Good afternoon, and welcome to today's event. With me today, I have Julie Brogren from Ascelia Pharma. She's the Deputy CEO of Ascelia Pharma. Topic for today is actually what happened during Q1, but actually a lot of stuff happened after the Q1. As always, I would like to welcome the audience for this session, and you are more than welcome to post your questions just below the video window here, and I will forward them to Julie during the presentation. As always, we will record this event, and afterwards, we will post it on our homepage, and I know Julie and Ascelia Pharma will post it on their page as well. So with that said, welcome, Julie, and please carry on.
Yes, thank you, Claus. It's good to be here. So I'll start with the highlights from Q1, and I'll be making some forward-looking statements, so please pay attention to this. Just for those of you who are not that familiar with Ascelia Pharma, we are focused on addressing unmet needs for patients who have rare cancer conditions. And we have two products in our pipeline. One is Orviglance, and that is now in the registration phase, so that's the one we'll be talking most about today. It's a first-in-class diagnostic agent to address a very specific unmet need with an FDA orphan drug designation, addressing an $800 million U.S. global annual market. And we will be talking about the successful phase III results and the completion of our clinical program.
The other asset is Oncoral, and it's a tablet, daily tablet formulation of a well-known chemotherapy, irinotecan. And we have an opportunity to help patients with better outcomes and starting in gastric cancer. And we have a collaboration with Taiho Oncology to start our phase II study for Oncoral. But again, I will not be talking so much about Oncoral today. And we're based in Malmö, Sweden, and we're listed on Nasdaq in Stockholm. So to the Q1 progress, the main event, as you mentioned, is actually came after the closing of the first quarter. That is really our fantastic phase III results for the Orviglance study, and that marks the completion of the clinical development for Orviglance.
Now, in terms of other news, in Q1, I think the main one here to mention is that we closed a financing agreement, in Q1, consisting of a portion of convertibles, and a loan, and we drew the second and last tranche of that loan, also, after the closing of the first quarter. But really, the main event here is that we finished the evaluation of the images for our phase III study with Orviglance, and we had very successful phase III headline results, meeting the primary endpoint. So that's what I'll be talking mostly about today. So just to go over what is Orviglance and the opportunity, for us, really. So Orviglance addresses a well-defined, unmet need in liver imaging.
So when you need a scan of the liver, and it's specifically helping patients who also have impaired kidney function. That market, addressable market, has a value of $800 million globally per year. We have finished the clinical development. Phase 3 study is successful. We have commercial scale manufacturing, so now we're entering the stage of regulatory filing, and approval. So I'll talk about the results and what the next steps are. But just to go a little bit back to how, Orviglance can help patients.
So, in cancer care, cancer in the liver is quite a significant problem, both because some patients have primary liver cancer, but also because many more patients have or are at risk of developing liver metastases, so they have another primary cancer, and it's common in many cancer types, for example, colorectal cancer and other cancer types. So there's a need to monitor and to scan and monitor and follow up on patients who have cancer in the liver for various reasons. So the gold standard for doing that today is to receive an MRI scan. And typically, when you have an MRI scan, you have a diagnostic drug, a contrast agent, that will improve the image of the MRI. And these are today used broadly based on gadolinium. Gadolinium is a toxic heavy metal and has been around for decades.
Now, we also know about gadolinium, that if you have severely impaired kidney function, you are at risk of some very severe side effects if you're exposed to these gadolinium contrast agents. And therefore, the gadolinium contrast agents have a black box warning, you can say, from the FDA, for use in these patients with severely impaired kidney function. Now, these are the patients we can help, because Orviglance is both a liver-specific imaging agent, but it's also based on manganese and not on gadolinium, and is not associated with these risks. So our aim with Orviglance is to be the imaging option for these specific patients, and that is where we have an unmet need and an orphan drug designation from the FDA to help these patients.
So with that in mind, we've been through clinical development, and now we had the readout from our phase III study early May, and it showed that we met the primary endpoint, and also that we had a very high statistical significance in meeting this primary endpoint. So just to talk a little bit more about what this means, is the primary endpoint for this study is to improve visualization. So that has some soft measurements, but it means, is the image with Orviglance better than the image in the MRI without a contrast agent? And that improvement in visualization had a very strong superiority for Orviglance compared to unenhanced. So that was the primary endpoint, and also the way it's determined is by having 3 independent radiologists look at all the images.
And all three of these said the image with Orviglance is significantly better than the image with unenhanced and also with a very high statistical significance. So this is fabulous data going into the next steps, both in terms of regulatory approval and in terms of commercialization.
Julie, isn't it true that actually only two of the three should show a significant enhancement, but all three did, so it really-
Yeah.
underscore how strong the data is?
