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1 Perpetual Pharmaceutical Pearl Provider 에이비온주식회사

2 Contents I. 회사개요 II. 신약개발프로젝트 - 다발성경화증단백질치료제 - 자궁경부암핵산치료제 - 난소암항체치료제 III. 지식재산권및국책연구과제현황 IV. CRO 서비스 2

3 개요 I. 회사개요 대표이사신영기설립일 2007 년 04 월 16 일 소재지 본사 부설연구소 제 2 연구소 서울시강남구개포로 34 길 5 창원빌딩 202 호 서울시구로구디지털로 242 한화비즈메트로빌딩 9 층 서울시관악구대학동서울대학교 20 동 318 호 자본금 33.6 억원 2013 년매출액 20 억원 ( 삼일회계법인적정의견 ) 인력구성 총 63 명 ( 임원 6 명, R&D 27 명, CRO 13 명, 영업 5 명, 경영지원 10 명, 인허가관리 2 명 ) 현재 주요연혁 회사설립 벤처기업인증 ISO9001 획득 LB인베스트먼트투자유치유방암진단키트 License-out IMM인베스트먼트투자유지 한국투자파트너스투자유치 KONEX 상장 나우IB캐피탈투자유치 부설연구소설립 기술혁신형중소기업 (INNO-BIZ) 인증 연구넷합병 구로사옥이전 사명변경중소기업기술혁신대전중소기업청장상수상 3

4 Business Partners I. 회사개요 R&D CRO Finance Legal & IP 4

5 조직도 I. 회사개요 이사회및자문위원회 부설연구소 (R&D Center 지점 ) 항체치료제개발 1 팀 항체치료제개발 2 팀 단백질치료제개발팀 핵산치료제개발팀 바이오마커개발팀 ( 바이오디펜스팀 ) 대표이사 관리총괄부사장 C R O (Abion CRO 지점 ) 분자유전분석팀 조직병리분석팀 세포면역분석팀 약물동태분석팀 감사 영업본부 (Abion Biomed 지점 ) 영업팀 학술팀 PACS 인허가관리본부 RA 팀 QA 팀 경영지원본부 관리팀 재무팀 사업개발 TFT * 부설연구소, CRO, 영업본부를별도지점으로등기하여구분손익을명확히하고있음 5

6 ABION Inc. II. 신약개발프로젝트 - 다발성경화증단백질치료제 (Carbiferon) - 자궁경부암핵산치료제 (2-in-1 sirna 치료제 ) - 난소암항체치료제 (B12) 6

7 R&D 특징 II. 신약개발프로젝트 Global Market Targeting Early License-out Business Model Perpetual Pharmaceutical Pearl Provider Target Specific New Bio Drug Companion Diagnostics Worldwide Open Innovation Patent Focus 7

8 Pipeline II. 신약개발프로젝트 Indication Candidate Type of Drug Multiple Sclerosis & Anti-virus Carbiferon Carbiferon-Peg Micelle R&D R&D Pre-Clinical Pre-Clinical Clinical Clinical Carbiferon (Protein) T16_426 R&D Pre-Clinical Clinical sirna Oncology OVC_CLD R&D Pre-Clinical Clinical mab-conjugated micelle SC_mCD R&D Pre-Clinical mab-protein SC_mT1 R&D Pre-Clinical Bispecific Ab-conj ugated micelle : License-out Carbiferon/PEG-Carbiferon: 단백질치료제로비임상단계준비중이며 2016년 License-out 예정 T16_426: sirna 치료제로비임상단계및외부기관승인을위한 CMC Data 준비중으로비임상이후부터조기 License-out 예정 OVC_CLD: 항체치료제로대량생산시스템확립후비임상단계진입을위한연구개발진행중 8

9 For Multiple Sclerosis Interferon-β (Carbiferon) 9

10 Background of Technology Problems of Conventional IFN-ß drugs (Betaseron, Rebif, Avonex ) 10

11 Background of Technology Conventional IFN-ß problems : Side Effects Side Effects of Conventional IFN-ß Drug Injection Site Reactions Flu-like Reactions; Depression; Headache; Fatigue Liver Test Abnormalities 11

