BioArctic AB

Thank you so much. Good morning and welcome to Biotic's presentation for the first quarter of 2025. Biotic has now entered a new era, an era of profitable growth, which is signified by an extraordinarily strong first quarter and a lot of activities throughout the different parts of the business. I will talk more about that in today's presentation and we take the next slide, please. I'm Gunilla Oswald, I'm the CEO of Biotic, and I will share today's presentation with our CFO, Anders Martin Lööf. Next slide, please. Biotic is listed at Nasdaq Stockholm large cap, and this is our disclaimer. Next slide, please. As I just said, Biotic has now entered a new era, an era of profitable growth with continuous and increasing royalties, also supported by milestones, which is reflecting the benefit of our successful partnership business model. This enables even further activities across the business as we develop new and better treatments for patients in an increasing number of severe brain disorders. Let's look at some of the highlights this year. First of all, Lekembe is now approved in all major markets, including US, Japan, China, and Europe. We see continued growth across markets and more and more patients are getting access to Lekembe. Continued development of more convenient dosing and more convenient diagnosis will continue to drive the growth going forward. We are very excited about the FDA recently approved the first blood test, which is used to diagnose Alzheimer's disease. So that means that even more patients will be able to be treated with Lecambi. I continue to be very excited about our brain transporter technology. The significant deal with Bristol Mild Squibs for our pyroglyph A-beta antibodies for Alzheimer's disease, including BAN 2803, which is utilizing our brain transporter technology, that has now come into effect since February. And this agreement also opens up for further partnerships utilizing our brain transporter technology. It's also great to see how Exidamnema progress. The phase 2a study started late last year and is progressing really well and according to plan. All patients have already completed dosing in the low dose part of the study and we have expanded the study to also include multiple systemic atrophy patients now. The quarter showed a record high profit due to several one-time events, including the upfront from BMS and milestone from ASI, as well as continuous royalties on Lecambi. Based on these achievements, Biotic has now entered the new era, and I call that Biotic Growth Area. And this is based on continuous and growing royalties, quarter on quarter, which gives us revenues. So we are not anymore dependent on irregular milestones. Biotic will get even more stable finances and we expect to be profitable from this year and onwards. Our brain transporter platform is validated with strong partnering interest and it could potentially lead to several partnerships in the future. Our Alphas Nuclein portfolio with Exidavnamab and BT2238 is also receiving a lot of interest amongst external partners. Next slide, please. Before I go into details, just a short reminder of the two platforms that are forming our company. We are among the world leaders as innovators in these two areas. The first one is highly selective antibodies targeting aggregated forms of misfolded proteins in Alzheimer's disease, Parkinson's disease and ALS. The other area is our brain transporter technology, which helps biological treatments to get better into the brain through the blood brain barrier. And that makes it higher concentrations in the brain of the treatment close to the target. This means that it has potential for even better effect, better safety profile, and lower doses, which makes it more convenient for patients. Our business model focus on innovation of new treatments for neurodegenerative disorders, but we are also now a platform company with the brain transporter technology. Next slide, please. Now I would like to start with Lekembe, where our partner ASI is doing a tremendous work on the regulatory side. The European Commission finally granted the market authorization for Lecambi in Europe, 15th of April this year. And this enables potential access to Lecambi in additional 30 countries. It's indicated for the treatment of adult patients with a clinical diagnosis of mild cognitive impairment and mild dementia due to Alzheimer's disease. We call those together early AD. And it is for patients who are APOE for non-carriers or heterozygotes with confirmed amyloid pathology. So homozygotes, which is about 10 to 15% of the Alzheimer population, they are excluded in Europe. The next step now in Europe will be to work on the controlled access program and post-authorization safety study and education material for the introduction into the market, and then local price and reimbursement discussions in each country. In total, Lekembe is now approved in 44 countries, and we are awaiting decision in 12 additional countries. The European regulator approval that may serve as a reference for regulator authorities in other countries where submissions are filed. In order to make Lecambi administration even more convenient for patients, ASI are progressing different lifecycle management activities. The first one is the IV maintenance dosing less frequently and that will then be able to give it every fourth week and that has now been approved in the US. This means that after 18 months of dosing patients they could consider to transfer into dosing every four weeks instead. This supports long-term treatment. And this is important to continue the treatment, to continue to clear the toxic species of amyloid also after that the plaques are cleared. And this is a clear benefit for Lekembe. ASI are also preparing for a broader submission of IV maintenance across markets. Another benefit for Lekembe is the subcutaneous autoinjector, which is making treatment even more convenient for patients. The FDA has accepted the filing for maintenance dosing with the PDUFA date of August 31st this year, so end of August. ASA is also preparing for a supplementary