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2024-06-13

申办/合作机构

Biogen, Inc.

NCT05399888 / 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 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.

NL-OMON55789 / RecruitingN/A

A Randomized, Double-Blind, Placebo-Controlled Study, Followed by an Open-Label Extension, to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 814907 in Patients with Mild Alzheimer*s Disease - ISIS 814907-CS1

开始日期2018-01-04

申办/合作机构

Ionis Pharmaceuticals, 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）

2023-12-01·JAMA neurology

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

Article

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

2024-05-16

·BioPharmaDive

Biogen, Ionis shelve ALS drug following study failure

Biogen and Ionis Pharmaceuticals are stopping development of an experimental drug for ALS following new clinical trial results that didnt meet the companies expectations.The trial, which enrolled almost 100 people with the nerve-destroying disorder, found the drug actually worked as intended. Biogen and Ionis had designed it to lower levels of ataxin-2, a protein tied to amyotrophic lateral sclerosis, and on that measure it was successful. High-level results released Thursday showed participants who received the drug, named BIIB105, rather than a placebo experienced significant decreases in the protein over a six-month treatment period.However, the drug didnt appear to change the trajectory of the disease. It didnt impact breathing, strength and overall function key health measures that diminish as ALS progresses. BIIB105 also didnt substantially reduce a different protein, neurofilament light chain, that recently became important across this area of research. Qalsody, another ALS medicine developed by Biogen and Ionis, won marketing approval last year based on its ability to lower that protein.“While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process,“ said Stephanie Fradette, head of Biogens Neuromuscular Development Unit, in a statement.In the trial, participants who completed the main six-month treatment period were then offered entry into an open-label extension phase, in which they all could receive BIIB105. Biogen and Ionis said data from that phase were similar what was seen earlier in the study, with sustained reductions in the ataxin-2 protein but no impact on neurofilament or clinical outcome measures.The companies said there was no evidence that any subgroup of drug-treated patients benefited clinically.Analysis of the data remains ongoing. Biogen and Ionis plan to present more detailed study results next month at an ALS research convention in Stockholm.The shelving of BIIB105 is the latest setback for a field of drug development marred by failure. ALS, like many central nervous system disorders, is complex and difficult to combat. Scientists first discovered the disease over a century ago; and yet, just a few medicines have ever been approved to treat it. (The list got even smaller this year, as a once-promising medication from Amylyx Pharmaceuticals is being pulled from the market.)Approved in April 2023, Qalsody is the newest offering, though its for a very small fraction of ALS patients with specific genetic mutations. Biogen and Ionis collaborated on early development of the therapy, using the latters antisense oligonucleotide drugmaking technology.Qalsody essentially gums up the production of an ALS-associated protein called SOD1. The therapy actually failed to hit the central goal of the study that led the Food and Drug Administration to clear it. In that trial, Qalsody wasnt any better than a placebo at slowing disease progression. It was, however, better at lowering neurofilament light chain a biological marker of neurological damage.The FDAs decision effectively created a new route to approval for ALS drugmakers, who, since the Qalsody decision, have prioritized showing an effect on neurofilament in their clinical tests.So far, Qalsody is the only clear victory to come out of Biogen and Ionis ALS research. In 2022, the partners discontinued another antisense drug, code-named BIIB078, after an early-stage trial disappointed. Also on Thursday, Ionis revealed that Biogen has opted against licensing a drug the companies have been working on for Angelman syndrome, despite results Ionis described as positive.The longstanding partnership has been fruitful in other areas, though. Its produced a blockbuster medicine in Spinraza, a treatment for a rare, muscle-wasting illness. And over the past couple years, Biogen has highlighted the potential of another experimental drug, BIIB080, which is in mid-stage testing as a possible treatment for Alzheimers disease. '

