Arrowhead Pharmaceuticals initiates phase III PALISADE study of ARO-APOC3.

ARO-APOC3 is the company’s investigational RNA interference (RNAi) therapeutic that is designed to inhibit the production of apolipoprotein C3 (APOC3), a key regulator of triglyceride metabolism.

ARO-APOC3 is currently being investigated in multiple clinical studies, including the Phase III PALISADE study in patients with FCS, the Phase IIb SHASTA-2 study in patients with severe hypertriglyceridemia (SHTG), and the Phase IIb MUIR study in patients with mixed dyslipidemia.

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “Initiating the Phase III PALISADE study is an important milestone for the ARO-APOC3 program, for patients with FCS, and for Arrowhead. Patients with genetically defined FCS, and those who are phenotypically similar, tend to have extremely high triglycerides, which can lead to an elevated risk of recurrent and painful bouts of pancreatitis. These patients currently have very limited treatment options. In the previously conducted Phase 1/II clinical study of ARO-APOC3 in 4 genetically confirmed FCS patients, ARO-APOC3 treatment led to a maximal mean reduction in APOC3 of up to 98%, a maximal median decrease in triglycerides of up to 91% and was generally well-tolerated. These changes are similar to those observed in 26 participants that had SHTG and similar baseline triglyceride levels but did not carry biallelic mutations consistent with FCS. These results were presented in November 2021 at the American Heart Association Scientific Sessions meeting1. We look forward to investigating ARO-APOC3 in the larger Phase III PALISADE study to further assess its efficacy and safety.”

Arrowhead is partnering with Ambry Genetics to provide no-cost genetic testing to patients who may be at risk of severe hypertriglyceridemia and FCS.