Pharming Group N.V. (PHAR) Q2 2026 Earnings Report, Transcript and Summary

Thank you, operator. Good morning and good afternoon, everyone, and welcome to our Q2 2026 earnings call. I'll be joined on this call today by Leverne Marsh, our Chief Commercial Officer; Anurag Relan, our Chief Medical Officer; and Kenneth Lynard, our Chief Financial Officer. In 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 today's press release, while we lowered our full-year revenue guidance, we are encouraged by the resilience of RUCONEST with robust underlying demand indicators. Joenja continued to deliver strong growth, and we are approaching a number of significant near-term catalysts. Before I go into more detail on the quarter, let me take a step back and put these developments into the context of the ongoing transformation of Pharming. We are evolving Pharming into a more diversified rare disease company with an increasingly attractive long-term growth profile. This is supported by three key pillars, RUCONEST, which provides a durable source of cash flow; Joenja, a high-growth asset still early in its life cycle with significant commercial opportunities ahead; and a high-value pipeline with two potential billion-dollar opportunities. Each of these pipeline programs has the potential to materially increase our scale and are important steps forward in our ambition to make Pharming a leading global rare disease company. We've made important progress across the business during the second quarter despite a 3% year-over-year decline in total revenue. RUCONEST revenue declined to $72.3 million compared with $80.4 million in the second quarter of last year. However, we are encouraged by the underlying performance indicators, which demonstrate the resilience of RUCONEST in an evolving on-demand HAE market. One year after the launch of the first oral on-demand HAE treatment, the RUCONEST active patient base remained at 93% of the level a year ago. We are also seeing strong new patient enrollments, which have returned to levels close to those of a year ago, and we expect this to support momentum over the coming months as these patients initiate treatment. We also continue to see new prescribers using RUCONEST, which underscores RUCONEST's differentiated value proposition for high-burden patients. Leverne will provide further detail on RUCONEST's performance later in the call. Turning to Joenja. We delivered another quarter of strong growth, with revenue increasing 40% to $17.9 million. We remain focused on expanding the opportunity for Joenja in APDS through both label and geographic expansion, and we are pleased to see commercial momentum accelerating in Europe. Beyond APDS, we see a potentially significant opportunity for leniolisib in broader primary immunodeficiencies, including CVID. This indication represents an addressable patient population up to 40 times larger than APDS. Anurag will provide more detail on the mechanistic rationale underpinning our excitement in these additional indications. With greater visibility into the current HAE market dynamics, which is much clearer a full year after the launch of the first oral on-demand treatment, we have decided to lower our full-year revenue guidance by $30 million. Nevertheless, based on the underlying trends we are seeing, we expect RUCONEST revenue to stabilize and return to growth during the second half of 2026. Importantly, our increasingly disciplined operating model has enabled us to maintain positive cash flow from operations. We have also reduced our full-year operating expense guidance by $15 million, to a range of $315 million to $320 million. As we position Pharming for an important second half of 2026, our priorities are clear. First, we will continue to reinforce RUCONEST's differentiated value proposition for high-disease-burden patients and build on the solid underlying performance indicators that we have seen in the second quarter. Second, we will continue to drive the growth of the Joenja franchise, supported by the potential pediatric label expansion in the U.S. and further geographic expansion into new markets, including Japan. And importantly, we expect to advance our pipeline at pace, with the readout in Q4 of the two Phase II studies for leniolisib in much broader CVID patient populations. Success in this indication could expand the annual sales potential of Joenja to more than $1 billion and establish Joenja as a blockbuster franchise. With that, I will turn the call over to Leverne to discuss our progress on the commercial front.

