A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

开始日期2022-05-05

申办/合作机构

Incyte Corp.

NCT05099445

/ Completed临床1期

A Phase 1 Study to Evaluate the Pharmacokinetics, Safety, and Pharmacodynamics of INCB000928 in Participants With Impaired Renal Function and Hemodialysis

This is a multicenter, open-label parallel-group to evaluate single oral doses of INCB000928 in participants with varying level of renal function or impairment.

开始日期2021-12-14

申办/合作机构

Incyte Corp.

NCT04582539

/ Terminated临床1/2期

A Phase 1/2, Open-Label, Multicenter Study of INCB000928 Administered as a Monotherapy in Participants With Anemia Due to Myelodysplastic Syndromes or Multiple Myeloma

This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

开始日期2021-08-19

申办/合作机构

Incyte Corp.

100 项与 Zilurgisertib 相关的临床结果

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100 项与 Zilurgisertib 相关的转化医学

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100 项与 Zilurgisertib 相关的专利（医药）

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项与 Zilurgisertib 相关的文献（医药）

2026-05-01·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Effects of Renal and Hepatic Impairment on the Pharmacokinetics of Zilurgisertib

Article

作者: Phillip Wang ; Yan-ou Yang ; Xiang Liu ; Jay Getsy ; Kevin Rockich ; Yunlan Fang

Aim:

Small‐molecule activin receptor‐like kinase‐2 inhibitor zilurgisertib (INCB000928) is under investigation in a pivotal trial for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultrarare genetic condition. This analysis assessed effects of renal and hepatic impairment on zilurgisertib pharmacokinetics.

Methods:

Two open‐label, parallel‐group studies were conducted. Participants with renal or hepatic impairment were matched with healthy controls and received a single 200‐mg dose of zilurgisertib; those with end‐stage renal disease (ESRD) received doses before and after haemodialysis. Plasma concentrations were quantified via validated assay; pharmacokinetic parameters were derived from non‐compartmental analysis. Geometric mean ratios (GMRs) and 90% confidence intervals (CIs) for C max , AUC 0‐t and AUC 0‐∞ were compared by ANOVA.

Results:

Compared with matched controls, GMRs (90% CIs) for C max and AUC 0‐∞ were as follows: mild renal impairment, 0.92 (0.64–1.34) and 0.98 (0.77–1.24); moderate, 1.22 (0.92–1.63) and 1.64 (1.28–2.10); severe, 1.00 (0.74–1.35) and 1.70 (1.33–2.16), respectively. Among participants with ESRD, values were the following: before haemodialysis, 1.17 (0.91–1.49) and 1.47 (1.15–1.88); after, 1.32 (1.00–1.75) and 1.68 (1.29–2.19), respectively. Among participants with hepatic impairment, values were as follows: moderate, 1.06 (0.80–1.42) and 1.13 (0.93–1.38); severe: 1.36 (1.05–1.76) and 1.25 (1.06–1.48), respectively.

Conclusions:

Zilurgisertib exposure was unchanged with mild renal or moderate hepatic impairment, but increased by 30%–70% with moderate or more severe renal impairment and by 25% with severe hepatic impairment. These findings, alongside the therapeutic index, to be defined by the ongoing pivotal FOP trial (NCT05090891), will guide dosing in these populations.

2026-02-01·Clinical Pharmacology in Drug Development

Evaluation of clinical cardiac safety of zilurgisertib, an activin receptor‐like kinase‐2 (ALK2) inhibitor, in healthy participants

Article

作者: Liu, Xing ; Yang, Hong ; Gong, Xiaohua ; Yang, Yan‐ou ; Getsy, Jay ; Rockich, Kevin

Abstract:

