Full Press Release Details
Second quarter 2025 total revenues increased by 26% to US$93.2 million, compared to the second quarter 2024, driven by strong RUCONEST® and Joenja® revenue growth
RUCONEST® second quarter revenue increased by 28% to US$80.4 million, compared to the second quarter 2024, reflecting strong growth in patients and prescribers
Joenja® (leniolisib) second quarter revenue increased by 15% to US$12.8 million, compared to the second quarter 2024, with a further acceleration in patient uptake
Second quarter operating profit amounted to US$10.8 million compared to a US$3.1 million loss in the second quarter 2024
Study published in leading peer-reviewed journal Cell identifies new variants leading to PI3Kδ pathway hyperactivity, supporting reclassification of VUS patients to APDS and suggesting up to a 100-fold increase in APDS prevalence
Submitted new drug application for leniolisib for the treatment of APDS in Japan
2025 total revenue guidance raised to US$335 million - US$350 million, up from prior US$325 million - US$340 million
Overall cash and marketable securities increased to US$130.8 million at the end of the second quarter 2025 from US$108.9 million at the end of the first quarter 2025, primarily due to cash generated from operations
Pharming to host a conference call today at 13:30 CEST (7:30 am EDT)
Leiden, the Netherlands, July 31, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM/Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the second quarter and first half year ended June 30, 2025.
Chief Executive Officer, Fabrice Chouraqui, commented:
“We’ve delivered a strong second quarter, with 26% total revenue growth and positive operating profit, building further on our momentum. RUCONEST® continues to show very significant growth, reflecting its unique value proposition for patients in the on-demand HAE market. We’re also seeing continued acceleration in Joenja® uptake, with first half 2025 patient growth already surpassing the total for all of 2024. Based on our strong first half performance and prospects for the second half of the year, we are raising our full-year revenue guidance.
The newly published study in the peer-reviewed journal Cell highlights the potential to significantly expand Joenja®’s addressable APDS patient population. The data support reclassifying patients with variants of uncertain significance (VUS) as APDS beginning in the second half of 2025. Notably, the study also suggests APDS prevalence may be up to 100 times higher than previously thought.
In recent months, we’ve made meaningful progress in executing our strategy. We launched Joenja® in the U.K., with the first patients now on commercial therapy, and we submitted a regulatory filing for leniolisib in Japan. Enrollment in both proof-of-concept trials studying leniolisib in larger PID indications is progressing well. Finally, we dosed the first patients in the second wave of the pivotal FALCON clinical trial for KL1333 in primary mitochondrial diseases.
We are working on options to mitigate the impact of recently announced U.S. tariffs. Although some uncertainties remain—such as potential tariff exclusions—we do not expect a material impact on our business.
The strength of our commercial portfolio and advancing pipeline—targeting significant, underserved markets—continues to validate our long-term strategy. We are also on track to deliver on the previously announced reduction in G&A expenses, enhancing our ability to invest in future growth. I’m grateful to our teams whose expertise and commitment drive our progress and success.”
Second quarter and first half 2025 highlights
Commercialized assets
RUCONEST® marketed for the treatment of acute HAE attacks
The strong RUCONEST® growth continued in the second quarter of 2025, with revenue of US$80.4 million, a 28% increase compared to the second quarter of 2024. Revenue for the first half of 2025 was US$149.0 million, a 37% increase compared to the same period in 2024.
In the U.S. market, we continue to grow the prescriber and patient base and see strong in-market demand over 10 years post-launch, reflecting RUCONEST®’s unique position in the on-demand HAE market. Unit sales volume in the U.S. increased by 27% in the second quarter and 31% in the first half.
Joenja® (leniolisib) marketed for the treatment of APDS
Joenja® revenue increased to US$12.8 million in the second quarter of 2025, a 15% increase compared to the second quarter of 2024. Revenue for the first half of 2025 was US$23.3 million, a 13% increase compared to the same period in 2024. Unit sales volume increased by 10% in the first half of 2025, primarily due to the increase in patients on paid therapy in the U.S.
The U.S. market contributed 92% of second quarter revenues, while the EU and Rest of World contributed 8%.
As of June 30, 2025, we had 114 patients on paid therapy in the U.S., representing a 25% increase from the 91 patients at the end of the second quarter of 2024. We made strong progress finding, enrolling and transitioning additional patients to paid therapy during the second quarter, increasing the number of patients by 12 versus 6 in the first quarter of this year. The increase of 18 patients during the first half of 2025 exceeded the total increase in patients in 2024. We expect that reclassifications of patients with a variant of uncertain significance, or VUS, to APDS will contribute to additional patient growth beginning in the second half of this year.
