Swedish Orphan Biovitrum AB (publ)

STO:SOBI

Earnings Call Analysis

Sobi Reports Strong Q3 Growth, Upgrades Guidance for 2024

Sobi achieved exceptional growth in Q3 with a revenue increase of 39%, driven by significant performance from Beyfortus and a solid launch of Altuvoct, capturing a 6% market share in Germany within weeks. The company reported an EBITDA margin of 43% and upgraded its 2024 revenue guidance from low double-digit to mid-teens growth. Additionally, Sobi announced an impressive 68% reduction in proteinuria from its nephrology pipeline, while maintaining its adjusted EBITDA margin guidance in the mid-30s percent. The overall portfolio, excluding seasonal effects, showed a robust growth of 16% during the quarter.

Robust Growth in Q3 Performance

In the third quarter of 2024, Sobi demonstrated impressive financial performance, with revenues reaching SEK 6.9 billion, marking a 39% increase year-over-year at constant currency rates. This growth was primarily fueled by significant contributions from products like Beyfortus, Doptelet, and the recently launched Altuvoct. Notably, Beyfortus royalties played a crucial role, contributing to an adjusted EBITDA margin of 43%, a significant jump from 30% during the same period last year. Importantly, when removing the seasonal impact of the RSV franchise, the underlying portfolio still achieved a solid 16% growth this quarter. Over the year-to-date, revenue growth stands at 24%, with a remarkable 22% growth when excluding the RSV effect, showcasing the company's continued expansion and steady performance across its product range.

Pipeline Advancements and Strategic Portfolio Growth

Sobi's strong performance is also reflected in its strategic growth portfolio, which constituted 56% of total business in Q3 and grew by an outstanding 113%. The successful launch of Altuvoct is a key part of this strategy, and the company has seen an increase of 6% in market share in Germany within just 2.5 months of its launch. This is particularly notable as it coincides with the competitive launch of similar products. Furthermore, the impressive 68% reduction in proteinuria observed in the Phase III VALIANT study for Aspaveli positions Sobi strongly for market opportunities in the nephrology sector, indicating further bullish prospects for future revenue generation.

Guidance Adjustments for 2024

Sobi has raised its revenue growth guidance for 2024 from low double-digit increases to mid-teens growth at constant exchange rates. Despite this upgrade, the company expects a slowdown in growth in the fourth quarter compared to previous months due to challenging comparables from the preceding year and the absence of manufacturing revenues. The adjusted EBITDA margin is anticipated to remain in the mid-30s percentage of revenue, reaffirming stability in profit margins amid elevated R&D expenditures, particularly related to exciting new drug submissions expected in the near future.

Market Drivers: Hematology and Immunology Innovations

In hematology, Sobi has seen consistent growth surrounding Doptelet, Aspaveli, and Altuvoct, with Doptelet experiencing a notable 65% growth in revenues, thanks to increased demand and expanded market presence. Immunology was similarly buoyed by the remarkable 96% growth attributed to Beyfortus, showcasing Sobi's impactful innovations in addressing unmet medical needs. Additionally, the newer product, Vonjo, reported a 12% quarter-on-quarter growth, indicating signs of stabilization and improved brand recognition as healthcare providers become more familiar with the treatment, which is critical in capturing a larger patient base going forward.

Strategic Positioning Against Competition

Sobi is strategically positioned against competitors, especially concerning its hemophilia products. While Altuvoct poses a shift in patient dynamics from Elocta, Sobi emphasized that Elocta is also experiencing growth independently of the new product. Management indicated confidence in maintaining a significant share of a complex market landscape, buoyed by favorable patient and physician feedback following product launches. Furthermore, the anticipated entry of competitors like clesrovimab may create pricing pressures, but initial confidence in Beyfortus's efficacy remains strong.

