Hello, and welcome to day one of the Jefferies New York Healthcare Conference. I'm Brian Balchin from Jefferies, mid-cap biopharma analyst, and it's my pleasure to have with me here today, Camurus. We've got the CEO, Fredrik Tiberg, who will be giving us a small presentation, and then we'll get into Q&A.
Yeah.
Ready.
Thank you so much, Brian, and welcome everybody. I'll give a quick intro to Camurus, and then after that, an update on our most important recent developments. So, we have, of course, forward-looking statements in this presentation. For you who have not been encountering us before, we are a rapidly growing commercial stage company based on our long-acting injectables for treatment of opioid dependence, primarily right now. We also have an advancing pipeline, from early stage up to registration. We have one program in registration as we speak, and most of our developments are based on our unique Fluid Crystal technology platform, which is a long-acting injectable technology platform.
Based on the significant progress we have had in terms of the commercial development, we have also developed our financial position and been profitable since 2022, and this is being sustained as we go forward. Looking at the recent progress, we are working here on the commercial side, establishing a leadership position in the opioid dependence treatment space with our long-acting products, Buvidal and Brixadi. We have had a very robust and substantial growth in our sales in Europe and Australia, which has been our first important markets. Now we see a very strong momentum for Brixadi, which is the U.S. trade name for the same product in the U.S. And then on top of that, we are in the process of building our own commercial organization for our next launch, which is CAM2029.
It's called Oakleys, and where we are targeting a launch in acromegaly late this year, early next year. Aside from the commercial progress, we are also advancing our pipeline. As you can see, we have ongoing U.S. Regulatory Submissions and European for CAM2029 in acromegaly. It's a long-acting octreotide product. We have completed recruitment in a large neuroendocrine tumor study, and that is progressing towards results next year. We have also a study in polycystic liver disease, where there's currently no treatment. In addition to that, we announced recently that we have positive preclinical assessments of a long-acting injectable GLP-1 candidate. We're actually have several in development, and this is a once-monthly formulation of semaglutide.
As I already talked about, we have a strong financial position, disciplined capital allocation, and a robust cash position of about SEK 2.3 billion, without any debt in the company. So we're looking at the development over the last few quarters here. This is Q1 2024 versus previous years. We had a significant growth of about 37%, compared to last year, and also our profit grew up to about near SEK 100 million. And that's very much in line with our expectations for the full year, as you can see from the outlook for 2024. So our most important commercial asset today is Buvidal, called Brixadi in the US. It consists of weekly and monthly injectables for treatment of opioid dependence. We've had these products in Europe and Australia, as I said, and had very strong success.
We have countries in Europe, like Finland, where we now have 70% of all patients being treated with this product. Australia is another success, where we have more than 25% market share of all patients and dominate also the long-acting space very significantly. This is a product that gives and has been proven to give superior treatment outcomes compared to standard of care. And it importantly blocks subjective opioid effects in patients being treated with this and co-using illicit opioids. So we have multiple other important benefits, but we can go into that at a later stage.
Looking at the growth of the market here, you can see that Europe, which is on the right-hand side, sales in Europe have grown very steadily and significantly over the last five years since our launch and had a 106% CAGR rate. Also, I would like to say that what is most important right now is that the development in the U.S. is going very strongly. We have had a successful start with strong payer coverage or good payer coverage, and in the last quarter, we had a threefold increase in net sales and in our royalties from our U.S. partner Braeburn Pharmaceuticals, who are commercializing the product here. Market expansion is the future for us here.
We are working hard to obtain our goal of having 100,000 patients in treatment with Buvidal in 2027 and reach peak sales in Europe and Australia of more than EUR 300 million. In the US, we estimate the peak sales to significantly above $1 billion. So the other very important development is our phase 3 and registration programs for CAM2029, which is a long-acting depot of octreotide, developed for acromegaly, as well as gastroenteropancreatic neuroendocrine tumors and polycystic liver disease. This product is designed for two things, both enhanced efficacy but also improved patient convenience, and it will be the first long-acting product that will be conveniently administered by patients. Here we have a number of studies ongoing, two in acromegaly called ACRINOVA 1 and 2.
The first one is a randomized controlled study against active control or against placebo, I should say, which has been completed successfully with positive outcomes, and we are in the midst of our NDA review period here. The second study is a long-term safety study, extension study, also including a large number of new patients, where we studied safety, of course, but also the transition and long-term efficacy in both new and transferred patients from the ACRINOVA 1 trial. In addition, I already mentioned that we have the GEP-NET study ongoing, progressing well. It's the largest study ever performed with somatostatin analog in this indication. We completed recruitment of patients, 332 patients before Christmas last year, and are now just sampling the PFS events, progression-free survival events. The study runs against standard of care with a superiority endpoint.
