Dose Escalation Approved: PYC-003 Moves to 1.2 mg/kg in Phase 1a Study
PYC Therapeutics has secured approval to escalate dosing in its Phase 1a trial of PYC-003 for Polycystic Kidney Disease, moving closer to patient dosing later this year.
- Safety Review Committee approves dose increase from 0.4 mg/kg to 1.2 mg/kg
- Dosing in healthy volunteers progressing as planned in Phase 1a Single Ascending Dose study
- Patient dosing for PKD expected to begin in the second half of 2025
- Phase 1a/1b studies to be followed by registrational Phase 2/3 trial
- PYC-003 targets underlying cause of Polycystic Kidney Disease, a condition with no current treatments
Clinical Trial Progress
PYC Therapeutics Limited (ASX: PYC) has announced a significant milestone in the development of its drug candidate PYC-003, designed to treat Polycystic Kidney Disease (PKD). The company’s Safety Review Committee (SRC) has approved an escalation in dosing from 0.4 mg/kg to 1.2 mg/kg in the ongoing Phase 1a Single Ascending Dose (SAD) study involving healthy volunteers. This approval follows a positive safety review of four-week data from the initial dosing cohort.
Advancing Toward Patient Dosing
With dosing now underway in the second cohort, PYC Therapeutics anticipates that, pending a successful safety review in July, the trial will escalate further to 2.4 mg/kg. Crucially, this progression will also trigger the initiation of dosing in patients with PKD in Part B of the Phase 1a study, expected in the second half of 2025. This marks a pivotal step as the company moves from healthy volunteers to the target patient population.
Strategic Clinical Development Pathway
The Phase 1a SAD study is part of a broader clinical development plan that includes an Open-Label Extension (OLE) study and a Phase 1b Multiple Ascending Dose (MAD) trial. These studies aim to refine dosing regimens and further evaluate safety, tolerability, and early efficacy signals. Successful completion of these early phases will pave the way for a combined Phase 2/3 registrational trial, which is critical for supporting a New Drug Application for PYC-003.
Addressing an Unmet Medical Need
Polycystic Kidney Disease affects over 10 million people worldwide and currently lacks effective treatment options. PYC Therapeutics’ approach targets the genetic root cause of PKD, leveraging its proprietary RNA therapy platform. This precision medicine strategy could represent a breakthrough for patients who have long awaited viable therapeutic alternatives.
Looking Ahead
As PYC Therapeutics advances through these critical clinical milestones, the biotech sector and investors will be watching closely. The company’s progress not only reflects its scientific capabilities but also the growing promise of RNA-based therapies in tackling complex genetic diseases.
Bottom Line?
PYC’s dosing escalation approval sets the stage for patient trials that could redefine treatment for Polycystic Kidney Disease.
Questions in the middle?
- Will the safety data from the 1.2 mg/kg cohort support further dose escalation and patient dosing?
- How will PYC-003 perform in terms of efficacy once tested in PKD patients?
- What regulatory hurdles remain before PYC-003 can advance to Phase 2/3 trials?
