Welcome to Egetis Therapeutics Q1 Report 2025. 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 #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, operator. Good morning and a warm welcome to Egetis Q1 Results Call planned for the coming 30 minutes. For those I haven't had the privilege to meet before, I'm Nicklas Westerholm and the CEO of the company. With me today, I also have Yilmaz Mahshid, Chief Financial Officer; Henrik Krook, our Head of Commercial Operations; and Karl Hård, Head of Investor Relations. Let's turn our attention to the topics covered today. First, let me begin saying that I'm incredibly proud of the full approval of Emcitate in the European Union on the 13th of February, which represents the single most important milestone in Egetis' history and also marks a major step forward in building a sustainable rare disease company.
We are very much delighted, of course, to bring this much-needed new treatment to patients in Europe through the upcoming launch in the first country, Germany, that takes place tomorrow, the 1st of May. It also, of course, makes a start of a transition from a late-stage development biotech company to a commercial-stage company. What we will cover during the call today is the progress in building a rare disease company, key milestones delivered during 2025 year to date, recap on the US regulatory pathway, and then the submission planned for 2025, and the ReTRIACt status, the pivotal study required for completing the NDA dossier. I will then hand over to Henrik that will elaborate further on the commercialization in Europe, but also the launch preparations in the US.
Subsequently, Henrik will hand over to Yilmaz, who will give an update on the financials, and we'll be finishing off with upcoming key value-enhancing milestones. With the European approval of Emcitate in February and the subsequent launch in Germany tomorrow, we're making strides to become a sustainable rare disease company with focus on late-stage development for commercialization. Furthermore, the pivotal study in the U.S., the ReTRIACt study, is well underway to complete the U.S. NDA dossier with the planned submission during this year. Preparations for the EU launch of Emcitate, focusing initially on EU4 countries, are on track, and the U.S. launch preparations, including the stepwise build-up of a commercial and medical affairs organization, are also on track, ensuring that we'll maximize the Emcitate opportunity.
For markets outside Europe and the US, we have a license agreement with Fujimoto Pharmaceutical Corporation for the development and commercialization of Emcitate in Japan. Discussions are currently ongoing with the Japanese Regulatory Authority, PMDA, regarding the development pathway required to obtain approval for Emcitate in Japan. In the Middle East, North Africa, and Turkey region, discussions are underway with potential distribution partners to enable access to Emcitate. Henrik here will provide further information and elaborate on these topics later in the presentation. In addition, and as previously communicated in December last year, we have decided to explore RTH Beta as the next indication for tiratricol, Emcitate, where we see a significant unmet medical need coupled with a significant market opportunity. MCT8 deficiency and RTH Beta are two distinct indications with no overlap in patient population.
As mentioned before, we are considering to support a multi-centered investigator-initiated phase two study in patients with RTH Beta in collaboration with academia. Last but not least, we have, during the last few years, been able to attract and also retain a very seasoned and experienced workforce covering late-stage orphan clinical development, registration, and commercialization, which makes us well positioned for the future. Reflecting on the progress made over the period, it's worthwhile mentioning a couple of highlights here. You can see it depicted on the slide, but one of them worthwhile mentioning is, of course, the ETA guidelines issued in July last year, recommending all patients with MCT8 deficiency to be treated with Emcitate. Also worthwhile calling out is the data from the retrospective reward cohort study where it was investigated the effects of tiratricol on mortality.
Tiratricol-treated patients had an approximately three times lower risk of all-cause mortality, which is, of course, very valuable data going forward, both for payer interactions as well as the upcoming New Drug Application in the U.S. Foremost, as I said before, I'm very proud of the European approval of Emcitate. Grateful for all the hard work of the Egetis employees, our partners over the years, in which we have invested more than EUR 100 million in making this happen and ensuring that we have the first and only approved treatment for patients with MCT8 deficiency available. It's also worthwhile stressing that this is a full Marketing A uthorization in Europe, so it's not a conditional approval, which means no further clinical trials will be necessary in the European Union.
