에이비온주식회사 - kr.abionbio.com

1 Copyright 2014 ABION Inc. All Rights Reserved PerpetualPharmaceutical Pearl Provider2014. 2014 ABION Inc. All Rights Reserved ContentsI. II. - - - III. 3 Copyright 2014 ABION Inc. All Rights Reserved I. 2007 04 16 34 5 202 242 9 2 20 318 2013 20 ( ) 63 ( 6 , R&D 27 , CRO 13 , 5 , 10 , 2 ) 2007. 4 2009. 3 2009. 12 2010. 8 (INNO-BIZ) 2010. 9 ISO9001 2013. 1 2013. 2 IB 2013. 4 2013. 9 2013. 12LB IMM 2012. 12( ) 2011. 9 License-out 2014. 7 KONEX 2014. 6 4 Copyright 2014 ABION Inc. All Rights Reserved BusinessPartnersI. FinanceLegal & IPR&DCRO5 Copyright 2014 ABION Inc.

2 All Rights Reserved C R O 1 2 ( ) PACS RA QA TFTI. (R&D Center )(Abion CRO )(Abion Biomed )* , CRO, 6 Copyright 2014 ABION Inc. All Rights Reserved II. ABION (Carbiferon)- (2-in-1 siRNA )- (B12)7 Copyright 2014 ABION Inc. All Rights Reserved R&D Global Market Targeting Early License-out Business Model Perpetual Pharmaceutical Pearl Provider Target Specific New Bio Drug Companion Diagnostics Worldwide Open Innovation Patent FocusII. 8 Copyright 2014 ABION Inc. All Rights Reserved Pipeline Carbiferon/PEG-Carbiferon: 2016 License-out T16_426: siRNA CMC Data License-out OVC_CLD: Indication Candidate 201120122013201420152016 Type of Drug Multiple Sclerosis & Anti-virusCarbiferonCarbiferon-PegMicell eCarbiferon(Protein)OncologyT16_426siRNA OVC_CLDmAb-conjugated micelleSC_mCDmAb-ProteinSC_mT1 Bispecific Ab-conjugatedmicelle: License-outClinicalPre-ClinicalR&DClinic alPre-ClinicalR&DClinicalPre-ClinicalR&D Pre-ClinicalR&DPre-ClinicalR&DClinicalPr e-ClinicalR&DII.

3 9 Copyright 2014 ABION Inc. All Rights Reserved Interferon- (Carbiferon) For Multiple Sclerosis10 Copyright 2014 ABION Inc. All Rights Reserved Problems of Conventional IFN- drugs (Betaseron , Rebif , Avonex )Background of Technology11 Copyright 2014 ABION Inc. All Rights Reserved Conventional IFN- problems : Side Effects Side Effects of Conventional IFN- Drug Injection Site Reactions Flu-like Reactions; Depression; Headache; Fatigue Liver Test AbnormalitiesBackground of Technology12 Copyright 2014 ABION Inc. All Rights Reserved IFN- Mutein : Bio-better (Abion s Candidate Carbiferon, R27T) One/Two Additional Glycosylation from Conventional Interferon- 1a Creation of Low Immunogenicity by Minimal Amino Acid Change Creation of Prolonged Half Life Time by Increased Absorption Time Technology Highlights of Abion s Candidate13 Copyright 2014 ABION Inc.

4 All Rights Reserved Target Disease : Multiple Sclerosis (MS) Patient seconomicburdendecrementImprovedpatient` scomplianceImprovedtolerability(immunoge nicity)MarketAdvantageConclusion14 Copyright 2014 ABION Inc. All Rights Reserved PatentTitleCountryPatent DateApplication Interferon-Beta MuteinKorea , Switzerland, France, United Kingdom, Hungary, Ireland,Finland, Portugal, Italy, Spain, Portfolio15 Copyright 2014 ABION Inc. All Rights Reserved Analysis SWOTS trengthOpportunityThreatWeakness Superior Patent Portfolio Guaranteed Safety from Biobetter Improved Patient s Convenience Lower Cost Clinical Trial for Efficacy FDA Approval Follow-Up Development Limitation in Drug Modification Expanding MS Drug Market Patent Expiration of Original Drug Needs for Biobetter Price Competition Unbalance in Inter-National Approval Possibility of Superior New Drug Market Competition Barrier byOriginal BlockbusterSWOT16 Copyright 2014 ABION Inc.

