Egetis Therapeutics AB (publ) (EGTX) Earnings Call Transcript

Welcome to Egetis Therapeutics Q3 Report 2025. [Operator Instructions]. 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 Quarter 3 results call planned for the coming 30 minutes. For those who I haven't had the privilege to meet before, I'm Nicklas Westerholm. I'm the CEO of the company. With me today, I also have Yilmaz Mahshid, Chief Financial Officer; Henrik Krook, Vice President, Commercial Operations; Karl Hard, Vice President, Investor Relations; and Christian Sonesson, Vice President, Product Strategy and Development. So 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 tiratricol on the 14th of July this year. We had a very successful pre-NDA meeting on October 21 with the FDA with agreement that Egetis can submit the NDA for Emcitate based on currently available data. Subsequently, we completed the ReTRIACt study of Emcitate 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. So let's start off with the breakthrough designation granted by the FDA on the 14th of July. In the first quarter 2025, we received the entire data set from the Erasmus Medical Center, also called EMC, in Rotterdam, with survival data from more than 600 patients with MCT8 deficiency. Approximately 1/3 of these patients have been treated with tiratricol and 2/3 had not. We submitted a Breakthrough Therapy Designation application based on our own detailed analysis of the survival data, which confirmed a significant and substantial improvement in survival in tiratricol treated patients. The breakthrough designation underscores both the urgent need for an effective treatment for patients with MCT8 deficiency, and the clinically meaningful evidence demonstrated today with tiratricol. Receiving a breakthrough designation this late in the 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 Emcitate in the U.S. with a special focus on the clinical data package, including the role and position of the ReTRIACt study. Importantly, and as agreed with the FDA, the NDA for Emcitate will be based on currently available clinical data. Furthermore, the ReTRIACt 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 ReTRIACt 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 usually large robust clinical data set from 6 different sources. This includes Triac Trial I with 46 patients, Triac Trial II with 22 patients, the ReTRIACt study with 15 patients, the EMC cohort 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 ]. So 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 Emcitate in the U.S. based on currently available clinical data. Let's now turn our attention to the positive outcome of the ReTRIACt study. On the 14th of November, we issued a press release announcing positive top line results from the ReTRIACt study with Emcitate. Based on the comments from the FDA at the pre-NDA meeting, the statistical analysis plan of the ReTRIACt 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 consider 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 criteria, 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 ReTRIACt study. And 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 tiratricol with a p-value of 0.034. And 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 the 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. And what we observed on T3 movements is exactly what we expected. When withdrawing tiratricol, the T3 levels in all placebo patients increase, but not much happens in the tiratricol group. In fact, there is a complete separation of the 2 groups with all patients randomized to placebo having a larger increase in T3 than all 7 patients randomized to tiratricol. So what does this really tells 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 tiratricol group. There was a statistically significant difference between placebo and tiratricol 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 criteria and no patients randomized to tiratricol met the rescue criteria. One patient, though, randomized to tiratricol discontinued during the RTP. This discontinuation was not drug related, and the patient marked with D in the left-hand figure, as you can see, experienced no change in T3 during the RTP. However, as per the SAP, this tiratricol patient was still imputed as a rescue for the analysis of primary endpoint 2, leading to a 4 placebo versus 1 tiratricol comparison for primary endpoint 2, which is not statistically significant. However, the sensitivity analysis below only considered the observed cases of rescue leads to comparison of 4 patients meeting the rescue criteria on placebo versus 0 patients randomized to tiratricol with a p-value of 0.070. Although not shown on this slide, after the RTP was completed, patients randomized to placebo reinitiated tiratricol treatment and they all showed an expected decrease in T3 levels in the follow-up period. So to conclude, we are very pleased with the outcome from the pre-NDA meeting with the FDA, and subsequently the positive ReTRIACt study outcome. As previously communicated, we now plan to submit an NDA for Emcitate with currently available clinical data. We have a very robust data set 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 updated -- and an anticipated completion of the FDA review process in quarter 3, 2026. With that, I will hand over to Henrik for an update on commercialization in Europe and the launch preparations in 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. So 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 commercialization of Emcitate 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 Er-Kim for Turkey. I'm very pleased with how this collaboration works. The Er-Kim team is in dialogue with several physicians and with payers related to funding of Emcitate for identified patients. In October, we announced our second distribution deal, an agreement with Taiba to enable Emcitate access and named patient sales in the Gulf region; initially, Saudi Arabia, United Arab Emirates, Qatar, Oman and Bahrain. And the ambition is to extend to more countries over time. So 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 Emcitate 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 controlled data. We are now preparing a resubmission early 2026 to address the payer objections and will then include new available data such as the recently announced positive placebo-controlled data from the ReTRIACt trial. In this process, on top of new data, we also have great support by physicians who already have patients on Emcitate 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 named patient sales in additional countries. To date, this approach has enabled us to successfully provide Emcitate to patients in Poland, Austria and Switzerland. So overall, we are delivering tangible results, expanding patient access and building momentum for Emcitate across Europe. Let me also walk through our U.S. prelaunch progress, where we continue to execute with strong discipline. We have made significant strides in expanding our reach among health care 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 rising awareness and supporting families. This momentum is translating into real impact, validating our awareness efforts. So 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 U.S. organization, establishing core leadership and expanding our field medical capabilities to ensure the clinical community is prepared to diagnose and treat patients once tiratricol becomes approved by the FDA. So in summary, we are executing our prelaunch strategy across 4 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 9 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 were SEK 17.4 million versus SEK 9.4 million in the same period last year, of which all is attributed to Emcitate. 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 9 months and the third quarter. This comes back to the regulatory success we had with Emcitate in Europe and starting of the depreciation. As a consequence, the COGS line item numbers not only includes the recurring royalty payments to the founders of 3% and the Erasmus Medical Center of 10%, but also nonrecurring 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 that the depreciation is a noncash 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 onetime payment and the depreciation, adjusted gross profit is SEK 35.3 million for the first 9 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 9 months at minus SEK 219.6 million versus SEK 224.7 million in the corresponding period. Results after tax for the 9 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 9 months 2025 figures for the revaluation were plus SEK 6 million versus plus SEK 3.1 million. Remember that this is also a noncash item. For the first 9 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 9 months. This is an item which did not exist in the corresponding period. All in all, the cash flow for the first 9 months were minus SEK 200.6 million versus minus SEK 176.2 million. And the cash position in September 2025 was SEK 145.7 million versus SEK 129.9 million last year. So in comparative period, we are in a better shape there. And 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 should 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. And to conclude, let's move on to the next slide, please. So 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 Emcitate 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 2 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 designation granted leading to 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 Emcitate, again, as the first and only approved therapy for MCT8 deficiency in the U.S. So with that, operator, please go ahead, and we'll take some questions.

