Ladies and gentlemen, welcome to Acquisition on CTI BioPharma conference call and live webcast. I'm Andre, the Chorus Call operator. I would like to remind you that all participants will be 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 one on your telephone. For operator assistance, please press star zero. The conference must not be recorded for publication or broadcast. At this time, it's my pleasure to hand over to Guido Oelkers. Please go ahead, sir.
Thank you so much and welcome everybody. Thanks for your interest in Sobi and our latest activity in our effort to acquire CTI BioPharma Corporation. We would want to talk a little bit more about this deal and lead you into our rationale. Before I do this, please take note of the forward-looking statement as per usual. On the next page, you know, you find important information with regard to this potential transaction. I just want to draw your attention to the fact that we have signed a definitive agreement, but obviously the deal has not been closed. With this said, you know, please move to the next page.
You know, who is going to share, you know, their sixth senses and yeah, their views on this deal is first myself, then followed by Tony, our new Head of R&D and Medical Affairs, and then Henrik, you know, will talk about the financials of the transaction, our Chief Financial Officer. Let's move to the next slide. You know, and here you can see a little bit, you know, in a nutshell, you know, why this is we believe a marriage in heaven, why this is such an important deal for Sobi. As usual, it starts with the patients. You know, just think about myelofibrosis.
These are 78,000 patients and worldwide, you know, 21,000, give or take, in the U.S., of which one-third are severe, you know, with a very poor prognosis, less than two years of survival. When you go to the left, you know, you see VONJO, it's a differentiated asset. It's a differentiated asset by this, by the virtue of pharmacological differentiation, meaning it's active at the JAK2 level and it's JAK1 sparing, and it is differentiated on the strengths of clinical data that Tony is going to share with you later. For us, it's an exciting asset because it speaks to our strengths. It's very complementary to what we are doing in hematology already, particularly with our TPO-RA. It basically is really about, you know, how one plus one could yield more than two.
We feel very excited about, you know, the expertise also that comes with this asset. This asset is going to be highly accretive in terms of revenues and margins in the near term. Let's go to the next slide. Who is CTI? I mean, CTI is a company with 144 employees, founded in 1991, is listed on the Nasdaq. You know, the key asset is VONJO, the JAK2 and IRAK1 inhibitor. It's approved in the U.S. and got a fast-track approval and, let's say, you know, based on the spleen volume reduction that Tony is going to show you the data later. An ongoing phase III trial, PACIFICA, and VONJO is the only agent specifically studied for cytopenic myelofibrosis. Next slide.
It's obviously, you know, it's a super fit to our strategy. I mean, it's quite intuitive. You know, it connects to our ambition in hematology because it addresses a severe unmet medical clinical need. It is about bringing this product global. Obviously, the U.S. is the most important part of this business, but given our strengths also beyond Europe and the U.S., we clearly want to take this product internationally. There are some markets where this can be done in a relatively quick sequence. We think that we want to take this product beyond the current indication. You know, there are also some ideas in this regard. Next slide, please. What is the rationale of this transaction? First of all, I talked about it's about the asset. Clinically differentiated, pharmacologically or by mode of action differentiated.
We like the team, you know, the team that who we have met as part of this diligence. This is a very highly skilled workforce in the area of hematology-oncology, and we think that this would do us extremely well, you know, to bring in this more scientifically medically oriented knowledge into our company. The product and the company is going to be a very significant accelerator of our growth prospects midterm because first of all, it will accelerate our U.S. business and as I said, you know, connects very well with what we are doing today. We want to take this product, and it's no surprise, international. The stepwise approach, we want to take also this product beyond current label.
Because we are in adjacencies in terms of commercialization, but we also think that we understand it medically and scientifically, is I'm convinced that we can be confident about impactful execution. Next slide. What it does is it takes really our ambition for hematology even broader. You know, on the left, our hemophilia franchise, and hopefully very soon with our new extended Factor VIII product, ALTUVIIO. You know, we are in the midst of large preparation for it. In London, they have already first patients in the Middle East, obviously it will add a very important medicine as described.
With this said, I would like now to refer to Tony, who will bring this product a little bit more alive from the disease area, but also from a mode of action and clinical perspective. Tony.
Thank you, Guido.
Yeah, right.
Here we go. What I would like to do first is to talk a little bit about myelofibrosis. It's a rare disease, obviously a rare form of bone marrow cancer, which has very, very serious impact on patients. Guido shared that with you. The best data we could get, it's about 78,000 patients worldwide. In the U.S., we have a substantial number, pardon me, about 20,000 patients. What it does, really, it leads to impaired blood cell production, and as a consequence, of course, we have anemia and thrombocytopenia. That plays a particular role, perhaps in later discussions also, how this product is differentiated. Myelofibrosis is often characterized by an upregulation, where we have basically a JAK-STAT target genes of a JAK target genes.
