作者: Lopez-Jaime, Francisco J ; Windyga, Jerzy ; Matytsina, Irina ; Mahlangu, Johnny ; Chowdary, Pratima ; Nielsen, Anne Louise ; Lentz, Steven R ; Gil, Lidia

BACKGROUND:

Mim8 (denecimig) is a factor VIII (FVIII) mimetic bispecific antibody in development for the treatment of hemophilia. Data from the phase 1 part of FRONTIER1 (EudraCT: 2019-000465-20, NCT04204408, and NN7769-4513) suggested that Mim8 was well tolerated in healthy participants and exhibited pharmacokinetic (PK) properties consistent with dose proportionality.

OBJECTIVES:

The partially randomized, phase 2, multiple ascending dose (MAD) part of FRONTIER1 aimed to evaluate the safety, PK, pharmacodynamics (PD), and exploratory efficacy of Mim8 in participants with hemophilia A with or without FVIII inhibitors.

METHODS:

The MAD part of FRONTIER1 consisted of 42 participants, assigned to 5 cohorts, with participants in cohorts 3 and 4 randomized 1:1 to dosing weekly or every 4 weeks, respectively. Four of the 42 participants (9.5%) had FVIII inhibitors prior to study enrolment. The primary endpoint was treatment-emergent adverse events (TEAEs). PK and PD were evaluated by Mim8 plasma concentration and thrombin generation, respectively. Exploratory efficacy was assessed via the number of treated bleeds. Safety and PD parameters were also evaluated from an exploratory cohort treated with emicizumab.

RESULTS:

Mim8 was well tolerated, with 1 serious TEAE (anxiety-related chest pain) deemed unrelated to Mim8. There was no dose dependency on the number, causality, type, or severity of TEAEs. PK/PD properties supported weekly to monthly dosing approaches, and few participants experienced treated bleeds beyond the lowest dose cohort (1 in cohorts 2 and 3, and 3 in cohort 5).

CONCLUSION:

These data support the continued clinical development of Mim8, and FRONTIER1 has proceeded onto an extension phase.

2023-11-01·Haemophilia : the official journal of the World Federation of Hemophilia

A next generation FVIII mimetic bispecific antibody, Mim8, the impact on non‐factor VIII related haemostasis assays

Article

作者: Hickey, Kieron ; Ezban, Mirella ; Kitchen, Steve ; Bowyer, Annette Elizabeth

Abstract:

Introduction and aims:

Mim8 is a next generation bispecific antibody developed for the treatment of haemophilia A (HA). Mim8 has an increased potency compared to first generation molecules. The impact on Mim8 on non‐FVIII measuring haemostasis assays was assessed in plasma containing Mim8.

Methods:

Congenital severe HA plasma was spiked with increasing concentrations of Mim8 (0–20 μg/mL). 28 routine and specialist haemostasis assays were used to measure activities. These included tests for prothrombin time (PT), fibrinogen, thrombin, D‐dimer, anti‐Xa, heparin induced thrombocytopenia (HIT), clotting factors II–XII, factor XIII, von Willebrand factor (VWF), thrombophilia and DRVVT.

Results and conclusions:

Less than 10 % difference was calculated between plasma without Mim8 and plasma spiked to 15 μg/mL Mim8 in all assays except thrombin time (−10.5%), APTT‐based factor IX, XI and XII, Werfen VWF:RCo (10.6%) and Siemens LA1 (−26.4%) and LA2 (−16.9%). At the expected therapeutic steady state levels of Mim8 (5–8 μg/mL), less than 10% difference was calculated for thrombin time and Werfen VWF:RCo.APTT‐based assays of FIX, XI and XII are significantly elevated in the presence of Mim8 and should not be performed. A chromogenic FIX assay could be used to accurately measure FIX activity in the presence of Mim8. There was some interference in the DRVVT method we used so local assessment of other DRVVT methods is advised. Differences in all other tests would not be predicted to affect patient management.

