Welcome to Egetis Therapeutics Q3 report 2025. During the Q&A 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, Operator, and good morning, everyone, and a warm welcome to Egetis Therapeutics' Q3 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, Vice President Commercial Operations; Karl Hård, Vice President Investor Relations; and Christian Sonesson, Vice President Product Strategy and Development. Let's turn our attention to the topics to be covered today. I am very pleased with the great progress made during the quarter and post the period. Amongst others, FDA awarded us a Breakthrough Therapy designation for Triac on the 14th of July this year. We had a very successful pre-NDA meeting on October 21st with the FDA, with the agreement that Egetis can submit the NDA for MCT8 deficiency based on currently available data.
Subsequently, we completed a ReTRIEVE study of MCT8 and announced positive results, which leads to us being able to commence a rolling NDA submission in December this year. Furthermore, we will also cover the commercialization in Europe and launch preparations in the U.S., as well as a financial update and leave ample time for questions. Let's start off with the Breakthrough Therapy designation granted by the FDA on the 14th of July. In the first quarter 2025, we received the entire dataset from the Erasmus Medical Center, also called EMC, in Rotterdam, with survival data from more than 600 patients with MCT8 deficiency. Approximately one-third of these patients had been treated with Triac, and two-thirds had not. We submitted the Breakthrough Therapy designation application based on our own detailed analysis of this survival data, which confirmed a significant and substantial improvement in survival in Triac-treated patients.
The Breakthrough Therapy designation underscores both the urgent need for an effective treatment for patients with MCT8 deficiency and the clinically meaningful evidence demonstrated today with Triac. Receiving a Breakthrough Therapy designation this late in a clinical development program is, of course, very encouraging for the forthcoming NDA process, as these designations are typically awarded at an early stage in development. Let's now focus on the outcome of the pre-NDA meeting with the FDA. The objective of the pre-NDA meeting was to seek FDA advice and agreement on the overall content to support our New Drug Application for MCT8 deficiency in the U.S., with a special focus on the clinical data package, including the role and position of the ReTRIEVE study. Importantly, and as agreed with the FDA, the NDA for MCT8 deficiency will be based on currently available clinical data.
Furthermore, the ReTRIEVE study is no longer considered as pivotal for the NDA, which it was when the study was designed. FDA agreed that in the NDA submission, data from the ReTRIEVE study will complement the existing data package from the EMC survival study and other clinical components. The FDA also recommended that the statistical analysis plan, the SAP, for the trial should be revised. Recognizing that MCT8 deficiency is an ultra-rare condition, we have an unusually large, robust clinical dataset from six different sources. This includes TRIAL-1 with 46 patients, TRIAL-2 with 22 patients, the ReTRIEVE study with 15 patients, the EMC core study with 67 patients, and also the EMC survival study with over 600 patients.
Furthermore, we also reached an agreement with the FDA that we can commence a rolling NDA submission in December 2025, targeting a complete NDA submission in early 2026 and anticipating completion of the FDA review process in the third quarter of 2026, as we expect a priority review. In summary, as I'm sure you all appreciate, we are very pleased with the outcome of the pre-NDA meeting and the clear path to an NDA submission for MCT8 in the U.S. based on currently available clinical data. Let's now turn our attention to the positive outcome of the ReTRIEVE study. On the 14th of November, we issued a press release announcing positive top-line results from the ReTRIEVE study with MCT8. Based on the comments from the FDA at the pre-NDA meeting, the statistical analysis plan of the ReTRIEVE study was revised with the intention to close down the study prematurely.
The revised SAP focused on more efficient use of the data collected during the randomized treatment period, or RTP, using all T3 data, not only the binary information on rescue yes or no. That's why we considered the rate of change in T3 during the RTP, using all patient data on T3 collected to increase the statistical power of the study. Subsequently, and therefore a primary endpoint 1 evaluating the rate of change in T3 during the RTP was added to the previously described primary endpoint based on T3 rescue criterion, from here on referred to as primary endpoint 2. Type 1 error is controlled via alpha recycling, testing primary endpoint 1 first, and if primary endpoint 1 is met, primary endpoint 2 will then be tested. The revised statistical plan was submitted to the FDA before database lock and unblinding of the ReTRIEVE study.
As announced, the positive top-line results demonstrate a statistically significant difference in the rate of change in T3 in patients randomized to placebo versus patients continuing on Triac, with a p-value of 0.034. If you furthermore look at the results depicted here on the slide, what you see depicted to the left is the ranking of patients according to their change in T3 during the 30 days randomized treatment period. Displayed with a dashed line is the line of no change from baseline. Hence, the patients with the largest increase in T3 during the RTP is in the top of the figure, and the patients with the largest decrease is at the bottom of the figure. What we observed on T3 movements is exactly what we expected.
