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Abstract Details

Phase 3 EPIC (EPIlepsy Cell Therapy) Clinical Trial Design: NRTX-1001 GABAergic Interneuron Cell Therapy for the Treatment of Drug-resistant Mesial Temporal Lobe Epilepsy (MTLE)
Epilepsy/Clinical Neurophysiology (EEG)
P11 - Poster Session 11 (11:45 AM-12:45 PM)
10-006

To present the Phase 3 EPIC (EPIlepsy Cell Therapy) trial design evaluating NRTX-1001 cell therapy for the treatment of seizures due to drug-resistant mesial temporal lobe epilepsy (MTLE).
GABAergic cortical interneurons from the medial ganglionic eminence (MGE) are critical for regulating the excitability of cortical circuits. We developed a cortical MGE-type GABAergic interneuron cell therapy candidate, NRTX-1001, derived from human pluripotent stem cells for single-dose administration into seizure foci.  Two Phase 1/2 multicenter, open-label trials evaluating NRTX-1001 are enrolling 38 adults with unilateral (NCT05135091) and bilateral (NCT06422923) MTLE with or without mesial temporal sclerosis (MTS). Results from 18 adults with unilateral MTLE with MTS showed NRTX-1001 was well-tolerated, with no treatment-related serious adverse events (SAEs). NRTX-1001 has demonstrated potential for clinically significant and durable seizure reduction, without neurocognitive decline, supporting advancement to Phase 3.   
The Phase 3 EPIC trial is a multicenter, double-blind, randomized, sham-controlled study enrolling adults with drug-resistant MTLE.  Following a 10-week baseline period, subjects randomize 2:1 to undergo NRTX-1001 administration into the affected hippocampus or a sham procedure with active or placebo immunosuppression, respectively. Subjects will be individually unblinded at their 6-month primary endpoint.  Sham procedure subjects will be offered NRTX-1001 and initiate immunosuppression; NRTX-1001 subjects taper off immunosuppression at one year. 
The primary efficacy endpoint is the difference between study arms in median percent change in seizure frequency (mean per 28-day average) from baseline to 4-6 months post-treatment. Secondary and exploratory endpoints include responder rates, safety, EEG, neurocognitive measures, mood, and quality of life. 
The Phase 3 EPIC study will assess the safety and efficacy of NRTX-1001 for drug-resistant MTLE. The study is anticipated to begin treating subjects in early 2026. A single administration of NRTX-1001 cell therapy may offer seizure control for patients who are not eligible for, or interested in, tissue resection or ablation.
Authors/Disclosures
Rose Larios, PhD (Neurona Therapeutics)
PRESENTER 		Dr. Larios has received personal compensation for serving as an employee of Neurona Therapeutics.
Manher Joshi, MD (Atara Biotherapeutics Inc.) Dr. Joshi has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Joshi has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Atara Biotherapeutics. Dr. Joshi has stock in Atara Biotherapeutics.
Gautam Banik (Neurona Therapeutics) Gautam Banik has received personal compensation for serving as an employee of Neurona Therapeutics.
Marina Bershteyn (Neurona therapeutics) Marina Bershteyn has received personal compensation for serving as an employee of Neurona therapeutics. Marina Bershteyn has stock in Neurona therapeutics. Marina Bershteyn has received intellectual property interests from a discovery or technology relating to health care.
Alessandro Bulfone, MD (Neurona Therapeutics) Dr. Bulfone has received personal compensation for serving as an employee of Neurona Therapeutics Inc.
Brianna Feld (Neurona Therapeutics) Brianna Feld has received personal compensation for serving as an employee of Neurona Therapeutics.
Luis Fuentealba (Neurona Therapeutics) Luis Fuentealba has nothing to disclose.
John D. Hixson, MD (Neurona Therapeutics) Dr. Hixson has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Hixson has received personal compensation for serving as an employee of Nile Ai. Dr. Hixson has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for NextSense Inc.. Dr. Hixson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Seer Medical. Dr. Hixson has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Expert Institute. Dr. Hixson has received personal compensation in the range of $5,000-$9,999 for serving as a Reviewer with Epilepsy Study Consortium.
Stephanie M. Hughes, PharmD Dr. Hughes has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Hughes has received personal compensation for serving as an employee of Neurona Therapeutics.
Ji-Hye Jung (Neurona Therapeutics) Ji-Hye Jung has nothing to disclose.
Tia Kowal (Neurona Theraputics) Tia Kowal has received personal compensation for serving as an employee of Neurona Tharapeutics. Tia Kowal has received personal compensation in the range of $100,000-$499,999 for serving as a Scientist with Neurona Theraputics.
Sonja Kriks (Neurona Therapeutics) Sonja Kriks has received personal compensation for serving as an employee of Neurona Therapeutics. Sonja Kriks has stock in Neurona Therapeutics. Sonja Kriks has received intellectual property interests from a discovery or technology relating to health care.
Ngoc Minh D. Le, MD Dr. Le has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Le has stock in Neurona Therapeutics.
Seonok Lee (Neurona Therapeutics) Seonok Lee has received personal compensation for serving as an employee of Neurona Therapeutics.
Sheri Madrid (Neurona Therapeutics, Inc) Mrs. Madrid has nothing to disclose.
Yves Maury (Neurona therapeutics) Yves Maury has received personal compensation for serving as an employee of Neurona Therapeutics. Yves Maury has stock in Neurona therapeutics.
Catherine Priest, PhD (Neurona Therapeutics, Inc) Catherine Priest has received personal compensation for serving as an employee of Neurona Therapeutics. Catherine Priest has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novo Nordisk. The institution of Catherine Priest has received research support from CIRM.
Cory R. Nicholas, PhD (Neurona Therapeutics) Dr. Nicholas has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Nicholas has stock in Neurona Therapeutics. Dr. Nicholas has received intellectual property interests from a discovery or technology relating to health care.