Emcitate® a first-in-class drug candidate, is being developed for the treatment of MCT8 deficiency, a rare inborn error of thyroid hormone signaling. A first clinical trial in patients suffering from MCT8 deficiency of all ages has been concluded with significant and clinically relevant results. A pivotal clinical trial looking at early intervention in very young affected children is about to start later this year. Emcitate® has been granted Orphan Drug Designation in the US and EU.
Aladote® a first-in-class drug candidate, is being developed to reduce the risk of acute liver injury associated with acetaminophen/paracetamol poisoning. A proof of principle study has been successfully completed and the design of the next study is being finalised. Aladote® has been granted Orphan Drug Designation in the US. Design of pivotal Phase IIb/III study for Aladote® finalized after completed interactions with FDA, EMA and MHRA