排名前五的靶点	数量

KCa4.1(potassium sodium-activated channel subfamily T member 1)	3
Nav1.2(II型钠通道蛋白α亚基)	2
Sodium channels(钠通道)	1
T-type calcium channel(电压门控性T型钙通道家族)	1
VGSCs(电压门控钠离子通道)	1

关联

项与 Praxis Precision Medicines, Inc. 相关的药物

Elsunersen

靶点

Nav1.2

作用机制

Nav1.2阻滞剂

在研机构

Praxis Precision Medicines, Inc.

原研机构

Praxis Precision Medicines, Inc.

在研适应症

癫痫性脑病 [+2]

非在研适应症-

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期-

Ulixacaltamide

靶点

T-type calcium channel

作用机制

T-type calcium channel阻滞剂

在研机构

Praxis Precision Medicines, Inc. [+1]

原研机构

Praxis Precision Medicines, Inc.

在研适应症

特发性震颤 [+1]

非在研适应症-

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期-

Relutrigine

靶点

Sodium channels

作用机制

钠通道阻滞剂

在研机构

Praxis Precision Medicines, Inc.

原研机构

Praxis Precision Medicines, Inc.

在研适应症

婴儿癫痫性运动障碍性脑病 [+2]

非在研适应症-

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期-

项与 Praxis Precision Medicines, Inc. 相关的临床试验

CTIS2025-521640-38-00

/ Recruiting临床3期

Open Label Extension Clinical Trial of Vormatrigine in Adult Patients with Epilepsy.

开始日期2025-10-16

申办/合作机构

Praxis Precision Medicines, Inc.

NCT07019922

/ Recruiting临床3期

A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy

A Randomized, Multi-Center, Double-Blind, Sham-Procedure-Controlled Clinical Trial to Investigate the Efficacy and Safety of Elsunersen in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy

开始日期2025-08-13

申办/合作机构

Praxis Precision Medicines, Inc.

NCT07010471

/ Recruiting临床3期

A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants With DEE Followed by an Open-Label Extension

开始日期2025-07-09

申办/合作机构

Praxis Precision Medicines, Inc.

100 项与 Praxis Precision Medicines, Inc. 相关的临床结果

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0 项与 Praxis Precision Medicines, Inc. 相关的专利（医药）

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项与 Praxis Precision Medicines, Inc. 相关的文献（医药）

2025-11-01·JAMA Neurology

Five-Year Outcomes from Deep Brain Stimulation of the Subthalamic Nucleus for Parkinson Disease

Article

作者: Duker, Andrew P. ; Metman, Leo Verhagen ; Luca, Corneliu C. ; Phibbs, Fenna T. ; Cooper, Scott E ; Jagid, Jonathan R. ; Pahwa, Rajesh ; Revuelta, Gonzalo J. ; Galifianakis, Nicholas ; Tatter, Stephen B. ; Gostkowski, Michal ; Goldberg, Edward ; Takacs, Istvan ; Leichliter, Timothy A. ; Pourfar, Michael H. ; Tagliati, Michele ; Karl, Jessica A. ; San Luciano, Marta ; House, Paul A. ; Okun, Michael S. ; Machado, Andre G. ; Neimat, Joseph ; Khandhar, Suketu M. ; Hebb, Adam O. ; Durphy, Jennifer ; Pilitsis, Julie G. ; Mogilner, Alon Y. ; Mandybur, George T. ; Foote, Kelly D. ; Zadikoff, Cindy ; Ponce, Francisco A. ; Starr, Philip A. ; Haq, Ihtsham ul ; Rosenow, Joshua M. ; Farris, Sierra M ; Whiting, Donald M. ; Tröster, Alexander I. ; Lyons, Kelly ; Schrock, Lauren E ; Ramirez-Zamora, Adolfo ; Shivacharan, Rajat S. ; Park, Michael C. ; Mamelak, Adam N. ; Vitek, Jerrold L. ; Ostrem, Jill L. ; Wharen, Robert ; Moguel-Cobos, Guillermo ; Gross, Robert E. ; Sedrak, Mark ; Nazzarro, Jules M. ; Siddiqui, Mustafa S. ; Uitti, Ryan J. ; Sani, Sepehr ; Buetefisch, Cathrin M. ; Giroux, Monique L.

