Our lead product candidate, Emcitate, is an oral small molecule that has been shown to restore dysfunctionsl thyroid hormone signalling in several situations. Studies have shown that Emcitate can enter into cells regardless of a functioning MCT8 transporter. We are developing Emcitate for the treatment of MCT8 deficiency, a syndrome resulting from mutations in the gene encoding for the MCT8 transporter, resulting in endogenous thyroid hormone being unable to pass across the cell membrane of many cell types, including the blood brain barrier. In several animal models of the disease, treatment with Emcitate has been shown to restore a normal phenotype if initiated very early in life.
As part of our collaboration with Erasmus Medical Center in Rotterdam, we have concluded a first clinical trial of Emcitate in MCT8 deficiency. In the study, 46 patients with MCT8 deficiency from 9 countries across the world were treated with Emcitate for one year. Treatment was found to be safe and well tolerated and was able to rapidly restore and maintain normal peripheral thyroid hormone levels and alleviate key symptoms of thyrotoxicosis. The full results of this clinical trial were published in the Lancet Diabetes & Endocrinology in August 2019.
In parallel, we are preparing for a second clinical trial, looking at early intervention in very young patients with MCT8 deficiency (<36 months of age). The study will have centres in both the EU and US and is expected to start recruiting in the second half of 2020.