Welcome to Egetis Therapeutics Q2 Report 2025. During the questions- and- answers session, participants are able to ask questions by dialing pound key five 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 Nicklas Westerholm. Please go ahead.
Good morning, everyone, and a warm welcome to our Q2 results call planned for the coming 30 minutes. For those who haven't had the privilege to meet before, my name is Nicklas Westerholm and I'm the CEO of Egetis Therapeutics. With me today, we also have Yilmaz Mahshid, Chief Financial Officer; Henrik Krook, Vice President, Commercial Operations; as well as Karl Hård, Vice President and Head of Investor Relations. Let me now turn our attention to the topics to be covered today. First, let me begin by saying that I am incredibly proud of the breakthrough therapy designation granted by the FDA in July this year, as well as the initiation of the launch of Emcitate as the first and only approved treatment for MCT8 deficiency in the first country, Germany, on the 1st of May.
This truly marks the start of a transition for Egetis from a late-stage development company to a commercial-stage company. What we'll cover today is an update focusing on the breakthrough designation granted by the FDA and the submission of a pre-NDA meeting request, commercialization in Europe, and launch preparations in the U.S. by Henrik. Our CFO, Yilmaz, will provide a financial update and will summarize it at the end with the key value-enhancing milestones during 2025 and 2026 as part of our exciting journey ahead of us. I'll also take a short opportunity to provide an update on the development of Emcitate in Japan. As many of you are aware, we have an exclusive license agreement with Fujimoto Pharmaceutical Corporation to develop and commercialize Emcitate in Japan. This will be an important market going forward.
In the MCT8 deficiency space, we are aware of 67 patients identified and diagnosed with MCT8 deficiency. So far this year, we have had two positive interactions with the Japanese Pharmaceutical Regulatory Authority Agency, PMDA, regarding the regulatory development pathway required to obtain approval of Emcitate in Japan. This is in light of the new guidelines issued by MHLW in October 2024, where for orphan drugs that meet certain criteria, it may be possible to submit a marketing authorization application without clinical trial results in Japanese patients. Let's focus on the breakthrough designation and what that means. In the first quarter of 2025, we received the entire dataset from the Erasmus Medical Center in Rotterdam with the survival data from more than 600 MCT8 deficiency patients who had been treated with tiratricol compared to the ones not treated with tiratricol.
The initial survival analysis, as many of you recall, was published by the Erasmus Medical Center as an abstract ahead of the European Thyroid Association in September 2024. We submitted a breakthrough therapy designation application largely based on our own analysis, detailed analysis of this survival data, which confirmed a significant and substantial improvement in survival in tiratricol-treated patients. Based on that, the FDA granted us a breakthrough therapy designation in July this year. The breakthrough therapy designation very much underscores both the urgent need for an effective treatment for patients with MCT8 deficiency and the clinically meaningful evidence demonstrated to date with tiratricol. Receiving a breakthrough therapy designation this late in a clinical development program is for us very encouraging for the forthcoming New Drug Application process, as these designations are typically awarded at an early stage in development.
At the moment, there are 15 evaluable patients in the ReTRIACt study, up from 12 when we reported our quarter one results. In light of this and the granted breakthrough therapy designation based on our own detailed analysis of the entire survival dataset, we have submitted a pre-NDA meeting request, a so-called type B meeting in August to the FDA to discuss the contents and timing of the U.S. NDA submission for tiratricol, including the role and position of the ReTRIACt study, together with the already robust dataset generated in this ultra-orphan disease. To remind you, the data generated so far includes Triac trial I, where the effects of 12 months of tiratricol treatment on T3 concentrations and clinical endpoints related to peripheral thyrotoxicosis were established in 46 patients.
The Erasmus Medical Center cohort study confirmed these treatment effects in a real-life setting in 67 patients with treatment up to six years. The Triac trial II in 22 patients, which although missed its primary endpoint, provided important safety data in young patients below 30 months of age, which enabled a label in Europe with no age restrictions. We also have an extensive natural history study of 151 untreated patients, which enables an indirect comparison to assess the clinical relevance of the treatment effects seen with tiratricol. For those who are not familiar with the regulatory framework in the United States, let me just give you a brief explanation. A pre-NDA meeting, type B meeting, is a formal and very valuable discussion between a pharmaceutical company and the FDA before submitting a new drug application.
