PYC Therapeutics (ASX:PYC) has announced it plans to progress investigational drug candidate PYC-002 into human clinical trials for Phelan-McDermid Syndrome (PMS).
The company said the nomination of PYC-002 follows successful studies in both patient-derived and animal models.
PMS is caused by an inability of neurons within the brain to communicate with one another due to insufficient expression of the SHANK3 protein at the communication junction between the neurons known as a synapse.
PMS patients have a loss of function mutation in one of the two copies of the SHANK3 gene, causing half as much SHANK3 protein as is required within the neuronal synapse.
PYC said the pre-clinical studies demonstrated that PYC-002 reaches the critical regions of the brain implicated in PMS in vivo and corrects the underlying cause of PMS in neurons derived from patients in vitro.
PYC CEO Dr Rohan Hockings said, “We are very pleased for the PMS community to be progressing this drug candidate into human trials. The data supporting this milestone show great potential for the first RNA therapy in this indication. The read-through benefits from other clinically-validated drug candidates within this class in different diseases occurring in the same target cell and organ provide a clear path to the patient-impact that we are striving for.”
