Friday, May 8, 2026 – Week 19
CURE SYNGAP1 STRATEGY WORKING
Our strategy has always been to derisk SYNGAP1 so that industry will invest and bring their skill and capital to bear. We leverage donor dollars to maximize impact. Yesterday was a great example:
EARNINGS REPORTS
CAMP4 1Q26 Results: $99M https://investors.camp4tx.com/news-releases/news-release-details/camp4-reports-first-quarter-2026-financial-results-and-corporate
We submitted our first regulatory filing for CMP-002 in Australia which positions us to initiate a global first-in-human Phase 1/2 clinical trial in the second half of 2026.
Stoke 1Q26 Results: $411M https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-first-quarter-2026-financial
Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic neurodevelopmental disease.
Praxis 1Q26 Results: $1.4B https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-19
Praxis remains on track to nominate a development candidate for each of its three early stage ASO therapeutic initiatives in the first half of 2026: PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs.
#CompetitionIsGoodForThePatient
KCNT1 Big Day today – Congratulations
Post: https://www.linkedin.com/posts/graglia_kcnt1-share-7458560855744618496-X10d
Press Release: https://www.linkedin.com/posts/kcnt1-kcnt1-epilepsy-ugcPost-7458487179149787136-kH7c/
WATCH & SHARE OUR VIDEO
Watch and share the new SYNGAP1 Video, it is excellent and helps explain our cause to families and friends. We need to turn them into supporters and donors. cureSYNGAP1.org/Burden
INAUGURAL SF NIGHT OF IMPACT, CA – 20 days
Join us this is our only Gala for 2026!
cureSYNGAP1.org/SF26
5TH SCRAMBLE FOR SYNGAP, SC – 148 days
Classic case of a small event becoming an institution!
cureSYNGAP1.org/Scramble26 USA:
Use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/https://onlinelibrary.wiley.com/doi/10.1002/epi.70142?__cf_chl_rt_tk=a9Yy35L40S_zHePCHihyHLVwynhiuCb5KVI_JizfWTU-1778315038-1.0.1.1-VlV_e1OFhzWgMvHWmUCVL_jsXa8dN3WqNP0rhKWfMP41…
PUBMED
Pubmed 2026 is at 28. +9 vs the week. (61 last year was +9)
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=datehttps://pubmed.ncbi.nlm.nih.gov/42093… ADHD in patients with #DLG2 #NRXN1 #SHANK3 #SYNGAP1
SOCIAL MATTERS
4,940 LinkedIn. https://www.linkedin.com/company/cureSYNGAP1/
1.569 YouTube. https://www.youtube.com/@cureSYNGAP1/
11.1k Twitter https://twitter.com/cureSYNGAP1/
44k Insta https://www.instagram.com/cureSYNGAP1/
$CAMP closed at $4.46. https://www.google.com/finance/beta/quote/CAMP:NASDAQ
$STOK closed at $32.89. https://www.google.com/finance/beta/quote/STOK:NASDAQ
$ACAD closed at $22.40. https://www.google.com/finance/beta/quote/ACAD:NASDAQ
$PRAX closed at $330.02. https://www.google.com/finance/beta/quote/PRAX:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 207 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy
Below is a transcript from the video:
[ 00:00:01 ] Hello, hello, hello, Syngap land. My name is Michael Graglia, and this is episode 207 of the CURE SYNGAP1 podcast. Today is Friday, May 8th. We are in the 19th week of the year 2026. I know I just did a pod two days ago, so you might be surprised that I’m doing another one. But let me tell you, yesterday was a very big day for Syngap.
[ 00:00:25 ] You may or may not know why. You may think I’m talking about our video, which I will get to, but that’s not what I’m talking about. I want to remind you that the strategy of CURE SYNGAP1 has always been to de-risk Syngap to get industry to invest in Syngap, to bring their expertise, to bring their capital, to bring their networks, to bring their arms of employees to work on SYNGAP1.
And for us to do that, we have made it easier to work on SYNGAP1. We have organized the community. We have been very public. We’ve been incredible—incredibly collaborative. We call people back. When there was too many people for me to call back, we hired more people. We had Kathryn. We have Virginie now. We’ve always had Lauren, thank God.
