Grant Award Details

Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Grant Type: 
Grant Number: 
CLIN2-13355
Project Objective: 
  • The objectives of this project are 1) to conduct and complete a Phase 1, first in human study of NRTX-1 in subjects with drug-resistant mesial-temporal lobe epilepsy (MTLE), 2) to demonstrate disease-modifying activity of the GMP grade clinical lots in a preclinical model, and 3) carry out activities in preparation for the next, Phase 2 study. NRTX-1 is a cellular product consisting of human embryonic stem cell (ESC)-derived inhibitory GABA-ergic interneurons.
Investigator: 
Name: 
Institution: 
Type: 
PI
Disease Focus: 
Epilepsy
Neurological Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
Award Value: 
$7,999,999
Status: 
Active

Grant Application Details

Application Title: 
  • Phase 1/2 Study of NRTX-1001 Neural Cell Therapy in Subjects with Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Public Abstract: 

Therapeutic Candidate or Device

NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells.

Indication

Focal epilepsy; drug-resistant mesial temporal lobe epilepsy.

Therapeutic Mechanism

NRTX-1001 is an inhibitory neuron cell therapy derived from human embryonic stem cells (hESCs) for the prospective treatment of drug-resistant focal epilepsy (MTLE). NRTX-1001 is delivered as a single dose by intracranial injection into the seizure focus, and is intended to distribute locally, functionally integrate, and release the inhibitory neurotransmitter GABA. NRTX-1001 is expected to persist long-term following a single dose and not require repeated administration.

Unmet Medical Need

Current treatments for drug-resistant MTLE include surgical resection and ablation; both are tissue-destructive and can cause serious, irreversible adverse effects. There is a clear need to develop targeted, non-tissue-destructive, and long-lasting therapies that are safe and effective for MTLE.

Project Objective

Initiate/conduct Phase 1; Plan for Phase 2

Major Proposed Activities

  • Initiate and conduct FIH Study of NRTX-1001 in subjects with drug-resistant MTLE (NCT05135091). Includes preclinical and technical support.
  • Clinical operations supporting planning of Stage 2. Includes preclinical and technical support.
Statement of Benefit to California: 

Epilepsy affects more than 3 million people in the United States; one-third of epilepsy patients are drug-resistant. Transplantation of inhibitory neuron cell therapy is a novel therapeutic strategy that has shown promise in preclinical studies and could potentially provide a non-tissue-destructive therapeutic option for suppressing seizures in people with drug-resistant focal epilepsy. California medical institutions will participate in the NRTX-1001 clinical trial.

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