PYC Therapeutics (ASX:PYC) has announced progress in the clinical development of its investigational drug candidate PYC-001.
The therapy is being developed to address the underlying cause of Autosomal Dominant Optic Atrophy, a condition that leads to progressive vision loss and currently has no approved treatment options.
The company revealed that an independent Safety Review Committee has completed its assessment of early-stage trial data and cleared the way for the next phase of testing.
After reviewing four-week safety and tolerability outcomes in patients who received a 60 microgram dose, the committee approved advancing this dose to a multiple-dose study, marking a key transition from the initial safety evaluation toward broader clinical investigation.
This development builds on an ongoing Phase 1 program that initially explored single ascending doses. With the new approval, PYC will now expand its work to include repeated dosing at 60 micrograms, alongside existing cohorts receiving 10 and 30 micrograms. The shift is intended to generate a more comprehensive understanding of how the drug performs over time, both in terms of safety and potential therapeutic effect.
The company is positioning this stage of the trial as a step toward establishing clinical proof of concept. If successful, the data generated could underpin progression into a global registrational study aimed at supporting a New Drug Application. PYC has indicated that safety and efficacy results from the ongoing study will be released progressively across 2026 and 2027.
Autosomal Dominant Optic Atrophy affects approximately one in 35,000 people and is caused by genetic mutations that impair the function of retinal cells. By targeting the disease at a molecular level, PYC’s approach aligns with a broader push in precision medicine to develop RNA-based therapies for conditions that have historically lacked viable treatment options.