Focused on therapeutic aptamers, aptaTargets is currently developing ApTOLL, an immune modulator which represents a novel approach to treat ischemic stroke in the acute phase by substantially reducing the brain damage. After the successful development in nonclinical stages and First-in-Human trial (Phase I), company is advancing up to a clinical Proof-of-Concept in patients.
ApTOLL is a first-in-class aptamer (single-stranded DNA molecule) targeting TLR4, a receptor that triggers the immune response at the first steps of the inflammatory pathway.
Due to aptamers’ three-dimensional conformation, they have a great affinity, selectivity, and biological action on their targets.
Antagonizing TLR4 allows ApTOLL to modulate the immune response, preventing the first wave of the inflammatory cascade that occurs after stroke onset. Experimental models in ischemic stroke show an outstanding neuroprotective effect, reducing brain damage up to 65% with an effective time-window of 12 hours. Preclinical and early clinical data confirms ApTOLL’s excellent safety profile.
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.