排名前五的靶点	数量

AVPR2(血管加压素V2受体)	3
NMDA receptor(谷氨酸[NMDA]受体复合体)	3
PAR-2(蛋白酶活化受体2)	2
Reactive oxygen species(活性氧)	2
5-HT2A receptor(5-羟色胺2A受体)	2

关联

项与 ACADIA Pharmaceuticals, Inc. 相关的药物

Trofinetide

曲非奈肽

靶点

IGF-1R

作用机制

IGF-1R激动剂

在研机构

ACADIA Pharmaceuticals, Inc.

原研机构

Walter Reed Army Institute of Research

在研适应症

Rett综合征

非在研适应症

脑震荡 [+2]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2023-03-10

Pimavanserin tartrate

酒石酸匹莫范色林

靶点

5-HT2A receptor

作用机制

5-HT2A receptor反向激动剂

在研机构

ACADIA Pharmaceuticals, Inc. [+1]

原研机构

ACADIA Pharmaceuticals, Inc.

在研适应症

妄想 [+11]

非在研适应症

躁动 [+7]

最高研发阶段批准上市

首次获批国家/地区

美国

首次获批日期2016-04-29

Carbetocin

卡贝缩宫素

靶点

OXTR

作用机制

OXTR激动剂

在研机构

ACADIA Pharmaceuticals, Inc. [+1]

原研机构

Ferring BV

在研适应症

出血 [+3]

非在研适应症-

最高研发阶段批准上市

首次获批国家/地区-

首次获批日期-

项与 ACADIA Pharmaceuticals, Inc. 相关的临床试验

NCT05999240 / Not yet recruiting临床2期IIT

A Target Engagement Study of Pimavanserin for Behavioral Inflexibility With Open Label Trial for Rigid Rigid-compulsive Behavior in Adolescents and Adults With Autism

This Phase 2 study examines the safety, tolerability, and preliminary efficacy of pimavanserin in individuals with Autism Spectrum Disorder. Male or female participants aged 16 to 40 years of age will be randomized to receive single doses of either placebo or pimavanserin in this randomized, placebo-controlled, cross-over designed study, followed by open label extension.

开始日期2024-09-15

申办/合作机构

The New York State Psychiatric Institute

[+4]

NCT05895513 / Recruiting临床2期IIT

Effect of Pimavanserin on Aggression and Social Cognition.

The investigators are studying how certain drugs can reduce anger outbursts in people with anger problems. In this study the investigators seek to determine if a single 34 mg (two 17 mg tablets) oral dose of the 5-HT2a receptor blocker, pimavanserin, will reduce aggressive responding in individuals with impulsive aggression (Intermittent Explosive Disorder: IED) on a laboratory task that assesses aggression (Taylor Aggression Paradigm: TAP). We will also be examining how this drug impacts hostile social cognition e.g., hostile attribution). If pimvanserin reduces aggression in this study a next step would be a placebo-controlled treatment trial of pimavanserin in study participants with IED. Participation will first involve a remote (e.g., TEAMS) screening session. If potential study participants appear eligible they will come into the lab for an in-person session where participants will complete interviews and questionnaires and have a medical evaluation (including a physical exam, electrocardiogram, and screens for alcohol and drug use). During the next study session, participants will complete a diagnostic interview and a series of questionnaires, all of which can all take place on-line. During the next two sessions (which will be in-person) participants will undergo two (2) study sessions during which study participants will be given a study drug (orally). The drug given, pimavanserin, is currently available and is known to block serotonin receptors thought to be involved in regulating anger. After participants take the study drug, study participants will complete questionnaires and computer tasks for assessment of aggression and of hostile social cognition. Each of these two in-person study sessions will take at least eight (8) hours. A final on-line session will be done to make certain the investigators have all the data required by the study protocol.

开始日期2024-08-01

申办/合作机构

The Ohio State University

[+1]

NCT06194799 / Enrolling by invitation临床3期

A 52-Week, Open-Label Extension Study of ACP-204 in Adults With Alzheimer's Disease Psychosis

This 52-week, open-label extension study is to evaluate the long-term safety and tolerability of ACP-204 in subjects with ADP.

开始日期2024-04-23

申办/合作机构

ACADIA Pharmaceuticals, Inc.

