Great. Welcome, everyone. My name's Jessica Fye. I'm a biotech analyst at J.P. Morgan, and we are continuing the 44th Annual Healthcare Conference with Stoke. First, you're going to hear a presentation from the company, and then we're going to get into some Q&A. So for those of you here in the room, if you want to ask a question, just raise your hand. Someone will bring over a microphone. Or alternatively, if you're listening online, you can submit questions to the portal, and I'll ask them up here on stage. But with that out of the way, let me pass it over to the company's CEO, Ian Smith.
Thank you, Jess, and good afternoon and good evening to some of those that may be listening on webcast over on the East Coast. I want to say thank you to my friends and family that appear to have shown up in the room today to fill some seats, so welcome to you all. Those include investors and analysts as well, but thank you to my friends and family that are here. My name's Ian Smith. I'm the CEO of Stoke Therapeutics. I've been in the role since March of 2025, and I want to tell you, spend the next 20 minutes giving you a brief update on Stoke Therapeutics, who we are, what we're doing, and where we are as a business, and how we experienced a tremendous growth period in this 2025 period and what we're going to do as we go into 2026.
So as usual, SEC statements, forward-looking statements. Please refer to our SEC filings for any forward-looking statements that I will be making. Stoke Therapeutics is a company that I'm going to keep it in plain English, simple. We focus on upregulating protein expression. We focus on what's known as haplo-insufficient diseases and upregulate protein that is lacking in those diseases. And if we upregulate enough protein, we can cause a person with a genetic disease to live a more normal life. That's exactly what we're doing, and the diseases we're focused in. We have a primary focus at this point with a lead medicine, zorevunersen, that is treating Dravet syndrome, where we upregulate SCN1A gene to produce more NaV1.1 expression and have the potential to not only just reduce seizures in these patients, but potentially put a young child on a more normal path of neurodevelopment.
That platform that has led to the creation of zorevunersen is also being used in a progressive loss of eyesight known as Autosomal Dominant Optic Atrophy. We're about to go into the clinic with that medicine. We're also focused on another disease, which is similar to Dravet syndrome. It's a more neurodevelopmental disease called SYNGAP1, where we're focused on the gene of SYNGAP1. There are many more diseases that we're looking at and are in the early stage of our pipeline. Focusing primarily today on Dravet syndrome, we are in Phase III. We've made tremendous progress. We initiated the study in the middle of 2025, where we had the first patient dosed.
We've now, in a study that's sized at 150 patients, we have recruited and randomized approximately 60 patients, and we have another 60 patients that are in an eight-week screening period that will move into the study subject to screen fails and potentially have 120 patients randomized and dosed come early March. This is kind of a funnel, if you think about it. We identify them for the trial. There's approximately 300 of those patients. They then move into screening, and then they move into randomization for dosing, and they move through the trial, which is a 52-week trial. And I'll explain more about that later. The progress in the study has been tremendous.
Just this last weekend, we provided an announcement and a study update timeline with the expectation of completing enrollment of Q2 of 2026, which importantly means that if that's the last patient in, that would support an NDA, it would be the last patient out would be Q2 2027, which then, as you think about timelines, will allow you to plot out a timeline using a rolling submission and therefore approval in 2027 or potentially early 2028, so we've made tremendous progress with a genetic medicine that may change the trajectory of these children's lives, and I'll talk a little bit more about that later as well. We have four years with the data.
Unusually in this disease, because of the way the trials, the early trials were designed, we have studied these children, 2 through 18, but we've studied them in a Phase I/2 study and given them the opportunity to roll over into an OLE study. These children have now been followed for up to four years, and what we've been able to see is a durability and seizure reduction while also seeing some neurocognition benefits in terms of behavior, and I'll talk a little bit more about that later as well. We continue to look at other haplo-insufficient diseases. As I said, ADOA and also SYNGAP1 are next up behind Dravet. And we've got a strong balance sheet that allows us to continue to invest in these medicines, and we have approximately $400 million as we complete 2025, which takes us all the way through to 2028.
And so we're very well capitalized to continue to invest behind the front disease of Dravet and also the pipeline. Dravet is often thought of as seizure disease. It's actually way more than seizures. To give you a description of this disease, if you can imagine a child that more neurotypically develops through two years of age, maybe 18 months to two years of age, but then they plateau. And what I mean by plateau is they're forever a two-year-old. So as a 20-year-old, you stay as a two-year-old. It's a devastating disease. Yes, it has seizures, and they're traumatic and multiple and frequent. But the neurodevelopment of these children is also devastating. So you live in an adult body as a two-year-old, and you still have seizures. And this is all because of a lack of NaV1.1 in the brain. We aim to change that.