Let me now review the regulatory pathway for Emcitate or tiratricol, with focus on the United States, with a New Drug Application planned for 2025. Our Marketing Authorization Application, or MAA, which formed the basis for the approval in February this year in the European Union, consisted of data from the studies denoted in green on this slide. In the first study, TRIAL-1, the effects of 12 months of Emcitate treatment on thyroid hormone T3 concentrations and clinical endpoints related to peripheral thyrotoxicosis were established. 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 third study included an extensive natural history study of untreated MCT8 deficiency patients, which enabled an indirect comparison to assess the clinical relevance of the treatment effects seen with Emcitate.
During the review process of the MAA, the top-line data from the TRIAL-2 was included, which, although missed its primary endpoint, provided very important safety data in young patients below 30 months of age, which was requested by the MAA. We were also able to include the encouraging survivor results from the abstract that was published as part of the ETA last year in September by EMC. Focusing on the New Drug Application in the U.S., the FDA has requested the placebo-controlled withdrawal study to verify the effects seen on T3 in single-arm studies, TRIAL-1, and the EMC cohort study. This is what we refer to as the ReTRIACt study. I will shortly give an update on the status of the same on the next slide.
Worthwhile recognizing here is that we also plan to include the full and detailed data set from the survival study in the U.S. New Drug Application later this year. In summary, it should be noted that we have a large and robust data set for an ultra-orphan investigational drug set for the NDA. If you move to the next slide, please. Let's now turn our attention to the ReTRIACt study. As agreed with the U.S. FDA, we're conducting the pivotal study ReTRIACt study in at least 16 available patients with MCT8 deficiency to support the New Drug Application in the U.S. As mentioned before and at previous calls, we opened four additional clinical study sites during 2024 in the U.S. to increase the recruitment capacity: one in Texas, one in Georgia, one in North Carolina, and back end of last year, one also in Florida.
Right now, we have seven study sites open to recruit patients. The current study status is that we have 12 available patients who have completed the randomized treatment phase. One patient is currently in the randomized phase. One patient is in the run-in period, and two patients are planned for screening, with further patients being evaluated for screening. As previously communicated, we continue to have full focus on the ReTRIACt study. We will update the market as the recruitment of the study is closed, and at that point in time, we will be able to provide information on when to expect top-line results and when in 2025 we plan to submit the New Drug Application. With that, I will hand over to Henrik to further give some granularity on the commercialization in Europe and the launch preparations in the U.S. Henrik.
Thanks, Nicklas, and good morning, everyone. We are gearing up for the commercialization of Emcitate through our dedicated team at Egetis in Europe and the US. For the rest of the world, we intend to collaborate with partner companies to ensure broad access to Emcitate for patients. In Japan, we have secured a licensing agreement for development and commercialization with Fujimoto Pharmaceutical Corporation. We have, over the last months, deepened our dialogues with potential partner companies for the Middle East, North Africa, and Turkey. In some of the Middle East countries and in Turkey, it is possible to provide access and sell Emcitate on a so-called named patient basis, and that is based on the EU regulatory approval and based on the German GBA and price.
For the Middle East and Turkey, we interacted with more than 10 companies, all being very interested in Emcitate, and we have now advanced these dialogues and come to a short list of companies that we believe would maximize the Emcitate opportunity. Over the next months, we will continue to do diligence and negotiations with these companies related to collaboration and financial terms with the ambition to close partnering deals. I would again like to take a moment to highlight the European Thyroid Association's guidelines, which were published in July 2024. These guidelines outline the management and diagnosis of MCT8 deficiency, aiding the diagnosis of more patients. We are delighted to see that the ETA guidelines recommend Emcitate as a long-term therapy for all patients with MCT8 deficiency. It is remarkable that the treatment is included in such guidelines even before launch.