Importance:

The Implantable Neurostimulator for the Treatment of Parkinson’s Disease (INTREPID) trial was a randomized, double-blind, sham-controlled study of subthalamic nucleus (STN) deep brain stimulation (DBS) for the treatment of Parkinson disease (PD).

Objective:

To evaluate the long-term (5-year) outcomes and safety of STN-DBS for PD.

Design, Setting, and Participants:

This was a prospective, randomized (3:1), 12-week double-blind sham-controlled study at 23 movement disorder centers across the US with an open-label 5-year follow-up. Patients were implanted and followed up with the Vercise DBS system from May 2013 to December 2022. Eligibility required diagnosis of bilateral idiopathic PD with more than 5 years of motor symptoms, more than 6 hours per day of poor motor function, modified Hoehn and Yahr Scale scores higher than 2, Unified Parkinson’s Disease Rating Scale (UPDRS-III) score of 30 or higher (medication-off state), and 33% or higher improvement in UPDRS-III medication-on score.

Intervention:

Bilateral STN-DBS for moderate to advanced PD.

Main Outcomes and Measures:

Primary outcomes included changes in UPDRS and dyskinesia scores, quality-of-life measures, and safety assessments. Exploratory analyses included medication reduction and DBS association with motor signs.

Results:

A total of 313 patients were enrolled with 191 receiving the DBS system, and 137 participants (72%) completed the study. The study population had a mean (SD) age of 60 (7.9) years, with 139 (73%) male participants. Motor function without medication as measured by UPDRS-III improved from a mean (SD) of 42.8 (9.4) to 21.1 (10.6) at year 1 (51%; 95% CI, 49%-53%; P &lt; .001) and 27.6 (11.6) at year 5 (36%; 95% CI, 33%-38%; P &lt; .001). Activities of daily living without medication as measured by UPDRS-II improved from a mean (SD) of 20.6 (6.0) to 12.4 (6.1) at year 1 (41%; 95% CI, 38%-42%; P &lt; .001) and 16.4 (6.5) at year 5 (22%; 95% CI, 18%-23%; P &lt; .001). Dyskinesia scores decreased from 4.0 (5.1) to 1.0 (2.1) at year 1 (75%; 95% CI, 73%-75%; P &lt; .001) and to 1.2 (2.1) at year 5 (70%; 95% CI, 63%-75%; P &lt; .001). The levodopa equivalent dose was reduced by 28% at year 1, remaining stable at year 5 (28%; 95% CI, 26%-31%; P &lt; .001). The most common serious adverse event was infection (9 participants). Ten deaths were reported, none related to the study.

Conclusions and Relevance:

Although STN-DBS outcomes declined slightly, possibly due to the progressive nature of the disease, patients with PD sustained significant improvement in motor and activities of daily living scores, along with a stable reduction in anti-parkinsonian medication over the 5-year follow-up period.

2025-01-07·BRAIN

Nav1.2 channel mutations preventing fast inactivation lead to SCN2A encephalopathy

Article

作者: Howell, Katherine B ; Tao, Elaine ; Corry, Ben ; Andersen, Erik ; Berecki, Géza ; Petrou, Steven ; Kahlig, Kris ; Wolff, Markus ; Coorg, Rohini K

Abstract:

