Pharming Group N.V. Q4 and Full Year 2025 Earnings Call - Joenja Accelerates, RUCONEST Keeps Cash Flowing as Company Turns Profitable

Operator: Good day, thank you for standing by. Welcome to the Pharming Group N.V. fourth quarter 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. I would now like to hand the conference over to your first speaker, Mr. Fabrice Chouraqui, CEO. Please go ahead, sir.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 Marsh, 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 fourth quarter of 2025, and by 27% for the full year. Thanks to disciplined cost management, we delivered $26 million of operating profit in 2025, compared to a loss in 2024. We also significantly increased our cash position.

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 the fourth quarter. 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.

Joenja grew 29% year-over-year and by 53% in the fourth quarter, fueled by the acceleration of new patients on drug in the U.S., but also increased demand in international markets, including in the U.K., 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 to 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.

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 KL1333 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 million and $425 million this year, representing an 8%-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.

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 U.S. and potential future regulatory approval internationally. Regarding the U.S. 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. 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 lower prevalence PIDs, and we expect a top-line data readout in the second half of this year. 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 and make Pharming a leading rare, ultra-rare disease company and deliver sustainable value for our shareholders. Let me now turn to LaVerne Marsh for deeper insights on the performance of our commercial portfolio.

LaVerne Marsh, Chief Commercial Officer, Pharming Group N.V.: Good day, everybody. Let me start with RUCONEST, where the U.S. 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 5 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 agents enter the market. For the full year, RUCONEST delivered 26% global revenue growth and a volume growth in the U.S. of 20%. A clear indicator of the strong and durable demand for RUCONEST in the acute segment.

In quarter four, we delivered 9% global revenue growth versus the prior year quarter and a 2% volume growth in the core U.S. market, continuing the upward trajectory of RUCONEST. As expected, through Q4, we began to observe impact of newly launched therapies for HAE in the U.S. market, with some patients trialing and some already returning to RUCONEST. Despite these competitive dynamics, we welcomed over 60 new enrollments in the U.S. in the fourth quarter, 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.

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 U.S. 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.

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 fourth quarter, building on the momentum we established throughout the year. Revenue grew 53% compared to the fourth quarter in 2024, reaching $19.8 million globally. For the full year, Joenja 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. 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.

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 U.S. 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 U.S. are working. Outside the U.S., 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.

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 U.K., where Joenja launched in April 2025, we have seen solid early uptake and strong engagement for complexity. 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. This will give us a running start and positions us for early momentum once the label is approved. Beyond the U.S., 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. 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. 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.

Anurag Relan, Chief Medical Officer, Pharming Group N.V.: 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.

Based on this understanding of APDS, we have two ongoing phase II 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 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. 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.

The prevalence figures here are for the U.S., 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 5-26 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 variants 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 remain a significant unmet need and actually a public health problem for clinicians and patients.

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.

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 4-11, 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.

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 four 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 four to eleven. 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 KL-one triple three. Napazimone 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.

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 U.S. 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 futility. 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. With that, I’ll turn it over to Kenneth now to discuss our financial results.

Kenneth Lynard, Chief Financial Officer, Pharming Group N.V.: 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 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 the fourth quarter. Adjusted operating profit was broadly stable year-over-year after several offsetting one-off items.

Fourth quarter 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 the fourth quarter of 2024, operating profit in the fourth quarter of 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.

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

In total, adjusted operating profit, which excludes 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 totals 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.

Turning to our 2026 outlook, we reaffirm our expectation for total revenues of EUR 405 million-EUR 425 million, representing full year growth of approximately 8%-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%-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.

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 U.S. approval timeline for patients aged 4 to 11 following the upcoming FDA Type A meeting. In the meantime, U.S. pediatric revenues are excluded from our 2026 guidance. For operating expenses, we anticipate a range of EUR 330 million-EUR 335 million, including more than EUR 60 million of incremental R&D investments. This guidance 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.

Because Joenja revenue is not expected to exceed EUR 100 million in 2026, we do not assume the EUR 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.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: Thank you, Kenneth. In summary, we are pleased to report another strong quarter and a record year for Pharming in 2025 with $76 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 HAE patients, underpinning a strong revenue base. We see clear revenue catalyst 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.

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.

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.

