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Supplemental New Drug Application (sNDA) submission anticipated in 3Q 2025

预计将在2025年第三季度提交补充新药申请（sNDA）。

NEW YORK

纽约

,

，

March 03, 2025

2025年3月3日

(GLOBE NEWSWIRE) --

（环球新闻社）--

Axsome Therapeutics, Inc.

Axsome Therapeutics, Inc.

(NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced that it has received formal pre-New Drug Application (NDA) meeting minutes from the

(NASDAQ: AXSM)是一家引领中枢神经系统（CNS）疾病治疗新时代的生物制药公司，今天宣布已收到正式的新药申请（NDA）前会议纪要。

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) supporting a supplemental NDA (sNDA) submission for AXS-05 in Alzheimer’s disease agitation. AXS-05 (dextromethorphan-bupropion) is a novel, oral, investigational NMDA receptor antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor. The purpose of the meeting was to reach agreement with the FDA on the proposed content and format of the Company’s planned sNDA submission including the clinical and nonclinical requirements..

美国食品药品监督管理局（FDA）支持提交AXS-05的补充新药申请（sNDA），用于治疗阿尔茨海默病患者的激越症状。AXS-05（右美沙芬-安非他酮）是一种新型、口服、在研的NMDA受体拮抗剂、σ-1激动剂和氨基酮类CYP2D6抑制剂。会议的目的是与FDA就公司计划中的sNDA提交内容和格式达成一致，包括临床和非临床要求。

Based on the feedback from the FDA, the Company’s regulatory data package would be sufficient for the submission of an sNDA for AXS-05 for the treatment of Alzheimer’s disease agitation. Axsome anticipates submitting the planned sNDA in the third quarter of 2025. Acceptance of the final sNDA will be subject to the FDA’s review of the complete filing.

基于美国食品药品监督管理局（FDA）的反馈，公司的监管数据包将足以支持提交AXS-05用于治疗阿尔茨海默病躁动的补充新药申请（sNDA）。Axsome预计将于2025年第三季度提交计划中的sNDA。最终sNDA的接受将取决于FDA对完整申报材料的审阅。

AXS-05 was granted Breakthrough Therapy designation for the treatment of Alzheimer’s disease agitation in .

AXS-05 被授予治疗阿尔茨海默病躁动的突破性疗法称号。

June 2020

2020年6月

.

。

“We are pleased with the pre-NDA meeting feedback we received from the FDA, which confirmed the studies and data to be presented in our planned sNDA submission of AXS-05 for the treatment of Alzheimer’s disease agitation,” said

“我们对我们从FDA收到的pre-NDA会议反馈感到满意，该反馈确认了我们计划在AXS-05用于治疗阿尔茨海默病躁动的sNDA提交中呈现的研究和数据，”

Herriot Tabuteau

埃里奥·塔布托

, MD, Chief Executive Officer of Axsome. “Alzheimer’s disease agitation is a serious, distressing, and prevalent condition with limited treatment options. If approved, AXS-05 would represent a new mechanism of action for the treatment of Alzheimer’s disease agitation with the potential to address this high unmet medical need.

，医学博士，Axsome首席执行官。“阿尔茨海默病的激越症状是一种严重、令人痛苦且普遍存在的状况，治疗选择有限。如果获得批准，AXS-05将代表一种新的作用机制，用于治疗阿尔茨海默病的激越症状，并有潜力满足这一高度未满足的医疗需求。

As an FDA Breakthrough Therapy designated program, the application for AXS-05 will be eligible for Priority Review. We look to complete the sNDA submission in the third quarter of 2025, with the goal of making this important new potential therapy available to patients living with Alzheimer’s disease as quickly as possible.”.

作为FDA突破性疗法指定项目，AXS-05的申请将符合优先审查资格。我们计划在2025年第三季度完成sNDA提交，目标是尽快将这一重要的潜在新疗法提供给阿尔茨海默病患者。"

The comprehensive clinical development program of AXS-05 in Alzheimer’s disease agitation includes four completed pivotal, Phase 3, placebo-controlled efficacy trials, and a completed long-term safety trial. AXS-05 demonstrated statistically significant improvement in Alzheimer’s disease agitation, assessed using the Cohen-Mansfield Agitation Inventory (CMAI), compared to placebo, in three Phase 3 efficacy trials: the ADVANCE-1 parallel-group trial (p=0.010), the ACCORD-1 randomized withdrawal trial (p=0.014), and the ACCORD-2 randomized withdrawal trial (p=0.001).

