PYC Therapeutics (ASX:PYC) has reported encouraging long-term data from its ongoing Phase 1/2 clinical program for VP-001, an investigational treatment targeting Retinitis Pigmentosa type 11 (RP11).
The company states that patients receiving the therapy are experiencing sustained improvements in vision for up to 18 months after treatment, an outcome that positions VP-001 as a potential first-in-class therapy for a disease currently lacking approved options.
RP11 is a childhood-onset, hereditary retinal disorder caused by a mutation in a single gene, leading to progressive vision loss and ultimately resulting in blindness. VP-001 is designed to correct the underlying defect by rescuing the gene expression deficit responsible for the disease. As one of three clinical-stage programs being advanced by PYC, the candidate represents a key focus of the company’s precision medicine pipeline.
According to PYC, data from the Phase 1/2 studies show that the treated eye consistently outperforms both the untreated 'fellow' eye and the outcomes seen in the natural history control group. Improvements span all registrational endpoints measured, including low-luminance visual acuity and microperimetry. Patients have demonstrated meaningful gains in visual function, such as improvements of 10 or more letters in low-luminance acuity, and enhanced retinal sensitivity, with multiple retinal loci showing increases of at least 7 decibels.
Safety results also continue to be favourable. Across all patients treated to date, no treatment-related serious adverse events have been reported, supporting VP-001’s potential as a well-tolerated therapy for a genetically defined population with significant unmet need.
PYC is preparing to engage with the US Food and Drug Administration to finalise its registrational trial design, with plans to commence the pivotal study in 2026. The company notes that timelines remain subject to the usual development risks and regulatory considerations.