Lucy Codrington, Analyst, Jefferies: 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 considered versus how many have been 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.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 a highly reliable treatment.

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 U.S. as well as growth increasing in international markets. It’s obviously the growth in international markets will come from the U.K., where the drug has been launched, new launch countries.

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 going to be gradual. When it comes to more specifics on Joenja’s patient funnel, I’ll ask LaVerne to comment.

LaVerne Marsh, Chief Commercial Officer, Pharming Group N.V.: 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.

Kenneth Lynard, Chief Financial Officer, Pharming Group N.V.: Maybe, Lucy, this is Kenneth speaking. Maybe I can just add, out of the expected Joenja growth in 2026, it will probably be around about 70%-75% that will come from the US.

Lucy Codrington, Analyst, Jefferies: 70-75% from the U.S.?

Kenneth Lynard, Chief Financial Officer, Pharming Group N.V.: Of the growth in 2026 for Joenja. Yes.

Lucy Codrington, Analyst, Jefferies: Got it. Thank you very much.

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.

Joseph Pantginis, Analyst, H.C. Wainwright & Co.: Hey, everybody. Good morning. Thanks for taking the questions. I know might not be able to give color here ahead of the Type A, but I’m going to 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.

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

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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. And once again, we look forward to engaging with them. When it comes to RUCONEST, I’ll let LaVerne answer your question.

LaVerne Marsh, Chief Commercial Officer, Pharming Group N.V.: Joe, thanks for your question. An important one. As we’ve seen new agents entering the HAE market in the U.S. between June and August last year, as expected, we saw some trialing of both acute agents and new prophylactic agents in the U.S. 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.

Joseph Pantginis, Analyst, H.C. Wainwright & Co.: Appreciate the color. Thank you.

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

Jeff Jones, Analyst, Oppenheimer: 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.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 U.S., 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 international. When it comes to higher prevalent PMDs, I’ll ask Anurag to elaborate.

Anurag Relan, Chief Medical Officer, Pharming Group N.V.: Hi, Jeff. On the question about the 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.

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, Analyst, Oppenheimer: Great. Appreciate the color, guys. Thanks.

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

Sheila Hernandez, Analyst, VLK: 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? And 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.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: When it comes to timeline, 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. We will 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.

This should happen very soon. It’s absolutely essential, and you can count on transparency here. For the second part of your question, I’ll let LaVerne elaborate.

LaVerne Marsh, Chief Commercial Officer, Pharming Group N.V.: Certainly. As Fabrice 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.

Sheila Hernandez, Analyst, VLK: 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.

Natalia Webster, Analyst, RBC Capital Markets: Hi there. Thanks for taking my questions. My first one is a follow-up on Joenja and the patients on paid therapy in the U.S. 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 6%-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.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 or ultra-rare disease player.

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. 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.

Kenneth Lynard, Chief Financial Officer, Pharming Group N.V.: 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 the Abliva, so non-recurring transaction cost of about EUR 10 million. We also communicated earlier that we had about EUR 4 million in costs related to the G&A reduction program. When you’re looking into the more than EUR 60 million incremental investments in 2026, we of course see the EUR 9 billion of savings in G&A come fully through. 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, but, I think, you know, picture speaks for itself that we’re fueling where we’re seeing the opportunities to advance, in this case, 2026, the pipeline, and are very diligent around spend discipline across all other areas.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 continue 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 patient on therapy in the US that 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.

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

Natalia Webster, Analyst, RBC Capital Markets: Great. Thank you.

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

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.0: 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. And 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. And if so, why do you think you saw such a large inventory build up towards the tail end of last year?

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: Thank you, Simon. Let’s start with VUS. Clearly this is a sizable opportunity and we are really committed to helping those patients accessing the right diagnostic. Anurag?

Anurag Relan, Chief Medical Officer, Pharming Group N.V.: 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. But as this work gets underway, over the coming months, we should be able to provide more details around it.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.0: Okay, thanks.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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 you know general movement. 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. 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 now, and that inventory levels are just kind of returning to a little bit more of the normalized level.

Impact wise it’s a little bit higher, but the mechanics of how the quarters are fluctuating and the fact that their inventory impact in the first quarter are not new to us.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.0: Okay. Thanks very much.

Fabrice Chouraqui, Chief Executive Officer, Pharming Group N.V.: 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.

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