Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in pediatric patients from 6 months to less than 2 years old with genetically confirmed CDD during a 12-week, DB phase. Pharmacokinetic (PK) assessments and population PK analyses will also be performed during this time. The DB phase will be followed by an optional long-term OL phase at which time all participants will receive GNX as an adjunct to their standard anti-seizure medication. Efficacy, safety and tolerability, and PK assessments will continue to be performed.

开始日期2024-12-01

申办/合作机构

Marinus Pharmaceuticals, Inc.

NCT05814523

/ Withdrawn临床3期

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus

This is a multicenter, double-blind, randomized, placebo-controlled study that will evaluate the efficacy, safety, and tolerability of intravenous (IV) ganaxolone versus placebo co-administered with IV antiepileptic drug (AED) according to standard of care for the treatment of RSE. Approximately 70 participants will be randomized in a 1:1 ratio to receive ganaxolone IV solution or placebo IV solution along with standard of care (SOC) IV AED.

开始日期2024-03-01

申办/合作机构

Marinus Pharmaceuticals, Inc.

CTR20233334

/ Recruiting临床3期

一项在结节性硬化症（TSC）相关癫痫儿童和成人患者中评价Ganaxolone（GNX）添加治疗的开放标签III期研究（TrustTSC OLE）

主要目的：评估GNX作为儿童和成人TSC相关癫痫发作的添加治疗的长期安全性和耐受性。

开始日期2023-12-05

申办/合作机构

Altasciences Co., Inc.

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100 项与加那索龙相关的转化医学

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100 项与加那索龙相关的专利（医药）

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259

项与加那索龙相关的文献（医药）

2025-05-01·NEUROPHARMACOLOGY

Effect of two novel GABAA receptor positive allosteric modulators on neuropathic and inflammatory pain in mice

Article

作者: Wu, Jian-Hong ; Li, Jun ; Chun-Ge ; Zou, Jian-Jun ; Xu, Chu ; Yu, Hui ; Liu, Yun-Xin

Loss of GABAergic inhibition in the spinal dorsal horn (SDH) is implicated in central sensitization and chronic pain. Both agonists and positive allosteric modulators (PAMs) of GABAA receptor are found to be effective in the management of chronic pain. In addition to benzodiazepines, neuroactive steroids (NASs) also act as PAMs through binding to unique sites of GABAA receptors. Thus, it is worth investigating whether these NASs can attenuate chronic pain. This study tested the antinociceptive properties of two novel NAS PAMs, ganaxolone and zuranolone, in segmental spinal nerve ligation (SNL)-induced neuropathic pain and complete Freund's adjuvant (CFA)-induced inflammation pain models. Spinally administered ganaxolone and zuranolone both exhibited dose-dependent analgesic effects but with quite different durations. This antinociceptive effect might be generated from elevated GABAergic inhibition, as the PAMs both enhanced GABA-evoked currents in SDH neurons, and the K⁺-Cl^- cotransporter isoform 2 (KCC2) antagonist reversed the analgesic effect of the PAMs. Different from ganaxolone, zuranolone produced a durable increase in the surface expression of GABAA receptors and of the amplitude of spontaneous inhibitory currents, which may contribute to the long-lasting analgesic effect. Furthermore, the PAMs alleviated SNL-induced mechanical allodynia synergistically with diazepam or GABAA receptor activator muscimol at inactive doses, consistent with the non-competitive activity and distinct binding sites from benzodiazepines. In summary, our findings suggest that NASs may not only acutely modulate GABA receptor activity but also induce sustained metabotropic effects on GABAA receptors and thus exert long-lasting antinociceptive effects.

2025-02-01·Continuum (Minneapolis, Minn.)

Update on Antiseizure Medications 2025

Review

作者: Abou-Khalil, Bassel W.

ABSTRACT:

OBJECTIVE:

This article is an update from the article on antiseizure medication therapy published in the three previous Continuum issues on epilepsy and is intended to cover the vast majority of agents currently available to neurologists in the management of patients with epilepsy. This article addresses antiseizure medications individually, focusing on key pharmacokinetic characteristics, indications, and modes of use.

LATEST DEVELOPMENTS:

Since the most recent version of this article was published, one new antiseizure medication, ganaxolone, has been approved by the US Food and Drug Administration (FDA), and the indications of some approved medications were expanded. Older antiseizure medications are effective but have tolerability and pharmacokinetic disadvantages. Several newer antiseizure medications have undergone comparative trials demonstrating efficacy equal to and tolerability at least equal to or better than older antiseizure medications as first-line therapy for focal epilepsy. These agents include lamotrigine, oxcarbazepine, levetiracetam, topiramate, zonisamide, and lacosamide. Pregabalin was found to be less effective than lamotrigine. Lacosamide, pregabalin, and eslicarbazepine have undergone successful trials of conversion to monotherapy for focal epilepsy. Other newer antiseizure medications with a variety of mechanisms of action are suitable for adjunctive therapy.

ESSENTIAL POINTS:

Knowledge of antiseizure medication pharmacokinetics, efficacy, and tolerability profiles facilitates the choice of appropriate antiseizure medication therapy for patients with epilepsy. Rational antiseizure medication combinations should avoid antiseizure medications with unfavorable pharmacokinetic interactions or pharmacodynamic interactions related to mechanism of action.

2025-01-24·Current Neuropharmacology

Antiseizure Medications: Advancements, Challenges, and Prospects in Drug Development

Article

作者: Ahmed, Qamar Uddin ; Feroz, Shevin Rizal ; Lam, Su Datt ; Sarian, Murni Nazira ; Ng, Yan Hong ; Latip, Jalifah ; Jamil, Siti Nur Hidayah

Abstract::

Epilepsy is a neurological disorder affecting millions of people worldwide. Antiseizure medications (ASM) remain a critical therapeutic intervention for treating epilepsy, notwithstanding the rapid development of other therapies. There have been substantial advances in epilepsy medications over the past three decades, with over 20 ASMs now available commercially. Here we describe the conventional and unique mechanisms of action of ASMs, focusing on everolimus, cannabidiol, cenobamate, fenfluramine, and ganaxolone, the five most recently marketed ASMs. Major obstacles in the development of ASMs are also addressed, particularly drug-resistant epilepsy as well as psychiatric and behavioral adverse effects of ASMs. Moreover, we delve into the mechanisms and comparative efficacy of ASM polytherapy, with remarks on the benefits and challenges in their application in clinical practice. In addition, the characteristics of the ideal ASM are outlined in this review. The review also discusses the development of new potential ASMs, including modifying existing ASMs to improve efficacy and tolerability. Furthermore, we expound on the modulation of γ- aminobutyric acid type A receptor (GABAAR) as a strategy for the treatment of epilepsy and the identification of a GABAAR agonist, isoguvacine, as a potential ASM.

