EMA Approves Phase 3 Trial for AriBio's AR1001 Alzheimer's Therapy

AriBio Co., Ltd., a biopharmaceutical firm headquartered in South Korea with a presence in the United States, has secured authorization from the European Medicines Agency (EMA) to proceed with a pivotal phase 3 clinical trial named POLARIS-AD for the treatment of early Alzheimer’s disease. This approval follows similar endorsements from the U.S. Food and Drug Administration (FDA) and the UK Medicines and Healthcare Products Regulatory Agency (MHRA), underscoring a unified international approach to the clinical trial's authorization.

The EMA's final approval was given on April 24th, paving the way for the trial to be conducted in several European countries including the Czech Republic, Denmark, France, Germany, Italy, Spain, and The Netherlands. The trial is designed to enroll approximately 1,150 participants across 200 global sites.

POLARIS-AD is structured as a double-blind, randomized, placebo-controlled study. It aims to assess the efficacy and safety of AR1001, also known as Mirodenafil, in patients with early Alzheimer’s disease who exhibit confirmed amyloid pathology. The trial will rely on the Clinical Dementia Rating Scale–Sum of Boxes (CDR-SB) as its primary endpoint, a measure recognized by both the FDA and EMA. Secondary endpoints will encompass a range of cognitive and functional assessments, as well as biomarker evaluations in cerebral spinal fluid and plasma.

James Rock, AriBio's Chief Clinical Officer, highlighted the significance of the EMA's approval, noting that it follows the UK's acceptance and marks a milestone in the global registration program for AR1001. He emphasized that the trial is set to begin enrolling participants in the second quarter of 2024, with the company dedicating substantial resources to ensure a swift and efficient expansion of the trial's reach. Rock also noted the heightened global interest in AR1001, driven by a strong desire for alternative Alzheimer’s treatments and the potential for worldwide access should the drug prove effective and safe.

AR1001 is an investigational oral agent developed as a phosphodiesterase-5 (PDE5) inhibitor for Alzheimer’s disease. Pre-clinical research has indicated that AR1001 possesses neuroprotective properties, capable of inhibiting neuronal cell death and enhancing synaptic plasticity, thereby suggesting its potential to influence the disease's progression positively.

AriBio is dedicated to the advancement of innovative treatments for neurodegenerative disorders, with a particular focus on Alzheimer’s disease. The company's commitment to the POLARIS-AD trial reflects its dedication to improving patient outcomes and contributing to the global fight against Alzheimer’s.