Good afternoon, everyone. Thank you for joining the 24th Annual Needham Healthcare Conference. My name is Joey Stringer, and I'm one of the biotech analysts at Needham & Company. It's my pleasure to introduce our next presenting company, Ionis Pharmaceuticals. Joining us today from Ionis is Head of Investor Relations, Wade Walke, Eric Swayze, Executive Vice President of Research, and Kyle Jenne, Chief Global Product Strategy Officer. For those of you joining us on the webcast, if you would like to ask a question, please do so at any time. You can submit a question using the chat box at the bottom of your screen. With that, we'll get started. Wade, Eric, and Kyle, thank you so much for joining us today.
Good to be here. Thanks for having us on.
We'll start off with olezarsen, your program for FCS and sHTG. I guess let's focus on sHTG for now. Your phase III program consists of three separate trials: CORE, CORE2, are the pivotal ones, and you've got a supportive safety study at Essence. For CORE and CORE2, data coming second half of this year, it'll be a big readout for you guys, and Essence data coming middle of this year. With that backdrop, I want to focus on sHTG. It's a much larger indication relative to FCS, but what have you learned about the FCS launch so far? How's it been playing out, and how's it helping set the commercial stage for sHTG?
Oh, thanks for the question, Joey. I think, if you don't mind, before we start talking about sHTG, I love people talking about sHTG. We think it's a great space for us to be in, but I kind of want to back up just a little bit and set the stage for Ionis as a whole and kind of where we're heading. This is a big deal for us because with olezarsen and the TRYNGOLZA launch in FCS, that's our first independent commercial launch and represents really the pinnacle of the first true Ionis-only step into us being a fully integrated commercial biotech. That is why we're talking about sHTG, and we think it's a great place to be. We are pretty excited about that. We think it's obviously a big change for Ionis.
It's a change in our business model, and we think that it's the right avenue for patients and shareholders as we go forward. TRYNGOLZA is really just beginning to talk about sHTG and TRYNGOLZA. We also have a PDUFA date coming up in August for donidalorsen, which is the potential treatment for Hereditary Angioedema , where we have some great-looking phase III data and really fantastic switch data. We have a late-stage program in zilganersen for Alexander disease, which is a rare leukodystrophy, key for our neurology programs that we envision as fully owned. Other than that, is our Angelman Syndrome program, which we also intend to commercialize ourselves and bring forward for, again, a disease that has no therapy. We're hoping to get enrollment for that completed next year.
These kind of really set the stage, and we'll talk about sHTG in a minute, for Ionis' fully owned commercial programs, which we think can return some tremendous value to patients and shareholders. If you want to talk about sHTG and the commercial phase, that's why we got Kyle on, and he can start to talk about that.
Yeah, thanks, Eric, and thanks, Joey, and team for joining the call today. I'll just mention briefly, as you asked, about the FCS launch. It's going extremely well. We did a great job of building commercial infrastructure on the efforts of WAINUA first and our co-commercialization effort, but then being ready for our first fully owned launch with TRYNGOLZA and FCS, which we received approval at the end of last year. The performance is very impressive from the commercial team and the medical affairs group so far. We have product in channel before the end of the year. We had patients on drug before the end of last year, and we are continuing to identify, get patients diagnosed, and get patients onto both commercial and Medicare drugs. We are very pleased with how this is going so far in the execution on the plans.
As it relates to the synergies that you brought up around FCS and sHTG, there are a number of those. When you think about the sHTG indication, which is the much larger indication you referenced, we're talking about triglyceride levels of 500 mg per deciliter and above. This is due to primary and secondary causes. This includes things like diet and lifestyle and other medical conditions and potentially certain medications that patients are on that create this issue. These patients are at high risk of acute pancreatitis, and the number one goal is to reduce that risk of pancreatitis, and it's the most important reason to treat sHTG.
A lot of physicians understand this, and they understand the risk, but unfortunately, in today's situation, they've got very few options available to them, and many patients, even on standard of care today, are unable to get their triglycerides down to the goal due to effective products. In terms of our infrastructure, as I mentioned in the synergies there, it seems like our market access teams, our operational groups, and the basic foundations, kind of the back office staff for commercialization, there are a lot of synergies there. As it relates to the sales organization, we have a great opportunity. We've got about 30 people or so in the field today, and we'll have the ability to scale that probably in the range of about 200 people or so as we get to sHTG in order to capitalize on the common call points that are out there.
