Pharming Group (PHARM) Corporate presentation summary

Commercial and pipeline assets

• RUCONEST® and Joenja® drive strong commercial performance, with both products showing significant revenue growth and market expansion.

• RUCONEST® remains a key on-demand therapy for hereditary angioedema (HAE), with robust U.S. demand and high patient retention, especially among those with severe or frequent attacks.

• Joenja® (leniolisib) demonstrates strong uptake in APDS, with a 35% YoY revenue increase and expanding patient base, including pediatric label expansion under FDA priority review.

• Pipeline includes leniolisib for broader PIDs/CVID and KL1333 for mitochondrial disease, both with significant revenue potential and ongoing pivotal trials.

• Positive interim results for KL1333 and ongoing regulatory reviews for leniolisib support future growth.

Financial performance and outlook

• Preliminary 2025 revenues reached $376M, up 27% from 2024, exceeding guidance.

• Operating profit of $30M and operating cash flow of $44M reported for the first nine months of 2025.

• Operating expenses for 2025 guided at $304-308M, with a focus on cost discipline and capital deployment to high-growth areas.

• Available cash and future cash flows expected to support current pipeline and pre-launch costs.

• 22% CAGR in total revenues since Joenja® launch, with both RUCONEST® and Joenja® contributing to growth.

Clinical and pipeline developments

• Leniolisib sNDA for pediatric APDS under FDA priority review, with global regulatory submissions planned for 2026.

• Phase II trials for leniolisib in PIDs/CVID and KL1333 pivotal trial for mitochondrial disease are progressing, with key readouts expected in 2026 and 2027.

• Joenja® clinical data show significant improvements in immune dysregulation, reduction in infection rates, and favorable safety profile.

• KL1333 demonstrated efficacy in reducing fatigue and improving muscle function in mitochondrial disease patients, with no serious adverse events.

• Expanded APDS prevalence and new genetic findings may significantly increase the addressable patient population for Joenja®.