When you look at some of the longer-term data, yes, we have reduced seizures. In our optimal dose group that we've taken into Phase III study, we're seeing seizure reductions on top of standard of care seizure medicines in Dravet, but we're seeing seizure reductions that are close to 80%. So they come into the trial on standard of care medicines in the eight-week pre-screen period. They are screened to be on a stable background of standard of care medicines, and then they receive our medicine, and we have seen 80%–85% reduction in seizures. What is also quite astounding about that result is not that we just reduced the seizures by 85%, is that we're now seeing durability of those reduced seizures out to three years, which is really four years because this is three-year data plus the one-year data from the Phase I/2.
And so there is a durability of seizure reduction, which is still one of the main implications and symptoms of Dravet. I also said that these children, unfortunately, grow to two, but then stagnate. And this is a chart of wonderful colors, but what's more wonderful about this chart is actually the lines that extend left to right. I'm just going to take a moment to explain what this is. So as the children come from the Phase I/2 study and into an OLE study, we continue to track them. We do questionnaires that's called Vineland. And Vineland is, if I look out to my friends and family in the audience here and I say, "Hey, Doug, can you hold your hand up?" He holds his hand up. That is a question that is in the Vineland study.
If I ask him again, "Hey, Doug, can you hold your hand up?" He holds his hand up. He's done it frequently now, so he scores a two. So when you look at this chart and you see receptive communication or expressive communication, when they do it frequently, then you score a two. If you do it sometimes, you score a one. If you do it never, which is what Dravet children never do, you score a zero. So when you look at the results that we're gaining, when you go through a questionnaire such as the Vineland-3, you're seeing these kind of results. But as you maintain the therapy that we have over a chronic dosing, you see this continuous improvement year-after-year.
So if I was to go back a couple of slides just for a moment and just maybe dream a little, and this is where the forward-looking statements are, you're a Dravet child on that orange line. You've stagnated at two years old. You now become 20 years. You're still a two-year-old. We in this room are that blue line where we develop and we stabilize. What we may just have the opportunity to do with our medicine is put a child somewhere between those lines. So instead of being a two-year-old when you're 20, is it possible that you're a 10-year-old when you're 20 years old? Now, we don't know the ultimate outcome, but that's what our medicine is doing from neurocognition and behavior. So it's quite amazing.
The company, long before I was involved, made a tremendous decision to actually track these patients in this long period of time. We now have data that goes out to four years. We will have data in a few months that will go out to five years. I hope that the next color is just as bright and is just as long and even longer going to the right-hand side because that will show continuous rate of neurodevelopment of these children. Recently, we had a medical conference in December. We decided to do an analysis that compared our study results from the Phase I, two, and the OLE to a study that we ran a couple of years ago called Natural History. It was a natural history study of measuring children over a 24-month period to see how they may have developed or not developed.
On the left-hand side of this slide, you can see that, and the seizures, you can see that the seizures were pretty much maintained over this 24-month period when they were using our drug. When you propensity match these patients between natural history and also study group, which was only five patients, we achieved a p-value of 0.018. That's with a p with an N of five. We're running a Phase III study with a primary endpoint, and that Phase III primary endpoint of seizures, and our N of the Phase III study is 150 patients. We've planned the study using this natural history data and also the Phase I, two results.
We also looked at the neurocognition and behavior in terms of matching, looking at the same endpoints and the key domains of Vineland and matching the natural history propensity matching the patients between natural history and study group. Again, the N was low. It was around eight patients, or it is eight patients, but it was seven in some. But when you compare that to natural history, what you see is more neurotypical development of these children. Again, compare that to natural history where you're not seeing any development in these children, and you get p-values even with an N value of eight, seven, and eight. So again, Phase III study that we're running is actually 150 patients. Safety profile of the medicine. Well, again, the company has tracked these patients for four years now.
This is four years' worth of data, 800 doses, initially 91 patients, of which 90% of those 91 patients went into the OLE study. We've been able to track those. Yes, some of them have dropped out over the four years. Unfortunately, some of them have died. SUDEP is a very common occurrence. SUDEP is a sudden death because of Dravet. There is approximately 70 patients still in that OLE study. We've been able to track them, and the safety profile, in the summary, is they're very well tolerated drug. We do see CSF protein elevation, but it doesn't manifest itself in any clinical symptoms. Quickly, to just help you understand the design of the Phase III study, it is a 52-week study.
