Ladies and gentlemen, welcome to the Sobi Q4 2024 Report Conference Call and Live Webcast. I'm Sandra, the Chorus Call operator. I would like to remind you that all participants will be in a listen-only mode. And the conference is being recorded. The presentation will be followed by a Q&A session. [Operator Instructions] 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, sir.

Yes. Thank you. Hello, everyone. This is Guido Oelkers, CEO of Sobi. We are delighted to welcome you to the fourth quarter and the full-year 2024 conference call for investors and analysts. We posted, as mentioned, this presentation to sobi.com earlier today. Let's move to Slide 2. Forward-looking statement as per usual, unless otherwise stated, we will be making comments that mostly relate to the third quarter at constant – to the fourth quarter at constant exchange rate in million Swedish krona. Today, we plan to cover the key aspects of the Q4 report. I'm joined by Henrik Stenqvist, our CFO; Lydia Abad-Franch, our Head of R&D and Chief Medical Officer. We plan to review the presentation first and then have Q&A until around 3

30 Swedish time or Central European Time. [Operator Instructions] Let me propose that you ask only one or two questions at a time. Let's move to the [indiscernible]. This is our overview on the performance of Q4. We are very pleased with the performance of the portfolio in the fourth quarter and overall for the full-year. The full-year was announced a few weeks ago, our internal expectations with SEK26 billion in revenues, representing 19% growth at CER and the full-year adjusted EBITDA was 36%. In the quarter, we saw growth at 8%, and adjusted EBITDA margin was 34%. This reflects a very strong performance across the portfolio. Please bear in mind that the RSV impact by Beyfortus had very strong quarters in Q3 and Q4, Synagis in Q4 2023 was also extremely significant. Hence, there was a negative variation. The underlying business in Q4, excluding RSV has been growing over 20%. Haematology growth was driven by continued growth of Doptelet, Aspaveli and the launch of Altuvoct. There has been a strong interest in Altuvoct and just the first half of this year on generating SEK300 million in the quarter. We continue to build the launch of Vonjo and see consistent quarter-on-quarter progress, and we are continuing to build awareness of product and the unmet medical need in myelofibrosis treatment. In Immunology, it is reflected very substantial. Obviously, as mentioned Beyfortus royalty income involved in Q3 and Q4. In terms of the pipeline, our progress continues with the filing of Gamifant in the U.S. for HLH/MAS in Still's disease and we are happy to announce that we have also submitted Aspaveli in Europe for C3G and IC-MPGN. We look forward to continuing the discussion with health authorities and bringing these important medicines to patients in these disease areas. We have very strong momentum, as you can see in the Sobi business, both commercially and in development. And 2025 will be a busy year with multiple filings ongoing, preparing the organization for a number of new launches, whilst we bring Sobi to the next level. With that in mind, our outlook for 2025 is that we expect revenue growth at highly single-digit, high single-digit at CER and adjusted EBITDA margin at mid-30s, reflecting the investment that is necessary. Let's move over to the overall business growth. Let's look at – and this is the next slide. Let's look at the overall performance in more detail. Can you move to the next slide, yes. Our growth of 8% in the quarter was driven primarily by Haematology sales, while strong Beyfortus royalty income was not able to offset Synagis negative evolution. We saw a very strong performance in Haematology with 22%. Overall the full-year, all areas delivered strong performance with Haematology with 24% and Immunology, 11%. In the second half of the year, the U.S. business was impacted by the loss of Synagis, as already mentioned. When Gamifant and Kineret have grown strongly throughout the year. Europe saw a strong growth of 14% for the full-year, reflecting the launch of Altuvoct and other strategic growth products. In the U.S., we saw, as I mentioned earlier, the impact of Synagis. And International is continuing its robust growth with 14% in 2024, while if you take away the Fosun effect, it has grown actually at 43%. When you think about the composition of our portfolio, regional portfolio, it just shows that our internationalization strategy that we laid out in 2017 is now showing the fruits when you just think about the composition of North America, including the Beyfortus royalty and this very dynamically growing international business. Let's move to the next slide. I go a little bit more deeper. Focusing a bit more on the portfolio in 2024, we have seen a strong momentum across the portfolio with already significant contributions from the Altuvoct launch. We are starting to see an impact on Elocta, as expected from Altuvoct. However, we have seen 30% growth across the Haemophilia franchise in 2024. Doptelet showed continuous growing demand across all regions with 58% growth in 2024 and Aspaveli continued to grow double-digit in 2024 with 76% growth. We do expect increasing competitive pressure from orals in 2025. But in those markets where we have seen this – we are facing this competition the product has shown good perseverance in these markets so far. Vonjo continues sequential quarter-on-quarter growth, and I will comment in more detail. The Kineret grew at an impressive 24% in Q4, driven by growing