Long-term Outlook and Emerging Opportunities

Looking longer term, Sobi is committed to expanding its portfolio and geographic presence, particularly in the nephrology market avenues with Aspaveli and SEL-212. The company recognizes the underdiagnosed nature of conditions like C3G and IC-MPGN, projecting at least 8,000 diagnosed patients in Europe, suggesting a strong growth potential aligned with effective market strategies. With proactive measures in enhancing treatment accessibility and outreach efforts, Sobi remains optimistic about fulfilling unmet medical needs and capturing incremental market share.

Earnings Call Transcript 2024-Q3

[Operator]

Ladies and gentlemen, welcome to the Q3 2024 Report Conference Call and Live Webcast. I am Alice, the Chorus Call operator. [Operator Instructions] The conference is being recorded. [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.

[Guido Oelkers, executive]

Yes. Thank you so much, and welcome, everybody. This is Guido Oelkers, CEO of Sobi. We are delighted to welcome you to the third quarter 2024 conference call for investors and analysts. We posted this presentation earlier today on the sobi.com.

Please turn to Page #2, as per usual forward looking statement, please take a 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 our Q3 report. I'm joined by Henrik Stenqvist, our CFO; Lydia Abad-Franch, our Head of R&D and Chief Medical Officer; and during the Q&A session by Armin Reininger, our Senior Scientific Adviser. We plan to review the -- during the presentation first and then have a Q&A thereafter, and this session will take around. It will take until 3 p.m. [Operator Instructions]

So let's go straight to Slide $4. And we are pleased with the performance in the third quarter, and the overall strong first 9 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 Beyfortus in the U. S. Excluding this seasonal RSV franchise, the portfolio grew 16% in the quarter, reflecting a strong base of our business.

When you look at the first 9 months, we grew 24% year-to-date and excluding the RSV effect, 22%, demonstrating the progress of the company. Haematology growth was primarily driven by Doptelet, Aspaveli and the launch of Altuvoct. We have materially improved our market share in hemophilia A prophylaxis within the first 2.5 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 Vonjo and we saw consistent quarter-on-quarter progress and we are continuing to build awareness around the product and the unmet medical need in myelofibrosis. Immunology reflected a very substantial Beyfortus 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 hemophilia A and the filing of SEL-212 in the first half of the year with very encouraging readout of Aspaveli 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 Sobi 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 EBITDA.

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 Beyfortus immunology grew 96%.

The U.S. and European business are growing strongly with 14% 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 #6. This growth strategy has led to 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%. Altuvoct now on the market is starting to deliver and on the way to become a very important medicine for Sobi going forward.

Please turn to Slide #7. 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 Track Designation for Gamifant in secondary HLH in Q2 and are preparing to complete this filing by end of year. For SEL-212, we are continuing with the submission to the U.S. FDA.

In Q3, we had a positive readout for Aspaveli in nephrology as mentioned. At the same time, we continue building our geographic footprint with the launches of Aspaveli, Doptelet, Vonjo, Zynlonta and Gamifant in new markets. This puts Sobi into a position to create strong mid-term growth driven by new products, execution on our development commitments.

Aspaveli in nephrology and SEL-212 in chronic refractory gout are of substantial relevance for Sobi 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. While Gamifant in 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 8, Altuvoct. We are very pleased with the first few months of the market with Altuvoct. As communicated at previous events, Altuvoct has a unique profile allowing it to normalize patients for the majority of the week is 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 confirms the relevance of this important medicine.

Our ongoing hypothesis was that this treatment would create or transform patients' life and the expectation about life. When reviewing the response 2.5 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, Elocta and Altuvoct in hemophilia increased by 6 points in Germany within 2.5 months of launch. And please bear in mind that obviously we also took share of our Elocta that basically is not considered in this number. We look forward to bring this important medicine to more markets across Europe and Sobi territories as the launch progresses.

Please turn to Slide #9, Aspaveli. The launch of Aspaveli in PNH continued well with quarterly sales of SEK 270 million and 66% growth, continuing a solid launch trajectory and strong momentum across all regions. We also announced a partnership with Enable Injections to bring more much more convenient self administration experience for patients with the enFuse injector, which we aim to bring to the market for both PNH and nephrology.