100 项与 ACADIA Pharmaceuticals, Inc. 相关的临床结果

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0 项与 ACADIA Pharmaceuticals, Inc. 相关的专利（医药）

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200

项与 ACADIA Pharmaceuticals, Inc. 相关的文献（医药）

2024-10-01·Med

Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study

Article

作者: Percy, Alan K ; Youakim, James M ; Barrett, Amy M ; An, Di ; Berry-Kravis, Elizabeth M ; Benke, Timothy A ; Glaze, Daniel G ; Marsh, Eric D ; Bishop, Kathie M ; Neul, Jeffrey L

BACKGROUND:

Trofinetide was approved for the treatment of Rett syndrome (RTT) in patients aged ≥2 years based on the results of the 12-week, randomized, phase 3 LAVENDER study. In LILAC, a 40-week, open-label extension study of LAVENDER, trofinetide continued to improve the symptoms of RTT, with a similar safety profile as LAVENDER. Here, we report long-term safety and efficacy results of LILAC-2, a 32-month, open-label extension study.

METHODS:

Females aged 5-22 years who completed LILAC were eligible to enter LILAC-2. Safety and tolerability were assessed with the incidence of adverse events (AEs). Efficacy was assessed with Rett Syndrome Behaviour Questionnaire (RSBQ) and Clinical Global Impression-Improvement (CGI-I) scores. Caregiver interviews explored the patient's experience with RTT and the efficacy of trofinetide during study participation.

FINDINGS:

In total, 77 participants were enrolled in LILAC-2. The most common AEs were diarrhea (53.2%), COVID-19 (27.3%), and vomiting (19.5%). The mean (standard error [SE]) change in RSBQ score from LAVENDER baseline to week 104 of LILAC-2 was -11.8 (2.45). The mean (SE) CGI-I score from LILAC baseline to week 12 of LILAC-2 was 3.1 (0.10). Most caregivers (96%; n = 24/25) were satisfied or very satisfied with the benefits of trofinetide.

CONCLUSIONS:

Long-term treatment with trofinetide continued to improve RTT symptoms, without new safety concerns. Caregivers reported satisfaction with trofinetide related to improvements that were meaningful for their child and themselves.

FUNDING:

The study was supported by Acadia Pharmaceuticals (San Diego, CA, USA). This study was registered at ClinicalTrials.gov: NCT04776746.

2024-09-01·Drugs-Real World Outcomes

Falls and Fractures among Nursing Home Residents Treated with Pimavanserin versus Other Atypical Antipsychotics: Analysis of Medicare Beneficiaries with Parkinson’s Disease Psychosis

Article

作者: Gopal, Daksha ; Rashid, Nazia ; Doshi, Dilesh ; Rajagopalan, Krithika

BACKGROUND:

Reducing falls and fractures remains an important clinical goal in managing older residents with Parkinson's disease psychosis (PDP) in long-term care/nursing home (LTC/NH) settings.

OBJECTIVES:

This analysis examined risk of all-cause falls or fractures among PDP residents on continuous monotherapy with pimavanserin (PIM) versus (i) other atypical antipsychotics (AAPs) [quetiapine (QUE), risperidone (RIS), olanzapine (OLA), aripiprazole (ARI)] and (ii) QUE.

METHODS:

A retrospective analysis of parts A, B, and D claims from a 100% Medicare sample (2013-2019) in LTC/NH settings was conducted. LTC/NH residents in the USA initiating continuous monotherapy (PIM versus other AAPs; PIM versus QUE) for ≥ 6 months between 01 January 2014 and 31 December 2018 were 1:1 propensity score matched (PSM) on 31 variables (age, sex, race, region, and 27 Elixhauser comorbidities). Outcomes included three measures: risks of falls only, fractures only, and falls/fractures during 6-months follow-up. Demographic characteristics were described using chi-square and t-tests. Generalized linear models were used to assess difference in risks of falls/fractures.

RESULTS:

Of 7187 residents, 47.59% (n = 3420) were female and mean age was 78.8 (± 7.75) years. In total, 14% (n = 1005) were on PIM and 86% (n = 6182) were on other AAPs. After PSM, falls only among PIM residents (n = 1005) was 4.58% (n = 46) versus 7.66% (n = 77) for other AAPs (n = 1005) [relative risk (RR) = 0.63 (0.46, 0.86), p < 0.05] and 8.26% (n = 83) for QUE (n = 1005) residents (p < 0.05). Fractures only among PIM residents was 1.39% (n = 14) compared with 2.09% (n = 21) for other AAPs (p = 0.31) and 1.89% (n = 19) for QUE (p = 0.49), respectively. Taken together, falls/fractures among PIM residents were 5.67% (n = 57) versus 9.05% (n = 91) for other AAPs [RR = 0.63 (0.46, 0.86), p < 0.05] and 9.55% (n = 96) for QUE (p < 0.05), respectively.