interest in the IL-1 mechanism in multiple settings, some phasing and some gross to net adjustments. For Gamifant, we are happy to have filed in secondary HLH indication. And we see – and this will mean obviously an inflection point for the product as it will significantly broaden the overall potential for the product. Beyfortus continued its strong demand as seen in Q3, with Q3 and Q4, reflecting the peak of the RSV season. Let's move to the next slide. And on the next slide, you can see our portfolio approach of today's and tomorrow's breadwinners. Whilst the foundation business is very resilient and has shown 6% growth. This used to be essentially the DNA of Sobi. Today, it is 50% of revenues while the other half is on the right hand, where you have a nice blend of catalysts that allow us the transformation of Sobi and investing into our pipeline and into our strategic growth product and the new royalty streams from Beyfortus and also Altuviiio to help on this in this regard. And when you look at the strategic growth products, they have grown very substantially. So they are 35% today, including the royalty stream is basically half of this portfolio. And in this half was 55% in Q4. And then we have in the left [indiscernible] and this is, for us, obviously super important as we have very significant products that will determine the future of Sobi. So we received fast track designation for Gamifant in secondary HLH in Q2, and we have now submitted a filing package as indicated in December. For SEL-212, we are continuing the submission to the U.S. FDA, and we will complete the filing before Q2. In Q3, we had the positive readout of Aspaveli nephrology, and we have now submitted the filing to EMA and look forward to receiving the CHMP decision later during the year. At the same time, we continue building on our geographic footprint with the launches of Aspaveli, Doptelet, Zynlonta, Gamifant and Vonjo. This puts Sobi into a new phase of sustained growth, driven by new products, execution on our development commitments, and we have an exciting launch in progress with Altuvoct and 2 substantial indication in our hands with Aspaveli in nephrology and NASP in chronic refractory gout, which will be – which we will now prepare in 2025. Let's move to Altuvoct. We are very pleased with our first half year on the market with Altuvoct. Altuvoct has seen a rapid adoption in Germany and our market in haemophilia A in Germany has grown for Elocta and Altuvoct by 15 percentage points, essentially in the first 5.5 months after launch. Our haemophilia A sales grew as a consequence in Q4 at 9%. To date, in Germany, we have seen around 50% of the switches from Elocta while the others are coming from competitive products. And the last three months clearly favor that we are taking significant share – more share from competition. We look forward to bringing this important medicine or to more markets in Europe and other Sobi territories in 2025. Let's move to Aspaveli. As you have seen, we had very strong sales evolution of Aspaveli in PNH. However, the main part of this franchise future is clearly related to nephrology. And we are very pleased to have submitted the filing package for nephrology of Aspaveli in EU after an impressive readout in 2024. The combination of very significant reduction in proteinuria clearance of C3 staining from the kidney and in a high percentage of patients and the stabilization of eGFR at such an early time makes about the potential of this medicine that we were bringing to patients. But for us, this product is really right at the heart of what Sobi is about, given the fact that many of those patients are kids and to prevent them from progressing to end-stage renal disease, we feel is an obligation and mission. We look forward to working with the regulatory authorities to move this forward in parallel we were building the internal infrastructure in nephrology and prepare for the launch. We believe that Aspaveli has the potential to become a blockbuster product for Sobi. Let's move to Vonjo. Vonjo sales were SEK416 million in the quarter with 27% growth at constant currency, and 6% quarter-on-quarter growth, showing continued momentum with the product. Growth is primarily coming from the labeled below 50,000 platelet population, and we are continuing to work to expand the use according to the NCCN guidelines in myelofibrosis. We have recently hired a new U.S. Chief Medical Officer, Jamie Freedman with a hem-onc specialist who is leading our medical organization in the U.S. and we're expecting for further impulses from him to help us to expand the product. We are continuing to roll out our strategy to increase awareness of the product and the unmet medical need with the NCCN guidelines and recommendations. The second pillar of our strategy is to bring patients outside of the U.S. this year and we will launch in selected markets, which will follow the FDA label and ultimately want to bring it to Europe and Japan. And Japan, once we have the PACIFICA trial concluded. The next step is to broaden the use, and we have initiated a Phase II study in VEXAS, an area with currently no approved treatment, which Lydia is going to come in her presentation. And we have also made an important strategic step to form a research collaboration to get CMML on label. And this is also another demonstration that we stand by the product. We want to further develop the product and bring it to the level that we earlier indicated when we consummated the transaction. On this note, I'd like to hand over now to our CFO, Henrik Stenqvist.

