This is a single dose, placebo-controlled study. Male subjects aged 18 to 40 years (inclusive) with a diagnosis of FXS. Eligible subjects may enroll in this study comprised of two in home and two in clinic visits each 14 days apart, for a total of four visits. Subjects will be given single dose gaboxadol (10 mg) or matched placebo at each of these visits to take orally. Thus, all enrolled subjects will receive placebo at home and in clinic and receive gaboxadol at home and in clinic in a blinded fashion.

开始日期2024-01-29

申办/合作机构

Children's Hospital & Medical Center

[+1]

NCT04823052

/ Withdrawn临床2期

A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS)

This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.

开始日期2022-05-25

申办/合作机构

Healx Ltd.

NCT04106557

/ Completed临床3期

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome

The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.

开始日期2019-09-09

申办/合作机构

Ovid Therapeutics, Inc.

100 项与加波沙朵相关的临床结果

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100 项与加波沙朵相关的转化医学

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100 项与加波沙朵相关的专利（医药）

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812

项与加波沙朵相关的文献（医药）

2026-08-01·MOLECULAR PSYCHIATRY

Hyperactivation of distinct thalamic nuclei differentially impairs sleep physiology in rats

Article

作者: Mendes Duarte, Joana ; Bielser, Thomas ; Gross, Simon ; Bainier, Marie ; Schöppenthau, Stephanie ; Künnecke, Basil ; Debilly, Sébastien ; Schulz, Jan M ; Janz, Philipp ; Redondo, Roger L ; Bruns, Andreas

Sleep disturbances and altered sensory processing are key features of neurodevelopmental and neuropsychiatric disorders (NDDs/NPDs). Clinical assessments of brain function in these conditions reveal increased connectivity between the thalamus and cortical areas, alongside changes in restorative sleep patterns that rely on thalamic function, like decreased sleep spindle density and slow-wave oscillations. To assess the role of individual thalamic nuclei in such impairments, we activated thalamic nuclei within three different thalamocortical circuits in rats using the chemogenetic receptor hM3Dq. Activation of the mediodorsal thalamic (MDT) nuclei impaired restorative sleep by reducing spindle density and slow-wave oscillations, recapitulating phenotypes associated with NDDs/NPDs. Conversely, activating ventral posterior thalamic (VPT) and ventromedial thalamic (VMT) nuclei increased wakefulness, reducing sleep spindle density and alpha/beta band power during sleep. To examine downstream network effects, we used perfusion-based pharmacological MRI; MDT activation significantly modulated neural activity in MDT and interconnected regions, including prelimbic cortex and thalamic nuclei. Finally, we tested whether pharmacologically increasing inhibition in the thalamus using the GABA_Aδ agonist gaboxadol and positive allosteric modulator DS-2, could counter phenotypes driven by thalamic hyperactivity. Gaboxadol partially normalized the balance between light and deep NREM sleep in the MDT-hM3Dq group, and in VPT- and VMT-hM3Dq animals, partially normalized resting-state oscillations but failed to rescue sleep physiology. DS-2 had minimal effects across groups. Collectively, our findings reveal that thalamic nuclei-specific activation impacts restorative sleep, and that this thalamocortical circuit model may provide valuable insights into the neural mechanisms underlying dysfunctions associated with NDDs/NPDs and neuropsychiatric conditions.

2026-08-01·EPILEPSIA

Region‐dependent differences in tonic inhibition underlie epileptic features in Angelman syndrome model mice

Article

作者: Cook, James M. ; Poe, Michael Ming ; Nakajima, Midori ; Miyoshi, Ryoko ; Mondal, Prithu ; Watanabe, Miho ; Sharmin, Dishary ; Hiramatsu, Yasuyoshi ; Goto, Takeru ; Egawa, Kiyoshi ; Manabe, Atsushi ; Ueda, Yuki ; Shiraishi, Hideaki ; Nakakubo, Sachiko

Abstract:

Objective:

Angelman syndrome (AS) is a neurodevelopmental disorder caused by loss of function of the maternally expressed UBE3A gene. Epilepsy and abnormal electroencephalographic (EEG) rhythms are key features, but their mechanisms and treatment remain unclear. Previous work showed that extrasynaptic γ‐aminobutyric acid type A (GABA A ) receptor‐mediated tonic inhibition is reduced in cerebellar granule cells of AS model mice, contributing to motor deficits. Here, we evaluated tonic inhibition across brain regions and tested whether its dysregulation drives epilepsy, EEG abnormalities, and behavioral deficits in AS.

