Swedish Orphan Biovitrum AB (publ)

Ladies and gentlemen, welcome to the Q3 2024 Report Conference Call and Lab Webcast. I am Alice, the Coral School Operator. I would like to remind today all participants will be listed in only mode any conference is being recorded. The presentation will be followed by a question and answer session. You can register for questions at any time by pressing star and 1 on your telephone. For operator assistance, please press star and 0. The conference must not be recorded for publication or broadcast. At this time, it's my pleasure to hand over to Brida Erkers, CEO. Please go ahead, sir.

Yeah, thank you so much and welcome everybody. This is Guido Erika, CEO of Sobi. We are delighted to welcome you to the third quarter 2024 conference called for investors and analysts. We posted this presentation earlier today under sobi.com. Please turn to page number two and as per usual, forward-looking statement. Please take note of it. And unless stated otherwise, we are making comments that most relate to the third quarter at constant exchange rate in million Swedish krona. Today, we plan to cover the key aspects of the Q3 report. I'm joined by Henrik Stenqvist, our CFO, Lydia Bart-Franch, head of R&D, and chief medical officer. And during the Q&A session, by Armin Reininger, our senior scientific advisor. We plan to review during the presentation first and then cover Q&A thereafter, and this session will take until 3 p.m. For those on the phone, as usual, please join the queue for questions by pressing star 1 And we would really recommend to only ask one or maximum two questions at a time. So let's go straight to slide number four. And we are pleased with the performance in the third quarter and the overall strong first nine months of the year. This quarter is about top line growth, strong earnings, progress of recent launches, and important milestones of our pipeline. We saw very significant top-line growth. Growth in Q3 was 39%. EBITDA margin adjusted was 43% for the quarter. This reflects a strong performance across the portfolio and significant seasonal royalty from Bifortis in the US. Excluding this seasonal RSE franchise, the portfolio grew 16% in the quarter, reflecting a strong base of our business. When you look at the first nine months, we grew 24% year-to-date, and excluding the RSV effect, 22%, demonstrating the progress of the company. Hematology growth was primarily driven by Dr. Dett, Asper Valley, and the launch of Altuvox. We have materially improved our market share in Haemophilia A prophylaxis within the first two and a half months of launch. This is even more impressive as Q3 is typically a more difficult period for switching patients in view of the holiday season. We will talk more about this important launch later. We continue to systematically build the launch of Wonjo, and we saw consistent quarter-on-quarter progress, and we are continuing to build awareness around the product and the unmet medical need in myofibrosis. Immunology reflected a very substantial by Fortis royalty income, as mentioned before, and strong growth of Kineret. In terms of the pipeline, our progress continues after the approval of Altuvoct in the EU for Haemophilia A and the filing of SEL212 in the first half of the year, with very encouraging readout of Aspervalli Nephrology, demonstrating a highly significant reduction of 68% in proteinuria in these rare, hard-to-treat diseases. We look forward to sharing the full data set this weekend at ASN. We have a very strong momentum in the SOBE business, both commercially and in development, and we are pleased to update our guidance for 2024 We expect mid-teen growth, previously was low double-digit, and mid-30s for adjusted MTA. Let's move to the next slide and look at our overall performance in more detail. Our growth of 39% in the quarter was driven by both hematology and immunology sales. We saw very strong performance in hematology with 18%. and due to the contribution of bifortis, immunology was 96%. The US and European business are growing strongly with 40 to 15% in the quarter. We look forward to continuing to deliver on this progress through the remainder of the year. Please turn to slide number six. This growth strategy has led to a strong results primarily driven by our strategic growth portfolio. 56% of our total business has been driven by this portfolio in Q3. The strategic growth portfolio has been growing by 113%. Altamogt now on the market is starting to deliver and they're on the way to become a very important medicine for Surrey going forward. Please turn to slide number seven. This slide depicts our power station generating the energy for Sobi's growth. It depicts a healthy portfolio of today's and tomorrow's breadwinners. As outlined, the strategic portfolio has generated impressive growth rates so far this year. On the left, we have the pipeline and the major achievement it has delivered this year. We have received fast rate designation for GAMI Fund and secondary HLH in Q2. and are preparing to complete this filing by end of year. For Cell 212, we are continuing with the submission to the US FDA. In Q3, we had the positive readout for Aspiravalli and Nephrology, as mentioned. At the same time, we continue building our geographic footprint with the launches of Aspiravalli, Doctylid, Wondro, Cylontar, and Gamifant in new markets. This puts SOBE into a position to create strong mid-term growth driven by new products, execution and our development commitments. ASPA Valley in Nephrology and Cell 212 in Chronic Refractory Gout are of substantial relevance for SOBE and will be prepared for the remainder of 2024 and 2025. We are extremely excited to build relevant launch platforms to bring them to patients who are in need of new treatments in these diseases. Whilst Gummy Fund and secondary HLH will significantly enlarge the accessible patient pool for the product, we will rely on our existing go-to-market organization. Please turn to slide eight. Title work. We are very pleased with the first few months of the market with Altuvox. As communicated at previous events, Altuvox has a unique profile allowing it to normalize patients for the majority of the week with a once a week administration. Its molecular structure is creating the foundation for unprecedented levels of protection. Two recent articles in the New England Journal of Medicine confirmed the relevance of this important