Yes, absolutely. The criteria for passing the endpoint, as you say, is that two of the three readers have a superior visualization with Orviglance than without. So, the fact that all three have, and with high statistical significance for all three, is... It's fantastic data. That's probably the shortest version. Yeah.
It's fair, fair to say.
Yeah. So just to mark, to sum up on the clinical development, so this was the last of nine studies, and the full clinical package is then completed. Almost 300 patients have now been exposed to Orviglance, and we are ready for the next steps. And the next steps include collecting and you can say, compiling the clinical, non-clinical, and CMC data, the data that will support manufacturing. And our objective is, of course, to have a timely submission to the FDA as an orphan drug, and also with the optimal label to use in this patient population, our target patient population.
The steps for that is, first, that we have the full study report early Q4 this year, and then that we have the conclusions from a pre-submission meeting, interaction with the FDA early next year. And these are standard steps of a regulatory process, and then that we submit the file in the middle of next year for the approval.
Julie, if I may-
Mm-hmm.
Just, just to dwell a little on this slide, because this is crucial information. First of all, you got this orphan drug designation, which is, you know, a voucher that you receive due to the unmet need for the patients you can treat. So maybe you can just explain that a little. And what risks do you see in this process? You have strong data, you have a strong... and, and sorry, you have strong data, but you will, you will not see the full study report before Q4. And in the full study report, you will also see the secondary endpoint. Isn't that true, Julie? Maybe you could explain a little about that, if you don't have it on slides further ahead.
Yeah. So the full study report is sort of mostly the drilling down of the details in the data where we've seen the high-level conclusions. But of course, when you submit it to the FDA, you need all the levels of detail in the data. So that's the main purpose of the full study report. A clinical study also has secondary endpoints. That's true. And that's part of the process as well, that we have the readouts for the secondary endpoints. So to go a little bit back to, as you said, the regulatory process and the Orphan Drug Designation. So the Orphan Drug Designation, as you said, is for small patient populations, and it will grant certain advantages both in the development phase.
You can have a smaller clinical program, you can have fewer clinical programs. And then also it gives some advantages when you launch. And one is, for example, that you have a seven-year exclusivity for that specific label in the U.S.. That's the way it works in the U.S., it's slightly different in other markets. But that's the main, the main elements. And then, of course, commercially, orphan drug designation makes a difference also for some stakeholders, both in terms of discussion with guideline committees and key opinion leaders for clinical practice or payers. For them, and for the orphan drug designation, the key thing is that we are addressing a significant unmet need, and that it's for well-defined, and also in this case, a small patient population.
So that's sort of the key part of the impact of the Orphan Drug Designation when it comes to the launch and the commercial uptake.
Thank you.
Did you mention anything else in the question?
No, then there, there's some questions out there, but also, you know, just to maybe dive a little bit with what risks do you see in the process going forward as before you launch? Because you have this interaction with FDA-
Yeah.
and there's always some kind of risk, but what, what is your view on that?
The key thing is that with this strong data and the clinical package we have, we are in a very strong position. So that is the key, high statistical significant, strong superiority. That it was a very clean and strong outcome of the phase III study. That is the main thing. Of course, in a regulatory process, there are many steps. So the way to address risk management in a regulatory process is that we have had a dialogue with the FDA throughout the years about the Orphan Drug Designation, which is granted by the FDA, in terms of recognizing the unmet need. Then you have some conversations about how to design your phase III study, what other data they will be asking for.
So you can say, the product and the processes evolved with the FDA. So I think we're in a very, very strong position now based on these dialogues, based on where we are now with the strong data. What is interesting, going ahead, and as this slide is also saying, it's really also now about the optimal label. So we are addressing an unmet need for these patients population, covered under the Black Box warning. But when you come to negotiating, you can say, or agreeing, let's call it that, on a label with the FDA, then, of course, you want to make sure you can help as many patients as possible. And I think I would rather call it the opportunities of the regulatory process, is really to have the best possible label.
You can say also, of course, in the fastest possible time. And that's what our focus is now, is to make sure it's a high quality file, where we really prepare to get the best optimal labels as fast as possible.
That's really, thanks a lot, Julie, for shedding some light on that.
So let's talk a bit, little bit about the market. Orviglance addresses an $800 million market annually, globally. Half of that is in the U.S.. And again, this is a launch focusing specifically on the patients who need liver imaging and who have severely impaired kidney function. Our strategy is to launch with commercialization partners. And I'll talk a little bit more about that in a minute. But just to go back to this, addressable market, we've done a lot of research and collected what we call real-world data, so that's actual procedures performed in the U.S., so you can look into the databases and see the patients and the procedures. And we can see that around 100,000 procedures per year are performed for the patients falling under the Black Box warning in liver imaging.
