PYC Therapeutics (ASX:PYC), a clinical-stage biotechnology company, has announced the nomination of its fourth clinical drug candidate, PYC-002, aimed at treating Phelan-McDermid Syndrome (PMS). PMS is a severe neurodevelopmental disorder caused by insufficient SHANK3 expression. PYC-002 is designed to target this root cause.
PYC Therapeutics is focused on developing precision RNA therapies for genetic diseases with unmet needs, utilizing a proprietary drug delivery platform. The nomination of PYC-002 for Phelan-McDermid Syndrome is a significant step, with preclinical studies showing increased SHANK3 expression in critical brain regions. The company aims to start IND-enabling studies in 2025 and human trials by 2026. PYC's pipeline also includes treatments for Retinitis Pigmentosa Type 11, Autosomal Dominant Optic Atrophy, and Autosomal Dominant Polycystic Kidney Disease, with various trials and data expected by 2026.
The nomination of PYC-002 marks a significant milestone for PYC Therapeutics as we continue to advance our pipeline of precision RNA therapies aimed at addressing the root cause of genetic diseases with high unmet medical needs. Our preclinical studies have demonstrated promising results for PYC-002, and we are excited to progress this program towards human trials.