SCN2A gene-related early-infantile developmental and epileptic encephalopathy (EI-DEE) is a rare and severe disorder that manifests in early infancy. SCN2A mutations affecting the fast inactivation gating mechanism can result in altered voltage dependence and incomplete inactivation of the encoded neuronal Nav1.2 channel and lead to abnormal neuronal excitability.In this study, we evaluated clinical data of seven missense Nav1.2 variants associated with DEE and performed molecular dynamics simulations, patch-clamp electrophysiology and dynamic clamp real-time neuronal modelling to elucidate the molecular and neuron-scale phenotypic consequences of the mutations.The N1662D mutation almost completely prevented fast inactivation without affecting activation. The comparison of wild-type and N1662D channel structures suggested that the ambifunctional hydrogen bond formation between residues N1662 and Q1494 is essential for fast inactivation. Fast inactivation could also be prevented with engineered Q1494A or Q1494L Nav1.2 channel variants, whereas Q1494E or Q149K variants resulted in incomplete inactivation and persistent current. Molecular dynamics simulations revealed a reduced affinity of the hydrophobic IFM-motif to its receptor site with N1662D and Q1494L variants relative to wild-type. These results demonstrate that the interactions between N1662 and Q1494 underpin the stability and the orientation of the inactivation gate and are essential for the development of fast inactivation. Six DEE-associated Nav1.2 variants, with mutations mapped to channel segments known to be implicated in fast inactivation were also evaluated. Remarkably, the L1657P variant also prevented fast inactivation and produced biophysical characteristics that were similar to those of N1662D, whereas the M1501V, M1501T, F1651C, P1658S and A1659V variants resulted in biophysical properties that were consistent with gain-of-function and enhanced action potential firing of hybrid neurons in dynamic action potential clamp experiments. Paradoxically, low densities of N1662D or L1657P currents potentiated action potential firing, whereas increased densities resulted in sustained depolarization.Our results provide novel structural insights into the molecular mechanism of Nav1.2 channel fast inactivation and inform treatment strategies for SCN2A-related EI-DEE. The contribution of non-inactivating Nav1.2 channels to neuronal excitability may constitute a distinct cellular mechanism in the pathogenesis of SCN2A-related DEE.

2023-01-01·Frontiers in neurology

SCN1A channelopathies: Navigating from genotype to neural circuit dysfunction.

Review

作者: Petrou, Steven ; Bryson, Alexander

The SCN1A gene is strongly associated with epilepsy and plays a central role for supporting cortical excitation-inhibition balance through the expression of Na_V1.1 within inhibitory interneurons. The phenotype of SCN1A disorders has been conceptualized as driven primarily by impaired interneuron function that predisposes to disinhibition and cortical hyperexcitability. However, recent studies have identified SCN1A gain-of-function variants associated with epilepsy, and the presence of cellular and synaptic changes in mouse models that point toward homeostatic adaptations and complex network remodeling. These findings highlight the need to understand microcircuit-scale dysfunction in SCN1A disorders to contextualize genetic and cellular disease mechanisms. Targeting the restoration of microcircuit properties may be a fruitful strategy for the development of novel therapies.

