RADNOR, Pa.--(BUSINESS WIRE)--Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that the Phase 3 TrustTSC trial evaluating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) in children and adults did not meet the primary endpoint of percent change in 28-day TSC-associated seizure frequency.

宾夕法尼亚州拉德诺（商业新闻短讯）--致力于开发治疗癫痫发作的创新疗法的制药公司Marinus Pharmaceuticals，Inc.（Nasdaq:MRNS）今天宣布，TrustTSC第三阶段试验评估口服甘纳唑酮治疗儿童和成人结节性硬化症（TSC）相关癫痫发作，未达到28天TSC相关癫痫发作频率百分比变化的主要终点。

While reductions in seizure frequency favored the ganaxolone arm, the primary endpoint did not achieve statistical significance..

虽然癫痫发作频率的降低有利于ganaxolone组，但主要终点没有达到统计学意义。。

“As the first controlled trial in TSC that allowed enrollment of patients taking a range of concomitant antiseizure medications, which included mTOR inhibitors and cannabidiol, these data represent a significant advancement in our understanding of the use of ganaxolone with other standard of care treatments,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.

Marinus董事长兼首席执行官ScottBraunstein医学博士说：“作为TSC的第一个允许患者同时服用一系列抗癫痫药物（包括mTOR抑制剂和大麻二酚）的对照试验，这些数据代表了我们对使用甘纳酮与其他标准护理治疗的理解的重大进步。”。

“We are disappointed that the results of the TrustTSC trial are not likely to be sufficient for an sNDA filing. We are deeply grateful for the contributions of the clinical trial investigators, patients, and caregivers whose support and participation made the study possible.”.

“我们对TrustTSC试验的结果不太可能足以提交sNDA感到失望。我们非常感谢临床试验研究人员，患者和护理人员的贡献，他们的支持和参与使研究成为可能。”。

TrustTSC (NCT05323734) was a global Phase 3 randomized, double-blind, placebo-controlled clinical trial of adjunctive oral ganaxolone treatment in children and adults with TSC-related epilepsy (n=129).

TrustTSC（NCT05323734）是一项全球3期随机，双盲，安慰剂对照临床试验，用于辅助口服甘纳酮治疗TSC相关性癫痫（n=129）的儿童和成人。

Topline data demonstrated that:

Topline数据表明：

The trial did not achieve statistical significance on its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures: median reduction was 19.7% for ganaxolone compared with 10.2% for placebo (p=0.09).

该试验的主要终点TSC相关癫痫发作28天频率降低百分比没有统计学意义：甘纳酮的中位数降低19.7%，而安慰剂的中位数降低10.2%（p=0.09）。

Ganaxolone was generally well-tolerated with a safety profile consistent with previous clinical trials. The most frequent adverse event was somnolence: 28.1% for ganaxolone compared with 16.9% for placebo.

Ganaxolone通常耐受性良好，安全性与以前的临床试验一致。最常见的不良事件是嗜睡：ganaxolone为28.1%，安慰剂为16.9%。

Marinus will continue to support and invest in the commercial growth of ZTALMY® (ganaxolone) oral suspension CV, which is FDA-approved for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older, where more than 200 patients are receiving treatment..

Marinus将继续支持和投资ZTALMY®（ganaxolone）口服混悬液CV的商业增长，该混悬液经FDA批准用于治疗两岁及以上患者的CDKL5缺乏症相关癫痫发作，其中200多名患者正在接受治疗。。

As a result of the TrustTSC outcome, Marinus is discontinuing further ganaxolone clinical development and is taking additional steps to reduce costs, including a reduction in its workforce. The Company has also commenced a process to explore strategic alternatives with the goal of maximizing value for its stockholders.

由于TrustTSC的结果，Marinus正在停止进一步的ganaxolone临床开发，并正在采取其他步骤来降低成本，包括减少劳动力。该公司还开始探索战略替代方案，以实现股东价值最大化。

Marinus has engaged Barclays as an advisor to assist in reviewing its strategic alternatives. There can be no assurance that the exploration of strategic alternatives will result in any agreements or transactions, or as to the timing of any such agreements or transactions. Marinus does not intend to discuss or disclose further developments regarding the exploration of strategic alternatives unless and until its board of directors has approved a definitive action or otherwise determined that further disclosure is appropriate or required by law..

