Hemlibra (emicizumab-kxwh) / Roche, JW Pharma - LARVOL DELTA

Targeting early immunologic decline in acquired hemophilia A: A case for prophylactic rituximab (ASH 2025) - "We present the case of an 81-year-old man with idiopathic AHA and a mechanical aortic valve on chronic coumadin...Following treatment with triple immunosuppressive therapy including prednisone, rituximab, and cyclophosphamide, he achieved remission...Beyond its clinical impact, this approach may also offer significant cost savings by reducing the need for costly agents such as recombinant FVIII (obizur) or emicizumab. Further investigation is warranted to define relapse thresholds, evaluate safety, and determine the broader applicability of prophylactic immunosuppression in AHA patients."

Clinical • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases • Thrombocytopenic Purpura

Efficacy, quality of life, and safety of non-clotting factor prophylaxis in Hemophilia A and b: A meta-analysis across inhibitor status (ASH 2025) - "Eligible studies included double-arm randomized controlled trials (RCTs) comparing non-clotting factor prophylaxis (emicizumab, fitusiran, or concizumab) with on-demand treatment in patients with hemophilia A or B, regardless of inhibitor status. Compared to on-demand therapy, non-factor prophylactic therapies significantly reduce bleeding episodes, improve quality of life, and increase the likelihood of zero bleeds in patients with hemophilia. Although associated with a higher rate of adverse events, the therapies themselves did not increase the risk of serious adverse events. While efficacy outcomes were similar across agents, cost differences may influence treatment selection.These findings support the clinical utilization of non-factor prophylaxis in managing hemophilia A and B, regardless of inhibitor status."

HEOR • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Clinical experience with efanesoctocog alfa in patients with uncover needs on emicizumab (ASH 2025) - "In these patients, efanesoctocog alfa improved hemostatic control and joint status in individuals with HA and unmet needs under emicizumab, without safety concerns. Longer follow-up is necessary to confirm these findings with additional data."

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Orthopedics • Rare Diseases • Rheumatology

Hodgkin lymphoma in a patient with Hemophilia A on emicizumab prophylaxis: A case report (ASH 2025) - "He completed five cycles of chemotherapy with brentuximab, doxorubicin, etoposide, prednisone, cyclophosphamide and vincristine. As emicizumab is intended for long term maintenance therapy, it is critical to report any significant adverse events. This case further suggests that continued use of emicizumab during cancer-directed therapies is safe and effective for patients with hemophilia A."

Case report • Clinical • Classical Hodgkin Lymphoma • Hematological Malignancies • Hemophilia • Hemophilia A • Hemophilia B • Hodgkin Lymphoma • Human Immunodeficiency Virus • Infectious Disease • Lymphoma • Rare Diseases • Thrombocytopenia

Management of acute coronary syndrome and surgical revascularization in a patient with severe Hemophilia A on emicizumab (ASH 2025) - "Following an initial diagnosis of non-ST elevation myocardial infarction (MI), he received aspirin and therapeutic enoxaparin with FVIII replacement to mitigate bleeding risk. Shortly afterward, his clinical status deteriorated to an ST elevation MI, necessitating the addition of clopidogrel and use of emergent fibrinolytic therapy with further FVIII replacement to target a trough FVIII activity above 100% in the immediate post-lysis period...This case highlights the complex interplay between HA and ACS, balancing thrombotic and bleeding risks, as well as the nuances of lab monitoring for patients on emicizumab. Early identification of these high-risk patients and transfer to a facility with access to specialized laboratory monitoring and multidisciplinary care are required to optimize patient outcomes."

Clinical • Acute Coronary Syndrome • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Human Immunodeficiency Virus • Infectious Disease • Myocardial Infarction • Rare Diseases • Rheumatology • Thrombosis

Single-center experience with emicizumab in Chinese children under 3 years with severe Hemophilia A (ASH 2025) - "Emicizumab prophylaxis demonstrated excellent safety and efficacy in children under 3 years of age with severe hemophilia A. Most patients remained bleed-free or experienced only minor, trauma-associated bleeding events. The only serious spontaneous bleed occurred in a patient who had discontinued treatment, emphasizing the importance of strict adherence to the prophylactic regimen. These findings support the early use of emicizumab in infants and toddlers and reinforce the need to maintain continuous prophylaxis without unplanned interruptions to achieve optimal outcomes."