临床结果寡核苷酸上市批准

2024-04-24

·FierceBiotech

To be better at neuroscience, Biogen will invest outside the therapeutic area

Biogen already signaled a foray into rare disease last year, where CEO Chris Viehbacher said the company has “tremendous commercial capability.” To be a better neuroscience company, Biogen is going to have to diversify—outside of the therapeutic area that has in recent years defined the company. “We always call ourselves a neuroscience company, but the reality of neuroscience is that this is a high-risk area,” CEO Chris Viehbacher said on the drug developer’s first-quarter earnings call Wednesday morning. And so Biogen will be focused on business development outside of the neuroscience arena, which in turn, will support the expensive, lengthy trials required of the beat. “While we remain committed to neuroscience, my personal view is that is not diversified enough for a company of our size,” the CEO said. Biogen already signaled a foray into rare disease last year, where Viehbacher said the company has “tremendous commercial capability.” Thanks to the slow buildout of Leqembi in Alzheimer’s disease, Biogen knows a thing or two about launching medicines in difficult areas where diagnosis is a challenge and hurdles with payers are rampant. Viehbacher also sees further potential for expansion in multiple sclerosis. “We are very well placed in neurology … but the reality is that we sit there with programs that are very difficult to predict, very expensive, very long-running. And when you actually look at the ability to do external deals, that field is also very narrow. There's just not that many people working in the CNS space,” Viehbacher said. “So we're by no means abandoning it. The fact that we go after these really tough diseases is really a source of pride within Biogen.” He continued: “But the reality is we need to have more predictable results out of R&D.” Biogen's industry peers have found plenty to buy in the space. Bristol Myers Squibb bought Karuna Therapeutics and its schizophrenia med KarXT for $14 billion while AbbVie picked up CNS-focused Cerevel Therapeutics for $8.7 billion, all at the end of 2023. Speaking of deals, Biogen is unlikely to take on a huge acquisition this year, given the company’s balance sheet, the CEO said. “If something really extraordinary came along, you’d look at it,” he said. Chief Financial Officer Michael McDonnell said the balance sheet is “in a very good spot” in terms of net debt for the first quarter. He says there’s about $4 billion to $5 billion to play with for an acquisition target. In 2025 or beyond, that number could grow. As for the pipeline, the executives had little to report with respect to concrete data, but Viehbacher promised more to come as three key readouts draw closer. A big one is Biogen’s anti-tau agent for Alzheimer’s disease called BIIB080, followed by phase 3 assets dapirolizumab pegol and litifilimab, both for systemic lupus erythematosus. Biogen has developed some tough go-no-go criteria to ensure those programs are only advanced if they prove promising, said Priya Singhal, M.D., head of development. “I think the next job is really now to build out the pipeline,” Viehbacher said. “We want to diversify our business a little bit more than we have in the past.”

并购临床3期

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

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适应症	最高研发状态	国家/地区	公司	日期
阿尔茨海默病引起的痴呆症	临床2期	美国	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	日本	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	澳大利亚	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	比利时	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	加拿大	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	丹麦	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	德国	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	意大利	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	波兰	Biogen, Inc.	2022-08-24
阿尔茨海默病引起的痴呆症	临床2期	西班牙	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	網鑰遞糧顧繭範鏇憲鹽(襯醖選蓋鏇齋製衊鬱衊) = 繭鏇壓淵廠艱範網願鬱鏇製襯鬱廠鑰膚窪齋範 (顧壓鏇糧鹹鏇窪築餘艱, 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	憲窪構製齋憲艱獵築膚(糧齋憲築夢衊膚鏇觸艱) = 鹹齋淵蓋淵淵獵壓繭繭築糧廠願窪憲觸淵鹹簾 (膚襯膚繭廠選繭夢餘窪 )	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)	窪鹽餘衊憲鹹淵網築獵(膚鹽選鏇築壓選網積壓) = 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. 繭壓鏇遞遞製壓築蓋廠 (獵觸築壓獵齋壓鏇膚膚 ) 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1/2期	阿尔茨海默症	-	IONIS-MAPT Rx	淵鏇襯膚齋簾廠願餘膚(蓋願鏇窪觸餘鹹築築獵) = 築獵蓋積壓餘鑰顧夢簾積鏇製餘製襯蓋廠積糧 (窪膚齋築範網鬱齋淵鏇 )	积极	2018-04-10
ANA2017	N/A	阿尔茨海默症	-	Ionis-MAPTRx	齋醖範顧選獵鏇餘築鹹(壓範壓艱繭觸鏇積鬱構) = 憲窪醖淵膚構廠鏇齋製範夢製膚廠選築醖觸觸 (顧衊淵膚醖鬱夢觸獵鹽 )	-	2017-10-15
Ionis-MAPTRx-CS1 (AAIC2017)	N/A	阿尔茨海默症	-	Ionis-MAPTRx	獵簾遞襯繭襯鹹觸糧範(選艱鹽鏇糧艱齋製廠構) = 鏇襯構觸壓襯蓋鹽壓糧鹹顧製顧製鹽獵淵鏇蓋 (膚製簾夢觸夢顧壓鹽鏇 )	-	2017-07-01