medicine. Our in-going hypothesis was that this treatment would create or transform patients' lives and the expectation about life. When reviewing the response two and a half months after the launch in Germany, in terms of market share gain and positive feedback from prescribers and patients, our belief to change hemophilia A therapy of tomorrow has further grown. Our market share combined in Locta and Altobox in hemophilia increased by six points in Germany within two and a half months of launch. And please bear in mind that obviously we also took share of our Locta that basically is not considered in this number. We look forward to bring this important medicine to more markets across Europe and Soviet territories as the launch progresses. Please turn to slide number nine, Asper Valley. The launch of Asper Valley in P&H continued well with quarterly sales of 270 million sec and 66% growth, continuing a solid launch trajectory and strong momentum across all regions. We also announced a partnership with Enable Injections to bring much more convenient self-administration experience for patients with the infused injector, which we aim to bring to the market for both PNH and nephrology. As previously emphasized, Aspergeri's future growth will be primarily driven by the new nephrology indications. The highlight of the quarter from a development perspective was undoubtedly the very strong data we have seen in nephrology in two rare kidney diseases, C3G and ICMPGN. showing a very significant 68 percent reduction in proteinuria in the primary endpoint of the phase three valium study. We are excited to be sharing the full data set this weekend at the ASN Congress in San Diego. Please turn to slide number 10. As we discussed internally, to understand the epidemiology of this product, It's quite challenging. We thought it's important that you get our view on these two indications. We are very excited on the opportunity in nephrology, and I wanted to take some time to outline our view. We believe this could represent a blockbuster opportunity for SOBI, considering there are an estimated 8,000 diagnosed patients in Europe with C3G and ICMPGN. Most diseases are significantly underdiagnosed. With an approved treatment, there's an opportunity to increase the number of diagnosed and treated patients. There's a significant work to be done in the coming months to understand the full patient journey with different stages and severity of the disease. This will be key to unlock this important market opportunity. You're in a great position As the drug has been studied in both indications, adults and adolescents, C3G patients, as well as in pre- and post-transplant. Our endpoint reduction of proteinuria of 68% has impressed quite a few experts in the field, particularly in view of the conclusion of a Spanish group, Lawson. 50%, they concluded that 50% reduction in proteinuria is a threshold for clinically meaningfulness in this setting. Please turn to slide number 11. Dr. Lett. Dr. Lett had another strong quarter with 65% growth. We did book the milestone payment from our partner in China on approval of the ITP indication in China during this quarter. Sales growth was driven by increased uptake in the US and ongoing launches in the regions Europe and international. In the West, there's a continued positive evolution of new patients, new prescribers, higher market share. There's also accelerated growth in Europe and international driven by our ongoing launches and continued increased market share in the earlier launched countries. Please turn to slide number 12, Wanzhou. I'm very pleased to provide you with a positive view on Wonjo after some really difficult times. As we discussed, a combination of internal challenges and a new competitive launch eroded part of our new patient base. The economic consequences you can see on the slide in Q4 2023 and Q1 2024. However, it is gratifying to see that we have been making material progress over the last two quarters, and in particular in Q3. Bonjour sales were 379 million SEC in the quarter, with 12% quarter and quarter growth, showing continued momentum with the product reflecting a high level of field activity we have now in the market. This has been seen in September VENT impact data showing Wonjo to have more than 70% awareness now with HCPs. Our thesis to create value by broadening the product in myofibrosis in the U.S., internationalize Wonjo, and expand the product by indication expansion has remained unchanged. Julia will later cover two exciting diseases as potential new indications for Bonjo. Vexus is a disease with no approved therapy currently on the market and a highly unmet medical need. CMML, a rare hematological malignancy, also with a high unmet medical need. We are continuing in the momentum we have now with the product to further expand in myofibrosis and further tap the potential with global launches and the new indication as mentioned previously. Let's turn to Elocta and Alprolex. We are continuing to expand the leading presence of Elocta and Alprolex to more patients in new territories. As discussed earlier, Elocta saw some switches as expected to Altowogt in Germany, however, remains a key medicine in many countries. And I think it's worth noting that if you take away the replacement or the substitution by Altowogt, Elopta actually has been growing. And what you see as a sales effect is more related to phasing of international tenders. Main impact, this quarter, as mentioned, the launch, but also the order phasing. For Alpalix, we continue seeing growth in the number of patients and the geographic expansion, which is partially offset by continued price pressure in many regions and some ordering phasing in the Middle East. Please turn to slide number 14, Gummy Fund and Kinneret. We see continued growth of new patients for Gamifund as we passed in a more important milestone with 1,000 patients treated during the Q3. The quarter was impacted by the ways of treatment as well as some destocking. Competitively, the product is in a very good shape and we look forward to expand later. We are looking forward to submit secondary HLH indication by end of year. And regarding Kineret, we see fantastic growth of 21% in Q3, further reinforced by elevated interest in the IL-1 mechanism, and the demand comes across all regions. Please turn to slide number 15. And at this point of time, I'd like to hand over to Hendrik for the financials.