In April, we launched Joenja® in the U.K., with the National Institute for Health and Care Excellence (NICE) issuing positive final guidance recommending Joenja® (leniolisib) for reimbursement and use within the National Health Service (NHS) in England and Wales for adult and pediatric patients 12 years of age and older. The first patients are now on commercial therapy in the U.K.
APDS patient finding
As of June 30, 2025, we have identified 971 diagnosed APDS patients of all ages globally, including 257 patients in the U.S. Of the identified patients in the U.S., 165 patients are 12 years of age or older and eligible for treatment with Joenja®, while 52 are between 4 and 11 years of age and would become eligible pending regulatory approval, demonstrating continued progress identifying additional patients.
VUS patient reclassification and APDS prevalence
There are currently over 1,400 known U.S. patients with a variant of uncertain significance, or VUS, in the PIK3CD and PIK3R1 genes implicated in APDS.
In June, a study performed by researchers at Columbia University was published in the leading peer-reviewed journal Cell, supporting reclassification of VUS patients and expanding the characterization of APDS. The study identified over 100 new variants leading to PI3Kδ pathway hyperactivity. Data suggest that VUS patients with these gain-of-function variants should be functionally classified as APDS patients. We expect genetic testing laboratories to utilize these data to independently re-assess VUSs and reclassify patients to APDS in the second half of 2025, adding an additional growth driver for Joenja®. We are committed to expanding studies to assess additional variants and estimate that 20% of VUS patients could ultimately be diagnosed with APDS.
By analyzing very large datasets of patients who agreed to have their genetic testing linked to their medical records, the research team at Columbia University also concluded that the real prevalence of APDS may be up to 100 times higher than previously estimated. We are now preparing population-based studies to further investigate the genetic prevalence and clinical phenotype of APDS in the newly identified variants, and to better understand the potential to expand Joenja®’s addressable patient population.
Joenja® (leniolisib) development
As of June 30, 2025, there are 185 APDS patients in either a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a named patient program.
Pediatric label expansion
We remain on track to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for leniolisib for the treatment of children aged 4 to 11 years diagnosed with APDS in the third quarter of 2025. An approval decision from the FDA is expected in the first half of 2026.
In June 2025, we submitted a new drug application (NDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for the approval of leniolisib for the treatment of APDS in adult and pediatric patients 4 years of age and older. A decision from the PMDA is expected in nine months based on priority review of the application, due to orphan drug designation (ODD) granted by the Ministry of Health, Labour and Welfare of Japan (MHLW) for the treatment of APDS.
European Economic Area (EEA)
We are on track to complete the manufacturing activities requested by the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP) as part of the ongoing review of the leniolisib Marketing Authorisation Application (MAA) for APDS patients 12 years of age and older, and to submit a response by the January 2026 deadline.
Leniolisib for additional primary immunodeficiencies (PIDs)
Two Phase II clinical trials are evaluating leniolisib for additional primary immunodeficiencies, or PIDs, which affect significantly more patients than APDS. These include (i) genetically identifiable PIDs with immune dysregulation linked to altered PI3Kδ signaling and (ii) common variable immunodeficiency, or CVID, with immune dysregulation identified independently of genetics. We continue to anticipate trial read-outs in 2026.
KL1333 for mitochondrial DNA-driven primary mitochondrial disease
KL1333 is currently in a pivotal clinical trial in primary mitochondrial diseases and has the potential to significantly enhance our future growth. We continue to anticipate trial read-out in 2027 with potential FDA approval by end of 2028.
The pivotal FALCON clinical trial is evaluating KL1333 in adult patients with genetically confirmed primary mitochondrial disease (PMD) with mitochondrial DNA (mtDNA) mutations who experience consistent, debilitating fatigue and muscle weakness (myopathy), and reduced life expectancy. The FALCON clinical trial is proceeding as planned with additional clinical sites now active and the first patients in wave two of the trial having been dosed.
Acquisition of Abliva AB
In the period from February 14, 2025, to June 18, 2025, Pharming acquired the remaining 11.1% interest in the voting shares of Abliva, thereby increasing its ownership to 100%.