178

项与 Praxis Precision Medicines, Inc. 相关的新闻（医药）

2025-12-06

·知乎专栏

2025年12月6日全球新药进展早知道

→，专注技术创新的Agent平台为以下生物医药提供更专业详细的研发数据和报告支持!来自智慧芽Eureka Agent平台药物研发进展1. 百时美施贵宝CD19 CAR-T在美获批新适应症，治疗边缘区淋巴瘤12月4日，百时美施贵宝（BMS）宣布，美国食品药品监督管理局（FDA）已批准其CD19 CAR-T细胞疗法，用于治疗既往至少接受过两种系统性治疗的复发或难治性（R/R）边缘区淋巴瘤（MZL）成年患者，成为首个也是唯一一个获批用于该患者群体的CAR-T细胞疗法。Breyanzi的此项批准是基于一项名为TRANSCEND FL的开放标签、多中心、多队列、单臂研究中MZL队列的结果。在纳入主要疗效分析集（n=66）且接受三线及以上治疗的Breyanzi患者中，总体缓解率为95.5%（95% CI：87.3-99.1）。完全缓解率为62.1%（95% CI：49.3-73.8）。中位缓解持续时间尚未达到（95% CI：25.59-NR），90.1%的缓解者在24个月时仍保持缓解。2. 君实生物IL-17A单抗申报上市12月5日，君实生物发布公告，宣布其收到国家药品监督管理局核准签发的《受理通知书》，用于治疗适合系统治疗或光疗的中度至重度斑块状银屑病的成人患者的新药上市申请获得受理。本次新药上市申请主要基于一项多中心、随机、双盲、平行、安慰剂对照的关键注册性III期临床研究（研究编号：JS005-005-III-PsO），研究结果显示，偌考奇拜单抗治疗12周显著改善了银屑病面积与严重程度指数（PASI）75/90/100 和静态医师全面评估（sPGA）评分0或1，疗效显著优于安慰剂并在52周治疗期间维持稳定，且总体安全性良好。除本次上市申请适应症外，偌考奇拜单抗用于治疗活动性强直性脊柱炎的II期临床研究的所有参与者已完成治疗，进入安全随访期。不过，该靶点领域竞争激烈，此前已有5款靶向IL-17A的单抗在中国获批上市。3. 治疗痤疮，歌礼1类新药申报上市12 月 4 日，CDE 官网显示，歌礼的 1 类新药上市申请获得受理。根据该药的临床研究进度，推测本次申请的适应症为用于治疗中重度寻常性痤疮。地尼法司他 (Denifanstat，ASC40) 是一款潜在 First-in-class、每日一次口服脂肪酸合成酶 (FASN) 抑制剂。该药最初由 Sagimet Biosciences 开发，歌礼拥有其在大中华区的独家权益。地尼法司他已在治疗中重度寻常性痤疮的 III 期临床试验（NCT06192264）中达到所有主要、关键次要及次要终点。这是一项在中国开展的随机、双盲、安慰剂对照、多中心的临床试验，旨在评估地尼法司他每日一次口服片在 480 例中重度寻常性痤疮患者中的安全性和疗效。4. 潜在First-in-Class小分子注册临床结果积极，即将申报上市12月5日，Praxis Precision Medicines宣布，其在研小分子药物在EMBOLD研究注册队列中取得积极结果。该研究评估relutrigine用于治疗携带SCN2A和SCN8A基因变异的发育性和癫痫性脑病（DEEs）患者。基于积极的疗效结果，独立数据监查委员会已建议提前因疗效原因终止试验。公司已确认将在未来数周内与美国FDA召开会议，审阅相关数据并讨论后续监管路径，Praxis将在会议后就relutrigine的新药申请（NDA）提交时间作出最终决定。Relutrigine是一款潜在“first-in-class”在研小分子药物。其作用机制为优先抑制持续性钠电流，而这一电流已被证实是严重DEEs患者癫痫发作症状的重要驱动因素。5. 多肽疗法3期积极结果登《新英格兰医学杂志》12月4日，Sobi宣布，《新英格兰医学杂志》发布了3期VALIANT研究的积极结果。该研究评估其与Apellis Pharmaceuticals联合开发的用于治疗C3肾小球病（C3G）及原发性免疫复合物膜增生性肾小球肾炎（IC-MPGN）的疗效。Pegcetacoplan是一款靶向C3补体蛋白的聚乙二醇化（PEGylated）双环肽疗法。研究显示，pegcetacoplan在多个关键疾病指标上均带来积极且具有临床意义的获益，包括使蛋白尿水平下降68%、帮助维持肾功能稳定，并显著清除肾脏中的C3补体沉积。目前，pegcetacoplan治疗这两类肾病的监管申请已由欧洲药品管理局（EMA）以及其他监管机构受理。此前于2021年5月，pegcetacoplan获FDA批准用于治疗阵发性睡眠性血红蛋白尿症（PNH），商品名Empaveli。6. 和黄医药第三代BTK抑制剂启动首个Ⅲ期临床12 月 5 日，药物临床试验登记与信息公示平台官网显示，和黄医药启动了一项国内Ⅲ期临床，以评估联合 R-GemOx 治疗复发/难治弥漫大 B 细胞淋巴瘤（DLBCL）患者的疗效和安全性。HMPL-760 是一种新型、非共价的第三代 BTK 抑制剂。临床前研究显示，作为一种选择性和可逆性的 BTK 抑制剂，HMPL-760 可同时靶向野生型 BTK 和 C481S 突变型 BTK，旨在克服患者对初代 BTK 抑制剂的耐药性。和黄医药本次登记的是一项随机、双盲、阳性对照国内 III 期研究（CTR20254776），旨在评估 HMPL-760 联合 R-GemOx 对比安慰剂联合 R-GemOx 在复发/难治 DLBCL 患者的疗效、安全性、药代动力学。7. 中国生物重磅产品获批新适应症12 月 4 日，中国生物宣布，药监局正式批准中国生物兰州生物技术开发有限公司研发的注射用新增原发性腋窝多汗症适应症。该产品成为国内首个获批用于治疗该适应症的 A 型肉毒毒素，是继暂时性改善眉间纹和成人脑卒中后上肢（腕部和手部）痉挛适应症获批后又一新突破。A 型肉毒毒素可以暂时性抑制乙酰胆碱的释放，抑制外分泌汗腺过度分泌汗液。研究表明，当多汗症患者用局部止汗剂无效且注射肉毒毒素后，汗液减少明显。在临床实践中，用药后 7-10 天可见症状明显减轻，疗效持续 4-25 个月，患者满意度高。行业资讯1. 翰思艾泰通过港交所聆讯，即将上市12月3日，港交所IPO申请通过聆讯，即将上市。翰思艾泰成立于2014年，2016年起开始搭建研发管线，核心管线为一款PD-1/SIRPα双抗，目前处于二期临床阶段。后续管线还有CTLA-4/SIRPα双抗、OX40双表位双抗、TRBV12单抗、NKG2A单抗、PD-1/VEGF双抗、PD-L1/VEGF双抗等。翰思艾泰成立以来经历多轮融资，2024年8月完成B+轮融资，投后估值16.15亿元。本文内容由智慧芽Eureka——赋能研发技术创新的AI Agent 技术平台提供支持。找技术方案、找研发灵感、关注技术专利保护，统统交给→！小tips：Eureka这个词源自古希腊语，是阿基米德发现浮力原理时的感叹，意思是“我找到了”或者“我发现了”。希望每个研发用户都能在Eureka平台获得技术灵感并解决难题~智慧芽Eureka平台指路→

