Medivir AB (publ) ($MVIR)

Welcome to Medivir Q1 Report. [Operator Instructions] Now I will hand the conference over to CEO, Jens Lindberg. Please go ahead.

Thank you, operator. Welcome, everyone, to the Medivir Q1 2026 Results Webcast. We look back at a transformative quarter where we've strengthened our financial position. We've opened up a new rare disease opportunity with MIV-711. And we just came back from a successful and quite invigorating trip to Korea, where we visited a number of the highly engaged hospitals and investigators, now sort of eager to get going in the FLEX-HCC study. Pia will share more on that particular topic in just a little bit. We've also seen our partner, Vetbiolix in France make great progress in the past quarter. So we are looking ahead to the remainder of 2026 with great anticipation. As mentioned, sort of we are -- we've just come back from Korea. And the interest in the FLEX-HCC study is very strong among the hospitals and the interest to participate in the study is quite high. So the Korean Cancer Study Group, they have added 4 additional hospitals in the quarter. And the interest as we were sort of visiting them was quite palpable when we had the opportunity to meet with 8 of the hospitals face-to-face. We also do understand that sort of perhaps the greatest interest, however, in the study is when is it starting, and we're happy to be able to convey today that we've just sort of gotten news that the first 2 sites are now open to start enrolling patients. So our trip to Korea came at a perfect time where we're able to engage with them just about as the study is about to open and they're about to start enrolling patients. We have also spent quite a bit of time in the last quarter of engaging externally with clinical and regulatory experts as well as patient organization in the field of Osteogenesis Imperfecta to kind of confirm and finalize study design plans for the proof-of-concept study in the disease. We have been positively surprised by the great interest and willingness to engage across sort of all the stakeholders. And perhaps most importantly, while we have made some -- those interactions have led to some adjustments in our plans, the overall plan is sort of lying firm, and we've been sort of been strengthened in our belief in the potential of 711 -- MIV-711 in Osteogenesis Imperfecta. And then finally, as I mentioned, our partner, Vetbiolix, continues to rapidly progress their clinical efforts with MIV-701 or as they call it, VBX-1000 for periodontal disease in dogs. And as of sort of end of April, they have now recruited more than 40% of the subjects in their ongoing study to confirm that MIV-701 has the opportunity to be the first disease-modifying treatment for the disease, which is a critical step to unlock the blockbuster potential of the drug. We and they expect results in Q4 this year, and that sort of will be a significant potential inflection point for the program and then also for us, of course. Today, we will focus on those -- so these are our programs, as you can see sort of the in-house programs at the top, fostrox for liver cancer and MIV-711 for Osteogenesis Imperfecta. We will touch more on those 2 programs today and the progress we've made. And we'll also sort of touch on the outlicensed program of Vetbiolix and hence, those that are marked in green. One minor comment to make regarding one of the other programs, and that is remetinostat is that following on from us making the agreement with Biossil last year, the Gorlin Syndrome Alliance, which is a not-for-profit organization in the U.S., supporting patients, families and health care providers, they have sort of actively reached out to us and then to Biossil eager to -- sort of as they heard of the news, eager to support the development in patients specifically with Gorlin syndrome. And Gorlin syndrome patients sort of genetically driven disease and that manifests predominantly as patients get multiple, multiple sort of BCC lesions where surgery is many times not sort of a good option. So for them, development of remetinostat in that particular rare disease subpopulation is critical. And that's also our understanding from discussions with Biossil as they license the drug that might well be sort of their first development path. So interesting to see interest in that drug as well, and we look forward to hearing more about their plans as we move forward. Today, I am joined by our CMO, Pia Baumann; our new CFO, Patrik Norgren Noren, who joined us sort of just a month back-ish; and then our CSO, Fredrik Öberg. But with that said, let me hand over to Pia to share an update on our efforts with both fostrox as well as MIV-711 including then our recent engagements on the ground in Korea. So Pia, over to you.

