Egetis Therapeutics AB (publ) ($EGTX)

Welcome to Egetis Therapeutics Q1 Report 2026. [Operator Instructions] Now, I will hand the conference over to CEO, Nicklas Westerholm. Please go ahead.

Good morning, and a warm welcome to our quarter 1 results plan for the coming 30 minutes. For those who haven't had the privilege to meet before, my name is Nicklas Westerholm, I'm the CEO of the company. With me today, we also have Yilmaz Mahshid, Chief Financial Officer; Henrik Krook, Vice President, Commercial Operations; Anny Bedard, President of our U.S. Business; and Karl Hard, Vice President and Head of Investor Relations. Let's turn to our attention to the topics covered today depicted on the slide in front of you. To summarize, quarter 1 has been historical for Egetis with several key milestones delivered. Amongst others and most importantly, the FDA acceptance of our new drug application for Emcitate for the treatment of MCT8 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, 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 1, building on the great momentum in 2025 and position us well for the Emcitate U.S. launch in quarter 4 this year. We completed the U.S. new drug application, i.e., NDA for Emcitate in January. The NDA for Emcitate 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 Emcitate available to MCT8 deficiency patients in the U.S. with an expected launch in quarter 4 this year. Furthermore, in March, the U.S. Patent and Trademark Office issued a Notice of Allowance for an MCT8 deficiency composition patent. They allowed claims cover, among other things, a method of treating MCT8 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 SEK 350 million. The issue was priced at SEK 5.25 per share, corresponding to 0 discount to market [ close ] and more importantly, with very strong participation from new and existing investors focused on the U.S. biotech investors like Frazier Life Sciences, Invus, Woodline Partners and Parkman Healthcare Partners. This means that we are now well placed to execute on our strategy of progressing our lead asset, Emcitate through U.S. approval and launch. And with that, I will now hand over to Anny Bedard to provide an update on our launch preparations in the U.S., our most important market. Anny, 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 Nick said, the U.S. launch is on track for Q4 2026, and this is supported by expanding patient identification, increasing experience with tiratricol 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 health care professionals, sharing emerging data, advancing the earlier recognition and diagnosis of MCT8 deficiency. Just as importantly, we continue to build strong relationship with the MCT8 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 tiratricol. 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 1. 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 tiratricol and having constructive early discussions with payers. This supports our confidence in a successful launch at approval and over time, establishing tiratricol as the standard of care with MCT8 deficiency. Thanks for your attention, and I'll now turn it over to Henrik for an update on commercialization in Europe.

Thank you, Anny, 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 Emcitate revenue was SEK 13.4 million. This was 9% higher than Q1 2025 at constant exchange rates, but lower than Q4 2025. The difference versus Q4 was primarily for 2 reasons: normal fluctuations in ordering patterns and accrual of rebates in Germany as we await 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 MCT8 deficiency. And during the quarter, we exhibited at 4 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 Q1, 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 Er-Kim in Turkey plus Central Eastern and Southeastern Europe and by Taiba rare in the Gulf region. Both Er-Kim and Taiba rare are actively identifying patients and initiating processes aiming for funded treatment, which contributed to named 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, Yilmaz Mahshid.

Thank you, Henrik. To start off with all numbers unless called out are in operating currency, Swedish krona. Revenue for the first 3 months were SEK 13.4 million versus SEK 12.7 million in the same period last year, corresponding to a year-over-year growth of 9% in constant exchange rates. As you can see, the gross profit is negative SEK 0.2 million. This comes back to the continued depreciation of balance sheet R&D, a noncash item. Excluding the quarterly depreciation of SEK 10.1 million, gross profit would have been SEK 9.9 million, corresponding to an adjusted gross margin of approximately 74%. Quarter 1 2026 operating results were negative SEK 92.9 million versus negative SEK 62.9 million in the prior period. The lower results are due to our continued investments and work with the U.S. NDA, U.S. commercial buildup and corresponding prelaunch activities. The management, the Board and our main shareholders are aligned that investing in the U.S. 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 SEK 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 ReTRIACt Study and the hard and intensive work conducted by the team for the U.S. NDA in quarter 4. For the first quarter, cash flow from operating activities were at negative SEK 64.3 million versus negative SEK 66.1 million. We ended the quarter with a cash position of SEK 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 SEK 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 SEK 5.25, corresponding to a 0 discount to the market close the same day. Interest to participate came largely from foreign specialists and long-only investors such as Frazier Life Sciences, Invus, Woodline Partners and Parkman Healthcare Partners, but also from the Swedish health care specialist Linc AB. These investors have great insights into the U.S. pharmaceuticals market and what it takes to succeed there. With that, I hand back to Nicklas.