The main focus is on endocrinology and cardiology and lipidology. They're the ones that are treating this condition and see these patients. We also have some other synergies as it relates to our Ionis Every Step program, which is our patient support program. I mentioned the co-commercialization of WAINUA. We have the lead in that with AstraZeneca, and we've been able to build a really strong and capable team around our patient services group. These are a group of nurse case managers that are actually Ionis employees. They're able to offer a full suite of services, kind of a white glove service for these physicians and patients. For patients, we're able to do disease state, diet education, nutrition education, and also obviously patient support around injection training and also reimbursement support if needed.
On the physician side, we're able to do things like streamline the prescribing process, support the insurance authorizations, and do coverage assistance and things. In terms of synergies, I think in terms of the market itself and what we're seeing in the market, and also in terms of our capability in commercial infrastructure, there are a lot of commonalities here and a lot of good things happen.
Now, on the clinical side of things, CORE and CORE2, primary endpoint is percent change in fasting triglycerides, but can you talk about the importance from both the clinical and commercial perspective, the key secondary endpoint, in particular the acute pancreatitis? How big of a deal is AP for patients and payers?
Yeah, Eric, I'll take this one as well and talk through this a little bit. First, we're really looking forward to learning more about the benefit that olezarsen can bring to patients with sHTG as we report on both CORE and CORE2 data in the second half of this year. It is coming up here fairly promptly. The CORE and CORE2 studies are powered for the primary endpoint, which is percent triglyceride reduction from baseline compared to placebo at six months. However, the positive thing that we've got going right now is we've got the results that we saw in the FCS Balance study. This gives us great confidence in terms of potential to see a benefit in AP in CORE and CORE2, be it in trends and/or statistical significance.
It's important to note as well in this category that there are already established guidelines in both endocrinology and cardiology in place for sHTG, and treating physicians firmly believe that reducing triglycerides as much as possible, that you need to reduce triglycerides as much as possible because of the severely elevated triglycerides and the risk, again, of pancreatitis. In CORE and CORE2, we've enrolled over 1,000 patients in these two studies. Baseline triglycerides were 836 mg per deciliter in CORE and 749 in CORE2. In the combined study, we've got about 40-50% of the patients, excuse me, the study participants, at greater than 880 mg per deciliter. The study is designed, and really the baseline demographics from these phase III studies really increases our confidence in terms of the potential to generate robust data in these patient populations.
Unlike FCS, patients that have sHTG, they've got functional levels of LPL, which means they've actually got greater ability to metabolize triglycerides than people with FCS who don't have LPL activity. What we believe that we can reasonably expect are higher levels of triglyceride reductions in sHTG, and we also expect a little bit of a lower rate of AP just because of the greater impact of triglyceride lowering that the much larger size of the study represents. We've got a study size that's about 10 times the size of study size that was in FCS. That gives us some confidence that we can actually see a benefit on AP in sHTG patients, but we'll wait and see how the data reads out, obviously. These patients are at risk of AP.
We know that the need is to bring these patients down and get them to goal to prevent the initial AP attack from ever happening if patients haven't had one before or reduce the risk of a second or third attack, which actually increases exponentially after you've had your first attack. We believe the AP trends can be beneficial to payers in the US specifically, but we don't think we need that to achieve statistical significance in order to get reimbursement and access to the drug based on our market research and the time that we've spent with payers thus far.
Do you have to hit the primary endpoint in both CORE and CORE2 for regulatory approval? How do you think regulators will view a successful outcome?
Yeah, as I mentioned a minute ago, we're really well-powered in each of these studies to hit the primary endpoint, which is triglyceride lowering. Both studies enrolled considerably more patients than we needed for our powering assumptions. TG lowering is important because of the medical guidelines that I was referencing just a few seconds ago. Things are in place right now, and we've been able to demonstrate in FCS that patients with TG lowering results in reductions in AP events as well. We've got that already. From a regulatory standpoint, we'll just need to hit the primary endpoint and demonstrate triglyceride lowering in order to potentially get the product approved.
Got it. From a statistical analysis plan for the two CORE phase III trials, can you walk us through that on the primary trig endpoint and the AP secondary endpoint? You touched on it a little bit before.
I can take that if you want. On CORE and CORE2, we've published fairly recently in the American Heart Journal some of the study design, the powering assumptions, and baseline characteristics of the patient population. You can look online at the American Heart Journal website and pull up that paper. Basically, we've got the powering assumptions in the study to show with fewer patients than we actually enrolled that we're able to get 90% powering to hit a 60% reduction in triglycerides or greater in the patient population in both CORE and CORE2, which, since we over-enrolled in both those studies, we're well-powered to do that in both studies. We're pretty confident that the studies are designed appropriately.
