/PRNewswire/ -- GRIN Therapeutics, Inc., a leader in the development of therapies to treat serious neurodevelopmental disorders, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation for its investigational drug, radiprodil, for the treatment of GRIN-related neurodevelopmental disorder (NDD).

/PRNewswire/ -- GRIN Therapeutics, Inc. 是一家致力于开发治疗严重神经发育障碍疗法的领先企业，今天宣布美国食品药品监督管理局（FDA）已授予其研究药物 radiprodil 用于治疗 GRIN 相关神经发育障碍（NDD）的孤儿药资格。

Radiprodil is designed as a potent negative allosteric modulator selectively targeting the N-methyl-D-aspartate receptor subtype .

Radiprodil 被设计为一种有效的负变构调节剂，选择性靶向 N-甲基-D-天冬氨酸受体亚型。

(NR2B or GluN2B). The company is on track to initiate a pivotal Phase 3 trial for radiprodil in mid-2025 for the treatment of GRIN-related NDD with GoF mutations and has an ongoing global open-label clinical trial of radiprodil for the treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II..

（NR2B 或 GluN2B）。该公司有望在 2025 年年中启动 radiprodil 的关键 III 期试验，用于治疗携带功能获得性突变的 GRIN 相关神经发育障碍 (NDD)，并且正在进行一项全球开放标签的 radiprodil 临床试验，用于治疗结节性硬化症 (TSC) 和局灶性皮质发育不良 (FCD) II 型。

'We are making rapid progress in our efforts to bring a first-ever treatment for GRIN-related neurodevelopmental disorder to patients. Supported by our promising clinical data, the FDA's decision to grant Orphan Drug designation to radiprodil is the latest milestone in that effort,' said

“我们正在迅速推进为GRIN相关神经发育障碍患者带来首个治疗方案的努力。在我们充满希望的临床数据的支持下，FDA授予radiprodil孤儿药资格的决定是这一努力中的最新里程碑，”

Michael A. Panzara

迈克尔·A·潘扎拉

, MD, MPH, Chief Medical Officer at Neurvati Neurosciences and GRIN Therapeutics. 'As we plan to launch our pivotal Phase 3 clinical trial to evaluate the impact of radiprodil on seizures, behavioral abnormalities and functional outcomes associated with GRIN-related NDD in the coming months, we are very grateful to the patients and their families who continue to support this important research effort.'.

医学博士、公共卫生硕士、Neurvati神经科学和GRIN治疗学首席医学官。 “在我们计划于未来几个月启动关键的III期临床试验，以评估radiprodil对与GRIN相关的神经发育障碍（NDD）引发的癫痫发作、行为异常及功能结果的影响之际，我们非常感谢那些持续支持这项重要研究工作的患者及其家属。”

Orphan Drug designation is designed to support innovation and research that can lead to more new treatments for diseases that affect fewer than 200,000 people in

孤儿药指定旨在支持创新和研究，以期为影响美国不到20万人的疾病带来更多的新疗法。

the United States

美国

. The designation grants drug developers the potential for seven years of market exclusivity for their drug after approval, during which time the FDA will generally not approve an application for the same drug targeting the same disease or condition. In

该指定授予药物开发者在药物获批后长达七年的市场独占期，在此期间，FDA通常不会批准针对相同疾病或病症的同一药物的申请。

February 2025

2025年2月

, the FDA granted radiprodil Breakthrough Therapy designation for the treatment of seizures associated with GRIN-related NDD in patients with GoF mutations.

，FDA授予radiprodil突破性疗法认定，用于治疗与GRIN相关神经发育障碍（NDD）相关的癫痫发作，针对具有GoF突变的患者。

'We look forward to continuing to engage with the FDA through the means offered by Breakthrough Therapy designation, as we advance our Phase 3 program with a goal of bringing radiprodil to patients,' said Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS, Senior Vice President, Development, Regulatory, and Quality at Neurvati Neurosciences and GRIN Therapeutics.

“我们期待通过突破性疗法指定提供的途径继续与FDA保持沟通，同时推进我们的III期项目，目标是将radiprodil带给患者，”Neurvati Neurosciences和GRIN Therapeutics的开发、监管和质量高级副总裁Anne-Marie Li-Kwai-Cheung（MChem，MTOPRA，RAPS）表示。

'We have heard from so many clinicians and patients about the urgent unmet need in the treatment of GRIN-related disorders and our entire team is dedicated to advancing this development program through late-stage clinical research and regulatory review as rapidly as possible.'.

“我们听到了许多临床医生和患者关于GRIN相关疾病治疗中迫切未满足的需求，我们的整个团队致力于尽快通过后期临床研究和监管审阅来推进这一开发项目。”

About GRIN-related neurodevelopmental disorder

关于GRIN相关神经发育障碍

GRIN-related neurodevelopmental disorder is a family of rare, genetically defined pediatric neurodevelopmental disorders caused by mutations in GRIN genes. While symptoms of GRIN-related neurodevelopmental disorder can present as early as infancy, a diagnosis is often not confirmed until age two or later when a child fails to reach developmental milestones.

