Rett syndrome (RTT) is a rare genetic neurodevelopmental disorder mainly affecting female individuals. Trofinetide was recently approved as the first treatment for RTT, largely on the basis of results from the phase 3 LAVENDER trial, in which trofinetide showed improvements in core symptoms of RTT compared with placebo. However, gastrointestinal (GI) symptoms such as diarrhea and vomiting were commonly reported side effects, and taste was also a reported issue. The objective of this article is to describe the perspectives of five caregivers of girls in trofinetide clinical trials as well as those of three nurse trial coordinators, with a focus on management of GI symptoms of trofinetide treatment.Audio Abstract available for this article. Audio Abstract: Jane Lane provides an overview and discusses key findings of the article titled "Managing Gastrointestinal Symptoms Resulting from Treatment with Trofinetide for Rett Syndrome: Caregiver and Nurse Perspectives." (MP4 83274 KB).

2024-02-16·Advances in therapy

Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome.

Article

作者: Mona Darwish ; Julie Passarell ; James M Youakim ; Heather Bradley ; Kathie M Bishop

INTRODUCTION:

Trofinetide was recently approved for the treatment of Rett syndrome (RTT) on the basis of the efficacy and safety findings of the phase 3 LAVENDER study, which used a body weight-based dosing regimen. Exposure-response (E-R) efficacy modeling was used to characterize relationships between trofinetide exposure measures (maximum drug concentration and area under the concentration-time curve for the dosing interval of 0-12 h [AUC_0-12]) and efficacy endpoints in RTT clinical studies to support the trofinetide dosing regimen.

METHODS:

Efficacy endpoints were modeled using trofinetide exposure measures predicted from the population pharmacokinetic model and Bayesian estimates. The analysis population for each E-R model comprised individuals receiving placebo or trofinetide who had available trofinetide exposure measures. Efficacy endpoints were scores from the Rett Syndrome Behaviour Questionnaire (RSBQ), the Clinical Global Impression-Improvement, the Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist (CSBS-DP-IT) Social Composite, and the Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC).

RESULTS:

Higher trofinetide exposure was associated with improvements in RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores. Assuming target trofinetide AUC_0-12 values of 800-1200 μg·h/mL, the reductions in RSBQ total scores at week 12 were approximately five- to seven-fold greater with trofinetide (range 3.55-4.94) versus placebo (0.76). Significant E-R relationships were also found for the CSBS-DP-IT Social Composite and RTT-COMC scores.

CONCLUSION:

E-R efficacy modeling demonstrated significant relationships between trofinetide exposure and RSBQ, CSBS-DP-IT Social Composite, and RTT-COMC scores. Trofinetide is efficacious within the target exposure range, supporting the approved dosing regimen for trofinetide.

TRIAL REGISTRATION:

NCT01703533, NCT02715115, NCT04181723.

2024-02-01·Clinical therapeutics

A Physiologically Based Pharmacokinetic Modeling Approach to Assess the Potential for Drug Interactions Between Trofinetide and CYP3A4-Metabolized Drugs.

Article

作者: Mona Darwish ; James M Youakim ; Inger Darling ; Viera Lukacova ; Joel S Owen ; Heather Bradley

PURPOSE:

Trofinetide is the first drug to be approved by the US Food and Drug Administration for use in the treatment of patients with Rett syndrome, a multisystem disorder requiring multimodal therapies. Cytochrome P450 (CYP) 3A4 metabolizes >50% of therapeutic drugs and is the CYP isozyme most commonly expressed in the liver and intestines. In vitro studies suggest the concentration of trofinetide producing 50% inhibition (IC₅₀) of CYP3A4 is >15 mmol/L; that concentration was much greater than the target clinical concentration associated with the maximal intended therapeutic dose (12 g). Thus, trofinetide has a low potential for drug-drug interactions in the liver. However, there is potential for drug-drug interactions in the intestines given the oral route of administration and expected relatively high concentration in the gastrointestinal tract after dose administration.

