Data presented at ACR Convergence 2025 highlight Sobi’s commitment to advancing care across rare and underserved inflammatory conditions

NASP data highlight reduction in disease burden among people living with uncontrolled gout

Gamifant (emapalumab) data showed response rates in patients with MAS in Still’s disease across MAS subtypes, including in those with recurrent disease

Discussion of the first ever international, randomized, multicentre, double-blind, placebo-controlled phase 2 dose finding trial of Vonjo® (pacritinib) in adult patients with VEXAS syndrome

WALTHAM, Mass., Oct. 25, 2025 (GLOBE NEWSWIRE) — Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced the presentation of data across its immunology portfolio highlighting the company’s continued leadership in addressing rare and underserved inflammatory conditions. A total of 15 scientific abstracts, including six oral presentations featuring new data from completed and ongoing studies of Nanoecapsulated Sirolimus plus Pegadricase (NASP, formerly SEL-212), Vonjo® (pacritinib), and Gamifant® (emapalumab-lzsg), are being presented at the annual American College of Rheumatology (ACR) Convergence 2025 meeting in Chicago, October 24-29, 2025.
                                                                          
“Uncontrolled gout is a distinct and severe form of gout, marked by intense pain and an increased risk of mortality. Yet, despite the urgent need, there have been no new FDA-approved treatment options in over a decade,” said Laura Saltonstall, MD, Vice President of Immunology, Medical Affairs at Sobi North America. “We’re encouraged by the new data presented for our novel, investigational therapy, NASP, in uncontrolled gout, including the observed effect on painful flares and disease burden.”

Presentations of post hoc analysis of the DISSOLVE Trials investigating NASP in uncontrolled gout

DISSOLVE I and II were Phase III replicate, double-blind, placebo-controlled studies evaluating the efficacy and safety of NASP over 24 weeks. DISSOLVE I included a blinded extension phase for an additional 24 weeks.

Patients were randomized 1:1:1 to receive high-dose (HD) NASP: sequential infusions, 0.15 mg/kg NAS and 0.2 mg/kg pegadricase; low dose (LD) NASP: sequential infusions, 0.10 mg/kg NAS and 0.2 mg/kg pegadricase or placebo every four weeks. In the DISSOLVE I extension phase, NASP was generally well tolerated with low discontinuation rates. No new safety signals were reported.

In two oral presentations, data presented from post-hoc analyses evaluated serum uric acid (sUA) levels, joint exam findings, health-related quality-of-life (HRQOL) outcomes, and gout flares in the subgroup of patients with uncontrolled gout who received six doses of NASP or placebo.

The U.S. FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 27, 2026, following the acceptance of the BLA submission on September 10, 2025.

Nanoencapsulated Sirolimus Plus Pegadricase Reduced Disease Burden in Patients With Uncontrolled Gout: Results from the Phase 3 DISSOLVE Trials (Abstract Number: 2587)

• In the NASP arms, reductions in sUA levels were noted immediately after the first dose, and mean sUA generally remained ≤2.0 mg/dL throughout the study, with the placebo arm staying generally >6 mg/dL.
• From baseline to Week 24, median sUA level reduced by 97% and 98% in HD and LD NASP, respectively, and increased by 1.5% in placebo; ~2 fold reductions in the number of tender and swollen joints were observed with HD NASP and LD NASP compared to placebo.
• NASP treatment was associated with improvements in SF-36 PCS Score and HAQ-DI Pain Score from baseline to Week 24 that were ≥ 2-fold higher than changes in patients treated with placebo.

Nanoencapsulated Sirolimus plus Pegadricase (NASP) Demonstrates a Reduction in Gout Flares: Results from the Phase 3 DISSOLVE Studies (Abstract Number: 2588)

• During the first month of treatment (weeks 1-4), the proportion of patients experiencing a gout flare was similar for NASP and placebo, 23.8%, 28.6% and 20.9% in patients receiving HD NASP, LD NASP and placebo, respectively.
• During the trial, the number of flares continued to decrease in patients treated with NASP, while an increase in the number of gout flares was observed in patients receiving placebo.
• During weeks 13–24, the average number of gout flares per patient was 2.3 and 5.7 times fewer on HD and LD, respectively, vs PBO. This translates to 12 flares in 42 patients and 4 in 35 patients in the HD NASP and LD NASP, respectively vs and 44 in 67 pts in placebo.
• Across all treatment arms, most flares ranged from mild to moderate intensity.

Additional key data from select NASP poster sessions highlighted:

Reduction in Tophi Observed in Patients with Uncontrolled Gout Treated with NASP: Results from Phase 3 DISSOLVE Studies (Abstract Number: 1998)

• Tophus outcomes from the pooled Phase III studies: Observed complete resolution of tophus at Week 24 was 31% of patients receiving HD NASP and 48% receiving LD NASP and 5% with placebo.

