PYC Therapeutics has successfully completed the first cohort dosing of healthy volunteers in its Phase Ia single ascending dose (SAD) trial for PYC-003, a drug candidate targeting the underlying cause of polycystic kidney disease (PKD). The Safety Review Committee (SRC) has reviewed the initial four-week safety data and authorized dose escalation for the second cohort.
Safety Review Committee Approves Dose Escalation
The SRC approved a dosage escalation from 0.4mg/kg to 1.2mg/kg for the second cohort. The committee is scheduled to meet again in July to review data from this cohort. A positive outcome will trigger further dose escalation to 2.4mg/kg in healthy volunteers and initiate dosing at 0.4mg/kg in PKD subjects.
The company has begun dosing the second cohort in the SAD trial's part A and expects to begin part B in the third quarter, pending SRC approval of the safety and tolerability data from the second cohort.
Trial Design and Future Development Plans
Following parts A and B, PYC Therapeutics plans an open-label extension (OLE) trial and a Phase Ib multiple ascending dose (MAD) randomized controlled trial to further assess the drug's optimal dosing regimen, tolerability, efficacy and safety.
The completion of the Phase Ia/Ib trial is expected to pave the way for a registrational combined Phase II/III trial, which will support a new drug application for PYC-003.
Primary and Secondary Endpoints
The primary goal of the Phase Ia SAD trial is to assess the tolerability and safety profile of the candidate, with a secondary aim to measure its efficacy when the trial extends to PKD subjects in the second half of 2025.
Company Platform and Focus
PYC Therapeutics leverages its drug delivery platform to improve the precision medicine potency within the RNA therapeutic class. Its drug development programmes focus on monogenic conditions.