Thank you, Guido, and hello, everyone. Please turn to Slide 12. We will now take a look at some key financial metrics for the quarter. So in Q4, our revenues of SEK7.4 billion meant that we exceeded SEK7 billion of revenue in an individual quarter for the first time, while also making significant progress on our pipeline assets. Revenue growth for the quarter was 8% at constant currencies, and we delivered an adjusted EBITDA margin of 34%. For the full-year 2024, we achieved revenues of SEK26 billion, corresponding to a growth of 19%, as outlined in our earlier announcement, as well as an EBITDA margin of 36%, in line with the guidance of adjusted EBITDA in the mid-30s. So if we look at the bar on the left, with revenues by quarter and business area, we see the solid growth in Haematology of 22% in the quarter, driven by all of Doptelet Haemophilia, Vonjo and Aspaveli. On the other hand, Immunology experienced a decline of 12% in the quarter as a result of the much lower sales of Synagis and the sales of Beyfortus royalties this year with a clearly larger share of royalties in Q3 than in Q4. Referring back to the table on the right, the adjusted gross margin of 78% in the quarter compared to 80% in Q4 2023, royalties in general were net positive to our gross margin in the quarter, but offsetting this was the loss of Synagis sales. Synagis was a high-margin product with significant sales in Q4 2023. Gross margin was also negatively impacted by other product mix items but to the lesser degree. The adjusted EBITDA margin reached 34% compared to 38% last year, a drop of 4 percentage points was due to 2 percentage points lower gross margin, as already discussed and higher OpEx driven by our investments in pipeline and launch products. Looking at the operating expenses for the quarter. We observed a 11% growth at CER compared to the same period in 2023. SG&A excluding non-recurring items and amortization increased by 10% at CER in the quarter, driven by launch and prelaunch costs for Altuvoct, Aspaveli in nephrology, NASP and Vonjo. We've managed to partly offset the impact of these activities on the SG&A line through savings in the Synagis organization as we have reported in prior quarters. R&D expenses increased by 13% at CER, excluding nonrecurring items, mainly due to post-approval activities for Altuvoct and development programs in Gamifant, Vonjo and Zynlonta. The operating cash flow for the quarter increased by 67% to SEK1.8 billion, benefiting from less capital tied up in receivables from Synagis than a year-ago. And if we turn to Slide 13, we see on the right-hand side of the slide that net debt went from SEK16.9 billion at the beginning of the quarter to SEK15.2 billion corresponding to a net debt-to-EBITDA ratio of 1.6x. And to the left, we see the debt reduction or more than SEK4 billion during the year and the leverage journey from 2.5x to 1.6x during the same period, driven by the improvement in EBITDA and strong cash flow. Please turn to Slide 14. And we will now discuss the financial outlook for the full-year 2025. And as usual, this outlook is based on revenue growth at constant exchange rates and adjusted EBITDA margin. For the full-year 2025, we anticipate revenue to grow by high single-digit percentage at CER and an adjusted EBITDA margin in the mid-30s percentage of revenue. On the revenue guidance, we look forward to continuing to launch Altuvoct into new markets in 2025, growing the haemophilia A franchise as well as continued progress with our existing commercial portfolio. We expect a continued strong contribution from Beyfortus royalties, although this is a factor where we do not control the outcome ourselves. In regards to our EBITDA margin guidance, we will continue to invest in our prelaunch assets, specifically NASP and Aspaveli in nephrology. In R&D, we will also continue with post-approval studies for Altuvoct and the new studies for Vonjo in VEXAS and CMML as well as major registrational efforts for Aspaveli, Gamifant and NASP. And with the outlook covered, I will now hand over to Lydia. Thank you.

