Ladies and gentlemen, welcome to the Q3 2023 report conference call and live webcast. I am Sandra, the Chorus Call operator. I would like to remind you that all participants are in listen-only mode, and the conference is being recorded. The presentation will be followed by a Q&A session. You can register for questions at any time by pressing Star and One on your telephone. For operator assistance, please press Star and Zero. 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.
Thank you so much, Sandra. Hello, everyone, this is Guido Oelkers, CEO of Sobi. We are delighted to welcome you to the third quarter 2023 conference call for investors and analysts, and as you can take from our previous announcement, we are pleased with our performance in 2023 so far. We posted this presentation to sobi.com earlier today. Please turn to slide 2. Forward-looking statement as per usual. We would like to remind you of those provisions in line with normal procedures. Unless stated otherwise, we will be making comments that mostly relate to the third quarter performance in 2023 at constant currency rate and in million SEK. Please turn to slide 3. Today, we will cover the key aspects of our quarterly report.
I'm joined by Henrik Stenqvist, our CFO, and Lydia Abad-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 the presentation first and then do a... around 2:00 P.M. Swedish time. For those on the phone, please join the queue, in the queue for questions by pressing Star One. We propose you ask only one or two questions at a time. Please turn to slide 4. We successfully delivered on our strategy in the last quarter. We saw a significant top line growth of 23%, 29% reported, and 11% for the first nine months, 19% recorded. This reflects a strong performance in both hematology, hematology and immunology, with a growing contribution from our launch medicines in all regions, primarily in the US and in international.
In addition, we received a strong contribution from Vonjo as well as Beyfortus. The stable growth of Elocta and Alprolix provides us with a solid basis from which we are expanding our position with our launch medicines. Hematology revenues were driven by the first full quarter of Vonjo, strong sales for Elocta, and continued growth of Doptelet, and the launch of Aspaveli. Immunology growth reflects strong Gamifant sales and first royalties of Beyfortus. We continue to build the leading rare hematology franchise. This quarter, we focus on the integration of Vonjo, which offers considerable strategic opportunities. Our R&D pipeline progressed with five milestones. We launched the Phase 3 B FREEDOM trial for efanesoctocog alfa, our Doptelet ITP studies for Japan and for the pediatric indication, and these are fully enrolled.
We announced positive phase two data for the pegcetacoplan, for pegcetacoplan in C3G and IC-MPGN, and nirsevimab received approval in the US. We have strong momentum in the Sobi business. We thus maintain our guidance that was raised in July. We continue to expect high single-digit growth and low thirties for the adjusted EBITDA margin. All four elements of the Sobi strategy contribute to this performance. Leading-- building a leading position in rare hematology. Our pipeline is progressing, our immunology franchise continues to grow, and our regional expansion contributes to the overall growth and launch success. Please turn to slide five. We continue to be excited about the CTI acquisition, which has been the key focus of our company activities in Q3. The rights issue was fully subscribed, and we used the proceeds of SEK 6 billion to partially finance the acquisition.
Vonjo will be a highly accretive product for us in terms of revenues and margins. We now recorded the first full quarter of sales with SEK 347 million, which is an increase of 13% quarter on quarter. The integration has been going on very smoothly. This one was one of our focus areas. We successfully retained key talents. We continue with our thoughtful strategy to maximize Vonjo's potential and synergies beyond cost savings. We have ambitious near-term goals for our hematology franchise and see exciting LCM development opportunities. Please turn to slide 6. Let's look at our quarterly performance. Our growth of 23% was driven by both hematology and immunology, especially our launch brands. For the first nine months, revenues grew 11%.
If we exclude the impact from COVID-19 on Kineret and sales to our Chinese partner, Doptelet, we grew by 15% in a year-to-year setting, year-to-date setting on a constant currency basis. Both North America and International grew significantly in the quarter, while Europe remained at the same level of last year. In the first nine months, International North America grew a lot fastest, as you can see from this picture. Please turn to slide number 7. We have been pushing growth in two dimensions or along two vectors. Geographic expansion, extending the market coverage of our medicine, pipeline development, bringing new products to the market, extending indications. For the geographic expansion, we have streamlined our organization so that we are able to process multiple applications in international markets in parallel, prepare international supply, and can rapidly deploy our commercial capabilities.
Aspaveli, Doptelet, Zynlonta, Gamifant, and Vonjo are examples of our current and anticipated growth in new markets. For the pipeline growth, our recent reorganization in R&D and medical affairs gives us improved reliability and scalability to master new products. Efanesoctocog alfa, SEL-212 will be launched soon, and Beyfortus just have a rousing start in North America. We are working on expanding the indication for Aspaveli, Zynlonta, Gamifant, and... This puts Sobi into a new phase of sustained growth, driven by a lot of new products, all on the stable basis of our established franchise. Please turn to slide number eight. This growth strategy has led to strong results, which is now primarily driven by our launch products, launch and host products, or as we call them in the report, launch medicines.
