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.
Yes, 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. 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 Abad-Franch, Head of R&D and Chief Medical Officer. We plan to review the presentation first. Then have a Q&A until around 3:00 P.M. CET. For those on the phone, please join the queue. For questions, as mentioned, please press star one. 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.
The second quarter, when you elevate yourself, was really for us about. Strong economics. Drive important pipeline milestones, and create economic preconditions to support our future growth ambitions. With regard to the first subject, strong economics, we delivered SEK 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 is accounting now for 55% of total revenues and grew impressively 65%. This supports our quest of transforming Sobi into the next phase. The growth was driven by Altuviiio, Doptelet, Aspaveli, and Gamifant. Vonjo 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 Gamifant in HLH, MAS in Still's disease by FDA, marking a significant expansion of our potential market. Because we were able to complete the filing for NAS, this is the FDA, in an uncontrolled gout. We were also able to present very strong 52-week data for Aspaveli 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 Apellis. 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. We are very confident about our business also growing this into the future. 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. Let's turn to slide number five. Let's look at the overall performance in more detail. We saw Sobi's continued performance in hemophilia with strong 27% growth, and the combined hemophilia franchise growing at 24%, portraying a robust growth of Altuviiio in hemophilia A. Immunology has grown at 11% by strong performance of Gamifant and Kineret. Geographically, Europe's growth of 21% has been propelled primarily by Altuviiio and Doptelet during the second quarter. North America and international have been contributing growth of 16% and 44%, respectively. The growth of North America is primarily driven by Doptelet and Gamifant. 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 Altuviiio launch.
Within the hemophilia A franchise, we have demonstrated growth of 32%. We believe that this is really just the beginning for Altuviiio because the growth in the first, let's say, two quarters was really primarily driven by three markets. We have now 17 markets on stream, but most of these markets are at a very early stage, so their best is still to come. Doptelet showed continued strong demand across all regions, including the U.S., with 43% growth in 2024. This highlights a 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 PNH space by orals. 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. Vonjo.
Experienced a 4% decline. While demand was increasing significantly versus Q2 last year, the decline is attributed to gross-to-net adjustments, which we are closely monitoring. Gamifant and Kineret continue to show good demand in the U.S. Gamifant 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, Altuviiio. Let us turn now our attention to the continued success of Altuviiio. In the second quarter, hemophilia A, as reported, of Altuviiio and Elocta grew impressively 32%. This growth is a testament to the strength of our portfolio and the successful launch of Altuviiio. Focusing on Altuviiio specifically, the product achieved sales of SEK 627 million in Q2, reflecting a strong growth momentum. Launches are progressing well across Europe and the Middle East.
As mentioned, we have now 17 countries on stream, whilst the actual sales are really primarily driven at this juncture by Germany, Switzerland, and Spain. We are also excited to announce a full launch in the U.K. this July, which we anticipate will further drive adoption. Overall, the launch of Altuviiio 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, Aspaveli. Let's now review the latest updates on Aspaveli, starting with PNH. 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 variant 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. Lydia will cover the results in more detail in her presentation. Additionally, we have made a significant update to our royalty agreement with Apellis. 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 upfronts by having lower royalties both from a cash flow perspective on our P&L moving forward. This deal reaffirms our commitment to our ongoing partnership with Apellis and our belief in the product's potential to deliver significant benefits to patients and to Sobi's long-term growth. Let's turn to Vonjo, where we continue to see a positive momentum despite external challenges. Demand for Vonjo increased quarter on quarter, reflecting its growing adoption. However, this growth was outweighed by the continued impact of healthcare reforms, primarily to mention here the reform in Medicare and the 340B scheme. In patients with below 50,000 platelets, Vonjo has achieved a significant market share, underscoring its strong position in this patient population. Looking ahead, our strategic focus for Vonjo 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 50-100,000 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 broaden the product on a global scale. Finally, we are exploring new indications. In this context, we need to mention the PACS study where we start to enroll. These initiatives reflect our commitment to maximizing Vonjo's long-term potential and delivering meaningful benefits to patients across a range of indications. Let's turn to slide number 10, Gamifant. I'm thrilled to share a significant milestone for Gamifant and the patients we serve.