The primary endpoint is seizures at week 28, and the secondary endpoint is week 52, where we measure the five key domains of Vineland-3. Then the patients have the opportunity to roll over into an OLE. We are doing two loading doses of 70 milligrams on day one, week eight. Then, as you can see, we have a 45 milligram maintenance dose at week 24 and week 40. It's 150 patients. The powering of the study is powered to a p-value of 0.1 with a 90% confidence interval for the Vineland-3 results. It's a very well-powered results and a very well-designed study, Barry. We've made great progress. I mentioned earlier, if you're trying to measure it in terms of how far through the Phase III we are, we're approaching closure of enrollment of the 150 patients.
That means the readout could be as early as Q2 2027 and then move using a rolling submission with the FDA, which we can do because we have Breakthrough Designation. That will allow us to potentially have approval before the end of 2027, maybe 2028, but it certainly could be in 2027. Just quickly on the commercial opportunity. Feels bad talking about, feels bad to me talking about commercial opportunity, but what is the opportunity to treat these people? How many people are suffering from this disease? It's approximately 40,000 patients in the seven key geographies. The way that splits up is it's around 15-20,000 in the U.S., North America, and it is 15-20,000 in Europe, Japan, and the U.K. That 40,000 number or 38,000 number does not include Brazil and China and other countries.
This is a large patient population that we have the opportunity to treat. As I said, it's a genetic medicine. If it works by upregulating protein in the younger children, it should continue to work as they progress through their lives. Just quickly, in the U.S., as I said, it's around 16,000 patients. The reason why I'm focused on the U.S. is we do have a wonderful working collaboration with Biogen. Biogen licensed the commercial rights to zorevunersen in February 2025. They fund 30% of R&D development, and they own the commercial rights outside North America. We receive a royalty, but the collaboration is actually less about the financials. It's actually more about the capabilities of the combined companies. We brought the drug. They brought their capabilities. Biogen was our chosen partner in what was a competitive process early in 2025.
Biogen was our chosen partner before we actually chose them because of their capabilities with zorevunersen. They are global with zorevunersen. They manufacture it. It's an ASO. Our medicine is an ASO. And so they're our right partner. And so we're happy to be working alongside Biogen. They give us lots of good advice. We work for them at some times. They work for us sometimes. It's a good working relationship. Quickly, just before I close, we do have a pipeline. One could say Dravet is a proof of concept of our platform for haplo-insufficient diseases. If we can do it in Dravet, why can we not do it in SYNGAP1? SYNGAP1 is very similar. We understand the target. We can upregulate. Potentially, we can change the neurodevelopment pathway of SYNGAP1 patients. And I'm sure a lot of you have been hearing more about SYNGAP1 recently.
If you watch CNBC in the mornings, it is getting more and more aware for that disease. And we hope we can do something in that disease area. And we look forward to close. Just in 2026, key milestones for the company is completion of enrollment in the Phase III. Why is that important? Because when your company is at this stage in development, you should be able by month, maybe even week, to actually pinpoint when you finalize your study, when you file your medicine, your NDA, and when you potentially could have approval. And your activities should be planned out to that manner. So look for the announcement of completion of enrollment because that should set the timeline through to NDA filing, submission, and approval. So that's one of the bigger milestones for this year.
And we will continue to talk and work with the FDA to help them understand what Dravet syndrome is and how our medicine can address Dravet syndrome, looking at the pathophysiology of the disease and how our mechanism of action can address that pathophysiology of the disease. We expect to continue to develop medicines behind zorevunersen, specifically in ADOA. ADOA, we expect the first dose of patient in this first half of this year. And in SYNGAP1, we expect to be identifying a clinical candidate in the first quarter of this year. So more to come to you later this year on SYNGAP1 as well. As I said earlier, we're very well financed. No anticipation of going to the market to raise money. We have been using an ATM, but that was more about building an investor base appropriate for our stage of company.
We have sold some stock from our ATM. In fact, in Q4, we sold approximately $100 million to two tier one long investors that we brought into our investor base. And again, that strengthened our financial position. Thank you for your time. I'm now looking forward to Q&A. I always prefer Q&A rather than presentations. And over to you, Jess.
Great. And just a reminder for everyone in the room, if you do have a question, just raise your hand and someone will bring you a microphone. But I'll go first. So you talked about having a meeting with the FDA to discuss potential expedited paths to bring zorevunersen to patients. Have you gotten the meeting minutes back from that?
