Pharming Group (PHARM) Investor presentation summary

Commercial foundation and product portfolio

• RUCONEST remains a cornerstone therapy for acute hereditary angioedema (HAE), showing durable cash flow and resilience despite competition, with most patients staying on therapy one year after oral competitor launch.

• Joenja (leniolisib) is approved for APDS, with strong global revenue growth (+40% YoY in Q2 2026) and ongoing geographic and pediatric expansion, including recent FDA approval for children aged 4–11.

• Napazimone (KL1333) is in pivotal clinical development for mtDNA-driven primary mitochondrial disease, with positive interim futility analysis and a readout expected in 2027.

• 2026 revenue guidance is $375–395M, with Joenja growth accelerating and RUCONEST revenue stabilizing.

Pipeline and clinical development

• Joenja is expanding from APDS into broader immunology indications, targeting genetic PIDs and CVID with immune dysregulation; two Phase II trials are fully enrolled with readouts in Q4 2026.

• Napazimone’s FALCON study is enrolling 180 patients globally, with both primary endpoints passing futility at interim analysis and no changes to study design.

• Joenja’s mechanism targets PI3Kδ-driven immune dysregulation, validated by sustained efficacy and safety in APDS clinical trials and real-world evidence.

Financial outlook and strategy

• Operating expenses for 2026 are projected at $315–320M, with $40M allocated to R&D and $9M in G&A cost reductions.

• Available cash of $159.5M and future cash flows are expected to cover the current pipeline and pre-launch costs.

• Focus on disciplined operating model, scalable organization, and value creation to support long-term growth.