175

项与加那索龙相关的新闻（医药）

2025-02-19

·化学经纬

2022年FDA获批新药合成方法

聚焦新药早研早聊学以致用侃侃而言 DOI: 10.1021/acs.jmedchem.3c02374 2022年，共有23个新的小分子化学实体（NCEs）获得美国FDA、欧盟EMA、日本PMDA、和中国NMPA批准为药物。 2024年3月15日，辉瑞联合默沙东、武田制药在J. Med. Chem.上发文，分析汇总了基于专利、和主要文献的以上新药大规模合成方法。 01 2022年获批小分子新药总览 2022年，共有23个新的小分子化学实体获批为药物，除此之外，本文还包括了于2021、2020年获批的11种药物，在此前的系列年度综述中没有提及，在此一同汇总。因此，本文共汇总了34种药物的大规模合成方法，以及它们的治疗应用和作用机制。其中，从治疗领域的角度来看：有5种药物用于抗感染，包括抗病毒、抗细菌、抗真菌；有4种药物用于心血管和血液领域；有4种药物用于中枢神经系统疾病；有3种药物用于免疫疾病；有2种代谢药物；有15种肿瘤领域药物（包括诊断和治疗药物）；有1种呼吸及肺部药物。具体药物结构如下图： 02 抗感染/抗生素药物 2.1、Azvudine (1) Azvudine(1)是中国真实生物技术公司生产的抗病毒药物。它于2021年在中国获得批准，成为治疗人类免疫缺陷病毒（HIV）的首创新药。它具有双重功能，作为抗转录酶抑制剂，靶向病毒感染因子/载脂蛋白B mRNA编辑酶，催化亚单位3G(Vif/A3G）蛋白-蛋白相互作用。 2022年，该药物在中国被重新批准，用于治疗Covid-19。其作为RNA依赖性RNA聚合酶（RdRp）抑制剂，机制与先前批准的莫硝帕韦和瑞德西韦相同。除抗病毒活性外，胸腺内的药物浓度表明其具有免疫靶向活性；这种双重功能在RdRp抑制剂中独一无二。其百克级合成路线如下： 2.2、Lenacapavir (Sunlenca) Lenacapavir（2）于2022年首次在欧盟获得批准，与其他抗逆转录病毒药物联合使用，用于患有多药耐药HIV感染的成年人，否则无法构建抑制性抗病毒方案。该药后来也在加拿大和美国获得了类似适应症的批准。作为首个已知能抑制HIV-1衣壳的获批药物，获得首创药物认定。 HIV-1衣壳是一种包含病毒遗传物质的蛋白壳，被认为与HIV生命周期的多个阶段有关。该药物可口服、可注射治疗，作为HIV-1低释放、长效治疗，当与抗逆转录病毒药物联合使用时，可能需要每6个月给药一次，目的是提高患者的依从性和药物依从性。其大批量合成路线涉及多个关键中间体的合成，路线如下： 2.3、Ensitrelvir Fumaric Acid (Xocova) 富马酸Ensitrelvir(3)，也被称为S-217622，是由Shionogi公司开发的非共价口服SARS-CoV-2主蛋白酶（Mpro）抑制剂。该药物已被日本PMDA批准用于治疗由SARS-CoV-2（COVID-19）感染引起的疾病。该药物对许多最常见的Mpro突变体保持一定的抑制活性，并对多种流行的SARS-CoV-2变种表现出抗病毒活性。Ensitrelvir是第二款被批准用于治疗COVID-19引起的疾病的Mpro抑制剂。与首个批准的治疗药物Paxlovid不同，Ensitrelvir不需要与CYP3A4抑制剂一起给药来减弱体内代谢此外，Ensitrelvir与其他三种人类感染冠状病毒（MERS-CoV， SARS-CoV和HCoV-NL63）的主要蛋白酶复合物的晶体结构表明该药物具有共价相互作用。由Shionogi报道的公斤级合成路线如下： 2.4、Contezolid (Youxitai) Contezolid(4)，也被称为MRX-I，是由上海盟科药业开发的口服给药恶唑烷酮抗菌剂。Contezolid克服了结构相似的利奈唑胺的单胺氧化酶（MAO）抑制限制的局限性。该药物用于治疗多重耐药革兰氏阳性细菌感染引起的复杂皮肤和软组织感染，包括甲氧西林敏感金黄色葡萄球菌（MSSA）、耐甲氧西林金黄色葡萄球菌（MRSA）、化脓性链球菌、无乳链球菌和耐万古霉素肠球菌。其合成路线如下： 2.5、Oteseconazole (Vivjoa) Oteseconazole(5)获得美国FDA批准，用于治疗有外阴阴道念珠菌病病史，且没有生育潜力的女性复发性外阴阴道念珠菌病。其他侵袭性和机会性感染以及甲真菌病的进一步研究正在进行中。该药物的抗真菌活性是由于抑制甾醇14-α去甲基化酶。这种细胞色素P450（CYP）酶51（CYP51）参与麦角甾醇的生物合成，麦角甾醇是一种通过维持真菌细胞膜完整性来满足真菌生长所需的成分。它在引起外阴阴道念珠菌病的多种念珠菌之间具有活性。在结构上，由于使用了四唑基团作为金属结合基，所以其他四唑与母唑衍生的抗真菌药是独特的。与含有咪唑或三唑甲基结合基团的抗真菌药相比，四唑的金属亲和力较低，可以通过减少与人CYP酶血红素铁的相互作用来提高选择性。其低于克级的合成路线如下： 03 心血管/血液病药物 3.1、Hetrombopag Olamine (Hengqu) Hetrombopag olamine(6)是由江苏恒瑞制药公司开发的非肽类血小板生成素受体（TpoR）激动剂，2021年在中国获批，用于治疗慢性原发性免疫性血小板减少症（ITP）和严重再生贫血的成人患者，这些患者对治疗反应不佳。该药物与其他TpoR激动剂一样，通过与TpoR起源细胞的跨膜结构域结合，诱导巨核细胞，从而增加血小板的产生。其公斤级规模合成方法如下： 3.2、Tolvaptan sodium phosphate (Samtasu) Tolvaptansodium diumphosphate(7)，也称为Samtasu和OPC-61815，是一种选择性血管抑制V-2受体的小分子拮抗剂，由大冢制药公司开发。该药物是通用托伐伐坦（7.13）的前药，改善了水溶性。该药物于2022年在日本获批，用于口服托伐他坦有困难的患者。该药物抑制肾小管的水重吸收，从而在不消耗电解质的情况下增强水利尿。它有效地治疗心力衰竭患者，并已显示出临床益处，如体重减轻、呼吸困难和其他体征和症状的减少。托伐普坦对早期急性心力衰竭也有效，静脉注射托伐普坦可在早期迅速引起心衰。