A35-009 (ORION) is a Phase 2b/3 trial to evaluate the efficacy and safety of AMX0035 in participants with Progressive Supranuclear Palsy (PSP), consisting of randomized, double blind placebo controlled phases, followed by an optional open-label extension phase.

开始日期2023-12-21

申办/合作机构

Amylyx Pharmaceuticals, Inc.

CTIS2023-504397-38-00

/ Not yet recruiting

- A35-016

开始日期2023-11-08

申办/合作机构

Amylyx Pharmaceuticals, Inc.

CTIS2023-504393-37-00

/ Not yet recruiting

- A35-015

开始日期2023-11-02

申办/合作机构

Amylyx Pharmaceuticals, Inc.

100 项与苯基丁酸钠/牛磺脱氧胆酸相关的临床结果

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100 项与苯基丁酸钠/牛磺脱氧胆酸相关的转化医学

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100 项与苯基丁酸钠/牛磺脱氧胆酸相关的专利（医药）

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项与苯基丁酸钠/牛磺脱氧胆酸相关的文献（医药）

2025-01-01·EXPERIMENTAL NEUROLOGY

Potential role of solid lipid curcumin particle (SLCP) as estrogen replacement therapy in mitigating TDP-43-related neuropathy in the mouse model of ALS disease

Article

作者: Lee, Yi-Chao ; Ashan, Asmar ; Hsieh, Yi-Chen ; Majumder, Pritha ; Huang, Chi-Chen ; Hsu, Tsung-I ; Hu, Chaur-Joug ; Huang, Jeffrey K ; Ahsan, Asmar ; Huang, Jeffrey K.

BACKGROUND:

Amyotrophic lateral sclerosis (ALS) was first identified in 1869, but it wasn't until the 2014 Ice Bucket Challenge that widespread attention was drawn to the disease. Since then, substantial research has been dedicated to developing treatments for ALS. Despite this, only three drugs - riluzole, edaravone and AMX0035, have been approved for clinical use, and they can only temporarily alleviate mild symptoms without significant disease modification or cure. Therefore, there remains a critical unmet need to identify disease modifying or curative therapies for ALS. The higher incidence and more severe progression of ALS and FTLD (frontotemporal lobar degeneration) observed in men and postmenopausal woman compared to young women suggests that sex hormones may significantly influence disease onset and progression. In both animal models and human clinical studies, 17β estradiol (E2) has been shown to delay and improve the outcomes of many neurodegenerative diseases. Here, we examined the role of TDP-43 in the regulation of estrogen-related enzymes, CYP19A1 and CYP3A4. In addition, we examined the impact of curcumin on the regulation of estrogen E2 levels and TDP-43-associated neuropathy as a potential therapeutic strategy for the treatment of FTLD and ALS.

METHODS:

Prp-TDP-43^A315T mice was used as a model of ALS/FTLD to examine the expression patterns of E2 and its biosynthesis and degradation enzymes, CYP19A1 and CYP3A4. Moreover, the molecular mechanisms and the potency of solid lipid curcumin particles (SLCP) as an E2 replacement therapy for TDP-43 associated neuropathy was analyzed. We further examined the survival rates and the pathological TDP43 patterns in female and male Prp-TDP-43^A315T mice administrated with or without SLCP. In addition, the changed expression levels of enzymes corresponding to E2 biosynthesis and degradation in the spinal cord of female and male Prp-TDP-43^A315T mice with or without SLCP were determined.

RESULTS:

We found that in addition to E2, the expression patterns of CYP19A1 and CYP3A4 proteins differed between Prp-TDP-43^A315T mice compared to wild-type control, suggesting that toxic phosphorylated TDP43 oligomers may disrupt the balance between CYP19A1 and CYP3A4 expression, leading to reduced estrogen biosynthesis and accelerated degradation. In addition, we found that oral administration of SLCP prolonged the survival rates in female Prp-TDP-43^A315T mice and significantly reduced the pathological insoluble phosphorylated TDP-43 species. Furthermore, SLCP attenuated disease progression associated with TDP-43-related neuropathies through modulating estrogen biosynthesis and the activity of CYP450 enzymes.

