EAN 2026 highlights: New therapies and evolving approaches to neurological care

Highlights from the European Academy of Neurology (EAN) 2026 Congress included new phase 3 data in ocular myasthenia gravis, emerging migraine treatments, longer-term results in multiple sclerosis, and advances in deep brain stimulation for Parkinson’s disease.

The 12th Congress of the European Academy of Neurology (EAN), held from June 27–30, 2026, in Geneva, Switzerland, brought together new clinical and translational research spanning a broad range of neurological diseases.

Among the congress highlights were positive phase 2 and 3 trial results for emerging therapies, longer-term data for investigational treatments, and studies exploring how established approaches to disease monitoring and treatment delivery could be refined. Here, we highlight selected presentations with particular relevance to current and future neurological practice.

Efgartigimod shows phase 3 efficacy in ocular myasthenia gravis

Ocular myasthenia gravis (oMG) can cause disabling ptosis and diplopia, yet targeted treatment options specifically evaluated in this population remain limited. Interim results from the phase 3 ADAPT OCULUS trial (NCT06558279) therefore provided important new evidence in this setting.^1,2

Adults with oMG and a Myasthenia Gravis Impairment Index patient-reported outcome (MGII [PRO]) ocular score of ≥6 were randomized to receive four once-weekly injections of subcutaneous efgartigimod PH20 1,000 mg or placebo. Efgartigimod inhibits neonatal Fc receptor (FcRn)-mediated immunoglobulin G recycling, reducing circulating pathogenic immunoglobulin G antibodies.

The trial met its primary endpoint. At Week 4, the least-squares mean change in MGII (PRO) ocular score was −4.04 points with efgartigimod compared with −1.99 points with placebo (p=0.012), with improvements reported across ocular symptoms. No new safety concerns were identified.¹

ADAPT OCULUS is the first registrational trial designed specifically to evaluate a targeted therapy in oMG.¹ Longer-term data will be important in determining the durability of response, but the findings suggest that the role of FcRn inhibition could extend beyond generalized myasthenia gravis.

Targeting pituitary adenylate cyclase-activating polypeptide opens another avenue for migraine prevention

Calcitonin gene-related peptide (CGRP)-targeting therapies have transformed migraine prevention, but some patients continue to experience substantial disease burden despite existing preventive treatments. Data presented at EAN highlighted pituitary adenylate cyclase-activating polypeptide (PACAP) as a potentially distinct therapeutic target.

Bocunebart (Lu AG09222) is an investigational monoclonal antibody designed to inhibit PACAP. Its phase 2 development program has included the HOPE trial (NCT05133323) and the phase 2b PROCEED trial (NCT06323928) in people with migraine who had experienced failure of previous preventive treatments.^3–5

In the intravenous portion of PROCEED, the primary endpoint was met. Monthly migraine days decreased by 4.24 days with the selected bocunebart dose compared with 2.86 days with placebo over Weeks 1–12 (p=0.0178).³

A pooled analysis of participants with chronic migraine across HOPE and PROCEED also showed greater reductions with bocunebart compared with placebo in monthly migraine days (−5.94 versus −3.63; p<0.001), monthly headache days (−6.90 versus −4.32; p<0.001), and migraine days requiring acute medication (−5.21 versus −3.53; p<0.05). No new safety signals were identified.³

The findings provide further support for PACAP inhibition as an emerging migraine prevention strategy. Importantly, the pathway is mechanistically distinct from CGRP, raising the possibility of another therapeutic approach for people whose migraine remains inadequately controlled with existing preventive treatments.

Frexalimab maintains low multiple sclerosis disease activity at 3 years

Longer-term phase 2 data were presented for frexalimab, an investigational monoclonal antibody targeting CD40 ligand (CD40L), a pathway involved in both innate and adaptive immune activation.

