Axsome Therapeutics宣布 Solriamfetol在成人注意力缺陷多动障碍（ADHD）的 FOCUS三期试验达到主要终点-动脉网

Axsome Therapeutics Announces FOCUS Phase 3 Trial of Solriamfetol in Adults with Attention Deficit Hyperactivity Disorder (ADHD) Achieves Primary Endpoint

Axsome Therapeutics 等信源发布 2025-03-25 19:03

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Demonstrated substantial and statistically significant improvement in ADHD symptoms as measured by the AISRS total score compared to placebo (p=0.039, primary endpoint, 150 mg solriamfetol)

与安慰剂相比，通过AISRS总分测量的ADHD症状表现出显著且统计学上显著的改善（p=0.039，主要终点，150毫克solriamfetol）。

Statistically significant reduction in overall ADHD disease severity as measured by the CGI-S score compared to placebo (p=0.017, key secondary endpoint, 150 mg solriamfetol)

与安慰剂相比，通过CGI-S评分测量的ADHD整体疾病严重程度显著降低（p=0.017，关键次要终点，150 mg solriamfetol）。

Statistically significant rate of clinical response on the AISRS compared to placebo (p=0.024, 150 mg solriamfetol)

与安慰剂相比，AISRS 上的临床反应率具有统计学显著性（p=0.024，150 mg solriamfetol）

Onset of action as early as Week 1 compared to placebo (p=0.036, AISRS, 150 mg solriamfetol)

与安慰剂相比，早在第1周就起效（p=0.036，AISRS，150 mg solriamfetol）

Well tolerated with safety profile generally consistent with prior solriamfetol trials

耐受性良好，安全性与之前的索利安非托尔试验基本一致。

NEW YORK

纽约

，

March 25, 2025

2025年3月25日

(GLOBE NEWSWIRE) --

（环球新闻社）--

Axsome Therapeutics, Inc.

(NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced that the FOCUS Phase 3 trial of solriamfetol in the treatment of attention deficit hyperactivity disorder (ADHD) achieved its primary and key secondary endpoints demonstrating statistically significant improvements in ADHD symptoms and disease severity with solriamfetol compared to placebo.

(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，solriamfetol 在治疗注意力缺陷多动障碍 (ADHD) 的 FOCUS III 期试验中达到了主要和关键次要终点，与安慰剂相比，solriamfetol 在 ADHD 症状和疾病严重程度方面表现出统计学上显著的改善。

The FOCUS study was a randomized, double-blind, placebo-controlled, multicenter, .

FOCUS研究是一项随机、双盲、安慰剂对照、多中心的研究。

U.S.

美国

trial, in which 516 adults with ADHD were randomized to receive solriamfetol 150 mg, solriamfetol 300 mg, or placebo, once daily, for 6 weeks.

试验中，516名成人ADHD患者被随机分配每日一次接受150毫克索利氨非妥、300毫克索利氨非妥或安慰剂，持续6周。

The study achieved the primary endpoint by demonstrating a statistically significant reduction in the Adult ADHD Investigator Symptom Rating Scale (AISRS) total score compared to placebo at Week 6, with mean reductions from baseline of 17.7 points for solriamfetol 150 mg and 14.3 points for placebo (p=0.039).

该研究达到了主要终点，与安慰剂相比，在第6周时，成人ADHD调查者症状评定量表（AISRS）总分显著降低，其中150毫克solriamfetol组较基线平均减少17.7分，而安慰剂组平均减少14.3分（p=0.039）。

Overall, the improvement with solriamfetol at Week 6 represents a 45% mean reduction from baseline in ADHD symptoms. Improvements in the AISRS total score were greater with solriamfetol compared to placebo starting at Week 1 (p=0.036). Clinical response, defined as ≥30% improvement from baseline in the AISRS total score, was achieved by a statistically significantly greater percentage of patients treated with solriamfetol 150 mg (53.5%) compared to those treated with placebo (41.3%) at Week 6 (p=0.024)..

总体而言，第六周时使用索利安非托尔的症状改善表现为ADHD症状较基线平均减少了45%。从第1周开始，与安慰剂相比，索利安非托尔在AISRS总分上的改善更大（p=0.036）。在第6周时，临床反应定义为AISRS总分较基线至少改善30%，使用150毫克索利安非托尔的患者中有53.5%达到了这一标准，相比接受安慰剂的患者只有41.3%，二者之间的差异具有统计学意义（p=0.024）。

The study also achieved the key secondary endpoint by statistically significantly reducing overall ADHD disease severity compared to placebo, as assessed by the Clinical Global Impression of Severity (CGI-S) for ADHD, at Week 6 (p=0.017). Results on the primary and key secondary endpoints for the exploratory 300 mg solriamfetol dose were numerically superior compared to placebo but were not statistically significant..

