Hympavzi (marstacimab-hncq) / Pfizer - LARVOL DELTA

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors (clinicaltrials.gov) - P3 | N=245 | Recruiting | Sponsor: Pfizer | N=145 ➔ 245

Enrollment change • 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

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. The majority of bleeds across both OD and RP groups during the BASIS study (OP and ATP) were spontaneous, treated, and occurred in joints, most commonly the ankle/foot, elbow, and knee. In the OD group, marstacimab prophylaxis resulted in a greater proportion of pts without any bleeding events and a lower proportion with treated bleeds during the ATP vs the OP. Of pts who had bleeding events, the RP group had a relatively greater proportion of treated bleeds (86.9%) vs the OD group (48.6%)."

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 | "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

U.S. FDA Approves Pfizer’s HYMPAVZI (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors (Pfizer Press Release) - "Pfizer Inc...announced today that the U.S. Food and Drug Administration (FDA) has approved HYMPAVZI (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) without factor VIII (FVIII) inhibitors, or hemophilia B (congenital factor IX deficiency) without factor IX (FIX) inhibitors....Results from the Phase 3 BASIS trial...supported the approval of HYMPAVZI in the U.S. in adults and adolescents with hemophilia A or B without inhibitors."

FDA approval • Genetic Disorders • Hemophilia • Hemophilia A • Hemophilia B

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 16-19 September 2024 (European Medicines Agency) - "Hympavzi (marstacimab), received a positive opinion for the treatment of bleeding episodes in patients aged 12 years and older with severe haemophilia A or B, two types of a rare inherited bleeding disorder."

CHMP • Genetic Disorders • Hemophilia • Hemophilia A • Hemophilia B

SUSTAINED EFFICACY OF MARSTACIMAB IN ADULTS AND ADOLESCENTS WITH SEVERE HEMOPHILIA A OR B WITHOUT INHIBITORS (EHA 2024) - P3 | "Long term treatment with once weekly SC marstacimab demonstrates continued safetyand sustained efficacy for treated & total ABR in adults and adolescents with HA or HB without inhibitors. Nearly all non-inhibitor participants have transitioned from the pivotal study to the OLE study. Participants arehighly compliant with therapy."

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

Coagulation biomarker levels in subjects with severe hemophilia receiving acute treatment for bleeding episodes experienced during prophylactic marstacimab treatment (ISTH 2024) - "In general, elevated levels of peak thrombin, D-dimer and PF 1+2 were not seen following bleed treatment per protocol specified recommended guidelines (sample collection within and outside 4-day window). Biomarkers levels in patients were generally lower than or similar to those seen in healthy volunteers. Except for transient increases, biomarker levels were generally below 120 nM for peak thrombin, below 1 µg/mL for D-dimer and below 1200 pmol/L for PF 1+2."

Biomarker • Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Rare Diseases • Thrombosis

SAFETY AND EFFICACY OF THE ANTI-TISSUE FACTOR PATHWAY INHIBITOR MARSTACIMAB IN PARTICIPANTS WITH SEVERE HEMOPHILIA WITHOUT INHIBITORS: RESULTS FROM THE PHASE 3 BASIS TRIAL AND ONGOING LONG-TERM EXTENSION STUDY (THSNA 2024) - P3 | "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 for 12 months in BASIS, and in an additional 16 months in the LTE study."

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

Dose escalation of the anti-tissue factor pathway inhibitor marstacimab in participants with severe hemophilia without inhibitors: results from the phase 3 BASIS and long-term extension trials (WFH 2024) - No abstract available

P3 data • Hematological Disorders • Hemophilia • Rare Diseases

Dose escalation of the anti-tissue factor pathway inhibitor marstacimab in participants with severe hemophilia without inhibitors: results from the phase 3 BASIS and long-term extension trials (WFH 2024) - No abstract available

P3 data • Hematological Disorders • Hemophilia • Rare Diseases

A substudy to evaluate the feasibility and effectiveness of marstacimab administration using a prefilled pen injection device (WFH 2024) - No abstract available

Benefits and risks of non-factor therapies: Redefining haemophilia treatment goals in the era of new technologies. (PubMed, Haemophilia) - "With the advent of novel therapeutic agents including factor concentrates with ultra-long half-life and improved FVIIIa mimetics aimed at raising the bar of protection into the non-hemophilic range redefinition of haemophilia treatment goals is eagerly needed."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

BASIS: Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (clinicaltrials.gov) - P3 | N=175 | Active, not recruiting | Sponsor: Pfizer | Trial completion date: Sep 2024 ➔ Jun 2025 | Trial primary completion date: Sep 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Hematological Disorders • Hemophilia • Rare Diseases

Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B. (PubMed, Cochrane Database Syst Rev) - "Evidence from RCTs shows that prophylaxis using non-clotting factor therapies compared with on-demand treatment may reduce bleeding events, increase the percentage of individuals with zero bleeds, increase the incidence of non-serious adverse events, and improve HRQoL. Comparative assessments with other prophylaxis regimens, assessment of long-term joint outcomes, and assessment of economic outcomes will improve evidence-based decision-making for the use of these therapies in bleed prevention."

Journal • Review • Cystic Fibrosis • Fibrosis • Genetic Disorders • Hematological Disorders • Hemophilia • Immunology • Oncology • Pain • Pulmonary Disease • Rare Diseases • Respiratory Diseases

Joint health in participants with hemophilia A and hemophilia B without inhibitors treated with marstacimab from the phase 3 BASIS trial (EAHAD 2024) - No abstract available

P3 data • Hematological Disorders • Hemophilia • Rare Diseases

Immunogenicity and safety of marstacimab, an anti-tissue factor pathway inhibitor, in participants with hemophilia A or B and without inhibitors (EAHAD 2024) - No abstract available

Clinical • Hematological Disorders • Hemophilia • Rare Diseases

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 • 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 • Inflammation • Pain • Pruritus • Rare Diseases

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (clinicaltrials.gov) - P3 | N=173 | Active, not recruiting | Sponsor: Pfizer | Recruiting ➔ Active, not recruiting

Enrollment closed • Hematological Disorders • Hemophilia • Rare Diseases

Evolution of Antidrug Antibody Assays During the Development of Anti-Tissue Factor Pathway Inhibitor Monoclonal Antibody Marstacimab. (PubMed, AAPS J) - "Additional criteria for validation were dilution linearity (Methods 1 and 2) and low positive control concentration, prozone effect, plate homogeneity, and robustness (Method 3). The three methods met validation criteria and are a potentially valuable tool in detecting the induction of marstacimab ADAs during treatment in patients with hemophilia."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of marstacimab in Chinese participants with severe haemophilia. (PubMed, Haemophilia) - No abstract available

Journal • PK/PD data • Hematological Disorders • Hemophilia • Rare Diseases

"#Pfizer anuncia resultados positivos de marstacimab en el ensayo pivotal de fase 3 de hemofilia A y B @pfizer https://t.co/dHEvo4st7s" (@ReporteSalud)

Hemophilia