The FDA approved Gamifant as the first-ever treatment for adults and children with macrophage activation syndrome in Still's 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 Gamifant's potential to transform the lives of patients with HLH and MAS in Still's 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 Gamifant 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 Gamifant and secondary HLH. Aspaveli is on track, and a CHMP opinion is expected by end of year. Four innovative development programs are on track with first patient in achieved for the PACS study in VEXAS 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. Now I'd like to hand over to Henrik, our CFO.
Thank you, Guido. Hello, everyone. Please turn to slide 13, and we will take a look at some key financial metrics for the quarter.
In Q2, our revenues of SEK 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 Altuviiio, as well as Doptelet and Gamifant. In addition to strong revenue growth, we delivered an improved Q2 adjusted EBITDA margin of 34% compared to 28% last year. If we look at the bar chart on the left with revenues by quarter and business area, we see a solid growth in hematology of 27% in the quarter at CER. Including our hemophilia A products, Elocta and Altuviiio, increasing by 32% at CER. We continue to see strong momentum for Altuviiio in the markets where we have launched to date.
In addition, we've continued strong growth in Doptelet of 43% at CER due to growth both in the U.S. and ex-U.S., as well as with Aspaveli, driven in large part by our international region. Vonjo demand decreased quarter on quarter but was negatively impacted by gross-to-net adjustments, mainly related to Medicare redesign. Immunology grew at 11% in the quarter due to double-digit growth in Kineret and our highest quarter of sales to date in Gamifant. Sales in our international region was another positive for Gamifant. As a reminder, Beyfortus sales and the royalties we received 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 product and country mix effects, offset by product's return. In synergies. Looking at the operating expenses for the quarter, we observe 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 Altuviiio, Aspaveli, and Nephrology, and NASP. 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. This was partially offset by post-approval development costs for Altuviiio and development costs for Gamifant and Vonjo. We are also investing in the four innovative programs for Vonjo in VEXAS syndrome and CMML, Altuviiio in Synovitis, and Gamifant in IDS. Operating cash flow for the quarter was SEK 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 SEK 11.4 billion, corresponding to a net debt-to-EBITDA ratio of 1.1 times. Please turn to slide 14. I would like to take a moment to address the restructuring charges of SEK 208 million recorded this quarter. You will find additional detail on page three of our report. This was a proactive and strategic decision aimed at positioning our organization for long-term success. As Guido mentioned earlier, we are in the 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. 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 % at CER and an adjusted EBITDA margin in the mid-30s % of revenue. The key considerations for our outlook have not changed since Q1. To start, the royalties we receive from Sanofi for Bay 42 sales remain the largest uncertainty both for revenue and EBITDA margin guidance.
While our view on Bay 42 is clearly positive, we do not control the outcome. 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 Altuviiio 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 Aspaveli 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.
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. I will now hand over to Lydia. Thank you.
Hello, everyone, and thank you, Henrik. We 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 Aspaveli, in June, we presented at ERA the 52-week data of the pivotal VALIANT study in C3G and ICMPGN. They showed 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 two study and a strategic assessment of the treatment landscape, we decided together with our partner, Apellis, to discontinue the development for Pegcetacoplan in this indication. In the phase two study, Pegcetacoplan demonstrated favorable safety and tolerability, consistent with its established safety profile. Moving to Gamifant, the FDA extended the indication for Gamifant to HLH MAS in Still's 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 Glucocorticoids. 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 Vonjo, the VEXAS syndrome proof-of-concept study called PACS 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. Lastly, we continue our focus on joint health for Altuviiio . Bleeding into joints is the major and most common clinical manifestation in hemophilia patients. Preventing them is the focus of Sobi's phase four activities for Altuviiio . The ALTITUDE study is a low-interventional study to understand how Altuviiio can prevent joint bleeds in the real-world setting, and we enrolled the first patient already in May. Next slide, please. We presented new Aspaveli sets of data from the VALIANT phase three pivotal study at the European Renal Association Congress in the late breaking session.
