Fierce Biotech April 7, 2024
Ionis previously touted that olezarsen was 100% effective in a rare metabolic disorder after a phase 3 readout. Now, the full dataset is slightly denting that statistic while leaving plenty of room for a planned regulatory filing later this year.
Olezarsen is being tested in adults with familial chylomicronemia syndrome (FCS). An early late-stage readout in September 2023 linked a high 80 mg dose of the investigational ligand-conjugated antisense medicine to a significant reduction in triglyceride levels and a 100% drop in acute pancreatitis (AP) events compared to 11 for placebo, but didn’t provide a deeper look at the data. AP events are a serious complication of FCS in which the pancreas suddenly becomes inflamed.
Now, Ionis has shared the...
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