Pharming Group (PHARM) Jefferies London Healthcare Conference 2024 summary

Strategic vision and business pillars

• Focused on building a global rare disease biopharma company with three pillars: Ruconest, Joenja, and pipeline expansion.

• RUCONEST® remains a cornerstone for acute HAE, with strong U.S. demand, revenue growth, and new enrollments up 25% in FY23.

• Joenja® (leniolisib), launched in April 2023 for APDS, is the first and only FDA-approved treatment, expanding globally and driving growth.

• Pipeline development targets new indications for Joenja® and additional rare disease assets in immunology, hematology, respiratory, and gastroenterology.

• Business development is active in seeking clinical-stage products to leverage commercial infrastructure.

Product performance and market positioning

• Ruconest sales exceeded $227 million in the first nine months, serving high-frequency, severe HAE patients, especially those unresponsive to targeted therapies.

• Joenja® generated $31.9 million in the first three quarters of 2024 (+210% YoY), with U.S. 3Q24 revenue at $11.2 million (+73% YoY); global expansion ongoing.

• Joenja® is the first PI3K delta inhibitor approved for APDS, showing a benign safety profile and long-term patient benefit.

• Patient finding strategies include genetic and family testing, medical education, and VUS resolution, with significant pools of potential new patients identified.

• Regulatory progress for Joenja® includes U.K. and Israel approvals in 2024, with filings in Australia and Japan expected in 2025.

Growth drivers and expansion plans

• Expectation to double the Joenja® patient pool in 2025–2026 through VUS clarification and pediatric studies.

• Pediatric studies for Joenja® are advancing, with enrollment complete for ages 4–11 and ongoing for 1–6 years; filings to begin 2H 2025.

• Geographic expansion planned for Europe, UK, Japan, and regulatory milestones anticipated in 2025–2026.

• Leniolisib Phase II trial for PIDs with immune dysregulation started in October 2024, targeting a prevalence of ~7/million, five times that of APDS.

• Ongoing regulatory activities in EU, Canada, and Australia, and continued business development for new assets.