Pharming Group (PHARM) investor relations material

Business transformation and growth

• Transitioned from a single-asset to a dual-commercial asset company, with both RUCONEST and Joenja showing double-digit growth and a high-value pipeline with two programs each having over $1 billion sales potential.

• Strong momentum with 26% sales growth in the last quarter, meaningful operating profit, and raised revenue guidance to $335–$350 million for the year.

• RUCONEST, a legacy product for hereditary angioedema, continues to grow 28% year-on-year, driven by new prescribers and patients, and is manufactured via a unique process using transgenic rabbits.

• Joenja, approved for APDS, is the only drug for this rare immunodeficiency and has seen accelerated patient uptake, with more new patients in the first half of the year than all of last year.

• Both commercial assets provide strong, predictable cash flows to self-fund pipeline development and launches.

Pipeline and near-term catalysts

• Joenja's growth is expected to accelerate with three catalysts: reclassification of VUS patients, pediatric label expansion (NDA submitted, decision expected first half next year), and geographic expansion (recent UK launch, NDA filed in Japan, EU approval expected next year).

• Recent research identified 100 new APDS variants, potentially increasing disease prevalence up to 100 times, offering significant upside for Joenja.

• Two Phase 2 trials for Joenja in adjacent primary immunodeficiencies and SIBD are underway, with readouts expected in the second half of next year.

• KL1333, acquired from Ableva, is in Phase 2 for primary mitochondrial disease, with successful interim analysis and final readout expected in 2027.

• Both Joenja and KL1333 are positioned as potential billion-dollar programs, with multiple short- and long-term growth drivers.

Financial outlook and strategy

• Sales revenue grew 33% in the first half of the year, with updated guidance reflecting confidence in both near- and long-term growth opportunities.

• Operating expenses include non-recurring acquisition costs, but cash flows from RUCONEST are expected to sustain ongoing development and launches.

• Focus remains on leveraging proven capabilities in clinical development, supply chain, and commercial execution to become a world leader in rare diseases.

• No need for external funding anticipated as pipeline and launches are expected to be self-funded.

• Multiple near-term catalysts, including trial readouts and regulatory decisions, are expected to drive further value creation.