To the 44th Annual JPMorgan Healthcare Conference. My name is Anupam Rama. I am one of the senior biotech analysts here at JPMorgan. I am joined by my squad, Rati Pinhe, Joyce Zhou , and Priyanka Grover. Our next presenting company is Camp4, and presenting on behalf of the company, we have CEO Josh Mandel-Brehm.
Thanks, Anupam. Thanks to the JP Morgan team for inviting us here. It's a real treat to be here again, and I have to say, coming into this year, having been at Camp4 for nine years, this is one of the most exciting years that I think we're all looking forward to, and I'm looking forward to telling everybody about that today, so Camp4 is using antisense oligonucleotides to very selectively increase gene expression, and I think it's really important to note that we are a product company with a powerful platform, and I'll talk a little bit more about that today, so I have three things I'm going to focus on today. The first is our flagship program in SYNGAP1-related disorders, or SYNGAP as I'll refer to it today. This is a genetic haploinsufficiency CNS disorder. This is a devastating disease. There are no approved treatments for this disease.
There's no disease-modifying therapies in development. We will be the first program to be entering the clinic with a disease-modifying approach, and there are tens of thousands of patients suffering from SYNGAP around the world. Our lead program for SYNGAP is CMP-002. This is an antisense oligonucleotide designed to specifically and selectively increase SynGAP protein and restore healthy protein levels, which we believe will have a disease-modifying impact for SYNGAP patients. What makes us really excited about this program is all the preclinical data we've generated to date, and as I'll point to as we go through it, a lot of this data we think is highly translatable to the clinical readouts. This program is currently finishing its ongoing GLP-tox studies, and we are moving heaven and earth to get this into the clinic later this year. We will provide some more details on that later in the presentation.
And then lastly, as I referred to, we have a platform, and that platform is what we're using to build a deep pipeline behind SYNGAP. There are other SYNGAP-like opportunities: developmental epileptic encephalopathies, or DEEs. These are very similar in SYNGAP in that they are haploinsufficiencies, they are devastating diseases, they have no approved treatments, and they're the ideal approach for our platform. But those aren't the only types of things we can do with our platform, and business development is going to be very important for us as well, and you'll see more of that in the future. So I want to start with one of the most important insights that makes us special and why I make the claim that we can develop many different types of products, and that starts with the science. So what I'm showing on this screen is called regulatory RNAs.
This was an insight developed by Camp4 and one of our co-founders, Dr. Rick Young out of MIT. Regulatory RNAs arise out of enhancers and promoters. Every protein-coding gene in our body has its own unique set of regulatory RNAs that it relies on to control its expression. So that's insight number one. Insight number two is that we discovered at Camp4 that when you drug these regulatory RNAs using antisense oligonucleotides at very precise locations, you can drive a very, very selective increase in a protein-coding gene, and that comes in very handy for the types of diseases where you're missing healthy levels of protein. We've built a platform called the RAP platform that allows us to take advantage of those two insights. So the first thing that we do is that we map cells.
We take any human cell type, we apply our platform to that cell type, and we generate billions of different data points that then go into a system that essentially turns gene expression into an in silico exercise. That allows us to create a catalog of regulatory RNAs for any particular cell type so that anytime we have a protein-coding gene of interest that we want to upregulate, we go into our system and we can identify the dominant regulatory RNA that controls that gene. Once we have that in hand, we use state-of-the-art antisense oligonucleotide chemistry to precisely drug that regulatory RNA and get a maximal increase in the protein-coding gene. And then lastly, depending on the tissue or cell type that we're going to, we can optimize that chemistry to have the desired pharmacology and safety effects for that particular disease.
One of the taglines that I think is really important for Camp4, and it's about the patients and it's about our shareholders, is that we've told our investors, "We want you to buy the assets and invest in the pipeline, but we're going to use our platform to essentially fund the future start of the pipeline that we want you to buy into." So as I mentioned before, we have our flagship SYNGAP1 program. That tox study is ongoing and will be concluding by the middle of this summer, but at the same time, we're already talking to regulatory agencies about entering the clinic later this year. We do intend to announce at least one additional DEE program behind SYNGAP later this year. We are actively working on quite a few different programs that will follow suit to that.