Again, most importantly, the severe thrombocytopenia in these patients is an important factor of this disease. We know patients who have severe thrombocytopenia have a pretty poor overall prognosis. You, you see here we have some data referenced at the 15-month point. What we can see, having talked to quite a number of experts, having looked at the field in quite some depth, there's a very large unmet medical need. There's super limited treatment options available, in particular for patients with this severe thrombocytopenia. If you go to the next slide, again, we can highlight that a little bit. It's about 30% of these patients who suffer from quite low platelet counts. These low platelet counts, when you look at the available therapies, there's really only one that's indicated for less than 50,000 platelet counts, and that's VONJO.
Again, this is directly linked with the prognosis if you have a really effective therapy available here. The clinical manifestation of the disease, we can talk a little bit more about that in the Q&A if you like, is this very, very large increased spleen. You know, when you think about a spleen that has a super increased size, that has all sorts of complications, whether it's problems with breathing, problems with eating, and. There's a very significant and very clear endpoint, by the way, also for clinical studies. As I said before, many of these patients who have very low platelets, they wouldn't be eligible to be treated with anything else. Because, one, there is pretty much nothing that's labeled for these low platelets.
Two, because of the mechanism of action, many of the available therapies actually have a negative effect on platelet counts. That's where we believe we have medically a truly differentiating asset and medication that can make a true difference for these patients. If you go to the next slide, we can talk a little bit about why this mechanism is so different. Here we have a drug that does not inhibit JAK1, and that's actually a very important aspect from a pharmacological perspective, because when you spare JAK1, then you avoid drug-induced myelosuppression, like some of the competitor drugs definitely do. Then we inhibit JAK2, and that actually has the potential to reduce these or many of the myelofibrosis-related symptoms. There's two other pathways that play a role here, IRAK and the IRAK pathway and also targeting ACVR1.
What we know, there's a lot of recent data out there that both of these pathways may have a beneficial effect regarding myelofibrosis, in particular to anemia and myelofibrosis-related symptoms. In combination, this sparing of JAK1, this inhibition of JAK2, and really having an effect on IRAK, IRAK1 and ACVR1, we believe is a pretty unique differentiator from the pathophysiologic perspective. And as we said, that has also led the FDA to give the company this license, and it's the only one that's licensed below 50,000 platelets. That being said, let's move on to the next slide. Here we go. Here is data that was pretty relevant for the license of this product. You see here, on these two charts or on the left-hand side on these two bars-
You see that we have patients, the percentage of patients with platelet counts less than 50,000 that achieved more than 35% spleen volume reduction. This is clinically and medically extremely significant. You see pacritinib was compared to the best available therapy, and best available therapy is a large number of options. None of them are really making a major difference, at least in my view. You see what difference that has made on a clearly measurable, objective endpoint that can be verified easily.
Right.
The other question that's very important here, have we seen any alarming, any critical safety signals? The answer is no. We have not seen that. We believe safety is very manageable, from what the FDA has reviewed, what we have reviewed. As we said, we have here probably the only JAK inhibitor that demonstrates a really meaningful difference in these low platelet patients. That we are very confident makes a lot of difference, both for the patients and for the company, in combination with everything else we get here, including skills and the ongoing evidence generation. That being said, I think I will turn it over to Henrik so that he can talk a little bit about the other aspects of the deal.
Thank you, Tony. Regarding the transaction terms, you will have seen that this is an all-cash offer of $9.10 per share with a transaction value of $1.7 billion on a fully diluted basis. In order to work out the math here, it's obvious that in addition to the basic shares outstanding, there is also options and convertible notes corresponding to about 53 million shares to be added to the fully diluted number of shares. In terms of the funding, it's a 100% cash acquisition funded by committed debt financing to carry out the transaction. This financing has been provided by Bank of America and Danske Bank. We anticipate to fund up to half of the transaction via an equity issue, a rights issue, post the acquisition.
Our main shareholder, Investor AB, has undertaken to subscribe their share of such issue. We expect to have a leverage ratio of below 3 x after completion of the rights issue. This together with an expected additional strong cash flow generation from this acquisition will support a rapid deleveraging and preserve our financial flexibility for future growth. In terms of the financial benefits, this acquisition is expected to be a significant accelerator for our top-line growth, but also be accretive to our adjusted EBITDA margin already near term, that is in 2024. This is an acquisition driven by the growth potential in hematology and through the addition of complementary reach and expertise, and it's not primarily by cost cutting.