Thank you, Guido. Hello, everyone. We will now review some key financials for Q3. We move to slide 16. So referring to the table on the right, the revenue of 6.9 billion corresponds to 39% growth at constant currencies. Main drivers were, before this, royalties, Toptelet, Altevoxt, and Asta Valley. The improved Adjusted gross margin of 81% in the quarter compared to 78% in Q3-23 is mainly due to the impact of the Bay Fortress royalties. The adjusted EBITDA margin reached 43%, up from 30% last year, also influenced by the higher sales and high gross margin just mentioned. Looking at operating expenses for the quarter, we observe 12% growth at cost of currencies compared to the same period last year. SG&A, excluding non-recurring items and amortization, increased by 11% at CER in the quarter, driven by launch and pre-launch costs for Altevox, Bonjo, and SAM212. We've managed to partly offset the impact of these activities on the SG&A in the synergies organizations as reported already in Q1. R&D expenses increased by 16% at CR, mainly due to post-approval costs for Altevoxt and on-due activities. With operating cash flow for the quarter at 1.2 billion, net debt at the end of the quarter was 16.9 billion. This included milestones to Sanofi of 1.8 billion following EMA approval of Altevox, as well as a milestone of about 300 million to Cartesian Therapeutics following the rolling submission to the FDA for Cell 212. This corresponded to a net debt to EDTA ratio of approximately 1.8 times down from 2.6 zero times the quarter before. Please turn to the next slide. And we will discuss the financial outlook for the full year 2024. As usual, this outlook is based on revenue growth at constant rates and adjusted beta margin. As you will have seen, we are upgrading our revenue guidance for the full year, anticipating mid-teens revenue growth at CER. And we are reiterating an adjusted EBITDA margin in the mid-30s percentage of revenue. Regarding upgrading revenue guidance and considering the year-to-date performance, the new guidance translates into a slowdown of growth in the fourth quarter compared to so far this year. And this is expected to result from challenging comps from Q423 in general. but in particular in RSV, not at least through synergies, which had very material sales in Q4 last year, and hemophilia with positive facing effects in Q4 last year, as well as the fact that we no longer have manufacturing revenue. The margin guidance remains unchanged at mid-30s percentage of revenue, given the year-to-date margin of 37% and our expectations for upcoming Q4. To reiterate what I said last quarter, we will have continued elevated spending R&D related mainly to multiple filings in CEL 212, county fund secondary HLH, and Estabelli C3G and ICMPGN, as well as accelerated Altevoxt Phase 4 studies. And in SG&A, launch and pre-launch costs for Monjo, Altevoxt, and CEL 212. With the outlook covered, I hand over to Lydia. Thank you.

