AAN 2026: Patient-Centered Progress on Many Fronts

AAN 2026 highlighted wide-ranging advances, from the treatment of neurodegeneration and movement disorders to meaningful progress in epilepsy and rare disease. And for our team, the meeting reinforced strong alignment between where neurology is headed and our areas of therapeutic expertise.

Alzheimer's Disease

Alzheimer's disease (AD) remained front and center at AAN 2026. Anti-amyloid therapies were an important foundation in this year's discussions, including the importance of early diagnosis and intervention, appropriate patient selection, safety considerations, and the possibility of next-generation monoclonal antibodies. Presenters and attendees shared their experiences with and insights into the practical implementation of these therapies.

While semaglutide failed to demonstrate efficacy in a phase 3 trial in symptomatic AD, interest in GLP-1s for AD (and other neurodegenerative diseases) treatment persists. Early-phase studies, epidemiologic data, and mechanistic rationale all suggest that this class of drugs could play a role in risk reduction, prevention, or combination therapy. Additional studies are needed to understand where these medicines may bring value.

Blood-based biomarkers (BBMs) are quickly making their way into clinical practice. Experts shared how they are using BBMs to facilitate earlier diagnosis and assess appropriateness of anti-amyloid therapy.

Parkinson's Disease

Parkinson's disease education at AAN 2026 showcased how rapidly the treatment landscape is diversifying beyond traditional dopaminergic approaches. Non-dopaminergic strategies generated strong interest, particularly therapies aimed at reducing OFF time, improving non-motor symptoms such as sleep, and minimizing dyskinesia risk. These approaches reflect an important shift toward whole-disease management versus motor symptoms alone.

Equally exciting were updates on dopamine-sparing strategies, highlighting the goal of maintaining symptom control earlier in the disease while delaying complications associated with long-term levodopa use.

Perhaps most intriguing were presentations around regenerative cell therapy, underscoring the possibility of restoring—not just supplementing—some level of dopaminergic function in patients with uncontrolled motor symptoms. While it's still very early, these programs could provide new options for the Parkinson's community.

Epilepsy

Innovation in epilepsy is alive, targeted, and increasingly patient-centric. Late-breaking phase 3 results for a novel Kv7 potassium channel activator, azetukalner, demonstrated robust seizure reduction in highly treatment-resistant populations, with additional long-term data supporting sustained safety and efficacy. The absence of a titration requirement, combined with once-daily dosing, supports ease of use for patients.

Sparking interest in regenerative therapy are phase 1 and 2 trials of a GABAergic interneuron allogeneic cell therapy in drug-resistant mesial temporal lobe epilepsy. A phase 3 trial is scheduled to begin this year.

Essential Tremor

While essential tremor (ET) is common, effective pharmacologic treatments are lacking. Data from a phase 3 trial of ulixacaltamide—an investigational, T-type calcium-channel modulator—demonstrated improvement and sustained response in activities of daily living, suggesting it could provide relief for some patients. Ulixacaltamide is under FDA review, with a decision expected in January 2027.

Wearable and device-based therapies continued to generate interest for tremor control with favorable tolerability—potentially offering options for patients who are not candidates for invasive procedures or who struggle with medication side effects.

The growing emphasis on functional improvement and quality of life reflects how the field is evolving. The excitement around scalable, patient-controlled solutions suggests that ET is entering an era where treatment choice and personalization will be expanding.

Tourette Syndrome

We were encouraged by a phase 3, double-blind, placebo-controlled, randomized withdrawal trial of ecopipam, a selective D1 receptor antagonist. The data suggest a durable impact on tics without clinically relevant metabolic adverse events. FDA approval could represent a meaningful development for the Tourette syndrome community.

Rare Neurologic Disease

Discussion of rare neurologic diseases was woven throughout the meeting—an encouraging sign of how integral these conditions have become to the neurology research ecosystem.

Advances in genetic diagnostics, pathway-specific therapies, and platform technologies continue to shorten the distance between disease biology and therapeutic action. And importantly, rare disease programs are increasingly influencing broader neurology innovation, from trial design to regulatory science. What we learn in rare disease is shaping the future of neurology as a whole.

Going Forward

AAN 2026 left us energized, optimistic, and deeply aligned with where neurology is headed. We are inspired by the science, affirmed in our strategy, and excited to continue contributing to a future where neurologic diseases are treated earlier, more precisely, and more effectively than ever before.

Reported by The HWP Group's Elizabeth Rappa, PharmD