Ulixacaltamide

BOSTON, Dec. 29, 2025 (GLOBE NEWSWIRE) -- 波士顿，2025年12月29日（环球新闻社）--Praxis Precision Medicines 实践精准医学, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for ulixacaltamide, a differentiated and highly selective small molecule inhibitor of T-type calcium channels, for the treatment of patients with essential tremor (ET).. Inc.（纳斯达克股票代码：PRAX）是一家临床阶段的生物制药公司，致力于将基因洞察转化为针对中枢神经系统 (CNS) 疾病的治疗方法，这些疾病以神经元兴奋-抑制失衡为特征。该公司今天宣布，美国食品药品监督管理局 (FDA) 已授予 ulixacaltamide 突破性疗法认定 (BTD)。Ulixacaltamide 是一种差异化且高度选择性的小分子 T 型钙通道抑制剂，用于治疗原发性震颤 (ET) 患者。“The granting of the Breakthrough Therapy Designation for ulixacaltamide, based on the Essential3 program, further underscores its potential to address the substantial unmet need in patients with ET. We recently completed a series of positive interactions with the FDA, that, together with this BTD, are enabling us to advance this promising treatment faster to patients. “基于Essential3项目，Ulixacaltamide获得突破性疗法认定，进一步突显了其在满足特发性震颤（ET）患者重大未满足需求方面的潜力。我们最近与FDA完成了一系列积极的互动，这些互动连同此次突破性疗法认定，正在使我们能够更快地将这一有前景的治疗方案带给患者。”We are diligently preparing for the filing of the ulixacaltamide NDA, which we expect in early 2026,” said Marcio Souza, president and chief executive officer.. “我们正在为乌利沙卡酰胺的新药申请（NDA）提交做认真准备，预计将在2026年初完成，”总裁兼首席执行官马西奥·苏扎表示。The BTD enables expedited development and regulatory review for drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The BTD for ulixacaltamide was based on the positive topline data from the Essential3 program, consisting of two pivotal Phase 3 studies of ulixacaltamide in ET.. BTD使得针对治疗严重疾病的药物能够加快开发和监管审查，初步临床证据表明，该药物可能在临床上重要的终点上比现有疗法显示出显著的改善。Ulixacaltamide的BTD是基于Essential3项目中的积极顶线数据，该项目包括两项关于Ulixacaltamide在ET中的关键性III期研究。In December 2025, Praxis announced the successful completion of its pre-NDA meeting with the FDA, including the receipt of written feedback and an in-person meeting. The Company has aligned with the FDA on the content of the NDA and expects to submit the ulixacaltamide NDA in early 2026. 2025年12月，Praxis宣布成功完成了与FDA的NDA前会议，包括收到书面反馈和进行面对面会议。公司已就NDA的内容与FDA达成一致，并预计在2026年初提交乌利沙酰胺的NDA申请。About Essential Tremor (ET) 关于原发性震颤 (ET) Essential Tremor is the most common movement disorder, affecting roughly seven million people in the United States alone, representing a multi-billion dollar commercial opportunity. ET is characterized by involuntary rhythmic movement in the upper limbs, with or without tremor in other body locations such as the head, vocal cords, or legs. 特发性震颤是最常见的运动障碍，仅在美国就影响了大约七百万人，代表着一个价值数十亿美元的商业机会。特发性震颤的特点是上肢出现不自主的节律性运动，头部、声带或腿部等其他身体部位可能伴有或不伴有震颤。These tremors significantly disrupt daily living and are progressive in nature, with increases in tremor severity and amplitude commonly observed over the course of the disease. Propranolol, a beta-blocker, is the only approved pharmacotherapy for ET, offering limited efficacy and poor tolerability and is also contraindicated for comorbidities that affect a significant share of the ET population. 这些震颤会显著扰乱日常生活，并且具有进行性特点，通常在病程中观察到震颤严重程度和幅度的增加。普萘洛尔（Propranolol），一种β受体阻滞剂，是唯一获批用于治疗特发性震颤（ET）的药物疗法，但其疗效有限、耐受性差，并且对于影响相当一部分特发性震颤患者的合并症来说是禁忌的。