PYC Therapeutics (ASX:PYC) says it has reached regulatory alignment with the United States Food and Drug Administration on the design of a registrational study for its investigational therapy VP 001, a drug candidate that could become the first approved treatment for patients with the rare genetic eye disease Retinitis Pigmentosa type 11.
The Perth and San Francisco-based biotechnology company announced that discussions with the FDA confirmed key elements of the clinical trial required to support a future New Drug Application for the therapy. The company is developing VP 001 to address the underlying genetic cause of RP11, a progressive condition that leads to severe vision loss and blindness.
According to the company, the therapy “has the potential to become the first approved treatment option for patients with the blinding eye disease Retinitis Pigmentosa type 11.”
The outcome of the FDA meeting establishes the framework for a pivotal study designed to measure whether the therapy can improve vision in people living with the disease. The trial will evaluate the mean change from baseline in low-luminance visual acuity after 36 months of treatment, with a target of a 15-letter improvement in favour of VP 001 compared with the sham-treated group.
Secondary measures will assess both improvement and protection against further vision loss. These include the proportion of patients achieving a ten-letter improvement in visual acuity as well as the proportion who avoid losing ten letters over the same period. Additional outcomes will monitor retinal sensitivity and the rate of structural changes in photoreceptors over time.
Regulators indicated that the study outcome would not be judged solely on whether the primary threshold is reached. The FDA confirmed that failure to meet the 15-letter benchmark would not necessarily prevent approval if the broader results supported the therapy’s safety and effectiveness. Regulators said the final decision would depend on the “totality of evidence” once the trial is complete.
The agency also agreed that PYC may combine results from its ongoing natural history study of RP11 patients with data from the randomised sham control group in the registrational trial to strengthen the interpretation of treatment effects.
As part of the regulatory pathway, the FDA highlighted the option of a Special Protocol Assessment process. This would allow the company to formally confirm details of the final study design, including the clinical protocol and statistical analysis plan, before launching the registrational trial.
PYC expects additional long-term clinical data from its ongoing Phase 2 study of VP 001 later this year. The update will include results from patients who have received the therapy for more than twelve months and will help determine the next steps toward initiating the pivotal study.
The company describes itself as a precision medicine developer focused on genetic diseases that currently have no effective treatment options. Alongside the RP11 program, PYC is advancing additional RNA-based therapies targeting the underlying causes of inherited disorders.