These Emcitate supporting ETA guidelines will be further utilized in our disease awareness and also in the pricing and reimbursement efforts throughout Europe. We are executing our pricing and reimbursement strategy in two waves, starting with the four largest EU countries. The French reimbursement dossier was submitted during the first week of April. The German dossier was submitted today. We are excited about our first launch of Emcitate in Germany as of tomorrow. I will soon give you some more details related to the German process and launch. However, first continuing with a brief update on the other EU4 countries. For Spain and Italy, we are on the way to initiate pricing and reimbursement processes. In France, Spain, Italy, and also in other European countries, these pricing and reimbursement processes normally take one to two years.
We are collaborating with experienced local pricing and access advisors to optimize the pricing and reimbursement processes. National pricing and reimbursement is negotiated country by country, and since the 27 EU countries have different processes and timelines, this is relatively complex and takes time. However, there are some key themes that we are focusing on in all countries. In the pricing and reimbursement processes, we deliver the Emcitate value proposition where we describe MCT8 deficiency and its rarity. In addition, we outline the high burden of MCT8 deficiency, which also is confirmed by the Egetis-sponsored caregiver study. The significant unmet medical need in MCT8 deficiency, with Emcitate being the first and only approved treatment.
Last but not the least, the benefits of Emcitate treatment as per the label or the EU SmPC, which is supported by publications and the earlier mentioned ETA guidelines. Let's turn our attention to Germany, where the pricing and reimbursement process lasts for one year in the so-called unknown process. As mentioned earlier, we started this process today by submitting our reimbursement dossier to GBA. The German process is very beneficial from an access to treatment perspective since it is possible for physicians to prescribe the commercial PACs with reimbursed costs also in parallel with the ongoing process. This means that as of tomorrow, which is the official German launch date, we can start to sell reimbursed commercial PACs in Germany at an initial price.
However, after the 12-month unknown process, if the final negotiated price is lower than the initial price, we will be required to pay back the delta between the initial price and the final negotiated price. However, this will only have to be done for the excess amount received during the latter six months of this 12-month period. The German launch strategy is focused on building an expert base that will support other HCPs in Germany related to the management of Emcitate deficiency. We are collaborating with experts to enhance disease awareness and enable diagnosis in Germany. Our efforts are centered on diagnosis, monitoring, and providing treatment guidance for Emcitate deficiency. We emphasize the importance of local publications and clinical training in managing this condition.
We are collaborating with physicians at specialized pediatric centers, the SPZs, and rare disease centers, the ZSEs, both being involved in the management of potential Emcitate deficiency patients. Our goal is to increase disease awareness and foster discussions in local educational training sessions with multidisciplinary teams at these centers. Additionally, we are developing customized awareness campaigns for HCPs and patient support materials in collaboration with disease experts. It is encouraging to see that our strategy works because over the last months, the German team has had over 130 interactions with physicians, including several meetings at many of these specialized pediatric centers, the SPZs depicted on the slide. These engagements resulted in that we became aware of additional patients diagnosed with Emcitate deficiency that soon, hopefully, can be helped by Emcitate.
We are thrilled to now be on our way out of the starting blocks for the launch of Emcitate. This launch represents a significant milestone in our journey to improve the lives of patients affected by this debilitating condition. Up to now, physicians have had to go through some extra work and administration to make their patients become a part of the Emcitate managed access program. However, as of tomorrow in Germany, they can instead simply prescribe Emcitate as any other available pharmaceutical drug, which makes the access to Emcitate easier. For us as a company, the launch also means that we will start to promote Emcitate through branded materials at, for example, congresses, in addition to the disease awareness materials, which have been our focus up to now.
Raising disease awareness will continue to be a key focus area for us in Germany, as well as in other countries, to enable fast and accurate diagnosis of Emcitate deficiency. A few months back, we sponsored an episode on U.S. national TV focused on Emcitate deficiency. The episode became very informative and featured both real-life stories with patients and caregivers living with Emcitate deficiency. In addition, there was contribution from leading endocrinologists and the international patient advocacy group, the MCT8- AHDS Foundation. There were a lot of viewers when the program aired in February and March, but also afterwards, with more than 800,000 social media impressions and over 300,000 video views. This has been a great success. The link to the video has been spread through social media, and the episode is still available through our educational websites, mct8deficiency.com and mct8deficiency.eu.