Clinical • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Rare Diseases

Pharmacokinetic and pharmacodynamic characteristics of emicizumab in Chinese pediatric patients with hemophilia A: A retrospective single-center study (ASH 2025) - "Conclusion This first real-world PK/PD study of emicizumab in Chinese pediatric patients demonstrates high exposure, predictable pharmacokinetics, and excellent bleeding control. The findings support early initiation, individualized dosing, and population-specific monitoring strategies in pediatric hemophilia A care."

PK/PD data • Retrospective data • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Pediatrics • Rare Diseases

Evaluating the safety and tolerability of switching from emicizumab to fitusiran prophylaxis in adult males with severe hemophilia A, with or without inhibitors: SWITCH study, a trial in progress (ASH 2025) - "This poster will present early clinical trial date from a study assessing the safety of switching from emicizumab to fitusiran in patients with hemophilia A who are on emicizumab."

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

Platelets stabilize factor VIIIa against loss of activity through subunit dissociation (ASH 2025) - "We wished to determine whether FVIII activity on platelet binding sites is enhanced bystabilization against A2 dissociation and/or protease cleavage.Like FVIII, the bispecific antibody emicizumab (Emi) serves as a cofactor that facilitates activation of FX byFIXa on either vesicles or platelets...Full-length recombinant factorVIII (octocog alfa (Antihemophilic Factor), Advate) was also used as a control (FVIII).Plasma clotting assays were performed using delipidated factor VIII-deficient plasma with corn trypsininhibitor to inhibit contact pathway activation...We are currently investigating the effect of the reported slower IIacleavage of full-length FVIII at residue 1689 as a possible explanation. Further experiments will probe therole(s) of FIXa and FX on decay of B domain deleted FVIII activity on platelets vs PLV.Our data shows that platelet binding sites provide enhanced factor VIII activity compared to phospholipidvesicles and suggests that stabilization of FVIIIa..."

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

Mim8 improves the In Vivo hemostasis of select hemophilia B causing factor IX variants (ASH 2025) - "Mim8 (denecimig) is a next-generation FVIIIa-mimetic with distinct binding epitopes and increased potency compared toemicizumab; it is in late-stage clinical development for HA. This clot size approaches the clot size inour previous studies of wild-type mice (platelets 400 ± 40 µm2 and fibrin 280 ± 40 µm2).ConclusionMim8 improves the in vitro and in vivo hemostatic activity of select HB-causing FIX variants. Themagnitude of this improvement for some variants—such as R333Q and I397T, which account for 5.7% ofpeople with HB—is likely therapeutically relevant."

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

Retrospective cohort analysis of treatment patterns and bleeding outcomes in the Korean bleeding disorder registry (KBDR) (ASH 2025) - "A total of 31 patientsreceived emicizumab, with a median of 10.7 administrations annually.Joint bleeding outcomes were favorable: in patients with severe hemophilia A, the median number ofannual joint bleeds was 0 (IQR: 0–10), with a mean of 13.6...The data demonstrate a high rate of prophylaxis use, particularly in younger and more severelyaffected patients, and favorable bleeding control across the cohort. As the registry moves into itsprospective phase, it is expected to provide further insights into long-term outcomes, optimizeindividualized treatment strategies, and ultimately improve the quality of life and clinical outcomes forpeople living with hemophilia in Korea."

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

Rates of inhibitor development and immune tolerance in children with severe Hemophilia A on emicizumab prophylaxis (ASH 2025) - "Our data suggest that FVIII inhibitor rates may be lower in PwHA initially started onemicizumab prophylaxis, though these PwHA may not have yet reached sufficient FVIII EDs. In addition,the elevated number of Black study participants adds critical data given racial disparities in research oninhibitor development. Ongoing analyses will include a comparison of the timing of FVIII EDs and inhibitordevelopment in PwHA on emicizumab prophylaxis to determine if inhibitor rates upon reaching 50 FVIIIEDs are different than for those on FVIII."

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Rare Diseases • F8

Assessment of coagulation potential of concomitant factor VIII administration in patients with haemophilia a receiving emicizumab prophylaxis (CAGUYAMA Study): A multicenter, open-label, non-randomized clinical trial (ASH 2025) - "No abnormalities in platelets counts, coagulation or fibrinolysis laboratory markers wereobserved during the follow-up.ConclusionIn PwHA receiving emicizumab prophylaxis, FVIII supplementation at 30±5 U/kg was safe and effective forbreakthrough bleeding and surgical hemostasis. These findings could support clinical use of FVIII withconsideration of emicizumab's baseline effect."