Hello, Henrik, and hello, everyone. So, let's start with the pipeline milestone on the next slide, please. We continue our solid pipeline progress in the third quarter. In hematology, the New England Journal of Medicine published the Extend Kids Study Results on Agaltuboct in Haemophilia A, confirming that the path to normalization is also possible in the pediatric and adolescent population. We also announced the very encouraging VALIANCE Phase III top-line data for pexitacoplant in C3G and ICMPGN, and a partnership for a pexitacoplant injection device. And I will get back to this in a minute. In August, we filed the Japanese application for doptalatin ITP, and in September, we submitted the pediatric ITP application for the United States. In immunology, We initiated the rolling BLA submission for cell 212 in chronic refractory gout at the end of June, and it's on track. So let's have a look at some of this, as well as some new perspectives for emapalumab and pacretinib. Next slide, please. In August, we announced very positive data from the VALIANT Phase 3 study of pexetacoplatin C3G and ICMPGN. It met the primary endpoint with highly statistically and clinically significant proteinuria reduction of 68% compared to placebo in a broad patient population of 124 patients aged 12 and above. The positive effects were consistent across all subgroups, including C3GNIC and BGN, adolescent and adult patients, and native and post-transplant kidneys. PEXA-TACO plan also demonstrated the statistically significance on the key secondary endpoints, such as proteinuria reduction and EGFR. The study also demonstrated a favorable safety profile consisting with established profiles for PEXA-TACO plan. And as Guido mentioned, the full data has been accepted as an oral late breaker at the American Society for Nephrology Congress this week in the high impact clinical trial session and we will invite for a call on Tuesday, October 29th. Next slide, please. We also enter a partnership with Enable Injections to use the infuse injector for subcutaneous delivery of PEX-ETACO plan, enhancing the patient experience and thus supporting adherence. This device is already approved for use in the U.S. by our partner, Appelis, and we plan to have it in all Soviet territories for patients with PNH and C3 and IC and PGN. Next slide, please. Moving on to emapalumab, we want to share that the first patient has been dosed in a proof-of-concept study for prevention of CAR-T side effects. CAR-T is a growing blood cancer treatment, and while it's highly effective, it comes with important toxicities. Most common is hyperinflammation in the form of cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. And both conditions require intensive monitoring and multidisciplinary management to mitigate their potential life-threatening effects. Interferon gamma plays a key role in these toxicities, and it has been shown that blocking interferon gamma could prevent side effects without hindering the efficacy of the CAR-T construct. Since semapalumab targets interferon gamma, there has been interest in studying it. The Massachusetts General Hospital and SOVI are working together in a research collaboration, which is expected to see its results next year. Next slide, please. For patritinib, on the top of other areas we have previously communicated, we also see new emerging areas of interest, namely vexes and CMML. PEXS is caused by a somatic gene mutation and manifests as a wide range of inflammatory and hematological symptoms. It was first described in 2020 and therefore has a potentially high number of underdiagnosed patients. Prevalence may be as high as 1 in 4,000 males over age 50 in the United States. There is no standard treatment available for PEXS, though almost all patients require corticosteroids. JAK inhibitors offer some efficacy, but they might not target root inflammatory pathways or address cytopenias. We believe that pacretinib could be beneficial because it may target additional pathways beyond JAK. And SOVI is supporting an investigator-initiated sponsored trial at the Washington School of Medicine. CMML is a blood cancer with features of both myelodysplastic syndrome and myeloproliferative neoplast. Cytopenias and organomegaly are common, and the median survival is 20 to 40 months, and its prevalence is one in 100,000 patients. The only curative treatment is hematopoietic stem cell transplantation. Preclinical and clinical data show a potential benefit of JAK inhibitors. STOI supports an investigator-sponsored study at the Icahn School of Medicine. Both indications show the potential of pacretinib beyond myelofibrosis, and we are looking forward to more clinical data substantiating the benefit of pacretinib in these disease areas. Next slide, please. As Guido said, Altubox is rapidly being adopted in the haemophilia community. The results from the Phase III Extend Kids Study, excuse me, have been also published in the New England Journal of Medicine. This is the first assessment of once-weekly ultraviolet prophylaxis. Oops, I'm really sorry. Just give me a second. Sorry, I've been having a cold. As I was saying, this is the first assessment of once-weekly ultraviolet prophylaxis in previously treated children. And the results indicate highly effective bleed protection in kids under 12 years of age with severe haemophilia A. Since haemophilia is a congenital bleeding disorder, early prophylaxis is crucial. However, the clearance of administered coagulation factor concentrates is known to be greater in children than in adults. And this usually leads to burdensome treatment regimens involving multiple injections per week. But the X10 Kids data shows that Altavolt is highly effective with once-weekly dosing in patients preventing bleeds, with 64% of patients having no bleeding episodes in this 52-week study, 82% of patients having zero joint bleeds, and 88% zero spontaneous bleeds. Going forward, joint health and physical activity will be a focus of our data generation activities. Just one bleed can lead to a vicious cycle of bleeding synovitis and damage of the cartilage, ultimately leading to arthropathy. And continuous prevention of joint bleeds is therefore paramount for haemophilia patients. Freedom is an example of this as we are monitoring joint health and tracking which activities are enabled by the high sustained factor VIII levels. The effect on overall health outcomes is also part of this phase 3D study. It will help to define the future of joint health in haemophilia. And we will be releasing more information on our Facebook program soon. Next slide, please. Summing up the year so far, we have delivered on all our major milestones. Still understanding the submission of GAMI funds in secondary HLH format. Sorry. Sorry. the submission of gamifan in secondary HLH for macrophage activation syndrome in Stills disease, and the doptellate pediatric filing in the EU. Our momentum will continue in 2025. Next slide, please. We will continue to generate data for Altuvox, and next year we expect initial data from the FREEDOM phase 3B study. For Astadeli, we aim to submit our nephrology application in Europe and Japan. GAMIFAN will see U.S. regulatory decision on secondary HLH mass, as well as submission in Japan. FDA will decide on CELTO-124 chronic refractory doubt. And we also expect Kineret to be ready for the submission in STILs disease in Japan. With that, I would like to hand back to Guido. Thank you.

