Swedish Orphan Biovitrum AB (publ)

The conference must not be recorded for publication or broadcast. At this time, it's my pleasure to hand over to Guido Oelkers, CEO. Please go ahead.

Yeah, thank you. Hello, everybody. This is Guido Oelkers, CEO of SOBI. We are delighted to welcome you to the second quarter 2025 conference call for investors and analysts. We posted this presentation to SOBI.com earlier. forward-looking statement as per usual. Please take note of this. And with this, let's go right into the next slide. Today, we plan to cover key aspects of our Q2 report. I'm joined by Henrik Stenqvist, our CFO, and Lydia Abarth-Franch, head of R&D and chief medical officer. We plan to review the presentation first and then have a Q&A until around 3 p.m. CET. For those on the phone, please join the queue. And for questions, as mentioned, please press star one. And we ask you to only ask, really request only to ask one, maximum two questions at a time. Let's go to slide number four. You know, the second quarter, when you elevate yourself, was really for us about, you know, strong economics, drive important pipeline milestones, and create economic preconditions to support our future growth ambitions. So with regard to the first subject, strong economics, we delivered 6.2 billion revenues representing a 22% growth at constant currency and our adjusted EBITDA was at around 34%. I think it's important to note that our strategic growth portfolio or portfolio is accounting now for 55% of total revenues and grew impressively 65%. And this supports, you know, our quest of transforming Sobi into the next phase. And the growth was driven by Altuvox, Dr. Lead, Asper Valley and Gamifund. Wonjo had a slower quarter and showed a 4% decline. We'll talk more about it because it's primarily driven by the gross-to-net effect. On the R&D milestone side, we were able to show an approval of Gummy Fund in HLH, MAS, and SILS disease by FDA, marking a significant expansion of our potential market. We were able to complete the filing for NASP with the FDA in an uncontrolled gout. And we were also able to present very strong 52-week data for Asper Valley in the two nephrology indications. As regards to economic preconditions, to support our launches and our ongoing R&D projects primarily, we were able to sign a royalty agreement that we will talk more about with Appellis. And we were taking out costs in the organization in a very selective way, and Henrik will talk more about this. On guidance, we have obviously a strong momentum, and we are very confident about our business also growing this into the future. But at this moment of time, we feel it's prudent, let's say, to confirm the guidance that we set out earlier and not going ahead of ourselves. So let's turn to slide number five. Let's look at the overall performance in more detail. We saw SOBE's continued performance in Haemophilia with strong 27% growth and the combined Haemophilia franchise growing at 24%, portraying a robust growth of Althovox in Haemophilia A. Immunology has grown at 11% by strong performance of Gramifan and Kineret, And geographically, Europe's growth of 21% has been propelled primarily by Altovocht and Doctolet during the second quarter. North America and international have been contributing growth of 16% and 44% respectively. And the growth of North America is primarily driven by Doctolet and Camifant. Let's move to the next slide. Focusing a bit more across the portfolio in the second quarter, we have seen good growth momentum and strong progress for Altovox launch within the Haemophilia A franchise. We have demonstrated growth of 32%. And we believe that this is really just the beginning for Altovox, because the growth in the first, let's say, two quarters was really primarily driven by three markets. and we have now 17 markets on stream, but most of these markets are at a very early stage, so the best is still to come. Dr. Lett showed continuous strong demand across all regions, including the US, with 43% growth in 2024. This highlights the continuous confidence in the product's efficacy and in expanding reach. For Aspaveli, we are pleased to see continued growth in the number of patients across markets, even though we are facing competitive pressure in the P&H space by Orwells. We are seeing more pressure in Europe than in international markets, but believe that we can continue driving growth with this important product for us. Wonjo experienced a 4% decline, whilst demand was increasing significantly versus Q2 last year. The decline is attributed to gross to net adjustments, which we are closely monitoring. Camifund and Kineret continue to show good demand in the US. Camifund delivered an exceptional performance in Q2 with 33% growth driven by robust demand. Overall, these results underscore the strength of our portfolio and our ability to navigate in competitive markets effectively. Please turn to slide number seven. So let's turn our attention to the continued success of Altevocht. In the second quarter, hemophilia as reported of Altevocht and Elocta grew impressively 32%. This growth is a testament to the strength of our portfolio and the successful launch of Altevocht. Focusing on Altovox specifically, the product achieved sales of 627 million SEC in Q2, reflecting a strong growth momentum. Launches are progressing well across Europe and the Middle East. And as mentioned, we have now 17 countries on stream, while the growth of the actual sales are really primarily driven at this juncture by Germany, Switzerland, and Spain. And we are also excited to announce a full launch in the UK this July, which we anticipate will further drive adoption. Overall, the launch of AltaWorks is progressing exceptionally well, and we remain focused on expanding access and delivering meaningful benefits to patients in our territories, and look forward to continuing to add launch countries in the second half of this year. Please turn to slide number eight, Aspar Valley. Let's now review the latest updates on Aspar Valley. Starting with P&H, we continue to see strong year-on-year growth across markets, driven by increasing adoption of therapy. However, we are also navigating growing competitive pressure in Europe. Moving to nephrology, we are excited about the progress in this area. In the EU, we anticipate a CHMP opinion by the end of 2025, which will be a key milestone for our portfolio. Our confidence in the product is bolstered by the 52-week VALIANT data, which were presented at ERA 2025. These results reinforce the product's best-in-class profile, positioning it as a leading option for patients in this space. Lilia will cover the results in more detail in her presentation. Additionally, we have made a significant update to our royalty agreement with Appellus. Under this new agreement, we have achieved a 90% reduction in ex-US royalty obligations until defined caps are reached. After these caps are achieved, the royalties will revert to original license agreement. This agreement involved an upfront payment of 275 million in cash, with an additional 25 million contingent upon EMA approval in nephrology. We will offset the upfront by having lower royalties both from cash flow perspective on our P&L moving forward. This deal reaffirms our commitment to our ongoing partnership with Appellus and our belief in the product's potential to deliver significant benefits to patients and to SUBI's long-term growth. Let's turn to Wonjo, where we continue to see a positive momentum despite external challenges. Demand for Wonjo increased quarter on quarter, reflecting its growing adoption. However, this growth was outweighed by continued impact of healthcare reforms, primarily to mention here the reform in Medicare and the 340B scheme. In patients with below 50,000 platelets, Wonjo has achieved a significant market share underscoring its strong position in this patient population. Looking ahead, our strategic focus for Wonjo is firmly focused on a number of elements. First, we want to continue to grow in the below 50,000 platelet population whilst expanding into the 50 to 100,000 platelet population in alignment with the NCCN guideline. Second, we are committed to generating additional data to support potential label expansion and to complete the pacifica trial which is critical for achieving full approval status and broadly internationalizing the product third we plan to leverage the pacifica data as mentioned to drop to broaden the product on a global scale and finally we are exploring new indications and in this context we need to mention the pax study where we start to enroll. These initiatives reflect our commitment to maximizing Wanzhou's long-term potential and delivering meaningful benefits to patients across a range of indications. Let's turn to slide number 10, Gummy Fund. I'm thrilled to share a significant milestone for Gummy Fund and the patients we serve. The FDA approved Gummy Fund as the first ever treatment for adults and children with macrophage activation syndrome in stills disease. This approval addresses a critical unmet medical need, where we have demonstrated compelling results. 54% of patients achieved a complete response, highlighting the therapy's effectiveness in resolving MAS symptoms at week eight, and 82% of patients achieved clinical MAS remission. This approval not only validates the strength of our clinical data, but also reinforces Gamifran's potential to transform the lives of patients with HLH and MERS-instilled disease. We look forward to further unlocking the unique anti-interferon gamma mechanism of this molecule in the future. Let's turn to slide number 12. You have seen in the first quarter that we have now made tremendous progress in our key building blocks with the approval of Gummy Fund and completing the filing in the U.S. with NASP. We continue to focus on two launches in the future now on three launches including Gummy Fund and secondary HLH. ASPA Valley is on track and a CHMP opinion is expected by end of year. And four innovative development programs are on track with first patient in achieved for the PECSIS-30 in Vexus during this quarter. These initiatives reflect our commitment to drive innovation, expand our portfolio, and to deliver transformative treatments and therapies to patients worldwide. With these investments, we are well poised to achieve sustained growth and make a meaningful impact in the years ahead. Let's turn to slide number 12, and now I'd like to hand over to Henrik, our CFO.

