A Phase 3 Study With an Open-Label Extension in Pediatric Participants to Evaluate the Safety, Efficacy, and Pharmacokinetics of Diroximel Fumarate and Dimethyl Fumarate for the Treatment of Relapsing Forms of Multiple Sclerosis

In this study, researchers will learn more about the study drugs diroximel fumarate (DRF) and dimethyl fumarate (DMF) in children with MS who may be experiencing relapses.
Participants will be divided into 2 groups based on their weight:
* Group A will include children who weigh 40 kilograms (kg) or less. They will receive DRF in both Part 1 and Part 2 of the study.
* Group B will include children who weigh more than 40 kg. They will be randomly assigned to receive DRF, DMF, or fingolimod. Fingolimod is a drug already used to treat MS in adults. In Part 2, they will receive DRF.
This is a 2-part study. Part 1 treatment will last 96 weeks. Participants who complete Part 1 may move to Part 2. Part 2 is an extension period. Treatment will last another 96 weeks and will help researchers learn about the long-term safety and effects of treatment.
The main goal of the study is to learn about the safety of DRF and DMF and compare their effect on relapses and brain lesions with fingolimod.
The main questions researchers want to answer are:
* How many participants have adverse events and serious adverse events?
* How often do participants relapse after treatment with DRF and DMF compared to fingolimod?
Researchers will take brain imaging scans to check for any new areas of brain inflammation and compare the brain lesions before and after treatment.
Researchers will also measure the amount of drug in the blood to understand how the body processes it. To check safety, they will monitor participants' growth and development, and compare changes in heart tests, vital signs, and lab tests. They will also use rating scales to monitor depression symptoms.
The study will be done as follows:
Part 1 (Treatment Period)
* After screening, participants will join Part 1 and be divided into 2 groups based on their weight.
* Participants in Group A will receive DRF.
* Participants in Group B will be randomly assigned to receive either DRF, DMF, or fingolimod.
* Neither the researchers nor the participants will know which drug or dose the participants will receive in Group B. Participants in Group B will also receive a placebo so they do not know which drug is being given. A placebo looks like a study drug but contains no real medicine.
* All study drugs will be taken by mouth, once or twice a day. Participants who take DRF or DMF will start with a lower dose during the 1st week, then move to a standard dose.
* Treatment in Part 1 will last for 96 weeks. Participants will have up to 11 study visits and 7 phone calls.
* Participants who do not move onto Part 2 will also have a safety follow-up period of 4 to 8 weeks. This will include 1 study visit and 1 phone call.
* The total length of Part 1, including the screening, treatment, and follow-up, will be up to 108 weeks.
Part 2 (Extension Period)
* Participants who complete Part 1 can move on to Part 2 of the study.
* All participants in Part 2 will receive DRF by mouth for 96 weeks.
* Participants will have up to 10 more study visits and 1 telephone call during treatment.
* They will also have a 4-week safety follow-up, including 1 study visit and 1 phone call.
* The total length of Part 2 will be up to 100 weeks.

开始日期2026-11-16

申办/合作机构

Biogen, Inc.

NCT07275515

/ Not yet recruiting临床2期IIT

DIROXIMEL FUMARATE TO REDUCE PERIHAEMATOMAL OEDEMA IN INTRACEREBRAL HAEMORRHAGE: A DOUBLE BLIND RANDOMIZED CLINICAL TRIAL (DARLENE)

Spontaneous intracerebral haemorrhage (ICH) is a life-threatening condition, still devoided of specific treatment. Peri-haematomal oedema (PHO) develops in the ensuing days after ICH onset and worsens functional outcome. Hence, PHO is a promising therapeutic target but until now there is no specific treatment for PHO. The occurrence and growth of PHO is mainly mediated by inflammation. We hypothesize that a modulation of inflammation is effective in reducing PHO growth, therefore improving the functional outcome of ICH patients. From animal studies to human post-mortem studies, our team has demonstrated a key role for erythroid-related nuclear factor 2 (Nrf2) in PHO. Indeed, this transcription factor promotes the protective effect of inflammation: Nrf2 activation enhances antioxidant defenses and increases rates of blood resorption. Therefore, Nrf2 emerges as a promising and innovative therapeutic target. Taking into account the prolonged time interval between de novo drug discovery and use in clinical practice, drug repurposing is an interesting option for the unmet clinical need of reducing PHO. We chose Diroximel Fumarate (DRF) which is a safe and effective Nrf2 activator widely used in multiple sclerosis (dimethyl fumarate is on the market since 2013, and DRF since 2019) to modulate inflammation and to establish the efficacy of Nrf2 activation in reducing PHO growth and, ultimately, in improving the functional prognosis after ICH.