And then behind that, as I mentioned, there's a lot of potential for our platform that we want to unlock. We just in December announced a formative relationship with GSK. It's a two-target deal. One of the targets is a large disease in the CNS. The other is in the kidney, an area that we are building out in conjunction with GSK. So those types of deals bring us non-dilutive capital. They allow us to apply our platform in areas that we otherwise would not have done right now. And very importantly, it allows us to bring forward other programs by virtue of the other partner carrying them forward where we collect milestones and royalties.
I grew up. I started my career at Genzyme, and one of the things I learned there is that we start with the patient and we do the same thing at Camp4, and a picture is worth more than words. This is Tony. Tony is 11 years old. He suffers from SYNGAP. Tony was diagnosed with SYNGAP once he missed developmental delays and had his first seizures. He suffers from learning disabilities, seizures, cognitive impairment, anger, and is a big part of this because they can't communicate very well. On the right, I'm showing you Tony's lovely family. In fact, Tony's father, Mike Graglia, is the President and CEO of Cure SYNGAP1. He's a force of nature. I always tell people to go talk to him.
A lot of what we're going to talk about today. Mike had a large hand in creating awareness for this disease, but I also want to point out this just isn't a disease about the patients. It's about their families and caregivers. If you go up and you talk to any SYNGAP parent, ask them to roll up their sleeves, and you'll see the bite marks on their arms from having to really try and restrain and help their children who are just frustrated and having to deal with this disease. So this is devastating not only at the patient level, but the caregiver level and the family level. I'm going to talk in the next few slides about the disease itself, the epidemiology, how it's diagnosed, then I'm going to shift to some of our preclinical data that has everybody so excited and giving hope to these families.
And then we're going to talk a little bit about what's to come in the next few years. One thing I want to point out on this slide is, as I mentioned earlier, SYNGAP is a haploinsufficient disease. What that means is one of your two alleles is no longer functional. It has a mutation, and in this case, you only make 50% of the amount of protein you need to be healthy. So this is a really nice slide summarizing at a higher level what these patients are suffering from. Many people think about SYNGAP as an intellectual disability as well as a genetic epilepsy. That is 100% true. However, these patients are affected in every aspect of their life. They have severe behavioral problems. They cannot sleep. They have gait and motor problems. They have limited communication. And unfortunately, there's just nothing available for these patients.
Everything I'm showing on the right, these are really treatments that are trying to address some of the symptoms. They're not disease-modifying. Many times these patients are on polypharmacy, rotating through, and even more so, a lot of these treatments over time lead to other types of side effects. You get GI problems. You get brittle bones. So we'd really prefer to give the patient something that's disease-modifying. We are very fortunate, and I think this is true of rare diseases that have gone on to be very successful in driving treatments, that they have a very strong, motivated, and engaged patient organization. So it used to be that the diagnostic journey was a real oddity. I'm showing it at the top of the screen.
In fact, we have talked to many families about this, and it's a nightmare scenario trying to get your child diagnosed with this disease and just not know what's going wrong. That's changed, which is a great thing, and there's still more work to do there, and why that's changed is the patient organization and creating awareness. There's also been a rapid expansion of ways to test this disease, in particular whole genome screening or exome screening, which is becoming more common. The costs are lower. This is a very definitive way to diagnose the disease, so now it's not so uncommon to see that when these children miss their milestones or have epilepsy, that they immediately get referred to getting one of these panels, and that's pretty good at diagnosing the disease, so that works in our favor, and that's good for everybody.
But how many patients are out there? And this is always a question with rare diseases, and what I can tell you is there's a lot of patients out there with SYNGAP, and they are showing up all over the place. For those of you, for example, who watch Squawk Box in the morning, you may have seen last Thursday that Becky Quick came out and talked about CNBC Cures and having a daughter with SYNGAP. So this is more common than people think. We believe in the U.S. and the EU5, there's at least 20,000 patients. We feel very strongly and good about that number. There may be a lot more than that. And on the right, what I'm showing is even though there are that many patients out there, we still have work to do in the diagnostic journey.
Probably around 25% of those patients are identified, diagnosed today. Many more are out there, and it's very common in these types of diseases when you start getting attention of pharma companies and biotechs, when we start developing drugs, that diagnostic rate tends to go up, right, because there's more for physicians to talk to those patients about as well. Again, a lot of credit to Cure SYNGAP for all the work they've done, but also a lot of the leading KOLs and physicians and families have spent a lot of time working on this. So I want to shift to talking a little bit about the biology and what's happening in this disease. What I'm showing on the left, excuse me, are synapses, and essentially SYNGAP is a negative regulator of the AMPA receptor.
