Oryzon Genomics Sa · BME:ORY
Oryzon Genomics Sa — Investor Presentation (February 2016)
32 slides from Oryzon Genomics Sa's investor presentation, Feb 8, 2016
Slide 1: A Global Leader in Epigenetics - Investor Presentation
A G L O B AL L E AD E R I N E P I G E N E T I C S I N V E S T O R P R E S E N TAT I O N MADX: ORY N e w Yo r k C i t y F e b r u a r y 2 0 1 6
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 in the Madrid Stock Exchange since December 14th 2015 Trading started at €3,39 = €96,5M Market Cap A clinical stage biopharmaceutical company developing innovative therapies in oncology and neurodegeneration leading the field of Epigenetics Two
Slide 4: Epigenetics: The Critical Role of Histone Coding
E P I G E N E T I C S : THE CRITICAL ROLE OF HISTONE CODING Epigenetics − the study of heritable changes in genome function that occur without a change in DNA sequence These changes mainly occur due to variations in the structure of chromatin that silence or activate whole regions of the chromosome
Slide 5: Epigenetics Competitive Landscape
EPIGENETICS COMPETITIVE LANDSCAPE EPIGENETICS is a new Space being explored by the Pharma Industry: Clinical Programs are still in Early Phases. ORYZON, Epizyme and Constellation are the only Biotechs developing more than one compound. GSK, Roche, Merck, Pfizer, Celgene and other Big Pharmas are als
Slide 6: Extensive Pipeline: Programs in Clinic with Multiple Indications
EXTENSIVE PIPELINE : 2 PROGRAMS IN CLINIC WITH MULTIPLE INDICATIONS 6
Slide 7: LSD1 Program: An Epigenetic Eraser
L S D 1 P R O G R A M : AN EPIGENETIC “ERASER” Lysine-specific histone demethylase 1 (LSD1 or KDM1A) is an enzyme that demethylates histones (removes methyl groups), specifically mono and di-methylated H3K4 and H3K9 LSD1 belongs to the family of FAD dependent amine oxidases, which include known drug
Slide 8: Oryzon's Clinical Oncology Program: ORY-1001
ORYZON’s CLINICAL ONCOLOGY PROGRAM: O R Y - 1 0 0 1 KDM1A is a key effector of the differentiation block in MLL leukemia KDM1A sustains expression of the MLL-AF9 oncogenic program Nanomolar KDM1A inhibitor concentrations induce differentiation of human AML cells KDM1A inhibition in vivo targets MLL-
Slide 9: ORY-1001: Oncology Program
O RY- 1 0 0 1 : O N C O L O G Y P R O G R A M ORY-1001 a highly potent and selective LSD1 inhibitor with orphan drug status granted by the European Medicines Agency (EMA) Pharmacological Properties • High druggability • Optimal ADMET and PK profiles • Orally bioavailable once daily • Easy to scale u
Slide 10: ORY-1001: Roche Partnership
O R Y - 1 0 0 1 : ROCHE PARTNERSHIP In April 2014, Oryzon and ROCHE entered into a global collaboration to research, develop and commercialize LSD1 inhibitors, including ORY-1001, for oncology, hematology and non-malignant conditions Licensed compounds are covered by 2 patents in the Oryzon IP portf
Slide 11: Phase I Highlights: ORY-1001 Leukemia
P H A S E I H I G H L I G H T S : ORY-1001 LEUKEMIA TRIAL DESIGN Refractory & Relapsed Multiple Ascending Dose (8 Multi-Center (5) Acute Leukemia Cohorts) PRIMARY ENDPOINT Evaluate Safety (hematological and non-hematological toxicities) and Tolerability SECONDARY ENDPOINTS Assess Responses Evaluate
Slide 12: Phase IIA: ORY-1001 Leukemia
PHASE IIA: O R Y - 1 0 0 1 L E U K E M I A After the MRD , an Expansion arm (Phase II-A) to include patients with target mutations (MLL and others) to evaluate preliminary signs of efficacy CENTERS 9 Patients to be included Status: 6 patients enrolled and actively recruiting Completion Date: 2Q-2016
Slide 13: LSD1 Additional Indications
LSD1 Additional indications Beyond onco-hematology there are a number of growing additional indications for LSD1i: Specific solid tumors are a growing field for the clinical expansion of LSD1 inhibition and ORY- 1001 Small Cell Lung Cancer GSK is in Phase I with a LSD1 inhibitor (A DNA Hypomethylati
Slide 14: ORY-1001 Clinical & Market Potential
ORY-1001 CLINICAL & MARKET POTENTIAL ORY-1001 market capture opportunity above $1.8 billion A number of scientific reports point out the potential of LSD1 inhibition as a target in a number of solid tumors Non oncological diseases as SCD and others may also be a CDP option Acute Myeloid Leukemia Sma
Slide 15: Role of Epigenetics: Neurodegenerative Disorders
R O L E O F E P I G E N E T I C S : NEURODEGENERATIVE DISORDERS Studies suggest epigenetic modifications that induce alterations in gene expression programs contribute to neurodegenerative disorders: • Alzheimer’s Disease (AD) • Parkinson’s Disease (PD) • Huntington’s Disease (HD) ENVIRONMENT GENES
Slide 16: Role of Epigenetics: Neurodegenerative Disorders
R O L E O F E P I G E N E T I C S : NEURODEGENERATIVE DISORDERS HDACi improves HD symptoms in animal models HDAC2 inhibition recovers memory on the bi- transgenic CK-p25 Tg mouse model HDAC inhibition improves FTD Efforts to develop Selective HDACi Selective HDAC2 i in HDAC-6 i in neurodegeneration
Slide 17: LSD1 in the Nervous System
LSD1 IN THE NERVOUS SYSTEM LSD1 is a key component of the LSD1-REST- CoREST-HDAC1/2 repressor complex involved mainly in controlling developmental programs and modulating neuronal morphology in the CNS LSD1 is known to be an important regulator in the maintenance of pluripotency and in specification
Slide 18: ORY-2001: CNS Program
O R Y - 2 0 0 1 : CNS PROGRAM ORY-2001 is a highly selective dual LSD1-MAO-B inhibitor Preclinical Proof of Concept: LSD1 Against AD and HD Clinical development • Phase I in Healthy Volunteers Alzheimer’s Disease is lead indication Potential for additional indications: PD, HD and others Exclusively
Slide 19: PoC Studies in SAMP8 Mice
PoC studies in SAMP8 mice The SAMP8 mouse is an excellent model to examine the pathophysiology of early defects seen in Alzheimer’s disease. They develop accelerated aging and senescence and show deficits in learning and memory as well as other similarities to pathology of AD ORY-2001 cognitive effe
Slide 20: PoC Studies in SAMP8 Mice: Dissecting LSD1 and MAOB
PoC studies in SAMP8 mice Dissecting the LSD1 and MAOB components LSD1+MAOB LSD1+MAOB LSD1 alone MAO MAOB LSD1 aloneLSD1 MAOB inhibition alone shows a trend LSD1+MAOB alone MAOB alone alon alone on cognitive improvement on the *** SAMP8 animals but it is not *** significant • p=0.12 at 2h *** • p=0.
