Q4 2025 Pharming Group NV Earnings Call

March 12, 2026

Operator: Good day and thank you for standing by. Welcome to the Pharming Group N.V. Q4 and full year 2025 financial results conference call and webcast. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, please press star one one on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star one one again. Please note that today's conference is being recorded.

Speaker #1: At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. To ask a question during the session, please press star 11 on your telephone.

Speaker #1: You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. Please note that today's conference is being recorded.

Operator: I would now like to turn the conference over to your first speaker, Mr. Fabrice Chouraqui, CEO. Please go ahead, sir.

Speaker #1: I would now like to end the conference over to your first speaker, Mr. Fabrice Chouraqui, CEO. Please go ahead, sir.

Fabrice Chouraqui: Thank you, operator. Good morning and good afternoon, everyone, and welcome to our Q4 and Full Year 2025 Earnings Call. I'll be joined on this call today by Laverne March, our new Chief Commercial Officer, Anurag Relan, our Chief Medical Officer, and Kenneth Lynard, our Chief Financial Officer. On this call, we will be making forward-looking statements that are based upon our current insights and plans. As you know, these may differ from future results. As you saw in our press release, Pharming ended 2025 on a strong note, operationally and financially. Total revenues grew by 15% in the Q4 of 2025, and by 27% for the full year. Thanks to disciplined cost management, we delivered $26 million of operating profits in 2025 compared to a loss in 2024. We also significantly increased our cash position.

Speaker #2: Thank you, Operator. Good morning and good afternoon, everyone, and welcome to our Q4 and full year 2025 earnings call. I'll be joined on this call today by Leaven March, our new Chief Commercial Officer; Anurag Relan, our Chief Medical Officer; and Kenneth Leonard, our Chief Financial Officer.

Speaker #2: On this call, we will be making forward-looking statements that are based upon our current insights and plans. As you know, these may differ from future results.

Speaker #2: As you saw in our press release, farming ended 2025 on a strong note. Operationally and financially. Total revenues grew by 15% in the fourth quarter of 2025 and by 27% for the full year.

Speaker #2: Thanks to disciplined cost management, we delivered €26 million of operating profits in 2025, compared to a loss in 2024. We also significantly increased our cash position.

Fabrice Chouraqui: Operating cash flow came in at $55 million in 2025, putting our cash position at year-end above that of the end of 2024 level before the acquisition of Abliva. 2025 was marked by significant growth of our two commercial assets, RUCONEST and Joenja. RUCONEST grew 26% year-over-year and by 9% in Q4. With its efficacy, with its reliability, and its rapid onset of action, RUCONEST is poised to remain an established on-demand treatment option for difficult to treat patients in an evolving HAE treatment landscape. Laverne will talk more about RUCONEST performance and its unique value proposition.

Speaker #2: Operating cash flow came in at $55 million in 2025, putting our cash position at year-end above that of the end of 2024 level. This is before the acquisition of Abriva.

Speaker #2: 2025 was marked by significant growth of our two commercial assets, Rukanest and Joanja. Rukanest grew 26% year on year and by 9% in the fourth quarter.

Speaker #2: With its efficacy, its reliability, and its rapid onset of action, Rukanest is poised to remain an established on-demand treatment option for difficult-to-treat patients in an evolving HA treatment landscape.

Speaker #2: Leaven will talk more about Rukanest's performance and its unique value proposition. Joanja grew 29% year on year and by 53% in the fourth quarter.

Fabrice Chouraqui: Joenja grew 29% year-over-year and by 53% in Q4, fueled by the acceleration of new patients on drug in the US but also increased demand in international markets, including in the UK, where the drug was launched last spring, and in other countries through purchases under government-supported access program. These results underscore Pharming's transformation from a single asset company into a highly profitable high-growth biotech with two commercial products and a late-stage pipeline with two programs offering billion-dollar sales potential. We highlighted these programs at our Investor Day last month, offering investors unique insights into our high-value pipeline. RUCONEST is the foundation of our portfolio and a reliable cash engine for the future, even in a more crowded HAE on-demand market, given its efficacy profile on the difficult to treat patient sub-subpopulation.

Speaker #2: Fueled by the acceleration of new patients on drug in the US, but also increased demand in international markets including in the UK where the drug was launched last spring, and in other countries through purchasers and the governance-supported access program.

Speaker #2: These results underscore farming's transformation from a single asset company into a highly profitable, high-growth biotech with two commercial products and a late-stage pipeline with two programs offering billion-dollar sales potential.

Speaker #2: We highlighted these programs at our Investor Day last month, offering investors unique insights into our high-value pipeline. Ruconest is the foundation of our portfolio and a reliable cash engine for the future.

Speaker #2: Even in the more crowded HAE on-demand market, given its efficacy profile on the difficult-to-treat patient subpopulation. Joanja is just at the beginning of its life cycle with multiple growth catalysts in APDS, international geographic expansion, and potential expansion into much larger PIDs.

Fabrice Chouraqui: Joenja is just at the beginning of its life cycle, with multiple growth catalysts in APDS, international geographic expansion, and potential expansion into much larger PIDs. Napazimone, which we used to call KL-one triple three for primary mitochondrial disease, is another billion-dollar-plus opportunity, with the registrational study now well underway. This combination of durable revenue, first in disease innovation, and an advancing late-stage pipeline positions Pharming well for substantial near-term and long-term value creation. Let me now turn to our outlook for the remainder of 2026. As announced at our Investor Day in February, we expect 2026 revenue between $405 and $425 million this year, representing 8 to 13% growth, with operating expenses increasing at a slower overall pace, even with substantially higher R&D investment in our pipeline to fuel future growth.

Speaker #2: Nepazimone which we used to call KL1333 for primary mitochondrial disease is another billion-dollar-plus opportunity. With the registration study now well underway. This combination of durable revenue, first in disease innovations, and an advancing late-stage pipeline positioned farming well for substantial near-term and long-term value creation.

Speaker #2: Let me now turn to our outlook for the remainder of 2026. As announced at our investor day in February, we expect 2026 revenue between 405 and 425 million this year.

Speaker #2: Representing an 8 to 13% growth with operating expenses increasing at a slower overall pace even with substantially higher R&D investment in our pipeline to fuel future growth.

Fabrice Chouraqui: We expect continued RUCONEST growth for the reason mentioned earlier, and accelerating Joenja growth fueled by the uptake in APDS patients above 12 years in the US and potential future regulatory approval internationally. Regarding the US pediatric label extension, we've been working to address the FDA request, and we expect to have greater clarity on the resubmission requirements and timeline following the FDA Type A meeting, which is now scheduled at the end of March. 2026 is an important year for our pipeline, with the materialization of potential value inflection points. We have now completed the enrollment of two leniolisib phase 2 trials for larger prevalence PIDs, and we expect a top-line data readout in the second half of this year.

Speaker #2: We expect continued Rukanest growth for the reason mentioned earlier. And accelerating Joanja growth. Fueled by the uptake in APDS patients above 12 years in the US, and potential future regulatory approval internationally.

Speaker #2: Regarding the US pediatric label expansion, we've been working to address the FDA request and we expect to have greater clarity on the resubmission requirements and timeline following the FDA Type A meeting which is now scheduled at the end of March.

Speaker #2: 2026 is an important year for our pipeline with the materialization of potential value inflection points. We have now completed the enrollment of two Leniolisib Phase 2 trials for larger prevalence PIDs and we expect a top-line data readout in the second half of this year.

Fabrice Chouraqui: We also expect to complete enrollment in the napazimone pivotal study this year to be in a position for a data readout at the end of 2027. We are clearly determined to maintain strong financial discipline to optimize capital allocations on our growth drivers. This is critical as we strive to build an efficient and scalable organization, make Pharming a leading rare, ultra-rare disease company, and deliver sustainable value for our shareholders. Let me now turn to Leverne Marsh for deeper insights on the performance of our commercial portfolio.

Speaker #2: We also expect to complete enrollment in the Nepazimone Pivotal Study this year to be in a position for a data readout at the end of 2027.

Speaker #2: We are clearly determined to maintain strong financial discipline to optimize capital allocations on our growth drivers. This is critical as we strive to build an efficient and scalable organization and make farming a leading rare ultra-rare disease company and deliver sustainable value for our shareholders.

Speaker #2: Let me now turn to Leaven March for deeper insights on the performance of our commercial portfolio.

Laverne March: Good day, everybody. Let me start with RUCONEST, where the US business delivered another year of strong and resilient performance. In 2025, despite the first new wave of treatment options in the HAE market in almost five years, RUCONEST continued to grow as an essential therapy for patients living with severe high-frequency HAE attacks. Our strategy remains consistent. Focus on high-attack patients who require fast, reliable, on-demand treatment and ensure that we continue to execute against that differentiated value proposition even as new engineered agents enter the market. For the full year, RUCONEST delivered 26% global revenue growth and a volume growth in the US of 20%. A clear indicator of the strong and durable demand for RUCONEST in the acute segment.