So when you don't make enough SYNGAP, you essentially have too many receptors in your post-synapse. That then leads to way too much post-synaptic firing, leads to tight junctions, and as a consequence of that, you get all this sequelae that I referred to on the previous slides. Our approach is simple but quite elegant. We take CMP-002. We direct it towards the regulatory RNA that is controlling SYNGAP, and we use that to teach SYNGAP to make even more protein, thus restoring its activity, driving down the amount of receptors in the post-synaptic surface, and restoring neuronal excitability, which we believe will have transformative effects for the patients, and I'll show you that data momentarily.
So there are, I'd say, three bodies of evidence that got people really excited: the patient groups, ourselves, investors, shareholders, and that we're really riding high on and that we are driving towards the clinic based on this data set. The first is that actually the great majority of these patients are homogeneous. They're very severe, but there is a small percentage of the population, maybe around 2%, that has a slightly milder mutation on exons 1 through 4. These patients are about 75% SynGAP levels instead of 50%, which would equate to about a 1.5-fold increase in the SYNGAP protein level. They have more communication. They can go to special schools. They can respond to some other therapies.
So we know that this isn't a zero or one in terms of how sick these patients are and that it's probably based on the genetics, something where it's a spectrum, and as we can increase protein, we can start to shift them from being very severe to milder. I will say that our own data tells us we can get the gene back to normal healthy levels. So that is our intention. The second piece of data which I'll go through is that there is a mouse model that replicates the disease, many features of it, and we were able to show using our drug at doses that we'll be bringing into the clinic that we could actually reverse the phenotype across many different domains of the disease.
And then lastly, and very importantly, we were able to then go into primates, which is using the route of clinical administration, that being intrathecal administration, lumbar puncture, and we showed a statistically significant dose response in these primates in regions of the brain that we believe are strongly correlated with the disease, so at Camp4, we really try to do the most robust science and leave no stone unturned as we advance things towards the clinic, so in this case, we started with patient-derived iPSC cells that I'm showing on the left. In purple is a familial control, so these have normal levels, if you will, of SYNGAP1. In gray, 50% of what you'd expect to have is a heterozygous iPSC-derived patient cell, and then green is that same cell that is then delivered using our CMP-001 where we're able to restore function back to the normal level.
On the right, this is a humanized mouse, meaning we actually took the human SYNGAP1 gene, put it in a mouse, and then we were able to use our human drug. And what you can see there is that in purple is a homozygous control. In gray, we're showing the SYNGAP1 heterozygous without any drug, and that's actually a little lower than 50%. These things are always a little imperfect. And what you can see in a dose-responsive way is that we can increase the SYNGAP protein, essentially doubling it from what the ACSF heterozygous cell line showed in this mouse. I should say we're showing protein here, but we've also always checked the mRNA. So the mRNA and the protein are always at the same levels when we increase. This same mouse that's a heterozygous humanized also has a phenotype. It shows learning and memory. It shows motor function.
It shows hyperactivity. If you recall what I told you about the SYNGAP patients, these are definitely defining features of the disease, so what I'm showing from left to right in gray, this is a homozygous control. In purple, this is a heterozygous without any treatment, and in turquoise, this is the same mouse with our drug, and what you can see is across all three of these domains, we were able to reverse and restore function back to the normal levels as compared to the homozygous control. This was incredibly exciting data. I think this is the first time anybody's really shown this before with something that actually is moving forward and has disease-modifying capability to it, and then, as I mentioned earlier, primates are really important.
One of the reasons primates are important is when you're dealing with oligonucleotide technology, whether it be siRNAs or antisense oligonucleotides, when these technologies are used in primates against monogenic single-gene targets, and they tend to show this type of data, that has high translatability to the clinic. So this was a really important study that we did. On the left, what I'm showing is three different doses of our drug, and we're showing the PK distribution in different regions of the brain. These are all regions that are very important for SYNGAP, and as you can see, we get a nice dose response, and these are all doses in which can be therapeutically relevant. On the right, just as important, we actually looked at the gene expression. Now, I want to point out these are healthy monkeys, so they're not sick. They have two functional alleles of SYNGAP.