其百克级合成方法如下： 3.3、Mavacamten (Camzyos) 马伐卡坦(8)是β-心肌蛋白的小分子调节剂，可减轻肥厚性心肌病（HCM）的过度收缩。肥厚性心肌病是由肌粒蛋白突变引起的单基因心血管疾病，特别是肌球蛋白，可能高达每200人中就有1人被发现。马伐卡坦通过两种方式影响心肌蛋白：减少肌球蛋白-肌球蛋白过桥形成和抑制心肌肌球蛋白ATP酶。两者都通过调节肌球蛋白功能导致收缩性降低。该药物是由百时美施贵宝的子公司Myokardia开发的，被美国FDA批准用于治疗有症状的纽约心脏协会（NYHA） ii-iii级阻塞性肥厚性心肌病的成人。其克级合成路线如下图： 3.4、Mitapivat (Pyrukynd) Mitapivat(9)是由Agios Pharmaceutics开发，在美国和欧盟被批准用于治疗溶血性贫血和丙酮酸激酶缺乏症（PKD），这是一种罕见的与慢性溶血性贫血相关的遗传性疾病。以往PDK的临床治疗仅限于与短期和长期风险相关的支持性治疗，包括红细胞输注和脾切除术。该药物是一种首创的红细胞丙酮酸激酶激活剂，在未接受常规红细胞输注的患者中已被证明可增加血红蛋白水平。除了被批准用于治疗PKD外，目前还在临床研究中用于治疗镰状细胞病和地中海贫血。其收敛、高产率合成路线如下图： 04 中枢神经系统疾病药物 4.1、Ganaxolone (Ztalmy) 加那洛酮（10），首创新药，是一种神经活性甾体γ -氨基丁酸（GABA）受体阳性调节剂，用于治疗与周期蛋白依赖性激酶样物质5（CDKL5）缺乏症（CDD）相关的癫痫患者，年龄≥2岁。 CDKL5缺乏性疾病是一种罕见的神经发育疾病，由CDKL5基因的致病性变异引起，发病率从4万分之一到6万分之一不等。虽然很少见，但它是遗传性癫痫最常见的形式之一。其工业化合成路线如下图： 4.2、Clazosentan Sodium (Pivlaz) 克唑生坦钠（11）是由Idorsia Pharmaceuticals开发的小分子内皮素（ET）受体选择性拮抗剂。该药物抑制ETA受体，可减少动脉瘤性蛛网膜下腔出血后可能发生的ET-相关脑血管痉挛。该药物在日本被批准用于预防脑血管痉挛、血管痉挛相关脑梗死和动脉瘤性蛛网膜下腔出血后的脑缺血症状。其最初是罗氏发现的，Axovan在2022年获得了该药物的全球权利，Axovan在2003年被Actelion收购。2017年6月，从Acetelion制药公司剥离旗下子公司后，Idorsia在全球范围内保留了对唑生坦的使用权力。其合成路线目前还没有明确披露工艺规模，但已经披露了发现阶段规模的药物和结构类似物的合成路径，如下图： 4.3、Sodium Phenylbutyrate and Taurursodiol (Relyvrio) Relyvrio是一个组合用药，包含两种主要成分（12A:12B,3:1质量比），如下图。由Amylyx Pharmaceuticals市场化，获批用于治疗肌萎缩性侧索硬化症（ALS），这是一种进行性神经退行性疾病，攻击和杀死控制随意肌的神经细胞。这种联合用药治疗ALS的作用机制目前尚不清楚。其中苯基丁酸钠（12A)是一种单一芳香脂肪酸，获批用于治疗神经循环障碍，且可大批量商业获得。另一种有效成分（12B），这是一种自然产生的胆酸，有各种同义词，包括牛磺酸和牛磺酸去氧胆酸（TUDCA）。虽然人类只含有少量的这种胆汁酸，但已知熊的胆汁酸含量很高。化合物12B的公斤级合成路线如下图： 4.4、Daridorexant (Quviviq) Daridorexant(13)，是一种口服双羟色胺（OX） 1型和2型受体拮抗剂（DORA）治疗失眠的药物。 OX1和OX2是在大脑中表达的G蛋白偶联受体（GPCR），它们的内源性配体sorexin A和dorexin B通过与OX1和dox 2相互作用促进清醒。该药物阻断了促进清醒的神经肽的结合，以减少过度活跃的清醒。研究发现，发作性睡病与产生OX1的神经元有关，阻断OX1和OX2可以改善睡眠测量。其合成路线见下图： 05 炎症/免疫药物 5.1、Tapinarof (Vtama) Tapinarof(14)，也称为GSK2894512，由葛兰素史克发现，并于2020年由Dermavant Sciences （Riovant Sciences的子公司）授权使用。该药物是一种首创的局部芳烃受体（AhR）激动剂，批准用于治疗成人斑块性银屑病，通过抑制关键促炎细胞因子如IL-17和IL-22而具有药理作用。其也可以一直T细胞扩增和TH17细胞分化，然而，确切的作用机制尚不清楚。其合成路线如下图： 5.2、Deucravacitinib (Sotykto). Deucravacitinib（15），第一类酪氨酸激酶2（TYK2）抑制剂，批准用于治疗中度至重度斑块性银屑病。自2013年以来，在12种获批药物中，靶标类提供的JAK1、JAK2、JAK3和Tyk2药物干预治疗多种炎症性疾病的影响不能被夸大。由百时美施贵宝公司（BMS）发现的deucravacitinib(15)，也被称为BMS-986165，是第一个含氘的JAK抑制剂，也是第一个被批准的Tyk2选择性抑制剂（相对JAK1、JAK2、JAK3，超5万倍选择性）。该药物通过与Tyk2的JH2结构域内的同源口袋结合，实现了高度的选择性，这可能导致与其他可用的JAK抑制剂相比，存在安全性差异。其吨级合成路线如下图： 5.3、Carotegrast methyl (Carogra) Carogra（16）是一种小分子α4-整合素拮抗剂，由EA Pharma开发用于治疗溃疡性结肠炎。溃疡性结肠炎是炎症性肠病的一个亚类，在美国和世界各地影响着1 - 200万患者。甲基酯功能被水解成具有生物活性的羧酸，其选择性地抑制α-4整合素与细胞粘附分子的结合，从而防止结肠炎的发生。该药物首次在日本获批，用于治疗对5-氨基水杨酸反应不足的中度溃疡性结肠炎患者。其合成路线如下图： 06 代谢药物 6.1、Chiglitazar (Bilessglu) Chiglitazar（17）是一种新型的α,δ和γ过氧化物酶体增殖物激活受体（PPARs）的非噻唑烷二酮泛激动剂，已显示出治疗2型糖尿病的希望。 