We have already mentioned that the baseline characteristics indicate that we have a pretty severe patient population with only 50% of the patients in each study having triglycerides above 880. They are pretty severe. They have pretty high triglycerides. Once you get above 880, your chance for acute pancreatitis increases, that risk increases in an exponential manner. We think it is a good population to have enrolled, and it would be a very steady design.
Sorry if I missed this before, but are the patients stratified between CORE and CORE2 on trig levels?
They are. We stratified them above 880 or above and below 880, and we have a secondary analysis that looks at triglyceride lowering in those two different populations.
Okay. Got it. What are your updated thoughts on pricing strategy for the larger FCS indication? What are reasonable costs to think about on price?
Yeah. So we've priced FCS as an ultra-orphan or ultra-rare disease. So we're close to $600,000 annually on the FCS population, which has about 3,000 patients in the U.S. For the sHTG population, we're talking about potentially addressing 1 million- 3 million patients. So we'll need to bring the price down, obviously, to probably specialty pricing. So the $10,000-$20,000 range is kind of directionally where we're headed. We've got a lot more work to do. We also need to see how the CORE and CORE2 studies read out, see what data we have to differentiate the profile of the drug and work with payers on that. That range of $10,000-$20,000 is kind of directionally, hopefully, helpful for you guys.
Yeah. The FCS market, do you think that's a winner-take-all market, or what product profile will ultimately win majority market share?
Yeah, that's a great question. First, we are super excited that we've got first-mover advantage in this market. Currently available products, unfortunately, just aren't significant enough in terms of triglyceride lowering to help physicians and patients reach the goal and to come out of harm's way of having acute pancreatitis or other events, essentially. First-mover advantage here, we've got about an 11-month advantage in FCS that we're capitalizing on now. We've had about a two-year advantage in sHTG. I think first-mover advantage is a key part to this. The other thing I'll just mention is the product profile for olezarsen and TRYNGOLZA is very, very strong. We've got substantial and sustained reductions in ApoC-III. We've got substantial and sustained reductions in triglycerides. We've got the acute pancreatitis data in the label for FCS today, which is supportive evidence as to why these patients need to be treated.
We've got a strong safety profile. We've got the ability to self-administer with an auto injector similar to what we have with WAINUA. Very strong data and evidence, and we're anticipating the same from CORE and CORE2 as those studies read out. The final thing I'll mention is we've got a really strong commercial team and medical affairs group. We have very strong talent that's joined the organization with numerous years of experience, and they know how to build a commercial team and launch execution, and they really know how to make a difference for these patients and physicians. I'm really encouraged by the commercialization efforts as well.
Got it. And speaking of WAINUA, obviously approved in December of 2023, had most of solid $42 million in the fourth quarter of 2024 sales. Can you talk about some of the launch metrics and how these are trending in the drug's first full-year launch?
Yeah. We have been so pleased, really, with the launch in polyneuropathy, and we're really excited that we're on track as well for the data in cardiomyopathy in the second half of 2026, which is obviously a much larger indication in the ability to serve two indications. AstraZeneca has already provided peak sales potential in the multi-billion dollar range for WAINUA. We've got very attractive royalties on this program up to the mid-20% range in the U.S., in the mid-teens ex U.S. It is going to translate into substantial royalty revenue for Ionis. As it relates to WAINUA, as you were asking about, we've gained a lot of momentum last year throughout the year to serve the polyneuropathy indication specifically. We've captured just over 50% of the total U.S. growth in polyneuropathy product sales from Q3 to Q4.
New to brand share is over 40% now, so we are competing very, very effectively in this space. It is great to see sequential growth throughout the year. We have strong demand, as you mentioned, in the fourth quarter, with sales nearly doubling compared to the third quarter. Upkeep continues to be strong, not only in the centers of excellence, but we are now starting to see the broadening years in the community setting for this patient population. The main focus on this launch is newly diagnosed patients. We really are looking at naive patients to get new to diagnosed patients started on WAINUA. That is where AstraZeneca's focus is. We are seeing some switches. We are seeing some patients that are used in combination with some of the stabilizers that are out there.
