Neurona Therapeutics Announces Positive NRTX-1001 Trial Results for Drug-Resistant Epilepsy at 2024 AES Meeting

Neurona Therapeutics, a biotherapeutics firm specializing in regenerative cell therapies for epilepsy and other neurological disorders, has reported promising results from its ongoing Phase 1/2 clinical trial of NRTX-1001. This investigational cell therapy aims to treat drug-resistant mesial temporal lobe epilepsy (MTLE). The findings, which will be presented at the American Epilepsy Society (AES) Annual Meeting in Los Angeles, cover both low and high dose cohorts of the therapy.

The company’s Co-Founder and CEO, Cory R. Nicholas, Ph.D., expressed enthusiasm about the trial's progress, highlighting the preliminary efficacy data that show a decrease in seizure frequency following NRTX-1001 administration. Dr. John Hixson, Neurona’s Senior Medical Director and a neurologist at UCSF, noted significant seizure reductions in the low dose cohort, with four out of five subjects experiencing more than a 75% reduction in disabling seizures from baseline during the primary efficacy evaluation period of 7-12 months post-administration. Additionally, the first two subjects treated in the low dose cohort have maintained over 97% seizure reduction after two years, one of whom has been completely seizure-free for approximately 16 months.

Dr. Derek Southwell, Assistant Professor of Neurosurgery at Duke University, emphasized that NRTX-1001 could offer a non-destructive treatment alternative for drug-resistant temporal lobe epilepsy, traditionally managed by more invasive and tissue-destructive methods like brain resection and laser ablation. He pointed out that the cell therapy is administered via a minimally invasive procedure, potentially making it a frontline treatment for drug-resistant focal epilepsy.

The data revealed that the low dose cohort experienced a median seizure reduction of 92% from baseline, with 80% of subjects reporting more than a 75% reduction in seizures between 7-12 months post-therapy. Long-term results showed that in the two initial subjects, seizure reduction exceeding 97% persisted 13-24 months after the single administration of NRTX-1001. In the high dose cohort, there was a 78% median reduction in seizures from baseline, with 60% of participants seeing more than a 75% decrease at the interim evaluation period of 4-6 months post-therapy.

Safety assessments indicated that NRTX-1001 has been well-tolerated in both dose cohorts, with no adverse events attributed directly to the cell therapy. Mild to moderate adverse events related to the procedure and temporary immunosuppression were noted, but these were transient and resolved over time. Serious adverse events, including status epilepticus in two subjects, were deemed related to their underlying disease rather than the cell therapy.

Regarding cognitive function and quality of life, the trial reported no significant impairments, with some subjects even showing improvements in their test scores.

The Phase 1/2 trial aims to evaluate the safety and efficacy of NRTX-1001 in patients with drug-resistant unilateral MTLE, expanding to include subjects with mesial temporal sclerosis (MTS) as identified by MRI. A third cohort will assess the therapy in adults with drug-resistant MTLE without MTS, and another trial is planned for bilateral MTLE. The study's goal is to enroll up to 18 subjects across the two dose cohorts, with an additional cohort planned to include up to 10 more subjects.

In June 2024, the FDA granted NRTX-1001 the Regenerative Medicine Advanced Therapy (RMAT) designation, with a pivotal trial anticipated to start in 2025.

Neurona Therapeutics is focused on developing off-the-shelf, regenerative neural cell therapy products capable of providing targeted and long-lasting repair of the nervous system. Their lead product, NRTX-1001, composed of GABAergic inhibitory interneurons, is being tested in Phase 1/2 trials for both unilateral and bilateral drug-resistant MTLE. The phase 1 trial for unilateral MTLE is supported by an $8 million grant from the California Institute for Regenerative Medicine.