A Phase 1, Open-Label Study to Determine the Biodistribution, Safety, and Tolerability of a Microdose of Radiolabeled BIIB080 Co-administered With BIIB080 in Healthy Adults

In this study, researchers will learn more about a study drug called BIIB080. BIIB080 is currently a drug under investigation for treatment of Alzheimer's disease. The main question researchers are trying to answer in this study is how radiolabeled BIIB080 distributes in the brain and spinal cord. To help answer this question, researchers will use positron emission tomography (PET) scanner that can detect radiolabeled BIIB080 after a single injection of a small dose of radiolabeled BIIB080 ([89Zr]Zr-DFO-BIIB080) and a dose of BIIB080 together via an intrathecal (IT) injection in healthy volunteers. Researchers will also learn about the safety of injecting radiolabeled BIIB080 and BIIB080 together.

开始日期2025-01-30

申办/合作机构

Biogen, Inc.

NL-OMON57028

/ RecruitingN/A

Moses II - Measuring Elecsys β-Amyloid (1-42) II and Phospho-Tau (181P) in CSF Samples supporting Phase 2 Clinical Trial of 247AD201 (BIIB080) - Moses II

开始日期2023-02-21

申办/合作机构-

NCT05399888

/ Active, not recruiting临床2期

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects With Mild Cognitive Impairment Due to Alzheimer's Disease or Mild Alzheimer's Disease Dementia

In this study, researchers will learn more about a study drug called BIIB080. The study will focus on participants with mild cognitive impairment or mild dementia due to AD.
The main question researchers are trying to answer is if BIIB080 can slow the worsening of AD more than placebo. It will focus on what dose of BIIB080 slows worsening of AD the most.
To help answer this question, researchers will use the Clinical Dementia Rating-Sum of Boxes, also known as the CDR-SB.
* Clinicians use the CDR-SB to measure several categories of dementia symptoms.
* The results for each category are added together for a total score. Lower scores are better.
Researchers will also learn more about the safety of BIIB080.
The study will be split into 2 parts. The 1st part is the Placebo-Controlled Period. The 2nd part is the Long-Term Extension (LTE) Period. The 2nd part of the study will help researchers learn about the long-term safety of BIIB080, and how it affects the participant's daily life, thinking, and memory abilities in the longer term.
A description of how the study will be done is given below.
* After screening, participants will first receive either a low dose or high dose of BIIB080, or a placebo, as an injection into the fluid around the spinal cord (cerebrospinal fluid). A placebo looks like the study drug but contains no real medicine.
* Participants will receive BIIB080 or placebo once every 12 weeks or 24 weeks.
* After 76 weeks of treatment in the Placebo-Controlled Period, eligible participants will move onto the Extension Treatment period, which will last 96 weeks.
* In the extension period, participants who received placebo will be switched to high dose BIIB080 every 12 or 24 weeks.
* Participants may be in the study for up to 201 weeks, or about 4 years. This includes the screening and follow-up periods.
* Participants can continue to take certain medications for AD. Participants must be on the same dose of medication for at least 8 weeks before the screening period.
* After the screening period, most participants will visit the clinic every 6 weeks.

开始日期2022-08-24

申办/合作机构

Biogen, Inc.

100 项与 ISIS-814907 相关的临床结果

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100 项与 ISIS-814907 相关的转化医学

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100 项与 ISIS-814907 相关的专利（医药）

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项与 ISIS-814907 相关的文献（医药）

2025-05-07·Science Translational Medicine

PET imaging of antisense oligonucleotide distribution in rat and nonhuman primate brains using click chemistry

Article

作者: Amatruda, Mario ; DuBois, Jonathan M. ; Jiang, Di ; Peterson, Emily A. ; Datta, Prodip ; Girmay, Sara ; Delavari, Armin ; Boscutti, Giulia ; Halldin, Christer ; Forsberg Morén, Anton ; Klein, Stephanie K. ; Muste, Cathy ; Kaliszczak, Maciej ; Ochniewicz, Piotr ; Plisson, Christophe ; Tang, Sac-Pham ; Martarello, Laurent ; Nag, Sangram ; Pickel, Thomas C. ; Yuan, Long ; Zhang, Qize ; Khani Meynaq, Yasir ; Beshr, Rouaa ; Cook, Brendon E. ; Bolduc, Philippe N. ; Polyak, Ildiko ; Shoroye, Adebowale

