PYC Therapeutics Achieves Key Preclinical Milestone for Phelan-McDermid Syndrome Program

PYC Therapeutics (ASX: PYC), a clinical-stage biotechnology company focused on RNA-based precision medicine, has announced promising preclinical results supporting the progression of its investigational drug candidate, PYC-002, into human trials for Phelan-McDermid Syndrome (PMS). PMS is a severe neurodevelopmental disorder that affects approximately one in every 10,000 children and currently has no approved treatment.

PYC-002 targets the underlying genetic cause of PMS by increasing the expression of the SHANK3 protein in neurons. The company’s latest data from non-human primate studies confirms the drug’s favorable safety profile, effective distribution to critical brain regions, and encouraging comparisons with a clinically validated RNA therapy in a similar disease area.

Importantly, the concentration of PYC-002 in primate brains exceeded levels required to restore SHANK3 expression and achieve functional improvement in patient-derived brain cells, reinforcing its disease-modifying potential. PYC also demonstrated that the drug upregulated target gene expression in both rats and primates, and that a single dose led to broad brain distribution and improved synaptic signaling.

PYC plans to initiate clinical trials in 2026, with Investigational New Drug filings expected following the successful completion of toxicology studies.

These results place PYC-002 among the most advanced programs globally targeting PMS, offering new hope to patients and families facing this challenging condition.