Pharming Group reports fourth quarter and full year 2024 financial results and provides business update
Pharming Group reported strong financial results for Q4 and full year 2024, with total revenues increasing 21% to US$297.2 million, exceeding guidance. This growth was driven by record RUCONEST® revenue of US$252.2 million (up 11%) and strong Joenja® performance of US$45.0 million (up 147%).
Q4 2024 highlights include total revenues of US$92.7 million (up 14%) and operating profit increase to US$6.7 million from US$1.1 million in Q4 2023. The company generated operating profit and positive cash flows for two consecutive quarters.
Key developments include two ongoing Phase II trials of leniolisib for additional primary immunodeficiencies, the completion of Abliva AB acquisition adding KL1333 to the pipeline, and the appointment of Fabrice Chouraqui as CEO. The company provided 2025 revenue guidance of US$315-335 million.
Pharming Group ha riportato risultati finanziari solidi per il quarto trimestre e l'intero anno 2024, con ricavi totali in aumento del 21% a 297,2 milioni di dollari USA, superando le previsioni. Questa crescita è stata guidata da un fatturato record di RUCONEST® pari a 252,2 milioni di dollari USA (in aumento dell'11%) e da un forte rendimento di Joenja® di 45,0 milioni di dollari USA (in aumento del 147%).
I punti salienti del Q4 2024 includono ricavi totali di 92,7 milioni di dollari USA (in aumento del 14%) e un aumento dell'utile operativo a 6,7 milioni di dollari USA rispetto a 1,1 milioni di dollari USA nel Q4 2023. L'azienda ha generato un utile operativo e flussi di cassa positivi per due trimestri consecutivi.
Tra gli sviluppi chiave ci sono due trial di Fase II in corso di leniolisib per ulteriori immunodeficienze primarie, il completamento dell'acquisizione di Abliva AB che aggiunge KL1333 al pipeline, e la nomina di Fabrice Chouraqui come CEO. L'azienda ha fornito una previsione di ricavi per il 2025 di 315-335 milioni di dollari USA.
Pharming Group reportó resultados financieros sólidos para el cuarto trimestre y el año completo 2024, con ingresos totales aumentando un 21% a 297,2 millones de dólares estadounidenses, superando las expectativas. Este crecimiento fue impulsado por unos ingresos récord de RUCONEST® de 252,2 millones de dólares estadounidenses (un aumento del 11%) y un fuerte rendimiento de Joenja® de 45,0 millones de dólares estadounidenses (un aumento del 147%).
Los aspectos destacados del Q4 2024 incluyen ingresos totales de 92,7 millones de dólares estadounidenses (un aumento del 14%) y un aumento en las ganancias operativas a 6,7 millones de dólares estadounidenses desde 1,1 millones de dólares estadounidenses en el Q4 2023. La empresa generó ganancias operativas y flujos de efectivo positivos durante dos trimestres consecutivos.
Los desarrollos clave incluyen dos ensayos de Fase II en curso de leniolisib para deficiencias inmunitarias primarias adicionales, la finalización de la adquisición de Abliva AB que agrega KL1333 al pipeline, y el nombramiento de Fabrice Chouraqui como CEO. La empresa proporcionó una guía de ingresos de 2025 de 315-335 millones de dólares estadounidenses.
Pharming Group은 2024년 4분기 및 연간 강력한 재무 결과를 보고했으며, 총 수익이 21% 증가하여 2억 9,720만 달러에 달했습니다. 이는 예상을 초과한 수치입니다. 이 성장세는 RUCONEST®의 기록적인 수익 2억 5,220만 달러(11% 증가)와 Joenja®의 강력한 성과 4,500만 달러(147% 증가)에 의해 주도되었습니다.
2024년 4분기 하이라이트에는 총 수익 9,270만 달러(14% 증가)와 운영 이익이 1,110만 달러에서 670만 달러로 증가한 것이 포함됩니다. 이 회사는 두 분기 연속으로 운영 이익과 긍정적인 현금 흐름을 창출했습니다.
주요 개발 사항으로는 추가적인 원발 면역 결핍증을 위한 leniolisib의 두 개의 진행 중인 2상 시험, KL1333을 파이프라인에 추가하는 Abliva AB 인수 완료, Fabrice Chouraqui를 CEO로 임명한 것이 있습니다. 이 회사는 2025년 수익 가이드를 3억 1,500만 - 3억 3,500만 달러로 제공했습니다.
Pharming Group a rapporté des résultats financiers solides pour le quatrième trimestre et l'année complète 2024, avec des revenus totaux en hausse de 21 % à 297,2 millions de dollars US, dépassant les prévisions. Cette croissance a été soutenue par des revenus record de RUCONEST® de 252,2 millions de dollars US (en hausse de 11 %) et une forte performance de Joenja® de 45,0 millions de dollars US (en hausse de 147 %).