Determination of a drug’s biodistribution is critical to ensure that it reaches the target tissue of interest. This is particularly challenging in the brain, where invasive sampling methods may not be possible. Here, we present a pretargeted positron emission tomography (PET) imaging methodology that uses bioorthogonal click chemistry to determine the distribution of an antisense oligonucleotide (ASO) in the brains of rats and nonhuman primates after intrathecal dosing of ASO. A PET tracer, [ 18 F]BIO-687, bearing a click-reactive trans -cyclooctene was developed and tested in conjunction with a test Malat1 ASO conjugated with a methyltetrazine group. PET imaging in rats demonstrated that the tracer had good kinetic properties for PET imaging in the rodent central nervous system and could react to form a covalent linkage with high specificity to the methyltetrazine-conjugated ASO in vivo. Furthermore, the amount of PET tracer reacted by cycloaddition with the methyltetrazine was determined to be dependent on the concentration of ASO-methyltetrazine in rat brain tissue, as determined by comparing the PET imaging signal with the liquid chromatography–mass spectrometry signal in the tissue homogenates. The approach was evaluated in cynomolgus macaques using both the Malat1 test ASO and a candidate therapeutic ASO, BIIB080, targeting the microtubule-associated protein tau ( MAPT ) gene. PET imaging showed favorable tracer kinetics and specific binding to both ASOs in nonhuman primate (NHP) brain in vivo. These results suggest that the PET imaging tracer [ 18 F]BIO-687 could show the distribution of intrathecally delivered ASOs in the rat and NHP brains.

2023-12-01·JAMA neurology

Exploratory Tau Biomarker Results From a Multiple Ascending-Dose Study of BIIB080 in Alzheimer Disease

Article

作者: Martarello, Laurent ; Kordasiewicz, Holly ; Wu, Shuang ; Mignon, Laurence ; Ziogas, Nick ; Beaver, John ; Hutchison, R. Matthew ; Budd Haeberlein, Samantha ; Li, Yumeng ; Lin, Lin ; DuBois, Jonathan ; Lane, Roger ; Junge, Candice ; Edwards, Amanda L. ; Collins, Jessica A. ; Shulman, Melanie ; Graham, Danielle

Importance:

Accumulation of hyperphosphorylated, tangled microtubule-associated protein tau (MAPT) is a pathological hallmark of Alzheimer disease (AD) associated with disease progression and cognitive decline.

Objective:

To evaluate the effect of tau synthesis reduction on tau biomarkers in patients with mild AD.

Design, Setting, and Participants:

This randomized clinical trial was a double-blind, placebo-controlled 36-week multiple-ascending dose (MAD) phase 1b trial (October 2017 to September 2020), followed by a 64- or 71-week open-label long-term extension (LTE) (October 2019 to May 2022). After being assessed for eligibility at 12 sites in Canada and Europe, participants with mild AD and confirmed amyloid pathology were randomized 3:1 (BIIB080:placebo) in 4 dose cohorts.

Intervention:

Intrathecal administration of BIIB080, a MAPT-targeting antisense oligonucleotide, or placebo. Active dose arms included 10 mg every 4 weeks, 30 mg every 4 weeks, 60 mg every 4 weeks, and 115 mg every 12 weeks during the MAD period and 60 mg every 12 weeks or 115 mg every 12 weeks during the LTE.

Main Outcome and Measures:

The original primary end point was safety. Additionally, BIIB080, total tau (t-tau), and phosphorylated tau 181 (p-tau181) cerebrospinal fluid (CSF) concentrations were evaluated. Tau positron emission tomography (PET) was collected in a substudy, and standard uptake value ratios (SUVRs) were calculated in a priori-defined composite regions of interest.

Results:

Of 102 participants assessed for eligibility, 46 participants with mild AD were enrolled; 23 (50%) were female, and mean (SD) age was 65.8 (5.70) years. BIIB080 was generally well tolerated and was associated with a dose-dependent reduction in CSF t-tau and p-tau181 in the MAD period (56% reduction; 95% CI, 50% to 62%; and 51% reduction; 95% CI, 38% to 63%, of CSF t-tau in the 2 higher-dose cohorts) that continued and/or was maintained through quarterly dosing in the LTE. Tau PET demonstrated reduced accumulation vs placebo at week 25 (n = 13). At week 100, tau PET showed a reduction from baseline across all regions assessed (n = 12), with the largest reductions from baseline observed in the temporal composite (−0.71 SUVR; 95% CI, −1.40 to −0.02). A moderate correlation was observed between model-predicted cumulative CSF drug exposure and tau PET change.

