Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — North American Neuro-Ophthalmology Society (NANOS) 2026 Conference
16 slides from Pyc Therapeutics's conference presentation, Mar 24, 2026
Slide 1: ADOA Program - Presentation at NANOS 2026
24 March 2026 ADOA PROGRAM - PRESENTATION AT NANOS 2026 • PYC is a precision medicine company dedicated to changing the lives of patients with genetic diseases who have no treatment options available • One of PYC’s clinical-stage programs is an investigational drug candidate (known as PYC-001) that
Slide 2: About PYC Therapeutics and forward looking statements
PYC recently commenced a repeat dose study of PYC-001 in ADOA patients and expects to present clinical safety and efficacy data from this study in H2 CY265. About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a clinical-stage biotechnology company creating a new generation of RNA therapies to chan
Slide 3: A Phase 1A Single Ascending Dose Study of PYC-001 in ADOA
A Phase 1A Single Ascending Dose Study To Evaluate Safety Of PYC-001; A Peptide-Conjugated Oligonucleotide Designed To Treat For personal use only OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy Clare Fraser NANOS March 2026 Disclosures: Clare Fraser – assistance with travel costs from PYC
Slide 4: 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 5: OPA1 protein deficiency triggers a cascade of bioenergetic deficits
OPA1 protein deficiency triggers a cascade of bioenergetic deficits leading to vision loss in ADOA patients Unaffected individual ADOA patient For personal use only Wild type OPA1 protein OPA1 Haploinsufficient expression (100%) ~50-70% expression Cristae Disorganized, wide Slim and tight mitochondr
Slide 6: PYC-001 Mechanism of action
For personal use only PYC-001 Mechanism of action A) The 5’UTR of the OPA1 mRNA contains a stem-loop structure that hinders protein translation B) The phosphorodiamidate morpholino oligonucleotide (PMO) structure of PYC-001 (depicted in red) binds to the 5’UTR of OPA1 mRNA, disrupting the stem-loop
Slide 7: PYC-001 Background research
PYC-001 Background 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 Enhanced mitochondria
Slide 8: PYC-001 Background research
PYC-001 Background research Single dose of PYC-001 increased OPA1 protein PYC-001 increases OPA1 protein in iPSC-RGCs For personal use only (stained red) expression 1.6-fold in NHP retina 2.0 Fold change of OPA1 over untreated ✱✱✱✱ (normalized to total protein) 1.5 1.0 0.5 1.0 1.4 0.0 Untreated 2.25
Slide 9: By restoring OPA1 protein expression, PYC-001 could address the underlying cause
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: SUNDEW: Phase 1A First-In-Human study of PYC-001
SUNDEW : Phase 1A First-In-Human study of PYC-001 in OPA1 mutation associated ADOA patients Phase 1a Single Ascending Dose Study For personal use only Study Design Ocular Assessments • N=9 ; n=3 patients/cohort Objective Subjective Cohort 3: S 24-week • Unilateral IVT injection of 30 µg R follow up
Slide 11: Sundew - patient overview
For personal use only Sundew – 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 Cohort Dose Average age
Slide 12: Sundew - safety outcomes
For personal use only Sundew – safety outcomes ✓ PYC-001 was safe and well-tolerated in ADOA patients ✓ No Treatment Emergent-Serious Adverse events (TE-SAEs) observed in any subject dosed with PYC-001 to date1 ✓ Treatment-Emergent Adverse Events (TE-AEs) were primarily mild and procedure related1 ✓
Slide 13: ADOA eyes treated with PYC-001 show improvement in visual acuity
ADOA eyes treated with PYC-001 show improvement in visual acuity Mean change in Visual Acuity (best corrected and low contrast ETDRS letters) at Week 4 1 For personal use only 6 Mean change from baseline in visual acuity Week 4 after dosing (+SEM) 4 PYC-001 Treated Untreated Fellow 2 0 -2 BCVA LCVA
Slide 14: Number of eyes showing clinically meaningful change in LCVA at Week 24
Week 24 follow-up Number of eyes showing clinically meaningful1 change in LCVA (ETDRS letters) For personal use only at Week 242 9 Number of eyes 6 3 0 PYC-001 Treated Fellow (n=9) (n=9) Gain (>10 letters) Stable (+9 to -9 letters) Loss (>10 letters) 1. PYC A >10 letter change in visual acuity is co
Slide 15: MYRTLE: Phase 1b multi-ascending dose trial is now underway
MYRTLE: Phase 1b multi-ascending dose trial is now underway Progress to date For personal use only Phase 1a SAD Study Phase 1b MAD Study2 Phase 2/3 Study SUNDEW Myrtle Cohorts 60 mcg 60 mcg (Q12W) Objectives of Phase 1/2/3 trials of PYC-001 • Safety & Tolerability • Efficacy 30 mcg 30 mcg (Q8W, Q12W
Slide 16: Questions
Questions For personal use only Myrtle PYC THERAPEUTICS
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