Pharming (PHAR) Q2 2025: Ruconest Drives 28% Growth as APDS Pipeline Expands
Pharming delivered a breakout quarter, propelled by double-digit Ruconest growth and accelerating Joenja patient uptake, as the company leverages rare disease expertise to expand its APDS franchise. New genetic findings and regulatory progress unlock further addressable market upside, supporting upgraded revenue guidance and sustained pipeline investment. Investors should watch for the pace of VUS reclassifications and pediatric label expansion as key catalysts in the next 12 months.
Summary
- Ruconest’s Unique Market Position: Continued double-digit growth confirms its role as a reliable cash engine funding pipeline expansion.
- Joenja’s Patient Uptake Accelerates: APDS franchise momentum builds with new patient identification and geographic launches.
- Pipeline and Label Expansion Catalysts: Recent genetic discoveries and regulatory filings set the stage for further addressable market gains.
Performance Analysis
Pharming’s Q2 saw revenue up 26% YoY, with operating profit swinging to $12.9 million from a prior-year loss, reflecting disciplined execution and robust commercial momentum. Ruconest, the company’s legacy hereditary angioedema (HAE) therapy, delivered 28% revenue growth (to $80.4 million in Q2), driven by a steady increase in prescribers—averaging 21 new additions per quarter for the past six quarters. This consistent expansion in the HAE patient base underscores Ruconest’s differentiated value for moderate to severe, frequently relapsing patients who have failed alternative therapies.
Joenja, the only disease-modifying therapy (DMT) approved for activated PI3K delta syndrome (APDS), posted 15% sequential revenue growth and an inflection in patient uptake: 18 new treated patients in H1 2025, already surpassing the total for all of 2024. The UK commercial launch and further expansion into new markets signal the early stage of Joenja’s lifecycle, with over 900 global patients identified and 185 on therapy via various access channels. Gross profit margin remained robust, and operating expenses were tightly managed, with most OPEX increases tied to non-recurring acquisition costs (Abliva), leaving underlying cost discipline intact.
- Cash Generation Strength: Cash and marketable securities rose to $130.8 million, mainly from operational cash flow, despite $66 million spent on the Abliva acquisition.
- Inventory Dynamics: Joenja’s revenue growth lagged patient uptake due to prior-year inventory builds, not conversion issues.
- OPEX Control: Excluding acquisition-related items, expense growth was minimal, with a 15% G&A reduction plan in progress.
Overall, Pharming’s dual-engine model—steady Ruconest cash flows and accelerating Joenja adoption—supports both near-term performance and long-term pipeline investment.
Executive Commentary
"Over the past two years, farming has evolved from being a one asset company to having two fast-growing commercial products and a high-value late-stage pipeline with two assets with over $1 billion potential each."
Fabrice Chiracchi, CEO
"In fact, we've added an average of 21 new Ruconest prescribers in each of the past six quarters. And this leads directly to the continuing increase in new patient enrollments, which obviously translates to a volume increase over prior year, which is 27% in the U.S."
Steve Thor, Chief Commercial Officer
Strategic Positioning
1. Ruconest as a Durable Cash Flow Foundation
Ruconest, recombinant C1 esterase inhibitor for acute HAE attacks, remains the cornerstone of Pharming’s financial model. Its unique efficacy profile (97% of attacks resolved in a single dose, half within 4.5 hours) and appeal to patients with frequent, severe attacks who have failed other therapies cement its role as a cash generator. Physician engagement continues to rise, and new competitors are not expected to meaningfully disrupt the core patient segment, as most Ruconest users are refractory to other modalities.
2. Joenja’s Expanding APDS Franchise
Joenja, PI3K delta inhibitor for APDS, is in the early innings of growth, with a multi-pronged expansion strategy: (1) reclassification of VUS (variant of uncertain significance) patients using new genetic data, (2) pediatric label expansion, and (3) launches in additional geographies (UK, Japan, EU, Canada). The addressable patient pool could increase substantially, with management citing a potential 100-fold higher prevalence suggested by recent Cell publication findings.
3. Pipeline Value Creation and Capital Allocation
The pipeline is anchored by two late-stage assets with $1 billion-plus potential: expanded Joenja indications (primary immune deficiencies, CVID) and KL1333 (primary mitochondrial disease, acquired via Abliva). Both programs are on track, with regulatory and clinical milestones expected through 2027. Capital discipline is evident, with a 15% G&A cut underway and cash flows expected to fund current pipeline and pre-launch costs without additional dilution.