Lastly, I would like to say a few words about how our disease awareness and our expanded access program helped in the preparations for the launch in the US. Firstly, we see how our activities year by year make us aware of more patients being diagnosed with MCT8 deficiency. Secondly, related to the expanded access program, that was an ask from FDA, this helps diagnosed patients to get access to Emcitate. We are happy to be able to support the families in this way, and there are currently 13 EAP centers in the US, and the number is growing with interest from more centers. The existence of the EAP also helps more physicians to gain experience of Emcitate. Furthermore, it is a great opportunity to generate real-world data that can support upcoming regulatory and payer interactions.
At the future anticipated US approval, the EAP patients will be converted to reimbursed commercial Emcitate packs over the first six months period after US approval. Please note that there are some key and principal differences between sales uptake in the US compared to Europe, and these are actually quite significant. In Europe, the uptake tends to be slower due to relatively lengthy pricing and reimbursement processes. As mentioned, it typically takes one to two years before getting reimbursement. On the other hand, the sales uptake in the US is faster and larger once approval is granted, and this is due to rapid reimbursement and also a potential for higher pricing. Overall, the total potential for Emcitate is clearly largest in the US. In parallel with the launch activities in the EU, we are stepwise establishing our US organization to seize the great US opportunity.
With that, I would like to hand over to Yilmaz for the financial update.
Thank you, Henrik, and good morning to everyone on the call. Revenues for the first quarter of the year were SEK 12.7 million versus SEK 12.1 million in the same period last year, of which SEK 12.6 million is attributable to Emcitate's managed access program. The gross profit margin is visibly lower than historical figures, but comes back to the success we have had with Emcitate in Europe. As a consequence, the COGS line item not only includes the recurring royalty payments to Erasmus Medical Center of 10%, but also non-recurring milestones of approximately SEK 3.5 million to Erasmus Medical Center. In addition, the European approval has also triggered the initiation of monthly SEK 3.4 million of intangible R&D depreciations. Hence, one month of depreciation was included in this quarter.
As many of us know, this is a non-cash item. In an apples-to-apples year-over-year comparison, excluding the last two items mentioned, the gross profit margin would have been slightly better than the Q1 figure, Q1 2024 figure. Results after tax for the first quarter of the year were minus SEK 62.9 million versus minus SEK 75 million. The higher amount versus prior period is mainly driven by the finance line items and specifically the revaluation of the convertible right, which fluctuates with the share price. Q1 2025 figures were plus SEK 10.2 million versus the minus SEK 3.4 million in Q1 2024. This is a total year-over-year difference or deviation of SEK 13.6 million. Remember that this is also a non-cash item.
It is better to look on the operating cash flow for the first quarter of the year, which were SEK -66.1 million versus SEK -55.4 million in the same period last year. The difference, as you can see, is mainly explained by the working capital line items, where we had a negative change of SEK 7.9 million in Q1 2025, while the Q1 2024 numbers were positive SEK 10.3 million. This is a year-over-year difference of SEK 18.2 million. The cash position at the end of March was SEK 273 million versus SEK 252 million last year, giving us a better start at the next quarter with a better footing. With that, I would like to hand back to Nicklas.
Thank you very much, Yilmaz. Thank you very much, Henrik.
To summarize, we have a very exciting period ahead of us with quite a few milestones, such as the European launch starting with Germany tomorrow, 1st of May. We're also very much looking forward to the top line results of the ReTRIACt study following by the New Drug Application in the U.S. this year. This means that we expect the U.S. approval and launch of Emcitate in the U.S. during 2026. With that, I would like to hand over the call to the operator and start the Q&A session. Operator, please go ahead.
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 Arvid Necander from Carnegie. Please go ahead.
