An Open-Label Study to Assess the Effects of VMX-C001 in Combination With an Oral FXa DOAC on the Efficacy of Unfractionated Heparin and of VMX-C001 Alone on the Efficacy of Low Molecular Weight Heparin in Healthy Subjects

A single centre, open-label study to assess the effects of VMX-C001 in combination with an oral FXa DOAC on the efficacy of Unfractionated Heparin (UFH) and of VMX-C001 alone on the efficacy of Low Molecular Weight Heparin (LMWH) in healthy subjects conducted in two parts:
UFH cohort: Subjects will be administered 2 single doses of 5000 IU UFH i.v. on Day 1 and Day 5, oral doses of the DOAC Rivaroxaban once daily from Day 2 until the morning of Day 5, and one single dose of 170 mg VMX-C001 i.v. on Day 5.
LMWH cohort: Subjects will be administered 2 single doses of 40 mg Enoxaparin s.c. on Day 1 and Day 4, and one single dose of 170 mg VMX-C001 i.v. on Day 4.

开始日期2024-08-14

申办/合作机构

VarmX BV

NCT06372483

/ Active, not recruiting临床1期

A Randomised, Double-Blind, Placebo-Controlled, Single Dose Trial Evaluating Different Doses of Intravenously Administered VMX-C001 and to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of VMX-C001 in Healthy Subjects (Part 1) and in Combination with a Selected FXa Direct Oral Anticoagulant (DOAC) in Healthy Older Subjects (Part 2)

A single centre, double-blind, randomized, placebo-controlled single dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of VMX-C001, conducted in two parts:
Part 1: Single dose of VMX-C001 or placebo in healthy volunteers.
Part 2: Single dose of VMX-C001 or placebo in combination with a selected factor 10a (FXa) direct oral anticoagulant (DOAC) in healthy older subjects.

开始日期2024-02-21

申办/合作机构

VarmX BV

100 项与 Recombinant coagulation factor X (VarmX) 相关的临床结果

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100 项与 Recombinant coagulation factor X (VarmX) 相关的转化医学

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100 项与 Recombinant coagulation factor X (VarmX) 相关的专利（医药）

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项与 Recombinant coagulation factor X (VarmX) 相关的文献（医药）

2023-05-01·Current cardiology reports

When and How to Use Reversal Agents for Direct Oral Anticoagulants?

Review

作者: Lecumberri, Ramón ; Suárez-Gea, Mª Luisa ; Gómez-Outes, Antonio

PURPOSE OF REVIEW:

Our objective is to describe currently available reversal agents for direct oral anticoagulants (DOACs), their target population, the available clinical practice recommendations and future directions.

RECENT FINDINGS:

Specific (idarucizumab for dabigatran and andexanet alfa for direct factor Xa inhibitors) and non-specific (prothrombin complex concentrates) reversal agents are effective in neutralizing the anticoagulant effect of DOACs. New investigational antidotes such as ciraparantag and VMX-C001 offer an alternative to andexanet alfa in reversing the anticoagulant activity of direct oral factor Xa inhibitors, but more clinical data are needed before they could be licensed for use. Specific reversal agents are recommended for use in clinical situations within their licensed indications (i.e.: reversal of DOACs in patients with severe uncontrolled or life-threatening bleeding or in need of emergency surgery or other invasive procedures), while non-specific reversal agents may be used when specific antidotes are not available or indicated.

2011-04-01·Journal of thrombosis and haemostasis : JTH2区 · 医学

Prothrombin complex concentrate and recombinant prothrombin alone or in combination with recombinant factor X and FVIIa in dilutional coagulopathy: a porcine model

2区 · 医学

Article

作者: Streif, W. ; Innerhofer, P. ; Danninger, T. ; Fries, D. ; Klima, G. ; Mitterlechner, T. ; Hansson, K. ; Lodl, M.

BACKGROUND:

This study was conducted to assess whether newly developed recombinant clotting factor concentrates enable the reversal of dilutional coagulopathy.

METHODS:

In 50 anesthetized pigs, ~60% of the blood volume was withdrawn and replaced with hydroxyethyl starch. Pigs were randomized to receive either 200 mg kg(-1) fibrinogen (n = 10), fibrinogen and 35 IU kg(-1) prothrombin complex concentrate (PCC) (n = 10), fibrinogen and 4 mg kg(-1) recombinant human factor II (rhFII) concentrate (n = 10), fibrinogen and a three-factor combination (3F) of 4 mg kg(-1) rhFII, 0.006 mg kg(-1) recombinant human FVIIa and 0.32 mg kg(-1) recombinant human FX (n = 10), or saline (n = 10). Thereafter, a standardized liver laceration was performed to induce uncontrolled hemorrhage. Survival time and blood loss were determined, and standard coagulation tests and thrombelastometry were performed.

