PDUFA in a week. Pos is 90%. In my opinion Still a 2x if they successfully commercialize in USA and good m&a candidate. https://genestreet.substack.com/p/mct8-deficiency-the-worst-of-both
The disease. MCT8 deficiency, also known as Allan–Herndon–Dudley syndrome, is a rare X-linked disorder (\~1 in 70,000). A broken transporter starves the brain of thyroid hormone while the rest of the body is flooded with it, because the normal feedback system fails. The result is lifelong disability and a median life expectancy in the mid-30s.
The drug. Emcitate (tiratricol) is the only approved treatment. It is a modified T3 that enters cells without the MCT8 transporter, so it still works when the normal route is broken, bringing thyroid hormone levels in the body back down and easing the peripheral thyrotoxicosis that drives many symptoms and early deaths.
The evidence. The T3-lowering effect is clear, consistent and durable: serum T3 fell \~60% into the normal range. The ReTRIACt withdrawal study confirmed causality — patients switched to placebo separated sharply from those kept on drug (p=0.034).
The limitation. Emcitate controls the overload outside the brain but cannot undo brain damage, which is largely set before birth. CHMP therefore limited the EU approval to peripheral thyrotoxicosis — not a safety concern, just an honest reflection of what the drug does.