As in every combination of companies, there will be overlaps and potential to realize efficiencies, that is something that we will come back to. In terms of the timing, we will shortly initiate a tender offer with the purpose of acquiring all outstanding shares, we expect the transaction to close in Q3, subject to customer closing conditions, including HSR clearance. If we go to next slide and look at an overview of the financing. This is, as I said, fully committed bank financing consisting of long-term bank debt with an equivalent to $2.9 billion and a short-term equity bridge of SEK 8 billion. The equity bridge as said to be refinanced through a rights issue. The long-term debt will be syndicated in the bank market.
With this setup, we keep our strong balance sheet in order to be able to continue the strategy of growth of the company. I give back to Guido.
Thank you. Henrik, you know, let's round it off with the last slide. Next slide, please. In conclusion, you know, what do we like to communicate? You know, obviously differentiated asset clinically, but also by the virtue of mode of action in a highly unmet medical need area. It's we are looking forward to working with a highly skilled workforce in the area of hematology-oncology and bringing them in. As, you know, Henrik pointed out and, you know, and I mentioned, this is all about acceleration of Sobi's growth in our core business, and it is, you know, really making sure that this will stimulate our midterm growth ambition significantly and materially. It is about obviously the fact that we are confident that we can execute this transaction.
We need the help, obviously, of the CTI colleagues or employees at the right time, and we can work with them and, you know, we will try, You know, entice them to be part of Sobi, and we think that this is a financially attractive deal for Sobi and, you know, that this will be also recognized over time. Yeah. With this said, I think we can now go into the Q&A. I understand, you know, that there's already, you know, an order of certain questions that have been registered, you know, prior to this call already. Jennifer, you know, I think the first one was Yun. Yeah, is this correct?
Mm-hmm.
Yeah, Yun, maybe you fire off and then, Jen, you guide us through the order because we have some difficulties to see this here. Yun?
We will now begin the question and answer session. Anyone who wishes to ask a question may press star and one on their touchtone telephone. You will hear a tone to confirm that you have entered the queue. If you wish to remove yourself from the question queue, you may press star and two. Questioners on the phone are requested to use only handsets while asking a question. Anyone who has a question may press star and one at this time. The first question comes from the line of Eun Yang with Jefferies. Please go ahead.
Thank you. Two questions. One is on the patent life. Looking at the competition around patent life, it seems like in the U.S., it starts expiring in 2028, outside the U.S. starting in 2026. I would like to ask you what your assumptions for market exclusivity are understanding that it has orphan designation. The second question is, CTI has about 144 employees currently, I think it's based in Seattle. Are you planning to keep that facility in Seattle with the employees? Thank you.
Hi, Eun. Thanks for the questions. You know, regarding the patent life, we think that we see on the strength or by the virtue of the NDA submissions that they have, that we can look forward to a patent protection till 2034. With regard to the location, you know, we will have discussions with the team, what makes sense and what is a sensible setup. You know, we clearly excited about the team and, you know, and at this stage, obviously, we have not been able to be in discussions with the organization. You know, as I said, you know, our base assumption is to make sure that, you know, the team, you know, feels comfortable and welcome at Sobi.
you know, and this will really depend now on the forthcoming discussions. I don't wanna speculate. Eun?
The-.
Thank you.
2034 is in the U.S. and both the U.S. and outside the U.S.
This is, in the U.S., That's what.
Thank you.
That's base. You're welcome, Eun. Maybe next question.
The next question comes from the line of Christopher Uhde with SEB. Please go ahead.
Hi there. Thank you very much for taking my questions. Got an awful lot, so I'll ask a couple and then get back in the queue. The first question is sort of on financials here. I haven't seen you... You know, you mentioned sort of short or, yeah, near term synergies. I haven't seen anything about the impact on the outlook for 2023. What can you say about that? Secondly, would you please help us quantify the level of synergies you see short term and long term? Let's say, to be specific, coming year and then over the longer term.
For the second question, in terms of the Trexel transaction structure, why was it a rights issue that was picked instead of a directed share issue? Then, you know, as it relates to that, would it be correct to assume these will be preferential rights? Would you convene an EGM pre or post-deal? I'm just trying to get a sense of when this will sort of stop being a question in the equity story over the, you know, short term. Thanks.
Yeah. Thank you, Christopher. Maybe I'll start, you know, and then I refer some of the others, you know, questions to Henrik. You know, basically with regard to the outlook of this year, the reason why we haven't provided this is simply because, you know, it will depend obviously on the closing. We otherwise, we don't think that there are a lot of FTC issues to clarify. You know, we didn't want to speculate either. I mean, you know, everybody I think has seen the consensus for the franchise. It's not gonna be, it not gonna be a miracle to figure out when we have the right, you know, how much we are going to consolidate this.