Methods:

Tonic inhibition was measured in principal neurons of the neocortex, hippocampus, and thalamus in maternal Ube3a knockout mice. We examined the effects of MP‐III‐022, an α5‐containing GABA A receptor–selective positive allosteric modulator, and gaboxadol, a δ‐containing GABA A receptor agonist, on EEG, seizure threshold, and anxiety‐like behavior.

Results:

Tonic inhibition was reduced in cortical layer 5 and hippocampal CA1 pyramidal neurons but preserved in thalamic relay neurons. This reduction correlated with elevated GAT1 expression in the cortex and hippocampus but not the thalamus. MP‐III‐022 reduced abnormal slow‐wave EEG activity, increased seizure thresholds, and improved anxiety‐like behavior. In contrast, gaboxadol enhanced slow‐wave activity and lowered seizure thresholds.

Significance:

Deficits in tonic inhibition in AS mice are region‐specific. These region‐dependent differences in tonic inhibition, rather than a global loss, likely underlie EEG abnormalities and heightened seizure susceptibility. α5‐containing GABA A receptors may offer a promising therapeutic target for adjunctive AS treatment.

2025-07-01·PAIN

Molecular and cellular targets of GABAergic anesthetics in the mesopontine tegmentum that enable pain-free surgery

Article

作者: Baron, Mark ; Minert, Anne ; Ibraheem, Angham ; Vaso, Kristina ; Devor, Marshall

Abstract:

The mesopontine tegmental anesthesia area (MPTA) is a focal brainstem locus which, when exposed to GABAergic agents, induces brain-state transitioning from wakefulness to unconsciousness. Correspondingly, MPTA lesions render animals relatively insensitive to GABAergic anesthetics delivered systemically. Using chemogenetics, we recently identified a neuronal subpopulation within the MPTA whose excitation induces this same pro-anesthetic effect. However, very few of these “effector-neurons” express synaptic γ2-containing GABAA receptor isoforms and none express extrasynaptic δ-subunit containing receptors, suggesting that they are not the direct cellular target of GABAergic agents. Here we used pharmacological tools in rats to define the molecular target(s) of GABAergics in the MPTA. GABA microinjected into the MPTA at nanomolar concentrations, selective for GABAAδ-Rs, proved to be pro-anesthetic as was blocking GABA reuptake. Likewise, low-concentration gaboxadol/THIP, also selective for GABAAδ-Rs, was effective, whereas benzodiazepines and zolpidem, which selectively target GABAAγ2-Rs, were not. The GABAergic anesthetics pentobarbital and propofol proved pro-anesthetic when applied to the MPTA at the low concentrations present in the brain after systemic dosing. Glycinergic agonists which are inhibitory, but infective on GABAAδ-Rs, and other non-GABAergic agonists tested, were at most only marginally effective. We conclude that GABAAδ-Rs are the primary molecular target of GABAergic anesthetics in the MPTA. Immunolabeling revealed that this GABAA-R isoform is expressed exclusively by a distinct subpopulation of MPTA “δ-cells” that reside in close apposition to effector neurons. This suggests that during wakefulness, δ-cells serve as inhibitory interneurons which, when silenced by GABAergic agents, disinhibit (excite) the effector-neurons, triggering transition to unconsciousness.