Thank you, Fabrice. Good morning and good afternoon, everyone. Let me begin with RUCONEST. This quarter, RUCONEST revenue was $72.3 million, down 10% year-over-year, but up sequentially by 24%. The year-over-year decline reflected continued competitive dynamics in the U.S., completion of our planned withdrawal from international markets, and temporary inventory normalization. I am pleased to report that nearly one year after the launch of a new oral on-demand treatment, the active RUCONEST patient base remains highly resilient at approximately 93% of the prior-year level. While some patients are evaluating new treatment options, the vast majority have continued on RUCONEST, as the drug remains especially important for patients who have more frequent attacks or severe attacks who need reliable on-demand treatment. Also notable, we've not seen a reduction in RUCONEST utilization among patients receiving newer prophylactic treatments. This reinforces that preventive and on-demand therapies play complementary roles in managing HAE. We're also encouraged by the upticks in new demand. During the quarter, we recorded 84 new patient enrollments, up substantially from approximately 50 in the first quarter and close to the high level we received during 2025, quarter 2. We also added 17 new prescribers, suggesting continued physician confidence even following the addition of a new competitor. Importantly, we see underlying U.S. demand improving. The resilient patient base, improvement in new enrollments, and continued addition of prescribers support our belief that revenue will stabilize and RUCONEST is expected to return to growth during the second half of the year. Next slide, these patient retention and demand dynamics reflect the distinct role that RUCONEST continues to play in high-burden HAE patients. HAE is challenging to treat as it affects patients differently and no single treatment meets every patient's needs. Convenience matters, but for patients with more frequent attacks, with more severe attacks, reliability, rapid onset, and confidence in their treatment are critical, particularly when attacks occur in high-risk locations or other therapies have not provided adequate control. RUCONEST is well-suited to meet these needs. By replacing deficient or dysfunctional C1 esterase inhibitor, it helps restore control of the pathways that drive HAE attacks while providing the efficacy and reliability of a self-administered IV treatment. This differentiated clinical profile explains why physicians could continue to prescribe RUCONEST, including for new patients with more severe disease. And, separately, RUCONEST's highly specialized manufacturing process limits the potential for direct biosimilar competition, supporting its durable commercial position well into the future. Next slide, please. The data shown here bring that differentiated profile into focus. In clinical studies, 97% of acute attacks required only one dose of RUCONEST, and RUCONEST stopped 93% of attacks for at least three days. Importantly, RUCONEST also demonstrated a median time to complete attack resolution of approximately four and a half hours, a key point of differentiation versus competition. Taken together, HAE patients with a high disease burden, this combination of efficacy, reliability, and rapid and complete attack resolution provides the confidence to both patients and physicians in choosing RUCONEST as their on-demand treatment. And these data reinforce our belief that RUCONEST will remain an important on-demand therapy for high-burden HAE patients and a durable part of Pharming's commercial portfolio. Next slide, please. Turning now to Joenja. We delivered another strong quarter and continue to build momentum across the U.S. and international markets. Global revenue increased 40% year-over-year to $17.9 million, while the U.S. revenue grew 31% to $15.4 million and international revenue increased 150% to $2.5 million, reflecting growing uptake and high adherence. In the U.S., new patient starts continue to drive sales growth. By the end of the quarter, 132 patients were on paid therapy, up 16% year-over-year, and 5 patients sequentially over quarter 1. We are also continuing to expand the diagnosed APDS population. During the quarter, the number of identified U.S. patients increased by 16 to 298, including 60 patients between the ages of 4 and 11, which is a key catalyst for future growth in APDS. As we identify more patients, we see meaningful room for continued growth in patients on therapy, both in the U.S. and internationally. Next slide, please. Looking ahead, we see several opportunities to continue expanding Joenja. In the U.S., sources of growth in APDS include continued patient identification, increased genetic testing, and continued efforts to reclassify VUSs. Pediatric expansion is the next important growth opportunity. We are awaiting our October 24 PDUFA date for approval of the higher 40 and 50 milligram doses. And I am pleased to report the filing of the sNDA for the lower 20 and 30 milligram doses will be submitted to the FDA today. We are also expanding internationally. Our first European launch is now underway in Germany, and we are preparing to launch in Japan in the third quarter. Together with continued growth in the U.K. and other markets, these launches significantly increase the number of patients we are positioned to reach. Finally, our phase II studies in genetic PIDs and CVID with immune dysregulation are evaluating substantially larger patient populations for Joenja beyond APDS. Together, these opportunities provide multiple sequential drivers of growth for Joenja, supporting our ambition to build a broader immunology franchise and positioning Joenja for potential blockbuster status. With that, I will now turn over to Anurag.

