CAMP4 Therapeutics Investor Day 2026
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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 would 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 SYNGAP1. 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 am going to take some time to make some briefly prepared remarks, but then you will have the opportunity to hear from Beata, a parent and caregiver to their daughter, Kasia, about their family's experience living with SYNGAP1. This will be followed by our CSO, Dan Tardiff, and our CMO, Yuri Maricich. We are going to review our preclinical data and provide an overview of our ASCEND phase I/II clinical study.
Next, you are going to hear from the CEO of CURE SYNGAP1, Mike Graglia, who will share his thoughts on the importance of our phase I/II study and how we collaborated with Mike and the SYNGAP1 community. Finally, we are quite pleased to welcome Dr. Stéphane Auvin, a leading child neurologist and epileptologist, who will provide a clinical perspective on SYNGAP1 and his views on the potential for a disease-modifying therapy. I am 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 ASCEND 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 five years is that we believe we can continuously design ASO therapeutics that upregulate genes underlying 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. I am quite excited to have the opportunity to speak later about our newest pipeline programs after the main focus of today, that being SYNGAP1. As many of you know, we now have the approval to begin our clinical study in Australia, Argentina, as well as the U.K. We have an additional regulatory filing underway in review in the E.U. Site activities are ongoing, and we have begun pre-screening patients in a few regions with the expected goal of dosing the first SYNGAP1 patient during the fourth quarter of this year.
Yuri's 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 a 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 SYNGAP1 protein. 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. Before I transition, I want to again thank the CURE SYNGAP1 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 SYNGAP1 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 SYNGAP patients. To kick us off today, I'm pleased to introduce Beata, a loving mother of Kasia, to talk about her family's story living with the disease.
My name is Beata Tarasiuk, and I'm most known as Kasia's mom. Kasia is a 9-year-old Syngapian, a 9-year-old little girl living with SYNGAP1-related disorders. I want everybody to understand that SYNGAP1 is a devastating disease. She's 9 years old, and compared to a typical 9-year-old, she's very far behind. Her abilities are about 12-month-old, sometimes 18-month-old child, but she's in the body of 9-year-old little girl. She is not able to do anything for herself, and she needs assistance with all daily living activities.
She is fully non-verbal. She has no words. She has a way to communicate with us, and we can understand some of her communication, but it is a very simple communication. "I want this, I want that." It feels like those kids with SYNGAP1, they do have depth to them, the depth of thought, but they cannot truly communicate. It is 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 is 150 seizures a day that you cannot really see. We couldn't see them back then when she was diagnosed at three years old. She has daily seizures. They are changing now. She is changing. She is not able to feed herself. She is not able to go to the toilet by herself. She is fully diapered. She needs help 24/7 with all her daily living activities.
She has also GI issues. She suffers from gas pain and acid reflux, because of all the medications she is on. She is suffering, and there is nothing. I feel sometimes there is just nothing else we can do. There is no way with the medicine that we have available today, there is really nothing else we can do. The biggest, we had to change 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. Now any other choice, her needs are so great, and my husband will continue his career. He is a physician. It made sense for our family that he will be the one to continue and support family financially, so we just do not collapse under the weight of this disease.
Kasia has trouble sleeping, so we do and I have to sleep. I cannot be on 24/7, so he does give me the certain time during the night that he stays up with Kasia, and he watches her, and she has trouble sleeping. He takes care of her, and I can sleep during that time. 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. We do not have a regular home. Our home is open to caregivers and therapists, and we have people in our home seven days a week. We never have any privacy in our home.
Again, it is 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 CSO, Dan Tardiff.
All right. Thank you for the introduction, Josh. I think it's pretty clear in watching this video of Kasia that we have very much focused on being able to help this patient population. Today, before we get into the ASCEND trial, and before Yuri 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 CMP-002 to the patient community. Here at Camp4, we are targeting non-coding regulatory RNAs to increase gene expression. What are these RNAs? These are non-coding RNAs that are transcribed from both promoters and enhancers of all genes.
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. 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.
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