Egetis Therapeutics AB (publ)

Welcome to Agedis Therapeutics Q1 Report 2026. For the first part of the conference call, the participants will be in listen-only mode. During the questions and answers session, participants are able to ask questions by dialing pound key 5 on their telephone keypad. If you are listening to the presentation via webcast, you can ask written questions using the form below. Now I will hand the conference over to CEO Nicholas Westerholm. Please go ahead.

Good morning, and a warm welcome to our quarter one results plan for the coming 30 minutes. For those who haven't had the privilege to meet before, my name is Nick Rousseff, and I'm the CEO of the company. With me today, we also have Yilmaz Mashi, Chief Financial Officer, Henrik Lund, Vice President, Commercial Operations, Annie Bedard, President of our U.S. business, and Karl Hahn, Vice President and Head of Investor Relations. Let's turn our attention to the topics covered today, depicted on the slide in front of you. To summarize, Quarter 1 has been historical for EGTIS, with several key milestones delivered. Amongst others, and most importantly, the FDA acceptance of our new drug application for the treatment of MCTA deficiency. Where the Agency also granted us a priority review, with a so-called PDUFA target action date, i.e. decision date, set to September 28, 2026. We will also provide an update on the launch preparations in the U.S., as well as the commercialization in the European Union. And furthermore, a financial update will be covered by our CFO. And we aim to leave ample time for Q&A at the end of the session. So let's turn our attention to the highlights of the quarter. The very strong execution continued in quarter one, building on the great momentum in 2025. And positionals well for the MC-8 US launch in quarter four this year. We completed the US new drug application, i.e. NDA, for MC-8 in January. The MDA for MCTA was accepted by the FDA on the 27th of March. We were also granted a priority review and the PDUFA target action date set to 28th of September. Furthermore, the agency stated that it's not currently planning to hold an advisory committee meeting to discuss our application. So all in all, this is a major achievement and represents an important step in making an incitate available to MCT deficiency patients in the U.S. with an expected launch in Q4 this year. Furthermore, in March, the U.S. Patent and Trademark Office issued a notice of allowance for an MCT deficiency composition patent They allow claims to cover, among other things, a method of treating MCT deficiency with a pharmaceutical composition, encompassing theoretical, including dosing regimens, a theoretical composition with specific excipients. This represents a significant milestone in strengthening our intellectual property, and we expect the resulting patent to be listed in the Orange Book, with an anticipated expiration date in 2045. Last but not least, in April, we successfully carried out an oversubscribed directed share issue amounting to 350 million Swedish kronor. The issue was priced at 525 Swedish kronor per share, corresponding to zero discount to market close, and more importantly, with very strong participation from new and existing investors, focused on the U.S. biotech investors like Fraser Life Sciences, InWoods, Woodline Partners, and Parkman Healthcare Partners. This means that we are now all placed to execute on our strategy of progressing our lead asset, MCTA, through U.S. approval and launch. And with that, I'll now hand over to Annie Bedard to provide an update on our launch preparations in the U.S., our most important market. Annie, please.