Les faits marquants du Q4 2024 incluent des revenus totaux de 92,7 millions de dollars US (en hausse de 14 %) et une augmentation du bénéfice d'exploitation à 6,7 millions de dollars US contre 1,1 million de dollars US au Q4 2023. L'entreprise a généré un bénéfice d'exploitation et des flux de trésorerie positifs pendant deux trimestres consécutifs.
Les développements clés comprennent deux essais de Phase II en cours de leniolisib pour des immunodéficiences primaires supplémentaires, l'achèvement de l'acquisition d'Abliva AB ajoutant KL1333 au pipeline, et la nomination de Fabrice Chouraqui en tant que PDG. L'entreprise a fourni une prévision de revenus pour 2025 de 315 à 335 millions de dollars US.
Pharming Group hat starke finanzielle Ergebnisse für das 4. Quartal und das Gesamtjahr 2024 berichtet, mit einem Gesamtumsatz von 21% auf 297,2 Millionen US-Dollar, was die Prognosen übertrifft. Dieses Wachstum wurde durch rekordverdächtige Umsätze von RUCONEST® in Höhe von 252,2 Millionen US-Dollar (ein Anstieg von 11%) und eine starke Leistung von Joenja® in Höhe von 45,0 Millionen US-Dollar (ein Anstieg von 147%) angetrieben.
Die Höhepunkte des 4. Quartals 2024 umfassen einen Gesamtumsatz von 92,7 Millionen US-Dollar (ein Anstieg von 14%) und einen Anstieg des Betriebsergebnisses auf 6,7 Millionen US-Dollar von 1,1 Millionen US-Dollar im 4. Quartal 2023. Das Unternehmen erzielte zwei aufeinanderfolgende Quartale mit Betriebsergebnis und positiven Cashflows.
Wichtige Entwicklungen umfassen zwei laufende Phase-II-Studien zu leniolisib für zusätzliche primäre Immundefizienzen, den Abschluss der Übernahme von Abliva AB, die KL1333 in die Pipeline aufnimmt, und die Ernennung von Fabrice Chouraqui zum CEO. Das Unternehmen gab eine Umsatzprognose für 2025 von 315-335 Millionen US-Dollar bekannt.
- Record RUCONEST® revenue of US$252.2 million, up 11% year-over-year
- Strong Joenja® growth with revenue up 147% to US$45.0 million
- Operating profit increased to US$6.7 million in Q4 2024
- Positive cash flow from operations for two consecutive quarters
- 96 patients on paid Joenja therapy in US with additional patients pending authorization
- 11% increase in RUCONEST physician prescriber base
- Ongoing regulatory review delays for leniolisib in EMA requiring additional manufacturing activities
- Extended timeline for Canadian regulatory submission to February 2026
Insights
Pharming Group's Q4 and FY2024 results demonstrate exceptional commercial execution with 21% YoY revenue growth to $297.2 million, meaningfully surpassing their guidance of $280-295 million. The company's transformation from a single-product to multi-product company is progressing successfully, with Joenja generating $45 million in its first full year, a 147% increase over 2023.
The fourth quarter shows accelerating momentum with total revenue of $92.7 million (up 14% YoY) and operating profit of $6.7 million, a substantial improvement from $1.1 million in Q4 2023. This marks the second consecutive quarter of operating profitability and positive operating cash flow, signaling a potential inflection point for sustainable profitability.
RUCONEST continues to show remarkable durability with 11% growth to $252.2 million, significantly outperforming management's mid-single-digit growth expectations. The 6% unit sales growth and 24% increase in new patient enrollments suggest effective commercial execution in a mature HAE market.
The 2025 revenue guidance of $315-335 million represents 6-13% growth, which appears achievable given the strong commercial momentum. With the Abliva acquisition adding KL1333 to the pipeline without requiring external funding, Pharming demonstrates both commercial success and disciplined pipeline expansion while maintaining financial strength.
Pharming's clinical development strategy is showing substantial progress with a multi-pronged approach for leniolisib expansion. The positive topline results for the pediatric Phase III trial (ages 4-11) provides a clear path to expand the addressable patient population, with regulatory submissions expected in H2 2025.
The company's indication expansion for leniolisib into additional primary immunodeficiencies represents a significant market opportunity. The two ongoing Phase II trials target populations with 7.5 patients per million for PIDs linked to PI3Kδ signaling (versus 1-2 per million for APDS) and 39 patients per million for CVID with immune dysregulation. If successful, these expansions would transform leniolisib from a ultra-rare disease treatment to a potential blockbuster with substantially larger addressable markets.
The Variant of Uncertain Significance (VUS) resolution strategy is particularly noteworthy, with potential to significantly expand the diagnosed APDS population. The completed high-throughput screening study identified novel variants leading to PI3Kδ hyperactivity, and the company's support for clinical laboratories to reclassify variants could lead to amended genetic testing reports and new patient identifications.
