OV350 showed a good safety profile, supporting the advancement of the Company’s KCC2 portfolio, including the first oral direct activator, OV4071

OV350 表现出良好的安全性，支持了公司 KCC2 产品组合的进展，包括首个口服直接激活剂 OV4071。

There were no treatment-related laboratory findings, no safety findings, and no treatment-related serious adverse events (SAEs)

与治疗相关的实验室结果、安全性结果以及与治疗相关的严重不良事件（SAEs）均未出现。

Exploratory quantitative electrophysiology results suggest OV350 had central activity and spectral power consistent with expected physiological effects of KCC2 modulation; aligned with expected drug exposure in the brain

探索性定量电生理学结果显示，OV350具有中枢活性，其频谱功率与预期的KCC2调节生理效应一致；与预期的脑部药物暴露相符。

Pharmacokinetics for OV350 were as predicted, and will inform dosing strategies for future KCC2 development programs

OV350的药代动力学结果与预测一致，这将为未来KCC2开发计划的剂量策略提供依据。

OV4071 (oral) is on track for regulatory submission for a Phase 1/1b clinical trial in Q1 2026

OV4071（口服）预计将于2026年第一季度提交1/1b期临床试验的监管申请。

NEW YORK, Dec. 18, 2025 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (Nasdaq: OVID), a biopharmaceutical company developing small molecule medicines to treat brain disorders and symptoms caused by excess neural excitability, today announced results from its Phase 1 study of OV350, the first-ever KCC2 direct activator known to be dosed in humans.

纽约，2025年12月18日（环球新闻社）——Ovid Therapeutics Inc.（纳斯达克股票代码：OVID）是一家开发小分子药物以治疗由神经过度兴奋引起的脑部疾病和症状的生物制药公司，今天宣布了其OV350一期研究的结果，OV350是已知首个在人体中给药的KCC2直接激活剂。

The study met its primary objectives to evaluate safety, tolerability and pharmacokinetics. Results from this intravenous program support the advancement of the Company’s portfolio of oral KCC2 direct activators into the clinic..

该研究达到了评估安全性和耐受性及药代动力学的主要目标。此静脉注射项目的结果支持了公司将口服KCC2直接激活剂组合推进到临床阶段。

“OV350 is a valuable tool program that supported human safety for drugging KCC2, an entirely new therapeutic target in the brain, which could be a master switch to curb neural hyperexcitability,” said Meg Alexander, President and Chief Operating Officer of Ovid Therapeutics. “The results from this study give us confidence that this new mechanistic class is amenable for further development and reinforces our decision earlier this year to invest in the development of additional direct activator molecules and formulations for chronic use, including OV4071, the first oral KCC2 direct activator.

“OV350 是一个重要的工具程序，支持了针对 KCC2 的药物安全性，KCC2 是大脑中一个全新的治疗靶点，可能是抑制神经元过度兴奋的主控开关，”奥维德治疗公司总裁兼首席运营官梅格·亚历山大表示。“这项研究的结果使我们相信，这种新的机制类别适合进一步开发，并且巩固了我们今年早些时候的决定，即投资开发更多的直接激活分子和配方以供长期使用，包括首个口服 KCC2 直接激活剂 OV4071。”

We expect to submit our regulatory application for a Phase 1/1b study of OV4071 in Q1 2026 and plan to initiate clinical studies in Q2 2026.”.

我们预计将在2026年第一季度提交OV4071的1/1b期研究监管申请，并计划在2026年第二季度启动临床研究。

Study Design and Key Results

研究设计与关键结果

OV350 was evaluated in an exploratory randomized, placebo-controlled, single-ascending dose study in 16 healthy participants (six active, two placebo per cohort). Doses of 50 mg and 100 mg were administered by IV infusion over ten minutes, with pharmacokinetic sampling conducted up to 48 hours post-dosing.

OV350 在一项探索性随机、安慰剂对照、单剂量递增研究中对 16 名健康参与者（每组六名活性药物，两名安慰剂）进行了评估。50 毫克和 100 毫克的剂量通过静脉输注十分钟给予，药代动力学采样在给药后长达 48 小时内进行。

In addition to laboratory and clinical results, exploratory quantitative electroencephalography (qEEG) endpoints were collected..

除了实验室和临床结果，还收集了探索性的定量脑电图 (qEEG) 终点。

Key findings include:

关键发现包括：

At 50 mg and 100 mg doses of OV350, exposure levels achieved expected pharmacologically active concentrations reinforcing the potential for further clinical development of KCC2 direct activators, including OV4071, which performs with twenty-fold greater potency than OV350 in pharmacodynamic models.

在50毫克和100毫克剂量的OV350中，达到的暴露水平实现了预期的药理活性浓度，这进一步证实了KCC2直接激活剂（包括在药效学模型中表现出比OV350强二十倍效力的OV4071）进行进一步临床开发的潜力。

The most frequent treatment-emergent adverse event (AE) associated with OV350 was headache.