Speaker #3: Good day, everybody. Let me start with Rukanest. We're the US business delivered another year of strong and resilient performance. In 2025, despite the first new wave of treatment options in the HAE market in almost five years, Rukanest continued to grow as an essential therapy for patients living with severe high-frequency HAE attacks.

Speaker #3: Our strategy remains consistent: focus on high-attack patients who require fast, reliable on-demand treatment, and ensure that we continue to execute against that differentiated value proposition even as new agents enter the market.

Speaker #3: For the full year, Rukanest delivered 26% global revenue growth and a volume growth in the US of 20%. A clear indicator of the strong and durable demand for Rukanest in the acute segment.

Laverne March: In Q4, we delivered 9% global revenue growth versus the prior year quarter and a 2% volume growth in the core US market, continuing the upward trajectory of RUCONEST. As expected, through Q4, we began to observe impact of newly launched therapies for HAE in the US market, with some patients trialing and some already returning to RUCONEST. Despite these competitive dynamics, we welcomed over 60 new enrollments in the US in Q4, slightly above Q3. RUCONEST added both new patients and new prescribers in the quarter, underscoring the resilience of our position in the difficult-to-treat patient segments and the clinical trust placed in RUCONEST in real-world settings. That said, the high burden of HAE matters for patients who have high-frequency attacks. Attacks are often unpredictable and potentially life-threatening, and symptom severity often escalate within hours.

Speaker #3: In quarter four, we delivered 9% global revenue growth versus the prior year quarter and a 2% volume growth in the core US market continuing the upward trajectory of Rukanest.

Speaker #3: As expected, through Q4, we began to observe the impact of newly launched therapies for HAE in the US market, with some patients trialing and some already returning to Ruconest.

Speaker #3: Despite these competitive dynamics, we welcomed over 60 new enrollments in the US in the fourth quarter, slightly above Q3. Rukanest added both new patients and new prescribers in the quarter underscoring the resilience of our position in the difficult-to-treat patient segments and the clinical trust placed in Rukanest in real-world settings.

Speaker #3: That said, the high burden of HAE matters for patients who have high-frequency attacks. Attacks are often unpredictable and potentially life-threatening and symptom severity often escalates within hours.

Laverne March: Importantly, for patients experiencing multiple attacks per month or patients who experience suboptimal responses to other options, dependable rapid relief is not optional, it is essential. This high-frequency attack segment is precisely where we have seen consistent, durable use of RUCONEST over time and where we expect RUCONEST to remain highly relevant even as new treatments enter the market. To that end, a meaningful proportion of new patient enrollments in the US are switches to RUCONEST from other on-demand therapies, further emphasizing the continued need that high-attack patients have for an effective, reliable one-and-done therapy like RUCONEST. When you put this together, the unpredictability of the disease, the high burden carried by certain patient segments, the limitations of some other acute therapies, and consistently strong clinical performance associated with RUCONEST, the unique value proposition for RUCONEST remains clear.

Speaker #3: Importantly for patients experiencing multiple attacks per month or patients who experience suboptimal responses to other options, dependable rapid relief is not optional. It is essential.

Speaker #3: And this high-frequency attack segment is precisely where we have seen consistent durable use of Rukanest over time and where we expect Rukanest to remain highly relevant even as new treatments enter the market.

Speaker #3: To that end, a meaningful proportion of new patient enrollments in the US are switches to Rukanest from other on-demand therapies. Further emphasizing the continued need that high-attack patients have for an effective, reliable, one-and-done therapy like Rukanest.

Speaker #3: And so when you put this together, the unpredictability of the disease, the high burden carried by certain patient segments, the limitations of some other acute therapies and consistently strong clinical performance associated with Rukanest the unique value proposition for Rukanest remains clear.

Laverne March: Looking ahead, we foresee some pressure on our growth early in the year, but with no change in the need for an effective, rapid onset, reliable one-dose treatment like RUCONEST. With that in mind, let me turn to Joenja. For Joenja, we delivered a strong Q4, building on the momentum we established throughout the year. Revenue grew 53% compared to Q4 in 2024, reaching $19.8 million globally. For the full year, Joenja generated $58 million, representing 29% growth year-over-year and demonstrating both sustained utilization from patients and the expanding clinical recognition and treatment of APDS. In the United States, patient growth remained a central driver of performance. By the end of 2025, we had 120 patients on paid therapy, representing a 25% increase over year-end 2024.

Speaker #3: Looking ahead, we foresee some pressure on our growth early in the year but with no change in the need for an effective, rapid-onset reliable one-dose treatment like Rukanest.

Speaker #3: With that in mind, let me turn to Joanja. For Joanja, we delivered a strong fourth quarter building on the momentum we established throughout the year.

Speaker #3: Revenue grew 53% compared to the fourth quarter in 2024 reaching $19.8 million globally. For the full year, Joanja generated $58 million representing 29% growth year-on-year and demonstrating both sustained utilization from patients and the expanding clinical recognition and treatment of APDS.

Speaker #3: In the United States, patient growth remained a central driver of performance. By the end of 2025, we had 120 patients on paid therapy representing a 25% increase over year-end 2024.

Laverne March: This steady expansion of our treated population reflects strong physician confidence, consistent engagement with patient communities, and a team that executes with discipline and with urgency. Equally important, we made significant progress in broadening the pool of identified APDS patients, one of the most critical leading indicators in an ultra-rare disease. In 2025, the number of US patients we identified diagnosed with APDS increased by 40, more than double the increase of 18 we saw in 2024. This growth in identified patients with APDS shows that our educational efforts, our diagnostic partnerships, and our medical engagement in the US are working. Outside the US, we saw strengthening demand across international markets, including a solid first-year uptake in the United Kingdom following the launch in April 2025. We also benefited from government-supported access programs, which allowed us to reach more patients who currently have limited therapeutic options.

Speaker #3: This steady expansion of our treated population reflects strong physician confidence, consistent engagement with patient communities, and a team that executes with discipline and with urgency.

Speaker #3: Equally important, we made significant progress in broadening the pool of identified APDS patients. One of the most critical leading indicators in an ultra-rare disease.

Speaker #3: In 2025, the number of US patients we identified diagnosed with APDS increased by 40 more than double the increase of 18 we saw in 2024.

Speaker #3: This growth in identified patients with APDS shows that our educational efforts, our diagnostic partnerships, and our medical engagement in the US are working. Outside the US, we saw strengthening demand across international markets including a solid first-year uptake in the United Kingdom following the launch in April 2025.

Speaker #3: We also benefited from government-supported access programs which allowed us to reach more patients who currently have limited therapeutic options. Taken together, these results give us a strong platform for Joanja in the years ahead.

Laverne March: Taken together, these results give us a strong platform for Joenja in the years ahead. Finally, we expect geographic expansion and the anticipated 4- to 11-year approval in the United States to be meaningful contributors to growth. These two catalysts remain materially important to increase the number of patients who can benefit from Joenja and expand the global footprint of the APDS business. Internationally, we've already demonstrated our ability to execute. In the UK, where Joenja launched in April 2025, we have seen solid early uptake and engagement from the community. That success reinforces that our teams have the capability, the infrastructure, and the strategic focus needed to deliver in new markets. In the United States, our commercial teams are fully prepared to launch Joenja for children ages 4 to 11, pending FDA approval.

Speaker #3: Finally, we expect geographic expansion and the anticipated four to 11-year approval in the United. States to be meaningful contributors to growth. These two catalysts remain materially important to increase the number of patients who can benefit from Joanja and expand the global footprint of the APDS business.

Speaker #3: Internationally, we've already demonstrated our ability to execute. In the UK, where Joanja launched in April 2025, we have seen solid early uptake and an encouragement for complicity.

Speaker #3: That success reinforces that our teams have the capability, the infrastructure, and the strategic focus needed to deliver in new markets. In the United States, our commercial teams are fully prepared to launch Joenja for children ages 4 to 11, pending FDA approval. Importantly, we already have 52 eligible patients identified, one-third of them currently on therapy through our early access program, ready to transition at approval.

Laverne March: Importantly, we already have 52 eligible patients identified, one-third of them currently on therapy through our early access program, ready to transition at approval. This will give us a running start and positions us for early momentum once the label is approved. Beyond the US, our international organization is deeply engaged in progressing regulatory submissions and ensuring that reimbursement discussions can start when approvals are granted across Europe, Japan, and Canada. Across these three regions, we have over 80 patients already receiving Joenja through early access mechanisms, awaiting full regulatory approval and commercial availability. This represents a significant built-in foundation for launch acceleration once those approvals are secured. We're stepping into 2026, we do so with confidence.

