更新于：2024-11-01

FLNA

更新于：2024-11-01

基本信息

别名

ABP-280、actin binding protein 280、Actin-binding protein 280

+ [15]

简介

Promotes orthogonal branching of actin filaments and links actin filaments to membrane glycoproteins. Anchors various transmembrane proteins to the actin cytoskeleton and serves as a scaffold for a wide range of cytoplasmic signaling proteins. Interaction with FLNB may allow neuroblast migration from the ventricular zone into the cortical plate. Tethers cell surface-localized furin, modulates its rate of internalization and directs its intracellular trafficking (By similarity). Involved in ciliogenesis. Plays a role in cell-cell contacts and adherens junctions during the development of blood vessels, heart and brain organs. Plays a role in platelets morphology through interaction with SYK that regulates ITAM- and ITAM-like-containing receptor signaling, resulting in by platelet cytoskeleton organization maintenance (By similarity). During the axon guidance process, required for growth cone collapse induced by SEMA3A-mediated stimulation of neurons (PubMed:25358863).

关联

项与 FLNA 相关的药物

Sumifilam

靶点

FLNA

作用机制

FLNA modulators [+1]

在研机构

Cassava Sciences, Inc.

原研机构

Cassava Sciences, Inc.

在研适应症

阿尔茨海默症

非在研适应症-

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期1800-01-20

WO2023004096

专利挖掘

靶点

FLNA

作用机制-

在研机构

Cassava Sciences, Inc.

原研机构

Cassava Sciences, Inc.

在研适应症

痴呆

非在研适应症-

最高研发阶段药物发现

首次获批国家/地区-

首次获批日期1800-01-20

PTI-609

靶点

FLNA x μ opioid receptor

作用机制

FLNA modulators [+1]

在研机构-

原研机构

Cassava Sciences, Inc.

在研适应症-

非在研适应症

炎症 [+1]

最高研发阶段无进展

首次获批国家/地区-

首次获批日期1800-01-20

项与 FLNA 相关的临床试验

NCT06390410 / Recruiting临床1期

A Pharmacokinetic Study of Simufilam in Subjects with Impaired Hepatic Function

Evaluate simufilam levels in the blood of hepatically impaired individuals compared to Healthy individuals of similar demographics

开始日期2024-06-26

申办/合作机构

Cassava Sciences, Inc.

NCT06195319 / Completed临床1期

A Phase 1, Open-label Study of the Absorption, Metabolism, and Excretion of [14C]-Simufilam Following a Single Oral Dose in Healthy Male Subjects

To evaluate the absorption, metabolism, and excretion of [14C]-simufilam in healthy male subjects following a single oral administration of 100 mg of (approximately 100 µCi) [14C]-simufilam.

开始日期2024-02-07

申办/合作机构

Cassava Sciences, Inc.

NCT05575076 / Enrolling by invitation临床3期

An Open-label, Long-term Extension Study to Evaluate the Safety and Tolerability of Simufilam 100 Mg Tablets in Participants with Mild to Moderate Alzheimer's Disease

The goal of this open-label extension study is to assess long-term safety and tolerability of simufilam 100 mg in subjects who have completed the RETHINK-ALZ or REFOCUS-ALZ Phase 3 clinical trials.

开始日期2022-11-07

申办/合作机构

Cassava Sciences, Inc.

100 项与 FLNA 相关的临床结果

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100 项与 FLNA 相关的转化医学

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0 项与 FLNA 相关的专利（医药）

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1,388

项与 FLNA 相关的文献（医药）

2024-12-31·Cancer Biology & Therapy

HSPB8-BAG3 chaperone complex modulates cell invasion in intrahepatic cholangiocarcinoma by regulating CASA-mediated Filamin A degradation

