PYC Therapeutics Ltd. announced that it has completed single-dose Good Laboratory Practice (GLP) toxicology studies to enable progression of its investigational drug candidate (VP- 001) for the treatment of Retinitis Pigmentosa type 11 (RP11) into first in human studies. PYC combines two complementary platform technologies - RNA drug design capabilities and a proprietary drug delivery technology - to create a new generation of RNA therapeutics to change the lives of patients with genetic diseases. The results of the single-dose GLP toxicology studies showed no observed adverse effects at any of the assessed doses.

The competitive advantages of PYC's proprietary RNA therapeutics platform for the treatment of retinal disease are now apparent across both efficacy and safety/tolerability dimensions. These toxicology studies were completed under GLP conditions, a standard required for submissions to regulatory bodies including the United States (US) Food and Drug Administration (FDA). PYC anticipates filing an Investigational New Drug (IND) application in support of VP-001 in Fourth Quarter 2022 and, if successful, progressing to human clinical trials in First Quarter 2023.

Two single-dose GLP toxicology studies were conducted, one in rabbits and the other in non-human primates (NHPs). In each case, low, medium, and high doses of VP-001 were administered and evaluated following a single dose of VP-001 in addition to a control group. All dosing was bilateral, and administration of VP-001 was by injection into the vitreous of the eye, the same route of administration anticipated to be used in human clinical trials.

The single-dose animal evaluations were conducted for a duration of 12 weeks. The information reported here contains data through completion of the studies at week 12 following single doses of VP-001 at each of the three doses assessed in both species. Safety results from the GLP toxicology study in rabbits, which were administered at up to 10 µg/eye of VP-001, showed that no drug-related mortality, changes in health and behaviour or visual function were observed through the 12-week study period.

Transient anterior segment inflammation was observed in some animals administered 10 µg/eye, the higher dose evaluated, on Day 3 that rapidly resolved by Day 8. Safety results from the GLP toxicology study in NHPs, which were administered up to 50 µg/eye of VP-001, showed that no drug-related mortality, changes in health and behaviour or visual function were observed through the 12-week study period. Transient anterior segment inflammation, on Days 3 through 15, which was observed in animals administered 50 µg/eye, the higher dose, resolved by week 4. All doses tested in these single-dose GLP toxicology studies in rabbits and NHPs were well tolerated and safe through-out the 12-week study. The higher tested dose of VP-001 in each species was considered to be the no observed adverse effect level (NOAEL) for the purpose of submitting the IND application.

These results represent the final piece of PYC's non-clinical data pack required for the IND in support of VP-001. Progression of VP-001 into a combined phase 1/2 clinical study will represent the first potentially disease-modifying therapy for RP11 to progress into clinical development. PYC is looking forward to completing its transition to a clinical-stage and multi-asset drug development company, and to scaling its drug discovery and development capabilities across multiple programs within its pipeline to change the lives of patients with major unmet needs in rare genetic diseases.