• PYC is developing a drug candidate that addresses the underlying cause of Phelan-McDermid Syndrome (PMS)
• PMS affects 1 in every 10,000 children and there are no approved treatment options
• PYC announces successful Non-Human Primate (NHP) studies that support progression of this drug development program into human trials

PYC Therapeutics is developing a drug candidate (PYC-002) that addresses the underlying cause of Phelan-McDermid Syndrome (PMS), a severe neurodevelopmental disorder affecting 1 in every 10,000 children. PMS is caused by a mutation in or deletion of one copy of the SHANK3 gene, leading to SHANK3 protein haploinsufficiency and impaired synaptic signalling and plasticity. PYC-002 works by increasing expression of SHANK3 from the remaining 'good' copy of the gene to compensate for the decreased expression caused by the mutation. PYC has now announced successful Non-Human Primate (NHP) studies that demonstrate PYC-002 is safe and well-tolerated, effectively distributes to key brain regions affected in PMS, and shows potency comparable to a clinically-validated RNA therapy targeting a different neurodevelopmental disorder. Importantly, the concentration of PYC-002 achieved in the NHP brain at safe and well-tolerated doses is higher than what was required to restore SHANK3 expression and achieve functional rescue in PMS patient-derived neurons in vitro. PYC also showed that a single dose of PYC-002 can upregulate multiple SHANK3 isoforms and cause dose-dependent SHANK3 upregulation in the prefrontal cortex of NHPs. These pre-clinical data support progression of PYC-002 into human clinical trials, which PYC expects to initiate in 2026, subject to regulatory approvals.

PYC expects to progress its investigational drug candidate for PMS, PYC-002, into clinical trials in 2026, subject to successful completion of Good Laboratory Practice toxicology studies and necessary regulatory approvals.