When we draw in Triac, the T3 levels in all placebo patients increase, but not much happens in the Triac group. In fact, there is a complete separation of the two groups, with all patients randomized to placebo having a larger increase in T3 than all seven patients randomized to Triac. What does this really tell us, and what does this mean for the results on the primary endpoint? On the right-hand side, the upper table shows the results for primary endpoint 1, the rate of change in T3 during the RTP. For the placebo group, the T3 rate of change was 1.590, which means a 59% increase in T3 over 30 days, whereas there was essentially no change for the Triac group.
There was a statistically significant difference between placebo and Triac, expressed here as the ratio of the T3 rate of change, which was 1.494, with a p-value equal to 0.034. In the lower table, you can see the results for primary endpoint 2. Four patients randomized to placebo met the rescue criterion, and no patients randomized to Triac met the rescue criterion. One patient, though, randomized to Triac discontinued during the RTP. This discontinuation was not drug-related, and the patient is marked with a D in the left-hand figure, as you can see. Experienced no change in T3 during the RTP. However, as per the SAP, this Triac patient was still imputed as a rescue for the analysis of primary endpoint 2, leading to a 4 placebo versus 1 Triac comparison, for primary endpoint 2, which is not statistically significant.
However, the sensitivity analysis below only considered the observed cases of rescue, which leads to a comparison of four patients meeting the rescue criterion on placebo versus zero patients randomized to Triac, with a p-value of 0.070. Although not shown on this slide, after the RTP was completed, patients randomized to placebo reinitiated Triac treatment, and they all showed an expected decrease in T3 levels in the follow-up period. To conclude, we are very pleased with the outcome from the pre-NDA meeting with the FDA and subsequently the positive ReTRIEVE study outcome. As previously communicated, we now plan to submit an NDA for MCT8 deficiency with currently available clinical data. We have a very robust dataset for such an ultra-rare genetic disorder, including data from the trials previously mentioned and could be visible here on the slide.
Our plan is to commence a rolling submission in December 2025, targeting complete NDA submission in early 2026, and an anticipated completion of the FDA review process in quarter three 2026. With that, I will hand over to Henrik for an update on commercialization in Europe and the launch preparations in the U.S.
Thank you, 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 Triac 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 Triac for patients. We have started the execution on this strategy by initiating the commercialization of Triac in Europe and started the market preparations in the United States.
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 and agreement with Erkim for Turkey. I'm very pleased with how this collaboration works. The Erkim team is in dialogue with several physicians and with payers related to funding of MCT8 for identified patients. In October, we announced our second distribution deal, an agreement with Taiba to enable MCT8 access and name patient sales in the Gulf region, initially Saudi Arabia, United Arab Emirates, Qatar, Oman, and Bahrain. The ambition is to extend to more countries over time. All in all, we continue to deliver on our commercialization strategy with our focus on reaching patients with urgent unmet medical needs.
As we continue to execute our European commercialization strategy, I'm pleased to report steady progress across key markets, with pricing and reimbursement processes moving forward in parallel. In Germany, we have successfully transitioned all managed access patients to commercial Triac product. Our ongoing dialogues with physicians are delivering greater awareness of MCT8 deficiency, resulting in new patient identifications and treatment initiations. In France, our first reimbursement submission was rejected in September by the French Health Authority, HAS. This is not uncommon when it comes to orphan drugs in France, since the HAS ideally would like to see randomized control data. We are now preparing a resubmission early 2026 to address the payer objections, and we'll then include new available data, such as the recently announced positive placebo control data from the ReTRIEVE trial.
In this process, on top of new data, we also have great support by physicians who already have patients on Triac treatment. In Italy, the pricing and reimbursement process is underway, and commercial product is now available to patients. In Spain, we recently held a successful advisory board with physicians and have support to generate local data to optimize our upcoming pricing and reimbursement submission. Beyond our core European markets, we are actively utilizing alternative funding pathways to facilitate name patient sales in additional countries. To date, this approach has enabled us to successfully provide Triac to patients in Poland, Austria, and Switzerland. Overall, we are delivering tangible results, expanding patient access, and building momentum for Triac across Europe. Let me also walk through our U.S. pre-launch progress, where we continue to execute with strong discipline.
We have made significant strides in expanding our reach among healthcare providers and accelerating the diagnostic pathway for MCT8 deficiency. We have grown our engagements across endocrinology, genetics, and neurology, with increased diagnostic considerations for MCT8 deficiency. Our partnerships with key opinion leaders and patient advocacy groups remain vital in raising awareness and supporting families. This momentum is translating into real impact, validating our awareness efforts. We are now aware of around 140 diagnosed patients in the U.S. Our expanded access program is active at 15 sites, providing early access for eligible patients, generating valuable clinical insights, and building operational experience ahead of launch. On market access, we have advanced our readiness by engaging with payers and stakeholders, directly informing our launch pricing strategy and strengthening our overall framework for distribution and patient services.