5 All Rights Reserved 17 Copyright 2014 ABION Inc. All Rights Reserved ANTI-E6/E7 (2-IN-1) siRNA SENSITIZERIn HPV-related Cancers18 Copyright 2014 ABION Inc. All Rights Reserved Cervical Cancer: Medical Unmet Needs19 Copyright 2014 ABION Inc. All Rights Reserved Development of 2-in-1 siRNA CandidatesBackground of TechnologyDerivatives Production byChemical modifications:2 -O-Me modified nucleotides2 -F modified nucleotides20 Copyright 2014 ABION Inc. All Rights Reserved Development of 2-in-1 Candidates and siRNA Pool (SP)HITS creening LeadDerivativeCandidatesiRNAPool (SP)Anti E6/E7(2-in-1)siRNAHPV18 Type 10 Type siRNA Type 10 Type siRNA IPatent IIPatent III*COMPOSITION FOR TREATMENT OF CERVIX CANCER -Registered in South Korea-Under prosecution in USA*COMPOSITION FOR TREATMENT OF HPV RELATED CANCERS-Registered in South Korea-PCT*COMPOSITION FOR TREATMENT OF HPV RELATED CANCERS-Under prosecution in South Korea-PCT21 Copyright 2014 ABION Inc.

6 All Rights Reserved Power of 2-in-1 siRNA Combination with cisplatin and HPV18/16 type lead siRNA Combination with irradiation and HPV18/16 type lead siRNA Proof-of-concept and Mode of actionin combination therapy with 2-in-1 siRNA Synergism analysis in combination therapy with lead (2012 Apr 15;130(8):1925-36) -Submission Human papillomavirus E6/E7-specific siRNA potentiates the effect of radiotherapy for cervical cancer in vitroand in vivo (2012 Apr 15;130(8):1925-36) Combinationwith Cisplatin The synergistic therapeutic effect of cisplatin with Human Papillomavirus E6/E7 short interfering RNA on cervical cancer cell lines in vitro and in vivo 22 Copyright 2014 ABION Inc. All Rights Reserved Comparison with Conventional ( )Vaccine SmithKline Beecham Biologicals (Belgium)2JP2004-315433 ( )Pharmaceutical composition for preventing or treating papilloma virus-induced disease Containing antigenic protein of papillomavirus and cpg-oligodeoxynucleotide3US7956177 ( )dsRNA compositions and methods for treating HPV Pharmaceuticals, Inc.

7 (USA)4KR10-2009-0005011 ( )HPV16-based Papillomavirus vaccineTransgene (France)5EP2112157( )Long peptides of 22-45 amino acid residues that induce and/or enhance antigen specific immune responsesacademisch ziekenhuis leiden (Netherland)6KR10-2013-0040675 ( )BIFIDO BACTERIUM ADOLESENTIS SPM 1005-A HAVING ANTIVIRAL ACTIVITY FOR HUMAN PAPILLOMAVIRUS, AND COMPOSITION FOR PREVENTING OR CURING OF HUMAN PAPILLOMAVIRUS INFECTION COMPRISING BIFIDO BACTERIUM ADOLESENTIS SPM 1005-A OR CULTURED MATERIAL OF THE SAME SAHMYOOK UNIVERSITY (Korea)7US8258111 ( )Compositions and methods related to miRNA modulationof neovascularization or angiogenesisThe Johns Hopkins University ( ) There has been no report about siRNA therapeutics development in HPV-related cancers as combinational agent approach23 Copyright 2014 ABION Inc. All Rights Reserved Possessed Technology Status siRNAs are regulated as drugs : -Under FDA s Center for Drug Evaluation and Research [CDER] siRNAs are synthetic molecules (cGMP) Polymer basednanoparticle(non-viral vector): (Easy to commercialize)Easy Development `2 in 1 siRNA system`: simultaneously inhibit E6 and E7 oncogene Synergistic therapeutic effects with chemo-/radio-therapy Play a role as Sensitizer Application inHPV-related cancer research(Anal cancer, Head and Neck cancer, Oral cancer, Penile cancer, Lung cancer, etc.)

