Pharming N.V.(US:PHAR)2025-03-13 23:44Financial Data and Key Metrics Changes - Full-year 2024 revenues increased by 21% to $297 million, exceeding guidance, with strong performance in Q4 [7][42] - Operating profit increased by $5.6 million, marking the second consecutive quarter of positive operating profit [43] - Net results shifted from a loss in Q4 2023 to a net profit in Q4 2024, with positive operating cash flow for the second quarter in a row [44] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 11% to $252 million for the year, with a 9% increase in Q4, driven by new patient enrollments and prescriber base expansion [7][11] - Joenja revenue surged by 147% to $45 million in 2024, with a 65% increase in Q4 [9][12] Market Data and Key Metrics Changes - The prescriber base for RUCONEST increased by 11% and new patient enrollments rose by 24% [11] - Joenja's patient pipeline is expanding, with over 240 identified patients in the US, 40% of whom are on paid therapy [20] Company Strategy and Development Direction - The company aims to develop a leading global rare disease portfolio, leveraging a proven clinical development and commercial infrastructure [7] - The acquisition of Abliva is seen as a strategic move to enhance the company's pipeline and revenue potential [6][46] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in RUCONEST's continued growth despite increasing competition, citing its unique profile and strong patient experience [50][79] - The company anticipates total revenues for 2025 to be between $315 million and $335 million, reflecting continued growth in RUCONEST and Joenja [47] Other Important Information - The company is actively monitoring potential US tariffs on drugs and is taking steps to mitigate any impact on its supply chain [70] - The anticipated operating expenses related to the Abliva acquisition are estimated at $30 million for 2025, including $17 million for R&D [49][69] Q&A Session Summary Question: Insights on KL1333 and patient population - The addressable population for KL1333 is estimated at 30,000 patients in the US and Europe, focusing on those with specific mitochondrial DNA mutations [59] Question: Breakdown of patients in the expanded access program - Patients in the expanded access program are in multiple countries, with some on paid therapy through name patient programs, but specific numbers are not disclosed [63] Question: Recurring costs from Abliva acquisition - Of the $30 million anticipated OpEx from the Abliva acquisition, approximately $17 million will be for R&D, with the remainder being non-recurring costs [69] Question: Impact of potential US tariffs - The company is monitoring the situation regarding US tariffs and is exploring ways to minimize potential impacts on its supply chain [70] Question: RUCONEST's growth despite competition - Management believes RUCONEST will remain a treatment of choice due to its unique profile and strong patient experience, supported by market research [79][81] Question: Timeline for CVID trials - A Phase 2 study is anticipated for CVID, with a Phase 3 study likely required for registration [102] Question: Future expectations for CVID and fast-track designation - The company plans to explore fast-track designation options for CVID, given the severity of the disease [105]