Polaris Group. Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers

Transcription

1 北極星藥業集團 2016/1/29

2 Polaris Group Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers Late Stage Clinical Development Already in multiple global Phase II, III clinical studies Fully Integrated Pharmaceutical Company Early stage R&D, worldwide clinical trials, cgmp production

3 Polaris Group Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers Late Stage Clinical Development Already in multiple global Phase II, III clinical studies Fully Integrated Pharmaceutical Company Early stage R&D, worldwide clinical trials, cgmp production

4 Arginine Metabolism in Normal Cells Internal: Urea Cycle CELLS External: FOOD In normal cells, arginine can be obtained via two different routes: 1. External nutritional intake 2. Internal synthesis via urea cycle

5 Arginine Metabolism In Cancer Cells With No Urea Cycle Internal: Urea Cycle X CELLS External: FOOD Some cancer cells lack an urea cycle enzyme: ASS (argininosuccinate synthetase) and cannot make arginine internally These cells rely exclusively on external supplies of arginine for survival and growth

6 ADI-PEG 20 A Novel Treatment For ASS-deficient Cancer Cells Internal: Urea Cycle X CELLS X External: FOOD Arginine deiminase (ADI) is a microbial enzyme that catalyzes arginine into citrulline ASS-deficient cancer cells are fatally affected by ADI treatment because they cannot obtain arginine internally and externally

7 ADI-PEG 20 Does Not Affect Metabolism of Normal Cells Internal: Urea Cycle CELLS X External: FOOD Normal cells are not affected by ADI because they can produce arginine internally via the urea cycle ADI-PEG 20 could be a safe and effective treatment for cancers

8 ADI-PEG 20 Development Background Exploiting amino acid deprivation as treatment for cancer began in the 1960s About 50% of children with ALL responded to Asparaginase When used in combination with chemotherapies, about 90% of the children responded for over 20 years, making childhood ALL a curable disease First batch of ADI PEG 20 was manufactured by Phoenix Pharmacologics in Lexington, KY in Asparaginase, an asparagine processing enzyme, was developed to treat acute lymphoblastic leukemia (ALL) Led by Dr. Lloyd Old at Memorial Sloan- Kettering (MSKCC) and Ludwig Institute For Cancer Research (LICR) in the 1970s Dr. Old initiated ADI project in 1990 at LICR. Early research focused on HCC and melanoma

9 ASS-deficiency Has Been Detected in Multiple Cancer Types Cancer Type No. of Tumor Samples Analysed % of ASS-Deficient Samples Prostate Cancer % Renal Cell Carcinoma % Melanoma > % Lymphoma % Sarcoma % Pancreatic Cancer 47 87% Acute Myelogenous Leukemia 53 87% Hepatocellular Carcinoma 44 75% Mesothelioma 82 63% Gastric Cancer % Small Cell Lung Cancer 16 44% Breast Cancer % Non Small Cell Lung Cancer 90 31% Glioblastoma 55 71% Colorectal Cancer 31 3% ASS deficiency as detected by immuno-histochemical (IHC) staining of biopsy samples

10 Selected Polaris Group Collaborators Institute Cancer Type Institute Cancer Type City of Hope, National Medical Center Breast Cancer Johns Hopkins University Head & Neck Cancer Albert Einstein College of Medicine, New York Barts and The London School of Medicine Baylor University Brighton and Sussex Medical School Cambridge University Cedars-Sinai Medical Center Osteosarcoma Bladder Cancer, AML, NHL, Mesothelioma Nutrition, bowel function Breast Cancer Hypoxia and cancer Ovarian Cancer King's College London Mayo Clinic MD Anderson Cancer Center National Health Research Institutes (Taiwan) Scott & White Clinic, Texas University of California, San Diego Ovarian Carcinoma Renal Cell Carcinoma Melanoma Pancreatic Cancer Prostate Cancer, Breast cancer AML Uveal Melanoma, Glioblastoma Charité, University in Berlin. Melanoma University of California, Davis Prostate Cancer Pancreatic Cancer Chi-Mei Medical Center Myxofibrosarcoma, Bladder Cancer VA Medical Center and University of Miami Melanoma Chulabhorn Research Institute Biliary Cancer University of Leicester Colon Cancer Cleveland Clinic Sarcoma University of Mainz, Germany Neutrophil function Imperial College London Glioma Washington University AML, Sarcoma Uveal Melanoma

