Oryzon Genomics SA · BME:ORY
Oryzon Genomics SA — Investor Presentation (September 2022)
23 slides from Oryzon Genomics SA's investor presentation, September 2022
Slide 1: Pioneering personalized medicine in epigenetics
Pioneering personalized medicine in epigenetics C O R P O R AT E P R E S E N TAT I O N September 2022 ORY:SM / ORY.MC
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: Epigenetic Champion Developing New Therapies in CNS and Oncology
Epigenetic Champion Developing New Therapies in CNS and Oncology Focus on developing 2 Programs with well- Growing epigenetic Listed in Europe (Madrid) highly potent and defined registrational platform with an MK Cap ∼$150M selective LSD1 pathways: expanding pipeline to inhibitors with best-in- Iada
Slide 4: Multiple Shots on goal & main investment thesis in the short-mid term
Multiple Shots on goal & main investment thesis in the short-mid term Note: Finalized clinical trials for iadademstat and vafidemstat are not shown. See www.oryzon.com for more details 4
Slide 5: ORYZON has an ambitious epigenetic program in Oncology
ORYZON has an ambitious epigenetic program in Oncology IADADEMSTAT A Phase II LSD1 inhibitor in Oncology
Slide 6: Iadademstat: first and potentially best-in-class LSD1 inhibitor in oncology
Iadademstat: first and potentially best-in-class LSD1 inhibitor in oncology • A unique asset to address specific cancers • Focusing on clinical execution in AML AML hemato-oncology and solid tumors with a registrational plan ODD • Reinforcing institutional granted SCLC collaborations • Exploring nic
Slide 7: Iadademstat: first and potentially best-in-class LSD1 inhibitor in AML
Iadademstat: first and potentially best-in-class LSD1 inhibitor in AML • The most potent (nM) oral inhibitor of the lysine histone demethylase LSD1 in clinical development The asset • +100 pts treated with iadademstat in 4 Phase I/II oncology trials – safety profile well established • LSD1 is requir
Slide 8: FRIDA: a Phase Ib trial in R/R AML
FRIDA: a Phase Ib trial in R/R AML as a foundation for an accelerated development R/R-AML a Market opportunity • In a competitive market, R/R AML is an underserved population: Majority of AML patients relapse after 1L treatment and require further treatment Best route to • FLT3 is the most common mu
Slide 9: Iadademstat in SCLC and other Neuroendocrine tumors
Iadademstat: potentially first and best-in-class LSD1 inhibitor in SCLC and other Neuroendocrine tumors • LSD1 is required for survival & proliferation of neuroendocrine/SCLC tumor cells A Proof of Concept (CLEPSIDRA) • Iadademstat blocks LSD1’s actions and promotes neuroendocrine/SCLC tumor Mechani
Slide 10: A future Phase Ib/II trial in 1L ED-SCLC
A future Phase Ib/II trial in 1L ED-SCLC with potential for accelerated development A strong • Iadademstat and ICIs show a strong synergism, suggesting potential clinical benefit rationale • LSD1i enhances the immunological response by with ICIs overexpressing MHC-1 class genes and deferring lymphoc
Slide 11: Collaborative PoC basket trial in NETs with iadademstat
Neuroendocrine Tumors: a Collaborative PoC basket trial in NETs with iadademstat A Phase Ib/II study of iadademstat in combination with synergistic agents in platinum R/R SCLC and extrapulmonary high grade neuroendocrine carcinomas • Label expansion opportunity • High unmet medical need: Treatment o
Slide 12: ORYZON, the only company developing epigenetic drugs in CNS
ORYZON, the only company developing epigenetic drugs in CNS VAFIDEMSTAT A Phase II LSD1 inhibitor for CNS diseases
Slide 13: CNS disorders: a field evolving towards Precision Medicine
CNS disorders: a field evolving towards Precision Medicine LSD1 is key for the function of the CNS and is involved in multifactorial CNS disorders and monogenic syndromes Small / rare monogenic LargeMultifactorial Large multifactorial indications Small/rare monogenic indications Indications indicati
Slide 14: Vafidemstat: an LSD1 inhibitor for BPD and schizophrenia
Vafidemstat: an LSD1 inhibitor to treat large multifactorial CNS indications including borderline personality disorder (BPD) and schizophrenia (SCZ) Safe • A potent (nM) oral inhibitor of LSD1 with high BBB penetration Mechanism • LSD1i induces the neuronal plasticity & downregulates neuroinflammati
Slide 15: Borderline personality disorder: a snapshot
Borderline personality disorder: a snapshot A Prevalent & impairing disease Two main types of symptoms No approved drugs yet 9 million in US & EU Unstable-extreme Patients in off-label anti- interpersonal relationships psychotics + Aggression & self-aggression Vafi improves these symptoms in PC mode
Slide 16: PORTICO: Phase IIb in Borderline Personality Disorder
Ongoing Study: a Phase IIb in Borderline Personality Disorder PORTICO: An adaptative randomized double blind Phase IIb trial with vafidemstat in Borderline Personality Disorder patients • High unmet need: no drugs specifically approved for BPD. 1.4 million patients in US are being treated with off-l
Slide 17: EVOLUTION: ongoing PoC Schizophrenia study with vafidemstat
EVOLUTION an ongoing PoC Schizophrenia study with vafidemstat EVOLUTION: An adaptative randomized double blind Phase IIb trial with vafidemstat in schizophrenia patients • Strong rationale: LSD1i restores phenotypes in various SCZ mice models • High Unmet Need: No drugs approved yet for cognitive im
Slide 18: ORYZON is pioneering personalized medicine in CNS
ORYZON is pioneering personalized medicine in CNS VAFIDEMSTAT A Phase II LSD1 inhibitor for CNS diseases
Slide 19: Vafidemstat targeting genetically defined neurodevelopmental syndromes, e.g., Kabuki
Vafidemstat: an LSD1 inhibitor targeting genetically defined neurodevelopmental syndromes, e.g., Kabuki • LSD1i can compensate complex phenotypes caused by single gene Mechanism deficiencies that are the cause of some rare neurodevelopmental syndromes of Action • Specifically, in Histone Methyltrans
Slide 20: HOPE: a Phase Ib/II trial in Kabuki syndrome patients
HOPE: a Phase Ib/II trial in Kabuki syndrome patients HOPE: An adaptative randomized double blind Phase I/II trial with vafidemstat in KS Type 1 patients • Phase Ib objectives: evaluate safety/tolerability, and determine the RP2D • Phase II objective: evaluate efficacy of vafidemstat at the RP2D • K
Slide 21: 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 lysine • Shank3 : Shankopaties – PMS / ASD • Rett Syndrome 4 to activate pr
Slide 22: Thank you: Value Creation in 2022-23
ORYZON Thank you Value Creation in 2022-23 A unique dual EPIGENETIC proposition Multiple inflection points in CNS and ONCOLOGY • A validated approach with multiple • Final data for 1L AML PoC ALICE shots on goal • 2L AML and NET trials preliminary readouts in 2023 • One and only epigenetic company i
Slide 23: Pioneering personalized medicine in epigenetics
Pioneering personalized medicine in epigenetics
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