Yeah. Just for everybody's benefit, our medicine is defined as a Breakthrough Designation class medicine. When you have a Breakthrough Designation, you have a requirement from the FDA to actually meet with them in what's called a multidisciplinary meeting. In that meeting, you get to discuss the pathophysiology of the disease. You get to discuss the mechanism of action of the drug. And you get to bring the efficacy and the safety data you have at that point to actually help the FDA, work with the FDA to actually expedite development, see if you can accelerate the development of a medicine. That's the reason you get Breakthrough Designation. We actually had that multidisciplinary meeting in December. What was unusual about that meeting for us is we took four-year data to that meeting.
In our briefing book and our first question, we did ask the question, can we file on Phase I, 2, and OLE data? Because that was four-year data. It was mature four-year data. It was also four-year safety data that showed the drug was well tolerated. So we did ask that question of the FDA. We had a productive discussion. There were some challenges with the discussion. I would say the main challenge with the discussion is the data I showed you in this slide deck where the FDA did ask about whether our analysis of propensity matched patients was a post-hoc analysis. We acknowledged it was a post-hoc analysis. However, it did have predetermined endpoints. The patients were matched between natural history and study. So there was an integrity and a credibility to the data, and the FDA were intrigued by it.
And they have subsequently come back to us and asked for more data. Actually, they've asked for our algorithms, our per-patient data, and our SAP plan for how we analyze that data, which was an interesting request. We've submitted that information, and they subsequently provided the minutes. Yes, Jess. So they provided the minutes. And in those minutes, they opened up the discussion both formally and informally to continue to discuss the one key question that we've asked them. I will use this opportunity, though, Jess, as we talked just the other day, to say that because of the shortening of the timeline of the Phase III and that we could be fully enrolled in Q2 of this year, the idea of filing around a Phase I, two, and OLE data doesn't have as much benefit on timeline as it once may have.
And so the spread between running a Phase III and filing and getting an approval versus filing on Phase I, 2 data at this point in time might be about nine months. So as we burn down those months working with the FDA, there's less benefit on timeline. And I would offer there's more that continues to be risk on label. So when you don't go to the FDA with a Phase III, a fully designed Phase III program, there is potential that you don't get all the information on your label that you believe you deserve given the profile of the medicine.
And so I would offer that we have to. I've been waving my hands around the last few days where I say, if you could think about a graph where one of the axes is risk of not getting the optimal label versus timeline saving, you've got to continue to evaluate those. And in the extreme, if we save three months but got a suboptimal label, that would be a bad decision to go file three months saving. And so we're in that mode now because of the acceleration of the Phase III. But your simple question, Jess, yes, we've received back the minutes. But we continue to work with the FDA. They are open for discussion, and they've requested more information. And so we will continue to talk with them. I'll just give you one other color point from that FDA meeting as well.
And I mentioned this to you the other day, Jess. The FDA did offer us to change the trial and move patients from a control arm onto drug and measure seizures at six months. That would have dramatically shortened the timeline because we're already closing on enough patients in our study now to move everybody onto seizures measures at six months. We declined in the meeting. It may have saved us up to a year, but we declined in the meeting because this is more than a seizure medicine. So the FDA is exhibiting regulatory flexibility. And we've seen that with some other companies recently in the neurodevelopmental seizure space. And so they are showing us flexibility. But we're very protective of ensuring our label is representative of the medicine we've created.
This timeline that you're talking about now with the completion of enrollment of 150 patients in the second quarter, does that mean that the 150th patient is starting their eight-week screening or they have completed their eight-week screening and they're getting a dose of whatever they're getting?
It means they're being dosed. Yeah, so I think of it as a funnel. The wide end of the funnel, we have 330 patients just coming into the funnel. And from the funnel, they get put in an eight-week screen period. That's actually the initial start of the study. We have got currently 60 patients in the screen period. We have a very low screen fail rate. So most of those 60 patients will be on drug in eight weeks from now. A lot of them will be on sooner, but no later than eight weeks, which means that when you add eight weeks to today, mid-January, then by mid-February, we should have approximately 120 patients on drug and randomized. And we're trying to get 150 onto drug and randomized. And our target is no later than Q2.
Okay. And the extra sort of 20 patients for Europe, do they factor into your US timelines in any way? And it looks like they're starting enrollment in Q2. So how does that kind of play into this?