It serves as a critical checkpoint to ensure the sponsor or the pharmaceutical company is on track with their data, the regulatory requirements, and submission strategy before formally filing the NDA. This meeting helps also to identify and address potential issues earlier on, potentially also streamlining the approval process. For a type B meeting such as this with the FDA, the agency will aim to schedule the meeting within 60 days of receiving the meeting request. If granted, this would be in October for us. Without further ado, I will now hand over to Henrik for an update on commercialization in Europe and the launch preparations in the United States. Henrik, over to you.
Thanks, Nicklas, and good morning, everyone. I would like to start by giving a reminder and the status on our commercialization strategy. We plan to commercialize Emcitate or tiratricol through the Egetis team in Europe and the U.S. For other regions, we intend to collaborate with partner companies to ensure broad access to Emcitate for patients. We have started the execution on this strategy by initiating the launch of Emcitate in Germany in May. We have also initiated market preparations in other European countries and in the U.S. For the rest of the world, we have the license deal with Fujimoto for development and commercialization in Japan. In June, we announced our first distribution deal, an agreement with Erkim for Turkey. The Erkim team has started the work with Emcitate, ordered the dialogues related to the initiation of Emcitate with physicians having MCT8-deficient patients.
We have also advanced our work for a planned deal for the Middle East and North Africa region. We are executing our EU pricing and reimbursement strategy in two waves, starting with the four largest EU countries. The French and German reimbursement dossiers were submitted in April. For Spain and Italy, we are preparing for the initiation of national pricing and reimbursement processes. Our choice to submit at different time points in the different countries is based on our ambition to optimize the total outcome in line with our global commercialization strategy. In parallel with the national pricing and reimbursement processes in the EU four countries, we have started the work in some other European countries with the ambition to make it possible to sell Emcitate through alternative funded routes.
This is to accelerate revenue generation, and it is possible in some countries on a patient-by-patient basis and funded through insurance systems or hospital budgets. I'm excited to share some updates on our initial German launch execution, which has been delivering early wins while we navigate the pricing and reimbursement process. As of May 1, Emcitate was launched and is now available in Germany. This marks a major milestone for us as we continue to expand our reach and provide a much-needed treatment option for patients with MCT8 deficiency. We initiated the pricing and reimbursement process on April 30, and it will be finalized by May 2026. Parallel with the ongoing pricing and reimbursement process, we have, in line with what the German system allows, already started sales of commercial Emcitate packs.
This dual approach allows us to provide immediate access to the treatment and generate sales in parallel with the reimbursement process. I'm pleased to report that almost all of our managed access patients have been successfully converted to commercial packs. We expect all managed access patients to have been converted during Q3. On top of the converted patients from the managed access program, Emcitate treatment has been initiated in a few new patients. Our engagements with physicians at center meetings at congresses have been productive, driving awareness of MCT8 deficiency, finding more patients, and leading to initiation of treatment with Emcitate. We have exhibited at seven congresses in the DACH region year to date, and DACH stands for Germany, Austria, and Switzerland. To complement our in-person meetings, we have also launched promotional educational initiatives through various channels, including virtual meetings, web mailings, and publication-related work.
These efforts are part of our comprehensive strategy to ensure that Emcitate is widely recognized and accessible to all who need it. We are thrilled with the progress we have made so far and remain dedicated to serve more patients in Germany going forward. I believe all of you are aware that the U.S. is expected to be the largest market for tiratricol, and I'm pleased to share that our U.S. team is doing an excellent job in the market preparations. On the patient front, we continue to deepen our understanding of the journey for people living with Emcitate deficiency. Like most rare diseases, the care pathway is complex, and we are using real-world data analytics to gain a more granular view of the diagnostic journey and patterns of disease management. This work has already helped us identify actionable signals that can support earlier recognition and referral of patients.