Good day, everyone. So let's now turn to the United States, our most important commercial market and a key driver of long-term growth. As Dick said, the U.S. launch is on track for Q4 2026. and this is supported by expanding patient identification, increasing experience with Teratricol in early access program, and continued progress on market access readiness. Starting with patient identification, we've now identified more than 140 genetically confirmed patients, and that number continues to grow each quarter. Our field medical team is now fully deployed engaging specialist centers that are central to patient identification and treatment. They're engaging with physicians across endocrinology, neurology, and clinical genetics with a focus on centers already managing known patients, increasing disease awareness, and enabling meaningful clinical dialogue and accelerating diagnosis and referral pathways. We're also expanding our presence at key scientific meetings, including the American College of Medical Genetics, which took place in the first quarter, and the Pediatric Endocrine Society, which is taking place this week and is our most important meeting. These engagements are critical to connecting with healthcare professionals, sharing emerging data, advancing the earlier recognition and diagnosis of MCTA deficiencies. Just as importantly, we continue to build strong relationships with the MCTA caregiver and advocacy community. This engagement helps us better understand the patient journey, ensure caregiver perspectives inform our plans, and strengthen the trust with the families. Our U.S. Early Access Program continues to expand with 50 patients now receiving treatment across 20 specialist centers. This program enables access for patients with significant unmet need, while also providing physicians with real-world experience managing teraprical. These insights are helping us inform our implementation approach, deepen engagement with specialist centers, and better define the support that will be required for both patients and providers over time. We're also making solid progress in building the access and patient support infrastructure that are needed for launch. Our work has now moved from implementation to execution, with national account directors now engaging Medicaid and commercial payers to communicate the seriousness of the disease, the lack of approved treatment options, and to strengthen our clinical and real-world evidence. In parallel, we've selected our 3PL and specialty pharmacy partners, and we're advancing our patient support program, which will include the specialty pharmacy integration, benefit verification, and the patient services to ensure that we have seamless access for both patients and physicians from day one. Finally, we're building the team to deliver on this opportunity. We're establishing a U.S. organization led by experienced rare disease experts across medical affair, market access, and commercial with a strong track record in rare disease and first-in-class launches. Over the past quarter, we've continued to expand these functions to ensure launch readiness, and we remain on track to reach approximately 25 U.S. colleagues at launch. what we believe is the right scale to capture the opportunity while maintaining capital discipline. So together, this progress is translating into strong launch readiness. We're gaining greater visibility into the patient population, seeing strong engagement from specialists. We're addressing the needs of patients with high unmet medical need with terapicals. and having constructive early discussions with payers. This supports our confidence in a successful launch at approval, and over time, establishing Teratrical as the standard of care with MCTA deficiency. Thanks for your attention, and I'll now turn it over to Henrik for an update on commercialization in Europe.

Thank you, Anne, and good morning, everyone. In Europe and for our international business, we continue to build momentum heading into the year, supported by increasing patient identification, expanding access, and a broader geographic footprint. In Q1, the entity's revenue was 13.4 million SEK. This was 9% higher than Q1 2025 at constant exchange rates, but lower than Q4 2025. The difference versus Q4 was primarily for two reasons. Normal fluctuations in ordering patterns and accrual of rebates in Germany as we raised the final reimbursed price. Importantly, these factors affect the timing of recognized sales in the quarter. They do not change our view of the substantial medical need or the momentum we are seeing. Overall, the commercial fundamentals continue to move in the right direction with more identified patients and increasing contribution from a broader set of countries and prescribers. With that, we expect the Q2 revenue numbers to be higher than the Q1 numbers. Then briefly on Germany. The pricing and reimbursement process is progressing, and we expect it to conclude in Q2. We continue to develop the market through disease awareness, physician engagement, congress presence, and education. In February, we launched a web-based certified continuous medical education program focused on MCTA deficiency, and during the quarter, we exhibited at four scientific congresses across Germany and Austria. Over the past year, several new patients have been identified in Germany, with patients progressively moving to treatment initiation. Beyond Germany, we are expanding access across additional European markets. For France, Spain and Italy, we are advancing the national pricing and reimbursement dossiers for upcoming submissions over the next months. In parallel with these national pricing and reimbursement processes, we have successfully established regionally reimbursed routes in Spain and Italy that we can leverage until the national processes have concluded. In June 1, we generated our first sales revenue in Italy through this regionally reimbursed pathway. We have also successfully implemented alternative reimbursement solutions in certain additional countries, including Switzerland and Austria, ensuring access and helping generate revenue. We also see growing reach through our distribution partners. We are supported by Elkim in Turkey, plus Central, Eastern and Southeastern Europe, and by Caibare in the Gulf region. Both Herr Kim and Thay Barber are actively identifying patients and initiating processes aiming for funded treatment, which contributed to main patient sales revenue in Poland and Turkey in the quarter. In addition to our existing distribution partnerships, we remain in active dialogue with potential partners to extend access in additional geographies over time. And with that, I would like to hand over to our CFO, Ylva Smarsson.