CONCLUSIONS:

Our results showed that Prp-TDP-43^A315T mice exhibit altered estradiol levels. Moreover, we demonstrated the efficacy of SLCP as an estrogen replacement therapy in mitigating TDP-43-associated disease progression and pathogenesis. These findings suggest that SLCP could be a promising strategy to induce E2 expression for the treatment of ALS and FTLD.

2024-12-12·BRITISH MEDICAL BULLETIN

New developments in the diagnosis and management of motor neuron disease

Review

作者: Shaw, Pamela J ; O’Brien, David

Abstract:

Introduction:

Motor neuron disease (MND) is a devastating neurodegenerative disease characterized by progressive muscle weakness.

Sources of data:

PubMed, MEDLINE, and Cochrane databases were searched for articles to March 2024. Searches involved the terms ‘motor neuron disease’ or ‘amyotrophic lateral sclerosis’ and ‘epidemiology’, ‘diagnosis’, ‘clinical’, ‘genetic’, ‘management’, ‘treatment’, or ‘trial’.

Areas of agreement:

Evidence-based management involves riluzole, multidisciplinary care, provision of noninvasive ventilation and gastrostomy, and symptomatic treatments. Tofersen should be offered to treat SOD1-MND.

Areas of controversy:

Edaravone and Relyvrio are approved treatments in the USA, but insufficient evidence was found to support approval in the UK and Europe.

Growing points:

The discovery of neurofilaments as MND biomarkers, growth of platform trials and development of novel therapies provide optimism for more powerful neuroprotective therapies.

Areas timely for developing research:

Further work should focus on the elucidation of environmental causes of MND, gene–environment interactions, and advanced cellular models of disease.

2024-10-01·CURRENT OPINION IN NEUROLOGY

Ultra-high dose methylcobalamin and other emerging therapies for amyotrophic lateral sclerosis

Review

作者: Oki, Ryosuke ; Kaji, Ryuji ; Izumi, Yuishin

Purpose of review:

Recent development in understanding the pathophysiology of amyotrophic lateral sclerosis (ALS) has led to increasing number of promising test drugs in the pipeline along with the existing ones. We will review these agents focusing on ultra-high dose methylcobalamin, which is pending approval in Japan. Clinical trial design best suited for ALS will also be discussed.

Recent findings:

The most recent phase 3 trial (JETALS) of ultra-high dose methylcobalamin demonstrated significant slowing of ALSFRSR changes (0.5/month), with marked reduction of serum homocysteine levels in the initial double-blind period. The post hoc analysis of the previous phase 2/3 study (E761 trial; Eisai) showed that it prolonged survival of ALS patients, if started within 1 year of onset, but the previous studies suggested its efficacy even in later stages, depending upon the rate of progression. Phase 3 trial of AMX0035 or Relyvrio on the other hand showed negative results despite the promising phase 2 data. The latter did not adjust the disease progression rate before entry.

Summary:

Ultra-high dose methylcobalamin is not a vitamin supplement but a novel disease-modifying therapy for ALS, and it emphasizes homocysteine as a key factor in the disease process. Clinical trial design must include entering patients early and with similar rates of progression using pretrial observation periods for meaningful results, since ALS is a chronologically heterogenous condition with similar phenotypes.

