Pharming Group (NASDAQ:PHAR) executives outlined commercial performance, pipeline priorities, and regulatory updates during a presentation at Oppenheimer’s 36th Annual Healthcare Conference, highlighting continued growth from its hereditary angioedema (HAE) franchise and expansion efforts for its immune deficiency therapy.
Company overview and commercial performance
Chief Financial Officer Kenneth Lynard said Pharming combines marketed products with pipeline assets the company believes are positioned for future value creation. He pointed to RUCONEST, which has been on the market for about 10 years, and Joenja, launched in 2023 for activated PI3K delta syndrome (APDS), as key commercial drivers.
Looking ahead, Lynard said Pharming expects 2026 revenues of $405 million to $425 million, or 8% to 13% growth. He noted this guidance excludes any contribution from potential U.S. sales of Joenja in pediatric patients aged four to 11, which would depend on U.S. Food and Drug Administration (FDA) approval and could represent upside.
For the first nine months of the year, Lynard said Pharming generated operating profit of $30 million and operating cash flow of $44 million, which he characterized as a significant improvement versus prior years. He said full-year details will be provided during the company’s earnings call scheduled for March 12.
RUCONEST: differentiated profile and market “stickiness”
Chief Medical Officer Anurag Relan reviewed RUCONEST’s role in treating acute attacks of hereditary angioedema, describing it as the only recombinant C1 inhibitor replacement therapy available. He said it is administered intravenously, is labeled for self-administration, and offers immediate bioavailability and rapid onset of action.
Relan highlighted clinical trial data showing that one dose was sufficient to treat 97% of attacks, and that patients experienced no symptoms in 93% of treated attacks lasting at least three days. He also discussed RUCONEST use in patients with HAE with normal C1 inhibitor, describing this population as having more severe attacks and, in some cases, failure on other on-demand therapies.
Relan also pointed to RUCONEST’s manufacturing process—production in the milk of transgenic rabbits—saying it makes the product less susceptible to biosimilar development.
In addition, Relan discussed an indirect treatment comparison presented at the American College of Allergy, Asthma and Immunology meeting in November, comparing RUCONEST with sebetralstat. He emphasized the comparison was not head-to-head and said it showed a significantly greater proportion of attacks achieving complete symptom resolution by 24 hours with RUCONEST. He said patients were “four and a half times more likely” to achieve complete symptom resolution with RUCONEST than with sebetralstat in that analysis.
In a Q&A session, Oppenheimer analyst Jeff Jones asked why RUCONEST demand has remained resilient despite oral options entering the market. Relan said Pharming often sees RUCONEST patients as those who have taken a “long journey” through multiple prophylactic agents and at least one acute agent before arriving at RUCONEST. He suggested that once these patients find a therapy that manages their disease, they may be less willing to switch to another option that may not work for them.
Joenja and leniolisib: APDS and expansion into related immune disorders
Relan reviewed Joenja (leniolisib) and the biological rationale for targeting PI3K delta signaling, describing PI3K delta as a master regulator of immune cell processes. He said hyperactivity in this pathway leads to clinical complications associated with APDS, including lymphadenopathy and splenomegaly, autoimmune complications, gastrointestinal disease, and lung disease.
Relan provided a patient example from the leniolisib clinical trial program, describing improvements over more than six years of treatment, including cessation of immunoglobulin replacement therapy, improved fatigue, no hospitalizations, and improvements in blood counts.
Relan also discussed Pharming’s view of APDS prevalence, citing an estimate of approximately one to two patients per million and noting that about 30% of patients are pediatric and currently outside the label. He described efforts to reclassify patients with inconclusive genetic testing results and referenced a publication in Cell suggesting APDS prevalence could be higher due to additional gene variants leading to pathway hyperactivity.
Pharming is also studying leniolisib in two phase II proof-of-concept programs beyond APDS:
- Primary immune deficiencies with immune dysregulation related to abnormal signaling
- Common variable immune deficiency with immune dysregulation
Relan said six patients have been treated under an expanded access program in other primary immune deficiencies, with “general improvements” across endpoints including biomarkers, lymphoproliferation, organ disease, and measures such as well-being and fatigue. He emphasized the expanded access experience was not a clinical trial but supported the rationale for formal studies. He said both phase II studies are underway, with readouts expected in the second half of this year.
Napazimone (formerly KL-1333): mitochondrial disease program and timeline
Relan also discussed napazimone, previously known as KL-1333, which Pharming obtained through its acquisition of Abliva. He said the therapy is being developed for primary mitochondrial disease driven by mitochondrial DNA mutations and could become a first disease-modifying treatment for these patients.
Relan said napazimone is intended to address an abnormal NAD+ to NADH ratio associated with impaired mitochondrial function, and he cited evidence from in vitro data, animal models, and patients. He described the addressable population as more than 30,000 diagnosed patients with mtDNA disorders across the U.S. and Europe, and noted that most are treated at centers of excellence.
Relan outlined the ongoing registrational Phase II FALCON study, noting it includes clinically relevant endpoints of fatigue and muscle strength and that the company has had discussions with FDA on endpoints. He said a positive interim analysis showed both primary endpoints “cleared futility,” allowing the study to continue, and that the data monitoring committee reviewed safety and tolerability and permitted continuation without design changes. He said the trial’s sample size has been confirmed at 180 patients, with patients treated for 48 weeks. Relan said Pharming expects to complete enrollment this year and expects readout at the end of next year.
Regulatory updates and 2026 expense outlook
Relan said Pharming recently received a Complete Response Letter for the supplemental NDA seeking to expand leniolisib use to APDS patients aged four to 11 years, and that the company has requested a Type A meeting with FDA to discuss next steps.
In Europe, Relan said Pharming has responded to CHMP questions, and that the European Medicines Agency canceled a previously scheduled oral explanation as unnecessary. He said the company now expects a March CHMP opinion and potential approval about two months later, which would fall in the first half of the year. He added that reviews in Japan are also ongoing and “on track” for approval later this year.
On the financial side, Lynard reiterated 2026 operating expense guidance of $330 million to $335 million, up from the company’s 2025 expense outlook. He said the company expects about $60 million of incremental R&D investment in 2026 to advance its pipeline and referenced $9 million of structural G&A cost reductions announced in October 2025, along with other optimizations, as helping moderate overall expense growth.
Asked about commercial planning in Europe following a strategic move to withdraw from RUCONEST commercialization outside the U.S., Lynard said Pharming has not eliminated all infrastructure and can leverage parts of it as it builds the Joenja franchise, though additional investment may still be required as the business grows.
Lynard also said business development remains a key element of Pharming’s strategy, with an active team evaluating in-licensing and M&A opportunities on a selective basis.
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.