项与加波沙朵相关的新闻（医药）

2026-08-04

·创药网

天使综合征药物研发进展

天使综合征是一种罕见的神经发育障碍性疾病，主要表现为严重的发育迟缓、言语障碍、睡眠障碍、癫痫发作、运动和平衡障碍等。该病对患者的认知功能、生活能力及长期预后产生深远影响，同时，也给家庭和社会带来沉重负担。尽管近年来分子生物学与基因技术快速发展，但截至目前，全球范围内尚无获批的能从根本上改变疾病进程的治疗方法，临床干预仍以对症及支持治疗为主。作为典型的罕见遗传性疾病，该病已纳入我国《第一批罕见病目录》。本文对天使综合征的药物研发情况进行概述，以期为相关研究提供参考。天使综合征（Angelman syndrome）是一种由于母源15q11-13染色体区域的UBE3A基因表达异常或功能缺陷引发的神经发育障碍性疾病，主要表现为精神发育迟滞或智力低下，语言、运动或平衡发育障碍，快乐行为（如频繁发笑、微笑或兴奋），小头畸形，癫痫等。该病被收录于我国《第一批罕见病目录》。作为一种罕见的神经遗传病，天使综合征在全球范围内影响着约1.5万至50万人。其中，该病在欧美人群的患病率为1/24000～1/12000；国内保守估计至少数十万。2014年，复旦大学附属儿科医院启动召开中国首届天使综合征学术会议及病友大会；2017年起，儿科医院在王艺教授的牵头下，积极开展天使综合征的国际临床注册研究，并通过科技部重点研发计划的精准医学课题建立天使综合征300余例的专病队列，开展多组学和神经影像学研究，积极推动基因修正治疗的研究，并积极争取“天使之翼”慈善专项基金的支持。遗传机制目前，天使综合征已明确的4种主要分子遗传机制为母源15q11～13缺失、父源15号染色体存在单亲二体（Uniparental disomy，UPD）、母源15q11.2～q13印记缺陷（imprinting defect）和母源UBE3A基因发生致病突变（表1）。其中，母源15q11～13缺失最常见，多数约长5～7Mb。表1：天使综合征的4种主要遗传学病因药物研发状况目前，天使综合征尚无治愈手段，临床主要依赖抗癫痫药物、物理治疗和睡眠管理。当前研发以靶向UBE3A为核心，技术路线呈现多元化，其中全球进展最快的药物已进入Ⅲ期临床阶段，包括反义寡核苷酸（ASO）类药物（如ION582和Apazunersen等）以及小分子药物（如Gaboxadol）。而基因疗法、基因编辑疗法、miRNA、多肽等疗法多处于早期或临床前研究阶段。从研发机构来看，目前该领域仍以国外企业为主导，如Ionis、Ultragenyx等在相关管线布局与临床推进方面处于领先地位；相比之下，国内机构虽已开始布局，但整体仍处于早期研发探索阶段，尚未形成成熟的系统性管线开发能力（表2）。表2：天使综合征代表性在研药物 01 ASO疗法在神经元中，UBE3A的父源性等位基因是完整的，但其表观遗传学上是沉默状态。UBE3A-ATS是一种长链非编码的RNA反义转录物，通过干扰UBE3A转录本在父源等位基因的延伸，阻断了父源等位基因的UBE3A表达。因而，通过抑制UBE3A-ATS重新激活父源UBE3A等位基因可作为天使综合征治疗的一个重要方向。 ION582是一款通过脊椎穿刺鞘内给药的在研ASO疗法，用于治疗天使综合征。该疗法旨在抑制UBE3A反义转录本（UBE3A-ATS）的表达，并激活来自父体的UBE3A等位基因以增加患者大脑中UBE3A蛋白的产生。