Welcome to Egetis Therapeutics Q4 report 2024. 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 Nicklas Westerholm. Please go ahead.
Thank you very much, Operator, and a warm welcome to Egetis Q4 and full-year results planned for 30 minutes. For those I have not had the privilege to meet, my name is Nicklas Westerholm, and I am the CEO of the company. With me today, I also have Yilmaz Mahshid, Chief Financial Officer; Henrik Krook, our Vice President, Commercial Operations; and Karl Hård, Head of Investor Relations. Let's turn our attention to the topics to be covered today. First, let me begin with saying that I am very proud of the full approval of Emcitate in Europe received on the 13th of February. This represents the single most important milestone in Egetis' history and a major step forward for the company building a sustainable rare disease business. We are also very much delighted to bring this much-needed new treatment to patients with MCT8 deficiency in Europe.
It also, of course, marks a start of a transition for the company from late-stage development company to commercial-stage company. What we will cover during the presentation today is a reflection on the key milestones delivered during 2024 and 2025 year-to-date, including the European Commission's approval of Emcitate. We'll update on the ReTRIACt status, the pivotal study required for the NDA submission in the U.S. Henrik Krook will update on the commercialization progress and timelines in Europe, and we will then hand over for an update from Yilmaz Mahshid on the financial status of the company and wrap it up with key value-enhancing milestones expected during 2025 and 2026. First, though, let me start with a short reminder of MCT8 deficiency, a detrimental condition with significant unmet medical need.
MCT8 deficiency is a thyroid hormone signaling disorder that causes simultaneously too high and too low thyroid hormone levels in different tissues. Really, a disease with two distinct clinical phenotypes. The first one is thyrotoxicosis, or too much thyroid hormone in the peripheral tissues, that actually causes lifelong morbidity and other very detrimental cardiovascular symptoms, such as, and as you can see on the right-hand side of the slide here, severe underweight, cardiac arrhythmias, median life expectancy of 35 years, and 30% of the patients are dying in childhood, with the main cause of mortality being sudden cardiac death. The second clinical phenotype is hypothyroidism, i.e., not enough thyroid hormone in other parts of the body, like the central nervous system and brain, which causes permanent and severe developmental delays. Here, most patients are never able to sit or walk and have limited ability to communicate.
Therefore, we are, of course, very proud of Emcitate being approved as the first and today only treatment for patients with MCT8 deficiency in Europe. Let's turn our attention then to reflect on the progress made in 2024 and 2025 year-to-date, focusing on the several important milestones delivered. The first one is the European Thyroid Association issuing guidelines. Why is this important? First of all, these are guidelines for diagnosis and treatment for MCT8 deficiency, stipulating the use of tiratricol as a long-term therapy for all patients. Worthwhile recognizing is that these guidelines were developed by independent key opinion leaders, both from Europe and in the U.S., and were commissioned by the European Thyroid Association in July. This is somewhat unusual. The reason for it being unusual is that MCT8 deficiency is an ultra-orphan condition, and the guidelines were commissioned before an approved treatment.
This is something, of course, we're very pleased with. This will be very supportive for us now moving into commercialization in Europe, and Henrik will reflect on this more in the coming sessions. The next important milestone was new data that was presented through an abstract and subsequently physically presented in September at the European Thyroid Association, illustrating that treatment with Emcitate in patients with MCT8 deficiency is associated with a three times lower risk of mortality. This is important, coming back to the disease characteristics, where median life expectancy is shorter, 35 years, and 30% of patients dying in early childhood. These are very valuable data for us, both for the forthcoming New Drug Application in the U.S., but also for upcoming payer interactions.
Further details of this data, the survival data, is under review and will be published in a peer-reviewed publication in the not too distant future. Also, at the European Thyroid Association, new data was presented from a post-hoc analysis that reported effects of Emcitate on patient-centered outcome measures in patients with MCT8 deficiency. These are data from Triac Trial I. In Triac Trial I, interviews were carried out with parents and caregivers about the most prominent changes, both positive and negative, during Emcitate treatment. Improved interactions, alertness, motor skills, and sleep were mentioned, according to the numbers you see here on the left-hand side of the slide. In one patient, increased constipation and increased unsettledness were reported. Most important here is all parents, 40 out of 40, preferred to continue with Emcitate treatment.