Conclusions and Relevance:

In this randomized clinical trial, BIIB080 reduced tau biomarkers, including CSF t-tau, CSF p-tau181, and tau PET, which is associated with cognitive decline, in participants with mild AD. Effects of BIIB080 on biomarkers and clinical outcomes are being further evaluated in a phase 2 trial.

Trial Registration:

ClinicalTrials.gov Identifier: NCT03186989

2023-05-04·Expert opinion on drug discovery

The development of peptide- and oligonucleotide-based drugs to prevent the formation of abnormal tau in tauopathies

Review

作者: Sardone, Rodolfo ; Dibello, Vittorio ; Castellana, Fabio ; Panza, Francesco ; Lozupone, Madia ; Bellomo, Antonello ; Bortone, Ilaria ; Altamura, Mario ; Solfrizzi, Vincenzo ; Daniele, Antonio ; Zupo, Roberta ; Lampignano, Luisa ; Stallone, Roberta

INTRODUCTION:

Tauopathies represent clinicopathological entities with increased and abnormal glial and/or neuronal inclusions of tau, a microtubule-binding protein. Antisense oligonucleotides (ASOs) are a promising therapeutic approach for treating tauopathies as they can target tau mRNA to reduce total human tau expression or tau exon 10 expression and 4 R tau. Additionally, targeting the tau specifically with peptides may be a unique pharmacological approach, between small molecules and proteins.

AREAS COVERED:

The present review investigates the chemical basis of designing ASOs and peptides currently known to treat tauopathies. Among ASOs targeting tau expression, BIIB080 was the first to enter clinical trial development for treating mild Alzheimer's disease (AD). Furthermore, the therapeutic potential of peptide 021 (P021, Ac-DGGLAG-NH2) in tauopathies is discussed based on preclinical studies.

EXPERT OPINION:

ASOs are a promising therapeutic approach for tauopathies, particularly because ASOs may suppress the expression of harmful genes and are directly delivered to the brain, showing little systemic side effects. However, whether a generalized brain tau decrease will produce positive clinical effects remains unclear. A Phase II trial of BIIB080 is ongoing in mild AD. Neurotrophic and neurogenic peptide mimetic compounds have also shown potential as treatment options for AD and other tauopathies.