Good morning and thanks for taking my questions.
First off, can you provide any more detail on the progress in Germany ahead of the launch? How many centers or physicians you believe will be ready to begin prescribing and treating patients from tomorrow? Yeah, perhaps I'll start there.
Thank you, Arvid, and good morning to you as well. I think you saw that on one of the slides Henrik elaborated on. We have started, actually, the launch preparations already two years ago, engaging with the different sites, identifying the specialty sites here for rare diseases and engaging with the specific physicians on those sites together with advisory boards. I don't know, Henrik, if you want to provide some more granularity on that.
Yeah, of course, I can elaborate a little bit further.
We do not disclose numbers of patients, but as you can imagine, we have some patients in the managed access program in Germany, and they will be swiftly converted now to reimbursed commercial PACs. In terms of activities, we have, as Nicklas said, been working, preparing the German market over the last two years, and we have held advisory boards with key experts in Germany and have now, over the last months, also extended our presence to more centers. It is really encouraging to see that we all the time also have the possibility to find new patients still in Germany. That is, of course, a key focus area for now in the launch phase to really make sure that we also drive disease awareness and enable fast and accurate diagnosis of more patients.
Okay, fair enough. Just a quick follow-up on that.
I assume you're sort of starting by addressing higher volume centers that can have a sort of regional center of excellence position. Can you provide any granularity on that? How many centers are you really targeting in full effect in the initial launch?
Yeah, we are focusing on these specialized pediatric centers, SPZs, and the rare disease centers, because these are the centers where most of these kind of patients will appear. There we are working from the experts, those that we already now have experience with Emcitate through the managed access program. We leverage them to also educate more physicians and also at other centers. In total, it's roughly 150 SPZs and a bit more than 50 rare disease centers. Those are the ones that we are focusing on.
Maybe calibrate that somewhat further, Arvid.
It's about engaging the key opinion leaders locally in Germany, getting their help for further education. As Henrik said, we have been actually over the last 12 months present at the majority of these centers as well, making sure that we can drive disease awareness and also encourage the upcoming launch.
Perfect. Thanks for that additional color. Just an additional one, if I may. I guess in light of the expected tariffs on pharmaceuticals and related ingredients, could you just walk us through your production chain and comment on potential impacts?
Yeah, I think you're referring to the potential U.S. tariffs on pharmaceuticals. You were breaking up a bit, Arvid, just so you know. The potential U.S. tariffs and how do we address that? Obviously, we all know with the new administration that it's very dynamic, so to say.
It's difficult to predict what our formal final outcome will be. Our current supply chain setup is as such that we have API production and drug product production in Europe. What we're looking at ahead of a US launch is potentially having secondary packaging in the US that might potentially mitigate some of the effects we potentially will see from tariffs.
With that, thank you, Arvid. Let's move on to the next question, if you please.
The next question comes from Suzanna Queckbörner from Handelsbanken. Please go ahead.
Hello, Susanna Queckbörner, Handelsbanken. I'd like to follow up on the situation in Japan. Perhaps you can give us an update whether your partner is going to require further clinical trials and what's the timeline here.
Thank you, Suzanna, and good morning to you as well. This is obviously very exciting.
I think you're referring back to the fairly recently issued new guidelines from PMDA that came into place in October last year, where the guidelines now stipulate a somewhat more relaxed approach to a regulatory pathway in Japan. The guidelines stipulate that if you fulfill certain criteria, such being a rare disease product, such being having a pivotal study or an approval elsewhere, and together with the combination of it's difficult to execute a clinical trial, you can get an exemption for a local clinical trial in Japan. We are currently actually together with our partner in the middle of discussions with PMDA on this topic. We will have to get back to you at some point during the summer on how the regulatory pathway will look like.
Obviously, where we're coming from is that we very much meet those criteria stipulated in new guidelines and should be exempted for a local clinical trial.
Okay, thank you. Also, to follow up on the managed access program, is it right to assume that once you've launched in a country, those patients will no longer have access?