AXS-05针对阿尔茨海默病躁动的综合临床开发计划包括四项已完成的关键性3期安慰剂对照有效性试验，以及一项已完成的长期安全性试验。在三项3期有效性试验中，AXS-05显示出与安慰剂相比，在使用科恩-曼斯菲尔德躁动量表（CMAI）评估的阿尔茨海默病躁动方面取得了统计学上显著的改善：ADVANCE-1平行组试验（p=0.010）、ACCORD-1随机撤药试验（p=0.014）和ACCORD-2随机撤药试验（p=0.001）。

Additional controlled safety and supportive efficacy results are provided by a fourth trial (ADVANCE-2). The long-term safety and tolerability of AXS-05 was also evaluated in more than 300 patients treated for at least 6 months and more than 100 patients treated for at least 12 months. AXS-05 was well tolerated with long-term dosing.

第四项试验（ADVANCE-2）提供了更多的受控安全性和支持性疗效结果。还评估了AXS-05在300多名患者中至少6个月和100多名患者中至少12个月的长期安全性和耐受性。AXS-05在长期给药过程中耐受性良好。

There were no deaths in patients receiving AXS-05 and AXS-05 was not associated with increased risk of falls, sedation, or cognitive decline as assessed by the Mini-Mental State Examination (MMSE)..

接受AXS-05治疗的患者中没有出现死亡病例，且根据简易精神状态检查（MMSE）评估，AXS-05未增加跌倒、镇静或认知能力下降的风险。

About Alzheimer’s Disease Agitation

关于阿尔茨海默病的躁动症状

Alzheimer’s disease (AD) is the most common form of dementia, affecting approximately 7 million people in

阿尔茨海默病（AD）是最常见的痴呆形式，大约影响700万人

the United States

美国

.

。

1

1

Agitation is reported in up to 70% of patients with AD and is characterized by emotional distress, verbal and physical aggressiveness, disruptive irritability, and disinhibition.

据报道，高达70%的AD患者会出现躁动，其特征为情绪困扰、言语和身体攻击性、破坏性易怒以及抑制解除。

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1,

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2

AD agitation has been associated with accelerated cognitive decline, increased caregiver burden, earlier nursing home placement, and increased mortality.

AD躁动与认知能力加速下降、护理人员负担加重、更早进入疗养院以及死亡率升高有关。

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About AXS-05

关于AXS-05

AXS-05 (dextromethorphan-bupropion) is a novel, oral, investigational N-methyl-D-aspartate (NMDA) receptor antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor under development for the treatment of Alzheimer’s disease (AD) agitation and smoking cessation. AXS-05 utilizes a proprietary formulation and dose of dextromethorphan and bupropion, and Axsome’s metabolic inhibition technology, to modulate the delivery of the components.

AXS-05（右美沙芬-安非他酮）是一种新型的、口服的、在研的N-甲基-D-天冬氨酸（NMDA）受体拮抗剂、σ-1激动剂和氨基酮CYP2D6抑制剂，正在开发用于治疗阿尔茨海默病（AD）激越和戒烟。AXS-05利用右美沙芬和安非他酮的专有配方和剂量，并结合Axsome的代谢抑制技术，以调节各成分的递送。

The dextromethorphan component of AXS-05 is an uncompetitive NMDA receptor antagonist, also known as a glutamate receptor modulator, and a sigma-1 receptor agonist. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan and is a norepinephrine and dopamine reuptake inhibitor.

AXS-05的右美沙芬成分是一种非竞争性的NMDA受体拮抗剂，也被称为谷氨酸受体调节剂，以及σ-1受体激动剂。AXS-05的安非他酮成分用于提高右美沙芬的生物利用度，同时也是去甲肾上腺素和多巴胺再摄取抑制剂。

AXS-05 is covered by a robust patent estate extending out to at least 2043. AXS-05 was granted .

AXS-05 拥有一个强大的专利组合，有效期至少延长至 2043 年。AXS-05 已获批准。

U.S.

美国

FDA Breakthrough Therapy designation for the treatment of Alzheimer’s disease agitation in

FDA授予治疗阿尔茨海默病躁动的突破性疗法认定

June 2020

2020年6月

.

。

About

关于

Axsome Therapeutics

Axsome Therapeutics

Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes.

Axsome Therapeutics是一家引领中枢神经系统（CNS）疾病治疗新时代的生物制药公司。我们通过识别护理中的关键缺口，开发具有新颖作用机制的差异化产品，从而实现科学突破，为患者带来有意义的治疗进展。

Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the .