As previously emphasized, Aspaveli's future growth will be primarily driven by the new nephrology indications. The highlight of the quarter was a development -- from a development perspective was undoubtedly the very strong data we have seen in nephrology in 2 rare kidney diseases, C3G and IC-MPGN, showing a very significant 68% reduction in proteinuria in the primary endpoint of the Phase III VALIANT study. We are excited to be sharing the full data set this weekend at the ASN Congress in San Diego.

Please turn to Slide #10. As we discussed internally to understand the epidemiology of this product, it's not -- it's quite challenging. We thought it's important that you get our view on these 2 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 IC-MPGN. Both diseases are significantly under diagnosed. 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 as to understand the full patient journey with different stages and severity of the disease. This will be key to unlock this important market opportunity.

We are 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, GLOSEN, 50%, they concluded that 50% reduction in proteinuria is a threshold for clinically meaningfulness in this setting.

Please turn to Slide #11, Doptelet. Doptelet 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 U.S. And ongoing launches in the regions Europe and International.

In the U. S, there is a continued positive evolution of new patients, new prescribers, higher market share. There is 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 #12, Vonjo. I'm very pleased to provide you with a positive view on Vonjo after some really difficult times. As we discussed, a combination of internal challenges and a new competitive launch a rooted path 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 2 quarters and in particular in Q3.

Vonjo sales were SEK 379 million in the quarter with 12% quarter-on-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, Brandimpact data showing Vonjo to have more than 70% awareness now with HCPs.

Our thesis to create value by broadening the product in myelofibrosis in the U.S., internationalize Vonjo and expand the product by indication expansion has remained unchanged. Lydia will later cover 2 exciting diseases on -- as a potential new indications for Vonjo.

VEXAS 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 the momentum we have now with a 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 Alprolix. We are continuing to expand the leading presence of Elocta and Alprolix to more patients in new territories. As discussed earlier, Elocta saw some switches as expected to Altuvoct 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 Altuvoct, Elocta actually has been growing. And what you see as a sales effect is more related to phasing of international tenders.

Main impact this quarter were, as mentioned, the launch but also the order phasing. With Alprolix, 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 #14, Gamifant and Kineret. We see continued growth of new patients for Gamifant 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 #15. And at this point of time, I'd like to hand over to Henrik for the financials.

[Henrik Stenqvist, executive]

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 SEK 6.9 billion corresponds to 39% growth at constant currencies.

Main drivers were Beyfortus royalties, Doptelet, Altuvoct and Aspaveli. The improved adjusted gross margin of 81% in the quarter compared to 78% in Q3 '23 is mainly due to the impact of the Beyfortus 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 observed 12% growth at constant 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 Altuvoct, Vonjo and SEL-212. We've managed to partly offset the impact of these activities on the SG&A line through savings in the synergies organization, as reported already in Q1.

R&D expenses increased by 16% at CER, mainly due to post-approval costs for Altuvoct and Vonjo activities. With operating cash flow for the quarter at SEK 1.2 billion, net debt at the end of the quarter was SEK 16.9 billion. This included milestones to Sanofi of SEK 1.8 billion, following EMA approval of Altuvoct, as well as a milestone of about SEK 300 million to Cartesian Therapeutics, following the rolling submission to the FDA for SEL-212. And this corresponded to a net debt to EDTA ratio of approximately 1.8x, down from 2.0x 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 EBITDA margin.

As you would 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 our 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. This is expected to result from challenging comps from Q4 '23 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 spend in R&D, related mainly to multiple filings in SEL-212, Gamifant secondary HLH, and Aspaveli, C3G and IC-MPGN, as well as accelerated out-of-the-box Phase IV studies, and in SG&A launch and pre-launch costs for Vonjo, out-of-the-box, and SEL-212.