You know, we are very comfortable with the guidance that the organization has set out. With regard to the, you know, to the growth, you know. You know, I think when you know, as we told you, that we think that we can pull three levers. You know, first of all, we think it's very synergistic to what we're already doing in the U.S. We think that we can further accelerate this, given also our more commercial focus on businesses and our focus on further medicalization. We think that there's a lever to be pulled, you know, in the midterm with regard to further internationalization, and we clearly want to broaden the product beyond the current label.
We think that, you know, when you think about, you have seen consensus, how it's playing out. Already at consensus, if you overlay this with your own expectations on Sobi, it's a very material accelerator, you know, for the years to come. If you pull some of these levers, you know, we hope that this, that we can further stimulate growth for the group, so making Sobi a really attractive group and truly growing into the size of our aspiration, becoming a leader in rare disease. Now, with regard to the cost synergies, that's correct. We have not quantified this and we won't today. Simply because, you know, we want to get to know the team. As I said, you know, this deal is about acceleration of growth.
It's much less, you know, about cost, but there will be some efficiencies, I think is worth saying. You know, it is about stimulation of growth, so one plus one can be more than two. You know, at the right time, we will, you know... Please bear in mind, you know, we don't own the company. Yeah. I mean... It is You know, we are still, you know, we have just signed an agreement, and we need to wait for closing. It would be also very premature to do this at this stage. With regard to the transaction structure, I like to refer to Henrik, who will talk to you about the merits of a rights issue.
I personally like it a lot that our main shareholder has committed with his share and equity contribution, it shows confidence in the transaction. Maybe, Henrik, you talk about this and, you know, and how we arrived at this at this juncture.
Yeah. Yeah. As Guido, you know, says, the equity issue will be a preferential rights issue for existing shareholders, which is the standard way of raising capital for Swedish public companies. We will initiate that post-closing of the transaction. Yes, we will call for an EGM in due course.
Yeah.
Okay. Thanks very much.
Thank you. Thank you. Maybe next question.
The next question comes from the line of Erik Hultgård with Carnegie. Please go ahead.
Yes. Hi there. Thanks a lot for taking my questions. I also have quite a few, but I will shoot a couple and then get back in the queue. First, I was wondering, maybe for Tony, your confidence level in hitting the primary endpoint in the confirmatory phase III study to get the full U.S. approval. I know the study is powered to show a 16% difference compared to physician choice on SVR. And based on the phase II data, it looks like you have a margin of error. But I was just wondering if there is any meaningful differences in baseline characteristics in the phase III study compared to the phase II. And also differences in physician choice that could impact the SVR rates in the phase III study. That's my first question.
Then maybe a question for Guido on the potential for early approval in Europe that you see, or if you need to wait for confirmatory data at the end of 2026 before submissions. Thank you.
Thank you, Erik. Tony.
Yeah, I'm happy to take the first question. Thank you for asking that. When we look at the PACIFICA trial, that's what you're referring to, we have two co-primary endpoints. One is, as you said, the spleen volume reduction, greater than 35%. As you said, it's an objective one, so that's MRI or CT measured. I think we will have no issues there, and I'm gonna talk about the power in a second. Secondly, we have patient-reported outcomes there, where we want to have a clear benefit. Now, when we look at the power of the study, we've looked very carefully and checked kind of which data they have from prior experience. We believe the study, if anything, is very well powered to hit both of these endpoints.
The other piece that plays a role here, how can you make these objective? Spleen volume reduction is clear, right? That's MRI or CT. Then patient-reported outcomes is something where there will be an external or an independent, if you want, interview who will verify these reported outcomes. From a power perspective, given on the patients that we've been able to analyze beforehand, we believe we are in very good shape for both of these endpoints and also from a perspective of making these very objective. The other piece that we have to keep in mind, which was probably also part of your question, this is a large international study with many international centers. There may be slight differences in baseline therapy or in best available therapy.
As far as we could study and look into what's available, we do not see anything that might even come close in the effect size regarding these endpoints. That's the best answer I can give you at this stage.
Yeah. Erik, with regard to the potential approval in Europe, or early approval in Europe, I mean, we, you know, we have done obviously quite a bit of diligence on this. You know, there are limitations to how far you can go in a diligence process. You know, basically, you know, an assumption is right now that obviously the PACIFICA trial would be useful. Having said this, you know, I mean, there's a high unmet medical need in the United States, but there's a high unmet medical need also in Europe for patients with myeloproliferative. You know, particular when they have a platelet count below 50,000. The same applies. We would feel very encouraged once this transaction has been closed, to engage with the European regulator and explore those.
There is nothing that we could already, you know, announce at this point of time. You know, Tony and his team and our regulatory obviously experts reporting to Tony will be engaging with the EMA, and then we will come back to you.