2型糖尿病影响全球超过3.74亿患者，并且在全球范围内发病率和患病率持续上升。该药物优先调节ANGPTL4和PDK4基因的表达，这两个基因与葡萄糖和脂质代谢有关。其合成路线如下： 6.2、Dorzagliatin (HuaTangNing) Dorzagliatin（18）由华领医药研发，用于治疗糖尿病肾病（DKD）、1型糖尿病（T1DM）和2型糖尿病（T2DM）。这种小分子口服葡萄糖激酶激活剂（GKA）于2022年9月在中国首次获批，用于T2DM单药治疗和二甲双胍（一种降糖药）联合治疗的成人患者对于2型糖尿病患者，葡萄糖激酶的表达降低，因此，多扎格汀等药物是一种新型的抗糖尿病治疗方案。其合成路线如下： 07 肿瘤药物 7.1、Adagrasib (Krazati) Adagrasib(19)是Mirati Therapeutics公司开发的KRASG12C的不可逆共价口服抑制剂，被FDA批准用于治疗伴有KRASG12C突变的局部晚期或转移性非小细胞癌（NSCLC）。 KRAS是所有癌症中最常见的突变癌基因，KRAS是一种G蛋白，在调节多种涉及细胞增殖和死亡的途径中起重要作用。导致KRAS过度激活的突变与许多癌症密切相关。 Adagasib靶向突变KRAS G12蛋白的半胱氨酸残基，并且不影响野生型KRAS，导致癌细胞死亡和肿瘤消退。其工业化合成路线如下： 7.2、Savolitinib (Orpathys) Savolitinib（20）是间充质上皮转化因子（MET）癌基因的小分子抑制剂，于2021年在中国有条件地批准，用于治疗MET外显子跳跃突变的非小细胞肺癌患者。这种突变导致MET稳定性和表达增加，从而导致多种下游信号通路的延长，这些信号通路调节关键的细胞功能，包括增殖、分化和细胞存活。MET外显子14突变在大约3-4%的非小细胞肺癌患者中出现。该药物目前正在研究用于治疗其它形式的癌症，包括乳头状和透明细胞癌、胃癌、和结直肠癌。其由和黄医药与AstraZeneca合作开发。 AstraZeneca披露的克级至公斤级合成路线如下： 7.3、Furmonertinib (Ivesa) Furmonertinib（21）是一种选择性表皮生长因子受体（EGFR）酪氨酸激酶抑制剂（TKI），由Allist制药公司开发，用于治疗EGFR突变阳性的非小细胞癌（NSCLC）。虽然EGFR TKIs已经推出了多种治疗非小细胞肺癌阳性突变形式的抑制剂，但耐药性的发展是一个挑战。临床最重要的耐药突变是在基因790的苏氨酸向蛋氨酸替代（T790M）。这种突变对第一代和第二代EGFR TKIs耐药，促使Furmonertinib成为第三代治疗TKIs。 2021年该药物在中国获得有条件的批准，用于治疗在EGFR TKI治疗期间或之后发生进展的T790M突变的，局部晚期或转移性NSCLC患者。其公斤级合成路线如下： 7.4、Lazertinib (Leclaza) Lazertinib（22）是一种口服、中枢神经系统渗透的第三代EGFR-TKI，由Janssen和Yuhan开发。该药物已被韩国卫生和安全部批准用于治疗既往接受EGFR-TKI治疗的EGFR T790M突变阳性非小细胞肺癌（NSCLC）患者。EGFR-TKIs是突变激活EGFR的NSCLC患者的一线治疗方法。尽管NSCLC肿瘤对第一代和第二代EGFR-TKIs的反应最初有所消退，但由于耐药，大多数患者在9-13个月后发生疾病进展。 EGFR T790M突变占对第一代、第二代EGFR-TKIs耐药的肿瘤的50-60%。Lazartinib已被证明可以不可逆地抑制这种突变，不影响野生型EGFR，促进EGFR T790M阳性肿瘤消退。其多克级合成路线如下： 7.5、Olutasidenib (Rezlidhia) Olutasidenib(23)，一种异柠檬酸脱氢酶-1（IDH1）抑制剂，用于治疗IDH1易感突变的复发或难治性急性髓性白血病（AML）成人患者。该药物最初是由Rigel Pharmaceuticals开发的，随后与Forma Therapeutics （Novo Nordisk子公司）达成许可协议，在美国进一步开发、制造和商业化。由Rigel Pharmaceuticals披露的合成路线如下： 7.6、Olverembatinib Olverembatinib（24）是由亚盛医药开发的可口服、第三代酪氨酸激酶抑制剂（TKI），用于治疗慢性髓性白血病（CML）、急性髓性白血病、急性淋巴细胞白血病（ALL）和实体瘤。该药物于2021年11月在中国获得首次批准，并被批准用于具有TKI耐药CML-慢性期和加速期的成人，其中包含T315I“看门人”突变。目前的CML治疗主要以TKIs为中心，然而，TKIs治疗的耐药性，通常是通过BCR-ABL1激酶域点突变，仍然是一个挑战。 Olverembatinib通过作为野生型BCR-ABL1激酶和广泛相关的突变体（包括T315I）的ATP结合位点抑制剂，保持其有效性，否则会对第一代和第二代TKIs产生抗性。其合成路线如下： 7.7、Valemetostat Tosylate (Ezharmia) Valemetostat Tosylate（25）由Daiichi Sankyo公司开发，于2022年9月在日本获批，用于治疗复发或难治性成人细胞白血病-淋巴瘤（R/RATL）患者，这种癌症现有治疗方案很少且预后不良。该药物是一种口服的选择性双抑制剂，可抑制表观遗传靶点EZH1（zeste同源1增强子）和EZH2 （zeste同源2增强子），它们与多梳抑制复合体2（PRC2）相连接。EZH1/2通过组蛋白甲基化调节基因表达，从而抑制与肿瘤抑制和细胞分化相关的基因。虽然EZH2选择性抑制剂之前已经开发出来，但EZH1可以补偿EZH2的损失，因此需要开发双抑制剂。与EZH2选择性抑制剂他zemetostat相比，Valemetostat能显著降低组蛋白H3赖氨酸27（H3K27）三甲基化。其公斤级合成路线如下： 7.8、Fuzuloparib (AiRuiYi; Formerly Fluzoparib) 福唑帕利（26）是一种口服活性聚（ADP-核糖）聚合酶（PARP）抑制剂，由江苏恒瑞开发，被批准用于治疗铂敏感复发性卵巢癌、输卵管癌或原发性腹膜癌患者，这些患者患有生殖系乳腺癌基因（BRCA）突变，接受过二线或以上化疗。 