“We have new hope for patients suffering from gout, where there is an unmet need due to disease burden. The reduction in uric acid levels, gout flares, and size of tophi highlight the potential of NASP in optimizing the treatment of uncontrolled gout,” said Puja P Khanna, MD, MPH, Professor of Medicine, University of Michigan Medical School.

Presentations on Gamifant (emapalumab-lzsg) in hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still’s disease

• Data were pooled from two open-label, single-arm interventional studies (NI-0501-06 [NCT03311854] and NI-0501-14 [EMERALD; NCT05001737]) in 39 patients with MAS in Still’s disease who had an inadequate response to high-dose GCs.
• The primary efficacy endpoint was complete response (CR) at Week 8 (resolution of clinical signs according to investigator assessment [visual analog scale (VAS) ≤1/10 cm]) and normalization of seven MAS-related laboratory parameters. Partial response (PR) was defined as VAS <4 cm and normalization of ≥3 abnormal baseline laboratory parameters included in the composite primary endpoint. Clinical remission was defined as VAS ≤1 cm.
• The most common adverse reactions (≥10%) for Gamifant included viral infection (44%), rash (21%), anemia (18%), leukopenia (15%), thrombosis (15%), bacterial infections (13%), headache (13%), hyperglycemia (13%), infusion-related reactions (13%), abdominal pain (10%), hypertension (10%), pyrexia (10%), and thrombocytopenia (10%).
• The U.S. Food and Drug Administration approved Gamifant as the first-ever treatment for adults and children with Macrophage Activation Syndrome in Still’s disease in June 2025.

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 (Abstract Number: 1671)

• In this post hoc analysis, the authors concluded that Gamifant demonstrated efficacy across diverse MAS presentations, including in recurrent cases.
  • Across all MAS presentations, Gamifant was associated with response rates consistent with the established efficacy profile.
  • Glucocorticoid doses tapering were similar in patients across MAS presentations.

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 (Abstract Number: 0780)

• Gamifant treatment was associated with reductions in key PD markers of IFNγ activity, including CXCL9 and ferritin.
  • CXCL9 reduced by -99% and ferritin by -92% at week 8 in the subgroup of patients with complete or partial response.

Presentations on Vonjo (pacritinib) and VEXAS Syndrome
PAXIS: A Randomized, 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

• An oral presentation on the design of a randomized, double-blind, placebo-controlled, dose-finding Phase 2 study of pacritinib in patients with VEXAS syndrome.
  • PAXIS is the first randomized, double-blind, placebo-controlled pharmacotherapy trial for this complex, refractory, and sometimes fatal condition.

Development of a Consensus Definition of VEXAS Flare for Use in Clinical Research (Abstract Number: 0260)

• Data presented reviewed the development of consensus criteria to establish a standardized definition of “flare” in VEXAS for clinical research and patient care.
  • The consensus was developed by a 9-member international expert panel using modified Delphi methodology and was based on literature review and iterative consensus rounds.
  • This consensus definition provides a uniform framework for identifying disease activity and guiding future research for a disease with significant morbidity and mortality.

Development of a Disease Activity Index for the Assessment of VEXAS Syndrome (VEXAS-DAI) (Abstract Number: 0777)

• Additional data presented in a poster session highlighted the development of a new, comprehensive and standardized disease activity index (DAI) for measuring disease activity in VEXAS syndrome.
  • The VEXAS-DAI was developed based on input from 24 expert physicians worldwide to measure active inflammation in patients with VEXAS syndrome.
  • Work is ongoing to validate the VEXAS-DAI instrument in the context of a clinical trial and evaluate its potential in advancing the development of therapies in this area of high unmet medical need.

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GAMIFANT (emapalumab)

INDICATIONS

Gamifant (emapalumab-lzsg) is an interferon gamma (IFNγ)-neutralizing antibody indicated 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.
• HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.

IMPORTANT SAFETY INFORMATION

Infections

Gamifant may increase the risk of fatal and serious infections with pathogens including mycobacteria, herpes zoster virus, and histoplasma capsulatum. Do not administer Gamifant in patients with these infections until appropriate treatment has been initiated.

In patients with primary HLH receiving Gamifant in clinical trials, serious infections such as sepsis, pneumonia, bacteremia, disseminated histoplasmosis, necrotizing fasciitis, viral infections, and perforated appendicitis were observed in 32% of patients.

In patients with HLH/MAS in Still’s disease receiving Gamifant in clinical trials, serious infections such as pneumonia, cytomegalovirus infection, cytomegalovirus infection reactivation, and sepsis were observed in 13% of patients.

