-- First quarter 2026 total revenues were US$72.4 million, an 8% decrease
compared to the first quarter 2025
-- RUCONEST$(R)$ revenue was US$58.4 million, a 15% decrease compared to the
first quarter 2025, mainly due to anticipated inventory drawdowns and the
planned exit from non-U.S. markets
-- Joenja(R) revenue was US$14.1 million, a 34% increase compared to the
first quarter of 2025, reflecting strong U.S. and international momentum
-- Reaffirmed 2026 total revenue guidance of US$405 - US$425 million (8% -
13% growth)
-- Generated positive net cash flow from operations of US$2.0 million in the
quarter
-- Joenja(R) approved in Japan and received positive CHMP opinion for APDS
-- Resubmitted pediatric sNDA to the FDA for Joenja(R) (leniolisib) for
highest doses; plan additional sNDA this summer for lowest doses
-- Pharming to host a conference call today at 13:30 CEST (7:30 am EDT)
Leiden, the Netherlands, May 7, 2026: Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary unaudited financial report for the three months ended March 31, 2026.
Chief Executive Officer, Fabrice Chouraqui commented:
"The first quarter demonstrated important progress across the business while also reflecting revenue variability for RUCONEST(R). Joenja(R) delivered strong revenue growth of 34% year over year, driven by robust patient uptake, reinforcing its role as an important growth driver still early in its lifecycle. We also made meaningful regulatory progress, including approval in Japan for APDS patients aged 4 and older and a positive CHMP opinion in Europe. In the U.S., constructive dialogue with the FDA following receipt of the CRL enabled us to already resubmit our pediatric sNDA for the two highest doses, which cover a meaningful proportion of children aged 4 to 11, and plan an additional sNDA submission for the lowest doses this summer.
First--quarter RUCONEST(R) revenue was impacted by several factors we had largely anticipated and incorporated into our full-year guidance, notably specialty pharmacy inventory drawdowns and our strategic exit from non-U.S. markets. We continue to see the overwhelming majority of patients stay on RUCONEST(R) nine months after the launch of a new oral treatment. New patient enrollments and growing prescriber engagement further validate RUCONEST(R)'s strong value proposition for high-burden patients.
We also advanced our key pipeline value drivers, including enrollment in the pivotal napazimone (KL1333) trial in primary mitochondrial disease and preparations for Phase II readouts later this year for leniolisib in broader primary immunodeficiencies. The encouraging compassionate-use experience with leniolisib in patients with CVID and immune dysregulation, which will be presented today at the Clinical Immunology Society Annual Meeting, further supports our view on the program's potential.
Finally, our focus on financial discipline also delivered tangible results, including positive net cash flow from operations in the quarter despite quarterly revenue variability. Taken together, our commercial execution, regulatory progress, pipeline advancement, and disciplined financial management position Pharming well for sustained long-term growth and value creation."
First quarter 2026 highlights
Commercialized products
RUCONEST(R) marketed for the treatment of acute HAE attacks
RUCONEST(R) revenue in the first quarter of 2026 was US$58.4 million, a 15% decrease compared to the first quarter of 2025. RUCONEST(R) revenue in the current quarter compared to the first quarter of 2025 was impacted by inventory drawdowns at U.S. specialty pharmacy customers, which were anticipated and reduced revenue by 8%, our previous decision to withdraw the product from non-U.S. markets, which reduced revenue by 3%, and competitive market dynamics in the U.S.
With its efficacy, reliability and rapid onset of action via IV administration, RUCONEST(R) remains a trusted on-demand treatment option for patients experiencing more severe or frequent attacks who have failed other on-demand medications. We continued to enroll new patients and add new prescribers in the quarter, with the vast majority of RUCONEST(R) patients showing limited interest in trialing or switching to alternative treatment options. Among patients who have trialed the new oral treatment option, we have observed that a meaningful proportion, particularly those experiencing more frequent attacks, have already made the decision to return to RUCONEST(R).
Joenja(R) (leniolisib) marketed for the treatment of APDS
Joenja(R) revenue increased to US$14.1 million in the first quarter of 2026, a 34% increase compared to the first quarter of 2025. Revenue growth was driven by a significant increase in patients on paid therapy in the U.S., offset by greater inventory drawdowns in the current quarter, and increased demand in international markets, including strong patient uptake in the U.K. following the April 2025 launch and significant growth in the number of patients on government-supported access programs.
