Egetis continues to increase disease awareness about MCT8 deficiency

Stockholm, Sweden — Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) announced that the Company, as part of its initiatives to increase disease awareness about MCT8 deficiency, has participated and informed about MCT8 deficiency, at the following medical conferences during the first quarter of 2023:

British Paediatric Neurology Association (BPNA) Annual Conference
January 21-25, 2023 in Edinburgh, Scotland
11th International Meeting of Paediatric Endocrinology
March 4-7, 2023 in Buenos Aires, Argentina
Arbeitsgemeinschaft für pädiatrische Stoffwechselstörungen (APS) Jahrestagung
March 7-10, 2023 in Kassel, Germany

During the second quarter 2023 the Company will participate at the following medical conferences:

American Academy of Neurology (AAN) Annual Meeting
April 22-27, 2023 in Boston, MA, USA
American Association for Clinical Endocrinology (AACE) Annual Meeting
May 4-6, 2023 in Seattle, WA, USA
US Pediatric Endocrine Society (PES)
May 5-8, 2023 in San Diego, CA, USA
Local Control of Thyroid Hormone Action (LOCOTACT)
June 7-10, 2023 in Essen, Germany
Endocrine Society (ENDO)
June 15-18, 2023 in Chicago, IL, USA
15th Congress of the European Paediatric Neurology Society (EPNS)
June 20-24, 2023 in Prague, Czech Republic

Nicklas Westerholm, CEO of Egetis, commented: “It’s important to increase disease awareness about MCT8 deficiency among specialized doctors who might encounter patients suffering from this rare and debilitating disease, as we approach submissions for marketing authorizations in Europe and the USA in 2023 for Emcitate.”

Egetis has also produced a new video about the daily life of a family living with a patient suffering from MCT8 deficiency. To watch the video, please follow this link ( To watch a video about the background to MCT8 deficiency and how the disease affects the body, please follow this link (

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 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 T3 levels and secondary clinical endpoints. As a result of fruitful regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in the second quarter of 2023 based on existing clinical data.
In the US, after discussions with the FDA, Egetis will conduct a small randomized, placebo-controlled 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 the second half of 2023 under the Fast-Track Designation granted by FDA.
Emcitate is currently being investigated in the Triac Trial II, a Phase II/III study in very young MCT8 deficiency patients (<30 months of age) exploring potential disease modifying effects of early intervention from a neurocognitive and neurodevelopmental perspective. The recruitment target was achieved in the second quarter 2022 and 22 patients have been included in the study. Results are expected mid 2024 and are expected to be submitted post-approval to regulatory authorities.
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.
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 during 2023. 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


Nicklas Westerholm, CEO
+46 (0) 733 542 062
[email protected]

Karl Hård, Head of Investor Relations & Communications
+46 (0) 733 011 944
[email protected]