Article

作者: Lin, Ronghua ; Wen, Yu ; He, Chao ; Li, Fazhao ; Luo, Cailan ; Shu, Bo

The incidence of intrahepatic cholangiocarcinoma (ICC) is steadily rising, and it is associated with a high mortality rate. Clinical samples were collected to detect the expression of HSPB8 and BAG3 in ICC tissues. ICC cells were cultured and transfected with plasmids that overexpressed or silenced specific genes to investigate the impact of gene expression alterations on cell function. qPCR and Western blot techniques were utilized to measure gene and protein expression levels. A wound healing assay was conducted to assess cell migration ability. The Transwell assay was used to assess cell invasion ability. Co-IP was used to verify the binding relationship between HSPB8 and BAG3. The effects of HSPB8 and BAG3 on lung metastasis of tumors in vivo were verified by constructing a metastatic tumor model. Through the above experiments, we discovered that the expressions of HSPB8 and BAG3 were up-regulated in ICC tissues and cells, and their expressions were positively correlated. The metastatic ability of ICC cells could be promoted or inhibited by upregulating or downregulating the expression of BAG3. Furthermore, the HSPB8-BAG3 chaperone complex resulted in the abnormal degradation of Filamin A by activating autophagy. Increased expression of Filamin A inhibits the migration and invasion of ICC cells. Overexpression of HSPB8 and BAG3 in vivo promoted the lung metastasis ability of ICC cells. The HSPB8-BAG3 chaperone complex promotes ICC cell migration and invasion by regulating CASA-mediated degradation of Filamin A, offering insights for enhancing ICC therapeutic strategies.

2024-12-01·Modern Pathology

Targeted DNA Sequencing in Diagnosis of Malignant Phyllodes Tumors With Emphasis on Tumors With Keratin and p63 Expression

Article

作者: Bean, Gregory R ; Allard, Grace ; Wang, Aihui ; Schwartz, Christopher J ; Chen, Yunn-Yi ; Krings, Gregor ; Rutland, Cooper D ; Peng, Yan ; Ye, Julia ; Vohra, Poonam ; Theparee, Talent ; Hosfield, Elizabeth M

The differential diagnosis of malignant spindle cell neoplasms in the breast most frequently rests between malignant phyllodes tumor (MPT) and metaplastic carcinoma (MBC). Diagnosis of MPT can be challenging due to diffuse stromal overgrowth, keratin (CK) and/or p63 immunopositivity, and absent CD34 expression, which can mimic MBC, especially in core biopsies. Distinction of MPT from MBC has clinical implications, with differences in surgical approach, chemotherapy, and radiation. In this study, we evaluated MPTs (78 tumors, 64 patients) for stromal CK, p63, and CD34 expression and profiled a subset (n = 31) by targeted next-generation DNA sequencing, with comparison to MBC (n = 44). Most MPTs (71%) were CK+ and/or p63+, including 32% CK+ (25/77 focal) and 65% p63+ (32/66 focal, 10/66 patchy, and 1/66 diffuse). Thirty percent of MPTs expressed both CK and p63 (20/66), compared with 95% of MBCs (40/42, P < .001). CK and/or p63 were positive in CD34+ and CD34- MPTs. Recurrent genetic aberrations in MPTs involved TERT, TP53, MED12, CDKN2A, chromatin modifiers, growth factor receptors/ligands, and phosphoinositide-3 kinase (PI-3K) and MAPK pathway genes. Only MED12 (39%, 12/31) and SETD2 (13%, 4/31) were exclusively mutated in MPTs and not MBCs (P < .001 and P = .044, respectively), whereas PIK3R1 mutations were only found in MBCs (37%, 13/35, P < .001). Comparative literature review additionally identified ARID1B, EGFR, FLNA, NRAS, PDGFRB, RAD50, and RARA alterations enriched or exclusively in MPTs vs MBCs. MED12 was mutated in MPTs with diffuse stromal overgrowth (53%, 9/17), CD34- MPTs (41%, 7/17), and CK+ and/or p63+ MPTs (39%, 9/23), including 36% of CD34- MPTs with CK and/or p63 expression. Overall, MED12 mutation and/or CD34 expression were observed in 68% (21/31) MPTs, including 61% (14/23) of CK+ and/or p63+ tumors. Our results emphasize the prevalence of CK and p63 expression in MPTs and demonstrate the diagnostic utility of next-generation DNA sequencing, especially in MPTs with confounding factors that can mimic MBC.