Speaker #3: This will give us a running start and positions us for early momentum once the label is approved. Beyond the US, our international organization is deeply engaged in progressing regulatory submissions and ensuring that reimbursement discussions can start when approvals are granted across Europe, Japan, and Canada.

Speaker #3: Across these three regions, we have over 80 patients already receiving Joenja through early access mechanisms, awaiting full regulatory approval and commercial availability. This represents a significant built-in foundation for launch acceleration once those approvals are secured.

Speaker #3: And so, as we're stepping into 2026, we do so with confidence. For Joenja, we have the right growth catalysts in front of us, and we have an organization that has demonstrated that it can execute launches with precision and continuity.

Laverne March: For Joenja, we have the right growth catalysts in front of us, and we have an organization that has demonstrated that it can execute launches with precision and continuity. This gives us confidence not just in the next quarter, but in the sustained global expansion of Joenja over the coming years. With that, I'll now hand over to Dr. Anurag Relan, our Chief Medical Officer, who will walk you through our progress across the pipeline and the upcoming development and regulatory milestones.

Speaker #3: This gives us confidence not just in the next quarter but in the sustained global expansion of Joanja over the coming years. And with that, I'll now hand over to Dr. Anurag Relan, our chief medical officer, who will walk you through our progress across the pipeline and the upcoming development and regulatory milestones.

Anurag Relan: Thank you, Laverne. As we discussed at our Investor Day in February, PI3K delta is a master regulator of the immune system, and imbalances here contribute to immune dysregulation in a number of primary immune deficiencies or PIDs. This understanding serves as the foundation and rationale for our Joenja development efforts. APDS, where Joenja is currently approved, is a primary immune deficiency caused by a genetic defect that leads to PI3K delta hyperactivation. This results in the dysfunction of the immune system and is characterized by frequent and severe infections and a wide array of immune dysregulation consequences, as you see here. APDS is a progressive disease and leads to early mortality due to these complications, with unfortunately about 25% of patients dying by the age of 30.

Speaker #1: Thank you, Laverne. As we discussed at our investor day in February, PI3K-Delta is a master regulator of the immune system, and imbalances here contribute to immune dysregulation in a number of primary immune deficiencies, or PIDs.

Speaker #1: This understanding serves as the foundation and rationale for our Joanja development efforts. APDS, where Joanja is currently approved, is a primary immune deficiency caused by a genetic defect that leads to PI3K-Delta hyperactivation.

Speaker #1: This results in the dysfunction of the immune system and is characterized by frequent and severe infections and a wide array of immune dysregulation consequences as you see here.

Speaker #1: APDS is a progressive disease and leads to early mortality due to these complications, with unfortunately about 25% of patients dying by the age of 30.

Anurag Relan: Based on this understanding of APDS, we have two ongoing phase 2 proof-of-concept clinical trials evaluating leniolisib in more prevalent PIDs, which share unmet medical needs, mechanisms, and disease pathology with APDS. These include the genetically identified primary immunodeficiencies with immune dysregulation linked to altered PI3K delta signaling and common variable immunodeficiency, or CVID, with immune dysregulation, which is identified independently of genetics. As Fabrice mentioned, both of these studies have now completed enrollment. As you see here, APDS falls under the broader CVID umbrella diagnosis, and Joenja, in fact, serves as a proof of concept for the work we are doing now in these much more prevalent PIDs. This slide highlights the opportunity now to broaden the use of Joenja.

Speaker #1: Based on this understanding of APDS, we have two ongoing Phase 2 proof-of-concept clinical trials evaluating leniolisib in more prevalent PIDs, which share unmet medical needs, mechanisms, and disease pathology with APDS.

Speaker #1: These include the genetically identified primary immune deficiencies with immune dysregulation linked to altered PI3K-Delta signaling and common variable immune deficiency or CVID with immune dysregulation which is identified independently of genetics.

Speaker #1: And as Fabrice mentioned, both of these studies have now completed enrollment. And as you see here, APDS falls under the broader CVID umbrella diagnosis and Joanja, in fact, serves as a proof of concept for the work we are doing now in these much more prevalent PIDs.

Anurag Relan: The prevalence figures here are for the US, but they illustrate the larger opportunity to serve patients and underpin peak sales potential above $1 billion. We're very excited about the work that I just discussed to study Joenja in these additional PIDs with immune dysregulation beyond APDS. These address significantly larger patient populations, which are 5 to 26 times the prevalence of APDS. In APDS, Laverne already discussed progress with patient identification and our commercial preparations for geographic and pediatric expansion. Let me update you now on the variant of uncertain significance project and the opportunity there. Following various discussions over many months involving Columbia and genetic testing labs, it became clear that the labs require additional evidence to reclassify VUSs. Understanding the consequences of VUSs remains a significant unmet need and actually a public health problem for clinicians and patients.

Speaker #1: This slide highlights the opportunity now to broaden the use of Joanja. The prevalence figures here are for the US, but they illustrate the larger opportunity to serve patients and underpin peak sales potential above 1 billion dollars.

Speaker #1: We're very excited about the work that I just discussed to study Joanja in these additional PIDs with immune dysregulation beyond APDS these address significantly larger patient populations which are 5 to 26 times the prevalence of APDS.

Speaker #1: In APDS, Laverne already discussed progress with patient identification and our commercial preparations for geographic and pediatric expansion. But let me update you now on the variants of uncertain significance project and the opportunity there.

Speaker #1: Following various discussions over many months involving Colombia and genetic testing labs, it became clear that the labs require additional evidence to reclassify the USs.

Speaker #1: Understanding the consequences of the USs remains a significant unmet need and actually a public health problem for clinicians and patients, and as you see, the number of patients with the US continues to grow.

Anurag Relan: As you see, the number of patients with VUS continues to grow. To complement the significant first batch of data which were published in Cell, we are now planning new experiments to generate the data needed to allow genetic testing labs to evaluate VUSs. Following completion of these experiments, we plan to provide an estimate how many VUSs may be reclassified and how many patients may be ultimately diagnosed with APDS. In addition, the work published in Cell also suggests that the prevalence of APDS could be significantly higher than we currently estimate. We convened a global advisory board and are now initiating work to explore and better understand the prevalence of APDS, as well as the spectrum of disease. I will now cover the progress we are making in APDS, including some of our near-term regulatory milestones.

Speaker #1: To complement the significant first batch of data, which were published in Cell, we are now planning new experiments to generate the data needed to allow genetic testing labs to evaluate the USs.

Speaker #1: Following completion of these experiments, we plan to provide an estimate how many the USs may be reclassified and how many patients may be ultimately diagnosed with APDS.

Speaker #1: In addition, the work published in Cell also suggested the prevalence of APDS could be significantly higher than we currently estimate. We convened a global advisory board and are now initiating work to explore and better understand the prevalence of APDS as well as the spectrum of disease.

Speaker #1: I'll now cover the progress we are making in APDS including some of our near-term regulatory milestones. Overall, we made good progress in APDS during 2025 and early this year.

Anurag Relan: Overall, we made good progress in APDS during 2025 and early this year. While we are disappointed in the complete response letter we received from FDA in January regarding our regulatory submission for the pediatric label expansion for Joenja for the treatment of APDS in children ages 4 to 11, we believe we can address both the clinical pharmacology and analytical batch testing methodology issues outlined in the letter. A Type A meeting has now been scheduled with FDA for later this month, and we expect to discuss the agency's feedback and align on the path forward for resubmission. In Europe, we have filed a marketing authorization application for leniolisib for patients 12 years and older, and now responded to CHMP's questions on manufacturing activities and quality controls and believe we have addressed their concerns.

Speaker #1: While we are disappointed in the complete response letter we received from FDA in January, regarding our regulatory submission for the pediatric label expansion for Joanja, for the treatment of APDS in children ages 4 to 11, we believe we can address both the clinical pharmacology and analytical batch testing methodology issues outlined in the letter.

Speaker #1: A Type A meeting has now been scheduled with the FDA for later this month, and we expect to discuss the agency's feedback and align on the path forward for resubmission.

Speaker #1: In Europe, we have filed a marketing authorization application for Leniolisib for patients 12 years and older and now responded to CHMP's questions on manufacturing activities and quality controls and believe we have addressed their concerns.