12 Technology Highlights of Abion s Candidate IFN-ß Mutein : Bio-better (Abion s Candidate Carbiferon, R27T) One/Two Additional Glycosylation from Conventional Interferon-β 1a Creation of Low Immunogenicity by Minimal Amino Acid Change Creation of Prolonged Half Life Time by Increased Absorption Time 12

13 Conclusion Target Disease : Multiple Sclerosis (MS) Market Advantage Patient s economic burden decrement Improved patient`s compliance Improved tolerability (immunogenicity) 13

14 IP Portfolio Patent Title Country Patent Date Application No. / Patent No. Status Korea Granted PCT Granted US Granted JP Granted Brazil PI Publication Human Interferon- Beta Mutein China CN B Granted India 3732/DELNP/2007 Applied EP Granted Sweden, Switzerland, France, United Kingdom, Hungary, Ireland, Finland, Portugal, Italy, Spain, Germany EP_Turkey TU T4 Granted EP_Greece Granted EP_Austria Granted EP_Denmark Granted 14

15 SWOT Analysis Strength Weakness Superior Patent Portfolio Guaranteed Safety from Biobetter Improved Patient s Convenience Lower Cost Expanding MS Drug Market Patent Expiration of Original Drug Needs for Biobetter Price Competition Opportunity S W O T Clinical Trial for Efficacy FDA Approval Follow-Up Development Limitation in Drug Modification Unbalance in Inter-National Approval Possibility of Superior New Drug Market Competition Barrier by Original Blockbuster Threat 15

16 한국보건산업진흥원기술사업화과제선정 16

17 In HPV-related Cancers ANTI-E6/E7 (2-IN-1) sirna SENSITIZER 17

18 Cervical Cancer: Medical Unmet Needs 18

19 Background of Technology Development of 2-in-1 sirna Candidates Derivatives Production by Chemical modifications: 2 -O-Me modified nucleotides 2 -F modified nucleotides 19

20 Development of 2-in-1 Candidates and sirna Pool (SP) Patent I Patent II Patent III HIT Screening Lead Derivative Candidate sirna Pool (SP) Anti E6/E7 (2-in-1) sirna HPV 18 Type 10 Library HPV 16 Type 10 Library T No.5 HPV 18 Type T No.4 sirna Pool T No.2 T No.2 T No.2 HPV 16 Type sirna Pool *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 - PCT 20

21 Power of 2-in-1 sirna Proof-of-concept and Mode of action in combination therapy with 2-in-1 sirna Synergism analysis in combination therapy with lead sirna Combination with Cisplatin Int.J.Cancer (2012 Apr 15;130(8): ) The synergistic therapeutic effect of cisplatin with Human Papillomavirus E6/E7 short interfering RNA on cervical cancer cell lines in vitro and in vivo Combination with cisplatin and HPV18/16 type lead sirna - Int.J.Cancer (2012 Apr 15;130(8): ) Combination with irradiation and HPV18/16 type lead sirna - Submission Human papillomavirus E6/E7-specific sirna potentiates the effect of radiotherapy for cervical cancer in vitro and in vivo 21

22 Comparison with Conventional Technology There has been no report about sirna therapeutics development in HPV-related cancers as combinational agent approach No. Country Patent Title 1 KR 2 JP 3 US 4 KR 5 EP 6 KR 7 US ( ) ( ) ( ) ( ) ( ) ( ) ( ) Vaccine SmithKline Beecham Biologicals s.a. (Belgium) Pharmaceutical composition for preventing or treating papilloma virus-induced disease Containing antigenic protein of papillomavirus and cpgoligodeoxynucleotide dsrna compositions and methods for treating HPV infection. Alnylam Pharmaceuticals, Inc. (USA) HPV16-based Papillomavirus vaccine Transgene S.A. (France) Long peptides of amino acid residues that induce and/or enhance antigen specific immune responses academisch ziekenhuis leiden (Netherland) 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) Compositions and methods related to mirna modulation of neovascularization or angiogenesis The Johns Hopkins University ( 미국 ) 22