We can also see that these patients are typically found at the larger hospitals, who are also quite specialized. So we can reach 75% of the market potential by addressing or launching Orviglance into 400, you can say, accounts or facilities. And also, we have worked specifically with market access and pricing experts to determine what is the optimal pricing, addressing this patient population with their unmet need. And what we have reached is a price point between $3,000-$4,500 in the U.S. per dose. And we looked also at other, you could say, innovative diagnostics and their pricing, and also how it would be covered by payers and so forth.
So really, you could say, groundwork in terms of understanding where are the patients, what are the payers willing to pay? So back to our commercialization strategy. We want to launch with partners. That is really, for us, the optimal balance between the investment required and the future revenues. And it means also that we can leverage the capabilities for our commercialization, existing in another organization. So the arms and legs, and the back office, and all the systems and procedures. So that's how we really see the optimal value, for Ascelia. That being said, we are all going to share the strategy and the data.
That's also a way of optimizing the outcomes globally, that we share all the clinical data we have developed, and all the value we have created so far will be available for this partner or these partners. So, so far, it's progressing well. Our work is recognized in the industry, and we have a very constructive dialogue with experts that will be able to help us as we progress. So just to round off a bit on the financials and a summary. So, in Q1, we spent almost SEK 17 million, it's a little higher than in Q4, but that's related to this completion of our SPARKLE image evaluation and also to the preparations for the NDA, for the regulatory filing.
And then, at the end of the first quarter, we had SEK 27 million. On top of that, we did draw the second tranche of the Formue Nord financing that we announced in February. So we drew the second part of the loan here in April. And that means we have with this financing, we have a runway into Q2 next year. So that covers the time-critical activities for the NDA. So to summarize, if we look at Orviglance, we have two key priority areas.
One is to advance Orviglance to approval, and the first step is to do the submission, and we are looking into a couple of milestones before that, including the full study report, including conclusions from a dialogue with the FDA in the beginning of next year or Q1 next year. And then the other focus area is to progress the commercialization readiness. That includes preparing the launch, making sure we are ready for a launch for a partner, with a partner. But it still means Orviglance needs to be ready in terms of manufacturing, supply chain, the data needs to be available, and we can progress some a number of things that are very valuable for the partner that we will work with. And then, of course, entering into this partnership is a key priority as well.
In terms of Oncoral, which we also have in our pipeline, we have an opportunity to demonstrate strong efficacy and safety in a phase II study, and we want to initiate that when the right timing comes, and the financing at some point. So, that's what I had here, and you started some of the questions, but I think there are maybe some more.
Thanks a lot, Julie. Yeah, there's a lot of questions coming in here, and...
Good.
Let's start off with the commercialization phase, because there's actually a lot of questions about that.
Yeah.
So, Julie, those strong headline data, do they change your plans for partnership? Or is it still the same strategy as you talked about? You changed it last year, I know. So it's still the same strategy, you're looking for a partner in U.S., you're looking for a partner for the rest of the world. Could this partner be the same partner?
Yes, it could be the same partner. It could be the same.
Yeah.
Our objective is that we find a strong partner to work with, with of course the right terms, and who sees the value in Orviglance, and we can have a successful launch.
Yes. Do you have any partner negotiations already now, or, or is it something coming later down the road?
So what we've said is that it's an ongoing process, because to get the best possible agreement, we need to time those conversations. So that's what we are saying. I think with phase III study in the hand, it's a really good time, right? Because we have de-risked the asset, we have very strong evidence, clinical development is completed. Now, it's packaging everything and full steam ahead into the submission and approval phase. So fantastic timing to continue those conversations.
There's a very direct question in relation to a partner, but... And you can probably not mention it, but, or you can comment on it, either yes or no. But is GE HealthCare a good partner for Orviglance and Ascelia Pharma?
We can't really comment on specific partner candidates. Of course, there are many types of partnering candidates for Orviglance. The gadolinium manufacturers are those with diagnostic drugs and radiology. That's some of them, and there are many. There are others where Orviglance, because we're addressing a well-defined unmet need, can be beneficial and see synergies to their strategy or capabilities. So there are many options here. I think the key thing in terms of capabilities, or where you would typically see some synergies, is where, in terms of commercialization, you are addressing hospitals, you are addressing diagnostic drugs, perhaps, or at least an area related to Orviglance.
But also that you share the strategy, that this is for a well-defined unmet need, an orphan drug designation, which also comes with a high value per patient. So it means it's a launch that is also a lot focused on the market access piece and working with key opinion leaders. But
Thanks a lot, Julie. So with these strong data at hand, of course, it's much easier to find a suitable partner. But if you have to choose, so if you have a partner that is very strong in U.S., and you have a partner, you know, that is very strong in the rest of the world, would you focus on U.S. first, and then take Europe down the road or sorry, the rest of the world down the road?