89% of the absolute growth is derived from this part of our portfolio, which you can see in orange here. This reflects the strong momentum. It also shows that despite our growth rate, we are still have further headroom because there's still a lot of potential connected to these products from being early in the launch phase, or benefiting from further geographic expansions, or line extensions, or life cycle management. Please turn to slide nine. Our business continues to be underpinned by stable hemophilia growth at 4% in the first nine months of 2023. We expand the leading presence of Elocta and Alprolix to more patients in new territories. This confirms the community's confidence in these treatments and has been able to overcompensate for price developments that we experienced, particularly in Europe.
For Elocta, we continue seeing growth in patients, benefits from geographic expansion, and favorable phasing of deliveries. Government interventions compress prices in various European markets, while we see the positive evolution on consumption and patient expansion in Europe. As a result, Elocta grew by 4% during the first nine months. Alprolix was supported this quarter by phasing in the Middle East, slightly offset by unfavorable price developments. It grew 8% in the first nine months. Please turn to slide 10. Doptelet is up 15% in the quarter and 21% in the year to date. Sales growth was strong, driven by increased uptake in the US, ongoing launches in the regions Europe and International. In the US, there is a continued positive evolution of new patients, new prescribers, shares and duration of treatment. There's also an accelerated growth in Europe and International.
Key growth drivers were Spain, UK, Italy, and Germany. As expected, there were no sales of Doptelet to our partner in China in the quarter. Excluding sales to China in the third quarter, 2022, sales grew 50%-57% at CER in Q3 2023. We expect this positive trend to continue even without China sales. During the quarter, Doptelet has also become available in Japan. Please turn to slide 11. The launch of Aspaveli continue doing very well, with quarterly sales of SEK 169 million, continuing a solid launch trajectory. We have launched in Germany, the UK, France, and parts of the Middle East.... Australia, as well as Canada and more European countries. We also have early sales in a number of other countries, where reimbursement is starting to come in one at a time. Aspaveli is one of our candidates for new indication.
Here, we have just released positive top line data of the phase two NOBLE study. This is a very encouraging sign for our phase three program, and we expect it to read this one out next year. Please turn to slide number 12. Immunology is also doing very well, with a growth of 27% in the quarter and 9% in the first nine months. Gamifant, we saw in the second quarter of significant improved Gamifant sales. This is the result of an enhanced go-to-market model, more experience with Gamifant, and the recent publication of new data. Physicians have gotten more comfortable with the benefit risk ratio of Gamifant and are increasingly prescribing it for their patients. They are doing this also for a longer period of time. All of this has contributed to a very encouraging quarter for Gamifant, with growth of 112%.
As in Q2, more than half of this is from new patients. There is some volatility between the quarters because of a large number of high dosing patients, which depends on the patient's body weight. Kineret is now, let's say, flushing out the COVID effects and returns to normal growth as there's a more elevated interest in the IL-1 mechanism. Growth is driven by the U.S. and international. Sales for the first nine months are down 3%, but are up 5% when excluding the COVID-19 effect. Please turn to slide number 13, and now is the time to hand over to Henrik, our CFO.
Thank you, Guido, and hello, everyone. So please turn to slide 14, and we go to the key financials for the quarter. So Q3 was another very strong quarter with solid business performance. Looking at the bars to the left, we see the consistent trend in hematology, where the contribution to growth, not only from the addition of Vonjo, but also from hemophilia and Doptelet, outside of China. In immunology, we are glad to deliver another very good quarter for Gamifant, while the Beyfortus royalties more than compensate for lower synergies sales, for which we see a decline in Q3, impacted by the late start of the RSV season. So back to the table, revenue reached almost SEK 5.2 billion, and that was the highest Q3 ever, as reported in SEK, and this was driven by the underlying performance, but also benefiting from currency tailwinds.
Reported growth was 29%, and that corresponded to 23% at constant currency. The adjusted gross margin in the quarter of 78% is a slight improvement compared to the same period last year, and this is mainly due to the absence of the low margin sales of Doptelet to China in the quarter, and also FX effect. The adjusted EBITDA margin reached 30%, slightly below last year on 31%, and this is explained by the increase in operating expenses in the quarter, driven by, first and foremost, the addition of the CTI business, which was not there before. But also in marketing and sales, higher spend related to the launches of Doptelet, Aspaveli, and Zynlonta, and the pre-launch activities for efanesoctocog alfa.
In R&D, the increase relates also to volume activities, but also to the indication expansion of Aspaveli and the phase 3b trials on the efanesoctocog alfa. The non-recurring costs or items affecting comparability of SEK 102 million in Q3 relate to the ongoing integration following the CTI acquisition, and we expect some further non-recurring costs in the coming quarters, but at lower amounts. For details on items affecting comparability in the quarter, please see page 3 in the Q3 report. Operating cash flow in the quarter was just above SEK 1 billion, 42% higher than the same quarter last year, reflecting an improved working capital. The net debt at the end of the quarter was SEK 20 billion, a reduction from SEK 27 billion at the end of Q2, reflecting the completion of the SEK 6 billion rights issue in Q3.