The oral, small molecule inhibitor of activin receptor‐like kinase‐2, zilurgisertib (INCB000928), is under evaluation in fibrodysplasia ossificans progressiva. Cardiac safety was assessed using electrocardiogram (ECG) parameters and a plasma concentration‐heart rate‐corrected QT (C‐QTc) interval analysis of pooled data from single ascending dose (SAD) and multiple ascending dose (MAD) studies of zilurgisertib in healthy adult participants (SAD: 10‐500 mg; INCB00928‐102: 50‐400 mg QD, 300 mg BID). Overall, 91 (SAD) and 79 (MAD) participants provided at least one pair of PK/ECG data. As both studies indicated a dose‐dependent effect of zilurgisertib on heart rate, individualized QT correction (QTcI) was used as the primary endpoint for QTc analysis. Estimated population slope of the individualized C‐ΔQTc (C‐ΔQTcI) relationship was shallow (0.02 ms per µ m [90% CI, −0.60, 0.65]) and not statistically significantly different from 0; treatment effect–specific intercept was small and not significant (−0.83 ms [90% CI, −2.26, 0.61]). No significant relationship between zilurgisertib plasma concentration and change in QTcI was identified; zilurgisertib did not have a clinically relevant effect on QTc prolongation. QT effect > 10 ms could therefore be excluded within the dose range studied (up to 300 mg BID). No clinically meaningful effects on cardiac conduction (PR and QRS intervals) or any categorical PR or QRS outliers were observed. These data support further clinical development of zilurgisertib.

2025-11-13·ACS Medicinal Chemistry Letters

Discovery and Characterization of Zilurgisertib, a Potent and Selective Inhibitor of Activin Receptor-like Kinase-2 (ALK2) for the Treatment of Fibrodysplasia Ossificans Progressiva

Article

作者: Sylvie Teixeira-Fouchard ; Alexandra Hinniger ; Victoria Head ; Ina Kramer ; Michaela Kneissel ; Sven Weiler ; Luca Arista ; Thomas Ullrich ; Nikolaus Stiefl ; Sabine Guth ; Carien Dekker

Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disease leading to progressive soft tissue heterotopic ossification (HO) with no curative treatment available to date. It is caused by gain-of-function mutations in the activin A type-1 receptor ACVR1/ALK2, a member of the bone morphogenetic protein (BMP) type I receptor family. Most recent clinical trials in FOP have adopted for the first time on-target therapies to normalize the aberrant ALK2 receptor activity. Here we describe the discovery and preclinical characterization of zilurgisertib, a novel small-molecule inhibitor of ALK2 kinase with high biochemical and cellular potency, selectivity over other BMP and TGFβ signaling receptor kinases, and excellent oral bioavailability in preclinical species. Zilurgisertib fully suppresses HO in a pediatric mouse model of injury-induced FOP and therefore holds great potential as a novel targeted disease-modifying therapy for FOP. The candidate is being evaluated in clinical trials.