So, with the outlook covered, I hand over to Lydia. Thank you.

[Lydia Abad-Franch, executive]

Thank you, 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 XTEND-Kids study results on out-of-the-box hemophilia A, confirming that the path to normalization is also possible in the pediatric and adolescent population.

We also announced the very encouraging VALIANT Phase III top-line data for pegcetacoplan in C3G and IC-MPGN, and a partnership for a pegcetacoplan injection device, and I will get back to this in a minute.

In August, we filed the Japanese application for Doptelet in ITP, and in September, we submitted the pediatric ITP application for the United States. In immunology, we initiated the rolling BLA submission for SEL-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 pacritinib.

Next slide, please. In August, we announced very positive data from the VALIANT Phase III study of pegcetacoplan in C3G and IC-MPGN. 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 C3G and IC-MPGN, adolescent and adult patients, and native and post-transplant kidneys.

Pegcetacoplan 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 pegcetacoplan. 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 29.

Next slide, please. We also entered a partnership with Enable Injections to use the enFuse injector for subcutaneous delivery of pegcetacoplan, enhancing the patient experience and thus supporting adherence. This device is already approved for use in the U.S. by our partner Apellis, and we plan to have it in all Sobi territories for patients with PNH and C3G and IC-MPGN.

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 constructs. Since emapalumab targets interferon gamma, there has been interest in studying it. The Massachusetts General Hospital and Sobi are working together in a research collaboration, which is expected to see its results next year.

Next slide, please. For pacritinib, on the top of other areas we have previously communicated, we also see new emerging areas of interest, namely VEXAS and CMML. VEXAS 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 VEXAS, 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 pacritinib could be beneficial, because it may target additional pathways beyond JAK. And Sobi is supporting an investigator-initiated sponsored trial at the Washington School of Medicine.

CMML is a blood cancer with features of both myelodysplastic syndromes and

myeloproliferative neoplasm. Cytopenias and organomegaly are common and the median survival is 20 to 40 months and its prevalence is 1 in 100,000 patients. The only curative treatment is haematopoietic stem-cell transplantation. Preclinical and clinical data show a potential benefit of JAK inhibitors. Sobi supports an investigator-sponsored study at the Icahn School of Medicine. Both indications show the potential of pacritinib beyond myelofibrosis. And we are looking forward to more clinical data substantiating the benefit of pacritinib in these disease areas.

Next slide, please. As Guido said, Altuvoct is rapidly being adopted in the hemophilia community. The results from the Phase III Kids -- XTEND-Kids study have been also published in the New England Journal of Medicine. This is the first assessment of once weekly Altuvoct prophylaxis pumps.

As I was saying, this is the first assessment of once weekly Altuvoct prophylaxis in previously treated children. And the results indicate highly effective bleed protection in kids under 12 years of age with severe hemophilia A. Since hemophilia 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 XTEND-Kids data shows that Altuvoct 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 0 joint bleeds and 88% 0 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 hemophilia 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 IIIb study. It will help to define the future of joint health in hemophilia. And we will be releasing more information on our Phase IV program soon.

Next slide, please. Summing up the year so far, we have delivered on all our major milestones. Still outstanding are the submission of Gamifant in secondary HLH for macrophage activation syndrome in Still's disease, and the Doptelet pediatric filing in the EU. Our momentum will continue in 2025.

Next slide, please. We will continue to generate data for Altuvoct. And next year, we expect initial data from the FREEDOM Phase IIIb study. For Aspaveli, we aim to submit our nephrology application in Europe and Japan. Gamifant will see U.S. regulatory decision on secondary HLH mass as well as submission in Japan. FDA will decide on SEL-212 chronic refractory gout. And we also expect Kineret to be ready for the submission in Still's disease in Japan.

With that, I would like to hand back to Guido. Thank you.