If we go to slide 15, we have an illustration of our recent debt and leverage development, resulting from the financing of the $1.7 billion acquisition of CTI. This acquisition was initially all debt financed and brought net debt up to SEK 27 billion at the end of Q2, and this was equivalent to the net debt to EBITDA ratio of about 3.7 times. In Q3, the SEK 6 billion rights issue and the underlying cash flow brought net debt down to SEK 20 billion and leverage to about 2.7 times. And this is a comfortable takeoff point for further deleveraging and further expansion.... Please go to slide 16. We now go to the financial outlook for the full year, as usual, for revenue growth at constant exchange rates and adjusted EBITDA margin.
We confirm the guidance for the full year from Q2, and that is that revenues are anticipated to grow by high single-digit % at CER, and that the adjusted EBITDA margin is anticipated at the low 30s % of revenue. This outlook is, of course, a reflection of the year-to-date performance and our continued investment in launches and pipeline acceleration. Needless to say, the main uncertainty to our forecast is what will happen to the RSV season in the short term and how that will play out for synergies and our royalties from Beyfortus. We believe this could be both an upside and a downside risk to this forecast. With the outlook covered, I will now hand over to Lydia. Thank you.
Thank you, Henrik, and hello, everyone. So let's start with the pipeline milestones on slide 18, please. Thank you. So we hit five key R&D milestones for our latest stage pipeline this quarter. In July, the first patient was dosed in the hemophilia efanesoctocog alfa FREEDOM trial. This is the first phase IIIb study we conduct after the ones that are required for filing, and it evaluates both changes in physical activity patterns and long-term joint health, which are pressing concerns for people with hemophilia and for healthcare providers. Also, in July, the Doptelet Japan study in immune thrombocytopenia was fully enrolled. This study is required to file Doptelet for ITP in Japan, which is planned for 2024. Later in August, we completed enrollment for the Doptelet pediatric study in ITP, which is a global phase IIIb study.
Together with our partner, Apellis, this month we announced positive phase II data for pegcetacoplan in C3G and immune complex membranoproliferative glomerulonephritis. The data will be presented in early November at the Annual Congress of the American Society of Nephrology Kidney Week. The results show that pegcetacoplan is clearing the deposits that are causing kidney damage and may block further damage from occurring. The study also show improvements across key clinical measures of kidney function. This data give us confidence for the ongoing phase III VALIANT study, which will read out next year. And finally, the FDA approved Sanofi's and AstraZeneca's Beyfortus for the prevention of RSV in early July. Please turn to slide 19. As Guido said earlier, we do have a rich pipeline with a lot of expected news upcoming.
Most significantly, we are preparing for the submission of Gamifant in secondary HLH macrophage activation syndrome in Still's disease in the US. It is a rare, life-threatening complication of a rheumatic disease with poor prognosis. Gamifant operates at the cutting edge of science and thinking evolves with growing experience. During our ongoing conversations with FDA, we were recently requested that longer-term safety and efficacy data on secondary HLH MAS to be included in a supplementary BLA filing. We expect to cover this with the available data from our ongoing study. I would like to highlight that in October, we also completed enrollment of the full cohort 1 in Still's disease in the Emerald study. We intend to use the data from this full cohort in the filing, so including the total of data from 25 patients.
This allows for a more robust and complete data set to be included in the supplementary BLA filing in 2024. We also were able to accelerate the Kineret regulatory process for sJIA in China, which we now expect by the end of this year. Please turn to slide 20. With that, I hand back over to Guido.
Thank you so much, Lydia. As you can sense, you know, we are really pleased with Sobi's development during this quarter and the first nine months. You know, just to reiterate, we saw significant top-line growth of 23% at constant and 29% reported in the quarter, 11% at constant, and 19% reported for the first nine months. This is due to our steady base in hemophilia, as well as our expansion position in launch medicines that contributed over 89% to our total growth first nine months. Our R&D pipeline progressed with our key milestones for efanesoctocog alfa, Doptelet, Aspaveli, and nirsevimab. We have a strong momentum in the Sobi business, and we thus maintain our guidance that we raised in July. Please turn to slide 22. We now go to the Q&A session.
We will. For those on the phone, please re-remember to press star one to ask questions. Can I please remind everybody to limit questions to one or two, to be fair to all callers? Thanks in advance. Perhaps it's now time to take the first question.
... The first question.
Operator, maybe you can-
The first question comes from Gonzalo Artiach from ABG Sundal Collier. Please go ahead.
Hi, can you hear me?
Yes.
Great, great. Thank you very much for taking my questions. The first one is on Beyfortus. I was wondering if you could give us some color on how to look at the potential royalties from this drug in Q4. I mean, for synergies, we have seen historically that from Q3 to Q4, there has been a high jump in sales due to the RSV season. Should we see Beyfortus in the same way or the number reported in Q3 somewhat showing hospital stocking for Q4? And if so, why it would be different from the trends we have seen historically with synergies?