项与 Zilurgisertib 相关的新闻（医药）

2026-09-28

·Firstwordpharma

Mirum tees up filing for hepatitis delta hopeful after Phase III win

Mirum Pharmaceuticals said Monday that brelovitug passed a key test on its way to become a new treatment to patients with chronic hepatitis delta virus (HDV), with the antibody meeting the primary endpoint in the Phase III portion of AZURE-1. The findings put Mirum a step closer to a potential US filing."These results were achieved in a broad patient population, including those with significant liver inflammation, cirrhosis and clinically significant portal hypertension," said Nancy Shulman, EVP of clinical development at the company. "We look forward to reporting topline results from AZURE-4 later this year and advancing toward our planned BLA submission in the first half of 2027."The Phase III portion of AZURE-1 enrolled 153 treatment-naive patients who were randomised to a self-administered, 300-mg subcutaneous brelovitug injection once weekly, 900 mg once every four weeks, or delayed treatment starting at week 24.According to the company, 56% of patients in the weekly arm and 45% in the monthly arm achieved the primary endpoint, which required both a virologic response and ALT normalisation at week 24, compared to 0% in the delayed-treatment group (p<0.0001 for both comparisons). Virologic response — defined as undetectable HDV RNA or decline in HDV RNA ≥2 log10 IU/mL — was seen in 86% and 85% of patients in the two brelovitug groups, respectively, while ALT normalised in 63% and 54%.There were no serious adverse events (AEs) in either treatment group, nor any drop-outs as a result of AEs. Mirum said the drug was well tolerated in both dose arms.Deepening responsesMeanwhile, Mirum also reported longer-term data from the 53-patient Phase IIb portion of AZURE-1, with responses generally deepening over time. In the 300-mg weekly group, the proportion achieving the combined endpoint rose from 45% at week 24 to 55% at week 48, while the proportion with HDV RNA below 10 IU/mL went from 45% to 80% at those two timepoints.Among those receiving 900 mg every four weeks, the combined response rose from 35% to 55%, while HDV RNA below 10 IU/mL increased from 10% to 40%.ALT normalisation increased from 45% to 55% in the 300-mg weekly group and from 40% to 55% in the 900-mg every-four-week group between weeks 24 and 48."Seeing virologic response alongside ALT normalisation is encouraging in a long-term therapy," commented Norah Terrault, an AZURE-1 investigator. "For patients facing the most aggressive form of viral hepatitis, these are important results."Monday's readout comes on the heels of an FDA approval for Mirum just last week. On Friday, US regulators cleared its oral drug Atebrioz (zilurgisertib) for fibrodysplasia ossificans progressiva.Upcoming Vir readoutsCommenting on the AZURE-1 results, Stifel analysts said Mirum will face competition from Vir Biotechnology, which is expected to report Phase III data for a combination regimen consisting of investigational siRNA elebsiran with mAb tobevibart. Topline data from ECLIPSE 1 are due in the fourth quarter, followed by results from ECLIPSE 2 and ECLIPSE 3 in early 2027. Vir's regimen is administered once monthly as two separate sequential subcutaneous injections.Vir has said 96-week data from the Phase II SOLSTICE study data showed that 88% (28/32) of patients receiving the elebsiran/tobevibart combo achieved undetectable HDV RNA, compared to 53% (17/32) of those on tobevibart monotherapy.Meanwhile, Gilead Sciences' Hepcludex (bulevirtide-gmod) became the first treatment greenlit in the US for chronic HDV infection, after the FDA granted it accelerated approval earlier this year. However, a poll conducted by FirstWord suggested a lack of disease awareness could be a hurdle to uptake (see – Physician Views Results: Hepcludex marks meaningful advance in HDV, but first it must find its people).