2025年9月，ION582获美国FDA授予的突破性疗法认定，其得益于ION582的Ⅰ/Ⅱ期HALOS研究的积极数据，该研究评估了ION582在天使综合征患者中的安全性、耐受性、药代动力学和初步疗效，结果显示：ION582不仅耐受性良好，而且在多个关键功能领域展现出令人鼓舞的临床改善，包括沟通能力、认知功能和运动功能。此前，ION582已获得FDA和欧洲药品管理局（EMA）的孤儿药资格认定，以及FDA的快速通道认定和罕见儿科疾病认定，多重加速通道的加持，无疑将进一步加快其上市进程。目前，一项全球多中心、随机、双盲、安慰剂对照的Ⅲ期REVEAL研究（NCT06914609）已启动，旨在进一步确认ION582在更大规模天使综合征患者群体中的疗效和安全性，预计将于2026年完成患者招募。这一关键性临床研究的顺利推进，将为ION582的最终获批上市提供坚实的数据支持。 Apazunersen（GTX-102）也是一款用于治疗天使综合征的ASO疗法，其通过鞘内给药方式递送，该疗法可促进神经元细胞中父系UBE3A的等位基因表达，产生患者体内所缺失的关键蛋白产物。2025年7月，Apazunersen获FDA授予突破性疗法认定。该疗法已进入Ⅲ期临床研究阶段。此前，Apazunersen在Ⅰ/Ⅱ期临床试验中取得积极数据：接受Apazunersen治疗的扩展队列A和B的患者，在第170天显示出具有临床意义的多领域功能改善。部分剂量递增队列患者在第758天显示出长期和持续的临床益处，远超过自然历史数据。 Rugonersen最初由罗氏研发，是一种具有选择性的ASO疗法，它具有提高UBE3A的mRNA和蛋白表达水平的潜力。2025年Rugonersen的Ⅰ期临床研究结果在Nature Medicine上发表。这项名为TANGELO的Ⅰ期、多中心、开放标签、多剂量递增临床试验，旨在研究Rugonersen在1～12岁的天使综合征儿童患者（共61人，男女分别为33人和28人）中的安全性和耐受性（主要终点）以及药代动力学（次要终点）。结果显示：该临床试验达到了主要终点，Rugonersen具有可接受的安全性和耐受性特征。对探索性终点的分析表明，Rugonersen可导致天使综合征相关脑电图异常出现剂量依赖性的部分正常化，并显示出核心症状的临床改善的信号，超出了自然病史数据的预期。2024年，罗氏在财报中宣布放弃Rugonersen项目。2025年4月，Oak Hill Bio公司宣布与罗氏达成独家许可协议，获得了Rugonersen的独家开发和商业化权利，并计划于2026年初启动关键性的Ⅲ期临床试验。 02 基因疗法MVX-220 腺相关病毒（AAV）基因疗法MVX-220由宾夕法尼亚大学开发，并得到了天使综合征治疗基金会（FAST）的全力支持。该项目随后授权给FAST药物开发加速器AS 2 Bio的投资组合公司MavriX Bio，以便与专注于开发转化基因疗法并确保全球可及治疗的GEMMABio公司合作进行临床转化。MVX-220旨在通过单次枕大池内注射将人类UBE3A基因递送至大脑神经元，以恢复UBE3A基因的功能表达。 2025年11月，MavriX Bio宣布MVX-220的I/Ⅱ期临床试验ASCEND-AS已成功完成首例患者给药。这不仅是MVX-220的首次人体试验，更是全球首个针对天使综合征的基因疗法临床试验，标志着这一罕见神经遗传病的治疗正式迈入基因时代。该疗法已获得美国FDA授予的“孤儿药”资格。 03 小分子药物Gaboxadol Gaboxadol是由Ovid Therapeutics研发的一种小分子突触外γ-氨基丁酸A型受体激动剂。