In the phase 2 study (NCT04879628), participants with relapsing multiple sclerosis (MS) initially received intravenous or subcutaneous frexalimab before continuing treatment in an open-label extension. At EAN 2026, investigators reported outcomes through Week 144.^6,8

Magnetic resonance imaging (MRI) disease activity remained low, with a mean of 0.1 gadolinium-enhancing lesions in the intravenous group and 0.0 in the subcutaneous group at Week 144. Overall, 86% of participants remained relapse-free. Treatment continued to be generally well tolerated, and lymphocyte counts remained stable.⁶

Supporting biomarker analyses showed reductions in plasma neurofilament light chain (pNfL) of approximately 45–47% from Baseline through Week 144.⁷ With intravenous treatment, brain volume changes from Baseline to Week 144 were -0.84% for whole brain, -1.18% for the thalamus, and −0.85% for the cerebral cortex.⁷

The sustained suppression of disease activity is encouraging, particularly because CD40L inhibition represents an immune-modulating strategy that does not rely on lymphocyte depletion. Phase 3 studies are evaluating frexalimab in relapsing MS and nonrelapsing secondary progressive MS.

Could annual magnetic resonance imaging be reduced during long-term ocrelizumab treatment?

While emerging therapies attracted considerable attention at EAN, other studies questioned whether established approaches to disease monitoring could be made more personalized.

Investigators from the Amsterdam MS Cohort examined 1,639 MRI scans from 342 people with relapsing or primary progressive MS receiving ocrelizumab over a median treatment duration of 49 months.⁹

Significant radiological disease activity was detected in only 27 scans from 23 patients, representing 1.8% of scans and 6.8% of patients. Importantly, the three patients whose MRI findings prompted a treatment switch had developed clinical symptoms before undergoing imaging.⁹

The number needed to scan to identify disease activity increased with treatment duration, particularly after 2 years. The investigators therefore proposed that MRI intervals could potentially be extended in clinically stable, asymptomatic patients receiving longer-term ocrelizumab, while maintaining closer surveillance during early treatment and in patients who develop new symptoms.⁹

The study does not establish a new monitoring schedule, and prospective validation will be required before practice recommendations change. Nevertheless, the findings raise an important question about whether uniform annual MRI surveillance remains necessary for every patient receiving highly effective therapy.

Neurofilament light chain and glial fibrillary acidic protein could provide complementary information on multiple sclerosis progression

Another potential route toward more individualized monitoring is the use of fluid biomarkers.

Investigators compared neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP) measurements from 1,709 participants in the Swiss MS Cohort and 620 participants in the University of California, San Francisco EPIC cohort.¹⁰

An elevated Baseline NfL Z score >1.5 was associated with an increased risk of relapse during the following year in both cohorts, with an odds ratio of 2.00 in the Swiss cohort and 1.69 in EPIC.¹⁰

GFAP appeared to capture a different aspect of disease activity. A Baseline GFAP Z score >1.0 was associated with a 45% higher risk of progression independent of relapse activity (PIRA) at the subsequent visit in the Swiss cohort and a 36% higher risk in EPIC.¹⁰

Changes in GFAP during treatment may also prove informative. Each annual one-unit reduction in GFAP Z score during the first 2 years of treatment was associated with a 54% lower subsequent risk of PIRA among patients treated with fingolimod and a 67% lower risk among those receiving B-cell-depleting therapy.¹⁰

Together, the findings suggest that NfL and GFAP may provide complementary information: NfL appears particularly relevant to inflammatory activity and neuroaxonal injury, whereas GFAP may offer additional insight into relapse-independent progression. Whether these biomarkers can ultimately be incorporated into routine treatment decisions will depend on prospective validation, assay standardization, and the establishment of clinically meaningful thresholds.

Remote deep brain stimulation programming is noninferior in Parkinson’s disease

The EAN program also highlighted how technology could change the delivery of established neurological treatments.

Deep brain stimulation (DBS) is an established treatment for selected people with advanced Parkinson’s disease, but successful therapy requires specialist programming, often involving repeated visits to expert centers.

A prospective, randomized, multicenter noninferiority trial (NCT05193825) conducted across DBS centers in Germany compared conventional in-person programming with programming delivered remotely using a secure telemedicine platform.^11,12

Of 52 patients randomized, 48 were included in the per-protocol analysis. At 3 months, median improvement in Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III scores was −23.0 points with conventional programming and −20.5 points with remote programming. The median between-group difference was −2.5 points (95% CI −9.0 to 2.0), meeting the prespecified noninferiority criterion.¹¹

Improvements in total MDS-UPDRS scores, Hoehn and Yahr stage, and Parkinson’s Disease Questionnaire-39 outcomes were also similar, and adverse and serious adverse events were comparable between groups.¹¹

Although longer-term studies and broader implementation data will be required, the results provide prospective controlled evidence that remote DBS programming can achieve comparable short-term motor outcomes to in-person care. This could be particularly relevant for people living far from specialist centers or whose disability makes frequent travel challenging.

