• Presented positive data from ongoing phase 1/2 studies for Retinitis Pigmentosa type 11 drug candidate
• Commenced dosing of patients with first drug candidate for Autosomal Dominant Optic Atrophy
• Completed pre-clinical studies and submitted regulatory application for Polycystic Kidney Disease drug candidate

PYC Therapeutics Ltd provided an update on the progress made in all four of its drug development programs during the fourth quarter of 2024. In the Retinitis Pigmentosa type 11 (RP11) program, the company presented data from its ongoing phase 1/2 studies demonstrating that patients were improving on two registrational endpoints following treatment with the investigational drug candidate. PYC also received Orphan Drug Designation and Rare Pediatric Disease Designation from the US FDA for this program. In the Autosomal Dominant Optic Atrophy (ADOA) program, the company commenced dosing of patients with the first drug candidate that addresses the underlying cause of this disease. For the Polycystic Kidney Disease (PKD) program, PYC completed pre-clinical studies and submitted a regulatory application to progress the drug candidate into clinical trials. In the Phelan-McDermid Syndrome (PMS) program, the company nominated a clinical development candidate following successful pre-clinical studies. PYC remains on track to deliver the objectives outlined in its operational roadmap, with plans to engage with the FDA, initiate registrational trials, and generate human safety and efficacy data across its pipeline in 2025.

As of 31 December 2024, PYC had $49.3 million in cash on hand. The company received a $17.3 million R&D tax rebate during the quarter. PYC expects to initiate registrational trials for its RP11 drug candidate, generate human safety and efficacy data for its ADOA and PKD programs, and commence IND-enabling studies for its PMS program in 2025, subject to regulatory approvals and the risks outlined in the company's ASX filings.

PYC remains on track to deliver the objectives outlined in its 4-year operational roadmap, with plans to establish clinical proof of concept, initiate registrational trials, and progress its pipeline of precision medicines for patients with genetic diseases in 2025 and beyond.