Neuren Pharmaceuticals has announced the expansion of its neurodevelopmental disorders pipeline for NNZ-2591 to include SYNGAP1 related disorder (SRD). This decision follows encouraging results obtained in a pre-clinical model. Neuren Pharmaceuticals is a biopharmaceutical company focused on developing novel therapies to treat neurological disorders. The company is dedicated to addressing unmet needs in neurodevelopmental disorders through innovative research and development.
SRD is a genetic condition affecting approximately 1 in 16,000 individuals and currently has no approved treatments. The condition arises from a mutation in the SYNGAP1 gene. Neuren’s pre-clinical research has yielded positive outcomes, suggesting that NNZ-2591 could potentially address the underlying neuronal dysfunction associated with SRD.
According to the company, in an in-vitro model of SRD utilising human iPSC-derived neurons, treatment with NNZ-2591 effectively reversed the neuronal dysfunction caused by SYNGAP1 haploinsufficiency. These findings provide a basis for further investigation into the therapeutic potential of NNZ-2591 for individuals affected by SRD.
Neuren is actively developing NNZ-2591 to address various neurodevelopmental disorders lacking approved treatments. The company is also preparing to initiate a Phase 3 clinical trial for Phelan-McDermid syndrome, marking a significant step forward in its clinical development program. The addition of SRD to its pipeline underscores Neuren’s commitment to expanding the application of NNZ-2591 to a wider range of neurodevelopmental conditions.