378

项与苯基丁酸钠/牛磺脱氧胆酸相关的新闻（医药）

2024-12-24

·氨基观察

CNS的冰与火之歌

氨基观察-创新药组原创出品作者 | 武月过去这一年，CNS领域大爆发，精神分裂症迎来破冰，阿尔兹海默症再迎重磅新药…… 大药企对于这一领域的热情，更是空前，10亿美元以上的合作，接二连三。然而，与鲜花、掌声相伴的，还有无法摆脱的挫折与失败。尤其艾伯维，11月份，就经历了三次临床失败，在精神分裂症领域的重磅押注，从高点直接掉头向下，转瞬成空。 2024年的CNS领域开启了过山车模式。当然，包括KarXT等在内的那些突破性新药的问世，依然让这场过山车之旅产生的刺激多于颠簸。不过，这种曲折，依然十分清晰地提醒着我们，大脑是一个多么复杂又精密的黑匣子。而对增长的渴望，依旧鼓励着药企在这一领域持续探索，无视失败、前赴后继。 / 01 / 精神分裂症的火焰与海水一半火焰，一半海水。 9月26日，精神分裂症领域迎来历史性一刻，Karuna研发的KarXT获得FDA批准上市。这是35年来首款精神分裂症新药的问世，也验证了去年底，百时美施贵宝以140亿美元收购Karuna决策的正确性。对比之下，与百时美施贵宝前后脚斥巨资收购Cerevel，押注CNS的艾伯维，却被投资者质疑眼光欠佳。就在11月11日，艾伯维宣布收购Cerevel得到的“王牌项目” 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。消息一出，艾伯维股价暴跌12.57%，一夜之间市值蒸发400多亿美元。 Emraclidine和KarXT原本的剧本相似，都是被大药企看中的精神分裂症核心资产，且都是毒蕈碱受体，彼时的不同点是，前者只针对M4受体，后者则针对M1、M4受体。当然，如今最大的不同是，二者的结局截然相反。尽管艾伯维承诺将继续分析数据，但仍有不少投资者认为，此次数据出炉之后，艾伯维多半会放弃管线以节省资源。与此同时，百时美施贵宝则公布了KarXT长期研究积极数据。这进一步证实其能够为精神分裂症治疗带来显著的改变。两相对比之下，艾伯维87亿美元收购案被再次推至风口浪尖，甚至质疑其长期神经板块布局的战略。面对质疑，艾伯维表示，“这家公司（Cerevel）的管线仍有更多潜力”。一个月之后，艾伯维宣布Cerevel的另一核心管线帕金森药tavapadon在关键3期TEMPO-2试验中获阳，算是为公司挽回了些许“颜面”。 Tavapadon是全球首款选择性靶向多巴胺D1/D5受体亚型的部分激动剂，每日一次口服治疗帕金森病。此前II期研究显示，tavapadon可显著提高运动机能的运动障碍-统一帕金森病评定量表（MDS-UPDRS）第III部分评分，且安全性与耐受性表现良好。在最新的TEMPO-2 III期研究中，tavapadon组在治疗第26周时，MDS-UPDRS第II、III部分综合评分较显著降低（安慰剂组:-1.2；tavapadon组:-10.3；p<0.0001）。艾伯维预计明年递交上市申请，如果tavapadon能够顺利上市，将与其现有针对症状性的晚期帕金森病疗法管线成为互补。只不过，这并不能改变，艾伯维在其重磅押注的精神分裂症市场，坐了一场过山车，并付出了不小的代价。 / 02 / 阿尔兹海默症的喜与悲下半年，除了精神分裂症领域在上演过山车，阿尔兹海默症（AD）等领域也不例外。 7月2日，礼来宣布，FDA已批准donanemab用于治疗早期阿尔茨海默病的成年患者。三期临床数据显示，与安慰剂组相比，donanemab延缓了患者35%的认知衰退，以及40%的日常活动下降。单从数据来看，这个结果已经是人类开始挑战阿尔茨海默病以来最好的一次。尽管安全性问题不可忽视，但FDA独立科学顾问委员一致投票通过，认为当前证据足够表明该药有效、该药益处大于风险。背后的核心是，AD苦新药空窗期已久。此前，Lecanemab就在安全性和有效性尚未完全证实的情况下，基于其降低患者大脑淀粉样蛋白沉积的治疗效果，获得了FDA的加速批准。而这极大激励着后来者，角逐这一万亿市场。其中，Alector与艾伯维合作开发用于治疗AD的AL002，被海外媒体评为2024年四季度值得关注的9项临床试验之一。早在2017年，艾伯维以2.05亿美元的预付款和高达2000万美元的潜在未来股权投资与Alector达成协议，用于开发和商业化AD候选药物AL-002和AL-003的全球选择权。 2022年7月，艾伯维决定放弃AL003，保留AL002的选择权，其看中的是AL002的作用机制。