开始日期2026-02-01

申办/合作机构

La Santé

IRCT20200105046010N142

/ SuspendedN/A

In vivo bioequivalence study of Diroximel fumarate 231 mg EC .Capsules of zist daru danesh as Test Drug in compared with the reference Drug in Iranian Healthy Volunteers

开始日期2025-07-23

申办/合作机构-

100 项与富马酸地罗西美相关的临床结果

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100 项与富马酸地罗西美相关的转化医学

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100 项与富马酸地罗西美相关的专利（医药）

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项与富马酸地罗西美相关的文献（医药）

2026-03-01·Multiple Sclerosis and Related Disorders

Retrospective review of lymphocyte changes when switching between fumarates in patients with multiple sclerosis

Article

作者: Schneider, Megan ; Choi, Leena ; Zuckerman, Autumn ; DeClercq, Josh ; Banks, Aimee ; Cruchelow, Katie R

BACKGROUND:

When treating multiple sclerosis, lymphopenia is an established side effect of fumaric acids. Research demonstrating the impact of switching fumarates on lymphocyte counts is scarce. The objective of this study is to characterize the incidence and severity of lymphopenia in patients transitioning from dimethyl fumarate (DMF) to monomethyl fumarate (MMF) or diroximel fumarate (DRF).

METHODS:

A single center, retrospective review of adult medical records at the Vanderbilt Multiple Sclerosis Clinic who filled prescriptions for DMF then either DRF or MMF between January 1st, 2019 and February 28th, 2023.

RESULTS:

Patients who switched to DRF experienced a greater median percent change in absolute lymphocyte count (ALC) [45% decrease (IQR -95 to -14)] compared to those who switched to MMF [6% increase (IQR -4 to 17)]. Patients who switched to MMF, on average, had an ALC at follow-up 1.8 times higher than those switched to DRF (95% CI 1.4 to 2.3; p < 0.001). Additionally, patients switched to DRF were more likely to experience grade 2 or 3 lymphopenia compared to patients switched to MMF.

CONCLUSION:

Our study demonstrated that patients switched to DRF had a significantly lower ALC at follow-up and were more likely to have lymphopenia compared to patients switched to MMF.

2026-01-01·INTERNATIONAL IMMUNOPHARMACOLOGY

Effect of diroximel fumarate on gut dysbiosis and autoimmunity in the central nervous system

Article

作者: Ito, Naoko ; Kalina, J Tamar ; Rosario, Yaritza ; Yadav, Sudhir Kumar ; Dhib-Jalbut, Suhayl ; Ito, Kouichi ; Suresh, Shradha ; Shankar, Sai L

Diroximel fumarate (DRF) is an approved treatment for relapsing-remitting multiple sclerosis (RRMS). It has demonstrated better gastrointestinal tolerability compared to dimethyl fumarate (DMF). Recent studies indicate that gut dysbiosis may contribute to the development of multiple sclerosis (MS) and that reducing gut dysbiosis may be beneficial for disease progression. This study investigated the effects of DRF on gut dysbiosis and neuroinflammation in experimental autoimmune encephalomyelitis (EAE), an animal model of MS. We examined the effect of DRF on the expression of α4β1 and α4β7 integrins on CD4 + T cells, which are essential for guiding their migration into the central nervous system (CNS) and the gut, respectively. The expression of α4β1 increased in lymph nodes when EAE was induced, but DRF treatment reduced its expression and the production of IL-17. The expression of α4β7 on T cells was also upregulated in the lymph nodes, and the infiltration of α4β7 + CD4+ T cells into the gut increased following EAE induction. DRF treatment reduced the development of α4β7 + CD4+ T cells in the lymph nodes and their infiltration into the small intestine. This led to a decrease in the gut dysbiosis biomarker, lipocalin 2 (Lcn2), and a reduction in pathogenic bacteria from the Desulfovibrionaceae family in EAE mice. In patients with relapsing-remitting multiple sclerosis (RRMS), treatment with DRF showed a trend toward decreased levels of α4β1 + CD4+ T cells and endotoxin, which is a blood marker of intestinal permeability. Furthermore, DRF reduced the production of CCL22, a chemokine derived from macrophages. Overall, our findings suggest that DRF can reduce neuroinflammation by lowering the production of IL-17, inhibiting the development of α4β1 + CD4+ T cells and α4β7 + CD4+ T cells in the lymph nodes, and alleviating dysfunction associated with gut dysbiosis and intestinal permeability.

2025-11-15·Gut and Liver

Early Oral Refeeding in Patients with Post-Endoscopic Retrograde Cholangiopancreatography Pancreatitis: A Randomized Controlled Trial

Article

作者: Jang, Dong Kee ; Choi, Young Hoon ; Jung, Min Kyu ; Lee, Sang Hyub ; Han, Sung Yong ; Lee, Jae Min ; Choe, Jung Wan ; Kim, Ha Yan ; Jo, Jung Hyun ; Kim, Eui Joo

Background/Aims:

To assess the safety and efficacy of early oral refeeding (ERF) versus delayed refeeding (DRF) in patients with mild post-endoscopic retrograde cholangiopancreatography pancreatitis (PEP).

Methods:

Eligible patients were randomly assigned in a 1:1 ratio to the ERF or DRF group. Eligible patients were randomly assigned in a 1:1 ratio to the ERF or DRF group. In the ERF group, feeding began 24 hours after the diagnosis of PEP; in the DRF group, feeding commenced once normal bowel sounds returned and pain had decreased to a visual analog scale score of <2. The diet was advanced from clear fluids to soft foods according to patient tolerance. Refeeding was temporarily halted if the visual analog scale score reached ≥5 points or if intake was refused due to pain. Resumption required normal amylase/lipase levels, pain relief, and bowel movement restoration. Discharge criteria included patient well-being >24 hours post-diet. The primary outcome was PEP hospitalization duration, and secondary outcomes were the incidence of severe acute pancreatitis, readmission rate (<30 days), and PEP-related mortality rate.