BLA submission to the FDA for the subcutaneous autoinjector for induction treatment after the approval of the maintenance treatment. Next slide, please. Lekembe data were presented at the latest key Alzheimer Congress, the ADPD Congress. I think it was very reassuring to hear that real-world use confirms FDA prescribing recommendations with similar rates of side effects as in the Clergy-AD study when it's now being used in the real world. Data from the UK and the EU indicated population, which is excluding APOE4 homozygotes, which I said is about 10-15% of the population. Data from that population were also presented, confirming that the effects of leucanumab were similar to the overall population in the CLARITY-AD study, and with lower risk of area events. We are now looking forward to coming congresses. And the next one is AAIC in July in Canada, where ASI will present data from CLARITY-AD open label extension study, supporting early treatment and maintenance dosing. And now we expect four year data to be presented and more real world evidence data. On the commercial side, ASI are progressing commercialization of Lecambia across markets, and this is then adjusted to each market depending on the time point for market entry. The first phase ASI call and that's now where EU and Asia is. And that's the areas where Lecambia just recently has been approved. Then when marketing continues, then they come into the demand creation phase. And this is like where China is now, which was launched last year. And then the third phase is demand expansion phase. And here we see Japan having had the best launch. And now the US is also entering into the demand expansion phase. In EU, ASI aims to start launch later this year. As for most launches, the first countries tend to be Germany and Austria. And at Biotic, we are preparing for a Nordic launch together with ASI, pending local reimbursement discussions. And the launch is estimated to start first half of next year. Next slide, please. I want to point out three major things that could broaden the use of Lekembe and accelerate the uptake. The first one is more convenient dosing. So less frequent IV dosing is now approved in the US. And the next step is subcutaneous autoinjector. And that will make it even more convenient for the patients. The first BLA has been submitted for maintenance dosing. After the approval that we're hoping for end of August, then ASI plan to submit a supplementary BLA for subcutaneous autoinjector for the induction phase. The subcutaneous autoinjector will make the treatment much more convenient for the patients and for caregivers. So it's more possible to give this as home administrations with less requirements and lower costs for administration compared to IV infusions. The second part is the simplified diagnosis based on blood-based biomarkers. It's very exciting with the first approval by the FDA last week of the first blood-based biomarkers, which has been accepted as confirmational for the diagnosis, making the diagnosis considerably simplified and increasing opportunities for primary care to make the diagnosis. This could also shorten in queues and freeing up time for specialists to focus on eligible patients. EISA is also increasing focus on primary care in different approaches. And also working on direct-to-consumer advertisement- in order to increase access to early patients that could benefit from Lekemvi. The third aspect is broadened indication to even earlier stages of Alzheimer's disease. And now we talk about pre-symptomatic individuals. So even before symptoms, but the individuals have elevated levels of a beta in the brain. The ongoing AHEAD 345 study will evaluate the effect when starting treatment of leucanumab very early. I think this is a great opportunity, especially based on all the data that has been presented at congresses on early patients, alluding towards even better effect when you start treatment early in the disease. Next slide, please. Then let's turn into our brain transporter technology. The license agreement with Bristol Myosquibs is a key event for BioRTIC. It's a global license for our pyroglyph beta antibody programs, and it includes both band 1503, the naked antibody, and band 2803, which is utilizing our brain transporter technology. This partnership was effective from the 20th of February this year. I think BMS is a great partner for our Pyroglyph Abita program with patients in focus. We are now preparing for BMS to take over the projects and drive it towards patients. This is our largest agreement which has been signed so far and one of the largest globally for such an early project. It includes 100 million US dollars upfront and another 1.25 billion US dollars in milestones, plus tiered low double-digit royalties on global sales. Importantly, BioRTIC retains all other rights to the brain-transported technology outside of the pyroglycemic beta field. And we have several interested partners and good discussions ongoing. At BioRTIC, we also continue to develop the brain-transported technology further, and we are also working on broadening it into other modalities like enzymes and antisense oligos. Next slide, please. Now let's turn to Exidavnimab, which is targeting toxic aggregated forms of alpha-synuclein. And it's a potential disease-modifying treatment for several different neuronal synucleinopathies. There are no existing disease-modifying treatments and a huge medical need with opportunities to help patients with Parkinson's disease, Lewy body dementia, Parkinson's disease with dementia, and multiple system atrophy. Exedavnamab is the most selective alpha-s-nuclein antibody that I'm aware of, with more than 100,000-fold selectivity for the pathological forms of alpha-s-nuclein while sparing the physiological monomers. We have very strong preclinical data showing reduction of the toxic forms of alpha-s-nuclein and delaying disease progression and increasing lifespan in very tough Parkinson's disease mice models. The two phase one studies performed showed excellent PK profile, and Exedabnema was well tolerated, supporting progression