CAMP4 Therapeutics Corporation Common Stock Investor Day 2026

Good afternoon, and welcome to the Camp4 Therapeutics Analyst event. At this time, all attendees are in a listen-only mode, and a question-and-answer session will follow the formal presentations and fireside chat. To our analysts joining us live, please use the raise-hand feature to indicate you have a question. As a reminder, this call is being recorded and a replay will be made available on the Camp4 website following the conclusion of the event. I'd now like to turn the call over to Josh Mandel-Brehm, Chief Executive Officer at Camp4 Therapeutics. Please go ahead, Josh. Thank you very much.

Good afternoon, everybody, and thank you for joining us for our first-ever Analyst event to discuss CMP-002 for the treatment of syngab1. We are going to be making some forward-looking statements today as part of the discussion, and I encourage everybody to visit our public filings for more information. We have a wonderful agenda for you all today. I'm going to take some time to make some briefly prepared remarks, but then you'll have the opportunity to hear from Beata, a parent and caregiver, to their daughter, Kasha, about their family's experience living with syngab1. This will be followed by our CSO, Dan Tardiff, and our CMO, Yuri Marasich, who are going to review our preclinical data, and provide an overview of our SEND phase 1, 2 clinical study. Next, you're going to hear from the CEO of Cure syngab1, Mike Rallia, who will share his thoughts on the importance of our phase 1, 2 study, and how we collaborated with Mike and the syngab1 community. Finally, we are quite pleased to welcome Dr. Stefano Vin, a leading child neurologist and epileptologist who will provide a clinical perspective on syngab1 and his views on the potential for a disease-modifying therapy.

I'm then going to close with an update on our preclinical pipeline. Many of you on this call today are familiar with Camp4, but before I provide a brief update of where we are today with regards to our SEND study, I want to talk about the ambitions we have as a company. We built Camp4 to be a product company with a platform behind it. One that is able to continuously generate life-altering drugs for patients in need. What we have learned over the past 5 years is that we believe we can continuously design ASO therapeutics that upregulate genes, undermine diseases, to promote healthy, protein expression. Our platform is broadly applicable, however, for our purposes, we are building a pipeline of upregulation therapeutics to solve genetic diseases of the brain. And I'm quite excited to have the opportunity to speak later about our newest pipeline programs after the main focus of today, that being syngab1. As many of you know, we now have the approval to begin our clinical study in Australia, Argentina, as well as the UK. And we have an additional regulatory filing underway and in review in the EU. Site activities are ongoing, and we have begun pre-screening patients in a few regions with the expected goal of dosing the first syngab1 patient during the fourth quarter of this year.

Yuri is going to spend a lot more time reviewing the clinical trial design data with you, but I did want to talk about our timeline for the data readout. As well as our overarching goal with this trial to get an answer to, does CMP-002 represent a disease-modifying therapy for this terrible disease? This is a first-of-its-kind therapy for this type of disease, as well as its first-of-its-kind clinical study. We're going to learn a lot along the way in terms of how and when these patients may respond to our therapy aimed to increase the expression of syngab1 protein. Now, we expect these top-line results to be available in the first half of 2028. The design of our clinical study may also allow us an opportunity to provide safety and pharmacodynamic updates prior to the full data readout. Where we stand today, just starting this journey, we do not know what kinds of specific, meaningful updates we might be able to provide or when, other than the top-line readout in 2028. However, we will look to provide meaningful updates on our progress when we can for the benefit of all of our stakeholders, including the investment community and, importantly, our patient, caregiver, and clinician community.

We are excited to embark on this new clinical development phase at Camp4, and we appreciate your willingness to spend time with us today to learn more. Now, before I transition, I want to again thank the Cure syngab1 community and all the entire patients for their courage, bravery, and collaboration to help get us to this point. We are inspired by you, and we hope we can make a big impact on you and your lives. As CEO, I have the privilege of being able to talk about syngab1 to investors and companies alike. However, there are, of course, times when words and pictures on a slide do not do the disease justice. Therefore, we felt it was important for everyone listening in to have the opportunity to meet one of these brave syngab patients, so to kick us off today, I'm pleased to introduce Beata, a loving mother of Kasha, to talk about her family's story living with the disease.