We have chosen to start with the U.S. because as a single market, it's the biggest opportunity, both in terms of value of the market opportunity, but also in terms of the, you can say, you know, the way the market dynamics work in terms of pricing, access, how to adopt new technologies. I would say what we know from our market research is the unmet need is well recognized, in generally, not only in the U.S., but if when you add that to the picture, U.S. is by far the biggest opportunity, and that's where we started. Now, many markets have patients who could benefit from Orviglance, and then we will progress those, as we move along and potentially with a partner.
You asked also if we would prefer one or the other. I think time will tell.
Mm.
You know, we will prefer where we get the best mutual value with a partner.
Yes.
Yes.
And just to take one more question on the partner deals. So, you know, a package is normal, some royalties or milestones in terms of royalties and some upfront payment.
Mm-hmm.
Don't you think you get a better deal now, you know, with the strong data than before you released the data?
I think for sure the asset is significantly de-risked.
Mm.
And that can also always be a good thing. So it will typically always progress the way, the more value we add, that we have invested in, the more value we will get in return-
Mm
... with a, in a partnering situation. So that is always, as you say, the development, we invest more, we get more. So I think it's a good—we're in a good space or in a good situation in terms of progressing with Orviglance towards regulatory approval, but also in terms of partner.
So when you do your submission, if you get a priority review from FDA, do you then expect to be commercial with Orviglance in 2025?
In terms of timing, that will depend on a couple of things. What is interesting about the Priority Review is, just for those who are not familiar with it, the standard review timeline from the FDA is 10 months, Priority Review is 6 months for this type of, so that's the setting for this asset. Priority Review is something we can apply for or request, and then the FDA will make a decision. We don't need to request or decide now. It's something we do, we submit. And of course, you also want to be fully ready for the launch to make most out of this Priority Review, and you also need to be prepared to answer the questions from the FDA in a shorter period of time.
If you count, you know, from middle of next year plus six months, that's the way I would count, so I will leave the conclusions to-
Yeah, no-
... But that's how the math works, you could say. So depending on when we submit, it's not an impossible scenario.
Good. Thanks a lot. Then, just talk a little further down the road, you know, a couple of years from now. You have another very interesting product, Oncoral.
Yeah.
When will you start to speed up the process in Oncoral again? Is that after you make the submission? Because as I understand it, you guided for sufficient capital until mid-2025 with your current plans.
Yeah, into two, yeah.
Yeah, yeah, sorry.
Yeah. Mm-hmm.
So, when we look into Oncoral, when will we start to, you know, to see some progress in Oncoral? Can you just briefly touch on that, Julie?
Yeah, we haven't guided specifically, but I think we are in a really strong position because we have the IND for the phase II study approved. It's ready to advance into the phase II study. And we also have a clinical development agreement with Taiho. So we're in a very good position to move ahead with Orviglance when we're ready. And I think when we are ready will depend on how things evolve with Orviglance. But it's a very interesting product with the potential to help patients, because we know chemotherapy today, clinically, is administered typically every three weeks with a high IV injection with a very high exposure for a short period of time.
And imagine if we could help patients have a more stable and constant exposure to the chemotherapy, that has the potential to improve both the outcome, clinical outcomes, but certainly, of course, also the safety, and most importantly, those two things in combination. So, it's very attractive, and we're, it's all set up to be ready to go when timing is right.
Good. Thanks a lot. And there's a final question here, and please, if you have any questions, just post them, and I will forward them to Julie. But we get back to Orviglance's, a little about the market share and penetration curves. Is that something you can elaborate a little on? You know, usually we see with these products that have a unmet potential, and with the orphan drug designation, that you can capture a very large part of the market very fast. Is that your expectation, Julie? And are we talking in the ballpark of 30%? I understand if you don't want to comment on that, but please, could you try to elaborate a little on your thoughts?
Yeah, we are preparing for, we're hoping for, and as you said, is that our expectations? Yes, of course. My expectation is that Orviglance will be used for the target patient population as soon as it can be used after launch. So we haven't specifically communicated our uptake expectations, but I think what is key is that we understand the stakeholders, we understand where the patients are, and we understand how they think around the unmet need. And we've invested a lot in this because to get the optimal uptake at launch really has a lot to do with what you prepare in advance. And I think that's no different between a larger product launch or a well-defined orphan drug launch.
And that is really key to the uptake, is that you are ready to go with the stakeholders, whether it's payers or, or the leaders in clinical practice. That is key to the uptake, and that is why it's important that we have these investments are in place, because that will help us and our partner. So yeah, and so are we, it's our expectation, of course, that Orviglance is successful, and I think we are on the right track.
Thanks, Julie, for shedding some light on that. But that said, I think we more or less answered all questions. And if not, please, contact me or Julie afterwards, and we'll do our best to answer your questions. Julie, thanks a lot for participating in this event. And also to the audience, thanks a lot for all the good questions. And by that said, I will close the session, and enjoy your day.
Thank you.