马力努斯聘请巴克莱作为顾问，协助审查其战略替代方案。无法保证战略替代方案的探索会产生任何协议或交易，也无法保证任何此类协议或交易的时间安排。马力努斯不打算讨论或披露有关探索战略替代品的进一步发展，除非董事会批准了最终行动或以其他方式确定进一步披露是适当的或法律要求的。。

About the TrustTSC Trial

关于 “信托信托基金 ”试验

TrustTSC (NCT05323734) was a global Phase 3 randomized, double-blind, placebo-controlled clinical trial of adjunctive oral ganaxolone treatment in children and adults with TSC-related epilepsy. The trial consisted of a four-week baseline period, followed by a 16-week double-blind treatment phase, which included a 28-day titration period followed by a 12-week maintenance period.

TrustTSC（NCT05323734）是一项全球性的3期随机、双盲、安慰剂对照临床试验，旨在对患有TSC相关癫痫的儿童和成人患者进行口服甘纳可龙的辅助治疗。试验包括为期四周的基线期和为期 16 周的双盲治疗期，其中包括 28 天的滴定期和 12 周的维持期。

Participants then had the opportunity to enter the ongoing long-term open-label extension. The primary endpoint was percent change in 28-day TSC-associated seizure frequency. TrustTSC enrolled participants at sites in the U.S., Western Europe, Canada, Israel, Australia and China. To be eligible, participants were required to have inadequate seizure control and have been treated with at least two prior antiseizure medications..

然后，参与者有机会进入正在进行的长期开放标签扩展。。TrustTSC在美国、西欧、加拿大、以色列、澳大利亚和中国的网站招募了参与者。为了符合资格，参与者被要求癫痫发作控制不足，并且已经接受了至少两种先前的抗癫痫药物治疗。。

About Tuberous Sclerosis Complex

关于结节性硬化症

Tuberous sclerosis complex (TSC) is a rare, multisystem genetic disorder caused by inherited mutations in the TSC1 gene or TSC2 gene. It is often characterized by non-cancerous tumors, skin abnormalities, and severe neurological manifestations, including refractory seizures and neurodevelopmental delays.

。它通常以非癌性肿瘤，皮肤异常和严重的神经系统表现为特征，包括难治性癫痫发作和神经发育迟缓。

The condition is a leading cause of genetic epilepsy, often occurring in the first year of life as either focal seizures or infantile spasms. While the disease phenotype can be extremely variable, neurologic manifestations such as epilepsy can be seen in up to 90% of TSC patients..

这种情况是遗传性癫痫的主要原因，通常在生命的第一年发生为局灶性癫痫发作或婴儿痉挛。虽然疾病表型可能变化很大，但在高达90%的TSC患者中可以看到癫痫等神经系统表现。。

About ZTALMY® (ganaxolone) oral suspension

关于ZTALMY®（甘纳索隆）口服混悬液

ZTALMY (ganaxolone) is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects. It is a prescription medicine that has been approved by the U.S. Food and Drug Administration and the European Commission for appropriate patients with CDKL5 deficiency disorder..

ZTALMY（ganaxolone）是一种神经活性类固醇GABAA受体调节剂，作用于已知具有抗癫痫作用的大脑中特征明确的靶标。它是一种处方药，已被美国食品和药物管理局和欧盟委员会批准用于适当的CDKL5缺乏症患者。。

U.S. Prescribing Information for ZTALMY® (ganaxolone) oral suspension CV.

U、 S.ZTALMY®（ganaxolone）口服混悬液CV的处方信息。

European Union Summary of Product Characteristics for ZTALMY.

ZTALMY的欧盟产品特性总结。

About Marinus Pharmaceuticals

关于Marinus Pharmaceuticals

Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company’s product, ZTALMY® (ganaxolone) oral suspension CV, is an FDA-approved prescription medication introduced in the U.S. in 2022. For more information, please visit www.marinuspharma.com and follow us on Facebook, LinkedIn and X..

Marinus是一家商业阶段的制药公司，致力于开发癫痫发作的创新疗法。该公司的产品ZTALMY®（ganaxolone）口服混悬液CV是一种FDA批准的处方药，于2022年在美国推出。有关更多信息，请访问www.marinuspharma.com，并在Facebook、LinkedIn和X上关注我们。。