CONCLUSIONS:

In this analysis of LTC/NH residents with PDP, PIM had a 37% and 41% lower risk of all-cause falls/fractures versus other AAPs and versus QUE, respectively.

2024-09-01·Med

Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study

Article

作者: Percy, Alan K ; An, Di ; Berry-Kravis, Elizabeth M ; Glaze, Daniel G ; Benke, Timothy A ; Marsh, Eric D ; Bishop, Kathie M ; Youakim, James M ; Neul, Jeffrey L

BACKGROUND:

Trofinetide was approved for the treatment of Rett syndrome based on the results of the phase 3, randomized, placebo-controlled, 12-week LAVENDER study. Rett syndrome is a chronic disorder requiring long-term treatment. We report the efficacy and safety results of LILAC, a 40-week, open-label extension study of LAVENDER.

METHODS:

Females with Rett syndrome aged 5-21 years received open-label treatment with trofinetide for 40 weeks. The primary endpoint was long-term safety of trofinetide; secondary endpoints included the change from baseline at week 40 in the Rett Syndrome Behaviour Questionnaire score and the Clinical Global Impression-Improvement score at week 40.

FINDINGS:

Overall, 154 participants were enrolled and treated with trofinetide in LILAC. The most common adverse events in LILAC were diarrhea (74.7%), vomiting (28.6%), and COVID-19 (11.0%). Diarrhea was the most common adverse event leading to treatment withdrawal (21.4%). The Rett Syndrome Behaviour Questionnaire mean score (standard error) improvement from the LAVENDER baseline to week 40 in LILAC was -7.3 (1.62) and -7.0 (1.61) for participants treated with trofinetide and placebo in LAVENDER, respectively. Mean Clinical Global Impression-Improvement scores (standard error) at week 40 rated from the LILAC baseline were 3.1 (0.11) and 3.2 (0.14) for participants treated with trofinetide and placebo in LAVENDER, respectively.

CONCLUSIONS:

Treatment with trofinetide for ≤40 weeks continued to improve symptoms of Rett syndrome. Trofinetide had a similar safety profile in LILAC as in LAVENDER.

FUNDING:

The study was supported by Acadia Pharmaceuticals Inc. (San Diego, CA, USA). This trial was registered at ClinicalTrials.gov (NCT04279314).

209

项与 ACADIA Pharmaceuticals, Inc. 相关的新闻（医药）

2024-10-17

·BioSpace

Acadia Pharmaceuticals to Announce Third Quarter Financial Results on November 6, 2024

Company to host conference call and webcast on Wednesday, November 6, 2024, at 4:30 p.m. Eastern Time SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that it will report third quarter financial results on Wednesday, November 6, 2024, after the close of the U.S. financial markets. Acadia’s management team will also host a conference call and webcast on November 6, 2024, at 4:30 p.m. Eastern Time to discuss financial results and operations. The conference call will be available on Acadia’s website, Acadia.com under the investors section and will be archived there until December 6, 2024. The conference call may also be accessed by registering for the call here . Once registered, participants will receive an email with the dial-in number and unique PIN number to use for accessing the call. About Acadia Pharmaceuticals Inc. Acadia is advancing breakthroughs in neuroscience to elevate life. Since our founding 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 approved drug in the United States and Canada for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on 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 X . Contacts Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858) 261-2872 ir@acadia-pharm.com