Thank you, Henrik. To start off with, all numbers, unless called out, are in operating currency Swedish krona. Revenue for the first three months were 13.4 million versus 12.7 million in the same period last year, corresponding to year-over-year growth of 9% in constant exchange rates. As you can see, the gross profit is negative 0.2 million. This comes back to the continued depreciation of balance sheet R&D, a non-cash item. Excluding the quarterly depreciation of 10.1 million, gross profit would have been 9.9 million, corresponding to an adjusted gross margin of approximately 74%. Quarter 1, 2026 operating results were negative 92.9 million. versus negative 62.9 million in the prior period. The lower results are due to our continued investments and work with the US NDA, US commercial build-up, and corresponding freelance activities. The management, the board, and our main shareholders are aligned that investing in the US is a key priority, as we believe this will be by far our most important market and where we need to be successful. I would also like to highlight, despite our higher year-over-year cost base, our total operating expenses are sequentially down by approximately 32 million. Almost all the lower cost base is R&D related, and specifically to all the costs that were taken for the wind-down of the retry study and the hard and intensive work conducted by the team for the USMDA in quarter four. For the first quarter, cash flow from operating activities were at negative 64.3 million versus negative 66.1 million. We ended the quarter with a cash position of 142.5 million. And it was gratifying to see that we did strengthen the cash position further on April 21. In total, we did raise 350 million on a gross basis. The high demand for the shares offered were depicted by the fact that they printed at a share price of 5.25, corresponding to a zero discount to the market close the same day. The influx of participants came largely from foreign specialists and long-running investors such as Fraser Life Sciences, Inverse, Woodland Partners and Parkman Health Partners, but also from the Swedish healthcare specialist Link AB. These investors have great insights into the U.S. pharmaceuticals market and what it takes to succeed there. With that, I am back to Niklas.

Thank you, Jonas. So let's turn our attention to the future. Looking ahead, we have a very exciting period ahead of us. We have the PDUFA date set by the US FDA for the 28th of September. And upon approval, we expect to be granted a priority review voucher, which we're likely to monetize in quarter four. Of note here is that the PRVs sold in 2025 and 2026 have fetched in the region of 150 to 200 million US dollars each. Furthermore, and as mentioned before, we expect the launch of MCPAY in the US in quarter four. And in parallel with that, we're going to prepare and drive forward indication expansion into resistance to thyroid hormone beta, our next pipeline opportunity. So with that, all in all, a very successful quarter and a very exciting period ahead of us. I will now hand over to the operator for question and answers. Operator, please go ahead.

If you wish to ask the question, please dial pound key 5 on your telephone keypad. To enter the queue, if you wish to withdraw your question, please dial pound key 6 on your telephone keypad. The next question comes from Chiara Monteroni from Van Lanchet Kempin. Please go ahead.

Hi, good morning. Congrats on the successful quarter, and thank you for taking my question. I have a couple, if I may. So the first one would be any update on when we can expect the publication of the survival data by Erasmus? Do you expect that to be before the TUFA date in September? And another one would be around FTH-beta. So I was wondering how are you thinking about the design of this trial? and the timing, and any learning from MCTAs that you can directly apply to FDA's data. Thank you.

Thank you, Chiara, and good morning, and also grateful that you acknowledged our very successful quarter. So the first question you had was around the publication of the so-called survival data. The survival data form a vital part of the NDA submission submitted to the U.S. FDA. There, I hope the audience will appreciate that the survival data is owned by the Erasmus Medical Center and subsequently they are directing the upcoming publication. What we know and what I can share is that the manuscript has been completed and submitted to a high-profile journal, and it's out for review. So I don't have any further insights than that, but hopefully it will be published in the not too distant future. Then when it comes to RTH beta, Chiara, we're very excited about this opportunity. It's a substantial unmet medical need also in this patient population where no treatment exists. Here we are actually have started together with key opinion leaders discussing a design of a pivotal study. That is important, recognizing that the safety data we have generated today in MCP8 deficiency will be supportive also of an RTHB opportunity, driven by a very much similar dosing. So the next step here is to conclude a design of a pivotal study. As I'm sure you have seen here, we have a new incoming CMO. This will be his first task, with the ambition to conclude a study design at some point towards the end of the summer, and subsequently then engage with both EMA and FDA to agree on an endpoint in such a study. Our mission is to start a pivotal study at some point during 2027.

Thank you, Chiara.

The next question comes from Arvid Nikatter from DNB Carnegie. Please go ahead.

Good morning, guys, and thanks for taking my questions. So the first one on sales, I was just wondering if you can provide a little bit more granularity on the bits that go into the quarter of a quarter decrease. and then just on the four-year sequencing you said you expect higher sales in Q2 assuming that reimbursement is achieved at an adequate level in Germany you expect the initial stocking up effects that centers that are sort of already up and running with NCT8 deficiency programs so should we expect a more linear sequential growth through 2026 And second one on R&D cost, if I may, how much is left to pay off from the retry study and with recent capital rates intended to support the development in RTH beta? Can you comment anything on what you are budgeting for in terms of costs for this in 2026? I'll start there. Thanks.

