In the phase 3 PALISADE trial, the primary endpoint of lowering triglycerides was met with the RNAi-based therapy plozasiran in patients with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS).

Treatment with plozasiran significantly reduced triglycerides up to 80% and APOC3 up to 94% at 10 months in patients with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome (FCS) in the phase 3 PALISADE trial. Moreover, treatment with plozasiran significantly reduced the incidence of acute pancreatitis compared with placebo.

The primary endpoint of the PALISADE trial was placebo adjusted median change in triglycerides at 10 months. A total of 75 subjects were randomized to receive 25 mg plozasiran, 50 mg plozasiran or matching placebo once every three months. At month 10, median triglyceride reduction of 80% and 78% were observed with quarterly doses of 25 and 50 mg plozasiran, respectively, and a maximal reduction of 98% was seen. In the placebo group, median triglyceride reduction was 17%. Median reductions in APOC3 at month 10 were 88% and 94% with 25 and 50 mg plozasiran, respectively.

All key secondary endpoints were met for plozasiran and demonstrated significance compared with the placebo group.

The safety profile in the PALISADE study was favorable. And the number of subjects with AEs was similar in the plozasiran and placebo groups. Most common AEs reported were abdominal pain, COVID-19, nasopharyngitis, headache and nausea.

Source: press release Arrowhead Pharmaceuticals, June 3, 2024.