They included several new subgroup analyses 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 Aspaveli. I will present this on the next slide. Looking at the various patient groups, Aspaveli 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. Aspaveli 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 C3G and primary ICMPGN patients are currently treated with immunosuppressive therapies. On the third column, you see how Aspaveli is clearly efficacious across all parameters shown, regardless of whether patients are already on immunosuppressions or not. Finally, both C3G and primary ICMPGN 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 Aspaveli. Next slide, please. The effects I just described were sustained at one year.
Patients receiving Aspaveli for a year saw a proteinuria reduction of 67%. They continue 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 switched from placebo to Aspaveli at the start of the open-label period experienced a similar magnitude of benefit in proteinuria reduction and 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 NASP regulatory review, while we expect a decision for Doptelet in pediatric ITP very soon. In Europe, we anticipate the CHMP opinion on Aspaveli in nephrology by the end of this year. We also anticipate an EU decision for our newly partnered product, Olezarsen. In Japan, we plan to submit Gamifant in HLH MAS, as well as Aspaveli in C3G and primary ICMPGN. Furthermore, we anticipate the submission of Kineret in Still's disease and a decision on Doptelet in ITP. We are also planning to advance the clinical projects with a host of exciting developments. This includes the first phase two-way data on Gamifant 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 LOTIS-5 study of Zylonta 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. 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. I mean, they speak for themselves, and they demonstrate that the company has a very material momentum. Our R&D pipeline has shown tremendous progress. We have continued launch success with Altuviiio in Europe. As you have seen, I mean, three products, three countries are currently the main source of Altuviiio's growth. Seventeen have come on stream. U.K. comes on stream now in July.
That gives you a bit of a flavor that the best is still to come. We've got the approval of Gamifant for MAS. In the U.S., we have completed the filing of NASP in uncontrolled gout in the U.S. We look forward to complete the review of NASP with the FDA and Aspaveli 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 project. It was gratifying to see that we had the first patient in this quarter for Vonjo in the PACS study for the potential treatment of VEXAS. We are also making good progress with the IDS study. With all these activities, we are preparing the organization and ensuring and prioritizing that we are fit for purpose.
Also with the economic space that we have created, 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 Sobi business, as you have seen, in the 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. 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 one 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 Artiach from Danske Bank. Please go ahead.
Hi. Hello, everyone. Thank you for taking my questions. I have a couple of them. The first one is on your guidance. Does the lack of top-line guidance upgrade come only due to questions or uncertainty on B42's 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?
My second question, also related to guidance, is 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 Cell 212, which would imply, I would say, high OPEX already in H2 ahead of launch in early 2026. 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-2026, if I'm not wrong? Would it make sense to assume you would push SG&A to next year, which would raise your adjusted EBITDA margin for 2025 mechanically? Thank you.
Yeah, thank you. I mean, this is, let's put it this way. With regard to the guidance, we are not aware.
That products are under threat or under pressure that should make us more cautious about the prospects of our product beyond what we are disclosing, obviously, in the report. With regard to Beyfortus, I mean, the only thing I can say is that it's in the hands of Sanofi. They seem to be confident about the product. Not heard anything else. If they are confident, I have no reason to be not confident simply because they used to be the largest vaccine company in the world and probably are still in this prevention market now. There is nothing to it. It's more prudent at this stage, let's say, because there are so many elements, but clearly not related to our portfolio.
The EBITDA margin, it's true to say that when you believe in an influx of Beyfortus coming in in the second half and you have the effects, as we pointed out, that. It may be beneficial to have EBITDA margins. 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. We are understanding more of the competitive dynamics. We have a potential readout on IDS. For all of these reasons, you want us to spend some money. That's the reason why we are not as specific on the mechanics of the uplift of the guidance. Everything we are doing right now is pointing as positive, and we will reinforce those investments. We are not spending freely.
I hope that gives you a flavor for the company. Next question.
The next question comes from Mattias Häggblom from Handelsbanken. Please go ahead.
Yes. Good afternoon. Mattias Häggblom from Handelsbanken. Thanks so much for taking my questions, two please. Firstly, Roche presented data from a phase I/II study for its next-generation bispecific. The majority of patients developed antidrug 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. Secondly, with regards to Vonjo, I can't recall we spoke about the confirmatory PACIFICA trial for a long period of time. Since the trial's ongoing, this is reading out late 2026. 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 2026. That's it. Thanks so much.