项与 ISIS-814907 相关的新闻（医药）

2025-05-30

·药时代

再砸10亿美金，Biogen进军CNS研发黑洞

正文共2000字预计阅读时间6分钟2025年5月27日，渤健与成立仅7个月的RNAi技术公司City Therapeutics达成协议，共同开发新型中枢神经系统疗法。根据协议，City将获得1600万美元预付款和3000万美元的投资，并有资格获得高达10亿美元的里程碑付款以及销售分成。通过此次合作，City Therapeutics将利用其下一代的RNAi工程技术，结合Biogen专有的组织增强型递送技术，实现RNAi疗法的系统性给药，并优先攻克中枢神经系统疾病（CNS）。该合作最初将集中在一个介导关键中枢神经系统疾病的单一靶点上。Biogen则将负责IND赋能研究和全球临床开发，以及任何监管提交和所有与商业化相关的活动。值得一提的是， City执行主席John Maraganore是RNAi治疗领域的先驱，曾领导Alnylam将全球首款RNAi疗法ONPATTRO™推向市场。核酸疗法攻克研发黑洞根据WHO评估，全球范围内超过10亿人口饱受CNS疾病困扰，而我国帕金森病（PD）患者超250万，阿尔茨海默病（AD）患者接近1000万，CNS市场空间巨大。然而，中枢神经系统药物研发成功率仅为6%，不足其他药物研发成功率的一半，素有“新药研发黑洞”之称。从机制不明，到动物模型难验证、临床开发难，每一项都是CNS药物开发的拦路虎。反义寡核酸（ASO）、小干扰RNA（siRNA）等类型的小核酸药物，被认为将带来“除小分子药和抗体药之外的第三次制药浪潮”。与前两者作用于蛋白质层面不同，小核酸药物直接靶向mRNA层面。因此，小核酸药物的靶点丰富，可治疗的疾病谱范围也非常广。RNAi的基因沉默机制，为传统难以成药的蛋白靶点（如tau蛋白、α-突触核蛋白）提供了新思路，也为CNS治疗带来了新的路径。2025年4月2日，Biogen在CNS小核酸疗法领域传来了喜讯。Biogen公司宣布，美国食品药品监督管理局（FDA）已授予BIIB080快速通道资格认定，用于治疗阿尔茨海默病（AD）。BIIB080是首个进入AD临床开发阶段的针对tau蛋白的反义寡核苷酸（ASO）疗法，旨在通过RNA水平的调节减少tau蛋白的生成。目前正在2期临床研究中。此外，今年2月，Biogen豪掷5亿美元引进Stoke Therapeutics旗下的zorevunersen，这是一款针对SCN1A基因的反义寡核苷酸（ASO），用于治疗 Dravet （遗传性癫痫）综合征。该产品有望成为首个针对Dravet综合征根本原因的疾病修饰药物。Biogen布局CNS喜忧参半在医药界的波澜壮阔中，Biogen以其对神经退行性疾病治疗领域（CNS）的深刻洞察和大胆投资策略而闻名。然而，Biogen布局CNS喜忧参半。Biogen和Eisai联合开发的阿尔茨海默病（AD）单抗药物Leqembi，第一季度业绩强劲，在全球销售额约9600万美元，保持稳步增长。今年1月，Leqembi静脉注射制剂（IV）在美国新增早期阿尔茨海默病适应症。Biogen也正在申请Leqembi的皮下注射剂型用于维持治疗，预计审批日期为今年8月31日。如果获批，Leqembi将成为首款可通过自动注射器，在家进行皮下注射的阿尔茨海默病治疗药物，注射过程预计平均仅需15秒。今年年初，Biogen宣布将以总估值4.42亿美元收购神经科学治疗公司Sage Therapeutics但被制止。尽管Sage此间经历了一系列临床试验失败股价暴跌近75%，但仍不愿此时断臂求生，已向特拉华州法院对Biogen提起诉讼，试图阻止这笔交易。而早在5年前（2020年11月），Biogen就以31.25亿美元的交易总价，从Sage手中买了两款CNS候选产品。如今，SAGE-324已终止临床试验， Zuranolone虽上市，但痛失在成人抑郁症MDD的巨大市场。此次收购失败，Biogen与Sage的爱恨纠葛还需观望一下。不过Biogen也有押对宝的时候。2023年7月28日，Biogen用73亿美元收购Reata，充实神经系统研发管线。令人诧异的是，在宣布收购的3天前，Biogen刚发布2023年H1财报，并宣布裁员1000人以节约10亿美元。如此反常的举动是冲着Skyclarys去的，这是全球首款用于治疗FA（弗里德赖希氏共济失调症）的产品，是一种Nrf2刺激剂。2025年第一季度Skyclarys销售额达1.24亿美元，同比增长59%；也算不负Biogen砸锅卖铁也要它的偏爱。结语今年CNS的运势低迷。今年3月3日，CNS研发药企经历“黑色星期一”。Biohaven、Neumora、Lexicon三家药企同日宣布临床失败或调整。随后，阿斯利康在Q1财报会上高调宣布，将全面退出CNS（中枢神经）领域，终止多个管线项目。此时，Biogen仍有斥资10亿合作CNS领域之举，或许呼吁了巴菲特的那句：“在别人贪婪时恐惧，在别人恐惧时贪婪”。攻克研发黑洞，Biogen正在开启神经科学的新时代。参考资料：1.Biogen官网2.其他公开资料图片来源：豆包AI特大爆发！一天11款创新药获批上市！NMPA重磅官宣2025-05-292期临床失败、暴跌60%，AVV基因疗法如何走出Blue时刻？2025-05-29从0%到42%，中国创新药创造了历史！2025-05-28版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn点击这里，查看更多精彩！