临床结果临床3期上市批准临床2期

2026-09-28

·从凌开始管好自己

ABC康谈早知道 · 第017期

2026年9月28日星期一｜秋分（第6天）早上好！今天9点30分，《ABC康谈早知道》如约而至。秋分第6天，新的一周，早晨出门凉意更重了，记得添衣。今天医讯里，2027年度居民医保集中缴费已在多地启动，一年一度的"续保提醒"来了；技术板块有一台国内首例的生物人工血管手术，还有让儿童1型糖尿病"按下暂停键"的新疗法；生物医药板块，FDA一天批准了两款口服新药；科普讲讲1型和2型糖尿病到底有什么不一样；文末的健脑小游戏换成"健康小算盘"，算一算藏在生活里的健康数字！上一期答案揭晓 · 第016期（歇后语填空）上一期我们玩的是歇后语填空，五条歇后语的"下半句"，你补全了几条？ 1. 第1题（★☆☆）：芝麻开花—— 节节高。点评：芝麻由下往上一节一节开花，日子越过越好的意思，最常用的一条。 2. 第2题（★☆☆）：小葱拌豆腐—— 一清二白。点评：青（清）白分明，借颜色说做人清白、账目清楚。 3. 第3题（★★☆）：八仙过海—— 各显神通。点评：八位神仙各凭法宝渡海，比喻各拿出看家本领。 4. 第4题（★★☆）：泥菩萨过江—— 自身难保。点评：泥菩萨入水就化，比喻自顾不暇、顾不上别人。 5. 第5题（★★★）：姜太公钓鱼—— 愿者上钩。点评：直钩离水三尺钓鱼，钓的不是鱼而是明主，比喻心甘情愿入局。今日医讯医保更新 1. 2027年度城乡居民医保集中缴费启动：个人最低400元、财政补助不低于724元，断缴有等待期进入9月以来，全国多地陆续启动2027年度城乡居民基本医疗保险集中征缴，集中缴费期多为2026年9月1日至12月31日。国家划定的最低个人缴费标准为每人每年400元，财政补助标准不低于每人每年724元——即个人缴400元、财政配套724元，全年筹资合计1124元（各地标准不同，如宁波成年居民个人缴800元、补助2120元；呼和浩特低保、特困等困难群体个人只缴200元）。需要特别提醒的是：未在集中征缴期续保的视为断保，重新参保将设置待遇等待期（固定3个月、每多断一年再加1个月），等待期内看病医保不能报销；而连续参保满4年后每多缴一年、以及当年零报销，次年大病保险最高支付限额都能提高至少1000元。（来源：各地医保、税务部门通知；腾讯新闻、今日头条9月26日报道） → 对普通人意味着什么：家里老人、孩子的居民医保，最近就适合办了。一年400元、财政补700多元，是杠杆最高的一笔保障支出；千万别为省这笔钱断缴——断缴后错过等待期，一场住院可能全部自费。缴费认准当地医保局、税务局的官方渠道。 2. "半年一针"的降脂针英克司兰，医保报销明确"限二线" 国家医保局在8月下旬发布的政策文件中，把"半年打一次"的降脂针英克司兰钠注射液增补进医保报销"限二线使用"的药品范围：只有在他汀等一线降脂治疗疗效不佳或不耐受时，才可由医保报销；直接当"一线用药"使用则医保不予支付。该药2026年以来已纳入医保，价格从每针9988元降至约2790元，单药使用可使低密度脂蛋白胆固醇（LDL-C）下降约50%。（来源：国家医保局官网文件；腾讯新闻9月25日报道） → 对普通人意味着什么：降脂针再方便，也绕不开"先他汀"这条规则。体检发现血脂高，第一步仍是改善生活方式加规范使用他汀；确实控制不住或不耐受，再由医生评估是否启用二线方案，别急着"扎一针了事"。医疗技术 1. 