Oryzon Genomics Sa · BME:ORY
Oryzon Genomics Sa — Investor Presentation (April 2019)
28 slides from Oryzon Genomics Sa's investor presentation, Apr 8, 2019
Slide 1: Oryzon April 2019
ieee SEU MADX: ORY Ste S April 2019 . m_
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: Company Highlights
Company Highlights MADX: ORY A publicly traded company on the Spanish Stock Exchange Integrated in the IBEX Small Cap Index A clinical stage biopharmaceutical company developing innovative therapies in the field of Epigenetics A competitive EPIGENETIC PLATFORM validated scientifically and clinically
Slide 4: Epigenetic & LSD1 in human diseases
EPIGENETIC & LSD1 in human diseases Epigenetic mechanisms regulate the function of chromosomes Chromatin remodeling enzymes are a central component of the epigenetic regulation Lysine specific histone demethylase 1 (LSD1), aka KDM1A, removes methyl marks at mono- and dimethyl-H3K4 (histone H3 lysine
Slide 5: Extensive pipeline: 2 programs in clinic with multiple indications each
Extensive pipeline : 2 programs in clinic with multiple indications each A Productive Epigenetic Platform A strong focus on LSD1 5
Slide 6: Vafidemstat: A Global Leader in CNS Epigenetics
A GLOBAL LEADER IN CNS EPIGENETICS V A F I D E M S TAT a P h a s e I I C l i n i c a l S t a g e C o m p o u n d w i t h a b r o a d developability in CNS diseases
Slide 7: Vafidemstat (ORY-2001): a "Neuron-fixer"
Vafidemstat (ORY-2001): a “Neuron-fixer” A small molecule that selectively inhibits LSD1 and MAO-B (IC50 s of 100nM in LSD1 / 75 nM in MAOB) Covalent binder. Excellent Pharmacology. High oral bioavailability Demonstrated target engagement in various animal species Characterized in 6 and 9 months PC
Slide 8: Preclinical results suggestive of disease modifying potential
Vafidemstat restores cognition measured by NORT in SAMP8 AD model Preclinical results suggestive of Disease modifying potential (Similar memory restoration results observed with vafidemstat in the R6/1 HD model. Positive effects in memory also described recently in the NMDA receptor-hypofunction mic
Slide 9: Vafidemstat reduces aggression in mice Alzheimer's Disease model
Vafidemstat reduces aggression in mice Alzheimer’s Disease model In the cognition tests we noticed that SAMP8 male mice treated with vehicle aggressed cage mates while vafidemstat-treated animals did not. Later we confirmed this in proper Resident- intruder aggression tests. SAMP8 animals treated wi
Slide 10: Vafidemstat Clinical Development
Vafidemstat CLINICAL DEVELOPMENT
Slide 11: REIMAGINE - a Basket trial in aggression
Vafidemstat: REIMAGINE - a Basket trial in aggression REIMAGINE Study A single center open label exploratory PhIIa basket trial to assess the effect of vafidemstat in reducing aggression in patients with two neurodegenerative and three neuropsychiatric indications N=30, with 6 participants per condi
Slide 12: REIMAGINE first proof of concept for vafidemstat in human patients
REIMAGINE the first proof of concept for vafidemstat in human patients The study has met the primary and secondary endpoints in BPD patients Borderline Personality Disorder (BPD) patients treated with vafidemstat showed a reduced aggressivity and a better overall performance in the core scales of th
Slide 13: Vafidemstat is efficacious in aggression
Vafidemstat is efficacious in aggression CGI-S CGI-I Secondary endpoints: Efficacy 8 p=0.0109 5 p=0.0175 Vafidemstat produced significant improvements on the 6 4 CGI values CGI values Clinical Global Impression (CGI) CGI-S and CGI-I scales 4 3 2 2 Vafidemstat also produced significant improvements o
Slide 14: Vafidemstat may be a therapeutic option for BPD
Vafidemstat: a CNS compound with broad development potential Vafidemstat may be a therapeutic option for BPD BPD prevalence ranges between 0.5%-1.4%1 of the total population (≤ 9.1M in US+EU5) The treatment of BPD is largely based on psychotherapeutic interventions. It represents a significant unmet
Slide 15: Vafidemstat: a new therapeutic option for Alzheimer's disease
Vafidemstat : a new therapeutic option for Alzheimer’s disease Alzheimer’s, the huge need 45 million people affected worldwide The Global cost of AD is $605 billion/year No therapeutic options so far The recent failures of BACE-i and mAb’s against Abeta (Aducanumab, Crenezumab and others) have final
Slide 16: ETHERAL: Epigenetic Therapy in Alzheimer's Disease
