Swedish Orphan Biovitrum (Sobi) will present new clinical data and research outcomes at the annual European Congress of Rheumatology (EULAR 2025) in Barcelona from June 11-14, 2025. The presentations will include clinical trial outcomes on the efficacy and safety of Gamifant in treating macrophage activation syndrome, updates on Vonjo's potential treatment of VEXAS syndrome, and analysis on uncontrolled gout management.
EULAR 2025 Symposium and Presentations
Sobi will host a symposium focusing on the dermatologic, rheumatologic, and hematologic features of VEXAS syndrome during the congress. The symposium will be chaired by Dr. Sophie Georgin-Lavialle MD, PhD from the French National Reference Centre for auto-inflammatory diseases and inflammatory amyloidosis, followed by a panel discussion and Q&A session.
"We are delighted that our continued advancements in treating rare disease, including those suffering with the most debilitating rheumatology conditions will be presented at this year's EULAR conference," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. "Sobi's presentations will provide insights and treatment options for those working with patients with rheumatological conditions, providing them with the most up-to-date clinical data and approaches."
Gamifant for Macrophage Activation Syndrome
Gamifant (emapalumab) is currently indicated for treating adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. The drug is an anti-interferon-gamma antibody that demonstrated efficacy and safety in a phase 2 pilot study in patients with macrophage activation syndrome (MAS) in Still's disease who had inadequate response to high-dose glucocorticoids.
MAS represents a potentially life-threatening complication of Still's disease characterized by interferon-gamma-driven systemic hyperinflammation. More than one-third of patients inadequately respond to high-dose glucocorticoids, highlighting the significant unmet medical need in this patient population.
VEXAS Syndrome and Vonjo Investigation
VEXAS syndrome is a disease causing inflammatory and hematologic manifestations, caused by mutations in the UBA1 gene of blood cells and acquired later in life. The condition is not genetically inherited. Vonjo (pacritinib), a kinase inhibitor currently indicated in the US for treating adults with intermediate or high-risk primary or secondary myelofibrosis with platelet counts below 50 × 10⁹/L, is being investigated as a potential treatment for VEXAS syndrome.
Novel Gout Treatment Approach
The presentations will also include analysis on NASP (formerly SEL-212), a novel investigational medicine designed to reduce serum urate levels in people with uncontrolled gout. NASP is administered every four weeks as a sequential, two-component infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) and pegadricase (P). The therapy aims to mitigate anti-drug antibody formation while reducing serum uric acid, potentially reducing harmful tissue urate deposits that can lead to debilitating gout flares and joint deformity.
Anti-drug antibodies develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability across multiple therapeutic modalities and disease states including uncontrolled gout.
Company Overview
Sobi is a global biopharma company focused on unlocking breakthrough innovations for people living with rare diseases. The company has approximately 1,900 employees across Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion, with shares listed on Nasdaq Stockholm.
