Imagine a world where rare blood disorders are no longer a life sentence. This is the bold vision driving Sobi, a global biopharma company, as they prepare to unveil groundbreaking advancements at the 65th American Society of Hematology (ASH) Annual Meeting in Orlando, Florida, from December 6th to 9th, 2025. But here's where it gets exciting: Sobi isn't just presenting data; they're showcasing a commitment to transforming lives through 19 scientific abstracts, including two highly anticipated oral presentations.
These presentations delve into the latest clinical data and insights from ongoing and completed studies across Sobi's innovative portfolio. Think efanesoctocog alfa, pegcetacoplan, avatrombopag, emapalumab, and pacritinib – therapies targeting rare and severe blood disorders like hemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), and myelofibrosis.
And this is the part most people miss: these aren't just scientific findings; they represent potential life-changing breakthroughs for patients grappling with daily challenges and limited treatment options.
Dr. Lydia Abad-Franch, Sobi's Head of R&D and Medical Affairs, and Chief Medical Officer, highlights the significance of their pacritinib research: “Our post-hoc analysis reveals pacritinib's ability to shrink or stabilize enlarged spleens, improve blood cell counts, and alleviate myelofibrosis symptoms in high-risk patients. These findings are crucial because they target the root causes of suffering, impacting daily life and overall quality of life.”
The ASH meeting also serves as a platform for Sobi to share the latest research on other promising therapies like emapalumab, efanesoctocog alfa, pegcetacoplan, avatrombopag, and loncastuximab tesirine.
Diving Deeper into the Science:
Sobi's presentations at ASH 2025 cover a wide spectrum of hematological conditions and treatment approaches. Here's a glimpse into the key highlights:
Efanesoctocog Alfa (ALTUVOCT®):
Long-term efficacy: Data from the XTEND-ed study will showcase the sustained benefits of once-weekly efanesoctocog alfa prophylaxis in adults, adolescents, and children with severe hemophilia A, up to four years.
Real-world impact: Several poster presentations will explore the real-world experience of efanesoctocog alfa in the US, including treatment patterns, bleeding outcomes, and patient-reported quality of life improvements.
Pegcetacoplan (Aspaveli®/Empaveli®):
Broadening treatment horizons: Studies will present data on the consistent benefits of pegcetacoplan in PNH patients with and without a history of aplastic anemia, as well as real-world treatment outcomes across different regions.
Expanding applications: Research will also delve into the potential of pegcetacoplan in C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
Avatrombopag (Doptelet®):
Treatment switching: The real-AVA 3.5 study will shed light on treatment patterns and outcomes for ITP patients switching from eltrombopag or romiplostim to avatrombopag.
Safety and efficacy: A systematic review and meta-analysis will evaluate the real-world safety and efficacy of avatrombopag in adults with ITP.
Emapalumab (Gamifant®):
- Rapid and sustained benefits: Pooled analysis of clinical trials will demonstrate the rapid and sustained benefits of emapalumab in various subgroups of patients with primary hemophagocytic lymphohistiocytosis (HLH), including those with central nervous system involvement.
Pacritinib (Vonjo®):
Real-world insights: Studies will explore real-world treatment patterns and outcomes in myelofibrosis patients treated with pacritinib, including those with thrombocytopenia and anemia at treatment initiation.
Expanding treatment options: Research will also investigate the use of pacritinib in patients with advanced proliferative chronic myelomonocytic leukemia (CMML) and those switching from ruxolitinib.
Beyond the Data: A Collaborative Effort
Sobi's success in developing these innovative therapies is fueled by strategic collaborations. Their partnerships with Sanofi for efanesoctocog alfa and Apellis for pegcetacoplan exemplify the power of combined expertise in bringing life-changing treatments to patients worldwide.
But the question remains: How will these advancements translate into tangible improvements in the lives of patients with rare blood disorders? The answers, we hope, will emerge from the insightful discussions and collaborations fostered at ASH 2025.
About Sobi:
Sobi is a global biopharma company dedicated to unlocking the potential of breakthrough innovations for people living with rare diseases. With a workforce spanning Europe, North America, the Middle East, Asia, and Australia, Sobi is committed to transforming lives through its innovative therapies.
For more information, visit: https://www.sobi.com/en
Contact:
- Investor Relations: https://www.sobi.com/en/investors
- Media Relations: https://www.sobi.com/en/media