Let's turn our attention to another milestone, and this is the submission of the patent application to the U.S. Patent and Trademark Office. As many of you are aware, today, Egetis holds Orphan Drug Designation, or ODD, for Emcitate for MCT8 deficiency in the U.S. and Europe. This currently provides marketing exclusivity of seven and ten years, respectively, from the dates of regulatory approval. Egetis has, over the last couple of years, invested substantially in updating and refining our manufacturing process, ensuring it's robust and up to the current quality standards. This has also led to generation of new data, which led to this patent application. If granted, this would be a significant patent for Egetis. Generally, the exclusivity term of a new patent is 20 years from the date on which the application for the patent was filed in the U.S.
We were also delighted to share that we carried out a directed share issue amounting to approximately $30 million at the back end of September last year. This was led by Frazier Life Sciences with a $10 million investment, and Frazier's ownership is now 17% of the company. Circling back to the highlight over the period, which is, of course, the European Commission approval of Emcitate as the first and only treatment for patients with MCT8 deficiency. It is worthwhile stressing here that the marketing authorization is a full marketing authorization. It is not conditional approval, which means no further clinical trials will be necessary in Europe. As mentioned, by far, this is the single most important milestone in Egetis' history and a major step forward in building a sustainable rare disease company.
Egetis expects to launch Emcitate in the first country, Germany, in Europe during the second quarter 2025, and Henrik will elaborate further on this shortly. Let's now turn our attention to the ReTRIACt study. As agreed with the U.S. FDA, we're conducting a pivotal randomized placebo-controlled study, the ReTRIACt study, in at least 16 evaluable patients with MCT8 deficiency to support the submission of a new drug application in the U.S. To increase the recruitment capacity in the study, four additional clinical study sites were opened in the U.S. during 2024, and the last one in December, one each in Texas, Georgia, North Carolina, and Florida. At the moment, we have seven open sites in the study, two in Europe, and five in the U.S.
The current status of the study is that 19 patients have been included, whereof 11 patients have completed the randomized treatment phase and are evaluable, and three patients are also in the so-called running period. Several additional patients are currently being evaluated for screening, with one patient being scheduled for screening tomorrow. Please note that for any clinical studies, there will be a few dropouts, and hence not all patients will be randomized. As previously communicated, we have full focus on the recruitment on the ReTRIACt study. We will update the market as soon as the recruitment of the study is closed. At that time, we will also provide information when we expect top-line results and when we plan to submit the New Drug Application during 2025. With that, I will now hand over to Henrik Krook to elaborate further on the commercialization in E.U.
Henrik, over to you.
Thanks, Nicklas, and good morning, everyone. As you can imagine, these are exciting and busy times for us, thanks to the recent EMA approval. Given that MCT8 deficiency is such a devastating disorder with no other approved treatments available, we are very excited to soon be able to launch Emcitate for the benefit of the affected patients. During the presentation, I will give you a status update on where we are with our European launch preparations for Emcitate. However, first, I will just remind everyone of the big picture. We are preparing for commercialization of Emcitate through our own team at Egetis in Europe and the United States. In the rest of the world, we plan to work through partner companies to secure broad access to Emcitate. For Japan, we have a license deal for development and commercialization by Fujimoto Pharmaceutical Corporation.
We are also in dialogues with other potential partners for other geographies, such as the cluster Middle East, North Africa, and Turkey, where we intend to sign partner agreements later this year. Next, I would like to take a moment to reflect on these European Thyroid Association guidelines that were published in July 2024. The guidelines describe how to manage MCT8 deficiency, how to diagnose, et cetera. They will contribute to disease awareness and support the diagnosis of more patients. As mentioned earlier by Nicklas, we are, of course, very pleased to see that the ETA guidelines recommend Emcitate as long-term therapy for all patients with MCT8 deficiency. It is extraordinary that a treatment is included in these kinds of guidelines even before marketing authorization. It will be great to leverage these guidelines in our upcoming dialogues with payers in the pricing and reimbursement processes.