Results:

A total of 80 patients (40 in each ERF and DRF group) were enrolled across nine referral centers. Baseline characteristics, procedural parameters and initial PEP severity were not significantly different between the two groups. Four ERF and three DRF patients had refeeding interruptions. ERF significantly reduced PEP hospitalization duration compared to DRF (2.93±1.59 days vs 3.78±1.97 days: relative risk, 0.75; 95% confidence interval, 0.59 to 0.97; p=0.026). Rates of severe acute pancreatitis, readmission, and mortality/morbidity related to PEP were similar between the two groups.

Conclusions:

ERF effectively shortens hospitalization in mild PEP patients without increasing safety risks (ClinicalTrials.gov identifier NCT04750044).

项与富马酸地罗西美相关的新闻（医药）

2026-03-18

·丁香园 Insight 数据库

全球神经科学 TOP5 药企

神经科学（CNS）药物因开发难度高、投入大，一度被药企视为最难啃的骨头之一。不过近些年，随着阿尔茨海默病、偏头痛等细分领域新疗法涌现，全球 CNS 交易并购明显回暖，这个领域正在重新成为跨国药企的必争之地。跨国药企中，谁已经提前完成布局，谁又在押注下一轮增长？本文基于 2025 年财报，梳理出了当前全球神经科学领域的头部药企，供参考。来源：Insight 整理（文末可下载各家的财报 PPT）罗氏 2025 年，罗氏以 118.88 亿美元（+11%）的收入稳坐 CNS 领域王者宝座。多发性硬化（MS）是罗氏 CNS 业务的核心支柱，Ocrevus（奥瑞利珠单抗）2025 年狂揽 84.74 亿美元，成为罗氏最稳定的现金牛产品，并牢牢占据 MS 市场龙头地位。在罕见神经疾病领域，罗氏布局的 Evrysdi（利司扑兰）作为全球首款口服脊髓性肌萎缩症（SMA）治疗药物，打破了 SMA 治疗依赖注射给药的局限，2025 年销售额达到 21.24 亿美元，与渤健 Spinraza（诺西那生）形成分庭抗礼的格局。罗氏并不满足于此，在 CNS 领域布局了丰富的管线。其中，在 AD 领域，罗氏利用「脑穿梭」技术开发的 Trontinemab 已进入 III 期临床，且牵手 Sangamo Therapeutics 开发静脉输注 AD 基因疗法。在 PD 领域，靶向 α-突触核蛋白的单抗 Prasinezumab 也已启动 III 期临床。艾伯维艾伯维曾公开表示要做全球最大的神经科学公司。2025 年，艾伯维神经科学业务收入首次突破百亿美元，同比增长 19.6% 达 107.67 亿美元，成为公司增长最快的板块，占公司总收入的比重达到 17.6%。这得益于多款产品的持续发力，帕金森病新药 Vyalev 正在快速放量，2025 年销售额达到 4.82 亿美元，成功接棒已上市超 20 年的老帕金森药物 Duodopa；精神分裂症药物 Vraylar（卡利拉嗪）表现同样亮眼，年销售额突破 36 亿美元。在细分赛道上，艾伯维的产品展现出强大的市场竞争力。以偏头痛业务为例，Ubrelvy（急性偏头痛）、Qulipta（偏头痛预防）、Botox Therapeutic（慢性偏头痛）已分别占据各细分赛道领先地位，均实现了两位数的增长，合力贡献了超 60 亿美元。