Anurag Relan: We now expect a CHMP opinion on the MAA to take place at their meeting later this month, with potential EC approval in the first half of this year. Regarding the Japan NDA for leniolisib for the treatment of APDS in patients 4 years of age and over, the Pharmaceutical Affairs and Food Sanitation Council meeting has recommended approval. This news was covered in The Pink Sheet, who importantly noted that this would represent the first approval for children with APDS ages 4 to 11. We now await the formal decision by the PMDA by the end of March. I'll now turn to our next pipeline asset, napazimone, or formerly known as KL1333. Napazimone has also progressed significantly in the past year.

Speaker #1: We now expect a CHMP opinion on the MAA to take place at their meeting later this month, with potential EC approval in the first half of this year.

Speaker #1: Regarding the Japan NDA for Leniolisib for the treatment of APDS in patients 4 years of age and older, the pharmaceutical affairs council meeting has recommended approval.

Speaker #1: This news was covered in the pink sheet who importantly noted that this would represent the first approval for children with APDS ages 4 to 11.

Speaker #1: We now await the formal decision by the PMDA by the end of March. I'll now turn to our next pipeline asset, the Pazimome or formerly known as KL1333.

Anurag Relan: This is being developed for primary mitochondrial diseases, which is a group of rare disorders where mutations in mitochondrial DNA lead to significant fatigue and muscle weakness. Napazimone addresses this underlying disorder by normalizing the NAD+ to NADH ratio, which is abnormally low in these patients. There are a large number of these patients already diagnosed across the US and large European countries, where they are treated at centers of excellence, and part of strong advocacy groups. Here we have a registration-enabling study underway with endpoints agreed upon with FDA. Importantly, there was a blinded interim analysis in which both endpoints passed utility. Since completing the acquisition of Abliva last year, we are making good progress in the second wave of the study and are on track to complete enrollment later this year, with readout in 2027 and potential approval later in 2028.

Speaker #1: Pazimome has also progressed significantly in the past year. This is being developed for primary mitochondrial diseases, which is a group of rare disorders where mutations in mitochondrial DNA lead to significant fatigue and muscle weakness.

Speaker #1: The Pazimome addresses this underlying disorder by normalizing the NAD+ to NADH ratio, which is abnormally low in these patients. There are a large number of these patients already diagnosed across the US and large European countries, where they are treated at centers of excellence and are part of strong advocacy groups.

Speaker #1: And here we have a registration-enabling study underway, with endpoints agreed upon with the FDA. And importantly, there was a blinded interim analysis in which both endpoints passed futility.

Speaker #1: Since completing the acquisition of Ableva last year, we are making good progress in the second wave of the study and are on track to complete enrollment later this year with readout in 2027 and potential approval later in 2028.

Anurag Relan: With that, I'll turn it over to Kenneth now to discuss our financial results.

Speaker #1: And with that, I'll turn it over to Kenneth now to discuss our financial results.

Kenneth Lynard: Thank you very much, Anurag. I'm pleased to now provide some color on our strong 2025 financial performance and our outlook for 2026. Q4 was a strong finish, with revenues of EUR 106.5 million, being 15% growth versus Q4 of 2024. This was driven by continued momentum across our portfolio, including a 9% growth for RUCONEST and a 53% growth for Joenja. Notably, Joenja annual revenues exceeded EUR 50 million for the first time, triggering our first EUR 5 million sales milestone payment in the quarter. As a reminder, this milestone is recorded in cost of goods sold, and therefore affected gross margin for Q4. Adjusted operating profit was broadly stable year-over-year after several offsetting one-off items.

Speaker #2: Thank you very much, Anurag. I'm pleased to now provide some color on our strong 2025 financial performance and our outlook for 2026. The fourth quarter was a strong finish with revenues of 106.5 million being 15% growth versus Q4 of 2024.

Speaker #2: This was driven by continued momentum across our portfolio including a 9% growth for Brukunest and a 53% growth for Joanja. Notably, Joanja annual revenues exceeded 50 million for the first time triggering our first 5 million sales milestone payment in the quarter.

Speaker #2: As a reminder, this milestone is recorded in cost of goods sold and therefore affected gross margin for the fourth quarter. Adjusted operating profit was broadly stable year over year after several offsetting one-off items.

Kenneth Lynard: Q4 2025 expenses include EUR 9.3 million in expenses related to Abliva and napazimone following the completion of the Abliva acquisition this year, as well as the EUR 5 million Joenja sales milestone payment just mentioned. Excluding these items to compare operating profit on a like-for-like basis to Q4 2024, operating profit in Q4 2025 would have been EUR 14 million higher. Finally, cash and marketable securities increased by EUR 12.2 million, from EUR 168.9 million at the end of Q3 to EUR 181 million at year-end, primarily driven by positive operating cash flow, reflecting the strength of our commercial performance. Turning to our full year 2025 results, our financials shows the continued strong execution of our strategy.

Speaker #2: Fourth quarter 2025 expenses include $9.3 million in expenses related to Ableva and Pazimome following the completion of the Ableva acquisition this year, as well as the $5 million Joanja sales milestone payment just mentioned.

Speaker #2: Excluding these items to compare operating profit on a like-for-like basis to the fourth quarter of 2024, operating profit in the fourth quarter of '25 would have been 14 million higher.

Speaker #2: Finally, cash and marketable securities increased by 12.2 million from 168.9 million at the end of Q3 to 181 million at the year end primarily driven by positive operating cash flow reflecting the strength of our commercial performance.

Speaker #2: Turning to our full year 2025 results, our financials show the continued strong execution of our strategy. Total revenues increased 27% to €376.1 million, driven by robust double-digit growth from both Brukunest and Joenja.

Kenneth Lynard: Total revenues increased 27% to $376.1 million, driven by robust double-digit growth from both RUCONEST and Joenja. Gross margin remained stable at approximately 88%, despite the $5 million Joenja sales milestone recorded in Q4. Operating expenses rose to $311.3 million. Excluding $4.1 million of one-off restructuring costs related to our G&A reduction program announced in October, operating expenses were $307.2 million, and within our previously communicated guidance range of $304 to 308 million. Importantly, when also excluding the full $29.7 million of Abliva-related expenses, operating expenses increased only 2% on a like-for-like basis. This reflects disciplined cost management.

Speaker #2: Gross margin remained stable at approximately 88% despite the 5 million Joanja sales milestone recorded in the fourth quarter. Operating expenses rose to 311.3 million excluding 4.1 million of one-off restructuring costs related to our GNA reduction program announced in October operating expenses were 307.2 million and within our previously communicated guidance range of 304 to 308 million.

Speaker #2: Importantly, when also excluding the full 29.7 million of Ableva related expenses operating expenses increased only 2% on a like-for-like basis this reflects disciplined cost management.

Kenneth Lynard: In total, adjusted operating profit, which exclude non-recurring Abliva acquisition-related costs and other offsetting items, was EUR 36.4 million, compared with a loss of EUR 8.6 million in 2024. Excluding recurring Abliva expenses and the EUR 5 million Joenja sales milestone, operating profit for 2025 would have been EUR 24.4 million higher. Cash flow from operating activities totaled EUR 54.7 million versus being slightly negative in 2024, showing the improved profitability and cash generation of the business. Cash and marketable securities increased EUR 11.7 million to EUR 181.1 million, despite EUR 68 million used for the Abliva acquisition. Again, highlighting the strength of our underlying operational cash generation.

Speaker #2: In total adjusted operating profit which exclude non-recurring Ableva acquisition related cost and other offsetting items was 36.4 million compared with a loss of 8.6 million in 2024.

Speaker #2: Excluding recurring Ableva expenses and the 5 million Joanja sales milestone operating profit for 2025 would have been 24.4 million higher. Cash flow from operating activities totaled 54.7 million versus

Speaker #1: Just being slightly negative in 2024 , showing the improved profitability and cash generation of the business Cash and marketable securities increased 11.7 million to 181.1 million , despite 68 million used for the A-b-e-b-a acquisition .

Speaker #1: Again highlighting the strength of our underlying operational cash generation. Turning to our 2026 outlook, we reaffirm our expectation for total revenues of €405 to €425 million, representing full-year growth of approximately 8 to 13% versus 2025.

Kenneth Lynard: Turning to our 2026 outlook, we reaffirm our expectation for total revenues of EUR 405 to 425 million, representing full year growth of approximately 8% to 13% versus 2025. The growth is expected to be driven by continued growth for RUCONEST in the US, partly offset by declining ex-US revenue as we exit those markets and accelerated growth for Joenja. For RUCONEST, quarterly revenue typically fluctuates with patient ordering patterns and channel inventory movements, with the first quarter usually being the lowest. In Q1 2026, inventory drawdowns are expected to impact US RUCONEST revenue growth by 7 to 9% year-over-year as market dynamics settle. This is factored into our full year guidance, which assumes mid-single-digit RUCONEST growth at the midpoint of the range.