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

Quality of life and functional improvements with efanesoctocog alfa in patients with moderate-to-severe Hemophilia A: A real-world survey (ASH 2025) - "The most recent treatments prescribed prior to Efa were standard half-life products (64.5%), extended half-life products (45.2%), emicizumab (32.3%), and others (6.5%) (groupsexceed 100% as multiple treatments were used either prophylactically or on-demand). This is the first real-world, patient-reported outcomes study of prophylactic Efa use amongpatients with moderate-to-severe HA. Most patients rated Efa more favorably for hemophilia controlcompared to previous regimens. Improvements were observed in daily functioning, participation inphysical activity, and a reduced need for preventive measures and additional prophylactic doses."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Cardiovascular disease risk assessment in Japanese adults with hemophilia: Interim results of a multicenter study (ASH 2025) - "Inhemophilia B, 15 (88.2%) were severe and 2 (11.8%) were moderate, with inhibitors in 2 patients.Treatment regimens for hemophilia A included emicizumab (n=26), prophylaxis with extended half-lifefactor VIII (EHL-FVIII; n=41), prophylaxis with standard half-life FVIII (SHL-FVIII; n=18), and on-demandtherapy (n=24)...Aging, hypertension, dyslipidemia, and diabetes were identified as major risk factorsfor CVD. Additionally, TNF-α and baPWV emerged as potential markers for identifying individuals atmoderate or higher risk of coronary artery disease, warranting further investigation."

Clinical • Acute Coronary Syndrome • Atherosclerosis • Cardiovascular • Chronic Kidney Disease • Coronary Artery Disease • Diabetes • Dyslipidemia • Heart Failure • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Human Immunodeficiency Virus • Hypertension • Infectious Disease • Metabolic Disorders • Nephrology • Rare Diseases • CRP • ICAM1 • IL6 • TNFA

Pharmacodynamic biomarkers in people with hemophilia A receiving multiple ascending doses of NXT007 (ASH 2025) - P1/2 | "NXT007 was engineered and optimized based on emicizumab. PD markers were well correlated with NXT007 concentrations and demonstrated stableactivity throughout the maintenance-dosing period, while safety biomarkers remained unaffected. InPwHA receiving prophylaxis with NXT007, the TG peak height during the steady state was in the non-hemophilia range and comparable to that obtained in participants with FVIII levels in the normal range."

Biomarker • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • SPDEF

Real-world outcomes of PK/PD-guided individualized emicizumab dose reduction in Chinese pediatric patients with hemophilia A: A prospective observational study (ASH 2025) - "These findings highlight the potential of PK/PD-guided dosing to improve accessand cost-efficiency of emicizumab in settings where treatment affordability is a concern. Larger-scale,multi-center studies with extended follow-up are needed to confirm these promising results and supportbroader adoption of this strategy."

Clinical • Observational data • PK/PD data • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Pediatrics • Rare Diseases

Exploratory analysis from HAVEN 1–4 to further contextualize injection-site reactions among people with hemophilia A receiving emicizumab (ASH 2025) - P3 | "These data in PwHA receiving emicizumab prophylaxis across the four Phase III HAVEN 1–4clinical studies show that the proportion of participants that experienced ISRs declined over time to <1%,with 0.75% of over 42,000 injections being associated with an ISR. However, it is important toacknowledge that changes in visit schedules beyond the main phase of the studies may introduce areporting bias. The results from this analysis expand our understanding of the tolerability of emicizumabinjections in PwHA."

Dermatology • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Lentiviral vector transduced autologous CD34+ cells with FVIII transgene for gene therapy of Hemophilia A with history of inhibitors (ASH 2025) - "He was then started on immune toleranceinduction (ITI) with 25 IU/kg of extended half-life FVIII (Eloctate®) three times a week in December, 2022.For a trauma related oral bleeding in January, 2023, he was given one dose of emicizumab 3mg/kg withwhich his bleeding stopped within 24 hours...The conditioning protocol for the HSCT consisted ofmyeloablative doses of treosulfan along with fludarabine followed by infusion of the transduced CD34+gene therapy product cryo-preserved from November, 2022...This case demonstrates the first successful sustained expression of measurable FVIII activity in plasmaafter gene therapy for hemophilia A with history of inhibitors which had resolved after immune toleranceinduction. Gene therapy with LV transduced autologous CD34+ cells technology holds promise forhemophilia A patients with inhibitors and needs to be further explored."

