Eye health has been in and out of sight and mind for some time now, urging organisations to demand greater investment and attention towards Aussies’ unmet needs. In 2018, 93% of Aussies aged over 65 reported vision disorders, and the pandemic did little to help by not being a sight for sore eyes. If that wasn’t enough, leaving these issues unaddressed has not been cheap. A report found that vision loss costs the Aussie economy over $25 billion every year. Circumstances like these shine a light on the importance of greater research into vision loss, and companies like Biotech company PYC Therapeutics (ASX: PYC) have taken matters into their own hands.

PYC has successfully given the first patient a dose of its experimental drug VP-001 as part of the Platypus Study. This study is focused on evaluating the safety and tolerability of VP-001 in a specific eye disease called Retinitis Pigmentosa Type 11 (RP11).

The patient with RP11 received a single dose of 3 μg of VP-001 through an injection into the eye. Two more RP11 patients are expected to receive the same low dose (3 μg) as part of this phase of the study. After that, there will be a 4-week period to review the safety data. The Safety Review Committee will assess the data from the first dose group and decide if the next group, which will receive a slightly higher dose (10 μg), can proceed. The outcome of this Safety Review Committee will be an important milestone in the study, and the Company will inform the market about it.

PYC’s CEO Dr Rohan Hockings commented, “We’re incredibly eager to see the potential benefit our VP-001 drug candidate can deliver for RP11 patients who have a substantial unmet need. This is an outstanding milestone for PYC that is the culmination of an incredible amount of scientific nous and hard work from the team.”

VP-001 represents a potential treatment for Retinitis Pigmentosa Type 11 (RP11), a debilitating eye disease that starts during childhood and eventually causes legal blindness in middle age. RP11 is a rare condition, affecting approximately 1 in every 100,000 people. It is characterised by insufficient expression of the PRPF31 gene in the retina. Currently, there are no available treatment options for RP11 patients, and no treatments are being developed in clinical trials.

VP-001 is an innovative precision therapy designed to address this unmet medical need.

The treatment’s primary objective is to restore the expression of the PRPF31 gene to the levels necessary for the normal functioning of the retina. What sets VP-001 apart is its utilisation of PYC’s proprietary drug delivery technology, which overcomes a significant challenge faced by RNA drugs. This technology ensures that the drug effectively reaches its target within the affected cells of RP11, increasing the chances of a successful treatment outcome.

The Safety Review Committee (SRC) responsible for overseeing the study. Once the final patient in a particular group has completed the safety review process, the trial will progress to the next group and dose level. After the highest tolerated dose cohort has received their doses, a 24-week safety follow-up assessment will be conducted to monitor any treatment-related serious adverse events that may emerge during that period.

This assessment will provide additional information about the safety profile of VP-001 and its potential effects over an extended timeframe.

Having begun its clinical trials in full force, PYC’s VP-001 is edging closer to commercialisation. Plus, that it’s attempting to enter a $1 billion addressable market is incentive enough to make sure all goes well.