METHODS:

Using a validated physiologically based pharmacokinetic (PBPK) model, deterministic and stochastic simulations were used for assessing the PK properties related to exposure and bioavailability of midazolam (sensitive index substrate for CYP3A4) following an oral (15 mg) or intravenous (2 mg) dose, with and without single-dose and steady-state (12 g) coadministration of oral trofinetide.

FINDINGS:

Following coadministration of intravenous midazolam and oral trofinetide, the PK properties of midazolam were unchanged. The trofinetide concentration in the gut wall was >15 mmol/L during the first 1.5 hours after dosing. With the coadministration of oral midazolam and trofinetide, the model predicted increases in fraction of dose reaching the portal vein, bioavailability, C_max, and AUC_inf of 30%, 30%, 18%, and 30%, respectively.

IMPLICATIONS:

In this study that used a PBPK modeling approach, it was shown that CYP3A4 enzyme activity in the liver was not affected by trofinetide coadministration, but trofinetide was predicted to be a weak inhibitor of intestinal CYP3A4 metabolism after oral administration at therapeutic doses.

项与曲非奈肽相关的新闻（医药）

2024-04-02

·药明康德

阿尔茨海默病、帕金森病……研发格局一览

▎药明康德内容团队编辑随着Leqembi获批为阿尔茨海默病（AD）治疗领域带来的显著进展，神经病学（Neurology）领域近年来受到业界的高度关注。这一趋势不仅促进了大型交易的频繁发生，也推动了临床研究积极进展。近日，行业媒体Evaluate发布了一份行业报告，深入分析了神经病学领域内的交易动态和研发趋势。在今天的文章里，药明康德内容团队将结合公开资料，与读者分享其中的精彩内容。点击文末“阅读全文/Read more”，即可访问报告原文网址。近年神经病学领域的大型收购与交易趋势神经病学是一门专注于神经系统疾病的医学分支，涵盖了从退行性疾病（如阿尔茨海默病和帕金森病）到癫痫、抑郁症、偏头痛、疼痛管理，乃至众多罕见疾病的广泛领域。相较于如癌症这样的高关注度领域，神经病学在过去数十年里获得的投资相对缺乏，这导致了大量未竟医疗需求。这种投资不足的背景，结合持续的精神健康危机和有限的治疗选择，为神经病学领域带来了独特的市场机遇和挑战。图片来源：123RF2022-2023年间，神经病学领域的并购活动活跃，其中较为突出的例子包括百时美施贵宝（Bristol Myers Squibb）以约140亿美元收购Karuna Therapeutics，此举旨在将精神分裂症疗法KarXT（xanomeline-trospium）纳入其产品管线，该药物的新药申请（NDA）已被美国FDA受理，有望在今年获得批准。艾伯维（AbbVie）则在去年12月以约87亿美元收购Cerevel Therapeutics，进一步加强艾伯维在神经病学领域的布局。此外，渤健（Biogen）以约73亿美元收购Reata Pharmaceuticals，获得了其针对弗里德赖希共济失调症的药物Skyclarys（omaveloxolone）。辉瑞（Pfizer）公司也加大了在神经病学领域的投入，于2022年以总计约116亿美元收购Biohaven Pharmaceutical，增强了其偏头痛治疗产品组合。这一系列并购案例不仅展示了大型医药企业在神经病学领域的布局，也凸显了该领域积极的发展前景。尽管过去神经病学曾经面临投资不足的挑战，但如今它正吸引着大型药企的集中关注和大规模投资。▲2014-2024年间，神经病学领域內＞10亿美元的交易活动（药明康德内容团队制表，数据来源：参考资料[1]）回顾近年来神经病学领域的交易活动，显而易见的一种趋势是医药公司对于“低风险、后期研发阶段项目”的偏好。神经病学药物开发的历程充满了挑战，多个项目在研发过程中曾遭遇了不可预见的挫折，凸显了该领域内药物开发的固有不确定性和高风险。