ESPEYB17 14. The Year in Science and Medicine (1) (16 abstracts)
14.9. Resmetirom (MGL-3196) for the treatment of non-alcoholic steatohepatitis: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
Harrison SA , Bashir MR , Guy CD , Zhou R , Moylan CA , Frias JP , Alkhouri N , Bansal MB , Baum S , Neuschwander-Tetri BA , Taub R & Moussa SE
To read the full abstract: Lancet. 2019 Nov 30;394(10213):2012-2024. doi: 10.1016/S0140-6736(19)32517-6.
Resmetirom (MGL-3196) is a liver-directed, orally active, selective thyroid hormone receptor-β agonist. This 36-week long randomised, placebo-controlled trial in 348 US adults with biopsy confirmed non-alcoholic steatohepatitis (fibrosis stages 1–3) shows that Resmetirom reduced hepatic fat by -37.3% (compared to -8.5% on placebo). Adverse events were mostly mild or moderate and were balanced between groups.
The effects of thyroid hormones on reducing LDL cholesterol (by suppressing its generation by the liver) are long established. Notably, before the availability of thyroid hormone assays, Lawson Wilkins used changes in plasma cholesterol levels to monitor treatment of hypothyroidism with thyroid extract. Indeed, Resmetirom was originally designed to treat dyslipidamia and reduces circulating LDL cholesterol levels by ~30%. Furthermore, it shows high selectivity for thyroid hormone receptor-β and has no impact on the central thyroid axis.
These findings are exciting as there are currently no effective pharmacological treatments that effectively prevent the progression to fibrosis in non-alcoholic steatohepatitis. Resmetirom is currently in large scale phase 3 trial with this histological endpoint, and another promising option in late stage trials is obeticholic acid, a semi-synthetic bile acid analogue.
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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