2024-11-01·Radiology Case Reports

A familial case of diffuse periventricular nodular heterotopia identified prenatally: Filamin A defect as the probable cause

Article

作者: Sá, Graça ; Loureiro, Teresa ; Neves, Mariana Tomásio ; Borges, André Luís ; Martins, Guilherme ; Moldovan, Oana

Periventricular nodular heterotopia (PNH) is a neuronal migration defect characterized by the presence of ectopic grey matter nodules adjacent to the walls of the lateral ventricles. The main genetic etiology of PNH are variants in the Filamin A gene (FLNA, MIM #300049), located in the X chromosome. It affects mostly females (embryonic lethality in males), with about 50% of cases inherited from healthy mothers or with a mild phenotype. It is associated with epilepsy (75%-90%), cardiovascular (65%) and pulmonary pathologies (25%). A 28-year-old primigravida was referred for prenatal care in obstetrics department because of personal history of obliterative bronchiolitis. She has a family history of asthma (mother and sister) and adulthood-onset epilepsy (father). The pregnancy was uneventful up to 20 weeks and 3 days when bilateral periventricular irregularities and mega cisterna magna were identified on ultrasound in a female fetus. Neurosonography was performed, which led to the hypothesis of diffuse PNH, supported by MRI. The hypothesis of PNH associated to the FLNA gene was made. Brain MRI on the pregnant woman was requested, which confirmed a similar pattern of PNH. The arrayCGH (PerkinElmer, Prenatal filter 37K) was normal, and whole exome sequencing identified the likely pathogenic c.1554del p.(Val519fs*) variant in the FLNA gene. We present a case of X-linked hereditary PNH that highlights the value of fetal neurosonography in making a putative diagnosis. The diagnosis was supported by MRI in both fetus and mother. The investigation was supplemented by genetic studies, which confirmed the diagnosis.

