Pharming Group (PHARM) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Strategic vision and commercial highlights

• Focus on building a leading global rare disease company with a diverse portfolio and strong presence in large markets.

• Achieved $376 million in sales in 2025, representing 27% growth over 2024, driven by RUCONEST and Joenja.

• RUCONEST continues to deliver strong growth, with a unique value proposition and high patient retention.

• Joenja, launched in 2023 for APDS, is seen as a significant near-term growth catalyst.

• Strategic withdrawal from RUCONEST commercial activities outside the US, leveraging existing infrastructure for Joenja in Europe.

Product and pipeline developments

• RUCONEST is the only recombinant C1 inhibitor replacement therapy for hereditary angioedema, showing rapid onset and high efficacy.

• Joenja (leniolisib) targets APDS and related immune deficiencies, with ongoing efforts to expand its label to pediatric populations and other indications.

• Two phase II proof-of-concept studies for Joenja in broader immune dysregulation are expected to read out in the second half of 2024.

• Napazimone (formerly KL-1333) is in phase II for primary mitochondrial disease, with positive interim results and a pivotal readout expected at the end of 2027.

• Active business development continues, with a focus on in-licensing and M&A to expand the pipeline.

Financial performance and guidance

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

• 2026 revenue guidance of $405–$425 million, representing 8–13% growth, excluding potential upside from pediatric Joenja approval.

• Operating expenses for 2025 guided at $304–$308 million, rising to $330–$335 million in 2026 due to increased R&D investment.

• $9 million in structural G&A cost reductions implemented in October 2025 to offset expense growth.

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