Thank you, Henrik, and hello, everyone. So we start with the pipeline milestones on the next slide, please. We kept advancing our pipeline projects hitting important milestones in the fourth quarter. The very positive Phase III data for Aspaveli in C3G and IC-MPGN was presented at the ASN Kidney Week in October as an oral presentation during the High-Impact Clinical Trial session. And very importantly, as you have heard, based on this data, we submitted the EU application for a label extension to EMA this week. We are also launching a Phase II clinical trial for Vonjo in VEXAS syndrome after receiving the IND from FDA, and I will come back to this in a minute. We also submitted the Gamifant supplemental BLA to FDA for the treatment in HLH/MAS in Still's disease. And in Zynlonta, LOTIS-5 study is now fully recruited. With this Phase III study, we are confirming the conditional EU approval, and we plan to extend the indication from third-line therapy to second line in combination with rituximab. All of these show our ambition to drive innovation for patients and extend the reach of our medicines. Next slide, please. In December, we submitted a supplemental biologic license application for Gamifant for the indication of HLH macrophage activation syndrome in Still's disease to FDA. HLH/MAS, a form of secondary HLH is a rare systemic autoinflammatory disorder for which there is currently no approved therapy. It is a life-threatening complication of a Still's disease where patients can experience intensive systemic hyperinflammation and even multiple organ failure. Emapalumab specializes in interferon-gamma and blocks the expression of inflammatory cytokines, which drives the hyperinflammation of HLH/MAS. The application is based on the pool analysis from 2 clinical trials presented at ACR in November which show that emapalumab rapidly controls signs and symptoms of HLH/MAS. 85% of patients achieved complete response at any time during the study and the median time to achieve an overall response was below three weeks. These 2 clinical trials, which evaluated emapalumab are the first completed prospective clinical trials in this rare disease patient population. With the potential U.S. approval later this year, we hope to provide patients with HLH/MAS in Still's disease, the first approved medication for this potential fatal condition. Next slide, please. Thank you. And speaking of first, we are launching the first randomized clinical trial in VEXAS. PAXIS will be a randomized Phase II study to assess the safety and efficacy of Vonjo in disease, which currently has no approved therapy. An IND to study this indication was granted in December, and we are planning to start activating sites soon. VEXAS syndrome is caused by an acquired gene mutation and manifest in a wide range of auto inflammatory symptoms across the entire body. It has first been described in 2020 and therefore has a potentially high number of undiagnosed patients. The causative mutation has a prevalence of approximately 1 in 4,000 men's over age 50 in the United States. There is no standard treatment available for patients with VEXAS syndrome, though may require high dose steroids to control their inflammation. While JAK inhibitors and cytokine blockers offer some efficacy, they do not target route inflammatory pathways, and they may even worsen cytokines. Pacritinib is a JAK2 inhibitor that additionally targets NF-kappaB mediated inflammation via IRAK1 an inflammatory anemia via ACVR1. Due to this unique profile, pacritinib could be both more effective and safer than other therapies that have been used to treat VEXAS. Next slide, please. We are very proud of what we have achieved in 2024 hitting major milestones. Outlook was approved by EMA with a strong positive reaction by physicians and patients. Aspaveli's EU label in PNH was expanded to first line, and we got very promising data in C3G and IC-MPGN. We continue to grow Doptelet's reach in region and international. We submitted the supplementary BLA for Gamifant, HLH/MAS to FDA. We initiated the FDA enrolling submission for NASP and Kineret is another good example of our life cycle management and geographic expansion. Next slide, please. Looking ahead, we will be busy with the submissions to the major regulatory agencies, FDA, EMA and PMDA for our key assets, Gamifant, Aspaveli and NASP. In the U.S., the focus will be on the approval of Gamifant for HLH/MAS in Still's disease and on finalizing the NASP rolling submission for chronic refractory gout data. In Europe, the focus will be on the approval of Aspaveli in the nephrology indications, C3G and IC-MPGN. And in Japan, we will be submitting three dossiers, Aspaveli for the nephrology indications, Gamifant for HLH/MAS in Still's disease and Kineret for Still's disease. And with that, I would like to hand back to Guido. Thank you very much.

Yes. Thank you, Lydia. As you can sense, we are very pleased with Sobi's development in 2024. We saw a significant topline growth of 19%, driven by positively – driven positively by growth in our portfolio, particularly related to our strategic growth portfolio that has more at 87% in 2024. Our R&D pipeline has shown tremendous progress with the approval and successful launch of Altuvoct in Europe, the filing initiation of NASP in chronic refractory gout in the U.S. And in Q4, the filing of Gamifant in the U.S. and secondary HLH continued in general – in February by our filing of Aspaveli in nephrology. These opened tremendous opportunities for Sobi in the coming years, and we are actively preparing these new launches in 2025, continuing the expansion of ongoing launches with our strategic growth portfolio. We have a very strong momentum. We have a lot to do in our pipeline development and we look forward to this journey with our colleagues and stakeholders across the globe. And as you can sense, the outlook is a reflection of this work. And also, as you know us, we are not the guys who want to impress you by what we forecast, but more by what we are doing. And we have a lot on the plate. We need to do a lot because we are less by a fantastic pipeline. In this regard, I'd like now to refer to the Q&A session. We will now answer your questions. [Operator Instructions] Thanks in advance. And let's proceed. Please go back to the operator.

We will now begin the question-and-answer session. [Operator Instructions] The first question comes from Mattias Haggblom from Handelsbanken. Please go ahead.

Thank you so much. Two questions, please. So Beyfortus royalties made up 1/3 of your 2024 EBITDA in light of a potential competitive launch by Merck ahead of the 2025, 2026 RSV season and the 100% margin contribution a royalty stream provides. I was curious how you incorporated that potential competitive launch into your 2025 guide. Does guidance include at Merck launches and grab a fair share of the U.S. market season 1? And as a consequence, Beyfortus royalties have now peaked in contrast to what consensus models or what is baked into your guidance? Secondly, investors appear to expect that Sobi will host a CMD later this year and perhaps provide 2030 revenue targets. Is that fair assumptions? Or are you more likely to do selective deep dives on select assets rather than host a full-blown CMD? Thanks so much.