项与 FLNA 相关的新闻（医药）

2024-10-08

·GBIHealth

4000万美元代价！Cassava Sciences旗下阿尔兹海默病候选药物Simufilam涉嫌论文造假事件持续发酵

药械追踪 No.1 / 基因泰克获得锐格医药下一代CDK抑制剂，8.5亿美金首付款创纪录近日，锐格医药宣布已与罗氏（SW：ROG）子公司基因泰克达成最终购买协议，基因泰克将从锐格医药收购用于治疗乳腺癌的下一代CDK抑制剂产品组合。根据协议条款，锐格医药将获得8.5亿美金首付款，创下了迄今为止中国生物制药公司授权交易预付款金额的最高记录。此外，锐格医药还有资格基于预设里程碑达成情况获得额外付款。基因泰克将负责相关产品的全球临床开发、生产和商业化。锐格医药将继续管理正在进行的两项临床Ⅰ期试验直至结束，罗氏将接手后续开发和生产。 ->点击阅读原文，解锁完整双语新闻 No.2 / 正大天晴10.15亿元获友芝友生物CD3/EpCAM双抗M701中国大陆权益 2024年10月7日，武汉友芝友生物制药股份有限公司（友芝友生物，2496.HK）宣布将CD3/EpCAM双特异性抗体M701的中国大陆权益授予正大天晴。正大天晴将根据研发进展情况，就许可产品向友芝友生物支付约3.15亿元的首付款及研发里程碑款项，并支付最高不超过7亿元的销售里程碑款项，同时按年净销售额的个位数至低双位数百分比向友芝友生物支付分层特权使用费。 M701是友芝友生物自主研发的生物1类新药，拟用于治疗肿瘤引起的恶性胸水和恶性腹水，目前处于临床试验III期，是国内首个自主开发并进入临床试验阶段的CD3/EpCAM双特异性抗体。 ->点击阅读原文，解锁完整双语新闻企业动态 No.1 / 学术不端事件持续发酵，Cassava Sciences支付4000万美元配合调查近日，Cassava Sciences旗下阿尔兹海默病候选药物Simufilam涉嫌论文造假事件持续发酵，继美国司法部、FBI以及学界人士之后，美国证券交易委员会（SEC）也对Cassava Sciences发起声讨。 SEC在9月26日发布的诉状文件显示，Cassava Sciences已同意支付4000万美元来处理Simufilam的II期临床试验涉嫌学术不端的问题；这不代表Cassava Sciences承认或否认虚假陈述，仅代表公司愿意配合SEC调查并采取相应措施。 Simufilam是一种靶向filamin A的小分子药物，旨在通过恢复大脑中支架蛋白filamin A的正常形状和功能，阻断淀粉样蛋白-β42（Aβ42）信号传导，进而延缓阿尔兹海默症病程。此次SEC发布的诉状进一步列举了Simufilam相关研究的5项疑点，具体包括违反双盲原则、未明确利益冲突、Hoau-Yan Wang的实验室缺乏资质、高管谎报临床数据哄抬股价、虚构入组排除标准且研究结果实际上并未达到统计学意义的改善。Cassava Sciences对质疑未置可否；公司仍在推进Simufilam的III期临床研究，并避免了Hoau-Yan Wang的参与。 ->点击阅读原文，解锁完整双语新闻 No.2 / 逾20亿美元！阿斯利康获得石药集团脂蛋白（a）抑制剂全球独家权益 2024年10月7日，石药集团（1093.HK）发布公告称，与阿斯利康订立独家授权协议，将旗下脂蛋白（a）抑制剂YS2302018全球独家开发、制造和商业化权益授予阿斯利康。根据协议，石药集团将获得1亿美元预付款，并有权收取最高3.7亿美元的潜在开发里程碑付款及最高15.5亿美元的潜在销售里程碑付款，以及根据该产品的年度销售净额计算的分层销售提成。作为领先的临床前候选药物，YS2302018是由石药集团AI驱动的小分子药物设计平台发现。