Arrowhead Pharmaceuticals, Inc.（ARWR）のニュースとイベントARWRArrowhead Pharmaceuticals, Inc.

Arrowhead Pharmaceuticals Announces Interim Results of ARO-MAPT Clinical Trial

Arrowhead Pharmaceuticals announced interim results from an ongoing Phase 1/2a study of ARO-MAPT, the company's investigational RNA interference, RNAi, therapeutic being developed as a potential treatment for Alzheimer's disease and other tauopathies. These results represent the first successful clinical validation of a platform to deliver siRNA across the blood-brain barrier and achieve substantial target gene knockdown in the brain. In healthy volunteers, subcutaneous administration of ARO-MAPT achieved deep and durable reductions in cerebrospinal fluid, CSF, total tau, with a mean reduction of up to 73% at Day 105 and a maximum reduction of 85%. Interim Healthy Volunteer Results: CSF total tau: ARO-MAPT administered at 2.1 mg/kg and 6.4 mg/kg on Days 1, 8, and 15 achieved mean reductions of 40% and 69%, respectively, at Day 105. ARO-MAPT administered at 12.8 mg/kg on Days 1 and 15 achieved a mean reduction of 73% at Day 105 with a maximum reduction of 85%; Durability: Day 105 represents 90 days following the final dose, with the sustained reduction in CSF total tau supporting the potential for dosing once every three months or less frequently.

Arrowhead Pharmaceuticals' plozasiran Enters FDA STAR Program

Arrowhead Pharmaceuticals announced that the United States Food and Drug Administration, FDA, has granted plozasiran entry in the Split Real Time Application Review, STAR, pilot program in connection with Arrowhead's upcoming supplemental new drug application, sNDA, for severe hypertriglyceridemias, HTG. The FDA STAR pilot program aims to shorten the time from the date of a complete sNDA submission to the action date for certain supplemental indications for already-approved drugs. Once accepted into the STAR program, a supplemental application qualifies for a 6-month Priority Review compared to a 10-month standard review. Arrowhead is on schedule to submit a sNDA for plozasiran in sHTG to the FDA by year-end 2026 and plans to seek regulatory approval with additional global regulatory authorities thereafter.

Arrowhead Pharmaceuticals Announces Interim Data for ARO-DIMER-PA Clinical Trial

Arrowhead Pharmaceuticals announced interim topline data from a Phase 1/2a clinical trial of ARO-DIMER-PA, the company's first investigational RNA interference, RNAi, therapeutic dimer being developed as a potential treatment for atherosclerotic cardiovascular disease, ASCVD, due to mixed hyperlipidemia. In participants with mixed hyperlipidemia, ARO-DIMER-PA single doses achieved dose-dependent mean maximal reductions in serum PCSK9 of 72% and APOC3 of 88%; Silencing of both the PCSK9 and APOC3 genes led to favorable changes in lipids and lipoproteins including the following mean maximal reductions: LDL-C reduction of 54%, Triglyceride reduction of 73%, Non-high density lipoprotein cholesterol reduction of 61%, Apolipoprotein B reduction of 50%.