Pharming Group (NASDAQ:PHAR) outlined its 2026 financial guidance and provided updates on its commercial portfolio and late-stage pipeline during its 2026 Investor Day, emphasizing continued revenue growth, disciplined spending, and multiple clinical readouts expected over the next two years.
Commercial portfolio: RUCONEST durability and Joenja growth catalysts
CEO Fabrice Chouraqui said the company has evolved from a single-asset business into a biotech with two commercial products growing at double-digit rates and a late-stage pipeline with two programs the company believes each carry more than $1 billion in sales potential.
On Joenja, management said the product is early in its lifecycle with multiple potential growth drivers. Chouraqui said the company was disappointed by the FDA’s response to its submission for pediatric label expansion in activated PI3K delta syndrome (APDS), but remains committed to pursuing that population and plans to request a Type A meeting that is expected to be scheduled in March. He also cited published data suggesting higher APDS prevalence and the potential for patients with variants of uncertain significance (VUS) to be reclassified over time, alongside plans to explore broader primary immunodeficiency (PID) populations.
2025 preliminary results and 2026 guidance
CFO Kenneth Lynard said 2025 results were preliminary and unaudited, with final figures to be provided on the company’s fourth-quarter and full-year 2025 earnings call scheduled for March 12, 2026. Pharming reported preliminary 2025 revenue of approximately $376 million, up 27% versus 2024.
- RUCONEST: $318 million in 2025 revenue, up 26% year over year.
- Joenja: $58 million in 2025 revenue, up 29% year over year.
Lynard said Joenja revenue surpassed $50 million in 2025, triggering a $5 million milestone payment to Novartis that was booked in cost of goods sold. Excluding that milestone, he said gross margin is expected to be approximately 88% to 89% for 2025. Pharming reiterated expectations for 2025 operating costs of $304 million to $308 million, excluding a one-time $7 million restructuring charge tied to structural G&A reductions announced in October 2025. The company ended 2025 with $181 million in cash and marketable securities, according to Lynard.
For 2026, Pharming guided to total revenue of $405 million to $425 million, implying 8% to 13% growth over 2025 and above an analyst consensus estimate cited by management of about $398 million. The company expects mid-single-digit growth for RUCONEST, driven by continued U.S. volume growth, partly offset by declining ex-U.S. revenue as Pharming exits those markets. Management also forecast “significant” Joenja growth, with acceleration versus 2025.
Notably, Lynard said Pharming has conservatively excluded any 2026 revenue contribution from a potential U.S. pediatric APDS approval for ages four to 11 until there is greater clarity on timelines. The company similarly said it is not assuming a $10 million Novartis milestone tied to Joenja revenue exceeding $100 million, nor other ex-U.S. rollout-related milestones in 2026.
Operating expenses are expected to be $300 million to $335 million in 2026, reflecting more than $60 million of incremental R&D investment to advance the pipeline, a $9 million favorable impact from the October 2025 G&A reductions, and stable marketing and sales spending. Pharming estimated cost of goods sold at roughly 10% of revenue, implying a 90% gross margin. Lynard said available cash and future cash flows are expected to fund the current pipeline and related pre-launch costs.
FDA pediatric APDS questions and RUCONEST market dynamics
In Q&A, management said it has substantial data in hand to address FDA questions related to pediatric dosing for leniolisib (Joenja) and will engage the agency at the upcoming Type A meeting. Chief Medical Officer Anurag Relan said the FDA’s concerns related to the lowest two pediatric doses (20 mg and 30 mg) used in children weighing less than 27 kg, and that efficacy data in those dose groups showed comparable efficacy across doses. He said the company expects to speak more confidently after the March meeting.
On RUCONEST, management said it implemented a 3% price increase “in line with CPI,” while also expecting volume growth beyond pricing. Chouraqui said RUCONEST growth is expected to come entirely from the U.S. market. Responding to questions about switching to oral therapies, he said Pharming has seen “a few” switches among milder patients trying oral options, along with anecdotal reports of patients returning, and said the company has not observed a significant overall impact. He described RUCONEST as entrenched in a subset of on-demand patients who often have failed other treatments and require rapid, reliable control.
On the potential contribution from reclassification of VUS patients as APDS, Pharming said it has not included VUS-driven contributions in its guidance given uncertainty over timing.
Pipeline update: leniolisib expansion studies and napazimone pivotal program
Relan said Pharming completed responses to all outstanding questions from the European Medicines Agency for leniolisib and expects an approval decision in the first half of the year. He added that the company expects additional approvals in Japan and other countries in 2026. Pharming is also running two phase II studies exploring leniolisib in broader PID populations with immune dysregulation, with top-line readouts expected in the second half of the year.
External speaker Dr. Jocelyn Farmer discussed the clinical and biological overlap between APDS and common variable immune deficiency (CVID), emphasizing that CVID is the most common PID worldwide and lacks FDA-approved therapies for non-infectious immune dysregulation complications. She cited data indicating CVID patients with non-infectious complications face an 11-fold higher risk of death and described parallels in clinical presentation and B-cell pathway biology, including PI3K/mTOR signaling. Farmer said work from the New England Immune Deficiency Consortium suggested a large share of CVID patients show an “APDS-like” clinical pattern, which could inform endpoints such as lymphadenopathy, splenomegaly, cytopenias, and inflammatory organ disease.
Dr. Rebecca Marsh said Pharming has treated five CVID patients to date under single patient IND access. She outlined two open-label, dose-escalation phase II studies (12 patients each): one in genetic PIDs linked to PI3K signaling and one in CVID with immune dysregulation. The studies emphasize safety, dose strategy, PK/PD, and preliminary efficacy assessments across clinical manifestations such as lymphoproliferation, cytopenias, lung disease, and liver disease.
On napazimone (KL1333), Pharming and external expert Dr. Amel Karaa described primary mitochondrial disease as a multisystemic, genetically heterogeneous metabolic disorder with no broadly approved therapies and fatigue and muscle weakness as key patient-reported priorities. Program lead Magnus Hansson said the registrational phase II FALCON study is a randomized, blinded, placebo-controlled pivotal design targeting mitochondrial DNA-related disorders that impair oxidative phosphorylation. He said the trial includes two alternative primary endpoints tied to fatigue and lower extremity myopathy, and that an independent data monitoring committee conducted an interim analysis that cleared futility on both primary endpoints and recommended continuation. Management said more than 20 sites are active, enrollment is expected to complete this year, and a readout is planned for late 2027. The company also plans to open an open-label extension in the second quarter of the year.
In closing remarks, Chouraqui reiterated expectations for two leniolisib phase II readouts in the second half of the year and a napazimone pivotal study readout by the end of next year, framing both as potential value drivers alongside continued RUCONEST growth and accelerating Joenja momentum.
About Pharming Group (NASDAQ:PHAR)
Pharming Group N.V. is a clinical-stage biopharmaceutical company headquartered in Leiden, the Netherlands, with a primary focus on developing and commercializing innovative protein replacement therapies for patients living with rare diseases. The company employs a proprietary transgenic technology platform designed to produce recombinant human proteins in the milk of transgenic animals, enabling scalable and cost-efficient manufacturing of complex therapeutic proteins.
The company’s lead product, RUCONEST (recombinant human C1 esterase inhibitor), is approved for the treatment of acute hereditary angioedema (HAE) attacks in multiple markets, including the United States and Europe.
