Pharming Group reports fourth quarter and full year 2024 financial results and provides business update

• 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 by 9% 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 by 66% 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)

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 21% to US$297.2 million and exceeding our revenue guidance range of US$280-$295 million. This achievement underscores the continued importance of RUCONEST® in the HAE treatment landscape as well as the strong commercial performance of Joenja® in just its first full year of sales.

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$79.6 million, a 9% increase compared to the fourth quarter of 2023. RUCONEST® revenues for the full year 2024 were a record US$252.2 million, an 11% increase compared to 2023, significantly above our expectation for mid single digit percentage growth.

The U.S. market contributed 98% of 2024 revenues, while the EU and Rest of World contributed 2%.

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 6% unit sales growth vs. 2023. We achieved over 100 new patient enrollments in the fourth quarter and total enrollments in 2024 were up 24% vs. 2023. We also increased the RUCONEST® physician prescriber base by 11% during the year, in many cases adding previously unknown HAE prescribers.

Joenja® (leniolisib) marketed in the U.S. - the first and only approved disease modifying treatment for APDS

Joenja® revenues increased to US$13.1 million in the fourth quarter of 2024, a 66% increase compared to the fourth quarter of 2023. This increase was mostly driven by higher volume from the increase in patients on paid therapy in the U.S. compared to the fourth quarter of 2023, and a significant increase in revenues from EU and Rest of World which are from product provided on a named patient basis. Revenue for 2024, the first full year of sales following launch in April 2023, was US$45.0 million, compared to US$18.2 million in 2023.

The U.S. market contributed 90% of 2024 revenues, while the EU and Rest of World contributed 10%.

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 20% of VUS patients being reclassified to APDS, suggesting that there could be a significant increase in the number of APDS patients in the US once those patients with a VUS are reclassified.

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$66.1 million. Abliva's lead product KL1333 is currently in a pivotal clinical trial in mitochondrial DNA-driven primary mitochondrial diseases and has the potential to significantly enhance our future growth trajectory. We announced that we did not require external funding to fund the acquisition and development costs for KL1333.

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 90% and thereby initiated the necessary activities to delist the Company from the Nasdaq Stockholm exchange. Following delisting, we expect to be able to start the second wave of patient recruitment for the ongoing pivotal FALCON clinical trial for KL1333 for the treatment of mtDNA-driven primary mitochondrial diseases. Pharming has initiated a compulsory acquisition procedure in respect of the remaining shares in Abliva under the Swedish Companies Act. On March 3, 2025, Nasdaq Stockholm approved Abliva's application for delisting and the last day of trading will be March 17, 2025. With these events, the acquisition of Abliva is now completed.

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$66.1 million acquisition price to be allocated to the fair value of the acquired identifiable assets and liabilities, with any excess to be recorded as goodwill. We do not expect any P&L impact at the acquisition date, besides recognition of acquisition costs incurred in 2025.

Financial Summary

Fourth quarter 2024

Revenues in the fourth quarter of 2024 increased by 14% to US$92.7 million from US$81.2 million in the fourth quarter of 2023 (US$74.8 million in the third quarter of 2024). RUCONEST® revenues amounted to US$79.6 million, a 9% increase compared to the fourth quarter of 2023. A volume increase in the U.S., and a U.S. price increase in line with CPI, were the primary factors behind this increase in RUCONEST® revenues. Joenja® revenues amounted to US$13.1 million in the fourth quarter of 2024, a 66% increase compared to the fourth quarter of 2023. This increase was primarily driven by an increase in volume.

Gross profit increased by US$6.4 million or 9% to US$80.5 million (4Q 2023: US$74.1 million), mainly due to the increase in revenues. This was partially offset by increased cost of sales, in part due to a one-off inventory impairment due to a power outage during the production process (US$ 1.1 million).

The operating profit amounted to US$6.7 million compared to an operating profit of US$1.1 million in the fourth quarter of 2023. This increase was primarily due to the increase in gross profit mentioned above, offset by the increase in operating expenses from US$73.6 million in the fourth quarter of 2023 to US$73.9 million. Fourth quarter 2024 operating expenses increased by US$9.2 million from US$64.7 million in the third quarter of 2024. Part of this 2024 increase was caused by one-off expenses, totaling US$6.2 million, including the full impairment of US$5.1 million (4Q 2023: US$4.7 million) on the leased DSP facility at Pivot Park in Oss, the Netherlands. Also contributing to the fourth-quarter increase were legal and advisory fees associated with the acquisition of Abliva AB, totaling US$1.1 million for the fourth quarter of 2024.

The net finance result amounted to a gain of US$0.4 million compared to a loss of US$3.8 million in the fourth quarter of 2023. This was primarily driven by favorable EUR/USD exchange rate developments, resulting in a foreign currency gain of US$2.6 million compared to a loss of US$2.9 million in the fourth quarter of 2023. Interest expense increased by US$1.5 million in the fourth quarter of 2024 compared to the previous year, following the convertible bond issuance in the second quarter of 2024.

In the fourth quarter of 2024, a net profit of US$3.7 million was realized, in contrast to a net loss of US$3.1 million in the fourth quarter of 2023. This improvement was primarily driven by the rise in gross profit supported by favorable EUR/USD exchange rate developments, partially offset by the increase in operating expenses.

Cash and cash equivalents, including restricted cash and marketable securities, decreased from US$173.3 million at the end of third quarter of 2024 to US$169.4 million at the end of the fourth quarter of 2024. This decrease was primarily driven by EUR/USD exchange rate developments (vast majority of cash and marketable securities position is EUR denominated), offset by positive cash flows from operations of US$9.3 million (4Q 2023: US$11.6 million), which includes a deduction of US$1.7 million in paid taxes (4Q 2023: US$0.7 million).

Full year 2024

In 2024, Pharming revenues increased by 21% to US$297.2 million. However, the operating loss declined to a loss of US$8.6 million, compared to a loss of US$5.4 million in 2023. Similarly, the net loss declined to US$11.0 million, compared to a net loss of US$10.5 million in 2023.

This section will further elaborate on Pharming's financial performance in 2024.

Revenues and Gross Profit

Total revenues for 2024 grew by 21%, reaching US$297.2 million, compared to US$245.3 million in 2023. Total RUCONEST® revenues were 11% higher at US$252.2 million, compared to revenues of US$227.1 million for 2023. Joenja® revenues amounted to US$45.0 million in 2024, a 147% increase compared to 2023 (first sales commenced at the start of the second quarter of 2023). This increase was primarily driven by an increase in volume.

Cost of sales increased by 40% from US$25.2 million in 2023 to US$35.4 million in 2024. Cost of inventories recognized as expenses in 2024 amounted to US$25.6 million compar