The strategic acquisition of Abliva brings KL1333 for mitochondrial DNA-driven primary mitochondrial diseases into the late-stage pipeline. With the FALCON pivotal trial advancing and blockbuster potential mentioned, this acquisition aligns with Pharming's rare disease focus while leveraging existing operational capabilities in specialized markets.
- Full year 2024 total revenues increased by
21% to US$297.2 million and exceeded our guidance, driven by record RUCONEST® revenue and strong Joenja® (leniolisib) growth - Fourth quarter 2024 total revenues increased by
14% to US$92.7 million , compared to the fourth quarter 2023 - RUCONEST® full year revenue increased by
11% to US$252.2 million and fourth quarter revenue increased by9% to US$79.6 million , compared to the fourth quarter 2023 - Joenja® revenue increased by
147% to US$45.0 million in the first full year post-launch and fourth quarter revenue increased by66% to US$13.1 million , compared to the fourth quarter 2023 - Fourth quarter operating profit increased to US
$6.7 million from US$1.1 million in the fourth quarter 2023 - Generated operating profit and positive net cash flows from operations for the second quarter in a row
- Two ongoing Phase II clinical trials of leniolisib for additional primary immunodeficiencies (PIDs) with immune dysregulation, including common variable immunodeficiency (CVID)
- Completed the acquisition of Abliva AB, adding KL1333 for mitochondrial DNA-driven primary mitochondrial diseases to Pharming’s late-stage clinical pipeline, in line with our vision to become a leading global rare disease company
- Fabrice Chouraqui appointed as Chief Executive Officer and Executive Director at EGM held on March 4, 2025
- 2025 total revenue guidance of US
$315 million – US$335 million - Pharming to host a conference call today at 13:30 CET (8:30 am EDT)
Leiden, the Netherlands, March 13, 2025: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM / Nasdaq: PHAR) presents its preliminary (unaudited) financial report for the three months and full year ended December 31, 2024.
Chief Executive Officer, Fabrice Chouraqui commented:
“I am delighted to have been appointed as CEO of Pharming and am deeply excited for the opportunities that lie ahead. The strength of the results presented today, with record RUCONEST® revenue and strong Joenja® growth, is testament to Pharming’s momentum.
We ended 2024 on a strong note, growing total revenues by
We enter 2025 with a number of regulatory reviews for leniolisib ongoing as we prepare for launches in key markets and for pediatrics. We also advanced our efforts to expand the addressable patient population for leniolisib, with two Phase II trials in additional PIDs now underway. The genetically identifiable PIDs indication and the CVID indication first announced today represent significantly larger market opportunities than APDS, with blockbuster revenue potential.
We are also strengthening our clinical pipeline with the acquisition of Abliva, which has now been completed. This adds a potential first-in-disease treatment for primary mitochondrial diseases, KL1333, which has the opportunity to further transform Pharming’s growth trajectory as it too has blockbuster potential. The acquisition aligns with our vision to become a leading global rare disease company and is well aligned with our operational capabilities. We are now moving to start the second wave of patient recruitment for the pivotal FALCON clinical trial as soon as possible.
Pharming’s 2024 financial performance was also noteworthy, with the company generating an operating profit and positive net cash flows from operations in the last two quarters of the year. This performance highlights the financial strength of our core commercial business.
Looking to 2025, we will continue to invest diligently to grow our portfolio in the U.S. and Joenja® in key countries, to reach more patients with APDS, and to progress the significant opportunities in our R&D pipeline for PIDs and primary mitochondrial diseases.
I firmly believe that Pharming is well-positioned to build on this positive momentum and embrace a new cycle of growth, delivering strong long-term value creation for all stakeholders and continuing our mission to serve the unserved rare disease patients. I look forward to working with the Pharming team to make this a reality.”
Fourth quarter and full year 2024 highlights
Commercialized products
RUCONEST® marketed for the treatment of acute HAE attacks
RUCONEST® demonstrated significant strength in the fourth quarter of 2024, with record revenues of US
The U.S. market contributed
The strong RUCONEST® revenue growth in 2024 can be attributed to strong performance in new physicians prescribing RUCONEST®, new patient enrollments, and the total number of patients, which together resulted in over
Joenja® (leniolisib) marketed in the U.S. - the first and only approved disease modifying treatment for APDS
Joenja® revenues increased to US
The U.S. market contributed
As of December 31, 2024, we had 96 patients on paid therapy in the U.S. and an additional five patients enrolled and pending authorization, representing an increase of active patients during the fourth quarter and continued progress enrolling and transitioning eligible patients to paid therapy.
APDS patient finding
As of December 31, 2024, Pharming had identified over 880 diagnosed APDS patients in global markets, including over 240 patients in the U.S. Of the identified patients in the U.S., over 150 patients are 12 years of age or older and eligible for treatment with Joenja®.
Pharming continues to advance several initiatives to diagnose additional APDS patients, including a sponsored genetic testing program in the U.S. and Canada, partnerships with several genetic testing companies who undertake their own testing efforts, and family testing programs.
