• Presented positive phase 1/2 data for VP-001 in Retinitis Pigmentosa Type 11
• Commenced dosing of first patient with PYC-001 for Autosomal Dominant Optic Atrophy
• Completed preclinical studies for PYC-003 in Polycystic Kidney Disease

PYC Therapeutics, a clinical-stage biotechnology company, reported significant progress in its pipeline of precision medicines for genetic diseases during the six months ending 31 December 2024. The company presented data from ongoing phase 1/2 studies demonstrating that patients were improving on two registrational endpoints following treatment with the company's investigational drug candidate for Retinitis Pigmentosa Type 11 (RP11), which also received Orphan Drug Designation and Rare Pediatric Designation from the US FDA. PYC also commenced dosing in patients with the first drug candidate (PYC-001) that addresses the underlying cause of Autosomal Dominant Optic Atrophy (ADOA), which received Rare Pediatric Disease designation from the FDA. Additionally, the company completed preclinical studies for PYC-003, a drug candidate with disease-modifying potential in Polycystic Kidney Disease (PKD), and received regulatory approvals to commence human trials for this program. PYC also nominated a clinical development candidate for the Phelan-McDermid Syndrome (PMS) program following successful preclinical studies. Subsequent to the end of the reporting period, the company launched a $146 million capital raising to fund progress in all four pipeline programs through to FY27, providing sufficient runway to deliver critical human safety and efficacy data in the company's three clinical-stage drug development programs.

The company reported a consolidated cash position of $49,253,334 as at 31 December 2024. Expenditure for the half-year was $38,558,169, with the increase due to the continued progression of the company's pipeline of drug candidates through translational studies and clinical trials.