NRTX-1001 / Neurona Therap - LARVOL DELTA

A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE) (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Neurona Therapeutics | Trial completion date: Jul 2040 ➔ Jun 2042 | Trial primary completion date: Jul 2026 ➔ Jun 2027

Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy

Neurona Therapeutics Presents Positive Clinical Data Update from NRTX-1001 Cell Therapy Trial in Drug-resistant Epilepsy at American Academy of Neurology Annual Meeting (GlobeNewswire) - P1/2 | N=58 | NCT05135091 | Sponsor: Neurona Therapeutics | "EFFICACY: 92% median reduction in disabling seizures during 7-12-month efficacy endpoint period, and 80% (4/5) of patients responding with >75% seizure reduction in Cohort 1; DURABILITY: All responders (4/4) in Cohort 1 continue to have durable seizure control for 18-24 months after a single-dose of NRTX-1001; COGNITION AND QUALITY OF LIFE: All patients (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no patient experiencing persistent decline in cognition; SAFETY: NRTX-1001 continues to be well-tolerated at both high and low doses with no adverse events attributed to the cell therapy; NEXT STEPS: Phase 3 EPIC trial planned to start enrolling in 2H 2025, as aligned with FDA under RMAT designation, to support submission of a Biologics License Application (BLA)."

New P3 trial • P1/2 data • Epilepsy

MTLE: FIH Study of NRTX-1001 Neural Cell Therapy in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (clinicaltrials.gov) - P1/2 | N=58 | Recruiting | Sponsor: Neurona Therapeutics | N=40 ➔ 58 | Trial completion date: May 2026 ➔ Feb 2042 | Trial primary completion date: May 2025 ➔ Feb 2028

Enrollment change • Trial completion date • Trial primary completion date • CNS Disorders • Epilepsy

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy - Updated Clinical Trial Results (AAN 2025) - P1/2 | "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."

Clinical • P1 data • CNS Disorders • Epilepsy

Neurona Unveils Phase 3 EPIC Study for NRTX-1001 Cell Therapy in Epilepsy (GlobeNewswire) - "Neurona Therapeutics...announced plans to initiate the Phase 3 EPIC clinical trial for its lead product candidate, NRTX-1001. NRTX-1001 is the first investigational human cell therapy for drug-resistant epilepsy. The EPIC trial is anticipated to begin in the second half of 2025 and is designed as a randomized, sham-controlled, double-blind study of NRTX-1001 in people living with drug-resistant mesial temporal lobe epilepsy (MTLE), which is the most common type of epilepsy in adults."

New P3 trial • Epilepsy

A Phase 1/2 Study of NRTX-1001 Neuronal Cell Therapy in Drug-Resistant Bilateral Mesial Temporal Lobe Epilepsy (MTLE) (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Neurona Therapeutics | Not yet recruiting ➔ Recruiting | Phase classification: P1 ➔ P1/2

Enrollment open • Phase classification • CNS Disorders • Epilepsy

First-in-human Trial of NRTX-1001 Gabaergic Interneuron Cell Therapy for Drug-resistant Focal Epilepsy – Updated Results (AES 2024) - P1/2 | "A first-in-human study of NRTX-1001 GABAergic interneurons for focal epilepsy is underway, and the preliminary safety and efficacy results are encouraging. One-time administration of NRTX-1001 cell therapy offers the potential for seizure control in patients with drug-resistant MTLE without removal or ablation of brain tissue."

P1 data • CNS Disorders • Epilepsy

Effect of NRTX-1001, a Cellular Therapy Product, for the Treatment of Refractory Bilateral TLE (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Neurona Therapeutics | Initiation date: Jul 2024 ➔ Nov 2024

Trial initiation date • CNS Disorders • Epilepsy

Speaker (Neuroscience 2024) - "This session will consider interneuron (IN) transplantation as a novel, cell-based therapy for human epilepsy. A panel with expertise in development, regenerative medicine, and surgical epilepsy management will discuss 1) cortical IN development and function, 2) preclinical studies of IN transplantation in animal models, 3) the generation of a clinical-grade human IN cell product (NRTX-1001), and 4) preliminary insights from a first-in-human study of IN transplantation for focal epilepsy."