4. Rare Disease Platform and Organizational Focus
Pharming’s rare disease commercialization platform, built over the past decade, is now a strategic asset. The company leverages deep expertise in patient finding, prescriber education, and access navigation—critical in ultra-orphan indications with fragmented patient populations. Employee engagement and retention were highlighted as a source of execution strength.
5. Regulatory and Scientific Tailwinds
Recent genetic research expands the APDS landscape, enabling labs to reclassify VUS cases and broadening the clinical phenotype. Management expects 20% of 1,400 identified VUS patients in the US to be reclassified as APDS, with reimbursement mechanisms already in place for rapid therapy initiation. Ongoing studies may further increase the eligible population without requiring new clinical trials.
Key Considerations
This quarter’s results reflect Pharming’s successful transition from a single-product company to a diversified rare disease platform with multiple growth levers.
Key Considerations:
- Ruconest’s Prescriber Growth: Sustained double-digit expansion in US prescribers signals ongoing relevance even as new competitors enter the HAE space.
- Joenja Patient Ramp: H1 2025 saw more new patients than all of 2024, with further upside from VUS reclassification and pediatric expansion.
- Regulatory Milestones: Japan and US pediatric filings, plus European and Canadian launches, provide near-term catalysts for Joenja adoption.
- Pipeline Execution: KL1333 pivotal trial reactivation and ongoing Phase II studies in broader immune deficiencies position Pharming for future blockbusters.
- Cost Discipline Amid Growth: Operating expenses remain tightly controlled, with non-recurring acquisition costs isolated and a 15% G&A reduction underway.
Risks
Key risks include the pace and completeness of VUS reclassification, which depends on external lab processing and physician follow-up, potentially leading to slower-than-expected Joenja uptake. Competitive dynamics in the acute HAE market remain a watchpoint, though management believes Ruconest’s patient segment is insulated. Pipeline execution risk exists, particularly in translating early genetic findings into label and market expansion without new clinical trials. Currency fluctuations and milestone-related OPEX (e.g., leniolafib) could also pressure margins.
Forward Outlook
For Q3 2025, Pharming guided to:
- Continued double-digit revenue growth for Ruconest and Joenja
- Ongoing patient additions from VUS reclassification and new market launches
For full-year 2025, management raised guidance to:
- Total revenue of $335 million to $350 million (up from $325 million to $340 million)
- Total operating expenses of $304 million to $308 million (reflecting FX impact and milestone costs)
Management highlighted several factors that will shape results:
- Timely execution of VUS reclassification and pediatric label expansion
- Progress on cost optimization and capital allocation discipline
Takeaways
Pharming’s Q2 results validate its dual-engine business model, with Ruconest providing cash flow stability and Joenja unlocking new rare disease growth. Scientific and regulatory catalysts set up multiple value inflections over the next year.
- APDS Franchise Expansion: The reclassification of VUS patients and new pediatric indications could meaningfully increase Joenja’s addressable market, supporting sustained top-line growth.
- Financial Resilience: Cash flow from operations and prudent OPEX management give Pharming flexibility to fund pipeline advancement without near-term financing risk.
- Investor Watchpoints: Monitor the pace of patient conversion from VUS and pediatric pools, as well as competitive responses in the HAE market, for signs of upside or emerging risk.
Conclusion
Pharming’s transformation into a multi-asset rare disease company is gaining traction, with commercial momentum, pipeline catalysts, and cost discipline all supporting its upgraded outlook. The next 12 months will be defined by execution on patient identification, regulatory expansion, and continued capital stewardship.
Industry Read-Through
Pharming’s results reinforce several rare disease sector themes: The ability to leverage genetic insights for label expansion and patient finding is increasingly a competitive differentiator, as seen with APDS. Commercial execution in ultra-orphan indications requires deep prescriber and patient engagement, not just product differentiation. Pipeline acquisitions (like Abliva) and disciplined OPEX management are essential to balancing growth with financial sustainability. Other rare disease players should watch for the impact of real-world genetic data on market size and the importance of building platforms that can deliver both cash flow and pipeline optionality.