In early December, the Neumirna team attended the Americal Epilepsy Society (AES) Annual Meeting in Atlanta, Georgia. AES is one of the most important international forums for epilepsy research, bringing together scientists, clinicians, and patient-focused organizations from around the world.
At the 2025 meeting, the Neumirna team presented two scientific posters highlighting different aspects of our lead program, NMT.001, in development for patients with drug-resistant epilepsy.
Building confidence in NMT.001
Our first poster focused on the foundations of the NMT.001 program. NMT.001 targets a small regulatory molecule in the brain called miR-134. Research shows that this molecule is consistently elevated in both human epilepsy tissue and well-established animal models of the disease. By selectively targeting miR-134, NMT.001 aims to rebalance disease-relevant brain networks rather than simply suppress seizures.
The poster summarized results from early safety and toxicology studies. These studies are a critical step toward clinical development, helping to determine whether the therapy can be safely administered at doses that are effective. The data presented at AES demonstrated adequate safety margins across relevant preclinical studies, supporting continued progress toward the clinic.
In addition, the poster outlined the overarching framework and design for Neumirna’s planned Phase 1/2 clinical study with NMT.001 called MiRNova. The study is designed to optimize a potential patient benefit and to best inform a subsequent wider Phase 3 program. Our poster provided an update on the status of the program and sparked many discussions around study design and clinical translation.
Beyond seizures
While seizure reduction remains essential, epilepsy affects far more than seizure frequency alone. Anxiety, cognitive challenges, and other comorbidities can have a profound impact on daily life and are often insufficiently addressed by existing treatments.
Our second poster explored this dimension directly. Based on a well-validated mouse model of drug-resistant epilepsy, our study examined whether NMT.001 could influence epilepsy-related comorbidities, in addition to its previously demonstrated anti-seizure effects, speaking to its potential as a disease-modifying treatment.
The findings showed that treatment with NMT.001 was associated with improvements in anxiety-related behavior, locomotor abnormalities, and memory performance, and these effects were sustained weeks after a single administration. Importantly, NMT.001 did not alter behavior in healthy animals, indicating that the observed effects were specific to the epilepsy disease model.
These results support the idea that targeting core disease pathways may offer broader benefits for people living with drug-resistant epilepsy by addressing aspects of the condition that significantly impact patients and families, but are often overlooked in drug development.
Transforming epilepsy care
The feedback and discussions at AES helped sharpen our thinking and reinforce the importance of pursuing therapies that are driven by innovative RNA science and guided by patient-relevant outcomes.
The Neumirna team returned from AES energized by the engagement of the epilepsy community and encouraged by the growing interest in approaches that aim to move epilepsy treatment forward in a transformative way.
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