鉴于此，相较于那些仍处于探索初期、前景充满不确定性的新药项目，大部分医药公司更倾向于投资于那些已经进入到后期研发阶段、相对风险更低的项目。这种策略反映了企业对于风险管理的审慎态度，以及在资本投入时寻求更高确定性的倾向，有些公司甚至避免涉足处于中期临床研究阶段项目的风险，倾向于在药物研发进展到更加成熟和风险更可控的阶段进行投资或并购。除了频繁的并购活动外，近年来神经病学领域的融资活动也十分活跃。在2024年3月，Engrail Therapeutics完成了1.57亿美元的B轮融资。而就在不久前，Alto Neuroscience也成功完成了初次IPO交易。2023年9月，Neumora Therapeutics也实现了上市，募集资金高达2.5亿美元。Neumora的主要产品navacaprant目前正处于针对抑郁症的3期临床试验阶段，该公司预计这一关键试验结果将为预定于2025年递交的新药申请提供支持。这些融资活动凸显了产业在神经病学领域的高度活跃性，也展示该领域在医药研发和创新方面的巨大潜力。图片来源：123RF神经病学领域的新药研发趋势随着科技的不断进步以及我们对人脑复杂机制的深入理解，神经病学领域正处于一场创新疗法和药物开发的浪潮中。在此过程中，一些关键趋势尤为突出：首先，药物的选择性变得更加精准，能够特异性地调节特定的神经通路或受体，旨在降低副作用并提升治疗效果。其次，致幻药物在精神疾病治疗中的潜在应用正受到重新评估，特别是在治疗抑郁症和创伤后应激障碍（PTSD）方面。此外，人工智能（AI）技术正助力脑部药物的发现和开发，加速了新药候选分子的筛选和优化过程。同时，对新分子和创新作用机制的探索正不断扩大治疗工具箱，为患者提供更多的治疗选择。这些进展共同推动了神经病学领域的创新和发展，为未来治疗带来新的希望。1.新一代药物更具选择性在开发更有效和安全的中枢神经系统药物方面，药物选择性的提高是一个重要因素。以强生公司（Johnson & Johnson）的aticaprant和Neumora公司的navacaprant为例，这两种药物针对Kappa阿片受体（KORs）的选择性上优于对μ或δ等其他阿片受体亚型，展现了对KORs的高度特异性。这种特性使得这两种药物在治疗重度抑郁症的3期临床试验中表现出潜力，并且还有望扩展到治疗焦虑症和药物滥用等领域。这种对特定受体的高选择性不仅提高了治疗的针对性，也有望减少潜在的副作用。2.重新思考致幻药物的药用价值近来，迷幻药物的医疗用途得到了重新评估和认可。今年2月，美国FDA宣布受理Lykos Therapeutics（前MAPS公益公司）为3，4-亚甲二氧基甲基苯丙胺（MDMA）递交的新药申请，用于治疗PTSD。该申请还获得了FDA授予的优先审评资格，预计审评结果将于今年8月11日之前发布。新闻稿指出，若该药物获批，它将成为第一款MDMA辅助疗法，同时也是首个获批的迷幻药辅助疗法。此外，Compass Pathways公司的在研裸盖菇素（psilocybin，致幻蘑菇中的致幻成分）疗法在2期临床试验中取得了积极结果，目前已进入治疗耐药性抑郁症的3期临床试验。大麻也正式进入医疗领域。例如，Jazz Pharmaceuticals的癫痫发作药物Epidiolex（cannabidiol，大麻的一个主要非致幻成分）在2018年获得FDA的批准上市，治疗两类罕见而严重的癫痫。新闻稿指出，这是首款获美国FDA批准上市的含纯化大麻提取物的新药。人们对致幻剂疗法潜力的兴趣与日俱增，促使FDA在2023年年中起草了指南草案，以支持相关临床研究，这标志着迷幻药物作为治疗手段就正在逐步正规化和科学化。3.AI辅助神经药物开发2024年，Alto Neuroscience成功地募集到1.29亿美元上市。Alto公司以“人工智能驱动的大脑精准医疗”为其核心理念，专注于AI辅助精准药物开发。其成功的融资活动背后是基于该公司主打项目展示出的积极研究数据：公司的ALTO-300项目相关研究显示，采用脑电图（EEG）生物标志物辅助筛选的患者相较于未使用EEG筛选患者获得了更大的益处。在神经病学领域，精准医疗仍处于起步阶段。然而，新兴的测序技术、传感器工具以及更为强大的数据分析能力正助力科学家们深入解析大脑的复杂机制。值得关注的是，2024年初，美国FDA批准了一项名为BrainSee的人工智能临床测试，专门用于阿尔茨海默病。该测试结合大脑成像技术和认知评估，旨在预测患者从轻度认知障碍进展到痴呆症的可能性，标志着人工智能在神经病学领域应用的一大进步。图片来源：123RF4.新靶点，新机制随着基因研究在神经病学领域的深入，新的治疗靶点不断涌现。例如，Denali Therapeutics和渤健合作开发的LRRK2靶向抑制剂BIIB122/DNL151，目前处于治疗帕金森病的2b期试验阶段。另外，Alector Therapeutics正在与艾伯维合作进行一项2b期临床研究，评估其TREM2靶向疗法AL002用以治疗阿尔茨海默病的作用。此外，Alector还与GSK达成合作，共同开发两款progranulin靶向单抗AL001和AL101以治疗神经退行性疾病。Lexeo Therapeutics正在研发的基因疗法LX1001，旨在通过递送APOE2基因—一种具有保护作用的基因——来降低携带纯合APOE4基因患者罹患阿尔茨海默病的风险。