Jivi (damoctocog alfa pegol) / Bayer - LARVOL DELTA

A New Risk-Based Scoring Approach to Individualize Prophylaxis in Patients with Hemophilia A: Results from the PREDICT Study. (PubMed, Adv Ther) - P4 | "The PREDICT risk-based scoring system-guided individualized prophylaxis with damoctocog alfa pegol, yielding improved bleeding outcomes and reduced treatment burden in most participants. These findings support the use of structured, risk-adapted approaches to personalize prophylaxis when transitioning from SHL to damoctocog alfa pegol."

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

Sixth Interim Analysis of the HEM-POWR Study: Evaluating Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Patients With Severe and Nonsevere Hemophilia A in the United States (THSNA 2026) - P | "This subgroup analysis of PTPs from the US in the HEM-POWR study continues to support the favorable effectiveness and safety profile of damoctocog alfa pegol in PTPs with severe and nonsevere hemophilia A. The findings provide insights into real-world clinical practice and can help to inform US stakeholders. Funded by Bayer. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

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

COST-MINIMIZATION ANALYSIS OF DAMOCTOCOG ALFA PEGOL VERSUS EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH SEVERE HEMOPHILIA A WITHOUT INHIBITORS IN COLOMBIA, PERU AND COSTA RICA (ISPOR 2026) - "Cost minimization analysis presented indicates that damoctocog alfa pegol is a cost-saving alternative, offering over 70% annual cost savings compared to emicizumab in Costa Rica, Colombia, and Peru"

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

HA-SAFE: Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (clinicaltrials.gov) - P=N/A | N=62 | Active, not recruiting | Sponsor: Bayer | Trial primary completion date: Jun 2028 ➔ Mar 2027

HEOR • Real-world evidence • Trial primary completion date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A (clinicaltrials.gov) - P=N/A | N=20 | Recruiting | Sponsor: Bayer | N=600 ➔ 20

Enrollment change • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Plain Language Summary on Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study. (PubMed, Eur J Haematol) - P3 | "This is a plain language summary describing results from the Alfa-PROTECT study on safety and efficacy of damoctocog alfa pegol in previously treated children aged between 7 and 12 years with severe haemophilia A. Trial Registration: The Alfa-PROTECT trial is registered at ClinicalTrials.gov: NCT05147662."

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

HEM-POWR Kids: An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A (clinicaltrials.gov) - P=N/A | N=40 | Not yet recruiting | Sponsor: Bayer | Initiation date: Dec 2025 ➔ Apr 2026

Trial initiation date • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main Study. (PubMed, Eur J Haematol) - P3 | "These data confirm the safety profile of damoctocog alfa pegol in children aged 7 to < 12 years with severe haemophilia A. Secondary endpoints indicate treatment was efficacious."

Journal • P3 data • Hematological Disorders • Hemophilia • Hemophilia A • Immunology • Rare Diseases

HEM-POWR Kids: An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A (clinicaltrials.gov) - P=N/A | N=40 | Not yet recruiting | Sponsor: Bayer | Trial primary completion date: Oct 2029 ➔ Dec 2029

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

A risk-based scoring approach to individualize prophylaxis in patients with Hemophilia A: Results from the predict study (ASH 2025) - "The Phase 4 PREDICT study evaluated whether a structured risk-based scoring system could guide personalized prophylaxis in patients transitioning from standard half-life (SHL) FVIII products to the extended half-life (EHL) therapy damoctocog alfa pegol...Significant reductions in bleeding, infusion frequency, and near-complete resolution of baseline target joints (96%) were observed. These findings support the feasibility and clinical utility of a structured risk-based algorithm to optimize EHL prophylaxis, improve outcomes, and meaningfully reduce treatment burden during SHL-to-EHL transitions."

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

A risk-based scoring approach to individualize prophylaxis in patients with Hemophilia A: Results from the predict study (ASH 2025) - "The PREDICT scoring system helped to guide individualized prophylaxis regimens with damoctocog alfapegol, leading to favorable outcomes in 77% and 71% of participants, based on treated and total ABRs,respectively. Although limited by a small sample size, these findings provide early evidence that a risk-based scoring approach, incorporating patients' clinical and intrinsic characteristics, may support HCPsand patients globally as a valuable clinical guide in personalizing their prophylaxis regimen whentransitioning from an SHL to an extended-half-life (EHL)-based regimen, like damoctocog alfa pegol."