Thank you, Leverne. I'll start by reviewing the science supporting leniolisib's potential beyond APDS and the scientific foundation supporting our two phase II programs. Let's begin by reviewing the relevant biology, where PI3K delta is a central immune signaling molecule. Specifically, it regulates lymphocyte activation, proliferation, differentiation, trafficking, and survival via the AKT, mTOR, and FOXO pathways. Consequently, imbalance in the pathway drives immune dysregulation, which can be clinically manifest as lymphoproliferation, autoimmunity, and inflammatory end-organ disease, specifically in the lung, liver, and the GI tract. This mechanistic understanding forms the scientific rationale for Joenja or leniolisib in APDS and our development programs in more prevalent PIDs. Before turning to these broader patient populations currently under investigation, it's worth reviewing the clinical foundation we've already established in APDS with PI3K delta inhibition using leniolisib. Leniolisib has demonstrated consistent and durable reductions in lymph node and spleen volume, showing sustained benefits with up to seven years of therapy in an open-label extension follow-up relevant to other PIDs. The safety profile is also well-established. Joenja has truly delivered life-changing benefits for patients, including fewer infections and hospitalizations, and a substantial reduction in treatment burden. What we have achieved in APDS gives us confidence in the potential for leniolisib to deliver the same meaningful benefits via PI3K delta inhibition to many more primary immune deficiency patients with immune dysregulation. Other genetically defined PIDs, as well as CVID with immune dysregulation, have a similar underlying PI3K delta-driven biology. Because of this PI3K delta involvement, these patients share many of the same clinical manifestations I just mentioned: lymphoproliferation, autoimmunity, GI disease, and lung disease. In addition to the mechanistic basis and our APDS experience, as we presented at the CIS conference in May, we have seen encouraging results in a small cohort of CVID patients who were treated in an expanded access program. In this clinician-reported experience, the breadth and consistency of improvement across cytopenias, lymphoproliferation, and end-organ disease provides a compelling early signal. Our two phase II studies are designed to determine whether the same targeted approach can benefit a broader patient population and significantly expand the addressable market. Both phase II studies are now fully enrolled, with top-line results expected in the fourth quarter. The CVID study includes 20 patients and is being conducted across multiple centers. The second study is a 12-patient basket study at the NIH involving genetically defined PIDs associated with immune dysregulation. These studies are designed to answer two fundamental questions. Can leniolisib produce clinically meaningful improvements across the key manifestations of immune dysregulation, and which dose regimen provides the appropriate balance of clinical activity, safety, and pathway modulation to support further development? To answer these questions, we are evaluating lymph node and spleen size, blood cell counts, liver and lung involvement, as well as patient and clinician-reported outcomes and key biomarkers. With the endpoints in these two studies strategically very similar to those in our APDS clinical program. Improvement across these measures would establish proof of concept and guide the next stage of development, including a registrational trial endpoint in these substantially larger patient populations. Given that the PI3K delta pathway is seen as a shared driver of immune dysregulation, we currently anticipate conducting a single registrational phase III trial in the broader CVID indication, incorporating patient populations from both studies. With that, I'll turn it over now to Kenneth to review our financial results.