该候选药物目前正在进行1/2期临床试验，初步研究数据表明，接受低剂量LX1001治疗的患者脑脊液中的APOE2蛋白表达增加，在超过12个月的随访期内，两名患者的脑脊液中tau蛋白和磷酸化tau蛋白水平与基线相比降低，这两种蛋白是反映阿尔茨海默病患者大脑关键病理变化的生物标志物。图片来源：123RF神经病学领域的弄潮儿在神经病学领域的创新浪潮中，众多公司凭借其独特的技术优势和丰富的产品线，促进了该领域的快速发展，并为治疗神经病学相关疾病提供了更广泛的选择。接下来，我们将介绍几家近年来专注于神经病学领域的公司。Neurocrine BiosciencesNeurocrine Biosciences专注于为患有神经性、神经内分泌性及神经精神疾病的患者开发新型治疗方案。该公司的产品组合多样化，包括已经获得FDA批准的Ingrezza，这是一款已获批用于治疗迟发性运动障碍和亨廷顿病相关舞蹈症的药物。除此之外，Neurocrine还拥有一系列正处于中到后期临床开发阶段的候选疗法。▲Neurocrine Biosciences在研管线（图片来源：Neurocrine Biosciences官网）Acadia PharmaceuticalsAcadia公司已有2款药物获得FDA批准，分别是Nuplazid（pimavanserin）和Daybue（trofinetide）。根据Acadia公司官网，pimavanserin是美国FDA批准的首个用于治疗帕金森病精神病相关幻觉和妄想症状的处方药。值得一提的是，该药物针对精神分裂症的阴性症状的适应症目前正处于3期临床研究中，此前公布的2期试验结果表明，当与现有抗精神病药物联合用药治疗精神分裂症患者时，该药物显著改善了患者在NSA-16量表上的表现。Acadia预计在2024年获得pimavanserin的3期研究的顶线结果。Daybue则是Acadia公司的第二款获批产品，它在2023年3月获得FDA批准用于治疗Rett综合征成人和2岁以上儿童患者，成为FDA批准的首款治疗Rett综合征的药物。▲Acadia Pharmaceuticals在研管线（图片来源：Acadia Pharmaceuticals官网）Alector TherapeuticsAlector公司的研发管线包含3个处在临床阶段的潜在“first-in-class”项目，这些项目吸引了包括GSK和艾伯维在内的大型医药公司的合作。其中，AL001和AL101是与GSK合作开发的两种新型单抗。AL001目前正在进行一项关键性3期临床试验，用于治疗因GRN基因突变而存在额颞叶痴呆（FTD）风险、或患有额颞叶痴呆的患者。而AL101目前则处于2期临床试验阶段，旨在治疗阿尔茨海默病。此外，Alector管线中的AL002是一种针对TREM2的在研人源化单抗，TREM2是散发性AD的一个重要遗传风险因素。该产品由Alector和艾伯维合作开发，目前正在一项2期临床试验中接受检验。▲Alector Therapeutics在研管线（图片来源：Alector Therapeutics官网）渤健渤健在神经病学领域拥有深厚的研发历史和广泛的产品组合，包括19款已经上市的产品，涵盖了多个重要的治疗领域。其中包括首款获得FDA完全批准的阿尔茨海默病疗法Leqembi，以及基于生物标志物加速批准的首款基于生物标志物加速批准的肌萎缩侧索硬化症（ALS）疗法Qalsody（tofersen）。渤健的研发管线中也不乏创新药物，如针对tau蛋白的反义寡核苷酸（ASO）疗法BIIB080，在针对轻度阿尔茨海默病患者的1b期临床试验中展现了积极结果，使其成为首款被证明可以减少tau蛋白聚集、并展现对患者临床结局有利的趋势的tau蛋白靶向药物。此外，渤健还正在开发针对LRRK2的小分子抑制剂BIIB122，展现了其在神经病学领域研发的广度与深度。Denali TherapeuticsDenali Therapeutics致力于开发治疗神经退行性疾病和溶酶体贮积症疗法。Denali拥有多样化的产品管线，其中多个项目已经步入中后期临床开发阶段。包含与渤健合作开发用于治疗帕金森病的LRRK2小分子抑制剂DNL151，与赛诺菲（Sanofi）合作研发用于治疗多发性硬化的RIPK1小分子抑制剂SAR443820/DNL788，以及基于Denali的转运载体平台技术构建的融合蛋白药物DNL310。DNL310可被成功递送到大脑从而缓解II型黏多糖贮积症（MPS II）的中枢神经系统症状。目前，DNL310正在2/3期临床试验中接受评估，Denali公司的目标是在今年完成2/3期临床试验的患者注册。大家都在看▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] EVALUATING: Neurology，Retrieved April 2, 2024 from https://www.evaluate.com/thought-leadership/evaluating-neurology/?utm_source=Eloqua&utm_medium=Email&utm_campaign=202403-CMP-TL-NEUROLOGY&utm_content=EVALPHARMA-EMN-TL-2403GL-NEUROLOGY-DLRT免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

