Neurona Therapeutics reports Phase 1/2 data showing durable seizure reduction and favorable safety of rezanecel in drug-resistant mesial temporal lobe epilepsy at AAN 2026.
Written By: Karthik Teja Macharla, PharmD
Reviewed By: Pharmacally Editorial Team
Neurona Therapeutics has reported updated clinical findings from its ongoing Phase 1/2 trials (NCT05135091) of rezanecel (formerly NRTX‑1001), an investigational allogeneic interneuron cell therapy, in patients with drug‑resistant mesial temporal lobe epilepsy (MTLE), presented at the American Academy of Neurology Annual Meeting 2026.
The open‑label, multicenter program has enrolled 31 participants with unilateral or bilateral MTLE, with or without mesial temporal sclerosis (MTS), and includes 15 patients followed for more than 18 months, providing the most mature data to date on durability, safety, and clinical outcomes.
Across cohorts, rezanecel demonstrated consistent seizure reduction, particularly in unilateral MTLE with MTS. In this subgroup, low‑dose treatment achieved an 89% median reduction in disabling seizures during the 7–12‑month primary efficacy evaluation period and maintained a similar 100% median reduction beyond one year, with responder rates (≥50% seizure reduction) of 78% at 7–12 months and 89% beyond one year.
High‑dose treatment in the same population showed a 58% median reduction in disabling seizures with a 78% responder rate at the interim 4–6‑month evaluation period.
Interim data in smaller cohorts indicated a 100% median seizure reduction in unilateral MTLE without MTS and a 64% median reduction in bilateral MTLE, though follow‑up in these groups remains limited.
The therapy showed a favorable safety profile, with no serious adverse events attributed to rezanecel or its administration. Most immunosuppression‑related events were mild to moderate and resolved in the majority of patients, while serious adverse events were linked to underlying comorbidities or epilepsy‑related complications rather than the cell therapy.
Cognitive assessments showed no meaningful decline across key domains, including memory and language function, and no significant impairments were detected in either dose cohort.
According to Chief Medical Officer Eduardo Dunayevich, the data support durable seizure reduction beyond one year with a consistent safety profile, while early findings in bilateral MTLE suggest potential broader applicability in patients who are not adequately managed by current surgical or device‑based treatments.
The World Health Organization (WHO) has assigned rezanecel as the international non‑proprietary name (INN) for the therapy, replacing NRTX‑1001.
Neurona plans to initiate a randomized, sham‑controlled Phase 3 EPIC trial (EPIlepsy Cell therapy) with low‑dose rezanecel in drug‑resistant MTLE in the second half of 2026 to further evaluate its efficacy and safety.
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About the Writer
Karthik Teja Macharla, PharmD is a Pharm.D. graduate with a strong interest in clinical research, pharmacovigilance, and medical writing. In his words, he is passionate about converting complex medical information into clear, evidence-based scientific communication, committed to contributing to patient safety and advancing healthcare through accurate and impactful medical content.