Other beta blockers and anti-convulsants are used off-label, though similarly are characterized by limited efficacy and tolerability. The vast majority of patients are left without a treatment option, with estimated minimum of 2 million patients seeking treatment. In a patient survey, up to 77% of patients felt their ET is inadequately controlled and up to 50% of patients aren’t receiving treatment. 其他β受体阻滞剂和抗惊厥药物也被用于非标签用途，但同样疗效和耐受性有限。绝大多数患者没有治疗选择，估计至少有200万患者在寻求治疗。在一项患者调查中，高达77%的患者认为他们的特发性震颤（ET）未得到充分控制，多达50%的患者未接受任何治疗。Indeed, U.S. neurologists surveyed indicated that 85% of their visits are for patients seeking treatment, and 40% of their patients are not receiving any treatment. These findings underscore the need for more effective treatments for ET.. 事实上，接受调查的美国神经科医生表示，85%的就诊是为了寻求治疗的患者，而40%的患者没有接受任何治疗。这些发现强调了对特发性震颤（ET）更有效治疗的需求。About Ulixacaltamide 关于UlixacaltamideUlixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide is the most advanced program within Praxis’ Cerebrum™ small molecule platform.. 乌利沙卡酰胺是一种差异化且高度选择性的小分子T型钙通道抑制剂，旨在阻断与震颤活动相关的脑小脑-丘脑-皮质（CTC）回路中的异常神经元突发放电。乌利沙卡酰胺是Praxis公司Cerebrum™小分子平台中最先进的项目。About Praxis 关于实践Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis Precision Medicines是一家临床阶段的生物制药公司，致力于将遗传性癫痫的研究成果转化为针对中枢神经系统疾病（以神经元兴奋-抑制失衡为特征）的治疗方法。Praxis通过其专有的小分子平台Cerebrum™和反义寡核苷酸（ASO）平台Solidus™，利用对大脑中共有生物靶点和回路的理解，将基因洞察应用于罕见及更普遍的神经系统疾病的治疗发现与开发。Praxis has established a diversified, multimodal CNS portfolio including multiple programs across epilepsy and movement disorders, with four clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, LinkedIn and Twitter/X.. Praxis建立了一个多元化的、多模式的中枢神经系统（CNS）产品组合，涵盖癫痫和运动障碍的多个项目，拥有四个临床阶段的产品候选药物。欲了解更多信息，请访问www.praxismedicines.com，并在Facebook、LinkedIn和Twitter/X上关注我们。Forward-Looking Statements 前瞻性声明This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the development of Praxis’ product candidates and the anticipated timing of regulatory submissions and interactions, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.. 本新闻稿包含《1995年私人证券诉讼改革法案》及其他联邦证券法律意义下的前瞻性声明，包括明确或隐含的关于Praxis未来预期、计划和前景的声明，特别是关于Praxis产品候选物的开发及预期的监管提交与互动时间的声明，以及其他包含“预期”、“相信”、“继续”、“可能”、“努力”、“估计”、“预计”、“打算”、“或许”、“计划”、“潜力”、“预测”、“计划”、“寻求”、“应该”、“目标”、“将”或“会”等词汇及类似表达的声明，这些均构成《1995年私人证券诉讼改革法案》下的前瞻性声明。The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2024 and as updated in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as well as other filings made with the Securities and Exchange Commission. 本新闻稿中包含的明示或暗示的前瞻性陈述仅为预测，且受多种风险、不确定性和假设的影响，包括但不限于：临床试验固有的不确定性；临床试验、数据读取及其结果的预期时间，以及向监管机构或政府当局提交的审批或审查；进行试验所需的监管批准；以及有关Praxis项目和运营的其他风险，详见其截至2024年12月31日的年度报告Form 10-K，以及截至2025年6月30日的季度报告Form 10-Q中的更新内容，以及其他向证券交易委员会提交的文件。Although Praxis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. 尽管 Praxis 的前瞻性陈述反映了其管理层的善意判断，但这些陈述仅基于 Praxis 目前已知的信息和因素。因此，提醒您不要依赖这些前瞻性陈述。本新闻稿中发布的任何前瞻性陈述仅在其发布之日有效。Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.. 实践不承担公开更新或修改任何前瞻性声明的义务，无论是否由于新信息、未来发展或其他原因。Source: Praxis Precision Medicines 来源：Praxis Precision MedicinesWhatever your topic of interest, 无论你感兴趣的话题是什么，subscribe to our newsletters 订阅我们的新闻通讯to get the best of Drugs.com in your inbox. 获取 Drugs.com 的最新资讯到您的邮箱。