此外，艾伯维还有不少在研管线，包括口服帕金森病新疗法 Tavapadon（已申报上市）、斥资 12 亿美元收购的新一代迷幻药 Bretisilocin（II 期）、抗癫痫药物 Darigabat（II 期）、阿尔兹海默病双抗 ALIA-1758（I 期）等，未来增长潜力充足。强生强生 2025 年 CNS 业务收入 78.37 亿美元，同比增长 10.1%，保持稳健增长态势。与其他企业不同，强生聚焦难治性、未满足临床需求的精神疾病领域，凭借创新作用机制的药物，走出了差异化竞争路线。 Spravato（艾司氯胺酮）是全球首款获批的难治性抑郁症鼻喷药物，具备快速起效的核心优势，2025 年销售额达到 16.96 亿美元，同比增长超 50%，是全球增长最快的精神疾病药物之一。另一款产品 Caplyta（卢美哌隆）是强生斥资百亿美元收购 Intra-Cellular 获得的，已获批治疗精神分裂症、双相情感障碍、重度抑郁症。凭借代谢副作用小、安全性高的优势，Caplyta 快速抢占市场，2025 年销售额达 7.0 亿美元。而且，强生试图通过持续扩大适应症，延长产品生命周期，以求 Caplyta 价值最大化。2025 年 7 月，强生已向 FDA 提交了其用于预防精神分裂症复发的补充新药申请。除精神疾病外，强生还在积极布局慢性炎症性脱髓鞘性多发性神经病（ARISE）、AD、广泛性焦虑障碍、帕金森病等领域的创新产品，其中针对失眠的 Orexin-2 受体拮抗剂 Seltorexant 已进入 III 期临床，针对 AD 的 Tau 蛋白的单抗 Posdinemab 已进入 II 期临床。渤健渤健是榜单中唯一一家以 CNS 为核心业务的跨国药企。作为老牌玩家，渤健 2025 年 CNS 业务收入达到了 63.90 亿美元，位居全球第四。在 MS 领域，渤健打造了全矩阵产品组合，涵盖口服、注射等多种剂型，可满足不同类型、不同病程的 MS 患者的治疗需求，包括 Tecfidera（富马酸二甲酯）、Vumerity（富马酸地洛西美酯）、Tysabri（那他珠单抗）等。2025 年，尽管面临罗氏 Ocrevus 和诺华 Kesimpta 的激烈竞争，这些产品依旧合计贡献了超 40 亿美元。在 SMA 领域，作为全球首款获批治疗该疾病的靶向治疗药物，渤健的 Spinraza（诺西那生）尽管已经上市多年，2025 年仍实现了 15.47 亿美元的销售额。在 AD 领域，渤健和卫材联合推出了 Leqembi（仑卡奈单抗）。这款产品被渤健寄予厚望，视为 Growth Products 的 6 款关键新产品之一，有分析师预测这款产品销售额峰值有望达到 35 亿美元，将成为渤健业绩的核心动力。诺华诺华是 2025 年神经科学领域增速最快的企业，其 CNS 业务收入大涨 25%，达到了 59.93 亿美元，核心增长动力正来自于 MS 精准治疗药物 Kesimpta（奥法妥木单抗）和 SMA 药物 Zolgensma。 Kesimpta 是一款皮下注射 CD20 单抗，凭借给药便捷、疗效优异、安全性好的优势，快速抢占市场，2025 年销售 44.26 亿美元（+36%），贡献了诺华神经科学收入的七成以上。图片来源：诺华 2025 年财报（文末可下载）此外，诺华依托基因治疗、RNA 疗法等前沿技术平台，在 SMA、AD 等领域布局了多款创新产品，包括 EDK060、VHB937 等。未来凭借高增速、强管线的优势，诺华有望在 CNS 赛道更进一步。结语 2025 年全球神经科学市场呈现头部集中、差异化竞争的格局，罗氏、艾伯维稳居第一阵营，强生、渤健、诺华各凭核心单品实现稳健增长。伴随神经科学领域技术的持续迭代，单抗、基因疗法、小核酸等创新药物正逐步登上舞台。未来，管线差异化、产品多元化、全球商业化将成为跨国药企的决胜关键。推荐阅读：全球药企 TOP15 大洗牌狂飙 VS. 失速！制药巨头中国区业绩生变 2025 年制药巨头研发投入 TOP10 榜单出炉 Insight 数据库整理了 2025 年 MNC 的财报，可扫描下方二维码添加小音，回复关键词「MNC财报」免费获得。封面来源：站酷海洛免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：newdrug PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn

2026-03-13

·蒲公英创投

渤健生物，江河日下的MS龙头

⭐关注微信公众号，领取蒲公英创投天使投资直通车链接。一家在最擅长的领域节节败退，却在最难啃的骨头上押下了全部筹码的公司。如果你问制药圈的人，哪家跨国药企这几年活得最憋屈，渤健（Biogen）大概率名列前茅。不是因为它做错了什么，而是因为它赖以为生的那个市场，正在不可逆地萎缩。渤健已经连续5年显示出全年营收的下跌趋势：2020年营收134.45亿美元，2021年骤降至109.82亿美元，2022年进一步下滑至101.73亿美元，2023年为98.36亿美元，2024年再降至96.76亿美元。五年，从134亿跌到97亿，跌去了近三成。这条下行的曲线背后，是一个无情的现实：多发性硬化症（MS），这个渤健深耕了四十年的主战场，正在被仿制药和竞品双重蚕食。多发性硬化：四十年的荣光，与无法逃脱的宿命多发性硬化症，一种中枢神经系统慢性炎症性脱髓鞘疾病，患者的免疫系统错误攻击自身髓鞘，导致神经信号传导受阻，引发肢体麻木、视力障碍、认知障碍等一系列症状。这个病，渤健比任何一家公司都懂。从上世纪90年代开始，渤健就是MS治疗领域的绝对主导者。Avonex（干扰素β-1a）、Tysabri（那他珠单抗）、Tecfidera（富马酸二甲酯）……一代代重磅产品，让渤健在MS领域建立起几乎无可撼动的地位。但这种地位，现在正在动摇。 2024年，渤健MS业务全年总收入43.5亿美元，同比下降7%。其中Tysabri收入17.15亿美元，同比下降8.6%。 Tecfidera（富马酸二甲酯）则已经提前走完了它的生命周期——专利到期后，仿制药的涌入几乎是毁灭性的，这款曾经支撑渤健半边天的口服MS药，销售额在几年内从高峰跌入谷底。 Vumerity（富马酸地洛西美）作为Tecfidera的升级换代产品，2024年销售额6.28亿美元，同比增长9% ，是MS板块少数逆势上涨的产品，但体量不足以弥补整体下滑。更让渤健头疼的是竞争格局的变化：罗氏的Ocrevus（奥瑞珠单抗）以半年一次的给药方式和更广的适应症覆盖，正在成为MS领域新的标准治疗，抢走的正是曾经属于Tysabri的份额。渤健预计2025年全年总收入将继续下降，降幅为中个位数百分比，MS业务的进一步下滑将只能被部分新产品收入所抵消。 Leqembi：一张迟迟没能起飞的王牌渤健为这个困局押注了一张什么牌？答案是仑卡奈单抗（Lecanemab），商品名Leqembi。 2014年，渤健与日本卫材签署合作协议，双方共同开发和商业化这款靶向β-淀粉样蛋白（Aβ）的单克隆抗体，用于治疗早期阿尔茨海默病。这不是一个新故事，这是一个渤健在阿尔茨海默病赛道上走了很久、摔了很重之后，再次站起来的故事。渤健在AD领域的第一个尝试，是阿杜那单抗（Aduhelm）。那是一次充满争议的FDA批准——临床数据不够扎实，咨询委员会多数投反对票，FDA仍以”加速批准”放行，随后引发轩然大波，医保拒绝报销，销售额一败涂地。2024年，渤健宣布放弃Aduhelm，将全部资源集中于仑卡奈单抗。这一次，数据更硬。 III期CLARITY AD研究显示，仑卡奈单抗治疗18个月后，患者认知衰退速度较安慰剂组减缓27%，日常生活能力衰退减少37%。这是真实的疗效信号，不是统计噪音。截至2025年，仑卡奈单抗已在包括美国、中国、欧洲等在内的51个国家和地区获批。在中国，2024年1月国家药监局批准仑卡奈单抗注射液在华上市，商品名乐意保。该药尚未进入医保，患者自费一年的治疗费用约18万元人民币。在欧洲，仑卡奈单抗成为欧盟首个批准用于延缓早期阿尔茨海默病进展、针对疾病潜在病因的药物。 2025年1月，FDA又批准了仑卡奈单抗每4周一次静脉注射维持剂量的新方案——患者完成18个月每两周一次的起始治疗后，可转为每月一次的维持治疗，注射频率大幅降低。以上每一个里程碑，都写着”好消息”。但现实是：仑卡奈单抗2024年全年销售额2.81亿美元——相较2023年的1000万美元已大幅提升，但与行业此前对一款”阿尔茨海默病突破性疗法”的期待相去甚远。 