2023-11-01·The Lancet. Haematology

Emicizumab prophylaxis in patients with acquired haemophilia A (GTH-AHA-EMI): an open-label, single-arm, multicentre, phase 2 study

Article

作者: Sachs, Ulrich J ; Pilch, Jan ; May, Marcus ; Klamroth, Robert ; Pelzer, Fabius J ; Knöbl, Paul ; Dobbelstein, Christiane ; Pfrepper, Christian ; Tiede, Andreas ; Hart, Christina ; Möhnle, Patrick ; Werwitzke, Sonja ; Greil, Richard ; Miesbach, Wolfgang ; Oldenburg, Johannes ; Trautmann-Grill, Karolin ; Weigt, Carmen ; Schipp, Dorothea ; Schindler, Christoph ; Holstein, Katharina

BACKGROUND:

Acquired haemophilia A is caused by neutralising autoantibodies against coagulation factor VIII, leading to severe bleeding. Standard treatment involves immunosuppressive therapy, which is associated with adverse events and mortality in the frail population of patients with acquired haemophilia A. This study investigated whether emicizumab, a factor VIIIa mimetic antibody, protects patients with acquired haemophilia A from bleeding and allows deferral of immunosuppression during the first 12 weeks after diagnosis.

METHODS:

We report final results of an open-label, single-arm, phase 2 clinical trial. Adult patients with acquired haemophilia A from 16 haemophilia treatment centres in Germany and Austria were eligible if they had not previously received immunosuppression. Patients received emicizumab subcutaneously (6 and 3 mg/kg on days 1 and 2, 1·5 mg/kg weekly until week 12), but no immunosuppression. Follow-up was until week 24. The primary endpoint was the number of clinically relevant bleeds per patient-week until week 12. Emicizumab was considered effective if the mean bleeding rate was significantly below 0·15 bleeds per patient-week, the rate observed in a previous study of patients with acquired haemophilia A treated with bypassing agents and immunosuppression but no emicizumab. The study is registered with clinicaltrials.gov, NCT04188639 and is complete.

FINDINGS:

Of 49 patients screened from March 25, 2021, to June 10, 2022, 47 were enrolled (23 women, 24 men). Median age was 76 years (IQR 66-80), 46 (98%) of 47 patients were White, median factor VIII activity was 1·4 IU/dL (0·3-5·6), and median inhibitor concentration was 11·4 Bethesda units per mL (3·9-42·7). Mean breakthrough bleeding rate was 0·04 bleeds per patient-week (upper 97·5% CI 0·06). 33 (70%) of 47 patients had no bleeding events, seven patients (15%) had one bleed, six patients (13%) had two bleeds, and one patient (2%) had three bleeds. Adverse events of grade 3 or worse included COVID-19 (n=2), acute kidney injury (n=2), and stroke (n=1). Four of 47 patients died, including two deaths related to bleeding, one from COVID-19, and one from cardiac arrest (none were judged as related to emicizumab).

INTERPRETATION:

This study suggests that emicizumab prophylaxis prevents bleeding in patients with acquired haemophilia A and that immunosuppressive therapy can be deferred while patients are receiving this treatment. The low number of thromboembolic events, severe infections, and fatalities observed in this study are promising.

FUNDING:

This study was supported by funding from Hoffman-La Roche.

