Quince Therapeutics, a late-stage biotechnology company, announced safety data from its Phase 3 ATTeST clinical trial at the 53rd Child Neurology Society Annual Meeting. The trial assessed treatment-emergent adverse events (TEAEs) in Ataxia-Telangiectasia (A-T) patients treated with EryDex (intra-erythrocyte dexamethasone sodium phosphate) for one year versus a placebo. The poster presentation highlighted findings regarding the safety profile of intra-erythrocyte dexamethasone treatment in children with A-T.
Quince is enrolling patients in the pivotal Phase 3 NEAT clinical trial, a global, randomized, double-blind, placebo-controlled study evaluating EryDex’s neurological effects in A-T patients. Approximately 86 patients aged six to nine (primary analysis population) and about 20 patients aged ten and older are planned for enrollment. The trial, conducted under a Special Protocol Assessment (SPA) with the FDA, anticipates topline results in Q4 2025. Pending positive results, a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA are expected in 2026. The FDA has granted Fast Track designation to EryDex for A-T treatment, recognizing its potential to address an unmet medical need.
A-T, an inherited neurodegenerative and immunodeficiency disorder caused by ATM gene mutations, affects DNA repair and cell function. Early diagnosis typically occurs before age five as gait abnormalities and frequent falls emerge. Neurological symptoms progress, often leading to wheelchair dependence in adolescence. Recurrent infections, pulmonary issues, and malignancies characterize the teenage years. Life expectancy is typically 25-30 years, with infections and malignancy as primary causes of death. An estimated 4,600 patients in the U.S. and approximately 5,000 in the U.K. and EU4 countries have A-T. Currently, no approved treatments exist globally.
EryDex combines dexamethasone sodium phosphate (DSP), encapsulated within a patient’s red blood cells, with a specialized delivery device. DSP, a corticosteroid known for anti-inflammatory properties and dose-limiting toxicity due to adrenal suppression, is delivered via the patient’s red blood cells (autologous erythrocytes). This drug/device combination aims to provide corticosteroid efficacy while minimizing adverse effects associated with chronic use. The system utilizes Quince’s proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology, an automated process designed for drug encapsulation and delivery using autologous red blood cells. Red blood cells’ properties, including the potential for better tolerability, enhanced tissue distribution, reduced immunogenicity, and extended circulating half-life, make them a promising drug delivery vehicle. Quince’s AIDE technology harnesses these benefits, enabling chronic administration of drugs limited by toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