Thank you, Jens, and thank you for the introduction also about sort of what has been going on when it comes to fostrox. But just as a reminder, the FLEX-HCC study, that is the reason why we went to Korea is a randomized second-line study in liver cancer, and it's really there to strengthen the evidence that we have from our single-arm trial with fostrox plus LENVIMA in liver cancer and to show superiority over LENVIMA alone. So just as a reminder, what this study is, it is like fostrox is combined with LENVIMA in one arm, and it's randomized against LENVIMA monotherapy in the other arm. And it will be 80 patients that will be enrolled in this study, and they should have progressed or be intolerant to prior immunotherapy combinations. And as Jens said, the patients will now be enrolled at 12 sites instead of the original 8 sites due to the fact that it was a high interest. And the PI for the study is Professor Chon from CHA Bundang Hospital and he is doing this study in close collaboration with the Korean Cancer Study Group. And I think that is also the reason why it is a high interest because this group recently executed and also published actually the only prospective study with LENVIMA lenvatinib monotherapy in a similar patient population that will be enrolled in the FLEX-HCC. So it's a really, really nice setup. And here, the patient enrollment is expected to take around 12 months. And the primary endpoint will be evaluated by a blinded independent central review to make sure that we have the quality that is required to really show this difference. And since we hope this study will enroll and perform relatively quickly, the top line results is expected already in 2027. So we had the opportunity to visit 8 of the 12 sites last week in Korea. And right now, 2 of the sites are open for enrollment, and that is CHA Bundang, where Professor Chon is situated and Severance Hospital. And we know that they are super eager to have the first patient in. As you can see on this map here, if you can see it, the site is geographically spread across Korea also to have the best chance of capturing all the patients that could be eligible for this study. And since we had the opportunity now to discuss with the investigators, it became really, really clear that there is a very high interest in participating in this study and that they have quite a few second-line patients every week that potentially could be candidates to be enrolled. Overall, the support for the study and the PI, Professor Chon was overwhelming, and it confirms really the expertise and the quality his name is associated with when it comes to liver cancer studies. So overall, we were very welcome and they were extremely interested in fostrox, also as an alternative with another mechanism of action compared to both immunotherapy and TKI that is usually the one that you can use in this patient population. So if we can go to next slide. So to summarize the takeaway from this visit, we can conclude that, unfortunately, liver cancer continues to be a very common disease in Korea despite that they now have started vaccination, it's still very common. And that while there are treatment option in first line now with immune combinations primarily, there is a high unmet need in subsequent treatment line and particularly in the second-line setting where the patients -- many of them can tolerate second-line treatment, but still there is sort of no optimal treatment. So the sites are fully committed to the FLEX-HCC study, and it fits very well in with the standard treatment for advanced HCC. The majority will have received 80%, something like that, will have received Tecentriq/Avastin, the immune combination in first line and almost all of them is trying to have LENVIMA in the second-line setting. That's why this trial really fits well in. And most of the sites had no competing studies ongoing in HCC. And those who actually had studies ongoing, it was mainly cell therapy or bispecific targeting GPC-3, which I think that all of you are interested in HCC have heard about. It has been a target that has been discussed for quite a few years now, and we are waiting for better results, but it's mainly in the first-line population and selected patients there. So the interest in fostrox is also part built on the fact that it is an all-comers population. You don't need to do any biomarker testing, and it could be something that is available for more patients than those are selected in the other GPC trials. So high interest. We can go to the next slide, and I think I'll leave it to you, Jens.