The U.S. market contributed 82% of first quarter revenue, while the EU and Rest of World contributed 18%.
As of March 31, 2026, 127 patients were on paid therapy in the U.S., representing a 25% increase from the 102 patients at the end of the first quarter of 2025 and an increase of seven patients during the quarter.
APDS patient finding
As of March 31, 2026, we have identified 1,016 diagnosed APDS patients of all ages globally, including 282 patients in the U.S. and 385 in core markets outside of the U.S. Of the identified patients in the U.S., 187 patients are 12 years of age or older and currently eligible for treatment with Joenja(R), while 57 are between 4 and 11 years of age.
Joenja(R) (leniolisib) development
Leniolisib for APDS
As of March 31, 2026, there are 180 APDS patients in either a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a paid access program.
Pediatric label expansion
On January 30, 2026, we received a Complete Response Letter $(CRL)$ from the U.S. Food and Drug Administration (FDA) regarding our supplemental New Drug Application (sNDA) for Joenja(R) (leniolisib) for the treatment of APDS in children aged 4 to 11 years.
We held a Type A meeting with the FDA on March 26, 2026, to discuss the issues outlined in the letter and align on a path forward for resubmission. Upon the receipt of written feedback received from the FDA in the form of meeting minutes, we resubmitted the sNDA for the 40 mg and 50 mg twice-daily doses which would cover a meaningful proportion of the identified patient population, and plan to file a separate sNDA this summer for patients requiring a lower dose.
European Economic Area $(EEA)$
On March 26, 2026, the CHMP adopted a positive opinion recommending marketing authorization for Joenja(R) (leniolisb) in adult and pediatric patients aged 12 years and older. A final decision by the European Commission $(EC)$ on the marketing authorization is expected in the second quarter of 2026.
If approved, Joenja(R) would become the first approved treatment for APDS in the European Union. The centralized marketing authorization would be valid in all 27 European Union Member States, as well as Norway, Iceland and Liechtenstein.
Japan
In March 2026, the Japanese Ministry of Health, Labour and Welfare (MHLW) granted marketing authorization for Joenja(R) for the treatment of APDS in adult and pediatric patients aged 4 years and older. Joenja(R) is the first approved treatment for APDS in Japan and this approval is the first anywhere globally for children aged 4 to 11.
Leniolisib for additional primary immunodeficiencies (PIDs)
Two Phase II clinical trials are evaluating leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, including genetically identifiable PIDs linked to altered PI3K signaling and common variable immunodeficiency or CVID, which represent substantially larger patient populations than APDS. Patient enrollment in both clinical trials is complete and we anticipate trial read-outs in the second half of 2026, consistent with prior guidance.
A presentation at the 2026 Annual Meeting of the Clinical Immunology Society (CIS), taking place May 6-9, includes clinician expanded access experience with leniolisib to treat immune dysregulation in patients with CVID and CVID-like disorders. Clinician-reported outcomes demonstrated improvements, or no change or progression, in clinical manifestations of immune dysregulation and improvements in patients' quality of life.
Organizational updates
On January 1, 2026, Leverne Marsh joined the Company as Chief Commercial Officer, succeeding Stephen Toor.
Financial summary
Consolidated Statement of Income 1Q 2026 1Q 2025 ----------------------------------------- ------- ------- Amounts in US$m except per share data ----------------------------------------- ------- ------- Total Revenues 72.4 79.1 ----------------------------------------- ------- ------- Cost of sales (6.6) (8.3) ----------------------------------------- ------- ------- Gross profit 65.8 70.8 ----------------------------------------- ------- ------- Other income 0.4 0.4 ----------------------------------------- ------- ------- Research and development (25.6) (21.1) ----------------------------------------- ------- ------- General and administrative (15.2) (22.5) ----------------------------------------- ------- ------- Marketing and sales (30.3) (34.6) ----------------------------------------- ------- -------
(MORE TO FOLLOW) Dow Jones Newswires
May 07, 2026 01:00 ET (05:00 GMT)
Comments