVLAYAH, I think there is more than 50% of commercial lives and 14 state Medicare programs now have coverage. What is the typical timeframe from treatment to first infusion, and where does the access still break down, if at all?
Yeah. The short answer is it is variable.
Yeah.
Obviously, from state to state and from clinic to clinic. I think the two most, there is really three components. One is everything we can do to assess, but there is a limit to that. It is really the physician and the families that drive. That has been probably the most inspiring thing to see in this community is the drive to get these young patients, these young boys on medicine driven by their families and the physicians. Again, early innings, we do not have, and I do not even know if it is relevant, the revenue is ultimately what we calculate, but it has been exciting and inspiring to see that level of motivation to get access to medicine.
Sure. I think you have mentioned 375 eligible U.S. pediatric patients out of a potential of 500. Where is that other 25%, and what is the realistic addressability of the market?
Yeah. The other 25% is essentially adult. If you think about the prevalence of the 500 patients, the long term, you start to see that shift from 70% neuronopathic to 30% attenuated to then about 50/50 because the attenuated patients live much longer. So that's where the other portion of patients, and they're obviously not covered by label. A lot of interest from that community, and there we hope to unlock that with the COMPASS trial data.
Sure. Maybe talk us through the conversion from ELAPRASE. How does that dialogue go?
Yeah. I think the conversion from ELAPRASE, number one, you go back into the clinic. That's the process as you go through the dose escalation process. The 47 patients that were on the phase I/II trial, those patients eventually are now at home infused, and that's the goal. You go back to the clinic, and that we had assumed would probably be a barrier, but the motivation again from families and physicians to see the benefit that AVLAYAH can offer treating the neurologic manifestations. Part of that is just making sure we get to the optimal dose, and we just haven't seen that as a big barrier.
Now, there are probably going to be some patients that are less severe, or families are not as motivated as they've kind of hit a comfortable position of where they are, and that's, I think, where there'll be work to be done.
Okay. Still thinking about some of the launch dynamics here, how concentrated is the prescriber base, and how many treatment centers are needed to reach most eligible patients?
Yeah. It's pretty concentrated in what we'd call centers of excellence in different parts of the U.S. In fact, often families will relocate to be adjacent to those centers. That's critical. Many of them were involved in the phase I/II trial, have a lot of experience with AVLAYAH now. But then there are other areas where it's less so, and that's where work also needs to be done. It also comes down to how connected the families are with the MPS community. But I think we've estimated 80- 100 Centers of Excellence, of which we know many of them and then engage with many of them. That includes also worldwide, but it's pretty concentrated.
Sure. On worldwide, I think you've estimated 2,000 patients.
Right.
What's the ex-U.S. filing sequence, and does COMPASS gate those assumptions?
Right. If I look at the entire Hunter market, I think of the U.S., I think of Europe proper, and then rest of world. You can access most of rest of world now, and we are in that process of doing that with the data from the phase I/II trial, with the accelerated approval. With Europe, it is going to be unlocked ultimately with COMPASS. Really a third, a third, a third. Why do we have that type of visibility? It is because there is a standard of care for the last 20 years, and we have an idea where that medicine is prescribed and where it is used.
Sure. I do have more AVLAYAH questions, but I want to skip forward.
Okay.
Giving you said validation of the platform, I want to make sure we get to—
Great.
—some of these other programs, given we have 13 minutes. On DNL126 in Sanfilippo, the Transport Vehicle platform has its first validation. How should investors view Denali now? Is this a single asset launch story or biologics delivery platform for new medicines with scale?
Yeah. This is zafinofusp alfa, or as what we call zafi. We have its generic name. Simple. We had tivi, now we have zafi. Tivi is now AVLAYAH in terms of its commercial name. Zafi is. We presented eight patients' worth of data at WORLDSymposium this year. Those eight patients were really dose- finding, and it was really insightful because unlike Hunter, there's no standard of care. This is the first time that we're really exploring, and we can't really use a standard looking at what's out there. We explored every other week dosing, frankly, to be optimized for patient families. What we discovered is that actually weekly dosing is much better tolerated than every other week dosing, and that's because it's an enzyme, and these patients have no enzyme. In enzyme replacement therapies, you have to build tolerance, so you need drug on board.
That was point number one. You can look at the study design, the way that we presented it at WORLDSymposium, and this is some detail that wasn't really expressed at WORLDSymposium, but I think it's really interesting. In addition to that, we can drive a better dose with that weekly dosing. What we're able to achieve is greater than 80% reduction in CSF heparan sulfate. Patients can achieve normalization. Those that don't have, like you see with most enzyme replacement therapies, anti-drug antibodies, and so modulating that is, I think, a key aspect. We also see that six out of seven patients had normalized GM3, which is a downstream biomarker. Those are the eight patients that allow us to really think about the dosing regimen and the dose. We then expanded to an additional 12 patients.