Great. Thank you.
Yeah.
Just a quick follow-up on that. If I'm properly informed that the extension of the patent has been applied for but not granted, and it will take like 18- months until you know for sure if it's granted or not. Are you planning to invest and start large registrational studies to widen the indication before you get the clarity on the IP?
Yeah. I mean, we have done a thorough IP check. We are, you know... There is no guarantee, as you said, but there's a lot of confidence, yeah, around the IP. We have looked at this. I'm sorry we have a bit of background noise because, you know, we couldn't stop here the blinds. Basically, you know, the, so we don't think that there's any showstopper. We will, you know, regardless of the approval, final approval, we'll explore now opportunities, you know, on how to internationalize this franchise further. You know, whether it's bridging studies into Asian patients and so on and so forth, we will update you at the right time.
Yeah.
Thank you so much.
What I.
Oh, sorry.
Yeah, what I can add here in response to your question, the first priority is of course, that we take full control over the ongoing study so that we really have all the details and can manage that extremely well. In parallel, we will start exploring what can we do outside of the current scope as you suggested.
Thank you. May we come to the next question?
The next question comes to the line of Charlie Mellert with Morgan Stanley. Please go ahead.
Hi there. Yeah, Charlie Mellert from Morgan Stanley. Thanks for taking my questions. Firstly, you suggest that about a third of the population are highly thrombocytopenic, which is a significant portion, and these patients have really poor prognosis. Could you please first talk to how CTI has progressed around building awareness of the disease as the first specific approval here, and any data points you can point to here. Secondly, could you also give some context around what proportion of these patients are currently receiving the allogeneic HCT and what proportion are not transplant candidates? Thank you very much.
Tony, you wanna take this, or?
I cannot talk to how the awareness was created by CTI in detail. What I can tell you is they have very many patients treated with the drug. Also, after the National Comprehensive Cancer Network had issued their guidelines on the full knowledge of the label. The treatment experience, the treatment pattern has been really now seen in many patients throughout the United States. Of course, through the scientific dialogue and the increasing data set, the confidence in the drug and especially the safety in the drug has been growing very readily and very significantly. We've personally talked to a number of external experts who have been using the drug, and again, the comfort level there is very high.
That's what I can say from the medical, scientific perspective. To the degree kind of who goes to transplant and who goes not, I don't have that data readily available. If I can get it during the call, I will share that with you.
Yeah. You know, and I think to be honest, let's check our stats and, you know, we will also look at it. You know, at this stage, I mean, what we can say, you know what I mean, just look at it. I mean, this product now is less than a year on the market. This has been a company, you know, with, you know, that clearly comes from a clinical science background, then try to basically become commercial with medical education. Obviously, they did a lot of efforts. You know, it's also, I think, fair to say that, you know, that, you know, this company also had to think about, a lot about their burn rate and how to spend money, wisely whilst, you know, committed to development.
I would not, you know, see that this is now indicative of what the product can do. It's more indicative of what the selling company and CTI could afford. I think that is something that we met actually at various point of times in the diligence process.
This is a company who is very skilled, you know, they are very skilled individuals, and I have deep respect for what they have built up. I think if everybody or many of you would also appreciate, you know, currently, funding for companies that are not profitable has not been easy in today's world. You know, and that basically must have driven also some of their decisions. We hope that, you know, by providing, and you know that the JAK inhibitor market is pretty large and, you know, the leading one is selling SEK 4 billion, which also, you know, and most of these sales are coming outside of the U.S., I mean, at least more than 50%.
There's a large opportunity and you know, and despite the limitation of JAK1 inhibition, you know, clearly products are used here. This is I think, where we see opportunities for Vonjo, you know, to when the message is more communicated that, you know, usage is broadened and the product becomes, asserts itself in its rightful place. I'm, so we, you know, we'll provide you with the stats at the right time. I think, you know, this is how you have to bring it probably into perspective.
Thanks very much.
Thank you. Next question.
The next question comes from the line of Peter Östling with Pareto Securities. Please go ahead.
Thank you very much. A couple of quick ones. When you look, Guido, I think you mentioned, talked a little bit, alluded to the current consensus for the drug. When you look at those consensus, do you feel that they are like a base case scenario or do you expect what kind of comfort do you have when you look at those top line?
I mean, my base assumption is that those aspirations drive taking away certain discounts, drive current valuation, yeah. We pay the premium, so if you want to, we want to, let's say create value for our shareholders, we need to find ways to create value beyond. That's my assumption. Therefore, let's say I would see them also consensus as a base, you know, we would feel encouraged to add value beyond.
Okay, great. I guess, I think that CTI mentioned that they had around 1,000 patients on the drug at the end of 2022, and I guess that most if not all of them belongs to the one-third bucket of the 20,000 so patients in the U.S., Is it so or, are there other patients above the 53 also treated today?