into phase two. Last week, the patent for Exedabnema was also granted in Europe, and it has previously also been granted in the US and in Japan, and we have a long patent life, up to 2046, including extensions. Next slide, please. The phase 2a study is called EXIST and it's progressing really well. The low dose part in patients with Parkinson's disease has been fully recruited and dosed. We are now preparing for a safety readout this summer. We're excited also about that we now will include MSA patients into this trial and approval has been granted in both countries, both in Spain and in Poland. We're also pleased that the FDA has granted Exidavnimab Orifant Drug designation for MSA. I think Exidavnimab offers opportunities to support several different neuronal synucleinopathies like Parkinson's disease, Lewy body dementia, and multiple system atrophy. Next slide, please. A couple of words on MSA. There are no disease modifying treatments available and a huge unmet medical need. It's a tough disease. It's fatal and rapidly progressing with a lifespan from diagnosis of about six to eight years. Patient suffers from motor symptoms and autonomic dysfunction. It is an orphan drug indication and we have received orphan drug designation in the US for Exidamnab, which is beneficial for continued drug development. Now new biomarkers are being developed both for diagnosis and to follow disease progression. And this is also improving the possibilities for drug development. MSA alone as indication is of blockbuster level. And Exedabnimab has also other opportunities with other even larger indications. Next slide, please. So in summary, our portfolio is progressing really well from innovative discovery and all the way to market, helping patients with devastating diseases. And today in the presentation, I have highlighted Lekembe, Exedavnimab, Pyroglyoebita programs and our Brain Transporter platform. And I'm pleased to see that biotics innovation and research with high quality is also being recognized externally. Next slide, please. And by that, I hand over to Anders Martin Lööf for the financial summary.

Thank you. If we then turn to the next slide, starting with the Lecambi sales, the global Q1 sales were 14.7 billion yen. That's roughly $96 million. That's roughly an 11% increase from the fourth quarter last year, or a 380% increase for the first quarter of 2024. You see the royalty numbers in the graph to the left of the box. And they look a little bit weird. The recorded royalty for the quarter was 96 million Swedish. But that includes a currency effect from the Q4 royalty of 5.7 million Swedish. So the real royalty for Q1 was 101.7 million. So we saw solid growth in the underlying royalties in line with the sales, basically. If you then look at the different markets globally, I would really want to highlight Japan that has been incredibly successful. There we saw a 9% increase from the fourth quarter last year up to 4.4 billion yen or $29 million. We also saw solid growth in the US 4% growth up to $52 million. And then it looks like we have stellar growth in China going up 46% from the fourth quarter. But that's really choppy, so it's hard to draw that many conclusions from that. But it's really reassuring to see good growth in all the markets. And all in all, ASI beat their full year forecast by roughly 4%, reaching 44.3 billion yen, or roughly $290 million in full year sales in their fiscal year 2024. Looking forward a little bit, as Gunilla mentioned, the US market is now following Japan into the demand expansion phase, as ASIC calls it. And what that really is about is that they try to focus on leveraging the core data that emphasizes the benefit of early treatment of the patients. And the patients that are in the earlier stages in mild cognitive impairment, almost all of them are treated in primary care and the vast majority of them have not even been diagnosed. So it's a big imperative to really focus on increasing engagement with the primary care practitioners to encourage coordination with specialty care so you can get more of those patients into treatment. So ASI is now targeting roughly 2,000 primary care practitioners during the year with their sales force. And Learning from Japan, they're also now starting direct-to-consumer campaigns to increase brand awareness and disease awareness to really create more approval from the patients. And of course, this is facilitated by the streamlining pathway that Kinil also talked about. First of all, we'll see the start of the use of the blood-based biomarkers for diagnosis, starting already this year in 2025, with the first approval earlier this year. probably the inclusion in the treatment guidelines during the summer and as a second step the subcutaneous administration will be approved for maintenance in the third quarter of this year and hopefully also then for induction for in the first half of next year So I think we will see some effect of this increased focus on primary care practitioners in the US already this year. But I expect the full effect to be seen basically in 2026. So it will be very interesting to follow this going forward. Based on that, if we turn to the next slide, ASI has updated a forecast for their fiscal year 2025, where they believe the LeCambie sales will grow by 73% globally to 76.5 billion yen. As you can see in the chart, they expect 53% growth in the US, but stellar 88% growth in Japan, and then even higher growth in China and in other markets. If this happened, this would correspond to roughly 510 million Swedish krona in royalties during the same time period. And over time, ASI has issued a revenue simulation in March of roughly 250 to 280 billion yen in 2027. And if you look at the growth rates in the coming years that are necessary to reach that, they are basically expecting similar or even higher growth in the coming years. One question we always get is how likely is ASI to reaching this forecast? And we think it looks really good. They indicated during their quarterly update with analysts that Already in April, the sales in Japan, for example, were 2 billion