Key Highlights

  • The first patient has been dosed in the Part D open-label extension of the Phase 1b multiple-ascending-dose study of PYC-003 in polycystic kidney disease.
  • The extension allows dosing every six weeks for up to 24 months, mirroring the duration of the planned registrational trial.
  • Phase 1a data is expected in H2 CY26 and Phase 1b and extension data in CY27, with the registrational Phase 2/3 subject to regulatory alignment.

PYC Therapeutics Limited (ASX:PYC), a precision medicine company developing RNA therapies for genetic diseases, announced on 11 September 2026 that the first patient has been dosed in the Part D Open-Label Extension (OLE) of the ongoing Phase 1b multiple-ascending-dose (MAD) study of PYC-003 in patients with polycystic kidney disease (PKD). The program is registered as NCT06714006.

The extension study

The OLE enables patients who complete Part C of the Phase 1b trial to receive PYC-003 continuously for up to 24 months, mirroring the duration of the registrational trial planned after the Phase 1b study. The first enrolled patient has finished dosing in the MAD study, has been on the drug candidate for four months, and will continue to receive PYC-003 every six weeks for up to 24 months. Ethics committee and regulatory approval for the OLE was granted.

Objective of the Phase 1b study

The Phase 1b MAD study aims to determine the safety, tolerability and optimal dosing regimen of PYC-003 in a repeat-dose setting, ahead of a proposed move to a registrational trial. PYC-003 is aimed at PKD caused by a mutation in the PKD1 gene, and the company says it addresses the underlying cause of the disease.

Development pathway

The company describes a proposed pathway in which successful completion of the Phase 1b study, followed by alignment with regulatory authorities, leads to a registrational combined Phase 2/3 trial intended to support a New Drug Application. The release notes that this is subject to receiving regulatory approval for the proposed registrational requirements and to the risks set out in the company's 2 February 2026 disclosures.

Expected data timing

Safety and efficacy data from the Phase 1a single-ascending-dose study is expected to be presented in the second half of calendar 2026, and data from the Phase 1b MAD study and the extension is expected in calendar 2027. The release does not include any patient data, and PYC says it currently has three clinical-stage Drug Development programs and a proprietary delivery platform designed to enhance the potency of RNA medicines.

Study design and prior announcements

The Phase 1b study includes a multiple-ascending-dose stage followed by Part D, the open-label extension for participants who complete Part C. Details of the Phase 1a single-ascending-dose study were reported in company announcements between February 2025 and February 2026, and the OLE approval was announced on 22 July 2026. The company states that the registrational trial is subject to regulatory approval of the proposed requirements and to alignment with authorities.

Company programs

PYC describes itself as creating a new generation of RNA therapies using a proprietary delivery platform intended to enhance the potency of precision medicines. It focuses on monogenic diseases, which it says have the highest likelihood of success in clinical development, and has three clinical-stage programs, of which PYC-003 for PKD is one. The announcement contains forward-looking statements that depend on assumptions that may prove incorrect.