Sobi's presentations highlight research across multiple rare diseases in haematology including PNH, ITP, HLH and myelofibrosis.
STOCKHOLM, June 11, 2026 /PRNewswire/ -- Sobi (STO: SOBI) today announced that new clinical and real-world data will be presented at the 2026 Congress of the European Hematology Association (EHA2026), taking place June 11-14 in Stockholm, Sweden. The presentations include data across paroxysmal nocturnal haemoglobinuria (PNH), immune thrombocytopenia (ITP), primary haemophagocytic lymphohistiocytosis (HLH), and myelofibrosis.
The data include real-world evidence and post hoc analyses for Aspaveli (pegcetacoplan), Doptelet (avatrombopag), Gamifant (emapalumab), and Vonjo (pacritinib).
"As EHA comes to Stockholm this year, we are pleased to present new clinical and real-world evidence spanning multiple rare diseases," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. "These presentations reflect the breadth of our ongoing research across PNH, ITP, HLH and myelofibrosis, and continue to build understanding of treatment outcomes in areas of high unmet need."
Oral presentation | ||
Doptelet (avatrombopag) | Impact of protocol mandated dose holds on platelet response durability in pediatric ITP treated with avatrombopag: AVA 301 post hoc analysis | Session s429 June 12 |
Poster presentations | ||
Aspaveli (pegcetacoplan) | Sustained long-term real-world effectiveness and safety of pegcetacoplan in patients with PNH: COMPLETE Phase 4 study | Session 2 |
Aspaveli (pegcetacoplan) | Real-world hemoglobin concentrations and transfusion burden in patients with paroxysmal nocturnal hemoglobinuria receiving complement inhibitors: A US retrospective claims data analysis | Session 2 18:45 - 19:45 CEST |
Doptelet (avatrombopag) | Avatrombopag in TPO RA-naive adults in acute, persistent, and chronic phases of ITP: results from the REAL AVA 3.0 retrospective study | Session 2 |
Vonjo (pacritinib) | Consistency of response by baseline platelet count in pacritinib-treated patients in the real world: MY-PAC Analysis | Session 2 |
Publication-only abstracts | |
Aspaveli (pegcetacoplan) | Incidence rates of serious infections in patients with Paroxysmal Nocturnal Hemoglobinuria: A Real-World Comparison using data from TriNetX Across the US, Europe, and Other Regions |
Aspaveli (pegcetacoplan) | Case reports of pegcetacoplan treatment in paroxysmal nocturnal hemoglobinuria (PNH) - a systematic review |
Aspaveli (pegcetacoplan) | Real-world physicians and patients perspectives on adherence, satisfaction with pegcetacoplan, and health related quality of life across europe, the United States and Canada: A cross-sectional study |
Gamifant (emapalumab) | Emapalumab in treatment-experienced primary HLH: Safety, efficacy, and transplant outcomes from the open-label, single-arm post-authorization study Sobi.EMAPALUMAB-104 in Chinese patients |
Vonjo (pacritinib) | Real-world treatment patterns and outcomes in patients with myelofibrosis treated first-line with pacritinib or ruxolitinib |
About Aspaveli/Empaveli (pegcetacoplan)
Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy 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 United States, European Union, and other countries globally.
It is the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally.
About the Sobi and Apellis (now part of Biogen) collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
About Doptelet (avatrombopag)
Doptelet is an orally administered thrombopoietin receptor agonist (TPO-RA) that increases platelet count. It is approved in the United States, European Union and Japan for thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, and for ITP in adults according to local labeling. In the United States, Doptelet is also approved for pediatric patients 1 year and older with persistent or chronic ITP who have had an insufficient response to a previous treatment.
About Gamifant (emapalumab)
Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralising interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour.
Gamifant is approved in the US for the treatment of adult and paediatric patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and paediatric patients with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.
About Vonjo (pacritinib)
Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
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 2 000 employees across Europe, North America, the Middle East, Asia and Australia. In 2025, revenue amounted to SEK 28 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.
Contacts
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Visiting address
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+46 8 697 20 00
info@sobi.com
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