Yes. Just to sort of summarize, I mean, I think first and foremost, we are -- we were sort of highly encouraged sort of it's always good to meet face-to-face and sort of see that engagement commitment. Now with Bundang and Severance hospitals just now in open for enrollment with the other hospitals to follow sort of shortly, it feels quite good, and we're very much looking forward to the progress and with their engagement sort of also looking forward to sort of speed of recruitment. For everyone to sort of summarize, again, we are sort of -- we do have the first and only liver-activated small molecule targeting selectively targeted tumor cells in the liver, sparing healthy liver cells. And that's also sort of clearly from some of the hospitals we met new physicians as we go through the drug in a bit more detail than I have seen before, it is one of those areas, one of the features that they appreciate the most. So we continue to move forward. And as Pia said, there has been very little development in second line, very little competing studies. So again, our aim eventually to the right is to hopefully become the first sort of approved second-line treatment in advanced HCC, a market that is conservatively valuated at sort of USD 2.5 billion per year. But with that, and on the note of hospitals sort of opening up, we move to the second program. And let's talk a little bit about sort of what has happened with MIV-711 since we last spoke. Pia?

Yes. So for MIV-711, we are aiming to do a proof-of-concept study in Osteogenesis Imperfecta. And since we really need to have advice both from the scientific community, but also from other parts, we have, first and foremost, formed a scientific expert council, and they will provide advice and they have already provided advice. We have had our first advisory board and feedback on the development path and also on the study design and the study design now initially for the planned proof-of-concept Phase II study. So the members of this council are highly known global experts -- I'm sorry.

Go ahead.

Highly renowned global experts in Osteogenesis Imperfecta, and they have a background as many of those working with OI has endocrinologists, internists, rheumatologists, and we also have a pediatric endocrinologist part of this Advisory Council, the Scientific Expert Council since that is sort of where we aim at eventually since this is a genetic hereditary disease that affects the patients already from the childhood. All of them in this council have a vast experience in clinical study design, in translational research and also in preclinical research. And they have been part in centralizing the treatment and where the patients are taken care of at the hospital. And they have also been part of developing systemic treatment, which is sort of I think that we talked about it potentially last time, those who have not been successful when it comes to the primary endpoint, the anti-sclerostin, and they have been part of that development as well. So we are trying to take as much experience as possible from earlier treatment program to understand how we are going to be successful in the first part of the development for OI. And obviously, their expertise and advice will be crucial to finding the optimal study design and also to find the sites where we can enroll patients and in addition to that, provide the context of how standard of care -- the standard of care for these OI patients, how it looks like. So the study, this is a very schematic draft design of the Phase II randomized proof-of-concept study that we are showing you so far. And it is -- it will enroll patients that have experienced at least one fracture during the last 2 years, and they will be randomized 1:1 into a high or into a low dose arm of MIV-711. MIV-711 is an oral treatment. It is a once-daily treatment. And we can come back to that, but this is something that is really important for the OI patients as well that it is an oral treatment and not an intravenous treatment because they have seen the inside of hospitals their whole life. So this is a very positive part for them as well. The endpoints will include biomarkers of bone resorption and also measurement of bone mineral density. So the proof of concept will be built on these 2, but also other endpoints will be looked at, obviously, PK, safety, and we will also evaluate the number of fracture even if the follow-up time it's not going to be probably long enough in this study, but it will at least give us some idea. Enrollment will be in Europe. And since these patients are already known at the sites, since they have been identified already when there are children, most of them. We expect a really fast enrollment, and we are currently working on finalizing the study design. And in addition to our scientific expert council, we have contacted other important forums for advice. And these forums include some of them we see here, it represents a regulatory and patient organization. And first, you can see here, we met a former FDA regulatory expert that is also a pediatric physician who previously worked in the clinic with OI patients. And we received advice on particular filing for rare pediatric disease designation and also the next development step in both adult and pediatric OI. Since there is a need for treatment in the whole sort of segment from pediatric to adolescent to adult OI. We also met with the Chair of the Osteogenesis Imperfecta Federation, OIFE to better understand the patient perspective and the general unmet need. And in addition to that, we have participated in the annual meeting for the Swedish OI organization, where we had the opportunity to present our development plan for MIV-711 in OI, and that included also the proof-of-concept Phase II study design that we have drafted to receive the insights from the patients to understand if this is feasible or not and also to get a little bit more knowledge what is the most important question for them. So the key takeaways were that there is strong engagement from the external experts with a deep scientific support for the specific mechanism of MIV-711, the anti-cathepsin in OI. And there is a high interest in this proof-of-concept study. We also learned that the existing clinical and nonclinical package that we already have for MIV-711 together with a significant unmet need, really supports the development in a pediatric population and that it was confirmed that there is the potential for rare pediatric disease designation with a Priority Voucher. So last, the patient organizations emphasize -- really emphasized the significant medical need here, and it is across all subtypes of OI, and it includes also the mild Type 1. And they really supported our plan for this proof-of-concept study with MIV-711. And as I already said it, it was more important than we probably realized before we went there that it is an oral treatment that can be taken at home without having to go to the hospital. So overall, a really, really positive meeting sequence and important advice. And I will leave it to Jens.

