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Pharming Group (PHARM) investor relations material

Pharming Group Jefferies London Healthcare Conference 2025 summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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Jefferies London Healthcare Conference 2025 summary19 Nov, 2025

Company evolution and strategic focus

  • Transitioned from a single-asset pharma to a high-growth biotech with two marketed assets and a robust pipeline, each with $1 billion revenue potential.

  • CEO joined to unlock value and apply a 10x growth mindset.

  • Focused on disciplined capital allocation, recently cutting 20% of G&A headcount to redirect resources to growth areas.

  • Attracted a new CFO with a strong value creation background to optimize P&L management.

RUCONEST performance and market strategy

  • RUCONEST continues double-digit growth, serving severe HAE patients not controlled by other treatments.

  • Maintains a unique position due to its efficacy, fast onset, and IV administration for hard-to-treat cases.

  • U.S. market remains the primary revenue source; exited non-U.S. markets due to low sales and high administrative burden.

  • Focus is on sustaining growth and reinvesting proceeds into high-growth assets.

Joenja growth opportunities and expansion

  • Joenja, for APDS, is in early high-growth phase with accelerating patient uptake.

  • Pediatric label expansion expected by end of January, potentially increasing addressable market by 30%.

  • Reclassification of VUS patients could boost APDS market by 50%, with process underway following new scientific data.

  • Ongoing trials in adjacent immunodeficiencies with much larger prevalence, with readouts expected in H2 2026.

  • Successful U.K. launch; EMA and Japan approvals targeted for next year as part of a focused international expansion.

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Frequently asked questions

Pharming Group N.V. is a global biopharmaceutical company based in Leiden, the Netherlands. The company specializes in the development and commercialization of protein replacement therapies and precision medicines, particularly for rare diseases. Pharming's key product is RUCONEST, a plasma-free recombinant human C1 esterase inhibitor used for treating acute hereditary angioedema (HAE). The company also has several other products in various stages of development, including treatments for Pompe and Fabry diseases and other rare disorders. The company is headquartered in Leiden, the Netherlands, and its shares are listed on both the Amsterdam Euronext and Nasdaq.

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