我们行业领先的神经科学产品组合包括FDA批准的治疗重度抑郁症、与嗜睡症和阻塞性睡眠呼吸暂停相关的白天过度嗜睡以及偏头痛的药物，并且还有多个针对广泛严重神经和精神疾病的晚期开发项目，这些疾病影响着超过1.5亿人。

United States

美国

. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit the Company’s website at

。我们共同致力于解决大脑的一些最大问题，以便患者及其亲人能够茁壮成长。欲了解更多信息，请访问公司网站

www.axsome.com

www.axsome.com

.

。

Forward Looking Statements

前瞻性声明

Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements.

本新闻稿中讨论的某些事项属于“前瞻性陈述”。公司在某些情况下可能会使用“预测”、“相信”、“潜在”、“继续”、“估计”、“预期”、“计划”、“意图”、“可能”、“能够”、“也许”、“将”、“应该”等词语来传达未来事件或结果的不确定性，以识别这些前瞻性陈述。

In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the commercial success of the Company’s Sunosi®, Auvelity®, and Symbravo® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the.

特别是，公司关于趋势和潜在未来结果的声明是此类前瞻性陈述的示例。这些前瞻性陈述包含风险和不确定性，包括但不限于公司产品Sunosi®、Auvelity®和Symbravo®的商业成功，以及公司为solriamfetol和/或AXS-05获得任何额外适应症的努力的成功；公司维持和扩大支付方覆盖范围的能力；公司正在进行的临床试验及其预期临床试验的成功、时间安排和成本，包括有关试验启动时间、入组速度和完成情况的声明（包括公司完全资助其已披露临床试验的能力，这假设公司目前预计的收入或支出没有重大变化）、无效性分析及中期结果的接收，这些结果不一定代表公司正在进行的临床试验的最终结果和/或数据读出，以及支持公司当前任何产品候选者提交新药申请（“NDA”）所需的研究数量或类型或结果性质；公司为继续推进其产品候选者提供额外临床试验资金的能力；。

U.S. Food and Drug Administration

美国食品药品监督管理局

(“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; the Company’s ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license agreements; the acceptance by the market of the Company’s products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of Sunosi, Auvelity, and Symbravo and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic and other factors, including general economic conditions and regulatory developments, not within the Company’s control.

美国食品药品监督管理局（“FDA”）或其他监管机构对公司产品候选物的批准或其他相关行动，包括任何新药申请（NDA）提交的时间安排；公司成功捍卫其知识产权或以公司可接受的成本获得必要许可的能力（如果能够获得）；公司研发计划和合作的成功实施；公司许可协议的成功执行；市场对公司产品及其候选产品的接受程度（如获批准）；公司的预期资本需求，包括用于Sunosi、Auvelity和Symbravo商业化以及公司其他候选产品商业化（如获批准）所需的资本金额，以及可能对公司预期现金跑道产生的影响；公司将销售额转化为确认收入并保持有利的总销售额与净销售额比率的能力；由于国内政治气候、地缘政治冲突或全球疫情引发的意外情况或其他对正常业务运营的干扰，以及其他不受公司控制的因素，包括一般经济状况和监管发展。

The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance..

本文讨论的因素可能导致实际结果和发展与这些陈述所表达或暗示的有重大差异。前瞻性陈述仅在本新闻稿发布之日作出，公司不承担公开更新这些前瞻性陈述以反映后续事件或情况的义务。

Investors:

投资者：

Mark Jacobson

马克·雅各布森

Chief Operating Officer

首席运营官

(212) 332-3243

(212) 332-3243

mjacobson@axsome.com

mjacobson@axsome.com

Media:

媒体：

Darren Opland

达伦·奥普兰德

Director, Corporate Communications

企业传播总监

(929) 837-1065

(929) 837-1065

dopland@axsome.com

dopland@axsome.com

References

参考文献

Alzheimer’s Association. 2024 Alzheimer’s Disease Facts and Figures.

阿尔茨海默病协会。2024年阿尔茨海默病事实与数据。

Tractenburg, R.E. et al. Estimating the prevalence of agitation in community-dwelling persons with Alzheimer’s disease. J Neuropsychiatry Clin Neurosci. 2002 Winter;14(1):11-8.

特拉滕堡，R.E. 等。估计社区居住的阿尔茨海默病患者中激越症状的患病率。《神经精神病学与临床神经科学杂志》。2002年冬季；14(1):11-8。

Porsteinsson, A.P. and Antonsdottir, I.M. An update on the advancements in the treatment of agitation in Alzheimer’s disease. Expert Opin Pharmacother. 2017 Apr;18(6):611-620.

Porsteinsson, A.P. 和 Antonsdottir, I.M. 阿尔茨海默病患者躁动治疗进展的最新更新。《专家意见：药物治疗》2017年4月；第18卷第6期：611-620页。

Source: Axsome Therapeutics, Inc.

来源：Axsome Therapeutics, Inc.