Slide 21: PoC Studies in SAMP8 Mice: Dissecting LSD1 and MAOB
PoC studies in SAMP8 mice Dissecting the LSD1 and MAOB components LSD1+MAOB LSD1 alone MAOB alone ORY-2001 provides a robust protective effect in the medium and long-term memory of mice, compared to age-matched SAMP8 mice LSD1 inhibition alone is also able to produce an effect but less pronounced Pr
Slide 22: ORY-2001 Proof of Concept: Biomarkers in SAMP8 Mice
(POCD) and traumatic brain injury (TBI). In addition, knockout or knockdown of S100A9 has been since S100A9 is emerging as an important contributor to inflammation-related neurodegeneration. Down-regulation of the pro-inflammatory S100A9 protein by ORY-2001 is particularly interesting, has a pleiotr
Slide 23: ORY-2001 Development Timeline
ORY-2001 DEVELOPMENT TIMELINE 2015 2016 2017 3Q2015 4Q2015 1Q2016 2Q2016 3Q2016 4Q2016 1Q2017 2Q2017 CMC / 4W IB / IMPD Reg Tox CTA Phase I (SAD) Phase I (MAD) 6M Reg Tox Additional Preclinical Work to Broaden CDP Phase II Dossier Phase II AD Phase II Additional Indications 23
Slide 24: ORY-2001 Development Timeline
ORY-2001 DEVELOPMENT TIMELINE A Phase I study with 88 healthy volunteers, young and elderly. 24
Slide 25: ORY-2001 Development Timeline
ORY-2001 DEVELOPMENT TIMELINE A Phase I study with 88 healthy volunteers, young and elderly. CL01-ORY-2001 SAD CL01-ORY-2001 MAD 25
Slide 26: ORY-2001 Clinical & Market Potential
ORY-2001 CLINICAL & MARKET POTENTIAL ORY-2001 market capture opportunity above $3 billion. Further development may include Neuro- inflammatory disorders ALZHEIMER’S DISEASE 5.4 M people currently affected in US. By 2025 the number of patients will rise to 7.1 million in USA 1 8.7 million Europeans a
Slide 27: Financial Highlights
FINANCIAL HIGHLIGHTS Strong balance sheet with €+20m in cash $5 million payment from ROCHE in 2015 Secured €2.6M in public aids in 2015 Unused credit line of €6 M from commercial banks €10M in debt with low interest rates • Repayment terms over either 3-4y or 8-10y (commercial loans or Public R&D lo
Slide 28: Key Investment Highlights
KEY INVESTMENT HIGHLIGHTS EPIGENETICS • Global leader in Epigenetics, an emerging field recognized by the pharmaceutical industry as a novel approach to varying diseases and personalized medicine • 2 programs in clinical development EXTENSIVE • Both clinic programs can expand in multiple indications
Slide 29: Catalysts 2015-2016
CATALYSTS 2015 - 2016 ORY-1001: LEAD CANCER ASSET • Conclude Phase I dosing study • Receive recommended dose milestone payment from Roche • Phase IIA first patient-in • Complete Phase IIA and report target efficacy • Roche execute ongoing clinical development plan ORY-2001: LEAD CNS ASSET • Complete
Slide 30: International Research Network
INTERNATIONAL RESEARCH NETWORK 30
Slide 31: Oryzon Is a Transatlantic Company
ORYZON IS A TRANSATLANTIC COMPANY EUROPE UNITED STATES ORYZON ORYZON Corp. Sant Ferran, 74 245 First Street 08940 Cornellà de LL. Suite 1800 Barcelona, Spain Cambridge, MA 02142 Investor Relations Research and Development US Clinical Operations European Clinical Trials Business Development Intellect
Slide 32: Thank You Very Much
T H AN K Y O U V E RY M U C H ! CARLO S BUESA C.E.O. & President cbuesa@oryzon.com EMILI TORRELL ANNA K.BARAN THE RUTH GROUP BDO IR Director IR firm for USA etorrell@oryzon.com abaran@oryzon.com oryzon@theruthgroup.com
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