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You need a #CRID. SRF $1M to $CAMP noticed in Equity Research! #BeaconOfHope was great. #S10e183

Friday, September 26, 2025. Week 39.

In #S10e182 I told you about CAMP4, don’t miss that, watch here: https://www.youtube.com/watch?v=PZ0Oj-Zz-B0 Sharing research comments from William Blair & Wedbush

William Blair Initiation of Coverage: “Among several quality investors, the private placement included the SynGAP Research Fund, which is active in the SYNGAP1 patient community and will be an important resource in aiding patient identification and enrollment in the Phase 1/2 trial in our view.”

Wedbush
Ph1/2 Will Likely Begin From Ex-U.S., Aiming for Early Intervention. Citing precedents of other intrathecally delivered antisense oligonucleotide programs for CNS indications, where the FDA oftentimes required sponsors to begin at a dose level well below the efficacious dose, CAMP4 plans to begin patient dosing outside the U.S. for the potential to go directly to doses that are expected to show efficacy. The selection of patient age range will depend on regulatory discussions, and management highlighted the impact of the disease on neurodevelopment, so early intervention could allow patients to have a better opportunity to achieve as normal as possible development.
CAMP4 Will Have Access to Natural History Data being Collected by SRF and CHOP. According to management, a natural history study is being conducted by SynGAP Research Fund (SRF), which also participated in the private placement, in collaboration with Children’s Hospital of Philadelphia (CHOP), and CAMP4 will have access to data as well as patients for future clinical study enrollment.

Donate now: https://curesyngap1.org/donate/
Beacon of Hope was a great success, raised over $100k. We need to do this every year. Thanks to Navarros for getting this launched, also to Suzanne Jones, Emily Barnes, Peter Halliburton, & Kathryn Helde who helped make this event incredible.

Emmy’s video (top of) https://curesyngap1.org/resources/movies/
Blog: https://cureSYNGAP1.org/Beacon25
Pairs well with Gala Blog: https://curesyngap1.org/Gala25

Research is non-stop:

Episode 183 of #Syngap10 #CureSYNGAP1
#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1 #SynGAP #SynGAProMMiS

Below is a transcript from the video:

