Pharming Group (PHARM) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Strategic direction and business overview

• Building a global rare disease biopharma company with a strong U.S. commercialization base; over 95% of commercial business is U.S.-driven.

• Two marketed products: Ruconest (hereditary angioedema) and Joenja (APDS), with Ruconest showing 16% growth versus prior year.

• Joenja, in-licensed from Novartis, generated $20.7M in H1 2024 sales, with focus on patient identification due to disease rarity.

• Expansion plans include regulatory reviews in multiple territories, pediatric programs, and a near-complete Japanese trial.

• Actively seeking in-licensing or acquisition of rare disease assets to leverage commercialization infrastructure, focusing on immunology, hematology, respiratory, and gastroenterology.

Product performance and differentiation

• Ruconest is a recombinant C1 esterase inhibitor, uniquely positioned as a protein replacement therapy, unlike competitors.

• Ruconest remains a cornerstone for acute HAE, with FY23 revenue of $227.1M and 1H24 revenue of $109.0M, driven by increased patient enrollments and prescriber growth.

• Ruconest is essential for severe cases and patients unresponsive to other therapies, with 97% requiring only one dose per attack.

• Regulatory exclusivity for Ruconest expires in 2026, but no biosimilar competition is expected due to unique production platform.

• Competitors target only one pathway, leading to breakthrough attacks; Ruconest addresses broader disease mechanisms.

Joenja (leniolisib) development and market expansion

• Joenja is the first and only FDA-approved treatment for APDS, launched in the U.S. in April 2023, with FY23 revenue of $18.2M and 1H24 revenue of $20.7M (+44% vs. 2H23).

• Patient finding is critical; initiatives include genetic testing partnerships, family testing, and clarifying VUS cases.

• Expecting to identify 240-250 new U.S. patients from VUS clarification by year-end.

• Regulatory progress: EU approval expected Q1 2026, UK launch in H1 2025, Japanese filing early next year, and ongoing approvals in other regions, with Israel approval received.

• Pediatric studies nearing completion, aiming for broader label within 18 months.