Egetis Therapeutics

• FDA awarded tiratricol Breakthrough Therapy Designation (BTD) in July 2025, based on the Agency’s review of Egetis’ analysis of the survival data set from the international real-world cohort study by the Erasmus University Medical Center
• There are 15 evaluable patients in the ReTRIACt study
• In light of the above, Egetis has submitted a pre-NDA meeting request to the FDA to discuss the contents and timing of the US NDA submission for tiratricol, including the role and position of the ReTRIACt study
• The Company plans to initiate the submission of the NDA for tiratricol to the FDA during 2025

Stockholm, Sweden, August 18, 2025. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that it has submitted a pre-NDA meeting (Type B) request to the US Food and Drug Administration (FDA) to discuss the contents and timing, including the role and position of the ReTRIACt study, of its New Drug Application (NDA) for tiratricol in the US, in light of the Breakthrough Therapy Designation (BTD) awarded on July 15, 2025, by the FDA. A BTD is awarded to programs that have shown preliminary evidence of clinical benefit that results in a meaningful improvement of key signs and symptoms compared to existing therapies.

Egetis today also provided an update on the recruitment status of the ReTRIACt study (clinicaltrials.gov identifier NCT05579327), evaluating withdrawal of tiratricol in patients with MCT8 deficiency. There are 15 evaluable patients in the trial. The Company plans to initiate the submission of the NDA to the FDA during 2025.

Nicklas Westerholm, CEO of Egetis, commented: “The clinically meaningful evidence available from treating patients with MCT8 deficiency with tiratricol was recognized by the FDA Breakthrough Therapy Designation (BTD) awarded in July this year (link to Press Release). The BTD was granted based on the Agency’s review of Egetis’ detailed analysis of the entire survival data set from the international real-world cohort study by the Erasmus University Medical Center (for preliminary results, see Abstract in Ref. 1), demonstrating a significant and substantial improvement in survival in tiratricol treated vs untreated patients. Receiving a BTD this late in a clinical development program is very encouraging for the forthcoming NDA process, as these designations are typically awarded at an early stage in development.

“We have submitted a pre-NDA meeting request and look forward to discussing the NDA submission for tiratricol with the Agency, to bring this potential treatment to patients also in the United States, as soon as possible. Tiratricol treatment for MCT8-deficiency is already included in clinical guidelines by the European Thyroid Association (Ref. 2) and Emcitate® (tiratricol) was approved for the treatment for MCT8 deficiency in the European Union in February 2025.”

1: van der Most, F. et al. 2024 https://www.endocrine-abstracts.org/ea/0101/ea0101op-03-04

2: Persani, L. et al. 2024 https://etj.bioscientifica.com/view/journals/etj/13/4/ETJ-24-0125.xml