Yeah. That's correct. Yeah. They have actually quite a mix because, you know, the product has a broader utility based on the NCCN guidelines as Tony pointed out, where, you know, the patients up to 100,000 platelet count are covered. That is also reflected in the mix of patients, whilst obviously the company is strictly adhering to promotions and label.
Yeah. Do you know why-
Yeah.
Oh, sorry.
I just wanted to add something. You know, it's a very interesting phenomenon we have here, which highlights the huge unmet medical need. The NCCN issued guidelines that go beyond the low platelet count of 50,000 because they saw that there is such an underserved community of patients. That's exactly why Guido's answer was spot on. The experts treat patients beyond more or above 50,000 as well because of the benefit. It's a very interesting constellation where the scientific community is advancing very quickly and gaining more knowledge and broadening the use. At the same time, of course, we have to create the data as a company as well to go in parallel.
Okay. Do you know the PERSIST-2 study included patients with platelet count below 100,000? Do you know why the label ended up just covering patients below 50,000?
I can refer to the regulatory correspondence. Obviously, we haven't been part there. The highest benefit has been seen in patients below 50,000 platelet counts. We've seen a couple of other incredibly promising benefits, including no suppression of platelets and the likes, especially in this subset of the patients. That's, there was simply a benefit risk assessment by the FDA seeing the highest benefit in this patient group. Also realizing that there hasn't or up to today, there is no other drug licensed in this low platelet population.
Okay. Is the PACIFICA trial only including below 50,000 or is it?
Uh, the, the.
Common copy of PERSIST-2?
No. the PACIFICA actually, you're right. The PACIFICA trial includes patients less than 50,000. That's exactly what they did.
Okay. Okay. Okay. finally, As you know, one potential competitor to this drug will have a PDUFA date within a month or so. Momelotinib from Sierra Oncology and GlaxoSmithKline. Just wanted to hear your view on that drug and if it consists of a competitive threat or not.
Yeah. I mean, you know, I think you should never underestimate a competitor who is coming, and obviously GSK is a formidable company. We studied the available data extensively. We see that the specificity of VONJO to, you know, and the effect size for patients below 50,000 makes it rather unique, and it is more a JAK1 sparing from what we can see than the GSK compound. Tony, you want to comment on your view of the product?
Yeah. First of all, of course, right, we'll have to see how the regulatory process goes and how the data works out. When we look at the mode of action for momelotinib, we know that there is also a JAK1 piece there, right? Since we kind of have a unique differentiation there, also with some of the other pathways, as I described, at least from the pathophysiological perspective, we would see still a quite distinct advantage subject to verification, of course, as we go forward. I think pathophysiology, mode of action, would speak for us, and the rest we'll have to see.
Okay.
Yeah.
Thank you. Thank you, guys.
Thank you.
I guess back into the queue.
Thank you. Maybe the next question, if there's any.
The next question comes from the line of Alistair Campbell with RBC. Please go ahead.
Hi there. Thanks for taking the questions. Actually, I'm gonna start again with momelotinib, if I can. They've talked a lot about utility in patients that have anemia as a key differentiator versus the other JAKs that are approved. But also, you know, CTI put some very good data out at ASH last year on anemia and ACVR1. I'm just intrigued to know how you think you'll be able to utilize that to fend off momelotinib when it comes to market. Then just a bit of a follow-up on PACIFICA. I think CTI were having some recruitment issues. Just can you confirm when you think that trial is set to read out on its primary endpoints and also when you think it might read out for that secondary endpoint of overall survival?
Finally, can I just ask and just for clarification on the royalty situation, could you just clarify all the royalty pay ways that exist on the product, on VONJO? Thank you.
I mean, maybe I start with the first view at anemia. I think, in a wide sphere, not licensed. Obviously, the data that have been published at ASH are quite enticing. You know, and we will look at this opportunity as less of a commercial reality today, but it could be an opportunity for tomorrow, and we understand that this is an area obviously where GSK in particular thinks is the opportunity for their product. Maybe, Tony, you can provide your view in the, in from a differentiation, medical differentiation perspective.
Yeah. I would simply repeat what I said before. I believe from a mode of action, there is differentiation very clearly. I have every reason to believe at this point in time that that will show also in the clinical outcomes. Again, we'll have to see that. To your other question, PACIFICA. You know, just to recap, the commitment was by CTI to enroll 400 patients. As of today, the company has enrolled about 150 patients. That means there's quite some way to go. There have been several statements by the company by when they want it completed. My guess at this point in time is we're a couple of years out for the completion of the study. There have been a few tools deployed, including increasing centers.