23 Possessed Technology Status Originality of 2-in-1 sirna therapeutics 2-in-1 sirna Maximizing Stability `2 in 1 sirna system`: simultaneously inhibit E6 and E7 oncogene Synergistic therapeutic effects with chemo-/radio-therapy Play a role as Sensitizer Application in HPV-related cancer research (Anal cancer, Head and Neck cancer, Oral cancer, Penile cancer, Lung cancer, etc.) Reduced Toxicity Increased Efficacy Anti-E6/E7 (2-in-1) sirna Therapeutics Chemical modification of nucleotides by 2'-OMe: a increase in serum stability Formulation optimization - Polymer based delivery system Easy Development Chemical modification of nucleotides by 2'-OMe: a decrease in off-target effect sirna POOL technology Polymer based nanoparticle for enhanced drug delivery sirnas are regulated as drugs : - Under FDA s Center for Drug Evaluation and Research [CDER] sirnas are synthetic molecules (cgmp) Polymer based nanoparticle (non-viral vector) : (Easy to commercialize) 23

24 Patent Position Patent Title Country Date of Application Application No. Date of Patent Patent No. Status (Applied/ Approval) 1 st Patent COMPOSITI ON FOR TREATMENT OF CERVIX CANCER Republic of Korea Approval PCT KR USA / nd Patent 3 rd Patent COMPOSITI ON FOR TREATMENT OF HPV RELATED CANCERS COMPOSITI ON FOR TREATMENT OF HPV RELATED CANCERS Republic of Korea Approval PCT KR Republic of Korea PCT KR

25 한국보건산업진흥원기술사업화과제선정 25

26 For Ovarian Cancer B12 Antibody Drug 26

27 항체치료제프로젝트 (B12) B12 항체치료제 난소암세포의세포막에과다하게발현되는특정클라우딘 (CD3, CD4) 단백질에선택적으로반응하는 항체개발 타겟증상 난소암 : 초기경고증상이없으며효과적인진단능력이부족하여일단발병되면사망률이높음 시장성 : 2012년난소암치료및진단시장은 160억달러, 2013년 187억달러예상 (CARG 13.1%) 장점및개발목표 표적암세포만제거가능 : 일반합성항암제에비해낮은부작용과높은효과기대 다른암종 ( 대장암, 위암등 ) 에도적용이가능함 27

28 Anti-Claudin ADC 난소암치료제 Therapeutic Anti-CLDN3/4 mab Antigen Design Antibody Screening In vitro Assay In vivo Assay Antibody Drug Conjugated Micelle(Co-development with JPN Company) Bortezomib Polymer with Sensor Binding Linker Bortezomib, Docetaxel, Oxaloplatin, Paclitaxel.. Etc. 300~500 Drug molecules Drug Conjugated Polymer Drug Conjugated Micelle Active Targeting Micelle Anti-CLDN3/4 Antibody or Antibody Fragment 28

29 경쟁연구현황 개발 Group 개발단계 TRL 기준특허 Target Indication Screening 방법 / 문제점 Kohleisen Group (NIH, US) PJ Morin Group (NIH, US) Smith MD Group (Brescia Univ. Italy) Ando Group (Kyowa Hakko Kirin) Stepheen BH Group (Univ. of California) Hit 탐색 1 없음 CLDN3 CLDN4 CLDN 1 Pan-Carcinoma Affinity 측정없음 기초연구 1 없음 CLDN3, 4 Pan-Carcinama Affinity 측정없음 Hit 탐색 1 없음 CLDN3 Candidate 발굴단계 2 Hit 탐색 1 물질특허추정 물질특허추정 CLDN4 Ovarian Cancer Uterine Cancer Pancreatic Carcinoma Ovarian Cancer Janet A. Sawicki Group Hit 탐색 1 특허추정 CLDN3 Ovarian Cancer Kenji Group (Forerunner Pharma research Co., Japan) ABION Inc Cytotoxicity 낮음 CLDN3 Ovarian Cancer CPE 의 immunogenicity CLDN3 sirna 를처리시전이억제확인 / Proof of Concept Hit 탐색 1 타깃특허 CLDN3 Pan-Carcinama Affinity 측정없음 Candidate 발굴단계 2 스크리닝특허출원 CLDN3 CLDN4 Ovarian Cancer Pan-Carcinoma Companion Diagnostics 가능 / 천연형항원을이용한스크리닝기법 비교적초기단계의연구만진행중 진입가치충분함 29