PARP酶在细胞的DNA损伤修复（DDR）反应中起着重要作用，PARP结合DNA损伤位点，并催化PAR化，导致同源重组（HR）募集DNA修复机制。缺乏BRCA的癌细胞修复DNA双链断裂（DSB）的能力有限。 Fuzuloparib显示出强大的抗癌活性，可以抑制BRCA缺陷导致的HR缺陷。该药物抑制正常的PARP功能，使DSB在肿瘤细胞中逐渐积累，最终达到合成致死。由于具有显著的生存益处，一些PARP抑制剂已被FDA、EMA和NMPA批准用于甲状腺、乳腺癌、胰腺癌或前列腺癌患者。恒瑞披露的大规模合成路线如下： 7.9、Pamiparib (Partruvix) Pamiparib（27）是百济神州开发的口服活性、强效、高选择性PARP1和PARP2抑制剂。该药物于2022年在中国被批准用于治疗BRCA突变的复发性晚期卵巢癌、输卵管癌或原发性腹膜癌。 BRCA11和BRCA2是通过功能同源重组（HR）修复DNA双链断裂（DSB）的关键肿瘤抑制因子。据称该药物对治疗伴有脑转移的癌症患者表现出良好的脑穿透能力。百济披露了其工业化规模原料药合成路线如下： 7.10、Lutetium (177Lu)Vipivotide Tetraxetan (Pluvicto) Lutetium (177Lu)vipivotide tetraxetan(28)，也被称为177Lu-PSMA-617，是一种放射性药物，于2022年被批准用于治疗前列腺特异性膜抗原（PSMA）阳性的转移性去势抵抗性前列腺癌（mCRPC），并获得FDA认定为首创药物。该药物部分由普渡大学、海德堡大学医院和德国癌症研究中心的研究人员发现，最初授权给德国制药公司ABX，其早前于2017年被Endocyte收购，随后于2018年被诺华公司收购。该药物作为FDA批准用于治疗前列腺癌（PCa）的第一种放射性药物，该化合物由于其大分子量而具有较长的清除时间和有限的进入肿瘤的所有部分。研究人员在研究了一系列基于脲-DOTA偶联PSMA抑制剂，具有最低的药效要求，可以有效结合PSMA，同时改善药代动力学和组织分布，最终开发出了PSMA-617。该分子在与68Ga结合时显示出诊断性，与177Lu结合时显示出治疗性。由于其分子量、理化性质、和递送目标，该药物具有高度的效价和选择性，同时可进入广泛分布的组织腔室。其合成路线如下： 7.11、Disitamab vedotin (Aidixi) 迪西他单维多汀（29）是人表皮生长因子受体2（HER2）特异性抗体-药物偶联物（ADC），连接了缬氨酸-瓜氨酸可切割连接物（mc-Val -Cit-PABC）与细胞毒剂（有效载荷）单甲基auristatin E(MMAE)，抗体与连接物-有效载荷单元（mc-Val -Cit-PABC-MMAE, 29.3）的比例为1:4。该ADC于2021年首次在中国获批用于治疗HER2过表达（定义为免疫组织化学评分为2+/3+）的局部晚期或转移性胃癌（包括胃食管交界处腺癌）患者，这些患者之前至少接受过两次全身化疗。 MMAE是一种以auristatin为基础的微管抑制剂，已知可以诱导细胞凋亡和抑制靶向细胞的有丝分裂，使ADC内化，并从连接子释放细胞内。该ADC是由RemeGen和Seagen合作开发，双方已签订了开发和商业化的联合许可协议。其合成路线如下： 7.12、Pimitespib (Jeselhy) Pimitespib（30）已在日本被批准用于治疗化疗后进展的胃肠道间质瘤（GIST）。它的靶标是细胞休克蛋白90 (HSP90)，这是一种分子伴侣，涉及稳定几种负责肿瘤生长的蛋白质。与其他HSP90抑制剂相比，Pimitespib选择性靶向HSP90的α和β亚型，并且具有减少脱靶效应，特别是减少视觉干扰。 Taiho Pharmaceuticals披露的其合成路线如下： 7.13、Donafenib (Zepsun) 多纳菲尼（Donafenib，31）是多激酶抑制剂索拉非尼（sorafenib）的减氢衍生物，用于治疗晚期肝细胞癌（HCC）。 HCC是成年人最常见的原发性肝癌类型，也是全球癌症相关死亡的第三大原因。多纳非尼抑制Srf激酶和VEGFR酪氨酸激酶，从而阻止肿瘤细胞的增殖。该药物中氘代甲基的存在改善了代谢稳定性，半衰期延长，全身清除率降低，全身暴露增加。该药在中国首次获批永固治疗以前未接受过全身治疗的不可切除的HCC患者。其克级合成路线如下： 7.14、Futibatinib (Lytgobi) 福替替尼（Futibatinib, 32）用于治疗多种类型的癌症：乳腺癌、胆管癌、胃癌、非小细胞癌、食管癌和尿路上皮癌。口服小分子癌症治疗药物于2022年9月下旬在美国获得FDA加速批准，该药物是一种不可逆抑制剂和选择性共价结合成纤维细胞生长因子受体（FGFR)1-4。由于FGFR信号通路失调（可由染色体重排、突变、上调和扩增引起）会破坏组织稳态并最终导致癌变，因此抑制该受体是一种很有前景的癌症治疗方法。 Taiho Pharmaceuticals披露了其合成路线如下： 7.15、Pacritinib (Vonjo) Pacritinib（33）是一种联合Janus激酶2（JAK2）和McDonough肉瘤（FMS）样受体酪氨酸激酶3（FLT3）抑制剂，已被批准用于治疗血小板计数低的患者的黄纤维化。该药物的独特之处在于，它能抑制Jak2和FLT3信号通路的多个成员，并对野生型和突变型Jak2和FLT3细胞系失活。与其他治疗方案（如骨髓纤维化）不同，该药物与骨髓抑制无关，最常见的不良反应是可控制的胃肠道疾病。发现其可以减少贫血和血小板减少症患者的脾肿大和与骨髓纤维化相关的症状。其合成路线如下： 08 呼吸/肺部药物 8.1、Gefapixant (Lyfnua) Gefapixant(34)，也被称为MK-7264，优先于AF-219和RO-4926219，是一种用于治疗慢性咳嗽的P2×3拮抗剂，最近被日本批准。慢性咳嗽是患者求医问诊和不明确的最常见原因之一，在过去12个月内咳嗽≥8周。成人慢性咳嗽的患病率为5%，并可能与生活质量的恶化有关。其工业合成路线如下：