Thank you, Pia. And I think just sort of a little bit of a backtracking to sort of the last quarterly call we had we clearly, as a team, as we are embarking on this type of journey, we have done quite a bit of work on the molecule and in terms of planning. But there's always the element of as you sort of step up your external engagements, whether being clinical experts, regulatory experts and you start to deep dive, there's always the worry that sort of how on spot have we been. So it's been super, super encouraging to see that sort of where we have aimed the plans we had, they've been highly supportive of those plans, worked with us to do some further adjustments and tweaks. But overall, we were already at that time, sort of on the right path, and that has been confirmed in the interactions in the past few months, which is highly encouraging. So if we look at it from a market opportunity perspective, one other element that has been confirmed is that sort of we are the estimated sort of prevalent OI population, sort of roughly around 70,000 patients across U.S., EU, Japan and Korea, the significant unmet medical need across all subtypes. I think that's been a key element because sometimes the milder that we've heard indications that maybe the mild subtype 1, maybe the unmet medical need isn't so big, but that from both experts and from the patients, it's super clear that the milder group have a very significant unmet medical need and in need of new treatment options as well. So confirmation overall that sort of the market opportunity here is of similar size as we see with fostrox in the advanced liver cancer space, so equal size for the second program. So all in all, as we have shown previously, the plan continues to stay the same. There are no treatment options available, sort of particularly following sort of Ultragenyx failure in Phase III in quarter 4. So unmet need there, the potential to be the kind of first approved treatment option in this disease is still there, and we are eagerly looking forward to embark on the Phase II proof-of-concept study. So with that, just to sort of take a few words as sort of we continue to see progress with Vetbiolix as they are moving forward. And since the last study, they've continued to enroll patients. They've continued to enroll subjects or dogs, so more than 40% so far, and we eagerly look forward to the results in quarter 4. Just to sort of refresh from a memory perspective, what the drug is doing and what they are targeting sort of with MIV-711 of VBX-1000 in periodontal disease. It's in development for Stage 2 and Stage 3, i.e., before the need for surgical removal of the teeth. And it is the first disease-modifying treatment in development for periodontal disease and as such, has the potential to prevent significant suffering as well as very costly procedures such as sort of removing the teeth if the disease progresses too far. And as a disease-modifying treatment, if it hits, then clearly has the potential to transform how these dogs are treated moving forward. No change -- the study is just as we sort of shown last time, but again, targeting out of sort of 51 dogs to be enrolled, sort of already reached 22 thus far by end of April. From a financial sort of commercial potential perspective, as a company, our upside potential lies in royalty revenues and any sort of share of potential Vetbiolix partnership payments. And with more than 70 million dogs in U.S. and EU suffering from periodontal disease, the commercial potential is sort of clearly significant. And in our estimations, if positive outcome of the program and it gets to market as a disease-modifying treatment, we estimate the potential for us through annual royalty revenues of approximately SEK 700 million per year sort of after 5 years after launch. As we have focused sort of in the agreement with Vetbiolix, we have focused the financial terms on the agreement on upside upon approval and commercialization. So with that, then I hand over to Patrik for the financial details before we go to Q&A.