母源性UBE3A基因突变会引起γ-氨基丁酸转运载体1在突触水平的表达增加，从而引起γ-氨基丁酸再摄取增加。抑制性信号在维持神经元兴奋性平衡方面起着关键作用，而小脑颗粒细胞抑制性信号传导的受损可能与天使综合征患者的运动障碍机制有关。在一项Ⅱ期临床试验中，对天使综合征患者进行12周的治疗后，与安慰剂相比较，Gaboxadol显示出普遍耐受性和安全性，且患者的临床总体印象-改善量表评分有提高。然而，在Ⅲ期NEPTUNE临床试验中未达试验终点，试验中观察到的疗效不支持进一步研究。值得注意的是，Ovid还有一款针对天使综合征的药物——短发夹RNA（shRNA）药物OV882，旨在恢复功能性UBE3A蛋白的表达，从根本上解决疾病，但该药物目前未有最新进展报道。小结当前，针对天使综合征的药物研发正处于由对症治疗向核酸与基因层面精准干预加速转型的关键阶段。从作用机制来看，以UBE3A为核心靶标的治疗策略已成为研究主线，其中ASO疗法被认为是最具临床转化潜力的技术路径之一。以ION582、Apazunersen为代表的ASO药物已进入Ⅲ期临床阶段，标志着该领域从机制验证迈向关键性临床验证，显著提升了未来药物落地的可预期性。同时，小分子药物（如NNZ-2591）仍在持续探索中；基因治疗、基因编辑、RNA调控等新兴策略也在并行推进，不仅加深对疾病机制的理解，也推动相关技术持续迭代，并使疗效、安全性、递送效率及长期表达调控等关键问题成为研发竞争与突破的核心方向。在国内层面，随着罕见病政策的不断完善与基因检测技术的逐步普及，天使综合征的临床识别率与社会关注度逐步提升，但整体仍面临临床资源分布不均、专业诊疗体系尚不完善等现实挑战。近年来，国内科研力量逐步向机制性干预方向深入布局，部分研究团队已在基因编辑等前沿策略上取得初步进展，但距离系统性临床开发与产业化应用仍有较长距离。与此同时，在靶标机制研究方面，2019年，中国科学院脑科学与智能技术卓越创新中心（神经科学研究所）等机构在PNAS上发表了题为“UBE3A-mediated PTPA ubiquitination and degradation regulate PP2A activity and dendritic spine morphology”的研究论文，揭示了UBE3A-PTPA-PP2A信号轴在神经系统功能调控中的重要作用，不仅深化了对天使综合征发病机制的分子层面认识，也为相关疾病的药物靶标挖掘与治疗策略开发提供了重要理论依据。总体而言，尽管短期内实现天使综合征的治愈仍面临较大挑战，但随着关键技术的持续成熟与临床证据的不断积累，天使综合征正在向“可精准治疗”的新阶段演进，未来有望为患者提供更加有效且可持续的治疗方案。【参考资料】 1. Almeida JFM, Tonazzini I, Daniele S. Molecular aspects of Angelman Syndrome: defining the new path forward. Biomol Biomed, 2025, 25(9): 1928-1936. 2. 王玉玺, 钟敏. Angelman综合征的治疗研究进展. 国际儿科学杂志, 2025, 52(3): 160-164. 3. Insight数据库、药渡数据库等, 检索日期: 2026年6月8日. 4. 递因递药、罕见病信息网、药明康德等网络公开资源. 本文其它内容请见《全球药物创新快讯》2026年第4期（总第161期）。

