Pharming Group (PHARM) investor relations material

Strategic Vision and Leadership

• The company aims to become a global rare disease leader, leveraging a 5X, 10X growth mindset, efficient clinical development, and scalable infrastructure.

• Focus on efficient capital allocation, ongoing M&A, and business development to expand and de-risk the pipeline.

Portfolio and Commercial Performance

• The commercial portfolio includes RUCONEST® for HAE and Joenja® (leniolisib) for APDS, both driving significant near-term catalysts and expansion opportunities.

• RUCONEST® remains a durable cash engine for difficult-to-treat HAE patients, with stable demand, new prescriber additions, and continued patient enrollments despite new competitors and inventory drawdowns.

• Joenja® is the primary growth driver, showing strong double-digit growth in APDS, with 127 U.S. patients on therapy (+25% YoY), and expansion through new indications, pediatric approvals, and launches in the U.K., Japan, and Europe.

Pipeline and Clinical Development

• Pipeline assets include leniolisib for PIDs/CVID (Phase II) and napazimone (KL1333) for mtDNA mitochondrial disease, both with over $1B revenue potential.

• Leniolisib is being studied in higher prevalence PIDs/CVID, with Phase II studies fully enrolled and topline data expected in H2 2026.

• Napazimone targets primary mitochondrial diseases, with positive interim clinical data, validated endpoints, and pivotal trial enrollment completion expected by year-end; full readout anticipated in 2027.