Speaker #1: The growth is expected to be driven by continued growth for Ruconest in the US, partly offset by declining ex-US revenue, as we exit those markets, and accelerated growth for Georgia.

Speaker #1: For Ruconest quarterly revenue typically fluctuates with patient ordering patterns and channel inventory movements . With the first quarter usually being the lowest in Q1 2026 .

Speaker #1: Inventory drawdowns are expected to impact us Ruconest revenue growth by 7 to 9% year on year , as market dynamics settle . This is factored into our full year guidance , which assumes mid-single digit ruconest growth at the midpoint of the range .

Kenneth Lynard: For Joenja, we expect growth to accelerate with annual growth approximately 10 percentage points higher than in 2025. The pediatric APDS indication remains an important future growth driver, and we look forward to clarity on the US approval timeline for patients aged four to 11 following the upcoming FDA Type A meeting. In the meantime, US pediatric revenues are excluded from our 2026 guidance. For operating expenses, we anticipate a range of EUR 330 to 335 million, including more than EUR 60 million of incremental R&D investments. This guidance also reflects the EUR 9 million favorable impact from the 20% G&A headcount reduction program announced in October 2025, along with stable marketing and sales spending. Overall, we remain committed to financial discipline, prioritizing investment that drive near and long-term value creation for our shareholders.

Speaker #1: For Joenja, we expect growth to accelerate, with annual growth approximately ten percentage points higher than in 2025. The pediatric APDs indication remains an important future growth driver, and we look forward to clarity on the US approval timeline for patients aged 4 to 11.

Speaker #1: Following the upcoming FDA type A meeting . In the meantime . US pediatric revenues are excluded from our 2026 guidance for operating expenses .

Speaker #1: We anticipate a range of 330 to 335 million , including more than 60 million of incremental R&D investments . This guidance reflects also reflects the 9 million favorable impact from the 20% G&A headcount reduction program announced in October 2025 , along with stable marketing and sales spending Overall , we remain committed to financial discipline , prioritizing investment that drive near and long term value creation for our shareholders .

Kenneth Lynard: Because RUCONEST revenue is not expected to exceed $100 million in 2026, we do not assume the $10 million commercial milestone payments, which otherwise would be recorded in cost of goods sold. As communicated during our investor day in February and aligned with ex-US rollout plans, no additional milestone payments are expected. We estimate cost of goods sold at 10% of revenues corresponding to a gross margin of 90%. Finally, available cash and future operating cash flows are expected to fully support pipeline investments, including all pre-launch activities. With that, I will now hand over to Fabrice for his closing remarks.

Speaker #1: Because growing revenue is not expected to exceed $100 million in 2026, we do not assume the $10 million commercial milestone payments, which otherwise would be recorded in cost of goods sold as communicated during our Investor Day in February and aligned with ex-US rollout plans.

Speaker #1: No additional milestone payments are expected . We estimate cost of goods sold at 10% of revenues , corresponding to a gross margin of 90% .

Speaker #1: Finally , available cash and future operating cash flows are expected to fully support pipeline investments , including all pre-launch activities . With that , I'll now hand over to Fabrice for his closing remarks Thank you Kenneth .

Fabrice Chouraqui: Thank you, Kenneth. In summary, we are pleased to report another strong quarter and a record year for Pharming in 2025 with $376 million revenues and a shift to operating profitability and positive operating cash flow. Looking ahead, RUCONEST is poised to remain a cornerstone treatment for severe HAE patients, underpinning a strong revenue base. We see clear revenue catalysts ahead for Joenja with the product well positioned to generate a significant proportion of our revenues in the future. Our upcoming clinical data readouts, including the leniolisib phase 2 later this year and the napazimone pivotal study readout next year, each have the potential to unlock significant value and take the company to a whole new level. Finally, the decisive steps we have taken to improve financial discipline will support driving the positive bottom line.

Speaker #1: So in summary .

Speaker #2: We are pleased to report another strong quarter and a record year for farming in 2025 , with $376 million of revenues and a shift to operating profitability and positive operating cash flow Looking ahead , Ruconest is poised to remain a cornerstone treatment for severe Ha patients , underpinning a strong revenue base .

Speaker #2: We see clear revenue catalysts ahead for joint year with the product. Well positioned to generate a significant proportion of our revenues in the future.

Speaker #2: Our upcoming clinical data readouts , including the two Leniolisib phase two later this year and the pivotal study readout next year , each have the potential to unlock significant value and take the company to a whole new level And finally , the decisive steps we have taken to improve financial discipline will support driving the positive bottom line with strong commercial and development capabilities , a fit for purpose leadership team with strong new additions like Laverne and Kenneth , and a scalable organization .

Fabrice Chouraqui: With strong commercial and development capabilities, a fit-for-purpose leadership team with strong new additions like Laverne and Kenneth, and a scalable organization, we are committed to making 2026 another stepping stone in achieving our vision of becoming a leading global rare disease company. Let me now open the line for questions.

Speaker #2: We are committed to making 2026 another stepping stone in achieving our vision of becoming a leading global rare disease company. Let me now open the line for questions.

Operator: Thank you. As a reminder to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. Once again, please press star one one to ask a question and wait for your name to be announced. To withdraw your question, please press star one one again. Thank you. We are now going to proceed with our first question. The question comes from the line of Lucy Codrington from Jefferies. Please ask your question.

Speaker #3: Thank you As a reminder to ask a question , please press star one one on your telephone and wait for your name to be announced .

Speaker #3: To withdraw your question , please press star one one again . Once again , please press star one one to ask a question and wait for your name to be announced .

Speaker #3: To withdraw your question, please press star one. Once again, thank you. We are now going to proceed with our first question. And the questions come from the line of Lucy Codrington from Jefferies.

Lucy Codrington: Hi. Thank you for taking my questions. To begin with Joenja growth this year, how much of that are you expecting to come from the US market versus international markets? Secondly, looking at the US market, of those 61 eligible patients that are not on treatment in the US, how many of these do you think could be converted to paid therapy, or how many have been versus how many have been considered and ruled out? Finally, in terms of RUCONEST dynamics, you mentioned that you have heard of patients coming back to treatment, having tried the orals. Do you have any, I guess, it would be anecdotal, commentary on how quickly the patients return having tried the orals? Thank you.

Speaker #3: Please ask your question Hi .

Speaker #4: Thank you for taking my questions . To begin with , Joenja growth this year . How much of that are you expecting to come from the US market versus international markets ?

Speaker #4: And then secondly , looking at the US market of those 61 eligible patients that are not on treatment in the US , how many of these do you think could be converted to paid therapy ?

Speaker #4: Or how many have been versus how many have been considered and ruled out ? And then finally , in terms of ruconest dynamics , you mentioned that you have heard of patients coming back to treatment , having tried the orals .

Speaker #4: Do you have any , I guess it would be anecdotal . Commentary on how quickly the patient's return . Having tried the orals .

Fabrice Chouraqui: Very good. Thank you so much, Lucy, for those questions. I'll turn to Laverne in a minute, actually. Let me start with the last one on RUCONEST, and then we can take your two questions on Joenja. It is true that we've seen some patients trying and coming back. As you know and as Laverne reinforced, HA is a very severe disease, and patients got to be controlled. Especially patients who were used to highly reliable treatments.

Speaker #4: Thank you

Speaker #2: Very good . Thank you so much , Lucy , for for those questions . I'll I'll turn to to live in a minute .

Speaker #2: Actually , let me start with the last one on Ruconest and then we can take your two questions on , on , on Joenja .

Speaker #2: It is true that we've seen some patients trying and coming back As you know , and as Laverne reinforced , ha is a is a is a very severe disease .

Speaker #2: And , and and patients got to be controlled and specifically patients who were used to a high highly reliable treatments . And so when those patients try another treatment and see that their , their crisis is not properly controlled , they tend to to come back to , to their previous medication fairly quickly .

Fabrice Chouraqui: When those patients try another treatment and see that their crisis is not properly controlled, they tend to come back to their previous medication fairly quickly, because not controlling a crisis could be life-threatening. Now, when it comes to Joenja's growth, as we said, we see the growth in this year in 2026 being fueled both by the continued growth in the US as well as growth increasing in international markets. It's obviously the growth in international markets will come from the UK, where the drug has been launched, new launch countries.

Speaker #2: Because not controlling a crisis could be life threatening Now , when it comes to , to , to join growth , as we've said , we see the growth in this year , in 2026 being fueled both by the continued growth in the US as well as growth increasing in international markets It's obviously the growth in international markets will come from the UK , where the drug has been launched New launch countries and this will will happen in a staged fashion .