The next step now, after the regulatory approval, is to make sure to get the pricing and reimbursement right so that more patients can get access to treatment. National pricing and reimbursement is negotiated country by country, and since the 27 E.U. countries have different processes and timelines, this is relatively complex and takes time. There are, however, some common themes that it is critical to get right in all countries. To get reimbursement, it is important that payers understand the disease and support the benefits of treatment with Emcitate. In the pricing and reimbursement processes, we will deliver a solid value proposition to payers where we describe Emcitate efficacy and its rarity. In addition, we will demonstrate the burden of MCT8 deficiency, which is further supported by a recent Egetis-sponsored caregiver study.
We will also demonstrate the significant unmet medical need, and please again remember that Emcitate is the first regulatory-approved drug for MCT8 deficiency. Lastly, the benefits of Emcitate treatment. These benefits have actually, to some extent, already been validated by physicians through the European Thyroid Association guidelines and by regulators through the recent EMA approval. We are executing our pricing and reimbursement strategy in two ways, starting with the four larger E.U. countries. In Germany, the pricing and reimbursement process lasts for one year, but it is possible to start with reimbursed sales from the initiation of the process. For France, Spain, and Italy, the pricing and reimbursement negotiations typically take one to two years, but also in these countries, there are possibilities to get funded sales in parallel with the national pricing and reimbursement processes.
To optimize the pricing and reimbursement processes, we are working in collaboration with local pricing and access advisors, and these are people who have many years' experience of getting ultra-orphan drugs successfully reimbursed in the respective country. We are on good track to optimize pricing and reimbursement. Germany is the country where we expect the first reimbursement, so I will briefly walk you through this process. It is called the AMNOG process, and it's very well defined.
In addition to the pharma company submitting the dossier for pricing and reimbursement, there are three key parties involved: G-BA, which is the authority that sets the framework and issues the benefit statement; IQWiG, the Institute for Quality and Efficiency in Healthcare, which assesses the data, the patient population size, and the annual therapy cost, and then reports back to the G-BA; GKV, the National Association of Statutory Health Insurance Funds, with which the pharma company negotiates the final price. This process lasts exactly 12 months, during which the drug can be sold from day one at the price set by the pharmaceutical company, with GKV reimbursing it. However, after the 12-month period, if the final negotiated price is lower than the initial price, the pharma company is required to pay back the excess amount received during the latter six months of the 12-month period.
We started the work with preparing the Emcitate AMNOG dossier one year ago, and it is in good shape. What is happening now is that we are making fine-tunings based on the final approved Emcitate label, and when finalized, we will submit our dossier to the G-BA. Our preparations are progressing well, meaning that we will launch in Germany in Q2 this year. You can imagine that these are exciting times for us. Our team members have solid experience of successfully launching drugs in the ultra-rare setting, but it is, of course, an extra strong feeling to be a part of a company's first drug launch. I must say that it is a true privilege to prepare for the first Egetis launch together with our skilled German launch team and to soon be able to provide broader access to Emcitate to patients affected by MCT8 deficiency.
With that, I would like to hand over to you, Yilmaz, for the financial update.
Thank you, Henrik. I have one slide on the financials, and please call me or IR if you have any further questions. On this slide, we have revenues for the full year 2024, where SEK 43.1 million versus SEK 57.6 million in the same period last year. Revenues for the fourth quarter of the year were SEK 10.8 million versus SEK 32.6 million in the same period last year. The lower numbers for the full year 2024 versus 2023 are due to one-time license income in 2023 of SEK 14.5 million received for the Japanese rights. The same comparison relates to the fourth quarter, but also due to lumpy order patterns in Managed Access Program income.
Results after tax for the fourth quarter of the year were negative SEK 110.5 million versus negative SEK 86.3 million. The lower amount versus prior period is driven by increased investments in the commercial buildup, but also impacted by a couple of non-cash items, such as the IFRS 2 bookings for the ESOP employee stock option programs of negative SEK 8 million in the quarter versus negative SEK 3.8 million in the prior period, which is part of the administrative line item in the P&L. Also, revaluation of the convertible right related to the BlackRock debt of negative SEK 7.6 million versus negative SEK 2.7 million in the prior period. This one is part of the finance line items in the end. Operating cash flow for the fourth quarter of the year were negative SEK 53.6 million versus negative SEK 41.6 million in the same period last year.