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

First report of high-titer FVIII inhibitor development following AAV-mediated gene therapy in Hemophilia A (ASH 2025) - "Prior to enrollment, he was on damoctocog alfa pegol prophylaxis (>150 ED)...After the 3rd CS course discontinuation (D335), FVIII:Ccontinued to decrease (1.1 to 0.2 IU/mL) leading to the initiation of mycophenolate mofetil (MMF) and CSwere reintroduced...In response,MMF was increased to 1 g twice daily on D455 to support inhibitor eradication, and emicizumabprophylaxis was initiated on D460 (225 mg QW)...A positive total IgG titer was firstdetected on D219 (~4 months prior to the first positive inhibitor result), however a first IgG3 titer >positive cutoff was detected on D23. IgG1 and IgG3 were the predominant subclasses.To our knowledge this is the first reported case of high-titer FVIII inhibitor development following genetherapy in a patient with hemophilia A. Although this appears to be a rare event, potentially comparableto or even less frequent than inhibitor formation in previously treated patients receiving FVIIIprophylaxis, these results suggest that the..."

Gene therapy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Human Immunodeficiency Virus • Immune Thrombocytopenic Purpura • Infectious Disease • Rare Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Real-World Data on Hemophilia a Patients Transitioning from Bay 81-8973 to Bay 94-9027 and from Bay 14-2222 to Bay 81-8973 and then to Bay 94-9027 in the Athndataset (ASH 2023) - "Patients with Hemophilia A are transitioning more and more from standard half-life (SHL) products to extended half-life (EHL) recombinant factor VIII (rFVIII) productsObjective: To evaluate the effect of Hemophilia A patients transitioning from BAY 81-8973 (Kovaltry ®) to BAY 94-9027 (Jivi ®) and from BAY 14-2222 (Kogenate FS ®) to BAY 81-8973 and then to BAY 94-9027 in a real-world setting. The data show that hemophilia A patients who transitioned to BAY 94-9027 from BAY 81-8973, or those that transitioned from BAY 14-2222 to BAY 81-8973 and then to BAY 94-9027 did experience similar or decreased total annual bleed rates with a decrease in their prophylaxis regimen frequency when transitioning to BAY 94-9027 in the real world. The therapeutic burden of frequent infusions can be reduced when patients transition between rFVIII product classes (SHL to EHL), without change in their annual bleed rate. These data should be interpreted with caution owing to..."

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

Impact of Recombinant Factor VIII and Platelet Interaction on Platelet Functionality and Hemophilia a Treatment (ASH 2023) - "Simoctocog alfa demonstrated higher binding to activated platelets in vitro compared with efmoroctocog alfa, rurioctocog alfa pegol or damoctocog alfa pegol, resulting in an increased phenotype shift of platelets from the pro-aggregatory to the pro-coagulant state. The increased binding of simoctocog alfa was associated with a phenotypic shift in platelets as evidenced by increased exposure of PS on the platelet membranes. The binding of simoctocog alfa to platelets was disrupted when integrin αIIbβ3 activation was inhibited, suggesting a role of integrin αIIbβ3 signaling following binding of FVIII to platelets."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • ANXA5

Impact of Variable Recombinant Factor VIII Binding on Platelet Functions (ASH 2024) - "rFVIII-platelet binding : Activated platelets were incubated with simoctocog alfa (Nuwiq®), efmoroctocog alfa (Elocta®), rurioctocog alfa pegol (Adynovate®) or damoctocog alfa pegol (Jivi®). The rFVIII products explored in this study bound to platelets with varying strength, with simoctocog alfa demonstrating the highest amount of platelet binding, as well as the highest interaction with pro-aggregatory platelets. These findings indicate that variations in platelet binding may influence the efficacy of rFVIII products in the treatment of HA."

Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • ANXA5

Fourth Interim Analysis of the Hem Powr Study: A Post Hoc Analysis of Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Adolescent and Older Patients with Hemophilia a (ASH 2024) - P | "These findings can inform routine clinical use of damoctocog alfa pegol in adolescent and older patients. Funded by Bayer."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Retrospective data • Cardiovascular • Cerebral Hemorrhage • Epilepsy • Hematological Disorders • Hemophilia • Hemophilia A • Hypertension • Rare Diseases

Real-World Data on Hemophilia a Patient Usage of Bay 81-8973 and Bay 94-9027 Stratified By Body Mass Index Categories in the Athndataset (ASH 2024) - "Overall, a low number of bleeds were recorded in all BMI categories; however, the underweight and healthy weight patients numerically had the highest ABRs of all, using the highest amount of IU/kg but a smaller percentage infusing at the same frequency than the overweight and obese patients. Knowing that in vivo recovery with Factor VIII tends to be lowest in underweight patients, these data support previous findings that not only patients with high but also those with low BMIs should potentially be dosed according to different body measurements.These data should be interpreted with caution owing to limitations of real-world studies."