寡核苷酸临床2期核酸药物信使RNAsiRNA

2025-05-28

·同写意

正式涉足RNAi领域，渤健再燃CNS战火

同写意年度巨献！！大会特设“CNS新药开发”分会，7月25-26日，邀您相聚金鸡湖畔，与5000人一起为中国医药创新“同写意”！渤健正式涉足RNAi领域。5月27日，渤健与成立仅7个月的RNAi技术公司City Therapeutics达成协议，共同开发新型中枢神经系统疗法。这家公司由数名前Alnylam高管创立，将运用其RNAi工程能力，与渤健共同开发新型中枢神经系统疗法。根据协议，City将获得1600万美元预付款和3000万美元的投资，并有资格获得高达10亿美元的里程碑付款以及销售分成。此次合作发生在强生、艾伯维披露在CNS研发中的失败消息，辉瑞、安进、默沙东等药企相继缩减CNS投入，以及阿斯利康表示不再碰CNS的背景下。这是渤健寻求“外部机遇”战略转型的需要，但在高风险的CNS领域，能否获得高收益，仍需打一个问号。TONACEA01关键武器在渤健与City的合作中，RNAi技术成为解决CNS疾病的关键武器。这种技术通过沉默致病基因表达，为传统药物难以靶向的疾病靶点提供新解决方案。自2018年首款RNAi药物patisiran获批以来，全球已有近10款siRNA药物获批上市。据Evaluate Pharma数据，全球siRNA药物市场规模已突破50亿美元，预计2030年将达200亿美元。CNS领域应用RNAi技术长期面临血脑屏障的递送挑战。渤健将贡献其专有药物递送技术，与City的下一代RNAi工程技术平台结合，开发针对CNS疾病靶点的RNAi触发分子。City执行主席John Maraganore是RNAi治疗领域的先驱，曾领导Alnylam将首款RNAi疗法推向市场。渤健研发负责人Jane Grogan在合作声明中强调，此次合作突显了公司新的战略研究方法，即平衡差异化的内部能力与对尖端科学的外部投资。“我们正在扩展研发工具箱中的药物形式，通过添加基于RNAi的方法，更精确地实现目标。”Grogan道出渤健转型的核心。两年前，渤健就提出“Fit for Growth”计划，希望通过优化资源配置，将更多精力投入到外部合作和新兴技术领域。当时裁撤超过1000个岗位，约占员工总数的15%。今年1月，渤健的裁员范围扩大到研究部门。公司发言人表示，研究部门的新架构将匹配优先临床前项目的需求，并将资源转向外部机遇。面对核心产品专利悬崖和阿尔茨海默病药物Aduhelm的商业化挫折，这家麻省剑桥的生物技术巨头正在重塑研发管线。通过外部合作补充内部研发，重点布局神经科学领域的新型治疗模式。TONACEA02“走钢索”在专利悬崖阴影、研发难度攀升、成本高涨、医保支付压力大的困境下，大部分MNC都选择收缩战线、聚焦核心。比如，默沙东不断围绕K药做加法，对RNA、细胞治疗等领域投资较少；诺华在CGT、AI驱动靶点发现等方面持续大投入；阿斯利康直接砍掉CNS，继续加大肿瘤领域的投资力度，尤其是ADC。渤健则选择“走钢索”，在CNS领域不断加注。今年2月，渤健与Stoke Therapeutics就ASO疗法zorevunersen治疗特定遗传性癫痫达成合作。2024年10月，渤健与Neomorph达成14.5亿美元合作，共同开发治疗阿尔茨海默病等神经系统疾病的分子胶降解剂。同年8月，渤健与诺奖得主Craig Mello创立的Atalanta Therapeutics合作，针对亨廷顿病等CNS靶点开发RNAi疗法。2023年用73亿美元收购Reata，充实神经系统研发管线。虽然说，CNS拥有巨大的未满足需求，潜力无限，但有业内人士表示，这个领域五年内诞生重磅炸弹的概率相对其他领域更小。高风险是否会带来高利润，还不能下定论，但渤健的转型确实初见成效。2024年财报显示，渤健多发性硬化症业务收入43.50亿美元，同比下降7%；罕见病业务收入19.88亿美元，同比增加10%。今年第一季度继续延续上述势头，财报显示，针对罕见病、阿尔茨海默病和产后抑郁的新上市产品实现销售额翻倍，正在改变其商业化布局。渤健总裁兼首席执行官表示，第一季度业绩强劲，符合长期增长战略预期。