临床3期基因疗法寡核苷酸引进/卖出申请上市

2026-03-20

·allsci.com

Ovid Therapeutics raises USD 60m in private placement to expand epilepsy pipeline

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

引进/卖出临床2期临床3期

2026-03-18

·allsci.com

Ovid Therapeutics raises USD 60m in private placement

Ovid Therapeutics Inc. (Nasdaq: OVID), a New York-based biopharmaceutical company developing small molecule therapies for brain disorders, announced a USD 60 million private placement to fund the expansion of its lead clinical program, OV329, into additional neurological indications. The PIPE financing, expected to close on or about March 19, 2026, will support development of OV329 in tuberous sclerosis complex and infantile spasms, alongside general research and development activities. The company intends to combine the net proceeds with existing cash, cash equivalents, and marketable securities. Under the terms of the securities purchase agreement, Ovid is selling 19,154,321 shares of common stock at USD 2.01 per share and 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 including 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. Company overview and pipeline Ovid Therapeutics is 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 being developed as a potential therapy for treatment-resistant focal onset seizures and developmental and epileptic encephalopathies, including tuberous sclerosis complex and infantile spasms. The press release did not specify the current clinical phase of OV329 or provide a timeline for upcoming milestones in these new indications. Ovid’s broader pipeline includes OV4071 and a library of related compounds designed to directly activate the KCC2 transporter, targeting multiple central nervous system disorders. The company has previously advanced OV101 (gaboxadol) through Phase III testing in Angelman syndrome, with a completed randomized, double-blind study enrolling 104 patients and an open-label extension study (ELARA) enrolling 170 patients. OV101 was also evaluated in a completed Phase II trial in Fragile X syndrome. In July 2020, Ovid licensed exclusive European rights to OV101 for Angelman syndrome to Angelini Pharma. On the business development side, Ovid has undergone a period of portfolio restructuring. In June 2024, Takeda secured global development and commercialization rights to soticlestat (TAK-935), a cholesterol 24-hydroxylase inhibitor previously co-developed with Ovid for Dravet syndrome and Lennox-Gastaut syndrome. In June 2025, Ovid sold 100% of its ganaxolone royalty rights to Immedica Pharma AB for USD 7 million in cash. The company also holds a collaboration with Graviton Bioscience, established in May 2023, focused on developing brain-penetrant ROCK2 inhibitors for rare neurological diseases marked by seizures. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

临床2期临床3期

100 项与加波沙朵相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D04282	加波沙朵	-	DB06554

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
天使综合征	临床3期	美国	Ovid Therapeutics, Inc.	2019-09-09
天使综合征	临床3期	澳大利亚	Ovid Therapeutics, Inc.	2019-09-09
天使综合征	临床3期	德国	Ovid Therapeutics, Inc.	2019-09-09
天使综合征	临床3期	以色列	Ovid Therapeutics, Inc.	2019-09-09
天使综合征	临床3期	荷兰	Ovid Therapeutics, Inc.	2019-09-09
入睡和睡眠障碍	临床3期	-	H. Lundbeck A/S	2003-01-01
脆性X综合征	临床2期	美国	Ovid Therapeutics, Inc.	2018-09-17
重度抑郁症	临床2期	奥地利	H. Lundbeck A/S	2008-11-01
重度抑郁症	临床2期	俄罗斯	H. Lundbeck A/S	2008-11-01
癫痫	临床前	美国	Ovid Therapeutics, Inc.	-

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适应症

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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