We are actively incorporating these insights into our field and medical strategies. So far, we have identified well over 100 patients in the U.S., where several new patients this year. We are able to provide tiratricol to many of these patients through our expanded access program, which now is active at 40 sites nationwide. We are progressing well on our market access and pricing preparations. We know that speed and breadth of payer coverage will be critical at launch. Our team is working with payers and stakeholders to ensure a robust evidence package and a clear path to access, aiming for patients to benefit from treatment without unnecessary barriers. We continue to build the right infrastructure to support the community once we launch.
A central element of this is expanding our field medical presence, which will enable high-quality scientific exchange with healthcare providers and strengthen relationships across the rare disease ecosystem. In short, we are making disciplined, coordinated progress across patient identification, access, and field readiness. With these pillars in place, we are confident in our ability to deliver a strong launch and, most importantly, to finally bring a much-needed therapy to patients living with MCT8 deficiency also in the U.S. With that, I would like to hand over to Yilmaz for the financial update.
Thank you, Henrik. Now for a brief financial update. Revenues for the first half year were SEK 27.1 million versus SEK 25.9 million in the same period last year. Revenues for the second quarter of the year were SEK 14.5 million versus SEK 13.9 million in the same period last year, and it's attributable to the managed access program and commercial sales of Emcitate. The gross profit margin is visibly lower than historical figures, both for the half year and the second quarter, but comes back to the regulatory success we had with Emcitate in Europe. As a consequence, the COGS line item in the half- year numbers not only includes the recurring royalty payments to the founders of 3% and Erasmus Medical Center of 10%, but also non-recurring milestone payments of approximately SEK 3.5 million to Erasmus Medical Center, as well as SEK 13.5 million of intangible R&D depreciation.
A reminder for the first-time listeners is that the depreciation is a non-cash item and was initiated as a consequence of the approval in Europe. In an apples-to-apples year-over-year comparison, adjusting for the one-time payment and the depreciation, adjusted gross profit would be SEK 21.7 million for the half year, an improvement not only nominally but also on the gross margin level versus the corresponding period last year. While operating results were nearly the same for the half year at - SEK 143.1 million versus - SEK 143.8 million in the corresponding period, results after tax for the half year of the year were - SEK 140.5 million versus - SEK 146.9 million. The improvement versus prior corresponding period is mainly driven by the finance line items and specifically the revaluation of the convertible right, which fluctuates with the share price.
Half year 2025 figures were SEK 8.8 million versus SEK 4.7 million. Remember that this is also a non-cash item and will continue to fluctuate. Although cash flow before working capital saw a year-over-year improvement in the first half for the quarter due to our cost-conscious execution of operations, changes in working capital had a negative impact. Also, as you may have noticed, we are repaying our debt and report SEK 14.2 million in repayment for the half year, an item which did not exist in the corresponding period last year. All in all, the cash flow for the half year were -S EK 143.4 million versus - SEK 113 million, and the cash position end June 2025 was a healthy SEK 202.6 million versus SEK 192.6 million last year. With that, I hand it back to Nick.
Thank you, Yilmaz. Let me take the opportunity to summarize this very shortly before we move into Q&A. We are very pleased with the milestones delivered so far, noting the Emcitate launch in Germany, which truly marks a major milestone for us as we continue to expand our reach and provide the first and only approved treatment options for patients with MCT8 deficiency in Europe. I'm also very proud of the breakthrough therapy designation granted by the U.S. FDA in July and want to thank the team in making this happen. We have an exciting period ahead of us with many upcoming value-enhancing milestones, such as the filing of a New Drug Application in the United States. With that, I would like to hand over the call to the operator and start the Q&A session of this call. Operator, please go ahead.
If you wish to ask a question, please dial pound key five on your telephone keypad to enter the queue. If you wish to withdraw your question, please dial pound key six on your telephone keypad. The next question comes from Suzanna Queckbörner from Handelsbanken. Please go ahead.
Hello, Suzanna Queckbörner here, Handelsbanken. I'd like to ask a question about the managed access program. In the past, you've reported a number of patients that are in the program. Perhaps we could get an update and maybe a breakdown of what share is based in the U.S. and what share is in the Europe top five, roughly.