yen. So they're already at the level that would indicate an annual level of 24 billion yen. So if they keep growing in Japan, it looks possible to actually beat the forecast in Japan. And hopefully, we will have a similar situation in the other countries. So we remain very confident that they can reach this global forecast. Turning to our financials on the next slide. On the left-hand side, you see that our revenues have increased from 30 million last year to 1,290 million Swedish this year. Fairly large increase. This is, of course, driven by the upfront payment from BMS of $100 million. But we also see the milestone payments from ASI of 10 million euros, which was a sales milestone. The EU approval milestone of 20 million euros was not recorded in the first quarter, but it will be recorded in the second quarter. This is also supported by the royalty revenues. I've already mentioned that there were 96 million, and we also had some co-promotion revenue, so 3 million. But over time, as we have said in the past, these recurring revenues will become more and more important. But in this quarter, they are really dwarfed by the enormous upfront payments and milestone payments. Looking a little bit at the costs in the middle, the operating expenses increased to 203 million in the quarter. The big increases is mainly explained by an increase in other operating costs, which was 72 million. And this is basically a currency effect on the upfront payment from BMS. If you deduct that and just look at the underlying operating costs, they were 131 million, which is roughly 30% higher than the year before. And out of that, R&D makes up 65%. So the vast majority of our costs are spent on R&D and really accelerating our portfolio. and the costs are expected the underlying costs I should say are expected to increase during 2025 as our project portfolio progresses and we will also increase our commercial expenses when we intensify the preparations for the launch of the Cambrian and Nordics I have previously said that I expect that the cost to increase by roughly 50 to 80 percent in this year I should probably revise the lower The lower limit there to roughly 60% because we will have this, I did not anticipate this currency effect. So if I would guess today, I would say that our costs will increase by roughly 60 to 80% this year compared to last year. Then on the right hand side, you see our operating profit, which was almost 1.1 billion Swedish in the first quarter. I'm sad to say, but the remaining quarters of the year will be slightly less profitable than that. So we expect the full year profit roughly in line with the first quarter. But as Gunilla already mentioned, we expect to remain profitable for the coming year. So it's really a big shift for us becoming more of a profitable company going forward. On the last slide that I will show on the next slide, you see the net result. It's roughly 55 million Swedish below the operating profits. And that explained by a financial net of a negative 9 million. And we have also recorded a tax of 44 million in the quarter. The cash flow is much weaker than the result, and that is also explained by the upfront payment from BMS. It was recorded in the first quarter, but we received the money in April. So we didn't see the cash effect of that. But we did get the money for the sales milestone of 10 million euros. So at the end of the first quarter, our cash balance was roughly 800 million Swedish. But already a couple of weeks after the closing of the quarter, we received a billion more. So following us in the remainder of the year, you will see that our financial position will strengthen in the coming quarters. from a cash perspective. So all in all we are in really good shape and are really looking forward to investing more and working hard to accelerate our portfolio and focus on the launch of Lecambri in the Nordics. With that I turn back to Gunilla for some final remarks.

Thank you so much, Anders. So we're coming to the final part of the presentation with upcoming news flow and some closing remarks. Next slide, please. But before that, I want to welcome you all to our first Capital Market Day, which is planned for 2nd of June. It will be live in Stockholm and it will also be live streamed. We're looking forward to giving our shareholders an in-depth look at our business and our ongoing research. It will be presented by some of my colleagues in the management team. And there will also be an opportunity to listen to a distinguished scientist and clinician who has treated several hundred patients with Lekembe in the US. If you're interested, then please register at www.bioartic.com. The last day of registration is 28th of May. Next slide, please. So our upcoming news flow. I think that this second quarter has started really well with several regulator approvals for Lecambi in, for example, EU. And we're looking forward to more regulatory responses. The next important congress is AAIC, where we look forward to several presentations on Lecambi. During the third quarter, That's actually where AAIC is, the third quarter. That's, of course, the regulator response on subcutaneous autoinjector as maintenance treatment in the US, and ASI subsequent filing for induction treatment with the subcutaneous autoinjector. And we also look forward then to the Exidamnumab safety review and progression into the high-dose part in Parkinson's disease and also in MSA in the EXIST phase 2A study. Next slide, please. So in summary, this quarter was an amazing quarter with a record high profit. Lecambi is now approved in all major markets and more and more patients are getting access to treatment. We concluded our first license agreement utilizing our brain transporter technology and it continues to generate great external interest. The Exidabnumab indications are now expanded and we are preparing for several different opportunities for Phase 2b. The profits so far this year are great and it will make Biotic highly profitable for this year. Next slide, please. So by that I say thank you for your attention and we're happy to take some questions.