AL002靶向骨髓细胞触发受体2（TREM2），TREM2作为一种跨膜受体，在一部分先天性免疫细胞上表达，并在小胶质细胞上特异性地表达，这些细胞共同构成大脑的免疫系统，随着TREM2功能的降低可能会导致AD和其他形式的痴呆症。然而，这一机制在理论上的潜力，并没有在实际临床中得到验证。11月25日，Alector宣布，AL002的II期INVOKE-2研究失败，没有显著减缓阿尔茨海默病患者的临床进展。并且，AL002对AD的液体生物标志物没有显著影响，淀粉样蛋白PET成像显示脑淀粉样蛋白水平没有与治疗相关的降低。基于此，Alector决定停止对AL002的长期扩展研究，同时计划裁员约20%。Stifel Partners分析师称INVOKE-2的读数是Alector的“彻底失败”。而在此之前，分析师认为，基于对目标的强烈遗传和生物标志物证据，AL002在阿尔茨海默病的试验中有很大的成功机会。没错，艾伯维再次在CNS领域，坐了一次过山车。当然，今年以来，在AD领域的失意者也不止Alector与艾伯维。几乎同一时间，Cassava Sciences公司宣布，其备受争议的阿尔茨海默症药物simufilam在三期临床试验ReThink-ALZ中失败。与安慰剂相比，simufilam未能减少轻度至中度阿尔茨海默症患者的认知或功能衰退。更早前，CNS药物开拓者Sage的核心在研管线Dalzanemdor也折戟AD。加上此次临床失利，今年来Dalzanemdo先后在帕金森、阿尔茨海默病、亨廷顿病领域尝到三次败绩，公司也决定终止其研发。 / 03 / 刺激多于颠簸除此之外，肌萎缩侧索硬化症（ALS）也在2024年，经历了一场过山车。 2022年，FDA顶着压力批准了ALS新药Relyvrio的上市申请，尽管定价高达15.8万美元，ALS也属罕见病，但Relyviro仍创造了一个市场奇迹，2023年销售额达3.8亿美元。 Relyviro也曾被不少人视为ALS患者的救星。然而，Relyvrio虽在早期研究中展现了一定潜力，但在更大规模、更长时间的试验中，其疗效并未得到充分验证。 3月8日，Amylyx宣布Relyvrio在3期临床试验失败，；4月4日，Amylyx也宣布了Relyvrio退市的决定。当然，CNS领域也并不是没有好消息。除了精神分裂症、AD这些大市场，一些罕见的神经系统疾病也在2024年迎来了突破。比如，3月份，FDA批准了Orchard开发的基因疗法Lenmeldy，这是第一种治疗变色性白细胞营养不良症（MLD）的基因疗法，MLD是一种罕见且危及生命的机体代谢系统遗传性疾病，根据现有文献估计发生率约为1/10万新生儿。 6月份，FDA批准了首款杜氏肌营养不良（DMD）基因疗法Elevidys的扩展适应症，将年龄至少为4岁的DMD患者纳入其中。无论患儿是否能够行走，尽管该疗法对不可行走的患者，尚未确认试验中满足主要疗效终点；9月份，FDA还批准了首个治疗C型尼曼-匹克病的药物Miplyffa上市。总的来说，CNS过山车在2024年产生的刺激多于颠簸。与此同时，所有人都清楚，CNS新药开发难度巨大，但另一面也代表着药物研发的高壁垒与严重未满足的临床需求，一旦研发成功，创收不可估量。因此，那些突破性新药的接连成功，激励着药企无视失败、前赴后继。比如，虽然Sage失败了，但亨廷顿领域开始看到更多的牵引力。Prilenia Therapeutics、Wave Life Sciences和uniQure都在关注批准可以解决疾病根本原因的疗法的途径。投资也在增加。近期，诺华与PTC Therapeutics公司达成价值近30亿美元的合作，推进后者亨廷顿病候选药物PTC518研发。今年，诺华还与Voyager公司达成13亿美元交易，推进亨廷顿等疾病的研究。再比如，虽然AD领域的失败率极高，但药企依然在坚定布局。不怕输的艾伯维，10月份再次斥巨资押注阿尔兹海默症，以14亿美元收购Aliada，后者的核心资产是ALIA-1758，目前处于临床I期试验开发阶段的抗淀粉样蛋白抗体。武田则以1亿美元预付款+最高21亿美元里程碑款，拿到AC Immune靶向有毒形式淀粉样蛋白（Aβ）的免疫疗法，包括用于治疗AD的ACI-24.060的全球独家选择和许可权。渤健/卫材的Lecanemab和礼来的donanemab在过去两年中分别获批，成为首批针对阿尔茨海默病的Aβ单抗疗法。显然，其他大药企也不想错过这一块大蛋糕。不过，过去一年，整个领域过山车般的曲折，依然提醒着我们，大脑是一个多么复杂又精密的黑匣子。期待过往几十年的探索与失败，能够为我们剔除一个又一个错误选项，最终推动整个行业的渐进进步，为更多患者带去曙光。 PS：欢迎扫描下方二维码，添加氨基君微信号交流。

