Pharming Group Q2 Earnings Call Highlights

Key Points

• Pharming cut its 2026 outlook, forecasting revenue of $375 million to $395 million after second-quarter revenue fell 3% to $90.2 million. The company also lowered its operating-expense forecast by $15 million, while expecting RUCONEST sales to stabilize and resume growth in the second half of 2026.
• Joenja remained the growth driver, with second-quarter revenue up 40% to $17.9 million. Pharming is advancing launches in Germany and Japan, pursuing additional pediatric doses in the U.S., and expects high-30% Joenja revenue growth in 2026.
• Pharming expects fourth-quarter Phase II results for leniolisib in broader primary immunodeficiency conditions, including CVID, which management estimates could represent an addressable population up to 40 times larger than APDS. Despite positive operating profit, cash and marketable securities declined to $159.5 million at quarter-end.

Pharming Group NASDAQ: PHAR reported second-quarter revenue of $90.2 million, down 3% from a year earlier, as growth from its Joenja treatment partly offset lower sales of hereditary angioedema therapy RUCONEST. The company reduced its full-year revenue outlook by $30 million but said it expects RUCONEST sales to stabilize and return to growth during the second half of 2026.

The company now expects 2026 revenue of $375 million to $395 million, representing approximately flat to 5% growth versus 2025. It also lowered its operating-expense forecast by $15 million to a range of $315 million to $320 million.

Management said the revised outlook reflects a clearer view of the competitive hereditary angioedema, or HAE, market nearly a year after the launch of the first oral on-demand treatment. Pharming continues to position RUCONEST as an on-demand option for patients with frequent or severe attacks, while seeking to expand Joenja in activated phosphoinositide 3-kinase delta syndrome, or APDS, and potentially in broader immune-deficiency indications.

RUCONEST Sales Decline, but Patient Metrics Improve

RUCONEST revenue fell 10% year over year to $72.3 million in the second quarter, though sales rose 24% sequentially. Chief Commercial Officer LaVerne Marsh said the year-over-year decline reflected U.S. competitive dynamics, the completion of Pharming’s planned withdrawal from international markets, and temporary inventory normalization.

Marsh said the active RUCONEST patient base remained at approximately 93% of its level a year earlier. The company recorded 84 new patient enrollments during the quarter, compared with about 50 in the first quarter and close to the level reported in the second quarter of 2025. Pharming also added 17 new prescribers.

“The resilient patient base, improvement in new enrollments, and continued addition of prescribers support our belief that revenue will stabilize,” Marsh said, adding that RUCONEST is expected to return to growth in the second half.

Management said new RUCONEST enrollments increasingly involve patients with high disease burdens, including those with frequent or severe attacks and concerns about high-risk attack locations. Marsh also said the new-enrollment figure excludes patients restarting therapy.

In response to an analyst question, Chief Financial Officer Kenneth Lynard said the company expects inventory patterns to be within normal seasonal ranges for the rest of 2026 after inventory reductions earlier in the year.

Joenja Growth Continues as International Launches Advance

Joenja revenue rose 40% year over year to $17.9 million. U.S. revenue increased 31% to $15.4 million, while international revenue climbed 150% to $2.5 million.

At the end of the quarter, 132 patients were on paid Joenja therapy in the U.S., up 16% from a year earlier and by five patients from the first quarter. The number of identified U.S. APDS patients rose by 16 during the quarter to 298, including 60 patients between ages 4 and 11.

Pharming is awaiting an October 24 PDUFA date for U.S. approval of higher 40-milligram and 50-milligram pediatric doses of Joenja. Marsh said the company submitted a supplemental new drug application for lower 20-milligram and 30-milligram doses on the day of the call.

Outside the U.S., the company launched Joenja in Germany on July 1 and said it is preparing to launch in Japan during August after progressing through pricing and reimbursement steps. Marsh said Pharming had identified approximately 387 potentially eligible APDS patients across eight core markets outside the U.S.

Management expects Joenja’s annual revenue to grow in the high-30% range in 2026, supported by Germany, Japan and the anticipated U.S. pediatric expansion. Lynard said this compares with 29% growth in 2025.

Phase II Readouts Expected in Fourth Quarter

Chief Medical Officer Anurag Relan said Pharming’s two fully enrolled Phase II studies of leniolisib, the active ingredient in Joenja, are expected to report top-line results in the fourth quarter. The programs are evaluating the treatment in broader primary immunodeficiency populations, including common variable immunodeficiency, or CVID, with immune dysregulation.

The CVID study includes 20 patients across multiple centers, while a second 12-patient basket study at the National Institutes of Health is examining genetically defined primary immunodeficiencies associated with immune dysregulation. The studies are assessing lymph node and spleen size, blood-cell counts, liver and lung involvement, patient and clinician-reported outcomes, and biomarkers.

Relan said Pharming currently anticipates that positive Phase II results could support a single registrational Phase III study in a broader CVID population. The company said the potential CVID addressable population could be up to 40 times larger than APDS, though management said it does not expect CVID sales before a Phase III trial and regulatory approval.

Pharming also expects results in 2027 from the pivotal FALCON study of napazimone in patients with primary mitochondrial disease.

Profitability and Cash Position

Lynard said reported and adjusted operating profit were positive in the second quarter despite lower revenue and nearly $9 million in incremental investments compared with the prior-year quarter. Adjusted operating profit declined by $5 million year over year, primarily due to lower revenue and higher research and development spending.

Operating cash flow was an outflow of $9.7 million in the quarter, driven by working-capital changes, including a strategic inventory build, and operating performance. Cash and marketable securities totaled $159.5 million at quarter-end, down $12.3 million from the end of the first quarter.

For the first half, revenue totaled $162.7 million, down 6% year over year. Joenja revenue increased 37%, while RUCONEST revenue declined 12%. The company increased research and development investment by $13 million in the first half while adjusted operating expenses rose 1%.

Pharming expects cost of goods sold, including a $4.9 million manufacturing-inventory impairment recorded during the second quarter, to be approximately 11% of full-year revenue, implying a gross margin of about 89%. Lynard said the impairment related to RUCONEST manufacturing inventory that may not be available for commercial use, with the company expecting to conclude its assessment before the end of the third quarter.

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.

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