Thank you, Guido, and hello, everyone. So please turn to slide 13, and we will take a look at some key financial metrics for the quarter. So in Q2, our revenues of 6.2 billion corresponded to a revenue growth of 22% at constant currencies with growth across the portfolio. Growth has been driven by a hemophilia franchise, including a continued strong launch of Altobox, as well as Topselet and Gamifund. In addition to strong revenue growth, we delivered an improved Q2 adjusted EBITDA margin of 34% compared to 28% last year. So if we look at the bar chart on the left with revenues by quarter and business area, we see the solid growth in hematology of 27% in the quarter at CER, including our hemophilia A products, Eloxta and Altubox, increasing by 32% at CER. And we continue to see strong momentum for out-of-the-box in the markets where we have launched to date. In addition, we've continued strong growth in top-to-net of 43% at CER due to growth, both in the U.S. and ex-U.S., as well as with Asper Valley, driven in large part by our international region. Bonjo demand decreased quarter-on-quarter, but was negatively impacted by gross-to-net adjustments, mainly related to Medicare redesign requirements. Immunology grew at 11% in the quarter due to double-digit growth in Kindred and our highest quarter of sales to date in Gamifant. And sales in our international region was another positive for Gamifant. And as a reminder, Bay Fortis sales and the royalties we receive from Sanofi are seasonal in the second half of the year. Referring back to the table on the right and the adjusted gross margin of 77% in the quarter, a slight improvement from Q2 last year. Gross margin was positively impacted by products and country mix effects, offset by products return in synergies. And looking at the operating expenses for the quarter, we observed a 7% growth at CER compared to the same period in 2024. SG&A, excluding non-recurring items and amortization, increased by 14% at CER in the quarter, driven by launch and pre-launch costs for Altevocht, Asper Valley in nephrology, and NASP. And this was partially offset by lower costs for synergies and Elocta. R&D expenses declined by 8% at CER, excluding non-recurring items, mainly due to NASP programs that are now complete. And this was partially offset by post-approval development costs for Altevoxt and development costs for Gamifund and Bonjo. We're also investing in the four innovative programs for Bonjo in Vexus and CMML, Altevoxt in Sinobitis, and Gamifund in IDS. Operating cash flow for the quarter was 1.4 billion. The decrease from last year is mainly due to the seasonal shift of RSV revenues to the second half of the year, partially offset by higher operating profit and lower interest payments. Net debt continues to go down, ending the quarter at $11.4 billion, corresponding to a net debt to EBITDA ratio of 1.1 times. Please turn to slide 14. And I would like to take a moment to address the restructuring charges of 208 million recorded this quarter. And you will find additional details on page three of our report. And this was a proactive and strategic decision aimed at positioning our organization for long-term success. As Guido mentioned earlier, we are in a phase with two major launches, three key filings, and four high-priority development programs. These initiatives require focused investments and organizational agility. We see it as a reallocation of resources, prioritizing areas that will drive innovation and deliver the greatest impact. While these decisions are never easy, they are necessary to ensure responsiveness and focus on the right key priorities going forward. Next slide, please. And we will now discuss the financial outlook for the full year 2025. As usual, this outlook is based on revenue growth at constant exchange rates and adjusted EBITDA margin. For the full year 2025, our outlook is unchanged. We anticipate revenue to grow by a high single-digit percentage at CER and an adjusted EBITDA margin in the mid-30s percentage of revenue. The key considerations for our outlook have not changed since Q1. To start, the royalties we received from Sanofi for Bay Fortis sales remain the largest uncertainty, both for revenue and beta margin guidance. While our view on Bay Fortis is clearly positive, we do not control the outcome. And there are unknowns related to new competition in this space, as well as other factors within the U.S. healthcare landscape. However, we are continuing to see strong patient uptick in out-of-box markets where we have launched, with more major markets launching in the second half of the year. We also expect continued progress with our current commercial portfolio. In regards to our EBITDA margin guidance, we will increase investments in our pre-launch assets, specifically NASP and S&LE in nephrology in the second half of the year. In R&D, we have the four priority development projects. On a final note, these are uncertain times for forecasting with everything that is going on in the world and in our industry, and many of these topics are still unknown and speculative, making them difficult to quantify and comment on. Having said that, and as evidenced by our Q2 report, we have very strong momentum in our business going into the second half of 2025. And I will now hand over to Lydia. Thank you.

