Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
Exploratory Objectives:
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months

Start Date01 Feb 2026

Sponsor / Collaborator-

NCT06703606

/ RecruitingPhase 1

A PHASE 1B OPEN-LABEL NON-RANDOMIZED STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MARSTACIMAB TREATMENT FOLLOWING THE DISCONTINUATION OF EMICIZUMAB THERAPY IN ADOLESCENT AND ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA A WITH OR WITHOUT INHIBITORS.

The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A. A rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut.
These patients can be with or without inhibitors who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines.
This study is seeking for participants:
* with severe Hemophilia A who are on emicizumab treatment for at least 6 months.
* must be 12 to less than 75 years old
* must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study.

Start Date13 May 2025

Sponsor / Collaborator

Pfizer Inc.

NCT05611801

/ RecruitingPhase 3

AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY =2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
This study will enroll participants who:
* have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
* have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
* if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
* if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.

Start Date09 Dec 2022

Sponsor / Collaborator

Pfizer Inc.

100 Clinical Results associated with Marstacimab-hncq

100 Translational Medicine associated with Marstacimab-hncq

100 Patents (Medical) associated with Marstacimab-hncq

Literatures (Medical) associated with Marstacimab-hncq

03 Aug 2025·Expert Review of Hematology

Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis

Review

Author: Mokresh, Sena ; Mokresh, Muhammed Edib ; Caliskan, Cagla Sumeyye ; Alomari, Omar ; Ilter, Merve Kabasakal

BACKGROUND:

Hemophilia A and B are life-threatening congenital bleeding disorders traditionally managed with frequent factor replacement therapies, often complicated by breakthrough bleeds and inhibitor development. Marstacimab, a monoclonal antibody targeting TFPI, has emerged as a novel prophylactic treatment to reduce bleeding episodes in patients without inhibitors.

METHODS:

A systematic review was conducted following PRISMA guidelines. A comprehensive literature search was performed across multiple databases. Meta-analysis was conducted using R, applying a random-effects model.

RESULTS:

Nine manuscripts were included. Marstacimab significantly reduced the annualized bleeding rate (mean difference: -16.30; 95% CI: [-18.46, -14.15], p < 0.001) and showed a favorable safety profile with most adverse events being mild or moderate, and no thrombotic events reported. The use of a prefilled pen device further highlighted the therapeutic benefits and ease-of-use of Marstacimab.

CONCLUSIONS:

This meta-analysis reinforces the efficacy and safety of Marstacimab in reducing bleeding rates in severe hemophilia A and B. The findings support its role as a promising, transformative alternative to conventional factor replacement therapies.

REGISTRATION:

The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number CRD42024620215.

03 Jul 2025·Expert Review of Hematology

Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations?

Review

Author: Mahlangu, Johnny

INTRODUCTION:

Non-factor therapies were developed to address the shortcomings of clotting factor concentrates (CFCs) used for hemophilia bleed management. These CFC unmet needs include high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Anti-tissue factor pathway inhibitors (anti-TFPIs) that have completed Phase 3 clinical studies are concizumab and marstacimab. The role of these anti-TFPIs in the hemophilia treatment armamentarium remains unclear.

AREAS COVERED:

This review critically appraises data published in PubMed, World of Science, and peer-reviewed congress presentations to determine whether anti-TFPIs merely supplement current treatment options or represent a disruptive shift in the treatment paradigm for hemophilia. It underscores the unmet needs of replacement therapies and compares the pharmacokinetic, efficacy, and safety data of anti-TFPIs and selected FVIII and FIX products.

EXPERT OPINION:

As hemophilia treatment goals continue to evolve, the role of currently developed anti-TFPIs is still not fully defined. This review comprehensively summarizes the clinical trial data, which shows that anti-TFPIs are not intended to replace the standard of care CFCs but to expand the therapeutic arsenal for patients with hemophilia treated with these therapeutic agents.

01 Apr 2025·The Journal of the Association of Physicians of India

Revolutionizing Treatment Strategies through Inhibition of Tissue Factor Pathway Inhibitor: A Promising Therapeutic Approach for Hemophilia Management.

