A GLOBAL LEADER IN EPIGENETICS I N V E S T O R P R E S E N TAT I O N M A D X : O R Y N e w Yo r k C i t y F e b r u a r y 2 0 1 6
L E G A L N O T I C E 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 this document have not been verified by independent third parties; therefore, no express or implied warranty is made as to the impartiality, accuracy, completeness or correctness of the information or the opinions or statements expressed herein. Oryzon genomics, S.A. does not assume liability of any kind, whether for negligence or any other reason, for any damage or loss arising from any use of this document or its contents. Neither this document nor any part of it constitutes a contract, nor may it be used for incorporation into or construction of any contract or agreement. Information in this document about the price at which securities issued by Oryzon Genomics, S.A. have been bought or sold in the past or about the yield on securities issued by Oryzon Genomics, S.A. cannot be relied upon as a guide to future performance. IMPORTANT INFORMATION This document does not constitute an offer or invitation to purchase or subscribe shares, in accordance with the provisions of Law 24/1988, of 28 July, on the Securities Market, Royal Decree-Law 5/2005, of 11 March, and/or Royal Decree 1310/2005, of 4 November, and its implementing regulations. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any other jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration. FORWARD-LOOKING STATEMENTS This communication contains forward-looking information and statements about Oryzon Genomics, S.A., including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon Genomics, S.A. believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon Genomics, S.A. shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon Genomics, S.A., that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon Genomics, S.A. to the Comisión Nacional del Mercado de Valores, which are accessible to the public. Forward-looking statements are not guarantees of future performance. They have not been reviewed by the auditors of Oryzon Genomics, S.A. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon Genomics, S.A. or any of its members, directors, officers, employees or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon Genomics, S.A. on the date hereof. Except as required by applicable law, Oryzon Genomics, S.A. does not undertake any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise 2
COMPANY HIGHLIGHTS MADX: ORY A publicly traded company in the Madrid Stock Exchange since December 14 th 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 therapeutic programs in clinical development with multiple indication opportunities & additional assets in preclinical development Signed global strategic partnerships with ROCHE for ORY-1001 valued at 500M USD Strong IP portfolio with technology developed in- house Strong financial profile with € +20M cash on balance sheet with runway till 2018 Experienced management team 3
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 and all the genes that reside in this region These variations are caused by post-translational modifications on histones, the proteins that serve as scaffold for the DNA to conform the chromatin Lysine methylation and demethylation is one of the key epigenetic modifications of the Histone tails 4
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 also entering in the field either through their own programs or through alliances. C O M P A N Y C O M P O U N D D E S C R I P T I O N I N D I C A T I O N S T A T U S Reverlogix RVX-208 BET bromodomain inhibitor Atherosclerosis -Diabetes Phase II b Acetylon (ACY-1215) lic to Oral selective HDAC6 inhibitor Multiple myeloma (MM) Phase I/II Pharmaceuticals Celgene Lysine-specific demethylase 1 ORY-1001 lic. to Roche Acute myelogenous leukemia (AML) Phase I/IIa (LSD1) inhibitor Oryzon Genomics Alzheimer's Disease ORY-2001 LSD1-MAOB dual inhibitor Phase I Other Neurodegenerative disorders CPI-1205 EZH2 inhibitor lymphoma Phase II Constellation Progressive Lymphoma Pharmaceuticals CPI-0610 BET bromodomain inhibitor AL, MDS, myeloproliferative neoplasms Phase I Multiple myeloma non-Hodgkin B-cell lymphoma Phase I/II EPZ 6438 Tazemetostat EZH2 inhibitor Synovial Sarcoma IND / Phase I Epizyme EPZ-5676 lic. to Celgene HMT DOT1L inhibitor MLL- AML Phase I/II Tensha Therapeutics TEN-010 BET bromodomain inhibitor Cancers including NUT midline carcinomas Phase I 5
EXTENSIVE PIPELINE : 2 PROGRAMS IN CLINIC WITH MULTIPLE INDICATIONS 6
L S D 1 P R O G R AM : 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 targets such as MAO-A and MAO-B LSD1 is located in the nucleus, unlike MAOs LSD1 expression has a high correlation in many solid tumors In some aggressive Leukemia, Leukemia Stem Cells are addicted to LSD1 activity The pan-MAO inhibitor tranylcypromine: a chemical starting point to design covalent LSD1 inhibitors. Protected by 19 patent families filed globally with 10 granted in US 7
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-AF9 cells, but spares normal repopulating cells. Oryzon’s LSD1 inhibitor (OG-86) blocks progression of leukemia cells into the circulation MLL-AF9 mice model Oryzon’s LSD1 inhibitor (OG-86) targets leukemia stem cells but spares normal hematopoietic stem cells Modified from Harris et al., Cancer Cell. 2012: 21(4):473-87) 8
O R Y- 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 up • Good pharmaceutical properties Currently in Phase I/IIA • Completed Part 1 of the study (Phase I) in acute leukemia • Extension Arm (Phase II-A) ongoing Potential for additional indications, such as solid tumors (like SCLC) and sickle cell disease Global strategic collaboration with ROCHE valued >500M USD 9
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