Pipeline

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

Clinical Trials

Our lead product candidate, Emcitate, is an oral small molecule that has been shown to restore dysfunctional thyroid hormone signalling in several situations.

A Phase IIb clinical trial (Triac Trial I; NCT02060474) in MCT8 deficiency has been completed which showed significant and clinically relevant treatment effects on key aspects of the disease. The Triac Trial I results have been published in The Lancet D&E in 2019.

In October 2021, strong data from long-term treatment in patients with MCT8 deficiency up to 6 years, with Emcitate was published in the Journal of Clinical Endocrinology & Metabolism. The data comes from an investigator-initiated real-life cohort study at 33 sites conducted by the Erasmus Medical Center, Rotterdam, The Netherlands, where the efficacy and safety of Emcitate was investigated in 67 patients with MCT8 deficiency.

Emcitate is currently being investigated in the Triac Trial II (NCT02396459), 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.

The NDA to the US FDA is targeted for mid-2023 under the Fast Track Designation granted by the FDA. As previously communicated, the NDA requires a small, randomized, placebo-controlled trial with 16 patients, and up to 30 days treatment duration with either Emcitate or placebo, to be conducted to verify the results on thyroid hormone T3 levels seen in previous clinical trials and publications. The design of this ReTRIACt trial has been agreed with the FDA and can be found on clinicaltrials.gov under identifier NCT05579327.

 

 

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