RESULTS:

Fibrinogen combined with rhFII or PCC improved survival. Blood loss was significantly decreased in all groups as compared with the animals receiving saline. Clotting time was significantly shortened in the animals treated with fibrinogen and PCC, as well as in those treated with fibrinogen and 3F. One animal died after administration of fibrinogen and PCC.

CONCLUSION:

Following hemodilution, a combination of fibrinogen and PCC, rhFII or 3F enhances coagulation and final clot strength. Mortality was reduced statistically significantly only in the animals treated with fibrinogen and rhFII or PCC, whereas administration of the combination of fibrinogen and PCC caused a fatal thromboembolic complication. The combination of fibrinogen and rhFII might be effective in reversing dilutional coagulopathy and may reduce blood loss in cases of dilutional coagulopathy.

项与 Recombinant coagulation factor X (VarmX) 相关的新闻（医药）

2025-09-17

·PMLiVE

VarmX and CSL to collaborate in research deal worth up to $2.1bn

VarmX, a Dutch biotech company, has entered a strategic collaboration with CSL. Under the agreement, CSL will fully fund VarmX’s phase 3 trial of its lead candidate, VMX-C001, and retains an exclusive option to acquire all outstanding shares in the company, subject to trial outcomes. VMX-C001 is a single-dose, modified recombinant Factor X protein designed to bypass the effects of direct oral anticoagulants (FXa DOACs) and restore coagulation in patients requiring urgent surgery. The candidate recently received Fast Track Designation from the US Food and Drug Administration (FDA), in recognition of the significant unmet need in this patient population. CSL will fully fund the EquilibriX-S phase 3 study, which will evaluate the efficacy of VMX-C001 in patients taking FXa DOACs who require urgent surgeries. In addition to trial costs, CSL will fund post-trial activities, including manufacturing, commercial operations, medical affairs, and late-stage development. “The collaboration with CSL represents a transformative step for VarmX,” explained John Glasspool, CEO at VarmX. “By securing full funding for the registrational trial, product development, CMC and pre-launch activities, we are well positioned to bring VMX-C001 to patients. We are proud to partner with CSL, whose expertise and global reach will be invaluable as we move forward.” Following an upfront payment of $117m, CSL also has the exclusive option to acquire all issued and outstanding shares of the company, representing a full buy-out. This option is dependent on the results of the EquilibriX-C trial – if the option is exercised and certain milestones achieved, VarmX shareholders will be issued an additional $388m up to the launch of VMX-C001. Following its launch, sales-based milestones could offer up to $1.7bn for shareholders. In both cases, payments are reliant of regulatory clearance. Dr Paul McKenzie, CEO of CSL, commented: “We are excited to partner with VarmX to develop a novel treatment and address a significant unmet need aligning strongly with our strategic ambition to deliver enduring patient impact. It also aligns with our portfolio of medicines designed to minimise bleeding, preserve a patient’s own blood supply, improve surgical and medical outcomes and support global public health approaches to patient blood management.”

