PYC Therapeutics (PYC) Investor presentation summary

Disease background and prevalence

• PRPF31-related retinitis pigmentosa (RP11) is a rare inherited retinal disease caused by haploinsufficiency of the PRPF31 gene, affecting vision through impaired splicing of key retinal proteins.

• In Western Australia, 40 individuals are affected, extrapolated to over 400 in Australia and 4,500 in the USA.

• Non-penetrance in RP11 is linked to increased expression of the wild-type PRPF31 allele, suggesting a therapeutic approach.

VP-001 mechanism and preclinical data

• VP-001 is a peptide-oligonucleotide conjugate designed to upregulate PRPF31 expression and rescue haploinsufficiency in RP11.

• In patient-derived models, VP-001 increased PRPF31 mRNA and protein to wild-type levels and improved cellular structure.

Clinical development and trial design

• The clinical program includes open-label Phase 1/2 studies (Platypus, Wallaby, Dingo) with endpoints focused on safety and low-luminance visual acuity (LLVA).

• 17 patients have received ≥4 doses of VP-001 with at least 7 months of follow-up.

• A potential registrational trial is planned with 90 patients, primary endpoint of mean change in LLVA, and a 48-month duration.