Press release

Egetis to participate at medical conferences to increase disease awareness about MCT8 deficiency

Stockholm, Sweden, September 12, 2023. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) today announced that the Company, as part of its initiatives to increase disease awareness about MCT8 deficiency, will participate at the following medical conferences during the remainder of 2023:

45th Annual Meeting of the ETA (European Thyroid Association)
September 10-13, 2023 in Milan, Italy

60th ESPE Annual Meeting (European Society of Pediatric Endocrinology)
September 21-23, 2023 in The Hague, The Netherlands

92nd Annual Meeting of the American Thyroid Association
September 27- October 1, 2023 in Washington DC, USA

CNS – 52nd Child Neurology Society Annual Meeting
October 4-7, 2023 in Vancouver, BC, Canada

Recently, the Company also presented a poster at the
SSIEM - Society for the Study of Inborn Errors of Metabolism
August 29- September 1, 2023 in Jerusalem, Israel
The poster entitled “Landscape of genetic testing for monocarboxylate transporter 8 (MCT8) deficiency” can be found here.

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 is under development for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, a highly debilitating rare disease with no available treatment. In previous studies (Triac Trial I and a long-term real-life study) Emcitate has shown highly significant and clinically relevant results on serum thyroid hormone T3 levels and secondary clinical endpoints. As a result of regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) on October 9, 2023, based on existing clinical data.
After a dialogue with the FDA, Egetis is conducting a small randomized, placebo-controlled pivotal study in 16 patients to verify the results on T3 levels seen in previous clinical trials and publications. Egetis intends to submit a new drug application (NDA) in the US for Emcitate in mid 2024 under the Fast-Track Designation granted by FDA.
Emcitate holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH-beta) in the US and the EU. MCT8 deficiency and RTH-beta are two distinct indications, with no overlap in patient populations. Emcitate has been granted Rare Pediatric Disease Designation (RPDD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval. This voucher can be transferred or sold to another sponsor.
The drug candidate Aladote 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 and the design of the upcoming pivotal Phase Iib/III study with the purpose of applying for market approval in the US and Europe for Aladote has been finalized after completed interactions with FDA, EMA and MHRA and study start is planned after Emcitate submissions have been completed. Aladote has been granted ODD in the US and in the EU.
Egetis Therapeutics (STO: EGTX) is listed on the Nasdaq Stockholm main market. For more information, see www.egetis.com