Leqembi起飞慢：一道真实的商业化难题仑卡奈单抗的商业化困境，不是药的问题，而是这个病的问题。阿尔茨海默病的诊断门槛极高——在用药之前，患者必须通过PET扫描或脑脊液检测确认脑内淀粉样蛋白的存在，而这类检测目前在大多数医院都不是常规项目，费用高昂，基础设施严重不足。换句话说：即便这款药有效，患者也很难走完”诊断→确认→用药”的完整路径。渤健和卫材仍在大力推进Leqembi的后续开发。皮下注射制剂（SC）稳步推进，用于维持治疗的Leqembi SC注射笔已在美国申报上市，用于初始治疗的皮下注射笔有望在2026年上半年获批。从静脉注射到皮下注射，这是Leqembi商业化的关键一跃——皮下注射可以居家自行操作，不再需要每两周跑医院挂点滴，患者依从性和可及性将发生质的飞跃。这一步若能实现，Leqembi的放量速度有望迎来真正的拐点。砍掉4条管线：不是认输，是校准 2024年财报，渤健同时宣布了一个让人五味杂陈的消息：终止开发4款中枢神经系统药物——针对早期阿尔茨海默病的BIIB113（口服TAU聚集抑制剂）、针对帕金森病的BIIB094（靶向LRRK2的反义寡核苷酸）、针对多系统萎缩症的BIIB101（靶向α-突触核蛋白的反义寡核苷酸），以及针对糖尿病周围神经性疼痛的BIIB143。四条管线，全部在中枢神经系统领域，全部是渤健花了多年时间和大量资金培育的项目。 2024年，渤健研发投入20.42亿美元，同比下降17.1%。研发投入下降，不是因为渤健不再相信科学，而是因为它必须把有限的子弹，打在最有可能命中的靶上。这是一家公司在资源约束下不得不做的取舍，也是渤健试图用更高效的资本配置走出困境的信号。脊髓性肌萎缩：另一块被低估的压舱石在讨论渤健的困境时，很多人忘了提一件事：这家公司在脊髓性肌萎缩症（SMA）领域，同样是全球先行者。诺西那生（Spinraza），全球第一款获批的SMA治疗药物，靶向SMN2基因的反义寡核苷酸疗法，在这个此前几乎没有任何治疗手段的罕见病领域，曾经是绝对的革命性产品。但SMA赛道的竞争格局同样在剧烈变化——诺华的基因治疗Zolgensma和罗氏的利司扑兰（Evrysdi）相继上市，前者一针治愈、后者口服便利，都在持续蚕食Spinraza的市场份额。渤健罕见病业务2024年收入19.88亿美元，同比增长10%。 10%的增长，是渤健整体业绩里难得的亮点。但Spinraza的长期走势，和MS产品一样，同样面临着被更新技术路线替代的压力。老王国的黄昏，与新大陆的黎明前从2020年的全球制药巨头排名第20位，到如今下滑至第25位。这个排名的变化，是渤健这几年处境最直观的注脚。但排名下滑，不等于这家公司失去了价值。渤健在神经科学领域深耕40年积累的know-how，是任何一家公司都无法在短时间内复制的。它在MS领域建立的临床数据库、患者网络、医生关系，构成了一道低调但真实的护城河。它与卫材合作打造的仑卡奈单抗，是人类历史上第一款被大规模临床证明能够减缓阿尔茨海默病进展的药物——无论商业化爬坡多慢，这个科学价值本身不会消失。渤健现在的处境，像极了一个守着一座老矿的矿主——老矿的出矿量在逐年下降，但他刚刚在旁边发现了一座可能更大的新矿，只是还没有找到最好的开采方式。 MS，是那座正在枯竭的老矿。 Leqembi，是那座还没有完全打开的新矿。老矿能支撑多久，新矿何时真正爆发，是渤健现在最重要的两个问题，也是这家公司在接下来几年里最值得持续观察的剧情。 ⭐点击关注，获得完整版融资案例蒲公英创投（OurVentures），是中国制药产业生态圈领先的科技服务平台“蒲公英Ouryao”旗下的创业投资孵化平台，建设有蒲公英生物医药孵化器，专注生物医药领域早期投资，寻找好项目、挖掘好苗子，联动蒲公英十大板块（创投、教育、媒体、论坛、产品、园区、联盟、商城、人才、咨询）资源，为蒲友创业赋能，为企业发展助力，长期陪伴共成长。地址：深圳市南山区学苑大道1001号南山智园2期D3栋22层