项与 Mim8 相关的新闻（医药）

2024-05-18

·CPHI制药在线

超60亿美元！恒瑞GLP-1产品组合有偿许可给美国Hercules公司；诺和诺德血友病双抗Ⅲ期研究成功 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点不少。首先是审评审批方面，多个药取得进展。国内来看，很值得关注的有两个药物动态，即百济神州泽布替尼新适应症获批上市，癌症患者中位PFS延长近1.7倍以及优卡迪生物新型CAR-T疗法拟纳入突破性治疗品种，ORR超90%。国外而言，最热的就是，度普利尤单抗在美申报新适应症，或成为首个针对12-17岁青少年CRSwNP患者的疗法；研发方面，多个药取得不错的进展，比如，诺和诺德血友病双抗Ⅲ期研究成功，年内申报上市；交易及投融资方面，恒瑞医药GLP-1产品组合有偿许可给美国Hercules公司，总交易额超过60亿美元。本周盘点包括审评审批、研发和交易及投融资三大板块，统计时间为5.13-5.17，包含25条信息。审评审批NMPA上市批准1、5月13日，NMPA官网显示，百济神州的泽布替尼胶囊新适应症获批，联合奥妥珠单抗适用于既往接受过至少二线系统性治疗的复发或难治性滤泡性淋巴瘤（R/R FL）患者。泽布替尼（商品名：百悦泽）是一款布鲁顿氏酪氨酸激酶抑制剂（BTKi）。截至2024年4月，该产品已在全球70个市场获批多项适应症。2、5月13日，NMPA官网显示，ViiV Healthcare的卡替拉韦钠片和卡替拉韦注射液同时获批，用于有感染风险的成人和青少年（体重≥35kg）进行HIV-1暴露前预防（PrEP），以降低性传播感染HIV-1的风险，结合安全的性行为措施以达到更好的HIV-1 PrEP效果。这是一款无需每日服药就能预防HIV感染的疗法。在一项Ⅱb/Ⅲ期临床研究中，与对照组相比，卡替拉韦长效注射液组的HIV感染风险降低91%。申请3、5月14日，CDE官网显示，阿斯利康的本瑞利珠单抗（benralizumab）注射液新适应症申报上市，推测适应症为6-11岁嗜酸性粒细胞性哮喘患者的附加维持治疗。Benralizumab最初由协和麒麟开发，属于一种IL-5Rα单抗，2023年6月，benralizumab首次在国内申报上市，目前正在审评中。4、5月15日，CDE官网显示，再鼎医药的艾加莫德α注射液（皮下注射）补充生物制剂上市许可申请（sBLA）申报上市，并于2024年5月11日纳入优先审评，用于治疗慢性炎性脱髓鞘性多发性神经根神经病（CIDP）。临床批准 5、5月15日，CDE官网显示，中美瑞康（Ractigen Therapeutics）的一款小核酸药物RAG-17获批临床，拟用于治疗成人因超氧化物歧化酶1（SOD1）突变引起的肌萎缩侧索硬化症（ALS）。RAG-17是一款以SOD1为靶基因的双链小干扰RNA（siRNA）。6、5月15日，CDE官网显示，英诺湖医药的注射用ILB-3101获批临床，用于治疗晚期实体瘤。ILB-3101是全球率先进入临床阶段的一款以eribulin为有效载荷且靶向B7-H3的ADC，在临床前研究中展示出显著的抗肿瘤活性，并在三阴性乳腺癌（TNBC）、肉瘤、卵巢癌、急性髓系白血病（AML）等多个肿瘤动物模型中显示出显著优于对照药物的肿瘤杀伤作用。7、5月17日，CDE官网显示，LIB Therapeutics的1类新药lerodalcibep获批临床，拟用于成人患有动脉粥样硬化性心血管疾病（ASCVD）、ASCVD极高危或高危，包括杂合子型家族性高胆固醇血症患者的治疗，以进一步降低低密度脂蛋白胆固醇水平。lerodalcibep是LIB Therapeutics开发的一款第三代长效PCSK9抑制剂，每月仅需注射一针。