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Abstract Details

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy - Updated Clinical Trial Results
Epilepsy/Clinical Neurophysiology (EEG)
P8 - Poster Session 8 (8:00 AM-9:00 AM)
9-003
To present updated findings from a first-in-human, open-label study of NRTX-1001, an allogeneic GABAergic interneuron therapy, for unilateral drug-resistant temporal lobe epilepsy with mesial temporal sclerosis (NCT05135091).
NRTX-1001 is an investigational GABAergic interneuron product derived from human pluripotent stem cells. Preclinical studies demonstrated that hippocampal transplantation significantly reduces seizure frequency in a mouse kainic acid model of focal epilepsy (Bershteyn et.al., Cell Stem Cell 30:1-20, 2023). Between June 2022 and May 2024, ten subjects were enrolled in the ongoing first-in-human study designed to assess the safety and efficacy of NRTX-1001.    
Adults participants with drug-resistant unilateral TLE with mesial temporal sclerosis were administered NRTX-1001 via stereotactic surgery into the hippocampal head and body with intraoperative imaging. Immunosuppression was initiated one week prior to the procedure and is scheduled for tapering after one year. The primary endpoint is safety, with secondary endpoints of seizure frequency, neuroimaging, EEG, cognitive function, and visual fields at one-year post-treatment.
As of July 2024, five subjects in the low-dose cohort were at least 6 months post-treatment, with two subjects completing 24 and 19 months of follow-up. The low-dose cohort demonstrated an 82% reduction in disabling seizure frequency over the 4-6-month post-treatment period, with 80% achieving a ≥50% responder rate. No treatment-related serious adverse events (SAEs) have been reported. For all treated subjects, three non-treatment-related SAEs were observed. These were not determined to be related to the cells, procedure, or immunosuppression. Preliminary cognitive assessments revealed improvements in select measures of memory function for some subjects.  Updated results for all subjects will be presented.
NRTX-1001 administration has been well-tolerated in ten patients with drug-resistant TLE, with preliminary data suggesting significant reductions in seizure frequency and potential cognitive improvements. Unlike current surgical treatment options, NRTX-1001 may provide a non-destructive, cell-based therapeutic approach, potentially providing a restorative option for focal epilepsy.  
Authors/Disclosures
John D. Hixson, MD (Neurona Therapeutics)
PRESENTER 		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.
David C. Spencer, MD, FAAN Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz Pharmaceuiticals. Dr. Spencer has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NeuroPace Inc. Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Various. The institution of Dr. Spencer has received research support from NIH. Dr. Spencer has received publishing royalties from a publication relating to health care. Dr. Spencer has received personal compensation in the range of $500-$4,999 for serving as a Faculty in Teaching Course supported by unrestricted educational grants with Creative 好色先生 Concepts.
Kim Burchiel Kim Burchiel has nothing to disclose.
Harish Babu Harish Babu has nothing to disclose.
Robert Beach, MD, PhD, FAAN (Upstate Medical University) The institution of Dr. Beach has received research support from Neurona.
Jerry J. Shih, MD, FAAN (UC San Diego Health) Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurelis. Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Neurelis. Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for SK Life. Dr. Shih has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sunovion. The institution of Dr. Shih has received research support from Neurona. The institution of Dr. Shih has received research support from NCGS, Inc. Dr. Shih has a non-compensated relationship as a Test Writing Committee Member with ABPN that is relevant to AAN interests or activities.
Sharona Ben-Haim No disclosure on file
Rebecca O'Dwyer, MD (Rush University Medical Center) Dr. O'Dwyer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Pharma. Dr. O'Dwyer has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for SK Life Sciences.
Sepehr Sani The institution of Sepehr Sani has received research support from NIH. Sepehr Sani has received intellectual property interests from a discovery or technology relating to health care. Sepehr Sani has received publishing royalties from a publication relating to health care.
Derek Southwell (Duke University) Derek Southwell has nothing to disclose.
Matthew W. Luedke, MD (Duke University Hospital Dept. of Neurology) Dr. Luedke has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Aucta Pharmaceuticals. Dr. Luedke has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. Dr. Luedke has received personal compensation in the range of $0-$499 for serving as a Consultant for Alphasights . Dr. Luedke has received personal compensation in the range of $0-$499 for serving as a Consultant for Clarivate. Dr. Luedke has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Starnes Davis Florie LLP. Dr. Luedke has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Huff Powell Bailey. The institution of Dr. Luedke has received research support from US NAVY. The institution of Dr. Luedke has received research support from Neurona. The institution of Dr. Luedke has received research support from Xenon.
David E. Blum, MD Dr. Blum has received personal compensation for serving as an employee of Neurona Therapeutics. Dr. Blum has stock in Neurona Therapeutics.
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.
Holly Finefrock (Neurona Therapeutics) Ms. Finefrock has received personal compensation for serving as an employee of Neurona Therapeutics.
Luis Fuentealba (Neurona Therapeutics) Luis Fuentealba has nothing to disclose.
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.
Rose Larios, PhD (Neurona Therapeutics) Dr. Larios has received personal compensation for serving as an employee of 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.
Sergei Shevchuk (Neurona Therapeutics) Sergei Shevchuk 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.
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.