APDS patient finding - VUS resolution
Pharming is aware of approximately 1,200 patients in the U.S. with a Variant of Uncertain Significance, or VUS, in the PIK3CD or PIK3R1 genes and is supporting validation studies with various laboratories to confirm which of these variants are pathogenic for APDS. Patients with disease-associated variants would receive a molecular diagnosis of APDS and, therefore, potentially be eligible for Joenja® treatment. Based on data from Pharming’s navigateAPDS sponsored genetic testing program, PIK3CD and PIK3R1 VUSs are found at four times the frequency of those mutations currently classified as pathogenic / likely pathogenic for APDS. Furthermore, a completed literature review and pilot study resulted in
As previously communicated, Pharming is supporting independent research to evaluate large numbers of VUSs without the need for additional patient testing. VUS resolution via high throughput screening methods is an established approach that is accepted as strong functional evidence for variant classification by various expert organizations including the American College of Medical Genetics (ACMG) and ClinGen (a National Institutes of Health-funded resource). One in vitro high throughput screening study was completed in the fourth quarter of 2024, identifying many novel variants leading to PI3Kδ hyperactivity. Pharming is now supporting clinical genetics laboratories across the US to be able to use their independent variant interpretation to reclassify variants and thus issue amended genetic testing reports for any variants these laboratories deem to be disease-causing. We anticipate that these endeavors will lead to the identification of new patients with APDS.
Joenja® (leniolisib) development
Leniolisib for APDS
During 2024, we received regulatory approvals for leniolisib for patients 12 years of age or older in the U.K. and Israel, and advanced additional regulatory filings, to bring leniolisib to APDS patients outside of the U.S. We also made strong progress in our clinical trials to support APDS marketing approval for leniolisib in Japan and pediatric label expansion.
In total, there are currently 188 patients in a leniolisib Expanded Access Program (compassionate use), an ongoing clinical study, or a named patient program.
Pediatric clinical development
On December 11, 2024, we announced positive top line results for the multinational Phase III clinical trial for children 4 to 11 years of age, which has been evaluating leniolisib tablets in 21 children with APDS. The data are consistent with the improvements seen in the previously reported randomized controlled trial in adolescent and adult APDS patients. Global regulatory filings are planned to begin with a U.S. submission in the second half of 2025.
Japan clinical trial
We completed an interim analysis, after 12-weeks of treatment, for the Phase III clinical trial in Japan evaluating leniolisib for the treatment of APDS in adult and pediatric patients 12 years of age and older. The study’s safety and efficacy findings were in line with data from the randomized controlled trial used to support approvals in adolescent and adult APDS patients in the U.S. and other countries, and support a regulatory filing with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) which is planned for mid-2025. An approval decision would be expected in nine months based on priority review of the application due to orphan drug designation (ODD) 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) in regard to the ongoing review of the Marketing Authorisation Application (MAA) for leniolisib for the treatment of adult and pediatric patients 12 years of age and older and submit a response prior to the January 2026 deadline.
United Kingdom
On September 25, 2024, the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Joenja® (leniolisib) for the treatment of APDS in adult and pediatric patients 12 years of age and older. Leniolisib is currently under evaluation by the National Institute for Health and Care Excellence (NICE) regarding reimbursement within the National Health Service (NHS) in England and Wales.
Additional markets
In Australia, we filed a regulatory submission for patients 12 years of age and older in the third quarter of 2023 and, after positive feedback from the Australian Advisory Committee on Medicines, expect a final decision by mid-March 2025.
We filed a regulatory submission in Canada for APDS patients 12 years of age and older in the third quarter of 2023. We have had ongoing interactions with Health Canada that recently granted an extension to February 2026 to respond to a request for additional CMC data, in line with the EMA extension. We plan to respond in early 2026 and expect a regulatory decision in 2026.
Leniolisib for additional primary immunodeficiencies (PIDs)
We made strong progress during 2024 on our leniolisib indication expansion efforts. We are developing 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 started the Phase II clinical trial evaluating leniolisib for PIDs with immune dysregulation linked to altered PI3Kẟ signaling in October 2024, and started a Phase II clinical trial for CVID with immune dysregulation in February 2025.
Primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kδ signaling
On October 10, 2024, Pharming announced the start of a Phase II, proof of concept, clinical trial evaluating leniolisib in PIDs with immune dysregulation linked to PI3Kẟ signaling in lymphocytes, with similar clinical phenotypes and unmet medical needs to APDS. The first patient was dosed in the study on October 29, 2024 and enrollment is progressing. The clinical trial includes PID patients with ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency, among others. Epidemiology suggests a prevalence of approximately seven and a half patients per million in this targeted PID population, compared to one to two patients per million for APDS.
The Phase II clinical trial is a single arm, open-label, dose range-finding study to be conducted in approximately 12 patients. The objectives for the trial are to assess safety and tolerability, pharmacokinetics, pharmacodynamics, and explore clinical efficacy of leniolisib in the targeted PID population.