Fabrice Chouraqui: This will happen in a staged fashion, since once we receive marketing approval, we'll have to negotiate a price and reimbursement. Ultimately a bigger portion of the growth will come from international market, but that's gonna be gradual. When it comes to more specificities on Joenja's patient funnel, I'll ask Laverne to comment.

Speaker #2: Since once we receive marketing approval . We'll have to to negotiate a price and reimbursement . So ultimately a bigger portion of of the growth will come from international market .

Speaker #2: But that's going to be gradual when it comes to more specificities on , on , on joint jazz patient funnel I'll ask Laverne to comment

Laverne March: Thank you. Thank you, Lucy, for your question. How we would think about the patients considered for Joenja, or eligible for Joenja in the US and the pull-through to patients on therapy, that there would be a lag there as patients are going through the patient journey, a fairly complicated patient journey, seeing multiple physicians, the enrollment, and then the reimbursement process, before we get to patients on paid therapy. How I would think about the delta there and the opportunity, there's still a substantial proportion of patients both eligible and potentially reimbursable that would drive growth for us in the US in 2026.

Speaker #5: Thank you . Thank you , Lucy , for for your question . How we would think about the patient's considered for joenja or eligible for Joenja in the US .

Speaker #5: And the pull-through to patients on therapy—that there would be a lag there as patients are going through the patient journey. A fairly complicated patient journey.

Speaker #5: Seeing multiple physicians . The enrollment and then the reimbursement process Before we get to patients on paid therapy . And so how I would think about the delta there and the opportunity , there's still a substantial proportion of patients , both eligible and potentially reimbursable , that would drive growth for us in the US in 2026 .

Kenneth Lynard: Maybe, Lucy, this is Kenneth Lynard speaking. Maybe I can just add, out of the expected Joenja growth in 2026, it will probably be around about 70, 75 percent that will come from the US.

Speaker #1: Maybe , Lucy , this is Kenneth speaking . Maybe I can just add out of the expected growth in 2026 . It will probably be around about 7,075% .

Lucy Codrington: 70 to 75% from the US?

Speaker #1: That will come from the US

Kenneth Lynard: Of the growth in 2026 for Joenja, yes.

Lucy Codrington: Got it. Thank you very much.

Speaker #4: So, 70, 75% from the US.

Speaker #1: Of the growth in 2026 for Joenja . Yes .

Operator: We are now going to proceed with our next question. The question comes from the line of Joseph Pantginis from H.C. Wainwright & Co. Please ask your question.

Speaker #4: Got it . Thank you very much

Speaker #3: We are now going to proceed with our next question And the questions come from the line of Zhou Panjwani from H c Wainwright .

Joseph Pantginis: Hey, everybody. Good morning. Thanks for taking the questions. I know you might not be able to give color here ahead of the Type A, but I'm gonna ask anyway. Obviously, the reasons, you know, for your discussions, as Anurag said, were the clinical pharmacology and the analytical batches. Is there anything that you would consider sort of the lead rate-limiting step here? That's question number one. Question number two regarding RUCONEST. You touched upon this a little bit, but I guess, you know, the RUCONEST case can be split into two components in my belief. First, you have the medical component, which continues to make the case, and I'd like to touch upon the investor component and specifically your comments that, you know, patients are still seeing switches from the new therapies to RUCONEST.

Joe Pantginis: Hey, everybody. Good morning. Thanks for taking the questions. I know you might not be able to give color here ahead of the Type A, but I'm gonna ask anyway. Obviously, the reasons, you know, for your discussions, as Anurag said, were the clinical pharmacology and the analytical batches. Is there anything that you would consider sort of the lead rate-limiting step here? That's question number one. Question number two regarding RUCONEST. You touched upon this a little bit, but I guess, you know, the RUCONEST case can be split into two components in my belief. First, you have the medical component, which continues to make the case, and I'd like to touch upon the investor component and specifically your comments that, you know, patients are still seeing switches from the new therapies to RUCONEST.

Speaker #3: Please ask your question

Speaker #6: Hey , everybody . Good morning . Thanks for taking the questions . So I know might not be able to give color here ahead of the type A , but I'm going to ask anyway .

Speaker #6: Obviously the reasons you know , for your discussions as Anurag said , were the clinical pharmacology and the analytical batches . Is there anything that you would consider sort of the lead rate limiting step here ?

Speaker #6: That's question number one . Question number two , regarding Ruconest , you touched upon this a little bit , but I guess , you know , the Ruconest case can be split into two components in my belief .

Speaker #6: So first you have the medical component which continues to make the case . And I'd like to touch upon the investor component and specifically your comments that , you know , the patients are still seeing switches from the new therapies .

Joseph Pantginis: I was hoping you can provide some additional color as to why those switches are taking place. Thanks a lot.

Joe Pantginis: I was hoping you can provide some additional color as to why those switches are taking place. Thanks a lot.

Speaker #6: To ruconest . So I was hoping you can provide some additional color as to why those switches are taking place . Thanks a lot

Fabrice Chouraqui: Thank you, Joe, for these questions. On the Type A meeting, it's very difficult to speculate. I think Anurag has been clear on the questions that were raised by FDA, and we are really looking forward to engage with the agency later this month to address their feedback and discuss a path forward. Too soon to tell. Clearly, given what they've raised, we feel actually that these questions are addressable. Once again, we look forward to engaging with them. When it comes to RUCONEST, I'll let Laverne answer your question.

Speaker #2: Thank you . Joe , for those for these questions . So on the Taipei meeting , it's very difficult to speculate . I think Anurag has been clear on on the questions that were raised by by FDA .

Speaker #2: And we are really looking forward to to engage with the agency later this month to address their feedback and , and discuss a path forward .

Speaker #2: So too soon , too soon to tell . Clearly , given what they've raised , we feel actually that these questions are addressable .

Speaker #2: And once again , we look forward to , to engaging with them when it comes to , to ruconest , I'll I'll let Leven answer your question .

Laverne March: Joe, thanks for your question. An important one. As we've seen new agents entering the HAE market in the US between June and August last year, as expected, we saw some trialing of both acute agents and new prophylactic agents in the US market. What we're observing in our data currently, and it's still early, is within about three to six months, some of these patterns may start to shift. We've seen some return of RUCONEST patients that have originally adopted or trialed a different product coming back to RUCONEST. Again, early days, and we're monitoring this closely, and we continue to execute competitively, based on RUCONEST's very different value proposition for patients, specifically in the high attack segment, where they are increasingly concerned with reliability and a fast on-demand treatment like RUCONEST.

Speaker #5: So thanks for your question . An important one . So as we've seen , new agents entering the market in the US between June and August last year , as expected , we saw some trialing of both acute agents and new prophylactic agents in the US market .

Speaker #5: What we're observing in our data currently , and it's still early , is within about 3 to 6 months . Some of these patterns may start to shift .

Speaker #5: And we've seen some return of ruconest patients that have originally adopted it or trialed a different product . Coming back to Ruconest . But again , early days and we're monitoring this closely and we continue to execute competitively based on Ruconest is very different value proposition for patients , specifically in the higher tax segment , where there are increasingly concerned with reliability , reliability and a fast on demand treatment like Ruconest

Joseph Pantginis: Appreciate the color. Thank you.

Joe Pantginis: Appreciate the color. Thank you.

Operator: We are now going to proceed with our next question. The question comes from the line of Jeff Jones from Oppenheimer. Please ask your question.

Speaker #6: Appreciate the color. Thank you.

Speaker #3: We are now going to proceed with our next question and the questions come from the line of Jeff Jones from Oppenheimer . Please ask your question .

Jeff Jones: Good morning, guys, and congrats on the great year 2025. A couple of questions from us. You spoke a little bit to the cadence of moving patients from early access onto paid therapy. Any notable variations between Europe, Japan, Canada as you look to that? And then as you look at the other primary immune deficiencies for Joenja and the phase 2 readouts that you're anticipating, can you speak a little bit about next steps, what types of additional trials might be needed, and how to think about the path forward there? Thank you.

Speaker #7: Good morning guys . And congrats on the great year 2025 . A couple questions from us . You spoke a little bit to the cadence of moving patients from early access onto paid therapy .

Speaker #7: Any notable variations between Europe , Japan , Canada , as you look to that and then as you look at the other primary immune deficiencies for Joenja and the phase two readouts that you're anticipating , can you speak a little bit about next steps ?

Speaker #7: What types of additional trials might be needed , and how to think about the path forward ? There ? Thank you

Fabrice Chouraqui: Thank you, Jeff, for these questions. When it comes to access to paid therapies for Joenja in international markets, most of these markets have centrally driven access. The dynamic is different compared to the US, where in a sense, each patient, you know, needs to deal with different payers. In a centralized access system, things are slower at the beginning because you need to negotiate, obviously, with the authorities for reimbursement. Once you get reimbursement, then afterwards, the reimbursement process is extremely efficient. We don't expect to see the same type of dynamic internationally. When it comes to higher prevalent PMDs, I'll ask Anurag to elaborate.

