• PYC is developing a drug candidate that addresses the underlying cause of Phelan-McDermid Syndrome (PMS)
• PMS affects 1 in 10,000 children and has no approved treatment options
• PYC's drug candidate restores missing gene expression and rescues functional deficits in neurons derived from PMS patients

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 10,000 children with no approved treatment options. The data PYC is presenting at the PMS Global Congress demonstrates that PYC-002 restores the missing gene expression that causes PMS in brain cells derived from patients and rescues the functional deficits in these neurons that underlie the neurodevelopmental delays that characterize the syndrome. In addition, PYC will present in vivo data showing that PYC-002 effectively controls target gene expression in the key regions of the brain implicated in PMS. PYC's PMS program is expected to advance into clinical trials in approximately 12 months, marking the fourth first-in-class drug candidate with disease-modifying potential that PYC has advanced into human trials.

PYC's PMS program is currently progressing through the final pharmacokinetic and dose-range finding studies required before initiating formal Investigational New Drug (IND)-enabling studies. The program is currently expected to enter human trials next year.

PYC-002 is a pre-clinical stage drug candidate that addresses the underlying cause of PMS by increasing SHANK3 protein expression in the target cells within the brain. The drug candidate has demonstrated efficacy in restoring SHANK3 levels and reversing key PMS-related neuronal deficits in patient-derived neurons, as well as a favorable safety and tolerability profile in vivo.