Yeah. Thank you so much for your, for your question. I mean, you, you have seen probably some announcement of CDC that is a little bit worried, you know, about the availability of stocks for Beyfortus in a broader setting. There's a high demand for the product. Due to competitive reasons, we cannot exchange information with them on Beyfortus, so I think this is mostly really a question that you should ask Sanofi. We can give you a take that we think, you know, for our synergies, given the constraints that were outlined by CDC, we think that we would probably have a more normal season.
But, you know, please remember that season started this year rather late, so, you know, so there's still some uncertainty related to synergies performance, as Henrik pointed out. But Beyfortus, you know, it's, it's obviously doing extremely well. And, and, you know, the RSV season, while it has started very slowly, is now becoming more epidemic in many more states in the U.S. So, you know, you would-- but we cannot comment on the, on the stock situation at Sanofi.
Great. Thank you. And a second question is on the guidance. You did not upgrade it. You're maintaining the expected top line growth at high single-digit%. So my question here is, having in mind the positive numbers seen in the Q3 report today, could you give us some words on the trends that you are currently seeing in Q4, which make you believe that the top line growth guidance should stay as it is now? Thank you.
Yeah, thank you. I mean, you know, the... As usual, you know, Sobi is not known for being, you know, too forthcoming or too extravagant with regard to forecasting. We like to deliver what we forecast. We have increased the guidance in Q2. We have a little bit of uncertainty, you know, in Q4. And just to remember, you know, that we had a really very significant quarter last year for synergies and in Q4. And, you know, so it is a, you have to make this one up in the first place. We see, though, you know, if you take away the RSV part, we see a very strong momentum across all businesses.
We think that this is gonna continue, but we have a little bit of a question mark at this juncture. But, you know, it's not like we are lacking confidence. It's more that we don't want to be caught out because we cannot oversee all the elements. That's the reason why we left it, but we are confident that we can make guidance and with a bit of luck, maybe do better. Yeah.
Great. Thank you very much.
Thank you.
The next question comes from Charles Mayne from Morgan Stanley. Please, go ahead.
Hi, Charles Mayne from Morgan Stanley. Thanks for taking my questions. Two questions, please. Firstly, on VONJO, following the integration of the sales forces and the first full quarter of sales, could you talk to your expectations for the ramp of the product over the coming 18 months? How much improvement do you think you can drive by simply reaching a broader set of prescribers than CTI? Is potential contracting with Doptelet an advantage? Secondly, Q4 is always a higher cost quarter, but bearing that in mind, do you believe Q3 is a good guide for the OpEx we should expect into 2024, given CTI has now been fully integrated? Thanks very much.
Thank you. Yeah, let's go right into, VONJO and, and Doptelet. We think it's a strength to have VONJO and Doptelet, and we have also integrated the field force. I think this was an important step to have, you know, to get the more breadth and depth, for both products, which are very complementary and, benefit from a very, you know, big overlap in terms of target audience. We make sure that we don't forget the specificities of the product by a very strong medical team, and we think that the combination now makes us very competitive also, also with regard to new competitors, new entrants. Mm. We think that we will, see an improvement of ramp-up, pretty soon, let's say, and, you know, because, you know, the team has worked, you know, during this, period of discontinuities quite well.
I mean, there's still a bit of work cut out for us, you know, to, to bring, to get really fully productive, but we should have some low-hanging fruits and, and, you know, we, we believe that VONJO will be a very material product for us next year, with a very strong year-on-year growth versus what we expect for this year. We are not yet giving guidance for VONJO in detail, but, you know, you can expect, you know, a further acceleration in the next, by year, next year. There are significant acceleration. Yeah. We think it's, we are on a good way. I mean, the team is super motivated, makes us very attractive as, also as an employer to have two products in the bag in this very competitive space of hematology.
With regard to the OpEx line, maybe Henrik, you want to comment on this, whether this is indicative for the next year?
Yeah, well, when it comes to volume costs, then you're right, that, of course, it is now fully absorbed in Q3. And, you know, that's an indication that we don't expect the sales and marketing costs to be any larger really in 2024. But that goes for Vonjo and, you know, the rest of 2024, we will guide for at a later stage.
Yeah, so we are ambitious. We see significant growth opportunities. We have now seven products in the stable that are still experience really exuberant growth. We need to make sure that they can further flourish and become a much bigger part of our entire corporation and driving further growth. But, we'll provide guidance on OpEx soon. Thank you again.
Thank you very much.
Yeah. Thank you.
The next question comes from Eun Yang from Jefferies. Please go ahead.
Hi, this is Melanie on for Brian. Thank you very much for taking our questions. So the first question is, you know, for Beyfortus, you mentioned that you mentioned that there's some shade. So maybe it's not for this year before, you know, the 2024 to 2025 RSV season. Do you expect synergies usage to be minimal? And if not, how do you expect it would take for, in terms of... How would you expect to replace before synergies. Thank you.