信使RNA

2025-02-11

·immedica.com

Immedica completes acquisition of Marinus Pharmaceuticals, Inc.

Stockholm, Sweden – February 11, 2025 – Immedica Pharma AB (“Immedica”), a leading global rare disease company, announced today the completion of the acquisition of Marinus Pharmaceuticals, Inc. (“Marinus”) (Nasdaq: MRNS) through its indirect wholly owned subsidiary Matador Subsidiary, Inc., a Delaware corporation (“Purchaser”). Following the completion of Immedica’s successful tender offer to purchase all outstanding shares of common stock of Marinus for USD 0.55 per share, net to the seller thereof in cash (the “Offer Price”), without interest and subject to any applicable withholding taxes, Immedica acquired all remaining shares of common stock of Marinus through a merger pursuant to Section 251(h) of the General Corporation Law of the State of Delaware. As a result of the transaction, Marinus has become an indirect wholly owned subsidiary of Immedica, and the common stock of Marinus will cease to be traded on the Nasdaq Global Market. At the effective time of the merger, and subject to any perfected appraisal rights, all of the remaining shares of common stock of Marinus not purchased in the tender offer were converted into the right to receive the Offer Price without interest and subject to any applicable withholding taxes. Forward-looking statements This press release contains forward-looking statements by Immedica and the acquisition of Marinus by Immedica (the “Transaction”) that involve risks and uncertainties and reflects Immedica’s judgment as of the date of this press release. These forward-looking statements generally are identified by words such as “believe,” “can,” “could,” “seek,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “might,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result” and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties, many of which are outside of Immedica’s control. Forward-looking statements are based on Immedica’s current expectations and beliefs, and Immedica cannot give assurances that its expectations or beliefs will be achieved. These forward-looking statements are not a guarantee of future performance and are subject to a number of known and unknown risks, uncertainties and other factors that could cause actual results or events to differ, possibly materially, from the expectations or estimates reflected in such forward-looking statements, including, among others: litigation relating to the Transaction; the ability of Immedica to retain key personnel from Marinus; competitive responses to the Transaction; the financial condition, results of operations and business of Immedica; the risk that Immedica may be unable to achieve the anticipated benefits of the Transaction; other business effects, including the effects of industry, economic or political conditions outside of the companies’ control; Immedica’s ability to achieve the growth prospects and synergies expected from the transaction, as well as delays, challenges and expenses associated with integrating Marinus with its existing businesses; and other risks described in Immedica’s prior press releases. Forward-looking statements speak only as of the date they are made. Immedica assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law. Immedica does not give any assurance that it will achieve its expectations. About Marinus Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. Marinus’ product, ZTALMY® (ganaxolone) oral suspension CV, is an FDA-approved prescription medication introduced in the United States in 2022. For more information, please visit www.marinuspharma.com and follow us on Facebook, LinkedIn and X. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care. Immedica was founded in 2018 and employs today around 130 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of Communications linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within genetic & metabolic diseases, hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

并购

2025-02-07

·immedica.com

Immedica successfully completes tender offer for all outstanding shares of common stock of Marinus Pharmaceuticals, Inc.

