Metabolic dysfunction-associated steatotic liver disease (MASLD) covers a series of pathogenic conditions including steatosis, inflammation and fibrosis with limited therapeutic options to date. Recent findings have shown upregulation of hepatic murine double minute 2 (MDM2) in patients with MASLD. Additionally, genetic deletion of hepatic MDM2 and its pharmacological inhibition were seen to improve steatosis and fibrosis in MASLD murine models.

As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.