Neurona Therapeutics Presents Positive Clinical Update: New Data from NRTX-1001 Cell Therapy Trial in Drug-resistant Epilepsy Announced at 2024 Annual Meeting of the American Epilepsy Society

– SAFETY: NRTX-1001 has been well-tolerated to date at both high and low doses. No adverse events have been attributed to the cell therapy

– LOW DOSE COHORT: 92% median reduction in disabling seizures from baseline, with 80% of subjects reporting >75% seizure reduction at the primary efficacy evaluation period of 7-12 months post administration (n=5) 

– HIGH DOSE COHORT: 78% median reduction in disabling seizures from baseline, with 60% of subjects reporting >75% seizure reduction at the interim 4-6-month period post administration (n=5)

– DURABILITY: The first 2 subjects who received low dose cell therapy continue to report >97% seizure reduction two years after a single administration of NRTX-1001

– COGNITION AND QUALITY OF LIFE: No clinically significant impairments detected in either dose cohort with significantly increased test scores for some subjects

– NEXT STEPS: FDA granted RMAT designation in 2024; pivotal trial planned for 2H 2025
 

San Francisco, CA, December 6, 2024 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for epilepsy and other disorders of the nervous system, today announced positive clinical data from the first (low dose) and second (high dose) cohorts in its ongoing multicenter, open-label Phase 1/2 clinical trial of NRTX-1001, an investigational allogeneic inhibitory neuron cell therapy candidate that is being developed for treatment of drug-resistant mesial temporal lobe epilepsy (MTLE). The data will be presented at the Annual Meeting of the American Epilepsy Society (AES), which is being held December 6-10, 2024, in Los Angeles, California.

“We’re delighted to present an update on our Phase 1/2 trial of NRTX-1001, which includes the first announcement of interim data from our high dose cohort, and an update on the durability of effect in our low dose cohort,” said Cory R. Nicholas, Ph.D., Neurona’s Co-Founder and Chief Executive Officer. “Preliminary efficacy data from both dose cohorts show reductions in seizure frequency post NRTX-1001 administration. This is in line with what we saw preclinically, where both the high and low dose levels performed similarly.”  

Dr. John Hixson, Neurona Senior Medical Director and UCSF Neurologist added, “As the NRTX-1001 low dose cohort data mature, we’re excited to report that four of five subjects had >75% disabling seizure reduction from baseline in months 7-12, which is the primary efficacy evaluation period. We’re also happy to report further durability data on the first two subjects who are now ~2 years post-NRTX-1001 administration and ~1 year after completion of immunosuppression. One of these subjects has been completely seizure-free for ~16 months and the other has been free from impaired-awareness seizures for ~2 years.”

“To date, the results of this trial indicate NRTX-1001 cell therapy could serve as a non-destructive treatment approach for drug-resistant temporal lobe epilepsy. Traditionally, surgeons have treated this condition with tissue-destructive methods, such as brain resection and laser ablation, that can adversely impact neurological functions. Cell therapy may offer a differentiated therapeutic profile that includes the preservation of brain tissue and function,” said Dr. Derek Southwell, Assistant Professor of Neurosurgery at Duke University, and investigator in the study. “NRTX-1001 is delivered via a minimally invasive procedure that could be integrated into practice settings as a frontline treatment option for drug-resistant focal epilepsy.”

Results:

Low Dose Effect on Disabling Seizures:

• Primary Efficacy Endpoint: 7-12 Months Post-Administration (n=5)
  • 92% median seizure reduction from baseline
  • 80% of subjects with >50% seizure reduction
  • 80% of subjects with >75% seizure reduction
  • 60% of subjects with >90% seizure reduction
• Long-Term Durability: 13-24 Months Post-Administration (n=2)
  • >97% seizure reduction from baseline was maintained after completion of immunosuppression regimen

High Dose Effect on Disabling Seizures:

• Interim Efficacy Endpoint: 4-6 Months Post-Administration (n=5)
  • 78% median seizure reduction from baseline
  • 100% of subjects with >50% seizure reduction
  • 60% of subjects with >75% seizure reduction
  • 0% subjects with >90% seizure reduction

Safety – Low and High Dose Cohorts:

• No adverse events related to the cell therapy to date
• Mild to moderate (non-serious) adverse events related to the procedure and temporary immunosuppression regimen
  • Adverse procedural-related events have been transient
  • Adverse immunosuppression-related events have resolved in the two subjects who completed immunosuppression per protocol
• Two subjects had serious adverse events of status epilepticus, one subject from each dose cohort, which were determined to be related to their underlying disease by the principal investigators, as both subjects experienced similar episodes before enrolling in the trial

Cognition and Quality of Life – Low and High Dose Cohorts:

• No clinically significant decline
• Statistically significant numerical increases from baseline for some subjects

Neurona Therapeutics’ multicenter, open-label Phase 1/2 clinical trial is designed to evaluate the safety and preliminary efficacy of a single-dose administration of NRTX-1001 inhibitory neuron cell therapy for drug-resistant unilateral MTLE. The ongoing trial has been expanded to include eight additional subjects and will enroll up to 18 subjects across two dose cohorts: nine subjects treated at a starting dose (Cohort 1) and nine at a higher dose (Cohort 2). This study focuses on MTLE patients with mesial temporal sclerosis (MTS) identified by MRI. Study subjects are being monitored for safety, tolerability, and effects on their epilepsy disease symptoms. A third cohort of up to 10 additional subjects will evaluate NRTX-1001 in adults with drug-resistant MTLE without MTS. In addition, a second multicenter, open-label Phase 1/2 clinical trial has been initiated to evaluate NRTX-1001 in adults with drug-resistant bilateral MTLE. For more information, please visit www.clinicaltrials.gov (NCT05135091 and NCT06422923). 

In June, the company announced that the U.S Food and Drug Administration (FDA) had granted NRTX-1001 the RMAT expedited program designation for drug-resistant MTLE. A pivotal trial is planned in 2025, pending alignment with the FDA.

AES Presentation Details:

Session: SIG | Translational Research: Fast Forward to Epilepsy Therapies in 2027
Title: Cortical Interneurons: From the Developing Brain to the Operating Room
Presenter: Dr. Derek Southwell, Assistant Professor of Neurosurgery, Duke University School of Medicine
Session: 408 - Level 2 - LACC
Date: Fri 12/6/2024
Presentation Time: 6:00 PM - 7:30 PM (PT)

Title: First-in-human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Drug-resistant Focal Epilepsy – Updated Results
Presenter: Dr. John Hixson, Senior Medical Director, Neurona Therapeutics; Professor of Neurology, UCSF
Poster Session #: 2.244
Date: Sunday December 8, 2024
Presentation Time: 12:00 PM - 2:00 PM (PT)

About Neurona Therapeutics

Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with the potential to provide long-term targeted repair of the nervous system following a single administration. The Company’s lead product candidate NRTX-1001, comprising GABAergic inhibitory interneurons, is currently being studied for safety and efficacy in two ongoing open-label multicenter Phase 1/2 trials (NCT05135091) for drug-resistant unilateral MTLE and (NCT06422923) for drug-resistant bilateral MTLE, with neocortical focal epilepsy and other indications planned in the future. The Phase 1 of the unilateral MTLE clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024. In February 2024, Neurona raised $120 million in a private financing co-led by Viking Global Investors and Cormorant Asset Management. For more information about Neurona, visit: www.neuronatherapeutics.com.
 

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