FCS  is a rare metabolic disease that prevents the body from digesting fats.

Related: What Arrowhead Pharmaceutical’s Interim Data Reveals About A RNAi Therapeutic For Rare Cholesterol Disorder

The study met the primary endpoint of lowering triglycerides and all key secondary endpoints, including reducing the incidence of acute pancreatitis compared to placebo.

Plozasiran is the company’s first investigational RNAi-based therapy to show clinical efficacy in a Phase 3 study.

The primary endpoint for the PALISADE study was placebo-adjusted median change in triglycerides at Month 10.

At that time point, patients treated with quarterly doses of 25 and 50 mg plozasiran achieved median triglyceride reductions of -80% and -78%, respectively, with a maximal reduction of -98%.

At month 12, patients treated with 25 and 50 mg plozasiran achieved median triglyceride reductions of -78% and -73%, respectively, with a maximal reduction of -99%.

These compared with median triglyceride reductions in placebo-treated patients of -17% at month 10 and -7% at month 12.

Mean reductions in Apolipoprotein C-III at month 10 were -88% and -94% at 25 and 50 mg plozasiran, respectively.

Plozasiran demonstrated a favorable safety profile. The most common AEs reported were abdominal pain, COVID-19, nasopharyngitis, headache, and nausea.

Arrowhead plans to highlight recent data for its cardiometabolic pipeline at its June 25, 2024, Cardiometabolic event as part of the 2024 Summer Series of R&D Webinars.

The company also plans to present full Phase 3 PALISADE study results at upcoming medical congresses.

Price Action: ARWR shares are up 7.58% to $24.69 at the last check on Monday.