They've obviously suffered from COVID, and they have a number of European sites which hasn't made life easier due to the political war kind of type situation in some of these countries. What we will do, and that's probably the best answer I can give you, as I said, we look very carefully into which sites are productive, which sites are not, and we'll make an effort to increase the speed of this trial. At this stage, it's really too early to give you a final timeline. My best guess, we're a few years out in order to both have a reading of each of the primary endpoints that I believe was your question. If you ask us that question in, you know, two or three months, then I think we have a pretty good read on the progress of the study.
Thank you, Tony. Maybe on the royalty, we have Henrik, who can provide you with an account.
Yeah. The royalty expired in February 2023.
The buyback. The buyback.
Yeah, the buyback. Yeah.
Yeah.
Yep.
Okay. Next. Thank you. Maybe we go to the next question.
The next question comes from the line of Mattias Häggblom with Handelsbanken. Please go ahead.
Yep. Thanks so much for taking my questions. I have two, please. Firstly, likely for Henrik. With regards to the fully diluted number of shares beyond outstanding shares and options, there seems to be roughly 30 million additional shares or $300 million worth of value to get to the equity value of $1.7 billion you referenced. The last part is not crystal clear to me what it represents. Any color on that would be helpful, whether that's related to the royalty rights or, you know, what contractual liabilities that may represent. Secondly, I know we're gonna read more about this in the SEC filing in a few weeks. I'm curious to understand when the dialogue started between you and the company.
I mean, maybe I start with this, you know. We have been following the company for quite a while, and we wanted to get, you know, and as we would do with so many other companies, and we have started the dialogue, you know, let's say in February this year. This was more of an exploratory discussion and then obviously it intensified over the last couple of weeks. You know, and obviously you will see the script when this is all opened up. Henrik, you wanna talk about the SEK 70 million?
Yes. In addition to the basic shares outstanding, there is about 53 million shares related to options and convertible notes, mainly.
That's helpful. Thank you so much.
Thank you. Maybe we open up for more questions.
The next question comes from the line of Viktor Sundberg with Nordea. Please go ahead.
Yes. Hi. Thanks for taking my questions. maybe a first one here on the myelofibrosis market. pacritinib is contraindicated for moderate or severe liver impairment or kidney disease. What is your feeling of how many patients with a low platelet count that could be contraindicated for this drug in this population? That would be helpful. Also here in first line patients with a low platelet count, what kind of opportunity is this when you have done your due diligence? Would be interesting to hear conclusions in the first line opportunity and also if you compare it to Jakavi maybe in that.
Yeah.
In that part of the world. Thanks.
What I can tell you is, you know, essentially we look at this as an opportunity, low platelet count in the U.S., give and take $1.2 billion-$1.4 billion. Basically, as we said, you know, there will be some utility in this, in this higher platelet count opportunity. The company, I think, is referring to the addressable market for the product, the seller at $3 billion, let's say myelofibrosis for them. At this stage, you know, we probably will not provide more guidance, you know, to how to size each of those segments at this juncture.
You know, I think that gives you a ballpark, you know, where this market, I think, is. You know, and obviously we would feel quite encouraged, you know, to take in the low platelet count a significant share and let's say, and then obviously trying to take this company, this product and company international to basically take advantage of other markets and patient numbers.
Okay. Thanks. In Europe, I noticed that, you know, that this product was withdrawn earlier. Do you plan to resubmit an application here before the confirmatory trial, or do you need more patient-reported outcomes before you can refile it in Europe?
Yeah.
To speak? Yeah.
I think, you know, we have a vested interest to take the product given the high unmet medical need also to European patients. At this stage, I think what, you know, before we have been able to conclude this transaction and close it, I don't think it's appropriate for us to engage the regulator. We were going to do this and once we have engaged the regulator and basically really understood what we can do as a company, because, you know, the evidence that has been generated looks pretty compelling to us. Then basically discuss whether, you know, the confirmatory trial is really mandatory or whether there are other pathways to achieve, you know, faster licensing.
Okay. Thank you very much. I'll jump back in the queue. Thank you.
Thank you.
The next question comes from the line of Emmanuel Papadakis with Deutsche Bank. Please go ahead.
Yes.
Thank you for taking the question and for hosting the call. Guido or Henrik, perhaps a question around margin implications. You understandably haven't given us any specific synergy-.
Mm-hmm.
Targets. You haven't really talked about accretion either. Perhaps you can help us with where margins go midterm. You previously, some time ago, talked about getting to back above 40% by 2025. Does this deal increase your certainty of achieving that? Does it perhaps even offer significant upside to that ambition? A second question on the funding. Apologies if I missed it. The 50% equity funding. How fixed is that 50% target ratio? Will you calibrate it according to market conditions? You alluded to, on the results call the other day, a potential monetization of the nirsevimab royalty stream. Could that potentially be used to adjust the size of the intended raise? You've helpfully disclosed AB will participate. Can you give us any opinion on AstraZeneca's intentions? Thank you.