The cash position at the end of December 2024 was SEK 351 million versus SEK 303 million last year, starting the new year on a better footing in 2025. It was gratifying to see that we had good support from existing and new shareholders, which did result in a net cash injection to the company of approximately SEK 281 million that occurred in the fourth quarter. During the fourth quarter, we did also issue 29 million C shares as hedge for the outstanding ESOP programs. These are held in company treasury and will only become outstanding common shares if any of the ESOP programs goes to exercise. With that, I'll hand the microphone back to Nicklas.
Thank you, Yilmaz, and thank you, Henrik. The last slide will focus on the future, looking ahead.
We have a very excited period ahead of us with many upcoming value-enhancing key milestones, such as the European launch starting with Germany in the second quarter. We very much also look forward to the top-line results of the ReTRIACt study, followed by the filing of a new application in the U.S. during this year. This means that we expect the U.S. approval and launch of Emcitate in 2026 in the U.S. With that, I would like to hand over the call to the operator and start the Q&A session for this call. Operator, please go ahead.
If you wish to ask a 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 Arvid Necander from Carnegie. Please go ahead.
Good morning, and thanks for taking my questions. First off, I was wondering if you could give us some perspective on your targeted launch and reimbursement timelines. I guess first, in Germany, I understand it's hard to give an exact estimate, but do you anticipate a, let's say, standard three to four-month HTA process with the G-BA, or is there something leading you to believe that it could be longer, for example, due to ongoing cost-benefit analysis or other factors? Secondly, when do you anticipate launching the remaining EU4 markets, and in which markets do you believe there are pathways that would allow you to launch before reimbursement is fully concluded? I'll start there. Thanks.
Thank you. Henrik, if you don't mind.
Yeah, thank you for the question, Arvid. First, starting then with Germany, there we intend to work according to the regular timelines there.
We assume that the final reimbursed price will be negotiated 12 months after our submission. To the second question related to the other EU4 countries, the timelines for the pricing and reimbursement processes are a little bit less structured than for Germany. We anticipate that these will take one to two years. For these countries, there are possibilities, as I mentioned, to get funded sales in parallel. That is possible thanks to routes through negotiating with local hospitals and regions. That is something that we are working on to make that happen.
Okay, great. Thank you. Lastly, just if I may, do you have visibility on how many patients you currently have on treatment in Germany and the EU4?
Yeah, thanks, Arvid. Good morning to you as well. We do not disclose how many patients we have per country.
I would like to circle back to the 230 patients in our Managed Access Program. The majority of the patients there are in Europe, and then population-based, one would assume that a major part would be in EU4. We do not disclose patient numbers identified today per country.
Okay. Just to finish, if I may follow up on that, have you seen any significant growth in terms of the patients on treatment in the EU4 markets over the past year, or has that mainly been outside these markets?
I think it is a mix. Not speculating on a specific market, but when we see patient identification, because I think it is key to understand patient identification versus patients under the umbrella of Managed Access Program, there we see across the world, regions and countries an uptick in the number of diagnosed patients.
This is obviously driven by a number of factors, our efforts in driving disease awareness in Europe, especially through the European Thyroid Association guidelines, etc. I think when it comes to number of identified patients being diagnosed, it's across the board.
Okay, perfect. Thanks, team. I'll jump back in the queue.
Thank you, Arvid.
The next question comes from Suzanna Queckbörner from SHB. Please go ahead.
Hello, Suzanna Queckbörner here from Handelsbanken. Thank you for taking my questions. Just to follow up on Arvid's question with regard to the German launch, do you have patients lined up for Q2? Also, how are you going to handle pricing and reimbursement negotiations in the EU4? Will you have people on the ground? I remember that you said you have 40 employees as of now. How are you thinking about it?
One final question regarding the ReTRIACt trial. Can you give me a view on how patient attrition is in these trials in terms of the run-up and screening, how long it takes, and how that impacts randomization?
Thank you. Good morning, Suzanna, and great to have your questions. I think the simple answer to the first question on patients lined up in Germany is yes, we of course do. It comes back to that we have a number of patients under the umbrella of the managed access patients being located in Germany. On top of that, we also have identified a few other patients in Germany currently not on treatment. We have a strong belief that already from launch and onwards, these patients will be converted into the so-called commercial space from the managed access program and also newly diagnosed patients.