2026-02-23

·BioSpace

QurAlis Demonstrates Effects on Disease Progression and Target Engagement in ANQUR Clinical Trial of QRL-201, a First-in-Class Precision Medicine in Development for Sporadic ALS

First clinical development program in sporadic ALS patients to show potential to restore STATHMIN-2 expression, a statistically significant effect on a neurofilament biomarker, and an overall trend of slowing disease progression as measured by the ALSFRS-R Subgroup analysis showed statistically significant effect on ALSFRS-R Interim data support including an open-label extended dosing period to ANQUR protocol; approval received in Canada and under regulatory review in other regions; preparations underway to advance QRL-201 into pivotal Phase 3 clinical trial in 2027 CAMBRIDGE, Mass.--(BUSINESS WIRE)-- QurAlis Corporation (“QurAlis”), a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that have the potential to alter the trajectory of neurodegenerative and neurological diseases, today announced interim data from its Proof-of-Concept Phase 1/2 ANQUR clinical trial of QRL-201 in people living with amyotrophic lateral sclerosis (ALS). Results from this study demonstrated QRL-201 had an effect on disease progression in ALS patients; this effect was confirmed by markers of clinical efficacy and changes in a clinically relevant biomarker, along with target engagement. QRL-201 is a first-in-class, potent antisense oligonucleotide (ASO) precision therapeutic in development to restore STATHMIN-2 (STMN2) expression in ALS patients with the aim to modify disease progression and improve outcomes. STMN2 is a protein important for muscle innervation that is regulated by TDP-43 and downregulated in the majority of ALS patients. ANQUR (QRL-201-01; NCT05633459) is the first-ever clinical trial to evaluate a potential therapy to rescue STMN2 expression in people with ALS. The ANQUR study is a double-blind, placebo-controlled study which dosed a total of 69 patients across the dose escalation (n=17) and dose-range finding (DRF) (n=52) phases of the study. “I am encouraged by these data from the ANQUR clinical trial of QRL-201. The combination of effects on target engagement biomarkers, disease biomarkers such as neurofilament, and clinical measures is very promising and remarkable to see from an early stage clinical trial,” said Merit E. Cudkowicz, M.D., M.Sc., director, Sean M. Healey & AMG Center for ALS and executive director of the Mass General Brigham Neuroscience Institute, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “ALS is a devastating, fatal neurodegenerative disease with a significant unmet medical need. Our mission is to make a meaningful difference for people living with ALS. We believe QRL-201 has the potential to modify disease progression and improve outcomes for sporadic ALS patients,” said Kasper Roet, Ph.D., CEO and co-founder of QurAlis. “These results from the ANQUR clinical trial are well aligned with our understanding of STMN2 biology and the potential of STMN2 restoration to have a positive impact on ALS disease progression. We are grateful to the ANQUR clinical trial team, participants and their families, and look forward to advancing the QRL-201 clinical program to deliver a much-needed precision medicine option for people living with ALS.” Results from the interim analysis of the ANQUR clinical trial show: Target engagement, confirmed through tissue analysis, with broad distribution across the spinal cord and motor cortex, statistically significant increased levels of STMN2, above the estimated therapeutic target, and correction of STMN2 mis-splicing, as compared to a cohort of natural history samples. A statistically significant and clinically meaningful reduction in phosphorylated neurofilament heavy (pNfH) in the low dose group. An encouraging trend of slowing decline in ALSFRS-R in sporadic patients, including a strong trend on the gross motor sub-score, which is critical for patient independence. In a post-hoc analysis excluding patients with the highest levels of baseline neurofilament light, which is associated with faster disease progression, there was a statistically significant and clinically meaningful slowing of decline in ALSFRS-R at the six-month (24 week) timepoint in treated sporadic ALS patients compared to placebo. QRL-201 was generally safe and well tolerated with most adverse events (AEs) reported as mild or moderate. During all phases of the study, and most recently in December 2025, the unblinded Data Safety Monitoring Board has recommended the study continue without modification. These data support an open-label extended dosing period to the ANQUR clinical trial protocol under which all eligible trial participants will be given the opportunity to receive QRL-201 at the low dose of the DRF portion of the study. The open-label extended dosing period to the ANQUR clinical trial has been approved in Canada and is currently under regulatory review in the European Union and the United Kingdom. In parallel, QurAlis is preparing to advance QRL-201 into a pivotal Phase 3 clinical trial in 2027. About STATHMIN-2 STATHMIN-2 (STMN2) is a well-validated protein important for neural repair, axonal stability, and muscle innervation and is the most significantly regulated gene by TDP-43 exclusively in humans. STMN2 has the most consistently decreased expression across multiple ALS RNA expression studies. Loss of nuclear TDP-43 leads to mis-splicing of the pre-mRNA of STMN2, resulting in loss of full-length transcript and protein. QRL-201 rescues STMN2 loss of function and neurodegenerative phenotypes in QurAlis’ ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. TDP-43 pathology is associated with nearly all ALS patients, approximately 50 percent of patients with frontotemporal dementia (FTD), the second most common form of dementia, and with about a third of Alzheimer’s Disease (AD) patients. There are currently no cures for ALS or FTD, and there are limited therapeutic options available for ALS and FTD patients who are in desperate need of effective therapies. About ANQUR Clinical Trial QRL-201 is being evaluated in the Phase 1/2 ANQUR clinical trial (QRL-201-01; NCT05633459), the first-ever clinical study designed to assess a potential therapy aimed at rescuing STATHMIN-2 (STMN2) expression in people with amyotrophic lateral sclerosis (ALS). The ANQUR trial completed the dose-escalation phase, based on pharmacokinetic analyses from Cohorts 1 and 2 that demonstrated cerebrospinal fluid exposure levels of QRL-201 met or exceeded the targeted therapeutic range. The study then advanced to the dose range-finding phase, which is evaluating two dose levels of QRL-201 and incorporates additional biomarker assessments. The study is currently active in Canada, the United Kingdom, and the European Union, and includes participants with both sporadic ALS and C9orf72-related ALS. More information about the ANQUR clinical trial can be found at . About QurAlis Corporation At QurAlis, we are neuro pioneers on a quest to cure, boldly seeking to translate scientific breakthroughs into powerful precision medicines. We work collaboratively with a relentless pursuit of knowledge, precise attention to craft, and compassion to discover and develop medicines that have the potential to transform the lives of people living with neurodegenerative and neurological diseases. QurAlis is the leader in development of precision therapies for amyotrophic lateral sclerosis (ALS). In addition to ALS, QurAlis is advancing a robust precision medicine pipeline to bring effective disease-modifying therapeutics to patients suffering from severe diseases defined by genetics and clinical biomarkers. For more information, please visit or follow us on X @QurAlisCo or LinkedIn . Contacts Kathy Vincent kathy.vincent@quralis.com