Organizational updates
On May 8, 2025, we announced a plan to reduce our general and administrative (G&A) expenses by 15% or US$10 million to optimize capital allocation to drive sustainable growth of our business. Work is on track to deliver on this objective.
| Consolidated Statement of Income | 2Q 2025 | 2Q 2024 | 1H 2025 | 1H 2024 |
| Amounts in US$m except per share data | ||||
| Total Revenues | 93.2 | 74.1 | 172.3 | 129.7 |
| Cost of sales | (9.0) | (8.0) | (17.3) | (16.4) |
| Gross profit | 84.2 | 66.1 | 155.0 | 113.3 |
| Other income | 1.8 | 0.9 | 2.2 | 1.3 |
| Research and development | (23.7) | (21.6) | (44.8) | (40.1) |
| General and administrative | (20.5) | (15.6) | (43.0) | (30.7) |
| Marketing and sales | (31.0) | (32.9) | (65.6) | (63.2) |
| Other Operating Costs | (75.2) | (70.1) | (153.4) | (134.0) |
| Operating profit (loss) | 10.8 | (3.1) | 3.8 | (19.4) |
| Fair value gain (loss) on revaluation | — | 5.1 | — | 5.1 |
| Other finance income | 0.7 | 1.2 | 1.3 | 2.9 |
| Other finance expenses | (4.7) | (2.9) | (9.8) | (4.5) |
| Finance result, net | (4.0) | 3.4 | (8.5) | 3.5 |
| Share of net profits in associates using the equity method | 0.3 | (0.4) | — | (0.8) |
| Profit (loss) before tax | 7.1 | (0.1) | (4.7) | (16.7) |
| Income tax credit (expense) | (2.5) | (1.1) | (5.6) | 3.0 |
| Profit (loss) for the period | 4.6 | (1.2) | (10.3) | (13.7) |
| Earnings per share | ||||
| Basic, attributable to equity holders of the parent (US$) | 0.007 | (0.002) | (0.015) | (0.019) |
| Diluted, attributable to equity holders of the parent (US$) | 0.006 | (0.002) | (0.015) | (0.019) |
| Segment information - Revenues | 2Q 2025 | 2Q 2024 | 1H 2025 | 1H 2024 |
| Amounts in US$m | ||||
| Revenue - RUCONEST® (US) | 79.6 | 61.6 | 146.2 | 106.4 |
| Revenue - RUCONEST® (EU and RoW) | 0.8 | 1.4 | 2.8 | 2.6 |
| Total Revenues - RUCONEST® | 80.4 | 63.0 | 149.0 | 109.0 |
| Revenue - Joenja® (US) | 11.8 | 10.2 | 21.3 | 18.7 |
| Revenue - Joenja® (EU and RoW) | 1.0 | 0.9 | 2.0 | 2.0 |
| Total Revenues - Joenja® | 12.8 | 11.1 | 23.3 | 20.7 |
| Total Revenues - US | 91.4 | 71.8 | 167.5 | 125.1 |
| Total Revenues - EU and RoW | 1.8 | 2.3 | 4.8 | 4.6 |
| Total Revenues | 93.2 | 74.1 | 172.3 | 129.7 |
| Consolidated Balance Sheet | June 30, 2025 | December 31, 2024 |
| Amounts in US$m | ||
| Cash and cash equivalents, restricted cash and marketable securities | 130.8 | 169.4 |
| Current assets | 245.8 | 278.4 |
| Total assets | 446.3 | 400.0 |
| Current liabilities | 87.9 | 73.8 |
| Shareholders' equity | 239.6 | 221.1 |
Financial highlights
For the second quarter of 2025, revenues increased by US$19.1 million, or 26%, to US$93.2 million, compared to US$74.1 million in the second quarter of 2024. RUCONEST® revenues amounted to US$80.4 million, a 28% increase compared to the second quarter of 2024. The increase in RUCONEST® revenues was primarily driven by an increase in volume. Joenja® revenues amounted to US$12.8 million in the second quarter of 2025, a 15% increase compared to the second quarter of 2024. This increase in Joenja® revenues was primarily driven by an increase in price, enhanced by gross-to-net adjustments that were elevated in the prior year.
Gross profit increased by US$18.1 million, or 27%, to US$84.2 million, compared to US$66.1 million in the second quarter of 2024, mainly due to the increase in revenues.
The operating profit amounted to US$10.8 million compared to an operating loss of US$3.1 million in the second quarter of 2024. Adjusted to exclude US$2.1 million of non-recurring Abliva acquisition-related expenses, of which US$1.9 million is included in General and administrative expenses and US$0.2 million is included in Research and development expenses, the operating profit amounted to US$12.9 million. The improved operating result was primarily driven by an increase in revenues, partially offset by higher operating expenses including US$2.1 million in non-recurring Abliva acquisition-related expenses.
The net finance result amounted to a loss of US$4.0 million compared to a gain of US$3.4 million in the second quarter of 2024. This decline was mainly driven by the absence of a one-time fair value gain recognized in the second quarter of 2024 following the reclassification of the convertible bond-related derivative to equity, as well as by unfavorable EUR/USD exchange rate movements in the second quarter of 2025.
The Company had a net profit of US$4.6 million, compared to a net loss of US$1.2 million in the second quarter of 2024. The change was primarily driven by increased revenues, partially offset by higher operating expenses and a change in the net finance result.