CNS Disorders • Epilepsy

Speaker (Neuroscience 2024) - "This session will consider interneuron (IN) transplantation as a novel, cell-based therapy for human epilepsy. A panel with expertise in development, regenerative medicine, and surgical epilepsy management will discuss 1) cortical IN development and function, 2) preclinical studies of IN transplantation in animal models, 3) the generation of a clinical-grade human IN cell product (NRTX-1001), and 4) preliminary insights from a first-in-human study of IN transplantation for focal epilepsy."

CNS Disorders • Epilepsy

Speaker (Neuroscience 2024) - "This session will consider interneuron (IN) transplantation as a novel, cell-based therapy for human epilepsy. A panel with expertise in development, regenerative medicine, and surgical epilepsy management will discuss 1) cortical IN development and function, 2) preclinical studies of IN transplantation in animal models, 3) the generation of a clinical-grade human IN cell product (NRTX-1001), and 4) preliminary insights from a first-in-human study of IN transplantation for focal epilepsy."

CNS Disorders • Epilepsy

Chair (Neuroscience 2024) - "This session will consider interneuron (IN) transplantation as a novel, cell-based therapy for human epilepsy. A panel with expertise in development, regenerative medicine, and surgical epilepsy management will discuss 1) cortical IN development and function, 2) preclinical studies of IN transplantation in animal models, 3) the generation of a clinical-grade human IN cell product (NRTX-1001), and 4) preliminary insights from a first-in-human study of IN transplantation for focal epilepsy."

CNS Disorders • Epilepsy

BTCR03. Cortical Interneurons: From the Developing Brain to the Operating Room — Derek G. Southwell (Neuroscience 2024) - "A panel with expertise in development, regenerative medicine, and surgical epilepsy management will discuss 1) cortical IN development and function, 2) preclinical studies of IN transplantation in animal models, 3) the generation of a clinical-grade human IN cell product (NRTX-1001), and 4) preliminary insights from a first-in-human study of IN transplantation for focal epilepsy...Learning Objective 2 Understand the key principles of in vitro MGE-type cortical interneuron derivation from pluripotent stem cells, and assess the validation and pre-clinical testing of an investigational human MGE-type interneuron cell product. . Learning Objective 3 Understand the clinical context, study design, and preliminary results of an ongoing first-in-human trial of MGE-type interneuron transplantation."

CNS Disorders • Epilepsy

Establishing assays to support the development of inhibitory cell therapy, NRTX-1001, for drug-resistant mesial temporal lobe epilepsy (MTLE) (Neuroscience 2024) - P1/2 | "Preliminary results suggest that MGE-pINs modulate burst frequency, network activity, and synchrony of glutamatergic cultures. These data support the ongoing clinical investigation of NRTX-1001 and will help to elucidate interneuron cell therapy mechanism of action."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Epilepsy

Neurona Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for NRTX-1001 in Focal Epilepsy (GlobeNewswire) - "Neurona Therapeutics...announced that the U.S Food and Drug Administration (FDA) has granted the company’s lead product candidate, NRTX-1001, the RMAT expedited program designation for drug-resistant mesial temporal lobe epilepsy (MTLE)....'In the near-term, we are focused on completing the ongoing Phase 1/2 study and engaging with the FDA to discuss our product development plan'....The ongoing multicenter Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant unilateral MTLE. The first stage of the trial is an open-label dose-escalation study in 10 subjects, recently expanded to up to 16 subjects..."

FDA event • Trial status • CNS Disorders • Epilepsy

Effect of NRTX-1001, a Cellular Therapy Product, for the Treatment of Refractory Bilateral TLE (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Neurona Therapeutics

New P1 trial • CNS Disorders • Epilepsy

Preclinical Characterization and Emerging Clinical Trial Results for NRTX-1001 - A Human Pluripotent Stem Cell-Derived Inhibitory Interneuron Cell Therapy for Drug-Resistant Chronic Focal Epilepsy (ASGCT 2024) - P1/2 | "NRTX-1001 is an investigational cellular therapeutic comprising GABAergic interneurons derived from human pluripotent stem cells, which is being evaluated for safety and efficacy in people with drug-resistant epilepsy. Preliminary data from the ongoing clinical trial (NCT05135091), as well as supporting preclinical data will be presented."

Preclinical • CNS Disorders • Cognitive Disorders • Epilepsy • Transplantation

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results (AAN 2024) - P1/2 | "NRTX-1001 transplantation was safely performed in 5 patients with drug-resistant TLE. Preliminary results at approximately 1 year (2 subjects) suggest NRTX-1001 transplantation may improve seizure control and memory dysfunction. In contrast to current surgical treatments (e.g., resection and laser ablation), NRTX-1001 could provide a non-destructive and functionally restorative approach to treating focal epilepsy."

