Axsome Therapeutics 宣布发作性睡病药物 AXS-12 的 3 期阳性结果

Axsome Therapeutics 的 3 期 SYMPHONY 试验显示 AXS-12（瑞波西汀）在减轻嗜睡症症状方面取得了积极成果。与安慰剂 (66%) 相比，AXS-12 显着减少了 83% 的猝倒发作，并且 33% 的患者获得了缓解。白天过度嗜睡的严重程度和认知功能也得到改善。 AXS-12 表现出良好的安全性，并伴有常见的轻度至中度不良事件。该药物的孤儿药资格和截至 2039 年的专利保护在 FDA 批准后提供潜在的排他性。

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

Axsome Therapeutics 报告发作性睡病药物 AXS-12 的 3 期阳性结果

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.

纽约，2023 年 3 月 8 日 - Axsome Therapeutics, Inc.（纳斯达克股票代码：AXSM）是一家专注于开发和商业化中枢神经系统疾病新疗法的生物制药公司，今天宣布其关键 3 期 SYMPHONY 试验评估的积极顶线结果AXS-12（瑞波西汀）用于治疗发作性睡病。

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.

随机、双盲、安慰剂对照 SYMPHONY 试验招募了 90 名发作性睡病患者。参与者接受为期五周的 AXS-12 或安慰剂治疗。主要终点是每周猝倒发作相对于基线的变化，这是由强烈情绪引发的肌张力突然丧失，这是发作性睡病的标志症状。

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 达到了主要终点，表明与安慰剂相比，第 5 周每周猝倒发作的统计显着减少。 AXS-12 组相对于基线的平均减少量为 83%，而安慰剂组为 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).

白天过度嗜睡（EDS）（发作性睡病的另一个核心症状）也得到了改善。接受 AXS-12 治疗的患者在 EDS 严重程度的临床医生总体印象量表上平均降低了 1.8 分，而安慰剂组则降低了 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。最常见的不良事件是口干、恶心和便秘，其严重程度一般为轻度至中度。该试验报告称，因不良事件而终止的比率较低。

"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 首席执行官 Herriot Tabuteau 医学博士表示：“这些积极的 3 期结果表明 AXS-12 具有显着减少发作性睡病患者的猝倒发作并改善 EDS 的潜力。” “猝倒事件的迅速和大幅减少尤其令人鼓舞，因为这种症状可能会使人衰弱，并且尽管有现有的治疗方法，但通常仍然持续存在。”

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已被美国食品和药物管理局（FDA）授予孤儿药资格，用于治疗发作性睡病。该公司还提交了 AXS-12 的新药申请（NDA），预计将在 2023 年下半年收到 FDA 的决定。如果获得批准，AXS-12 将成为超过一个世纪以来首个治疗发作性睡病的新疗法。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 计划在即将召开的科学会议上展示 SYMPHONY 试验的详细结果。该公司还正在进行 AXS-12 的开放标签安全性扩展试验，以进一步评估该药物的长期安全性和有效性。

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 年之前受到已颁发专利的保护，一旦获得 FDA 批准，就有可能在美国获得七年的独家营销权。

About Narcolepsy

关于发作性睡病

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.

发作性睡病是一种慢性神经系统疾病，其特征是白天过度嗜睡 (EDS)、猝倒和其他睡眠相关障碍。 EDS 是一种致残症状，可能导致注意力难以集中、记忆问题以及工作或学校表现受损。猝倒是一种肌肉张力突然丧失的情况，可由强烈的情绪（例如大笑或愤怒）引发。发作性睡病会对受影响的个人及其家庭的生活质量产生重大影响。

About Axsome Therapeutics

关于 Axsome 疗法

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 是一家生物制药公司，致力于开发和商业化中枢神经系统疾病的新型疗法。该公司的产品线包括几种处于临床开发阶段的候选产品，用于治疗抑郁症、偏头痛、嗜睡症和阿尔茨海默病。 Axsome Therapeutics 总部位于纽约市。

Forward-Looking Statements

前瞻性陈述

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.

本新闻稿包含涉及重大风险和不确定性的前瞻性陈述。这些陈述基于当前的预期和假设，可能会发生变化。由于各种因素，包括临床开发和监管审批流程固有的不确定性，实际结果可能与任何前瞻性陈述中预测的结果存在重大差异。 Axsome Therapeutics 不承担更新或修改任何前瞻性陈述的义务。