Marinus Pharmaceuticals Wraps Phase 3 Trial Enrollment for Tuberous Sclerosis Complex Treatment, Targets New Drug Application in 2025 Following Phase 3 Seizure Treatment Trial
Marinus Pharmaceuticals Wraps Phase 3 Trial Enrollment for Tuberous Sclerosis Complex Treatment, Targets New Drug Application in 2025 Following Phase 3 Seizure Treatment Trial
Marinus Pharmaceuticals, Inc. (NASDAQ:MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced completion of enrollment in the global Phase 3 TrustTSC trial evaluating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) in children and adults.
致力于开发治疗癫痫疾病的创新疗法的制药公司Marinus Pharmicals, Inc.（纳斯达克股票代码：MRNS）今天宣布完成了全球3期TrustTSC试验的注册，该试验评估了口服甘那唑酮用于治疗儿童和成人结节性硬化综合症（TSC）相关的癫痫发作。
"We are pleased to announce we have completed enrollment in our pivotal Phase 3 trial in tuberous sclerosis complex," said Alex Aimetti, Ph.D., Chief Scientific Officer at Marinus Pharmaceuticals. "The TrustTSC study enrolled highly refractory TSC patients experiencing a significant seizure burden despite treatment with multiple antiseizure medications, including the majority of patients having exposure to mTOR inhibitors. We would like to thank the TSC community for their support, trust and participation as we strive to bring a new treatment option to TSC patients."
Marinus Pharmicals首席科学官亚历克斯·艾梅蒂博士说：“我们很高兴地宣布，我们已经完成了结节性硬化综合症的关键3期试验的入组。”“TrustTSC研究招收了尽管接受了多种抗癫痫药物治疗但仍有严重癫痫发作负担的高难治性TSC患者，其中包括大多数接触过mTOR抑制剂的患者。我们要感谢TSC社区在我们努力为TSC患者带来新的治疗选择时给予的支持、信任和参与。”
Joseph Hulihan, M.D., Chief Medical Officer of Marinus added, "Based on the findings in Marinus' Phase 2 TSC trial, the titration schedule of ganaxolone was modified in the Phase 3 TrustTSC trial with the goal of reducing the frequency of somnolence-related adverse events and improving patients' response to treatment. With less than two percent of TrustTSC participants discontinuing due to somnolence-related adverse events, this improvement in tolerability may have the potential to yield meaningful efficacy outcomes in this patient population."
Marinus首席医学官约瑟夫·胡利汉医学博士补充说：“根据马里努斯2期TSC试验的发现，在3期TrustTSC试验中修改了加那索龙的滴定时间表，目的是降低与睡眠相关的不良事件的发生频率并改善患者对治疗的反应。由于不到2％的TrustTSC参与者因与睡眠相关的不良事件而中止治疗，这种耐受性的改善有可能在该患者群体中产生有意义的疗效结果。”
Topline data from the TrustTSC trial is anticipated in the first half of the fourth quarter of 2024. Marinus is targeting submission of a supplemental New Drug Application to the U.S. Food and Drug Administration in April 2025 with priority review requested.
TrustTSC试验的头条数据预计将在2024年第四季度上半年公布。Marinus的目标是在2025年4月向美国食品药品监督管理局提交补充新药申请，并要求进行优先审查。
Marinus also announced that the United States Patent and Trademark Office (USPTO) has granted a new method of use patent (U.S. Patent No. 11,980,625) for ganaxolone in the treatment of TSC, expiring in 2040. This is Marinus' second method of use patent granted for ganaxolone in TSC, further strengthening the Company's intellectual property position.
马里纳斯还宣布，美国专利商标局（USPTO）已批准一项用于治疗TSC的加那索隆的新使用方法专利（美国专利号11,980,625），该专利将于2040年到期。这是Marinus在TSC获得的第二项使用方法专利，进一步巩固了该公司的知识产权地位。