申请8、5月15日，CDE官网显示，博安生物的1类新药注射用BA1302申报临床。该药物是一款靶向黑色素转铁蛋白（CD228）的抗体偶联药物（ADC），也是国产首款CD228 ADC。截至目前，仅有两款靶向CD228药物进入临床阶段，分别为SGN-BB228和SGN-CD228A。突破性疗法9、5月16日，CDE官网显示，艾伯维的JAK1抑制剂乌帕替尼片（Upadacitinib片）拟纳入突破性疗法，用于12岁及以上的青少年和成人非节段型白癜风患者。2023年10月，乌帕替尼治疗非节段性白癜风的Ⅱb期研究达到了主要终点。目前，该药正在多种免疫介导性疾病中对其进行研究。10、5月16日，CDE官网显示，优卡迪生物医药公司的具有沉默白介素6（IL-6）表达功能的靶向CD19基因工程化自体T细胞注射液（ssCART-19）拟纳入突破性疗法，用于治疗难治/复发急性淋巴细胞白血病。这是优卡迪生物研发的一款具有IL-6敲减功能的CD19靶向CAR-T细胞，ssCART-19是其开发的第四代CAR-T产品。优先审评11、5月13日，CDE官网显示，加科思自主研发的一款强效、不可逆的KRAS G12C抑制剂枸橼酸格来雷塞片（代号：JAB-21822片）拟纳入优先审评，用于治疗既往接受过至少一线系统性治疗的KRAS G12C突变的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者。FDA上市申请12、5月14日，FDA官网显示，卫材（Eisai）和渤健（Biogen）的皮下注射仑卡奈单抗（lecanemab）申报上市，用于治疗早期阿尔茨海默患者（轻度认知障碍或轻度痴呆阶段患者），可在家中给药。该产品此前已经获FDA授予快速通道资格，适用于已完成双周静脉注射起始阶段的患者后续接受每周一次皮下注射。仑卡奈单抗是一款人源化抗可溶性β淀粉样蛋白（Aβ）单克隆抗体。优先审评13、5月13日，FDA官网显示，再生元和赛诺菲合作开发的IL-4Rα全人源单抗度普利尤单抗（商品名：Dupixent）补充生物制品许可申请（sBLA）获优先审评，用于治疗慢性鼻-鼻窦炎伴鼻息肉（CRSwNP）青少年患者，PDUFA日期为2024年9月15日。如果获得批准，度普利尤单抗将成为美国第一种针对12-17岁青少年CRSwNP患者的疗法。快速通道资格14、5月14日，FDA官网显示，迈威生物的9MW2821被授予快速通道资格，用于治疗既往接受过含铂化疗方案治疗失败的复发或转移性宫颈癌。该产品此前已获FDA快速通道资格和孤儿药资格，分别用于治疗晚期、复发或转移性食管鳞癌和食管癌。9MW2821是迈威生物开发的一款靶向Nectin-4的抗体定点偶联药物，由创新抗体分子、新型连接子以及新型载荷（TOP1i）共同组合而成。研发临床状态15、5月13日，药物临床试验登记与信息公示平台显示，赛诺菲的抗CD40L单抗SAR441344（frexalimab）在中国启动了Ⅲ期临床试验，旨在评估SAR441344在非复发性继发进展型多发性硬化成人患者中的安全性和有效性。研究的主要终点为6个月内患者发生经复合指标确认的残疾进展（cCDP）的时间。16、5月14日，药物临床试验登记与信息公示平台显示，和黄医药启动了一项Ⅱ/Ⅲ期临床试验，以评估和黄医药的索凡替尼、恒瑞医药的PD-1抗体卡瑞利珠单抗、白蛋白结合型紫杉醇和吉西他滨的联合疗法用于一线治疗转移性胰腺导管腺癌（PDAC）患者。首名受试者已于2024年5月8日接受首次给药治疗。17、5月15日，药物临床试验登记与信息公示平台显示，武田（Takeda）启动了TAK-279片的两项国际多中心（含中国）Ⅲ期临床研究，针对适应症为中重度斑块状银屑病。TAK-279是一款高选择性、口服别构酪氨酸激酶2（TYK2）抑制剂。