Orphan drug candidate restoring thyroid hormone signalling in cells with defect MCT8 transporter

Emcitate®- treatment of MCT8 deficiency (Allan-Herndon-Dudley Syndrome)

Emcitate is under development for the treatment of patients with 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 fruitful regulatory interaction Egetis submitted a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in October 2023.

In the US, after discussions with the FDA, Egetis is conducting a small randomized, placebo-controlled study (ReTRIACt) in 16 patients to verify the results on T3 levels seen in previous clinical trials and publications. Topline results from the ReTRIACt study are expected in the first half of 2024. Egetis intends to initiate the rolling submission of a new drug application (NDA) in the US for Emcitate mid 2024 under the Fast-Track Designation granted by FDA.

Emcitate is currently also being investigated in Triac Trial II, a Phase II 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.

Emcitate holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH- β) in the US and the EU. Emcitate has been granted Rare Pediatric Disease Designation (RPD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval.




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Phone: +46 8 679 72 10

E-mail: info@egetis.com