Thank you. Maybe Lydia, direct to you.
Yeah. I'm really sorry. I couldn't hear. You mentioned an ISDH presentation of data, but I couldn't understand which asset did you talk about? I'm sorry.
Yeah. Sorry for being confusing here. Roche Biospecific 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. 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. It is something that we are monitoring and that we are cautious. We still believe that the future, as also at ISDH, and there are some publications coming up on really the normalization and the new class of factory therapies to really bring the patients to those very high levels of protection. To me, it is a little bit early to comment on, but we will obviously keep monitoring these developments.
Pacifica had an update and timelines when we should expect data from this confirmatory trial for Vonjo?
For Vonjo, the confirmatory trial, we are seeing a further acceleration coming from international markets. We are adding additional markets where we are seeing a lot of traction, and we are very.
Hopeful that we will be bringing the timelines even further ahead of what we originally planned. It will depend if this acceleration that we are seeing now keeps continuing. I think it would be better, as we meet on a quarterly basis, we will continue confirming if that acceleration track is continuing and if we see this acceleration materializing. So far, yeah, we are very happy with the new additional countries that we are including. Yeah, that's why we expect data now, probably most likely in 2027.
Thanks so much.
The next question comes from Christopher Uhde from SEB. Please go ahead.
Hi there. Thanks for taking my questions. Two, if I may, Altuviiio the first and then the reorganization the second. Altuviiio continues to impress, of course. How much of the sequential growth is Germany switched to Linden?
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? My second question is, the market seems to be interpreting the reorganization and maybe in combination with the Apellis renegotiation pretty negatively, as if you're, I don't know, looking for ways to squeeze more margin for something you had not 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? 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.
Yeah, thank you, Christopher. With regard to the launches, I mean, Germany, Switzerland, and Spain is clearly the vast majority of our business right now. North of 80%. Yeah.
That is the reason why the next phase of launches will obviously do well for the overall performance of Altuviiio also moving forward, yeah, because there were some questions. Also now the U.K. is obviously expanding that pool of patients pretty materially. We expect also France coming on stream in the second half at one stage. This is also not part of the 17 countries right now. It is a normal European rollout, and that will propel it. It is really primarily driven by those three countries where the Spanish market is smaller, probably also partially driven by the history with the plasma-derived product years ago, where unfortunate things happened. Let's say, the best is de facto still to come. It is these three countries that are driving it.
The other markets will pay more dividends in the second half and then in the quarters to come. 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 is a trimming exercise, as many companies are doing on a regular basis every four or five years. It is just good practice to make sure that you create space, then also for a new talent base. The organization is changing. The business is changing. So do we. That means that we need to adjust. It creates, obviously, the space. I mean, we mentioned earlier the NASP launch where we need new talents. We probably need to expand a bit more in the C3G launch in Europe.
Then we need to also have some readiness should the IDS data come out well in the fourth quarter. We plan for success. Hence, we have created some space. This is not in proportion. It is not really extraordinary. Maybe, Henrik, you want to comment also?
Yeah. No, I think you covered it. It is getting the priorities right simply. A normal trimming exercise that most companies do. Nothing to add, really.
Good. I hope this gave you a flavor, Christopher. Maybe we move to the next question.
The next question comes from Harry Gillis 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. Of course, there is the uncertainty with Sanofi sales of Beyfortus being.
A major cause of uncertainty in the rest of the year. Would it be fair to say that the rest of the business has exceeded your expectations in the first half of the year? My second question is on Gamifant. You've seen very solid growth in Q1 and Q2, above 30%. I was just wondering how much of that is off-label use in the secondary HLH indication versus the original label. 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, we clearly, we were hoping for it, but we probably didn't plan all of that in. It's a very solid growth. You take away the extraordinaries that we had also.
In Q1. It is a very consistent, very strong underlying performance driven by the new products. Altuviiio launch is exceptional. As I said, we do not see here a plateauing effect. We see it. We keep it. We are expecting it to keep growing. With Gamifant, if you can afford to have a more, not quarter-on-quarter perspective, but more of an annual perspective, and this is what we have guided the market, we said essentially last year. The new indication is going to double the potential for Gamifant. Admitting, obviously, that some of the patients have been treated where the borderlines between primary and secondary HLH can be blurred and decisions have to be made. Physicians are making decisions. Yeah. Basically, partially, probably this is already in, but we think that.
