PYC Therapeutics Begins Global Dosing Trial for Inherited Blindness Treatment

PYC Therapeutics (ASX: PYC) has initiated a global Phase 1b repeat dose clinical trial for its lead investigational RNA therapy, PYC-001, designed to treat Autosomal Dominant Optic Atrophy (ADOA), a rare genetic eye disease with no approved treatment.

The trial, named the MYRTLE study, has officially dosed its first patient. The objective of the study is to establish the safety, tolerability and early efficacy signals of repeated dosing with PYC-001, with the aim of progressing to a registrational trial in 2027.

ADOA affects approximately one in 35,000 people and is caused by mutations in the OPA1 gene, leading to the gradual loss of retinal ganglion cells and progressive vision loss. PYC-001 targets the underlying genetic cause of the disease, offering a potential therapeutic breakthrough for an unmet medical need.

The MYRTLE study will evaluate three different doses—10, 30 and 60 micrograms per eye—across varying intervals of eight and twelve weeks. Most patients will receive three doses of the drug in one eye, administered via intravitreal injection. In the highest dose group, patients will first receive a single dose, and pending safety clearance, can roll into the repeat dosing arm.

Three patients will be enrolled in each dosing cohort. For the 60 microgram dose, a safety review committee will assess four-week follow-up data before allowing progression to repeat dose administration. The primary goal is to determine the optimal dosing regimen, with secondary measures focused on gathering preliminary evidence of clinical efficacy.

The treatment period is scheduled to conclude by the December quarter of 2026, with data across both safety and efficacy endpoints expected in the second half of the year.

Following the Phase 1b trial, PYC plans to conduct an open-label extension study to further build clinical proof-of-concept. This staged approach is intended to support a future New Drug Application for PYC-001.

PYC’s broader strategy focuses on developing RNA-based therapies for monogenic diseases—conditions caused by mutations in a single gene. The company uses a proprietary delivery platform to enhance the potency and cellular uptake of its drug candidates. With three clinical programs in development, PYC is positioning itself as a leader in the emerging field of precision RNA therapeutics.

As the MYRTLE study progresses, the company continues to engage with patients and global research centres in an effort to advance toward a commercially viable treatment for a condition that currently has no therapeutic alternatives.