并购临床3期临床2期申请上市临床成功

2024-12-13

·BioPharmaDive

CMS says private Medicare plans can’t automatically deny Biogen’s ALS drug

In a rare move, the U.S. agency behind Medicare is warning insurers who provide Medicare Advantage plans that they cant automatically deny coverage of Qalsody, a Biogen drug approved to treat amyotrophic lateral sclerosis.Medicare Advantage, or MA, plans are a privatized form of Medicare, the federal health insurance program for people aged 65 or older. These plans must cover the same drugs as traditional Medicare. And yet, the government agency that oversees the program says its observed some inappropriately denying coverage of Qalsody by classifying the drug as experimental and investigational.In a letter dated Dec. 9, the Centers for Medicare and Medicaid services wrote it is impermissible for MA plans to use this reasoning as the basis for blanket coverage policies that exclude Qalsody. Insurers can still use their own criteria to determine whether the drug is reasonable and necessary for any individual patient. But policies that outright reject coverage and dont consider the patients medical history would violate federal regulations, CMS wrote.In April 2023, Qalsody was granted a type of clearance that allows drugs with limited supporting data to enter the market while their developers collect more evidence to confirm they are effective. CMS notes how, in terms of coverage, there should be no difference between drugs given this so-called accelerated approval versus a traditional approval.Qalsody, which is administered by a healthcare professional, is specifically meant for patients with a rare genetic mutation. The Food and Drug Administration estimates less than 500 of the roughly 30,000 people in the U.S with ALS have these mutations. Qalsodys approval was based on data from a failed late-stage study, wherein the drug didnt significantly slow the progression of the disease but was able to lower levels of a protein tied to nerve cell damage.CMS now expects all MA plans that classify Qalsody as experimental and investigational to immediately stop, and to expeditiously contact enrollees who were denied coverage because of that classification and inform them the coverage policies have changed.This is a victory for the entire ALS community, said Calaneet Balas, CEO of The ALS Association, a patient advocacy group, in a statement.We hope this sends a message to the entire rare disease community and pharmaceutical industry that new treatments can be developed and made accessible to those who will benefit from them, Balas added. The ALS Association, which helped fundQalsodys development, says it played a key role in the new CMS directive by participating and advocating for the research, approval, and accessibility of the drug.ALS is a uncommon, fatal disease where the progressive erosion of nerve cells robs patients of the ability to walk, talk, eat and breathe. The disease advances fast, with most patients living just two to five years following a diagnosis. Its also proven exceedingly difficult to treat. At the beginning of this year, there were four main FDA-approved medicines for ALS, but that list got shorter in April when one, Relyvrio, was pulled from market.Priced at $158,000 for a years supply, Relyvrio also faced some insurance challenges before its withdrawal. One of the countrys largest insurers, Cigna, had by early 2023 changed its policy to not cover the drug. Cigna said at the time it considered Relyvrio experimental, investigational or unproven, despite the drug having received full FDA approval.When Relyvrio first debuted on the market, patients and caregivers expressed concerns about whether insurance companies would restrict access to it, as they had in the past with other ALS therapies. '