When it comes to the organization, I think that's a really good question and something that is very, very important to us when it comes to sufficient capability and capacity. We have a staff built up, as you probably know, our commercial and medical affairs infrastructure over the last years, initially focusing on Europe. The focus for the coming 12 months, as you heard from Henrik, is around pricing and reimbursement in EU4. There we have people on the ground covering both access, so pricing and reimbursement, and this is governed by our global head of market access being based here in Europe, but also supported by local consultants, experts in navigating the local reimbursement processes. When it comes to medical affairs, we also have people on the ground in the key countries EU4.
Last but not least, when it comes to the ReTRIACt study, your question was around the attrition, and they're coming back to 11 patients today just so we get the facts right. Our ambition is to have at least 16 available patients. Today, we have 11 patients that have completed the randomized treatment phase and are available. That's up by three from December, where we had eight. We have another three patients in the running period, and we have a number of patients being evaluated and planned for screening, one actually tomorrow. When it comes to the attrition, so to say, and dropouts, we recognize and we mentioned that at previous calls that we have had a few dropouts, not driven by study drug, but family circumstances and other aspects. Those are the, I would say, attrition one would see, which is not unusual for clinical trials.
When it comes to screening and screening failure, their attrition is somewhat lower, but there it's the majority of the challenges we have had has been driven more by family's ability to travel, etc., etc. I hope that answered your question, Suzanna, and happy to take a follow-up question offline. Thank you.
Thank you very much.
The next question comes from Oscar Haffen Lamm from Bryan Garnier. Please go ahead.
Hi, team. Also here from Bryan Garnier. Thank you for taking my questions. The first question may be on the launch in the EU. I was just wondering how quickly do you think the patients in the early access program will translate into commercial revenues upon launch and how we should think about pricing revenues in the four biggest European countries except Germany before final price and reimbursement negotiations have concluded.
Then as a second question regarding ReTRIACt, so far it looks like you have a good translation of, I mean, translation of patients between run-in to randomization. I was wondering how many patients have dropped out, if any, so far in the run-in period and in the randomization phase. Thank you.
Henrik, do you mind starting with the first question?
Yes, sure. Related to the transition from the Managed Access Program to commercial sales. Here, just to state first that we will not stop providing drug to any patients, and patients will be converted from managed access to traditional prescriptions once national reimbursement is secured. In Germany, this will happen immediately at the time of our launch in Q2 this year. For France, Spain, and Italy, the pricing and reimbursement negotiations typically take one to two years.
As mentioned, also in these countries, there are possibilities to get funded sales in parallel with the national pricing and reimbursement processes. I hand over to you, Nicklas, for the ReTRIACt-related question.
Yeah, thank you, Oscar. Your question was around dropouts, to my understanding, and what we have seen. I think we mentioned this at the quarter three call we had back in end of November, and there we have seen a few dropouts, very few, less than a handful that has been dropping out through the different phases of the study. This is not unusual, and I reiterate what I said to Suzanna's question, that this has been driven not by study drug. It has been driven by actually family circumstances not being able to continue on treatment any longer, or sorry, not continue to participate in the clinical study.
It is quite a few to answer your question.
Okay, great. Thank you.
Thank you, Oscar.
The next question comes from Fredrik Thor from Redeye. Please go ahead.
Hello, and thank you. My first question, and maybe you answered this, but when will we know about the initial price in Germany? I mean, the launch price, I'd say, before the final reimbursement.
Yeah, please, Henrik.
Yeah, the initial German list price will actually be publicly available at the time of launch, so from Q2 this year.
As you heard from Henrik during the presentation, that is the initial list price we have from the launch. Of course, during the very clearly defined stipulated AMNOG process, we will end up with a final negotiated price 12 months after the launch. Thank you, Frederick.
Got it.
Please, sorry.
I also had a question.
I also had a question about the rarity that this is a voucher program. If you could give us your view of the status of the program. I know that there have been some budget discussions about it and so on. What's your view?