Mike Graglia

[00:00:00] Hello, Syngap land, my name is Mike Graglia, and this is episode 183 of Syngap 10, aka the CURE Syngap1 podcast. Today’s Friday, September 26th, year 2025. This is week 39. In the last episode, episode 182, I told you about this incredibly exciting news about CAMP4 raising tons of money and really focusing on SYNGAP1. If, for whatever reason, you have not yet watched or listened to the previous episode to this one, go back and listen to that. Link is in the show notes. Since that announcement, I’ve seen a lot of investor, a lot of investment research that’s been published about this, and SRF has been named. I want to just read a couple quotes to you. William Blair initiated coverage, so when Initiation of coverage is like these investment banks will publish articles or reports rather about listed public equities, right? And so when they start coverage that’s a big deal because it’s someone saying, ‘Hey, this is a stock to watch and then you know, investors read those to learn what’s going on. So William Blair initiated coverage, and one of the things they said, quote, among several quality investors, the private placement included the Syngap Research Fund, which is active in the Syngap1 patient community, that would be the understatement of the decade, and will be an important resource in aiding patient identification and enrollment in the Phase 1-2 trial in our view, end quote. Yes, that is exactly what I said in the last podcast. This is exactly one of the big reasons why our investment with so much more than a million dollars is because it’s a signal that we are working closely with CAMP4 and we really want to ensure that this trial finds the patients it needs to find around the world and and is able to successfully demonstrate the success or failure of this drug, and based on all the pre-clinical data we’ve seen, we expect it to be successful, very exciting. Wedbush said the following this is a slightly longer quote, but it’s worth listening to. Because the details, the details matter. Phase 1-2 will likely begin from ex-US aiming for early intervention. What does that mean? It means ex-US means not in the States, aiming for early intervention, meaning they’re going to look at you. Younger kids, citing precedents of other intrathecally delivered antisense oligonucleotide programs for CNS indications, where the FDA oftentimes required sponsors to begin at a dose level well below the efficacious dose. This is one hell of a run-on sentence, Wedbush. CAMP4 plans to begin patient dosing outside the U.S. for the potential to go directly to doses that are expected to show efficacy. What did they just say? what they just said is, The FDA is really conservative, and they will make people start at a dose that is crazy low. This is exactly what happened with Stoke and their ASO for the Dravet syndrome, a. k. a. SCN1A. So because the FDA is going to be very conservative, insisting on starting with doses that really don’t make sense, and because the FDA is very slow, it is reasonable to assume that once the company is ready to launch this drug, once any company is ready to launch a drug, frankly, they’re going to go outside the U.S. if they think there’s a country or a geography where they can get into humans faster at a dose that makes more sense. I’m not being political. I’m not throwing the FDA under the bus. I’m citing well-established precedent that this very respected investment bank has also just talked about. To begin at a dose level well below the efficacious dose, right? That’s what they said. Expects trials to start outside the U. S., but expect them to get here soon. And hopefully, because of the work ex-US showing safety and efficacy at certain doses. Expect the doses here to work. And then, the other important word in that very long sentence was intrathecally. Remember, this will be an intrathecal dosing. There’s different ways you can dose. You can dose IV into the blood. You can dose ICM into the skull, or you can dose IT, which is a spinal tap. This is a good thing. This is a good thing, right? Because we only want this medicine right now to get to the brain. The easiest way to get it to the brain is to stick it in the spinal column. You don’t want them drilling a hole in the kid’s head. Much easier to get a needle in the back. Right? Well established. Happens all the time. It’s great. The selection of patient age range will depend on regulatory discussions, and management highlighted the impact of the disease on neurodevelopment, so early intervention could allow patients to have a better opportunity to achieve as normal as possible development. This is a great sentence. In other words, they’re not going to tell you what age they’re going after because they’re not going to know that until they talk to the regulators and see how young the regulators will let them go. That’s what it means. And they’re going to argue to go as young as possible. And the more conservative regulators are going to be like, start with adults. And they’ll be like, well, we might not see any changes in adults because that could be too late, right? We don’t know if it’s too late, but there’s a tension here between regulators saying start in adults and companies saying this is a neurodevelopmental disease. We want to start as young as possible. But the question I’ve gotten the most from families is, what’s the age range going to be? And the answer is we don’t know yet. Next paragraph, super exciting. CAMP4 will have access to natural history data being collected by SRF and CHOP. According to management and natural history studies being conducted by the SYNGAP Research Fund, which also participated in the private placement. Cha-ching! In collaboration with Children’s Hospital of Philadelphia. Go CHOP! And camp. Will have access to this. To the data as well as patients for future clinical study enrollment. Pretty exciting, guys. Pretty exciting. What this tells you is that what I’ve been telling you for years now. Is that by having a natural history study, by engaging closely with industry, we will be able to mobilize. Impact and get things done. And that is exactly what is happening here. That is exactly what leading investment banks are talking about. So good job, team. All of this takes money, guys. I’m not talking about the money we invested in CAMP4. We expect that to come back. But all these meetings, all this work. All the research that’s got us this far requires money. So please, it’s never too early to donate, curesyngap1.org/donate. And it’s never too early to think about Christmas season, Christmas cards. End of the year’s coming, guys. We got to raise more money. We have so much work to do. On that count, last week after I talked about CAMP4, I got on a Maybe I was already on a plane. Who knows? I went to Boston for the Beacon of Hope event, the inaugural Beacon of Hope event, and it was a great success. We raised over $100,000. We need to do this every year. I want to thank the Navarros for forcing us into that event and kicking it off and saying, ‘We’ve got to do something in Boston.’ And I also want to thank Suzanne Jones, our fearless board chair, Emily Barnes, Peter Halliburton, and Kathryn Halde, who all came and helped work hard to make sure it was successful. It was a great night. The Navarros, Anthony and Chelsea, we shared a video about their child at the event. That video is now on our website. Curesyngap1.org/resources/movies. All the family movies are on that page. Go to the top, read about Emmy or watch the Emmy video. It’s great. There’s also a blog about the event, curesyngap1.org/beacon25. Links in the show notes as per usual. Check it out. Check it out. It’s going to be great. The other link that’s in the show notes is curesyngap1.org/gala25. That’s going to be a great read and you can hear all about what happened in Jersey. So we had an event in Boston a few weeks earlier. We had an event in Jersey. Between the two of them, we brought in almost $200,000. We need that money, guys. We need that money to keep the research going. Speaking of research, speaking of research. It’s nonstop. It’s nonstop. I got to tell you this. And the reason it’s nonstop is because of you, because the patients show up and the patients share their data. So we just put out a blog. Is why every Syngap1 patient needs a CRID. CRID is C-R-I-D, Clinical Research Identifier, I guess it means. And what a CRID is and why you should, and you just go to the website. You type, type, type a few things and you get a CRID. And then what you do is you get that CRID. It’s like a secret number that ties to you. And only you know that. And I guess the CRID people, it doesn’t matter. So you take the CRID and then you call the Simon Searchlight people, the Citizen people, the RareX people, whatever other studies you’re in. You say, ‘Put my CRID in there.’ And what that allows us to do is in a safe, privacy-protected way, match up data between patients so that when we put data sets together, we don’t miss things. So read the blog. Every Syngap patient needs a CRID. If you can’t find the blog, go to the website. Our website has an amazing search function. Use it. CRID. Read the blog. The other thing that’s going on is ProMMis. As you know, this natural history study that these investment banks just talked about is a critical reason why people are investing in Syngap1. So, if you haven’t given yourself the gift of going and participating in the natural history study, and I’m not being glib there, guys. You want that data. You want to see those doctors. You want to get that information about your kids. And you want to share that data with the world. Sign up for ProMMis. We are seeing patients at Stanford, at Colorado, and at Children’s Hospital of Philadelphia. Get yourself into one of those sites. Also, and that will start measuring your child going forward. Going backward, looking at all their health records, that would be Citizen. Sign up for Citizen. It’s a huge data set. It’s incredibly valuable. And they’ve got a great AI advocates. You can now go in and like talk to the AI about your kid. The AI is reading your medical records and talking to you about your kid. It’s amazing. Citizen.health/partners/SRF. That’s where you sign up. And then the last thing on research I’m going to talk about is the DSC has been announced. Now, I got to be really careful about my 10-minute thing. Actually, I’m going to talk about the DSC in the next pod. I’m going to do a part two for this. So thanks for listening. This has been episode 183, and I’m just going to go right into episode 184 because I’m at 10 minutes.