PYC Therapeutics (ASX:PYC) has reported continued clinical and operational progress across its pipeline of RNA-based precision medicines, alongside a strong financial position, in its third-quarter 2025 shareholder update.
The company is advancing four drug development programs targeting rare genetic diseases with no approved therapies: polycystic kidney disease (PKD), two blinding eye diseases (RP11 and ADOA), and the neurodevelopmental disorder Phelan-McDermid Syndrome (PMS).
The company confirmed that human safety and efficacy data are expected across all programs over the next two years, with the first readouts anticipated in calendar year 2025.
Its most advanced kidney program has progressed to Part B of its single-ascending-dose study in PKD patients, while dosing in cohort 4 of Part A in healthy volunteers has been completed. Safety data from Part A were presented at the Australian and New Zealand Society of Nephrology conference earlier this year. PYC noted that human safety and initial efficacy data are expected in 2026, subject to regulatory and clinical outcomes.
In its RP11 program for retinitis pigmentosa, PYC has advanced to an open-label extension of its multiple-ascending-dose trial. The company is preparing for a Type D meeting with the U.S. Food and Drug Administration in the first quarter of 2026 to finalise the registrational trial design. Data from ongoing clinical trials have shown improvements in vision among treated patients, and the program is positioned as a front-runner for first approval in this rare blinding eye disease.
For autosomal dominant optic atrophy (ADOA), the company has completed dosing in its Phase 1 single-ascending-dose study and has commenced a global repeat-dose study. PYC expects this trial to provide the clinical proof-of-concept necessary to advance the program toward registration, with human safety and efficacy data expected in 2026.
In its pre-clinical PMS program, the company has generated non-human primate data to complement earlier patient-derived model results. This will support progression into Investigational New Drug (IND) enabling studies, with the first clinical trials planned for 2026 and data anticipated in 2027.
PYC ended the quarter with $135 million in cash and cash equivalents. It also expects to receive a further $20 million R&D rebate in early 2026. Net operating cash outflow for the quarter was $17.4 million, and the company reported an estimated 7.7 quarters of funding runway based on its current burn rate. Payments to related parties during the quarter were $157,000, covering director remuneration.
The company said the clinical progress across all four programs reflects the execution of its operational roadmap to bring first-in-class RNA therapies to patients with severe genetic diseases.
“Our goal is to create life-changing impact for patients with genetic diseases through the discovery and development of drugs that address their underlying cause,” the company stated in its quarterly update.