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

Welcome to Egetis Webcast. [Operator Instructions] Now I will hand the conference over to CEO, Nicklas Westerholm. Please go ahead.

Good morning, everyone, and welcome to Egetis Therapeutics webcast. My name is Nicklas Westerholm, CEO of Egetis Therapeutics. And with me today, I also have Kristina Sjöblom Nygren, Chief Medical Officer; Christian Sonesson, Vice President, Product Strategy and Development; Karl Hard, Head of Investor Relations; and Yilmaz Mahshid, Chief Financial Officer. This morning, we issued a press release announcing positive top line results from the ReTRIACt study of MCT8 in MCT8 deficiency. We are, of course, incredibly pleased with this positive study outcome. The positive top line results demonstrate a statistically significant difference in the rate of change in serum T3 in patients randomized to placebo versus patients continuing on tiratricol with a p-value of 0.034. The ReTRIACt study is the last clinical study to be reported for MCT8 in MCT8 deficiency and complement the EMC Survival Study data and other clinical data for the upcoming new drug application, as discussed with the FDA in our October 2025 pre-NDA meeting. Subsequently, we'll plan to start a rolling NDA submission in December this year, targeting a complete NDA submission in early 2026 with an anticipated completion of the FDA review process in the third quarter of 2026. Let's now turn our attention to the design of the ReTRIACt study. The ReTRIACt study is a double-blind, placebo-controlled randomized withdrawal study. Patients included in the trial could be either already on tiratricol treatment or retreatment naive. During the running period, patients were titrated to achieve stable serum T3 levels within the normal range. Once patients had a stable T3 level, they were randomized to either continue tiratricol treatment or to receive placebo. When patients are randomized to placebo, it's reasonable to expect a reversal of the tiratricol treatment effect and hence, increased T3 levels. As a result of the pre-NDA meeting we held with the FDA in October 2025, the NDA for Emcitate will be based on currently available clinical data, where data from the ReTRIACt study will complement the already robust and existing data set we have from the likes of EMC Survival Study and other clinical studies with tiratricol. Based on comments from the FDA, the statistical analysis plan of the ReTRIACt study was revised and the study was closed. A primary endpoint 1 evaluating the rate of change in serum T3 during the randomized treatment period 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 analysis plan was submitted to the FDA before database lock and the unblinding of the ReTRIACt study. Now let's turn to the results in the study. What you see depicted to the left is the ranking of patients according to their change in T3 during the 30-day randomized treatment period. Displayed with the dashed line is the line of no change from baseline. Hence, the patients with the largest increase of T3 during the randomized treatment period is in the top of the figure and the patients with the largest decrease is at the bottom of the figure. And what we have observed is exactly what we expected. When withdrawing tiratricol, the T3 levels in all placebo patients increase between 0.49 to 2.08 nanomole per liter. In the tiratricol group, not much happens when they continue the tiratricol treatment with the change in T3 ranging between negative 0.24 to 0.40 nanomole per liter. In fact, there is a complete separation of the 2 groups with all 8 patients randomized to placebo having a larger increase in serum T3 during the randomized treatment period than all 7 patients randomized to continue tiratricol. So what does this mean for the results of the primary endpoint? On the right-hand side, the upper table shows the results for primary endpoint 1, the rate of change in serum T3 during the randomized treatment period. For the placebo group, the rate of change was 1.590. This means once tiratricol is withdrawn, there is a 59% increase in T3 over 30 days, whereas there was essentially no change for the tiratricol group over the 30 days. Comparing the 2 groups, as per the definition of the primary endpoint 1 in the statistical analysis plan, 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. On the right-hand side, in the lower table, you can see the results for primary endpoint 2. 4 patients randomized to placebo met the rescue criteria. No patients randomized to tiratricol met the T3 rescue criteria. One patient randomized to tiratricol discontinued during the randomized treatment period. Discontinuation was not drug related. The patient is marked with D in the left-hand figure, as you can see. Also experienced essentially no change in T3 during the randomized treatment period. However, per the statistical analysis plan, this tiratricol patient was still imputed as a rescue for the analysis of primary endpoint.2, leading to a 4 versus 1 comparison for primary endpoint.2, which is not statistically significant. However, the sensitivity analysis below only considered the observed cases of rescue leads to a comparison of 4 patients meeting the rescue criteria on placebo versus 0 patients randomized to tiratricol with a p-value of 0.070. Furthermore, after the randomized treatment period was completed, patients randomized to placebo reinitiated tiratricol treatment, and they all showed as expected, a decrease in serum T3 levels in the so-called follow-up period. So to conclude, we are very, very pleased with the positive outcome of the ReTRIACt study. We now plan to submit an NDA for Emcitate with the current clinically available data. We have a very robust data set for such an ultra-rare genetic disorder, including data from Triac Trial I with 46 patients, Triac Trial II with 22 patients, now the ReTRIACt study with 15 patients, the EMC cohort study with 67 patients, also the EMC survival study with over 600 patients, by far the largest data set ever generated in this disease condition, together with the U.S. expanded access program with around 30 patients today. Our plan, as previously mentioned, is to commence a rolling submission in December 2025, targeting a complete NDA submission in early 2026 and an anticipated completion of the FDA review process in 2026 quarter 3. So with that, I would like to open up for questions. Operator, please go ahead.

The next question comes from Oscar Haffen Lamm from Stifel.

How do you expect the FDA to weigh the primary endpoint 1 versus the primary endpoint 2 of the study when they look at the data package as a whole?

Thank you, Oscar, and thank you for the question. And I think here, we need to take a couple of steps back. And I'm sure you appreciate that we received a granting of a breakthrough designation during the summer where the agency recognized the substantial mortality benefit seen in the survival data. Subsequently, we, of course, interacted with the FDA through a pre-NDA meeting in October to discuss the clinical content in the upcoming NDA and to get their view actually on the ReTRIACt study, given this new situation and then acknowledging the value of the survival data. As a result of that interaction, it was agreed to stop the ReTRIACt study immediately. And subsequently, we modified the statistical analysis plan because the ReTRIACt study is now seen as complementary additional evidence to the other clinical data generated. So all in all, positive.

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

Congratulations again on the update. I was wondering, do you already have a sales force in place in the U.S. And if not, how much do you think this will [indiscernible] for spending?