Speaker #2: Thank you . Jeff , for these questions . When it comes to access to to therapies for for joint in international markets , most of these markets are access centrally driven access .

Speaker #2: So the dynamic is different compared to the US where in a sense , each patient , you know , needs to deal with a different payers .

Speaker #2: So so in a in a centralized access system , things are slower at the beginning because you need to negotiate . Obviously with the authorities for reimbursement with once you get reimbursement , then afterwards the reimbursement process is extremely efficient .

Speaker #2: So . So we don't expect to see the same type of dynamic international when it comes to higher prevalence PMDs . I'll ask Anurag to elaborate .

Anurag Relan: Hi, Jeff. On the question about what happens next. As I mentioned, the studies have completed enrollment. We expect results later this year. The results that we're looking for, again, the endpoints that we're evaluating are clinically relevant endpoints similar to the ones that we looked at in APDS, as well as other clinically relevant endpoints. As we look at those endpoints, we'll plan to have a discussion with FDA and other regulators about the path forward. I think our base case here is that we would expect to do a phase 3 randomized type of study. However, I think you've also seen from FDA some openness and willingness to look at alternative mechanisms and pathways for patients with rare diseases, especially those where there's a plausible mechanism and there's a mechanism that's understood.

Speaker #2: Hi , Jeff . So on the question about .

Speaker #8: The what happens next ? So as I mentioned , the studies have completed enrollment . We expect results later this year . The results that we're looking for .

Speaker #8: Again , the endpoints that we're evaluating are clinically relevant endpoints similar to the ones that we looked at in APDs as well as other clinically relevant endpoints .

Speaker #8: As we look at those endpoints , we'll plan to have a discussion with FDA and other regulators about the path forward . I think our base case here is that we would expect to do a phase three randomized type of study .

Speaker #8: However, I think you've also seen from the FDA some openness and willingness to look at alternative mechanisms and pathways for patients with rare diseases, especially those where there's a plausible mechanism.

Anurag Relan: Those are the types of discussions that we would have with FDA, again, once we have the data to be able to plan the path forward.

Speaker #8: And there's a mechanism that's understood . So those are the types of discussions that we would have with FDA . Again , once we have the data to be able to plan the path forward

Jeff Jones: Great. Appreciate the color, guys. Thanks.

Speaker #7: Great . Appreciate the color , guys . Thanks

Operator: Thank you. We are now going to proceed with our next question. The question comes from the line of Sheila Hernandez from VLK. Please ask your question.

Speaker #3: Thank you . We are now going to proceed with our next question . And the questions come from the line of Sheila Hernandez from VLC .

Sheila Hernandez: Yes. Thank you for taking my questions. On Joenja, you mentioned different launch dynamics for international markets. What kind of timelines are you working with for getting these patients in Japan and Canada on paid therapy? In the UK, will you start reporting the numbers of patients on paid therapy? Currently, how many patients in the UK are on paid therapy, and how many have you identified? Thank you.

Sushila Hernandez: Yes. Thank you for taking my questions. On Joenja, you mentioned different launch dynamics for international markets. What kind of timelines are you working with for getting these patients in Japan and Canada on paid therapy? In the UK, will you start reporting the numbers of patients on paid therapy? Currently, how many patients in the UK are on paid therapy, and how many have you identified? Thank you.

Speaker #3: Please ask your question Yes .

Speaker #9: Thank you for taking my questions. You mentioned different launch dynamics for international markets. So, what kind of timelines are you working with for getting these patients in Japan and Canada on paid therapy, and in the UK?

Speaker #9: Will you also start reporting the numbers of patients on paid therapy? So currently, how many patients in the UK are on paid therapy, and how many have you identified?

Fabrice Chouraqui: When it comes to timelines, we've elaborated a bit on our expectations when it comes to regulatory timelines in the very near future, when it comes to CHMP opinion from Europe and PMDA approval in Japan. In Japan, specifically, since you asked the questions, we would be submitting a price very shortly for reimbursement, and it takes about three months, actually, for the price to be granted. Today we have launch timelines planned for the summer. We will be reporting more information on international market on Joenja in a pooled fashion as soon as we have launches in more than one country.

Speaker #9: Thank you

Speaker #2: So when it comes to to , to timelines , so we've elaborated a bit on our expectations when it comes to regulatory timelines in the very near future .

Speaker #2: When it comes to opinion from Europe and Pmda approval in Japan , we'll in Japan specifically , since you asked the questions , we will be submitting a price very shortly for reimbursement , and it takes about three months .

Speaker #2: Actually , for the price to be to be granted . So today we have launch timelines planned for for the summer We will be reporting more information on international market on Jahangir In a pool fashion , as soon as we have launches in more than one country .

Fabrice Chouraqui: This should happen very soon. It's absolutely essential, and you can again count on transparency here. For the second part of your question, I'll let Laverne elaborate.

Speaker #2: So this should happen very soon . It's it's absolutely essential and you can can count on on on , on transparency here for the second part of your question , I'll let Levent elaborate .

Laverne March: Certainly. As Fabrice Chouraqui has said prior, as we look at different approvals coming online at different times in this year, every country fundamentally will be a country-by-country process as the approval and reimbursement processes are quite unique for the countries that we discussed. How we thinking about conversion, is conversion will depend on physician experience, diagnostic confirmation and testing, and access. We are building those enablers systematically with the international teams to make sure that we are able to execute upon approval.

Speaker #5: Certainly as Reese has said prior , as we look at different approvals coming online at different times in this year , every country fundamentally will be a country by country process , as the approval and reimbursement processes are quite unique for the countries that we discuss .

Speaker #5: So how are we thinking about conversion ? Is conversion will depend on position experience , diagnostic confirmation and testing and access . And we are building those enablers systematically with the international teams to make sure that we are able to execute upon approval

Sheila Hernandez: Okay. That's clear. Thank you.

Sushila Hernandez: Okay. That's clear. Thank you.

Operator: We are now going to proceed with our next question. The question comes from the line of Natalia Webster from RBC Capital Markets. Please ask your question.

Speaker #9: Okay . That's clear . Thank you

Speaker #3: We are now going to proceed with our next question and the questions come from the line of Natalia Webster from LBC Capital Markets .

Natalia Webster: Hi there. Thanks for taking my questions. My first one is a follow-up on Joenja and the patients on paid therapy in the US. You added 18 in H1 2025 and 6 in H2. Appreciate that this takes time, but are you expecting for this rate to pick up into 2026? The second question is just on costs. You're guiding for 6% to 8% growth in OpEx in 2026. You mentioned some phasing considerations on the revenue side. Are there any particular phasing considerations for the costs through the year, particularly around the higher R&D costs and G&A cost savings? Just finally on M&A. In the release you mentioned continued focus on potential acquisitions and in-licensing opportunities. Any additional color on what your thoughts are there would be helpful. Thank you.

Speaker #3: Please ask your question .

Speaker #10: Hi there . Thanks for taking my questions . My first one is a follow up on Georgia and the patients on paid therapy in the US .

Speaker #10: You added 18 in H1 2025 and six in H2 . Appreciate that . This takes time , but are you expecting for this rate to pick up into 2026 ?

Speaker #10: Then the second question is just on costs . You're guiding to 6 to 8% growth in OpEx for 2026 . And you mentioned some phasing considerations on the revenue side .

Speaker #10: Are there any particular phasing considerations for the costs through the year, particularly around the higher R&D costs and DNA cost savings? And then just finally, on M&A, in the release you mentioned continued focus on potential acquisitions.

Speaker #10: And enlightening in-licensing opportunities. So any additional color on what your thoughts are there would be helpful. Thank you.

Fabrice Chouraqui: Very good. Thank you so much, Natalia, for your questions. I'll start with the last one when it comes to M&A. We have clearly a number of growth catalysts both in our commercial portfolio and in our pipeline in the years to come. Obviously there is no urgency to do any transaction hastily to compensate for any sort of a weakness. Yet, we clearly aspire to leveraging proven capabilities and a great growth platform to take that to a whole new level and make Pharming a leading rare disease, ultra-rare disease player.

Speaker #2: Very good . Thank you so much , Natalia , for your questions . So I'll start with the last one . When it comes to to M&A , we have clearly a number of growth catalysts both in our commercial portfolio and in our pipeline .

Speaker #2: In the years to come . So obviously there is no urgency to do to do any any transaction hastily to compensate for any sort of of weakness .