Gene therapy • Viral vector • Bone Marrow Transplantation • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Hypotension • Infectious Disease • Inflammation • Mood Disorders • Mucositis • Neutropenia • Rare Diseases • Respiratory Diseases • Septic Shock • Tuberculosis • CD34

The Mayo Clinic enterprise experience and outcomes of perioperative utilization of recombinant factor VIIa (rFVIIa, NovoSeven®) (ASH 2025) - "Adjunctive medications to control bleeding included emicizumab, FEIBA, and rituximab.Mean estimated blood loss was 136 mL (SD 211 mL), and mean length of stay was 5.7 days (SD 6.4).Surgical risk strongly correlated with perioperative outcomes: blood loss increased from 19 mL in verylow-risk surgeries to 352 mL in very high-risk surgeries, while mean length of stay increased from 4.5days (risk 1) to 19 days (risk 4) and 11 days (risk 5).Actual rFVIIa dosing ranged from 7.2 to 7211.5 mcg/kg per dose, with a median of 67.4 mcg/kg and amean of 852.8 mcg/kg, reflecting variability in clinical practice. In conclusion, perioperative rFVIIa was most frequently used for Factor VII deficiency but was alsoadministered to patients with Factor VIII inhibitor and Glanzmann's thrombasthenia, as well as severalrare congenital and acquired bleeding disorders. Postoperative thrombotic events were rare, while ISTH-defined bleeding complications and readmissions were common. Most..."

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

Use of emicizumab in patients with acquired hemophilia A: An interim safety analysis of a large-scale post‑marketing surveillance study (ASH 2025) - "A causal relationship with emicizumab was ruled out in all deaths.During emicizumab treatment, 21/51 (41.2%) patients used rFVIIa, 9/51 (17.6%) had transfusions, and7/51 (13.7%) received other hemostatic agents (FXIII, tranexamic acid). In this large-scale post-marketing study, emicizumab demonstrated a favorable safetyprofile in real-world clinical practice. While the study was not specifically designed to evaluate efficacy,the consistently low incidence of bleeding events observed supports the potential role of emicizumab ineffectively preventing bleeds in patients with AHA, reinforcing its positive risk–benefit profile at thisinterim analysis."

Clinical • P4 data • Biliary Cancer • Cholangiocarcinoma • CNS Disorders • Gastroenterology • Hemophilia • Hemophilia A • Immunology • Infectious Disease • Interstitial Lung Disease • Ischemic stroke • Lung Cancer • Nephrology • Peptic Ulcer • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Solid Tumor

Lower dose emicizumab prophylaxis in children with hemophilia A: Real - world outcomes , hemostatic profiles and cost- effectiveness from a resource limited setting (ASH 2025) - "Evidence on lower dose Emicizumabin real-world pediatric cohorts is limited.Objective:To assess clinical outcomes, thromboelastography (TEG) profiles, and cost-effectiveness in childrenreceiving lower dose Emicizumab compared to prior treatment regimens.This was a hybrid observational study comprising a retrospective clinical audit and a prospectivelydesigned laboratory substudy, conducted at a single tertiary care center in India of children with severeHemophilia A who initiated lower-dose Emicizumab prophylaxis between June 2019 and May 2024.Eighteen pediatric patients were included: 15 without inhibitors (on low- to intermediate-dose clottingfactor concentrate [CFC] prophylaxis) and 3 with inhibitors (previously on FEIBA on demand).Clinical Outcomes:Annualized bleed rates (ABR) and school absenteeism (days/year) were recorded for one year pre- andpost-Emicizumab. Lower dose Emicizumab significantly reduces bleeding and school absenteeism in children withHemophilia A,..."

Clinical • Cost effectiveness • Hematological Disorders • Hemophilia • Hemophilia A • Mood Disorders • Rare Diseases

Real-world use of prophylaxis in people with non-severe hemophilia in the United States: Insights from the athn transcends Study - hemophilia natural history arm (ASH 2025) - P | "In this analysis, we demonstrate that over one third of ATHN Transcends participants withnon-severe hemophilia were already receiving continuous prophylaxis, including a significant subset withmild disease. Prior publications reported prophylaxis use in fewer than one-third of moderate and under30% of mild cases*. This shift reflects growing recognition that bleeding risk may not correlate with factorlevels and supports a phenotype-driven approach to care."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Real-world treatment patterns for Hemophilia A since the launch of emicizumab in the US (ASH 2025) - "Among PwHA receiving newer therapies in the real-world setting, switching fills betweenemicizumab and efanesoctocog alfa was infrequent (2% vs 4%). The majority of PwHA initiated andpersisted with emicizumab prophylaxis, supporting its real-world effectiveness as the standard of care."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases