Executive Summary / Key Takeaways

• Pharming Group N.V. is transforming into a diversified rare disease leader, driven by the sustained growth of its established HAE treatment, RUCONEST, and the accelerating uptake of its APDS therapy, Joenja. This dual commercial engine is generating significant cash flow, enabling strategic pipeline investments.
• The company's core technological differentiation, particularly RUCONEST's comprehensive C1 esterase inhibition and Joenja's selective PI3Kδ inhibition, provides a strong competitive moat in their respective markets, offering superior efficacy and targeted treatment for severe rare diseases.
• Pharming has significantly raised its full-year 2025 revenue guidance to $335 million - $350 million, reflecting strong Q2 2025 performance and anticipated catalysts like Joenja's VUS patient reclassification and pediatric label expansion.
• Strategic pipeline expansion, including leniolisib for broader primary immune deficiencies (PIDs) and the recently acquired KL1333 for primary mitochondrial disease, positions Pharming for potential "blockbuster" assets and long-term value creation.
• While facing competitive pressures in HAE and operational challenges like potential tariffs and exchange rate impacts, Pharming's disciplined financial management, including G&A expense cuts, and its unique cash-funded pipeline development strategy underscore a compelling investment thesis.

A Rare Disease Powerhouse in the Making

Pharming Group N.V. is rapidly evolving from a specialized biopharmaceutical entity into a formidable global rare disease company. This transformation is anchored by two commercial products, RUCONEST and Joenja, and a burgeoning late-stage pipeline, all leveraging a robust rare disease capability platform. The company's strategic vision centers on developing a diverse portfolio and expanding its presence in major markets, supported by an efficient clinical development, supply chain, and commercial infrastructure. This foundational strength, coupled with a clear focus on unmet medical needs, positions Pharming for sustained growth and significant value creation.

The biopharmaceutical industry is undergoing dynamic shifts, with increasing demand for precision medicines and targeted therapies for rare diseases. Pharming's strategy aligns perfectly with this trend, emphasizing innovative solutions for patient populations often overlooked by larger pharmaceutical players. While broader industry trends like the rise of AI in drug discovery could indirectly benefit biopharma through advanced analytics, they also create indirect pressures on operational costs due to increased energy demands for data centers. Pharming's focus on efficient R&D and strategic partnerships could mitigate some of these cost pressures compared to competitors with broader infrastructures.

In the competitive landscape, Pharming operates alongside established players like BioCryst Pharmaceuticals (BCRX), CSL Behring , and Takeda Pharmaceuticals . BioCryst, with its oral HAE treatment Orladeyo, offers a convenience-focused alternative to Pharming's injectable RUCONEST. CSL Behring, a leader in plasma-derived therapies, and Takeda, with its expansive rare disease portfolio, represent larger, more diversified competitors. Pharming differentiates itself through its specialized recombinant technology and a strategic emphasis on partnerships, allowing it to maintain agility and focus on niche markets. While larger rivals may boast greater scale and broader market reach, Pharming's targeted approach and innovative pipeline aim to carve out significant value in specific rare disease segments.

Technological Edge: Precision and Efficacy in Rare Disease Treatment

Pharming's competitive advantage is deeply rooted in its differentiated technology, which underpins the efficacy and unique value propositions of its commercial products.

RUCONEST, a recombinant C1 esterase inhibitor, stands out in the hereditary angioedema (HAE) market due to its comprehensive mode of action. Unlike many acute HAE treatments that target a single pathway, RUCONEST blocks numerous enzymes across all three inflammatory cascades involved in HAE attacks. This broad mechanism translates into tangible and quantifiable benefits for patients: it delivers complete attack resolution in a single dose for 97% of attacks, with half achieving complete resolution within 4.5 hours and the vast majority within 24 hours. This rapid and sustained efficacy is particularly critical for patients experiencing moderate to severe, frequent attacks who have often failed other targeted acute therapies.

Joenja (leniolisib) represents another technological leap, being the first and only FDA-approved treatment for Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). This oral small molecule selectively inhibits PI3K delta, a hyperactive pathway in APDS patients. By normalizing this pathway, Joenja corrects underlying immune defects and restores balance to the immune system, offering a disease-modifying therapy where none existed before.

Pharming's R&D initiatives are strategically focused on expanding the reach of these precision medicines. The company is advancing leniolisib into Phase II proof-of-concept studies for other primary immune deficiencies (PIDs) with immune dysregulation, including a genetically defined group and Common Variable Immune Deficiency (CVID). These represent significantly larger patient populations than APDS, with the PID group estimated at least 2,500 patients and CVID at least 13,000 patients in the U.S. The stated goal is to propel Joenja to a "blockbuster" franchise, leveraging the molecule's proven mechanism in related immune dysregulation disorders.

Furthermore, the recent acquisition of Abliva introduced KL1333, a high-value late-stage asset for primary mitochondrial disease (PMD). KL1333 addresses the underlying disorder by normalizing the NAD+ to NADH ratio, which is abnormally low in PMD patients. The registrational trial for KL1333 has already passed an interim analysis, with a readout expected in 2027 and potential approval in 2028. This asset targets an addressable population of approximately 30,000 patients in the U.S. and large European countries.

For investors, these technological differentiators and pipeline advancements are crucial. They form the bedrock of Pharming's competitive moat, enabling premium pricing, driving market share in niche segments, and fostering long-term growth. The ability to develop and commercialize targeted therapies for severe rare diseases, where unmet needs are high, translates directly into stronger financial performance and a more resilient market position.

Financial Performance and Operational Momentum

Pharming has demonstrated robust financial performance, reflecting its strategic execution and the strength of its commercial assets. In the second quarter of 2025, total revenues grew by 26% year-on-year, reaching $93.2 million. This strong top-line growth fueled a meaningful operating profit of $12.9 million, a significant turnaround from a loss in the previous year, excluding $2.1 million in nonrecurring Abliva acquisition-related expenses. For the first half of 2025, total revenues increased by 33%, with gross profit rising by 37% compared to the first half of 2024.

RUCONEST continues to be a foundational revenue driver, achieving $80.4 million in Q2 2025, a 28% increase over Q2 2024. This consistent double-digit growth, even after 10 years on the market, is attributed to a steady increase in new prescribers (an average of 21 new prescribers per quarter over the past six quarters) and a 27% increase in new patient enrollments in the U.S. over the prior year. Joenja, while earlier in its lifecycle, is rapidly accelerating its patient uptake, with a 15% revenue increase to $12.8 million in Q2 2025. The increase of 18 patients on paid therapy in the first half of 2025 already surpassed the total increase for all of 2024.

Looking at the full year 2024, Pharming reported revenues of $297 million, a 21% increase over 2023, exceeding its guidance range. RUCONEST contributed $252 million (up 11%), and Joenja generated $45 million (up 147%). The company also achieved operating profit in the last two quarters of 2024, signaling improving profitability.

Pharming's liquidity position is also strengthening. Cash and marketable securities increased from $108.9 million at the end of Q1 2025 to $130.8 million by the end of Q2 2025, primarily driven by positive net cash flows from operating activities.

While the $66.1 million acquisition of Abliva shares in March 2025 impacted cash reserves, management asserts that available cash and future cash flows are expected to cover current pipeline and pre-launch costs, a "very unique position in the biotech environment."

Compared to competitors, Pharming's gross profit margin (TTM) stands at 89.31%, indicating strong product profitability. Its operating profit margin (TTM) of 4.30% and net profit margin (TTM) of -2.41% reflect ongoing investments in R&D and commercial expansion, particularly with the Joenja launch and Abliva acquisition. While direct comparative margins for specific rare disease segments are not always publicly detailed for larger, more diversified competitors like CSL Behring (CSLFY) or Takeda (TAK), Pharming's ability to generate positive operating cash flow for multiple consecutive quarters, even with acquisition-related expenses, demonstrates operational effectiveness.