Yeah. No, thank you very much, Suzanna. You're absolutely correct that up until the approval and launch of Emcitate in Europe, we have reported on a quarterly basis the number of patients in our managed access program, and we report over 230 patients earlier in the year. Going forward, this will be difficult in the sense that we are in Europe transitioning, as you heard from Henrik, patients from the managed access program to patients receiving commercial packs that generate revenue. We will be somewhat of a bumpy road going forward with the number of patients in the managed access program versus the number of patients actually entering Emcitate treatment or transitioning into Emcitate treatment from a commercial product perspective.
What one will see in Germany, for example, if I take Germany as an example, is that we had a number of patients moving into 2025 on the managed access program, which of course now is close to zero. That will be giving a somewhat of a wrong illustration of the number of patients identified. What we want to do instead is focusing more on conversion of those patients, as you heard from Henrik today, but also give some more granularity on one of the most important markets for us in the U.S. As you heard from Henrik, we have identified well more than 100 patients in the U.S. so far. I think that's the background, Susanna, of why not continuing to provide an update on that. I don't know, Henrik, if you have something to add.
I think you summarized it well, Nicklas.
Perhaps just to add, I mean, we will not stop providing drugs to any patients, and patients will be converted from the managed access to traditional prescriptions once national reimbursement is secured in that specific country. In Germany, as Nicklas said, this started immediately at the time of launching. Thank you.
Very good. Maybe then just to follow up, do you think you can provide an estimate of how many patients we should think about when thinking about Germany and the prioritized countries in Europe?
We haven't disclosed and didn't before either on a country-by-country basis. I will try to somewhat narrow it down for you. We reported 230 patients before the approval in Europe in the global managed access program or under the umbrella of the managed access program, and the majority has been in Europe out of those 230. We won't give any further disclosure on the number of patients per country at this point in time.
Okay, thank you very much.
Thank you. Thank you, Susanna.
The next question comes from Chiara Montironi from Van Lanschot Kempen. Please go ahead.
Hello, team. Congratulations on the update. Thanks for taking my question. I'm just trying here to put events on a timeline. Could you help us understand the timing of the type B meeting with the FDA in respect to top-line data release for tiratricol? Do you expect to meet the FDA before having completed the recruitment or before or after having the full dataset?
Yeah. We can start with timelines for the meeting. This is a type B type of meeting, which really stipulates that the agency, or FDA in this case, will aim to schedule the meeting within 60 days of receiving the meeting request. If granted, of course, this will mean October for us. When it comes to future predictions and giving guidance on when we have top-line results, as well as an ongoing regulatory interaction, unfortunately, Chiara, I know you're very much looking forward to it, but that's nothing we can disclose. All discussed with FDA, as mentioned, is, of course, in light of the breakthrough therapy designation together with where we are in the ReTRIACt study, discussing the position and role.
This is taken together with the robust dataset generated from Triac trial I, the Erasmus Medical Center cohort study, 67 patients up to six years, as well as the Triac trial II. I can't give more guidance than that at this point in time.
Yes, got it. Thank you very much.
Thank you, Chiara.
The next question comes from Arvid Necander from DNB Carnegie. Please go ahead.
Good morning, and thanks for taking my question. First off, it's early days, of course, but could you speak to how you are tracking compared to what you had expected as the launch was initiated? Can you give us any insight into launch metrics and how these are tracking, prescribing physicians, centers, et cetera? That would be very helpful. Thanks.
Thank you, Arvid. Henrik here. First of all, we are on track with the KPIs that we have. Our KPIs are, of course, related to the number of patients identified and the number of patients converted initially from the managed access program to commercial drug and then new patients initiated on Emcitate. We're happy with the dialogues that we have at the different centers. As we have talked about before, we are focusing on the pediatric disease centers in Germany, the SB Cells, and also the specific rare disease centers. There we have had good conversations where we have had meetings so far, and some of them are leading to that we become aware of new patients, and then we can have good conversations also about how to initiate treatment of those.
I'm pleased overall with what we are doing in Germany, and I look forward to continuing the work developing that market.