Thank you, Yilmaz. 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 U.S. FDA for the 28th of September. And upon approval, we expect to be granted a priority review voucher, which will likely to monetize in quarter 4. Of note here is that the PRVs sold in 2025 and 2026 have fetched in the region of USD 150 million to USD 200 million each. Furthermore, and as mentioned before, we expect the launch of Emcitate in the U.S. in quarter 4. 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.

[Operator Instructions] The next question comes from Chiara Montironi from Van Lanschot Kempen.

Congrats on the successful quarter. 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 PDUFA date in September? And another one will be around RTH-beta. So I was wondering how are you thinking about the design of this trial and the timing and any learnings from MCT8 that you can directly apply to RTH-beta?

Thank you, Chiara, and good morning and also grateful that you acknowledged our very successful quarter. So the first question you have 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, as 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 insight 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 MCT8 deficiency will be supportive also on an RTH-beta opportunity driven by very much similar dosing. So the next steps here is to conclude the design of a pivotal study. As I'm sure you have seen, Chiara, we have a new incoming CMO. This will be his first task with the ambition to conclude the 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. Ambition is to start a pivotal study at some point during 2027. Thank you Chiara.

The next question comes from Arvid Necander from DNB Carnegie.

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-over-quarter decrease. And then just on the full year sequencing, you said you expect higher sales in Q2, assuming that reimbursement is achieved at an adequate level in Germany. Do you expect the initial stocking up effect at centers that are sort of already up and running with MCT8 deficiency programs or should we expect a more linear sequential growth through 2026? And second one on R&D costs, if I may. How much is left to pay off from the ReTRIACt Study and with recent capital raise 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.

Thank you Arvid, and great to have you on the call. You have 2 questions, one around the European commercialization and the performance there and expectations in the future. And the second one was my understanding around R&D costs and potential additional payments for the closing down of the ReTRIACt Study. 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 accruals 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 2. We also mentioned, as you rightly said that we expect an increase in quarter 2 versus quarter 1 from a revenue perspective. This is very much driven on the totality, actually, our partnerships in Turkey, Gulf region and Central Eastern 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 what I just said.

Yes. I think you covered it well, Nicklas. 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, Henrik. And then we had a question around R&D costs, Yilmaz?

Thanks for the question. No, we don't have so much cost left for the wind down of the ReTRIACt Study, 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 RTH-beta at these levels.

Thank you Yilmaz and thank you, Arvid for your questions. Operator, next question, if you don't mind.

The next question comes from Oscar Haffen Lamm from Stifel.

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 -- yes, sorry, Oscar, 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 adequately accrued for what we believe will be a reasonable rebate in line with industry standards. That to aside, I can't share any more details.

Okay. No worries. Then maybe just a quick follow-up since we're still on 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 1st of May? Is it in line with your expectations? I mean how is the dynamic in terms of patient certification, how they are taking a new product, et cetera?

Yes. No, I think -- and again, I think it's aligned with our expectations. But bear in mind that our expectations have always been based on the [indiscernible] model, 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 mark 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 quite a few additional patients 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 color to this.

Yes. Thank you, Nicklas. And I agree, I think it's in line with our expectation. I mean, overall, Germany is 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 specialists 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 Emcitate treatment, the adherence to treatment has been very high. Both caregivers and physicians have expressed positive feedback regarding treatment effects.

Yes. Thank you so much, Henrik. And that takes us to the end of the call today. I just would 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.