Clinical-stage biotechnology company PYC Therapeutics (ASX:PYC) has announced the approval to escalate dosing in its ongoing clinical trial for Autosomal Dominant Optic Atrophy, a genetic eye disease that leads to progressive vision loss from childhood and has no current treatment.
The Safety Review Committee overseeing the single ascending dose Phase 1 study has authorized progression from cohort 2 (10 micrograms per eye) to cohort 3 (30 micrograms per eye) following a favourable review of 4-week safety and tolerability data.
The study evaluates PYC-001, a first-in-class investigational therapy designed to restore the expression of the OPA1 gene, which is deficient in ADOA patients.
The therapy uses PYC's proprietary delivery technology to effectively reach affected retinal cells, and has shown promise in both patient-derived retinal models and non-human primates.
The SAD trial is set to run through 2025, with a multiple ascending dose study and open-label extension planned to further assess safety, optimal dosing, and efficacy.
Success in these studies will support a Phase 2/3 registrational trial, with the OLE intended to inform the final trial design, pending regulatory approval.