NCT03882918 (ELARA, CTgov)	临床3期	天使综合征	141	OV101	淵齋築糧齋醖網衊齋淵 = 壓艱夢糧鑰築簾製遞願糧鏇窪築網夢淵廠觸鹽 (獵積觸鬱製繭顧鏇衊淵, 鹽襯簾鹹憲鬱窪淵獵觸 ~ 範憲齋窪夢衊築鹹簾艱) 更多	-	2025-07-18
NCT03697161 (ROCKET, CTgov)	临床2期	脆性X综合征	36	OV101 (Gaboxadol) (OV101 (Gaboxadol) (5 mg QD))	鬱壓廠廠範壓窪構網糧 = 鏇艱範膚糧獵範鹽艱夢齋廠憲構獵選鑰衊願鬱 (繭積顧願窪夢膚憲蓋積, 淵鬱鹹繭鑰鬱糧構獵築 ~ 壓觸蓋網蓋獵簾鑰齋遞) 更多	-	2024-01-23
NCT03697161 (ROCKET, CTgov)	临床2期	脆性X综合征	36	OV101 (Gaboxadol) (OV101 (Gaboxadol) (5 mg BID))	鬱壓廠廠範壓窪構網糧 = 獵壓淵蓋積積壓鹽遞廠齋廠憲構獵選鑰衊願鬱 (繭積顧願窪夢膚憲蓋積, 顧艱壓遞簾齋鬱醖淵鑰 ~ 鬱廠顧壓製壓鑰鏇淵鬱) 更多	-	2024-01-23
NCT02996305 (The STARS Phase 2 Study, CTgov)	临床2期	天使综合征	88	Placebo	糧衊鑰範蓋窪觸遞網構 = 願鏇膚築願觸製鹽齋艱醖醖淵願壓醖蓋簾醖壓 (繭衊醖構製蓋襯膚選構, 鹹憲觸憲鹹範糧襯製獵 ~ 壓憲觸製簾範構簾廠齋) 更多	-	2022-03-31
NCT03697161 (ROCKET, Pubmed \| Front Pharmacol) 人工标引	临床2期	脆性X综合征	23	OV101	艱艱範遞遞積製鏇繭衊(範蓋膚窪顧餘醖選鑰淵) = 顧窪糧願築積夢範鑰鑰遞選憲網鏇憲構網餘構 (艱願廠遞夢鹽築廠鏇鏇 ) 更多	积极	2021-10-08
NCT04106557 (NEPTUNE, GlobeNewswire) 人工标引	临床3期	天使综合征	97	OV101	積鹽鏇廠餘網艱鹹夢蓋(簾範糧選選觸製顧範鏇) = 遞廠構鏇窪鏇積顧鑰衊獵蓋膚壓鏇鹽鏇構艱鏇 (襯構齋蓋憲繭淵鏇醖繭 )	不佳	2020-12-01
NCT04106557 (NEPTUNE, GlobeNewswire) 人工标引	临床3期	天使综合征	97	Placebo	積鹽鏇廠餘網艱鹹夢蓋(簾範糧選選觸製顧範鏇) = 簾壓艱糧製築觸鬱鏇觸獵蓋膚壓鏇鹽鏇構艱鏇 (襯構齋蓋憲繭淵鏇醖繭 )	不佳	2020-12-01
STARS trial (AAN2020)	临床2期	天使综合征	-	Gaboxadol	範廠顧淵構繭築遞範淵(齋積廠範築鏇壓淵衊醖) = 鏇衊簾廠蓋網醖鹽淵鑰衊衊襯窪壓網蓋鹹壓鬱 (壓膚獵積顧築築鹹憲壓 ) 更多	-	2020-04-14
Pubmed \| Int J Neuropsychopharmacol	临床2期	重度抑郁症	490	Placebo	繭遞繭鑰範鏇網蓋鹹衊(窪餘網襯鏇鏇製網襯憲) = The most common adverse events reported in the active treatment groups for which the incidence was higher than that in the placebo group, comprised anxiety 餘鹹壓製繭獵簾鹹製積 (憲構壓網製壓築遞鑰廠 ) 更多	-	2012-07-01
Pubmed \| Int J Neuropsychopharmacol	临床2期	重度抑郁症	490	Escitalopram 20 mg		-	2012-07-01
NCT00807248 (CTgov)	临床2期	重度抑郁症	490	Placebo (Placebo (Orally, Once Daily))	積範廠蓋艱艱積簾膚簾(積鬱齋鑰窪製淵製顧繭) = 鑰願積糧範構廠網糧襯憲夢糧醖選範鏇積蓋膚 (鑰襯鹹網艱醖窪簾夢顧, 1.1) 更多	-	2011-08-11
NCT00807248 (CTgov)	临床2期	重度抑郁症	490	Escitalopram+Placebo (Escitalopram 20 mg and Placebo (Orally, Once Daily))	積範廠蓋艱艱積簾膚簾(積鬱齋鑰窪製淵製顧繭) = 鑰廠憲膚觸鹹鬱壓糧鏇憲夢糧醖選範鏇積蓋膚 (鑰襯鹹網艱醖窪簾夢顧, 0.9) 更多	-	2011-08-11
NCT00103818 \| NCT00095069 (0928-014, Pubmed)	临床3期	入睡和睡眠障碍	-	Gaboxadol 15 mg	繭鬱醖膚壓獵願範簾鹽(齋衊選鹹積鬱願網鹽觸) = 壓膚網壓繭觸艱膚網願餘鬱鹽觸壓糧壓蓋餘襯 (蓋壓鏇選廠窪糧構選網 ) 更多	-	2010-02-15