Pharming Group's 2024 Financial Results: A Blockbuster Year for Rare Disease Innovations

Pharming Group N.V. (Euronext Amsterdam: PHARM / Nasdaq: PHAR) has just released its preliminary (unaudited) financial report for the three months and full year ended December 31, 2024, and the numbers are nothing short of spectacular. The biopharmaceutical company, dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases, reported a 21% increase in total revenues for the full year 2024, driven by record RUCONEST® revenue and strong Joenja® (leniolisib) growth. This performance not only exceeded the company's guidance but also underscores Pharming's momentum in the rare disease treatment landscape.

The full year 2024 total revenues increased by 21% to US$297.2 million, significantly above the guidance range of US$280-$295 million. This achievement is a testament to the continued importance of RUCONEST® in the hereditary angioedema (HAE) treatment landscape as well as the strong commercial performance of Joenja® in just its first full year of sales. RUCONEST®, marketed for the treatment of acute HAE attacks, demonstrated significant strength in the fourth quarter of 2024, with record revenues of US$79.6 million, a 9% increase compared to the fourth quarter of 2023. RUCONEST® revenues for the full year 2024 were a record US$252.2 million, an 11% increase compared to 2023. The U.S. market contributed 98% of 2024 revenues, while the EU and Rest of World contributed 2%. The strong RUCONEST® revenue growth in 2024 can be attributed to strong performance in new physicians prescribing RUCONEST®.

Joenja® (leniolisib) revenue increased by 147% to US$45.0 million in the first full year post-launch and fourth quarter revenue increased by 66% to US$13.1 million, compared to the fourth quarter 2023. This remarkable growth demonstrates the strong commercial performance of Joenja® and its potential to become a significant revenue driver for the company.

Pharming's 2024 financial performance was also noteworthy, with the company generating an operating profit and positive net cash flows from operations in the last two quarters of the year. This performance highlights the financial strength of Pharming's core commercial business. Looking to 2025, Pharming has provided total revenue guidance of US$315 million – US$335 million, reflecting confidence in the continued momentum of its commercial portfolio and the potential for significant revenue growth from its strategic initiatives.

One of the key strategic initiatives that positions Pharming for long-term growth is the acquisition of Abliva AB. This acquisition adds KL1333 for mitochondrial DNA-driven primary mitochondrial diseases to Pharming’s late-stage clinical pipeline, in line with the company's vision to become a leading global rare disease company. KL1333 has the opportunity to further transform Pharming’s growth trajectory as it too has blockbuster potential. The acquisition aligns with Pharming's vision to become a leading global rare disease company and is well aligned with its operational capabilities.

Another strategic initiative that could significantly influence Pharming's revenue projections and market expansion strategies in 2025 and beyond is the ongoing Phase II clinical trials of leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, including common variable immunodeficiency (CVID). These trials represent a strategic effort to expand the addressable patient population for leniolisib, which is currently marketed as Joenja®. The genetically identifiable PIDs indication and the CVID indication, first announced in 2025, represent significantly larger market opportunities than APDS, with blockbuster revenue potential. Successful outcomes from these trials could lead to regulatory approvals in key markets and for pediatric use, further broadening the commercial potential of leniolisib.

In summary, Pharming Group's 2024 financial results and business update paint a picture of a company on the rise, with a strong commercial portfolio, strategic acquisitions, and promising clinical trials. The company's focus on rare disease treatments and its financial strength position it well for continued growth and success in the biopharmaceutical sector.