2025-12-05

·药明康德

产业新闻 | FDA批准CAR-T疗法扩展适应症；潜在“first-in-class”小分子注册性研究结果积极

FDA批准CAR-T疗法扩展适应症美国FDA近日批准百时美施贵宝（Bristol Myers Squibb）旗下Breyanzi（lisocabtagene maraleucel）扩展适应症，用于治疗在接受至少两线或以上既往治疗后复发或难治的成年边缘区淋巴瘤（MZL）患者。根据FDA新闻稿，Breyanzi为首个获批用于MZL治疗的CAR-T细胞疗法。此次批准主要基于一项开放标签、多中心、单臂临床试验的结果。入组的复发或难治性MZL成年患者均接受了至少两线系统治疗，或在接受造血干细胞移植（HSCT）治疗后复发。研究中，首先采集患者自体免疫细胞用于制备Breyanzi，随后在通过预处理化疗清除患者的淋巴细胞2至7天内接受单次Breyanzi输注。在77例完成细胞采集的患者中，共有66例按方案接受单次Breyanzi输注，其中95.5%的患者对治疗产生应答，62.1%的患者在影像学检查中达到完全缓解，未见边缘区淋巴瘤病灶。在中位随访21.6个月时，这些疗效仍具有良好的持久性。 Breyanzi是一种靶向CD19抗原的自体CAR-T细胞疗法，具有明确的组成和4-1BB共刺激域。这款疗法的独特之处在于精准控制CAR-T疗法中CD8阳性和CD4阳性T细胞的比例，从而可以更好地控制细胞疗法的毒副作用。4-1BB信号结构域则增强了CAR-T细胞的扩增和持久性。潜在“first-in-class”小分子注册性研究结果积极，有望递交上市申请 Praxis Precision Medicines近日宣布，其在研小分子药物relutrigine在EMBOLD研究注册队列中取得积极结果。该研究评估relutrigine用于治疗携带SCN2A和SCN8A基因变异的发育性和癫痫性脑病（DEEs）患者。基于积极的疗效结果，独立数据监查委员会已建议提前因疗效原因终止试验。公司已确认将在未来数周内与美国FDA召开会议，审阅相关数据并讨论后续监管路径，Praxis将在会议后就relutrigine的新药申请（NDA）提交时间作出最终决定。 Relutrigine是一款潜在“first-in-class”在研小分子药物。其作用机制为优先抑制持续性钠电流，而这一电流已被证实是严重DEEs患者癫痫发作症状的重要驱动因素。通过精确调控钠离子通道（NaV）活性，relutrigine有望在疾病状态下对NaV通道的过度兴奋性实现更高选择性调节，从而潜在为这类重症癫痫患者提供更具针对性的治疗选择。参考资料： [1] Praxis Precision Medicines Announces Positive Results from EMBOLD Study for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies. Retrieved December 5, 2025 from https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-positive-results-embold [2] FDA Approves First CAR T-Cell Therapy for Marginal Zone Lymphoma In the US. Retrieved December 5, 2025 from https://www.fda.gov/news-events/press-announcements/fda-approves-first-car-t-cell-therapy-marginal-zone-lymphoma-us 免责声明：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：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载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法免疫疗法上市批准ASH会议

