December 9, 2024 - 🧬 [nGram] Today’s News Scoop: Neurona's NRTX-1001 & More Epilepsy Advances


  1. Neurona Therapeutics presents positive clinical update on NRTX-1001 cell therapy trial in drug-resistant epilepsy
    • NRTX-1001 cell therapy shows promising results in reducing seizures in drug-resistant epilepsy patients.
    • Low dose cohort achieved a 92% median reduction in seizures, with 80% of subjects reporting over 75% reduction.
    • High dose cohort showed a 78% median reduction in seizures, with 60% of subjects reporting over 75% reduction.
    • FDA granted RMAT designation; pivotal trial planned for the second half of 2025.
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  2. Rapport Therapeutics presents data on seizure biomarker association
    • A 30% reduction in long episodes correlates with a 50% or greater reduction in clinical seizures.
    • The ongoing Phase 2a trial for RAP-219 in focal epilepsy uses long episode frequency as the primary endpoint.
    • Additional studies support RAP-219's potential as a transformative treatment for focal epilepsy.
    • Topline data from the Phase 2a trial is expected in mid-2025.
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  3. Stoke Therapeutics presents new open-label extension study data for zorevunersen in Dravet syndrome
    • Stoke Therapeutics shared data from Phase 1/2a and open-label extension studies of zorevunersen, showing significant seizure reduction in Dravet syndrome patients.
    • Patients treated with zorevunersen experienced an 87% median reduction in convulsive seizures at month eight of the OLE study.
    • Continuous improvements in cognition and behavior were observed over two years of treatment.
    • Zorevunersen was generally well-tolerated, with most adverse events being unrelated to the study drug.
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  4. SK Life Science presents data on XCOPRI at AES 2024
    • SK Life Science showcased data on XCOPRI (cenobamate tablets) at the AES 2024 Annual Meeting.
    • The data provided insights into cenobamate's dual mechanism of action, enhancing GABAergic inhibition and reducing neuronal excitability.
    • A study showed significant reduction in epileptiform events and clinically reported seizures with cenobamate treatment.
    • The potential for flexible and individualized dosing was highlighted, aiding in optimal seizure reduction.
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