Review

Author: Shaikh, Bilal Ahmed ; Aggarwal, Sunita ; Seth, Tulika ; Nigam, Rajendra K ; Jain, Ankur ; Baveja, Avriti ; Atri, Sudhir ; Sharma, Vishnu ; Digra, Sanjeev Kumar ; Vyas, Bhadresh ; Gupta, Naresh

Hemophilia, an X-linked genetic bleeding disorder, is caused by the deficiency of coagulation factors VIII (hemophilia A) or IX (hemophilia B). Regular replacement therapy with the missing clotting factor is an effective standard-of-care treatment. However, it comes with a significant fallout of frequent intravenous dosing with poor compliance, the risk of inhibitor development, and a substantial treatment burden. Research has progressed from missing clotting factors and factor VIII mimetics to the most recent rebalancing therapy that suppresses tissue factor pathway inhibitor (TFPI). Thrombin generation is restricted by TFPI, which inhibits the tissue factor-mediated activation of factor VII. This promising therapeutic approach rebalances hemostasis by inhibiting TFPI, a critical regulator of the extrinsic coagulation pathway, thereby increasing thrombin generation. Novel monoclonal antibodies (concizumab and marstacimab) enhance thrombin generation by blocking TFPI to restore hemostasis. Clinical trials have demonstrated good clinical efficacy and safety of these anti-TFPI, besides their convenient subcutaneous administration using pen devices. These innovative therapies have the potential to enhance the quality of life (QoL) of people with hemophilia. This review provides a comprehensive overview of the clinical development, therapeutic potential, challenges, and prospects of anti-TFPI in the management of hemophilia.

News (Medical) associated with Marstacimab-hncq

01 Aug 2025

·firstwordpharma.com

FDA okays broader indications for Novartis’ lipid-lowering drug, Novo’s haemophilia prophylatic

The FDA cleared additional indications for Novartis’s cholesterol-lowering drug Leqvio (inclisiran) and Novo Nordisk's haemophilia prophylactic Alhemo (concizumab-mtci), broadening their use to wider patient populations.Leqvio gets go-ahead as montherapyNovartis's twice-yearly injectable siRNA drug Leqvio was approved by the FDA as monotherapy alongside diet and exercise to lower low-denisty lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, thereby eliminating the previous requirement for concurrent statin therapy.“With this new indication enabling Leqvio's use as monotherapy…we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey," said Victor Bultó, president of Novartis US.The US regulator said Leqvio’s broader indication was approved based on strong LDL-C-lowering data from PCSK9-targeting therapies. Additional label updates include replacing "primary hyperlipidaemia" terminology with the more specific "hypercholesterolaemia" designation throughout the prescribing information.Novartis noted the decision also aligns with the 2025 American College of Cardiology/American Heart Association Clinical Practice Guidelines, recommending more aggressive treatment approaches for patients with acute coronary syndrome.Leqvio was discovered by Alnylam and co-developed with The Medicines Company, which Novartis acquired in 2020 for $9.7 billion. First approved in the EU for hypercholesterolaemia or mixed dyslipidaemia in 2020, the siRNA’s US approval faced initial delays owing to manufacturing issues, but was subsequently cleared by the FDA in 2021 for patients on maximally tolerated statin therapy.The Swiss drugmaker expects Leqvio not only to surpass $1 billion in sales this year, but potentially outperform consensus forecasts long term. For more, read – Spotlight On: Novartis' fastest growing drugs.Alhemo cleared for patients without inhibitorsThe FDA also okayed an expanded indication for Novo Nordisk's Alhemo (concizumab-mtci), extending its prophylactic use to adult and paediatric patients aged 12 and older with haemophilia A or B without inhibitors. The latest approval builds upon the tissue factor pathway (TFPI) inhibitor antagonist’s approval last year for patients with inhibitors after being rejected by the agency on the first attempt.Anna Windle, senior VP of clinical development, medical & regulatory affairs at Novo Nordisk, said the latest approval “marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option."The label expansion was supported by the Phase III explorer8 study, wherein Alhemo achieved an 86% reduction in annualised bleeding rate for patients with haemophilia A and 79% reduction for those with haemophilia B versus no prophylaxis.Following a positive recommendation by the EU drug advisory body last week, the anti-TFPI monoclonal antibody is also on the verge of approval in Europe for the same indication.With the additional indication, Alhemo emerges as a potential rival to Pfizer’s once-weekly Hympavzi (marstacimab-hncq), approved last year for patients without inhibitors. Pfizer also seeks Hympavzi’s expansion to patients with inhibitors following a successful Phase III study.