30 ABION Inc. III. 지식재산권및국가과제연구현황 30

31 IP 운영전략및사업화모델 III. 지식재산권및국책연구과제현황 1 단계 2 단계 CRO 기술개발 포트폴리오 구성 연구개발 국가과제 IP R&D 특허분쟁 특허 수행 특허 Infra 검토 도출 확보 에버그리닝 기술가치 특허환경분석 전략확보 평가수행 Perpetual Pharmaceutical Pearl Provider 고유 기술 특허권 기술 인력 고유기술과관련지재권 등의 License-Out 31

32 지식재산권현황 III. 지식재산권및국책연구과제현황 제목 출원 / 등록 국가출원 / 등록번호비고 Korea PCT PCT/KR/2005/ 차세대 Interferon-β 치료제개발자궁경부암을위한치료용조성물 HPV 감염과관련된암의치료용조성물 CD24를표적으로하는암억제방법 등록 등록중 등록 US 8,101,716 B2 Japan China ZL EU Brazil, Portugal, UK, Finland, Germany, Italy, Spain, Switzerland, Sweden, France, Ireland, Hungary Korea PCT 미국 등록 Korea 출원 PCT PCT/KR2011/ Korea PCT PCT/KR2013/ 등록 US 12/307,108 * 포괄적타겟특허 출원 PCT PCT/KR2007/ * Austria, Denmark, Greece, Turkey 개별등록완료 * 본특허로부터 sirna PK/PD/ADME 분석 Tool 개발하고, 2014 년초부터 CRO 서비스로서매출이발생하고있음 표준발현유전자를발굴하기위한유전자 발현데이터처리, 분석방법 등록 출원 Korea * 본특허에서개발된유방암진단키트를 2011년 PCT PCT/KR2007/ 월젠큐릭스로 License-out 하였으며, 관련된 CRO서비스매출이지속적으로발생하고 EU, Japan, US 있으며, 향후로열티수입기대됨 복합막횡단단백질의세포막외펩타이드 부위를항원으로하는항체개발 등록 Korea * 중기청과제종료후막단백질 Kit 을개발하여 2014 년 6 월부터매출발생하고있음 순환암세포분리용칩출원 Korea * 대형병원의요청으로 CTC 서비스제공준비중 32

33 연구개발을통한 CRO 사업화모델 III. 지식재산권및국책연구과제현황 신약개발과정을통한국내독점고부가가치 CRO 아이템개발및사업화 항체치료제개발서비스 ( 항체평가, 세포주개발 ) 항체치료제조직교차반응분석서비스 ( 신약개발업체, 연구단등에 10 여건제공 ) + 조직병리분석서비스 분자병리분석서비스 혁신적접근 : GLP 도입 C-LAB 혁신적진단검사센터운영 sirna 치료제개발및 PK/PD 분석서비스 (2 개제약사서비스수행중 ) 융합적접근 : 조직병리와분자병리동시접근 과제수행결과의활용 ( 축적된인력, 장비, knowhow 활용 ) 산업통상자원부과제 ( 난소암치료용항체치료제개발 ) 중기청기술혁신개발사업 ( 자궁경부암치료 sirna 개발 ) 식약처용역연구과제 ( 항체치료제조직교차반응시험법확립 ) 30 억원지원 (2010~2015 년 ) 확립기술 : - Phage library screening - scfv, Fab, full IgG 항체제작기술 - CHO 세포주확립 - In vivo / in vitro 효능실험 - CMC 자료확보 6 억원지원 (2010~2012 년 ) 확립기술 : - sirna design - sirna modification - sirna formulation/transfection - In vivo / in vitro 효능실험 - qrt-pcr 이용 PK 법 1 억원지원 (2010~2011 년 ) 확립기술 : - 항체치료제독상시험지표작성 - Microarray 법 - 동결조직분석법확립 - 반응성표준방법확립 33

