Arrowhead to submit NDA after pivotal rare disease trial meets all key endpoints

Arrowhead Pharmaceuticals is preparing to file a New Drug Application (NDA) to the FDA following the release of full data from a key Phase III trial of its lead drug candidate, plozasiran. The Palisade study (NCT05089084) successfully met its primary endpoint, showing significant reductions in fasting triglyceride levels. It also achieved key secondary endpoints, including decreases in apolipoprotein C-III (APOC3) and the incidence of acute pancreatitis (AP).

The trial focused on patients with familial chylomicronemia syndrome (FCS), a rare genetic condition with no current approved treatment in the US. Participants received either 25mg or 50mg doses of plozasiran or a placebo every three months. After ten months, the median triglyceride reduction was -80% in the 25mg group, -78% in the 50mg group, and -17% in the placebo group. APOC3 levels dropped by 93%-96% in the treatment groups.

Arrowhead aims to file the NDA by the end of 2024 and pursue further global regulatory approvals.