Hello, everyone, and thank you, Henrik. We'll start with the pipeline milestone on the next slide, please. I'm excited to share that we had a very productive second quarter with regulatory successes and important clinical progress. Starting with us, Paveli and Paveli, In June, we presented at ERA the 52-week data of the Pivotal Valiant Study in C3G and ICMPGM. They show sustained efficacy consistent with the 26-week results, and I will go into more detail in a minute. In TATMA, the Transplant-Associated Thrombotic Microangiopathy, following completion of the Phase II study and a strategic assessment of the treatment landscape, we decided together with our partner, PELIS, to discontinue the development for texetacoplant in this indication. In the full study, texetacoplant demonstrated favorable safety and tolerability consistent with its established safety profile. Moving to gamifan, the FDA extended the indication for gamifan to HLH mass in a stills disease at the end of June. Importantly, this is the first FDA approved treatment for this condition. which is serious and potentially life-threatening. We are truly excited to provide a new therapeutic option that helps control hyperinflammation and can reduce reliance on high-dose glucocorticoid steroids. Also, at the end of June, we completed the rolling submission for NASP in uncontrolled gout, and the next step will be the FDA validation of the application. For Bonjour, the Vexor syndrome proof-of-concept study called PAXIS enrolled their first patients. Notably, this is the first clinical trial for a treatment in this condition, and we see very high interest in the scientific community. And lastly, we continue our focus on joint health for Alpovox. Bleeding into joints is the major and most common clinical manifestation in hemophilia patients, preventing them is the focus of service phase for activities for ALTVOX. The altitude study is a low interventional study to understand how ALTVOX can prevent joint bleeds in the real-world setting. And we enrolled the first patient already in May. Next slide, please. We presented new PECS-ETACO plan sets of data from the VALIAN Phase III Pivotal Study at the European Renal Association Congress in the late breaking session. They included several new subgroup analysis at week 26 when the randomized placebo-controlled period ended, shown on this slide, and the final 52-week data, which also includes the additional 26-week open-label period when all patients received pexotacoplant. And I will present this on the next slide. Looking at the various patient groups, pexotacoplant shows consistent efficacy regardless of disease type, age, or transplant status. The first column on this slide shows the data for the overall population at week 26. And Astavel demonstrated a statistically significant 68 proteinuria reduction versus placebo, and improved estimated glomerular filtration rate, as well as histology improvement, with more than 70% of patients having no C3 staining in the biopsies, which is truly impressive. The new subgroup analysis at week 26, shown in the next three columns, makes it clear that the effects are very consistent. The second column shows patients in the nephrotic range, where kidney damage has already progressed, and we see a similar improvement across all parameters, proteinuria, EGFR, and histology. Many C3 and primary ICMPGN patients are currently treated with immunosuppressive therapies. On the third column, you see how pexotacoplant is clearly efficacious across all parameters shown, regardless of whether patients are already on immunosuppressants or not. And finally, both C3GM primary IC and PGM are often diagnosed in adolescence. As a reminder, 44% of valiant participants were between 12 and 17 years old, with the same picture of efficacy again, as shown on the right-hand side column on this slide. This robust reduction of proteinuria and stable kidney function across a broad patient population of patients is very encouraging and highlights the potential of Aspavelli. Next slide, please. The effects I just described were sustained at one year. Patients receiving pexitacoplant for a year saw a proteinuria reduction of 67%, and they continued to achieve a stabilization of kidney function as measured by the EGFR. Biopsies were only available at week 26, so there is no one-year data. According to the recently published Kidney Health Initiative consensus paper, a favorable effect on the three endpoints of one, proteinuria reduction, two, EGFR stabilization, and three, histopathologic improvement, provides convincing evidence of efficacy for treatments targeting the complement pathway. In addition, patients who switch from placebo to Aspavelli at the start of the open-label period experience a similar magnitude of benefit in proteinuria reduction and the stabilization of kidney function. With our submission to EMA at the beginning of this year, we hope to offer this new treatment option to patients very soon. Next slide, please. Looking ahead, we anticipate progress with the major regulatory submissions to FDA, EMA, and PMDA. In the U.S., the focus will be now on the NAST regulatory review, while we expect a decision for doctorate in pediatric ITP very soon. In Europe, we anticipate the CHMP opinion on Aspavelli in nephrology by the end of this year. We also anticipate an EU decision for our newly partnered product, Olesarsen. In Japan, we plan to submit Gamifan in HLH mass, as well as Aspavelli in C3G and primary ICMPGM. And furthermore, we anticipate the submission of Kineret in Steve's disease and a decision on Doctelet in ITP. We're also planning to advance the clinical projects with a host of exciting exciting developments. This includes the first phase 2a data on gamifan in interferon gamma-driven sepsis from our research collaboration that will inform our decision on the next development steps. Another milestone will be the readout of the LOTIS5 study of SYNLONTA in second line diffuse large B-cell lymphoma next year. And with that, I would like to hand back to Guido. Thank you.