国内首例！生物人工血管"搭出"透析生命线，落地博鳌乐城上海交通大学医学院附属瑞金医院海南医院（海南博鳌研究型医院）近日成功完成国内首例以特许器械Artegraft生物人工血管作为移植物的人工血管动静脉内瘘（AVG）手术，为患者建立了后续规律血液透析所需的稳定血管通路。这也是这款海外创新血管在国内血液透析通路领域的首次临床应用。该产品尚未在国内上市，目前仅获批在乐城先行区内使用，本次手术填补了国内生物源性人造血管在血透通路领域的临床应用空白。（来源：海南省人民政府网，9月报道） → 对普通人意味着什么：尿毒症患者做透析，前提是有一条可靠的"血管通路"。长期透析患者血管往往"越用越枯"，生物人工血管给血管条件耗竭的患者多了一条"生命线"选择；乐城先行区"先试先用"的机制，也让国际新产品更快触达国内患者。 2. 全国首例！儿童1型糖尿病"疾病修饰治疗"落地：还没发病，先按暂停键华中科技大学同济医学院附属同济医院8月24日为一名1型糖尿病2期患儿实施替利珠单抗疾病修饰治疗，是该药获批后在国内的首例儿童患者临床应用（湖南日报相关报道9月26日刊发）。1型糖尿病是自身免疫系统错误攻击胰岛β细胞所致，新版防治指南将其分为三期：1期已有抗体但血糖正常，2期糖代谢异常但仍无典型症状，3期才是传统意义上的"确诊发病"；进入2期后儿童中位进展至3期的时间仅约1.9年。疾病修饰治疗的目标是延缓进展、保护残余胰岛功能——本次接受治疗的11岁女孩此前已确诊2期，输注过程耐受良好，近日复诊指标平稳。（来源：湖南日报·今日头条，9月26日） → 对普通人意味着什么：过去孩子查出"糖尿病前期"样的2期，只能等发病再打胰岛素；如今在无症状阶段干预、把发病时间往后推甚至拦住，成为可能。家里孩子有多饮、多尿、体重下降，或尿糖阳性时，尽早去儿科内分泌专科筛查胰岛自身抗体，"早一步"现在真的有用。医疗与生物医药 1. FDA一天批准两款口服新药：罕见骨病有了"每天一片"，帕金森病新添多巴胺类似物 9月26日，美国FDA同日批准两款每日一次的口服新药：一是Mirum制药与Incyte合作的Atebrioz（zilurgisertib，ALK2抑制剂），用于12岁及以上进行性骨化性纤维发育不良（FOP）患者，减少异位骨化的新增体积——FOP患者肌肉、韧带会逐渐变成骨头，全球仅约900人，此前治疗选择极少；III期研究显示，用药24周新骨形成体积较安慰剂显著减少。二是艾伯维的tavapadon（商品名Juvmo），每日一次口服用于成人帕金森病，通过模拟多巴胺改善运动症状，III期项目显示症状改善。（来源：公司公告/美国FDA批准信息，Mirum与艾伯维9月25—26日发布；行业媒体9月26日报道） → 对普通人意味着什么：一款是"能把软组织变成骨头"的罕见病，一款是影响全球超千万人的帕金森病，这次都拿到了"每天一片"的口服方案。对帕金森病患者家庭来说，口服一天一次比多药错峰好管理；这类新药进入国内通常还需审批流程，请通过正规渠道关注，不要代购海淘。 2. 默沙东/第一三共主动撤回一款肺癌ADC的加速审批申请：FDA要求更硬的证据同日披露的另一则消息：默沙东与第一三共联合宣布，撤回其小细胞肺癌抗体偶联药物（ADC）ifinatamab deruxtecan在美国的加速上市申请——FDA经沟通认为其中期临床数据尚不足以支持"提前放行"，双方将继续推进已接近入组完成的III期研究，再用最终数据申请完全批准。