Highlighting Key Clinical and Research Trends From the 2026 AAN Annual Meeting

At the recently concluded 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18–22 in Chicago, Illinois, experts in the Clinical Trials Plenary Session highlighted late-stage and practice-informing studies evaluating novel therapeutics, disease-modifying strategies, and neuroprotective interventions across neurology. Codirected by neurointensivist H. E. Hinson, MD, MCR, FAAN, the session featured research spanning movement disorders, neuroimmunology, neurocritical care, and neurodegeneration. Overall, the studies reflected an ongoing shift toward targeted therapies, biomarker-informed endpoints, and more individualized approaches to neurologic disease management.

At the session, investigators presented a series of phase 3 and pivotal trial results with potential near-term clinical implications in neurological diseases. These included durability data for ulixacaltamide (Praxis Precision Medicine) in essential tremor from the ESSENTIAL3 study (NCT06087276); efficacy and safety findings for cemdisiran (Regeneron) in generalized myasthenia gravis from the phase 3 NIMBLE trial; and results of zilganersen (Ionis Pharmaceuticals) in Alexander disease (NCT04849741).^1,2

Additional presentations included ecopipam (Emalex Biosciences) for Tourette syndrome (NCT05615220), satralizumab (Enspryng; Genentech) in relapsing myelin oligodendrocyte glycoprotein antibody-associated disease from the METEOROID trial (NCT05271409), and neuroprotective and biomarker-based outcomes with privosegtor (Oculis) in acute optic neuritis.³ Neurocritical care and cerebrovascular presentations included the ICECAP trial (NCT04217551), examining cooling duration after cardiac arrest, and OCEANIC-STROKE (NCT05686070), evaluating factor XIa inhibition with asundexian (Bayer) in acute noncardioembolic stroke or high-risk transient ischemic attack.

Following the conclusion of the meeting, NeurologyLive^® spoke with Hinson, who serves as vice chair of the AAN Science Committee and a professor of clinical neurology at the University of California, San Francisco. In the conversation, she reflected on the overall atmosphere of the meeting, key scientific themes that emerged across sessions, and several late-breaking trials that may influence future neurologic practice. She also discussed the increasing emphasis on prevention, advances in interventional neurology, and how innovative trial designs presented at the meeting may help shape the next generation of neurologic research.

NeurologyLive: What was the vibe around this year’s AAN Annual Meeting and how did it differ from previous iterations?

H.E. Hinson, MD, MCR, FAAN: I think the vertical layout of this year's meeting, specifically that the convention center itself was quite vertical, allowed for a lot of interaction between the meeting attendees, even more so than usual. But this year it was extra vertical and extra interactive. That, plus the record-breaking attendance of this year's annual meeting, led to a buzzy, exciting atmosphere in the McCormick Convention Center. The walking distances felt a little bit shorter to me this year. It really felt almost impossible not to run into a colleague or a friend around every single corner. The buzz just felt especially palpable this year because of those 2 factors.

What were some of the main themes and focuses that emerged this year, and how do they speak to where neurology is going?

As a member of the Science Committee, I think my head is always in the science, and that's how I approach thinking about the annual meeting. When I reflect on the annual meeting this year, there are some topics that I think are shaping both medicine broadly and are also touching neurology specifically. These are topics that physicians are talking about really across fields in medicine. Some specific examples include the GLP-1s, artificial intelligence, and the N-of-1 rare genetic disease trials. We saw plenaries and scientific sessions throughout the meeting on all of these topics this year at the annual meeting.

One of the major themes is how incredibly interventional neurology has become throughout every subspecialty. We're seeing many phase 3 trials being presented at the annual meeting, particularly this year. I think that used to be somewhat less common back 10-15 years ago. We are able to offer not just drug treatments, but interventions for our patients in conditions where historically we really had very little to offer, maybe only symptom management. But now we're offering, if not outright cures, disease-modifying therapies that are changing lives. I think that's a big difference in the last decade.