商业产品组合的转型令人鼓舞，期内约45%的产品收入来自多发性硬化业务以外的重要药物。具体来看，第一季度总收入24.31亿美元，同比增长8%；产品收入17.27亿美元，同比增长3%。按业务划分，多发性硬化治疗产品收入同比下降10%至9.53亿美元，生物类似药收入同比缩减5%至1.81亿美元。罕见病业务同比增长36%，收入5.63亿美元；其中，用于治疗脊髓性肌萎缩症的Spinraza贡献了主要增量，销售额达到4.24亿美元。阿尔茨海默病药物Leqembi在全球销售额约9600万美元，保持稳步增长。渤健和卫材仍然在大力推进Leqembi的后续开发。今年1月，Leqembi静脉注射制剂（IV）在美国新增早期阿尔茨海默病适应症。渤健也正在申请Leqembi的皮下注射剂型用于维持治疗，预计审批日期为今年8月31日。如果获批，Leqembi将成为首款可通过自动注射器，在家进行皮下注射的阿尔茨海默病治疗药物，注射过程预计平均仅需15秒。此外，渤健还在进行Leqembi的III期临床试验，针对无症状阿尔茨海默病患者，预计2028年获得关键数据。其他阿尔茨海默病治疗药物的研发，如BIIB080已处在II期临床试验。同时，公司还在推进其他神经退行性疾病的研发，以进一步巩固其在CNS领域的地位，如针对帕金森病的BIIB122项目，目前处于II期临床阶段。TONACEA03CNS困局渤健的战略重组并非孤例，而将新的增长曲线寄希望于CNS的也不止渤健一家，这其中不乏百亿美元的资产迁移。2025年开年，强生豪掷146亿美元收购专注于CNS疾病治疗的Intra-Cellular Therapies公司，将其核心产品抗精神分裂症药物Caplyta收入囊中。在更早之前的2023年12月，BMS以140亿美元收购Karuna Therapeutics，获得70年来精神分裂症FIC新药Cobenfy（KarXT）。该产品已经于2024年9月获FDA批准上市，成为数十年来首个获FDA批准用于治疗精神分裂症的新作用机制药物。CNS的医疗需求和市场潜力毋庸置疑。数据显示，全球超过10亿人患有神经精神或神经退行性疾病——相当于每8人中就有1人受此困扰。仅强生收购的Caplyta，2023年销售额就达4.62亿美元，同比增长86%。强生预测这次收购获得的口服抗精神病产品Caplyta（lumateperone）销售峰值有望突破50亿美元。投资公司Jefferies也预计礼来的阿尔茨海默病新药Kisunla（Donanemab），在巅峰时期的年销售额接近30亿美元。然而，CNS药物的研发困境也显而易见。诺华以7.7亿美元收购Cadent Therapeutics获得的NMDAr变构调节剂管线，罗氏与Relay Therapeutics就SHP2变构抑制剂达成的合作相继败北。艾伯维87亿美元“打水漂”事件也足够让人在CNS领域犹豫一下——其收购Cerevel Therapeutics获得的M4选择性正向变构调节剂Emraclidine，未能在两项针对精神分裂症的II期研究中达到预期终点。去年10月，艾伯维又花费14亿美元收购Aliada，收获处于Ⅰ期临床的潜在BIC阿尔茨海默病疗法ALIA-1758。监管环境的日益严格，也促使MNC更加谨慎地对待研发项目。在CNS这个复杂领域，新药获批难度加大，使得MNC在权衡风险与收益时更加保守。不过，多种新型治疗模式有望重塑CNS药物研发格局。分子胶降解剂成为新宠。近两年该领域已产生超10笔交易，总金额超过百亿美元。2024年10月，诺华、渤健、辉瑞接连入场，一个月内达成三笔重磅合作。双抗药物在肿瘤领域大放异彩后，也开始向CNS扩展。4月，致力于开发用于免疫肿瘤学和神经退行性疾病的双特异性抗体技术公司ABL Bio，宣布与GSK达成协议，开发神经退行性疾病的新药。当传统药物形态难以突破CNS疾病治疗的瓶颈，核酸药物、蛋白降解剂等新一代治疗模式或将开启神经科学的新时代。参考文章：1、Biogen taps City Therapeutics in RNAi deal worth up to $1B；endpts2、强生放大招；同写意3、当阿斯利康向神经类药物说“再见”；新浪财经