Thank you, Luria. As you can see, at SUBI, we are very much dedicated to submission, even in difficult situations. We are very pleased with ISUBI's development, as you can see, for the first nine months of the year and with our performance in Q3. As you outlined, we have experienced very strong top-line growth, primarily driven by Fortis, but also the rest of the portfolio saw significant growth with 16% for the quarter and 22% for year-to-date. This reflects a very strong performance in our strategic portfolio, continued strong growth of our key products around the world. This has led to upgrade the revenue guidance from low double-digit growth to mid teens for the full year, and we maintain the guidance of mid 30s adjusted EBITDA margin. Our R&D pipeline so far this year has showed Tremendous progress with approval of AltaWorks in Europe, the filing initiation of CEL 212 of Kroenker Factory Goud in the US, the positive phase 3 variant data and nephrology for Asper Valley, and receiving two fast-track designations for Gamifund and CEL 212. We have strong momentum in the business pipeline development, and we look forward to continuing this journey with our colleagues and stakeholders around the globe. Let's go straight into Q&A.

We will now begin the question and answer session. Anyone who wishes to ask a question may press star and one on their touchtone 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 choose only answers and eventually turn off the volume from the webcast. In the interest of time, please limit yourself to one question only. Anyone who has a question may press star and 1 at this time. Our first question comes from the line of Brian Balchin, Jefferies. Please go ahead.

Thank you. Two, if I may. It's just on Bay Fortis and competition. So just first, your view on the extent to which that big 3Q Bay Fortis beat was pulled forward. Anything you can share on the 3Q, 4Q dynamic, particularly as it relates to the impact of 4Q consensus, which I believe is around SEC 1.4 billion, just how we should be thinking about that. And then secondly, on Claire's RoboMap, just your thoughts on the competitiveness of those data, particularly on hospitalisation reduction, differences on endpoints, impact and comparability, and your view on potential just ballpark market share that you think that could take into the 25-26 RSC season. Thank you.

Yeah, thank you. I mean, with regard to Bay Fortis, I mean, you know, we have to say that, you know, there will be obviously a Q&A tomorrow with Sanofi, who I know will update you directly. And, you know, they're not for obvious reasons and for competitive reasons can share all the information with us. But, you know, we, you know, let's put it this way. We are quite for Bifortis because I think at the very nature, you know, we think, you know, in a trial context, you should look at, you know, the primary endpoint and there, you know, Bifortis has a very strong advantage as opposed to post hoc analysis. And maybe, you know, Armin, you want to comment on this, you know, how you view the data because, you know, they were, but, you know, Granted, this is our view, and let's say that Sanofi will update you a little bit more. Armin?

Yes, of course. Thank you, Guido. Thanks for the question. I do believe that if you just look at what we can see here from the interim analysis and the published data so far, the primary endpoint is not as good as the one that you have seen for Bay Fortis. And if you look at the post hoc analysis, then with all due respect that we need to see the full data package. That one seems, at least on the slide that they showed, seems not to have the statistical significance. Yes, the number has, but if you look at the stringent and strict methodology that Befortis has, and it was also New England Journal of Medicine publication, just to remind you. And as well, we do have 90% hospitalization reduction in the real world, shown by the CDC report. I think that is a very strong standard and benchmark that needs to be met first.

I think the real world data from Sanofi that have been published, I think, speak for themselves. But, you know, Brian, I know that you want to know this, but I think, you know, we would refrain from speculating what this means for market share. I think you will have to directly ask Sanofi as we don't promote the product. But, you know, let's put it this way. I mean, we are at least still optimistic about the opportunities of Bifortis. because we have not got any indication that we have to think of a superior product coming our way. And, you know, and obviously, you know, don't forget, I mean, Sanofi, you know, before COVID, you know, used to be the largest vaccine company in the world. And I'm sure they have quite clued on what to do here. Maybe we go to the next slide, to the next questions.

The next question comes from Mattias Häggblom, Handelsbanken. Please go ahead.

Thanks so much for taking my questions. I have two, please. The first one, hemophilia in Germany. The six percentage point share you gained in one quarter. So help me think about what that means for peak market share potential in Germany going forward since patients don't see a position more than perhaps once or twice a year. And linked to that, what does it imply for other European markets? I believe Germany's story has been one of the strongest So should we temper our expectations since it's accomplished in Germany or opposite? Should we see the initial uptake as encouraging also for other European markets? And then second on systemic PEG-septic plan and kidney disease. Curious to hear if there's any feedback from European regulators in terms of timelines when you can submit. Does initial feedback suggest we'll have to wait for 12 months data or is there possibly a path to submit earlier given the magnitude of the effect on the primary endpoint?