The other big theme that you can see amongst the annual meeting and in neurology more broadly is the emphasis on prevention. I think that's another big difference. The concept of maintaining brain health is really permeating our specialty in neurology, and that space, specifically in brain health, has exploded in the last 5 years. One example of this is that the AAN is partnering with the American Heart Association to fund research awards called the Sacco Scholars Awards. This funds early-career researchers in brain health, and that unites a broad array of topics, everything from dementia to vascular health in the brain. We're going to start to see the fruits of this investment in the coming years, as the scholars start to report out the results of their work. I think we're going to start to see some really innovative science in the area of brain health. So those are the 2 major themes that I saw at the meeting this year.

Give a bit of an overview of the Clinical Trials Plenary Session—what trials stood out? Any therapeutics that we could see coming to patient care soon?

I love this session at the annual meeting. Several therapeutics are likely to be available soon. For example, we heard about the phase 3 NIMBLE trial in myasthenia gravis, testing a new drug, cemdisiran, which showed efficacy. We also heard about a new drug for essential tremor, ulixacaltamide, tested in the ESSENTIAL3 trial. Finally, there's also a new factor XI inhibitor that, when added to an antiplatelet agent, appears to reduce recurrent stroke or cardiovascular events over an antiplatelet alone. This was the result of the OCEANIC-STROKE trial, which was presented in the clinical trial session as well.

Two out of 3 of these trials were just recently published. The NIMBLE phase 3 trial came out in The Lancet, concurrent with the clinical trial session, and the OCEANIC-STROKE trial was published in The New England Journal of Medicine in the middle of April. With those recent publications, I think that it's very likely that some of these therapeutics will be coming to market soon. So, there is a lot to look forward to in emerging neurology therapeutics.

Did any of the trials presented stand out in terms of innovative design, end points, or use of biomarkers that may reshape how we approach future neurologic clinical trials?

Probably the best example of an innovative design was the ICECAP trial, which used an adaptive design. Here, the investigators examined the duration of cooling after cardiac arrest as a neuroprotective strategy. The adaptive design allowed the trial to start at a 12-hour duration of cooling at 33 degrees Celsius, and depending on efficacy, more arms would open at longer or shorter durations. However, this was masked to the investigators so as not to bias the results. Essentially, it operated like a dose-finding trial. Instead of dose-finding for a drug, it was dose-finding at a duration of exposure to cooling.

Ultimately, the trial found exposure to 33 degrees at various times was all similar, which was taken to mean, in the context of other hypothermia trials conducted previously, that a higher temperature goal is probably sufficient to maintain neuroprotection, though it will be helpful to read the full text of the publication when it comes out, which is not yet available. This is very interesting to us in the neurocritical care because it will likely impact the way we practice neuroprotection after cardiac arrest in the neuro ICU.

Editor’s Note: Hinson has disclosed receiving personal compensation for serving as an editor, associate editor, or editorial advisory board member for Neurology and for the American Heart Association.

Transcript edited for clarity. Click here for more coverage of AAN 2026.

REFERENCES
1. Cemdisiran, Dosed Subcutaneously Every 12 Weeks, Demonstrates Rapid, Deep and Sustained Disease Control in Generalized Myasthenia Gravis (gMG) Phase 3 Trial. Regeneron Pharmaceuticals. News Release. April 21, 2026. Accessed May 12, 2026. https://investor.regeneron.com/news-releases/news-release-details/cemdisiran-dosed-subcutaneously-every-12-weeks-demonstrates
2. Waldman A, Lynch D, Tonduti D, et al. Efficacy and Safety of Zilganersen, an Investigational RNA-targeted Antisense Therapy, in People Living with Alexander Disease: Results from a Pivotal Study. Presented at: 2026 AAN Annual Meeting; April 18-22; Chicago, Illinois. ABSTRACT 000941
3. Genentech’s Enspryng (Satralizumab) Reduces Risk of Relapses by 68% Demonstrating Potential to Become First Treatment for MOGAD. News release. Genentech. May 12, 2026. Accessed April 21, 2026. https://www.gene.com/media/press-releases/15108/2026-04-21/genentechs-enspryng-satralizumab-reduces