Pharming Group N.V. (PHAR) reported its first-quarter 2026 financial results, showing lower total revenue year over year but strong growth from Joenja (Ieniolisib), alongside continued regulatory and clinical progress across its rare-disease portfolio.
For Q1 2026, total revenues were $72.4 million, an 8% decrease from $79.1 million in Q1 2025.
Gross profit decreased to $65.8 million from $70.8 million a year earlier. Operating loss narrowed to $4.9 million, compared with $7.0 million in Q1 2025. Net loss improved to $5.2 million, versus $14.9 million in the prior-year quarter, helped by lower tax expense and favorable currency movements.
Cash generated from operations increased to $2.0 million, compared with $0.2 million in Q1 2025.
Pharming ended the quarter with $171.8 million in cash, restricted cash and marketable securities, down from $181.1 million at year-end 2025, mainly due to a $12.3 million lease-termination payment.
Commercial Portfolio
RUCONEST- Acute HAE treatment
RUCONEST revenue declined 15% to $58.4 million, from $68.6 million in Q1 2025. The decline reflected:
- 8% impact from U.S. inventory drawdowns
- 3% impact from withdrawal from non-U.S. markets
- Competitive pressure in the U.S. market.
Despite this, Pharming reported continued new patient enrollments and strong prescriber engagement, with most patients remaining on RUCONEST nine months after the launch of a new oral competitor.
Joenja (Leniolisib)- APDS Treatment
Joenja revenue, however, rose 34% to $14.1 million, up from $10.5 million in Q1 2025. Growth was driven by:
- Higher U.S. patient uptake (127 patients on paid therapy, up 25% year over year)
- Strong uptake in the U.K.
- Expansion of government-supported access programs internationally
The U.S. accounted for 82% of Joenja revenue, with 18% from Europe and the rest of the world.
Pipeline and Regulatory Update
Leniolisib for APDS- Pediatric Label Expansion Pharming resubmitted its pediatric sNDA to the FDA for the 40 mg and 50 mg twice-daily doses following a Complete Response Letter in January. A second sNDA for lower doses is planned for summer 2026.
Europe
The CHMP issued a positive opinion recommending Joenja approval for patients aged 12 and older. A final EC decision is expected in Q2 2026. If approved, Joenja would become the first approved APDS treatment in the EU.
Japan
Japan granted marketing authorization for Joenja for patients aged 4 and older- the first global approval for children aged 4-11.Leniolisib for Additional Primary Immunodeficiencies (PIDs)
Two Phase II trials in PIDs with immune dysregulation have completed enrolment, with top-line data expected in 2H 2026. Compassionate-use data presented at CIS 2026 showed improvements or stabilization in clinical symptoms and quality of life.
Napazimone (KL1333)- Primary Mitochondrial Disease
Enrollment continues in the pivotal FALCON study, with Phase II readouts expected later this year.
2026 Outlook
Pharming reaffirmed its full-year 2026 guidance:
- Total revenues between $405 million and $425 million represents 8% to 13% growth over the $376.1 million reported in 2025. - Total operating expenses between $330 million and $335 million, compared with $311.3 million last year.
- Continued RUCONEST and Joenja growth in the U.S. and internationally
- Additional regulatory approvals and launches expected in Japan, Europe, and the U.S.
- Key clinical readouts for Leniolisib in PIDs and progress in the KL1333 programThe company also reiterated its focus on disciplined capital allocation and potential in-licensing or acquisition opportunities in rare diseases.
PHAR has traded between $8.69 and $16.74 over the past year. The stock closed Wednesday's trading at $16.40, up 1.42%. In pre-market trading the stock is at $13.18, down 19.63%.
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