Thank you, Arvid, and great to have you on the call. I have two questions. One around the European commercialization and the performance there and expectations in the future. The second one was my understanding around R&D costs and potential additional payments for the closing down of the retail sector. I'll start off with European commercialization. As I'm sure you appreciate, we have seen a slight decrease quarter on quarter when it comes to revenue. As Henrik mentioned, this is very much driven by variation in order patterns. as well as accrues for rebates for the upcoming final price in Germany. And as Henrik mentioned, the pricing and reimbursement discussion is progressing well, and we expect an outcome of that in quarter two. We also mentioned, as you rightly said, that we expect an increase in quarter two versus quarter one. From a revenue perspective, this is very much driven on the totality, actually. Our partnerships in Turkey, Gulf region, and Central East, and Southern Eastern Europe are bearing fruit, and we expect an increase in all the demand there. But I'll hand over to you, Henrik, to provide somewhat more granularity to...

Yeah, I think you covered it well, Nick. And I think it's important to see that, I mean, our view on the underlying demand is the same. I mean, there is a substantial medical need, and the momentum we see in the market is positive. So, I mean, overall, the commercial fundamentals continue to move in the right direction. with more identified patients and increasing contribution from more prescribers.

So overall, positive outlook moving forward. Thank you, Elvig. And then we have a question around R&D costs.

Yes, hi, Elvig. Thanks for the question. No, we don't have so much cost left for the wind-down of the retry studies, pretty much negligible going forward from here. Most of the activities will be related to the NDA filing in the U.S. and starting the work for ArcGIS VISA at these levels.

Thank you, Yilmaz. And thank you, Arvind, for your questions. Operate the next question, if you don't mind.

The next question comes from Oscar Haytham Lamb from Stifel. Please go ahead.

Hi Tim, thanks for the question. In Germany, how confident are you that the final negotiated price will come in line with your expectations? I mean, are there different scenarios that we could expect?

Well, I think, yeah, sorry, I'll say, I think, thank you for the good question. Obviously, we don't give any guidance on our assumptions. As always in Europe nowadays, there is a challenging environment across all more or less countries when it comes to pricing and reimbursement. We have had to quickly accrue for what we believe will be a reasonable rebate in line with industry standards. That to a side, I can't share any more details.

Okay, no worries. Then maybe just a quick follow-up since we're still on the topic of Germany. I mean, we're now almost a year after your commercial launch. How would you say it's tracking since you've started on the first of May? Is it in line with your expectations? I mean, how is the dynamic in terms of, you know, patient satisfaction, how they are, you know, taking in the product, et cetera?

Yeah, and again, I think it's aligned with our expectations, but bear in mind that our expectations have always been based on the realism of how it works in Europe today, where it's tough, both pricing and reimbursement discussions as well as other components. But as we mentioned before, within a quarter we converted, within quotation marks, all the patients in our managed access program into commercial product. On top of that, as I'm sure we have discussed before, we also identified where we have also managed to get a few of them on therapy. So I think all in all, it's progressing in the right direction. But Henrik, you might want to add some more comment to this.

Yeah, thank you, Niklas. And I agree, I think it's in line with our expectation. I mean, overall, Germany's a little bit of a challenging environment. And that is also something that we hear from our networks at other pharma companies. There is a high workload on specialists involved in rare diseases. So this means that it is difficult for the pharma industry to get appointments with physicians. And more importantly, that it also can be difficult for new patients to get appointments with the right specialist within a short time frame. So this is unfortunate for some patients because it can result in slower initiation of new treatment. But overall, I mean, it's moving in the right direction, and it's important to note also that for patients who have initiated MCTA treatment, the adherence to treatment has been very high. Both caregivers and physicians have expressed positive feedback regarding treatment effects.

Thank you so much, Henrik. And that takes us to the end of the call today. I'd just like to thank the participants and for the educated questions during the call. And again, as depicted on the slide here in front of us, we have a very exciting period ahead of us with several key value enhancing milestones. Thank you, everybody, and I wish you a great rest of the day.