PYC Therapeutics Launches Global Repeat Dose Trial for Vision-Restoring Drug
PYC Therapeutics has kicked off a global Phase 1b repeat dose study of its investigational drug PYC-001 targeting Autosomal Dominant Optic Atrophy, a rare genetic eye disease with no current treatments. Initial dosing has begun, with key data expected in the second half of 2026.
- Initiation of global Phase 1b repeat dose MYRTLE study for PYC-001
- Targeting Autosomal Dominant Optic Atrophy (ADOA), a rare blinding disease
- Study to evaluate safety, tolerability, and efficacy across multiple dosing regimens
- Data anticipated in H2 2026 to establish clinical proof of concept
- Plans for registrational trial and New Drug Application submission in 2027
PYC Therapeutics Advances Clinical Program for Rare Eye Disease
PYC Therapeutics Limited (ASX – PYC) has announced a significant milestone in its pursuit to develop treatments for genetic diseases, initiating a global repeat dose Phase 1b clinical trial of its lead drug candidate, PYC-001. This investigational therapy targets Autosomal Dominant Optic Atrophy (ADOA), a rare inherited condition that leads to progressive vision loss and currently has no approved treatments.
The MYRTLE study, as it is called, marks the first time PYC-001 is being administered repeatedly to patients worldwide. The trial aims to assess the safety, tolerability, and preliminary efficacy of multiple dosing regimens, with patients receiving three doses in a single eye via intravitreal injection. Different dose levels; 10, 30, and 60 micrograms; and dosing intervals of 8 and 12 weeks will be evaluated to determine the optimal therapeutic approach.
Design and Expectations of the MYRTLE Study
The study design includes cohorts of patients receiving varying doses and intervals, with an initial safety review planned after early data from the highest dose group. The primary goal is to establish a safety profile and identify the best dosing strategy, while secondary objectives focus on gathering early signals of efficacy. The treatment period is expected to conclude by the fourth quarter of 2026, with data readouts anticipated in the latter half of the year.
Following the Phase 1b study, PYC plans to initiate an Open-Label Extension study to further evaluate PYC-001’s effects. These steps are critical to building clinical proof of concept ahead of a planned global registrational trial in 2027, which will support a New Drug Application submission for regulatory approval.
Implications for Patients and the RNA Therapeutics Landscape
ADOA affects approximately one in every 35,000 individuals and currently has no approved therapies, leaving patients with limited options. PYC Therapeutics’ approach leverages precision RNA medicine, aiming to address the underlying genetic cause of the disease rather than just managing symptoms. This trial represents a hopeful advance for patients and highlights the growing momentum of RNA-based therapies in treating rare genetic disorders.
With operations spanning Perth and San Francisco, PYC is positioning itself at the forefront of genetic medicine innovation. The company’s proprietary drug delivery platform enhances the potency of RNA therapies, targeting monogenic diseases where clinical success rates tend to be higher. The MYRTLE study’s progress will be closely watched by investors and the biotech community as a bellwether for PYC’s broader pipeline and strategic direction.
Bottom Line?
As PYC Therapeutics embarks on this pivotal trial, the coming year will be crucial in validating PYC-001’s potential to transform treatment for a rare blinding disease.
Questions in the middle?
- Will the MYRTLE study demonstrate a clear safety and efficacy profile for PYC-001?
- How will dosing intervals and amounts impact patient outcomes and tolerability?
- What regulatory hurdles might PYC face ahead of the planned 2027 registrational trial?