Thank you. Yeah, I think, you know, the, we told you tha you know, already in Q2, that what we are expecting with increasing usage of Beyfortus, we expected more confidence is built up for the product. And provided that their safety profile stacks up to what they have demonstrated in the clinical trials, then that should also have an impact on us. Yeah. Now we are, let's say, with the latest guidelines or with the latest recommendation from CDC, let's see how you know, I think we are still confident for this season, that we have respectable sales. But, you know, for next year, you would see at one stage, but we believe Beyfortus could play a larger role, which will benefit us in a different way.
Then the big question is, obviously, will their progress outside of the synergies indication be so large that it will, you know, balance the negative effects it will have on, we expecting for synergies. There is a good case to do for this, but, you know, at this stage, we are not providing guidance. This is a question mark. But, you know, we obviously, the team around synergies is still very confident. It's working the physicians right now. You know, that will, but, you know, over time, you know, whether it's now 2025 or 2026, 2024-2025, the season 2024-2025 or 2025-2026, you will see obviously an erosion of synergies at a more significant scale. Yeah, that's clear.
For Beyfortus, what's the anticipated patent expiration, and when does Sobi's entitlement to royalties from Sanofi expire? Thank you very much.
Yeah. Excuse me. I mean, it must, it might be my computer, but I have difficulties to hear you, to be honest.
What's for Beyfortus, what's the anticipated patent expiration, and when does Sobi's entitlement to royalties from Sanofi expire? Thank you so much.
Yeah. It is, you know, we, we have a perpetual license, perpetual agreement that basically makes us participants, that we have announced. And this royalty agreement goes from 25% in the earlier years, in the early, in the first two years, to up to 35% on sales. And, and this is not expiring. As far as the patent of Beyfortus is concerned, I mean, it's, it's, I, I can't tell you actually off the top of my head, to be honest, let's say, when the, when the patent is expiring, and, we'll provide this, you know, by the, either later part of this call or, or tomorrow when we have the call in a broader audience. Yeah.
Thank you.
Thank you.
The next question comes from Christopher Uhde from SEB. Please go ahead.
Hi there. Thanks for taking my questions. Christopher Uhde from SEB. On the pipeline, Gamifant, you I guess... Okay, well, can you tell us about the response rate in Emerald? Was it consistent with prior clinical data presented previously? For efanesoctocog alfa, you previously talked about the possibility of a relatively rapid review. You've got the 180-day questions in two weeks by my calculation. So have you any sense whether your optimism there was well-founded? For my second question, Elocta, can you quantify the order phasing impact? Thank you.
Mm-hmm. Yeah. Thanks, Christopher. You know, the, we cannot probably provide you with too many details on the response rate now, because we are still in discussions with FDA, and there's not yet a publication. But maybe what we could do is, Lydia, you can give maybe Christopher a sense on where we stand with Gamifant and give him his, you know, you know, because the overall response it was quite good for Gamifant. Yeah, so we were quite pleased with this. But, you know, maybe we can give you a sense and also with regard to alpha.
So with regards to Gamifant, and what we had was the interim analysis of 16 patients. But what we have achieved, as I mentioned before, was the full recruitment of cohort one, and that's what we want to analyze now, because it will make much more sense to present a full cohort instead of only the interim analysis. But of course, I cannot comment on the data, and we will be communicating as soon as possible, the data from the full cohort. When it comes to efanesoctocog alfa, you are right. We received 120 questions, and we are ready to answer all of those questions. We are collaborating closely with our co-development partner, Sanofi, and we are confident that we have all the data to answer the questions that were raised by CHMP, so.
Yeah. You know, I mean, I think the pipelining or the... I think this was more related, not to Elocta, but more to Alprolix. You know, this is not at a material scale. Really, I mean, it's more to be, you know, to do, to give justice. Henrik, you want to comment on this, on the adjustment, potential adjustment there?
Christopher, I think the question related to the phasing?
Yeah.
Is that right?
Correct.
Yeah. Well, we, there is some fluctuations, obviously from international markets due to, to tenders that we have in some quarters, but not in all quarters. You know, in this quarter, about 50% of the growth relates to these new markets.
Okay, great. Thank you very much.
Yeah.
The next question comes from Susanna Queckborner from Handelsbanken. Please go ahead.
Hello, Susanna Queckborner, Handelsbanken. I'd like to follow up on Beyfortus. Has Sobi started to adjust the costs associated with the field force supporting synergies? And then also, since Q3 net financials were burdened by the bridge loans costs prior to finalizing the rights issue, how should we think about Q4 net financials?
Mm-hmm. With regard to, you know, Synergis, we have, I mean, in a prudent way, you know, managed headcount over the last 12 months. You know, and have now a smaller head, team, obviously, than we had, maybe two years ago or three years ago. That's okay. It still allows us to be competitive. For us, it's important that the team feels that we have trust and faith in them managing this, period. You know, they do, so they're very fired up. We don't anticipate now any short-term, changes to this. We'll obviously review this again, you know, in Q1, and on the strengths of any further feedbacks that we may obtain from the market.
