• Positive topline results demonstrate a statistically significant (p=0.034) difference in the rate of change in serum T3 in patients randomized to withdrawal (placebo) vs. in patients continuing therapy (tiratricol)
• The ReTRIACt study is the last study to be reported for Emcitate® (tiratricol) in MCT8 deficiency and complement the EMC Survival Study and other clinical data for the New Drug Application (NDA), as discussed with the FDA in the October 2025 pre-NDA meeting
• A rolling NDA submission is planned to commence in December 2025, targeting a complete NDA submission in early 2026 and anticipated completion of the FDA review process in Q3 2026, if Priority Review is granted
• Conference call for analysts and investors to be held today at 10:00am CET. Further details below

Stockholm, Sweden, November 14, 2025. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced positive topline results from the ReTRIACt study of Emcitate® (tiratricol) in patients with MCT8 deficiency.

The ReTRIACt study (clinicaltrials.gov identifier NCT05579327) is a double-blind, multicenter, placebo-controlled, randomized, withdrawal study in patients with MCT8 deficiency. Males 4 years of age and above who demonstrated stable maintenance treatment with tiratricol, were randomized to continue tiratricol or receive placebo for 30 days or until reaching the rescue criterion (serum total triiodothyronine [T3] > upper limit of normal [ULN]).

As a result of the pre-NDA meeting Egetis held with the FDA in October 2025, on the back of the Breakthrough Therapy Designation granted in July 2025, FDA agreed that in the NDA submission, data from the ReTRIACt study will complement the existing data from the EMC Survival Study and other clinical components. Furthermore, based on comments from the FDA, the Statistical Analysis Plan (SAP) of the ReTRIACt study was revised, and the study was closed. A Primary Endpoint 1 (PE1), evaluating rate of change in serum total T3 during the double-blind Randomized Treatment Period, was added to the previously described Primary Endpoint 2 (PE2), based on the T3 rescue criterion. Type I Error is controlled via alpha recycling, testing PE1 first, and if PE1 is met, PE2 will then be tested.

The Primary Endpoint 1 (PE1), rate of change in serum T3 during the Randomized Treatment Period, showed a statistically significant difference between placebo and tiratricol (ratio of T3 rate of change (placebo/tiratricol): 1.494 [95% CI: 1.035; 2.155]; p=0.034). The results show a complete separation of the two groups with all 8 patients randomized to placebo having an increase in serum T3 during the randomized treatment period (range: 0.49 - 2.08 nmol/L), larger than all 7 patients randomized to continue tiratricol (range: -0.24 - 0.40 nmol/L). In the placebo group, 4 patients met the rescue criterion during the randomized treatment period compared to 0 patients randomized to tiratricol (p=0.070 on observed cases of rescue). One patient randomized to tiratricol discontinued (not drug related) during the randomized treatment period, which per the SAP was imputed as rescue in PE2, leading to a 4 (placebo) versus 1 (tiratricol) comparison for PE2 (p=0.182). All patients randomized to placebo showed an expected decrease in serum T3 levels following reinitiation of tiratricol in the follow-up period.

In line with the outcome of the pre-NDA meeting with the FDA in October 2025, Egetis plans to submit an NDA for Emcitate® (tiratricol) with currently available clinical data in patients with MCT8 deficiency, including data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program. The rolling submission will commence with the initial submission in December 2025, targeting a complete NDA submission in early 2026 and anticipated completion of the FDA review process in Q3 2026, if Priority Review is granted. As Emcitate® (tiratricol) has Breakthrough Therapy Designation, Egetis will request a priority review. Emcitate® (tiratricol) also holds Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation in the US. Emcitate® (tiratricol) was approved in the EU in February 2025 and launched in Germany in May 2025. Emcitate® (tiratricol) is not approved for use in the United States.

Nicklas Westerholm, CEO of Egetis, commented: “We are delighted with the positive topline results for the ReTRIACt study and plan to initiate a rolling NDA submission in December 2025, targeting a complete NDA submission in early 2026. We look forward to bringing this potential treatment to patients in the United States as soon as possible. Tiratricol treatment for MCT8-deficiency is already included in clinical guidelines by the European Thyroid Association and Emcitate® (tiratricol) was approved for the treatment for MCT8 deficiency in the European Union in February 2025.

“We are grateful to the patients, their families, and the investigators who have participated in the ReTRIACt study.”

The results will be presented at a forthcoming scientific meeting, submitted to a peer-reviewed medical journal and shared with regulatory authorities.

Information about webcast
If you wish to participate via webcast please use the link below. Via the webcast you are able to ask written questions.
https://egetis.events.inderes.com/webcast-nov-14-2025
 
If you wish to participate via teleconference please register on the link below. After registration you will be provided phone numbers and a conference ID to access the conference. You can ask questions verbally via the teleconference. 
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