（来源：行业媒体汇总，9月26日） → 对普通人意味着什么：这条新闻读起来是"挫折"，其实是监管在把关。加速审批通道允许"先用后补作业"，但数据不硬就该退回去补——这意味着最终走到患者手里的药，证据更扎实。新药消息别只看"获批多快"，也要看"证据多硬"。健康科普：都是糖尿病，1型和2型有什么不一样？昨天医讯里提到儿童1型糖尿病的"疾病修饰治疗"，不少读者可能纳闷：孩子也会得糖尿病？和家里老人那个"糖尿病"是一回事吗？今天一次说清。发动机不同，但结局相似。打个比方：血糖像锅炉里的蒸汽，胰岛素是"开阀门"的工人。1型糖尿病是"工人失踪"——自身免疫系统错误地攻击了胰岛β细胞，身体几乎生产不出胰岛素，阀门根本没人开，蒸汽（血糖）越积越高。它多发于儿童和青少年，起病急，一旦发病基本离不开胰岛素。2型糖尿病则是"工人还在，但门锈住了"——身体对胰岛素不敏感（胰岛素抵抗），工人拼命加班也拧不动阀门；它多发于中老年人，与超重、缺少运动、家族史相关，起病缓慢，很多人体检才发现。管理重点不一样。 1型是"缺什么补什么"：胰岛素是唯一的钥匙，每天多次注射或使用胰岛素泵，配合血糖监测、碳水计数，生活质量照样可以很好。2型是"先把门上的锈除掉"：第一阶段是减重、运动、饮食控制，多数人还需口服药，部分患者后来也需要胰岛素。两者共同的底线是：长期把血糖、血压、血脂管理好，防并发症。几个常见误区要澄清。其一，"孩子得糖尿病是因为糖吃多了"——不对，1型是自身免疫出了错，与吃糖无直接关系，患儿家庭不需要背负这种自责。其二，"2型是轻的，不用管"——错，2型患者基数大，长期不管同样会失明、肾衰、截肢。其三，"瘦子不会得2型"——体重正常者若有家族史、腹型肥胖、熬夜等风险因素同样可能中招。记住这几点： 1. "三多一少"别大意。多饮、多尿、多食、体重下降，无论孩子还是成人，出现就查血糖；儿童突发这些症状更要当天就医，警惕酮症酸中毒。 2. 2型糖尿病的"处方"一半在生活里。每周150分钟中等强度运动、腰围管住、少含糖饮料——对已确诊者是治疗，对没患病的人就是预防。科普不替代就诊，具体诊疗方案请咨询医生。健脑小游戏 · 第017期：健康小算盘今天换新玩法——健康小算盘！把健康知识变成几道小算术：会算，才能真吃透。请动笔算一算（提示已备好，答案明日揭晓）： 1. 小李体重65公斤、身高1.7米，请算出他的BMI（体重公斤数÷身高米的平方），并对照标准（18.5—23.9为正常）判断是否达标。　　难度：★☆☆ 2. 一瓶500毫升的可乐，含糖量约10.6克/100毫升，这一瓶总共含多少克糖？成人每日添加糖建议不超过25克，超了多少克？　　难度：★★☆ 3. 一种药说明书要求"每8小时服一次"，小张早上7:00服了第一次，一天三次，他第三次服药应该是几点？　　难度：★★☆ 4. 小王每天快走6000步，步幅约0.7米，他一天大约走了多少公里？　　难度：★★☆ 5. 一瓶500毫升、酒精度4%的啤酒，含纯酒精多少毫升、约多少克（纯酒精密度按0.8克/毫升计算）？与中国膳食指南"成人每日酒精摄入不超过15克"相比是超了还是没超？　　难度：★★★ 答案公布：明日（9月29日）《ABC康谈早知道》揭晓健脑小知识：把数字算清楚，是把健康知识从"听说过"变成"用得上"的过程——计算同时激活数字处理与逻辑推理脑区，属于对执行功能很好的日常锻炼。 · · · · · · · · · · 每日9:30更新

