PYC Therapeutics Confirms Safety Milestone for PKD Drug Candidate PYC-003
PYC Therapeutics reports positive safety outcomes from the initial phase of its clinical trial for PYC-003, a promising treatment for Polycystic Kidney Disease. The drug was well tolerated in healthy volunteers, paving the way for patient dosing and further trials.
- PYC-003 safe and well tolerated up to 4 mg/kg in healthy volunteers
- No serious adverse events reported in Phase 1a/1b Part A
- Dosing commenced in PKD patients for Part B of the study
- Repeat dosing in patients planned for first half of 2026
- Successful Phase 1a/1b completion to lead to combined Phase 2/3 registrational trial
Clinical Safety Milestone Achieved
PYC Therapeutics Limited (ASX – PYC) has announced encouraging safety results from Part A of its Phase 1a/1b clinical trial for PYC-003, an investigational drug targeting autosomal dominant Polycystic Kidney Disease (PKD). The Safety Review Committee overseeing the trial confirmed that single doses of PYC-003 up to 4 mg/kg were safe and well tolerated in healthy volunteers, with no treatment-emergent serious adverse events reported.
Advancing Into Patient Dosing
Following these positive safety findings, PYC has initiated dosing in PKD patients in Part B of the study at a dose of 1.2 mg/kg. This marks a critical transition from healthy volunteer testing to evaluating the drug’s effects in the target patient population. The company is also preparing to start repeat dosing in patients (Part C) in the first half of 2026, which will provide further insights into the drug’s safety and optimal dosing regimen over multiple administrations.
Pathway to Market
The Phase 1a/1b study is designed to assess both safety and preliminary efficacy, with successful completion expected to enable a combined Phase 2/3 registrational trial. This pivotal study aims to support a New Drug Application (NDA) for PYC-003, potentially bringing the first precision medicine treatment for PKD closer to regulatory approval. The company’s approach leverages RNA therapeutic technology to address the genetic root cause of PKD, a disease with limited treatment options.
Strategic Positioning and Future Outlook
PYC Therapeutics is positioning itself at the forefront of RNA-based therapies for monogenic diseases, which have a higher likelihood of clinical success. The company’s proprietary delivery platform enhances the potency of its precision medicines, aiming to transform patient outcomes. While the current announcement focuses on safety, upcoming data from patient dosing cohorts will be closely watched for signs of efficacy and longer-term tolerability.
Overall, these results represent a significant step forward for PYC-003 and reinforce PYC Therapeutics’ commitment to advancing innovative treatments for genetic diseases with unmet medical needs.
Bottom Line?
With safety confirmed, all eyes now turn to patient efficacy data and regulatory milestones in 2026.
Questions in the middle?
- Will PYC-003 demonstrate meaningful efficacy in PKD patients during repeat dosing?
- How will regulatory authorities respond to the combined Phase 2/3 trial design?
- What are the commercial prospects if PYC-003 gains approval as a first-in-class PKD therapy?