并购申请上市

2024-03-12

·MedCity News

Schizophrenia Trial Failure Ends Acadia’s Efforts to Expand Use of Its Flagship Drug

An Acadia Pharmaceuticals drug that’s already approved for treating psychosis in Parkinson’s disease patients could not beat a placebo in a pivotal schizophrenia study, spelling the end of efforts to develop a medication the company once envisioned as having potential applications across a range of neurological disorders. The clinical trial failure announced Tuesday evaluated the drug, pimavanserin, as a treatment for negative symptoms of schizophrenia. The main goal was to show a change in score according to an assessment used to measure the disease’s symptoms. According to the preliminary results, the pimavanserin arm achieved a numerical change in score that was similar to the change reported in a prior Phase 2 study. But San Diego-based Acadia also said the placebo effect in Phase 3 was higher. Consequently, the score change in the Phase 3 trial was not enough to be statistically significant. Pimavanserin was initially approved in 2016, a regulatory decision that made the drug the first treatment for the hallucinations and delusions experienced by some Parkinson’s patients. In this indication, the once-daily capsule is marketed under the brand name Nuplazid. The Acadia drug is a small molecule designed to bind to 5-HT2A, a serotonin receptor that plays a role in psychosis. Parkinson’s psychosis was just the lead indication for the drug. The company conducted additional clinical trials to support potential label expansion. In 2021, the FDA rejected Acadia’s application for pimavanserin in dementia-related psychosis, pointing to a lack of statistical significance in some patient subgroups. The following year, FDA turned an application for the drug as a treatment for psychosis in Alzheimer’s patients, asking the company to conduct another clinical trial. Acadia instead discussed a resubmission based on additional analyses of existing clinical data. That resubmission was also rejected, leading the company to cease efforts to develop the drug for Alzheimer’s psychosis. The latest failure in schizophrenia marks the last time the company will try to expand pimavanserin’s label beyond Parkinson’s psychosis. “We are disappointed the trial did not meet its primary endpoint given the significant unmet need in patients with negative symptoms of schizophrenia,” Acadia CEO Steve Davis said in a prepared statement. “We will continue to analyze these data with our scientific advisors, but we do not intend to conduct any further clinical trials with pimavanserin.” Nuplazid accounted for $549.2 million in revenue in 2023, a 6.1% increase over sales of the product in 2022, according to Acadia’s financial reports. The company also has a new contributor to revenue, Daybue. A year ago, this drug became the first FDA-approved treatment for Rett syndrome, a rare genetic neurological disorder. In 2023, Daybue accounted for $177.2 million in sales. Acadia reported a cash position of $438.9 million at the end of 2023. In addition to supporting commercialization of Nuplazid and Daybue, the capital will also support development of a pipeline of drug candidates in various stages of development for central nervous system disorders and rare diseases. After the wind down of pimavanserin, Acadia’s next most advanced program is ACP-101, a drug in Phase 3 clinical trials for treating the insatiable appetite caused by the rare disease Prader-Willi syndrome. The pipeline also includes ACP-204, a novel molecule that has reached Phase 2 testing in Alzheimer’s disease psychosis.

临床2期临床3期上市批准财报

2024-03-11

·BioSpace

Acadia Pharmaceuticals Announces Top-Line Results from Phase 3 ADVANCE-2 Trial of Pimavanserin in Negative Symptoms of Schizophrenia

- Study Did Not Meet Primary Endpoint SAN DIEGO--(BUSINESS WIRE)-- Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced top-line results from the Phase 3 ADVANCE-2 trial evaluating pimavanserin for the treatment of negative symptoms of schizophrenia. Pimavanserin did not demonstrate a statistically significant improvement over placebo on the study’s primary endpoint, the change from baseline to week 26 on the Negative Symptom Assessment-16 (NSA-16) total score (-11.8 vs. -11.1; p=0.4825; effect size=0.07). The safety and tolerability pro pimavanserin was consistent with previous clinical trials, showing a low rate of adverse events. “We are disappointed the trial did not meet its primary endpoint given the significant unmet need in patients with negative symptoms of schizophrenia,” said Steve Davis, Acadia’s Chief Executive Officer. “We will continue to analyze these data with our scientific advisors, but we do not intend to conduct any further clinical trials with pimavanserin. We want to thank the patients, their families, and the investigators for their contributions in this important study.” ADVANCE-2 was a 26-week double-blind, randomized, placebo-controlled study of 34 milligram pimavanserin in 454 adult patients with predominant negative symptoms of schizophrenia who had achieved control of positive symptoms with their ongoing antipsychotic treatment. ADVANCE-2 used the NSA-16 scale, designed to measure change on the wide range of predominant negative symptoms that patients experience, which looks at 16 different items in five subscales and covers symptoms such as blunted affect, poor socialization and lack of motivation. The change from baseline observed in the pimavanserin arm of ADVANCE-2 was similar to the change from baseline observed at the 34 milligram dose in ADVANCE-1 (-11.8 vs. -11.6); however, the placebo effect in ADVANCE-2 was higher than the placebo effect observed in ADVANCE-1 (-11.1 vs. -8.5). In ADVANCE-2, pimavanserin was well-tolerated with an adverse event rate of 30.4% compared with 40.3% for the placebo arm. About Acadia Pharmaceuticals Acadia is advancing breakthroughs in neuroscience to elevate life. For 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only FDA-approved drug to treat hallucinations and delusions associated with Parkinson’s disease psychosis and the first and only FDA-approved drug for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on treating, Prader-Willi syndrome, Alzheimer’s disease psychosis and multiple other programs targeting neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at Acadia.com and follow us on LinkedIn and Twitter. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as “intends,” “may,” “will,” “should,” “could,” “would,” “expects,” “plans,” “anticipates,” “believes,” “estimates,” “projects,” “predicts,” “potential” and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this press release, include, but are not limited to, statements about: (i) our clinical development plans related to pimavanserin and (ii) the safety pro pimavanserin. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties, assumptions and other factors include, but are not limited to: our dependency on the continued successful commercialization of NUPLAZID and DAYBUE and our ability to maintain or increase sales of NUPLAZID or DAYBUE; our ability to generate or obtain the necessary capital to fund our clinical development plans; the timing and results of our clinical trials; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission on February 28, 2024, as well as our subsequent filings with the Securities and Exchange Commission from time to time. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law.