Thank you, Mattias, for your questions. With regard to Beyfortus margin. I mean, obviously, we do our best possible judgment – and based also – we follow here some of these guidelines and you probably listened in to what Sanofi was outlining last week. I mean, just bear in mind, let's say that, obviously, our royalties, relatively speaking, are going to grow as a percentage of sales. And yes, we basically based it on what we think without having had any direct discussions from – with Sanofi to what we think their prospect is. And we are recognizing that the formidable competitor is coming in, but we also recognize – it's a product that is very strong – Beyfortus has very strong data in terms of real-world evidence and in terms of primary endpoint versus the product. So we don't see now a Cassandra scenario for this royalty stream. And so we have recognized this in our guidance. As far as the CMD is concerned, we were thinking about it, but we have actually a lot to share. So as opposed to now having one CMD, and we may end up there. But our current thinking is more to have a sequence of events and deep dives, where we want to update the audience on different items, definitely related on our pipeline. But also, for instance, related for sure at the right time to want and how to think about these new indications and other products. So this is probably more what we want to do as opposed to having one event and providing the different time points when new data arise and new endpoints have achieved that we basically provide an overview to keep you abreast with what we are doing. Thank you. Maybe next question?

A quick follow-up if I could. Sanofi said they expect to grow global sales of Beyfortus, but they refuse to comment on U.S. sales since you only have exposure to the U.S. portion, any insight to how you expect that market to progress in 2025?

No, we have made certain scenarios. And on that basis, we have forecasted. And I don't want to comment more on or what kind of other data points we may or may not have.

Excellent.

Thank you.

Your next question comes from Brian Balchin from Jefferies. Please go ahead.

Hey. Thanks. Just one question on margins. So clearly, 2025 margin guide mid-30s factors in the build-out of Aspaveli in nephrology and NASP in CRG but then you look at the 2026 consensus, margins are stepping up to 40%. So Guido, it would be great to just get your thoughts on that step up into next year, just how we should be thinking about margins going forward? Thank you.

Yes. Thank you. I mean we were happy already to provide guidance for this year. And obviously, customary is that we provide guidance for next year, at the same point of time in a year later. But when you think about this business and why – and I know that everybody wants us to have higher margins. But realistically, we have – so when you think about it, three products, very near-term in the pipeline, two of them extremely material to the future of the company. We have two products in the growth phase and Altuvoct being now commercialized all Europe and also internationalize in our territory during the next, let's say, 12 months. And then you have Vonjo where we shared the vision that we want to embark on label expansion new indications. So this is a lot for the size of the company. In fact, the opportunity is in terms of scale is probably for the size of company we are, is a massive work. That's the reason why we look at the royalty income and the income we derive from our foundation products as a source as a unique opportunity to catalyze this. But some of these building blocks and these expenses on a relative scale, a, because the company grows, b, because some of the expenses will come down – will allow us – should allow us to have margin expansion to what extent this will lead – will yield not to result in 2026. I think we have, obviously, certain assumptions. But that will be premature. I mean I think we – at this point of time, we want to demonstrate that we can be proud of our results in 2025. And as I said, we are – we want to shine by results, not by forecast. I think let me give you some color at least.

Thank you.

I think that may give you some color at least, Brian. Thank you. Maybe next question?

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

Thanks for taking the questions. I have a couple on haemophilia A, please. So given the strong Altuvoct launch in Germany, just wondering how you're thinking about your target market share. You've given the 30% to 40% range. But should we now sort of consider that you're aiming for the higher end of that range and perhaps is 35% now a reasonable floor. And then I know you've talked about considering Altuvoct and Elocta as a single haemophilia A franchise. So just wondering, do you think you can grow this franchise as a whole by double digits consistently over the next few years? Thank you.

Thanks for your question. I mean, yes, you can see the market share evolution has been very impressive, granted this is only Germany, one part of the franchise, and we want to make sure that this becomes a European and then an international launch very soon. So basically, the data and the evolution that we have seen and when you think about it, I mean, 15 points more in 5.5 months is really impressive. And I think any data point that we have a good start also in Switzerland and once we got the regional approvals in Spain, we also look forward to materialize sales there. So as you know, the data we are probably our confidence in that range has increased because the product gets a significant pool from the patient community, from the physicians and the feedback we get from patients is – and also physicians is very positive in terms of how it affects the daily life in terms of "I feel better, experience less pain." So all of these assumptions that we hoped for and that we saw initially from the trials is coming through. And we see that we have by far – we are really at the early start, but we think that these ideas are transferable. So clearly, we are probably more in the higher end of the range as far as our expectation is concerned. Yes. I think this gives you probably some color. I mean I don't want to be now setting out a singular number, I think, but higher end, I think, should give you some direction. Thanks a lot. Next question?

The next question comes from Christopher Uhde from SEB. Please go ahead.

