Pharming Group (PHARM) investor relations material

Financial performance and guidance

• Achieved 2025 revenues of $376 million, up 27% from 2024, with both commercial assets delivering double-digit growth and exceeding prior guidance.

• RUCONEST generated $318 million in 2025 (26% growth), while Joenja delivered $58 million (29% growth), triggering a $5 million milestone payment to Novartis.

• 2026 revenue guidance is $405–$425 million, representing 8–13% growth, driven by Joenja and RUCONEST.

• Operating expenses for 2026 are projected at $300–$335 million, mainly due to increased R&D for leniolisib and napazimone clinical trials, with $9 million in G&A cost reductions.

• Gross margin is expected to be around 90% in 2026, with available cash and future cash flows covering all pipeline and pre-launch investments.

Commercial and pipeline strategy

• Transitioned from a single-asset to a fast-growth biotech with two commercial assets and a late-stage pipeline, each with over $1 billion sales potential.

• RUCONEST remains a cornerstone for difficult-to-treat patients, with U.S. growth offsetting ex-U.S. market exits; Joenja is at the start of its lifecycle with multiple growth catalysts, including potential pediatric label expansion.

• Conservative 2026 guidance excludes revenues from Joenja pediatric APDS until regulatory clarity is achieved.

• Expansion into larger PIDs and CVID with leniolisib could unlock a much larger market, supported by strong clinical rationale and ongoing phase II studies.

• KL1333 (napazimone) for primary mitochondrial disease is in a pivotal phase II study, having cleared futility analysis, with readout expected by end of next year.

Pipeline and clinical development

• Advancing two major programs: leniolisib for primary immunodeficiencies (PIDs) with immune dysregulation and napazimone (KL1333) for mtDNA-driven mitochondrial disease.

• Leniolisib is being developed for high-prevalence PIDs and CVID, with two Phase II trials ongoing and top-line data expected in the second half of 2026.

• Compassionate use and early data in CVID patients show reduction in pathologic B cells and stabilization of organ function, supporting the mechanistic rationale.

• KL1333 (napazimone) targets NAD+/NADH imbalance in mitochondrial disease, with the FALCON pivotal study designed with dual primary endpoints and positive interim futility analysis.

• The FALCON study is on track to complete recruitment by year-end, with regulatory alignment on endpoints and design, and readout expected in 2027.