2022年12月，武田以40亿美元的预付款和20亿美元的商业里程碑付款收购了Nimbus Lakshmi公司及其开发的这款TYK2抑制剂。18、5月16日，药物临床试验登记与信息公示平台显示，加科思启动了格来雷塞联合JAB-3312对比替雷利珠单抗联合培美曲塞和卡铂一线治疗携带KRAS p.G12C突变的晚期非鳞状非小细胞肺癌（NSCLC）患者的Ⅲ期研究（n=392）。研究的主要终点是无进展生存期（PFS）。该研究也是加科思就格来雷塞启动的第一项Ⅲ期临床试验。19、5月16日，药物临床试验登记与信息公示平台显示，翰森制药的c-Met/EGFR双抗HS-20117启动Ib/Ⅲ期研究，成为开发进度最快的国产c-Met/EGFR双抗。HS-20117是豪森药业以14.68亿元从普米斯生物引进的一款c-Met/EGFR双抗，于2023年3月进入IND阶段，目前，全球共12款临床在研c-Met/EGFR靶向药物，其中7款为c-Met/EGFR双抗。临床数据20、5月13日，诺和诺德宣布，Mim8（NNC0365-3769/denecimig）治疗血友病A的Ⅲa期FRONTIER 2研究达到了主要终点。基于该积极结果，诺和诺德将在年内提交Mim8的上市申请。Mim8是诺和诺德利用Genmab的DuoBody技术平台开发的一款模拟凝血因子VⅢa（FVⅢa）作用的双抗药物。21、5月16日，罗氏宣布，双重GLP-1R/GIPR激动剂CT-388用于治疗肥胖和2型糖尿病的的I期临床试验取得了积极结果。研究发现，与安慰剂相比，健康肥胖成人受试者每周皮下注射一次CT-388，持续24周，可显著减轻体重。CT-388的减重效果具有临床意义，经安慰剂调整后的平均减重率为18.8% (p值<0.001)。在第24周，100%接受CT-388治疗的受试者体重减轻>5%，85%体重减轻>10%，70%体重减轻>15%，45%体重减轻>20%。交易及投融资22、5月14日，武田宣布，与AC Immune签署相关协议，获得后者一款靶向有毒形式淀粉样蛋白β（Aβ）的免疫疗法，包括用于治疗阿尔茨海默病（AD）的ACI-24.060，总额超过21亿美元。ACI-24.060是基于AC Immune公司SupraAntigen平台开发的抗Aβ疫苗，目前正在Ⅰb/Ⅱ期试验ABATE中受检视。23、5月15日，贝美药业宣布，其与Sol-Gel签署资产购买协议，贝美药业从Sol-Gel公司购得痤疮药物Twyneo在中国（含中国香港、中国澳门、中国台湾地区）和以色列地区的相关资产。该药已获得美国FDA批准上市，用于治疗九岁及以上儿童和成人患者的寻常痤疮。根据协议，贝美药业获得Twyneo在授权区域内的相关资产，包括但不限研发、商业化等方面的权益。24、5月16日，恒瑞医药宣布，GLP-1产品组合有偿许可给美国Hercules公司，美国Hercules公司将获得在除大中华区以外的全球范围内开发、生产和商业化GLP-1产品组合的独家权利。作为对外许可交易对价一部分，恒瑞医药将取得美国Hercules公司19.9%的股权，且将从美国Hercules公司获得GLP-1产品组合授权许可费。此外，根据公告，这项合作的首付款和各项里程碑付款最高超过60亿美元。25、5月17日，嘉越医药宣布，与美国Erasca共同宣布签署一项全球独家授权协议。根据协议条款，ERASCA公司将获得Pan-RAS（ON）抑制剂JYP0015中国内地和香港、澳门地区之外的全球独家研究、开发和商业化的授权许可。嘉越医药将有资格获得首付及潜在近期付款高达2000万美元，总计最高达3.45亿美元的潜在开发、商业化里程碑及全球行使权付款等，以及净销售额的分级特许权使用费。【智药研习社近期线下课程预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

