Pharming Group (PHARM) Jefferies 2024 Global Healthcare Conference summary

Strategic Vision and Product Portfolio

• Aims to build a leading global rare disease company, leveraging Ruconest and Joenja as core assets.

• Ruconest, approved for acute hereditary angioedema, generated $227.1 million in US sales in FY23 and continues to grow after a decade on the market.

• Joenja (leniolisib), in-licensed from Novartis, targets APDS and achieved $28 million in its first US market year, with $18.2 million in FY23 and $9.6 million in 1Q24.

• Expansion plans include new indications, pediatric studies, and global regulatory submissions for Joenja, with ongoing reviews in Israel, Europe, UK, Canada, Australia, and Japan.

• Focus remains on rare and ultra-rare disease opportunities, with a scalable commercialization infrastructure and strategic asset acquisitions.

Market Performance and Growth Drivers

• Ruconest Q1 2024 revenues reached $46 million, up 8% year-over-year, with increasing patient and prescriber numbers.

• Joenja is positioned as a disease-modifying therapy for APDS, with ongoing efforts to identify and confirm eligible patients through genetic testing and medical education.

• The MAVE study is expected to clarify APDS mutations by year-end, potentially expanding the US patient base significantly in 2025.

• Pediatric studies and a secondary indication for Joenja in immune dysregulation are expected to drive growth from 2026 onward, with Phase II trials underway.

• Global expansion is underway, with regulatory actions and clinical trials in Israel, Japan, UK, Canada, Australia, Asia Pacific, and the Middle East.

Competitive Landscape and Product Differentiation

• Ruconest serves patients with severe hereditary angioedema who have failed other therapies, offering near 100% efficacy as the only recombinant C1 esterase inhibitor actively marketed.

• New oral and subcutaneous therapies exist, but Ruconest remains essential for breakthrough attacks, with 97% of patients needing just one dose per attack.

• Joenja is the first and only FDA-approved PI3 kinase delta inhibitor for APDS, with a benign safety profile and long-term efficacy data.

• Extensive medical education and genetic testing programs are in place to identify APDS patients and expand the eligible patient base.