Yes. Let's go to Slide 22. And I will present the financial summary for Q1 and all the numbers are in million SEK. As you can see, the net turnover for Q1 was SEK 1 million, which was SEK 0.4 million higher than Q1 last year. Other external expenses were SEK 4.5 million and the personnel cost was SEK 5.5 million. And in total, the total operating costs were SEK 10 million, which is SEK 3.5 million lower than Q1 last year. It's mainly related to less employees and lower costs for clinical studies. The operating loss was minus SEK 9.4 million, which is almost SEK 4 million better than last year, Q1 2025. The cash flow from operating activities was minus SEK 13 million. And Medivir has a strong cash position. The cash balance for end of Q1 was SEK 149.1 million.

Okay. So with that, thank you, everyone, for listening to the presentation part of the webcast. And operator, we can now open up for Q&A.

[Operator Instructions] The next question comes from Richard Ramanius from Redeye.

I have a few questions. The first one is when exactly do you expect to dose the first patient in the fostrox study?

Pia, do you want to take it or should I take it?

So 2 sites are open, and obviously, we are hoping that will happen soon as eager as they are to enroll patients. But I cannot say, but any day now, I would say.

I think that -- I mean, clearly, we're prepared for it. So I mean, drug is on site in Korea as well. So they -- as they open the sites, as they open for enrollment, they are now ready to start sort of including and initiating patients.

Okay. I was also thinking about the study in Osteogenesis Imperfecta. What age group do you think you will target in the study you're designing? And what type of disease?

Yes. So we are -- this is still a draft, but I can say that we are aiming for an adult population in this first proof-of-concept study. And to find a relatively homogeneous population, probably the milder ones. But we will come back when we have a little bit more firm study design and when we have received all the feedback that we need.

All right. when do you think VBX-1000 could be on the market at the earliest?

I think that it's a bit difficult for us to speculate on. And I think that I would defer that sort of topic more to the animal health experts with regard -- and Vetbiolix in particular. And we are -- I mean, we've had discussions with actually the Vetbiolix team to see whether we should organize sort of as we progress our sort of as a study start for fostrox, we progress our plans and we firm up our plans on OI. We would also look to have a Capital Markets Day where we would include the Vetbiolix team. And I think that might be a better sort of suited topic for asking -- for answering that question in a bit more detail. But what I can say in terms of what they've communicated, they anticipate the readout of this study, which is an important one, and that will be a key inflection point for them. But they are also anticipating either themselves or sort of a partner, which wouldn't be surprising if they sort of move to an outlicense situation after this study. There is an anticipation of a registrational study as well as the next step before final approval and timing of that, I would be a -- bit difficult for me to speculate on, but it seems relatively clear that sort of as they run studies, they are able to recruit subjects quite quickly.

[Operator Instructions] The next question comes from Klas Palin from DNB Carnegie.

I would start with fostrox question. Nice to hear that you now have 2 sites open in Korea. But just a little bit curious why expanding the number of sites from 8 to 12? Is this purely driven by interest or anything else?

So this is collaboration with the Korean study group, which is a natural study group that just performed another study where they had actually 13 sites enrolled. I would assume that there was so high interest that it's a little bit difficult to maybe select those because all of them enroll patients, right? So -- and all of them are qualified and they are in different parts of Korea. So it is of interest they have expanded the sites, not that they are worried that they wouldn't enroll fast enough.

Okay. And you're sticking to the time line that you have previously announced?

Yes. And I would say, I mean, we can never -- we can just see how the environment looks like when we are at the sites, but it was encouraging to see how many second-line patients they have. Then, of course, we can never know if they are eligible or not since they are also a heterogeneous population in HCC. But it's very encouraging to see how many patients they have at these sites that we were. So I'm not worried about the time lines.