Thank you, Anurag. I will now cover our Q2 and H1 2026 results, along with our updated full year outlook. Q2 revenue was $90.2 million, down 3% year-on-year. RUCONEST declined, as you heard earlier, by 10% versus Q2 of last year, and Joenja increased 40% year-on-year. Our reported operating profit was positive in the quarter, despite lower revenues and incremental investments of nearly $9 million compared with the same quarter last year. Adjusted operating profit was also positive and declined by $5 million year-on-year, primarily impacted by the decrease in revenues and the higher R&D investments. It is important to note that the adjusted Q2 2025 figure excludes $2.1 million of non-recurring Abliva acquisition-related costs, and the adjusted Q2 2026 result excludes $6.5 million of non-recurring items, comprising $4.9 million related to the impairment of manufacturing inventory and $1.7 million associated with the planned closure of our production support site in Évry, in France, in the Q4 of 2026. Operating cash flow for the quarter was an outflow of $9.7 million, primarily driven by changes in net working capital, including a strategic inventory build to further strengthen supply security as well as operating performance. As a result, cash and marketable securities at quarter end were $159.5 million, a decrease of $12.3 million compared with the end of Q1 2026. Looking now at our performance for the first half of 2026, the trends we saw in the second quarter are also reflected in our year-to-date results. Revenue for the first six months was $162.7 million, representing a 6% decline compared with the first half of 2026. For RUCONEST, revenue was 12% lower than the prior year period, and Joenja continued to deliver strong growth, with revenue increasing 37% year-on-year. Reported operating profit was impacted by a decrease in revenue impacted by RUCONEST demand and inventory destocking at specialty pharmacy level, predominantly in Q1 2026, and the manufacturing-related impairment of inventories in the first half of 2026. Adjusted operating profit was $10.7 million, lower than in the first half of 2025. For comparability, the adjusted H1 2025 result excludes $9.9 million of non-recurring Abliva acquisition-related costs, while the adjusted H1 2026 result excludes $6.5 million, comprising of the $4.9 million impairment of manufacturing inventory and $1.7 million provision related to the site closure in France. Compared with the first half of last year, we increased investments in R&D by $13 million. We maintained strong cost discipline. On an adjusted basis, operating expenses increased by just 1% versus the first half of 2025, despite these incremental R&D investments. Finally, operating cash flow for the first half was an outflow of $7.7 million, reflecting the combined effect of net working capital changes, primarily the strategic inventory build mentioned, and lower revenues. Turning now to our outlook for the full year. We have revised our 2026 revenue guidance to $375 million-$395 million, a reduction of $30 million from our previous outlook, reflecting the RUCONEST stabilization and return to growth and strong Joenja growth as discussed earlier. This represents expected growth of approximately 0%-5% versus 2025. As Fabrice mentioned earlier, nearly a year after the launch of the first oral on-demand HAE treatment, we have much clearer understanding of the evolving market dynamics, giving us greater confidence in both our forecasting and our revised outlook. At the midpoint of our guidance range, RUCONEST U.S. revenue is expected to decline approximately 3% for the full year. For Joenja, we continue to expect annual revenue growth in the high 30%s as compared to 29% growth in 2025, with Germany, Japan, and the anticipated U.S. pediatric label expansion contributing. We remain committed to disciplined capital allocation to efficiently balance investments in our pipeline, combined with a strong focus on operational efficiency, driving short and long-term value creation. Accordingly, we have reduced our full-year operating expense guidance by $15 million to $315 million-$320 million, representing growth of 1%-3% versus 2025. This includes more than $40 million of incremental R&D investment to advance our pipeline and approximately -- the approximate $9 million benefit from the 20% structural reduction in G&A headcount that we announced last year in October. Marketing and sales expenditure are expected to be broadly stable. For the full year, we expect cost of goods sold, including the $4.9 million manufacturing inventory impairment recorded in the second quarter, to be approximately 11% of revenue, corresponding to a gross margin of around 89%. Finally, we remain confident that our existing cash resources, together with future operating cash flows, are sufficient to fund our current development pipeline and all associated pre-launch activities. With that, I'll hand the call back to Fabrice.

