Axsome Therapeutics 宣布發作性睡病藥物 AXS-12 的第 3 期陽性結果

Axsome Therapeutics 的 3 期 SYMPHONY 試驗顯示 AXS-12（瑞波西汀）在減輕嗜睡症症狀方面取得了積極成果。與安慰劑 (66%) 相比，AXS-12 顯著減少了 83% 的猝倒發作，並且 33% 的患者獲得了緩解。白天過度嗜睡的嚴重程度和認知功能也得到改善。 AXS-12 表現出良好的安全性，並伴隨常見的輕度至中度不良事件。該藥物的孤兒藥資格和截至 2039 年的專利保護在 FDA 批准後提供潛在的排他性。

Axsome Therapeutics Reports Positive Phase 3 Results for Narcolepsy Drug AXS-12

Axsome Therapeutics 報告發作性睡病藥物 AXS-12 的第 3 期陽性結果

NEW YORK, March 8, 2023 - Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company focused on developing and commercializing novel therapies for central nervous system disorders, today announced positive top-line results from its pivotal Phase 3 SYMPHONY trial evaluating AXS-12 (reboxetine) for the treatment of narcolepsy.

紐約，2023 年3 月8 日- Axsome Therapeutics, Inc.（NASDAQ：AXSM）是一家專注於開發和商業化中樞神經系統疾病新療法的生物製藥公司，今天宣布其關鍵3 期SYMPHONY試驗評估的積極頂線結果AXS-12（瑞波西汀）用於治療發作性睡病。

The randomized, double-blind, placebo-controlled SYMPHONY trial enrolled 90 patients with narcolepsy. Participants received either AXS-12 or placebo for five weeks. The primary endpoint was the change from baseline in weekly cataplexy attacks, a sudden loss of muscle tone triggered by strong emotions that is a hallmark symptom of narcolepsy.

隨機、雙盲、安慰劑對照 SYMPHONY 試驗招募了 90 名發作性睡病患者。參與者接受為期五週的 AXS-12 或安慰劑治療。主要終點是每週猝倒發作相對於基線的變化，這是由強烈情緒引發的肌張力突然喪失，這是發作性睡病的標誌症狀。

AXS-12 met the primary endpoint, demonstrating a statistically significant reduction in weekly cataplexy attacks at Week 5 compared to placebo. The mean reduction from baseline was 83% in the AXS-12 group versus 66% in the placebo group (p<0.001). Additionally, 33% of patients treated with AXS-12 achieved complete remission of cataplexy by Week 5, compared to 9.5% of patients receiving placebo (p=0.003).

AXS-12 達到了主要終點，顯示與安慰劑相比，第 5 週每週猝倒發作的統計顯著減少。 AXS-12 組相對於基線的平均減少量為 83%，而安慰劑組為 66%（p

Improvements were also observed in excessive daytime sleepiness (EDS), another core symptom of narcolepsy. Patients treated with AXS-12 experienced a mean reduction of 1.8 points on the Clinician Global Impression of Severity scale for EDS, compared to a 0.9-point reduction in the placebo group (p<0.001). Cognitive function, as measured by the Cognitive Function Items of the Functional Outcomes of Sleep Questionnaire, also improved significantly with AXS-12 treatment (p<0.001).

白天過度嗜睡（EDS）（發作性睡病的另一個核心症狀）也得到了改善。接受 AXS-12 治療的患者在 EDS 嚴重程度的臨床醫生總體印象量表上平均降低了 1.8 分，而安慰劑組則降低了 0.9 分（p

The safety profile of AXS-12 was favorable. The most common adverse events were dry mouth, nausea, and constipation, which were generally mild to moderate in severity. The trial reported low rates of discontinuation due to adverse events.

AXS-12 的安全性良好。最常見的不良事件是口乾、噁心和便秘，其嚴重程度一般為輕度至中度。該試驗報告稱，因不良事件而終止的比率較低。

"These positive Phase 3 results demonstrate the potential of AXS-12 to significantly reduce cataplexy attacks and improve EDS in patients with narcolepsy," said Herriot Tabuteau, M.D., Chief Executive Officer of Axsome Therapeutics. "The rapid and substantial reduction of cataplexy events is particularly encouraging, as this symptom can be debilitating and often persists despite existing treatments."

