Oryzon Genomics Sa · BME:ORY
Oryzon Genomics Sa — Investor Presentation (January 2022)
28 slides from Oryzon Genomics Sa's investor presentation, January 2022
Slide 1: Pioneering personalized medicine in epigenetics
Pioneering personalized medicine C O R P O R AT E P R E S E N TAT I O N in epigenetics January 2022 O R Y : S M / O R Y. M C
Slide 2: Legal Notice
Legal Notice DISCLAMER This document has been prepared by Oryzon Genomics, S.A. exclusively for use during the presentation. Oryzon Genomics, S.A. does not assume liability for this document if it is used with a purpose other than the above. The information and any opinions or statements made in thi
Slide 3: Oryzon: Epigenetic Champion Developing New Therapies in Oncology and CNS
Oryzon: Epigenetic Champion Developing New Therapies in Oncology and CNS Growing epigenetic platform with an expanding pipeline LSD1 is an “eraser” Developing highly potent and selective LSD1 inhibitors with best-in-class potential Two programs with positive Phase IIa data in AML and SCLC, and in ag
Slide 4: Potentially Best-in-Class LSD1i Addressing Unmet Medical Needs in Oncology and CNS
Potentially Best-in-Class LSD1i Addressing Unmet Medical Needs in Oncology and CNS ONCOLOGY CNS Iadademstat (ORY-1001) Vafidemstat (ORY-2001) MoA: Pro-Differentiation MoA: Prosynaptic Anti-cancer stemness Anti-neuroinflammatory Epigenetic • Potential first & best-in-class LSD1i in personalized • Pot
Slide 5: Deep Pipeline with Multiple Upcoming Milestones
Deep Pipeline with Multiple Upcoming Milestones 5
Slide 6: Iadademstat: A Phase II LSD1 inhibitor in Oncology
I A DA D E M STAT A Phase II LSD1 i n h i b i to r i n O n c o l o g y
Slide 7: Iadademstat: Potentially Best-in-Class LSD1 Inhibitor for Oncology
Iadademstat: Potentially Best-in-Class LSD1 Inhibitor for Oncology LSD1 plays a key role in numerous cancers; elevated levels often correlate with more aggressive forms or poor prognosis Iadademstat is a potent and highly selective, iadademstat investigational oral LSD1i with best-in-class potential
Slide 8: Iadademstat: Potentially Best-in-Class LSD1 inhibitor for Oncology
Iadademstat: Potentially Best-in-Class LSD1 inhibitor for Oncology • The most potent LSD1 inhibitor in clinical development • The most efficient in disrupting LSD1-transcriptional complexes CD11b expression 100 % CD11-positive cells 80 60 40 20 0 0.01 1 100 10000 Concentration (nM) cell assay Iada v
Slide 9: Multiple Commercial Opportunities in Heme-Onc and Solid Tumors
Multiple Commercial Opportunities in Heme-Onc and Solid Tumors Orphan Drug Designation granted for AML in US and EU • Clear PoC in ongoing AML Phase II study (ALICE) • Registrational strategy: Potential for expedited development in AML and SCLC in two indications • 2L in AML with FLT3 mutations Pati
Slide 10: Iadademstat MoA and Antileukemic Activity Observed in AML
Iadademstat MoA and Antileukemic Activity Observed in AML LSD1 inhibition LSD1 interacts with modulates the GFI1/1B AML is the most common GFI1/1B causing a block axis to induce terminal type of acute leukemia in in leukemia cell differentiation and adults differentiation control leukemia stem cell
Slide 11: ALICE: a PoC Phase IIa in AML
ALICE: a PoC Phase IIa in AML Trial Design / Overview Results Highlights • Open label, multicenter, single arm study • 78% ORR (21 out of 27 evaluable patients): • Newly diagnosed elderly or unfit AML patients • 62% CR/CRi (13/21): 6 CR & 7 CRi (3 still evolving); 8 PR • Iadademstat in combination w
Slide 12: Next Steps: A Phase Ib/II trial in 2L AML with registrational potential
Next Steps: A Phase Ib/II trial in 2L AML with registrational potential Phase Ib/II in FLT3 mut+ R/R AML patients combining iadademstat and gilteritinib (Xospata®) FRIDA: FLT3 mutated Relapsed/Refractory AML treatment with IaDAdemstat • 2L AML is an underserved population: 50% of AML patients relaps
Slide 13: Iadademstat is a Therapeutic Approach for SCLC With a Well-Defined MoA
Iadademstat is a Therapeutic Approach for SCLC With a Well-Defined MoA SCLC is very aggressive LSD1i acts in SCLC by LSD1i synergizes with and represents 15% of enhancing Hes1 and ICIs and boosts the host all lung cancers Notch1 pathways immune system • Iadademstat produces complete and durable tumo
Slide 14: Iadademstat SCLC Phase IIa CLEPSIDRA Trial Overview
Iadademstat SCLC Phase IIa CLEPSIDRA Trial Overview Trial Design / Overview Results Highlights • Open label, multicenter, single-arm study • Platinum/etoposide in combo with iadademstat showed hematotoxicity • Biomarker selected, platinum sensitive 2L ED- • Iada alone was generally well tolerated. T