Those patients are completing the 49-week now, and that was all pre-specified with the FDA, as we thought about heparan sulfate as the biomarker to drive accelerated approval. This program was selected for the START Pilot Program. We engage with the FDA quarterly in defining the path for approval for this medicine, and now we're in the process of officially locking the database, going through that process, preparing for the BLA filing. We look forward to sharing new data at WORLDSymposium next year, which is basically the additional patients on that trial. We then also plan to launch this medicine from our own commercial manufacturing in Salt Lake City, so this is part of what we were talking about before. One of the biggest factors for us building our own manufacturing.
That'll be the rate-limiting step for the BLA filing, but we're looking forward to 2027 and filing on zafi.
Right. I think you've just answered my next question, but I am going to ask it anyway just to make sure I got it. On [audio distortion] 126, produced an 80% reduction in HS in the CSF. Does AVLAYAH materially de-risk the accelerated approval process in Sanfilippo A, or must the FDA independently validate the surrogate in the disease?
Yeah. I think for the surrogate itself, heparan sulfate, and the belief that it is reasonably likely to predict clinical benefit, that is really solid.
I think what the FDA does is it reviews every application independently, right? The precedent is very important. I think it substantially increases probability of success as using this biomarker, including in Sanfilippo. The FDA got that. You can see what has happened over the last year and a half. I love sitting on this stage today versus last year and all the uncertainty as we are thinking about the path to approval. Our review division has been fantastic. They are rigorous. We spent probably three years arguing why heparan sulfate and CSF actually would be reasonably likely to predict clinical benefit. It was, frankly, the community that came around. The community being the treating physicians, families, and gathering with the FDA to say, "This is why heparan sulfate is the biomarker to predict clinical benefit." That foundation is laid.
I think the future for zafi is just defining the clinical, the to-be-marketed dose, getting the data, showing that, and then designing, importantly, and now very soon beginning our phase III trial to confirm efficacy. That is going to be key. I think that is the bar that the FDA holds, and we understand why that is the case.
Sure. Just a question on the competitive positioning. UX111 could establish a one-time gene therapy ahead of a potential launch of zafi . How do you think about the competitive positioning of zafi?
Yeah. Just to restate, there are gene therapy competitors in Hunter and in Sanfilippo. What's unique about this particular gene therapy is it's delivered systemically, not to the brain. We just feel like there's going to be unmet need even on gene therapy, so there's probably room for multiple, and that's how we thought about it with Hunter as well. Part of it is heterogeneity, but also we have biomarkers, and the biomarkers can tell us, are you really driving robust CSF reduction? If you are, great, but if you're not, we have this medicine that can really drive that with zafi.
Great. Thank you. Moving on to OTV:MAPT and ATV:Abeta programs, which are super exciting. AVLAYAH validated the TfR-mediated transport for enzyme in a lysosomal disease. What does that generally de-risk for oligonucleotides and antibodies in neurodegeneration, and what remains target- or modality- specific?
Yeah. If you look at the history of blood-brain barrier discovery, it was 1989 when the first paper was published using a transferrin receptor technology to get a medicine in the brain. Now fast-forward to 2026 to the FDA approval. I don't want to say it's embarrassing. I don't even know what I was doing in 1989, but that's not unusual for drug discovery and drug development. It's a long road where you're really defining the right properties to drive this. I think in many ways that FDA approval very much just unlocks the field of BBB back to our original question around competition. Everyone's going to start going after. We already see that. We already know that. Specifically for the Transport Vehicle, it's very important to now go after other targets. We have endless number of exciting targets to use the Transport Vehicle for.
First it's in enzymes, now it's in Alzheimer's, and there are many others that there's interest in, and we can do that in different ways. We can do it through partnerships. We can do it through small efforts within Denali. There are academic partnerships, and we'll see some of that, and you're seeing that really blossom throughout the field. But in Alzheimer's disease in particular, we now have a validated approach, and we can put it on the antibody. We can put it on oligonucleotide. Antibodies, we already know. We've done one antibody already. We worked on TREM2 way back when. We saw fantastic biomarker data, really robust activation at about 1/60 a naked antibodies approach in him. So we know it's going to get in brain. We also know that from some of the Roche data. The oligonucleotides is just the untapped potential.
Also highly competitive because now we know transferrin receptor works, and getting these oligonucleotides in the brain will be really exciting. Instead of having to do intrathecal delivery, which has its limitations with biodistribution, and everything that comes along with having to inject in the spinal cord, you can give systemically. So these are the two approaches in Alzheimer's targeting A-beta. People have known about this target, I think 1991 was when APP was first sort of discovered as a gene in Alzheimer's disease. And tau, the other histological hallmark of Alzheimer's, and those are the two targets that we're going after.
Sure. On those programs, which would you have the high conviction of? I do believe that we're going to see data for both programs next year.
That's right.
What data would you be looking for to justify broader development of either?