34 ABION Inc. IV. CRO 서비스 34

35 CRO 서비스항목 IV. CRO 서비스 Molecular Genetics FFPE based molecular genetics analysis Nucleic Acid (DNA/RNA) Isolation - Blood, Cell, Tissue (FFPE/Frozen/TMA) DNA Quantification (qpcr) RNA Quantification (qrt-pcr) RNA Validation (QC) Micro RNA - mirna Isolation - mirna RT-qPCR Quantitative Methylation-Specific PCR PK Analysis for sirna Therapeutics ( 식약처허가용 ) Gene Mutation Sequencing Microarray - Expression Array (1 or 2 color) - Array CGH - mirna Expression Next Generation Sequencing Cell Line Authentication - STR Analysis - Mycoplasma Test Histology Tissue Cross Reactivity Analysis ( 식약처허가용 ) FFPE/Frozen Tissue/Cell Block Hematoxylin & Eosin Staining Immunohistochemistry (IHC) Staining Special Staining TUNEL & BrdU Assay Tissue Microarray (TMA) Laser Micro-Dissection (LMD) FISH/IF Test and Diagnosis Pharmacokinetic LC-MSMS Analysis TK & PK Analysis Cell Immunology ELISA Analysis Customized ELISA Kit Development & Validation ( 식약처허가용 ) - Direct/Indirect Kit - Direct Sandwich Kit - Indirect Sandwich Kit Animal TK, Human PK Analysis for Protein Therapeutics ( 식약처허가용 ) Drug Discovery Customized Ab drug discovery Customized sirna drug discovery 35

36 ABION CRO 의특징 ( 차별점 ) IV. CRO 서비스 36

37 License & Certification IV. CRO 서비스 ISO 9001 유전자검사기관 ( 질병관리본부 ) IRB( 질병관리본부 ) 진행중인인증 : GLP (KFDA), ISO15189 (KOLAS) 37

38 고객맞춤형 sirna 치료제개발서비스 IV. CRO 서비스 ABN ABN ABN ABN Client s sirna Hits Lead Discovery sirna Modification sirna Candidates Preclinical/ Clinical trials ABN Client s sirna Hit identification MOA, Proof-of-Concept sirna Lead Library Screening Lead discovery Lead validation sirna Lead Optimization Derivatives Production sirna Candidate Candidates selection PK TK analysis service Verification of RNAi CRO service Services under full GLP compliant conditions Design more than 10 sirnas Verification of mrna/ protein level change In vitro efficacy assay In vivo efficacy assay Stem-loop RT-qPCR RACE-PCR In vivo efficacy assay GLP-PK TK analysis 38

39 고객맞춤형항체치료제개발서비스 IV. CRO 서비스 Antibody Development Service, Phase I - Whole IgG conversion - Stable cell line construction - Epitope mapping - Flow cytometric analysis - Affinity analysis(spr) - Specificity analysis - Cell based ELISA/SPR - Antibody maturation - Chemistry manufacturing and controls - Effector function analysis(adcc/cdc) - Tissue cross reactivity Whole IgG Conversion CDR analysis Affinity / Specificity analysis Lead Discovery (>5) Affinity maturation CMC, in vitro / in vivo efficacy, TCR Candidate Discovery( 2) Hit Discovery(>30) - Animal model preparation - In vivo efficacy assay - Design for optimized product condition (expression, cultivation and purification) R&D Design Antibody Development Service, Phase II - Mode of action - Proof of concept - Antigen design/preparation - Definition of hit, lead and candidate scfv Library screening, ELISA screening FACS/SPR screening ScFv Development Service - Cloning for High level expression - Flow cytometric analysis - Affinity analysis(srp) 39

40 국내일반 CRO 와의차별점 IV. CRO 서비스 생물의약품 ( 항체, 단백질, 핵산치료제등 ) 의비임상또는임상분석시험에특화된서비스제공 의료기기 ( 진단시약 ) 의 Analytical validation & Clinical validation 시험제공 분자유전분석의경우소량의임상검체를이용하여동시에빠르고정확하게다양한분석 (qpcr, Microarray, NGS 등 ) 제공 정확도, 정밀도, 재현성이높은결과확보 비임상시험관리기준 (Good Laboratory Practice, GLP) 매뉴얼에따라분석시험진행 한글 / 영문보고서제공 다양한 ELISA Kit 개발 & 시험물질의개발부터비임상및임상분석시험에필요한키트 (Lot 개념 ) 제작, 분석및보고서제공 LIMS(lab information management system) 보유 : FDA CFR 21 part 11 요구조건만족 시험물질 & 시약보관을위한온도모니터링시스템보유 시험물질이송시스템보유 40

41 Perpetual Pharmaceutical Pearl Provider ABION CRO 41