临床结果临床3期

100 项与富马酸地罗西美相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11154	富马酸地罗西美	L03	DB14783

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
复发-缓解型多发性硬化	欧盟	BIOGEN NETHERLANDS BV	2021-11-15
复发-缓解型多发性硬化	冰岛	BIOGEN NETHERLANDS BV	2021-11-15
复发-缓解型多发性硬化	列支敦士登	BIOGEN NETHERLANDS BV	2021-11-15
复发-缓解型多发性硬化	挪威	BIOGEN NETHERLANDS BV	2021-11-15
多发性硬化症	瑞士	Biogen, Inc.	2021-09-20
复发性多发性硬化	美国	Biogen, Inc.	2019-10-29

未上市

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
GM1神经节苷脂贮积病	临床3期	美国	Alkermes, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	美国	Biogen, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	比利时	Alkermes, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	比利时	Biogen, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	保加利亚	Alkermes, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	保加利亚	Biogen, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	加拿大	Biogen, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	加拿大	Alkermes, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	德国	Biogen, Inc.	2015-12-10
GM1神经节苷脂贮积病	临床3期	德国	Alkermes, Inc.	2015-12-10

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临床结果

适应症

分期

评价

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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


NCT05083923 (CTgov)	临床3期	复发性多发性硬化	136	DRF 462+DRF (Cohort 1: Japanese Participants)	鹹膚積淵廠鹽膚糧餘襯 = 壓獵餘築餘願繭築選餘齋鹹願網積鬱襯網膚窪 (製餘選顧願艱顧齋齋廠, 鑰廠窪選壓鏇選鏇鹹鬱 ~ 範遞範遞積鹽衊選築鏇) 更多	-	2025-10-24
				DRF 462+DRF (Cohort 2: Chinese Participants)	鹹膚積淵廠鹽膚糧餘襯 = 齋夢糧鹹衊憲淵齋繭顧齋鹹願網積鬱襯網膚窪 (製餘選顧願艱顧齋齋廠, 齋鬱膚醖膚憲淵壓選襯 ~ 獵衊築選窪壓淵觸獵鏇) 更多
NCT02634307 (EVOLVE-MS-1, AAN2025)	临床3期	GM1神经节苷脂贮积病 \| 复发-缓解型多发性硬化	1,057	Diroximel Fumarate (DRF)	築鹹襯醖獵餘衊遞壓襯(鏇襯鹽艱膚願襯鏇積襯) = 襯鑰艱積齋築衊鬱鏇鬱製築糧製鹹鏇製廠蓋艱 (範窪觸鏇獵鑰醖簾憲鏇, 3.8 ~ 8.7) 更多	积极	2025-04-07
				Ocrelizumab (OCR)	築鹹襯醖獵餘衊遞壓襯(鏇襯鹽艱膚願襯鏇積襯) = 鑰廠淵膚衊獵醖糧獵醖製築糧製鹹鏇製廠蓋艱 (範窪觸鏇獵鑰醖簾憲鏇, 7.1 ~ 11.1) 更多
NCT02634307 (EVOLVE-MS-1, Pubmed)	临床3期	复发-缓解型多发性硬化 lymphopenia \| ALC decline	-	Diroximel Fumarate	衊壓鬱餘獵衊顧鑰壓醖(網網顧願夢鏇鹹淵簾繭) = 艱遞鹽遞積夢遞積遞襯廠選鹹餘壓範顧廠構構 (顧憲膚製襯齋觸窪觸憲 )	积极	2024-06-27
NCT02634307 (EVOLVE-MS-1, Pubmed)	临床3期	复发-缓解型多发性硬化	1,057	Diroximel fumarate	淵醖觸糧鬱遞選製憲網(窪艱淵齋廠鹽遞淵願鹹) = 糧鬱醖壓餘築膚憲窪築艱繭壓遞構齋構憲構壓 (鹹餘淵憲鑰鹹夢夢餘鬱 )	积极	2024-06-15
				Dimethyl fumarate	淵醖觸糧鬱遞選製憲網(窪艱淵齋廠鹽遞淵願鹹) = 齋夢糧獵餘醖壓簾範廠艱繭壓遞構齋構憲構壓 (鹹餘淵憲鑰鹹夢夢餘鬱 )
AAN2024	N/A	多发性硬化症	-	Fumarates (Black)	遞夢範憲觸獵艱鹽齋觸(鏇鹽繭遞選壓觸選積鹹) = 淵餘鑰壓範夢襯衊齋鏇衊鏇鬱觸襯獵鏇壓獵衊 (廠鏇構築範膚鑰夢蓋簾, 73.7–80.3)	-	2024-04-09
				Fumarates (Hispanic)	遞夢範憲觸獵艱鹽齋觸(鏇鹽繭遞選壓觸選積鹹) = 壓鹽壓憲顧憲願簾繭醖衊鏇鬱觸襯獵鏇壓獵衊 (廠鏇構築範膚鑰夢蓋簾, 71.0–80.0)
ACTRIMS2024	N/A	多发性硬化症	-	Fumarates (Black)	網衊鬱觸積製鹽壓艱醖(網憲鹹網膚膚鑰鑰選願) = 醖範夢鏇鬱選艱範衊網鹹夢窪鬱選範鏇襯鹽壓 (願淵築選壓選構鑰膚簾 ) 更多	-	2024-03-01
				Fumarates (Hispanic)	網衊鬱觸積製鹽壓艱醖(網憲鹹網膚膚鑰鑰選願) = 廠構餘築選醖淵蓋壓願鹹夢窪鬱選範鏇襯鹽壓 (願淵築選壓選構鑰膚簾 ) 更多
SUNBEAM (RPC01-301), RADIANCE phase 3 (RPC01-201B), EVOLVE-MS-1 (ACTRIMS2023)	临床3期	复发-缓解型多发性硬化	-	Ozanimod 1.0 mg	蓋衊糧壓網鹽齋築構鏇(築築壓鏇醖糧衊製醖範): risk ratio = 0.924 (95% CI, 0.646 ~ 1.324) 更多	积极	2023-05-30
				Diroximel fumarate 462 mg
NCT02425644 \| NCT02634307 (EVOLVE-MS-1 \| OPTIMUM, Pubmed)	临床3期	复发性多发性硬化	-	Diroximel fumarate	襯顧願糧鹹積餘鑰憲鏇(鏇憲積壓蓋鬱範簾簾選) = 遞鹹蓋製醖襯獵夢鬱窪壓製範糧窪積範願鬱鏇 (築壓淵製鹽範製蓋顧憲 )	-	2023-05-08
NCT02634307 (EVOLVE-MS-1, AAN2023)	临床3期	复发-缓解型多发性硬化	239	Diroximel Fumarate	醖選鏇繭願壓餘壓願憲(餘繭廠鑰繭觸艱範餘簾) = 23 (9.6%) patients discontinued DRF due to adverse events 繭顧壓網鬱鏇簾觸鬱顧 (觸鑰構醖繭獵願齋壓淵 ) 更多	积极	2023-04-25
NCT02634307 (EVOLVE-MS-1, AAN2023)	临床3期	多发性硬化症	1,057	Diroximel fumarate	選積鬱壓淵夢壓窪製築(膚醖糧遞糧襯顧簾遞窪) = 繭鬱顧製鏇鏇糧膚夢衊膚鑰簾憲膚繭積夢醖醖 (糧構淵夢齋窪糧顧淵醖 ) 更多	积极	2023-04-25
				Placebo	選積鬱壓淵夢壓窪製築(膚醖糧遞糧襯顧簾遞窪) = 廠遞餘餘糧鏇繭鏇網餘膚鑰簾憲膚繭積夢醖醖 (糧構淵夢齋窪糧顧淵醖 ) 更多