该研究还通过临床全局印象严重程度量表（CGI-S）评估，在第6周时，与安慰剂相比，显著降低了整体ADHD疾病严重程度，达到了关键的次要终点（p=0.017）。探索性300毫克solriamfetol剂量在主要和关键次要终点的结果数值上优于安慰剂，但不具有统计学显著性。

Gregory Mattingly

格雷戈里·马廷利

, M.D., Associate Clinical Professor of Psychiatry at the

医学博士，精神病学临床副教授

Washington University School of Medicine and President

华盛顿大学医学院院长

的

the American Professional Society

美国专业学会

for ADHD and Related Disorders, commented, “ADHD substantially impairs social, academic, and occupational functioning, while negatively impacting patient quality of life and increasing the risk of morbidity and mortality. The results of the FOCUS trial demonstrate that solriamfetol was able to reduce mean ADHD symptom burden by nearly fifty percent, which contributed to significant reductions in disease severity.

针对ADHD及相关障碍，有评论指出：“ADHD会严重损害社交、学术和职业功能，同时负面影响患者的生活质量，并增加患病率和死亡率的风险。FOCUS试验的结果表明，solriamfetol能够将ADHD症状负担平均减少近百分之五十，从而显著减轻疾病严重程度。

These results are especially promising as part of a comprehensive wellness plan for individuals with ADHD. The symptom improvements observed with solriamfetol were accompanied by a favorable safety and tolerability profile. Based on these compelling data, solriamfetol has the potential to be an important new treatment option for adult patients living with ADHD.”.

这些结果作为针对ADHD患者的综合健康计划的一部分尤其令人鼓舞。使用索利氨呋妥观察到的症状改善伴随着良好的安全性和耐受性特征。基于这些令人信服的数据，索利氨呋妥有潜力成为成年ADHD患者的重要新治疗选择。"

Herriot Tabuteau

埃里奥·塔布托

, MD, Chief Executive Officer of Axsome, said, “We are pleased with the positive results of the FOCUS trial which provide the first evidence from a multicenter controlled trial of the efficacy of solriamfetol in the treatment of ADHD. ADHD is a serious, heterogenous, and prevalent condition. We look forward to advancing the development of solriamfetol as a new, differentiated potential treatment for the millions of patients living ADHD.

Axsome首席执行官MD表示：“我们对FOCUS试验的积极结果感到满意，这些结果提供了首个多中心对照试验的数据，证明了solriamfetol治疗ADHD的疗效。ADHD是一种严重、异质性强且普遍存在的疾病。我们期待推进solriamfetol的开发，为数百万ADHD患者提供一种新的、差异化的潜在治疗方案。”

With these results in the adult population in hand, we plan to initiate a trial in pediatric patients this year.”.

有了这些成年人群的研究结果在手，我们计划在今年开始针对儿科患者的试验。

Solriamfetol was safe and well tolerated in the trial, with a side effect profile that was consistent with the established safety profile of solriamfetol. Rates of adverse events were dose dependent. There were no serious adverse events reported in the trial.

在试验中，Solriamfetol 是安全且耐受性良好的，其副作用特征与已确立的 Solriamfetol 安全性特征一致。不良事件的发生率呈剂量依赖性。试验中未报告严重不良事件。

About the FOCUS Trial

关于FOCUS试验

FOCUS (Forward Treatment of Attention Deficit and Hyperactivity Using Solriamfetol) was a Phase 3, randomized, double-blind, placebo-controlled, multicenter, 6-week, parallel group trial to evaluate the efficacy and safety of solriamfetol in adults with ADHD in

FOCUS（使用索利安非托尔对注意力缺陷和多动障碍进行前瞻性治疗）是一项为期6周的第三阶段、随机、双盲、安慰剂对照、多中心、平行组试验，旨在评估索利安非托尔在成人ADHD患者中的疗效和安全性。

the United States

美国

. A total of 516 adult patients with a primary diagnosis of ADHD were randomized 1:1:1 to treatment with solriamfetol 150 mg, solriamfetol 300 mg, or placebo, once daily for 6 weeks. The primary endpoint was the change from baseline in the Adult ADHD Investigator Symptom Rating Scale (AISRS) total score at Week 6.

共有516名主要诊断为ADHD的成年患者被随机按1:1:1分配至每日一次服用150毫克索利安非托尔、300毫克索利安非托尔或安慰剂组，持续6周。主要终点是第6周时成人ADHD研究者症状评定量表（AISRS）总分较基线的变化。

Total scores on the AISRS range from 0 to 54, with 0 corresponding to total absence of symptoms and higher scores corresponding to greater symptom severity. Mean baseline AISRS total scores for the solriamfetol 150 mg, solriamfetol 300 mg, and placebo groups were 39.1, 38.3, and 37.9 respectively. The key secondary endpoint was the change from baseline in the Clinical Global Impression of Severity (CGI-S) for ADHD at Week 6..