First-in-class therapy shows promise in trigeminal neuralgia

A positive phase 2 study also provided an emerging therapeutic lead for trigeminal neuralgia, a condition characterized by recurrent, severe paroxysmal facial pain.

SR750 is a first-in-class negative allosteric modulator of α5-containing gamma-aminobutyric acid type A receptors being investigated for neuropathic pain. In a double-blind, randomized, placebo-controlled phase 2 trial (NCT06571448), 138 patients with primary trigeminal neuralgia were randomized to receive SR750 50 mg twice daily, SR750 150 mg twice daily, or placebo for 6 weeks.^13,14

At Week 6, least-squares mean reductions from Baseline in daily average paroxysmal pain scores were −0.89 with placebo, −1.83 with SR750 50 mg, and −1.97 with SR750 150 mg. Both SR750 doses were statistically superior to placebo, with between-group differences of −0.94 (p=0.031) and −1.08 (p=0.012), respectively.¹³

The trial was relatively small, and further studies will be needed to establish durability of response, longer-term safety, and the potential position of SR750 relative to existing treatments. However, a new mechanism of action is noteworthy in a condition in which pharmacological options can be limited by incomplete response or tolerability.

Tau-targeting antisense therapy advances toward phase 3 in progressive supranuclear palsy

Progressive supranuclear palsy (PSP) remains an area of substantial unmet need, with no approved treatment proven to modify the underlying neurodegenerative process. Results presented at EAN provided an early look at a therapeutic strategy designed to directly reduce tau production.

NIO752 is an antisense oligonucleotide (ASO) targeting MAPT messenger RNA, with the aim of reducing production of tau protein and potentially limiting the accumulation of pathological tau associated with PSP.¹⁵

In a first-in-human, randomized, double-blind, placebo-controlled phase 1 study (NCT04539041), 59 people with PSP-Richardson syndrome received intrathecal NIO752 or placebo. Forty-five participants received active treatment and 14 received placebo.^15,16

NIO752 demonstrated an acceptable safety profile, with most adverse events reported as mild or moderate. Serious adverse events occurred at similar rates in the treatment and placebo groups.¹⁵

The phase 1 study was not designed to establish clinical efficacy, and the EAN data did not demonstrate that NIO752 slows disease progression. However, the program is notable because NIO752 is the first MAPT-targeting ASO investigated as a potential treatment for PSP. The phase 3 PRESERVE trial (NCT07498426) is now evaluating the efficacy and safety of NIO752 in people with PSP-Richardson syndrome.^15,17

For a rapidly progressive primary tauopathy with no disease-modifying therapy, establishing whether direct suppression of tau production can alter the clinical course will be an important test of genetically and molecularly targeted approaches to neurodegenerative disease.

Looking ahead

Selected data from EAN 2026 reflected two parallel changes taking place across neurology. New therapeutic mechanisms are continuing to emerge, from FcRn inhibition in ocular myasthenia gravis and PACAP inhibition in migraine to CD40L targeting in MS, modulation of α5-containing gamma-aminobutyric acid type A receptors in trigeminal neuralgia, and tau-lowering strategies in PSP.

At the same time, advances are not limited to new medicines. Studies examining biomarker-guided assessment, reduced MRI surveillance, and remote DBS programming suggest that greater personalization could also change how established neurological treatments are monitored and delivered.

Not all of these approaches are ready for routine clinical practice. Several findings remain based on early-phase trials or require longer-term confirmation. Nevertheless, the breadth of research presented at EAN 2026 illustrates an increasingly mechanism-based and individualized approach to neurological disease, with potential implications across diagnosis, treatment, and long-term care.

