Safety First: What PYC-001’s Phase 1 Results Mean for Future ADOA Treatments
PYC Therapeutics has completed dosing in its Phase 1 study of PYC-001 for Autosomal Dominant Optic Atrophy, showing strong safety and early signs of improved vision. The company plans to launch a global Phase 1/2 trial later this year.
- Completed dosing in Phase 1 Single Ascending Dose study of PYC-001
- No serious adverse events reported in treated patients
- Early indications of improved visual acuity in treated eyes
- Presentation of clinical data scheduled for NOSA 2025 conference
- Global Phase 1/2 Multiple Ascending Dose study planned for Q4 2025
Background on ADOA and PYC-001
Autosomal Dominant Optic Atrophy (ADOA) is a progressive, inherited eye disease that leads to irreversible vision loss, typically manifesting in childhood. Affecting approximately 1 in 35,000 people, it currently has no approved treatments. The condition stems from mutations in the OPA1 gene, which impairs mitochondrial function in retinal ganglion cells, critical for vision.
PYC Therapeutics is developing PYC-001, an innovative RNA-peptide conjugate designed to increase OPA1 protein levels and restore mitochondrial health. Administered via intravitreal injection, PYC-001 aims to address the root cause of ADOA in a mutation-independent manner.
Phase 1 Single Ascending Dose Study Results
The company recently completed dosing in its Phase 1 Single Ascending Dose (SAD) study, enrolling nine patients across three dose cohorts. The study demonstrated a strong safety profile, with no treatment-emergent serious adverse events or intraocular inflammation reported. Most adverse events were mild and related to the injection procedure itself.
Importantly, early exploratory efficacy data showed encouraging trends toward improved visual acuity in the treated eyes compared to baseline and untreated fellow eyes. While preliminary, these findings suggest PYC-001 may positively impact vision in patients with this currently untreatable disease.
Next Steps and Clinical Development Pathway
PYC Therapeutics will present these clinical data at the Neuro-Ophthalmology Society of Australia (NOSA) conference in Auckland from 11-13 September 2025. Following this, the company plans to initiate a global Phase 1/2 Multiple Ascending Dose (MAD) study in the fourth quarter of 2025. This next trial will evaluate repeated dosing to better understand safety, tolerability, and efficacy, with the goal of establishing clinical proof-of-concept.
Success in the MAD study would pave the way for a registrational trial aimed at supporting a New Drug Application for PYC-001, potentially bringing the first treatment option to patients suffering from ADOA.
Broader Implications for RNA Therapeutics
PYC Therapeutics is leveraging its proprietary RNA delivery platform to target monogenic diseases with high unmet need. The progress of PYC-001 underscores the growing promise of RNA-based precision medicines to tackle genetic disorders that have long eluded effective treatment.
While challenges remain, including confirming long-term efficacy and navigating regulatory pathways, the encouraging early clinical data mark an important milestone for the company and the field.
Bottom Line?
PYC-001’s promising Phase 1 safety and efficacy signals set the stage for pivotal trials that could transform treatment for a rare blinding disease.
Questions in the middle?
- Will the Phase 1/2 MAD study confirm and extend the early efficacy signals seen in the SAD trial?
- What dosing regimen will optimize safety and visual improvement in ADOA patients?
- How will regulatory agencies view PYC-001’s clinical data in the context of no existing treatments?