AISRS总分范围为0到54，其中0表示完全没有症状，分数越高表示症状越严重。Solriamfetol 150毫克组、solriamfetol 300毫克组和安慰剂组的基线AISRS总分平均值分别为39.1、38.3和37.9。关键的次要终点是第6周时ADHD临床总体印象严重程度（CGI-S）相对于基线的变化。

About Attention Deficit Hyperactivity Disorder

关于注意力缺陷多动障碍

Attention deficit hyperactivity disorder (ADHD) is a chronic neurobiological and developmental disorder characterized by a persistent pattern of inattention, hyperactivity, or impulsivity, that interferes with functioning or development.

注意缺陷多动障碍（ADHD）是一种慢性神经生物学和发育障碍，其特征是持续存在注意力不集中、多动或冲动的行为模式，这种模式会干扰个体的功能或发展。

Impairments in cognition are apparent in attention, planning and problem solving, working memory, and behavioral inhibition.

认知障碍明显表现在注意力、计划与解决问题、工作记忆以及行为抑制方面。

，

An estimated 15.5 million adults and 7 million children in the

据估计，1550万成年人和700万儿童在

U.S.

美国

are affected by ADHD,

受到多动症的影响，

，

with approximately two-thirds or more of children with ADHD continuing to experience symptoms into adulthood.

大约三分之二或更多的ADHD儿童在成年后仍然会出现症状。

The total annual societal excess cost associated with adult ADHD in the

与成人ADHD相关的年度社会总超额成本

U.S.

美国

has been estimated at over

估计已经超过

$120 billion

1200亿美元

。

About Solriamfetol

关于Solriamfetol

Solriamfetol is a dopamine and norepinephrine reuptake inhibitor (DNRI), TAAR1 agonist, and 5-HT

Solriamfetol 是一种多巴胺和去甲肾上腺素再摄取抑制剂 (DNRI)、TAAR1 激动剂和 5-HT

agonist being developed for the treatment of attention deficit hyperactivity disorder (ADHD), major depressive disorder (MDD), binge eating disorder (BED), and excessive sleepiness associated with shift work disorder (SWD).

正在开发用于治疗注意力缺陷多动障碍（ADHD）、重度抑郁症（MDD）、暴食症（BED）以及与轮班工作障碍（SWD）相关的过度嗜睡的激动剂。

About

关于

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.

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差距来实现科学突破，并专注于新型作用机制，开发出差异化产品，从而推动患者治疗效果的显著进步。

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 .

我们行业领先的神经科学产品组合包括FDA批准的用于治疗重度抑郁症、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疗法，并且还有多个后期开发项目，针对影响超过1.5亿人的广泛严重神经和精神疾病。

the 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.

我们一起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。

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”、“打算”、“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.

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例子。前瞻性声明包含风险和不确定性，包括但不限于公司Sunosi的商业成功。

, Auvelity

，Auvelity

, and Symbravo

，以及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 timing of and the Company’s ability to obtain and maintain .

产品以及公司为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的能力；公司正在进行的临床试验及预计进行的临床试验的成功、时间安排和成本，针对公司现有候选产品的临床试验（包括关于试验启动时间、入组进度和完成情况的声明（包括公司能否全额资助其已披露的临床试验，假设公司目前预期的收入或支出没有重大变化））、无效性分析和中期结果的接收，这些结果不一定反映公司正在进行的临床试验的最终结果和/或数据读出结果，以及支持公司现有候选产品中新药申请（“NDA”）所需的研究所数量或类型或结果性质；公司为继续推进候选产品而资助其他临床试验的能力；公司获取和维持的时间和能力。

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.

（“FDA”）或其他监管机构对公司产品候选者的批准或许可，或对其采取的其他行动，包括任何新药申请（NDA）提交时间的声明；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，如果可能的话；公司研发计划和合作的成功实施；公司许可协议的成功；市场对公司产品及产品候选者的接受度（如获批准）；公司预期的资本需求，包括用于Sunosi、Auvelity和Symbravo商业化以及公司其他产品候选者商业发布（如获批准）所需的资本金额及其对公司预期现金跑道的潜在影响；公司将销售转化为确认收入并保持有利的总销售额至净销售额的能力；因国内政治气候、地缘政治冲突或全球疫情而引起或相关的意外情况或其他对正常业务运营的干扰，以及其他因素，包括一般经济状况和非公司可控范围内的监管发展。

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 circumstances..

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

mjacobson@axsome.com

Media:

媒体：

Darren Opland