Drug ApprovalPhase 3AcquisitionClinical ResultAHA

27 Jun 2025

·pmlive.com

Pfizer shares positive phase 3 results for haemophilia A and B therapy Hympavzi

Pfizer has shared positive top-line results from a late-stage study of its haemophilia A and B therapy Hympavzi (marstacimab). The phase 3 BASIS trial has been evaluating the drug in adult and adolescent patients with inhibitors, or antibodies, which neutralise factor replacement therapies and make them ineffective. The study met its primary endpoint, with once-weekly prophylactic treatment with Hympavzi resulting in a statistically significant and clinically relevant reduction in annualised bleeding rate (ABR) of treated bleeds compared to on-demand treatment. Patients were treated with Hympavzi during a 12-month period versus an on-demand intravenous regimen with bypassing agents, given as part of usual care in a six-month lead-in period. Hympavzi reduced ABR by 93% over 12 months and also demonstrated superiority across all bleeding-related secondary endpoints, including spontaneous bleeds, joint bleeds, target joint bleeds and total bleeds. Over 800,000 people worldwide are affected by haemophilia, a group of rare genetic blood disorders caused by a clotting factor deficiency. Alongside episodes of excessive and prolonged bleeding, patients can experience spontaneous bleeding in their joints and muscles without having had an injury, potentially leading to joint damage. Pfizer’s Hympavzi, given as a once-weekly subcutaneous injection using a pre-filled pen, works by reducing the amount of TFPI, a naturally occurring protein that prevents blood from clotting too much. This promotes the formation of thrombin, an enzyme critical in blood clotting. The drug already holds approvals to treat haemophilia A and B patients without inhibitors. However, around 20% and 3% of haemophilia A and B patients, respectively, are unable to continue taking factor replacement therapies because they develop inhibitors to factor VIII and factor IX. Michael Vincent, chief inflammation and immunology officer at Pfizer, said: “These encouraging results demonstrate Hympavzi’s potential to help people living with haemophilia A or B with inhibitors, meeting an important need for patients with antibodies that neutralise most factor-based prophylactic options used to manage bleeding episodes.” Pfizer said it is planning to discuss the latest results with regulatory authorities, “with the goal of initiating regulatory filings for Hympavzi for the treatment of patients living with haemophilia with inhibitors”.

Clinical ResultPhase 3Drug Approval

26 Jun 2025

·www.fiercepharma.com

Pfizer's hemophilia drug Hympavzi notches phase 3 win as pharma eyes label expansion

Trial results indicate that Pfizer’s Hympavzi is effective in patients with hemophilia A and B who are inhibitors, setting it up for a label expansion. The treatment was approved last year for hemophilia A and B patients who are non-inhibitors. Approved eight months ago for a certain class of patients with both hemophilia A or B, Pfizer’s Hympavzi has now shown its effectiveness in another group of patients with the bleeding disorder.That's according to new top-line results released June 26 from a phase 3 trial. This test was specifically in patients with hemophilia A or B who are classified as “inhibitors” and saw the drug hitting its primary endpoint and key secondary endpoints. The study showed the superiority of once-weekly injections of Hympavzi compared to the performance of on-demand treatment in improving bleeding outcomes, Pfizer announced.The results come on top of the FDA endorsing Hympavzi for patients with hemophilia A or B who are classified as “non-inhibitors.” In those with the disorder, inhibitors are patients who develop antibodies that counter the effectiveness of commonly used clotting factor replacement therapies to control bleeding.“Patients with inhibitors tend to face frequent complications and navigating the treatment landscape can introduce complexities and increase disease burden,” Davide Matino, M.D., the principal investigator in the Basis study and a professor at McMaster University, said in a release.Matino added that the bleed reduction demonstrated in the trial by Hympavzi “coupled with its weekly administration method, offers exciting potential for patients who are in critical need of treatment options.”In the trial, 48 patients were given Hympavzi during a 12-month period versus an on-demand intravenous regimen with bypassing agents, as part of usual care in a six-month lead-in period. Hympavzi reduced the annual bleeding rate by 93% over 12 months.Hympavzi also provided superior control in all bleeding-related secondary endpoint measures, including spontaneous bleeds, joint bleed, target joint bleeds and total bleeds. There were no deaths or thromboembolic events.Pfizer said it will reveal full data at an upcoming medical meeting and will discuss the data with regulatory authorities, with the “goal of initiating regulatory filings” for hemophilia patients who are classified as inhibitors. The only other treatment for hemophilia A and B which can be given to inhibitors and non-inhibitors is Sanofi’s Qfitlia, a small interfering RNA drug, which gained an FDA approval three months ago. It has an edge in that it is designed to be administered every two months, though the dosing period can be adjusted depending on the performance of the treatment.Several new hemophilia therapies have hit the market in recent years, including Novo Nordisk’s daily injectable Alhemo, which was approved in December for A and B patients who are inhibitors.Roche’s Hemlibra, which generated sales of $4.9 billion last year and can be used by inhibitors and non-inhibitors, rules the hemophilia A market but is being challenged by Sanofi and Sobi’s Altuviiio, which was approved in 2023 and achieved sales of $682 million last year. Hympavzi was one of two Pfizer hemophilia treatments approved by the FDA in 2024. The company has already removed the other—hemophilia B gene therapy Beqvez—from the market, saying it was unable to attract customers or physicians to the $3.5 million one-time treatment. Of the more than 800,000 people in the world with the disorder, roughly 20% of those with hemophilia A and 3% of those with hemophilia B are inhibitors, Pfizer said. Hemophilia inhibits the blood’s ability to clot properly, increasing the risk of repeated bleeding inside the joints, which can lead to hemophilic arthropathy, which may require surgery to repair or to replace damaged joints.

Clinical ResultPhase 3Drug ApprovalGene Therapy

100 Deals associated with Marstacimab-hncq

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KEGG	Wiki	ATC	Drug Bank
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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hemophilia A	United States	Pfizer Inc.	11 Oct 2024
Hemophilia B	United States	Pfizer Inc.	11 Oct 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia	NDA/BLA	China	Pfizer Inc.	13 Aug 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03938792 (BASIS, BusinessWire) Manual	Phase 3	Hemophilia B \| Hemophilia A	-	Hympavzi	uvqhcayget(ovibhqevkw) = zvyjbxgumy qdyfpqwgku (dbouwddgii ) Met	Positive	26 Jun 2025
				on-demand treatment	uvqhcayget(ovibhqevkw) = fbiguwhqyd qdyfpqwgku (dbouwddgii ) Met
ASH2024	Not Applicable	Hemophilia B \| Hemophilia A	-	Marstacimab 150 mg SC QW	bnvnzgwbif(dukbhdahxz) = upudtyjbmr joiaxojkpg (dtaxptmgfl )	-	09 Dec 2024
				Factor replacement therapy (OD or RP)	bnvnzgwbif(dukbhdahxz) = brfbhaippt joiaxojkpg (dtaxptmgfl )
NCT05145127 (FDA_CDER) Manual	Phase 3	Hemophilia B \| Hemophilia A	-	On-Demand Factor-Based Therapy	lmcgrsrzmx(ylkwwxpfmv) = amkgxhrcjn ymjircwbzl (stbvcjckcl, 5.09 - 10.61) View more	Positive	11 Oct 2024
				HYMPAVZI Prophylaxis	lmcgrsrzmx(ylkwwxpfmv) = gxmlcmxzsq ymjircwbzl (stbvcjckcl, 3.40 - 6.77) View more
NCT03938792 (FDA_CDER) Manual	Phase 3	Hemophilia B \| Hemophilia A	66	On-Demand Factor-Based Therapy	saufumrhzg(ebjmylsoag) = ncmhajrnbf gtxhphjowb (kkfpmgoyea, 31.03 - 46.54) View more	Positive	11 Oct 2024
				HYMPAVZI Prophylaxis	saufumrhzg(ebjmylsoag) = gbinzgzaau gtxhphjowb (kkfpmgoyea, 2.09 - 4.85) View more
NCT03938792 (BASIS, ASH2023) Manual	Phase 3	Hemophilia B \| Hemophilia A	128	Marstacimab (On Demand) (Observational Phase)	qbivnjihnc(rxzgeiucyx) = rtwqvakgjl eiveniqmdl (fomhcrvnzu, 31.03 - 46.54) View more	Positive	09 Dec 2023
				Marstacimab (Routine Prophylaxis) (Observational Phase)	qbivnjihnc(rxzgeiucyx) = ukhhwlltax eiveniqmdl (fomhcrvnzu, 5.09 - 10.61) View more
NCT04878731 (CTgov)	Phase 1	Hemophilia	6	PF-06741086 (Marstacimab)	yziqgyxazh = grdpiztjdf fcayzckhwx (vjqiowoino, nkhjjsoymd - leczdghewl) View more	-	07 Jul 2023
NCT03363321 (Pubmed) Manual	Phase 2	Hemophilia A	18	Marstacimab	xazbaslyde(yakrylmnnq) = ftkechfjjd dztgufcfvd (eepzfgyklq )	Positive	11 Oct 2022
NCT02974855 (CTgov)	Phase 2	Hemophilia	27	PF-06741086 (PF-06741086 300 mg SC QW Non-Inhibitor)	pbhvidfbbe = yermsbrqfd yvspnnefor (nstjstpbjv, qcmuyfkios - apzhyfzkza) View more	-	04 Dec 2019
				PF-06741086 (PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor)	pbhvidfbbe = fomqdrffam yvspnnefor (nstjstpbjv, vraxcellao - sgrxkmfbec) View more

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