Swedish Orphan Biovitrum (SOBI) investor relations material

Unmet medical need and market opportunity

• Uncontrolled gout is a high-impact condition causing severe pain, complications, and increased mortality, affecting a smaller but severely burdened patient subset, with global prevalence rising and higher rates in males and older adults.

• Systemic inflammation from uncontrolled gout increases risks for cardiovascular disease, diabetes, and chronic kidney disease.

• Chronic flares disrupt daily life, impair quality of life, and negatively impact mental health.

• The current market for uricase therapy exceeds $1 billion, but less than 5% of eligible patients receive it, with only about 15,000 U.S. patients as core candidates and less than half currently treated.

• Barriers to uricase use include safety concerns, need for systemic immunosuppression, frequent dosing, logistical challenges, and insurance hurdles, many of which are addressable.

Clinical data and product profile

• NASP is a monthly, sequential infusion therapy using nanoencapsulated sirolimus plus pegadricase, designed to reduce immunogenicity and avoid broad immunosuppression.

• Sirolimus induces targeted immune tolerance, while pegadricase extends drug half-life and reduces immune response.

• Phase 3 pooled data show 51% (high dose) and 43% (low dose) of patients achieved the primary endpoint vs. 8% for placebo.

• NASP led to rapid, sustained uric acid reduction (94–95% from baseline), high rates of tophi resolution (49–70% vs. 5% for placebo), and 94–95% of patients were flare-free in the final treatment period.

• NASP was well tolerated, with mostly mild to moderate adverse events, low infusion reaction rates, and no major renal, cardiovascular, or hepatic safety signals; in CKD Stage 3 patients, 52–61% achieved sUA response with stable or improved kidney function.

Strategic roadmap and commercialization

• Regulatory submission for NASP is complete, with FDA acceptance and a PDUFA date set for June 2026; Biologics License Application was submitted June 2025.

• Focus areas include highlighting unmet need, strengthening clinical value messaging, addressing financial/logistical barriers, and optimizing patient/provider experience.

• Ongoing data generation and upcoming presentations (e.g., ACR Congress) will support launch readiness.

• Market expansion is being evaluated in Japan, Brazil, Middle East, Canada, and potentially Europe, though U.S. commercialization is the primary driver.

• Lifecycle management and international market expansion plans are underway, positioning NASP as a key strategic growth driver.