So we are building the team. We are working on the science. We are activating the community. We are constantly asking you to take part in studies. And that is to make it easier for industry to focus on and invest in Syngap1. And that is how we take the millions of dollars we’ve raised. We’ve given about—we’ve given more than $8 million over the past year. So round numbers, a million bucks a year, right?
[ 00:01:28 ] Which we’ve asked for from you and taken from you and used to make this organization work. So by granting $8 million a year to scientists and partners
[ 00:01:37 ] and by keeping our team functioning, we have worked hard to encourage industry to work on SYNGAP1. And it would be fair question, is that working? How are we doing on that, Mike? Well, yesterday, three biotech companies posted earnings reports, three of them, and all of them talked about Syngap1, and I’m going to read those to you right now.
And I’m also going to tell you how much money these companies have in the bank. Because if you remember the early days when I was so excited about CAMP4, I was super excited. I was like, “These guys don’t have any money in the bank.” That’s not true anymore.
So the first results I want to read came from CAMP4. They have $99 million in the bank, so they got runway. The link is in the show notes to their press release. Let me read this quote. This is verbatim out of their press release. We have made, quote, “We have made significant progress year-to-date against our goal of bringing a potential first-in-class treatment for SYNGAP1-related disorder into the clinic,” said Josh Mandel-Brehm, President and CEO of CAMP4.
[ 00:02:31 ] Josh and I did a fireside chat interview at the end of the conference last year. It’s available on our YouTube channel. Quote, “We submitted our first regulatory filing for CMP-002 in Australia, which positions us to initiate a global first-in-human Phase 1-2 clinical trial in the second half of 2026. Additional filings with global regulatory agencies are planned through 2026. We are also excited to support the ProMMiS study through CURE SYNGAP1 and invest in the foundational science that will further validate our understanding of the natural history of Syngap1 and advance meaningful, potentially disease-modifying medicines for all patients affected by this disease.” End quote. Thank you, Josh.
[ 00:03:14 ] Thank you for filing in Australia and telling us you want to start Phase 1-2 clinical trials this year. Congratulations to our friends in Australia. You might remember in Episode 206, I was like, maybe they’re going to start in Australia. I actually didn’t know this press release was coming out.
[ 00:03:28 ] You guys got to listen to me. Anyway, this is amazing. A company has filed with a regulator in a country, saying they want to start disease-modifying therapies for our patients this year.
[ 00:03:42 ] Exceptionally exciting news. Phase 1-2 clinical trial. That is real patients will be dosed with real medicine. Um, additional filings with global regulatory agencies are planned throughout 2026. That means it’s not going to be just Australia. They’re going to do it in Australia and other places.
[ 00:04:00 ] We will wait and see for them to tell us where those places are. But as a Syngapian, as a parent of a Syngapian, as somebody who desperately needs better medicine for our children, this is amazing.
[ 00:04:12 ] Only way this could be better is if there were other companies doing this because competition is good for the patient. Competition drives for better products. Competition affects pricing. Competition affects everything. Guess what? Stoke Therapeutics yesterday.
[ 00:04:24 ] In their press release, they’ve got $400 million in the bank, by the way, because they’re working on Dravet. There’s a lot of money sloshing around over there. Quote: “Lead optimization is underway to identify a clinical candidate for the treatment of Syngap1 in 2026. Syngap1 is a severe and rare genetic neurodevelopmental disease.” Amen. Go Stoke. We’ve been talking about Stoke for a very long time. Stoke’s been thinking and working on Syngap1 for a very long time. Stoke is a reminder it’s 50-50 on this project with Acadia.
[ 00:04:53 ] All very exciting things.
[ 00:04:55 ] Let’s stay tuned and watch what’s going on with Stoke.
[ 00:04:59 ] Praxis Precision Medicine, who, by the way, has the Emerald Study with relutrigine, and is, um, doing a lot with a lot of diseases. They have grown tremendously lately. They have $1.4 billion with a B in the bank.
“Praxis remains on track to nominate a development candidate for each of its three early-stage ASO therapeutic initiatives in the first half of 2026.” Hey, guys, first half of 2026, you got like six, maybe seven weeks left. PRAX-080 is focused on PCDH19.