8 2-in-1 siRNA Chemical modification of nucleotides by 2'-OMe: a increase in serum stability Formulation optimization -Polymer based delivery systemMaximizing Stability Chemical modification of nucleotides by 2'-OMe: a decrease in off-target effect siRNAPOOL technology Polymer based nanoparticle for enhanced drug deliveryReduced ToxicityIncreased EfficacyAnti-E6/E7 (2-in-1) siRNAT herapeuticsOriginality of 2-in-1 siRNA therapeutics24 Copyright 2014 ABION Inc. All Rights Reserved Patent PositionPatentTitleCountryDate of ApplicationApplication PatentPatent (Applied/Approval)1stPatentCOMPOSITION FOR TREATMENT OF CERVIX CANCER Republic of Korea 2006. 11. 05. 0112/5133372ndPatentCOMPOSITION FOR TREATMENT OF HPV RELATED CANCERS Republic of Korea 2010. 01. FOR TREATMENT OF HPV RELATED CANCERS Republic of Korea 2014 ABION Inc. All Rights Reserved 26 Copyright 2014 ABION Inc.

9 All Rights Reserved B12 Antibody DrugFor Ovarian Cancer27 Copyright 2014 ABION Inc. All Rights Reserved (B12) B12 (CD3, CD4) : : 2012 160 , 2013 187 (CARG ) : ( , ) 28 Copyright 2014 ABION Inc. All Rights Reserved Anti-Claudin ADC Therapeutic Anti-CLDN3/4 mAbAntigen DesignAntibody ScreeningIn vitro AssayIn vivo AssayPolymer with Sensor Binding LinkerBortezomib,Docetaxel,Oxaloplatin, Conjugated PolymerDrug Conjugated MicelleActive Targeting MicelleAnti-CLDN3/4 Antibody or Antibody FragmentAntibody Drug Conjugated Micelle(Co-development with JPN Company)300~500 Drug moleculesBortezomib29 Copyright 2014 ABION Inc. All Rights Reserved Group TRL TargetIndicationScreening / Kohleisen Group (NIH, US) Hit 1 CLDN3 Pan-Carcinoma Affinity CLDN4 CLDN 1PJ Morin Group (NIH, US) 1 CLDN3, 4 Pan-CarcinamaAffinity Smith MD Group (Brescia Univ.)

10 Italy)Hit 1 CLDN3 Ovarian Cancer10-8 Uterine CancerAndo Group(Kyowa Hakko Kirin)Candidate 2 CLDN4 Pancreatic CarcinomaCytotoxicity Ovarian CancerStepheen BH Group (Univ. of California)Hit 1 CLDN3 Ovarian CancerCPE immunogenicityJanet A. Sawicki GroupHit 1 CLDN3 OvarianCancerCLDN3siRNA /Proofof ConceptKenji Group(ForerunnerPharma research Co., Japan)Hit 1 CLDN3 Pan-CarcinamaAffinity ABION 2 CLDN3 Ovarian CancerPan-CarcinomaCompanionDiagnostics / CLDN430 Copyright 2014 ABION Inc. All Rights Reserved III. ABION 2014 ABION Inc. All Rights Reserved IP CRO Infra IP R&D License-OutPerpetual Pharmaceutical Pearl Provider1 2 III. 32 Copyright 2014 ABION Inc. All Rights Reserved III.