[Guido Oelkers, executive]

Thank you, Lydia. As you can see, at Sobi, we are very much dedicated to the mission, even in difficult situations. We are very pleased with Sobi's development, as you can see, for the first 9 months of the year and with our performance in Q3.

As outlined, we have experienced very strong top-line growth, primarily driven Beyfortus, but also the rest of our 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 Altuvoct in Europe, the filing initiation of SEL-212 of chronic refractory gout in the U.S., the positive Phase III VALIANT data in nephrology for Aspaveli, and receiving 2 fast track designations for Gamifant and SEL-212.

We have strong momentum and 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.

[Operator]

[Operator Instructions] Our first question comes from the line of Brian Balchin, Jefferies.

[Brian Balchin, analyst]

Two, if I may. It's just on Beyfortus and competition. So just first, your view on the extent to which that big 3Q Beyfortus 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 SEK 1.4 billion. Just how we should be thinking about that?

And then, secondly, on clesrovimab, just your thoughts on the competitiveness of those data, particularly on hospitalization, production, differences on endpoints, impacts on comparability, and your view on potential just ballpark market share that you think that could take into the '25, '26 RSV season?

[Guido Oelkers, executive]

With regard to Beyfortus, I mean, we have to say that there will be obviously a Q&A tomorrow with Sanofi, who will -- who I know will update you directly. And they're not, for obvious reasons and for competitive reasons, can share all the information with us. But we -- let's put it this way. We are quite optimistic for Beyfortus, because I think that's the very nature.

We think that in a trial context, you should look at the primary endpoint. And there, Beyfortus has a very strong advantage as opposed to post-hoc analysis. And maybe Armin, you want to comment on this, how you view the data? Because they werel -- granted, this is our view. And let's say that Sanofi will update you a little bit more. Armin?

[Armin Reininger, executive]

Yes, of course. Thank you, Guido. 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 Beyfortus. And if you look at the post-hoc analysis, and 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 Beyfortus 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.

[Guido Oelkers, executive]

I think the real-world data from Sanofi that have been published, I think, speak for themselves. But Brian, I know that you want to know this, but I think 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 let's put it this way. I mean, we are at least still optimistic about the opportunities of Beyfortus, because we have not got any indication that we have to think of a superior product coming our way.

And obviously, don't forget, Sanofi, before COVID, used to be the largest vaccine company in the world. And I'm sure they are quite clued on what to do here. Maybe we go to the next slide, to the next questions.

[Operator]

The next question comes from the line of Mattias Haggblom, Handelsbanken.

[Mattias Häggblom, analyst]

Yes. I have 2, please. The first one, hemophilia in Germany. The 6 percentage point share you gained in 1 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 that -- does it imply for other European markets? I believe Germany historically has been one of Elocta's 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 pegcetacoplan 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-month data? Or is there possibly a path to submit earlier given the magnitude of the effect on the primary endpoint?

[Guido Oelkers, executive]

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 a net gain consolidated that we have done, that we have achieved in 2.5 months as switches from competition. And obviously, the product is much larger now in Germany, because the majority of patients came in addition from Elocta.

Now -- and this is all done within a relatively short period of time. And magnitudinally, I mean, there are close to 2,500 patients in prophylaxis in Germany. So that gives you a kind of taste for the magnitude now. Now is this -- so to be honest, we are very excited actually about this very accelerated launch. And -- 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 now also from physicians to provide the product. Is this now indicative for the other launches? We get -- we will get relatively soon out of the blocks in Switzerland, in Spain, in the U.K. And we will be happy to update you once we have finalized the agreements with the reimbursement authorities. But this is imminent. And the product and the patient needs will be the same.

We are quite competitive, as you said, in Germany. So is this a transferable achievement. We'll see. We will try everything. But we cannot promise this. This is traditionally more for, obviously, as you know, a conservative group. But we have switched patients, which is good from all from all product factor and also non-factor, 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 regarding the guidance that we have given the market, where we said, 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 Lydia. And maybe you can give shed some light.

