PYC Therapeutics (ASX:PYC) has taken a significant step forward in its goal to deliver the first treatment for Autosomal Dominant Optic Atrophy (ADOA), announcing the initiation of a global repeat-dose Phase 1b clinical study of its investigational RNA therapy, PYC-001.
The study, called MYRTLE, is designed to establish clinical proof of concept for the treatment, which targets the genetic cause of the disease. The first patient has now received their initial intravitreal dose of PYC-001.
The MYRTLE study will evaluate three different doses across 8- and 12-week dosing intervals. Initial single-dose cohorts will inform dose escalation, followed by repeat-dose cohorts to assess safety, tolerability and preliminary efficacy signals. The company expects data readouts in the second half of 2026, with plans to advance to a global registrational trial in 2027.
ADOA affects approximately one in 35,000 people worldwide and currently has no approved treatments. Caused by mutations in the OPA1 gene, it leads to progressive vision loss and, in many cases, legal blindness. PYC-001 aims to address the underlying cause of the disease. The company said the program reflects its broader commitment to developing precision RNA therapies for patients with no available treatment options.
Following the Phase 1b study, an Open-Label Extension is planned to build a more complete understanding of the therapy’s performance over time. These studies are intended to provide the foundation for a future New Drug Application for PYC-001.
PYC Therapeutics is advancing a pipeline of RNA therapies using its proprietary delivery platform, focused on monogenic diseases to maximise the likelihood of clinical success. If successful, PYC-001 could become the first approved treatment for ADOA, offering new hope to patients and their families.