Arrowhead Pharmaceuticals Announces ARO-MAPT Interim Phase 1/2a Clinical Data Demonstrating Deep and Durable Tau Reductions in Healthy Volunteers
Business Wire•14/10/2026•07:30 ET
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Key Highlights
- ➤ARO-MAPT cut CSF total tau 73% at Day 105, maximum individual reduction reached 85%
- ➤2.1 mg/kg and 6.4 mg/kg doses produced mean tau reductions of 40% and 69%
- ➤Day 105 results came 90 days after final dose, supporting potential quarterly dosing
- ➤No serious adverse events, deaths, discontinuations, persistent anemia, or neurobehavioral events reported
- ➤Arrowhead expects early Alzheimer’s disease data updates in 2027 and initiated regulatory discussions for additional studies
Expert Statements
Christopher Anzalone, President and Chief Executive Officer of Arrowhead Pharmaceuticals
“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, representing an important step toward developing a potential treatment for Alzheimer’s disease and other primary tauopathies.”
Christopher Anzalone, President and Chief Executive Officer of Arrowhead Pharmaceuticals
“Abnormal tau accumulation is a key feature of multiple devastating diseases, and we believe reducing tau production has the potential to offer a meaningful new approach for patients.”
Christopher Anzalone, President and Chief Executive Officer of Arrowhead Pharmaceuticals
“Equally important, these results provide clinical validation of our proprietary Targeted RNAi Molecule (TRiM(TM)) delivery platform to potentially address multiple diseases in the central nervous system (CNS).”
Christopher Anzalone, President and Chief Executive Officer of Arrowhead Pharmaceuticals
“This major scientific breakthrough further solidifies Arrowhead’s position as the leading innovator in siRNA therapies.”
Arrowhead Pharmaceuticals Announces ARO-MAPT Interim Phase 1/2a Clinical Data Demonstrating Deep and Durable Tau Reductions in Healthy Volunteers
* Arrowhead’s TRiM(™) platform achieves clinical validation for delivery of siRNA that crosses the blood-brain barrier after subcutaneous injection
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