与OV350相关的最常见的治疗中出现的不良事件（AE）是头痛。

Nausea and vomiting occurred in a subset of participants who experienced headache. These AEs coincided with the timing of food intake and are believed to be caused by secondary off-target pharmacology unique to OV350.

头痛受试者中有一部分人出现恶心和呕吐。这些不良事件与进食时间一致，被认为是由OV350独特的次级脱靶药理学引起的。

There were no treatment-emergent SAEs associated with OV350 and stopping criteria were not met in the study.

研究中未出现与OV350相关的治疗引发的严重不良事件（SAEs），且未达到停止标准。

The pharmacokinetics were as predicted for all doses.

所有剂量的药代动力学均符合预期。

qEEG findings supportive of central activity and spectral power relevant for the expected pharmacologic impact of KCC2 modulation were observed. These effects were contemporaneous with the expected exposure of OV350 in the brain.

观察到支持中枢活动和与KCC2调节预期药理作用相关的频谱功率的qEEG结果。这些效应与OV350在大脑中的预期暴露同时发生。

Results from the study will be submitted for a future congress.

研究结果将提交给未来的大会。

Earlier this year, Ovid prioritized and directed its capital resources to accelerate chronic (oral) formulations of its oral direct activator programs, including OV4071 and OV4041. Accordingly, the Company does not intend to advance OV350 IV further in the clinic.

今年早些时候，Ovid优先考虑并将其资本资源用于加速其口服直接激活项目（包括OV4071和OV4041）的慢性（口服）制剂开发。因此，公司不打算在临床上进一步推进OV350 IV。

Advancing a First-in-Class Portfolio of Oral KCC2 Direct Activators

推进首创口服KCC2直接激活剂组合

Ovid believes the results from OV350 support the advancement of Ovid’s portfolio of KCC2 direct activators, which contains multiple unique molecules. Most advanced in the portfolio is development candidate OV4071, an oral direct activator that is twenty-fold more potent than OV350 in pharmacodynamic disease models.

Ovid认为，OV350的研究结果支持了Ovid公司KCC2直接激活剂组合的进展，该组合包含多个独特的分子。在该组合中，最先进的开发候选药物是OV4071，这是一种口服直接激活剂，在药效学疾病模型中，其效力比OV350强二十倍。

Ovid expects to initiate a Phase 1/1b safety and proof-of-concept clinical study in Q2 2026 for the treatment of psychosis associated with Parkinson’s disease and Lewy body dementia. These conditions have high unmet need, validated endpoints, an established regulatory pathway, and traditional atypical antipsychotics are typically contraindicated.

Ovid预计将在2026年第二季度启动一项针对帕金森病和路易体痴呆相关精神病治疗的1/1b期安全性和概念验证临床研究。这些疾病具有高度未满足的需求、已验证的终点、明确的监管路径，并且传统非典型抗精神病药物通常为禁忌。

Additionally, the Company is planning to characterize the pharmacodynamic effects of OV4071 and relevant mechanisms in additional neuropsychiatric conditions, such as schizophrenia and psychoses or agitation associated with other neurodegenerative conditions including Alzheimer’s disease..

此外，公司计划进一步研究 OV4071 在其他神经精神疾病中的药效学作用及其相关机制，例如精神分裂症、精神病或与其他神经退行性疾病（包括阿尔茨海默病）相关的躁动症状。

Concurrent to the clinical translation of OV4071, Ovid is advancing next-generation KCC2 activators from its proprietary library of compounds. These molecules are designed for oral and injectable formulations.

与OV4071的临床转化同步，Ovid公司正在从其专有化合物库中推进下一代KCC2激活剂。这些分子设计用于口服和注射制剂。

About KCC2

关于KCC2

KCC2 is a neuron-specific chloride transporter that maintains inhibitory balance in the brain. The cotransporter plays a central role in regulating neuronal excitability by enabling gamma-aminobutyric acid (GABA) to exert its inhibitory effect. Direct activation of KCC2 represents a differentiated, mechanism-based approach to treating serious neurological and neuropsychiatric conditions in which neuronal hyperexcitability is central to disease and symptom manifestation.

KCC2是一种神经元特异性的氯化物转运蛋白，它维持着大脑中的抑制平衡。该共转运蛋白通过使γ-氨基丁酸（GABA）发挥其抑制作用，在调节神经元兴奋性方面起着核心作用。直接激活KCC2代表了一种基于机制的差异化方法，用于治疗以神经元过度兴奋为核心致病因素及症状表现的严重神经系统和神经精神疾病。

Ovid’s KCC2 programs are designed to build a first-in-class franchise targeting restoration of excitatory/inhibitory balance in the brain, which may offer therapeutic benefit across multiple neurological and neuropsychiatric disorders..