Thank you, Kenneth. Let me close by putting our updated outlook into context. While we have revised our revenue expectations for 2026, the fundamentals of our business remain solid, and we have several important growth opportunities and clinical milestones ahead. We continue to believe that RUCONEST will remain a cornerstone on-demand treatment for high-burden HAE patients and a durable cash engine over the long term. This is supported by the performance we began to see this quarter, specifically the limited erosion of the active patient base, the renewed strength in patient enrollments, and the continued adoption by new prescribers. Joenja, our key growth driver, is maintaining strong sales momentum and remains early in its commercial life cycle with significant near-term commercial opportunities ahead through geographic expansion and pediatric label expansion. We are also operating the company with much greater discipline. We have reduced our full-year operating expense guidance while continuing to invest in our most important commercial and clinical growth opportunities. We must continue to transform the organization and significantly increase our level of execution to fully capture the many opportunities in front of us. Looking ahead, we have a compelling series of near-term clinical catalysts. As you've seen in the fourth quarter of 2026, we expect to report results from two phase II studies evaluating leniolisib in broader primary immunodeficiency populations, including CVID. Positive results could significantly expand the opportunity for Joenja and support its potential to become a blockbuster franchise. This will be followed in 2027 by the readout of the FALCON pivotal study of napazimone in patients with primary mitochondrial disease. Another program with the potential to materially increase Pharming's scale. While we have rebased our near-term revenue outlook, we remain confident in the strength of our commercial foundations, the quality of our pipeline, and the multiple catalysts that can drive our next phase of growth. I believe these combinations position Pharming well to continue building a leading global rare disease company. Thank you. We'll now open the floor for questions. Operator?

[Operator Instructions] Our first question is going to come from Jeff Jones with Oppenheimer.

Congrats on the strength of RUCONEST and the quarter. Could you perhaps provide some color around the commercial launch efforts for Joenja in the E.U. and how you're anticipating these to build? Maybe speak a little bit more to the magnitude of the opportunity as you expand country to country. Then for a second question, can you speak to assuming a positive outcome from the 2 PIDs trials, what that looks like and next steps moving ahead into that pivotal study? Thank you.

Thank you, Jeff. I'll ask Leverne to answer the first part of your question and Anurag for the second part.

Thank you, Jeff, good morning, good afternoon. In terms of commercial launch preparation outside of the U.S. for Joenja, we have made substantial progress in patient identification across our core 8 markets. We've identified core 8 markets that will be key for us to launch in the next couple of years. To date, we've identified about 387 potentially eligible APDS patients across these markets. We have launched in the United Kingdom, where we have gained some scale in our capability, both from a commercial execution perspective. Our market access capabilities has grown, which we've scaled into the German launch and are preparing for the launch in Japan. The U.K. launch is proceeding at pace, and we're making good progress. German launch is underway since the 1st of July, and we have made substantial progress in acquiring funded patients on therapy in Germany. From a Japan perspective, we are in the final stages of securing our pricing and reimbursement following approval in the second quarter. And we look forward to launching Joenja in Japan during the August timeframe.

Jeff, on your question about the two phase II studies. Again, we're expecting those results in the fourth quarter. The studies, as I've reviewed today, they have very similar patient populations in terms of one being a group of patients that are genetically defined and the other are more clinically defined. However, they have similar clinical manifestations, and we see significant overlap in the enrolled population, especially from the first study into the broader CVID study, which again, is a patient population that's about 40 times the size of APDS. So, when thinking ahead now to what we can expect, once we have the results, we'll review those results. We're collecting a broad spectrum of endpoints to evaluate. We're also evaluating the dose regimen, and then we'll come up with a plan to review with FDA, starting with FDA, but also with regulators across Europe as well as Japan to see what a registrational study could look like. We do anticipate right now, again, pending the results of those two studies, that we would do a combined CVID study, and this would likely be a randomized control study similar to what we performed in APDS. So, I think we have a good blueprint based on the work that we've done in APDS, and again, now anxiously awaiting those final results.

The next question will come from Alyna Shamsi with Jefferies.

Hi. Thanks for taking my questions. Firstly, on RUCONEST, enrollments appear to have recovered to around pre-competitor launch levels. I was wondering if you could give a sense of what you've seen so far the first month of this quarter and whether the trend has continued. And then secondly, I just wanted to ask on Joenja. There appears to be roughly, I think, 30 identified pediatric patients in the U.S. who could be eligible under the expanded dosing schedule, if approved. How do you think we should think about the pace of converting those patients onto reimbursed therapy following that approval? Thank you.