Yes. Hi. Thank you for taking my questions. Two, please. First, just coming back to that 2026 margin, and I mean, noting that some of the R&D investments are obviously going to take a little time to kick off. I guess, a few trials yet to be put in. How – I mean, is there some indication you can give on sort of a long-term floor for R&D? And also as it relates to the margin going forward, you've been on pause for a little while for BD and M&A. Is that a factor when you are guiding on the margins? Or do you tend to exclude that? And then my second question then is on Altuvoct, can you give us a sense of the demand in markets you've entered outside Germany and what proportion of switches are non-factor therapies versus what are non-Elocta factor VIII switches? Thank you.

So let's start with the long-term margin and R&D expenses. I mean, historically, what we said is that R&D expenses as a floor probably around for the kind of model that we are running 13% to 15%, that's the range, yes. And in a very important year that at the upper limit, but normally probably we are at the low end, given the fact that we obviously in-source innovation. That's roughly where we are in this range. But we obviously – as you will see also during the later part of the year, we really want to make a difference. This is a very strong team under Lydia's leadership and that have demonstrated now to execute in line with expectations. And as far as M&A is concerned, we have excluded this simply because we don't know what comes our way and what kind of profile you're going to find and then it's very difficult to model this. But we are very mindful of the sensitivities on margins in today's environment. So for us to unload things now that with would mute our earnings expectations is very difficult undertaking and we – it would need a lot of conviction to do this. And as regard to Altuvoct switches, I mean, what we see here is as an example of Germany that at the beginning, it was majority Elocta and now is the vast majority, as I pointed out during the presentation is driven by competitive products, including non-factor products. So we try to be democratic and take from everybody.

Thanks very much.

Thank you.

The next question comes from Erik Hultgard from Carnegie. Please go ahead.

Yes. Hi. Two questions, if I may. First, on Vonjo, could you give us an update on the IP status for Vonjo and the patent extension application, if I remember it correctly, the application was pending the time of the acquisition and was expected to be approved in 2024? So I may have missed it, but I haven't seen an update on the matter. And then secondly, on Vonjo as well and the PAXIS study, is that – do you think registrational given the unmet medical need? Or should we sort of think that additional Phase III studies will be required as well? And then maybe the opportunity within this indication compared to MS, if you could categorize that, that would be helpful? Thank you.

Yes. Maybe I'll start with the IP status on Vonjo. I mean our assumption hasn't changed that we have a 2034 protection – until 2034 and that obviously predicates a patent extension that based on my knowledge, is not yet there, but we have not seen anything that would speak against this and so I think it's more of a formality than an if. And with regard to PAXIS, I mean, this is obviously a Phase II study depending on the results, that will be hopefully, a Phase IIb. I mean, Lydia, maybe you want to comment?

Yes, sure. So obviously, this is a dose finding study and with – in a disease with a very high unmet medical need and no available treatments for patients. So depending on the magnitude of the effect, what we have agreed with regulators is that we will have the discussions to see this could lead directly to a registration. What is positive is that we have started the discussions before in order to have a trial design that could meet all these expectations. But obviously, until we see the data and the results, we would not be able to finalize the strategy. But we are very eager on starting the trials activated and also the physicians, the PIs are really eager in starting the recruitment.

Yes. So there's a lot of unmet medical need, Erik, and a lot of excitement that was expressed to us at ASH, and the size of the opportunity is very material. So it is not in MF, you have around 17,000, 13,000 are treated by JAK inhibitors. Here, you have a patient population that are de facto there's no available treatment – effective treatment with around 5,000 in the U.S., and for a company that potentially is first in into the therapy. This would be a very meaningful and very sizable opportunity.

Right. Thank you.

Yes. Thank you.

The next question comes from Gonzalo Artiach from Danske Bank. Please go ahead.

Hi. Gonzalo Artiach Castanon from Danske Bank. Thank you for taking our questions. Two questions. First one is on Aspaveli. Apellis mentioned recently that they will aim to initiate two new Phase III studies in renal diseases in FSGS and DGF. So I was wondering what is your plan here? Do you have any intention to join on that given the strong data you saw in VALIANT or yes, I haven't heard anything from that from you guys. And the second question is on SEL-212. Based on the interactions you have had with clinicians, what is the feedback you get for a potential launch, yes, this year, beginning of 2026? Is it, I mean, because based on the way you have phrased it, it seems that you are more cautious in terms of that launch. So where do you see the initial patient indication? Is it in methotrexate intolerance or resistant patients or are you planning to compete straight with KRYSTEXXA and KRYSTEXXA methotrexate? Thank you.