加速审批上市批准

2024-12-12

·EndPoints

Corcept’s ALS drug fails to improve function in Phase 2 study

It’s been a tough few months for Corcept Therapeutics. In October, the company’s lead pipeline candidate missed the primary endpoint in a Phase 3 Cushing’s syndrome trial. Now, its only neurology asset has flunked a mid-stage study in amyotrophic lateral sclerosis, possibly marking the end of the road for the program. The California biotech’s dazucorilant did not meet the primary endpoint of change from baseline in an ALS functional rating scale in the 249 patient-strong placebo-controlled Phase 2 study. Subjects who received the treatment also had “substantially more” stomach upset than placebo patients, according to a company release Thursday. Corcept appeared to claim signs of a survival benefit for the 300 mg dose of dazucorilant, pointing out that no one in this arm died, compared with five deaths for placebo. However, in light of the primary endpoint failure, this is likely to carry little weight, particularly since survival was not a specified endpoint on the trial’s protocol as listed on clinicaltrials.gov. The company said it plans to share full Phase 2 results at a medical meeting in 2025. Meanwhile, its open-label extension study of the drug will continue as normal, with overall survival to be evaluated in March 2025. Dazucorilant, a selective cortisol modulator, binds to the glucocorticoid receptor while avoiding the body’s other hormone receptors, Corcept said. In February, CEO Joseph Belanoff told investors the drug had “shown great promise” in ALS animal models, improving motor symptoms and reducing muscular atrophy. ALS treatments have managed to clear regulatory hurdles in the past with less-than-stellar results. In 2022, the FDA approved Amylyx’s ALS drug Relyvrio based on results from a single mid-stage trial and post hoc analyses, after much back and forth and two advisory committee meetings. But the drug then failed a Phase 3 test earlier this year, prompting Amylyx to voluntarily pull it from the market and terminate around 70% of staff to help reduce costs.

临床2期临床3期上市批准

100 项与苯基丁酸钠/牛磺脱氧胆酸相关的药物交易

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研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肌萎缩侧索硬化	加拿大	Amylyx Pharmaceuticals, Inc.	2022-06-10

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10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
进行性核上性麻痹	临床3期	美国	Amylyx Pharmaceuticals, Inc.	2023-12-21
进行性核上性麻痹	临床3期	法国	Amylyx Pharmaceuticals, Inc.	2023-12-21
进行性核上性麻痹	临床3期	德国	Amylyx Pharmaceuticals, Inc.	2023-12-21
进行性核上性麻痹	临床3期	意大利	Amylyx Pharmaceuticals, Inc.	2023-12-21
进行性核上性麻痹	临床3期	西班牙	Amylyx Pharmaceuticals, Inc.	2023-12-21
Wolfram综合征	临床2期	美国	Amylyx Pharmaceuticals, Inc.	2023-03-03
阿尔茨海默症	临床2期	美国	Amylyx Pharmaceuticals, Inc.	2018-08-27

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临床结果

适应症

分期

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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