Thank you, Matthias. With regard to peak market share, I think one needs to, in order to bring the 6%, I think, which was probably not picked up by everybody. I mean, 6% is the net gain consolidated that we have done, that we have achieved in two and a half months as switches from competition. And obviously, the product is much larger now. in Germany because the majority of patients came in addition from Milokta. Now, and this is all done within a relatively short period of time and, you know, and magnitudinally, I mean, you know, there are close to 2,500 patients in prophylaxis in Germany. So that gives you a kind of taste for the magnitude. Now, is this, so to be honest, we are very excited actually about this very accelerated launch. But we didn't want to lay out now detailed numbers because then it just creates an entire industry for us. So we wanted to give you at least an indication the product is on track and has a faster uptake than we could probably have hoped for, which speaks to the product because even during this vacation period, we have a lot of ask from patients and also from physicians to provide the product. Is this not indicative for the other launches? We will get relatively soon out of the blocks in Switzerland, in Spain, in the UK, and we will be happy to update you once we have finalized the agreements with the reimbursement authorities, but this is imminent. The product and the patient needs will be the same. You know, we are quite competitive, as you said, in Germany. So is this a transferable, you know, achievement? We'll see. We will try everything. But, you know, we cannot promise this. This is traditionally more of a, obviously, as you know, a conservative group. But, you know, we have switched patients, which is good from all, you know, from all products, factor and also non-factor products. primarily non-factor, but still also quite a few already from the non-factor side. So I think we are on a very good track and we are very confident that, you know, regarding the guidance that we have given the market where we said, you know, that this is a product that will be a leader in prophylaxis and leaders in our world is between 30 to 40%. And if anything, this short period has confirmed our view, if not made us a little bit more optimistic, because it's really a very overwhelming positive feedback that we have obtained in this last couple of weeks. And with regard to the timelines on nephrology, I would like to refer to Livia, and maybe you can shed some light.

Yeah, sure. based on the magnitude of the fact that you were referring in your question, we expect to file in Europe in early 2025 and in Japan thereafter. So these are our plans and we're in discussions with EMA to fulfill these plans.

Can I squeeze in a follow-up, please, on hemophilia? Thanks so much for the clarification. But I think there is some investor concern around the impact Altevox has on Elocta. So if you gain net six percentage point share in Germany, I guess the factor eight franchise overall in that market must have grown like 20% year over year or so in the quarter.

It has very substantially grown. We don't guide typically on market, but it has very substantially grown. And I think that this was a little bit missed in the announcement because obviously There were some other effects, the phasing, as we said, of these international business primarily that basically insinuated a much bigger decline of a lockdown than actually has taken place.

That's helpful. Thanks so much.

Thank you.

Our next question comes from a line of Eric Turcord, Carnegie. Please go ahead.

Yes, hi, thanks for taking my questions. I have two. If I may first continue a bit on Altevox in Germany and the uptake. Could you specify a bit the proportion of patients on Altevox in the quarter that came from Elocta and competing products respectively? I think you mentioned that the majority was from other products than Elocta, but if you can be a little bit more specific on the proportion there.

uh and also whether any stocking including in in sales in the quarter for outbox and then i had to follow up on one new thank you hi eric yes thank you for your question so so i should precise the majority of our of our outdoor patients is from a locta the delta of six points market share is what we switched from other products, not a lockdown. So that gives you, let's say, a feel for this. And we don't want to go there now, how much is the majority. But it is clearly significantly more than 50%. So that gives you a feel for this. And there has been no stocking.

OK. Very loud and clear. So then on VONIO, I think you previously said that you expect to reach an infliction point in the uptake in the second half, and I was wondering if this is the case. It looks like you have some slight acceleration in the third quarter, but do you have any leading indicator data points such as prescription, new prescription data supporting that you're actually converting the

Brand awareness data that you highlight that this is actually converting to an acceleration in also prescriptions Yeah, what we I mean first of all, I mean we felt you know after you know 6% quarter-on-quarter growth in Q2 now 12% quarter-on-quarter growth in Q3 that this is at least you know in our books was an acceleration. This is now an inflection of I mean, for a product that is lifecycle, probably not to the full extent. But what we see is that this, our, I would say, consistent work should bear fruits and we should see further progress, let's say, at a significant rate in the quarters to come. We have some indications, you know, I mean, our input factors are significantly up, much more than these percentages would suggest. We have obviously improved the quality of the organization, and we see indicators. The indicators are tougher indicators because the product is in video. We have two channels, SD and SP channel. In the SD channel, it's more difficult to get detailed information on new patients and new prescribers but we see that the product is on the good way. It takes us more longer, but honestly, after, I would say, two more depressing quarters in Q4 and Q1, we feel now that we are coming out of this and we see significant growth. You have seen the indications that we are marking for the product. from Lydia's presentation and we are looking forward to more material international launches next year. Pacifica accelerating. So the combination actually makes us quite confident about the product that we are on the good path. But bottom line is it takes us longer. But you can expect strong growth also in Q4. That's clear.

Sounds good. Just if I can stick in the final short one on Ronjo. So could you say something about the proportion of patients starting on Ronjo that are above or below 15 in platelet count?

