Soticlestat

癫痫治疗在过去几十年取得了显著进步，但仍有约 30% 的患者属于药物难治性癫痫。因此，全球制药企业和科研机构仍在持续投入大量资源，开发新的抗癫痫治疗策略。 未来几年，癫痫药物研发正在出现几个明显趋势： 下面介绍未来五年最值得关注的 10个癫痫药物研发项目。 第一梯队： 新型离子通道调节剂（解决耐药性） 1. Azetukalner (XEN1101) — Xenon Pharmaceuticals 机制： Kv7 钾通道开启剂。 看点： 它是钾通道赛道的领头羊。目前的 III 期数据显示其对难治性局灶性癫痫具有极强的控制力,且每日只需服药一次,无需滴定。 预期： 极有可能成为未来 5 年内首个获批的新机制抗癫痫药。 2. BHV-7000 — Biohaven 机制： 高选择性 Kv7.2/7.3 调节剂。 看点： 与 XEN1101 类似,但它在设计上更专注于减少“中枢神经系统副作用”（如嗜睡、头晕）。如果临床数据证实其安全性更高,它将具有极强的市场竞争力。 第二梯队： 精准医疗与遗传性癫痫（靶向基因） 3. STK-001 — Stoke Therapeutics 机制： 反义寡核苷酸（ASO）疗法。 看点： 针对 Dravet 综合征。它不是单纯控制抽搐,而是通过增加 SCN1A 基因产生的健康蛋白质水平,从底层修复基因缺陷。 预期： 开启“基因补偿”疗法的新时代。 4. ETX101 — Encoded Therapeutics 机制： AAV 基因疗法。 看点： 同样针对 Dravet 综合征。这是一种“一劳永逸”的尝试,通过病毒载体将调节序列送入特定神经元。目前正处于早期临床,是全球基因治疗癫痫的风向标。 第三梯队： 神经炎症与免疫调节（寻找新靶点） 5. Darigabat — Cerevel Therapeutics (已被 AbbVie 收购) 机制：$\alpha 2/3/5$ 选择性 GABA_A 受体正向别构调节剂。 看点： 传统的安定类药物作用于所有 GABA 受体,导致严重的镇静副作用。Darigabat 避开了引起镇静的 $\alpha 1$ 亚型,试图实现“只抗癫痫、不催眠”的目标。 6. Atuzaginstat — Quince Therapeutics 机制： 蛋白酶抑制剂（针对神经炎症）。 看点： 该药原本研究阿尔茨海默病,但新证据发现其能通过抑制脑部特定细菌代谢物产生的炎症来控制癫痫,是典型的“新靶点”尝试。 第四梯队： 孤儿药与罕见综合征 7. Soticlestat (TAK-935) — Takeda (武田) 机制： 胆固醇 24-羟化酶 (CH24H) 抑制剂。 看点： 通过调节脑内谷氨酸水平来减少兴奋性。虽然近期在 LGS 的某些终点上表现反复,但在 Dravet 综合征中的潜力依然巨大,是目前唯一的酶抑制剂路径药物。 8. LP352 — Longboard Pharmaceuticals 机制： 5-HT2C 受体超选择性激动剂。 看点： 针对多种发育性癫痫性脑病（DEE）。它旨在复制芬氟拉明的疗效,同时去除心脏瓣膜损伤的风险。第五梯队：脑机接口与再生医学（终极方案） 9. NRTX-1001 — Neurona Therapeutics 机制： 抑制性中间神经元细胞疗法。 看点： 这是一个革命性的方案——直接向致痫灶移植产生的 GABA 神经元,让它们在那里扎根并抑制异常放电。 阶段： I/II 期临床中,部分受试者已实现长期无发作。 10. 智能植入式闭环系统（硬件+软件） — NeuroPace/Medtronic 机制： AI 驱动的响应式神经电刺激（RNS）。 看点： 虽然不是药物,但其算法更新速度极快。未来 5 年,集成深度学习算法的植入设备将能提前数分钟预测发作并精准放电阻断,这被称为“电子药物”。 总结：未来 5 年的三个关键词 脱离“钠通道”： 研发重心已全面转向钾通道和 GABA 亚型受体。 认知挽救： 评价一个好药的标准不再仅仅是“发作减少了多少”,而是“孩子的认知好转了多少”。 个性化： 基因检测将成为癫痫治疗的前置步骤,医生将根据基因报告在上述项目中选择最匹配的武器。 这些项目代表了从“改善症状”到“精准对因”甚至“疾病修饰”的范式转变。

Ovid Therapeutics Inc. (Nasdaq: OVID), a New York-based biopharmaceutical company developing small molecule therapies for neurological disorders, announced a USD 60 million private placement to fund the expansion of its lead epilepsy candidate, OV329, into two additional indications: tuberous sclerosis complex and infantile spasms. The PIPE financing, expected to close on or about March 19, 2026, follows an earlierprivate placement initiated in October 2025 to raise up to USD 175 million, reflecting the company’s ongoing capital requirements as it broadens OV329’s clinical development program across multiple developmental and epileptic encephalopathies. Under the terms of the securities purchase agreement, Ovid is selling 19,154,321 shares of common stock at USD 2.01 per share alongside pre-funded warrants to purchase up to 10,701,710 additional shares at a purchase price of USD 2.009 per pre-funded warrant. The pre-funded warrants carry an exercise price of USD 0.001 per share and are immediately exercisable. Point72 led the financing, with participation from existing investors Adage Capital Management, ADAR1 Capital Management, Affinity Asset Advisors, Ally Bridge Group, Balyasny Asset Management, Coastlands Capital, Eventide Asset Management, Janus Henderson Investors, and RA Capital Management. Leerink Partners served as lead placement agent, with Oppenheimer & Co. and LifeSci Capital acting as co-placement agents. The press release did not indicate participation by company insiders. Company overview and pipeline Ovid Therapeutics is a New York-based biopharmaceutical company focused on developing targeted small molecule candidates that modulate factors involved in neuronal hyperexcitability across multiple neurological and neuropsychiatric disorders. The company’s lead asset, OV329, is a next-generation GABA-aminotransferase inhibitor under development for treatment-resistant focal onset seizures and developmental and epileptic encephalopathies, including tuberous sclerosis complex (TSC) and infantile spasms (IS). The proceeds from this financing will support the expansion of OV329 into those additional indications. In a separate release, Ovid states it is planning to initiate a TSC-associated seizures study in Q4 2026, and an IS study in 2027. Beyond OV329, Ovid is developing OV4071 and a library of related compounds that directly activate the KCC2 transporter, targeting multiple central nervous system disorders. The KCC2 program originated from an exclusive license agreement with AstraZeneca completed in January 2022, under which Ovid paid USD 5 million in cash and USD 7.5 million in common stock, with AstraZeneca eligible for development and commercial milestones and royalties. Development of OV4071 is initially focused on psychosis associated with Parkinson’s disease and Lewy body dementia. Ovid’s earlier pipeline assets have largely been divested or out-licensed as the company has narrowed its focus toward epilepsy. In June 2025, Ovid sold its worldwide ganaxolone royalty rights and licensing to Immedica Pharma AB for USD 7.0 million. The company’s former collaboration with Takeda on soticlestat (TAK-935/OV935) for Dravet syndrome and Lennox-Gastaut syndrome was restructured in 2021 in a deal valued at up to approximately USD 856 million in milestones and royalties, with Takeda assuming Ovid’s 50% global share. Previously completed clinical trials include a Phase III study of OV101 (gaboxadol) in Angelman syndrome and a Phase II open-label extension of TAK-935 in rare epilepsies. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website