Yeah, it's a good question. That's one of the big changes for us. And we also discussed it with Biogen. The study's original design was 150 patients, and it was a lumbar puncture sham control. And that was going to be run in the U.S., U.K., Japan, and Europe. While we were agreeing protocols and having them reviewed by committees in Europe, we ran into a challenge where Europe wouldn't allow for a lumbar puncture sham control. So what we did is we actually changed the European part of the study to have needle prick control, so needle prick sham control. And so we provided those for the European countries, but we maintained the powering of 150. And so we moved up. So effectively, it was 130 and 20, but then it became 150 and 20.
So we maintained the powering for the lumbar puncture sham control in U.S., U.K., and Japan. And it's that 150 patients that we have seen this massive groundswell of people coming in and the acceleration of recruitment into the study. And a lot of credit goes to the education from our medical affairs group, the clinical ops group who have opened the sites. And it shows also the medical need that these families and the caregivers want to provide to their patients or their family members. And it's a really good marker of the acceptance of the medicine. But that's how the 150 and 20 to 170 adds up.
I guess putting aside that cohort, you're talking to the FDA about sort of potentially expediting the regulatory path in the U.S. Are you talking to the European regulators about that or talking to the Japanese regulators about that?
That's more of a discussion for Biogen. So I'd ask you to talk to Biogen about that. I mean, we take their guidance in their territories. Obviously, filing, registration, and approval and commercialization is their decision in their territories. So they would be better to guide you on that.
Okay. And you talked about starting your rolling submissions the first half of next year. When do you expect to complete the rolling submission?
It's a great question. I've got a couple of statistics around this one. We're going to do a rolling submission, which is what you can do when you have breakthrough designation. We will expect to start it in the early part of 2027. The early part of a rolling submission is always CMC and preclinical work. The final part of a rolling submission is usually clinical. It's your last clinical data point that comes into the rolling submission, which typically is around two months. I'm being precise now, but that's how I want to hold the organization accountable. Two months from the last clinical data point should be the completion of the NDA submission. The completion of the NDA submission would then give you a PDUFA date, which would be eight months.
But if you look at medicines that have Breakthrough Designation, they are typically approved within six months of completing the submission. Hence the timeline I gave earlier today, which could be Q4 approval, as early as Q4 approval.
So maybe if we think about kind of fast forward to the products approved, do you see differing degrees of uptake with respect to either ramp speed or peak sales? Should the Phase III demonstrate a strong seizure benefit with meaningful benefit on the Vineland measures versus without? And if those are different commercial scenarios, what do they look like?
So I want to take this opportunity to introduce you to Jason Hoitt. He's our Chief Patient Officer. It's a title that tries to define Jason's role and responsibility at Stoke, which is commercial, medical affairs, and manufacturing. And Jason, do you want to take that question?
Yeah, it's a great question. I think first and foremost, maybe I'll start with the latter first, which is where you have the label that I think we all anticipate, which is disease modification. So you would have seizures and Vineland data suggesting the neurocognitive improvements that we've seen in the Phase I, two. We hear things in market research that are along the lines of most clinicians would anticipate offering this drug to nearly all of their Dravet patients. So I think that is also reflected in what you see in terms of the 330 patients that are in pre-screening and just how quickly we've been able to fill the enrollment in the Phase III. So I think there's really robust demand for what could be the first disease-modifying treatment for Dravet syndrome.
Maybe then looking at the other scenario where it's more of a seizure focus, I think one of the other things we hear in market research when we ask about the unmet need associated with Dravet, whether you're talking to doctors or to caregivers, all will admit there is really substantial unmet need that exists today. And when you ask what the primary unmet need is, even today, with the prospect of disease modification on the horizon, the persistent seizure burden and lack of seizure freedom in patients is the number one unmet need both for healthcare providers and for patients and families, followed really closely by disease modification and targeting the underlying genetic cause.
And so what I would say is with us showing nearly 80% reductions in seizures on top of the best standard of care medicines, with about half of those patients being on Fintepla in the Phase I, two studies, we anticipate that there will be robust demand kind of irrespective of that. But obviously, the slope of that curve will be different if it's a seizure drug versus a true disease modifier like we anticipate.
How confident are you in the potential for a label for the treatment of Dravet syndrome versus when I looked at some other labels there to treat seizures associated with Dravet?
