Pharming Group (NASDAQ:PHAR) reported third-quarter revenue growth of 21% to $117.6 million, supported by higher sales of its RUCONEST hereditary angioedema treatment and Joenja therapy for activated phosphoinositide 3-kinase delta syndrome, or APDS.
Kenneth Lynard, interim co-CEO and chief financial officer, said the company released preliminary results earlier than its originally planned Nov. 5 reporting date because revenue and operating expenses came in above analyst consensus. He said the accelerated disclosure was not related to a transaction or restructuring.
The company also said its strategy, pipeline and commitment to patients remain unchanged following the Sept. 29 departure of former CEO Fabrice Chouraqui. Lynard and Leverne Marsh, chief commercial officer, are serving as interim co-CEOs while the board conducts a search for a permanent successor.
RUCONEST Returns to Growth
RUCONEST revenue rose 14% from a year earlier to $93.8 million during the quarter. Marsh said underlying U.S. demand stabilized, with volume returning to prior-year levels after declines reported in the first and second quarters.
However, the quarter’s revenue growth also included a seasonal increase in channel inventory that occurred earlier than expected. Marsh said the inventory movement contributed approximately 9 percentage points of RUCONEST’s 14% year-over-year revenue growth.
The company said it expects demand momentum to continue into the fourth quarter, though revenue could be affected by inventory timing. Marsh said Pharming has retained more than 90% of its active RUCONEST patient base more than a year after the launch of an oral on-demand competitor. Retention has been particularly strong among patients with more frequent or severe attacks, according to the company.
During the question-and-answer session, Marsh said new patient enrollments naturally vary by quarter and that the third quarter has historically been softer than the second quarter. She said the company considers patient retention, new enrollments and vial volumes collectively when assessing demand.
For 2027, Pharming is planning for the potential impact of another oral competitor that currently has a stated PDUFA date at the end of April, Marsh said. The company expects a pattern similar to the impact seen following the introduction of the first oral competitor.
Joenja Sales Rise as International and Pediatric Expansion Continues
Joenja revenue increased 58% year over year to $23.8 million. U.S. sales rose 19% to $15.9 million, while international revenue increased to $7.9 million from $1.7 million a year earlier.
Pharming ended the quarter with 136 U.S. patients on paid Joenja therapy, up 17% from a year earlier and four patients above the second-quarter total. Internationally, 75 patients were receiving paid therapy, including 30 in launch markets and 45 through paid-access programs in other markets.
The company launched Joenja in Germany in July and Japan in August, building on its presence in the U.K. Marsh said Japan became the first market where Joenja is commercially available for children ages 4 through 11.
International Joenja revenue included $2 million to $3 million in earlier-than-expected revenue recognition due to ordering patterns in certain markets, which the company had originally expected in the fourth quarter.
In the U.S., the Food and Drug Administration approved higher pediatric doses of Joenja on Sept. 11 for children ages 4 through 11 weighing at least 27 kilograms. Shipments began in October, so the third-quarter results did not include any contribution from the newly approved doses.
Pharming has identified more than 60 U.S. children ages 4 through 11 with APDS. The approved doses address 38 of those patients, and the company expects an initial fourth-quarter addition of 15 to 20 pediatric patients on therapy. A separate application for lower doses for children weighing 13 to less than 27 kilograms has a Jan. 30, 2027, PDUFA date after receiving Priority Review.
Pipeline Updates Include CVID Readout Expected in November
Chief Medical Officer Anurag Relan said Pharming expects to report top-line data in November from a 20-patient, single-arm, open-label study of leniolisib in common variable immune deficiency, or CVID, with immune dysregulation.
The company will evaluate lymphoproliferation, cytopenias, lung and liver disease, biomarkers, safety and clinician- and patient-reported measures. Relan said Pharming will look for a consistent direction of benefit across these measures and identify a dose to take into a potential larger study.
Pharming recently presented data from a Phase II study in genetically defined primary immune deficiencies with immune dysregulation. The trial enrolled 13 participants, with 12 completing treatment. The company said leniolisib was associated with improvements in spleen volume, lymph node size, lung gas exchange and liver stiffness, with no new safety signals reported. All 12 participants completing the study entered a three-year extension study.
The company said it anticipates a single Phase III study in the broader CVID indication that could incorporate patients from both of its Phase II programs. Relan noted that the physicians treating APDS patients are largely the same immunologists who treat CVID and other genetic primary immune deficiencies.
Pharming also said its registrational-enabling FALCON study of napazimone, or KL1333, in mitochondrial DNA-driven primary mitochondrial disease continues to enroll patients. More than 50 sites are recruiting, and the company expects 145 of the planned 180 participants to be enrolled by year-end, with enrollment completed in the first half of 2027. Top-line results are expected about one year after enrollment concludes.
Profitability and Outlook
Third-quarter operating profit increased 89% to $29.9 million. Excluding a $5.1 million one-time manufacturing-related inventory impairment, adjusted operating profit was $35 million, compared with $16 million a year earlier. Net profit was $22.4 million, compared with $7.5 million in the prior-year period.
Operating cash flow totaled $32.4 million, and cash and marketable securities increased by $31.4 million during the quarter to $190.9 million.
For full-year 2026, Pharming tightened revenue guidance to $380 million to $390 million, representing growth of 1% to 4%, and lowered operating-expense guidance to $310 million to $315 million. The company said its cash position and expected future operating cash flows are expected to cover current pipeline costs and pre-launch expenses without additional financing.
About Pharming Group (NASDAQ:PHAR)
Pharming Group N.V. is a biopharmaceutical company headquartered in Leiden, the Netherlands. The company develops and commercializes medicines for rare diseases and operates internationally, serving patients and healthcare providers in North America, Europe and other selected markets.
Pharming’s principal product is Ruconest, a recombinant C1 esterase inhibitor used to treat acute attacks of hereditary angioedema, a rare genetic disorder that can cause potentially serious swelling. The company also markets Joenja (leniolisib), an oral treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