Yeah, hello, Fredrik. This is Karl Hård speaking. I mean, we are following this, of course, with interest. Interestingly, the FDA has continued to issue rare pediatric disease designations also during 2025. That is a very positive sign. There is big activity going on in the Congress at the moment, both in the House and the Senate. We will see how that develops. It has been twice before that there has been a sunset clause coming, but then it has been renewed. We hope that the same happens here as well. Thank you.
Thank you. That's all from me.
The next question comes from Alexander Krämer from ABGSC. Please go ahead.
Yes, good morning. I have two questions. One financial question. I mean, today, of course, your OpEx went a little bit up. My question here is, as ReTRIACt progresses and, of course, then finalizes this year, are the costs expected to move down while, of course, SG&A costs will go up? Do you expect the total OpEx levels this year in 2025 to be net neutral, or do you expect a single or a single-digit growth in the total OpEx levels? I have a second question.
Thank you, Alexander. That's a really good question. We have shared this before, that if you look into 2025, first, let us be clear, we don't give any guidance on cash burn rate, but I will try to triangulate it for you together with Yilmaz here.
We see absolutely, as you said, that we see the R&D costs declining over the year, obviously, especially compared to last year where we concluded the first part of Triac Trial II. Now we're going to conclude within short the ReTRIACt studies. Of course, we believe that the R&D costs will decline over the year. That is offset, of course, by increased investments in the commercial and medical affairs space, both in Europe and in the U.S., of course, where we had our U.S. launch as well. Not giving any guidance, it's reasonable to assume that it won't be a substantial uptick in cost, but rather a moderate to flat uptick. Yilmaz, I don't know if you want to add anything to that.
No, not much really.
I mean, most of our investments are dependent on achieving certain operational milestones, such as the ReTRIACt study, as you all know. I mean, we probably accelerate beyond that, but the reasonable assumption is that our cash burn will see a minor uptick here at the beginning of the year, but nothing major.
All right. Thanks. The second question relates to your disease awareness activities in Germany. Are you going to medical conferences there? If yes, which ones? Also, as you have your people there outside in Germany, how good is your knowledge level of how the diagnosis and the screening of newborns work in Germany and how easy it is to identify newborn patients?
Yeah, so Henrik here, I can answer to those questions.
First, related to disease awareness and Congress attendance, we have planned to attend roughly 10 congresses in the German region this year. These are something that we have learned from the past that it's really good to attend because that is when we are able to have high-quality interactions with new physicians as well. We support the identification of new patients. Related to your second question about screening in Germany, there is no national program specifically for MCT8 deficiency, but Germany is one of the most advanced countries in Europe when it comes to look and do genetic testing once there comes a patient with an unclear diagnosis. Therefore, it's a well-developed country for diagnosing patients.
Maybe to add on that, on top of what Henrik said and reiterating, 10 conferences are planned for this year for Egetis attendance.
It is quite a few. As I'm sure you appreciate, Alexander, this is something that is important to us. It is a high return on investments with quality dialogues and patients being identified. We can reflect on that from the past experience. On top of that, we are also arranging a number of advisory boards with key opinion leaders to further have dialogues on the topic of diagnosis and treatment of MCT8 deficiency.
Thank you, Alexander. We are running a bit over time, but we have time for one last question from Patrik Ling, to my understanding, DNB.
The next question comes from Patrik Ling from DNB Markets. Please go ahead.
Yes, hi. Just a short follow-up, Nicklas, to your prepared remarks.
I mean, when you talked about the ReTRIACt study, did you say that you would communicate a timeline in 2025, or did you actually say that you will be filing in the U.S. in 2025?
Good question, Patrick, and good morning to you. I want to be very, very clear here. Good morning. We will file. That's what we plan in 2025.
Good. Good. That's what I thought I heard. That was actually all for me. All other questions have been answered. Thank you.
Thank you so much. That takes us to the end of the call. If you have any further questions that I was not able to answer, you can reach out to us through our normal contact channels. We'll take it from there. Wish you a great day.
Also a short reminder that Egetis is holding a key opinion leader event on Tuesday, the 4th of March, focusing on MCT8 deficiency, details on the disease characteristics, as well as experience in treating these patients with MCT8. Thank you very much, and I wish you all a good day.