Aribio: "AR1001 Demonstrates Efficacy for CAA, Overcoming Limitations of Existing Injection Therapies"

On May 28, Aribio announced that the results of a preclinical study demonstrating the efficacy of its Alzheimer's disease drug candidate AR1001 in relation to cerebral amyloid angiopathy (CAA) have been published in the international journal Frontiers in Aging Neuroscience.

This study was jointly conducted by Aribio's neuroscience research team and researchers from Hallym University. In an Alzheimer's disease model accompanied by CAA, AR1001 was found to show significant improvements in cerebrovascular stabilization, inflammation suppression, and cognitive function recovery.

CAA is a condition that accompanies more than half of Alzheimer's patients, in which amyloid proteins accumulate on the walls of cerebral blood vessels, increasing the risk of vessel rupture or hemorrhage. Existing antibody injection therapies have a high incidence of severe side effects such as cerebral edema (ARIA-E) or hemorrhage (ARIA-H) in this patient group. For this reason, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have mandated black box warnings and regular MRI monitoring.

The research team administered AR1001 to animal models carrying the Alzheimer's risk gene (ApoE4) and confirmed the following: a reduction in inflammatory cytokines, increased expression of vascular neuroprotein (CLN-5), suppression of blood-brain barrier (BBB) leakage, and a decrease in amyloid deposition in the hippocampus along with recovery of cognitive function. These results indicate that AR1001 acts directly on CAA pathology and can serve as an oral therapy to mitigate disease progression.

AR1001 is an oral therapeutic based on phosphodiesterase-5 (PDE5) inhibition. Unlike antibody injections, it does not rapidly remove toxic proteins. Instead, it gradually modulates pathology through multiple mechanisms, including improvement of cerebral blood flow, vascular stabilization, inflammation suppression, and neuroprotection.

Currently, AR1001 is undergoing a global Phase 3 clinical trial (POLARIS-AD) involving 1,450 patients across 13 countries, including the United States, Europe, and Korea. The target enrollment of 1,150 patients has already been exceeded, and enrollment has been completed. Topline results are scheduled to be announced in the first half of 2026.

Jaejoon Jung, CEO of Aribio, stated, "AR1001 represents a meaningful therapeutic alternative that can also be applied to high-risk Alzheimer's patients with CAA, and it may overcome the limitations of side effects seen with existing antibody injections. We plan to actively pursue additional indications and follow-up clinical trials."

Meanwhile, AR1001 has been selected by the global market research firm GlobalData as the "most promising next-generation Alzheimer's disease therapy." Aribio is currently pursuing a merger with the listed company Solux, with the merger date set for August 1.