PYC Therapeutics (ASX:PYC), a clinical-stage biotechnology company, has commenced a clinical trial for its drug candidate, PYC-001, targeting Autosomal Dominant Optic Atrophy (ADOA), a rare eye disease. The Single Ascending Dose (SAD) study aims to assess the safety, tolerability, and efficacy of the drug, with the first patient now dosed.
PYC Therapeutics has initiated a clinical trial for PYC-001, marking progress in addressing Autosomal Dominant Optic Atrophy (ADOA). The study is expected to provide insights by mid-2025, informing future trials, including a Multiple Ascending Dose study and a registrational trial aimed for 2026. PYC-001, which has received Orphan Drug and Rare Pediatric Disease Designations, seeks to restore OPA1 gene expression crucial for retinal function. PYC Therapeutics is also developing treatments for other genetic diseases, with ambitions for regulatory approvals and successful clinical advancements.
The initiation of this clinical trial is a significant milestone for PYC Therapeutics. It underscores our commitment to developing precision medicines for genetic diseases with unmet medical needs.