[Operator Instructions] The next question comes from Suzanna Queckbörner from Handelsbanken.

I'd like to start with 2 questions. So first, a question on the ReTRIACt 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?

Yes. No, 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 objective of the ReTRIACt study has been to confirm the effect on T3 seen in other clinical studies as attributed to tiratricol, hence, why the ReTRIACt study was placebo-controlled. The discussion we had, of course, with the FDA with their now agreement to submit the currently available data and subsequently the intention to close down the ReTRIACt study immediately. We considered, in discussion with the FDA, if there would be 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 1 as a rate of change in T3 over the randomized treatment period using all patient data on T3 collected to increase the statistical power. And there, Christian, I don't know if you have anything to add from your perspective there.

I think you articulated very well, Nicklas. I think it was a very good and clear guidance from the FDA at the meeting, which led us to amend the SAP accordingly. And just as you say, we targeted the most efficient endpoint and promoted that to primary endpoint 1, while at the same time, of course, keeping the original primary endpoint in the study as well.

Understood. And so then in terms of -- if we look into the rescue part of the trial and 4 patients required rescuing while 4 didn't. How do you think about this? Do you think that the 1-month period was not enough? Or what is your sort of interpretation of that?

Maybe I can start, Christian, and you can add. And of course, we are in the process of still analyzing all the data. But what we see is, of course, as I mentioned before, is that it's a clear separation between the 2 groups. All 8 patients randomized to placebo have a larger increase of T3 compared to the 7 patients randomized to tiratricol. So what we actually observed on T3 movements, it's exactly what we expected. Christian, anything to add from your perspective?

I think that based on what we see on the primary endpoint 1, we have a 59% increase in the placebo group over 30 days. We have a more or less stable tiratricol treatment T3 levels. So it's really a question of time before you would have all the placebo patients hitting the rescue criteria. And 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. But I think that the results on the primary endpoint 1 strongly suggest that if we had continued the trial for just a little bit longer, you would have seen all placebo patients rescued.

Thank you, Christian. And then 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 and most importantly, obviously, recognizing the survival benefits we have seen in patients treated with tiratricol versus untreated patients. And subsequently, the good discussion with the FDA that we are actually -- we are 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.

The next question comes from Chiara Montironi from Van Lanschot Kempen.