PYC Therapeutics Limited (ASX:PYC) today announced a significant advancement in its clinical development program for PYC-001, an investigational drug candidate targeting Autosomal Dominant Optic Atrophy (ADOA). PYC Therapeutics Limited is a clinical-stage biotechnology company that creates new generations of RNA therapies, leveraging its proprietary drug delivery platform to address genetic diseases. The company confirmed that the Safety Review Committee (SRC) governing the Phase 1 Single Ascending Dose (SAD) study of PYC-001 has reviewed the 4-week safety and tolerability outcomes for ADOA patients treated with a 60-microgram dose of PYC-001 and approved the progression of this dose to a multiple-dose study.
This approval is a crucial step for PYC-001, which addresses the underlying cause of ADOA, a blinding eye disease for which there are currently no approved treatment options available. ADOA affects approximately 1 in every 35,000 people globally. Following the SRC’s decision, PYC will now add the evaluation of the safety and efficacy profile of repeat doses of 60 micrograms of PYC-001 to the ongoing Phase 1/2 Multiple Ascending Dose (MAD) study. This new cohort will proceed alongside the existing 10 and 30 microgram cohorts within the same study.
The objective of the ongoing Phase 1/2 MAD study is to establish clinical proof-of-concept for PYC-001 before potentially advancing the drug candidate into a global registrational trial. Such a trial would be directed towards supporting a New Drug Application for PYC-001 in ADOA. PYC Therapeutics anticipates presenting safety and efficacy outcomes from this study throughout 2026 and 2027, providing further updates on the drug’s development trajectory.
