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Neurona Therapeutics’ Progress in Phase I/II Clinical Trial of NRTX-1001 Cell Therapy for Drug-resistant Focal Ep

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Neurona Therapeutics, a clinical-stage biotherapeutics company, recently announced the completion of enrollment for the first cohort in its ongoing Phase I/II clinical trial of NRTX-1001.

This investigational allogeneic cell therapy is being developed to treat drug-resistant mesial temporal lobe epilepsy (MTLE).

The first cohort consisted of five subjects who received a one-time dose of NRTX-1001, accompanied by a year of immunosuppression to encourage the integration and persistence of the therapy.

Key highlights from the trial include:

  1. Positive Outcomes in Initial Subjects: The first patient to receive NRTX-1001 experienced a stable seizure reduction of over 95% in the second year after treatment. Similarly, the second patient reported a 95% reduction in seizures a year post-treatment and has been seizure-free for the past six months.
  2. Promising Early Data: Cory R. Nicholas, Ph.D., CEO of Neurona, expressed optimism about the early results, noting the potential of NRTX-1001 to offer significant, long-lasting seizure reduction for patients with drug-resistant MTLE.
  3. Anticipated Updates: David Blum, M.D., Neurona’s Chief Medical Officer, highlighted the encouraging results from the first two subjects and mentioned that more updates on these patients, as well as data from the remaining participants in the first cohort, are expected in the fourth quarter of 2023.
  4. Background on Participants: The first two subjects in the trial had a history of significant monthly seizures that were not controlled by medication. Both reported a reduction of over 95% in overall seizure counts 16 months and one year after receiving NRTX-1001.
  5. About NRTX-1001: This regenerative neural cell therapy is derived from human pluripotent stem cells. The fully differentiated neural cells, known as interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). When administered as a one-time dose, these human interneurons aim to integrate into neural circuits and effectively silence seizure activity in the epileptic region of the brain.
  6. The Need for New Treatments: MTLE is a prevalent type of focal epilepsy in adults. Despite the availability of approved drugs, a significant number of patients continue to experience seizures. Current surgical options, which involve removing or ablating the damaged temporal lobe, are not suitable or effective for all patients and can have considerable side effects.

Neurona Therapeutics is dedicated to developing regenerative cell therapy candidates with the potential for a single-dose cure. The company’s investigational therapies are designed to provide long-term repair of dysfunctional neural networks for various neurological disorders.

For more details, visit Neurona Therapeutics.

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