Yeah. Historically, that rate was, you know, this is what we explained at one of the earning conference. Before we had, before the competitive launch, we used to be a 50-50. Now, this is probably more a 70-75 to 30 or 25 ratio favoring below 50 where we are mining and where we have strong preference shares, obviously. And our job that is cut out for us with the help of the clinical work is to appeal to a broader audience. and we make respective and to be in line with the NCCN guidelines. And that's basically the journey and that's the reason why we took us a bit of time even though we put a lot of effort behind it to dig ourselves out of this and now showing the quarter-on-quarter growth.

Perfect. Thank you so much.

Thank you, Erika. Very much appreciate your question. Next question.

As a reminder, in the interest of time, I can't ask you to limit yourself to one question, not only per person. And the next question is from Gonzalo Arteaga, Danske Bank. Please go ahead.

Hi, good afternoon. Gonzalo Arteaga from Danske Bank. One on Bay Fortus and Kles Rovima, the potential competitor from Merck. In case of this competitor receiving approval for next season, If you have any color, you could share on any potential pricing pressure that this could imply for before tools. If you could give us some color here would be great. And also on Bonjo, another question. I mean, you mentioned that the slower integration of CTIO in SOBI has been one of the reasons for sales taking, yeah, being slower than what initially expected, but Also, Jara, as it was mentioned previously, has had a good launch here. So I was thinking, how are you actually planning to convert this 70% market awareness that you just mentioned into sales, especially out of label, given the fact that Jara is doing quite well or they're establishing itself in the market? And also, if I heard correctly in the presentation, you mentioned that your long-term goals for Bonjo are untouched. Is it including new indications that you are starting now or excluding these? Thank you very much.

Thank you for your question. With regard to Merck, Sharp and Dome, I think you need to really ask them how they are thinking about pricing. Historically, at least, MSD was not a price fighter for innovative medicines, even though the degree of innovation I leave it up to the audience and KOLs to judge. It's very difficult to judge, but we'll figure it out. They're coming with a new medicine. Do they want to shrink the profit pool of this medicine? I'm not sure. But, you know, that is something you have to really ask MSD. And with regard to one rule, I mean, obviously, first comes awareness, then comes adoption. We have, you know, quite a few people now in the field. We have stepped up, as we previously reported, our digital interaction. And the reason why we left the goals that we had, you know, that we insinuated, historically unchanged is indeed because we see not everything has to work out. But the combination and using a probability adjustment makes us believe that we do not have to change our guidance or let's say all the magnitudes that people have associated and we have confirmed on Bonjour. So we think that this is a fantastic medicine for And yes, it took us a bit more time, but we will continue making progress in the U.S. We are going to make progress with new data generating and also with this broadening the product in myofibrosis, broadening the product in new indication, and broadening the footprint of the product by launching in other countries. And the combination of this is more than what we need to make our, uh, to achieve our, the expectations for the product. But you know, not everything is going to become true. Uh, so the, but you know, probability adjusted if we stick to the, to what you have set up.

Great. Thank you very much.

You're welcome. Uh, maybe more questions.

the next question comes from line of christopher at cb please go ahead uh hi there thanks for uh taking my questions uh so obviously really impressive start for altivox uh and coming off such a high a lot to share base it must be rewarding after years of analysts telling you russia's family was going to eat your lunch but i do want to get a little clarity around how to think about ilakta through the conversion process component of the launch given that you know your start you started talking about positive phasing impacts on Ilakta already back in Q1 23 that only started to unwind this quarter. And obviously we know payers wanted to ensure market stability, but the unwind does not seem to be factored into consensus. Can you help us understand what proportion of Altivax sales so far were switches from Ilakta and how much stocking unwind is left? Are we talking 500 million Kroner left or could it be more or less? You know, you had what truly looks like a phenomenal clinical result with Aspavelli. So kudos, of course. The share rose 15 to 20% on the back of the first blockbuster sell side forecast following the announcement, which you today are clearly backing. I know you hate when I ask long questions, so I'm sorry about that. But with up to 20% of market cap in play from this, the potential is pretty important to understand the number of, you know, and the number of addressable patients, especially because you used to say that there were up to 8,000 ex-US. Your partner still says 8,000 ex-US, while you now say 8,000 in Europe alone. Your share is up since then. Your partner's is down. Your presentations say the estimate is based on the literature. When I emailed Sobe to ask how it arrived at 8,000, the reply cited a review from Nature Reviews Nephrology summarizing data from Europe, and the reply specifically stated the French data set may be a good anchor. We have anchored on the 14 per million in the bottom range. So that's the estimate is based on that large French study and extrapolating that it takes us to 7,000 diagnosed patients in Europe, which is pretty close to that 8,000. But reading how the review publication calculated the 14 per million, they state point prevalence values were calculated as new cases per year divided by the size of the referral population, in other words, the population of France, times the population average life expectancy. They explicitly state, we assume there were no deaths from C3G. They also do not remove patients who went into kidney failure, but we have that kidney failure rate data for that study from, you know, from the publication. And if you redo their calculation and subtract patients going into kidney failure each year, you're left with only 2,000 patients. So, you know, and of course, more recent large registry-based data sets from UK, Spain, Italy all give similar results. Are you simply expecting to be able to treat all the patients after they have already gone into kidney failure? Or how much of your estimate relies on anecdotal evidence from KOLs and market research? Does Aspavelli need a total addressable market of at least 8,000 patients in Europe to become a blockbuster? Thank you very much for your patience and for the questions.