Stockholm, Sweden – February 7, 2025 – Immedica Pharma AB (“Immedica”), a leading global rare disease company, announced today that it has, through its indirect wholly owned subsidiary Matador Subsidiary, Inc., a Delaware corporation (“Purchaser”), successfully completed its tender offer to purchase all outstanding shares of common stock of Marinus Pharmaceuticals, Inc. (“Marinus”) (Nasdaq: MRNS) at a price of USD 0.55 per share, net to the seller thereof in cash, without interest and subject to any applicable withholding taxes. The tender offer expired as scheduled at 12:00 midnight, Eastern Time, at the end of the day on February 6, 2025. As of the expiration, 37,287,732 shares of common stock of Marinus have been validly tendered and not validly withdrawn, representing approximately 67.5 percent of Marinus’ outstanding shares of common stock, according to the depositary for the tender offer. The conditions to the tender offer were satisfied, and Immedica and Purchaser have accepted for payment and will promptly pay the depositary for all validly tendered shares. Immedica expects to complete the acquisition of Marinus on February 11, 2025, through a merger without a vote or meeting of Marinus’ stockholders pursuant to Section 251(h) of the General Corporation Law of the State of Delaware. At the effective time of the merger, and subject to any perfected appraisal rights, all of the remaining shares of common stock of Marinus not purchased in the tender offer will be converted into the right to receive the same USD 0.55 per share, net to the seller thereof in cash, without interest and subject to any applicable withholding taxes. Upon completion of the merger, Marinus will become an indirect wholly owned subsidiary of Immedica, and the common stock of Marinus will cease to be traded on the NASDAQ Global Market. On February 11, 2025, Immedica intends to file with the U.S. Securities and Exchange Commission (the “SEC”) an amendment to the tender offer statement on Schedule TO which sets forth the final results of the tender offer. Broadbridge Corporate Issuer Solutions, LLC is acting as depositary for the tender offer. For Immedica, MTS Health Partners, L.P. acted as the financial advisor and Gibson, Dunn & Crutcher LLP as the legal counsel. For Marinus, Barclays Capital Inc. acted as the financial advisor and Hogan Lovells US LLP as the legal counsel. Forward-looking statements This press release contains forward-looking statements related to Immedica, Marinus and the acquisition of Marinus by Immedica (the “Transaction”) that involve risks and uncertainties and reflect each of Immedica’s and Marinus’ judgment as of the date of this press release. These forward-looking statements generally are identified by words such as “believe,” “can,” “could,” “seek,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “might,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result” and similar expressions. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties, many of which are outside of Immedica’s and Marinus’ control. Forward-looking statements are based on Immedica’s and Marinus’ current expectations and beliefs, and neither Immedica nor Marinus can give assurances that its expectations or beliefs will be achieved. These forward-looking statements are not a guarantee of future performance and are subject to a number of known and unknown risks, uncertainties and other factors that could cause actual results or events to differ, possibly materially, from the expectations or estimates reflected in such forward-looking statements, including, among others: the ability to consummate the Transaction and to meet expectations regarding the timing and completion of the Transaction; litigation relating to the Transaction; the satisfaction or waiver of the other conditions to the completion of the Transaction on the terms expected or on the anticipated schedule; the financial condition, results of operations and business of Immedica and Marinus; the occurrence of any event, change or other circumstance that could give rise to the termination of the merger agreement entered into between the companies; the risk that Immedica may be unable to achieve the anticipated benefits of the Transaction; other business effects, including the effects of industry, economic or political conditions outside of the companies’ control; and other risks described in Immedica’s and Marinus’ respective prior press releases and listed under the heading “Risk Factors” in Marinus’ reports filed with the SEC, including current reports on Form 8-K, quarterly reports on Form 10-Q and annual reports on Form 10-K, the Schedule 14D-9 filed by Marinus and the Schedule TO and related tender offer documents filed by Immedica and Purchaser. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Investors are cautioned not to put undue reliance on forward-looking statements, and each of Immedica and Marinus assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law. Neither Immedica nor Marinus gives any assurance that either Immedica or Marinus will achieve its expectations. About Marinus Pharmaceuticals Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. Marinus’ product, ZTALMY® (ganaxolone) oral suspension CV, is an FDA-approved prescription medication introduced in the United States in 2022. For more information, please visit www.marinuspharma.com and follow us on Facebook, LinkedIn and X. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within genetic & metabolic diseases, hematology & oncology and specialty care. Immedica was founded in 2018 and employs today around 130 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com Immedica contact: Linda Holmström Head of Communications linda.holmstrom@immedica.com Marinus contact: Molly Cameron Director, Corporate Communications & Investor Relations Marinus Pharmaceuticals, Inc. mcameron@marinuspharma.com Information Agent for the offer: MacKenzie Partners, Inc +1 800 322 2885 Press release - Immedica successfully completes tender offer for all outstanding shares of common stock of Marinus Pharmaceuticals, Inc Immedica Pharma AB Solnavägen 3H SE-113 63 Stockholm Follow us to stay up to date with what is happening at Immedica. We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within genetic & metabolic diseases, hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com

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100 项与加那索龙相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D04300	加那索龙	-	DB05087

研发状态

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10 条最早获批的记录,

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适应症	国家/地区	公司	日期
癫痫发作	欧盟	Marinus Pharmaceuticals, Inc.	2023-07-26
癫痫发作	冰岛	Marinus Pharmaceuticals, Inc.	2023-07-26
癫痫发作	列支敦士登	Marinus Pharmaceuticals, Inc.	2023-07-26
癫痫发作	挪威	Marinus Pharmaceuticals, Inc.	2023-07-26
CDKL5缺乏症	美国	Marinus Pharmaceuticals, Inc.	2022-03-18