2025-12-05

·FierceBiotech

Praxis claims efficacy success in phase 2 seizure trial, setting up approval talks with FDA

Praxis Precision Medicines said the FDA has agreed to a meeting “in the coming weeks … to review the data and discuss next steps.”\n Having already claimed a win for the phase 2 study of its seizure drug, Praxis Precision Medicines is hoping fresh efficacy data will form the backbone of an approval application for the sodium channel inhibitor.Last year, the Boston-based biotech revealed that the Embold study of the drug, called relutrigine, had hit its primary endpoint of safety. The trial enrolled 16 individuals ages 2 to 18 with early-onset SCN2A-DEE or SCN8A-DEE, forms of epilepsy for which there are no approved treatments.Praxis has continued with the study, alongside discussions with the FDA about the possibility of using its efficacy results as the basis for an approval application that had been penciled in for early 2026.Against this backdrop, the company announced yesterday that it has ended the Embold study early on the recommendation of the trial’s data monitoring committee after it was clear relutrigine’s efficacy had been demonstrated.The company’s stock jumped 30% in premarket trading Friday to $247 per share from a Thursday closing price of $189.97.“SCN2A and SCN8A DEEs are devastating conditions with extremely high mortality due to the debilitating seizure burden they impose on patients, and there are currently no approved treatment options,” Praxis’ CEO Marcio Souza said in the Dec. 4 post-market release.“Our progress represents an important milestone towards delivering the first therapy ever designed for these children and their families,” added Souza, who said the company will share the results at the American Epilepsy Society Annual Meeting tomorrow. In the same release, Praxis said the FDA has agreed to a meeting “in the coming weeks … to review the data and discuss next steps.”“Praxis will make a determination on the timing for filing an NDA for relutrigine after the meeting,” the biotech added.When Praxis rolled out the top-line results in September 2024, the company explained in a presentation that SCN2A and SCN8A are among \"the most severe and refractory forms\" of developmental and epileptic encephalopathies (DEEs).There are roughly 5,000 patients in the U.S. with the SCN2A/SCN8A DEEs, representing a market opportunity of about $500 million, the company said at the time.Praxis launched another trial of relutrigine in patients across all DEEs this year. The hope is that data from the study—which is due to complete in the second half of 2026—would form the basis of a supplemental new drug application. In the September presentation, the company said the peak market opportunity for all developmental epilepsies with a high seizure burden could surpass $2 billion.Meanwhile, the company scored another midphase epilepsy win this year when its next-generation NaV blocker vormatrigine was linked to a 100% complete response rate in epilepsy patients with photoparoxysmal response, a form of photosensitivity.

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药物(靶点)	适应症	全球最高研发状态

Ulixacaltamide ( T-type calcium channel )	特发性震颤更多	临床3期
Relutrigine ( Sodium channels )	SCN8A突变癫痫性脑病更多	临床2/3期
Vormatrigine ( VGSCs )	特发全身性癫痫更多	临床2期
Elsunersen ( Nav1.2 )	性早熟更多	临床1/2期
SYNGAP1	癫痫更多	临床前