SEP 28 2026
Egetis Therapeutics — Emcitate (tiratricol) $EGTX
for MCT8 deficiency
Accepted MAR 27 2026.
About
MCT8 deficiency is a rare inherited disorder that severely disrupts brain development, leading to profound intellectual disability and major problems with speech and movement. Emcitate (tiratricol) is a thyroid hormone analog designed to bypass the body's defective transport system and deliver thyroid hormone activity directly to cells in the brain. This approach could potentially address the core neurological deficits of the condition, offering a new strategy to support development.
Pivotal trial
This is a double-blind, randomized phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. Male participants, from 4 years of age (at randomization) and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching the rescue criterion (serum total triiodothyronine \[T3\] \> upper limit of normal \[ULN\] of the participant's normal range, for a ...
Source: ClinicalTrials.gov