Mm-hmm. Thank you, and, you know, with regard to margin, I mean, I'll give you the first shot. You know, basically, the way you have to look at this product is obviously it, you know, it's given that it's a small molecule and the cost of manufacturing of COGS are relatively low. So it's gonna be very, gonna yield a very positive impact once it materializes on our gross margin. Given the fact that the product is extremely complementary to what we are currently doing and also the size of the indication rare disease and to the size of what the company is currently spending in this therapy field. You know, we think that's also obviously gonna be accretive, as we said, earlier on the earnings side.
Yeah. That clearly, you know, when you could look at this basically makes us more optimistic, you know, with our, reaching, coming closer to our ambition. You know, It's not the time to just now to tell you, what it is exactly. I mean, Henrik, you wanna comment on this?
I think it's exactly right what you say. This will be accretive, notably to the margin.
Yeah.
It will bring us closer to the ambition. I just echo what you say, you know.
Yeah. you know, we would not set out now, let's say, this target. you know, I think at this juncture, what we said is, you know, Henrik pointed this one out with the financing. We wanna raise SEK 8 billion of equity financing the rest by debt. That's our ambition. We know that our main shareholder has already committed their part. We have not discussed this, you know, given also the timeframe for this transaction, which was rather short term with any other shareholders. We hope that the shareholders will follow our rationale. We have, at this stage, not contemplated, you know, to use the nirsevimab royalty stream.
I think, you know, just, you know, I'm not saying that we would not do this one day or we're not contemplating on how to leverage this, but I think, when you just detach yourself, you know. I mean, I could personally quite like the nirsevimab royalty stream as it will bolster obviously, our overall capacity. If we would do so, we probably would do so once the product gets approved because, you know, clearly then, you know, the conditions would be much more favorable for us and, we could probably yield much higher returns on it. I think it's more speculative at this stage. We wanted to do, you know, a conservative way of funding it, trying to raise close to 50% with equity.
At least, you know, our main shareholder is very excited about this opportunity. Hopefully once we have the perspectives out and reach out to other shareholders, they will share this excitement. Henrik, you want to comment?
No, it's the usual way of raising equity, as I said. We feel that we have a very strong support with the main shareholder, undertaking to subscribe for close to 35% of the issuance.
Yeah. Maybe we have room for a last question today, and then we, you know, we may want to consider, you know, and we'll see how, when is a good time for this maybe to have an informal session again like we typically do after, you know, the quarterly events. You know, because I can sense that there may be, you know, more questions. You know, for us it's obviously, you know, very important that we bring you into the situation to fairly, to give this asset justice in this transaction. You know, As you may sense, you know, we have thought about this a little bit, and this is not something that fell, you know, from out of nowhere.
you know, it's, and we feel very passionate about this asset, that this will be the right asset for the company. Yeah. maybe, one more question and then we probably close the session.
The last question for today will be a follow-up question from Christopher Uhde with SEB. Please go ahead.
Okay. Thank you so much for taking my question. Just maybe, you know, you kind of asked this, but I think it was kind of asked, but maybe we'll ask it in a slightly different way. Up to 50% of the value could be covered by a rights issue. 0%-50% is a big interval. What factors would impact that range? Is further M&A a factor? And I guess then also, just what can you say about transaction fees and loan costs? Thanks very much.
Henrik.
What would be the factors influencing the size of the rights issue?
Yeah.
It would be market conditions, price, rebates, participation.
Yeah.
Thanks.
We have to make a perspective and then, obviously people will elect, or not, you know. That's basically what basically.
And then as usual.
Yeah.
There will be possibility also to subscribe without the preferential rights.
Okay, great. Thanks.
Yeah. I think maybe what we do at the end, you know, because we don't want to shortchange anybody, we will come back to you shortly with a proposal for another session, you know. If you don't have too many questions, we can keep it short. At least, you know, I want to be respectful that, you know, many of you have other appointments booked today. Thank you for your interest. You know, if you need any other, you know, information in the meantime, please reach out to our IR or to Henrik or myself and to Camilla. Yeah. Basically, you know, we are here to make sure that you get, you know, the right information to do your job.
Obviously we have a vested interest that you give justice. You are in a position to give justice to this asset. Anyway, thank you so much for your attention and for your time, I wish you a great day. Yeah. Thank you.
Ladies and gentlemen, the conference is now over. Thank you for choosing Chorus Call, and thank you for participating in the conference. You may now disconnect your lines. Goodbye.