Evaluate patients for tuberculosis risk factors and test for latent infection prior to initiating Gamifant. Administer tuberculosis prophylaxis to patients at risk for tuberculosis or known to have a positive purified protein derivative (PPD) test result.

Consider prophylaxis for herpes zoster, Pneumocystis jirovecii, and fungal infection while receiving Gamifant. Employ surveillance testing during treatment with Gamifant.

Closely monitor patients receiving Gamifant for signs or symptoms of infection, promptly initiate a complete diagnostic workup appropriate for an immunocompromised patient, and initiate appropriate antimicrobial therapy.

Increased Risk of Infection With Use of Live Vaccines

Do not administer live or live attenuated vaccines to patients receiving Gamifant and for at least 4 weeks after the last dose of Gamifant. The safety of immunization with live vaccines during or following Gamifant therapy has not been studied.

Infusion-Related Reactions

Infusion-related reactions in patients with primary HLH, including drug eruption, pyrexia, rash, erythema, and hyperhidrosis, were reported with Gamifant treatment in 27% of patients. In one-third of these patients, the infusion-related reaction occurred during the first infusion.

Infusion-related reactions in patients with HLH/MAS in Still’s disease, including pyrexia, headache, paresthesia, bone pain, pruritic rash, and peripheral coldness, were reported with Gamifant treatment in 13% of patients. Infusion-related reactions were reported as mild in 8% of patients and as moderate in 5% of patients.

Monitor patients for infusion-related reactions, which can be severe. Interrupt the infusion for infusion reactions and institute appropriate medical management before continuing infusion at a slower rate.

Adverse Reactions

Primary HLH

Serious adverse reactions were reported in 53% of patients. The most common serious adverse reactions (≥3%) included infections, gastrointestinal hemorrhage, and multiple organ dysfunction. Fatal adverse reactions occurred in 2 (6%) of patients and included septic shock and gastrointestinal hemorrhage.

The most common adverse reactions were (≥10%) for Gamifant included infection (56%), hypertension (41%), infusion-related reactions (27%), pyrexia (24%), hypokalemia (15%), constipation (15%), rash (12%), abdominal pain (12%), CMV infection (12%), diarrhea (12%), lymphocytosis (12%), cough (12%), irritability (12%), tachycardia (12%), and tachypnea (12%).

HLH/MAS

Serious adverse reactions were reported in 12 patients (31%), with the most common serious adverse reaction being pneumonia (5%). Fatal adverse reactions occurred in two patients (5%) and included multiple organ dysfunction and circulatory shock.

The most common adverse reactions (≥10%) for Gamifant included viral infection (44%), rash (21%), anemia (18%), leukopenia (15%), thrombosis (15%), bacterial infections (13%), headache (13%), hyperglycemia (13%), infusion-related reactions (13%), abdominal pain (10%), hypertension (10%), pyrexia (10%), and thrombocytopenia (10%).

Please see Full Prescribing Information for Gamifant.

VONJO (pacritinib)

Indication
VONJO is a kinase inhibitor indicated 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 (1). 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).

Important Safety Information

Hemorrhage
Avoid use in patients with active bleeding and hold VONJO prior to any planned surgical procedures. May require dose interruption, dose reduction or permanent discontinuation depending on severity.

Diarrhea
Manage significant diarrhea with anti-diarrheals, dose reduction, or dose interruption.

Thrombocytopenia
Manage by dose reduction or interruption.

Prolonged QT Interval
Avoid use in patients with baseline QTc >480 msec. Interrupt and reduce VONJO dosage in patients who have a QTcF >500 msec. Correct hypokalemia prior to and during VONJO administration.

Major Adverse Cardiac Events (MACE)
Risk may be increased in current/past smokers and patients with other cardiovascular risk factors. Monitor for signs, evaluate and treat promptly.

Thrombosis
Including deep venous thrombosis, pulmonary embolism, and arterial thrombosis may occur. Monitor for signs, evaluate and treat promptly.

Secondary Malignancies
Lymphoma and other malignancies may occur. Past/current smokers may be at increased risk.

Risk of Infection
Delay starting VONJO until active serious infections have resolved. Observe for signs and symptoms of infection and manage promptly.

Adverse Reactions
The most common (≥20% of patients) adverse reactions are diarrhea, thrombocytopenia, nausea, anemia, and peripheral edema.

Please see Full Prescribing Information for Vonjo.

About NASP, formerly SEL-212
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
Uncontrolled gout is the most common form of inflammatory arthritis with more than 8.3 million people in the US 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 (emapalumab-lzsg) 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.