PYC Therapeutics (ASX: PYC) has dosed the first patient in the Phase 1b Multiple Ascending Dose (MAD) study of PYC-003 for polycystic kidney disease (PKD).
The MAD study aims to establish safety and tolerability for repeat dosing, as well as to evaluate efficacy endpoints including urinary PC1 protein levels, total kidney volume on MRI, and estimated glomerular filtration rate.
Results from the Phase 1a Single Ascending Dose (SAD) study are anticipated in H2 CY26, with data from the Phase 1b MAD study expected in CY27.
Successful completion of this study, subject to regulatory alignment, would enable a transition to a registrational combined Phase 2/3 trial targeting a New Drug Application (NDA).
Ophthalmology Program Progress Highlighted
Interim Phase 1/2 ophthalmology clinical trial data for two investigational RNA-peptide therapies were presented at the ARVO 2026 conference (May 3-7, 2026).
These candidates are VP-001 for RP11 and PYC-001 for Autosomal Dominant Optic Atrophy (ADOA), both of which continue to show safety and tolerability.
For VP-001, no treatment-related serious adverse events have been reported to date, including repeat-dose patients.
The presentations highlighted emerging efficacy signals for VP-001, including clinically meaningful improvements in Low-Luminance Visual Acuity (LLVA) and retinal sensitivity.
The PYC-001 Multiple Ascending Dose (MAD) study for ADOA is progressing, with the company's Safety Review Committee giving its approval to progressing the 60 microgram dose cohort into the MAD study, alongside existing 10 and 30 microgram cohorts, following acceptable four-week safety outcomes.
Previous Funding and Development Milestones
PYC Therapeutics recently completed the retail component of its pro rata accelerated non-renounceable entitlement offer that raised approximately $47 million at $1.50 per new share.
Earlier, the PYC-003 Single Ascending Dose (SAD) study for PKD saw its Safety Review Committee approve dose escalation.
This followed a review of four-week safety data from the B1 and B2 cohorts, allowing progression to the B3 cohort.
Similarly, for PYC-001 in ADOA, the Safety Review Committee approved progression of the 60 microgram dose cohort from the Phase 1 SAD study into the Phase 1/2 Multiple Ascending Dose (MAD) study.
This was based on 4-week safety and tolerability outcomes.
Outlook for PYC Therapeutics
PYC Therapeutics is advancing its pipeline with key clinical trial initiations and progress updates across its PKD and ophthalmology programs.
The upcoming data readouts for both PYC-003 and PYC-001 will be critical in de-risking the development pathway and supporting the transition to registrational studies.