核酸药物引进/卖出siRNA上市批准临床2期

2025-04-10

·ETPharma

Biogen’s Investigational Alzheimer’s drug fast-tracked by FDA

Cambridge: US-based drugmaker Biogen announced that the US Food and Drug Administration ( FDA ) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy aimed at treating Alzheimer ’s disease by targeting the tau protein. BIIB080 is designed to reduce the production of microtubule-associated protein tau (MAPT) by targeting its mRNA. The candidate is currently being evaluated in a Phase 2 clinical trial (NCT05399888) in individuals with early-stage Alzheimer’s disease. Priya Singhal, Head of Development, Biogen, said, “We are encouraged by the FDA’s Fast Track designation for BIIB080. As an investigational antisense therapy, it represents a differentiated approach to targeting tau and holds promising potential for patients. We are advancing this program with urgency on behalf of people living with Alzheimer’s and their families.” Biogen acquired worldwide, exclusive, royalty-bearing rights to BIIB080 from Ionis Pharmaceuticals in December 2019, exercising a license option under their collaboration. As part of the agreement, Ionis received an upfront payment of $45 million (approx. ₹387 crore) and is eligible for up to $155 million (approx. ₹1,330 crore) in milestone payments, along with royalties in the low- to mid-teens on future sales. Besides this candidate Biogen had collaborated with Ionis Pharma to acquire the global marketing rights of its SMA drug Spinraza (nusinersen) in 2016. The Fast Track designation is intended to accelerate the development and review of investigational therapies that treat serious conditions and fulfill unmet medical needs. By Online Bureau ,

引进/卖出临床2期寡核苷酸快速通道

100 项与 ISIS-814907 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
认知功能障碍	临床2期	美国	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	日本	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	澳大利亚	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	比利时	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	加拿大	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	捷克	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	丹麦	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	芬兰	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	法国	Biogen, Inc.	2022-08-24
认知功能障碍	临床2期	德国	Biogen, Inc.	2022-08-24

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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


NCT03186989 (Literature) 人工标引	临床1期	阿尔茨海默症 Tau protein	46	BIIB080	遞構築醖齋夢鏇遞簾觸(選獵製醖簾夢顧願夢壓) = 網獵範鹹醖鑰衊夢衊襯廠獵憲構鹹夢鏇築觸艱 (廠廠網鬱艱憲襯膚遞衊, 50 ~ 62) 更多	积极	2023-10-30
NCT03186989 (Literature) 人工标引	临床1期	阿尔茨海默症 Tau protein	46	placebo	-	积极	2023-10-30
NCT03186989 (NEWS) 人工标引	临床1期	阿尔茨海默症	46	BIIB080	範糧餘廠繭網糧製壓簾(積網網鏇艱觸積鬱網襯) = 廠鹽淵糧範觸觸構艱築餘鑰窪襯醖艱製獵選願 (選鹹壓築憲餘鑰齋憲膚 )	积极	2023-10-26
NCT03186989 (PRNewswire) 人工标引	临床1期	阿尔茨海默症	46	BIIB080 (low dose group treated every four-weeks)	築憲願蓋積壓艱獵築鹽(網鹹遞襯憲築淵鏇襯構) = All adverse events were mild to moderate in severity with no serious adverse events occurring in any patients that received BIIB080. There were no deaths, dose-limiting adverse events or dosing discontinuations. 艱衊鹽範鹹觸襯築願膚 (糧簾選製繭衊窪築製選 ) 达到	积极	2023-08-16
NCT03186989 (PRNewswire) 人工标引	临床1期	阿尔茨海默症	46	BIIB080 (medium dose group treated every four-weeks)		积极	2023-08-16
NCT03186989 (Ionis-MAPT Rx CS1, AAN2018)	临床1期	阿尔茨海默症	-	IONIS-MAPT Rx	壓鏇願觸鬱壓繭齋選餘(蓋艱膚蓋範築艱蓋廠蓋) = 憲窪醖鬱遞顧鹽鬱憲遞鬱壓範簾鑰遞繭衊願淵 (夢夢構築獵壓艱構繭遞 )	积极	2018-04-10
Ionis-MAPTRx-CS1 (ANA2017)	N/A	阿尔茨海默症	-	Ionis-MAPTRx	觸構顧鹹膚繭艱鑰鹽蓋(製廠壓遞獵範餘蓋糧襯) = 獵鹽鹹壓顧夢淵獵壓蓋遞網鑰夢繭鏇醖構構膚 (選衊鹹餘鹹獵鑰鹹醖壓 )	-	2017-10-15
Ionis-MAPTRx-CS1 (AAIC2017)	N/A	阿尔茨海默症	-	Ionis-MAPTRx	製糧鹽鑰築壓觸鏇鹽衊(蓋觸簾廠糧遞鹹夢遞餘) = 衊廠鬱襯獵顧繭簾鏇壓夢壓衊糧網製獵廠膚淵 (獵獵網築範網壓憲築窪 )	-	2017-07-01