Hympavzi (marstacimab-hncq) / Pfizer - LARVOL DELTA

Consistent clinical factor VIII equivalency is unlikely for non-factor therapies in hemophilic mice. (PubMed, Haematologica) - "Strikingly, both emicizumab- and SIA-marstacimab-treatment resulted in spontaneous rebleeds in the TVT-, TAT- and tail-clip-models, further distinguishing them from FVIII-treatment. Our data suggest that a single FVIIIequivalence is unlikely to exist for emicizumab, SIA-marstacimab and similar molecules, because their activity is dependent on local conditions and severity of the injury."

Journal • Preclinical • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Novel drugs approved by the EMA, the FDA and the MHRA in 2024: A year in review. (PubMed, Br J Pharmacol) - "Some notable examples are the first drug successfully using a 'dock-and-block' mechanism of inhibition (zenocutuzumab), the first approved drug for schizophrenia designed as an agonist of M1/M4 muscarinic receptors (xanomeline), the first biparatopic antibody (zanidatamab), binding two distinct epitopes of the same molecule, the first haemophilia therapy that instead of relying on external supplementation of clotting factors, restores Factor Xa activity by inhibiting TFPI (marstacimab), or the first ever authorised direct telomerase inhibitor (imetelstat) that reprogrammes the oncogenic drive of tumour cells. In addition, an impressive percentage of novel drugs were first in class (28 out of 53 or 53% of the total) and a substantial number can be considered disease agnostic, indicating the possibility of future approved extensions of their use for additional indications. The 2024 harvest demonstrates the therapeutic potential of innovative pharmacological design,..."

European regulatory • FDA event • Journal • Review • CNS Disorders • Hematological Disorders • Hemophilia • Oncology • Psychiatry • Rare Diseases • Schizophrenia

LLUH becomes first in California to offer immunotherapy for Hemophilia B patients (Loma Linda University Health) - "For patients with Hemophilia B, managing their bleeding condition has long been a demanding and painful process. Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first medical center in California to initiate treatment with Marstacimab (Hympavzi), a newly FDA-approved subcutaneous immunotherapy. This breakthrough offers an alternative to the painful injections into the vein that has been a long-standing standard of care for patients from early childhood throughout the rest of their lives."

Launch US • Hemophilia B

Satellite Symposium 7: A new era of haemophilia care with marstacimab (EAHAD 2025) - "Symposium objectives: This symposium aims to illustrate some of the key practical considerations for patient management in the new era of haemophilia therapy. We will discuss clinical decision making, choosing the appropriate type of treatment within an evolving treatment landscape, understanding efficacy and safety, and associated informational needs."

Hematological Disorders • Hemophilia • Rare Diseases

Clinical considerations for practical implementation of marstacimab and other approved novel rebalancing therapies (EAHAD 2025) - "This symposium has been organised and funded by Pfizer and is intended for healthcare professionals only"

Clinical • Gene Therapies • Hematological Disorders • Hemophilia • Rare Diseases

Long-Term Efficacy of Marstacimab in Adults and Adolescents With Severe Hemophilia A or B Without Inhibitors Who Completed the BASIS Trial (EAHAD 2025) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics of Switching From Emicizumab to Marstacimab: In Vitro Data and Phase 1b Study Design (EAHAD 2025) - No abstract available

P1 data • PK/PD data • Preclinical

Surgical and Medical Procedures in Participants With Hemophilia A or B Without Inhibitors Receiving Marstacimab in the BASIS and Open-Label Extension Trials (EAHAD 2025) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Indirect Treatment Comparison of Marstacimab versus Emicizumab in Haemophilia A (EAHAD 2025) - No abstract available

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Exogenous Factor Consumption in Participants With Hemophilia A or B Without Inhibitors Receiving Marstacimab in the BASIS Trial (EAHAD 2025) - No abstract available

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Outcomes by Dose Escalation Criteria in Participants With Hemophilia A or B Without Inhibitors Receiving Marstacimab in the BASIS Trial (EAHAD 2025) - No abstract available

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Health-Related Quality of Life Outcomes for Marstacimab in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B Without Inhibitors: Results From the Phase 3 BASIS Trial (EAHAD 2025) - No abstract available

Clinical • HEOR • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A. (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Pfizer | N=15 ➔ 10 | Trial completion date: Jul 2025 ➔ Jun 2026 | Initiation date: Dec 2024 ➔ Apr 2025 | Trial primary completion date: Jul 2025 ➔ Jun 2026

Enrollment change • Trial completion date • Trial initiation date • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Marstacimab-hncq. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Marstacimab: First Approval. (PubMed, Drugs) - "Marstacimab has since been approved on 18 Nov in the EU for the treatment of adults and adolescents with severe hemophilia A or B without inhibitors. This article summarizes the milestones in the development of marstacimab leading to this first approval for hemophilia."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Pediatrics • Rare Diseases