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Obesity • Rare Diseases • Thrombosis

Indirect Comparison of the Efficacy and Therapy-Related Costs of a Pharmacokinetic and Individualized Prophylaxis Regimen with Simoctocog Alfa Versus Other Extended-Half Life Factor VIII Concentrates (ASH 2024) - "For comparison, aggregated data was obtained from the following trials with EHL concentrates : pathfinder2 (turoctocog alfa pegol, N = 175), A-LONG (efmoroctocog alfa, N = 117), PROTECT FVIII (damoctocog alfa pegol, N = 110), PROPEL (rurioctocog alfa pegol 1–3% and 8–12%; N = 57 and 58), and XTEND-1 (efanesoctocog alfa, Group A, N = 133). Conclusion : Albeit at a generally higher weekly dose, a PK-guided, individualized prophylaxis regimen with simoctocog alfa offered comparable or significantly improved zero bleed rates and significantly lower or comparable ABRs than prophylactic regimens with EHL rFVIII concentrates. Nevertheless, the estimated annual cost of a simoctocog alfa-based regimen is 20–55% lower than with the other concentrates."

Clinical • PK/PD data • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Role of Factor VIII As a Regulator of Angiogenesis and Promoter of Endothelial Barrier Stability (ASH 2024) - "HA BOECs were treated in vitro with the rFVIII products simoctocog alfa (Nuwiq®), efmoroctocog alfa (Elocta®), rurioctocog alfa pegol (Adynovate®), damoctocog alfa pegol (Jivi®), octocog alfa (Advate®), or emicizumab (Hemlibra®). Investigating the potential extra-coagulative role of FVIII could be crucial to understanding the key molecular targets at the cellular level which impair EC function in patients with HA. Knowledge of the possible effect of different rFVIII products and non-factor therapies on EC function can be used to optimize therapeutic approaches, which in turn may result in safer and more efficient treatment of HA."

Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Osteoarthritis • Rare Diseases

Preventing Bleeds in Pediatric Patients With Hemophilia A: Which Factor Replacement Therapy Offers the Best Protection and at What Cost? (ISPOR-EU 2025) - "Second lowest number of bleeds was achieved with efanesoctocog alfa (Altuviiio/Altuvoct) with 3.90 bleeds, following Efmoroctocog alfa (Elocta), turoctocog alfa pegol (Esperoct), turoctocog alfa (NovoEight), simoctocog alfa (Nuwiq), octocog alfa (Kovaltry), Afstyla (lonoctocog alfa) and octocog alfa (Advate) with 9.80, 9.85, 9.85, 11.85, 16.85, 18.45 and 18.75 bleeds, respectively. Prevention of bleeds is of utmost importance when treating pediatric patients with hemophilia A. Choosing a treatment with the lowest possible bleeding rates can support the physical development of the patient. This analysis showed that the lowest number of bleeds and lowest costs are estimated to be reached by damoctocog alfa pegol (Jivi)."

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

Changes in Physical Activity of Pediatric Patients Treated With Damoctocog Alfa Pegol During the Alfa-PROTECT Clinical Trial (ISPOR-EU 2025) - "Patients reported improvements in their overall physical and emotional health after initiating treatment with damoctocog alfa pegol. Intensity and frequency of sport activities increased as well, suggesting confidence in the bleed-prevention of the treatment."

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

Real-World Effectiveness and Safety of Damoctocog Alfa Pegol in Severe and Nonsevere Patients With Hemophilia A From the Prospective, Multinational, Ongoing HEM-POWR Study. (PubMed, Eur J Haematol) - P | "This updated analysis further demonstrated the effectiveness, acceptable safety profile, and tolerability of damoctocog alfa pegol for previously treated patients with severe and nonsevere hemophilia A from adolescence to older age in a real-world setting."

Journal • Real-world evidence • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

Canadian Clinical Experience on Switching From Standard Half-life Recombinant Factor VIII (rFVIII), Octocog Alfa, to Extended Half-life rFVIII, Damoctocog Alfa Pegol, in Persons With Haemophilia A ≥ 12 Years Followed in a Comprehensive Haemophilia Care Program in Canada. (PubMed, Haemophilia) - "This is a plain language summary on switching from the medicine octocog alfa to a new medicine damoctocog alfa pegol (BAY 94-9027, Jivi) for the treatment of haemophilia A in Canada."

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

A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A (clinicaltrials.gov) - P=N/A | N=600 | Recruiting | Sponsor: Bayer | Trial completion date: Jun 2026 ➔ May 2028 | Trial primary completion date: Mar 2026 ➔ Mar 2028

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

HEM-POWR Kids: An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A (clinicaltrials.gov) - P=N/A | N=40 | Not yet recruiting | Sponsor: Bayer | Trial primary completion date: Jan 2030 ➔ Oct 2029

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