临床结果临床3期上市批准

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KEGG	Wiki	ATC	Drug Bank
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研发状态

适应症	最高研发状态	国家/地区	公司
Rett综合征	批准上市	美国更多	ACADIA Pharmaceuticals, Inc. 更多
脆性X综合征	临床2期	美国更多	Neuren Pharmaceuticals Ltd. 更多
脑震荡	终止	美国更多	Neuren Pharmaceuticals Ltd. 更多
创伤性脑损伤	无进展	澳大利亚更多	Neuren Pharmaceuticals Ltd. 更多

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04279314 (CTgov)	临床3期	Rett综合征	154	Trofinetide	(淵鹹夢襯簾顧艱積夢顧) = 衊齋築夢齋襯網範獵蓋鬱鬱鬱觸願鬱選鑰窪醖 (鏇簾築鬱繭蓋夢簾齋觸, 構網齋衊範夢獵鹹製膚 ~ 齋遞襯積夢願顧鹹襯簾) 更多	-	2024-04-11
LAVENDER study (AAN2023)	临床3期	Rett综合征	-	Trofinetide	- 更多	积极	2023-04-25
				Placebo
NCT04181723 (FDA) 人工标引	临床3期	Rett综合征	187	DAYBUE	(齋觸獵壓蓋廠鬱艱廠範) = 網醖簾襯鬱獵壓顧鹹憲顧築繭艱糧構範鏇夢蓋 (窪襯願網觸鏇鏇窪繭餘, 0.08) 更多	积极	2023-03-10
				placebo	(齋觸獵壓蓋廠鬱艱廠範) = 鹽鏇繭襯網觸鹽顧糧繭顧築繭艱糧構範鏇夢蓋 (窪襯願網觸鏇鏇窪繭餘, 0.06) 更多
NCT04181723 (CTgov)	临床3期	Rett综合征	187	Trofinetide (Drug - Trofinetide)	築壓鏇積壓選襯願壓夢(淵夢願壓鹹繭餘鬱廠夢) = 壓鹽襯齋範繭築廠壓膚醖艱築鑰築艱顧夢遞築 (觸鹹範糧範鑰鹽選網築, 窪蓋鏇構襯選鹹簾範顧 ~ 壓糧構構築構積淵選積) 更多	-	2024-04-08
				Placebo (Placebo)	築壓鏇積壓選襯願壓夢(淵夢願壓鹹繭餘鬱廠夢) = 網選鑰範鬱構製醖廠鹹醖艱築鑰築艱顧夢遞築 (觸鹹範糧範鑰鹽選網築, 齋衊簾網蓋簾鏇鹹獵繭 ~ 醖鹽繭餘積憲糧衊壓衊) 更多
AAN2024 人工标引	N/A	Rett综合征	325	Trofinetide	(鏇鬱艱廠願顧繭顧膚憲) = 艱蓋範夢積積鹹廠遞獵鹽廠蓋憲範製積齋餘淵 (蓋願願鬱獵鬱襯齋蓋糧 )	积极	2024-04-13
NCT04988867 (DAFFODIL, AAN2024) 人工标引	临床2/3期	Rett综合征	12	Trofinetide	(夢選鹹餘遞選繭齋願鏇) = Diarrhea (80.0%) and vomiting (53.3%) were the most common TEAEs, all of mild/moderate severity. 蓋遞鏇淵餘獵醖醖餘憲 (觸簾艱蓋繭醖蓋鑰願壓 ) 更多	积极	2024-04-13