Retinitis pigmentosa type 11 (RP11) is a blinding eye disease that begins in childhood and ultimately leads to legal blindness in middle age. The disease affects approximately one in every 100,000 people and is caused by insufficient expression of the PRPF31 gene in the retina.
In Perth there is a well-characterised, identified patient population with RP11. They don’t have any treatment options and nor have there been any in clinical development. Associate Professor Fred Chen is changing this by dedicating his career to solving the mystery behind inherited retinal diseases, the leading cause of youth blindness.
Associate Professor Fred Chen is dedicating his research to finding the causes and treatments for inherited retinal diseases
New drug being trialled
His research has led to a drug called VP-001 being developed to help treat RP11. The drug commenced phase one human trials in 2023.
Vision Pharma, a joint venture between the Lions Eye Institute and PYC Therapeutics, is the company bringing this drug to market.
In November 2023, PYC announced that it completed dosing in the second cohort of patients in its phase one clinical trial. These patients have received the lowest dose of the drug as part of the single ascending dose phase one study. Once approval is received from the Food and Drug Administration, higher doses othe drug will be given to the next cohort.
This milestone marks the completion of dosing in the second of three planned cohorts of RP11 patients. PYC is accelerating plans to initiate a mid-late stage multiple-dose clinical trial of this drug and now plans to start the phase two trial
in 2024.
Philanthropy an essential ingredient
Philanthropic supporters, government grants and other grant funding have played a critical role in this groundbreaking scientific work. Philanthropic supporters include:
- Bowen Foundation
- Channel 7 Telethon Trust
- Constantine Family Foundation
- Norman and Freda Hogg
- McCusker Charitable Foundation
- Brad and Caroline Miocevich
- John and Lee Saleeba
- Rhonda Wyllie
