PYC Therapeutics Shows Durable Vision Gains and Gene Expression in ADOA Trials
PYC Therapeutics reports durable retinal gene expression and visual improvements from its PYC-001 candidate in Autosomal Dominant Optic Atrophy, supporting longer dosing intervals and expansion into glaucoma research.
- Non-human primate data shows sustained OPA1 protein increase at 4 months post-dose
- ADOA patients exhibit lasting visual acuity improvement and reduced retinal stress
- Favourable safety profile with no treatment-related serious adverse events
- Clinical protocols to be reviewed for extended dosing intervals beyond 4 months
- Pre-clinical glaucoma studies underway to explore broader applications
Sustained Retinal Gene Expression in Preclinical Models
PYC Therapeutics (ASX:PYC) has unveiled compelling non-clinical data showing that a single dose of its RNA therapy candidate PYC-001 leads to a sustained increase in OPA1 protein expression in the retina of non-human primates (NHPs) for at least four months. This protein is crucial because its deficiency underlies Autosomal Dominant Optic Atrophy (ADOA), a progressive blinding disease. The treated primates received a 15 microgram dose per eye, equivalent to the 30 microgram dose currently tested in human trials, and demonstrated statistically significant elevation in OPA1 levels compared to controls.
Clinical Trial Patients Show Lasting Vision Benefits
Complementing the preclinical findings, patients enrolled in PYC’s ongoing Phase 1/2 MYRTLE trial have exhibited sustained improvements in low contrast visual acuity alongside decreased retinal stress markers, as measured by flavoprotein fluorescence imaging. These functional gains have persisted following multiple doses of PYC-001 administered every 2-3 months. Importantly, the therapy continues to maintain a clean safety profile, with no treatment-related serious adverse events reported to date.
Extending Dosing Intervals and Expanding Indications
The prolonged gene expression observed in NHPs supports extending the dosing interval in clinical trials beyond the current 2-3 months to potentially once every four months or longer. PYC plans to amend its clinical protocols accordingly, subject to regulatory review and ongoing trial outcomes. This extension could improve patient convenience and adherence, a significant consideration in chronic genetic diseases.
Beyond ADOA, PYC is actively investigating PYC-001’s potential in other blinding eye conditions where OPA1 expression plays a role, notably glaucoma. Preclinical models are underway to evaluate neuroprotective effects, with plans to initiate Phase 2 studies in glaucoma patients if results are favourable. This strategic expansion reflects the broader translational promise of PYC-001 within ophthalmology.
Positioning in the RNA Therapeutics Landscape
PYC Therapeutics continues to carve out a niche in precision RNA medicines targeting monogenic diseases, leveraging proprietary delivery platforms to enhance potency. The company’s pipeline includes multiple clinical-stage programs, with PYC-001 at the forefront for genetic eye diseases. The latest data reinforce PYC’s commitment to addressing unmet needs in rare blinding disorders, an area with limited treatment options and significant patient impact.
Bottom Line?
PYC’s data suggest a promising step toward less frequent dosing and broader applications for PYC-001, though clinical protocol changes and glaucoma trials remain contingent on forthcoming regulatory and trial developments.
Questions in the middle?
- Will extended dosing intervals maintain efficacy and safety in larger patient cohorts?
- How quickly can PYC advance PYC-001 into Phase 2 glaucoma trials based on preclinical results?
- What regulatory hurdles might affect protocol amendments for dosing schedules in ADOA?