Yes. Thank you. So with regard to the two indications for Aspaveli in nephrology. We have – I mean, this is a partnership with Apellis, we are in discussions with them on how to best partner – you know that we have a mechanism in any case that allows us to opt in at the end of the Phase III development. And we have discussions on how we can – because we are excited about the opportunity of Aspaveli in the renal disease on nephrology and as a consequence, we have no update yet, but it's an ongoing dialogue with Apellis, and Apellis is a great partner, and we are super excited obviously about nephrology. As far as SEL-212 is concerned, we have done various surveys, as you can imagine. And the idea is clearly that we where we provide an alternative to patients because when you think about the dosing, the corresponding treatment, that you need to undergo with folic acid with methotrexate, and then twice a month with KRYSTEXXA versus our product, there is enough space for a product like SEL-212. And we would be daring enough to say that we want to be a relevant alternative. And when you think about the number of CKD patients suffering from this disease and patient groups who are also excluded from using methotrexate, we feel that there is a very material opportunity also to expand the market. And we gave guidance that we think that launched 15,000 patients could benefit or should benefit from PEGylated uricase treatment and KRYSTEXXA has probably around 6,500 to 7,000 patients currently treated. So there's still a very significant blue ocean out there, but we will not – and we understand that we will not win at all, but the SEL-212 for the patient as a very interest – and for the physician is an interesting profile. Our conservatism is more we want to complete our submission. And then we – but we are preparing success as we speak and building the teams. So we will not be shy. All right. Thank you. And then maybe we move to the next question.

The next question comes from Shirley Chen from Barclays. Please go ahead.

Hi. Thank you for taking my questions. I have one on Aspaveli. So we saw a slowdown of growth this quarter, and you mentioned about pegcetacoplan in PNH. Just want to – can you please talk about your observation so far Aspaveli competing with these recently approved drugs? And would you expect them to take a big role in – towards the first-line treatment? And also what your view on the sales growth for 2025? Will we see some initial uptake from C3G and IC-MPGN towards the very end of this year? Thank you.

Thank you. We have seen some initial impact in some of the markets where we were facing our competition. And that's what you see that trickling through to a certain degree in Q4, and there's also a little bit of phasing in Q4. But for us, Aspaveli in PNH is clearly a double-digit growth product. And we have, obviously, a significant growth still ahead of us, particularly also in international territories. I just want to outline that, obviously, the majority of our growth, if you think about when you can afford about looking out maybe five years will definitely come from nephrology because the opportunity is much larger and competition is less pronounced. And our data are so differentiated in nephrology, but the product is quite resilient, and this is what we can see. We can see also patients now switching back from oral treatment to pegcetacoplan also in PNH. So we are not giving up on PNH. So we think that we will have a good year ahead of us, and obviously, a more magnificent year than in the years to come with this significant opportunity. And to what degree we will see C3G and IC-MPGN patients ahead of registration we'll have to see. I'm sure there will be some requests in terms of early access, and we will have to treat this obviously using the appropriate standards and as we do usually. But you would probably not be able to avoid it simply because there are desperate patients in need. We look forward to protect themselves or in family situations to protect their children. I think that the data are so compelling, obviously. Thank you. Next question?

The next question comes from Viktor Sundberg from Nordea. Please go ahead.

Yes. Hi. Thank you for taking my questions. So one question on Gamifant. Before, there have been some uncertainty if the pool data from the open-label and EMERALD trials, you have submitted to the FDA for getting it approved in secondary HLH is enough for the agency to make a decision on this indication. I guess now with more interactions, have you got more confidence that the data you have at hand is enough for the FDA to take a decision on this? And then just secondly, I think a lot of the investor debate this morning was about your outlook on EBITDA margin in the mid-30s, with consensus being a bit higher here in 2025, especially as the guidance for 2024 set in the beginning of last year was a good indicator of the actual outcome last year, even if you have beaten your usual guidance quite often historically. But I just wanted to understand a bit more of a kind of moving parts you expect for EBITDA guidance this year, what could get you in the high end of margin or even surpassing in 2025? Thanks.

Yes. First, maybe about the confidence of secondary HLH. To be honest, if we wouldn't be confident, we wouldn't submit here. And – but that doesn't mean we get certainty. I mean, Lydia, you want to comment?

Yes, sure. Because obviously, we've been in conversations with FDA, and we are – in our submission package, we have a robust data from several studies and not only the study [06] and the EMERALD but also from [indiscernible], which is a historical control arm. So we are providing FDA with everything that has been requested. And the conversation, it is really in a positive framework so that we can – we are really confident that we will bring the product to patients with this high unmet medical need. So yes, I think the level of confidence is high because we have strong data to support the submission.