NCT05676034 (HELIOS, Biospace) 人工标引	临床2期	Wolfram综合征	12	AMX0035 (Week 24 ITT)	餘憲衊鬱選醖夢鑰構構(構簾鬱醖遞壓齋醖壓簾) = 餘膚遞膚網範夢廠衊積醖網鬱淵構鬱憲淵憲衊 (膚憲廠窪淵淵築醖鑰範, 19.3) 更多	积极	2024-10-17
				AMX0035 (Week 24 Per Protocol)	餘憲衊鬱選醖夢鑰構構(構簾鬱醖遞壓齋醖壓簾) = 廠憲廠繭繭鏇範鏇鹹願醖網鬱淵構鬱憲淵憲衊 (膚憲廠窪淵淵築醖鑰範, 11.2) 更多
NCT03533257 (PEGASUS, BusinessWire) 人工标引	临床2期	阿尔茨海默症	95	AMX0035	窪壓簾遞壓鑰遞壓廠襯(窪齋築糧艱顧選顧淵製) = The exploratory analyses showed that compared to placebo, AMX0035 reduced levels of p-tau181 and total tau. AMX0035 treatment also reduced levels of synaptic and neuronal degeneration biomarkers in the CSF, specifically neurogranin and FABP3, as well as YKL-40, a biomarker that has been shown to correlate with cortical volume loss and rate of cognitive decline. 構鏇夢繭襯鏇鑰積鬱膚 (繭醖鏇鏇淵簾選觸鏇衊 )	积极	2024-08-12
				Placebo
PHOENIX (Biospace) 人工标引	临床3期	肌萎缩侧索硬化	-	Relyvrio	糧網醖醖範襯鹽鹹鏇繭(膚願範製蓋糧鑰夢選選) = 選鹽鏇鹹鏇齋鑰窪遞顧遞鏇鏇齋繭糧選顧範製 (網積鏇鑰顧廠醖鏇觸淵 ) 未达到	不佳	2024-04-17
				placebo	糧網醖醖範襯鹽鹹鏇繭(膚願範製蓋糧鑰夢選選) = 齋夢襯願選夢淵觸範蓋遞鏇鏇齋繭糧選顧範製 (網積鏇鑰顧廠醖鏇觸淵 ) 未达到
AAN2024	N/A	肌萎缩侧索硬化	-	Relyvrio (Neuromuscular specialists)	淵艱構壓齋鬱襯淵壓夢(獵範範壓糧壓選鑰餘衊) = 壓淵選選鹹範齋繭築壓鏇糧醖範網繭齋獵選衊 (觸積餘廠網製醖衊遞襯 ) 更多	-	2024-04-09
				Relyvrio (ALS Center-affiliated neurologists)	淵艱構壓齋鬱襯淵壓夢(獵範範壓糧壓選鑰餘衊) = 築餘鹹廠襯積構餘壓鹽鏇糧醖範網繭齋獵選衊 (觸積餘廠網製醖衊遞襯 ) 更多
PHOENIX (Biospace) 人工标引	临床3期	肌萎缩侧索硬化	664	AMX0035	簾衊簾鹹窪鬱觸鑰範糧(鹹蓋網艱膚衊觸鹽膚膚): P-Value = 0.667 未达到更多	不佳	2024-03-08
				placebo
CENTAUR (Biospace) 人工标引	临床2期	肌萎缩侧索硬化 C-reactive Protein Expression	126	AMX0035	範觸範艱築網蓋獵憲範(網獵淵選顧願憲積憲鹽) = 觸糧壓簾網壓鏇窪窪餘夢糧糧願鑰鏇遞觸鑰鹹 (廠餘廠製範構願膚衊範 ) 更多	积极	2023-12-04
				placebo	-
NCT03127514 (CENTAUR, CTgov)	临床2期	肌萎缩侧索硬化	137	Placebo (Placebo)	製鑰窪夢顧獵鏇壓鹹窪(壓醖壓壓壓製製構願蓋) = 鑰淵鏇網鬱齋襯艱鏇築製壓窪鑰範觸窪淵膚製 (淵艱範製餘齋艱積膚簾, 壓積鹽觸蓋選簾窪艱鬱 ~ 遞淵齋願廠蓋遞蓋鏇鬱) 更多	-	2021-08-11
				AMX0035 (AMX0035)	製鑰窪夢顧獵鏇壓鹹窪(壓醖壓壓壓製製構願蓋) = 艱簾餘艱淵鏇觸築繭壓製壓窪鑰範觸窪淵膚製 (淵艱範製餘齋艱積膚簾, 夢齋窪築築壓壓蓋膚構 ~ 糧繭廠遞窪顧選繭製廠) 更多
CENTAUR (AAN2021)	N/A	肌萎缩侧索硬化	-	AMX0035	築築壓鏇製範獵鹹積壓(簾窪憲艱繭製壓艱觸齋): HR = 0.56 (95% CI, 0.34 ~ 0.92), P-Value = 0.023	-	2021-04-13
				Placebo
CENTAUR (MDA2021)	N/A	肌萎缩侧索硬化	137	AMX0035	築鹹蓋製網築範遞鬱鏇(範繭廠窪餘膚衊艱醖鹹): HR = 0.56 (95% CI, 0.37 ~ 0.86), P-Value = 0.008 更多	-	2021-03-01
				Placebo
NCT03127514 (CENTAUR, Pubmed \| Br Dent J \| N Engl J Med) 人工标引	临床2期	肌萎缩侧索硬化	137	Sodium Phenylbutyrate-Taurursodiol	壓鬱廠鏇淵鹹膚廠窪網(廠繭觸襯壓窪衊選繭鏇) = 製鑰願遞襯衊獵憲鹽衊獵鏇選夢夢艱艱齋遞襯 (餘鹽鹽衊鹹網簾鏇鏇衊 )	积极	2020-09-03
				Placebo	壓鬱廠鏇淵鹹膚廠窪網(廠繭觸襯壓窪衊選繭鏇) = 鑰積淵繭衊襯膚艱顧齋獵鏇選夢夢艱艱齋遞襯 (餘鹽鹽衊鹹網簾鏇鏇衊 )