Last year, at the start of the year, I published a blog of four research projects I was looking forward to updates in the new year. This year, I'm doing the same thing. I'm also including an update of the project from last year.
What I'm Watching In 2025
Vertex VX-264 is an encapsulated beta cell transplant that does not require immune suppression. The trial is expected to run until 2026, but the company has said that they expect to have some data to report in 2025. I hope so!
VCTX211 is another encapsulated beta cell transplant that does not require immune suppression.
Biomea BMF-219 is a drug that the company hopes is "a brake on beta-cell turnover and growth, supporting the notion that [it] could lead to the regeneration of normal, healthy beta cells". It is in the middle of a clinical trial which started in 2023 and is expected to finish in Sept-2025. I'm looking forward to data in 2025. I previously blogged on it here:
Ladarixin is a drug being developed by Dompé. It inhibits activity on parts of the immune system called the IL-8 receptor (which has two subtypes: IL-8a and IL-8b). Dompé hopes that this will stop the progression of type-1 diabetes. Ladarixin is in the middle of a phase-III trial, and they expect results in 2025. This is a big study. They have enrolled 327 people and 2/3s of them will get the treatment; with only 1/3 controls who get the placebo.
Siplizumab is a monoclonal antibody targeting CD2, which is part of the immune system. This study finished recruiting in Oct-2024, so I would expect results in late 2025. I blogged on this here:
https://www.clinicaltrials.gov/study/NCT06239636
What I Watched In 2024
The quick summary of 2024, is that, of the four research projects I focused on:
1. Semaglutide is big in T2D/Obesity, but did not make progress as a T1D cure.
2. Ladarixin progressed as expected, but the real news is expected in 2025.
3. Verapamil completed enrollment in 2024, so should have results by 2025, but has already led
to important follow-on work.
4. Diamyd was hoping for full enrollment, but did not make that milestone.
So overall, I would say 1 project did better than expected, 1 project did as expected, and 2 projects did worse.
Semaglutide (sold as: Ozempic, Wegovy and Rybelsus) had very strong results in a very small study of honeymoon T1Ds:
I was hoping to read about more research starting up in 2024 to confirm these results. Because Semaglutide is widely used to treat T2D and for weight loss, I think just one or two confirmation studies will be required to enable widespread use. If that gets us a year or two delay starting injections for newly onset T1D, that would be a huge win (and even more so if it could be combined with Teplizumab or other treatments).
Unfortunately, none of that happened. Semaglutide continues to blow up (in a good way) for T2D/Obesity, but does not move forward for T1D.
Ladarixin is a drug being developed by Dompé. I'm not sure why I put it in the 2024 list, because there was no expectation of results until the end of 2025. Nothing happened, but it is on my 2025 list.
Verapamil is another interesting "off label" drug. This is a drug which is already approved (since 1981) and widely used (3 million prescriptions a year) for high blood pressure. It is being tested on honeymooners. Therefore, if clinical trials show that it helps, it should be available quickly, especially for aggressive doctors who are willing to prescribe it off label.
Unfortunately, the ongoing study which was going to finish in 2024 is now going to finish in either 2025 or 2026, so we'll be waiting longer for results. I blogged about the start of this study here:
However this line of research has already led to the creation of the company TIXIMED working to start clinical trials on their drug TIX100, which is sort of a follow on to Verapamil. They have gotten FDA approval for the clinical trial, but have not yet started it.
Diamyd is another drug in phase-III trials. They hope to finish in 2025, but the news I was hoping for was that their clinical trial was fully enrolled in 2024. Unfortunately, it looks like that did not happen. The trial is still not fully enrolled, so I would not expect results in 2025.
Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My kid has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.
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