Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-MAPT for the Treatment of Alzheimer's Disease and Other Tauopathies

- ARO-MAPT utilizes the Targeted RNAi Molecule (TRiM™) platform designed for subcutaneous administration and systemic delivery to the CNS by crossing the blood-brain-barrier

- Study initiation further highlights Arrowhead's innovation and leadership in the delivery of siRNA

Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-MAPT, the company's investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies including Alzheimer's disease, a progressive neurodegenerative disease characterized by cognitive and functional decline. Alzheimer's disease is the most common cause of dementia, affecting an estimated 32 million people worldwide, and is part of a group of neurodegenerative diseases called tauopathies that are marked by the abnormal accumulation and formation of tau tangles in neurons.

ARO-MAPT is Arrowhead's first investigational RNAi-based therapy to utilize a new proprietary delivery system which, in preclinical studies, has achieved blood-brain-barrier penetration and deep knockdown of target genes across the central nervous system (CNS), including deep brain regions, after subcutaneous injection. This underscores Arrowhead's leadership in the delivery of siRNA to multiple tissues and cell types throughout the body utilizing its proprietary and differentiated Targeted RNAi Molecule (TRiM™) platform.

"Alzheimer's disease is the most common cause of dementia and is debilitating for patients and caregivers. Currently available disease-modifying treatments have focused on amyloid-beta plaque clearance but have shown limited clinical benefit. Tau related pathology may be a critical driver of neurodegeneration, and targeting tau is a promising strategy to potentially slow or stop cognitive and functional decline," said James Hamilton, M.D., Chief Medical Officer and Head of R&D at Arrowhead. "By silencing expression of the gene tha