Pharming N.V.(US:PHAR)2024-03-14 17:15Financial Data and Key Metrics Changes - The company reported over $227 million in revenue for 2023, representing a 10% growth compared to the previous year, significantly exceeding the expected single-digit growth for RUCONEST [6][45] - In Q4 2023, revenue grew by 49%, with RUCONEST achieving a record revenue of $73.3 million, reflecting a 34% increase in that quarter [26][33] - The total net loss for 2023 was $10.1 million, a decline from a net profit of $13.7 million in the previous year, primarily due to higher operating costs and adverse foreign exchange fluctuations [44] Business Line Data and Key Metrics Changes - RUCONEST's revenue growth was driven by increased patient reliance, with a 10% growth in 2022 and a record revenue since its launch in the US [45][56] - Joenja, launched in the US in April 2023, generated over $18 million in sales during its first nine months, with 81 patients on therapy by year-end [7][98] - Joenja's revenue grew by 21% compared to Q3 2023, reaching $7.9 million [98] Market Data and Key Metrics Changes - The company identified over 840 patients with APDS globally, with an estimated prevalence of approximately 1.5 patients per million [13] - The company expects continued growth in the US market for Joenja, supported by family testing and validation efforts [51] Company Strategy and Development Direction - The company aims to expand its portfolio through potential acquisitions and in-licensing of clinical-stage opportunities in rare diseases, particularly in immunology, hematology, respiratory, and gastroenterology [54][127] - A Phase 2 clinical trial for leniolisib in primary immune deficiencies with immune dysregulation linked to PI3K delta signaling is expected to significantly expand its long-term commercial potential [54][132] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about RUCONEST's growth despite increased competition, highlighting strong underlying market indicators [57][60] - The company provided revenue guidance for 2024, expecting between $280 million and $295 million, indicating a growth of 14% to 20% [48][134] Other Important Information - The company is actively engaged in genetic testing partnerships to identify patients with APDS and is working on a registry to follow APDS patients longitudinally [18][75] - The gross profit in Q4 2023 increased by $25.8 million compared to the same quarter last year, driven by higher revenues [33] Q&A Session Summary Question: Can we still expect seasonality for RUCONEST in first quarter sales as previous years? - Management indicated that similar patterns are expected, with strong underlying indicators continuing into the first quarter [35][44] Question: Can you provide any color on the duration of the patients on Joenja since its launch? - The company reported high adherence rates close to 90% and a steady inflow of new patients through family testing efforts [12][64] Question: What is the expected impact of the expiration of RUCONEST's exclusivity in the US? - Management expects no impact on RUCONEST, as there is no significant interest in developing a biosimilar for this product [116] Question: What is the risk profile for the second indication in primary immune deficiencies? - Management believes that many genetic dysfunction areas should respond positively, based on previous interactions with the immunology community [74] Question: When can we expect data from the registry for APDS patients? - The registry is just underway, with more substantial data expected in 2025, while expanded access program data will be shared at conferences this year [105][75]