Strategic Initiatives and Growth Catalysts

Pharming's future growth is underpinned by a series of clear strategic initiatives and catalysts across its commercial portfolio and pipeline.

For Joenja, significant expansion is anticipated. A new study published in Cell identified over 100 new variants leading to PI3K delta hyperactivity, suggesting a much higher prevalence of APDS, "perhaps even 100x greater." This scientific breakthrough is expected to facilitate the reclassification of approximately 20% of the more than 1,400 U.S. patients with Variants of Uncertain Significance (VUS) results as having APDS, with this process commencing in the second half of 2025. This represents a substantial expansion of the addressable patient pool. Furthermore, Pharming filed its regulatory application for Joenja in Japan in June 2025 and intends to file with the FDA for pediatric label expansion (ages 4-11) in Q3 2025, with an expected six-month review. Launches in the European Union and Canada are also anticipated over the next 12 months, following the successful U.K. launch in April 2025.

The pipeline for leniolisib is also progressing rapidly. Two Phase II proof-of-concept studies for primary immune deficiencies with immune dysregulation (genetically defined and CVID) are on track to read out in the second half of 2026. These indications target patient populations significantly larger than APDS, with CVID alone representing at least 13,000 patients in the U.S., underscoring the potential for leniolisib to become a "blockbuster" franchise. The registrational study for KL1333 in primary mitochondrial disease has resumed enrollment, with a readout expected in 2027 and potential approval in 2028.

Reflecting its strong growth momentum, Pharming was promoted to the Euronext AMX MidCap index, effective September 22, 2025. This increased visibility and attractiveness to investors is a testament to the company's financial and pipeline growth. On the financial discipline front, Pharming is implementing a sustainable 15% cut in G&A expenses, aiming for $10 million in annual savings, to optimize capital allocation towards business growth.

Outlook and Risk Assessment

Pharming has raised its full-year 2025 total revenue guidance to between $335 million and $350 million, up from the prior guidance of $325 million to $340 million. This implies a robust full-year revenue growth between 13% and 18%, driven by the continued strong performance of RUCONEST and the accelerating uptake of Joenja, particularly from the VUS patient reclassification and geographic expansion. Total operating expenses for 2025 are projected to be between $304 million and $308 million, a slight increase primarily due to the negative impact of a higher euro-dollar exchange rate, which the company is actively working to mitigate.

Despite this positive outlook, investors should consider several risks. The HAE market is becoming more competitive with new entrants like sebetralstat. While management believes RUCONEST serves a distinct patient population—those with more severe attacks who have failed other treatments—some market disruption is possible. The company is also monitoring the potential impact of U.S. import tariffs, evaluating mitigation strategies for its supply chain and manufacturing. Furthermore, while Joenja's patient identification and conversion rates are strong, the process can be non-linear due to the complexities of rare disease diagnosis and insurance authorization. The $5.3 million negative impact from the euro-dollar exchange rate on operating expenses highlights currency fluctuation as an ongoing financial risk.

Conclusion

Pharming Group N.V. is at a pivotal juncture, successfully transforming into a leading global rare disease company. Its core investment thesis is built upon the robust performance of its dual commercial engines, RUCONEST and Joenja, which generate significant cash flow to fuel an ambitious and high-value pipeline. The company's technological prowess in recombinant therapies and selective enzyme inhibition provides a strong competitive moat, delivering superior efficacy for patients with severe, life-threatening rare diseases.

With a raised 2025 revenue guidance and clear catalysts for Joenja's expansion—including VUS patient reclassification, pediatric label extension, and global launches—Pharming is poised for accelerated growth. The strategic investments in leniolisib for broader PID indications and KL1333 for primary mitochondrial disease hold the potential to create multiple "blockbuster" assets, significantly expanding the company's long-term value proposition. While competitive dynamics in HAE and macroeconomic factors present challenges, Pharming's disciplined financial management and unique cash-funded pipeline development strategy position it favorably. For discerning investors, Pharming offers a compelling opportunity to participate in the growth of a focused rare disease innovator with a clear roadmap for sustainable value creation.