Slide 15: Next Steps: A Phase Ib/II Trial in 1L ED-SCLC with registrational potential
Next Steps: A Phase Ib/II Trial in 1L ED-SCLC with registrational potential A randomized Phase Ib/II study of iadademstat plus a checkpoint inhibitor in 1L patients with metastatatic SCLC STELLAR: Synergistic Treatment with Epigenetics in front line small celL Lung cAnceR • High unmet medical need a
Slide 16: Vafidemstat: A Phase II LSD1 inhibitor for CNS diseases
VA F I D E M STAT A P h a s e I I L S D 1 i n h i b i to r fo r C N S d i s e a s e s
Slide 17: Vafidemstat: Neuron-resetting LSD1 Inhibitor in Phase II for CNS Disorders
Vafidemstat: “Neuron-resetting” LSD1 Inhibitor in Phase II for Multifactorial and Monogenic CNS Disorders Oryzon is the first and only company pursuing LSD1i in CNS diseases Vafidemstat is an investigational LSD1 inhibitor with high BBB penetration, optimized for the CNS Vafidemstat has been adminis
Slide 18: LSD1 Inhibition and Multifactorial CNS Disorders
LSD1 Inhibition and Multifactorial CNS Disorders • LSD1 is expressed in the developing and adult nervous system • LSD1 plays a critical role in neurogenesis, in neuronal differentiation, axonal navigation and regulating expression of key genes • Regulation of methylation has emerged as a top pathway
Slide 19: Vafidemstat: Aggression / Agitation Phase IIa REIMAGINE Trial Overview
Vafidemstat: Aggression / Agitation Phase IIa REIMAGINE Trial Overview Trial Design / Overview • Open label, single-center, Phase IIa study • Adult patients with BPD, ADHD and ASD with significant agitation and aggression • Received vafidemstat 1.2mg/day, 5 days per week for 8 weeks • 30 patients en
Slide 20: Vafidemstat for the treatment of Borderline Personality Disorder
PORTICO: Ongoing Phase IIb Trial in BPD • Double blind, placebo controlled adaptive design with interim analysis to assess statistical power. 156 patients to be enrolled • Two primary endpoints: overall clinical BPD improvement and improvement in aggression • CTA approved in Spain, Germany Bulgaria
Slide 21: Vafidemstat for the treatment of Schizophrenia
EVOLUTION: Ongoing Phase IIb Trial in SCZ • Double blind, placebo controlled adaptive design with interim analysis to assess statistical power. 100 patients to be enrolled in two arms • 2 arms, vafidemstat as add-on to SoC. 6 months of treatment • Primary endpoints: efficacy to address SCZ Negative
Slide 22: LSD1 and Precision Medicine in CNS
LSD1 and Precision Medicine in CNS 22
Slide 23: LSD1 Inhibition: Personalized Medicine in Rare Monogenic CNS Disorders
LSD1 Inhibition: Personalized Medicine in Rare Monogenic CNS Disorders • Dysregulated methylation plays an important role in the onset of certain neurodevelopmental disorders • Genetic single defects or loss of alleles can cause CNS disorders with complex phenotypes • Preclinical data has shown that
Slide 24: Methylation is involved in Kabuki Syndrome and LSD1 inhibition rescues phenotypes
Methylation is involved in Kabuki Syndrome and LSD1 inhibition rescues phenotypes in genetic models Kabuki syndrome (KS) is a congenital disorder characterized by intellectual disability, growth retardation, dysmorphic facial features and immune defects Mutations of MLL2 (KMT2D) cause Kabuki syndrom
Slide 25: HOPE: an adaptive Phase I/II trial with vafidemstat in Kabuki Syndrome patients
HOPE: an adaptive Phase I/II trial with vafidemstat in Kabuki Syndrome patients • A randomized, double blinded, multicenter study in KS type I (MLL defective) patients. 6-8 US centers to be included • Lead investigator: Dr. Jaqueline Harris, director of the Epigenetics Clinic at Kennedy Krieger Inst
Slide 26: Vafidemstat has a growing avenue for personalized therapy in CNS
Vafidemstat has a growing avenue for personalized therapy in CNS COMPASS-related Pathologies Glutamatergic pathway-related Pathologies Other yet unknown genetic relationships SET1 (COMPASS)-like complex methylates histone H3 • Shank3 : Shankopaties – PMS / ASD • Rett Syndrome lysine 4 to activate pr
Slide 27: Thank you: Value Creation in 2022
ORYZON Thank you Value Creation in 2022 A unique dual EPIGENETIC proposition Multiple inflection points in ONCOLOGY and CNS • A validated approach with multiple • Final data for 1L AML PoC ALICE shots on goal • New AML & SCLC trials to start in 2022 with potential to support • 2 Phase II programs re
Slide 28: Pioneering personalized medicine in epigenetics
Pioneering personalized medicine in epigenetics
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