I think maybe to build on that, Arvid, as Henrik mentioned before during the presentation, almost all patients have been converted during the quarter, and we expect all patients to be converted now during quarter three. That is a key KPI for us, as I'm sure you appreciate, together with identifying new patients that will be initiated or have been initiated on treatment.
Thanks, guys. That's very helpful. Just a second one, if I may. Sales in Q2 seem to have been roughly in line with consensus. How comfortable are you guys with the SEK 88 million estimates for the full year?
Sorry, Arvid, you were breaking up at the end. Comfortable with, and then I lost you.
Okay. Sorry. I'll repeat. At present, I think consensus is at SEK 88 million for the full year 2025. How comfortable are you guys with that estimate?
As you know, we haven't, and I'll ask Yilmaz to comment in a bit, but we haven't provided any revenue guidance this year. The reason is very simple, right? We're navigating through the pricing and reimbursement processes in France and in Germany, and now we're on the verge of submitting also the pricing and reimbursement discussions in Spain and Italy. It's complicated to give revenue guidance at this point in time, and hence why I can't comment on are we comfortable or not comfortable on the consensus that you were alluding to. Yilmaz, I don't know if you have anything to add.
No, it's just so early days. We just launched in May. I mean, sure, we have good conversions in Germany, but still early days with other reimbursement processes and countries. It's just hard to give guidance, and we're only launching one country, as you know, from the commercial side at the moment.
Thank you, Arvid. I think we have time for one last question. Operator, please.
The next question comes from Oscar Haffen Lamm from Stifel. Please go ahead.
Congrats on the progress this quarter. I'd just like to understand how the conversion from the managed access program to commercial package happens. I mean, in terms of what type of delays can happen, does the patient journey change, or any other things that might happen?
No, thank you. Maybe I can start, and Henrik, you can build. Obviously, we had the ability to provide commercial packs from the 1st of May, right? What usually is rate limiting is a really simple aspect, such as how much in stock within a specific patient do they have? Do a patient have stock of Emcitate from the managed access program at the 1st of May for the coming two months, for the coming three months? We don't, as I'm sure you appreciate, have any visibility on that. What we know is that, of course, when they are running out of stock, the only opportunity they have is, of course, to switch over to commercial product. It's very difficult for us. We don't have any insights of stock levels for individual patients, as I'm sure you can appreciate.
That is really what is driving the transition period rather than anything else. Henrik, I don't know if you want to add on that.
Yeah, then more. On the technical things, to be able to do this shift, there needs to be a drug price listed, which we did in time. The difference is that the physicians prescribe with a regular prescription, and then the caregiver to these patients can pick up the prescription. This has happened really seamlessly, which we are happy with.
Okay, thank you. It makes sense. Maybe a second question. Maybe it's too early to tell, but have you noticed any change in patient diagnosis dynamics in Europe since Emcitate was approved and launched? I mean, at least in Germany.
Related to patient diagnosis, it's a short time to say. I mean, it was two months in Q2 since we launched, and we have, over the time, been active with field medical in Germany. We have seen a continuous inflow of new patients and no dramatic shift in May. It's early to say.
I think this is an interesting question, Oscar. I think it comes back to what Henrik said, that, of course, we've been active, especially in Europe, over the last two years, participating in conferences, drive disease awareness, et cetera, et cetera. Since it's such a short time since the initial launch in Germany, i.e. 1st of May, it's difficult to see any major step change. However, we have a couple of good examples, and Henrik, I don't know if you want to share that.
Yeah, I think I personally attended the European Society of Pediatric Endocrinology, where we were able to, for the first time, exhibit with Emcitate. That was actually a big shift because it was much more interest and traffic to the booth, more dialogues with healthcare professionals. That is something that I really believe that we now have the opportunity to accelerate the awareness of both Emcitate deficiency and that Emcitate is available for these patients.
Okay, thank you very much.
Thank you so much. I apologize that we have been running over for four minutes. Due to that, we don't have time for any further questions. Without further ado, I would really like to thank the participants on the call and wish everybody a good rest of the day. Thank you.