Axsome Therapeutics 執行長 Herriot Tabuteau 醫學博士表示：“這些積極的 3 期結果表明 AXS-12 具有顯著減少發作性睡病患者的猝倒發作並改善 EDS 的潛力。” “猝倒事件的迅速和大幅減少尤其令人鼓舞，因為這種症狀可能會使人衰弱，儘管有現有的治療方法，但通常仍然持續存在。”

AXS-12 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of narcolepsy. The company has also filed for a New Drug Application (NDA) for AXS-12 and expects to receive a decision from the FDA in the second half of 2023. If approved, AXS-12 would be the first new treatment for narcolepsy in over a decade.

AXS-12已被美國食品藥物管理局（FDA）授予孤兒藥資格，用於治療發作性睡病。該公司還提交了AXS-12 的新藥申請（NDA），預計將在2023 年下半年收到FDA 的決定。如果獲得批准，AXS-12 將成為超過一個世紀以來首個治療發作性睡病的新療法。十年。

Axsome Therapeutics plans to present detailed results from the SYMPHONY trial at upcoming scientific meetings. The company is also conducting an ongoing open-label safety extension trial of AXS-12 to further evaluate the long-term safety and efficacy of the drug.

Axsome Therapeutics 計劃在即將召開的科學會議上展示 SYMPHONY 試驗的詳細結果。該公司還正在進行 AXS-12 的開放標籤安全性擴展試驗，以進一步評估該藥物的長期安全性和有效性。

AXS-12 is protected by issued patents until at least 2039, providing the potential for seven years of marketing exclusivity in the United States upon FDA approval.

AXS-12 至少在 2039 年之前受到已頒發專利的保護，一旦獲得 FDA 批准，就有可能在美國獲得七年的獨家行銷權。

About Narcolepsy

關於發作性睡病

Narcolepsy is a chronic neurological disorder characterized by excessive daytime sleepiness (EDS), cataplexy, and other sleep-related disturbances. EDS is a disabling symptom that can lead to difficulty concentrating, memory problems, and impaired performance at work or school. Cataplexy is a sudden loss of muscle tone that can be triggered by strong emotions, such as laughter or anger. Narcolepsy can have a significant impact on the quality of life of affected individuals and their families.

發作性睡病是一種慢性神經系統疾病，其特徵是白天過度嗜睡 (EDS)、猝倒和其他睡眠相關障礙。 EDS 是一種致殘症狀，可能導致注意力難以集中、記憶問題以及工作或學校表現受損。猝倒是一種肌肉張力突然喪失的情況，可由強烈的情緒（例如大笑或憤怒）引發。發作性睡病會對受影響的個人及其家庭的生活品質產生重大影響。

About Axsome Therapeutics

關於 Axsome 療法

Axsome Therapeutics is a biopharmaceutical company dedicated to developing and commercializing novel therapies for central nervous system disorders. The company's pipeline includes several product candidates in clinical development for the treatment of depression, migraine, narcolepsy, and Alzheimer's disease. Axsome Therapeutics is headquartered in New York City.

Axsome Therapeutics 是一家生物製藥公司，致力於開發和商業化中樞神經系統疾病的新型療法。該公司的產品線包括幾種處於臨床開發階段的候選產品，用於治療憂鬱症、偏頭痛、嗜睡症和阿茲海默症。 Axsome Therapeutics 總部位於紐約市。

Forward-Looking Statements

前瞻性陳述

This press release contains forward-looking statements that involve substantial risks and uncertainties. These statements are based on current expectations and assumptions that are subject to change. Actual results could differ materially from those projected in any forward-looking statements due to various factors, including the uncertainties inherent in clinical development and regulatory approval processes. Axsome Therapeutics undertakes no obligation to update or revise any forward-looking statements.

本新聞稿包含涉及重大風險和不確定性的前瞻性陳述。這些陳述基於當前的預期和假設，可能會發生變化。由於各種因素，包括臨床開發和監管審批流程固有的不確定性，實際結果可能與任何前瞻性陳述中預測的結果有重大差異。 Axsome Therapeutics 不承擔更新或修改任何前瞻性陳述的義務。