In February 2025, the FDA granted Fast Track designation for leniolisib for the treatment of PIDs linked to PI3Kẟ signaling. Fast Track is an FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
Common variable immunodeficiency (CVID) with immune dysregulation
CVID with immune dysregulation represents a much larger group of PID patients, which may be identified independently of genetics, and with a targeted population prevalence of approximately 39 patients per million. The majority of CVID patients with immune dysregulation exhibit a spectrum of noninfectious autoimmune and end-organ lympho-infiltrative disease manifestations with similarities to APDS.
We engaged with the FDA and EMA on the CVID indication, and received positive feedback regarding the large unmet medical need and the rationale for evaluating leniolisib in CVID patients with immune dysregulation. We have initiated a Phase II study for leniolisib in CVID patients with immune dysregulation, with the first patient expected to be dosed in late March 2025.
Acquisition of Abliva AB
In December 2024, we announced the proposed acquisition of Abliva AB, via a public cash offer to the shareholders to acquire all issued and outstanding shares of Abliva, for approximately US
KL1333 for mitochondrial DNA-driven primary mitochondrial diseases
KL1333 is in a pivotal clinical study (FALCON) 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. Over 30,000 patients diagnosed with mtDNA mitochondrial disease would be potentially addressable by KL1333 in the U.S., EU4 (France, Germany, Italy, Spain) and the UK, offering blockbuster potential for this product in the U.S. alone.
KL1333 has shown positive clinical effects in a proof-of-concept Phase 1b study, and a pre-planned interim analysis of the ongoing pivotal FALCON trial demonstrated promising differences over placebo in both alternate primary efficacy endpoints passing futility. KL1333 has received Fast Track designation in the U.S. and Orphan Drug Designation for the treatment of PMD in the U.S. and EU. We are now moving to start the second wave of patient recruitment for the pivotal FALCON clinical trial as soon as possible. We anticipate the trial to read-out in 2027 with potential FDA approval by end of 2028.
Organizational updates
Ms. Inés Bernal was appointed Chief People Officer, or CPO, as of December 1, 2024, to lead the development, execution and monitoring of Pharming's people and culture strategy.
On October 24, 2024, we announced that Mr. Sijmen de Vries, Executive Director and Chief Executive Officer, had informed the Board of Directors that he would not be available for reappointment at our next AGM.
Subsequent events
On January 21, 2025, we announced that the Board of Directors had nominated biopharmaceutical leader Mr. Fabrice Chouraqui as Pharming’s new Chief Executive Officer and Executive Director, succeeding Mr. Sijmen de Vries.
Mr. Chouraqui was appointed for a term of four years at the Extraordinary General Meeting of Shareholders (EGM) that took place on March 4, 2025.
Upon the appointment of Mr. Chouraqui, Mr. Sijmen de Vries resigned from the Board of Directors. To ensure a smooth hand-over of tasks and responsibilities, Mr. de Vries will remain a strategic advisor to the new CEO until December 31, 2025.
On February 20, 2025, we announced ownership of shares and voting rights in Abliva AB exceeding
The acquisition of Abliva will be accounted for as a business combination. Substantially all of the value of the acquisition is concentrated in a single asset, KL1333. Following delisting as expected in March 2025, the acquisition would be reflected in our financial statements beginning with the first quarter 2025. At this point we expect the US
Financial Summary
| Consolidated Statement of Income | 4Q 2024 | 4Q 2023 | 2024 | 2023 |
| Amounts in US$m except per share data | ||||
| Total Revenues | 92.7 | 81.2 | 297.2 | 245.3 |
| Cost of sales | (12.2) | (7.1) | (35.4) | (25.2) |
| Gross profit | 80.5 | 74.1 | 261.8 | 220.1 |
| Other income | 0.1 | 0.6 | 2.2 | 23.3 |
| Research and development | (22.3) | (11.6) | (83.2) | (68.9) |
| General and administrative | (24.6) | (24.1) | (70.6) | (55.9) |
| Marketing and sales | (27.0) | (37.9) | (118.8) | (124.0) |
| Other Operating Costs | (73.9) | (73.6) | (272.6) | (248.8) |
| Operating profit (loss) | 6.7 | 1.1 | (8.6) | (5.4) |
| Fair value gain (loss) on revaluation | (0.2) | (0.9) | 5.0 | (0.9) |
| Other finance income | 3.1 | 1.6 | 6.8 | 3.7 |
| Other finance expenses | (2.5) | (4.5) | (9.9) | (9.1) |
| Share of net profits in associates using the equity method | (0.5) | 0.7 | (1.8) | (0.3) |
| Profit (loss) before tax | 6.6 | (2.0) | (8.5) | (12.0) |
| Income tax credit (expense) | (2.9) | (1.1) | (2.5) | 1.5 |
| Profit (loss) for the period | 3.7 | (3.1) | (11.0) | (10.5) |
| Share Information | ||||
| Basic earnings per share (US$) | 0.005 | (0.004) | (0.016) | (0.016) |
| Diluted earnings per share (US$) | 0.005 | (0.004) | (0.016) | (0.016) |
| Segment information - Revenues | 4Q 2024 | 4Q 2023 | 2024 | 2023 |
| Amounts in US$m | ||||
| Revenue - RUCONEST® (US) | 78.2 | 71.9 | 246.6 | 221.2 |
| Revenue - RUCONEST® (EU and RoW) | 1.4 | 1.4 | 5.6 | 5.9 |
| Total Revenues - RUCONEST® | 79.6 | 73.3 | 252.2 | 227.1 |
| Revenue - Joenja® (US) | 11.8 | 7.6 | 40.5 | 17.9 |
| Revenue - Joenja® (EU and RoW) | 1.3 | 0.3 | 4.5 | 0.3 |
| Total Revenues - Joenja® | 13.1 | 7.9 | 45.0 | 18.2 |
| Total Revenues - US | 90.0 | 79.5 | 287.1 | 239.1 |
| Total Revenues - EU and RoW | 2.7 | 1.7 | 10.1 | 6.2 |
| Total Revenues | 92.7 | 81.2 | 297.2 | 245.3 |
| Consolidated Balance Sheet | December 31, 2024 | December 31, 2023 |
| Amounts in US$m | ||
| Cash and cash equivalents, restricted cash and marketable securities | 169.4 | 215.0 |
| Current assets | 278.7 | 316.3 |
| Total assets | 400.8 | 462.9 |
| Current liabilities | 73.8 | 78.0 |
| Equity | 221.9 | 218.8 |
Financial highlights
Fourth quarter 2024
Revenues in the fourth quarter of 2024 increased by
Gross profit increased by US
The operating profit amounted to US
The net finance result amounted to a gain of US
In the fourth quarter of 2024, a net profit of US
Cash and cash equivalents, including restricted cash and marketable securities, decreased from US
Full year 2024
In 2024, Pharming revenues increased by
This section will further elaborate on Pharming's financial performance in 2024.
Revenues and Gross Profit
Total revenues for 2024 grew by
Cost of sales increased by
Gross profit increased by US
Other income
Other income decreased to US
Operating Profit (loss) and Other Operating Costs
For 2024, the operating loss increased to a loss of US
Operating Profit (loss) excluding one-time events
The 2023 operating expenses included milestone payments for Joenja® of US
Finance income and expenses
The net finance result amounted to a gain of US
Income tax credit (expense)
Income tax credit (expense) shifted from a US
Net loss for the year
The Company had a net loss of US
Intangible assets
In 2024, intangible assets decreased by US
The amortization relates to regular amortization of software, the RUCONEST® licenses (US and EU) and the Joenja® license. The RUCONEST® license has a remaining amortization period of 13 years for the US and 7 years for the EU. The Joenja® license has a remaining amortization period of 12 years.
Property, plant and equipment
The value of property, plant and equipment decreased from US
Right-of-use assets
The right-of-use assets decreased from US
The decrease in the right-to-use assets is partially offset by additions of cars (US
Investments
Investments decreased by US
Inventories
Inventories decreased from US
Cash and cash equivalents and marketable securities
Cash and cash equivalents alone decreased by US
The combined total of cash and cash equivalents, together with restricted cash and marketable securities decreased from US
Shareholders’ equity
Shareholders’ equity increased by US
Convertible bond
The convertible bond position has decreased by US
Lease liabilities
Lease liabilities decreased by US
Outlook/Summary
For 2025, the Company anticipates:
- Total revenues between US
$315 million and US$335 million (6% to13% growth), with quarterly fluctuations expected. - Total operating expenses not to exceed the prior year pre-Abliva impact, and a preliminary estimate of US
$30 million in Abliva-related operating expenses, including research and development and non-recurring 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 diseases.
- 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 2025 is provided.
Additional information
Presentation
The conference call presentation is available on the Pharming.com website from 07:30 CET today.
Conference Call
The conference call will begin at 13:30 CET/08:30 am EDT on Thursday, March 13. 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.
Webcast Link:
https://edge.media-server.com/mmc/p/7qvoovac
Conference call dial-in details:
https://register.vevent.com/register/BI7d716758d12d4ad588b70fdbca665b6a
Additional information on how to register for the conference call/webcast can be found on the
Pharming.com website.
Financial Calendar 2025
Annual Report and 20-F 2024 April 3
1Q 2025 financial results May 8
Annual General Meeting of Shareholders June 11
2Q/1H 2025 financial results July 31
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
E: investor@pharming.com
FTI Consulting, London, UK
Simon Conway/Alex Shaw/Amy Byrne
T: +44 203 727 1000
LifeSpring Life Sciences Communication, Amsterdam, the Netherlands
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl
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. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision 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.