2026-09-28

·BioSpace

Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 AZURE-1 Study of Brelovitug in Chronic Hepatitis Delta Virus

– Phase 3 primary endpoint (virologic response and ALT normalization) achieved in both dose arms at Week 24 – 48-week data from Phase 2b portion of AZURE-1 demonstrate deepening viral suppression and increased rates of ALT normalization with longer-term treatment – Favorable safety and tolerability profiles observed; no new safety signals – Mirum to host conference call to discuss topline results today, September 28, at 8:30 a.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced the primary endpoint was met in the Phase 3 portion of the AZURE-1 study evaluating brelovitug, an investigational fully human monoclonal antibody that binds to hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV). In addition, 48-week data from the Phase 2b portion of AZURE-1 demonstrated deepening viral suppression, with a greater proportion of patients achieving HDV RNA below the lower limit of quantification (LLOQ, <10 IU/mL) and target not detected (TND), and increased rates of alanine aminotransferase (ALT) normalization. AZURE-1 is one of two pivotal Phase 3 studies intended to form the basis of Mirum’s U.S. registration package for brelovitug. “These results demonstrate the potential of brelovitug as a well-tolerated, convenient, single agent to deliver both deep viral suppression and ALT normalization, with responses that continue to deepen through 48 weeks of treatment. Notably, these results were achieved in a broad patient population, including those with significant liver inflammation, cirrhosis and clinically significant portal hypertension,” said Nancy Shulman, M.D., Executive Vice President of Clinical Development at Mirum. “We look forward to reporting topline results from AZURE-4 later this year and advancing toward our planned BLA submission in the first half of 2027.” “The goal of treating chronic HDV is to prevent progression to cirrhosis, liver cancer and liver failure, and that requires controlling both the virus and liver inflammation,” said Norah Terrault, M.D., MPH, Professor of Medicine and Chief of the Division of GI and Liver at the Keck School of Medicine of USC, and an AZURE-1 investigator. “ALT is a marker of liver injury, so seeing virologic response alongside ALT normalization is encouraging in a long-term therapy. For patients facing the most aggressive form of viral hepatitis, these are important results.” “Chronic hepatitis delta is a lifelong disease, and people living with it need treatment options that are effective, safe and tolerable enough to stay on over time. That’s especially true for patients with more advanced disease, who often have the fewest choices,” said Chari A. Cohen, DrPH, MPH, President of the Hepatitis B Foundation. “Results like these bring hope to a community that has had few options until recently.” Phase 3 AZURE-1 Results: Brelovitug Met the Primary Endpoint at Week 24 The Phase 3 portion of the AZURE-1 study enrolled 153 treatment-naive patients randomized in a 2:2:1 ratio to receive brelovitug 300 mg self-administered once weekly (QW) by subcutaneous (SC) injection, brelovitug 900 mg administered once every four weeks (Q4W) by SC injection, or delayed treatment starting at Week 24. This global study enrolled a broad patient population including patients with advanced disease. At Week 24, treatment with brelovitug met the primary endpoint, with 56% of patients in the 300 mg QW arm and 45% in the 900 mg Q4W arm achieving the combined endpoint of virologic response (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA [

临床结果临床2期突破性疗法临床3期孤儿药

100 项与 Zilurgisertib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12547 D12548	-	-	-

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
骨化性肌炎	美国	Incyte Corp.	2026-09-25

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
异位骨化	临床2期	美国	因塞特医药科技（上海）有限公司	2022-01-31
异位骨化	临床2期	美国	Incyte Corp.	2022-01-31
异位骨化	临床2期	澳大利亚	因塞特医药科技（上海）有限公司	2022-01-31
异位骨化	临床2期	澳大利亚	Incyte Corp.	2022-01-31
异位骨化	临床2期	巴西	因塞特医药科技（上海）有限公司	2022-01-31
异位骨化	临床2期	巴西	Incyte Corp.	2022-01-31
异位骨化	临床2期	加拿大	因塞特医药科技（上海）有限公司	2022-01-31
异位骨化	临床2期	加拿大	Incyte Corp.	2022-01-31
异位骨化	临床2期	智利	因塞特医药科技（上海）有限公司	2022-01-31
异位骨化	临床2期	智利	Incyte Corp.	2022-01-31

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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