Cash generated from operations amounted to US$11.7 million, compared to US$13.2 million used in operations in the second quarter of 2024. Cash and cash equivalents, including restricted cash and marketable securities, increased from US$108.9 million at the end of first quarter of 2025 to US$130.8 million at the end of the second quarter of 2025. This increase was primarily driven by the net cash flows generated from operating activities.
First half year 2025
Total revenues increased 33% during the first half of 2025 to US$172.3 million, compared to US$129.7 million during the first half of 2024. For the first half of 2025, total RUCONEST® revenues were 37% higher at US$149.0 million, compared to revenues of US$109.0 million for the first half of 2024. The increase in RUCONEST® revenues was primarily driven by an increase in volume. Joenja® revenues amounted to US$23.3 million in the first half of 2025, a 13% increase compared to the second half of 2024. This increase in Joenja® revenues was primarily driven by an increase in volume.
Gross profit increased by US$41.7 million, or 37%, to US$155.0 million, compared to US$113.3 million in the first half of 2024, mainly due to the increase in revenues.
Further details on revenue and gross profit segmentation is provided in Note 7. Segment information in the Notes to the condensed consolidated interim financial statements of this press release.
The operating profit amounted to US$3.8 million compared to an operating loss of US$19.4 million in the first half of 2024. Adjusted to exclude US$9.9 million of non-recurring Abliva acquisition-related expenses, of which US$7.6 million is included in General and administrative expenses and US$2.3 million is included in Research and development expenses, the operating profit amounted to US$13.7 million. The improved operating result was primarily driven by an increase in revenues, partially offset by higher operating expenses which include a total of US$15.0 million Abliva-related expenses.
The net finance result amounted to a loss of US$8.5 million compared to a gain of US$3.6 million in the first half of 2024. This decline was mainly driven by the absence of a one-time fair value gain recognized in the second quarter of 2024 following the reclassification of the convertible bond-related derivative to equity, as well as by unfavorable EUR/USD exchange rate movements in the first half of 2025.
The Company had a net loss of US$10.3 million, compared to a net loss of US$13.7 million in the first half of 2024. The change was primarily driven by increased revenues, partially offset by a change in the net finance result and higher operating expenses, including US$9.9 million non-recurring Abliva acquisition-related expenses, most of which were not tax-deductible.
Cash generated from operations amounted to US$12.0 million, compared to US$20.9 million used in operations in the first half of 2024. Cash and cash equivalents, including restricted cash and marketable securities, decreased by US$38.6 million to US$130.8 million from US$169.4 million at the end of 2024, primarily driven by purchases of Abliva shares totaling US$66.1 million and non-recurring Abliva acquisition-related expenses totaling US$9.9 million.
For 2025, the Company anticipates:
Total revenues between US$335.0 million and US$350.0 million (13% to 18% growth), with quarterly fluctuations expected.
Total operating expenses between US$304.0 million and US$308.0 million, assuming constant currency and US$10.2 million non-recurring Abliva-related transaction and integration expenses.
Significant progress finding additional APDS patients in the U.S., supported by VUS validation efforts and subsequently converting patients to paid Joenja® (leniolisib) therapy.
Increasing ex-U.S. revenues for leniolisib - driven by funded access programs and commercial availability in the U.K.
Progress towards additional regulatory approvals for leniolisib for APDS patients 12 years of age or older, and submitting regulatory filings in Japan and for pediatric label expansion in key global markets.
Advancing the two ongoing Phase II clinical trials in PIDs with immune dysregulation to significantly expand the long-term commercial potential of leniolisib.
Advancing the ongoing pivotal FALCON clinical study for KL1333 in mitochondrial DNA-driven primary mitochondrial disease.
Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases.
No further specific financial guidance for 2025 is provided.
Additional information
The conference call presentation is available on the Pharming.com website from 07:30 CEST today.
The conference call will begin at 13:30 CEST/07:30 EDT on Thursday, July 31. A transcript will be made available on the Pharming.com website in the days following the call.
Please note, the Company will only take questions from dial-in attendees.
Conference call dial-in details:
Additional information on how to register for the conference call/webcast can be found on the
Pharming.com website.
Financial Calendar 2025
3Q 2025 financial results November 6
For further public information, contact:
Pharming Group N.V., Leiden, the Netherlands
Michael Levitan, VP Investor Relations & Corporate Communications
T: +1 (908) 705 1696
FTI Consulting, London, UK
Simon Conway/Alex Shaw/Amy Byrne
LifeSpring Life Sciences Communication, Amsterdam, the Netherlands
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 are commercializing and developing a portfolio of innovative medicines, including small molecules and biologics. Pharming is headquartered in Leiden, the Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific.
For more information, visit www.pharming.com and find us on LinkedIn.