Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — BIO International Convention 2023
15 slides from Pyc Therapeutics's conference presentation, Jun 6, 2023
Slide 1: Phelan-McDermid Syndrome Presentation: Materials for BIO Conference
31 October 6 June 2023 2022 PHELAN-MCDERMID SYNDROME PRESENTATION MATERIALS FOR BIO CONFERENCE • PYC is currently attending BIO 2023 – one of the industry’s primary business development events - in Boston For personal use only • The Company’s presentation materials for the Central Nervous System pla
Slide 2: Forward looking statements
Forward looking statements Any forward-looking statements in this ASX announcement have been prepared on the basis of a number of assumptions which may prove incorrect and the current intentions, plans, expectations, and beliefs about future events are subject to risks, uncertainties and other facto
Slide 3: Phelan-McDermid Syndrome program
For personal use only Life-changing science Phelan-McDermid Syndrome program June 2023
Slide 4: Disclaimer
Disclaimer For personal use only The purpose of this presentation is to provide an update of the business of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification. A
Slide 5: Executive Summary
Executive Summary • Phelan-McDermid Syndrome (PMS) is a severe neurodevelopmental disorder affecting 1 in every 10,000 children¹ that can cause a wide range of medical, intellectual and behavioural challenges For personal use only • PMS is caused by haploinsufficiency of the SHANK3 gene • There are
Slide 6: There is a pressing need for a disease-modifying approach in Phelan-McDermid Syndrome
There is a pressing need for a disease-modifying approach in Phelan-McDermid Syndrome Phelan-McDermid Syndrome (PMS): Signs and symptoms of PMS For personal use only Poor head control • Is a rare and severe neurodevelopmental disorder causing life-long disability Intellectual disability • ~28,000 ad
Slide 7: PYC has designed an RNA therapy that addresses the underlying cause of PMS
PYC has designed an RNA therapy that addresses the underlying cause of PMS Phelan McDermid Syndrome (PMS) is caused by insufficient (~50%) expression of SHANK3 protein in neurons For personal use only PYC has designed an RNA drug candidate capable of increasing SHANK3 protein expression >2-fold in a
Slide 8: The RNA therapy rescues the underlying gene expression that causes PMS
The RNA therapy rescues the underlying gene expression that causes PMS by increasing translation from the healthy allele PYC’s RNA therapy for PMS (known as ‘PYC-002’) restores SHANK3 protein expression from the remaining healthy allele For personal use only PMS patient treated Unaffected individual
Slide 9: PYC has optimised two different chemistries/mechanisms of action in parallel
PYC has optimised two different chemistries/mechanisms of action in parallel - creating optionality in the development path RNA Chemistry/Mechanism 1 RNA Chemistry/Mechanism 2 2′MOE Clinically validated RNA chemistry PPMO Next generation RNA drug conjugate For personal use only Naked RNA drug Delive
Slide 10: PYC's 2′MOE RNA drug has an established path through clinical development
PYC’s 2′MOE RNA drug (chemistry/mechanism 1) has an established path through clinical development For this combination of: For personal use only • Chemistry: 2′MOE PS There is an established path through non-clinical • Administration: intrathecal species to clinical validation and commercial • Targe
Slide 11: The in vivo data pack for this RNA candidate sets the program up for success
The in vivo data pack for this RNA candidate links in to this pathway and sets the program up for success in the clinic For this combination of: For personal use only • Chemistry: 2’MOE PS There is an established path through non-clinical • Administration: intrathecal species to clinical validation
Slide 12: PYC's PPMO RNA drug has an improved in vivo PK/PD profile
PYC’s PPMO RNA drug (chemistry/mechanism 2) has an improved in vivo PK/PD profile relative to the 2′MOE candidate Brain regions implicated in PPMO shows enhanced target engagement profile (at lower tissue concentration) For personal use only PMS in the brain via a clinically relevant route of admini
Slide 13: PYC's preferred candidate is expected to have a differentiated clinical profile
PYC’s preferred candidate is expected to have a differentiated clinical profile due to its disease-modifying potential 1 2 3 4 For personal use only Symptoms that are Clinical endpoints tailored Non-clinical models linked Conviction in a potentially important to patients to the symptoms to clinical
Slide 14: Human safety and initial efficacy data are expected in 2025
Human safety and initial efficacy data are expected in 2025 2023 2024 2025 2026 For personal use only Lead Characterization PoC in Patient Derived Model IND Animal PoC, PK/PD, Tox Prelim Stability of DS/DP Phase 1 / 2 IND Enabling Studies Human safety data GMP DS & DP Initial efficacy data PYC THERA
Slide 15: FDA special designations assist in efficiently creating the first treatment option for PMS
FDA special designations assist in efficiently creating the first treatment option for patients with PMS Potential eligibility for 3 FDA special designations For personal use only Orphan Drug Designation (ODD)1 Rare Pediatric Disease Designation3 Accelerated Approval3 Orphan drugs address patient po