Yeah. So Jess, that's the data that I went through in the presentation. So we have designed the Phase III study with endpoints that would define a disease-modifying medicine. So if I contrast that to the other medicines you're referring to, those medicines' Phase III design was just measuring seizures. In fact, one of the seizure medicines actually tried to measure Vineland results and got a zero, unfortunately, and therefore was registered as a seizure reduction medicine in Dravet. We've designed our study specifically with seizure reductions and the neurocognition and behavior endpoints. That's the Vineland measures. Plus, we're also working with the FDA now to help them understand what observed data would be. It's a lot of detail, but people need to understand that a label is much more than just primary and secondary endpoints.
It has a section on the label called Section 14 where data that you observe in your clinical studies you can put on the label, which means you can also promote to it. You can also explain the medicine using that Section 14 observed data. And our observed data is actually that longitudinal data over four years, which might be the most striking data. If I was a parent of a Dravet child watching those lines go lower left to upper right and seeing that neurocognition and behavior developments in the observed data label, that would be very compelling for me to want my child to be on the medicine. It also helps with value, working with the payers to have that observed data on the label as well as the primary and secondary endpoints. So that's how we've designed the study to define this as a disease-modifying medicine.
Just to kind of double-click on that, is it your aspiration to get not only your Phase III data in Section 14 of the label, but also your Phase I, two data?
Potentially, yes. Yeah. And most importantly, I would like to see the OLE data that's measured longitudinally. The Phase I, two data I'd expect to be in there as well. The primary and secondary endpoints from the Phase III would be in the primary part of the label, primary and secondary part of the label upfront. So yes, all that data. The way we've been explaining it is we're asked questions. It's a common question, actually, we've been asked over the last couple of days. But it is the totality of the data being on the label, which gives us the opportunity to explain that this is more than just primary and secondary endpoints. It's actually observed data. And if you have that totality of data, it differentiates your medicine as a disease modifier.
Okay. What are the best price analogs to consider for a product like this?
Jason.
Yeah, I'm glad you asked that question because I think one of the misconceptions we've run into over time is that this will be anchored to the already approved drugs for Dravet syndrome right now. But the only drugs approved for Dravet syndrome right now are approved to treat the seizures associated with Dravet syndrome and not the syndrome itself. And so that nuance in the language, I think, is incredibly important. And we see this as a true disease-modifying treatment that offers significant value in addition to what's already available today, as evidenced by the robust results that you see from the Phase I, two, and OLE on top of that best standard of care. So I think the more appropriate analogs would be genetically targeted disease-modifying treatments for rare diseases.
I think products like Spinraza, the exon skippers from Sarepta, the CF drugs from Vertex are far more appropriate analogs than any of the anti-seizure meds, even Fintepla.
Can you give us more of an update on the STK-002 program for ADOA and just elaborate a little bit on the timelines for the OSPREY study and the next steps?
Yeah. So we've initiated a Phase I, 2 dose-escalating study with STK-002 in ADOA. That study should dose-escalate throughout 2026 with the potential of reaching efficacious doses towards the end of 2026 and therefore potentially seeing some efficacy results towards the end of 2026. Obviously, the earlier cohorts are more about safety as we dose-escalate. But as we end 2026 and potentially go into 2027, we may see the first efficacy signs. The medicine is actually designed to improve eyesight. It's not to stabilize the decline of eyesight. I want to be very clear on that. We took it into the clinic because of certain animal models, non-human primate models that we ran that showed, based on measuring certain biomarkers, that there was an improvement in vision in the eye that was injected.
And so we're highly hopeful of favorable progression to show improvement in eyesight as we get to the higher doses in this dose-escalation study this year.
Is that kind of a go, no-go threshold for you to advance it?
Yes, actually. It's a great question. These diseases are very difficult to develop medicines in. Stabilizing a disease and a slowly progressing loss of eyesight is a long development path. So yes, when you improve eyesight, I used to work at Vertex Pharmaceuticals, and we once thought about how you slow the progression of loss of lung function. But when we first went into the patients, we improved lung function, which allowed us to actually reduce the duration of clinical studies. This is the same thing. If we can improve eyesight, you will reduce the duration of clinical studies.
What could the regulatory path look like if you do see signals that you're improving vision?
Yeah, the next step out of the Phase I/2 would be figure out how we dose this drug chronically. And so we'd have to understand the equivalent of a half-life and the biodistribution and the maintenance of the drug in the eye and brain. But the next step would be to do chronic dosing.
Okay. Looks like we're just about out of time, so we'll stop there. Thank you.
Okay. Thank you, Jess, and thank you for everybody.