37th Annual J.P. Morgan Healthcare Conference TWO (Taipei Exchange) January 9, 2019

Transcription

1 37th Annual J.P. Morgan Healthcare Conference 4162.TWO (Taipei Exchange) January 9,

2 Mission & Vision Mission Unlock value for promising therapeutic candidates & Extend patients lives Vision Top tier innovative oncology drug development company 2

3 The year 2019 marks PharmaEngine s 16 th year committing to oncology new drug development Founded the Company Licensed-in PEP02 (Onivyde) Licensed-out PEP02 (Onivyde) in 2011 Turned profitable since 2014 Established PharmaEngine Europe in Paris in 2015 Onivyde approved in 38 countries Built marketing & sales in Taiwan in 2016 US$120mn cash and equivalent on hand Royalty plus US$165mn of milestones to be collected Generated positive data of PEP503 in STS pivotal trial in Establish a strong and diversified portfolio Build different business units to maximize global presence Raise funding if needed Become a world class drug development company 3

4 Market Capitalization ~US$550mn Market Cap Taipei Exchange: 4162.TW 30 People ~US$18mn Market Cap per Employee 4

5 Profitability Through successful out-licensing of ONIVYDE Eligible to receive up to US$266.5mn milestone plus tiered royalty US$101.5mn milestone achieved so far, US$165mn to go ~ US$120mn cash and equivalent on hand 5

6 Business Model NRDO + Drug Discovery Drug Development Marketing & Sales Target Validation Lead Optimization Pre-Clinical Phase I Phase II Phase III Approval Market In-licensing Partners Out-licensing Partners Taiwan Right Outsourcing CMO Partners Hospital Organizations CRO Partners International Partnership 6

7 Core Competence Strong Oncology Experts Network Product Project In-licensing Development Strategy & Out-licensing Evaluation Deals Execution Deals Negotiation Negotiation Scale up & Manufacturing Design Clinical Trials Design & Execution Global Regulations Compliance Financial Risk Management 7

8 Management Team (1) Grace Yeh, Ph.D. Founder, President & CEO Ph.D., Immunology, Medical U. of South Carolina, USA Blond McIndoe Center of Transplantation Biology, UK; INSERM, France T Cell Sciences; CytoMed; LeukoSite; Millennium (Cambridge, MA, USA) Alain Herrera, MD Head, Corporate Development; Managing Director, PharmaEngine Europe MD, Pitié-Salpétrière Hospital, France CNRS; Roger Bellon; Pierre Fabre; Chiron Global Oncology Business Strategy & Development, Sanofi; Alain Oncology Consulting 8

9 Management Team (2) Hubert Chan, MD and Ph.D. VP, Clinical & Regulatory Affairs MD, Chung Shan Medical School, Taiwan Ph.D., Pathobiology, U. of Minnesota, USA Certified Medical Oncologist, Cancer Centers; National Institute of Cancer Research, Taiwan Armand de Gramont, Ph.D. VP, Translational Science Ph.D. in Cellular Biochemistry at Pierre et Marie Curie University, France Postdoctoral fellowship in NIH, USA Head of the New Drugs Evaluation Laboratory, Lausanne University Hospital, Switzerland 9

10 Scientific and Clinical Advisors Head of Internal Medicine in Oncology, French-British Institute Hospital, Prof. Aimery de Gramont Paris, France Expert in gastrointestinal cancer (FOLFIRI, FOLFOX) Sponsor of the colorectal (PEPCOL) cancer study Prof. Li-Tzong Chen Director, National Institute of Cancer Research, Taiwan Expert in gastrointestinal cancer Participated in phase I, II, and III studies of PEP02 (Onivyde) Prof. Eric Raymond Head of Medical Oncology, Saint-Joseph Hospital Group, Paris, France Principal Investigator of > 50 clinical trials Dr. Shin-San Michael Su Chief Scientific Officer, Decibel Therapeutics, Inc., Cambridge, MA, USA Co-Founder & Senior VP, R & D, Agios Pharmaceuticals, Cambridge, MA, USA 10

11 Product Development Pipeline Product Mechanism Indication Preclinical Phase I Phase II Phase III Approval Commercial Rights Partner Pancreatic Cancer Approved (2nd-line) Topo 1 Pancreatic Cancer... ONIVYDE Ib/III Study Design Milestone (EU/Asia) Inhibitor (1st-line) Ipsen Royalty (EU/Asia) (PEP02) (Liposome Small Cell Lung Cancer (Licensee)... Ib/III Study Design Taiwan Sales Formulation) (2nd-line) PEP503 Radiotherapy Enhancer (Hafnium Oxide) Various ISTs Soft Tissue Sarcoma (RTx alone) Head and Neck Cancer (RTx alone) Head and Neck Cancer (RTx + Chemo) Liver Cancer (RTx alone) Rectal Cancer (RTx + Chemo) Prostate Cancer (RTx alone) Head and Neck Cancer & Lung Cancer (I/O + RTx) PEP06 NCE Solid Tumor Preclinical Preclinical Phase I/II IND Approved Phase II/III Phase I Phase II Phase III Approval Approval Asia Pacific Global excl. Mainland China Nanobiotix (Licensor) Guangzhou Bebetter (Licensor) 11

12 New Project Evaluation Strategy Repeat Our Success Oncology Project Source 1. Partnering meetings 2. Referral Medical Devices New Drugs (Small molecules+biologics) Classification First-in-class Best-in-class Mechanism Drug Resistance Cancer Metabolism Tumor Microenvironment Others Stage Enter into Phase I within 1 year or In Phase I Development Stage Preclinical Phase I Phase II 12

13 ONIVYDE is a liposome formulation Lipid membrane of irinotecan Sustained release profile PEG-DSPE Preferential tumor accumulation (EPR effect)* Site-specific activation** Note: * Enhanced Permeability and Retention effect ** Irinotecan is converted into the 100 to 1000-fold active metabolite, SN-38, by enzymes around tumors. Internal aqueous space 13

14 Financial Transactions Transaction value for Onivyde has reached around US$2 bn. Its market potential drive largecap peers to in-license it and help product development as well as sales. Large cap ex-us (ex-tw) US 2018 Sold Oncology Unit Acquired US + ex-us (ex-tw) TW 2014 Out-license Ex-US US$970 mn 2017 Sold Onivyde US$1.025 bn* 2011 Small Cap 2003 In-license EU/Asia US$3 mn Out-license EU/Asia (excl. TW) Milestone US$220mn Sublicense Rev US$46.5mn (2014) Tiered Royalty * Merrimack sold Onivyde plus generic version of DOXIL to Ipsen for up to $1.025bn 14

15 In the global Phase III study for Onivyde, PharmaEngine held responsibilities for Taiwan Clinical Trial Execution sites and recruited as many as 23% of patients, which was unprecedented considering the potential patient numbers in Taiwan is much less than in other populated areas. Phase III (NAPOLI-1) Enrollment for Onivyde 17% Canada USA 37% Czech Republic France Germany Hungary Italy Spain United Kingdom 9% South Korea 9% Korea 23% Taiwan 4% Argentina Brazil 10% Australia 15

16 NAPOLI 1: A Randomized, Open Label Phase 3 Study of MM-398, with or without 5- Fluorouracil plus Leucovorin, and 5-Fluorouracil plus Leucovorin in Patients with Metastatic Pancreatic Cancer Who have Failed Prior Gemcitabine-based Therapy Onivyde improved OS significantly Total Population (OS = 6.1 m vs. 4.2 m) OS was significantly longer in Asian subgroup Asian Subgroup (OS = 8.9 m vs. 3.7 m) 4.2 months 6.1 months months Extend OS from 4.2 months to 6.1 months after Gemcitabine failure One in four patients treated with ONIVYDE survived one year or more: Increased 1 Year Survival by 63% in Metastatic Pancreatic Cancer with a well-defined safety and tolerability profile. 16

17 Little Progress in the Pancreatic Cancer Treatment for the Past 20 Years Metastatic pancreatic cancer is a challenging malignancy and 5 year survival rates are only 8.5% The number #2 cancer in the number of deaths, although accounting for <5% of all cancer patients (US) Between 1997 and 2015, from a total of 35 unique therapeutic regimens evaluated in 39 Phase III pancreatic adenocarcinoma trials, only 11% had favorable outcomes. Notably, 85% of these Phase III trials were conducted despite preceding Phase II studies not meeting their primary endpoints 17

18 Onivyde - Approved in 38 countries for post-gem metastatic pancreatic cancer FIRST US FDA approved cancer drug from Taiwan (2015) MAA approval from EMA (2016), 31 countries Approval in Taiwan (2015), Korea (2017), Singapore (2017) Approval in Australia (2016), Canada (2017), Switzerland (2017) ESMO guidelines (2015), US NCCN guidelines (2016) 18

19 PEP503 Nanobiotix (FP: NANO) Licensing Partner Pioneering nanomedicine for over 10 years Based in Paris, France with more than 100 employees Incorporated in 2003 A spin-off from the State University of New York at Buffalo IPO in Oct (Euronext: NANO) PharmaEngine licensed the exclusive rights to develop and commercialize PEP503 in the Asia Pacific region from Nanobiotix in Aug Nanobiotix is eligible for US$57 mn, of which US$54 mn remains, plus up to double digit royalty. Chairman, Nanobiotix CEO, Nanobiotix 19

20 Radioenhancer: 9-fold Dose Enhancement A nanoparticle formulation of crystalline hafnium oxide (HfO2) - inert High Electron Density Classified in the EU as class III medical device Filed for CE Marking in Europe and currently under review Local treatment of tumors to enhance the efficacy of radiotherapy An increased probability of photoelectric interaction at lower energy levels due to Its high atomic number PEP503 Crystalline HfO2 20

21 PEP503 Radioenhancer: 9-fold Dose Enhancement Approximately 60% of patients with cancer require Radiotherapy alone radiotherapy Normal tissue toxicity limits the doses used in X-ray water e - Cell damage standard radiation therapy Efficacy Conventional Radiotherapy Radioenhancer Radiotherapy + PEP503 e - e - X-ray HfO e - 2 e - e - Cell damage Safety 21

22 PEP503 Soft Tissue Sarcoma Population: Patients with soft tissue sarcoma of the extremity and trunk wall Global Pivotal (Phase II/III) STS Study PEP503 injection Radiotherapy (standard of care) 25 daily sessions X 2GY per session surgery end of treatment week1 week2 week3 week4 week5 Soft Tissue Sarcoma 176 patients R 1:1 Study Arm (87 pts): PEP503 + RT (50 Gy 2 GyF) Comparator Arm (89 pts): RT alone (50 Gy 2 GyF) Source: Nanobiotix corporate presentation; 2017/3 Stratification Population: myxoid liposarcomas vs others 22

23 PEP503 Proof of Concept PEP503 Soft Tissue Sarcoma Global Pivotal Study in STS met primary and secondary endpoints Pathological Complete Response Rate (pcrr) Resection Margins (R0 Rate) p-value p-value Primary Endpoint Secondary Endpoint 23

24 PEP503 Immunotherapy PEP503 in Combination with Immunotherapy PEP503 plus radiotherapy could efficiently prime the adaptive antitumor immune response, turning cold tumors into hot tumors PEP503 could become a backbone in Immuno-oncology (I/O) Only 15-20% of non-small-cell lung cancer patients (NSCLC) respond to immunotherapy Only 13-22% of head and neck squamous cell carcinoma patients respond to immunotherapy IND application approved in the U.S. for a clinical trial with PEP503 in combination with I/O Source:Nanobiotix 24

25 2018 Key Events Onivyde Obtain reimbursement from Taiwan National Insurance Bureau Initiate frontline pancreatic cancer and second-line small cell lung cancer studies PEP503 Pivotal STS trial data Assess license-out opportunity Expand product portfolio License-in and initiate new development projects 25

26 5-year Planning Become a world class drug development company Competitiveness Globalization Financing Establish a strong and diversified portfolio Build different business units to maximize global presence Raise funding for new drug development projects, if needed List in other stock market 26