Clinical • P1 data • CNS Disorders • Epilepsy • Transplantation

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results (AES 2023) - P1/2 | "This first-in-human study of NRTX-1001 GABAergic interneurons for focal epilepsy is underway, and preliminary results are encouraging. One-time implantation of NRTX-1001 cells offers the potential for seizure control in patients with MTLE without removal or ablation of brain tissue."

Clinical • P1 data • CNS Disorders • Epilepsy

NRTX-1001: Human inhibitory interneuron cell therapy suppresses seizures and reduces histopathology in a mouse model of chronic focal epilepsy with high repeatability across multiple studies and manufacturing lots (Neuroscience 2023) - P1/2 | "The results of these preclinical studies support the ongoing phase I/II clinical trial (NCT05135091) to evaluate NRTX-1001 in people with drug-resistant temporal lobe epilepsy. Funding: CIRM (TRAN1-11611; CLIN2-13355)"

Preclinical • CNS Disorders • Epilepsy

Transplanted human pluripotent stem cell-derived GABAergic neuronal precursors develop electrophysiological properties of MGE pallial-type interneurons in host mouse brains (Neuroscience 2023) - "Neurona Therapeutics has developed a clinical cell therapy candidate, NRTX-1001, comprising human pluripotent stem cell (hPSC)-derived post-mitotic GABAergic interneurons of a specific MGE pallial-type lineage...We demonstrate the progressive functional maturation of the grafted human cells and protracted acquisition of two divergent firing properties and waveforms, including fast-spiking and non-fast spiking MGE pallial-type interneurons. Our data highlight the remarkable capacity for transplanted human interneuron precursors to mature and functionally integrate into adult circuits and support further preclinical and clinical development of human GABAergic interneuron cell therapy for neurological disorders characterized by deficient inhibitory function."

Preclinical • Alzheimer's Disease • CNS Disorders • Epilepsy • Psychiatry • Schizophrenia

Neurona Therapeutics Presents One-Year Data on the First Patient Treated with NRTX-1001 Cell Therapy in an Ongoing Phase I/II Trial for Drug-resistant Focal Epilepsy (GlobeNewswire) - P1/2 | N=40 | NCT05135091 | Sponsor: Neurona Therapeutics | "Neurona Therapeutics...announced the presentation of new data from the open-label, single arm, starting dose-level of its ongoing Phase I/II clinical trial of a one-time dose of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE). The preliminary data demonstrate promising reduction (>90%) in seizure frequency in the first and second patients at one year and seven-months post-treatment, respectively."

P1/2 data • CNS Disorders • Epilepsy

AAN to Mark 75th Anniversary at 2023 Annual Meeting (AJMC) - "Spencer’s abstract...highlighted in the AAN Annual Meeting Top Science Press Conference held last week. The other abstracts were...from Marianna Spatola, MD, PhD, Ragon Institute of Massachusetts General Hospital, Massachusetts Institute of Technology, and Harvard Medical School, and...from George Ambalathingal, PhD, QIMR Berghofer Medical Research Institute, Queensland, Australia."

Clinical data • CNS Disorders • Epilepsy

First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results. (AAN 2023) - P1/2 | "This first-in-human study of NRTX-1001 GABAergic interneurons for focal epilepsy is underway, and preliminary results are encouraging."

Clinical • Late-breaking abstract • P1 data • CNS Disorders • Epilepsy

Neurona Therapeutics Announces FDA Clearance of Expanded Inclusion Criteria for Phase I/II Clinical Trial of NRTX-1001 Regenerative Cell Therapy in Patients with Dominant Hemisphere Drug-Resistant Focal Epilepsy (GlobeNewswire) - "Neurona Therapeutics...announced that the U.S. Food and Drug Administration (FDA) has cleared an amendment to Neurona’s ongoing Phase I/II clinical trial of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE) enabling expansion of enrollment to include adults who have dominant-lobe disease, as well as those whose disease is focused in the non-dominant lobe. In addition, the independent Data and Safety Monitoring Board (DSMB) overseeing the clinical trial completed a prespecified review of data from the first two patients and unanimously recommended progression of the trial to enroll the remaining patients in the first dose cohort."

Clinical protocol • DSMB • CNS Disorders • Epilepsy