上市批准临床3期优先审批细胞疗法免疫疗法

2024-05-14

·FierceBiotech

Novo Nordisk's Hemlibra rival hits goals in phase 3, teeing up blockbuster showdown with Roche

Efficacy is one area where Novo Nordisk predicted it may have an edge. Novo Nordisk is coming for Roche’s Hemlibra. A phase 3 trial of the Danish drugmaker’s challenger to the blockbuster hemophilia drug hit its primary endpoint, clearing the path for a filing for approval around the end of the year. Roche’s hemophilia A treatment Hemlibra brought in (PDF) 4.1 billion Swiss francs ($4.5 billion) last year. Novo Nordisk is taking the same approach to preventing bleeds with Mim8, a bispecific antibody designed to bridge Factor IXa and X to replace missing Factor VIII. Roche’s Hemlibra showed (PDF) the approach can enable most people to be free from treated bleeds on its path to market. Now, Novo Nordisk has shared phase 3 data on Mim8. The trial met its co-primary endpoints by linking once-weekly and once-monthly Mim8 to statistically significant reductions in treated bleeding episodes. Novo Nordisk found the treatment safe and well tolerated, with no deaths or thromboembolic events reported in the trial. The combination of a primary endpoint hit and clean safety profile could be enough for Mim8 to get a favorable response when Novo Nordisk submits it to regulators late this year. The pharma will face a well-entrenched rival in Roche if it brings Mim8 to market, but analysts are upbeat. “Positive headline phase 3 for Novo's Mim8 in hemophilia A may near our ‘best’ case scenario, with convenient once-monthly dosing seeming similarly effective to once-weekly, and efficacy data that may challenge Roche's incumbent Hemlibra,” Jefferies analysts wrote in a note to investors. Efficacy is one area where Novo Nordisk predicted it may have an edge. Martin Lange, M.D., Ph.D., Novo Nordisk’s executive vice president of development, told attendees at the company’s Capital Markets Day in March that Mim8 has the potential to render more than 70% of patients free from treated bleeds. In patients with no prior prophylaxis treatment, 86% of people treated with once-weekly Mim8 and 95% of those treated with once-monthly Mim8 had no treated bleeds. Jefferies analysts compared the results to the 80% reported by Roche's HAVEN-6 Hemlibra trial at its interim readout and the 56% to 60% range in Roche’s HAVEN-3. Meanwhile, the zero treated bleed rates in people with prior coagulation factor prophylaxis were 66% in the once-weekly arm and 65% in the once-monthly cohort. The analysts compared the result to the 54% rate of zero treated bleeds seen in patients on prior coagulation factor prophylaxis in HAVEN-3. The similarity of the once-weekly and once-monthly results supports Novo Nordisk’s attempt to establish convenience as a differentiator versus Hemlibra. Patients receive Roche’s therapy once a week for four weeks, at which point they start taking maintenance doses either every week, every two weeks or every four weeks. Lange said Mim8 was designed to have a “true monthly profile.” Roche Pharmaceuticals CEO Teresa Graham sounded confident when asked about Mim8 on an earnings call in April, saying Hemlibra will remain the treatment of choice for patients switching to a prophylactic treatment. The confidence is built on real-world data showing 80% of patients have zero bleeds and a drug profile that means 60% of people take the medicine every two weeks or every four weeks.

临床结果临床3期

2024-05-14

·Firstwordpharma

Performance of Novo’s monthly haemophilia A therapy sets up challenge to Roche’s Hemlibra