Yeah, thank you, Lydia. So summing it up, let's move to the next slide. We are really pleased with Sobi's development in the second quarter and so far in the first half of this year. As you can see in quarter two, 22% growth, 65% growth of our strategic portfolio. They speak for themselves and they demonstrate that the company has a very material momentum. Our R&D pipeline has shown tremendous progress and we have continued launch success with Altuvox in Europe. You know, and as you have seen, you know, I mean, three products, the three countries are currently the main source of Altuvox's growth. 17 have come on stream. UK comes on stream now in July. So, you know, that gives you a bit of a flavor that the best is still to come. We've got the approval of Gamifund. for MAS in the US. We have completed the filing of NASP in uncontrolled gout in the US. So we look forward to complete the review of NASP with the FDA and ASPA Valley with EMA and bring these important medicines to the market in 2026. At the same time, we are continuing to push forward with our four priority development projects And it was gratifying to see that we had the first patient in this quarter for Wonjo in the Paxos study for the potential treatment of Vexus. And we are also making good progress with the IDS study. So with all these activities, we are preparing the organization and ensuring prioritizing that we are fit for purpose. And also with the economic space that we have created, that we can take advantage of these opportunities that are ahead of us. The organizational changes were necessary to do so, but they are only a retooling of the organization that allows us now to take advantage of these development projects that are ongoing, the ongoing launches and the forthcoming launches. We have a very strong momentum. in the SOVI business, as you have seen, is in pipeline development, and we look forward to continuing this journey with our colleagues and stakeholders around the globe. With this, I would like to open the call for questions. And I'd like to refer back to the operator.

We will now begin the question and answer session. Anyone who wishes to ask a question may press star and 1 on their telephone. You will hear a tone to confirm that you have entered the queue. If you wish to remove yourself from the question queue, you may press star and two. Questioners on the phone are requested to disable the loudspeaker mode and eventually turn off the volume from the webcast while asking a question. Anyone who has a question may press star and one at this time. The first question comes from Gonzalo Artiac from Danske Bank. Please go ahead.

