The 2025 Annual Meeting of the American Academy of Neurology in San Diego brought together over 14,500 neurology professionals from 110 countries and all 50 US states, alongside more than 300 exhibiting companies. It was a week filled with inspiring science, learning, and global collaboration—all in support of advancing brain health for all.
Among the many highlights were the late-breaking abstracts, showcasing some of the most exciting developments in neurology. From this impressive selection, we’ve chosen five standout presentations that reflect the innovation and momentum seen throughout the meeting.
Phase 3 results highlight telitacicept as a potential new option for generalized myasthenia gravis
Results from a phase 3 study showed that telitacicept, a novel B cell-targeting fusion protein, significantly improved symptoms in patients with generalized myasthenia gravis (gMG). After 24 weeks, patients receiving telitacicept saw notable reductions in MG-ADL and QMG scores compared to placebo, with 98.1% achieving a clinically meaningful improvement in daily living activities. The treatment also led to decreased immunoglobulin levels, with the most common adverse event being a reduction in IgM. These findings support telitacicept as a promising new therapy for gMG, with further data expected from the ongoing open-label phase.
Early promise for oral therapy in childhood cerebral adrenoleukodystrophy
Results from the open-label phase 2/3 NEXUS study show that leriglitazone, an oral PPARγ agonist, achieved disease arrest in a significant number of boys with childhood cerebral adrenoleukodystrophy (cALD). The treatment led to clinical and radiological stabilization without major functional disabilities or serious treatment-related adverse events. This marks a meaningful step toward a less invasive alternative to HSCT for this rare and devastating condition. Further insights into secondary and exploratory outcomes are expected.
Oral dopamine agonist shows promise in early Parkinson’s disease
Results from the phase 3 TEMPO-1 and TEMPO-2 trials presented at AAN 2025 highlight the potential of tavapadon, a once-daily oral D1/D5 partial dopamine agonist, as an effective monotherapy for early Parkinson’s disease (PD). TEMPO-1 investigated two fixed doses of tavapadon (5 mg and 15 mg), while TEMPO-2 evaluated a flexible dosing approach to identify the optimal tolerated dose for each patient. Both studies showed significant improvements in motor symptoms and daily functioning compared to placebo, as measured by MDS-UPDRS scores. Tavapadon was generally well tolerated, with mostly mild to moderate adverse events, and long-term data from the ongoing TEMPO-4 extension study will further inform its clinical potential.
Read the full abstract for TEMPO-1 and TEMPO-2 here.
First-in-class antibody shows early promise for acute spinal cord injury recovery
A phase I/II multicenter study presented at AAN 2025 investigated ALMB-0166, a novel monoclonal antibody targeting connexin-43 hemichannels, for the treatment of acute spinal cord injury (SCI). The trial assessed the safety, tolerability, and preliminary efficacy of single intravenous doses of ALMB-0166 in patients with cervical SCI, comparing outcomes to placebo. ALMB-0166 was well tolerated and associated with improvements in motor and sensory function, pain reduction, and AIS grade recovery by day 56. These early results support the potential of ALMB-0166 as a therapeutic strategy to enhance neurological recovery following acute SCI.
AXS-05 shows strong potential for managing agitation in Alzheimer’s disease
New results from the phase 3 ACCORD-2 study, presented at AAN 2025, highlight the efficacy and safety of AXS-05, an oral NMDA receptor antagonist/sigma-1 receptor agonist, in treating agitation associated with Alzheimer’s disease (AD). The randomized withdrawal trial evaluated patients who initially responded to AXS-05 during an open-label phase and were then assigned to continue treatment or switch to placebo. AXS-05 significantly delayed and prevented relapse of agitation, while also reducing the risk of overall clinical worsening. The treatment was well tolerated, with low rates of adverse events and no signs of sedation or cognitive decline, highlighting its potential as a much-needed option for this common and challenging AD symptom.
Disclosures: This article was created by the touchNEUROLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.
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