A pivotal study showed that both weekly and monthly regimens of Novo Nordisk’s factor VIIIa mimetic bispecific antibody Mim8 significantly decreased bleeding episodes compared with on-demand and previous prophylactic treatment in patients with haemophilia A. The results suggest that Mim8 could be a strong challenger to Roche’s Hemlibra (emicizumab), which is given once weekly.“Given the differing needs of people living with haemophilia A, a convenient once-weekly or once-monthly dosing provides optionality and flexibility,” remarked Martin Holst Lange, EVP for development at Novo Nordisk.The Phase IIIa FRONTIER 2 trial — comprising a 26-week main segment and an equally long extension portion — compared once-weekly and once-monthly Mim8 against no prophylaxis, and against prior coagulation factor prophylaxis in 254 patients with haemophilia A aged 12 years and above, with or without inhibitors. The study’s co-primary endpoints were the number of treated bleeds for Mim8 versus no prophylaxis and prior prophylaxis during the main phase.Main goals metThe headline results announced Monday showed that once-weekly and once-monthly Mim8 demonstrated significant 97% and 99% reductions in treated bleeds, respectively, compared with no prophylaxis. Additionally, 86% and 95% of patients receiving once-weekly and once-monthly Mim8 regimens, respectively, had zero treated bleeds versus none of the patients who did not receive prophylaxis. Jefferies analysts suggested that preventing at least 80% of patients from needing to be treated for bleeding is a key determiner of whether Mim8 poses a threat to Hemlibra. Thus, the FRONTIER 2 results “may challenge Roche’s incumbent,” the analysts said.In an intra-patient analysis of patients who had received prophylaxis earlier, once-weekly and once-monthly Mim8 showed superior reductions of 48% and 43% in treated bleeds, respectively, compared with prior prophylactic treatment. Furthermore, 66% of patients on weekly and 65% on monthly Mim8 had no treated bleeds.Marketing applications this yearMeanwhile, the drug’s safety and tolerability were consistent with previous studies, with no reports of deaths or thromboembolic events. The company noted that detailed data will be presented at upcoming medical congresses.Novo Nordisk’s FRONTIER programme is investigating Mim8 across four late-stage studies in haemophilia A. The company plans to file regulatory submissions towards the end of this year, which alongside FRONTIER 2, will also include data from the Phase III FRONTIER 3 study.In addition to Hemlibra and several recombinant coagulation factors, currently approved haemophilia A therapies include Sanofi and Sobi’s Altuviiio (efanesoctocog alfa) and the first gene therapy Roctavian (valoctocogene roxaparvovec) from BioMarin.

临床3期临床结果基因疗法临床2期

100 项与 Mim8 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
血友病A	临床3期	美国	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	中国	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	日本	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	奥地利	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	比利时	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	加拿大	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	捷克	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	丹麦	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	法国	Novo Nordisk A/S	2021-12-02
血友病A	临床3期	德国	Novo Nordisk A/S	2021-12-02

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适应症

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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


FRONTIER 2 (GlobeNewswire) 人工标引	临床3期	血友病A	254	Mim8 once-weekly	餘願簾膚蓋鹹鹽鑰積鬱(蓋鹹鬱繭選廠積觸廠繭) = 遞積窪窪範鬱膚鬱齋鏇築積憲憲淵簾醖網築鬱 (憲夢窪餘廠積觸遞壓鑰 ) 达到更多	积极	2024-05-13
				Mim8 once-monthly	餘願簾膚蓋鹹鹽鑰積鬱(蓋鹹鬱繭選廠積觸廠繭) = 鑰獵鹹網製憲餘夢憲夢築積憲憲淵簾醖網築鬱 (憲夢窪餘廠積觸遞壓鑰 ) 达到更多
ASH2023	N/A	血友病A	-	AAV-Mediated Expression of a Single-Chain FVIII Mimetic Antibody (AAV-Bi8 vector)	網餘顧網壓憲願鏇製齋(鏇壓艱夢壓願夢繭選壓) = 觸夢範鹽夢艱蓋齋範鏇糧壓顧夢鹽範構顧遞獵 (獵醖衊遞繭鏇憲構憲鑰 ) 更多	-	2023-12-10
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER 1, ASH2022) 人工标引	临床1/2期	血友病A	42	Mim 8	選製構網製醖蓋糧積艱(憲鏇製簾積鑰憲網願艱) = 窪願廠膚齋獵網憲壓淵膚鏇構選襯鏇遞蓋醖襯 (製糧醖積夢築廠艱衊襯 ) 更多	积极	2022-11-15
NCT04204408 (EudraCT:2019-000465-20, ISTH2022) 人工标引	临床1/2期	-	-	Mim 8 (SAD phase)	衊窪淵窪顧觸鹽願鬱鬱(鹽蓋醖淵選願糧糧顧築) = Mim8 was well tolerated following both single and multiple dosing, and no thromboembolic events or related serious adverse events were reported 構壓齋繭餘遞蓋襯鏇範 (齋積蓋窪選簾遞廠襯糧 )	积极	2022-07-11
				Mim 8 (MAD phase)