References

1. Claeys K, Juel V, Barnett-Tapia C, et al. Efficacy and safety of efgartigimod PH20 SC in adults with ocular myasthenia gravis: ADAPT OCULUS interim results. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr LB_11.
2. ClinicalTrials.gov. A study to assess efficacy and safety of efgartigimod PH20 SC in adults with ocular myasthenia gravis. ClinicalTrials.gov Identifier: NCT06558279. Available at: https://clinicaltrials.gov/study/NCT06558279 (accessed September 2, 2026).
3. Ailani J, Phul R, Florea I, et al. Bocunebart (anti-PACAP monoclonal antibody Lu AG09222) in adults with migraine and prior preventive treatment failures: Phase 2 program outcomes. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr LB_09.
4. ClinicalTrials.gov. A dose-finding trial with Lu AG09222 in adults with migraine who have not been helped by prior preventive treatments. ClinicalTrials.gov Identifier: NCT06323928. Available at: https://clinicaltrials.gov/study/NCT06323928 (accessed September 2, 2026).
5. ClinicalTrials.gov. A study with Lu AG09222 in adults with migraine who have not been helped by prior preventive treatments. ClinicalTrials.gov Identifier: NCT05133323. Available at: https://clinicaltrials.gov/study/NCT05133323 (accessed September 2, 2026).
6. Vermersch P, Krieger S, Wiendl H, et al. Efficacy and safety of frexalimab in participants with relapsing multiple sclerosis: 3-year results from the phase 2 open-label extension. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr EPO-0785.
7. Granziera C, Vermersch P, Djukic B, et al. Long-term treatment effects of frexalimab on NfL, CXCL13, and brain volume loss in relapsing multiple sclerosis. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr EPO-0790.
8. ClinicalTrials.gov. Proof-of-concept study for SAR441344 (frexalimab) in relapsing multiple sclerosis. ClinicalTrials.gov Identifier: NCT04879628. Available at: https://clinicaltrials.gov/study/NCT04879628 (accessed September 2, 2026).
9. Schoof L, Heijnen R, Hogenboom L, et al. Rethinking annual MRI monitoring in ocrelizumab-treated multiple sclerosis patients: A real-world number needed to scan analysis. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr EPO-0137.
10. Einsiedler M, Sandgren S, Schaedelin S, et al. Assessing the value of NfL and GFAP for individualized treatment decisions in two independent large observational cohorts. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr OPR-021.
11. Köglsperger T, Shaik J, Zhang W, et al. Remote deep brain stimulation programming for Parkinson’s disease: A prospective, randomized, controlled, multicentre, non-inferiority trial. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr OPR-132.
12. ClinicalTrials.gov. Remote deep brain stimulation programming for Parkinson’s disease. ClinicalTrials.gov Identifier: NCT05193825. Available at: https://clinicaltrials.gov/study/NCT05193825 (accessed September 2, 2026).
13. Fan B, Han Q, Jin L, et al. Safety and efficacy of SR750 in patients with trigeminal neuralgia: A double-blind, randomized, placebo-controlled, phase 2 study. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr LB_16.
14. ClinicalTrials.gov. Study of SR750 in patients with trigeminal neuralgia. ClinicalTrials.gov Identifier: NCT06571448. Available at: https://clinicaltrials.gov/study/NCT06571448 (accessed September 2, 2026).
15. Serban C, Höglinger G, Boxer A, et al. NIO752, a tau-targeting antisense oligonucleotide, in progressive supranuclear palsy: Phase I results and phase III design. Presented at: 12th Congress of the European Academy of Neurology, Geneva, Switzerland, June 27–30, 2026. Abstr OPR-065.
16. ClinicalTrials.gov. Safety, tolerability and pharmacokinetics of multiple ascending doses of NIO752 in progressive supranuclear palsy. ClinicalTrials.gov Identifier: NCT04539041. Available at: https://clinicaltrials.gov/study/NCT04539041 (accessed September 2, 2026).
17. ClinicalTrials.gov. A study to evaluate the efficacy of NIO752 in participants with progressive supranuclear palsy. ClinicalTrials.gov Identifier: NCT07498426. Available at: https://clinicaltrials.gov/study/NCT07498426 (accessed September 2, 2026).

Abstracts from the 12th EAN Congress were published in the June 2026 supplement of the European Journal of Neurology, Volume 33, Issue S1.