Yeah. The data next year is around dose. It is like what is the right dose to engage the target? So in one case, we want to remove amyloid plaque, in the other case, we want to reduce tau expression. I think no one would argue with me that right now the clinical data points really strongly towards amyloid as being a positive target. Now, there is some people that still question. I think prevention is going to be key, early intervention, removing amyloid before you have cognitive decline is going to be key. But the way that the data plays out right now is that it appears that plaque forms 20 years before we have cognitive decline. Then you start to see tau pathology rise, and it rises several years, and then its pathology correlates with cognitive decline.
It seems that A-beta is upstream, and it in many ways accelerates tau spreading histologically. They are both very promising targets, and I think much like a lot of disease areas that are complex, like cancer and otherwise, going after multiple targets. The way I describe it is that A-beta is the trigger and tau is the executioner. They both will be important targets. You need to get rid of the trigger. You need to get rid of the executioner.
Then you have a set of accelerators or contributors that many of which are immune modulators, but those are the two real hallmark targets.
Sure. Wonderful. Thank you, Ryan. Moving on to FTD-GRN, which we are calling DNL593 data. It is moved to the first half of 2027 to allow for longer neurofilament follow-up. What would constitute a convincing package? Progranulin restoration alone or do you need NfL movement?
Yeah. I think in this particular program, I would put in the category of high risk, but really high reward.
Part of it is, it is a neurodegenerative disease, but it acts like a lysosomal storage disease. From a biological perspective, it is very similar to what we are seeing with Hunter and Sanfilippo in the sense that the biology is a lysosomal biology. There is a set of lysosomal biomarkers. This is a heterozygous haploinsufficiency of loss of one copy of granulin, which codes for progranulin, a protein that produces 7.5 granulins. The idea is just like Hunter and Sanfilippo, restoring progranulin with a protein replacement. It frankly acts a lot like an enzyme in terms of its lysosomal function. So there, I think the data and the science is pretty clear. The challenge is that it is frontotemporal dementia, which is not Hunter or Sanfilippo. They are a little bit more challenging to diagnose these patients, to say the least.
It is a genetic subpopulation of FTD. When we look at the biomarkers, we realize that unlike heparan sulfate, that is tenfold or 20-fold elevated in Hunter and Sanfilippo respectively, the biomarkers in a haploinsufficient disease are much more modest. They are like 10%, 15%. They are not big differences, but NfL is about four-fold elevated. So this is a real biomarker of neurodegenerative disease. Our experience with NfL is that it can take time. So you can restore the lysosome, but in six months, you are asking a lot to see NfL decline. We have just said, "You know what? Before we see any of the data, let us focus on a big signal-to-noise, a high risk/benefit sort of a paradigm." We can do that. We wholly own the program now.
Yeah.
Gives us the ability to make the decision, now we're focusing on NfL as the endpoint for that.
Awesome. So two Alzheimer's programs, one on FTD. What's your thoughts around ongoing internal development of those programs versus partnering with Big Pharma, given that particularly Alzheimer's would be big studies?
Yeah. The other program that equally excited about, which we haven't mentioned yet, which is Pompe.
Pompe.
I bring that up just because there is a set of enzymes that we can develop on our own. We now have a commercial team. We can hopefully launch multiple enzymes. I think with these Alzheimer's programs, they're definitely the programs that we've had the most incoming interest. Not surprising. These are huge indications, a lot of rationale, a lot of competition, a lot of other cool approaches being taken around blood-brain barrier technologies, that interest remains high. I love a world where we're able to launch our enzymes, generate enough revenue, develop an Alzheimer's portfolio, launch an Alzheimer's. That would be brilliant. That would be brilliant for Denali. But I think realistically, we will continue to be opportunistic about the right partner.
That partner would have to be someone who shares our vision for the right risk-taking and has a set of resources that we do not have. Certainly, global is one consideration.
Right. Thank you, Ryan. I do have time just to go back to this AVLAYAH question.
Yeah.
You may have already answered it. How does COMPASS support the adult label expansion? Would the current [prior] to advanced neurological impairment language apply to adults?
Yeah. Okay, so two separate questions very quickly. COMPASS has Cohort A and Cohort B. Cohort A is ages two to six , and it is really focused on the neurologic manifestations. The other cohort is up to age 26, and that is looking for comparability with ELAPRASE. What is certainly the case is that even adult patients that are attenuated have neurologic manifestations. Hearing, absolutely, and that is some of the most robust data we have generated is looking at hearing benefit. In addition to that, cognitive decline and those aspects, it is really an area of unmet need, and we are excited to see the data and move AVLAYAH forward.
Sure. Well, we're right at time. Is there anything I didn't ask that I should have, Ryan, or a message you'd like to leave the audience with?
Yeah, I think I'll just end. For us, the story is our first product, which validates our platform, which drives our pipeline, and that platform is the Transport Vehicle. We're very excited to be in this very competitive field and seeing a number of companies explore biologics for the brain, something that was started in the late 1980s, and now we can bring it forward.
Wonderful. Well, thanks for your time today, Ryan, and thanks everyone for listening. Thank you.