So what was pleasantly surprising for us is that we could actually upregulate the healthy allele 1.5-1.7-fold in a statistically significant way, and that it was shown to be very safe. I'll point out every time we do this in a heterozygous setting, like I just showed on the previous slide, we get about a twofold increase. So we believe in the disease setting, we will be able to restore normal function back to the healthy levels. So now I'm going to shift a little bit towards the forward-looking clinical plans, and as the year goes on, we'll give more details here as we're interacting with regulatory agencies. As a reminder, there's no disease-modifying therapies available for these patients, and there's actually nothing in the clinic along the lines of what I showed you.
There is a very active natural history study, a few of them ongoing, which is terrific. We're getting a lot of data from that. It's helping to inform our studies. We're working closely with all of those KOLs that are doing those studies. That's in the U.S. and Europe as well. That also helps us identify patients that we can quickly enroll into the study. There is a global center of excellence here. There are thought leaders all over the globe. For example, one of the thought leaders that we intend to work with is in Australia. There's some in Europe. There's some in Latin America. So this is all very important because we want to run a global phase one-two study directly in patients.
Why that's important is that we know that many of these jurisdictions, in terms of being able to move forward, seek approvals, they want patients from those jurisdictions. So we're being very thoughtful about that. It's not just about enrollment. And there are, we think, multiple paths to approval here, which creates an opportunity for us and the patients as well. Some things are already defined, and some things we think just create those opportunities. So what I'm showing here are different endpoint categories that we are going to measure in our phase one-two, first-in-patient study. These range from seizures, of course, which is an already approvable endpoint, to sleep, to motor and gait, communication, behavior, development, cognition, etc. Many of these already have ways of being measured.
For example, we have a seizure plus category where we will follow both in terms of visualizing seizures as well as EEGs, but we're also going to look at neurodevelopmental endpoints because we believe our drug should work across all of these different domains and not just seizures. This isn't just a seizure disease, as the patients remind us, and we appreciate that. There's also other ways to approach this. If you look at, for example, Rett syndrome and Angelman, where there's some parallels here. For example, the FDA has been very forward-looking and actually called out different ways that the drug can get approved, quite frankly, by just showing one or two domains of the disease being addressed, using things like the Bayley for other cognitive communication endpoints, which we will deploy in this study as well. Our phase I-II. We have a few different goals.
One is rapid enrollment. Two is efficacy. Three is, well, in no particular way, safety is obviously first and foremost. We want to identify the optimal biological dose. But really importantly, we want to design this study in such a way that when it's done, it opens up possibilities to have different discussions with different regulatory jurisdictions about how quickly we can move forward. These patients badly need therapy, and so we take it very seriously in thinking about not just the first study, but what's the path to approval and commercialization. So we're taking that all into consideration as we think about this first study. As such, some of the things that we intend to do are go straight to a multiple ascending dose study instead of starting with the SAD. We think that that saves time, and we think that's also a lot more reasonable for the patients.
We are going to start with an enriched genotype-phenotype population. Although all of these patients are severe, 80% of them, for example, are missense mutations, and what that means is they're very homogeneous. Most of them have seizures. Most of them have other types of cognitive behaviors that we want to measure, so we're going to try to enroll many of them. Our aim is to go as young as possible. This is a neuro-arrest disease. It's not neurodegenerative, but we'd like to get there as young as possible before they start missing neurodevelopmental milestones, and we're going to do a whole bunch of comprehensive assessments that are going to match with the natural history, so we're taking full advantage of that, and lastly, it should go without saying, we will have an open label. We think every patient deserves to get a shot at getting on treatment.
And also, some of these domains of disease probably will take longer to measure, so we want to make sure that we give an opportunity to really follow those over time, and we've learned that by looking at other similar approaches in similar diseases as well. So this is a team we have at Camp4 to do this. They're going to run through walls to make this happen. Many of us have worked together for five-plus years. We've lived through hard times, and we're really committed to building this company together. And we're really fortunate. We have a great board that has a lot of experience in building companies, raising capital, designing clinical studies, all the types of tools that we need to augment our team and help us make great decisions. So in conclusion, 2025 was a banner year for Camp4.
We really transitioned the company to be a SYNGAP-first company, and we're driving forward as rapidly as possible to bring our program into the clinic. SYNGAP represents a major market opportunity on its own. We think it's a massive value-creating opportunity for our shareholders, starting with doing the right thing by the patients, but it also has major read-through to the other DEE programs that are haploinsufficiencies that we're working on. So we really think about it as a cornerstone program for Camp4. As such, you will see us building additional programs. We'll talk about that later this year, and you should expect to see us to start to do other partnerships like GSK. Ultimately, our belief is we can be the next Alnylam, and that's how we're thinking about building this company, and that's a goal we've set for ourselves. Thanks a lot. Look forward to taking questions.
