On November 20, 2023 Sobi reported that it will present new data at the 65th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Diego from the 9th to the 12th of December 2023 (Press release, Swedish Orphan Biovitrum, NOV 20, 2023, View Source [SID1234637877]). During the meeting several analyses will be presented in patients with haemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), relapsed or refractory diffuse large b-cell lymphoma, myelofibrosis, and haemophagocytic lymphohistiocytosis.

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"We take pride in our expanded commitment to individuals affected by rare diseases, which we are pleased to highlight at this year’s ASH (Free ASH Whitepaper) meeting, with research that spans a range of rare and debilitating conditions. We are continuing our strong legacy of elevating standards of care for haemophilia A with two oral presentations. Additionally, we mark our entry into the myelofibrosis field with several poster presentations," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "We look forward to collaborating and connecting in person at this year’s meeting."

Key data to be presented at ASH (Free ASH Whitepaper) 2023

Haemophilia A

Efanesoctocog Alfa

Once-Weekly Efanesoctocog Alfa Prophylaxis Provided High Sustained Factor VIII Activity Levels, Independent of Blood Group, in Children <12 Years of Age with Severe Hemophilia A

Oral presentation.

#506

Session: 322

Sunday, December 10, 2023, 12:00 PM – 1:30 PM

Presentation Time: 12:15 PM

Experiences with Efanesoctocog Alfa: Exit Interviews with Caregivers of Previously Treated Patients with Hemophilia A from the XTEND-Kids Phase 3 Clinical Trial

Oral presentation.

#507

Session: 322

Sunday, December 10, 2023, 12:00 PM – 1:30 PM
Presentation Time: 12:30 PM

Experience with Accelerometer Activity Tracking in Patients with Hemophilia A: Results from the XTEND-1 Efanesoctocog Alfa Phase 3 Trial

Poster presentation.

#2360

Session: 904

Saturday, December 9, 2023, 5:30 PM – 7:30 PM

Treatment of Bleeding Episodes with Efanesoctocog Alfa in Children with Severe Hemophilia A in the XTEND-Kids Phase 3 Study

Poster presentation.

#3993

Session: 322

Monday, December 11, 2023, 6:00 PM – 8:00 PM

Haemophilia A

Real-World Unmet Needs in Patients with Hemophilia A Without Inhibitors in the US: Results from the Picnic Health Database

Poster presentation.

#2380

Session: 904

Saturday, December 9, 2023, 5:30 PM – 7:30 PM

Investigating the Relationship between Endogenous Factor VIII Levels and Annual Bleed Rates and Health-Related Quality of Life in Patients with Hemophilia A Not Treated with Factor VIII Prophylaxis

Poster presentation.

#5125

Session: 904

Monday, December 11, 2023, 6:00 PM – 8:00 PM

Paroxysmal Nocturnal Hemoglobinuria

Empaveli (pegcetacoplan)

Efficacy and Safety Is Maintained in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan for up to 3 Years

Oral presentation.

#574

Session: 508
Sunday, December 10, 2023, 4:30 PM – 6:00 PM
Presentation Time: 5:15 PM

Immune Thrombocytopenia

Doptelet (avatrombopag)

Interim Baseline Characteristics of Adult Patients with Immune Thrombocytopenia Enrolled in the Observational Multicenter Phase 4 ADOPT Study to Evaluate the Use and Effectiveness of Avatrombopag

Poster presentation.

#1226

Session: 311

Saturday, December 9, 2023, 5:30 PM – 7:30 PM

Interim Analysis of Platelet Response in a Prospective Phase 4 Study in Adult Immune Thrombocytopenia (ITP) Subjects after Switching from Eltrombopag (ELT) or Romiplostim (ROM) to Avatrombopag (AVA)

Poster presentation. #2577

Session: 311, Sunday, 10 December 2023, 6:00 PM – 8:00 PM

Interim Analysis of Treatment satisfaction from a Prospective Phase 4 Study in Adult Immune Thrombocytopenia (ITP) Subjects after Switching from Eltrombopag or Romiplostim to Avatrombopag

Poster presentation.

#3954

Session: 311

Monday, 11 December 2023, 6:00 PM – 8:00 PM

Real-World Treatment Patterns and Outcomes in Patients with Immune Thrombocytopenia Treated with Avatrombopag in the United States: REAL-AVA 2.0 Study Design

Poster presentation.

#5127

Session: 904

Monday, December 11, 2023, 6:00 PM – 8:00 PM

Relapsed/Refractory Diffuse Large B-Cell Lymphoma

Zynlonta (loncastuximab tesirine)

Early and Sustained Circulating Tumor DNA Response Dynamics after Loncastuximab Tesirine for Relapsed/Refractory Diffuse Large B-Cell Lymphoma

Poster presentation.

#3133

Session: 627

Sunday, December 10, 2023, 6:00 PM – 8:00 PM

Myelofibrosis

Vonjo (pacritinib)

Impact of Symptom Benefit and Transfusion Response on Survival in Myelofibrosis Patients Treated with Pacritinib: PERSIST-2 Landmark Survival Analysis

Poster presentation.

#3207

Session: 634

Sunday, December 10, 2023, 6:00 PM – 8:00 PM

Platelet Response in Pacritinib-Treated Patients with Cytopenic Myelofibrosis: a Retrospective Analysis of PERSIST-2 and PAC203 Studies

Poster presentation.

#4554

Session: 634

Monday, 11 December 2023, 6:00 PM -8:00 PM

Retrospective Analysis of the Relationship between Transfusion Independence and Bone Marrow Fibrosis Reduction in Patients with Myelofibrosis Treated with Pacritinib Versus Ruxolitinib

Poster presentation.

#4566

Session: 634

Monday, 11 December 2023, 6:00 PM -8:00 PM

An Analysis of Ruxolitinib Dosing for Myelofibrosis in Real-World Practice

Poster presentation.

#5186

Session: 906

Monday, December 11, 2023, 6:00 PM – 8:00 PM

Haemophagocytic Lymphohistiocytosis

Gamifant (emapalumab)

Emapalumab, a Fully Human Anti-Interferon Gamma Monoclonal Antibody, in Pediatric Patients With Primary Hemophagocytic Lymphohistiocytosis: Long-Term Follow-up of a Phase 2/3 Study

Poster presentation.

#1174

Session: 203

Saturday, December 9, 2023, 5:30 PM – 7:30 PM

Real-World Treatment Patterns and Outcomes Among Patients with Primary Hemophagocytic Lymphohistiocytosis with and without Infection at Diagnosis and Treated with Emapalumab: The REAL-HLH Study

Poster presentation.

#2534

Session: 201

Sunday, December 10, 2023, 6:00 PM – 8:00 PM

Real-World Treatment Patterns and Outcomes Among Patients with Secondary Hemophagocytic Lymphohistiocytosis Treated with Emapalumab in the United States: The REAL-HLH Study

Poster presentation.

#3909

Session: 201

Monday, December 11, 2023, 6:00 PM – 8:00 PM

About efanesoctocog alfa
Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for a significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023.