Really, the focus is on growing this untapped market right now and getting WAINUA to be the product of choice. The reason that we're seeing physicians use this product as a first-line treatment of polyneuropathy is a couple of things. First, quality of life improvements. We're seeing the polyneuropathy be controlled and well-controlled month over month. The second thing is the profile with the ability to self-administer with the auto injector. This, again, allows a lot of flexibility for patients to manage and control their own disease. The third thing is around access. Both in the commercial and the Medicare setting, the majority of patients have a $0 out-of-pocket expense for WAINUA. It is very affordable for these patients to have access to. We are really encouraged that the profile is playing out very positively.
We're seeing the growth that I just explained, and the profile overall of the drug is competing very effectively in the market.
Yeah. Switching to cardiomyopathy, big picture question on the market. How big do you think the total market is in cardiomyopathy, just given what we know about tafamidis sales and some Pfizer commentary that the drug only has 20% market penetration?
Yeah. The market is definitely underserved across the globe. I think when Pfizer originally was launching tafamidis in this space, they said it was about 100,000 potential patients, I think. The estimates that we're hearing now are probably 300,000-500,000 patients potentially in cardiomyopathy. I think as you see more drugs come to market and more companies enter this space, I think you're going to continue to see the growth of this market. I think ultimately this is north of $15 billion. We're hearing somewhere between $15 billion-$20 billion in terms of the size of the market.
Again, what we're trying to do is use Ionis's deep knowledge in the ATTR space, combined with AstraZeneca's significant commercial reach globally, where they can hit the markets very fast and be able to get to these untapped markets over time and see WAINUA be accepted and ultimately become the treatment of choice for these patients all over the globe.
The cardiomyopathy space, it's competitive. Obviously, there are two approved oral drugs, stabilizers, silencer recently approved. I guess, big picture, how do you view this market, or how do you see this market playing out given the competitive dynamics around those three competitors? Also thinking about the potential for tafamidis LOE in 2028, and any other factors that we should consider that play into your long-term outlook?
Yeah. I think you're already seeing this with the new drug approved in the fourth quarter of last year, for example, that there is definitely an additional need for different therapies and different treatments in this space, not only from the stabilizer class, but obviously from the silencer class as well. We are really looking forward to seeing the data from our landmark study in the second half of 2026. Right? Just as a reminder, we've got the CARDIO-TTRansform study, which is the largest and the most comprehensive study in the ATTR-CM space to date. It's designed to deliver just a robust data set in a very broad population of patients, including those that are on or naive to stabilizers. That represents the dynamic and the evolving space that you were just referencing.
We expect, again, as I was talking about the profile of WAINUA earlier, the efficacy and safety, the monthly self-administration, the expertise that we have combined with AstraZeneca's reach and obviously cardiovascular leadership all over the globe, that we believe this can be a treatment of choice for ATTR patients. Again, I mentioned the market size, $15 billion+ , you're going to see multiple treatments, we believe, and do very well in this category. We believe that WAINUA is going to be one of those that performs very, very well, with estimates north of $5 billion globally, according to AstraZeneca.
Yeah. You guys mentioned that there is one person in the phase III cardiomyopathy trial. Can you just briefly outline the trial design there and highlight some of the key design elements that you think give you confidence that you're going to have the right approach here, number one, and the results set you up for long-term success in this space?
Yeah. I mean, as Kyle alluded to, it's the largest study by far in the state, so it's more than twice as big as other studies. We made that decision a while ago to increase the size of the study when it became apparent from the study that was out there that patients were doing a little better with cardiomyopathy and were more severely affected as, for example, with the tafamidis trial. I mean, as Kyle alluded to, as a result, we think we have the ability to deliver a really robust data set in a lot of patients, with patients both on monotherapy and also patients that are already on a stabilizer such as tafamidis. We've got a lot of patients in the trial in both situations. Primary endpoint is a composite outcome of cardiovascular mortality and frequency of recurrent events at week 140.
We think it's a pretty good trial. It can generate a lot of data, including on subgroups, those on stabilizers and naive to stabilizers, which kind of represent how patients get treated in the real world, we think. We also built in some nice, in my view, nice cardiac imaging sub-studies. There's an MRI sub-study and a scintigraphy sub-study, which we think will get additional valuable data generated about the potential benefits of [audio distortion] in patients with cardiomyopathy. We think it's a very nice program. It'll give us a great data set, and if it's successful, will set us up and AstraZeneca to bring the drug to patients.