On RUCONEST enrollments, Alyna, thank you for the question. You're 100% right. So, we've seen certainly Q2 with 84 enrollments getting fairly close to what we had in Q2 2025, which neared about 90 new enrollments. We are seeing consistency in the momentum into the third quarter. Now, in the HAE market overall, there is some seasonality. If you look back at new enrollments in the previous year or the year before that, you do see some changes in the rate of new enrollments as we go into the third quarter. As I said, it's quite consistent with what we're experiencing deep into July so far. Importantly, we continue to add new prescribers. And what we're seeing in these new enrollments is this entrenchment of the RUCONEST position in high-burden HAE patients. These are typically either HAE normal patients, where the burden of disease is quite high, or patients with more frequent and more severe attacks, and that's where we anticipate having a really durable position, even 4 quarters after competitor launch, to continue into the third quarter.

Joenja?

From a conversion perspective, as I mentioned before, we have a substantial number of patients identified in these core markets, including certainly the U.K., Germany we're launching now, and Japan launch coming. We are assuming a fairly similar conversion rate that we've seen in the U.S. as we secure reimbursement and the commercial teams execute against our plan in the various centers, both in Germany and upcoming in Japan. Part of that will be conversion of patients that are on our EAP programs in some markets, but also pretty clear -- pretty clear identification for us in where those patients are sitting in these respective markets and what we need to do to convert them as soon as we have reimbursement.

Specifically for U.S. pediatric?

For U.S. pediatrics, similarly, what we have today is 60 identified patients between 4 and 11 years old. As Anurag mentioned earlier, the submission is divided into the higher dose and the lower dose, and what we see in the split of those identified patients, it is about 50/50. So, 50 patients in the higher dose category, 50% rather, and 50% in the lower dose category. Again, we are assuming a similar uptake what we have seen with the adult population 12 and above, converting first those EAP patients in the first few months and then continuing to convert the remaining patients that we have identified in the centers that we have.

Thank you, Alyna.

Our next question is going to come from Whitney Ijem with Canaccord.

Hi, all. My congrats on the quarter. Just to go back to the 84 new RUCONEST patients, can you talk a little bit more about those patients in terms of what they look like, severity, attack rate, is that consistent with how the RUCONEST patient has looked historically? Are they all new to therapy or some returning back from maybe trying something else?

Thank you, Whitney. Leverne?

Thanks, Whitney. So, the 84 new enrollments, as I said before, these are -- the patient population for us in terms of new enrollments have evolved over time. Right, but now we're seeing a very clear trend emerging for these high-burden patients that have high-frequency attacks, more severe attacks, patients who are more worried about high-risk attack locations like laryngeal attacks, GI attacks, and these are just the new enrollments. So, the new enrollment number does not include restarts, as you've identified, i.e., patients who are returning back to therapy. So, pure new enrollments, unique new for the first time on RUCONEST.

Got it. That's really helpful. Then moving over to CVID and PIDs. In terms of the larger patient number, clearly, very large TAM assuming success there. I guess, can you talk about any efforts in terms of patient identification or just as we think about the diagnosis rate in those, given they are larger, but not genetic, I guess how well-characterized are those patients and under the care of physicians currently, in terms of being able to access them commercially?

Sure. Hi, Whitney. This group of patients, these CVID patients, is largely already identified, and that's very different from what we experienced with APDS. With APDS, it was a newly defined disease just in the last 15 years. It required genetic testing, and it required a considerable effort in terms of educating doctors also on the importance of genetic testing. CVID, however, is clinically diagnosed, and these group of patients that we're talking about are, again, already diagnosed. They're almost always on immunoglobulin replacement therapy because that's part of one of the key manifestations is that they have low IgG levels. And therefore, really, it's a different, let's say, mechanics in terms of diagnosis versus what we've seen with APDS.

The next question is going to come from Joe Pantginis with H.C. Wainwright.

