PYC Therapeutics (ASX: PYC) has completed dosing of the first patient with its drug candidate VP-001 as part of the Platypus Study – a Phase 1 Single Ascending Dose (SAD) study designed to assess the safety and tolerability of VP-001 in the blinding eye disease Retinitis Pigmentosa Type 11 (RP11).
The RP11 patient who received VP-001 was dosed with a three μg single administration to one eye via an intravitreal (into the eye) injection. A further two RP11 patients are expected to be dosed as part of the low (3 μg) dose cohort followed by a four-week safety review period. The Safety Review Committee will review the safety data from the first dose cohort at the completion of the review period and determine whether the next dosing cohort (mid dose – 10 μg) may proceed.
PYC will advise the market of the outcome of the Safety Review Committee as the next key milestone of this study.
About VP-001 – the first potential treatment for 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 ~one in every 100,000 people and is caused by insufficient expression of the PRPF31 gene in the retina.
There are currently no treatment options available for patients with RP11 nor are there any in clinical development.
VP-001 is a precision therapy that aims to restore the expression of the PRPF31 gene back to levels required for the normal function of the retina. VP-001 utilises PYC’s proprietary drug delivery technology to overcome the major challenge for RNA drugs by ensuring that sufficient drug reaches its target inside the cells affected by RP11.
About the Phase 1 Single Ascending Dose (SAD) Study
The Phase 1 open label study will be conducted to evaluate the safety and tolerability of a single dose of VP-001 to a single eye administered intravitreally in participants over the age of 18 with confirmed PRPF31 mutation-associated retinal dystrophy (RP11 patients).
Three groups of patients will be administered a single dose (low, mid and high dose) with each cohort consisting of three patients with RP11.
The Safety Review Committee (SRC) for the study will review the safety data for each cohort of patients dosed with VP-001, four weeks after the first dose is administered. When the final patient in the relevant cohort has progressed through the SRC, the trial will progress to the next cohort/dosing group.
On completion of the dosing of the highest tolerated dose cohort, a 24-week safety follow- up assessment will be conducted to assess treatment-emergent serious adverse events.
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,” CEO, Dr Rohan Hockings, said.
“This is an outstanding milestone for PYC that is the culmination of an incredible amount of scientific nous and hard work from the team.”