Hi, my name is Beata Tarasiuk, and I'm most known as Kasha's mom. So Kasha is a 9-year-old syngabian. A 9-year-old little girl living with syngab1-related disorders. I want everybody to understand that syngab1 is a devastating disease. She's 9 years old, and compared to a typical 9-year-old, she's very far behind. She, her abilities are about 12-month-old, you know, sometimes 18-month-old child, but she's in the body of 9-year-old little girl. So it's excuse me. She is not able to do anything for herself, and she needs assistance with all daily living activities. She does she's fully nonverbal. She has no words. She has a way to communicate, with us, and we can understand some of her communication, but it's a very simple communication, like I want this, I want that. It feels like those kids with syngab1, they do have depth to them the depth of thought, but they can't truly communicate and it's because she has daily seizures. Because her seizures are before she was medicated, they were every 10 minutes on the EEG. We observed, so it's 150 seizures a day that you can't really see. We couldn't see them back then when she was diagnosed at 3 years old.

So she has daily seizures. They're changing now as she's changing. She's not able to feed herself. She's not able to go to the toilet by herself. She's fully diapered. She's she needs help 24/7 with all her daily living activities. And she has also GI issues. She suffers from gas pain and acid reflux, you know, because of all the medications she's on. She's suffering and there's nothing I feel sometimes there's just nothing else we can do. And there's no way with the medicine that we have available today, there's really nothing else we can do. We well, the biggest we had to change all our whole life to be with my children and take care of my children, but I never imagined that I would have to completely give up my professional career. But now. Any other choice. Her needs are so great. And my husband will continue his career. He's a physician. It made sense for our family that he will be the one to continue and support family financially and so we just don't collapse under the weight of this disease. Kasha has trouble sleeping, so we do and I have to sleep. I can't be on 24/7, so he does give me the time the certain time during the night that he stays up with Kasha and he watches her and she has trouble sleeping.

He takes care of her. And I can sleep during that time. And so he had to adjust his schedule at work. He had to give up some of the work opportunities and some of the career opportunities that otherwise he would probably be able to take on. You know, we don't have a regular home. Our home is open to caregivers and therapists and we have people in our home 7 days a week. So we never have any privacy in our home. But again, it's a choice we made so we can have some resemblance of some normalcy in our life and our younger daughter can have some normal childhood.

All right. With that, we're going to hand it over to our CFO, Dan Tardiff.

All right. Thank you for the introduction, Josh. I think it's pretty clear in watching this video of Kasha that we have very, very much focused on being able to help this patient population so today, before we get into the essential trial and before Joey describes what we're going to be doing, I'd like to walk you through the underlying biology and the target that we're going after, as well as the preclinical data that is supporting our efforts moving forward to get CMP00 to the patient community. So here at Camp4, we are targeting non-coding regulatory RNAs to increase gene expression. So what are these RNAs? These are non-coding RNAs that are transcribed from both promoters and enhancers of all genes. And they actually help regulate transcription, and they do so by binding to both positive and negative acting transcription factors and helping maintain high local concentration of these factors so that they can interact with the DNA with the promoter to help regulate transcription. Again, all genes are generating these regulatory RNAs. And we have uncovered over the last number of years that we can target these non-coding RNAs with antisense oligonucleotides to increase gene expression. And what we have found is that by targeting these regRNAs, we influence their structure and change the proteins that they're interacting with.

