DOWN-AUTONOMY · LCTB-21: A DYRK1A protein kinase inhibitor for treatment of cognitive decline in Down Syndrome.

All people with Down Syndrome (DS) suffer from a certain level of impaired learning and memory (cognitive disorders) from childhood onwards, greatly impacting their autonomy and societal integration. This is becoming an increasingly urgent issue as people with DS are more often outliving their care takers, yet an adequate treatment has not yet been developed.Studies have revealed that the cognitive decline in people with DS is associated with overexpression of the DYRK1A protein kinase in the brain. Perha Pharmaceuticals has developed a novel therapeutic compound, Leucettinib-21 (LCTB-21), which acts as a DYRK1A inhibitor that can reduce cognitive decline in children with DS. Ultimately, this will increase the autonomy of people with DS resulting in better societal integration. Perha Pharmaceuticals aims to outlicense LCTB-21 to a large pharmaceutical partner (e.g. Otsuka Pharmaceuticals, Intra-Cellular Therapies) following a successful phase 2a clinical trial.