上市批准财报

2024-10-17

·Pharmaceutical Technology

Health Canada approves Acadia’s DAYBUE for Rett syndrome

Rett syndrome is a rare neurodevelopmental disorder that predominantly affects females. Credit: SewCreamStudio/Shutterstock. Acadia Pharmaceuticals has received marketing authorisation from Health Canada for DAYBUE (trofinetide) to treat Rett syndrome in adult and paediatric patients aged two years and above. Rett syndrome is a rare neurodevelopmental disorder that predominantly affects females. It is characterised by a significant regression in development after an initial two years of normal growth. The majority of individuals with Rett syndrome will require lifelong, 24-hour care. The authorisation, under the priority review process, marks DAYBUE as the first and only drug approved in the country for this purpose. Acadia CEO Catherine Owen Adams stated: “Health Canada’s authorisation of DAYBUE is a significant milestone for the Rett syndrome community in Canada and another step forward in Acadia’s commitment to increase access to this therapy for patients and their families. We look forward to bringing DAYBUE to Canadian patients with Rett syndrome as soon as possible.” The approval of DAYBUE in Canada is for use in patients who weigh a minimum of nine kilograms. See Also: Health Canada approves Acadia’s DAYBUE for Rett syndrome COPD market expected to grow to $30.8bn in the 7MM by 2033 It was based on the positive outcomes from the Phase III LAVENDER trial. The pivotal study assessed the safety and efficacy of trofinetide against a placebo. 187 girls and young women aged between five and 20 participated in the study. The study’s co-primary endpoints included the change in the total score from baseline in the Rett Syndrome behaviour questionnaire (RSBQ), as reported by caregivers, and the score of clinical global impression-improvement (CGI-I) scale, as evaluated by clinicians, at week 12. The RSBQ is a 45-item scale filled out by caregivers that evaluates symptoms of Rett syndrome, such as hand movements, repetitive behaviours, night-time behaviours and vocalisations. In March 2023, the therapy received approval from the US Food and Drug Administration for treating Rett Syndrome.