Finally, we have recently added employees to our US organization, establishing core leadership and expanding our field medical capabilities to ensure the clinical community is prepared to diagnose and treat patients once Triac becomes approved by the FDA. In summary, we are executing our pre-launch strategy across four pillars: medical awareness and diagnostic acceleration, early access, market access readiness, and scalable launch infrastructure. With these foundations, we are confident in our ability to, once regulatory approved, deliver a strong commercial launch and bring a transformative therapy to children and families living with MCT8 deficiency. With that, I would like to hand over to Yilmaz for the financial update.
Thank you, Henrik. If we look at the financial slides then, revenue for the first nine months were SEK 44.6 million versus SEK 35.3 million in the same period last year.
Revenue for the third quarter of the year was SEK 17.4 million versus SEK 9.4 million in the same period last year, of which all is attributed to MCT8. As mentioned in our half-year call and worth reminding everyone again, the gross profit margin is visibly lower than historical figures, both for the nine months and the third quarter. This comes back to the regulatory success we had with MCT8 in Europe and starting of the depreciation. As a consequence, the COGS line item numbers not only include 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 23.6 million of intangible R&D depreciation.
A reminder to listeners is that the depreciation is a non-cash item, and it was initiated at the end of Q1 as a consequence of our approval in Europe. In an apples-to-apples year-over-year comparison, adjusting for the one-time payment and the depreciation, adjusted gross profit is SEK 35.3 million for the first nine months. This is an improvement not only nominally, but also on the gross margin level versus the corresponding period, the Q3. Operating results were nearly the same for the nine months at minus SEK 219.6 million versus SEK 224.7 million in the corresponding period. Results after tax for the nine months of the year were minus SEK 222.8 million versus minus SEK 233.1 million. The improvement versus prior corresponding period is mainly driven by better cost control and the finance line items.
Specifically looking into the finance line items, you see the revaluation of the convertible right, which fluctuates with the share price. The nine months 2025 figures for the revaluation were plus SEK 6 million versus plus SEK 3.1 million. Remember that this is also a non-cash item. For the first nine months, cash flow from operating activities were almost completely the same at minus SEK 174.2 million versus minus SEK 174.3 million. Also, as you may have noticed, we do continue to repay our debt and report SEK 21.7 million in repayment for the first nine months. This is an item which did not exist in the corresponding period. All in all, the cash flow for the first nine months were minus SEK 200.6 million versus minus SEK 176.2 million. The cash position end September 2025 was SEK 145.7 million versus SEK 129.9 million last year.
In comparative period, we are in a better shape there. It was gratifying to see that we did strengthen the cash position further through the great support from our existing and new shareholders through the directed rights issue carried out post the closeout of period on October 2. This did result in a net cash injection to the company of SEK 172 million and should be added on top of the reported cash position in the earnings release. With this, I hand back to Nick.
Thank you, Yilmaz. To conclude, let's move on to the next slide call, please. In essence, I think we are very pleased with the productive year we have had to date.
We have delivered some historical milestones, such as Triac becoming approved in the European Union as the first and only approved treatment for the devastating condition of MCT8 deficiency and subsequently launched it in Germany. We have signed two partnerships to expand the access in Turkey and in the Gulf region. We had a very successful pre-NDA meeting on the back of the Breakthrough Therapy designation granted, leading to an NDA submission starting already this year. Going forward, even more exciting times are ahead of us with a potential U.S. approval and launch for Triac, again, as the first and only approved therapy for MCT8 deficiency in the U.S. With that, operator, please go ahead and we'll take some questions.
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.
Hi, Suzanna.
Hi. Great. I'd like to start with two questions. First, a question on the ReTRIEVE trial. I'd like to just follow up on the comments you received from the FDA in terms of what comments did they make that they wanted to change the statistical analysis. Perhaps you can sort of expand on that to start off with.
Yeah. No, we had, as you know, we had a very, very constructive and positive dialogue with the FDA. I really need to commend their collaborative spirit.
As you remember, the primary object of the ReTRIEVE study has been to confirm the effect on T3 seen in other clinical studies as attributed to thoracic wall, hence why the ReTRIEVE study was placebo-controlled. The discussion we had, of course, with the FDA, with their now agreement to submit with currently available data, and subsequently the intention to close down the ReTRIEVE study immediately, we considered in discussion with the FDA if there would be a more efficient use of the data collected during the randomized treatment period, i.e., using all T3 data collected, not only the one on information on rescue, yes or no, which is very much binary. That's why we changed the primary endpoint or added the primary endpoint one as a rate of change in T3 over the randomized treatment period, using all patient data on T3 collected to increase the statistical power.
There, I don't know, Christian, I don't know if you have anything to add from your perspective there.