临床3期快速通道

2025-09-16

·Medaverse

22.05亿美元！CSL引进一款凝血新药

9月16日，荷兰利登，VarmX是一家生物技术公司，致力于开发创新方法，绕过针对活化因子Xa（FXa-DOACs）和遗传性凝血障碍的直接口服抗凝剂，该公司已与全球生物技术领导者CSL（ASX:CSL）达成战略合作，以支持其主要资产VMX-C001的开发。VMX-C001是一种新型治疗方法，可帮助需要紧急手术或在使用FXa DOAC时出现严重出血的患者恢复凝血。CSL还与VarmX股东签订了独家期权协议，以收购该公司所有已发行和流通的股份。VarmX是莱顿大学医学中心（LUMC）分拆的公司，该中心由世界领先的止血和血栓专家Pieter Reitsma教授于2016年创立。VMX-C001 是一种改良的重组人凝血因子 Xa，即使在存在因子 Xa 凝血抑制剂的情况下也能实现正常的血液凝固。 Pieter Reitsma教授根据战略合作协议的条款，CSL将全额资助VarmX的全球3期EquilibriX-s试验，该试验评估VMX-C001在需要紧急手术的服用FXa DOAC的患者中的效果。CSL还将全力资助和支持VarmX的后期产品开发、制造以及上市前的商业和医疗事务活动。 CSL将在交易完成后向VarmX股东支付1.17亿美元的预付款，以获得收购该公司的独家选择权。CSL将有权在3期临床数据后行使期权。根据某些里程碑的实现情况，在行使期权和惯例监管许可后，VarmX股东将获得3.88亿美元的收购和额外付款，直至VMX-C001商业启动，此后将获得高达17亿美元的销售成功里程碑。 VarmX首席执行官John Glasspool表示：“与CSL的合作标志着VarmX迈出了变革性的一步。通过为注册试验、产品开发、CMC和上市前活动获得全额资金，我们完全有能力将VMX-C001带给患者。我们很自豪能与CSL合作，CSL的专业知识和全球影响力将在我们前进的过程中发挥不可估量的作用。” CSL首席执行官Paul McKenzie博士表示：“我们很高兴与VarmX合作开发一种新的治疗方法，并解决一个尚未满足的重大需求，这与我们提供持久患者影响的战略目标高度一致。它还与我们的药物组合相一致，旨在最大限度地减少出血，保护患者自身的血液供应，改善手术和医疗结果，并支持全球公共卫生方法进行患者血液管理。” CSL执行副总裁兼研发主管Bill Mezzanotte表示：“CSL对VMX-C001的独特作用机制特别感兴趣，与其他治疗的更一般作用相比，它如何特异性靶向Xa因子抑制剂，以及临床前和早期临床数据导致美国FDA批准直接进行单一3期研究。” VMX-C001是一种研究性重组修饰因子X蛋白，以快速单剂量给药，有效绕过FXa抗凝活性，并在紧急手术和严重出血情况下迅速恢复服用因子Xa抑制剂的患者的凝血。它有可能与所有FXa DOAC一起使用，并与肝素等其他常见抗凝剂一起使用，并且可能没有额外的血栓形成风险。到2030年，美国、欧洲和日本估计将有3000万患者服用Xa因子（FXa）直接口服抗凝剂（DOAC），用于预防房颤中风和治疗深静脉血栓形成等慢性适应症。虽然这些疗法非常有效，但每年有2-4%的患者面临严重的、危及生命的出血或需要紧急手术，每周有超过30000名患者受到影响。尽管临床需求未得到满足，但目前欧盟或美国还没有完全批准的治疗剂用于治疗服用Xa因子抑制剂的患者的急性大出血。正如2025年9月3日宣布的那样，VMX-C001最近获得了美国FDA的快速通道认证，承认其有可能解决需要紧急手术的FXa DOAC患者在恢复凝血方面尚未满足的关键医疗需求。预计2029年将进行商业化。 VarmX首席执行官John Glasspool表示：“我们感谢我们的投资者的持续支持，包括Sound Bioventures、EIC、EQT Life Sciences、INKEF、Lundbeckfonden BioCapital、Ysios Capital、BioGeneration Ventures、InnovationQuarter、LEH和UNIIQ，他们与我们有着共同的愿景，即改变患者的紧急护理，并在帮助公司发展和实现这一里程碑方面发挥了重要作用。” 关注下方公众号，带你看世界!

临床3期并购临床结果临床成功申请上市

2025-09-16

·EndPoints

CSL commits $117M to help bring VarmX’s blood clotting drug to market

CSL and VarmX have inked a $117 million deal focused on advancing the Dutch biotech’s blood coagulation candidate’s Phase 3 development. And if that trial yields positive results, the Australian drugmaker could end up buying the company altogether. CSL will fund an ongoing Phase 3 trial of VarmX’s recombinant Factor X protein called VMX-C001. It will support “late-stage product development, manufacturing and pre-launch commercial and medical affairs activities” for the program, according to a Tuesday release . If CSL exercises its exclusive option to acquire VarmX, the biotech could get up to $388 million ahead of the launch of the drug, which is anticipated in 2029. VarmX would also be eligible for up to $1.7 billion in sales milestones. VMX-C001 is a single-dose treatment designed to restore blood clotting in patients taking Factor Xa inhibitors, as certain patients can experience severe bleeding or need urgent surgery, according to VarmX. The biotech estimates around 30 million people in the US, Europe and Japan will be taking an oral Factor Xa inhibitor for conditions like stroke prevention or deep vein thrombosis by 2030. About 2% to 4% of them experience severe bleeding or need emergency surgery every year, the company added. There are currently no FDA-approved treatments for these patients. In 2023, VarmX secured €30 million in a Series B2 round to support the registrational trial of VMX-C001. The company was also awarded a €15 million follow-on equity investment from the European Innovation Council accelerator earlier this year. For CSL, the deal comes around a month after it announced a major overhaul of its business, including a spinout of its vaccines business and sweeping staff cuts.

临床3期并购

100 项与 Recombinant coagulation factor X (VarmX) 相关的药物交易

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