NRTX-1001

–EFFICACY: 92% median reduction in disabling seizures during 7-12-month efficacy endpoint period, and 80% (4/5) of patients responding with >75% seizure reduction in Cohort 1. –DURABILITY: All responders (4/4) in Cohort 1 continue to have durable seizure control for 18-24 months after a single-dose of NRTX-1001. –COGNITION AND QUALITY OF LIFE: All patients (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no patient experiencing persistent decline in cognition. – SAFETY: NRTX-1001 continues to be well-tolerated at both high and low doses with no adverse events attributed to the cell therapy. –NEXT STEPS: Phase 3 EPIC trial planned to start enrolling in 2H 2025, as aligned with FDA under RMAT designation, to support submission of a Biologics License Application (BLA). April 08, 2025 -- Neurona Therapeutics, a clinical-stage biotherapeutics company developing regenerative cell therapies for epilepsy and other disorders of the nervous system, today presented updated data from its open-label Phase 1/2 clinical trial of NRTX-1001 in adults with drug-resistant, unilateral mesial temporal lobe epilepsy (MTLE) at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, CA. The ongoing multicenter Phase 1/2 trial for unilateral MTLE has enrolled 18 subjects in Cohorts 1 and 2 across 13 comprehensive epilepsy centers in the United States, and 10 subjects have been followed for 9 to 24 months after NRTX-1001 administration. Patients in the low-dose Cohort 1 (N=5) achieved median disabling seizure reduction of 92% from baseline during the primary efficacy evaluation period in months 7-12 post-treatment, and a 97% median reduction of disabling seizures after month 13. In the high dose Cohort 2 (N=5), patients achieved median disabling seizure reduction of 72% from baseline in months 4-9, the most recent 6-month epoch, and have not yet reached month 12. “We are pleased with the results to-date from the ongoing trial of NRTX-1001 cell therapy,” said Dr. Jerry Shih, M.D., Neurologist and Director of the University of California San Diego (UCSD) Comprehensive Epilepsy Center, who has treated 4 subjects in the trial alongside UCSD Neurosurgeon and Surgical Director of Epilepsy, Dr. Sharona Ben-Haim, M.D. “Our first subject in the study is now 19 months post-cell administration and has experienced a 98% reduction in his drug-resistant disabling seizures. He successfully stopped the temporary immunosuppression regimen 6 months ago, and continues to have a durable outcome, free from any study-related adverse effects. If these results continue to be observed in the upcoming Phase 3 EPIC trial, cell therapy has the potential to become a paradigm-shifting treatment option for patients with drug-refractory focal epilepsy.” Said Dr. John Hixson, M.D., VP of Clinical Development at Neurona, “The results of the trial thus far point to the promise of NRTX-1001 being a cell therapy that can address a major unmet need for patients with drug-resistant focal seizures, providing durable seizure control and avoiding the significant risk of permanent neurocognitive impairment from current tissue-destructive surgeries like laser ablation and brain resection. To that end, NRTX-1001 could represent a new treatment option that preserves brain tissue and function.” Dr. Hixson continued, “We are pleased with the continued durability signal from the ongoing NRTX-1001 study, supporting a single ‘one-and-done’ dose. We are also very encouraged by the lack of cell-related adverse effects and absence of neurocognitive impairment thus far. In summary, the data give us confidence to proceed with our planned Phase 3 EPIC trial of NRTX-1001, as we seek to bring this novel therapy to patients as safely and expeditiously as possible.” Results: Low Dose Effect on Disabling Seizures (N=5): Months 7-12: 92% median seizure reduction Months 13+: 97% median seizure reduction Durability: All (4/4) patients who exhibited marked seizure reduction in first year continue to have durable seizure control in second year after completing the 12-month immunosuppression regimen, which is intended to facilitate long-term persistence of the allogeneic cell therapy Quality-of-Life: All patients have significantly improved Quality-Of-Life In Epilepsy (QOLIE) overall test scores from baseline levels High Dose Effect on Disabling Seizures (N=5): Months 4-9 (latest 6-month epoch to date): 72% median seizure reduction 80% (4/5) subjects with >50% seizure reduction from baseline Safety – Low and High Dose Cohorts (N=10): No adverse events determined to be related to NRTX-1001 Neurocognition – Low and High Dose Cohorts (N=10): No persistent decline in cognition based on word retrieval, verbal memory, and visuospatial memory batteries The open-label Phase 1/2 clinical trial was 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. This ongoing trial has been expanded to include 8 additional subjects for a total of 18 subjects across both cohorts: low-dose cohort 1 (N=9) and high-dose cohort 2 (N=9). Study subjects are being monitored for safety, tolerability, and effects on their epilepsy disease symptoms. 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 February, the company announced plans to initiate its Phase 3 EPIC (EPIlepsy Cell therapy) trial, which is expected to be a single pivotal study and primary basis of a future BLA submission for NRTX-1001 in drug-resistant MTLE, based on alignment gained with FDA under the RMAT designation. The EPIC trial is anticipated to begin in the second half of 2025 and is designed as a randomized, sham-controlled, double-blind study of NRTX-1001 in adults with drug-resistant MTLE, the most common type of epilepsy in adults. Earlier this month, Neurona closed an upsized and oversubscribed $102 million private financing with support from new and existing investors. AAN Presentation Details: Title: First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy - Updated Clinical Trial Results Presenting Author: John D. Hixson, M.D., VP of Clinical Development, Neurona Presentation: Tuesday April 8, 2025; Poster Session 8 (8:00 AM-9:00 AM); Number: 9-003 Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapies 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 interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials: NCT05135091 for drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), and NCT06422923 for drug-resistant bilateral MTLE, with expansion to neocortical focal epilepsy and other indications planned in the future. The Phase 1/2 MTLE clinical trials are supported by grants from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355 and CLIN2-17135). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024. The content above comes from the network. if any infringement, please contact us to modify.