Speaker #2: Yet we clearly aspire to leveraging a proven capabilities and great growth platform to take that to a to a whole new level and make farming a leading rare disease , ultra rare disease player .

Fabrice Chouraqui: We are constantly looking out for opportunities to expand our pipeline. Now, it is absolutely essential that these opportunities, if they were to materialize, would be value accretive. And that's really our commitment to our shareholders. It's not about actually leveraging any external growth opportunities, but making sure that anything that we would consider would be complementary, would fulfill our mission, and will be quickly accretive from a value perspective. From a cost perspective, I'll let Kenneth elaborate.

Speaker #2: And so we are constantly looking out for opportunities to expand our pipeline . Now , it is absolutely essential that these opportunities , if they were to materialize , would be a value accretive .

Speaker #2: And that's really our commitment to our shareholders . So it's not about actually leveraging any external growth opportunities , but making sure that anything that we would consider would be complementary would fulfill our mission and will be quickly accretive from a .

Kenneth Lynard: Yeah, thanks for the question. I think, you know, the way to think about it is in the 2025 baseline, we obviously had also some one-off costs that were related to the transaction of Abliva, so non-recurring transaction cost of about EUR 10 million. And we also communicated earlier that we had about EUR 4 million in costs related to the G&A reduction program. But then when you're looking into the more than EUR 60 million incremental investments in 2026, we of course see the EUR 9 million of savings in G&A come fully through. And, you know, then we have seen the impact in our planning of the strengthened capital allocation, which has allowed us to keep also marketing and sales costs flat. There are different dynamics that are playing in.

Speaker #2: Value perspective , from a cost perspective , I'll let Kenneth elaborate .

Speaker #1: Yeah , thanks for the thanks for the question . I think you know , the way to to think about it is in the 2025 baseline , we obviously had also some one off costs that were related to the transaction of the Oliver .

Speaker #1: So non-recurring transaction cost of about 10 million . And we also communicated earlier that we had about 4 million in cost related to the DNA reduction program .

Speaker #1: But then when you're looking into the more than 60 million incremental investments in 2026 , we of course see the 9 million of savings in DNA come fully through .

Speaker #1: And , you know , then we have seen the impact in our planning of the strengthened capital allocation , which has allowed us to keep also marketing and sales cost flat so there are different dynamics that are playing in , but I think , you know , picture kind of speaks for itself that we're fueling where we're seeing the opportunities to advance .

Kenneth Lynard: I think you know the picture speaks for itself that we're fueling where we're seeing the opportunities to advance, in this case, in 2026, the pipeline. We are very diligent around spend discipline across all other areas.

Fabrice Chouraqui: Lastly, coming to your question on Joenja, which is actually a very important question. Because, as I said, I really see Joenja taking a larger part of our revenues, as the drug continues to grow significantly and realize its full potential. You heard that last year we really accelerated the uptake of the drug. We had more new paid patients on therapy in the US than we had in 2024. Also, we've identified more APDS patients in the US in 2025 than we did in 2024. This is really fueling the growth.

Speaker #1: In this case, in 2026, the pipeline and are very diligent around spend discipline across all other areas.

Speaker #2: And so lastly , coming to your question on joint , which is actually a very important question , because as I said , I really see joint taking a larger part of our revenues as the the drug continue to , to , to grow significantly and realize its full potential .

Speaker #2: You heard that last year . We really accelerated the uptake of , of the drug . We had more new paid patient on therapy in the US that we had in 20 .

Speaker #2: For us also , we've identified more APDs patients in the US in 25 than we did in 24 . And so this is really fueling the growth .

Fabrice Chouraqui: This year we expect to continue to accelerate patient enrollment on Joenja and, as a consequence, accelerate our revenue growth.

Speaker #2: So this year we expect to continue to accelerate patient enrollment on on joint gel . And as a consequence accelerate our revenue growth .

Natalia Webster: Great. Thank you.

Operator: We are now going to proceed with our next question. The question's come from the line of Simon Scholes from First Berlin. Please ask your question.

Speaker #10: Great . Thank you

Speaker #3: We are now going to proceed with our next question . And the questions come from the line of Simon Scholes from First Berlin .

Simon Scholes: Yes. Hello. Thanks for taking my questions. I've just got two. So I was wondering if you could give us a timeline on the performance and collation of the results from these additional experiments you need to perform with regard to the variants of uncertain significance. Also, are you still confident that 20% of these VUSs will turn out to be APDS? And then just on RUCONEST, you were talking about a possible inventory-related decline in or inventory-related effect on sales in Q1. Is this an unusually large inventory drawdown that you're expecting to see in Q1? I mean, that was my impression. If so, why do you think you saw such a this, a large inventory build up towards the tail end of last year?

Speaker #3: Please ask your question .

Speaker #11: Yes . Hello . Thanks for taking my questions . I've just got two , so I was wondering if you could give us a timeline on the performance and collation of the results from these additional experiments .

Speaker #11: You need to perform with regard to the variance of uncertain significance . And also , are you still confident that 20% of these views will turn out to be APDs ?

Speaker #11: And then just on Ruconest , you were talking about . A possible inventory related decline in ore inventory related effect on sales in Q1 .

Speaker #11: Is this an unusually large inventory drawdown that you're expecting to see in Q1 ? I mean , that was my impression . And if so , how did why do you think you saw such a this large inventory build up towards the tail end of last year

Fabrice Chouraqui: Thank you, Simon. Let's start with the US. Clearly this is a sizable opportunity and we are really committed to helping those patients accessing the right diagnostic. Henry, right?

Speaker #2: Thank you . Simon . So let me let's start with with the US . Clearly this is a sizable opportunity and we are really committed to helping those patients accessing the right diagnostics .

Anurag Relan: Simon, we are now planning these new experiments. These experiments, again, we're working with Columbia. They're actually gonna be using new technology, new base editing technology to be able to generate different types of variants, generate more controls, as they go through this process. It's too soon to tell in terms of the timelines as well as the actual number of patients that will actually be reclassified. As this work gets underway, over the coming months, we should be able to provide more details around it.

Speaker #2: So I

Speaker #8: So Simon , we are now planning these new experiments . These experiments again , we're working with Colombia . They're actually going to be using new technology , new base editing technology to be able to generate different types of variants , generate more controls as they go through this process .

Speaker #8: It's too soon to tell in terms of the timelines as well as the actual number of patients that will actually be reclassified . But as this work gets underway over the coming months , we should be able to provide more details around it .

Simon Scholes: Okay, thanks.

Kenneth Lynard: Simon, on the inventory part, the way we're thinking about it is that there's always this quarterly fluctuation of the RUCONEST business given the patient ordering patterns and the general movements. We have also historically, if you go back in the previous years, seen that similar dynamics in the early part of the year. Now it's a little bit, let's say, higher in terms of inventory drawdown and dynamics this year, and we kind of attribute that simply to some of those market dynamics that are kind of settling now, and that inventory levels are just kind of returning to a little bit more of the normalized level.

Speaker #11: Okay , thanks .

Speaker #1: And Simon , on the on the inventory part , the way we're thinking about it is that there's always this quarterly fluctuation of the Ruconest business , given the patient ordering patterns and the general movements .

Speaker #1: And we have also historically , if you go back in the previous years , seen that , you know , similar dynamics in the early part of the year .

Speaker #1: Now it's a little bit , let's say , higher in terms of inventory drawdown and dynamics this year . And we kind of attribute that simply to some of those market dynamics are kind of settling .

Kenneth Lynard: Impact-wise, it's a little bit higher, but the mechanics of how the quarters are fluctuating and the fact that their inventory impacts in Q1 are not new to us.

Speaker #1: Now . And that inventory levels are just kind of returning to to a little bit more of the normalized level . So impact wise , it's a little bit higher , but the , the mechanics of how the quarters are fluctuating and the fact that their inventory impact in the first quarter are not new to us .

Simon Scholes: Okay, thanks very much.

Fabrice Chouraqui: Very good. Listen, I think with this we are going to close our call. Thank you so much for these additional questions and we look forward to keeping you closely informed on our plan. There are a number of important milestone coming up and so we look forward to reconnecting with you very soon. Thank you so much. Thank you, operator.

Speaker #11: Okay . Thanks very much

Speaker #2: Very good . So listen , I think with this , we are going to close our call . Thank you so much For these additional questions .

Speaker #2: And we look forward to keeping you closely informed on on our plan . There is a there are a number of important milestone coming up and so we look forward to reconnecting with you very soon .

Operator: Thank you. This concludes today's conference call. Thank you all for participating. You may now disconnect your lines. Thank you.

Speaker #2: Thank you so much . Thank you . Operator .

Q4 2025 Pharming Group NV Earnings Call

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