Egetis Therapeutics’ Annual Report 2025 published

Summary by AI ^BETAClose X

Egetis Therapeutics AB has made its 2025 Annual Report available, detailing significant progress in its drug development pipeline. The company's lead candidate, Emcitate®, received European Commission approval in February 2025 for treating MCT8 deficiency and launched in Germany on May 1, 2025, with a rolling New Drug Application submitted to the FDA in the US on January 29, 2026. Tiratricol, the active ingredient in Emcitate®, holds Orphan Drug Designation in both the US and EU for MCT8 deficiency and resistance to thyroid hormone beta, and has received Breakthrough Therapy and Rare Pediatric Disease Designations from the FDA, potentially leading to a Priority Review Voucher. The development of Aladote® for paracetamol overdose has been paused, though it also holds Orphan Drug Designation.

Disclaimer*

Stockholm, Sweden, March 24, 2026 – Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX) Annual Report for 2025 is available (in Swedish) attached to this press release and at the Company’s website, www.egetis.com. An English translation of the Annual Report will be published shortly.

About Egetis Therapeutics

Egetis Therapeutics is an innovative and integrated pharmaceutical company, focusing on projects in late-stage development for commercialization for treatments of serious diseases with significant unmet medical needs in the orphan drug segment.

The Company’s lead drug candidate Emcitate® (tiratricol) is developed for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In February 2025 the European Commission approved Emcitate® as the first and only treatment for MCT8 deficiency in EU. Egetis initiated the launch of Emcitate® in Germany on May 1, 2025. Emcitate® (tiratricol) is not approved in the USA.

The Company completed a rolling New Drug Application (NDA) for Emcitate® (tiratricol) in the USA on January 29, 2026. The FDA is expected to confirm within 60 days that the NDA submission is complete. As a designated Fast Track and Breakthrough Therapy, Egetis has requested Priority Review, and if granted, the FDA review should be completed within six months following the 60-day filing review period.

Based on feedback from the FDA, the NDA for Emcitate® (tiratricol) for treatment of MCT8 deficiency will be based on currently available clinical data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program.

Tiratricol holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Tiratricol has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation (RPDD) by the FDA, which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval.

The drug candidate Aladote® (calmangafodipir) is a first in class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed. The design of a pivotal Phase IIb/III study (Albatross), with the purpose of applying for market approval in the US and Europe, has been finalized following interactions with the FDA, EMA and MHRA. The development program for Aladote® has been parked. Aladote® has been granted ODD in the US and in the EU.

Egetis Therapeutics is listed on the Nasdaq Stockholm main market (Nasdaq Stockholm: EGTX).
For more information, see www.egetis.com