Hi. Hello, everyone. Thank you for taking the questions. I have a couple of them. The first one, it's on your guidance. Does the lack of top-line guidance upgrade come only due to questions or uncertainty on Bay Fortress performance in the second half now that there is a competitor around? Or is there anything else that we are missing or being over-optimistic right now and should be more cautious? And my second question also related to guidance, it's on your EBITDA margin. I mean, I guess that one of the main reasons for keeping it at mid-30s is due to the base assumption that you guys will receive priority review for CEL 212, which would imply, I would say, high OPEX already in H2 ahead of launch and early 2036. But if you guys do not secure priority review, what would be the impact in your adjusted EBITDA margin since the potential approval would come then in mid-2036, if I'm not wrong? Would it make sense to assume you would push SG&A to next year, which would raise your adjustability margin for 2025 mechanically? Thank you.

Yeah, thank you. I mean, this is, you know, let's put it this way. With regard to the guidance, we are not aware, you know, that products are under threat or under pressure. That should make us more cautious about the prospects of our products. beyond what we are disclosing obviously in the report. And let's say with regard to Bifortis, I mean, the only thing I can say is that, you know, it's in the hands of Sanofi, they seem to be confident about the product, not heard anything else. So if they are confident, I have no reason to be not confident simply because, you know, they used to be the largest vaccine company in the world and probably are still, you know, in this prevention market now. So, you know, there's nothing to it. It's more prudent at this stage, let's say, because, you know, there are so many elephants, but clearly not related to our portfolio. You know, the EBITDA margin, it's true to say that, you know, when you believe in an bolus of pay-forters coming in in the second half and you have the effects, as we pointed out, you know, that it may be beneficial to EBITDA margins. But, you know, we have not only the NASP launch that we may have to prepare. We have obviously the C3G launch where we may have to step up because the opportunity is just so vast and we are understanding more the competitive dynamics. And we have a potential readout on IDS But all of these reasons, you know, you want us to spend some money. So that's the reason why we are not as specific and, you know, on the on the mechanics of the uplift of the guidance. But, you know, everything we are doing right now is is, you know, it's pointing is positive and and we will influence those investments. So, you know, we are we are not spending freely. I hope it gives you a flavor for the for the company. Next question.

The next question comes from Mattias Hegblom from Handelsbanken. Please go ahead.

Yes, good afternoon. Mattias Hegblom, Handelsbanken. Thanks so much for taking my questions. I have two, please. So firstly, Roche presented data from a Phase I-II study for its next generation of specific remethyl at ISTH. The majority of patients developed anti-drug antibodies against the therapy to the extent that their PK data was affected. I was curious if Lydia had any thoughts at this stage of a candidate with such a profile and what to look for in the future programs. And then secondly, with regards to Vonja, I can't recall we spoke about the confirmatory Pacifica trial for a long period of time. The trial's got gone. This is us reading out late 26. I'm not sure that's up to date. How's the trial enrolling? What are the timelines? Have you activated more sites than originally planned? It's not listed as a pipeline event on the new slide, so I guess it's later than 26. That's it. Thanks so much.

Thank you. And maybe, Julia, direct to you.

Yeah, I'm really sorry. I couldn't hear. So you mentioned an ISTH presentation of data, but I couldn't understand which asset did you talk about? I'm sorry.

Yeah, sorry for being confusing here. So, Rosh, by specific, Next Generation 007, a majority of patients developed ADAs. I was just curious to hear how you thought about that to the extent that PK was affected of the therapy.

Yeah, yeah. Sorry. Sorry that I couldn't understand. So, yeah, our... We are always careful in analyzing and interpreting data from competitors, especially when it's so early. These are assets that initially are very promising, but as you referred to, there are many things that can happen during a development program. So it's something that we're monitoring and that we are cautious about. We still believe that the future, as also at ISTH, and there are some publications coming up on really the normalization and the new class of factor A therapies to really bring the patients to those very high levels of protection. So, to me, it's a little bit early to comment on, but we will obviously keep monitoring these developments.

And then, Pacifica, an update and timelines when we should expect data from this confirmatory trial for Bonjour.

So for Bonjour, the confirmatory trial, we are seeing further acceleration coming from international markets. So we are adding additional markets where we are seeing a lot of traction. And so we are very excited. hopeful that we will be bringing the timelines even further ahead of what we originally planned. But it will depend if this acceleration that we are seeing now keeps continuing. So I think it would be better, like as we meet on a quarterly basis, we will continue confirming if that acceleration track is continuing. and if we see this acceleration materializing. But so far, yeah, we are very happy with the new additional countries that we are including, and, yeah, that's why we expect data now probably most likely in 2027. Thanks so much.

The next question comes from Christopher Hude from SED. Please go ahead.

