[May 28, 2025]ARIBIO Publishes CAA Efficacy of AR1001 in International Journal Oral Drug AR1001 Overcomes Limits of Injectable Alzheimer’s Therapies

ARIBIO Presents CAA Treatment Potential at International Journal AR1001 Surpasses the Limitations of Existing Antibody-Based Alzheimer's Treatments AriBio’s Alzheimer’s treatment candidate, AR1001, has been recognized for its differentiated safety and efficacy in Cerebral Amyloid Angiopathy (CAA) compared to conventional antibody-based therapies. A joint study by AriBio’s neuroscience team and Hallym University has been published in the international journal Frontiers in Aging Neuroscience, highlighting AR1001’s mechanism of action in stabilizing brain blood vessels and suppressing inflammation—with significantly lower risk of ARIA (Amyloid-Related Imaging Abnormalities), a known side effect of antibody drugs like brain swelling or hemorrhage. CAA is a condition in which toxic proteins accumulate in cerebral blood vessel walls, increasing the risk of rupture or hemorrhage. It coexists in more than 50% of Alzheimer’s patients. The study demonstrated that AR1001 directly targets CAA by protecting the blood-brain barrier (BBB), stabilizing blood vessels, and reducing toxic protein accumulation in the brain. Using Alzheimer’s mouse models carrying the human ApoE4 gene—a known genetic risk factor for dementia—the researchers observed a significant decrease in inflammatory cytokines, increased activation of microglia, and elevated expression of CLN-5, a neurovascular protein. These changes helped restore the integrity of the BBB. Furthermore, toxic protein deposits (amyloid-beta) in the hippocampus were significantly reduced, and cognitive functions were recovered. Recently approved antibody-based Alzheimer’s therapies such as lecanemab and donanemab have demonstrated effectiveness in removing toxic proteins, but they come with high risks of serious side effects like ARIA (Amyloid-Related Imaging Abnormalities). Clinical trials show that patients suspected of having CAA experienced 2 to 3 times higher incidence of ARIA-E (edema) or ARIA-H (hemorrhage) compared to other patients. Fatal brain hemorrhages have also been reported. Accordingly, major regulatory bodies including the U.S. FDA and European Medicines Agency (EMA) have issued Black Box Warnings for these antibody therapies and mandated regular MRI monitoring. They also advise avoiding treatment in high-risk CAA patients. In contrast, AR1001 is an oral PDE5 inhibitor-based treatment, which does not abruptly remove toxic proteins. Instead, it improves cerebral blood flow, stabilizes the blood-brain barrier, reduces neuroinflammation, and protects brain cells through multi-modal mechanisms, gradually alleviating Alzheimer’s-related pathology. AR1001 is currently undergoing a global Phase 3 clinical trial (POLARIS-AD) across 13 countries including the United States, Europe, and South Korea. With over 1,150 patients enrolled, the recruitment target has been exceeded and enrollment was completed ahead of schedule. Topline results are expected in the first half of 2026. Ph.D Jai-Jun Choung, CEO of AriBio, stated: “While the currently approved antibody therapies are powerful injectable drugs that clear toxic proteins from the brain, they come with serious risks of edema and hemorrhage, especially in patients with CAA. In contrast, AR1001, as an oral therapy, may offer a safer and more sustainable option for Alzheimer’s patients with vascular complications like CAA. We will proactively pursue follow-up clinical trials and indication expansion based on these findings.” Meanwhile, AR1001 has been recognized by GlobalData, a leading market intelligence firm, as the next-generation Alzheimer’s drug with the highest clinical potential. Expectations are rising for exclusive global licensing deals and blockbuster commercialization. About AriBio AriBio Co., Ltd. is a biopharmaceutical company based in South Korea with offices in the United States. The company focuses on the development of novel therapies for neurodegenerative diseases including Alzheimer’s disease. The company continues to expand its partnerships to accelerate first-in-class treatment options and advancements in the field of neurodegeneration. About Hallym University College of Medicine Hallym University College of Medicine, established in 1982, has been dedicated to cultivating medical professionals who respect human dignity and the value of life under the founding philosophy of “Medicine as a benevolent art that saves lives.” With five affiliated teaching hospitals—Kangnam, Chuncheon, Dongtan, Hangang, and Kangdong Sacred Heart Hospitals—it offers an outstanding clinical education environment. The college also leads cutting-edge biomedical convergence research in collaboration with the Institute of Medical Science, Clinical Research Center, and AI Convergence Medical Research Center. Hallym University College of Medicine continues to pursue global research partnerships and educational innovation with the aim of nurturing creative and dedicated healthcare professionals equipped for the global stage. Contacts Fred Kim [email protected]

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