No, that's not fully correct. I can start, and Henrik can hand over, because of course, being a pharmaceutical company like ours, we have of course strong ethical commitments making sure that no patients on treatment today will be standing there without the treatment. I think it goes by country by country basis. If you think about Germany, as Henrik mentioned, there we will convert the managed access program patients as quickly as possible to so-called reimbursed patients.
In countries like France, for example, of course, they will continue to receive managed access product throughout the pricing and reimbursement process and until that is completed. Please, Henrik, if you want to elaborate further.
Yeah, I think you said it. I mean, we are committed to provide Emcitate to the patients in the managed access program until they have a reimbursed funding mechanism in their countries.
Thank you, Suzanna. Thank you, Henrik. Operator, can we move to the next question and potentially the last question since we are running out of time? I apologize for this.
The next question comes from Alexander Krämer from ABG Sundal Collier. Please go ahead.
Yes, good morning. I have a couple of questions, if I may, maybe a simple one to start with. Previously, you talked about the publication of the listed price in Germany in LAUER-TAXE .
Could you provide some color on when can we expect that? Like something like in May or like later? I have a couple of other questions.
Yeah, I think the simple answer to that, and Henrik can elaborate, is we as a company won't publish our prices publicly. There will be a price as of tomorrow in Germany that will be used when physicians prescribe Emcitate as of tomorrow. Did you have some follow-up questions? Yeah, Alexander?
Yes. I have two follow-up questions. One on the reimbursement process in Germany. My question is here for the treating physician, if it is a straightforward process actually to prescribe Emcitate from tomorrow, or if they also have to get approval from the health insurance company, so the Krankenkasse in Germany, and how this translates also into your sales estimates for 2025 in Germany.
I mean, of course, if there's some delay because of some bureaucratic processes, still, of course, should we expect to get first sales in Q2, or is it more like a story of Q3, Q4?
Yeah, thank you for the questions. In some countries, it is a little bit of a complex process, but in Germany, it's actually very smooth. The physicians can just prescribe now, and the drug will be reimbursed. Yes.
From tomorrow. I think it's important to stress Germany is maybe somewhat different to other countries, but in Germany, the process is very smooth. It's actually simpler to prescribe the reimbursed drug tomorrow compared to go through the managed access program. There shouldn't be any hurdles there.
Then related to your follow-up questions on the timing, I mean, we do not guide on sales, but here it depends, of course, related to when the physicians picked up the last PACs from the managed access program. When the patients run out of treatment, they will get new prescriptions and get the reimbursed drug from the pharmacies.
Great.
Thank you, Alexander. Maybe we have time for one last question. Operator, please go ahead.
The next question comes from Oscar Hafen Lamb from Bryan Garnier. Please go ahead.
Hi, team. Good morning. My question would be around the conversion rates that you have seen so far in the U.S. trial between the patients being screened and then being subsequently included in the run-in.
I guess my question would be, how confident can we be that the two patients that are currently being screened will eventually end up being included in the run-in?
I think thank you. I think this is a complex question in the sense of it depends on if it's a patient already on treatment, where we have, of course, incredibly high conversion rates, or if it's treatment-naive patients meeting all the eligible criteria required for an inclusion. I think I can't give you an exact number there, Oscar. Unfortunately, what I can say, though, is that we have confidence that we will be delivering results this year and also submission this year.
I think it's important to stress that if you look back, if you can use history as a bit of a proxy, we had eight patients complete the randomized treatment period in December as of last year. We now end of April, and now we have 12 patients complete the randomized treatment period. We have an additional patient in the randomized treatment phase. We have an additional patient in the run-in period or the titration period. We have additional two patients being planned for screening in the upcoming period. It is difficult to give you some granular detail around that because it is very much patient-by-patient dependent.
Okay, I understand. Thank you.
Thank you very much, Oscar. With that, thank you very much to all the participants on the call. Excellent questions, and we bid you a very good rest of the Wednesday. Thank you.