[Lydia Abad-Franch, executive]

Yes, sure. And basically, 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.

[Mattias Häggblom, analyst]

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 Altuvoct has on Elocta. So if you gain net 6 percentage points share in Germany, I guess the factor VIII franchise overall in that market must have grown by 20% year-over-year or so in the quarter?

[Guido Oelkers, executive]

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 Elocta than actually has taken place.

[Operator]

Our next question comes from the line of Erik Hultgard, Carnegie.

[Erik Hultgård, analyst]

I have 2. If I may first continue a bit on Altuvoct in Germany and the uptake. Could you specify a bit the proportion of patients on Altuvoct in the quarter that came from Elocta and competing products, respectively? I think Guido 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? And also, were there any stocking including in sales in the quarter for Altuvoct? And then I have a follow-up on Vonjo.

[Guido Oelkers, executive]

Yes. So I should be precise. The majority of our Altuvoct patients is from Elocta. The delta of 6 points market share is what we switched from other products, not Elocta. 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 more than -- significantly more than 50%. So that gives you a feel for this. And there has been no stocking.

[Erik Hultgård, analyst]

Okay. Great, loud and clear. So then on Vonjo, I think you previously said that you expect to reach an inflection 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 highlighted, that this is actually converting to an acceleration in also prescriptions?

[Guido Oelkers, executive]

Yes. I mean, first of all, I mean, we felt after 6% quarter-on-quarter growth in Q2, now 12% quarter-on-quarter growth in Q3, that this is, at least, in our books was an acceleration. This is now an inflection, 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 fruit, and we should see further progress, let's say, at a significant rate in the quarters to come. We have some indications, 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 have -- and we see indicators. The indicators are tougher indicators because the product is in Vonjo, we have 2 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 a good way, yes? So we have -- I mean, it takes us more longer, but honestly, after, I would say, 2 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 earmarking for the product with -- 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 the bottom line is it takes us longer, yes? So -- but you can expect strong growth also in Q4. That's clear.

[Erik Hultgård, analyst]

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

[Guido Oelkers, executive]

Yes. Historically, that rate was -- and 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 to 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 a respective and to be in line with the NCCN guidelines. And that's basically the journey and that's the reason why it 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. Next question?

[Operator]

[Operator Instructions] And the next question is from Gonzalo Artiach, Danske Bank.

[Gonzalo Artiach Castanon, analyst]

Gonzalo Artiach from Danske Bank. One on Beyfortus and Klezovimod, the potential competitor from Merck. In case of this competitor receiving approval for next season, do you have any color you could share on any potential pricing pressure that this could imply for Beyfortus? If you could give us some color here, it would be great.

And also on Vonjo, another question. I mean, you mentioned that the slower integration of CTI in Sobi has been one of the reasons for sales taking, yes, being slower than what initially expected. But also Jara, as it was mentioned previously, has had like a good launch here. So I was thinking how are you actually trying to convert this 70% market awareness that you just mentioned into sales, especially, yes, out of label, given the fact that the Jara is doing quite well or they're establishing itself in the market?

And also, I heard correctly in the presentation, you mentioned that your long-term goals for Vonjo are untouched. Is it including new indications that you are starting now or excluding these?

[Guido Oelkers, executive]

Yes. Thank you for your question. I mean, with regard to Merck Sharp & Dohme, 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'll leave it up to the audience and KOLs to judge. But that is -- it's very difficult to judge, but I would we'll figure it out. Yes, but -- they're coming with a new medicine. I mean, do they want to shrink the profit pool of this medicines? I'm not sure. But that is something you have to really ask MSD.

And with regard to Vonjo, I mean, obviously, first comes awareness, then comes adoption. We have 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 is 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 our let's say, or the magnitudes that people have associated and we have confirmed on Vonjo.

So we think that this is a fantastic medicine for Sobi. 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 myelofibrosis, 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 -- to achieve our -- the expectations for the product. But not everything is going to become true. So the -- but probability adjusted if we stick to what you have set out. Maybe more questions?