With regard to the loans, maybe, Henrik, you want to talk about this?
The net financials in Q4, it's you know natural that it will be slightly lower. But remember that you don't get an immediate effect. There is a bit of delay in the impact, but it's going to be slightly lower than what we saw in Q3.
Mm-hmm.
Okay. Thank you.
Thank you.
The next question comes from Neil Alexander from Deutsche Bank. Please go ahead.
Hi, everyone, Neil Alexander from Deutsche Bank. Thanks for taking my questions. It'd be good to get your views on the pricing outlook for hemophilia and your hemophilia products. And then second question on Altuviiio. Expectations of pricing once it's launched, will it be similar to Elocta? Thanks.
Yeah. What we're expecting in the hemophilia field is that we have a more, you know, a price reduction, more to the low to mid-single digit, as we had traditionally. There may be the one or the other outlier, you know, that we cannot anticipate at this stage right now, that will force us to object, to respond, but overall, as a franchise, I think this is where, where we—I would peg it. with regard to Altuviiio , with the, you know, I think we will be able to take a premium in some markets, but in most markets it will be at the same cost of therapy. That's cost anyway. Thank you. The case really lives from, you know, expansion of market share. Okay. Thank you. Next question.
The next question comes from Victor Sandberg, from Nordea. Please go ahead.
Yeah, hi. Thank you for taking my questions. Victor Sandberg, Nordea. Yeah, a question here on nirsevimab also. So we've seen, as you said, you know, the CDC priorities in nirsevimab to high-risk groups here for mainly the 100 milligram dose. But I guess the 50 mg dose is still quite available, if I read the guide correctly, which I guess could be used for premature infants or anyone weighing less than 5 kilograms. So could you elaborate a bit more why, why synergies could be protected via giving that overlap? I mean, the fear here, I guess, from my feedback, is that you don't get the upside from the 100 mg dose in kids weighing over 5 kilograms, but you could get cannibalized by the 50 milligram dose-
Yeah.
Maybe the 100 milligram dose in high-risk patients or, or patients with lung or heart conditions. So I just want to get your, your flavor on that a bit. Thanks.
Yes. Yes, thank you. You know, there is still a, you know, we will do still a bit of consultation with the decision makers, because there's a bit of an ambiguity, because obviously, what we hear in the market is also that, you know, the people are, you know, adding two doses of 50 to compensate for 100. So it is, you know, but, you know, it is not... This is what we hear from the market. This is not official Sanofi. And, and let's say, but, you know, to what extent there is this ambiguity, and unfortunately, we cannot completely remove this ambiguity right now, because, you know, I think, you know, there needs to be further clarification from the agencies.
And B, there is then the question, obviously, how physicians are responding. I mean, we, we have done a survey, as we reported, I think it previously, and that indicates that, you know, in the up to 29 weeks, there's a large group of physicians that sees utility of synergies. Yeah.
Okay, thanks. I also had a question here on Still's disease, or at the Emerald study. Since the FDA required more safety data, is that positive somehow? Because before, you were a bit uncertain if the data package you had would be enough for the FDA filing and subsequent label extension. I just wanted to, yeah, understand more here with your interactions with the FDA, if you're more certain now that maybe the data you have is enough, if you get the safety analysis to the agency. Thanks.
Yeah, I think it's a, you know, we see this more as a, you know, the interaction, as a benevolent interaction, given the high unmet medical need. And, you know, if anything, you know, the data set that we are now collecting should give us much more confidence. Do you want to comment on the specifics?
Yeah, sure. I agree, Guido. So, we're talking about rare diseases, very small patient population. So the more data that we can present to FDA, the higher chances we will have to really support the benefit risk profile of Gamifant. So, for us, it's good to present more larger data, and for longer period of time. So, we see this as something very positive for us.
Yeah. If I guess, I'll sneak one question in. I think you hinted that you needed more time to communicate something around synergies with CTI BioPharma. Is that something you plan or something you have some more visibility around here? Thanks.
Yeah. On the, on with regard to the synergies, yeah, I mean, there is a, you know, we have not yet announced this because, you know, we are, we obviously reaped quite substantial synergies. But, let's say we want to get now the, the overall trials going for, you know, for indication expansion and also for further lifecycle management. But, you know, we will provide you with an account, but today it was unfortunately not yet today.
Okay, thank you very much.
Thank you.
The next question comes from Erik Hultgaard from Carnegie. Please go ahead.
Yes, hi there. Thanks a lot for taking my questions, and two, if I may. First, a bit on Vonjo. If you could provide some type of early feedback from what you're seeing with the GSK launch of Ojjaara. I know it's early, six weeks post the U.S. approval, but what are you seeing? I know there appears to be some type of overlap with patients that you addressed, but although Ojjaara is more focused on anemia. Then secondly, if you could provide some sort of feedback on how resilient synergies is. And I think given the sort of strong demand message conveyed by Sanofi, it seems to be a lot of use already.