该平台利用AI技术分析目标蛋白与现有化合物分子的结合模式，对其成药性进行针对性优化，最终筛选出高效且具有优良开发性的脂蛋白（a）小分子抑制剂。 ->点击阅读原文，解锁完整双语新闻 No.3 / 因生物安全法案，药明康德拟出售部分关键业务根据上周英国金融时报报道，鉴于《生物安全法案》的压力，药明康德（2395.HK）正在寻求出售其位于费城的细胞和基因治疗制造部门WuXi Advanced Therapies，药明生物（2269.HK）则希望出售其在欧洲的部分生产基地。据悉，几个月前药明康德已将其细胞和基因治疗制造部门WuXi Advanced Therapies挂牌出售，该公司在费城运营着四个实验室和生产设施。药明生物正在与顾问合作，评估市场对其部分欧洲生产设施的兴趣。药明生物正在审查出售的资产，最主要的是其从拜耳购买的两处德国生产设施，该公司去年斥巨资扩建了这两处设施。药明生物在爱尔兰也有大型生产设施。但业内消息称，药明尚未确定出售欧洲资产，尽管最近存在地缘政治压力，但全球对药明服务的需求仍普遍存在。 ->点击阅读原文，解锁完整双语新闻行业政策 No.1 / 国家医保局发布长护险专家库管理办法及相关经办规程、协议范本近日，国家医疗保障局发布《长期护理保险专家库管理暂行办法》（医保办发〔2024〕20号，以下简称《暂行办法》），以及《长期护理保险经办规程（试行）》、《长期护理保险定点失能等级评估机构服务协议范本（试行）》、《长期护理保险定点护理服务机构服务协议范本（试行）》等多个文件，为长期护理保险（以下简称“长护险”）制度建设提供指导。《暂行办法》提出，国家医疗保障行政部门负责组建、管理国家长期护理保险专家库。省、地级医疗保障行政部门可参照组建、管理本级专家库，鼓励省级医疗保障行政部门统一组建、管理本地区专家库。各级医疗保障部门开展长期护理保险领域政策研究、意见咨询、课题委托、调查研究、宣传培训等时需要合作的专家，原则上应从本级或上级专家库中选取。《暂行办法》主要对入库条件与程序、专家权利义务、专家库的管理和维护等作出规定。 ▶一是明确入库条件。明确国家库专家分为核心专家和咨询专家，规定了2类组别专家应具备的基本条件。通过公开征选、定向邀请、单位推荐、个人自荐等方式组建和扩展。 ▶二是明确专家权责。确定入库专家应履行受委托开展课题研究与政策咨询、主动做好长期护理保险制度政策宣传、促进相关学科建设等义务，强调了工作、保密、廉洁等方面纪律要求。 ▶三是建立健全专家库管理机制。提出专家库实行准入、退出、评估等动态管理措施，细化了医疗保障行政部门管理职责、专家库清退规则、入库期限等规定。国家医保局将按照《暂行办法》有关规定，开展国家长期护理保险专家库组建工作。 ->点击阅读原文，解锁完整双语新闻 No.2 / CDE公开征求《生物类似药说明书撰写技术指导原则》意见近日，国家药监局药审中心（CDE）发布《生物类似药说明书撰写技术指导原则（征求意见稿）》，旨在通过明确生物类似药说明书撰写的详细技术要求，为医生和患者在用药选择、安全性监测及风险控制方面提供参考。征求意见时限为自发布之日起1个月。生物类似药说明书内容应充分反映参照药的安全性和有效性信息，参照药说明书中安全性和有效性信息可为医患提供所需的必要科学信息，帮助医患根据说明书中对适应证和给药方案等推荐做出临床用药决策。因此，建议在生物类似药说明书中纳入参照药说明书中的相关信息，并对这些信息进行适当修改，以助于临床用药决策，从而确保用药安全和有效，并减少可能的用药错误。 ->点击阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作 ▶ zhangxinyue13@baidu.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