I think the added comment, Klas, is that the -- and I think as Pia is alluding to, that the decision to include 4 additional sites that was a Korean Cancer Group study decision. That wasn't a push from us with regards to sort of speeding up time lines, et cetera. That came from them. We have an agreement and that agreement was sort of for 8 sites, and there's a budget for 8 sites, et cetera. This came from them, and this is a decision that they took and sort of -- it doesn't sort of add any kind of budgetary costs for us, et cetera. So it purely reflects their engagement and the interest from their hospitals that they felt that, okay, we want to include them. So for us, we see this as a pure positive. It can only speed up recruitment, but sort of rather than promising a quicker recruitment, we're sticking with the time lines, but hopefully, sort of this can contribute to recruiting even faster.

Okay. Perfect. And my next question is then related to Osteogenesis Imperfecta. And it relates to your interactions with experts, if you had a chance to discuss a potential primary endpoint for a pivotal study and also perhaps discussing the development that has been seen in osteoporosis where bone mineral density has emerged as a surrogate endpoint.

Thank you. It's a very good question. And yes, we have the possibility to discuss it with them. But just remember now, this is -- the first study we are doing is a proof-of-concept study. And bone mineral density is what you can use for that study together with other biomarkers for bone resorption. So to see that while our hypothesis holds. When it comes to the primary endpoint for a pivotal study, it is a huge interest also for the whole OI community and for everyone who is developing anything in this disease since the FDA has required previously to look at annual fracture rate. And we talked about this before, Ultragenyx study -- was not positive when it came to annual fracture rate. So this is something that is concerning, obviously, for the OI community since they want to have an approved drug. So this is a discussion that I know is going on with the regulatory authorities, also with the OI community. But so far, I haven't heard what that endpoint could be, if not a fracture rate. When it comes to bone mineral density, it is an approved endpoint in osteoporosis. And I think we have said that before as well, you could potentially consider OI and particularly in the adult population as osteoporosis on steroids. So -- and with the same sort of amount of bone mineral density loss. So I think it -- depending on data you can show also in the future where you can validate the bone mineral density against the fracture rate, I think that is going to be important. And the collective new data will potentially contribute to -- I don't know if it's ever going to be a surrogate endpoint for OI, but we have -- now I'm saying several things at the same time, I hear myself, but it is a good endpoint for showing proof of concept. But it has also been shown that, for example, for bisphosphonates, that even if you increase the bone mineral density, you have not shown that you reduce the fracture rate. And I think that is sort of the base for not having this endpoint accepted in OI. But if you can show that you still have a bone remodeling, which we hope to show with MIV-711 and you actually can see that you are at least having some kind of effect on the fracture rate, maybe it will be a possibility in the future.

There are no more questions at this time. So I hand the conference back to the speakers for any closing comments.

Thank you. And before we close the call, I mean we -- just to reiterate, we are very encouraged by the engagement in the FLEX-HCC study among the investigators and the hospitals. Usually sort of standard approach is for us to communicate via press release when we have the first patient dosed sort of in the study, and we are eagerly looking forward to that sort of as we have the quarterly call today, and we just sort of gotten the news that sort of we now have 2 hospitals open to start enrolling patients. We wanted to share that today, and we are super happy that those 2 first are up and running, and we have drug on site. And we look forward to sort of as Pia alluded to, sort of also sharing the update on that first patient is dosed and randomized. Over -- but we're very happy with the progress so far. Over the past months, our belief in MIV-711as a candidate for treatment of Osteogenesis Imperfecta has only grown stronger, and our development plans for MIV-711 has generated support from both clinical and regulatory experts, which has been a very nice progress. And we are super pleased to see the progress with MIV-701, eagerly anticipating the results Q4 this year. That event will sort of be a significant sort of potential inflection point for the program, of course. It will be an inflection point for Vetbiolix as a company and as a result, also for us here at Medivir. So on that note, with the progress made and with the sort of positive outlook to the future, we thank you for attending today's call and wish you all a great day. Thank you.