Risk profile
We continue to closely monitor and manage the key risks and opportunities, and will respond appropriately to any emerging risk. We will issue a full overview of our risk profile in our Annual report 2024 to be published on April 3, 2025.
Related party transactions
There are no material changes in the nature, scope, and (relative) scale in this reporting period compared to last year.
Auditor’s involvement
The Condensed Consolidated Interim Financial Statements have not been audited by the Company’s statutory auditor.
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 2023 Annual Report and the Annual Report on Form 20-F for the year ended December 31, 2023, 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 Financial Statements in U.S. Dollars (unaudited)
For the period ended December 31, 2024
- 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
| CONDENSED CONSOLIDATED INTERIM STATEMENT OF INCOME | ||
| For the period ended December 31 | ||
| Amounts in US$ ‘000 | 2024 | 2023 |
| Revenues | 297,200 | 245,316 |
| Costs of sales | (35,399) | (25,212) |
| Gross profit | 261,801 | 220,104 |
| Other income | 2,177 | 23,349 |
| Research and development | (83,161) | (68,914) |
| General and administrative | (70,619) | (55,877) |
| Marketing and sales | (118,819) | (124,049) |
| Other Operating Costs | (272,599) | (248,840) |
| Operating profit (loss) | (8,621) | (5,387) |
| Fair value gain (loss) on revaluation | 4,990 | (930) |
| Other finance income | 6,820 | 3,663 |
| Other finance expenses | (9,944) | (9,069) |
| Finance result, net | 1,866 | (6,336) |
| Share of net profits (loss) in associates using the equity method | (1,758) | (289) |
| Profit (loss) before tax | (8,513) | (12,012) |
| Income tax credit (expense) | (2,514) | 1,464 |
| Profit (loss) for the period | (11,027) | (10,548) |
| Basic earnings per share (US$) | (0.016) | (0.016) |
| Diluted earnings per share (US$) | (0.016) | (0.016) |
| CONDENSED CONSOLIDATED INTERIM STATEMENT OF COMPREHENSIVE INCOME | ||
| For the period ended December 31 | ||
| Amounts in US$ ‘000 | 2024 | 2023 |
| Profit (loss) for the period | (11,027) | (10,548) |
| Currency translation differences | (11,992) | 5,936 |
| Items that may be subsequently reclassified to profit or loss | (11,992) | 5,936 |
| Fair value remeasurement investments | 79 | 1,167 |
| Items that shall not be subsequently reclassified to profit or loss | 79 | 1,167 |
| Other comprehensive income (loss), net of tax | (11,913) | 7,103 |
| Total comprehensive income (loss) for the period | (22,940) | (3,445) |
| CONDENSED CONSOLIDATED INTERIM BALANCE SHEET | ||
| Amounts in US$ ‘000 | December 31, 2024 | December 31, 2023 |
| Non-current assets | ||
| Intangible assets | 61,039 | 71,267 |
| Property, plant and equipment | 7,752 | 9,689 |
| Right-of-use assets | 16,382 | 23,777 |
| Long-term prepayments | 90 | 92 |
| Deferred tax assets | 31,090 | 29,761 |
| Investment accounted for using the equity method | 466 | 2,285 |
| Investments in equity instruments designated as at FVTOCI | — | 2,020 |
| Investment in debt instruments designated as at FVTPL | 3,767 | 6,093 |
| Restricted cash | 1,505 | 1,528 |
| Total non-current assets | 122,091 | 146,512 |
| Current assets | ||
| Inventories | 55,724 | 56,760 |
| Trade and other receivables | 55,079 | 46,158 |
| Marketable securities | 112,949 | 151,683 |
| Cash and cash equivalents | 54,944 | 61,741 |
| Total current assets | 278,696 | 316,342 |
| Total assets | 400,787 | 462,854 |
| Equity | ||
| Share capital | 7,769 | 7,669 |
| Share premium | 488,990 | 478,431 |
| Other reserves | (222) | (2,057) |
| Accumulated deficit | (274,675) | (265,262) |
| Shareholders’ equity | 221,862 | 218,781 |
| Non-current liabilities | ||
| Convertible bonds | 78,154 | 136,598 |
| Lease liabilities | 26,968 | 29,507 |
| Total non-current liabilities | 105,122 | 166,105 |
| Current liabilities | ||
| Convertible bonds | 4,245 | 1,824 |
| Trade and other payables | 66,611 | 72,528 |
| Lease liabilities | 2,947 | 3,616 |
| Total current liabilities | 73,803 | 77,968 |
| Total equity and liabilities | 400,787 | 462,854 |
| CONDENSED CONSOLIDATED INTERIM STATEMENT OF CHANGES IN EQUITY | |||||
| For the period ended December 31 | |||||
| Attributable to owners of the parent | |||||
| Amounts in US$ ‘000 | Share capital | Share premium | Other reserves | Accumulated deficit | Total equity |