Got it. Great. Helpful discussion on WAINUA and the ATTR study. I want to switch to donidalorsen for HAE. Obviously, it's a new drug coming out in August of this year. Start off with a market size question. There are multiple prophylactic therapies that are approved, like TAKHZYRO, ORLADEYO, Haegarda. Can you outline what the current prophylactic market size is in terms of number of patients and sales?
Yeah. I'll start with the global market. This is north of $3 billion for HAE treatments globally. There are about 20,000 patients in the U.S. and Europe today that are diagnosed with HAE. The prophylactic market obviously continues to grow in the United States, which is primarily a switch market, right, because the majority of these patients, upwards of probably 70% of these HAE patients in the U.S., are on a prophylactic treatment today. Outside the U.S., it looks a little bit different. The prophylactic treatment's gaining ground, but it's more of a growth story. It's a marketplace to be developed. Our estimates for donidalorsen will be with peak sales north of $500 million.
As I mentioned, it's going to be a switch market, so it's going to take a little bit of time to transition these patients from their current therapies that they're on today and for physicians and patients to gain experience with donidalorsen. We will go after newly diagnosed or naive patients as well as switch patients. The bulk of these patients in the U.S. obviously will be switch patients. I think what we know about these patients in the U.S. is that many of them are unsatisfied with their current treatments. They're unsatisfied for really three different reasons. Either it's not effective enough, and they're having breakthrough attacks, and they're not well-controlled. They are not tolerating their treatment very well because some of these treatments have very large bolus injections.
You have to take them very frequently, which is the third thing, the convenience factor of having to administer this on an every two-week basis or if you're on an oral, having to do this every single day. But donidalorsen, I think, has an opportunity to bring something different for these patients, not only really strong efficacy and control as we've published, but also tolerability in terms of the ability to self-administer with the auto injector. And then also the convenience. We're shifting out the dosing regimen from every two weeks, which is the standard today on the injectables, to every four weeks or potentially every eight weeks with this treatment. We believe we're going to be able to compete effectively, and we've got some unique data sets and reasons why in terms of the product profile itself.
Now, HAE is a rare disease that kind of sits outside of Ionis's current commercial focus on CV metabolic and CNS programs. What gives you the confidence that you can execute commercially in this indication?
Yeah. I'll start with the fact that the performance in the clinical trials was so strong, right, to carry this program forward and for us to pursue the HAE marketplace. We had to have a convention product that we believed in, and we have exactly that, as I just mentioned. The data around the efficacy and the tolerability and convenience, the switch data that we've generated demonstrating that if patients move from one of the existing prophylactic treatments over to donidalorsen, they do better when they do switch, and they prefer donidalorsen once they're on it. When we started to see that data, thinking about our synergies that we do have, we talked about the synergies between FCS and sHTG earlier. When you think about the synergies in the back office, you think about our patient support program and also things like market access, etc.
This is a program that we can layer into our commercial portfolio and do really well with, north of $500 million, as I referenced, but also be able to have those other synergies. Where we will have to build is on the field force size, right? We will have a standalone field force dedicated to HAE. Being in this class of medication and how profitable this treatment is, we believe we can compete effectively and that it will be a valuable return for Ionis to be able to stay on this market, take this program forward.
Great. The pelacarsen program, partnered with Novartis. Can you briefly walk us through the economics to Ionis?
I'll take that one. We're really excited about pelacarsen. It's a medicine we discovered. It's a risk factor, cardiovascular risk factor called Lp(a) and it's one that Novartis has currently in phase III study that's on track to be out in the first half of next year. This is one where we've got with Novartis, Novartis has estimated that there's going to be peak sales in the multi-billion dollar range for the residents that gives us a royalty that is in the mid-teens to low 20% range. We're also eligible to receive more than $1.2 billion in payments for pelacarsen under our agreements with Novartis and also with Royalty Pharma on this program. Our next milestone payment for this would be a $50 million milestone payment on NDA acceptance.
Great. That's a very helpful Wade. Novartis stating up in late January that the top line data for the phase III CV outcomes trial for pelacarsen, it was shifted from the second half of 2025 to the first half of 2026 based on blinded event rates . I guess, how should we view the potential impact of that shift on the trial outcome?
Yeah, you bet. It is an event-based study. The only thing that's changed is the estimates when we achieved the events needed for the power assumptions. There was a paper published last week on the study design and baseline characteristics of this study. It indicated that it was powered at 90% power to achieve about an 80% or 20% risk reduction or hazard ratio, so about a 20% risk reduction in the overall patient population. The extension of the readout timing doesn't affect power assumptions, so the probability of success just meant that the initial estimate for the timing for reaching those events was off a little bit. We are very confident that the study design is appropriate for the powering.