Thank you, man. I'll ask the first couple of questions, and then there'll be an opportunity in the audience. If you want to ask a question, just raise your hand when prompted. Josh, we were just talking right before the presentation over here. You said one year ago you gave this presentation, and it felt like no one knew about SYNGAP, and now you're talking in this that the awareness has significantly increased in just 12 months. Walk us through what's happened here in the last 12 months to significantly increase SYNGAP awareness.
Yeah. I think there's a few different things, and I will, for a moment, just say, having been in rare diseases for as long as I have, this is very familiar to me that every few years, a rare disease comes along, and the first reaction is, "I've never heard of that." And then it becomes a disease that everybody's heard of. SMA was like that when I was at Biogen, and there's others like that. SYNGAP strikes me like that as well. I think there's a few reasons why things have shifted. I'd like to say Camp4 was part of that. When we came out and did our financing and told the world that we're prioritizing SYNGAP and whatnot, other companies also spoke out that they were going to start developing programs in SYNGAP. So that's good for everybody. That's good for the patients.
The Patient Foundation themselves has done an incredible job of getting out there and raising awareness. I think the field writ large of these genetic epilepsies, other companies working on other programs, Dravet syndrome, SCN2A, and whatnot, has also driven a lot of attention to these diseases. And so the KOL and physician community also has a tremendous amount of awareness. And then lastly, I think investors are extremely excited about what we're doing for the reasons that I said, and that builds awareness amongst the investor community, which is also very helpful.
And then you initiated the GLP-tox work here in October of last year. Where are you in this process, and would we be getting any updates outside of just that you've completed?
Yeah. Good question. So what we've guided people to say is it's going as planned. It's going well. Just to remind people the way these work is it's not just the in-life portion of the GLP- tox, but obviously we have takedown periods to follow up on the drug over time. So that takes a little bit more time than I think people might have expected for if you say you're doing a three-month GLP- tox, it takes longer than that. So we should be ready to go by the middle of the summer with all the takedowns and everything done. That said, we're having regulatory discussions in parallel. And to answer your question, ideally, I think the way we would talk about success there would be more towards thinking about the regulatory milestones.
Yep. Okay. Questions from the audience? Yeah, go ahead, man.
I have two questions.
Sure.
The first thing is your target... The first one is about your target discovery platform. I believe you have a unique platform versus the industry ABC model. And could you just show the differentiation? And the second one is, as all of your programs are CNS programs, are you considering collaboration or in-house development of blood-brain barrier penetration? Thank you.
Yeah, so I think your first question that you're asking me, how are we differentiated? Is that the effective?
Differentiation of the platform, the RAP platform.
Yeah, yeah.
Your platform of target discovery.
Yeah. So a couple of things. I mean, one, we are, I believe, the only ones who are currently working on regulatory RNAs. I think that'll probably change in the future, but part of the reason for that is we are a little stealthy about it. It helps that one of the key scientists working on it is also on our board, so we got a little bit of a head start there, and we built a moat. And these reg RNAs, they're not in the public domain yet. You can't just do RNA-seq. You actually need our platform to identify and characterize them. It's not as straightforward as one would believe. And then figuring out how to drug them, actually understanding what's real and what's not in the upregulation world takes a certain amount of time, sophistication. So we built models around that as well.
So I think there's a lot of know-how that underpins some intellectual property there. In terms of the chemistry, our view is we want to buy down as much risk as possible. Said differently, we'd like to not take risk where we don't need to. So what does that mean? We choose diseases where we think they're best suited for our platform, things where we can get twofold increase, things where there's a high unmet need, things where we can deliver the drug safely and effectively, for example, using intrathecal delivery. But we also use chemistry that's already in approved products like Spinraza. We're not reinventing chemistry right now. We think that chemistry works really well. We're just opening the aperture for it. To your point of the question about how we think about the, I think, doing other things like blood-brain barrier, that's a really good question.
I think what I can offer up, though, is that's one avenue we think about. Another is if you look at some of the chemistry advancements, if you can go from once every three months or six months to once a year dosing intrathecally, these intrathecal deliveries have gotten quite common for a lot of these physicians. Mainly due to the Spinraza, actually. We have to think that. So blood-brain barrier is one thing we're interested in, but there's other technologies on the chemistry side that allow us to make a more durable or potent next-gen that we're thinking about.