ETHERAL: Epigenetic THERapy in ALzheimer’s Disease 150 Mild to Moderate AD patients An ambitious Phase IIa study to Primary Objective: Safety & Tolerability provide useful information to design Secondary Objectives : future Phase II/III studies Cognition/Agitation/Apathy/QoL Volumetric MRI Biomarker
Slide 17: Vafidemstat ETHERAL Phase IIa: Biomarker strategy
Vafidemstat ETHERAL Phase IIa: Biomarker strategy S100A9 has been characterized as one of the TOP10 up-regulated genes in LOAD dataset S100A9 PROTEIN levels are significantly Human PFC (RNA) Human CSF (protein) increased in CSF from AD patients compared to age-matched controls S100A9 CSF PROTEIN lev
Slide 18: SATEEN: pilot study in MS
Vafidemstat : Phase IIa study in MS SATEEN A pilot study in MS to see a proof of biological activity SAfety, Tolerability and Efficacy in an EPIGENETIC approach to treat Multiple Sclerosis Randomised, double-blind, placebo-controlled, 3-arm, 36 weeks parallel-group study to evaluate the safety and t
Slide 19: Iadademstat (ORY-1001): A Phase II stage clinical compound
A GLOBAL LEADER IN EPIGENETICS Iadademstat (ORY-1001) A Phase II stage clinical compound
Slide 20: Iadademstat: the most advanced selective LSD1 inhibitor in clinic
Iadademstat (ORY-1001): the most advanced selective LSD1 inhibitor in clinic LSD1 is involved in different cancers. High levels of LSD1 often correlate with more aggressive forms of cancer and/or bad prognosis Iadademstat is a small molecule that selectively inhibits LSD1. Preclinical in-vivo positi
Slide 21: Iadademstat a flexible CDP for a Large Market Opportunity
Iadademstat a flexible CDP for a Large Market Opportunity MoA well characterized in SCLC, AML and Medulloblastoma Second Line Combination with SOC Small Cell Lung Cancer is the main SCLC indication under exploration Iadademstat Relapsed/ Biomarkers Refractory SCLC POTENTIAL IADADEMSTAT ONCOLOGICAL I
Slide 22: Iadademstat opportunity in SCLC
Iadademstat opportunity in SCLC LSD1 is a target well characterized in SCLC and validated in preclinical models. LSD1 inhibitors are effective in vitro and in-vivo xenograft models of SCLC Iadademstat produces complete and durable tumor regression in different chemoresistant PDX models Characterized
Slide 23: Iadademstat: SCLC Current Clinical Development Plan
Iadademstat: SCLC Current Clinical Development Plan CLEPSIDRA: A Combination trial of LSD1 and Etop-Platinum in Small Cell Lung Cancer in biomarker-ID Relapsed pAtients A Phase IIa study to assess the safety, tolerability, dose finding and efficacy of iadademstat (ORY-1001) in combination with plati
Slide 24: Iadademstat MoA: differentiating agent for Acute Leukemias
Iadademstat MoA: a potent differentiating agent to treat Acute Leukemias LSD1 is a target for HEMATOLOGICAL CANCERS, and in particular, for a subset of acute myeloid leukemia: mixed lineage leukemia MLL-AML Oryzon’s LSD1 inhibitors block progression of leukemia into the circulation in mice with expe
Slide 25: Iadademstat: AML Current Clinical Development Plan
Iadademstat: AML Current Clinical Development Plan Phase I/IIa previous data Safe and very well tolerated and therefore a meaningful candidate for combination with other agents PD Biomarkers identified in different subsets of leukemia Antileukemic activity observed in 29% of patients (12/41), includ
Slide 26: Recent & Anticipated Oryzon main catalysts: Iadademstat
Recent & Anticipated Oryzon main catalysts IADADEMSTAT (ORY-1001): lead CANCER asset Potential Conferences where data may be presented FPI Phase IIa in elderly unfit AML ALICE FPI Phase IIa in Open Label Studies SCLC CLEPSIDRA 2019 2020 BCN; Sept Orlando; Dec Period of Preliminary Clinical Read Outs
Slide 27: Recent & Anticipated Oryzon main catalysts: Vafidemstat
Recent & Anticipated Oryzon main catalysts VAFIDEMSTAT (ORY-2001): lead CNS asset Potential Conferences where data may be presented 2019 2020 Copenhaguen; Sept Athens; Oct Period of Preliminary Clinical Read Outs 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q Warsaw; Apr Lisbon; Apr Stockholm Sept San Diego; Dec March Ams
Slide 28: Oryzon: A Global Leader in CNS Epigenetics
ORYZON A GLOBAL LEADER IN CNS EPIGENETICS CARLOS BUESA ROGER BULLOCK CEO & President Chief Medical Officer [email protected] [email protected] TA M A R A M A E S M I C H A E L R O PA C K I Chief Scientific Officer VP Clinical Development [email protected] [email protected]