I think you articulated very well, Niklas. I think it was a very good and clear guidance from the FDA at the meeting, which led us to amend the SAP accordingly. Just as you say, we targeted the most efficient endpoint and promoted that to primary endpoint one, while at the same time, of course, keeping the original primary endpoint in the study as well.
Understood. In terms of if we look into the rescue part of the trial and four patients required rescuing while four didn't, how do you think about this? Do you think that the one-month period was not enough, or what is your sort of interpretation of that?
Maybe I can start, Christian, and you can add.
Of course, we are in the process of still analyzing all the data. What we see is, as I mentioned before, that there's a clear separation between the two groups. All eight patients randomized to placebo have a larger increase of T3 compared to the seven patients randomized to Triac. What we actually observe on T3 movements is exactly what we expected. Christian, anything to add from your perspective?
I think that based on what we see on the primary endpoint one, we have a 59% increase in the placebo group over 30 days. We have a more or less stable Triac treatment T3 levels. It is really a question of time before you would have all the placebo patients hitting the rescue criteria.
I think it becomes a little bit on the sort of interplay between where do you put the rescue criteria versus how long time you have. I think that the results on the primary endpoint one strongly suggest that if we had continued the trial for just a little bit longer, you would have seen all placebo patients rescue.
Thank you, Christian. Really, to summarize, one should also take a step back. We have an unusually large and robust data set. Again, Suzanna, coming back to the number of studies and number of patients we have in this ultra-rare condition where everything is pointing in the right direction. Most importantly, obviously recognizing the survival benefits we have seen in patients treated with Triac versus untreated patients.
Subsequently, the good discussion with the FDA that we are actually moving forward and agreed, that the agency agreed to submit based on currently available data. Maybe we should move to the next question. Thank you so much, Suzanna, for your questions.
Thank you.
The next question comes from Chiara Montironi from Van Lanschot Kempen. Please go ahead.
Hello, team. Thanks for taking my question. To start off, a clarification. You mentioned that all patients on placebo restarted thoracic wall and the T3 levels decreased again. Do you mean all patients on placebo or only the four that met the criterion?
I hope I understood the question, Chiara. I think I'm playing it back to you. You're referring to after the randomized treatment period, correct? And if they—
Correct yeah.
Yes, you're absolutely correct that it wasn't shown on the slide, but after the randomized treatment period was completed, patients that were randomized to placebo reinitiated Triac treatment, and they all showed, as expected, a decrease in T3 levels in the so-called follow-up period. Yes, you're correct.
All eight?
Yes, correct.
Okay, great. A second question, if I may. Could you give us a sense of how much establishing the US commercialization port will weigh on your spending?
We don't give guidance on cash burn rate and investment levels, but trying to triangulate this for you, Chiara, because obviously it's important, right? US will be the most important market for Egetis, for MCT8, as it is for many other companies.
Of course, we'll make sure that we have sufficient investments moving forward ahead of the launch and through the launch in the back end of 2026 or second half of 2026. Of course, you will see an increase in investments in the commercial and medical affairs infrastructure in the U.S. However, that will be offset by decreased R&D costs since we're now closing down the ReTRIEVE study. To calibrate a bit on the size of the infrastructure, we envisage to be around 25 FTEs at the point of launch. We have started to build that up already now in the second half of 2025, and we'll do further more of that in the first half of 2026.
Okay, thank you.
Thank you, Chiara.
The next question comes from Frederick Thor from Redeye. Please go ahead.
Hello, and thank you. My question was about you mentioned that all German patients on early access have been converted. How well is that reflected in the sales for the quarter? I mean, how early in the quarter were all converted and so on? Can you give us some more details on that?
I can start and Henrik, you can build, right? Obviously, we have mentioned this before. We do not give granular information on a patient-by-patient basis. We had the majority of the patients converted in quarter two. We had a few also converted in quarter three. More importantly, as Henrik mentioned, we have also identified further patients that have now also been prescribed treatment. It is work in progress, but it is definitely moving in the right direction.
Got it. A final question about the process in France. Can you give us some detail on the timeline there?
What is the best hope for getting approved reimbursement in France?
Yeah, Henrik, you're happy to answer to that. First of all, I just would like to repeat that it is not uncommon that the reimbursement submission initially is rejected in France since the HIS ideally would like to see randomized control data. We are now preparing for the resubmission early 2026 to address their objections. We will then include new data available, and that is, of course, both the positive ReTRIEVE data, but also more survival data. This process is expected to take one to two years, which normally is the case in France. Overall, I mean, in our resubmission, since it will consist of a much stronger data package, we see a much higher likelihood of getting reimbursed in France.
Got it. Thank you. That's all from me.
Thank you, Frederik. With that, we recognize that we're running over time. With that, we need to close down and thank the audience for their participation and wish everybody a great rest of the day. We are looking forward to an exciting 2026. Thank you.