[Operator]

The next question comes from the line of Christopher Uhde at SEB.

[Christopher Uhde, analyst]

So obviously, really impressive start for Altuvoct. And coming off such a high share base, it must be rewarding after years of analysts telling you, Russia's Hemlibra is going to eat your lunch. But I do want to get a little clarity around how to think about Elocta through the conversion process component of the launch, given that you started talking about positive phasing impacts on Elocta 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 Altuvoct sales so far were switches from Elocta and how much stocking unwind is left? Are we talking SEK 500 million left or could it be more or less?

Second, you had what truly looks like a phenomenal clinical result with Aspaveli. 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 addressable patients, especially because you used to say that there were up to 8,000 ex-U.S.

Your partner still says 8,000 ex-U.S. While you now say 8,000 in Europe alone. Your share is up since then your partner's is down. Your presentation say, the estimate is based on the literature. When I emailed Sobi to ask how it arrived at 8,000, the reply submitted review from Nature Reviews nephrology summarizing data from Europe and the reply specifically stated that the French data set maybe a good anchor. We have anchored on the 14 per million in the bottom range. So 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 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, and of course, more recent large registry based data sets from U.K., 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 Aspaveli 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.

[Guido Oelkers, executive]

I mean, look -- just 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 there? And let's see, I think what you should just life is sometimes in pharmaceuticals, I'm not seeing more than 30 years, yes? 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 2 major consulting firms, very significant resources over the last 0.5 year with super experts and key KOLs to understand this market. Our Head of Market Analytics is -- said, this is one of the most difficult case of Epidata to understand. We will not share all our market insight as you may appreciate with the word. But one thing we can guarantee you is, 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 going to change. There's no effective treatment. So let's leave it there. We also can tell you 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 going to change, the way diagnosis is going to 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 think I can reassure, yes.

But I understand that this is a lot and but that's the reason, to be honest, why we have spent on this subject matter more than 0.5 year with probably a 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 rig, but we will not be 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 with regard to Elocta, I just wanted to say Elocta's growth year-to-date 6%. Yes. So this doesn't look like a product that is in dire straits. And therefore what I said also here with regard to 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 meets more than 50% have been switched in Germany, let's say for a lot for Altuvoct. 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, if you survive. I have a vested interest to explain this to you properly. And we have obviously not yet been able to do this. Any other question?

[Operator]

Yes, we do have a question coming from the line of Harry Gillis, Berenberg.

[Harry Gillis, analyst]

Just a couple of short ones from me. Just back on to Aspaveli, just wondering what your latest base case is for your relative market share versus Novartis that you're including in this guidance for blockbusters opportunity?

And then just thinking about the patient pool that is there today, is there 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?

[Guido Oelkers, executive]

Yes. I think, the -- let's start with the easy there are quite a number of I mean, we did some quite extensive market research. There's quite a number of patients that are waiting. There's also a number of patients that are currently quite desperate and not getting adequate treatment. And so there will be a faster uptake and we are currently for a certain group of patients and then there will be -- 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 the data in particular era reductions, our assumption is that we would have a larger share than Novartis.

With all due respect, given the absolute amplitude and the effect that's our belief, obviously not underestimating that they are formidable competitor. And we look forward to the discussions at ASN. And let's say, so we are quite and with regard to the financing, I'd like to refer to Henrik.

[Henrik Stenqvist, executive]

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

[Guido Oelkers, executive]

Yes. So I think what we do is, so apologies if we were a little bit extensive. And let's say, we can if you have further questions, please do refer to Ravi Rao. Either we make meetings available separately or otherwise we can answer this. So as you can see, we are quite excited about this data. And because it's not just the Beyfortus story, it's carried by our broader portfolio.

And we look forward to update you. The next update, big update is on Tuesday when we have a call on C3G, IC-MPGN 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.

Gerard 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.

[Operator]

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