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.
致力於開發治療癲癇疾病的創新療法的製藥公司Marinus Pharmicals, Inc.（納斯達克股票代碼：MRNS）今天宣佈完成了全球3期TrustTSC試驗的註冊，該試驗評估了口服甘那唑酮用於治療兒童和成人結節性硬化綜合症（TSC）相關的癲癇發作。
"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首席科學官亞歷克斯·艾梅蒂博士說：“我們很高興地宣佈，我們已經完成了結節性硬化綜合症的關鍵3期試驗的入組。”“TrustTSC研究招收了儘管接受了多種抗癲癇藥物治療但仍有嚴重癲癇發作負擔的高難治性TSC患者，其中包括大多數接觸過mTOR抑制劑的患者。我們要感謝TSC社區在我們努力爲TSC患者帶來新的治療選擇時給予的支持、信任和參與。”
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首席醫學官約瑟夫·胡利漢醫學博士補充說：“根據馬裏努斯2期TSC試驗的發現，在3期TrustTSC試驗中修改了加那索龍的滴定時間表，目的是降低與睡眠相關的不良事件的發生頻率並改善患者對治療的反應。由於不到2％的TrustTSC參與者因與睡眠相關的不良事件而中止治療，這種耐受性的改善有可能在該患者群體中產生有意義的療效結果。”
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試驗的頭條數據預計將在2024年第四季度上半年公佈。Marinus的目標是在2025年4月向美國食品藥品監督管理局提交補充新藥申請，並要求進行優先審查。
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.
馬里納斯還宣佈，美國專利商標局（USPTO）已批准一項用於治療TSC的加那索隆的新使用方法專利（美國專利號11,980,625），該專利將於2040年到期。這是Marinus在TSC獲得的第二項使用方法專利，進一步鞏固了該公司的知識產權地位。
譯文內容由第三人軟體翻譯。