Thanks for all the updates today. Two questions. First on RUCONEST and then on Joenja. So, with regard to the current commercial profile coming into the second half, it was mentioned in the prepared comments, with the three factors. One of them was inventory normalization. I just wanted to know any anticipation for further optimization here or anything we can expect with regard to inventory and distribution going forward that might be relatively obvious to you.

Good morning, Joe. This is Kenneth here. We expect that we are in a year with a normal seasonality where we saw some inventory reduction in the first part of the year, and there will be a normal cycle with a bit of inventory build also in the year to go, but all within normal frame. Remember that when we commented on Q1, we said that 2025 was the unusual part. So, we had a normal year when it comes to the inventory.

Got it. Then for Joenja, I'm going to phrase my question very carefully. With the upcoming PIDs CVID data, which is obviously a major event for you guys, obviously you cannot market these data. However, you can leave or present these data to physicians as just sort of like a supplemental information concept. Do you have any views towards these data providing any sort of incremental boost to revenue prior to potential approval? Obviously, from an off-label standpoint.

Joe. Obviously, as Anurag said, we have those two phase II open label study that we read out in Q4, and we would be engaging with the FDA and our base assumption that we're going to have to conduct a phase III trial. Until the phase III trial read out and we receive proper approval from FDA, we won't be able to promote the data, and we don't expect any sales from CVID. This being said, there are a lot of opportunities in APDS, as we disclosed, the many opportunities in APDS in the U.S. in adult as well with the expected upcoming pediatric and the geographic expansion should allow us to fuel the growth of Joenja very significantly in the coming years as we wait for these new indications to materialize.

The next question is going to come from Sushila Hernandez with Van Lanschot Kempen.

Yes. Thank you for taking my questions. On RUCONEST and the on-demand HAE market, how do you expect that these dynamics on new patient initiations will play out or do you expect that patients will still find RUCONEST after they have tried other drugs?

Certainly. Thanks, Sushila. From a new enrollment perspective, we think about inflows in two different ways, right? So, new enrollments or brand new patient starts and restarts. When we look at new enrollments, the vast majority of those come from, in fact, patients who've had suboptimal responses to other therapies and now switching to RUCONEST. We expect that to continue because what we're seeing in patients who have high severity of attacks, patients who have more frequent attacks, two or more attacks per month, they do require a more reliable, faster onset HAE treatment and that is where RUCONEST is uniquely positioned compared with competitive set to continue to have a position.

Okay, that's clear. On Joenja, you mentioned the 8 core markets outside of the U.S. Could you also break down or give us more color on which of the markets will be key drivers for growth ex-U.S.? And also on the VUS reclassifications, what are the next steps and when could this meaningfully add to Joenja opportunity?

On the core 8 markets in the very short term, we are especially excited about the progress we're making in Germany. We're one month into that launch, so that is going to be key for us as our next ex-U.S. market, our first European launch. And subsequent to that, the Japan launch that we are planning for August will be another key growth driver for us in the short term. Additionally, from a U.K. perspective, we continue to make progress in adoption in APDS within the U.K. One additional piece that I'll add is especially excited about the Japan market because we do have an approval for ages 4 and above, which is our first pediatric launch that we will have before we anticipate U.S. approval later this year.

Sushila, on the VUS reclassification efforts, we actually have a number of efforts that are ongoing. We have the efforts that are trying to help clinicians reclassify VUSs on a one-by-one basis. And again, that's ongoing now and that involves collecting additional data, looking at literature, family testing that can also be helpful in resolving these VUSs. That's happening on an ongoing basis, and we're seeing patients getting reclassified as a result of that. On top of that, we have the much larger project that we talked about in the past that we had partnered with Columbia on, that work has also been initiated. Once I have a little more clarity on when that's getting wrapped up, we'll be able to give you more guidance on what the timing of that, but that work is well underway.

Okay, thank you for this additional color. Just the final question. For the genetic PID phase II study, could you remind me again, how did you select these specific genetic PIDs as there are so many and which ones are more directly or indirectly involved in a PI3K delta-driven immune dysregulation?