And then finally, we have an interesting development in the polycystic liver disease area, where there is currently no treatment here. It's a randomized controlled trial against placebo, which we expect will deliver results early 2025. So overall, the 2029 program is progressing well. We're estimating an approval decision at the PDUFA date of 21st of October this year, and we are planning for a U.S. launch around the end of 2024, early 2025. In the NET area, we are expecting to have top-line results in the first half of next year and NDA and MA submissions somewhere in 2025, end of 2025. And finally, you have the POSITANO study progressing, where we are having top-line results towards in the first half of next year. So we are addressing an interesting...
with CAM2029, a very interesting commercial opportunity, which is in the range of $1.5 billion to more than $2 billion, and a majority of this is in the neuroendocrine tumor space. So, of course, very important. But also, the acromegaly and PLD indications have significant potential. So we're looking at the future here. We are steadily establishing ourselves as the global leader in opioid dependence treatment. We have waiting for U.S. approval in acromegaly. We have top-line results from phase 3 trials coming out in the indication I talked about before. Inorganic growth and diversification through business development is ongoing. We have strengthened our balance sheet through more than SEK 1 billion directed share issue earlier this year.
Our U.S. Commercialization Organization, which is led by Richard Sheldon, who's sitting here in front of me, is moving well ahead and for the planned launch in at the end of the year. Going forward, we have an ambitious plan for 2027, a five-year plan, which included fivefold increase of our revenues, establishment of the U.S. Organization, of course, four new approvals, and the reaching of 50% operating margin. So that's something that we are ambitiously pursuing, and so far, we are well on track. With that said, I think I'll leave it over for questions.
Oh, yeah. So if you do have a question... Yeah, I think we've got one here. Can you get the mic, please?
Are you still doing contract formulation development for third parties? Sorry? Are you still doing contract formulation development for third parties?
Contract, well, it happens if we find an opportunity that we think is very interesting from our perspective, yes.
Yeah. Okay, so if there are no further questions, I'll just kick off with Buvidal, maybe.
Yeah.
So, yeah, so you're currently at 50,000 patients treated. I know you saw some softness in January and February, owing to seasonal variations in inventory. There was a temporary funding gap in the U.K. that are recovered in March. So, how has that been developing?
Well, we said, even in the first quarter, that we had an in-market growth of about 5%. So that's according to plan, I would say. So those effects were mainly due to the channel stocking that we had over Christmas.
Got it.
Seasonality. As I understand it, you know, things are progressing according, in the same direction right now.
Okay, perfect. And then maybe just sticking on Buvidal, your, your guidance is for over 100,000 patients treated by 2027.
Yeah.
So can you just talk us through how, kind of the steps to get from 50,000 to 100,000, would that come from simply driving penetration in existing markets or just, help us with that?
Yeah, I think so. I mean, one very important component is, of course, continued penetration of our markets and also growing the markets we see in some countries like Australia and Finland, that we are actually expanding the whole treatment landscape with new patients coming into treatment. We need to, that's been in our plan all the time, is to accelerate the growth, and that will mainly come through the larger countries and through our various initiatives that are going on in Germany, France, Spain, but also through a more aggressive transfer of patients from methadone over to long-acting buprenorphine treatments, where we see there is a large interest, both among patients but also treatment providers.
Got it. That's helpful. Maybe now shifting on to Brixadi in the U.S. So, as you mentioned, strong one Q, 7,000 patients, which is over three times greater than what you had at the end of four Q. And I don't think you saw any impact from the Change Healthcare cyberattack, as Indivior had pointed out. So it would just be great if you can share what the latest feedback is there, and then just how that continues to progress.
Yeah. So I mean, I have not commented on any cyberattacks, and I don't think that they had any material impact on the sales of Brixadi. So that's... But otherwise, you know, we are very pleased. We've got a good payer coverage, very high at this moment. And that's been, of course, important. We also see that the distribution chain that has been built up has been very effective. I think the real possibility in the U.S. lies in transferring more and more patients over from sublingual and building the market.
It's a huge opportunity, and if that's, so to speak, executed in the right manner, I think the overall potential for Brixadi is going to be significantly above $1 billion and maybe, you know, multifold in the best of circumstances, because that's still right, quite small market share we're talking about.
Yeah, and I mean, just to get to your over SEK 1 billion peak sales target, I think that's based on penetrating only 6%-7%.
Yes, I think that's correct. Somewhere in that range, 6%-7% corresponds to $1 billion. And of course, this opportunity is much larger, but, it needs to be followed. I mean, it's you need to establish the right patient pathways and have the support system that is needed for patients and build that out.
Yeah. Okay, great. Yeah. And it was interesting, I think Indivior had mentioned this morning that their physician feedback was that they see market share going up to 30%. So yeah, I mean, seems like there's potentially more in there than just the 6%-7% that we currently have. Okay, maybe now shifting on to acromegaly, CAM2029. So you've got your approval that's expected on the twenty-first of October. So just maybe you can give us the latest on how that launch is going, and then how we should be thinking about the pace of that launch, just given that that would be your first attempt?