▎药明康德内容团队编辑据创鉴汇不完全统计，上周（3月31日至4月6日）全球大健康领域共披露融资事件14起，总额超96亿元。按照金额划分，亿元及以上融资7起。按照已披露的融资轮次划分，早期融资（B轮以前）6起，中后期融资（B轮及以后）4起。生物医药领域共5家公司获融资，涉及药物类型包括基因疗法、细胞疗法、蛋白降解疗法等。#01Isomorphic Labs获得6亿美元融资关键词：AI药物最新融资：6亿美元A轮本轮投资机构：Alphabet、谷歌风投（GV）、Thrive Capital3月31日，人工智能药物设计和开发公司Isomorphic Labs宣布融资6亿美元。Isomorphic Labs旨在利用AI的力量重新构想和加速药物发现，为全球患者带来急需的治疗方法。该公司旨在应用其开创性的AI药物设计引擎来实现生物医学突破，并正在推进多个治疗领域和药物模式的药物设计计划。自成立以来，该公司开发了许多下一代AI模型，这些模型共同构成了其统一的AI药物设计引擎，适用于多个治疗领域和药物模式。其中一个突破性模型的例子是AlphaFold 3，它由Isomorphic Labs与Google DeepMind于 2024 年 5 月共同开发并发布，能够准确预测生命体的分子结构和相互作用。#02AIRNA完成1.55亿美元B轮融资关键词：RNA编辑疗法最新融资：1.55亿美元B轮本轮投资机构：ARCH Venture Partners、Forbion、ND Capital、Nextech Invest、RTW Investments等4月1日，AIRNA宣布完成超额认购的1.55亿美元B轮融资。该公司将利用本轮融资获得的资金推动其针对α1抗胰蛋白酶缺乏症（AATD）的首个RNA编辑候选药物AIR-001进入1/2期临床试验，并开发针对心血管代谢及其他疾病的一系列创新RNA编辑药物。AIRNA的独有平台利用天然机制以安全灵活的药物高效编辑目标RNA。精准的RNA编辑旨在通过修复有害基因变异或引入有益变异来实现对严重或慢性疾病的功能性治愈。新闻稿指出，该公司的主打项目有望成为治疗α1抗胰蛋白酶缺乏症的潜在“best-in-class”疗法。#03Atsena获得1.5亿美元C轮超额认购融资关键词：基因疗法最新融资：1.5亿美元C轮本轮投资机构：阿宾沃思、贝恩资本、Foundation Fighting Blindness、Hatteras Venture Partners、惠灵顿管理等4月2日，Atsena Therapeutics是一家临床阶段的基因治疗公司，专注于利用基因医学改变生活的力量来逆转或预防失明，宣布成功完成1.5亿美元的超额认购C轮融资。融资收益将用于推进Atsena的主导项目ATSN-201，用于治疗X连锁视网膜裂骨症 （XLRS），这是一种通常在儿童时期被诊断出来并在以后的生活中导致失明的遗传病。所得款项还将支持Atsena的潜在“first-in-class”临床前管线，扩大Atsena新型腺病毒载体的使用。#04Neurona Therapeutics获1.02亿美元融资关键词：细胞疗法最新融资：1.02亿美元本轮投资机构：Alexandria Venture Investments、富达、The Column Group4月4日，Neurona Therapeutics宣布筹集1.02亿美元，将其癫痫细胞疗法推向3期临床试验。这家加州生物技术公司将利用这笔资金来支持其针对慢性神经系统疾病的同种异体细胞疗法候选药物管线。其中，NRTX-1001目前正在耐药性内侧颞叶癫痫（MTLE）患者中进行1/2期试验。#05Prazer完成290亿韩元B轮融资关键词：蛋白降解疗法最新融资：290亿韩元B轮本轮投资机构：Company K Partners、K2 Investment、Kiwoom Investment、Mirae Asset Capital、Premier Partners等4月1日，Prazer Therapeutics宣布已完成290亿韩元的B轮投资，获得资金将用于新药研发和全球商业化。Prazer Therapeutics致力于开发基于靶向蛋白质降解（TPD）技术的新药。该公司表示，现有的TPD技术使用有限的E3泛素连接酶来降解蛋白质，而Prazer专有的SPiDEM平台可以使用各种E3连接酶实现更广泛的蛋白质靶向。该公司预计，这有望克服现有PROTAC和分子胶技术的局限性，并可针对以前难以靶向的靶点，例如蛋白质聚集体和膜蛋白。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐点击“在看”，分享创鉴汇健康新动态