PYC Therapeutics (ASX:PYC) has confirmed that the Safety Review Committee (SRC) governing its phase 1 clinical trial or patients with a blinding eye disease called Retinitis Pigmentosa type 11 (RP11) has approved the escalation of dosing to patient cohort 2.
This approval comes following review of the four-week safety/tolerability data from the first cohort of patients dosed with the investigational drug candidate.
PYC will now proceed to enrolment and dosing of patients in the second dose cohort evaluating a dose of 10 micrograms of the investigational drug in patients with Retinitis Pigmentosa type 11.
Subject to SRC approval, PYC remains on track to complete dosing for patients in cohorts 2 and 3 before the end of 2023.
PYC expects to transition to a Phase 2 multi-dose study beginning in the middle of next year on successful completion of the ongoing Phase 1 study.
Platypus Phase 1 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 thre 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, 4 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.
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 around 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.