临床3期上市批准临床结果优先审批

2024-10-04

·同写意

曙光初现，提前布局下一个“重磅药物摇篮”？

本届大会以“共建创新生态共赢产业未来”为主题，聚焦生物医药出海与国际化创新、创新药投融资、医疗器械等产业发展风向标，打造“高端化、国际化、专业化、立体化”的年度盛会。近日，礼来Kisunla（通用名：Donanemab，多奈单抗）在日本获批用于治疗早期症状性阿尔茨海默病，礼来的阿尔兹海默症药物版图全球化进程加速。暨2023年7月6日，卫材的Leqembi（通用名：Lecanemab，仑卡奈单抗）成为20年来首款获得FDA完全批准的阿尔茨海默病新疗法以来，阿尔茨海默病药物利好频传。除了仑卡奈单抗的全球化进展外，2024年7月FDA更是双批了礼来的多奈单抗Kisunla以及Alpha Cognition公司的Zunveyl（Benzgalantamine，苯加兰他敏）上市。这说明阿尔兹海默症市场近20年的沉寂终被打破。那么，在频频利好之下，投资者们不禁发问阿尔兹海默症市场会成为下一个“重磅炸弹药物”摇篮吗？ 1 潜力无限：足够大的摇篮深度众所周知，年销售额超过10亿美元的上市药物，被称为“重磅炸弹”。自第一个重磅炸弹药物“泰胃美”面世以来，洛赛克、耐信、阿司匹林一直到现今的K药、O药、司美格鲁肽，整个药物史，也可以说是这些“重磅炸弹”药物的江湖史。药物经济价值通常与其对应疾病的治疗价值挂钩。如果把药物看作武林高手，那么对症领域就是他们的门派根基。根据2024年柳叶刀杂志发表的全球疾病负担研究数据GBD 2021相关研究结果，阿尔兹海默症位于2021年全球DALY的前25个3级原因，排名第25位。（注：伤残调整生命年（Disability-Adjusted Life Year，DALY）‌是一种用于量化疾病负担的指标，它表示从发病到死亡所损失的全部健康寿命年，包括因早死所致的寿命损失年和疾病所致伤残引起的健康寿命损失年。）简单做个比较，前25大三级原因中肺癌排名第17位，糖尿病排名第7位。根据药智网文章数据显示，2024上半年全球销售额top100药物中，肿瘤药物占比约46%、而肺癌是其中的第一大癌种。此外，糖尿病药物占比约12%。经过十年的发展，同样跃迁至前25大致命因素的阿尔兹海默症其重要性与治疗价值不言而明。除了极高的治疗价值外，从市场的拓展性来讲，根据WHO的数据，随着社会老龄化，全球痴呆症患者人数预计将从2019年的5500万增加到2050年的1.39亿。预计到2030年，痴呆症相关的诊治费用也将增加一倍以上，从2019年的每年1.3万亿美元增加到2.8万亿美元，而阿尔兹海默症是痴呆症的主要类型。不仅如此，阿尔茨海默病（AD）是一种与年龄正相关的疾病。据文献统计，其在65-74岁人群中发病率约为5%，在75-84岁人群中发病率约为13.1%，在85岁以上人群中发病率约为33.3%。全球人口老龄化预示着目前患有这种疾病的人数将显著增加，并预测未来受影响的人数将激增。2015年至2050年间，世界60岁以上人口的比例将几乎翻一番，从12%增至22%，达到21亿人。结合阿尔兹海默症的治疗价值与市场拓展性来看，这个领域成为重磅药物炸弹摇篮的可能性可谓十拿九稳。但事实上从1906年发现该病至今，仍然未有重磅药物的诞生，原因究竟几何？ 2 药企们的“死亡之谷” 从1906年阿尔兹海默症被发现至今，涌现出了许多关于其发病机制的理论学说，如β淀粉样蛋白假说、Tau蛋白异常修饰学说、线粒体级联假说、遗传因素等。如此多的假说意味着阿尔兹海默症的研发之路并非坦途。根据美国药物生产与研发协会发布的研究报告，2000年到2017年，药企针对AD累计投入的研发费用已超过6000亿美元，但这一时期内仅有4款AD药物获得批准。即使对那些强大的MNC巨头们来说，阿尔兹海默症也是百年难以攻破的魔障。 2010年3月，辉瑞宣布同Medivation联合研制的阿兹海默症候选药物Dimebon（该项目是2008年辉瑞以7亿多美金的高价，从Medivation公司手中获得的），第三期临床试验失败。 2012年8月，强生与辉瑞宣布终止联合研发的Bapineuzumab静脉注射液III期临床研究，原因是该药在III期临床试验中对未携带ApoE4基因的轻度至中度阿尔茨海默病患者在功能恢复上未能达到预期目标。 2016年8月，礼来就已宣布FDA授予了口服BACE抑制剂AZD3293治疗阿尔茨海默氏症临床开发项目的快速通道地位。然而到2016年年底，其III期临床药物Solanezumab却没有达到主要临床终点，宣告失败。 2022年8月2日，罗氏Aβ单抗Crenezumab一项II期临床宣告失败，这项临床在2013年11月登记，研究历时超过8年。同年11月14日其在研的阿尔兹海默症（AD，老年痴呆）药物（ganteneruma）三期临床研究失败。尚未阐明的发病机制、难以测量的治疗效果，都让无数药企对这一块儿可望而不可及的香饽饽长吁短叹。有文献显示在2024年所统计的阿尔茨海默病药物开发管线中，目前有164项试验和127种药物，而2023年有187项试验评估141种AD药物。管线中的NCE数量不成比例地减少（从101个减少到88个，减少了13%）。 2024 年阿尔茨海默病药物开发管线；来源：Alzheimer's disease drug development pipeline: 2024 这也许意味着，很多企业在死亡之谷面前丧失了勇气。并且在过去一年的临床试验里，有10项终止，4项撤回，1项暂停，7项变为“未知”状态，如此高的异常率，也许更是说明了阿尔兹海默症的研发难度。曾有数据显示，阿尔茨海默症的药物研发失败率99.6%，为各领域新药研发失败率之首，与之相比,癌症的药物研究失败率也仅为81%。在如此高的研发失败率面前，阿尔兹海默症药物市场沉寂如此之久似乎也能理解。困境还不止如此，尽管前面我们提到今年阿尔兹海默症药物市场利好频频，但其中的潜在风险仍然值得关注。