Australian biotech PYC Therapeutics (ASX:PYC) has taken a major step forward with its lead program, presenting clinical data that signals a significant opportunity in the treatment of genetic eye diseases — a space where no treatments currently exist.

The company announced that interim results from its Phase 1/2 trials for VP-001 — a drug candidate targeting Retinitis Pigmentosa type 11 (RP11) — will be presented at two major international conferences in May.

Showcasing Clinical Success

PYC announced that VP-001 has demonstrated a favourable risk-benefit profile in ongoing open-label Phase 1/2 studies. 

The data reveal that treatment with VP-001 led to statistically significant improvements in vision on registrational endpoints. Specifically, patients showed notable gains in Low Luminance Visual Acuity and microperimetry compared to both their untreated ‘fellow’ eye and the natural history of the disease.

Delivering Clinically Meaningful Improvements

PYC highlighted that these improvements have already translated into meaningful day-to-day benefits for patients.

The company noted that patients who received the therapy experienced clearer central vision and reduced haze. Some even required updated eyeglass prescriptions due to the improvement in their treated eye.

One participant described the moment they first noticed the treatment’s effects: seeing the white lid of a coffee cup for the first time in years. Another reported being able to see airplanes and stars at night — everyday sights that had previously been invisible to them.

Prioritising Safety and Tolerability

Importantly, VP-001 appears to be safe and well-tolerated.

PYC noted that there have been no treatment-related or procedure-related serious adverse events reported in any patient who has received VP-001 to date.

This clean safety profile could set PYC’s proprietary RNA platform apart from other precision medicine approaches for blinding eye diseases, opening up future potential to expand into other indications with significant unmet need.

Accelerating Toward a New Drug Application

Following these promising results, PYC has confirmed a critical next step.

The company will meet with the US Food and Drug Administration (FDA) on 6 June 2025 to align on the design of registrational studies intended to support a New Drug Application (NDA) for VP-001.

PYC added that VP-001 has already received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA — a suite of regulatory advantages aimed at expediting the drug’s pathway to market.

Strengthening Market Position in RNA Therapeutics

PYC’s broader strategy leverages its proprietary drug delivery technology to enhance the potency of RNA therapies, a drug class that has gained significant traction globally.

The company’s focus on monogenic diseases — where a single gene mutation drives the condition — positions it in a high-probability development segment within biotech.

With three clinical-stage programs currently underway, PYC is working to carve out a leadership role in the treatment of severe, previously untreatable genetic disorders.

Preparing for Major Industry Showcases

The new data will be unveiled at two important events: the Foundation Fighting Blindness Retinal Therapeutics Innovation Summit on 2 May 2025, and the Association for Research in Vision and Ophthalmology (ARVO) conference between 4–8 May 2025, both in Salt Lake City, Utah.

If registrational studies proceed successfully, VP-001 could become the first approved treatment for RP11, offering a lifeline to patients who currently face inevitable vision loss.