Yeah. So I mean, obviously, we have a very strong commercial setup being founded in the U.S., and we have the team in place, so the core team is in place. We've been working very actively in the medical affairs arena for the past half a year or so, and above that, also a lot of payer interactions and payer interviews. We've had national, regional meetings, so we have had a lot of interactions with the physicians across the board. Feedback so far has been very good. There is a large interest. I think many people see this as a best of two worlds because you have a long-acting product that needs to be administered, you know, seldomly, but with the convenience of an auto-injector pen device.
Yeah. Okay, got it. Thank you. Maybe just on the competitive risk, what is your thoughts on Crinetics's oral paltusotine, particularly following the PATHFNDR- 2 data?
I think, yeah, I mean, paltusotine is definitely more perhaps competitive, I would say, than Mycapssa, which has been on the market for a couple of years. In the latter case, of course, it's a twice-daily administration with fasting conditions, whereas the paltusotine product requires only once daily administration. However, I think, I mean, the market is very, as I said before, long-acting injectables are dominating the market. We have very good positive feedback to the product profile and the clinical data that we have shown, including the improvement of symptoms compared to standard of care, and also improvement in terms of quality of life characteristics in those studies.
I do believe we have the data and the profile to support a successful launch in the US.
Okay, perfect. Thank you. Maybe now let's shift over to GEP-NET, given that you've just mentioned it on your slide was almost a $2 billion opportunity. So you, you've got your phase III. We get data for that in the first half of 2025?
Yes.
I think the aim is to show superiority versus Sandostatin and Somatuline. So can you just comment again on the kind of the specific reasons that underpin your confidence there? And then just on that 35% treatment difference that I think you, you've been assigned to, to hit, is this the, a, a bar that has been set by the FDA, or is it?
No. Well, first of all, so the hazard ratio of 0.65, which we have in the study, was a bar that we have set ourselves based on the data and the collective data that we had access to when we designed the study. So that's and that includes both exposure response data for PFS that were in our relationship with Novartis, but also literature data. And I think we do have confidence in reaching the 35% treatment difference. Of course, I think it's even a 25% improvement would probably or very likely result in a positive treatment, a positive statistical superiority outcome. So we feel that based on the data we have access to and the published studies, this seems to be a reasonable approach, well-founded.
Got it. Okay, that's super helpful. Thank you. And then just maybe on Somatuline and sandostatin, I think some are kind of viewing both of those comparators as quite a high bar, and I think they're just thinking about the phase III from both of those, so the CLARINET and the PROMID. But I think patients that were recruited into those were a lot healthier in terms of grade of GEP-NET.
Yeah.
It was more towards the 1s, whereas SORENTO is more 2, 3.
Yeah.
So if you can just, you know, talk a little bit about that.
Yeah. So, in our SORENTO trial, we are recruiting quite broadly in terms of patient characteristics with the GEP-NET. A majority, I can say, of our patients are Grade 2 patients, so with the Ki-67 values, which is the proliferation values between maybe 3% up to 15% is the most common range. And the data, looking at historical data, I think this is really a sweet spot in terms of being able to show improvements because the progression-free survival times are a bit shorter. And over the course of the study that we are anticipating, we believe that we'll be able to demonstrate the treatment difference here. So I think that's the main difference, whereas CLARINET was mainly patients below 2 or so in Ki-67 and Grade 1, as you said before.
Thank you. And then maybe just to round out GEP-NET, just your thoughts on Novartis' Lutathera, and yeah, what the competitive risk is there?
I think Lutathera is, I mean, obviously a very interesting product, and they, I think today they published or should have published at least the, the GEP, the NETTER-2 data in, in Lancet. So which shows that the, the product is very effective also in grade three patients. I think it's important to know that in those studies, octreotide is used together with Lutathera, and that's also in the label. I think somatostatin analogs first generation will remain first-line treatment for a majority of patients. However, if you go to more advanced disease, of course, it makes sense to start out treatment with Lutathera, and that's going to happen. It has no negative impact on us. I think it's more, more a synergy, I would say.
Got it. Fantastic. Maybe the last one just 'cause of the time. So onto your GLP-1, so you said it on the first Q call and presentation that you plan to initiate a phase I with Novo's semaglutide. So maybe you can just help us understand kind of what's going on there, the intention to potentially partner, and then potentially if we should expect broader application of the FluidC rystal tech to large molecules.
Yeah, I think we see an interest in the potential of developing a once-monthly semaglutide or other GLPs for that matter as well. And we have done a number of studies, preclinical studies in different models, and those have been positive to our understanding, so to speak. We the both pharmacodynamic response in terms of weight, in this case, it's not weight loss because you often have growing animals, but in terms of reduced weight gain and other parameters, tolerability have been positive. So based on that, we have decided to go and start a phase I trial, and with a certain treatment modality, you could say, or dose regimen, which we think we have optimized during the course of this work. But basically, it's of course an area with large interest, and there are other opportunities as well as in this space.
Okay, fantastic. I don't think we've got time for another one, so wrap it up there. Thank you very much.
Thank you.