PYC Therapeutics (ASX: PYC) has announced significant progress in their ongoing Phase 1/2 studies of their drug candidate VP-001. The data will be presented by Associate Professor Fred Chen at the Asia Pacific Vitreo-Retina Society scientific conference. VP-001 is designed to treat Retinitis Pigmentosa type 11 (RP11), a genetic blinding disease in children.
PYC Therapeutics is making notable strides with its VP-001 drug candidate aimed at treating Retinitis Pigmentosa type 11 (RP11). The company is moving towards a registrational trial with the FDA, expected to begin in mid-2025. VP-001 has shown positive results in improving vision in patients, with no serious adverse events reported. The drug has fast-track and orphan drug designation by the FDA, hinting at a potentially accelerated path to market. With an estimated market opportunity of over $1 billion annually, PYC's ambitions include delivering further human efficacy data in the next year and expanding their RNA therapy pipeline. Their approach targets genetic causes of disease, offering innovative solutions in the biotechnology field.
The ongoing Phase 1/2 studies of VP-001 have shown promising results in improving visual function in patients with RP11. We observed significant improvements in Low Luminance Visual Acuity and microperimetry compared to baseline and untreated eyes. Importantly, no treatment-emergent serious adverse events have been reported, which underscores the safety and tolerability of VP-001. We are excited to present these findings at the APVRS conference, as they represent a significant step forward in addressing a previously unmet medical need.