临床2期引进/卖出并购临床1期临床3期

2024-10-06

·循因缉药

Cassava离得谱中谱，方为人上人

环球视野，深度视角各位亲爱的股东们，大家早上好中午好晚上好。国庆七天乐，各位玩的开心吗？其实这篇早就写完了。。。反正也没人看，就算做个总结吧。在我脑子里面，Cassava Sciences是绝对已经上榜过的公司。然而，翻了翻...竟然没有。马上过节了，也不想过多的去深挖这家公司，就大概就着最近的事件聊一聊。大家也可以看下美国人民的语言艺术，那可一点都不比咱们差啊。 #01 渡劫 2024年9月26日，美国上市制药公司Cassava Sciences就一项疏忽信息披露的指控与SEC（美国证券交易委员会）达成和解。该项调查涉及公司就2020年针对该公司AD（ Alzheimer’s disease）的候选药物simufilam的临床二期实验内容（后面会看到），公司卷入其中的两位高级管理员工也一同达成和解。 Cassava既没有承认也没有否认SEC的指控，但是同意支付4000万美元来了结SEC的调查。公司完全配合SEC的调查，并且公司已经采取了补救措施。大家可以猜猜，SEC的指控是真是假。 Cassava还一并告知大家别太兴奋，因为还有个DOJ（司法部）的调查在正在进行，DOJ是否起诉还未可知。渡劫渡了一半？不知道，也许连一半都没有 Cassava由于simuflam的影响，股价从2021年年初的7美元飙升到2021年7月的135美元。人们迫切希望能看到AD在这家公司手上得到根本性的改善，诞生一款重磅炸弹似的药物。尤其，simuflam还是一个小分子口服药物。这一切，直到2021年8月一家名为 Labaton Sucharow向FDA递交请愿书，要求不要批准这款药物的临床试验而陷入舆论漩涡。当然，这不是我们的重点。我们首先需要了解下，这两个高级管理员工是谁呢？ #02 炸裂根据SEC的指控文件显示，分别是以17.5万美元和8.5万美元达成和解的前CEO Remi Barbier和高级副总裁Lindsay H. Burns。 CEO没啥好说的，这位Lindsay Burns是个人才。她不仅是一位神经科学家还是一位运动员，1996年巴塞罗那奥运会Lindsay拿到了双人赛艇的银牌... 是不是有点炸裂？当然还有更炸裂的，这俩人是夫妻.... 如果你觉得这就是尽头了，那就太天真了。 Remi Barbier是一位“成功的”创业者，1998年Remi Barbier创立一家名为Pain Therapeutics的公司。公司核心产品名为Remoxy ER (oxycodone)，该候选药物被描述为一种具有抗滥用特性的羟考酮。随便吃，止疼不上瘾，简直天才的想法。然而，2018年8月6日，美国FDA以14比3的高票...拒绝批准此款候选药物。呃，第四次拒绝。随后，公司股价暴跌。然后在CEO Remi Barbier的带领下，公司决定转型做Alzheimer’s disease药物，他们找了一款候选药物PTI-125。聪明的小伙伴已经猜到了，Pain Therapeutics就是如今的Cassava Sciences。而PTI-125就是如今的Simufilam。就问够不够炸裂...如果觉得还不够炸裂，那么坐稳扶好。 #03 “小分子炸弹” PTI-125（Simufilam）是由Lindsay Burns（Cassava的神经科学高级副总裁）、Hoau-Yan Wang（CUNY教授兼Cassava顾问）和Maya Frankfurt（CUNY）在PLOS One上发表了一篇论文，论文中报告了一种名为filamin A（FLNA）的300-kDa蛋白质与纳洛酮的结合，这种结合可以预防阿片类药物的耐受性和依赖性。作者们声称这是一项关键的发现，即某些阿片类拮抗剂（纳洛酮和纳曲酮）与FLNA的结合。FLNA是一种细胞骨架蛋白，对于维持细胞的形状和分裂至关重要。 Wang和Burns在阿尔茨海默病研究中的一个发现，他们发现FLNA蛋白质在Abeta42与α7烟碱型乙酰胆碱受体结合时起重要作用，并提出了PTI-125作为一种可能的治疗策略。这一发现被发表在The Journal of Neuroscience上，具体机制可以看上面的图，咱也不知道真假，毕竟第一篇论文在2022年被PLOS One撤回，第二篇杂志表达了“concern”。其实也没什么好concern的，这位Wang教授除了拿Cassava的资助以外，还拿了NIH的资助。 Wang教授现年67岁，被控涉嫌提交虚假数据以此获取数以百万计用以研究AD的基金。 2017-2021期间，Wang教授从NIH获得了约1600万美元的资金支持。 1600万美元的基金+数千万美元的临床试验花费攒出来的果然是一个“小分子重磅炸弹”，原来Wang教授才是最后的赢家... #04 离得谱中谱如果，你以为围绕这个公司的奇葩事就这么结束了就太天真了。在SEC的起诉书中，我们看到了更多奇葩的事。比如，虽然Cassava的临床试验虽然设盲，但是Lindsay Burns在Wan教授进行试验检测之前“不小心”让Wang教授知道了足够的信息能够部分“揭盲”样本。哦，对了，尽管Wang教授跟Cassava关系密切，他的实验室仍然被描述为“学术实验室”，潜台词是跟我们不熟悉，都是专业的。专业不专业咱不知道，据说Cassava自己在2022年对Wang教授实验室做过一次审计，结果是...“unacceptable”... 嚯，这审计员显然是不懂人情世故啊，估计这份报告写完也该下岗了吧。你说，就这样的实验室出来的结果他能可靠？这“小分子重磅炸弹”在临床上能出好结果？别担心，离谱操作还在继续。 Lindsay Burns在报告认知数据中删掉了40%的患者的数据，这些数据还是在她揭盲之后删掉的哦。这等于明白无误的表示她在操纵数...毕竟如果不删，与安慰剂相比PTI-125根本没有人知改善。当然，临床二期的记忆结果也一样被篡改，因为不改PTI-125就没有改善。所以，再看到这样的结果我总是心里打鼓。这到底是真的没有认知水平下降还是下降的人被移除了... 那么，就这么一家离得谱中谱的公司价值几何呢？截至完稿时，其市值仍然有13亿美元之多，股价高达28美元。你看，最离谱的根本不是这家公司，而是众多甘心奉献的股东啊。 END 啊对对对对，扫描这货能联系到我至此，各位股东星标了么？点赞了么？转发了么？在看了么？谢谢！所有内容均不作为投资建议，信息均来自公开资料。近期文章： NGS/NIPT谁能斩获2024诺奖？ Relay Therapeutics的裁员接力赛终于按下了暂停键 Cologuard Plus正式获得FDA批准！肠癌早筛新标杆！ Prime Medicine拿钱、股价暴涨、管线缩减一气呵成 Illumina 要发布个啥相关资料：注1：https://www.cassavasciences.com/node/17196/pdf注2：https://www.sec.gov/enforcement-litigation/litigation-releases/lr-26132注3：https://en.wikipedia.org/wiki/Lindsay_Burns注4：https://www.biospace.com/cassava-sciences-responds-to-allegations-that-data-is-flawed-and-defies-logic注5：https://en.wikipedia.org/wiki/Simufilam注6：https://www.sec.gov/files/litigation/complaints/2024/comp-pr2024-151.pdf