I mean, you're... So maybe what we do, because this was a lot to take in and probably more than we can cover now as part of this conference call. And the question was also, do you want to have an answer or do you want to make a point? And let's see, I think what you should just, life is sometimes, in pharmaceuticals, I'm not saying more than 30 years, yeah? And I can tell you one thing, just when you think you know something, there's always somebody who knows a little bit more. We have spent now with two major consulting firms, very significant resources over the last half a year with super experts and key KOLs to understand this market. Our head of market analytics said this is one of the most difficult cases of epi data to understand. You know, we will not share all our market insight, as you may appreciate, with the world. But one thing we can guarantee you is, you know, when you think about this market, there are 16,000 to 20,000 patients. There are 8,000 diagnosed in Europe. And obviously, today's treatment is not representative of what tomorrow's treatment is because it's gonna change. There's no effective treatment. So let's leave it there. We also can reassure you that there are many more patients outside of Europe and the data vary here more and we don't want to, but when you think about the value per patient, the way this treatment is gonna change, the way diagnosis is gonna change, if there is a hope for treating patients successfully, then we are very confident and we believe in the blockbuster potential and we do not need to treat all patients. I can reassure you. But I understand that this is a lot. But that's the reason, to be honest, why we have spent on this subject matter more than half a year with probably... the total number of people involved, 15 to 20 people. So I appreciate that this is difficult and we are happy to share with you within the, but we will not, you know, we are a public company. We will not stipulate something that we cannot stand behind. Life is too short for that. Let me be very clear. So in regard to Elocta, I just wanted to say Elocta's growth year-to-date, 6%. So this doesn't look like a product that is in dire straits yeah and uh and therefore what i said also here with regard to uh impact quarterly impact has to be taken into consideration because otherwise you get the impression that the product is worse off than it is but yes the majority of patients that means more than 50 percent uh have been switched in germany uh let's say for a lot for altoworked but still we have gained consolidated market share of more than 6%. So I think that we leave it like this, and then we have probably time for one more question from somebody else. Thank you. And we will come back to you. I have a vested interest to explain this to you properly, and we have obviously not yet been able to do this. Any other questions?

Yes, we do have a question coming from the line of Harry Gillis, Barenberg. Please go ahead, sir.

Hi, thanks for taking the questions. Just a couple of short ones from me. Just back onto Aspavelli, just wondering what your latest base case is for your relative market share versus Novartis that you're including in this guidance for blockbuster opportunity. And then just thinking about the patient pool that is there today, are you seeing a bolus of sort of well-identified patients that are sitting there waiting for new targeted therapies just so we can think about how quickly the sales might ramp And then a very quick one on the financials. Do you also expect a significant step up in OPEX in Q4 like last year, or was that sort of unique to last year? Thank you.

Yeah, I think, you know, let's start with the issue. There are quite a number of, I mean, it's quite extensive market research. There's quite a number of patients that are waiting. There's also a number of patients, you know, that are currently quite desperate and not getting adequate treatment. And so there will be a faster uptake in the economy for a certain group of patients, and then we will have, obviously, to do also some market preparations and making sure that people understand the opportunities to treat it adequately. Based on your data in proteinuria reductions, our assumption is that we would have a larger share than Novartis With all due respect, you know, given the absolute amplitudinal effect, that's our belief, obviously not underestimating, but they are formidable competitors. And we look forward to the discussions at ASN. And let's see, you know, so we are quite, and with regard to the financing, I'd like to refer to Enric.

Yes, you're right that mathematically we do expect to step up in OPEX in Q4 compared to Q3, just as we saw last year.

Yeah, so I think, you know, what we do is, you know, so apologies if we were a little bit extensive. And let's say we can, if you have further questions, please do refer to RARAC. Either we make a meetings available, you know, separately or otherwise we can answer this. So, you know, as you can see, you know, we are quite excited about this data. And because it's not just that my photo story is carried by our broader portfolio. And, you know, we look forward to update you with the next update. Big update is on Tuesday when we have a call on C3GIC and PGN and provide an update on the data from ASN to make sure that we democratize basically the information to these important data points. Thank you so much. I wish you a great week, and apologies if not everybody was able to ask questions. Jura or Jennifer, and we will make sure that you get connected with the experts, or they can probably address most of the questions in any case. Thank you.