PYC Therapeutics (ASX:PYC) has achieved notable progress in its Phase 1a Single Ascending Dose (SAD) clinical trial for its RNA-based therapeutic candidate, PYC-003, targeting autosomal dominant polycystic kidney disease (ADPKD).
As of August 8, 2025, the Safety Review Committee (SRC) has given the green light to escalate dosing to the fourth and final cohort in Part A of the SAD trial involving healthy volunteers. Simultaneously, the company has begun dosing ADPKD patients in Part B, marking the transition from healthy subjects to the targeted patient population.
This milestone comes on the heels of the successful completion of the first three dose cohorts, including healthy volunteer dosing and interim safety reviews, as announced on the ASX. The SAD study is designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of PYC-003, with preliminary data in healthy participants forming the foundation for patient dosing and subsequent trial phases.
Looking ahead, PYC Therapeutics expects to move into repeat-dose studies in PKD patients by the fourth quarter of 2025. These studies are critical in establishing the optimal dosing regimen and further evaluating safety profiles in patients.
PYC-003 represents a potentially disease-modifying treatment that targets the genetic mechanisms underlying PKD by restoring PC1 protein expression. If early clinical success continues, PYC is poised to progress to registrational combined Phase 2 and 3 trials