[ 00:05:32 ] PRAX-090 is designed to address SYNGAP1 loss-of-function mutations.
[ 00:05:37 ] PRAX-100 for SCN2A loss-of-function mutations. Amazing.
[ 00:05:42 ] Amazing. Does this guarantee that Praxis and Stoke will announce a candidate and prosecute it as aggressively as CAMP4 has? No. Maybe there’ll be more. Maybe they’ll be faster. Maybe not. Who knows? But the fact that three publicly traded companies put in these investor-facing reports—that they’re all working on SYNGAP1—should give a lot of hope to families.
And I want to underscore that CAMP4 mentioned us by name, right? Do not think for one nanosecond we are out of the woods. Do not think for one nanosecond we are out of the woods. The fact that these things are happening should make you want to double down and fundraise and volunteer more with CURE SYNGAP1, not less.
Because the amount of work that lies ahead to get through Phase 1-2, to get through—Phase 3, to do access, to argue about the pricing, to ensure that Medicaid covers the drugs, to unify the community, to support new patients, to understand what modified patients need, what adjunctive therapies other drugs kids who have received these ASOs need, the amount of work ahead of us is ginormous
[ 00:06:45 ] friends, and we are all in this for life together. So please
[ 00:06:52 ] do your bake sales, buy your Girl Scout cookies, send your check to the PTA, whatever. If you’ve got a Syngapian
[ 00:06:58 ] you have got to see this news and understand that CURE SYNGAP1 is effective and is working for you. And you need to work with us and help us raise more money to do more work to make all of this go faster.
[ 00:07:11 ] There is so much going on. I want to also mention KCNT1 today received an FDA approval for a drug. It used to be a terrible developmental, uh, an epileptic encephalopathy. Terrible disease. Very high mortality rate. And the leader of that is a dear friend of mine, Dr. Justin West. And it’s his story to tell, but fierce advocacy has played a huge role in that drug getting done, getting to this point, and we’ll do it and we’ll be a huge part of everything that comes next.
So just to say, it’s incredible what Praxis and Stoke and CAMP4 especially are doing right now. And it is true that the fact that they have CURE SYNGAP1 as a partner is a reason why it is happening. So if you’re a family out there, take a moment.
[ 00:08:00 ] Do some fundraising. How are you going to do fundraising? How are you going to explain to your donors, your friends, your family, how important this organization is and why they should write a check? Well, funny you should ask. The last link in my show notes before I get to the usual stuff, we recorded a video at the last conference. We gave all these families a chance to come in and record themselves.
And then we paid somebody to make a big long video and they got it kind of right. So we went through the whole thing. We went through all the raw footage. We gave them other clips. We had them do it again. Polish it a little bit. And we finally released that video
[ 00:08:30 ] just the other day. The link is dead simple: CureSyngap1.org/Syngap1.
[ 00:08:36 ] And that will point you to our YouTube channel where that is a featured movie.
[ 00:08:43 ] Here’s the deal, guys.
[ 00:08:45 ] CureSyngap1.org/Syngap1. Take that link, text it to your friends, to your family, to anyone you know, whoever has given a charitable donation in their life, and say, “Please donate to CURE SYNGAP1.” CureSyngap1.org/, you guessed it, donate.
[ 00:09:01 ] Let them watch this video. Watch this video tonight with your friends and your families and your other kids.
[ 00:09:08 ] Put it on YouTube. Check it out. It’s incredible. It’s incredible. It tells the story of our disease. It tells the story of our organization. And it tells the story of hope and why we’re all here working together.
This YouTube episode is a perfect place for me to mention that and this. Speaking of hope, in 20 days, we’re going to have a big fundraiser in San Francisco, the SF Night of Impact. If you can make it, if you’re near San Francisco, please come. In 148 days, the Scramble will be in South Carolina.
If you have a patient and you’re seeing doctors, make sure they include your ICD-10 code F78.A1. I want to mention PubMed is at 28. I have 10 seconds left for this. There’s a really cool paper in here about ADHD that just came on the wire. Check that out. Social media, make sure your friends and family are following us on LinkedIn and YouTube.
See you soon. This has been episode 207 of the CURE SYNGAP1 podcast, and it’s an exciting day.