临床2期

2024-09-26

·GlobeNewswire-Health Care

Cassava Sciences Announces Completion of an Interim Safety Review of Oral Simufilam On-going Phase 3 Trials

An Independent Data and Safety Monitoring Board (DSMB) Recently Evaluated the Interim Patient Safety Database for Oral Simufilam in On-going Phase 3 Trials. The DSMB Recommended Both Phase 3 Trials Continue as Planned, Without Modification. Final Clinical Safety Data for Simufilam Are Expected at the Conclusion of the Phase 3 Program. Sept. 24, 2024 -- Cassava Sciences, Inc. (Nasdaq: SAVA), a biotechnology company focused on Alzheimer’s disease, today announced the completion of a third interim safety review of simufilam in on-going Phase 3 clinical trials in patients with Alzheimer’s disease. A routine, scheduled meeting of a Data and Safety Monitoring Board (DSMB) resulted in a recommendation that both of Cassava Sciences’ on-going Phase 3 studies continue as planned, without modification. “We are pleased to have satisfactorily completed this safety review, the last before the expected top-line read out for our first Phase 3 study.” said Jim Kupiec, Chief Medical Officer. “We look forward to announcing top-line efficacy, safety and biomarker data for our 12-month Phase 3 study before the end of 2024.” The DSMB is composed of independent clinical research experts who periodically review interim patient safety data for Cassava Sciences’ on-going Phase 3 trials of simufilam in Alzheimer’s disease. This DSMB only reviews patient safety. It does not assess drug efficacy. Cassava Sciences’ simufilam is a novel, small molecule drug candidate for the proposed treatment of Alzheimer’s disease dementia. The drug is in late-stage clinical evaluation in a pair of pivotal Phase 3 trials. These Phase 3 trials are fully enrolled. Over 1,900 patients with mild-to-moderate Alzheimer’s disease who also met other study eligibility criteria were randomized into the trials. The first Phase 3 trial (NCT04994483) has a 52-week treatment period; 804 Alzheimer’s patients were randomized into this trial. Top-line results for the 52-week Phase 3 trial are currently expected by year-end 2024. The second Phase 3 trial (NCT05026177) has a 76-week treatment period; 1,125 Alzheimer’s patients were randomized into this trial. Top-line results for the 76-week Phase 3 trial are currently expected approximately mid-year 2025. Patients with mild-to-moderate Alzheimer’s disease dementia who met study eligibility criteria were recruited into the Phase 3 program from clinical sites in the U.S., Puerto Rico, Canada, Australia and South Korea. Cassava Sciences is conducting its on-going Phase 3 program in collaboration with Premier Research International, a global contract research organization (CRO). Today’s news follows interim safety MRI data announced in October 2023, which suggests simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities (ARIA). In addition, September 2023 and March 2024 meetings of the DSMB recommended that both Phase 3 trials continue as planned, without modification. Final safety data are expected at the conclusion of the Phase 3 program. Cassava Sciences is a clinical-stage biotechnology company based in Austin, Texas. Our mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. Simufilam is Cassava Sciences’ small molecule oral drug candidate currently in Phase 3 clinical trials for the treatment of Alzheimer's disease. Simufilam targets a specific site on filamin A, a scaffolding protein that is critical to certain receptor interactions in the brain. Cassava Sciences believes that simufilam interrupts amyloid-β42 binding to receptors in the brain and may affect the Alzheimer's disease process. Cassava Sciences owns exclusive, worldwide rights to its investigational product candidates and related technologies, without royalty obligations to any third party. The content above comes from the network. if any infringement, please contact us to modify.