Ovid的KCC2项目旨在建立一个首创的特许经营体系，针对恢复大脑中的兴奋/抑制平衡，这可能为多种神经和神经精神疾病提供治疗益处。

About OV350

关于 OV350

OV350 is a first-in-class, investigational direct activator of KCC2, the neuron-specific chloride transporter that restores inhibitory signaling and helps rebalance hyperexcitable neural circuits. Data from this tool program is informative in establishing safety, tolerability, pharmacokinetics and pharmacodynamic properties of this new mechanism of action for the brain..

OV350 是一种首创的、研究中的 KCC2 直接激活剂，KCC2 是一种神经元特异性氯化物转运蛋白，能够恢复抑制性信号传导并帮助重新平衡过度兴奋的神经回路。该工具计划的数据有助于确定这种针对大脑的新作用机制的安全性、耐受性、药代动力学和药效学特性。

About Ovid Therapeutics

关于Ovid Therapeutics

Ovid Therapeutics Inc. is a New York-based biopharmaceutical company dedicated to developing small molecule medicines for brain conditions and symptoms caused by excess neural excitability. Ovid is advancing a pipeline of novel targeted small molecule candidates that modulate the intrinsic and extrinsic factors involved in neuronal hyperexcitability causative of multiple neurological and neuropsychiatric disorders.

Ovid Therapeutics Inc.是一家总部位于纽约的生物制药公司，致力于开发针对大脑疾病和由神经过度兴奋引起的症状的小分子药物。Ovid正在推进一系列新型靶向小分子候选药物的管线，这些药物可调节导致多种神经和神经精神疾病的神经元过度兴奋的内在和外在因素。

Ovid is developing: OV329, a next-generation GABA-aminotransferase inhibitor, as a potential therapy for treatment-resistant seizures and other undisclosed indications; and is developing OV4071 and other candidates within a library of compounds that directly activate the KCC2 transporter for multiple CNS disorders.

Ovid 正在开发：OV329，一种下一代 GABA-氨基转移酶抑制剂，作为治疗耐药性癫痫发作及其他未公开适应症的潜在疗法；并正在开发 OV4071 及其他化合物库中的候选药物，这些化合物可直接激活 KCC2 转运蛋白，用于多种中枢神经系统疾病。

For more information about these and other Ovid research programs, please visit .

有关这些以及其他 Ovid 研究计划的更多信息，请访问 。

www.ovidrx.com

www.ovidrx.com

.

。

Forward-Looking Statements

前瞻性声明

This press release includes certain disclosures by Ovid that contain “forward-looking statements” including, without limitation: statements regarding the expected timing of initiation, completion, and results and data of Ovid’s ongoing and planned clinical studies; the potential use and development of OV329, OV4071 and other compounds from Ovid’s library of direct activators of KCC2; the potential therapeutic opportunity of OV329, OV4071 and other compounds from Ovid’s library of direct activators of KCC2; Ovid’s clinical pipeline strategy and plans for future clinical studies; the expected timing of IND-enabling and formulation efforts for molecules from its KCC2 direct activator library and related regulatory submissions; and other statements that are not historical fact.

本新闻稿包含Ovid发布的某些“前瞻性声明”，包括但不限于：关于Ovid正在进行和计划中的临床研究的预期启动时间、完成时间及结果和数据的声明；OV329、OV4071以及Ovid KCC2直接激活剂库中其他化合物的潜在用途和开发；OV329、OV4071以及Ovid KCC2直接激活剂库中其他化合物的潜在治疗机会；Ovid的临床管线战略及未来临床研究计划；其KCC2直接激活剂库中分子的IND支持和制剂工作的预期时间及相关监管提交；以及其他非历史事实的声明。

You can identify forward-looking statements because they contain words such as “anticipates,” “believes,” “expects,” “intends,” “may,” “plan,” “potentially,” and “will,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances). Forward-looking statements are based on Ovid’s current expectations and assumptions.

您可以识别前瞻性声明，因为它们包含诸如“预期”、“相信”、“预计”、“打算”、“可能”、“计划”、“潜在地”和“将”等词语，以及类似表达（以及其他引用未来事件、条件或情况的词语或表达）。前瞻性声明基于Ovid当前的预期和假设。

Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance.

由于前瞻性陈述涉及未来，它们受到固有的不确定性、风险和环境变化的影响，这些可能与前瞻性陈述中考虑的情况有重大差异，前瞻性陈述既不是历史事实的陈述，也不是对未来表现的保证或担保。

Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, uncertainties inherent in the preclinical and clinical development and regulatory approval processes, impediments to Ovid’s ability to achieve expected benefits of cost-savi.

可能导致实际结果与前瞻性声明中的结果存在重大差异的重要因素包括但不限于：临床前和临床开发以及监管审批过程中的固有不确定性，阻碍Ovid实现预期成本节约效益的能力。

Contact

联系

Investor Relations & Media

投资者关系与媒体

Victoria Fort

维多利亚堡

VFort@ovidrx.com

VFort@ovidrx.com

202.361.0445

202.361.0445

Source: Ovid Therapeutics Inc.

来源：Ovid Therapeutics Inc.