The genetic PID study, the genes that were selected for it were all selected on the fact that there was a connection to this pathway and that these abnormalities in these genes could lead to driving of that pathway, and there was published data on that. So, that's how that group or that basket of genes was selected.

[Operator Instructions] Our next question comes from Simon Scholes with First Berlin.

Yes, hello. Good morning and good afternoon. I've got three or four questions, if I may. The first one is on the new enrollments. You've pointed out that with 84 new enrollments with RUCONEST in Q2, I mean, you're quite close to the level you were seeing in the first half of last year, which is over 90. Of course, in the first half of last year, you were seeing U.S. growth in RUCONEST of 30%, and now it's retreating somewhat. So, I think it would be helpful if you could give us the net figure, i.e., the new enrollments, less switches and drop-offs. Then also, I was wondering if I'm correct in thinking that the next EU launch for Joenja after Germany will not be until 2027. And then I was also wondering if you could give us an idea of when you might see pediatric approval in the European Union. Just lastly, I was wondering if you could give us some more color on the manufacturing impairment of inventory, what exactly was happening there, and which product does that relate to. That's it from me.

Thank you, Simon. I'll start with the new enrollment and the outlook, the growth outlook for RUCONEST, as I understand your question well. As we've said, based on the robust underlying key performance indicator related to the resilience of RUCONEST, 93% of the active patient base maintained year-over-year, as well as the solid new enrollment that you pointed out. We expect to stabilize RUCONEST and have it return to growth. Yet, as I've explained, we see RUCONEST as a durable cash engine for Pharming with Joenja being the growth drivers for the years to come. So, that's how we are approaching our portfolio. That's how the company is evolving from becoming a one asset company to becoming a more diversified rare disease company with a longer-term growth profile, given the number of growth catalysts for Joenja, as well as very significant pipeline catalysts for Joenja as well as napazimone. When it comes to geographic expansion, I will let Leverne provide her perspective.

In the short term, of course, we are in our peri-launch phase in the U.K., German launch is underway, and then we're looking forward to the Japan launch in the August timeframe in ages 4 and above. For 2027, as we look into that timeframe, of course, we have announced both approvals in Canada and South Korea, and we are anticipating that we continue launch progress toward Australia and Italy in 2027. So, when we look in totality at the potential for continued growth and expansion in 2027, we plan to make good progress across those markets for Joenja pediatric and as well as Joenja adult. The second Joenja question, if you could repeat that.

Was about pediatric indication in Europe. I'll let Anurag.

Yes, that's right.

Hi, Simon. Obviously, as Leverne mentioned earlier, we've identified a large number of APDS patients outside of the U.S. in these core markets, and a significant number of those, similar to what we've seen in the U.S., are pediatric patients. So, the unmet need is very similar across Europe. We're planning to bring the same data set that we have from these pediatric studies to Europe as well. And we're going to do that by beginning in the U.K., as we did with the adolescent and adult population, then we'll continue to do that across Europe.

When it comes to the manufacturing impairments, Kenneth?

Yeah. This is a matter where we have impaired for manufacturing inventory throughout the manufacturing process related to RUCONEST. This is a matter that is not concluded upon, but the indicators are that we have inventory that may not be possible to use for commercial use. We will conclude on the matter before the end of Q3, but according to our normal process, we impair for such kind of issues when we are made aware of them.

[Operator Instructions] Thank you. I am showing no further questions in the queue at this time. I will now turn the call back over to Fabrice for closing remarks.

Thank you, operator. Thank you so much for all the relevant questions. We hope that we provided additional color on our Q2 earnings. Not only about the revised revenue guidance, but also about the business fundamentals and the fact that we continue to manage a much tighter operating model that has allowed us to make sure that we allocate capital more efficiently and fully capture the number of growth opportunities and pipeline catalysts that are ahead of us. So, we look forward to updating you on our plans as those catalysts will unlock in the coming weeks and months. Thank you very much.

This concludes today's conference call. Thank you for participating, and you may now disconnect.