| Balance at January 1, 2023 | 7,509 | 462,297 | (8,737) | (256,431) | 204,638 |
| Profit (loss) for the period | — | — | — | (10,548) | (10,548) |
| Reserves | — | — | — | — | — |
| Other comprehensive income (loss) for the period | — | — | 7,103 | — | 7,103 |
| Total comprehensive income (loss) for the period | — | — | 7,103 | (10,548) | (3,445) |
| Other reserves | — | — | (423) | 423 | — |
| Income tax benefit from excess tax deductions related to share-based payments | — | — | — | 204 | 204 |
| Share-based compensation | — | — | — | 9,251 | 9,251 |
| Options exercised / LTIP shares issued | 160 | 16,134 | — | (8,161) | 8,133 |
| Value of conversion rights of convertible bonds | — | — | — | — | — |
| Total transactions with owners, recognized directly in equity | 160 | 16,134 | (423) | 1,717 | 17,588 |
| Balance at December 31, 2023 | 7,669 | 478,431 | (2,057) | (265,262) | 218,781 |
| Balance at January 1, 2024 | 7,669 | 478,431 | (2,057) | (265,262) | 218,781 |
| Profit (loss) for the period | — | — | — | (11,027) | (11,027) |
| Reserves | — | — | 1,555 | (1,555) | — |
| Other comprehensive income (loss) for the period | — | — | (11,913) | — | (11,913) |
| Total comprehensive income (loss) for the period | — | — | (10,358) | (12,582) | (22,940) |
| Other reserves | — | — | (31) | 31 | — |
| Income tax benefit from excess tax deductions related to share-based payments | — | — | — | (66) | (66) |
| Share-based compensation | — | — | — | 11,248 | 11,248 |
| Options exercised / LTIP shares issued | 100 | 10,559 | — | (8,044) | 2,615 |
| Value of conversion rights of convertible bonds | — | — | 12,224 | — | 12,224 |
| Total transactions with owners, recognized directly in equity | 100 | 10,559 | 12,193 | 3,169 | 26,021 |
| Balance at December 31, 2024 | 7,769 | 488,990 | (222) | (274,675) | 221,862 |
| CONDENSED CONSOLIDATED INTERIM STATEMENT OF CASH FLOWS | ||
| For the period ended December 31 | ||
| Amounts in $’000 | 2024 | 2023 |
| Profit (loss) before tax | (8,513) | (12,012) |
| Adjustments to reconcile net profit (loss) to net cash used in operating activities: | ||
| Depreciation, amortization, impairment of non-current assets | 16,070 | 15,925 |
| Equity settled share based payments | 11,248 | 9,251 |
| Fair value loss (gain) on revaluation | (4,990) | 930 |
| Gain on disposal from PRV sale | — | (21,279) |
| Disposal of leases | 22 | — |
| Other finance income | (6,820) | (3,663) |
| Other finance expenses | 9,887 | 9,069 |
| Share of net profits in associates using the equity method | 1,758 | 289 |
| Other | — | (1,079) |
| Operating cash flows before changes in working capital | 18,662 | (2,569) |
| Changes in working capital: | ||
| Inventories | (503) | (14,434) |
| Trade and other receivables | (6,783) | (18,539) |
| Payables and other current liabilities | (2,769) | 16,228 |
| Restricted cash | (17) | (216) |
| Total changes in working capital | (10,072) | (16,961) |
| Interest received | 5,201 | 2,883 |
| Income taxes received (paid) | (15,584) | (655) |
| Net cash flows generated from (used in) operating activities | (1,793) | (17,302) |
| Capital expenditure for property, plant and equipment | (790) | (1,437) |
| Proceeds on PRV sale | — | 21,279 |
| Investment intangible assets | (6) | (27) |
| Disposal of investment designated as at FVOCI | 2,098 | — |
| Purchases of marketable securities | (284,314) | (382,014) |
| Proceeds from sale of marketable securities | 314,630 | 232,811 |
| Net cash flows generated from (used in) investing activities | 31,618 | (129,388) |
| Payment of lease liabilities | (4,008) | (4,038) |
| Interests on lease liabilities | (1,141) | (1,088) |
| Net proceeds of issued convertible bonds | 104,539 | — |
| Repurchase of convertible bonds | (134,924) | — |
| Interests on convertible bonds | (4,457) | (4,046) |
| Settlement of share based compensation awards | 5,579 | 8,133 |
| Net cash flows generated from (used in) financing activities | (34,412) | (1,039) |
| Increase (decrease) of cash | (4,587) | (147,729) |
| Exchange rate effects | (2,210) | 2,128 |
| Cash and cash equivalents at the beginning of the period | 61,741 | 207,342 |
| Total cash and cash equivalents at December 31 | 54,944 | 61,741 |
---ENDS---
Attachment
FAQ
What were Pharming Group's total revenues for full year 2024?
How did RUCONEST perform in Q4 2024 compared to Q4 2023?
What was Joenja's revenue growth in its first full year of sales?
What is Pharming's revenue guidance for 2025?
