Sobi will present new data from its hematology portfolio at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego, CA, from December 7-10, 2024. The presentations will cover a range of rare blood disorders, including hemophilia A, hemophagocytic lymphohistiocytosis (HLH), myelofibrosis, paroxysmal nocturnal hemoglobinuria (PNH), and immune thrombocytopenia (ITP).
Advancing Hemophilia A Treatment with ALTUVOCT
Three oral presentations will feature new data on ALTUVOCT® (efanesoctocog alfa) prophylaxis in hemophilia A. According to Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi, these presentations will reveal important new data about long-term outcomes with ALTUVOCT. Key presentations include:
- Association Between Hemophilia Joint Health Score and Quality of Life Using Results from the XTEND-1 Efanesoctocog Alfa Phase 3 Trial
- Clinical Outcomes Over 3 Years of Once-Weekly Efanesoctocog Alfa Treatment in Adults and Adolescents with Severe Hemophilia A: Second Interim Analysis from the Phase 3 XTEND-ed Long-Term Extension Study
- Real-world Experience of Switching to Prophylactic Efanesoctocog Alfa in Patients with Moderate or Severe Hemophilia A: An Analysis of the Adelphi Hemophilia Disease Specific Programme™
A poster presentation will also cover the cost comparison of efanesoctocog alfa with existing Factor VIII replacement therapies for major surgeries in people with severe hemophilia A.
Real-World Evidence for Doptelet in Immune Thrombocytopenia
Sobi will also present an oral presentation on the real-world effectiveness of Doptelet® (avatrombopag) in treating people with ITP. Additional presentations on Doptelet include:
- Use of Avatrombopag in Patients with Immune Thrombocytopenia: Interim Analysis of the Phase 4 ADOPT Study
- Analysis of Durability of Response to Avatrombopag (AVA) from a Phase 3b Multicenter, Randomized, Double-Blind, Placebo (PBO)-controlled, Parallel-group Trial to Evaluate the Efficacy and Safety of AVA for the Treatment of Pediatric Patients with Immune Thrombocytopenia
- Enhanced Patient Satisfaction and Stability of Platelet Counts Following a Switch from Eltrombopag (ELT) or Romiplostim (ROMI) to Avatrombopag (AVA) In Adult Idiopathic Thrombocytopenic Purpura (ITP): Post-hoc Analyses from a Prospective Phase 4 Study
- Results of a Prospective, Open-label, Phase 4 Study Evaluating the Safety, Efficacy, and Treatment Satisfaction in Adult Immune Thrombocytopenia (ITP) Subjects after Switching to Avatrombopag (AVA) from Eltrombopag (ELT) or Romiplostim (ROMI)
- Real-World Treatment Patterns and Outcomes in Patients with Primary Immune Thrombocytopenia Treated with Avatrombopag in the United States: REAL-AVA 2.0 Interim
Advancing Treatment for HLH and Myelofibrosis
Data on Gamifant® (emapalumab) in hemophagocytic lymphohistiocytosis (HLH) and Vonjo® (pacritinib) in myelofibrosis will also be presented. Presentations include:
- Transplant Related Outcomes in Patients with Hemophagocytic Lymphohistiocytosis Treated with Emapalumab as a Bridge to Hematopoietic Stem Cell Transplantation: The Real-HLH Study
- Patients (pts) with Optimized Hemophagocytic Lymphohistiocytosis (HLH) Inflammatory (OHI) Index-Confirmed Diagnosis of Malignancy-Associated HLH (mHLH) and Emapalumab Treatment
- Hematologic Improvement Experienced by Pacritinib-Treated Patients with Myelofibrosis in Real-World Clinical Settings
- Improvement in Serum Albumin as a Measure of Improved Metabolic Profile in Pacritinib-Treated Patients: a Retrospective Analysis of Patients Treated Across Three Clinical Trials
Real-World Outcomes in Paroxysmal Nocturnal Hemoglobinuria
Sobi will present real-world clinical outcomes for Aspaveli®/Empaveli® (pegcetacoplan) in paroxysmal nocturnal hemoglobinuria (PNH):
- Real-World Clinical Outcomes for Complement inhibitor Experienced and Naïve Paroxysmal NocturnalHemoglobinuria Patients Prescribed Pegcetacoplan in Europe and Canada
These presentations highlight Sobi's commitment to advancing innovative treatments for people with rare hematological diseases.