GRIN相关神经发育障碍是一组罕见的、由GRIN基因突变引起的儿童神经发育障碍。尽管GRIN相关神经发育障碍的症状可能早在婴儿期就会出现，但往往要到孩子两岁或更大时，因未能达到发育里程碑，才会确诊。

Individuals may experience developmental delay, intellectual disabilities, epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties and behavioral problems. There are currently no approved therapies for GRIN-related neurodevelopmental disorder..

个体可能出现发育迟缓、智力障碍、癫痫、肌张力减退、运动障碍、痉挛、进食困难和行为问题。目前尚无针对GRIN相关神经发育障碍的获批疗法。

About Radiprodil

关于Radiprodil

Radiprodil is an investigational, selective and potent negative allosteric modulator of the N-methyl-D-aspartate (NMDA) receptor subtype

拉迪普罗地尔是一种研究性、选择性和强效的N-甲基-D-天冬氨酸（NMDA）受体亚型的负变构调节剂。

(NR2B or GluN2B). In nonclinical studies, radiprodil has been shown to potently and selectively modulate GluN2B. Radiprodil has also demonstrated an antiseizure effect in a number of in vitro and in vivo preclinical seizure and epilepsy models and specifically in models characterized by an enhanced GluN2B NMDA transmission, which can occur with gain-of-function (GoF) mutations in GRIN-related neurodevelopmental disorder.

(NR2B 或 GluN2B)。在非临床研究中，拉迪普迪尔已被证明能够有效且选择性地调节 GluN2B。拉迪普迪尔还在多种体外和体内临床前癫痫发作和癫痫模型中显示出抗癫痫发作的作用，特别是在那些以 GluN2B NMDA 传递增强为特征的模型中，这种增强可能是由 GRIN 相关神经发育障碍中的功能获得（GoF）突变引起的。

In vitro analysis of brain tissues extracted from both tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) lesions has shown enhanced GluN2B NMDA expression supporting the potential ability of radiprodil to control seizures in these conditions..

从结节性硬化症复合体（TSC）和局灶性皮质发育不良（FCD）病灶中提取的脑组织的体外分析显示，GluN2B NMDA表达增强，这支持了radiprodil在这些情况下控制癫痫发作的潜在能力。

About GRIN Therapeutics

关于GRIN治疗学

GRIN Therapeutics is dedicated to the research and development of precision therapeutics for pediatric neurodevelopmental disorders with the goal of bringing hope to patients and caregivers. Late last year, GRIN Therapeutics reported promising topline data from a Phase

GRIN Therapeutics 致力于研究和开发针对儿童神经发育障碍的精准疗法，旨在为患者和护理人员带来希望。去年年底，GRIN Therapeutics 报告了其第二阶段令人鼓舞的初步数据。

/2a clinical trial (the Honeycomb Trial) evaluating radiprodil in GRIN-related neurodevelopmental disorder in patients with GoF variants, leading to the decision to advance to a Phase 3 trial.   The company has an additional ongoing clinical trial to evaluate radiprodil for the potential treatment of tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II.

/2a期临床试验（Honeycomb试验）评估了radiprodil在GRIN相关神经发育障碍伴GoF变异患者中的效果，推动了进入3期临床试验的决定。该公司还有另一项正在进行的临床试验，评估radiprodil用于治疗结节性硬化症（TSC）和局灶性皮质发育不良（FCD）II型的潜力。

GRIN Therapeutics is an affiliate of Neurvati Neurosciences, a portfolio company of Blackstone Life Sciences (BXLS). For more information, please visit .

GRIN Therapeutics 是 Blackstone Life Sciences (BXLS) 旗下投资组合公司 Neurvati Neurosciences 的关联公司。欲了解更多信息，请访问 。

About Neurvati Neurosciences

关于Neurvati神经科学

Neurvati Neurosciences, a portfolio company of Blackstone Life Sciences, identifies and advances the development of high-potential drug candidates across the neuroscience landscape. Neurvati employs a collaborative model that establishes fit-for-purpose affiliate companies, aligning dedicated resources with long-term strategic capital to catalyze innovative treatment options in areas of unmet need.

Neurvati Neurosciences是黑石生命科学旗下的一家投资组合公司，致力于在神经科学领域识别并推动高潜力药物候选品的开发。Neurvati采用协作模式，建立符合特定需求的附属公司，将专用资源与长期战略资本相结合，以在未满足需求的领域催化创新治疗方案。

Neurvati's team of experienced operators and drug developers seeks opportunities to challenge current treatment paradigms and make a difference for patients suffering from a wide range of neurological and psychiatric disorders. For more information, please visit .

Neurvati的经验丰富的操作员和药物开发团队寻求机会挑战当前的治疗模式，为遭受各种神经和精神疾病困扰的患者带来改变。欲了解更多信息，请访问。

About Blackstone Life Sciences

关于黑石生命科学

Blackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical technologies that improve patients' lives and currently has more than .

黑石生命科学是一个行业领先的私人投资平台，有能力在关键生命科学领域内的公司和产品的生命周期中进行投资。通过结合大规模投资与亲力亲为的运营领导，黑石生命科学助力将有前景的新药和医疗技术推向市场，改善患者生活，目前拥有超过 。