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Focused on therapeutic aptamers, aptaTargets developed ApTOLL, an immune modulator which represents a novel approach to treat ischemic stroke in the acute phase by substantially reducing the brain damage. ApTOLL is a first-in-class neuroprotective drug that acts on the Toll-like receptor 4 (TLR4) with high specificity, blocking the inflammatory response that occurs after an ischemic stroke.
TLR4 signaling is suggested to be central to the innate inflammation induced by acute ischemic injury and inhibiting TLR4 aims to down-regulate neuroinflammatory mechanisms. In a Phase 1b/2a clinical study, published in JAMA Neurology (1), ApTOLL demonstrated positive safety and efficacy results for acute ischemic stroke, including reduced mortality and improved functional recovery. In 2023 ApTOLL was granted a PRIME designation from the European Medicines Agency in 2023.
(1) Hernández-Jiménez, M., et al. (2023). Safety and efficacy of ApTOLL in patients with ischemic stroke undergoing endovascular treatment: A phase 1/2 randomized clinical trial. JAMA Neurology, 80(8), 779-788. https://doi.org/10.1001/jamaneurol.2023.1660
Additionally, aptaTargets aims to study the use of ApTOLL in other indications where TLR4 receptor plays a key role (intracerebral hemorrhage, myocardial infarction, and multiple sclerosis), and to launch other drug development programs with new aptamer drug candidates.