Also for Gamifant, in the next three to four years, there's very significant growth, while there's always a bit of risk of lumpiness on a quarterly basis given the small number of patients overall. Yeah. But the new indications are significantly increasing the potential for the product. If you have a more mid-term outlook, yes, it will propel the product to a larger level. I hope this gives you some flavor. Next question, maybe?
The next question comes from Alistair Campbell from RBC. Please go ahead.
Thanks for taking my question. I've got two, please, if possible. Just first of all, I wonder, Henrik, if you give us a bit of thinking about medium-term R&D expenditures. Obviously, stepped up quite a bit in the last couple of years, partly after the CTI deal.
What should I be thinking about absolute R&D expenditure maybe for the next sort of two, three periods? Is there much more to come in terms of an uplift there? Secondly, Guido, I'm sure your business development teams are busy as ever. I just wonder if 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.
Henrik?
Should I start on the expenditure going forward? You know that we talk a lot about upcoming launches in 2026 with both NASP and the nephrology for Aspaveli. When it comes to R&D, we also have these four priority development projects that we are running. There will be R&D next year as well.
For further, more specific guidance on margins, we will have to come back to that. As we usually do.
Yeah. And then maybe with regard to the learnings from the CTI year. We have now done 10 deals. I think nine of them 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 do these deals. We do these deals with a probabilistic approach. I mean, if you are the, if you can, and then you make decisions because if you want to have 100% certainty, that is unfortunately not part of the game. Where we have some learnings is clearly on the integration management side. I think it is there we could have done, on hindsight.
We could have institutionalized this better and let's be more welcoming to some of the team members and have been. Should have probably. Getting our arms around them in a better way. Then from the deal perspective. Yet on hindsight, it looks like we underestimated the opportunities related to the GSK product. Yeah. It's true. They got a much broader label. We did not see that coming. You could say be mindful when you're thinking about conditional approvals. It is pretty mute at the time. That did not look like a likely probability when we looked at this. I think it is not belaboring it. 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.
Right now, I mean, I would also like to say that we are not singing Sayonara to Vonjo by any respect. We are investing into PACS. We are working as we speak on label and guideline expansion. Once we can compete on equal footing, I think you see the competitive strength of Sobi. Right now, it's just a bit harder. Let's see what we can squeeze out internationally. I think, judge, it's not satisfactory. I see this. But judge us, give us a little bit more time. I know that in today's world, time is a luxury, but the case deserves a little bit more time. Yeah. There are some learnings, but it's not like, "Oh, we can point to a complete blunder." Yeah. All right.
Thanks.
Yeah. Maybe we have a— You're welcome. Yeah. Maybe another question.
The next question comes from Viktor Sundberg from Nordea. Please go ahead.
Yes. Hi. Thanks for taking my questions. I also have another Vonjo question. Yeah. Just on the same topic, you mentioned some of the unforeseen events here for Vonjo that we're now seeing in the numbers. 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 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. Just wondering how we should view that risk of any kind of trigger event for any impairment. Also a second one, perhaps on VEXAS 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 VEXAS diagnosis? 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. I mean, with regard to impairment, I think. Why don't we have this discussion. Let's say, because the team is bullish to make the product grow on a year-to-date basis, yeah, during the course of this year. They have been bullish before. Why this time? I mean, we made some changes. I think maybe we have some additional data. Give it a shot. I don't think it's a time to think about impairment. With regard to VEXAS. 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. As the increase 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, Lydia, you want to add something on the medical awareness of the disease?
We see, I think, two really indicators on how the interest in the scientific community is growing. On one end, it is the scientific communications at congresses. We saw it at EULAR before, and it is going to come as well at both ACR and ASH, how really the number of communications is exponentially increasing and the sessions are extremely well attended. The second thing that I would like to mention is that we have.
