STOCKHOLM, Oct. 24, 2025 /PRNewswire/ — Sobi® (STO: SOBI), a global biopharmaceutical company dedicated to delivering innovative treatments for patients with rare disease, will be presenting new data across its immunology portfolio at the annual American College of Rheumatology (ACR) Convergence 2025 meeting in Chicago, October 24-29. Sobi is presenting a total of 15 scientific abstracts, with six oral presentations featuring new clinical data from completed and ongoing studies of NASP, pacritinib, and emapalumab-lzsg.

“The data Sobi is presenting highlight the distinct clinical benefit of emapalumab-lzsg in MAS in Still’s disease, the ongoing study of pacritinib in VEXAS Syndrome, and the potential for NASP to redefine the treatment of uncontrolled gout. The results underscore the strength of our portfolio in advancing care for complex and rare inflammatory diseases where progress is urgently needed,” said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs, and Chief Medical Officer at Sobi.

Summary of full Sobi data to be presented at ACR 2025:

NASP

Nanoencapsulated Sirolimus plus Pegadricase Reduced Disease Burden in Patients with Uncontrolled Gout: Results from the Phase 3 DISSOLVE Trials

Presenting Author:  Puja P. Khanna, MD, MPH, Division of Rheumatology, University of Michigan, Ann Arbor, MI

Oral Presentation

Session: Abstracts: Metabolic & Crystal Arthropathies – Basic & Clinical Science

Date: Tuesday 28 October

Session time: 1:00 PM – 2:30 PM CT

Presentation time: 1:30 PM – 1:45 PM CT

Nanoencapsulated Sirolimus plus Pegadricase Demonstrates a Reduction in Gout Flares: Results from the Phase 3 DISSOLVE Studies*)

Presenting Author:  Angelo Gaffo, MD, Division of Rheumatology and Clinical Immunology, University of Alabama, Birmingham, AL

Oral Presentation

Session: Abstracts: Metabolic & Crystal Arthropathies – Basic & Clinical Science

Date: Tuesday 28 October

Session time: 1:00 PM – 2:30 PM CT

Presentation time: 1:45 PM – 2:00 PM CT

Nanoencapsulated Sirolimus plus Pegadricase Demonstrates Long Term Efficacy and Safety in Patients with Uncontrolled Gout: Results from the 24-week Double-blind Extension of the Phase 3 DISSOLVE I Study

Presenting Author: Alan Kivitz, MD, Altoona Center for Clinical Research, Duncansville, PA

Poster Presentation

Session: Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster I

Date: Monday 27 October

Presentation time: 10:30 AM – 12:30 PM CT

Characterisation of Infusion Reactions Within 1 Hour of Treatment with Nanoencapsulated Sirolimus Plus Pegadricase: Pooled Results from the Phase 3 DISSOLVE I and DISSOLVE II Trials

Presenting Author: Herbert S.B. Baraf, MD, FACP, MACR, The Center for Rheumatology and Bone Research, Wheaton, MD

Poster Presentation

Session: Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster II

Date: Tuesday 28 October

Presentation time: 10:30 AM – 12:30 PM CT

Reduction in Tophi Observed in Patients with Uncontrolled Gout Treated with NASP: Results from Phase 3 DISSOLVE Studies*

Presenting Author: Herbert S.B. Baraf, MD, FACP, MACR, The Center for Rheumatology and Bone Research, Wheaton, MD

Poster Presentation

Session: Poster Session C, (1990–2014) Metabolic & Crystal Arthropathies – Basic & Clinical Science Poster II

Date: Tuesday 28 October

Presentation time: 10:30 AM – 12:30 PM CT

Patient and Caregiver Perspectives on the Burden of Disease in Uncontrolled Gout: A Cross-Sectional Survey Study

Presenting Author: Angelo Gaffo, MD, Division of Rheumatology and Clinical Immunology, University of Alabama, Birmingham, AL

Poster Presentation

Session: Patient Outcomes, Preferences, & Attitudes Poster II

Date: Monday 27 October

Presentation time: 10:30 AM – 12:30 PM CT

A Real-World Survey on Physicians’ Perspectives of Uncontrolled Gout and Gout Management Practices

Presenting Author: John Botson, MD, RPh,CCD, Orthopedic Physicians, Anchorage, AK 

Poster Presentation

Session: Health Services Research Poster III

Date: Tuesday 28 October

Presentation time: 10:30 AM – 12:30 PM CT

The Care Pathway and Treatment Patterns in Patients with Uncontrolled Gout: A Real-World Survey of Physicians in the United States

Presenting Author:  John Albert, MD, Rheumatic Disease Center, Glendale, WI

Poster Presentation

Session: Health Services Research Poster II

Date: Monday 27 October

Presentation time: 10:30 AM – 12:30 PM CT

Management of Uncontrolled Gout Among Rheumatologists: Findings from a Medical Chart Audit

Presenting Author:  Hyon Choi, MD, MPH, DrPH, Massachusetts General Hospital, Boston, MA

Poster Presentation

Session: ARP Posters I

Date: Tuesday 28 October

Presentation time: 10:30 AM – 12:30 PM CT

Gamifant (emapalumab-lzsg)

Baseline Pharmacodynamic Markers and Response to emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials

Presenting Author: Ed Behrens, MD, Chief, Division of Rheumatology, Children’s Hospital of Philadelphia

Oral Presentation

Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases I: Focus on Auto-Inflammatory Diseases

Date: Sunday 26 October

Session time: 1:00 PM – 2:30 PM CT

Presentation time: 1:45 PM – 2:00 PM CT

Emapalumab Treatment for Patients with Differing Presentations of Macrophage Activation Syndrome (MAS) Secondary to Still’s Disease: Results from a Pooled Analysis of Two Prospective Trials

Presenting Author:  Alexei Grom, MD, Research Director, Division of Rheumatology, Cincinnati Children’s Hospital

Oral Presentation

Session: Abstracts: Pediatric Rheumatology – Clinical I

Date: Monday 27 October

Session time: 1:00 PM – 2:30 PM CT

Presentation time: 1:45 PM – 2:00 PM CT

Economic Burden of Macrophage Activation Syndrome (MAS) in Patients with Still’s Disease (Systemic Juvenile Idiopathic Arthritis (sJIA) and Adult-onset Still’s Disease (AOSD): Analysis of a US National Administrative Claims Database

Presenting Author:  Michael Marrone, Director of Epidemiology and Biostatistics, Sobi

Poster Presentation

Session: Health Services Research Poster III

Date: Tuesday 28 October

Presentation Time: 10:30 AM – 12:30 PM CT

Vonjo (Pacritinib)

Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS-DAI)*

Presenting Author:  Dr. Matthew J. Koster,

Assistant Professor of Medicine, Mayo Clinic Rochester

Oral Presentation

Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases I: Focus on Auto-Inflammatory Diseases

Date: Sunday 26 October

Session time: 1:00 PM – 2:30 PM CT

Presentation time: 1:00 PM – 1:15 PM CT

PAXIS: A Randomised, Double-Blind, Placebo-Controlled, Dose Finding Phase 2 Study (Part 1) Followed by an Open-Label Period (Part 2) to Assess the Efficacy and Safety of Pacritinib in Patients with VEXAS Syndrome*

Presenting Author:  Dr. David B. Beck, MD, PhD, Division of Rheumatology, NYU Langone

Oral Presentation

Session: Abstracts: Miscellaneous Rheumatic & Inflammatory Diseases III: End Organ Focus on Heart, Lung and Eye

Date: Wednesday 29 October

Session time: 11:30 AM – 1:00 PM CT

Presentation time: 11:30 AM – 11:45 AM CT

Development of a Consensus Definition of VEXAS Flare for Use in Clinical Research*

Presenting Author: Dr. Marcela A. Ferrada, MD, Clinical Associate Professor, University of Maryland School of Medicine 

Poster Presentation

Session: Miscellaneous Rheumatic & Inflammatory Diseases Poster I

Date: Sunday 26 October

Presentation time: 10:30 AM – 12:30 PM CT

*Signifies encore data presentation

About NASP

NASP is a novel investigational medicine designed to reduce serum uric acid (sUA) levels in people living with uncontrolled gout, potentially reducing harmful tissue urate deposits which when left untreated can lead to debilitating gout flares and joint deformity. NASP is administered every 4-weeks as a sequential, two-component, infusion therapy consisting of tolerogenic nanoencapsulated sirolimus (NAS) which mitigates the formation of anti-drug antibodies (ADAs) and a uricase, pegadricase (P), which reduces serum uric acid. ADAs develop due to unwanted immune responses to biologic medicines, reducing their efficacy and tolerability, which remains an issue across multiple therapeutic modalities and disease states including uncontrolled gout.

About Uncontrolled Gout

Gout is the most common form of inflammatory arthritis with more than 8.3 million people in the United States having been diagnosed. Gout is caused by high levels of uric acid in the body that accumulate around the joints and other tissues and can result in flares that cause intense pain. Approximately 200,000 people in the United States suffer from uncontrolled gout, with serum uric acid (sUA) levels above 6 mg/dL despite treatment with oral urate lowering therapies. This leads to debilitating flares and tophi. Elevated sUA levels have also been associated with diseases of the heart, vascular system, metabolism, kidney and joints.

About Gamifant (emapalumab-lzsg)

Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralizing 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 pediatric (newborn and older) patients with primary hemophagocytic 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 pediatric (newborn and older) patients with hemophagocytic 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 macrophage activation syndrome (MAS)

Macrophage activation syndrome (MAS) is a severe complication of rheumatic diseases, most frequently in Still’s disease including systemic juvenile idiopathic arthritis (sJIA) – a rare systemic disorder of auto-inflammatory nature with common clinical manifestations such as daily spiking fever, typical transient cutaneous rash, arthritis, lymphadenopathy, hepatosplenomegaly and serositis. MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities and hyperferritinaemia, possibly progressing to multiple organ failure and death. MAS is classified as a secondary form of haemophagocytic lymphohistiocytosis (HLH).

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).

About VEXAS

VEXAS syndrome is a disease that causes inflammatory and hematologic (blood) manifestations. The syndrome is caused by mutations in the UBA1 gene of blood cells and acquired later in life. The condition is not genetically inherited.

About 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 Europe, North America, the Middle East, Asia and Australia. In 2024, revenue amounted to SEK 26 billion. Sobi’s share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn.

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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