If you look at the baseline characteristics, it gives us also confidence because the median Lp(a) level is 108 per deciliter, well above the risk threshold of 50. Even above the higher risk threshold of 90 mg per deciliter, which is one of the subgroup analyses that we're looking at in this study. About 80% or so of the patients are above 90 mg per deciliter, in the higher risk subpopulation. Looking at the baseline characteristics, the study population, and the study design gives us confidence that this study can give us robust data on whether or not having substantial lowering of Lp(a) can reduce the risk of cardiovascular events.
Anything else weighed on the stats plan for the phase III? You touched on it a little bit, but my understanding of the trial is looking at CV risk reduction from the endpoint for both the 70+ and then the 90+ per deciliter. Can you explain the significance of these analyses? I guess, how will these be how do you think these will be viewed by regulators? What would this mean from a commercial opportunity perspective?
Sure. The first endpoint is in the total population, which is patients who have Lp(a) greater than 70 mg per deciliter. That is, again, 90% power to see a 20% risk reduction. In the subgroup that is greater than 90 from the study, we also have a primary endpoint there that looks at the CV risk reduction that is powered to give us 25% risk reduction at a 90% powering. Pretty good opportunity to hit that one as well. If we hit either one, it is a positive study, basically. I would say if we only hit it in the greater than 90 mg per deciliter, that would probably limit the label to patients greater than 90. Although you never know, given that there is no treatment for high Lp(a), exactly where that will end up in labeling, that is part of the discussion.
That could be a potential outcome if we only hit it in the greater than 90.
Got it. Last, we want to briefly touch on Angelman Syndrome, how they tier the program there? Can you briefly outline the MOA here and what is the commercial opportunity in Angelman Syndrome?
I'll start and jump into the commercial opportunity if I mangle it. Angelman Syndrome is a rare disease, but it's a pretty big rare disease. In research, kind of thought of it as a Spinraza-like type of opportunity. It has a huge unmet medical need. It's a debilitating neurodevelopmental disease where the patients really never develop beyond a very young cognitive age. It's a tremendous burden on families and caregivers. They're desperately in need of disease-modifying treatments, and that's what we set out to develop and what we think we have. Our drug raises the levels of UBE3A protein, which is what's deficient in the disease. We've shown that preclinically, and we showed that in our phase I of HALOS study where we demonstrated consistent and meaningful improvements relative to natural history on a whole bunch of functional areas.
More than 90% of the patients showed clinically meaningful improvements relative to natural history and overall Angelman Syndrome measure of test called AS-CGI. We also looked at specific measures in terms of communication, cognition, and motor function on things like the Vineland and the Bayley- 4, which would be used for primary on our phase III study, which we plan to get started the first half of this year and hopefully complete enrollment next year. There we have a cognitive expressive communication endpoint for the primary on the Bayley- 4 scale. We think it's a great opportunity with patients who really need to use the therapy.
What do you think differentiates Ionis's approach and program in Angelman relative to some of the competitors out there?
I'll start with the drugs. We use what I'll call a validated chemical class. It's the same type of molecule that led to Spinraza and the same type of molecule that is currently a phase I- B study run by Biogen with the MAPT tau-lowering drug BIIB080, which we have nice data on that has lowered target broadly in the CNS. You can see it with PET imaging. The class has a proven safety record, consistent and validated dosing ranges. We kind of know how to dose. We know the safety. We know the durability, which I really think helps us design our trials. We benchmark our drugs against everything out there in a model that predicts human performance or human gene in a mouse, and we think it's the best way to do this.
We're very, very happy with the activity and duration and profile of our drug. As far as the phase III trial, we have a placebo-controlled approach for our study, which we think is the gold standard to evaluate safety and efficacy in clinical trials. It also helps simplify the burden on the site a bit because you just need one team to measure the drug. Back to the molecule, we don't have to use a tilt table. We don't administer corticosteroids. We have no load. The profile of our drug and the way it's administered, we think it's consistent with the way we've done all of our intrathecal drugs based on our vast experience in the space. We think the molecule class is right for this patient population. We're clearly running out of time because our lights are going off .
Not moving around enough.
That's a timer.
No, we are up on time. It was good to see you coming in. Thank you, Wade, Kyle, and Eric for participating. It was a very helpful discussion.
Thanks for having us, Joe.
Thanks everyone else for joining on the webcast. Everyone have a good day and a good rest of the conference.