• Neuren announces addition of SYNGAP1-related disorder (SRD) to NNZ-2591 pipeline
• SRD is a rare genetic disorder with no approved treatments
• In pre-clinical studies, NNZ-2591 reversed neuronal dysfunction caused by SYNGAP1 haploinsufficiency

Neuren Pharmaceuticals (ASX: NEU) has announced the addition of SYNGAP1-related disorder (SRD) into its neurodevelopmental disorders pipeline for NNZ-2591, following positive results in a pre-clinical model. SRD is a rare genetic disorder caused by a variant on the SYNGAP1 gene, which is responsible for producing the SYNGAP1 protein. This protein acts as a regulator in the synapses, and insufficient production leads to impaired communication between neurons, resulting in various neurological issues such as intellectual disability, epilepsy, and autism spectrum disorder. In an in-vitro model of SRD in human iPSC-derived neurons, treatment with NNZ-2591 reversed the neuronal dysfunction caused by SYNGAP1 haploinsufficiency. Neuren is developing NNZ-2591 to treat multiple neurodevelopmental disorders for which there are no approved treatments and is preparing to commence a Phase 3 clinical trial in Phelan-McDermid syndrome.