Have you seen or what are you hearing in terms of switches to the forward season in your on-label population? That would be really helpful. And I guess that's sort of before the CDC recommendation on the supply constraints that was communicated.
Yeah.
Thank you.
Yeah, thank you, Erik, for your question. So let's... So with regard to VONJO and Ojjaara from GSK, I maybe, we, let's say, you know, basically, the big battlefield is, you know, we have a clear place in, in those patients below 50,000 platelets, irregardless whether they are anemic or not. They have now, for anemic patients, let's say, some support in the, in also in the guidelines. And, and basically, the battlefield is now, really what is more important is, is it the thrombocytopenia, or is it the anemia? When you look at mortality, you would think that, thrombocytopenia plays a more important role.
We think that based on the feedback, you know, with our-- we don't need to be, in this phase of our launch, we don't think that we need to be too clever. We have figured out that a lot of physicians were not covered. I think it's fair to say that CTI was more focused on academic centers. We basically, obviously, having now a much larger team, have an opportunity to go deeper and broader. We figure out that, you know, there is still an opportunity for many physicians who have not even heard about Vonjo, so we see this as an opportunity for us to make our claim. We think that the thrombocytopenia topic is of more serious concern.
Armin, you want to maybe comment on the, on the thrombocytopenia element?
Yes, thank you. Eric, thanks for the question. I think it's a very important one, because if you consider that VONJO is really helping those patients at 50,000 and below platelet count, and that literally cuts the survival time in half when you compare to higher platelet counts, or if, particularly if you compare to red cell counts, like, like anemia. If you also look into the papers, particularly the pivotal report on momelotinib, you see that their patients were in the range of 100-150,000 platelets per microliter, which is much less or severe state as the ones that we looked at for VONJO. The question is: What do the physicians want to see? Because there's also now a publication in Blood Advances, that VONJO also works well on transfusion avoidance and really on the anemia parts.
Now, the question is, if it also works on a much more severe patient group, isn't that a drug that you want to use, particularly if the other one has not that data, that they work also on the low platelet counts? So I think it's up to the physician to decide if they believe that one drug has advantages. But when it comes to the data and the facts, I think we are very confident that Vonjo has a very strong data set.
Yeah. We have not yet heard, you know, of a big shift in, we were yet last week in the US. In the US, obviously, there is a group of physicians that will embrace it. But, you know, we always said that the market is large enough. I think our focus right now is really on the more established checkpoint, as opposed to be too worried now about those who don't have patients yet. You know, that is obviously going to change, but we feel well equipped, cutting a long story short with the profile that we have.
With regard to synergies, we have, we, I mean, you know, obviously, as you protect the patients and apart from those patients that you're carrying forward, we have not heard so much of, of, you know, switching. Obviously, the product, let's say, of those patients, otherwise, you know, you, you vaccinate newly in the season. Let's say, with regard to synergies, so we think, you know, the in the most data points make us believe that we have, you know, yes, a shorter season in 2023, but more of a more normal like season. This is, you know, and obviously, we see the indication of shortage of supply at this juncture for synergies, you know, because we obviously also like to be beneficiaries of the nirsevimab stream.
For synergies, it's probably more of a sym-- or more of a positive than rather believe, you know, that, you know, the 1.4, 1.5% of patient population is where all these stocks of nirsevimab are going, yeah. I mean, that is, we think at this stage, you know, it is probably making more a point that there is, also based on the FDA comments, that we think that there is more clear pathway, at least for this till the end, for synergies than it may have been beforehand.
Great. Thank you so much.
Thank you. Next question.
The next question comes from Alistair Campbell, from RBC. Please go ahead.
Thanks very much for squeezing me in. Just a couple of quick questions. Gamifant, obviously, you've had two very strong quarters, the last couple of quarters. As we roll into Q4 and, more importantly, I guess, into 2024, I mean, should I be thinking that the current quarter run rate is a good base to be thinking of, or would you urge a bit more caution than that? Obviously, you've rightly called out volatility, so just a question on that. We're now obviously seeing Sanofi rolling out Altuviiio in the US, and so far it looks pretty strong and overall their hemophilia franchise is growing. Obviously, you know, you have differential royalties on those programs, so though that franchise is growing, is there anything specific we should think about, about the royalties you receive?
I mean, obviously, you've, you've delivered a good quarter, actually, in terms of royalties, but is there anything you'd, you'd call out there? Thanks.
