PYC Therapeutics Advances PKD Drug Candidate with Key Safety Approval
PYC Therapeutics has secured Safety Review Committee approval to escalate dosing in its PYC-003 clinical trial and will soon begin dosing patients with Polycystic Kidney Disease, marking a pivotal step forward.
- Safety Review Committee approves dose escalation to cohort 3 in healthy volunteers
- Initiation of Part B dosing in Polycystic Kidney Disease patients imminent
- On track for repeat dose studies in PKD patients by Q4 2025
- Progression toward registrational Phase 2/3 trial for PYC-003
- PYC-003 targets the underlying cause of Polycystic Kidney Disease
Clinical Progress in PKD Treatment
PYC Therapeutics Limited (ASX – PYC) has announced a significant milestone in the clinical development of its drug candidate PYC-003, designed to tackle Polycystic Kidney Disease (PKD) at its root cause. The company received approval from its Safety Review Committee (SRC) to escalate dosing to the third cohort in the initial phase of its Single Ascending Dose (SAD) study conducted in healthy volunteers.
This approval not only allows the company to increase dosage safely but also greenlights the commencement of Part B of the SAD study, which involves dosing actual PKD patients. This transition from healthy volunteers to patients marks a crucial step in evaluating the drug’s safety and efficacy in the target population.
Strategic Clinical Trial Design
The SAD study is structured in phases, with Part A focusing on dose escalation in healthy volunteers and Part B extending the investigation to PKD patients. Following these, PYC plans to initiate an Open-Label Multiple Ascending Dose (MAD) study to assess repeat dosing and optimal treatment regimens. This will be complemented by a Phase 1b randomized controlled trial to further evaluate safety and efficacy.
CEO Dr. Rohan Hockings highlighted the importance of the SRC’s decision, noting that the dose used in cohort 2 is expected to be at the upper end of the effective range based on preclinical data. Establishing safety at this level is a pivotal milestone that sets the stage for subsequent pivotal studies.
Looking Ahead – Toward Registrational Trials
Successful completion of these early-phase studies will pave the way for a combined Phase 2/3 registrational trial, which is critical for supporting a New Drug Application for PYC-003. The company remains on track to progress to repeat dose studies in PKD patients by the fourth quarter of 2025, maintaining momentum in its high-velocity clinical development program.
With no current treatments addressing the underlying cause of PKD, PYC-003 represents a promising advancement in precision medicine for patients with this genetic disease. The company’s proprietary RNA therapeutic platform underpins its approach, targeting monogenic diseases with high potential for clinical success.
Bottom Line?
PYC’s clinical progress with PYC-003 signals a promising trajectory toward a novel treatment for PKD, but upcoming trial results will be critical to watch.
Questions in the middle?
- How will PYC-003 perform in terms of efficacy and safety in PKD patients during Part B?
- What are the timelines and potential regulatory hurdles for the planned Phase 2/3 registrational trial?
- How does PYC-003 compare to other emerging therapies targeting Polycystic Kidney Disease?