And yeah, go ahead.
Thanks very much. A great presentation, and best of luck to you and the team.
Thanks.
I guess I'm curious about dose schedule. That would be really helpful for me to understand.
Yeah.
And I think you made mention of the fact that you wanted to go straight to a MAD.
Yeah.
Have you had any regulatory interactions to date, and what's the feasibility of that?
Yeah, good questions. So what we can say about the dosing is we expect it to be in line with what we've seen from some of the other oligos that are delivered in the CNS, so Spinraza, Stoke's program, a couple others where they do a monthly dose loading, and then you spread that out over to 3 months-6 months. Our own PK data in monkeys and mice tells us that we have a similar half-life, so we expect to be in line with other comparable products that have been delivered that way. You asked about the, oh, the...
Regulatory?
Yes. We are in the midst of getting advice, having different conversations. What I can tell you is there is definitely precedent for going directly into MAD, so that's, and actually a lot more precedent recently. Every jurisdiction is a bit different in how they approach these things, and so we have a plan for that as well. But what I can say is it's certainly not a novel idea to them that we're talking about this, and I think that's one important piece. The other is being able to start at a therapeutically relevant dose. We're thinking about the patients in this and making sure we're doing the right thing here to give them a shot at getting a benefit from it.
Additional questions from the audience? Then one from me in terms of the push-pull levers to kind of getting a phase I-II study started here, because the language is interesting. It says, "As early as." So there's some flexibility in that.
My analyst is teaching me. Yeah. We want to underpromise and overdeliver, so sometimes maybe we're too cute for our own good. We have every intention to initiate this study by the end of the year, and I say by the end of the year, not because it's December 31st, but when we put this out, we haven't had the formal regulatory interactions yet, and that will inform start time. That will inform if we can go directly to MAD. We feel more confident in a lot of the things we're talking about here based on precedent, based on some conversations we've had, but we will tighten that guidance up, but we said that earlier because we were a little bit far away, and we didn't want to overpromise and underdeliver in terms of being too specific.
Yeah. You talked about in your presentation the natural history work that's being done. You talked about how that could be a feeder for the clinical trial.
Yeah.
The range of things that you're trying to learn there, but also, could that be potentially, in a regulatory scenario, used as an external control?
Right. Really interesting question. So the data to date is mostly in children. It's really good data. My colleague Yuri Maricich, who's in the audience, our Chief Medical Officer, has been spending a lot of time going through that with the PIs. Some of the data, for example, that's really helpful for us is these seizures are not all drop seizures, meaning they can be an eyelid, it can be finger twitching. EEG is a very powerful tool we plan to use, and they have a lot of EEG data. So that's incredibly helpful for us, and will come in very handy. You asked about the ability to use that as a control. That's interesting because actually some are more progressive than others in terms of jurisdictions.
The U.K. has come out and said, "We are supportive of what's called twinning," which is exactly what you're talking about, which is matching a control. I don't think the U.S. is there yet, for example, but things change quickly here. So in terms of what I said before, we are very much being thoughtful about this study, anticipating that regulatory opportunities may open up for these types of things. So we're trying to design it with that in mind, and that may very well come in handy.
Questions from the audience? So you had a partnership, a collaboration announcement with GSK also focused on neurodegeneration, right?
Yep.
It's safe to assume there's not going to be a lot of target overlap here, right? The profile, you're talking about being a CNS company.
Completely.
Yeah.
There are over 30 haploinsufficiencies in the central nervous system. Most of them, if not all, have no approved treatments. We can subdivide that into DEE, neurodevelopmental, and then neurodegeneration. And by the way, some of those haploinsufficiencies are larger diseases, like in Parkinson's. We are holding on to the DEEs for ourselves. Things that we are partnering on, for example, are much larger diseases of neurodegeneration, if you will, where our platform could come in handy, but we would not prioritize those.
Got it. Questions from the audience? I have one more, and then maybe final one from me. You've had some recent financing activity. What's your cash runway sufficient to, and does that, you have a tranche available of $50 million as well, so is that included?
Yeah. So part of our banner year in 2025 was not just shifting to SYNGAP, but actually putting the company on stable ground as far as the funding goes. So right now we are funded to the end of 2027, and that does not include the second tranche of $50 million, which becomes unlocked after we get our first regulatory approval to initiate our first-in-human study later this year.
Okay. Thank you, Josh.
Thanks a lot, Anupam. Appreciate it.