Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025
"We will present evidence at ASH from a post hoc analysis demonstrating that pacritinib reduces or stabilises spleen size, improves haematologic parameters, and lessens myelofibrosis symptoms in patients with highrisk disease. These findings matter because they address key drivers of morbidity which affect daytoday functioning and quality of life. The ASH meeting also offers an opportunity to discuss the latest research on emapalumab, efanesoctocog alfa, pegcetacoplan, avatrombopag, as well as loncastuximab tesirine," said
Summary of full Sobi data to be presented at ASH 2025:
Overview of treatment advances with complement Inhibitors in patients with Paroxysmal Nocturnal Haemoglobinuria.
Optimising PNH treatment with the complement inhibitor Pegcetacoplan: A case report.
User experience with Pegcetacoplan on-body Injector in patients with Paroxysmal Nocturnal Hemoglobinuria.
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Efanesoctocog alfa |
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Clinical outcomes up to four years of once-weekly Efanesoctocog Alfa Prophylaxis in previously treated adults, adolescents, and children with severe Haemophilia A: Interim analysis of the Phase 3 XTEND-ed long-term extension study. |
Oral Presentation
Session
Date:
Time:
Presentation Time:
Room: OCCC – W304EFGH Publication Number: 539 |
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Understanding unmet needs for people with Haemophilia A receiving factor and non-factor treatments. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 Publication Number: 2679 |
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Real-world experience of Efanesoctocog Alfa in Haemophilia A patients in the US: A retrospective analysis. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 Publication Number: 1286 |
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Patient characteristics, treatment patterns, and bleeding in people with Haemophilia A without inhibitors initiating Efanesoctocog alfa in the US: An administrative claims analysis. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 Publication Number: 1290 |
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Quality of life and functional improvements with Efanesoctocog alfa in patients with moderate to severe Haemophilia A: A real-world survey. |
Poster Presentation Session name: Poster Session III
Date:
Time:
Room: OCCC - West Halls B3-B4 Publication Number: 4846 |
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Pegcetacoplan |
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Consistent benefits of Pegcetacoplan treatment in PNH patients with and without a history of Aplastic Anaemia in real world: Analysis of the ongoing COMPLETE Phase 4 observational study. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Early results from the ongoing Pegcetacoplan Silo of the International Paroxysmal Nocturnal Haemoglobinuria Interest Group Registry. |
Poster Presentation
Session
Date:
Time:
Location: OCCC - West Halls B3-B4 |
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Real-world clinical characteristics and treatment outcomes in PNH patients prescribed Pegcetacoplan: Insights of complement inhibitor-experienced and -naïve patients across |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Pegcetacoplan - Publication Only Abstracts |
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Real-world effectiveness of Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria: A systematic review of clinical and patient-reported outcomes. |
Publication only - published online on |
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Low risk for Meningococcal and encapsulated bacteria infections with systemically administered Pegcetacoplan in Paroxysmal Nocturnal Haemoglobinuria and C3 Glomerulopathies. |
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Real-world treatment patterns and outcomes among patients with immune thrombocytopenia (ITP) who switched treatment from Eltrombopag or Romiplostim to Avatrombopag in |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Real-world safety and efficacy of Avatrombopag in adults with Immune Thrombocytopenia: A systematic review and meta-analysis. |
Global Abstract
Session
Date:
Time:
Room: OCCC – West Halls B3-B4 |
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Patient-reported outcomes of Avatrombopag for Chronic Immune Thrombocytopenia: Interim analysis of the Phase 4 ADOPT Study. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Emapalumab |
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Use of Emapalumab is associated with rapid and sustained benefits in pHLH subgroups, including CNS involvement and previously untreated patients: Pooled analysis of prospective trials NI-0501-04, NI-050105 and NI-050109. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Emapalumab induces rapid, durable responses and reliable bridging to curative HSCT in patients with primary HLH: Pooled analysis of prospective trials NI-0501-04, NI-0501-05 and NI-0501-09. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Pacritinib |
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Real-world treatment patterns and outcomes in patients with myelofibrosis who presented with thrombocytopenia and anaemia at initiation of Pacritinib treatment. |
Oral Presentation
Session Malignancies: Real-World Experiences
Session date:
Session time:
Presentation time:
Room: OCCC - W414CD |
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Pacritinib in patients with high-risk myelofibrosis: Outcomes from post-hoc analyses of two Phase 3 studies. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Real-world treatment patterns and clinical outcomes in patients with Myelofibrosis treated with Pacritinib (PAC): Results from the MY-PAC Study. |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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PROSPERA (ABNL-MARRO 002): A randomised Phase 2 study of Pacritinib vs. Hydroxyurea in patients with Advanced Proliferative Chronic Myelomonocytic Leukaemia (CMML) |
Poster Presentation
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Treatment patterns and outcomes in patients with myelofibrosis treated with Pacritinib following a switch from Ruxolitinib: The MY-PAC Study. |
Session
Date:
Time:
Room: OCCC - West Halls B3-B4 |
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Incidence, prevalence, and clinical outcomes of Myelofibrosis with and without Cytopenia in |
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About ALTUVOCT ® (efanesoctocog alfa)
ALTUVOCT® (efanesoctocog alfa) is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (HA). ALTUVOCT can be used for all age groups and any disease severity.
About the Sobi and Sanofi Collaboration
Sobi and Sanofi collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially
About Aspaveli®/Empaveli® (pegcetacoplan)
Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b inhibitor designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the US,
About the Sobi and Apellis Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-
About Doptelet® (avatrombopag)
Doptelet (avatrombopag) is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments, and for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) scheduled to undergo an invasive procedure. Doptelet is also approved for the treatment of chronic ITP in pediatric patients.
About Gamifant® (emapalumab-lzsg)
Gamifant is an anti-IFNγ antibody that binds free and receptor-bound IFNγ, which when secreted in an uncontrolled manner can cause hyperinflammation. Gamifant is indicated for intravenous infusion over one hour and is FDA approved for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) in Still's disease.
About Vonjo® (pacritinib)
Vonjo (pacritinib) is a kinase inhibitor indicated in
Sobi®
Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 1,900 employees across
Contacts
For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here.
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Sobi to Showcase Scientific advances and Commitment to Haematology at ASH 2025 |
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