比如，欧洲药品管理局（EMA）于7月26日表示，应拒绝卫材和渤健阿尔茨海默药Leqembi的上市许可，这与美国FDA对这一药的态度相反。原因是在关键临床试验中，Leqembi仅略减缓了早期阿尔茨海默病患者的认知和功能衰退速度，与安慰剂相比降低了约27%，且可能导致ARIA包括脑肿胀或出血，在极少数情况下可能致命。药效的难以界定与副作用是阿尔兹海默症药物们在上市后同样会面临的问题，可以预见的是在未来很长一段时间里，药企们都在陷在死亡之谷里挣扎。 3 前奏已响：一小步即是一大步绝对潜力的市场与难度值点满的研发困境，摇篮曲还将奏响吗？答案是肯定的。引用批准Leqembi的FDA顾问小组成员西北大学范伯格医学院神经学教授Simuni博士的话，“每个人都在纠结的问题是其临床意义是什么，这绝对是一个很小的改变，但必须放在疾病早期这一背景下考虑。” 从当前FDA最新获批的几种阿尔兹海默症药物来看，其有效性与安全性仍然存疑。但是，在“稀缺法则”的加持下，任何的进步相较于此前的无能为力，其效用都是指数级增加的。以Leqembi为例，根据卫材2024年Q1财报显示，Leqembi在过去三个月内实现增长，三个月销售额约0.41亿美元，同比增长+8785%。其中美国市场收入约0.28亿美元，中国市场销售额约1000万元。并且，卫材预测Leqembi在 2024财年的全球收入将达到约3.7亿美元，在2030年Leqembi全球销售额或将达到70亿美元。在这样强劲的增长预期下，Leqembi作为先行者已经揭开了千亿市场的一角。不仅如此，从研发端口来说，除了近期上市药物所基于的Aβ抗体疗法外，新疗法也是迎来了新曙光。 2024年7月，在阿尔茨海默病协会国际大会（AAIC）上科学家们提到两种全新的疗法： 1、Lomecel-B治疗：Lomecel-B是从年轻健康成人捐献的骨髓中分离出来的多能干细胞和祖细胞，与安慰剂相比，接受Lomecel-B治疗的患者病情恶化速度总体放缓，目前2a期随机、安慰剂对照试验已经完成； 2、激动性抗TREM2抗体治疗：研究显示TREM2的功能丧失变异与AD的风险增加相关，通过TREM2抗体治疗，其可直接调动小胶质细胞的神经保护能力，以广泛对抗多种病理（Aβ蛋白，Tau磷酸化等），目前已进入早期临床试验阶段。此外，近日Cassava Sciences宣布其正在进行的Simufilam第三期临床试验已顺利通过数据和安全监测委员会（DSMB）的第三次中期阶段安全性审查。（Simufilam可通过靶向调节关键蛋白质——突触膜蛋白受体，有望恢复其正常构象，进而减少Aβ沉积，改善神经元通讯，最终缓解认知衰退症状）经clinicaltrials.gov数据统计，2024年至少有48项3期临床试验正在进行中，包括阿卡迪亚制药ACP-204、AriBio的AR1001、Avanir Pharmaceuticals的AVP-786等，新的强效药物可期。综上，在Leqembi作为先行者强力的增长预期以及新疗法的促动下，阿尔兹海默症市场里重磅炸弹药物们的诞生前奏已被吹响。前奏已响，未来可期。从经济与市场的角度来说，相信阿尔兹海默症一定会是未来重磅炸弹药物们的核心摇篮。而从造福人类的角度，对于阿尔兹海默症病人而言，活着的每一天，都意味着失去。一个领域是否能够出现数量众多的重磅药物炸弹，其显著的经济价值固然令人兴奋。但是，其代表着人类将有富有成效的武器抵御这些强大病魔侵扰的事实才更让人尊重其价值。参考文献：（上下滑动查看更多） 1、国际阿尔茨海默病协会 | 2023 年世界阿尔茨海默病报告 2、Global incidence, prevalence, years lived with disability (YLDs), disability-adjusted life-years (DALYs), and healthy life expectancy (HALE) for 371 diseases and injuries in 204 countries and territories and 811 subnational locations,1990–2021: a systematic analysis for the Global Burden of Disease Study 2021 3、Gill Livingston, Jonathan Huntley, Kathy Y Liu, et al. Dementia prevention, intervention, and care: 2024 report of the Lancet standing Commission. The Lancet. Published:July 31, 2024DOI:https://doi.org/10.1016/S0140-6736(24)01296-0 4、《12款阿尔茨海默病在研新药正在中国开展临床！来自礼来、强生、恒瑞医药等公司》，医药观澜 5、Alzheimer's disease drug development pipeline: 2024， https://doi.org/10.1002/trc2.12465 6、《关注政策出台催化创新药产业景气度提升》，东北证券 7、《“重磅炸弹药物”-医药工业兴衰录》，书籍 8、各公司官网 9、《阿尔茨海默病治疗又有新进展！这两个新疗法你知道吗？》，医学界神经病学频道同写意媒体矩阵，欢迎关注↓↓↓

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药物(靶点)	适应症	全球最高研发状态

曲非奈肽 ( IGF-1R )	Rett综合征更多	批准上市
酒石酸匹莫范色林 ( 5-HT2A receptor )	精神分裂症更多	临床3期
ACP-204	阿尔茨海默症更多	临床3期
卡贝缩宫素 ( OXTR )	普拉德-威利综合症更多	临床3期
NNZ-2591 ( IGF-1 )	Rett综合征更多	临床1期