Thank you. Yeah, no, with regard to Gamifant, I don't think we are yet at a point to say that, you know, the last 2 quarters are the new normal. Yeah, it's clear that what we are doing, you know, that this is not just serendipity, yeah. That, you know, that, let's say because, you know, the—I think what we, what we understand is, data and, you know, now the medical education programs that we are running and, you know, a newly composed team, with much more insights, is paying dividends. I think, is it already this? Clearly, it's an elevated level, you know. We see, we don't see now, we clearly have moved away from a Q1 situation, yeah, which was devastating.
let's say, so we think that we are on a much higher plateau, let's say, and basically are very confident that we can develop the business from here, particularly once we can publish also the Emerald data in its full. Yeah, I think this will be a big pull, also because if the patients, if physicians see more utility, and we have a couple of life cycle management programs right now for Gamifant in the pipeline, and one of them reading out mid of next year. Yeah, so that could also quite stimulate the debate quite a bit. So we think that we are quite positive in this regard. With regard to the royalties, you know, we think that Efa is a much bigger product, obviously, also for Sanofi.
Hence, you know, even though it's a differential royalty, it will be, when you look at the overall royalty stream, it should be good for us, yeah.
Thank you. Very clear.
You're welcome.
The next question comes from Liu Yifeng from HSBC. Please go ahead.
Hello, thanks for taking my question. Two questions, please. One is, so could you possibly give some timeline and just colors on the timeline of Altuviiio and the market and the market access in European countries and in the next year? And the second question is on your leverage, and obviously, you're coming down from 3.7 times to 2.7 times. Just in terms, how should we think about that going forward in the next few quarters? Thank you.
Thank you. Yeah, so we expecting to launch in Europe Efa mid of next year. Yeah, the first country, and it's no surprise that it's going to be Germany. Okay, there's holiday season, so we have to see, but you know, we are committed to bring this on the market as soon as possible because we have such a great demand for the product from associations, from patients that are individual as well as the healthcare professionals. And then basically, we will follow the flow. We had UK would have been also very fast. There's a bit of a queue right now with the agency, but you know, we should be able to resolve this also by end of year.
And then, you know, you have to, and then you have various early access programs and, and in various markets in addition, and, and so further on. So, you know, so full launch is really 2025, but, you know, you will see some, some significance, already through the course of 2024. And with regard to leverage, I can refer to Henrik. How do you see this?
Yeah, so the current situation after the rights issue is about 2.7 times. Obviously, what we foresee going forward is a further reduction due to our strong cash flows. You know, how fast the reduction is, we don't guide on, but we are creating, obviously, a headroom for further expansion.
All right.
Yeah. Thank you. Maybe we open up for the last question.
The last question comes from Christopher Uhde from SEB. Please go ahead.
Thanks for taking my questions again. So, yeah, just, could you comment on Gamifant? You, you did say about the average duration of therapy being longer, but, what, what are you seeing in familial and rheumatologic HLH, respectively? And Orfadin has also been quite flattish lately, despite generic competition, obviously. Is this the kind of run rate we should expect for the, you know, going forward for a while, at least? Thank you.
Yeah, I mean, maybe I talk to Gamifant and first, you know, with regard to Gamifant. Let's say we think that you know, we have a good growth path ahead of us. Sorry, Christopher, I didn't get probably this question completely right here. Can you get this repeated for Gamifant?
Yeah, sure. Just the average duration of therapy that you're seeing in,
Yeah.
Familial HLH and also rheumatologic HLH.
Yeah. So the duration is sometimes driven, obviously, you know, we have sometimes longer duration, particularly when these are heavier patients and they are, you know, in rheumatologic or conditions or, you know, in not necessarily primary settings, yeah, where the physician is making decisions. But, you know, what we see is also that the physician is dosing higher at the beginning because there's more confidence, and that basically also is impacting this. But, you know, we have not... I would say we don't have a good take on the data on duration, you know. But yes, there is a, there's a longer duration and there's also more units that are deployed. Yeah.
Okay, great. Thank you.
That's basic. Yeah, and with regard to Orfadin, yeah. I mean, you know, it's not, you know, as core anymore to our business, but, you know, we have now reached a point where we can effectively compete, and therefore it's a little bit more of a steady state, yeah.
Okay, great. Thanks very much.
Thank you. Armin, you have a point?
Yeah. Maybe, Christopher, just to give you a little bit the perspective, because those patients are so different and so difficult. Literally, ranges that we have seen are from 4-245 days, averaging at roughly 60 days. But, that is probably a difficult thing to say then, how does that translate to the large group? Unless we have more data and more to come with more experience. But that is just to give you a feel of what it could be.
Yeah.
Much appreciated.
Thanks, Armin. Thank you. Yeah, thank you so much for your interest. As you can see, we feel reasonably upbeat, you know. It feels quite nice, you know, to be at Sobi at, you know, quarter, quarter or two. The quarter growth of 23% doesn't feel so bad. Thank you for your interest in Sobi, and look forward to, for those who want to join us tomorrow to answer more questions in more detail. Thanks a lot, yeah. Wish you well. Thank you.
Ladies and gentlemen, the conference is now over. Thank you for choosing Chorus Call, and thank you for participating in the conference. You may now disconnect your lines. Goodbye.