Hympavzi (marstacimab-hncq) / Pfizer - LARVOL DELTA

Management of breakthrough bleeds in participants with hemophilia Α or Β without inhibitors receiving marstacimab prophylaxis in the phase 3 BASIS study (ASH 2025) - "Most bleeding episodes were managed with a single infusion of factor replacement therapy, and standard half-life products were the most common intervention. Importantly, no thrombotic or thromboembolic events were reported in association with concomitant factor replacement use with marstacimab prophylaxis."

P3 data • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Evaluation of the combined effects of emicizumab and marstacimab in plasma from Hemophilia A patients using thrombin generation assay (ASH 2025) - "Our data show that addition of marstacimab to plasma from patients on prophylaxis withemicizumab resulted in a dose-dependent increase in peak thrombin generation and ETP, while alsoreducing lag time. These changes consistently remained mostly within the range observed in healthydonors. Overall, no evidence of excessive thrombin generation was detected, even at the highestconcentrations of marstacimab tested."

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

Outcomes of marstacimab treatment in adolescent participants with Hemophilia A or B without inhibitors compared with prior routine prophylaxis: Results from the phase 3 BASIS trial (ASH 2025) - P3 | "Compared with previous RP therapy, SC QW marstacimab reduced bleeding in adolescent ptswith HA or HB without inhibitors and was generally well tolerated. No clinically relevant differences in PDendpoints were observed compared with adults and PK differences were explained by weight."

P3 data • Cardiovascular • Dermatology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Infectious Disease • Novel Coronavirus Disease • Otorhinolaryngology • Pruritus • Rare Diseases

Long term effect of marstacimab prophylaxis in hemophilia Α and Β on target joints: Results from BASIS and OLE studies (ASH 2025) - P3 | "Marstacimab prophylaxis was generally safe and led to sustained and clinically meaningfulreductions in TJ bleeding and high rates of joint resolution, even among patients with severe baselinejoint involvement. These findings reinforce the long-term efficacy of marstacimab in people with severeHA or HB without inhibitors."

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

Characterization of participants with elevated bleeding rates responding to prophylactic marstacimab treatment in the phase 3 BASIS trial (ASH 2025) - P3 | "However, most pts with an elevated ABR showed ABR reductions during theATP or OLE, suggesting these pts still responded to marstacimab. Dose escalation to marstacimab 300mg SC QW led to reduced ABR in all pts with an elevated ABR who escalated, suggesting this may be anoption for select pts."

P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Rheumatology

Real‑world experience with anti‑TFPI agent (Marstacimab) in severe and non‑severe hemophilia: First reported successful use in a female with non-severe hemophilia b (ASH 2025) - "Recent approval of rebalancing agents—includingMarstacimab, a tissue factor pathway inhibitor (TFPI) monoclonal antibody for people with hemophilia Aand B without inhibitors, along with Fitusiran and Concizumab—herald a new era of expanded, andeffective patient-friendly therapeutic options.To date, regulatory approvals and pivotal trials have focused predominantly on severe hemophilia (factorVIII or IX activity <1%), often excluding non-severe (1–5% activity) and female pwh...50% switchedfrom a CFC based prophylaxis while the remaining switched to weekly SQ regimen from on demandtherapy in the moderate cohort whereas 75% of severe Hemophilia A/B patients switched from CFC andremaining from previous Emicizumab prophylaxis... Our findings underscore the critical need to consider bleeding phenotype—not merely factorlevels—when selecting candidates for rebalancing therapies. Women with hemophilia endure recurrent,debilitating bleeds related to menorrhagia or obstetric..."

Clinical • Real-world • Real-world evidence • Cardiovascular • Gynecology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Diseases • Obstetrics • Rare Diseases • Rheumatology

Management of breakthrough bleeds in participants with hemophilia Α or Β without inhibitors receiving marstacimab prophylaxis in the phase 3 BASIS study (ASH 2025) - P3 | "No thromboembolic events were reported in association with concomitant FRT use.ConclusionsIn the BASIS study, for pts with HA or HB without inhibitors, acute breakthrough bleeding episodes weresuccessfully managed with episodic FRT during concurrent QW marstacimab prophylaxis andmarstacimab was generally safe. Most acute breakthrough bleeds were managed with a single infusion ofFRT, and the most common treatment agents were SHL FVIII products (recombinant or pd)."

P3 data • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Marstacimab prophylaxis in participants with Hemophilia A or B with inhibitors: Results from the Phase 3 BASIS trial (ASH 2025) - P3 | "No deaths or thrombotic events were reported.Conclusion In pts with HA or HB and inhibitors, subcutaneous marstacimab QW significantly reduced ABRfor all bleeding related endpoints vs prior OD therapy and improved HRQoL. Marstacimab demonstrateda favorable safety profile, consistent with the noninhibitor cohort and earlier studies."