Hi there. Thanks for taking my questions. Two, if I may. Altevox, the first, and then the reorganization, the second. So Altevox, it continues to impress, of course. How much of the sequential growth is Germany supposed to lend and how much the other countries? Or put another way, how do the initial launch trajectories in other markets, especially Spain, compare to that of Germany? And my second question is, so the market seems to be interpreting the reorganization and maybe in combination with the palace renegotiation pretty negatively, as if you're looking for ways to squeeze more margin for something you hadn't previously planned. Could you expand on the reasons for the reorganization and negotiation, particularly what you might use any added margin headroom for, if anything? And perhaps, given that you've got three launches, to what extent has your thinking and expectations changed around those launch costs over time.

Thank you. Thank you, Christoph. With regard to the launches, Germany, Switzerland, and Spain is clearly the vast majority of our business right now, north of 80%. That's the reason why the next phase of launches will obviously do well for the overall performance of the of also moving forward because there were some questions you know also now the UK is obviously expanding that pool of patients pretty materially and then we expect also France coming on stream in the second half at one stage so you know so this is which is also not part of the 17 countries right now so it's you know it's a normal European rollout and that will propel it, but it is really primarily driven by those three countries, while the Spanish market is smaller, you know, probably also partially driven by the history, you know, with the plasma-derived product years ago, you know, where unfortunate things happened. And, you know, the best is de facto still to come, but it is these three countries that are driving it, and And then the other markets will pay more dividends in the second half and then in the quarters to come. And with regard to the reorganization, I think this is a little bit misunderstood or blown out of proportion. I mean, it affects 5% of our headcount. It's a trimming exercise, as many companies are doing on a regular basis every four or five years. And it's just good practice to make sure that you... create space, then also for new talent base, the organization is changing. The business is changing. So do we. And that means, you know, that we need to adjust and it creates overseas the space, you know, I mean, we mentioned earlier the, the NASP launch where we need new talents. We probably need to expand a bit more in the, in the, uh, C3D launch in Europe. And then, you know, we need to also have some readiness should the IDS data come out well in the fourth quarter. So we plan for success. Hence, we have created some space. But this is not, you know, in proportion. It is not really extraordinary. Maybe, Henrik, you want to comment also?

Yeah, no, I think you're confident it's getting the priorities right. simply, and a normal trimming exercise that most companies do. So nothing to add really.

Good. I hope this gave you a flavor, Christopher. Maybe then we move to the next question.

The next question comes from Harry Gillies from Berenberg. Please go ahead.

Hi, thank you very much for taking the call. I had another one just on the top line guidance. which obviously you guys reiterated. And of course, there's the uncertainty with Sanofi sales of Bay Fortis, you know, being a major cause of uncertainty in the rest of the year. But would it be fair to say that the rest of the business has exceeded your expectations in the first half of the year? And then my second question is on GammaFant. You've seen very solid growth in Q1 and Q2 above 30%. So I was just wondering how much of that is off-label use in the secondary HLH indication versus the original label. And then is that a growth rate we should forecast to continue into the sort of near to medium term? Thank you.

Yeah, thank you. I mean, I think it's fair to say that the first half of most of our products, I mean, You know, we clearly, we were hoping for it, but we probably didn't plan all of that. So, you know, it's a very solid growth. You take away the extraordinaries that we had also in Q1. So, you know, it's a very consistent, very, very strong underlying performance driven by the new products. I2Work launch is exceptional, you know, and as I said, you know, we We don't see here a plateauing effect. We see it, we keep it, we will be expecting it to keep growing. With GAMI Fund, you know, if you can afford to have a more, you know, not quarter on quarter perspective, but more of an annual perspective, you know, and this is what we have guided the market. We said, you know, essentially last year, you know, the new indication is going to double the potential for GAMI Fund. and admitting obviously that some of the patients have been treated you know where you know the borderlines between primary and secondary age and age uh can be blurred decisions have to make it physicians are making decisions uh yeah so so basically this part partially probably this this is already in but we think that you know that also for gummy front is in the next three to four years is very significant growth Why is there always a bit of risk of lumpiness on a quarterly basis, given the small number of patients overall? But the new indications is significantly increasing the potential for the product. So if you have a more midterm outlook, yes, it will propel the product to a larger level. I hope that gives you some flavor. Next question, maybe?

The next question comes from Alastair Campbell from RDC. Please go ahead.

Thanks for taking my question. I've got two, please, if possible. Just first of all, I wonder, Hendrik, if you give us a bit of thinking about medium-term R&D expenditure. It's obviously stepped up quite a bit in the last couple of years, partly after the CTR deal. But what should I be thinking about absolute R&D expenditure maybe for the next two, three periods? Is there much more to come in terms of an uplift there? And then secondly, I'm sure your business development teams are busy as ever. I just wonder if, you know, what you've experienced with CTI. Are there any learnings from that acquisition from the process there that you've taken forward to inform BD activity and your thoughts on future deals? Thank you.

Henry?

Should I start on the expenditure going forward? Well, you know that we talk a lot about upcoming launches in 2026 with both NASP and the nephrology for Esper Valley. When it comes to R&D, we also have these four priority development projects that we are running. So, you know, there will be R&D next year as well.

um for you know further more specific guidance on on margins we we will have to come back to that you know as we usually do yeah and then and then maybe with regard to the learnings from the cti deal you know we have now done uh 10 deals i think nine of them have have uh have been very successful Now there are some question marks with regard to the CTI deal. So what are the learnings? I mean, first of all, you know, you do these deals, we do these deals in a probabilistic approach. I mean, if you are the, if you can, and you know, then you make decisions because, you know, if you want to have 100% certainty, you know, that is unfortunately not part of the game, man. So where we have some learnings is clearly on the integration management side. I think it is where we could have done on hindsight, we could have institutionalized this better and let's have been more welcoming to some of the team members and should have probably getting our arms around them in a better way. And then from the deal perspective, You know, in hindsight, it looks like we underestimated the opportunities related to the GSK product. Yeah, it's true. They got a much broader label. We didn't see that coming. So, you know, you could say be mindful when you're thinking about conditional approvals. But, you know, it's pretty mute, you know, at the time, you know, that didn't look like a likely probability when we looked at this. So, you know, I think it is not belaboring it. You know, we have, yeah, we could have done a bit better, but there is a residual risk with all of these deals. We have probably learned in terms of vigilance a bit, but we also need to take some chances. And right now, I mean, I would also like to say that, you know, we are not singing sayonara to Wonjo by any respect. You know, we are investing into Paxos. We are working as we speak on label and guideline expansion. Once we can compete on equal footing, you see the... I think you see the competitive strength of Sobe. Right now, we have... It's just a bit harder. And then let's see what we can squeeze out internationally. So I think... Judge, you know, it's not satisfactory. I see this. But, you know, judge us. Give us a little bit more time. I know that in today's world that time is a luxury. But, you know, the case deserves a little bit more time. So there are some learnings, but it's not like, oh, you know, we can point to a complete blunder. All right. Thanks. Maybe we have a... You're welcome. Maybe another question.

The next question comes from Victor Sandberg from Nordea. Please go ahead.

Yes, hi. Thanks for taking my questions. I also have another one-year question. Yes, on the same topic, you mentioned some of the unforeseen events here for one year that we're now seeing in the numbers. So I don't know if you can answer this, but just wondering what risks should we have in our model for any kind of impairment on the CTI I mean, if it keeps underperforming in H2, we could see negative growth on this asset on a year-over-year basis, which I guess was not part of your forecast when you acquired the asset. So just wondering how we should view that risk of any kind of trigger event for any impairment. And also second one, perhaps on VEXA syndrome also. Even if prevalence numbers are out there, I guess very few receive actual treatment today. Do you have any data on how many patients are confirmed by UBA1 gene testing and have a confirmed vexus diagnosis and how much work do you expect you need to kind of create awareness of this product and have doctors start testing for UBA1 gene to formally diagnose patients? Thank you.

Thank you. You know, with regard to impairment, you know, I think, why don't we have this discussion, let's say, because, you know, the team is bullish to make the product grow on a year-to-date basis, yeah, during the course of this year. And, you know, they have been bullish before. Why this time? I mean, we made some changes. I think, you know, maybe we have some additional data give it a shot. I don't think it's the time to think about impairment. With regard to vexers, you know, we have set out some prevalence number. There is an increasing interest in this, and we know now of diagnostic companies who are offering these tests. So as the increase of of awareness of the disease is happening. And we have a few years till we sell this. This is going to be a very material indication. I mean, Julia, you want to add something on the medical awareness of the disease?

So we see, I think, two really indicators on how the interest in the scientific community is growing. On one end, it's the scientific community communications at Congresses, and we saw it at Euler before, and it's going to come as well as both ACR and ASH, how really the number of communications is exponentially increasing, and the sessions are extremely well attended. But the second thing that I would like to mention is that we have, you know, as I mentioned, we have started recruiting patients, And we hosted investigator meetings in different geographies, regional ones. And I think it was the first time in probably many, many years that I saw that everyone attending were all the principal investigators together with co-investigators. There is a huge interest with the feeling that really this is we're making history. Obviously, we are running the trial because we believe in the potential of Bongio. You never know, as I mentioned before, until you finalize the study. But what is clear is that there is a very high awareness, and it's really increasing. So, also talking to investigators, once they know how to diagnose, it's very easy to have access to this genetic testing. We know that we need to invest in medical affairs, in disease awareness, but it's also clear that there is a trend in the community and this really interest on this new disease because it's really compromising patients and having an alternative treatment option and a clinical trial that they can participate. It's really driving a lot of this interest.

Yeah, to your point, I mean, I don't have a data point on hand, but, you know, based on the reasoning and let's say, you know, there will be, there is no shortage of the number of patients. It's then obviously the identification and having a more, you know, standardized diagnosis program and protocol is helping. I think, you know, this will, if we have strong data in our study, we don't have to worry about finding enough patients. Right now, we know that there are quite a few patients, but this is below 1,000 that will get some form of treatment, but not approved. So I think the opportunity is very material for us, and we just want to give it a go, but the risks... There's still obviously risk involved, but as Julia said, the ground is fertile. If you have strong data, I think what you will see is already that the product will get used. Thank you. You're welcome. Next question.

