Pharming Group N.V. (NASDAQ: PHAR)*
As of 14 March 2025
Company Performance
Full-year 2024 revenues increased 21% to $297 million, exceeding guidance
Q4 2024 showed strong 14% revenue growth compared to Q4 2023
Operating profit and positive operating cash flow achieved in last two quarters of 2024
2025 revenue guidance: $315-335 million (6-13% growth)
Product Performance
RUCONEST (for HAE):
11% growth to $252 million in 2024
9% growth in Q4
11% increase in prescriber base and 24% increase in new patient enrollments
Management expects continued growth despite new market entrants due to its unique profile
Joenja (leniolisib for APDS):
147% growth to $45 million in 2024
65% growth in Q4 to $13.1 million
96 patients plus 5 pending on paid therapy in US
Additional 188 patients on therapy globally under access programs and clinical trials
Pipeline & Growth Strategy
Joenja expansion plans:
VUS (variant of unknown significance) resolution program to identify new APDS patients by mid-2025
Pediatric indication (ages 4-11) with FDA filing expected in H2 2025
Geographic expansion starting with UK launch in 2025 (NICE draft guidance published)
Additional indications in development: APLS and CVID (Common Variable Immune Deficiency)
Abliva acquisition ($66.1 million):
Adds KL1333 for primary mitochondrial diseases (PMD)
Pivotal study underway with endpoints agreed with FDA
Addressable population of approximately 30,000 patients in US and major European markets
Expected OpEx impact: $30 million in 2025 ($17 million R&D, remainder in transaction costs)
Pipeline expansion into larger indications:
Phase 2 trials initiated for two additional indications for leniolisib
CVID represents a significant market opportunity with 40 patients per million prevalence
Corporate Strategy
Developing a global rare disease company with diverse portfolio and presence in large markets
Leveraging clinical development, supply chain, and commercial infrastructure
Focus on rigorous P&L management and continued pipeline expansion