Alzheimer’s Research  ·  Hawaii Memory & Alzheimer’s Research Unit  ·  Published in Nature 2026

University of Hawaii Professor & Researcher Publishes Buntanetap Study in Nature — A Novel Oral Therapy Targeting APP and Tau in Alzheimer’s Disease

npj Dementia, Nature  ·  April 2026  ·  Hawaii Memory & Alzheimer’s Research Unit

A landmark study co-authored by University of Hawaii Professor and researcher Dr. Kore Kai Liow, MD has been published in npj Dementia (Nature), reporting results from a Phase 2/3 randomized double-blind trial of Buntanetap — a novel oral small RNA targeting molecule — in patients with mild to moderate Alzheimer’s disease. The findings show meaningful cognitive benefits in amyloid-positive patients, with a promising safety profile, and have set the stage for a Phase 3 trial now underway here in Hawaii.

What Is Buntanetap?

Buntanetap is an orally available small molecule that works at the RNA level to inhibit the translation of multiple neurotoxic aggregating proteins — most critically Amyloid Precursor Protein (APP) and Tau, the two proteins central to Alzheimer’s disease pathology. Unlike many Alzheimer’s therapies that target a single protein after it has already aggregated, Buntanetap acts upstream to reduce the production of these harmful proteins before they accumulate.

This multi-target mechanism distinguishes Buntanetap from other disease-modifying approaches and may explain its activity across both Alzheimer’s and Parkinson’s disease, where overlapping protein aggregation pathways drive neurodegeneration.

The Phase 2/3 Study

This 3-month randomized, double-blind, dose-ranging study (NCT05686044) enrolled 351 patients with mild to moderate Alzheimer’s disease, equally randomized across four arms:

Key Findings

The study did not meet its co-primary endpoints (ADAS-Cog-11 and ADCS-CGIC) in the overall population — a finding the investigators attribute to the fact that 40% of enrolled participants lacked amyloid pathology, diluting the treatment signal in the intent-to-treat analysis.

Prior smaller studies in early Alzheimer’s disease showed trends toward cognitive improvement, and a Phase 2/3 study in early Parkinson’s disease demonstrated improved cognitive function in PD patients — reinforcing the drug’s multi-disease potential and the consistency of its mechanism across neurodegenerative conditions.

Hawaii Memory & Alzheimer’s Research Unit — Hawaii Pacific Neuroscience

Phase 3 Trial Now Underway in Hawaii

Building on these findings, a Phase 3 trial of Buntanetap is currently enrolling patients at the Hawaii Memory Disorders Center and Alzheimer’s Research Unit at Hawaii Pacific Neuroscience. This Phase 3 study is specifically designed to enroll amyloid biomarker-confirmed patients — directly addressing the limitation identified in the Phase 2/3 results and positioning the trial to deliver a definitive efficacy signal.

This is an extraordinary opportunity for Hawaii residents living with early Alzheimer’s disease to access a potentially disease-modifying oral therapy at the cutting edge of neurological research — without leaving the islands.

Hawaii Pacific Neuroscience continues to advance access to world-class neurological research for Hawaii’s communities — ensuring that patients across the islands can participate in the trials shaping the future of Alzheimer’s care.

Hawaii Memory & Alzheimer’s Research Unit  ·  Hawaii Pacific Neuroscience  ·  hawaiineuroscience.com