The next question comes from Kirstie Roth-Stewart from BNP. Please go ahead. Hi there.

Thank you for taking my questions. Kirstie Roth-Stewart from BNP Paribas. So just on your expectations for NASP, given that your submission is now completed, I was hoping you could talk to kind of your launch expectations for the product and also your longer-term confidence in exceeding the 500 million peak sales number that you've previously mentioned, especially in light of Cristexa, which is already doing over a billion. Or would you point to kind of some elements of differentiation in terms of maybe your expectations for positioning and uptake of the product? And then hopefully time for a quick last one, just on doptillate dynamics for the rest of the year. I understand that it's not that simple to switch from doptillate onto a generic or promactor. The currently treated population are relatively protected, but could you clarify that that's the correct understanding? And it would also be great if you could touch on your expectations to kind of grow this product beyond the 1.2 billion run rate you've seen over the last few quarters in light of the generic entry for promactor. Thank you very much.

Yeah, thank you. I mean, we, you know, with regard to NASP, you know, the, I mean, we believe that we have a unique proposition. They, you know, CRUS-Tex, Amgen with CRUS-Tex have around six, six and a half thousand patients. We believe based on market research that we conducted that around 15,000 patients would be eligible for peculated uricase therapy. As you know that Equisexa is mostly prescribed in conjunction with methotrexate, which then has some shortfalls and is contraindicated for a number of patients of this community. So what we would expect is a relatively quick uptake in patients that would be eligible for therapy, but currently cannot take it in conjunction with methotrexate. and have an immunogenicity issue. And so the hope is that we can position NASP very well in those patients. We also think that the competitive profile, given the dose advantage, is quite robust based on market research. So we think that we can attract some patients to switch. But there's a significant large blue ocean And with regard to uptake, I think we will do further studies. But, you know, we think that there is a group where we can penetrate quite quickly. And then, you know, we'll provide guidance at a further stage, particularly when we have got approval or when we know when approval has been given. With regard to Doctylid, we think the product is well differentiated. So with regard to the generic entry for Promecta, We think that in the United States that we can still hold on to the product, keep growing. Growth, obviously, then forward-looking will become more difficult, but we think that we can defend our patients. And then, obviously, then we have international end and also European market where there's you know, where we can keep driving the product. And, you know, we have exponential growth in those markets. So we think that the product, you know, and by the way, I mean, what we set out for Dr. Lit was, you know, excluding the CIT indication at the time that didn't come. We basically gave guidance to 500, and if you extrapolate where we are today and give us some credit for growth, then... Maybe this 500 million is not so far away. And so we believe that the product is meeting expectations for us. And we obviously took into consideration that there's going to be a generic with Promecta, but we think that the product profile will hold. Good. I think we are now, maybe we have room for one more question.

We have a follow-up question from Christopher Ude from SCB. Please go ahead.

Thanks very much for taking my follow-up question. I'll keep it very short. Could you just tell us what the bar for success is in your view for GammaFendt and IDS for the Phase II? Thank you.

Louis, do you want to comment?

Yes. So, as you know, this is a proof-of-concept study. meaning that we cannot do the standard calculations for the probability of success. We know that 20% of patients with sepsis probably is driven by this increased interferon gamma, so there is a very strong rationale, and that's why we have entered in this research collaboration to explore. But from there, to give you a specific perspective target number of what's the probability of success, that's something that we cannot do at this stage. No, yeah.

Sorry, that's actually not what I meant. I meant more in terms of what would you be looking to see, you know, in terms of driving a go, no-go decision on phase three. Sorry about that.

Ah, okay. So we're looking at the data in terms of how patients will respond. Obviously, the first thing is the safety, and that's what we're looking at. making sure that gamifan is not being detrimental for this group of very severe patients. And once we have established the safety profile and the dose response, then that's what is going to drive our goal-no-goal decision, making sure that there is no harm for patients and that we see a dose proportionality in the response so that then we can plan for the next phase-to-be program.

Thanks so much.

Yeah, thank you. Yeah, maybe we round it off, you know, given that our time is up. You know, when you think about, you know, us, I mean, 22% growth, you know, and 34% EBITDA at this point of time, we thought that, you know, this is more a reason to celebrate than anything else. But, you know, we understand the concerns. But I think when you look at the overall performance of the companies in a very robust state, we are delivering in our key milestones and we are taking action to be very competitive to take advantage of the opportunities ahead of us. And so we believe at least that industrially, even though we may have not done the perfect job to explain it, that industrially we do the right job to build this company for success. So I look forward to further interactions, and if you have further questions, please let us know, and we're happy to answer them. And thanks for your interest. Have a great day. Thank you.

Ladies and gentlemen, the conference is now over. Thank you for choosing Coruscall, and thank you for participating in the conference. You may now disconnect your lines. Goodbye.