PYC Therapeutics (ASX:PYC) has completed dosing for patients in cohort one of the Platypus clinical trial.
This early-phase single ascending dose study of the investigational drug candidate VP-001 is in patients with a blinding eye disease, Retinitis Pigmentosa type 11 (RP11).
RP11 is a blinding eye disease that begins in childhood and ultimately leads to legal blindness in middle age. It is caused by insufficient expression of the PRPF31 gene in the retina. VP-001 is a precision therapy that aims to restore the expression of the PRPF31 gene to levels required for the normal retina function.
The company said VP-001 is the first drug with disease-modifying potential to have entered human studies in patients. The US FDA recently granted it Fast Track status.
The company said the trial's Safety Review Committee (SRC) will meet in September to review the initial data generated for patients in cohort one and consider approval to escalate to dosing in cohort two.
Subject to SRC approval, PYC said it remains on track to complete dosing for patients in cohorts two and three before the end of 2023.
PYC said it expects to transition to a Phase 2 multi-dose study beginning in the middle of next year on successful completion of the ongoing Phase 1 study.