P3 data • Dermatology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Infectious Disease • Novel Coronavirus Disease • Rare Diseases • Respiratory Diseases

Exploring a Subcutaneous Treatment Option in Hemophilia Care with HYMPAVZI (marstacimab-hncq) (ASH 2025) - "Supported By Pfizer For in-person participants only"

Hematological Disorders • Hemophilia • Rare Diseases

fss25-110: Advancing Hemophilia Care—Uniting Expert Insights and Community Voices to Shape the Future of Non-Factor Replacement Therapy (ASH 2025) - "This dynamic event combines expert panel discussions, real-world case challenges, and insights from patients and community HCPs to explore the latest data on novel therapies including fitusiran, concizumab, marstacimab, and Mim8. Through interactive polling and audience Q&A, attendees will gain practical guidance on integrating these therapies into personalized care strategies for patients with moderate to severe hemophilia A and B. Discover how to navigate evolving safety profiles, improve joint health outcomes, and tailor treatment based on patient needs and preferences. This symposium offers a comprehensive view of current challenges, cutting-edge solutions, and future directions in hemophilia management, equipping you with actionable insights to improve clinical practice."

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

Efficacy and Safety of Marstacimab Prophylaxis in Hemophilia A/B With Inhibitors: Results from the Phase 3 BASIS Trial. (PubMed, Blood) - P3 | "Marstacimab may be a viable treatment option for people with hemophilia A or B with inhibitors. This trial is registered at www.clinicaltrials.gov as # NCT03938792."

Journal • P3 data • Cardiovascular • Dermatology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

HYMPAVZI (marstacimab) Reduced Bleeds by 93% Compared to On-Demand Treatment in Adults and Adolescents with Hemophilia A or B with Inhibitors (Businesswire) - "In the BASIS trial, 48 adults and adolescents living with severe hemophilia A or hemophilia B with inhibitors were treated with HYMPAVZI....A statistically significant and clinically meaningful 93% reduction in mean treated annualized bleeding rate (ABR) (1.39 [95% CI: 0.85-2.29] vs.19.78 [95% CI: 16.12-24.27]; p<0.0001), demonstrating superiority of HYMPAVZI over OD therapy.....Superiority (p<0.0001) of HYMPAVZI was also demonstrated across all bleeding-related secondary endpoints – spontaneous bleeds (ABR 0.87 vs. ABR OD 15.27), joint bleeds (ABR 1.10 vs. ABR OD 15.15), target joint bleeds (ABR 0.79 vs. ABR OD 6.42), and total treated and untreated bleeds (ABR 4.36 vs. ABR OD 27.29)."

P3 data • Hemophilia A • Hemophilia B

Pfizer…announced that Hympavzi (marstacimab)...has received marketing approval from China’s National Medical Products Administration (NMPA) for routine prophylaxis in patients aged ≥12 years and weighing ≥35 kg with severe Hemophilia A or B without inhibitors (flcube.com) - "Launch expected Q1 2026....Phase III BASIS study demonstrated 91% reduction in treated bleeds vs. on‑demand factor therapy; non‑inferior to prophylactic factor in Hemophilia A and B patients without inhibitors....Expected to be included in China National Reimbursement Drug List (NRDL) by 2027 based on novel mechanism and patient convenience..."

China approval • Launch non-US • Reimbursement • Hemophilia A • Hemophilia B

HIZ: A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan. (clinicaltrials.gov) - P=N/A | N=50 | Active, not recruiting | Sponsor: Pfizer | Not yet recruiting ➔ Active, not recruiting

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

Marstacimab, an antitissue factor pathway inhibitor, combined with bypassing agents: effects on thrombin generation and in hemostatically normal rats. (PubMed, Res Pract Thromb Haemost) - "Effects of the combination of marstacimab plus pdFVIIa/FX were limited to alterations in coagulation parameters and similar to those of pdFVIIa/FX alone. These findings suggest marstacimab in combination with pdFVIIa/FX can increase hemostasis without excessive in vitro thrombin generation, and marstacimab in combination with rFVIIa, aPCC, or pdFVIIa/FX is well tolerated in hemostatically normal rats."

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

Marstacimab, an Anti-Tissue Factor Pathway Inhibitor, in Participants with Hemophilia Α or B, with and without Inhibitors: An Integrated Analysis of Safety (ASH 2023) - P2, P3 | "Once-weekly SC marstacimab was well tolerated in participants with severe HA or moderately severe to severe HB, without inhibitors, with a low rate and mild severity of ISRs, transient ADAs, and no thromboembolic events with continuous treatment up to 28 months in the phase 3 program."

Clinical • Cardiovascular • Dermatology • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Inflammation • Pruritus • 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"

Efficacy and Safety of the Anti-Tissue Factor Pathway Inhibitor Marstacimab in Participants with Severe Hemophilia without Inhibitors: Results from the Phase 3 Basis Trial (ASH 2023) - P3 | "Compared with previous OD or RP therapy, once weekly subcutaneous marstacimab was safe and effective for reducing bleeding events in participants with severe HA or moderately severe to severe HB without inhibitors beyond 12 months in the phase 3 study and up to an additional 16 months in the LTE study."

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

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

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

Retrospective Study of Chinese Adults and Adolescent Patients with Hemophilia on Marstacimab in Bo'ao pilot zone (ChiCTR) - P=N/A | N=23 | Not yet recruiting | Sponsor: Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine Hainan Hospital (Boao Research Hospital, Hainan); RUIJIN-HAINAN HOSPITA

New trial • Hematological Disorders • Hemophilia • Rare Diseases

HIZ: A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan. (clinicaltrials.gov) - P=N/A | N=50 | Not yet recruiting | Sponsor: Pfizer

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