Advances in Development of Drug Treatment for Hemophilia with Inhibitors. (PubMed, ACS Pharmacol Transl Sci) - "More recently, emicizumab, a bispecific antibody that mimics the function of activated clotting factor VIII, has demonstrated favorable efficacy for prophylaxis in patients with hemophilia A and inhibitors, representing a promising new therapeutic strategy...This review summarizes the current understanding of the pathophysiology of inhibitor development in hemophilia, outlines existing treatment options, and discusses advancements in novel therapeutic biologics, including a recombinant activated clotting factor VII variant (marzeptacog alfa), a new bispecific antibody (Mim8), antitissue factor pathway inhibitor antibodies (concizumab and marstacimab), and small interfering RNA targeting antithrombin (fitusiran). All of these agents are administered subcutaneously, with some offering the convenience of less frequent dosing (e.g., weekly or monthly). These potential drug candidates may provide significant benefits for the prophylaxis or treatment of bleeding disorders in..."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

The Science Behind HYMPAVZI (Marstacimab-hncq): Introducing an Innovative Treatment Option from Pfizer (ASH 2024) - "Sponsored by Pfizer For in-Person participants only"

Gene Therapy and Hemophilia A: What Is the Future of Curative Therapy in the Age of Emicizumab? (ASH 2024) - "Additionally, the beneficial results seen in hemophilia A gene therapy clinical trials have occurred with meaningful challenges. This talk will review the risks and benefits of gene therapy for hemophilia A and consider them within the context of therapies (emicizumab and Fc-VWF-XTEN fusion protein-eht) that have shown consistent benefit compared with previously available factor VIII products as well as other promising therapies (Mim8, fitusiran, concizumab, and marstacimab) in late-stage clinical trials."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Hympavzi (Marstacimab-hncq). (PubMed, Clin Ther) - No abstract available

Journal

Marstacimab-hncq for Treatment of Hemophilia A or Hemophilia B (ASH 2024) - No abstract available

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Descriptive Characterization of Bleeding Events in Participants with Severe Hemophilia Α or B without Inhibitors, Receiving Prophylactic Marstacimab Treatment (ASH 2024) - P3 | "Background : Marstacimab is a monoclonal antibody targeted to the K2 domain of tissue factor pathway inhibitor to reduce inhibition of the extrinsic coagulation pathway and rebalance hemostasis independently of factor VIII (FVIII) and factor IX (FIX) activity. Of note, in pts with prior FVIII prophylaxis in the HAVEN-3 emicizumab study who had bleeds, 24% of total bleeds were treated (Callaghan et al, RPTH, 2022), suggesting differences in bleed management vs the BASIS study. Overall, these results demonstrate that a high proportion of bleeding events were treated with factor replacement therapy in the BASIS study, but that clinical management and treatment strategies may differ across trials."

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

A Fixed (Weight-independent) Subcutaneous Once-Weekly Dose for Marstacimab, an Anti-TFPI Monoclonal Antibody for the Prophylactic Treatment of Hemophilia Α and B (ASH 2024) - P2, P3 | "To date, no thromboembolic events have been observed in hemophilia patients at any of the clinical doses (EAHAD 2024 poster # P0186). Conclusions : A flat (fixed) dosing regimen for marstacimab, supported by comparable PD and ABRs across weight ranges, lack of safety concerns to date and an absence of a narrow therapeutic window profile, provides significant advantages of patient convenience, compliance, less risk of dosing errors as well as cost-effectiveness."

Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Safety Biomarkers in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B Without Inhibitors Receiving Prophylactic Marstacimab Treatment: Results From the Phase 3 BASIS Trial (ASH 2024) - P3 | "There was no clinically meaningful impact on aPTT. There were no reported adverse events related to CFB in safety biomarkers in any participants."

Biomarker • Clinical • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Hepatology • Inflammation • Rare Diseases

Non-factor Therapies for Hemophilia: Achievements and Perspectives. (PubMed, Semin Thromb Hemost) - "Factor VIII (FVIII)-mimetic agents, such as emicizumab, have transformed the management of hemophilia A with inhibitors, offering a lower treatment burden and an effective alternative for those without inhibitors as well. Rebalancing agents, including anti-tissular factor pathway inhibitor agents (concizumab and marstacimab) and serpin inhibitors like fitusiran, have shown promising efficacy for patients with hemophilia B with inhibitors and other hemophilia subtypes...Unresolved issues include optimal management strategies for major surgeries and tailored approaches for safe use in older populations. This review highlights the progress and future potential of NFTs in treating persons with hemophilia."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A. (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Pfizer

New P1 trial • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases