March 10th, 2017
Corporate Presentation Jin-San Yoo, Dr. rer. Nat. President & CEO of PharmAbcine
Executive Summary-1 1.
PharmAbcine is a leading clinical stage biotech with valuable Platform technologies for development of the next generation therapeutics.
2.
The best fully human antibody based therapeutics for unmet medical needs.
3.
Leading science and most innovative pipelines with strong IP position.
4.
Leadership, Management, Boards, and investors with top global standard.
5.
Plan for KOSDAQ IPO in 2018.
Executive Summary-2 1.
Tanibirumab phase I data was clean and currently, its phase IIa multi center trials in Australia are running smoothly with recurrent GBM patients with 13M PFS with tumor shrinkage at Melbourne site and other tumor shrinkage patient with >7M PFS at Perth site.
2.
This phase IIa is expected to be completed by this August.
3.
We are preparing for phase IIb recurrent GBM global trial including US, Europe, Australia, Japan and South Korea.
4. We also are preparing for multiple clinical trials with Tanibirumab single, and Tanibirumab + immune checkpoint blockade combination in refractory tumors and TNBC. 5. We will accelerate our PMC-001 IND and Phase I in US. 6. We will accelerate our development time of novel immune checkpoint blockade.
Contents
I.
Company Intro
II. People , R&D Asset
III. Marketability IV. Competition
V. Future Plan
Key Milestones 2009.09. Series A Fund - USD 6 M (Invested by OrbiMed, Novartis Venture Fund, GreenCross, DongYang etc.)
2012.04. ~ 2013.06. Series B Fund - KRW 10 B (Novartis Venture Fund, Hanwha-Oxford, DongYang, MiraeEsset, KB Investment etc)
2008.09. Company Establishment
2008
2008.10. 1st GATE Project Best Award (Sponsored by Novartis, KOTRA, Samsung,)
2009
2011
2011.07. Tanibirumab: Phase I IND approval in Korea
2012
2013
2013.09. Tanibirumab: Completion of Phase I
2015.01.06 KOSDAQ IPO Tech. Evaluation Passed 2016.02 Tanibirumab: Phase IIa FIH in Australia
2014
2015
2015.03 Series C Fund - KRW 2 B (IMM Investment)
2014.03 ~ 2014.12 Tanibirumab & PMC-001 License-Out (3SBio, Triphase etc.)
2016
2016.04. Preliminary Listing Application
2015.12.18 KOSDAQ IPO Tech. Evaluation Passed
Our Key Performances
• Patent Registration : 26 cases
• Fully Human Ab Tech. (Fully Human Phage Display Library& Innovative Selection System)
Platform Tech.
• Next-generation Bispecific Ab Tech. (DIG-bodyTM
& PIG-body
TM)
IP Rights
• Patent Application : 14 cases • Trademark Registration: 4 cases
• Co-development with
• Patients-derived CSC Library
Sanofi (‘12, 1 case)
and Disease-bearing Mouse System
• Domestic L/O (‘13, 1 case)
Business Results Human Resources • Specialized Research Group for Ab Dev. • Global-leveled BOD & SAB • Global Human Networks
• Domestic & Oversea L/O
(’14, 4 cases) • Government Grant (‘09~’15, $ 12 M)
Pipelines
• Tanibirumab (Phase IIa) • PMC-001 (Preclinical stage) • PMC-201 & PMC-102 (In vivo completed) • PMC-xxxs (>20 Hit candidates)
PharmAbcine’s People Honored
CEO: Dr. Jin-San Yoo •
PharmAbcine, CEO/President
•
KRIBB, Head of The Therapeutic Antibody Ctr.
•
LG Life Science, Head of Therapeutic Antibody Division
•
The SCRIPPS Research Institute, TSRI Senior Associate
•
Howard Hughes Medical Institute at Stanford Univ., HHMI Associate
•
Max-Planck-Institute (MS & Ph.D), MPI Fellow
•
Georg-August Univ. Goettingen (BS)
Global-leveled Management •
Finance and BD : Dr. Sung-Woo Kim
•
Management : Dr. Christopher Kim
PharmAbcine (KAIST/Tech. Analyst) Oxford Bioscience Partners (Texas
Univ./Carnegie Melon Univ., MBA) •
Berkeley MBA) •
R&D : Prof. Dong-Sup Lee Seoul Nat’l Univ. (SNU, MD/Ph.D)
Commendation from MFDS (‘16)
•
Commendation from Ministry of Science, ICT, and Future Planning (‘15)
•
Commendation from Ministry of Health and Welfare (‘14)
•
Commendation from Ministry of Trade, Industry and Energy (‘14)
•
TechConnect National Innovation Award (14’, TechConnect World)
•
Korea Eureka Day Award (14’, KIAT)
•
Pharma Idol (‘12, 7th Annual China Pharmaceutical R&D summit)
•
The 1st GATE project Best Award (’08, Novartis, KOTRA, Samsung etc.)
Innovative R&D Center
Global-leveled SAB • •
Management : Dr. Hongbo Lu OrbiMed (Washington Univ./UC
•
•
Chairman : Prof. Waun Ki Hong MD Anderson Cancer Center, USA Director : Prof. Dong Moon Shin Winship Cancer Institute, USA Director : Prof. Do Hyun Nam Samsung Medical Center, KOREA
•
Head : Dr. Weon Sup Lee PharmAbcine (KAIST)
•
Team 1 Leader : Dr. Jinsang Yoo PharmAbcine (SKKU)
•
Team 2 Leader : Dr. Keun Hee Oh PharmAbcine (SNU)
•
Team 3 Leader : Dr. Sung Ho Ahn PharmAbcine (Tokyo Institute of Technology)
PharmAbcine’s Strategic or financial Investors
And Angel investor!
Our Business Model Leading Global Antibody Therapeutics Management from Global Standard BOD
Profit Structures
(OrbiMed, Novartis, Oxford Biosciences) Internal Discovery and Pipeline Development • Licensing out after PK/Tox study • Profit from Upfront and Milestones (fixed and running royalties)
Innovative Technologies Fully Human mAb Tech.
Bispecific Ab Platform Tech. Cancer Stem Cell (CSC) Library ADC-based Pipelines
Co-Development with Strategic Partners • Build Competitive Pipelines • Licensing out after Phase I or Phase II • Profit from Upfront and Milestones (fixed and running royalties)
CAR-T/NK based pipelines Immune checkpoint blockades
Clinical Sciences from Global Standard SAB (MD Anderson Cancer Center, Winship Cancer Institute, Samsung Medical Center)
Fee based Service • OEM Service for Research Antibody Production • OEM Service for Antibody Biopanning • Profit from Fee for Service
I. PharmAbcine’s antibody library Fully Human Antibodies Highly Successful Screening Hit rate High Diversity (about 10^12) Living Antigen Customized Antibody Selection System Accumulated know-how and experience to handle right panning! Diverse antibodies with cross species cross reactivity, sub nanoM affinity and Biology against most druggable targets!
II. Fully human IgG pipeline, as best in class
Fully human IgG against:
KDR EGFR EGFRviii cMET ANG2 TIE2 VEGF-C
PD-1 PD-L1 CTLA-4 OX40 TIM3 VISTA PMC-1 PMC-2 PMC-3
Anti-cMET agonistic antibody was out-licensed to ViroMed who’s developing for ischemia, diabetic ulcer, and non oncology indications. http://immuno-oncologynews.com/2014/11/24/3sbio-pharmabcine-sign-agreement-tanibirumab/ http://eng.kddf.org/Tasks/?mode=view&TaskId=104&p=1&DiseaseId=Others
III. DIG-Body Platform and its pipeline:
IgG Backbone Primary target
Func. Domain secondary target
KDR EGFR cMET PD-1 PD-L1 PMC-1 PMC-2 PMC-3
TIE2 DLL4 EGFR cMET PD-1 PD-L1 PMC-1 PMC-2 PMC-3 PMC-4
IV. TIG-Body Platform and its pipelines:
IgG Backbone Func. Domain as primary target as secondary target KDR EGFR EGFRviii cMET PD-L1 PMC-1 PMC-2 PMC-3
DLL4 EGFR EGFRviii cMET PD-L1 PMC-1 PMC-2 PMC-3 PMC-4
Func. Domain as tertiary target CD3
V. PIG-Body Platform and its pipeline
IgG Backbone as primary target KDR
Func. Domain as secondary target cMET
VI. 3G-System Platform for the best performing: CHO cell based High Performance System
>3g/L
3G-Cell line Completed: Protein X Protein Y Avastin biosimilar Zaltrap/Eylea biosimilar On going: Humira biosimilar Xolair biosimilar
VII. Next Generation ADC and CAR-T/NK ADC technology with LinXis
CAR-T Technology with
PharmAbcine: anti-EGFRvIII gene sequence Partner: CAR-T technology
CAR-NK technology with KRIBB
PharmAbcine: anti-EGFRvIII mAb LinXis: Lx® Technology Product: LIN004 (Pre-IND) http://www.linxispharmaceuticals.com/technology/pipeline/
PharmAbcine: anti-cMET mAb-ADC Under the codevelopment partnership
PharmAbcine: anti-EGFRvIII gene sequence KRIBB: CAR-NK technology
Summary of Science & Technologies Commercialization Tech.
Platform Tech. Fully Human Ab Development Technology Bispecific Ab Development
•
Commercialization Technology
Tumor and tumor microenviroment targeting Ab: Tanibirumab, anti-Tie2, anti-Ang2, etc.
Phase IIa 1 case
•
ADC or CAR-T/NK cells: anti-EGFRvIII
L/O 3 cases
•
Immune checkpoint targeting: anti-Ox40, PDL1, Tim3, VISTA, etc.
•
Tumor and tumor microenviroment targeting Ab: PMC-001 (DIG-KT), PMC-201 (DIG-KN), PMC-404 (DIG-AVc), etc.
•
Technology Ab Therapeutics
Ref.
Immune checkpoint blockades:
L/O 2 cases
anti-PDL1 & CD47
•
Fee for service: 1. Ab discovery (Biopanning) 2. Cell line develop (3G Expression System) 3. Ab production (from lab to pilot scale) 4. Ab characterization
> 2 cases/year
Selected Pipeline roadmap summary Discovery (Early Stage)
`
Pre-clinic (Late Stage)
Phase I
Phase II
Phase III
recurrent GBM
2016 ~ 2019 AMD
2016 ~ 2018
Tanibirumab (Anti-VEGFR2)
TNBC
2018 ~ 2020 China IND 2017
PMC-001, (Anti-VEGFR2 & Tie2)
2018~2019
Gastric cancer
2020 ~ 2024
Pancreatic cancer
US IND 2018
2019~2020
2021 ~ 2022 Breast, Colorectal, Liver cancer
2021 ~ 2024
PMC-307, (Anti-VISTA)
IND 2018
2020
> 20 PMC-XXX, Anti-TME
IND 2019
2020
Tanibirumab Summary-1
A fully human mAb and targets VEGFR2 (KDR) which is major factor on tumor angiogenesis.
The only mAb therapeutics having murine cross-reactivity among VEGFR2 targeting mAbs under clinical development and therefore, is applicable to translational research using various disease bearing mouse model systems and as a result, can increase the possibility of success in clinical study.
Phase I study was successfully completed at Samsung Medical Center in Korea and Phase IIa with recurrent GBM patients is scheduled to initiate in Australia early this year. (http://clinicaltrials.gov/ct2/show/NCT01660360?term=tanibirumab&rank=1)
Received Innovation Award from TechConnect World which holds a technology exhibition annually in USA (‘14) (http://www.techconnectworld.com/World2014/participate/innovation/innovation_awards.html)
Selected as Best Project in National Research Project 2015 (‘15)
License-Out : TaeJoon pharma (Korea) – indication: Eye diseases, territory: Global 3Sbio (China) – indication: All diseases except eye diseases, territory: China, Russia, Brazil,
♦ IP status Registered
Thailand, Taiwan
Korea, Japan, China, Singapore, EP (15 countries), Australia, Canada, USA
Tanibirumab Summary-2 Preclinical result
Tanibirumab had shown Anti-cancer efficacies in various tumor-bearing mouse models. A549 Lung Cancer - Efficacy: Tanibirumab = Avastin - Apoptosis: Tanibirumab = Avastin MCF-7 Breast Cancer - Tanibirumab > Avastin MCF-7 Breast Cancer Metastasis - Tanibirumab > Avastin Colo205 Colon Cancer
Efficacy comparison between Tanibirumab and Avastin in U87-MG GBM orthotopic mouse model
Hep3B Liver Cancer
Phase I result
http://www.ncbi.nlm.nih.gov/pubmed/25942475 http://www.ncbi.nlm.nih.gov/pubmed/26325365
Up to cohorts 8 (24 mg/kg ) has no DLT observed.
Study of PK/PD from Phase I suggested that increase of some biomarkers (VEGF-A, sVEGFR-2, PlGF) were observed.
Displayed no common side effects like hypertension, bleeding, hemorrhage appeared in Cyramza, Avastin, Zaltrap, Sutent, Nexavar and other VEGF antagonists.
Shown several SD patients (~60%) from all terminal staged cancer patients in Phase I.
Revealed reversible capillary hemangioma on the skin to 50% of patients who were treated with Tanibiruamb http://meetinglibrary.asco.org/content/149434-156
PMC-001(DIG-KT) Summary-1 VEGF/VEGFR2 and Ang2/Tie2 pathways are well known as major factors that induce tumor angiogenesis and metastasis. It is expected to have superior anti-cancer activity to Avastin by simultaneously neutralizing VEGF/VEGFR2 and Ang2/Tie2 pathways. We have constructed global-leveled bispecific antibody by DIG-bodyTM platform technology and licensed-out in early developmental stage. We have received Korea Eureka Day Award from the Government (‘14). License-Out : 3Sbio(China) – All indications for human (China and Korea)
♦ IP Status Registered Pending
Korea, Australia, Singapore, Russia, China, Japan, EP USA, Canada, Brazil, India
PMC-001(DIG-KT) Summary-2 ďƒ˜ Preclinical Results
Recurrent GBM Orthotopic Model
Liver Cancer Xenograft Model
Anti-tumor efficacy test in Hep3B-xenografted mouse
Pancreatic Cancer Orthotopic Model
Anti-tumor efficacy test in Avastin resistant, U87-MGxenografted mouse
Pancreatic Cancer Metastasis Model
Anti-tumor and anti-metastasis efficacy test in CFPAC-1_Luc- xenografted mouse
Tanibirumab and PMC-001 are best candidate to combine with Immune Checkpoint blockade
Angiogenesis inhibitors like Avastin and Cyramza with Immune Checkpoint blockades combinations shows synergy of efficacy and manageable safety. Since Avastin, Cyramza, Tanibirumab and PMC-001 have tendency to normalize and stabilize disorganized , leaky and fragile tumor vessel temporary in early phase. During that period of time, immune checkpoint blockade can be delivered to tumor site more efficiently by using the temporary normalized vessel.
In the same way, the activated killer T cell can utilize this normalized vessel to reach tumor mass.
Tanibirumab and PMC-001 are best candidate to combine with Immune Checkpoint blockade
Inhibitors of VEGF-VEGFR2 pathway also help activated killer T cell to infiltrate into cancer cells. We strongly believe that Tanibirumab and PMC-001 will be the best candidate to combine with Immune Checkpoint blockades to perform the superiority to Avastin or Cyramza due to their limitation.
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