Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Neuro-Ophthalmology Society of Australia (NOSA) Annual Scientific Meeting 2026
16 slides from Pyc Therapeutics's conference presentation, Sep 3, 2026
Slide 1: ADOA Program - Data Presentation at NOSA 2026
3 September 2026 ADOA PROGRAM - DATA PRESENTATION AT NOSA 2026 • PYC is a precision medicine company dedicated to changing the lives of patients with genetic diseases who have no treatment options available • The Company is progressing a drug candidate (known as PYC-001) that addresses the underlyin
Slide 2: About PYC Therapeutics and Forward Looking Statements
About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a clinical-stage biotechnology company creating a new generation of RNA therapies to change the lives of patients with genetic diseases. The Company utilises its proprietary drug delivery platform to enhance the potency of precision medicines wit
Slide 3: Phase 1B Study of Intravitreally Administered PYC-001 in OPA1-Associated ADOA
A Phase 1B Open-Label, Randomized, Single Dose For personal use only and Repeat Dose Study to Evaluate the Single and Repeat Dose Safety and Tolerability of Intravitreally Administered PYC-001 in Participants with Confirmed OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy NOSA 2026 Clare Fr
Slide 4: PYC-001 in Confirmed OPA1
For personal use only PYC-001 in Confirmed OPA1 Sundew (single dose) Myrtle (multi dose) NOSA 2026 Clare Fraser Disclosures: – assistance with travel costs to NANOS 2026 from PYC
Slide 5: Overview
Overview • ADOA is a progressive and irreversible PYC-001 is a disease-modifying drug candidate for ADOA in clinical trials blinding eye disease with onset in childhood PYC-001 is a peptide-conjugated oligonucleotide administered intravitreally For personal use only • ADOA affects 1 in every 35,000
Slide 6: PYC-001 Mechanism of Action
PYC-001 3(b) PYC-001 to OPA1 bindsacts PYC-001 directly mRNAon Mechanism the in the toOPA1 5‘UTR of functional action facilitate transcript more to efficient upregulateribosomal OPA1 protein accessexpression and translation For personal use only A) The 5’UTR of the OPA1 mRNA contains a stem-loop str
Slide 7: PYC-001: Updated NHP research
only PYC-001: Updated NHP research ✱ 8×10 6 Representative images fluorescent intensity per RGC Untreated control RNFL use 6×10 6 RGCL (Mean + SEM) personal 4×10 6 PYC-001 15 μg Month 4 RNFL 2×10 6 RGCL For OPA1 RGCL = Retinal Ganglion Cell Layer OPA1 0 RNFL = Retinal Nerve Fiber Layer Cell nuclei (
Slide 8: PYC-001: Background human in-vitro research
PYC-001: Background human in-vitro research In ADOA patient-derived induced pluripotent stem cells (iPSC-RGC) 1 For personal use only a) Upregulates OPA1 protein in a mutation agnostic manner b) Restores mitochondrial structural defects c) Restores cellular bioenergetics Improved mitochondrial Enhan
Slide 9: By restoring OPA1 protein expression, PYC-001 could address the underlying cause of ADOA
By restoring OPA1 protein expression, PYC-001 could address the underlying cause of ADOA ADOA Patient Functional outcome ADOA Patient For personal use only + PYC-001 Haploinsufficient OPA1 protein OPA1 Restoration towards 100% restoration ~50-70% expression Disorganized, wide Cristae Slim, tight and
Slide 10: PYC-001: Patient overview
For personal use only PYC-001: Patient overview • >18 years • haplo-insufficiency OPA1 mutation • vision between 20/40 and 20/200 • mild to moderate RNFL loss (1-3 sectors) on Spectralis glaucoma module • not on idebenone, CoQ10, B vitamin supplements or other nutraceuticals PYC THERAPEUTICS
Slide 11: PYC-001: Safety outcomes
For personal use only PYC-001: Safety outcomes ✓ PYC-001 has been shown to be safe and well-tolerated in ADOA Dose of PYC-001 Number of patients patients1 (number of patients with TR-SAE (%) that have received)1,2 3 mcg (n=3) 0 (0%) ✓ No Treatment Related-Serious Adverse events (TR-SAEs) observed in
Slide 12: PYC-001: Results LCVA over time
PYC-001: Results LCVA over time Low-Contrast Visual Acuity data for ADOA patients enrolled in MAD meeting proposed registrational trial inclusion criteria1 For personal use only 20 Change from baseline in Low-Contrast 15 Improvement Visual Acuity (LCVA) letters 10 5 PYC-001 Treated Untreated 0 Line
Slide 13: PYC-001: Results
PYC-001: Results PYC-001 Treated Eyes Untreated Fellow Eyes For personal use only Improvement Decline Improvement Decline Improvement Improvement Decline Decline Reduced macular stress (FPF) correlates to improved LCVA1 PYC THERAPEUTICS 11 1. All data from MYRTLE patients that have received 10 mcg o
Slide 14: Planned progress: registrational trial to support the first approval in ADOA
Planned progress: registrational trial to support the first approval in ADOA1 2027 Progress to date For personal use only Phase 1a SAD Study Phase 1b MAD Study2 Phase 2/3 Study Cohorts 60 mcg 60 mcg (Q12W) Objectives of Phase 1/2/3 trials of PYC-001 • Safety & Tolerability • Efficacy 30 mcg 30 mcg (
Slide 15: FDA special designations intended to accelerate the path to approval
FDA special designations intended to accelerate the path to approval For personal use only FDA Special Designation Status Benefits Orphan Drug Designation Granted for OPA1- • Tax credits for qualified clinical trials associated vision loss1,2 • Exemptions from some regulatory fees • Potential for 7
Slide 16: PYC-001: Conclusions
PYC-001: Conclusions PYC-001 for Autosomal Dominant Optic Atrophy (ADOA) • PYC-001 is a drug candidate with first in indication potential for the treatment of ADOA1 For personal use only • A single dose of PYC-001: • 2-fold increase in OPA1 protein expression through 4 months in the NHP retina • Res