As I mentioned, we have started recruiting patients, and we hosted investigator meetings in different geographies. Regional ones. 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 we are making history. Obviously, we are running the trial because we believe in the potential of Vonjo. You never know, as I mentioned before, until you finalize the study. What is clear is that there is a very high awareness, and it is really increasing. Also, talking to investigators, once they know how to diagnose, it is very easy to have access to this genetic testing. We know that we need to invest in medical affairs, in disease awareness, but it is 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 see a I don't have a data point on hand, but based on the reasoning and let's say there will be there is no shortage of the number of patients. It's then obviously the identification and having a more standardized diagnosis program and protocol is helping. I think this will, if we have strong data in our study. We will not have to we don't have to worry about finding enough that there are 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. Yeah, but not approved. I think the opportunity is very material for us, and we just want to give it a go. There is still obviously risk involved. As Lydia said, the ground is fertile. We will, as soon as if we have strong data, but I think what you will see is already that the product will get used. Yeah.
Okay. Thank you.
You're welcome. Next question.
The next question comes from Kirsty Ross-Stewart from BNP . Please go ahead.
Hi there. Thank you for taking my questions, Kirsty Ross-Stewart from BNP Paribas.
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 Krystexxa, which is already doing over $1 billion. Would you point to kind of some elements of differentiation in terms of maybe your expectations for positioning and uptake of the product? Hopefully time for a quick last one just on Doptelet dynamics for the rest of the year. I understand that it's not that simple to switch from Doptelet onto a generic or Promacta to the currently treated population are relatively protected, but could you clarify that that's the correct understanding?
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 Promacta. Thank you very much.
Yeah. Thank you. I mean, with regard to NASP, I mean, we believe that we have a unique proposition. Amgen with Krystexxa have around 6,000-6,500 patients. We believed based on market research that we conducted that around 15,000 patients would be eligible for pegylated uricase therapy. As you know, Krystexxa is mostly prescribed in conjunction with methotrexate, which then has some shortfalls and is contraindicated for a number of patients of this community. What we would expect is a relatively quick uptake in patients that would be eligible for therapy.
Currently, cannot take it in conjunction with methotrexate and have an immunogenicity issue. 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. We think that we can attract some patients to switch, but there is a significant large blue ocean. With regard to uptake, I think we will do further studies. We think that there is a group where we can penetrate quite quickly, and then 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 Doptelet, we think the product is well differentiated. With regard to the generic entry for Promacta, we think that.
In the U.S. that we can still hold on to the product, keep it growing. Growth, obviously, then forward-looking will become more difficult, but we think that we can defend our patients. Let's say, obviously, then we have international and also Europe might be a market where there's, where we can keep driving the product, and we have exponential growth in those markets. We think that the product, and by the way, I mean, what we set out for Doptelet was, excluding the CIT indication at the time that didn't come, we basically gave guidance to $500 million, and if you extrapolate where we are today and you give us some credit for growth, then maybe this $500 million is not so far away. Yeah. We believe that the product is meeting expectations for us.
We obviously took into consideration that there's going to be a generic with Promacta, but we think that the product profile will hold. I think we are now. Maybe we have room for one more question.
We have a follow-up question from Christopher Uhde from SEB. 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 Gamifant and IDS for the phase two? Thank you.
Lydia, do you want to comment?
Yes. 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 increase in interferon gamma. There is a very strong rationale, and that's why we have.
Entered in this research collaboration to explore. From there, to give you a specific target number of what's the probability of success, that's something that we cannot do at this stage.
Oh, yeah. Sorry. That's actually not what I meant. I meant more in terms of what would you be looking to see in terms of driving a go, no-go decision on phase three. Sorry about that.
Okay. 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 are looking at, making sure that Gamifant is not being detrimental for this group of very severe patients.
Once we have established the safety profile and the dose response, then we will, that's what is going to drive our go, no-go 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 two B program.
Thanks so much.
Yeah. Thank you. Yeah. Maybe I'll be rounded off given that our time is up. When you think about us, I mean, 22% growers and 34% EBITDA at this point of time, we thought that this is more a reason to celebrate than anything else. We understand the concerns.
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. 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. I look forward to further interactions. If you have further questions, please let us know, and I'm happy to answer them. Yeah. Thanks for your interest. Have a great day. Yeah. Thank you.
Ladies and gentlemen, the conference is now over. Thank you for choosing Chorus Call, and thank you for participating in the conference. You may now disconnect your lines. Goodbye.