Ultimately, what this means is that there is an increase in gene expression. Again, importantly, there's a couple features here that are highly relevant. One, all genes make these RNAs. They also act very specifically. So when we are targeting a regulatory RNA with an oligonucleotide, we're only having an effect on the gene of interest. And also the effect size that we get is really well suited to haploinsufficiencies or diseases where there's about a 50% reduction in expression and we want to bring that back towards wild type levels. So SYNGAP1 related disorders caused by haploinsufficiency or mutations in the SYNGAP1 gene that result in about a 50% reduction in activity. SYNGAP1 is a post-synaptic protein. It is involved in regulating the insertion of receptors in the post-synaptic membrane. So what happens when there is a reduction in SYNGAP levels, there's a stabilization of the AMPA receptor at the post-synaptic membrane. This results in increased signaling glutamate signaling and neuronal hyperexcitability. So the synapses are too strong. They've matured too quickly. And this results in hyperexcitability and leads to all the different neuronal phenotypes that you have seen exhibited in the video of Kasha. So what we are doing is we're targeting the SYNGAP1 non-coding regulatory RNA with CMP002.

The idea here is that we will restore SYNGAP1 in the post-synapse, this will normalize, receptors at the post-synaptic membrane, and restore proper neuronal activity. So the data that we have generated to date establishes that targeting the SYNGAP1 regulatory RNA is able to increase expression and that this has a physiological benefit. So the data that I'm showing on this slide on the left is looking at an in vitro system. This is patient iPS-derived neurons. So we're looking at control neurons or with a wild type SYNGAP gene in black. And then in purple, is the patient iPS-derived neuron. So these cells have a mutation in the SYNGAP1 gene that results in a 50% reduction of expression. And you can see this. When we add in our CMP002 development candidate oligonucleotide, you can see that this SYNGAP1 levels are restored towards wild type. Now we've also been able to demonstrate a similar relationship in vivo. So how are we going to look at the activity of a human-specific oligonucleotide in mice? do this, we turn to a humanized SYNGAP1 mouse. So this is a model in which the human SYNGAP gene that includes the promoter and the regulatory elements that are transcribing the non-coding regulatory RNA have fully replaced the mouse gene.

So there's no more mouse SYNGAP remaining. There's only either two copies of human SYNGAP or a single copy of human SYNGAP. And you can see that comparing the black bar to the purple, that there's a 50% or more reduction in SYNGAP1 protein when there's only a single copy of the human SYNGAP1 gene. Single dose administration of CMP002 by intracerebral ventricular injection or ICV injection results in an approximately twofold increase in protein levels, restoring it back to wild type levels. So this nicely shows that both in vitro as well as in an animal system, we can administer an oligonucleotide, engage the regulatory RNA target, and increase SYNGAP protein back towards wild type levels. So what's critical as we develop CMP002 is also to show that the increase in expression that we're achieving is having a physiological benefit. So to do that, we again use this humanized mouse model. And looked at a wide range of phenotypes that are both neurobehavioral as well as seizures. As we heard during the video, there's a wide range of phenotypes and symptoms that these patients have. And exhibit. And by restoring SYNGAP, we would hope that we can have some impact in a broad way across the different domains of disease.

So we looked at learning a memory and motor function in a hyperactivity as some neurobehavioral assessments and phenotypes. And you can see in each case, that there is a significant difference or deficit comparing the haploinsufficient SYNGAP mice compared to their wild type two copy SYNGAP controls. And in each case, when we administer CMP002, we're bringing these phenotypes back towards the wild type level. So we're able to have broad reaching improvements in these neurobehavioral phenotypes in this humanized mouse model. We also wanted to explore seizures. So seizures is a significant component of the SYNGAP1 patient experience. These mice on their own do not exhibit over countable motor seizures. So to assess. So SYNGAP1 haploinsufficiency results in neuronal hyperexcitability and by adding PTZ or by injecting these animals with PTZ, which is a GABA antagonist or an inhibitor of inhibitory neurons, this further exacerbates or makes these animals even more susceptible to seizures. And the way we run this study is we give repeated administrations of PTZ every five minutes and then count score whether those animals have a seizure or not. And you can see that the wild type animals in the black line with the repeated administration do not exhibit any seizures.