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Abstract Details

Clinical Update: NRTX-1001 GABAergic Interneuron Cell Therapy for Bilateral Drug-resistant Focal Epilepsy
Epilepsy/Clinical Neurophysiology (EEG)
S41 - Epilepsy: Public Health and Epidemiology (1:24 PM-1:36 PM)
003
To present results from an ongoing open-label, multicenter Phase 1/2 trial (NCT06422923) evaluating NRTX-1001 cell therapy for the treatment of seizures due to bilateral 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. NRTX-1001 demonstrated encouraging results in an ongoing open-label Phase 1/2 clinical trial (NCT05135091) evaluating intra-hippocampal administration of cells in 18 adults with unilateral drug-resistant MTLE. NRTX-1001 has been well-tolerated, with preliminary efficacy data showing a substantial reduction in seizure frequency and no decline in neurocognitive performance. 
This Phase 1/2 study investigates bilateral intra-hippocampal administration of NRTX-1001 in 10 adults with bilateral drug-resistant MTLE. Immunosuppression is administered to promote allograft persistence, beginning preoperatively and tapering after year one. Quarterly visits monitor adverse events (AEs), seizure frequency, EEG, imaging, and neuropsychological metrics. The primary endpoint is one-year safety; the secondary endpoint is seizure frequency at months 7-12 post-administration. 
The first bilateral MTLE subject treated with NRTX-1001 achieved seizure freedom and continues to be seizure-free at 6 months (time of abstract submission). These data, and data for additional subjects treated, will be presented at the conference.
One-time NRTX-1001 administration offers the potential for durable seizure control in bilateral drug-resistant MTLE. NRTX-1001 cell therapy may represent an option for patients with bilateral seizure generation who are not eligible for, or interested in, resection, ablation, or neurostimulator implantation surgeries.
Authors/Disclosures
Peter C. Warnke, MD
PRESENTER 		Prof. Warnke has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Medtronic. Prof. Warnke has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Kline Specter.
Naoum P. Issa, MD, PhD (University of Chicago) The institution of Dr. Issa has received research support from NIH. Dr. Issa has received intellectual property interests from a discovery or technology relating to health care. Dr. Issa has received personal compensation in the range of $0-$499 for serving as a Study Section Member with NIH. Dr. Issa has a non-compensated relationship as a Scientific advisory board member with Theta Neurotech that is relevant to AAN interests or activities.
Kevin Graber, MD (Stanford University Medical Center) The institution of Dr. Graber has received research support from Neurona Therapeutics.
Vivek Buch (Stanford University) Vivek Buch has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Scientific. Vivek Buch has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neuropace.
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.
Nathan B. Fountain, MD, FAAN (University of Virginia) Dr. Fountain has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Fountain has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Coda. Dr. Fountain has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Fountain has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Lundbeck. Dr. Fountain has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Eisai. Dr. Fountain has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Fountain has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for BrainStorm. Dr. Fountain has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Shackleford. Dr. Fountain has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Acumen. Dr. Fountain has stock in Hexokine.
Shayan Moosa, MD Dr. Moosa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for RebrAIn.
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.
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.
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.
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.