Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — 2026 PMS Foundation Family Conference
19 slides from Pyc Therapeutics's conference presentation, Jul 20, 2026
Slide 1: Presentation at 2026 PMS Foundation Family Conference
20 July 2026 PRESENTATION AT 2026 PMS FOUNDATION FAMILY CONFERENCE • 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 developing a drug candidate (known as PYC-002) that addresses For person
Slide 2: PYC presents new PYC-002 data in patient-derived models
PERTH, Australia and SAN FRANCISCO, California – 20 July 2026 PYC Therapeutics Limited (ASX:PYC) (PYC or the Company) is a precision medicine Company dedicated to changing the lives of patients with genetic diseases who have no treatment options available. The Company currently has three clinical-st
Slide 3: PYC-002 restores neuronal communication deficits; next steps
Figure 2. PYC-002 restores neuronal communication deficits to ‘unaffected’ levels in PMS patient-derived neurons11 in a manner not seen with the Non-Targeting Control (NTC) oligonucleotide. For personal use only A full copy of the materials presented at the conference is attached to this announcemen
Slide 4: 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 5: PYC-002, a disease modifying RNA therapeutic approach to treat Phelan-McDermid syndrome
For personal use only Life-changing science PYC-002, a disease modifying RNA therapeutic approach to treat Phelan-McDermid syndrome 2026 PMSF Family Conference
Slide 6: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the business of PYC Therapeutics about the future, you are urged to view all forward looking statements contained in this For personal use only Limited (ASX:PYC) [‘PYC’]. These slides have been prepared as a presentation aid only
Slide 7: PYC-002 targets SHANK3 upregulation to treat Phelan-McDermid syndrome
PYC-002 is a disease modifying approach that targets SHANK3 upregulation to treat Phelan-McDermid syndrome • PYC is developing PYC-002, an antisense oligonucleotide for the treatment of For personal use only Phelan-McDermid syndrome (PMS). • PYC-002 is designed to target the underlying cause of PMS
Slide 8: PYC-002 targeting the root cause of PMS
PYC-002 targeting the root cause of PMS to restore neuronal structure and improve connection between brain cells PMS BIOLOGY: WHAT HAPPENS For personal use only 1 Reduced SHANK3 2 Fewer healthy 3 Reduced brain 4 Less effective 5 Challenges in daily life SHANK3 helps brain cells connections cell acti
Slide 9: PYC-002 is a precision ASO designed to restore SHANK3
PYC-002 is a precision ASO designed to restore SHANK3 What is an ASO? Why this approach? For personal use only An ASO is synthetic, short strings of RNA/DNA designed to Specifically target SHANK3 – the root target the disease at the RNA level—the direct middle step cause of PMS between our DNA bluep
Slide 10: PYC-002 is delivered by intrathecal injection
PYC-002 is delivered by intrathecal (IT) injection, allowing it to reach target brain regions to restore SHANK3 towards healthy levels For personal use only What is an IT injection? Why IT injection Our goal A small amount of drug is Allows the transport and To deliver the right amount gently inject
Slide 11: Encouraging evidence in pre-clinical studies
Encouraging evidence in pre-clinical studies supports PYC-002 moving towards clinical studies For personal use only • In studies using PMS patient-derived cells, PYC-002 restored SHANK3 protein expression toward healthy levels; and • Restoring SHANK3 protein helps brain cells form healthier connecti
Slide 12: Preclinical studies support potential of PYC-002 to address underlying cause of PMS
Preclinical studies support the potential of PYC-002 to address the underlying cause of PMS 1 2 3 4 Neuronal For personal use only SHANK3 protein Neuronal connections Neuronal activity communication SHANK3 protein Rebuilds connections More brain cells are active Stronger communication increase betwe
Slide 13: Restoring SHANK3 protein helps patient-derived brain cells communicate more effectively
Restoring SHANK3 protein helps patient-derived brain cells communicate more effectively Untreated Healthy control-derived neurons For personal use only Untreated PMS patient-derived neurons PYC-002 + PMS patient-derived neurons The upper plot shows the mean+SD of weighted mean firing rate (Hz), expr
Slide 14: Deficient SHANK3 levels in multiple brain regions disrupt neuronal communication
Deficient SHANK3 levels in multiple brain regions disrupts neuronal communication contributing to the PMS phenotype1-3 For personal use only Brain regions implicated in PMS and impact on phenotype 1. Hippocampus: critical for learning and memory processes (2) (3) 2. Prefrontal cortex: important for
Slide 15: PYC-002 distributes to all key brain regions implicated in PMS
PYC-002 distributes to all key brain regions implicated in PMS at safe and well-tolerated doses1 For personal use only Concentration of PYC-002 in the NHP brain after single dose2 Distribution of PYC-002 in the rat brain after single dose3 100000 Key regions contributing to Key regions contributing
Slide 16: PYC-002 increases SHANK3 protein at safe and well-tolerated doses
PYC-002 increases SHANK3 protein at safe and well-tolerated doses For personal use only SHANK3 protein increased up to 1.5-fold in A single dose of PYC-002 increases monkey brain following a single IT injection1 SHANK3 protein up to 2-fold in rat brains2 1. Concentration of SAHNK3 protein (pg protei
Slide 17: Where we are today and what's next
Where we are today — and what’s next 2026 2027 2028 2029 For personal use only IND-enabling Start NHS Start MAD Start OLE/LTE study enrollment treatment ~2H 2028 In progress ~Q4 2026 ~Q4 2027 IND-ENABLING STUDIES INTERVENTIONAL (MAD) OLE/LTE (LONG TERM EXTENSION) Completing key safety studies and pr
Slide 18: An Observational Study: Understanding PMS today to inform future clinical studies
HIPPOCAMPUS An Observational Study History of Progression in Phelan-McDermid: Observational Characterization and Assessment of Mapped Phenotypes Underlying SHANK3 Deficiency Understanding PMS today to inform future clinical studies For personal use only Follow-up every 6 months … Future PYC-002 Base
Slide 19: Does PYC-002 work? (conference poster)
PYC-002: an RNA drug candidate for the treatment of Phelan-McDermid syndrome Dulce B. Vargas-Landin, Abbie Francis, Sasiwimon “Fern” Utama, Donna Bezner, Wontaek Oh, Priyanka Sain, Rungtiwa Nutalai, Tracy Chai, Tanisha Verma, Dean De Alvis, Linda de Castro, Laura Florez, Philipp Bayer, Alexander de
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