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适应症	最高研发状态	国家/地区	公司	日期
癫痫	临床3期	美国	Marinus Pharmaceuticals, Inc.	2022-05-17
癫痫	临床3期	美国	元羿生物科技（上海）有限公司	2022-05-17
癫痫	临床3期	澳大利亚	Marinus Pharmaceuticals, Inc.	2022-05-17
癫痫	临床3期	澳大利亚	元羿生物科技（上海）有限公司	2022-05-17
癫痫	临床3期	比利时	元羿生物科技（上海）有限公司	2022-05-17
癫痫	临床3期	比利时	Marinus Pharmaceuticals, Inc.	2022-05-17
癫痫	临床3期	加拿大	Marinus Pharmaceuticals, Inc.	2022-05-17
癫痫	临床3期	加拿大	元羿生物科技（上海）有限公司	2022-05-17
癫痫	临床3期	法国	Marinus Pharmaceuticals, Inc.	2022-05-17
癫痫	临床3期	法国	元羿生物科技（上海）有限公司	2022-05-17

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04391569 (RAISE, Biospace) 人工标引	临床3期	癫痫	96	Ganaxolone	遞簾膚構壓網築襯繭膚(顧憲鹽淵築餘鹽鬱鏇構) = 憲遞蓋鏇構憲憲齋夢餘網夢觸築憲積齋簾衊窪 (齋顧顧壓齋願壓齋獵鏇 ) 达到更多	积极	2024-06-17
				Placebo	遞簾膚構壓網築襯繭膚(顧憲鹽淵築餘鹽鬱鏇構) = 膚築艱餘廠選廠簾襯廠網夢觸築憲積齋簾衊窪 (齋顧顧壓齋願壓齋獵鏇 ) 达到更多
NCT03572933 (MARIGOLD Study, AAN2024)	临床3期	CDKL5缺乏症	101	Valproate	鹽繭廠獵鑰憲襯顧選鏇(鹹襯鹹範憲顧鬱鑰遞鑰) = TEAEs and SAEs were similar in ganaxolone-treated patients across all concomitant ASM subgroups. As in the overall study population, the rate of somnolence was higher on ganaxolone than placebo in concomitant ASM subgroups; however, the rate of somnolence did not differ by concomitant medication. 鹽獵構夢鬱觸觸顧淵遞 (壓獵範鹹獵衊簾夢觸憲 ) 更多	积极	2024-04-09
				Levetiracetam
CNS2023	N/A	CDKL5缺乏症	101	Ganaxolone	遞選糧蓋淵選艱製齋築(選窪鑰夢夢願鑰構鏇衊) = There was one death reported, which was deemed unrelated to study treatment 願夢鬱網鑰範鑰醖鑰醖 (構窪獵廠鑰顧繭顧製網 ) 更多	积极	2023-10-01
IEC2023	N/A	CDKL5缺乏症	101	Ganaxolone	獵襯繭膚蓋餘製網醖襯(構簾齋膚鏇壓築糧積願) = 1 鑰醖願醖鑰夢鏇鬱鏇製 (壓襯窪壓齋蓋製夢衊憲 ) 更多	积极	2023-09-04
				Placebo
IEC2023	临床1期	-	36	IV ganaxolone 10mg	膚襯繭壓糧衊積夢蓋餘(範觸鏇積繭簾窪製觸醖) = No deaths were reported 窪簾獵鑰顧衊鏇鑰膚製 (構窪遞選齋鑰繭築網壓 ) 更多	-	2023-09-04
				IV ganaxolone 30mg
NCT00442104 (CTgov)	临床2期	婴儿痉挛	54	Ganaxolone	憲選繭襯製簾窪製積繭(蓋積鬱壓顧醖構鬱膚襯) = 鹹遞獵獵鹽築築糧夢顧窪憲鹽鹽鑰積鏇襯鏇繭 (糧繭願顧艱製艱積構選, 獵鬱範窪選選艱築選廠 ~ 構網壓鏇選構鹽繭壓鏇) 更多	-	2023-06-02
NCT01725152 (CTgov)	临床2期	脆性X综合征	59	Ganaxolone (Ganaxolone)	憲鬱糧襯廠築鏇遞襯壓(願鹽襯選廠襯鹹築鏇觸) = 製繭網繭襯廠襯壓鑰窪製膚繭壓醖淵構衊觸醖 (築鏇廠範糧獵壓築憲鏇, 製鏇鏇構顧築範積齋廠 ~ 獵選願選構餘廠鹹築衊) 更多	-	2023-04-10
				placebo+Ganaxolone (Placebo)	憲鬱糧襯廠築鏇遞襯壓(願鹽襯選廠襯鹹築鏇觸) = 範製鏇鹽構鑰構網憲醖製膚繭壓醖淵構衊觸醖 (築鏇廠範糧獵壓築憲鏇, 範窪積膚選製範窪壓艱 ~ 壓廠觸製艱淵鬱襯構窪) 更多
NCT04285346 (TSC, CTgov)	临床2期	结节性硬化症	23	Ganaxolone (Treatment Period: Ganaxolone)	淵憲構顧淵顧衊鏇願廠(夢鏇範繭夢鏇遞衊鹹製) = 獵製廠齋築願夢壓膚觸構鹽顧鑰範鏇網鹹觸網 (選願壓願觸壓鏇願夢廠, 餘鏇夢膚艱壓願窪獵遞 ~ 鑰淵鹽憲鹹鏇夢齋觸壓) 更多	-	2023-04-04
				Ganaxolone (Open Label: Ganaxolone)	壓淵夢淵鹽鏇選夢網遞(鏇餘鹽壓遞網憲獵鹹繭) = 鹽壓顧糧襯製遞艱廠壓窪製夢網獵餘遞夢鏇餘 (願願壓餘範膚鹹廠網鹹, 淵壓繭鏇壓鹽顧築構繭 ~ 蓋餘糧衊夢膚餘觸獵廠) 更多
NCT00465517 (CTgov)	临床2期	癫痫发作 \| 癫痫部分性发作	147	Ganaxolone	憲鑰網顧製鹽憲築鹽觸(築餘壓觸憲齋蓋廠鏇餘) = 壓餘壓憲壓鹽蓋膚淵艱繭獵憲窪醖齋襯壓餘範 (築壓淵願廠築簾鹽鑰窪, 積獵顧願觸範艱憲鏇簾 ~ 製構齋築鹽膚簾繭廠廠) 更多	-	2023-03-15