Yes. And with regard to the outlook before I hand over to Henrik, I mean, just bear in mind, Viktor, 2025 is a defining year for the company. We have – when you think about to keep it simple for everybody in U.S. dollar terms, roughly the company is a $2.5 billion business. At the Capital Market Day, many, many years back, now, we set out this vision of SEK25 billion by 2025. Now it is SEK26 billion by 2024, which, to be honest, is a big relief after we suffered a little bit for a couple of years from it. But having said this now on the earnings, I mean, we are launching two products. The largest two products where we said Altuvoct is growth price and Vonjo, the indications are out there, even though we owe you obviously more proof points that we can move the product into this direction. No question. But we are confident, then we have three products. One of them we said blocks a potential for Aspaveli. SEL-212 at the last earnings call, we already indicated that there is absolutely no reason to revise our ambition for SEL-212 from this Capital Market Day. We said this could be a $500 million product. And when you look at the latest report from Amgen on KRYSTEXXA, I understand there absolutely will be to revise our ambition. If anything, once we have submitted, maybe we can think about it, what more is possible? And then we have secondary HLH, which will essentially double our opportunity, as we previously pointed out for Gamifant. This is a year we don't want to make too many shortcuts and misses because it will be so important for the company to again change its layout totally, and basically have much more material products in the year to come. And that is basically driving our cautiousness, but we understand that probably today, margins are important. And you know us a little bit, we are not here to sit back and relax, and spend frivolously. But we are a company that is building businesses, and we will look opportunities to improve this company as we speak, and we are not the guys who are typically giving guidance lightheartedly. So we are more on the conservative side. I mean – maybe, Henrik, you want to comment what could influence guidance.

Yes. So I mean, we obviously have a lot of opportunities to pursue this year, and we need to give justice to the opportunities that Guido mentioned. Also I'd like to mention that margins are also a function of sales and sales growth. So of course, the continued momentum here is very important. And then we have a high degree of confidence. Then maybe just to reiterate that, obviously, Beyfortus is part of our P&L, and that's a factor that we don't control.

So I think this gives us a bit of color. I think if some of you are still up for it. I think we should be open for at least one question – two questions, yes. We have two questions for those who want to stay. Yes. Please. Maybe next question?

The next question comes from Alistair Campbell from Royal Bank of Canada. Please go ahead.

Hi, there. Thanks for fitting me in. To be honest, you have covered most of my questions, but if I could just have a follow-up on Vonjo and the PAXIS trial. VEXAS is a disease area, I'm frankly not very familiar with. But just to get a sense, as you launch that study given the biology of the disease, is that somewhere where you feel quite a high degree of confidence? Or is this more of a sort of – well, it might work, let's give a chance? I just want to get a sense of your confidence levels as you head into that trial. And then maybe just coming back to balance sheet. Guido, you obviously talked a lot about there's a lot going on internally. But with the balance sheet strengthening, do you think you would be looking at new deals and new opportunities going to this year? Or do you think focus will be very much internal? Thank you.

Thank you. Maybe Lydia on the confidence on VEXAS?

Yes, sure. So basically, we have very high level of confidence because of the unique kind of profile of Vonjo, as I've mentioned during the presentation. So this is something that could be very specific and targeted. And what was at the beginning, just a scientific concept, we already got a proof of concept with some patients that have been treated, and we already know the efficacy and the higher efficacy in those. Obviously, those are small and single center experience, and that's why we are running this clinical trial and with this high level of confidence. So based on the mechanistic effect of Vonjo and based on the preexisting proof of concept data available in the U.S. in few centers.

So obviously, it's a new disease. Time will tell, but the team has thought about this very carefully, and we have chosen very strong centers and a strong setup. So there is confidence we are even – probably even have a higher confidence or a higher probability of success in CMML, which is much more closely related to MF. And this is also not indication that would be underestimated and then we can talk about it another time. And then as firepower is concerned, yes, we are reviewing assets as we speak. But we are mindful of what we have on the play today, and we are very mindful on the earlier discussions on earnings. Maybe last question. Thank you.

The last question comes from Liu Yifeng from HSBC. Please go ahead.

Thanks for taking my question. Just one on Vonjo, specifically on CMML and you just talked about it. Could you give you a bit more color on the development time lines and when you're sort of planning to launch clinical trials and whether that's the trial has a registrational potential, how should we think of that in 2025 and 2026? Thanks.

Thank you. Lydia?

Sure. So what we are starting right now is a research collaboration with the international working group. So that's what is our first step based on the data that we see and on the results, we will be defining the path to registration. But again, based on the magnitude of the effect that we could see on the results. But what we are starting right now is a research collaboration.

Liu, this research collaboration could lead to a filing. That is basically because there's a huge unmet medical need, and it's a very significant patient population. I mean, today, the epi data vary quite a fair bit. But even in a conservative part, it's definitely worth our while.

Yes. So this maybe summarizes, let's say, our earnings call. I would like to thank you for your attention, and please reach out to us if you have further questions. I mean, time is always running out. But we wanted to give you some color at least. Really like to thank you all for your interest. And as you can see, we like this company and we like to build it. Thank you so much, and look forward to catching up with you in due course. Thank you.

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