Arrowhead Reports Positive Phase 1/2a Data For Alzheimer's Candidate ARO-MAPT

(RTTNews) - Arrowhead Pharmaceuticals, Inc. (ARWR) announced positive interim results from an ongoing Phase 1/2a study of ARO-MAPT, the company's investigational RNA interference (RNAi) therapeutic for Alzheimer's disease and other tauopathies.

According to the company, in healthy volunteers, subcutaneous ARO-MAPT administration achieved 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%, supporting the potential for once-quarterly dosing.

The abnormal buildup of tau protein is implicated in several neurodegenerative diseases, including Alzheimer's disease, MAPT-related frontotemporal dementia, and progressive supranuclear palsy.

ARO-MAPT is designed to reduce MAPT gene expression in the central nervous system, lowering production of all six tau protein isoforms.

Trial details

AROMAPT-SC-1001 is a phase 1/2a placebo-controlled dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MAPT in approximately 64 normal healthy volunteers (NHV) and up to 48 subjects with early Alzheimer's disease (AD).

In Part 1a, healthy volunteers will receive one to three subcutaneous doses of ARO-MAPT or placebo. In Parts 1b and 2a, healthy volunteers and patients with early Alzheimer's disease will receive up to five subcutaneous doses, including an initial two to three doses followed by one to two additional doses.

Key Trial Findings At day 105, 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.

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%.

Sustained reductions in CSF total tau through day 105, measured 90 days after the final dose, support the potential for once-quarterly or less frequent dosing, according to the company.

ARO-MAPT was generally well tolerated. Most treatment-emergent adverse events (TEAEs) were Grade 1 or mild.

The most common reported TEAEs were injection-site reactions, vessel puncture-site bruising, upper respiratory tract infection, back pain, and headache.

CEO Quote

"These early clinical data are exciting because they demonstrate that ARO-MAPT can achieve deep and durable reductions of tau expression in the brain following a simple subcutaneous administration," said Christopher Anzalone, President and Chief Executive Officer of Arrowhead.

Road Ahead Healthy volunteer enrollment is complete, with dosing ongoing and enrollment continuing in early Alzheimer's disease cohorts.

Data updates are expected in 2027, while preparations are underway for studies in MAPT-associated frontotemporal dementia and progressive supranuclear palsy.

Stock Performance

ARWR has traded between $36.02 and $95.49 over the past 52 weeks. The stock closed Tuesday's trade at $58.13.

ARWR is currently up 1.48% at $58.99.

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