NCT05090891 (PROGRESS, ENDO 12026 \| ENDO 22026)	临床2期	骨化性肌炎	63	Zilurgisertib 100 mg QD	餘襯艱憲餘齋製願觸鑰(蓋鏇糧糧積壓鏇觸鬱選) = 鹽糧廠選衊願獵鹽遞衊鑰糧遞膚選襯餘憲顧艱 (衊築簾襯簾壓淵淵窪製 ) 更多	积极	2026-06-14
				Placebo	餘襯艱憲餘齋製願觸鑰(蓋鏇糧糧積壓鏇觸鬱選) = 鬱膚糧構鬱襯鹹膚淵簾鑰糧遞膚選襯餘憲顧艱 (衊築簾襯簾壓淵淵窪製 ) 更多
NCT04455841 (INCB00928-104, ASH2025)	临床1/2期	贫血 \| 骨髓纤维化	84	Zilurgisertib alone (TGA)	廠糧築衊齋鏇簾簾觸積(積膚夢壓廠襯構膚艱顧) = 鏇壓齋鹹範醖鏇夢壓衊選簾廠鏇衊鑰艱顧艱鏇 (選簾鏇襯醖艱壓鹹觸選 ) 更多	不佳	2025-12-06
				Zilurgisertib + ruxolitinib (TGB)	繭壓網繭製艱築艱壓夢(窪艱觸網鹹夢糧醖艱顧) = 衊範簾齋衊鑰蓋夢壓廠構壓憲顧膚艱醖製範壓 (夢構鬱網選構衊獵獵選 ) 更多
NCT04582539 (LIMBER, CTgov)	临床1/2期	贫血 \| 多发性骨髓瘤 \| 骨髓增生异常综合征	22	Zilurgisertib (Zilurgisertib 50 mg QD)	鏇積範鬱廠獵餘廠選鹹 = 蓋窪蓋衊膚觸構積鏇蓋鑰積獵壓艱築齋壓遞簾 (窪簾製齋糧網鏇蓋顧艱, 顧鏇製蓋襯築窪醖襯願 ~ 鬱鹽齋憲壓製壓鏇觸鏇) 更多	-	2025-10-22
				Zilurgisertib (Zilurgisertib 100 mg QD)	鏇積範鬱廠獵餘廠選鹹 = 壓願構選鑰艱醖廠繭選鑰積獵壓艱築齋壓遞簾 (窪簾製齋糧網鏇蓋顧艱, 醖獵顧鑰鬱蓋壓淵鹽願 ~ 憲廠願壓膚築鏇淵獵鏇) 更多
NCT04455841 (INCB 00928-104, EHA2024) 人工标引	临床1/2期	贫血 \| 骨髓纤维化 hepcidin	45	Zilurgisertibb alone	-	积极	2024-05-14
				Zilurgisertibb wRuxolitinibinib	廠壓壓憲遞鹹憲積製選(築淵顧築鏇獵膚構遞選) = 窪觸糧壓築蓋淵觸膚選膚糧膚衊鏇艱鑰網獵壓 (繭糧膚艱鏇願艱製鹹範 ) 更多
NCT04455841 (INCB 00928-104, ASH2023) 人工标引	临床1/2期	贫血	36	zilurgisertib (TGA)	膚積鏇壓鏇願積願蓋憲(鬱醖製鬱齋構鑰網獵衊) = No DLTs occurred in either treatment group. 廠願繭顧淵選艱範糧襯 (餘鑰積餘襯鹽遞襯醖顧 ) 更多	积极	2023-12-10
				zilurgisertib+ruxolitinib (TGB)
NCT04455841 (ASCO 12023 \| ASCO 22023) 人工标引	临床1/2期	原发性骨髓纤维化	31	zilurgisertib (TGA)	窪憲鹽鏇鏇鏇醖壓積簾(淵製構構遞齋繭夢艱鑰) = No dose-limiting toxicities (DLTs) or study drug-related serious adverse events (AEs) occurred in either treatment group. 簾繭淵網膚艱鏇築觸願 (簾網鹹壓築壓繭膚襯廠 ) 更多	积极	2023-05-31
				ruxolitinib+zilurgisertib (TGB)
NCT04455841 (INCB 00928-104, ASH 12022 \| ASH 22022) 人工标引	临床1/2期	原发性骨髓纤维化	15	INCB000928	蓋醖餘壓窪築鹽獵繭簾(壓觸簾膚製鏇範廠窪餘) = 餘糧範築憲鹽窪顧窪獵齋蓋壓淵衊壓築壓醖顧 (艱艱遞蓋鑰構餘網鏇壓 ) 更多	积极	2022-11-15
				INCB000928+Ruxolitinib	蓋醖餘壓窪築鹽獵繭簾(壓觸簾膚製鏇範廠窪餘) = 遞遞壓鹹簾壓壓艱廠顧齋蓋壓淵衊壓築壓醖顧 (艱艱遞蓋鑰構餘網鏇壓 ) 更多