Pharming Group Reports First Quarter 2026 Financial Results On Track For Joenja® U.S. Pediatric Label Expansion And Launches In Japan And Europe In 2026

• First quarter 2026 total revenues were US$72.4 million, an 8% decrease compared to the first quarter 2025
• RUCONEST® 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® 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® approved in Japan and received positive CHMP opinion for APDS
• Resubmitted pediatric sNDA to the FDA for Joenja® (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®. Joenja® 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® 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® nine months after the launch of a new oral treatment. New patient enrollments and growing prescriber engagement further validate RUCONEST®'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® marketed for the treatment of acute HAE attacks

RUCONEST® revenue in the first quarter of 2026 was US$58.4 million, a 15% decrease compared to the first quarter of 2025. RUCONEST® 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® 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® 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®.

Joenja® (leniolisib) marketed for the treatment of APDS

Joenja® 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®, while 57 are between 4 and 11 years of age.

Joenja® (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® (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® (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® 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® for the treatment of APDS in adult and pediatric patients aged 4 years and older. Joenja® 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 PI3Kδ 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

Underlying figures are unrounded. Therefore, totals may differ slightly from the sum of individual items due to rounding effects in the presentation of this press release.

Financial highlights
For the first quarter of 2026, total revenues decreased by US$6.6 million, or 8%, to US$72.4 million, compared to US$79.1 million in the first quarter of 2025. RUCONEST® revenues amounted to US$58.4 million, a 15% decrease compared to the first quarter of 2025. This decrease in RUCONEST® revenues was primarily driven by volume decreases in the U.S. and internationally, following our decision to withdraw from all non-U.S. markets. Joenja® revenues amounted to US$14.1 million in the first quarter of 2026, a 34% increase compared to the first quarter of 2025. This increase in Joenja® revenues was primarily driven by an increase in volume.

Gross profit decreased by US$5.0 million or 7% to US$65.8 million (1Q 2025: US$70.8 million), mainly due to the decrease in revenues.

The operating loss amounted to US$4.9 million compared to an operating loss of US$7.0 million in the first quarter of 2025. Excluding US$7.8 million of non-recurring Abliva acquisition-related expenses, the first quarter of 2025 would have reflected adjusted operating profit of US$0.8 million. The adjusted operating profit declined due to the decrease in revenues, increased R&D spending mainly related to the addition of napazimone (KL1333) to our pipeline, and unfavorable currency translation effects, partially offset by other expense savings.

The finance result (net) and share of result in associates amounted to a gain of US$0.2 million compared to a loss of US$4.8 million in the first quarter of 2025. This improvement was primarily driven by favorable EUR/USD exchange rate movements, resulting in a foreign currency gain of US$2.4 million, compared to a loss of US$2.6 million in the first quarter of 2025.

In the first quarter of 2026, a net loss of US$5.2 million was realized, compared to a net loss of US$14.9 million in the first quarter of 2025. In addition to the aforementioned drivers, the net result was positively impacted by a lower tax expense of US$0.5 million compared to a tax expense of US$3.1 million in the first quarter of 2025.

Cash generated from operations amounted to US$2.0 million, compared to US$0.2 million in the first quarter of 2025. Cash and cash equivalents, including restricted cash and marketable securities, decreased from US$181.1 million at the end of fourth quarter of 2025 to US$171.8 million at the end of the first quarter of 2026. This decrease was primarily driven by a US$12.3 million settlement of the lease liability following the early termination of the DSP facility lease at Pivot Park in Oss, the Netherlands.

Outlook/Summary

For 2026, the Company anticipates:

• Total revenues between US$405 million and US$425 million (8% to 13% growth), with quarterly fluctuations expected.
• Total operating expenses between US$330 million and US$335 million (6% to 8% growth), including US$60 million incremental R&D expenses to advance the pipeline and US$9 million structural G&A cost reductions based on the plan announced in October 2025.
• Continued RUCONEST® growth, and significant and accelerating Joenja® U.S. and ex-U.S. growth.
• Progress towards additional regulatory approvals and commercial launches for leniolisib for APDS patients 12 years of age or older and for pediatric label expansion in key global markets.
• Top-line data readouts for the two ongoing leniolisib Phase II clinical trials in PIDs with immune dysregulation to expand the asset's addressable patient population.
• Completion of enrollment in the pivotal FALCON clinical study for napazimone (KL1333) in mitochondrial DNA-driven primary mitochondrial diseases.
• Enhancing capital allocation to drive growth and build a leading global rare disease company.
• Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases. Financing, if required, would come via a combination of our strong balance sheet and access to capital markets.

No further specific financial guidance for 2026 is provided.

Additional information
Presentation

The conference call presentation is available on the Pharming website from 07:30 CEST today.

Conference Call

The conference call will begin at 13:30 CEST / 07:30 EDT on Thursday, May 7. A transcript will be made available on the website in the days following the call.

Please note, the Company will only take questions from dial-in attendees.

Webcast Link:

Conference call dial-in details:

Additional information on how to register for the conference call/webcast can be found on website.

Financial Calendar 2026

Annual General Meeting of Shareholders May 28
2Q/1H 2026 financial results July 30
3Q 2026 financial results November 5

For further public information, contact:

Investor Relations
Michael Levitan, VP Investor Relations and Capital Markets
T: +1 (908) 705 1696
E: ...

Media Relations
Global: Saskia Mehring, Head of Corporate Communications
T: +31 6 28 32 60 41
E: ...

U.S.: Ethan Metelenis (Precision AQ on behalf of Pharming)
T: +1 (917) 882-9038

Netherlands: Leon Melens (LifeSpring Life Sciences Communication on behalf of Pharming)
T: +31 6 53 81 64 27

About Pharming Group N.V.

Pharming Group N.V. (EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. We develop and commercialize innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, with U.S. and European operations.

For more information, visit and find us on LinkedIn.

Forward-looking Statements

This press release may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management's current expectations and assumptions and involve known and unknown risks and uncertainties that could cause actual results, performance, or events to differ materially from those expressed or implied in these statements. These forward-looking statements are identified by their use of terms and phrases such as“aim”,“ambition”, ''anticipate'', ''believe'', ''could'', ''estimate'', ''expect'', ''goals'', ''intend'', ''may'',“milestones”, ''objectives'', ''outlook'', ''plan'', ''probably'', ''project'', ''risks'',“schedule”, 'seek'', 'should'', ''target'', ''will'' and similar terms and phrases. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory, commercial, competitive and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2025 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. All forward-looking statements contained in this press release are expressly qualified in their entirety by the cautionary statements contained or referred to in this section. Readers should not place undue reliance on forward-looking statements. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release. Pharming does not undertake any obligation to publicly update or revise any forward-looking statement as a result of new information, future events or other information.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

Pharming Group N.V.

Condensed Consolidated Interim Financial Statements in US Dollars (unaudited)
For the period ended March 31, 2026

• Condensed consolidated statement of income
• Condensed consolidated statement of comprehensive income
• Condensed consolidated balance sheet
• Condensed consolidated statement of changes in equity
• Condensed consolidated statement of cash flow
  

Attachment

• Pharming Group reports 1Q26 results_EN_07MAY26

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