Financial Performance - Total revenues for 2024 increased by 21% to US$297.2 million, exceeding guidance of US$280-$295 million, driven by record RUCONEST® revenue and strong Joenja® growth [3][8][52] - RUCONEST® revenue for the full year 2024 was US$252.2 million, an 11% increase compared to 2023, while Joenja® revenue reached US$45.0 million, a 147% increase in its first full year of sales [8][52] - Fourth quarter 2024 total revenues increased by 14% to US$92.7 million compared to the same quarter in 2023, with RUCONEST® revenue at US$79.6 million (9% increase) and Joenja® revenue at US$13.1 million (66% increase) [8][44][49] Clinical Development and Pipeline - The company is advancing regulatory reviews for leniolisib in key markets and for pediatric use, with two ongoing Phase II trials for additional primary immunodeficiencies (PIDs) [4][27] - The acquisition of Abliva has been completed, adding KL1333 for mitochondrial diseases to the clinical pipeline, which has blockbuster potential [5][34] - Positive top-line results were announced for a Phase III trial of leniolisib in children aged 4 to 11, with global regulatory filings planned for the second half of 2025 [21][19] Market and Product Insights - The U.S. market contributed 98% of 2024 revenues, with the company focusing on expanding its portfolio in the U.S. and Joenja® in key countries [10][8] - The company identified over 880 diagnosed APDS patients globally, with ongoing initiatives to diagnose additional patients through genetic testing programs [14][15] - The strong growth in RUCONEST® revenue is attributed to new physician prescriptions and patient enrollments, with a 24% increase in total enrollments in 2024 [11][9] Financial Outlook - For 2025, the company anticipates total revenues between US$315 million and US$335 million, representing a growth of 6% to 13% [78] - The company plans to continue investing in its product portfolio and R&D pipeline, particularly for PIDs and primary mitochondrial diseases [7][78] - Operating expenses are expected to remain below the prior year's levels, with an estimated US$30 million in Abliva-related expenses [78]
Pharming Group reports fourth quarter and full year 2024 financial results and provides business update