11 Polaris Group Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers Late Stage Clinical Development Already in multiple global Phase II, III clinical studies Fully Integrated Pharmaceutical Company Early stage R&D, worldwide clinical trials, cgmp production

12 ADI-PEG 20 Clinical Development History The first Investigation of New Drug (IND) in the USA was filed in 2001 ADI-PEG 20 has 4 active INDs in the US. Orphan drug status for HCC mesothelioma, and melanoma 11 completed and 11 ongoing clinical studies More clinical studies will be initiated in The first patent application for worldwide rights of ADI-PEG 20 was submitted in 1998 The first clinical study, a Phase I/II study for HCC, was initiated in 2001 at MD Anderson Cancer Center Over 1,000 patients have been treated with ADI-PEG 20 The drug has demonstrated safety and strong efficacy in multiple cancer types

13 Completed Clinical Trials: 11 Cancer Type Study Phase Clinical Centers Principal Investigator Subjects Total I/II MD Anderson Cancer Center Steven Curley 37 Hepatocellular Carcinoma I/II Pascale National Cancer Institute, Italy Francesco Izzo 19 IIB Pascale National Cancer Institute, Italy Francesco Izzo 76 Metastatic Melanoma II Eight clinical centers in Taiwan Li-Tzong Chen 71 I Indiana University, University of Miami Ted Logan (IU) 15 I/II Pascale National Cancer Institute, Italy Paolo Ascierto 24 II University of Miami Lynn Feun 39 Mesothelioma II Barts and 5 other centers in UK Peter Szlosarek 68 Small Cell Lung Carcinoma Pediatric Cancers I/II Memorial Sloan-Kettering & NYU Jedd Wolchok 34 Global study led by Memorial Sloan- II Lee Krug 21 Kettering I MD Anderson Cancer Center Cynthia E. Herzog 8

14 Ongoing Clinical Trials: 11 Study Phase III II II II I/IB I/IB Study Type Cancer Type Clinical Center(s) Monotherapy Monotherapy Monotherapy + TACE + Docetaxel + Cisplatin Hepatocellar Carcinoma Acute Myeloid Leukemia Non-Hodgkin's Lymphoma Hepatocellular Carcinoma Prostate Cancer Non-Small Cell Lung Cancer Cutaneous melanoma Uveal melanoma Ovarian Cancer Hepatocellular Carcinoma Cholangioarcinoma 75 centers worldwide, lead by MSKCC Multiple centers Taiwan Multiple centers Taiwan Multiple centers Taiwan University of California, Davis Principal Investigator Ghassan Abou-Alfa Initiation Date Patient # Jan July 2011 T. Y. Chen November 2013 T. Y. Chen P. J. Chen Lucky Lara November 2013 October 2014 August 2011 MD Anderson Siquing Fu November I/IB + Doxorubicin Breast MD Anderson Siquing Fu April I/IB + Sorafenib Hepatocellular Carcinoma I/IB I/IB I/IB + Gemcitabine + nab-paclitaxel + Cisplatin + Pemetrexed Pancreatic Cancer MSKCC Eileen O'Reilly January Mesothelioma Non-Small Cell Lung Cancer Uveal Melanoma Glioma Hepatocellular Carcinoma MSKCC Univ. of Washington + FOLFOX Hepatocellular Carcinoma MSKCC Barts Cambridge Kings College Ghassan Abou-Alfa Ghassan Abou-Alfa Peter Szlosarek December 2014 January 2015 July

15 ADI-PEG 20 Clinical Study Overall Observations ADI-PEG 20 has been clinically tested in 22 clinical studies globally in patients with a variety of end stage cancer types. Over 1,000 patients have received ADI- PEG 20 as monotherapy or in combination with other treatment. All clinical studies have been conducted under US FDA INDs. Half of the studies were led by Memorial Sloan-kettering Cancer Center or MD Anderson Cancer Center, two of the most prestigious cancer centers in the world. ADI-PEG 20 has demonstrated apparent anti-tumor activity and excellent safety Most patients taking ADI-PEG 20 can continued treatment without sacrificing quality of life. The duration of treatment ranged from weeks to 4 years. ADI-PEG 20 is administered intra-muscularly. Patients typically receive treatment once a week as out-patients, and are observed for 1 hour, and then released. They do not require extensive hospital stays for treatment.

16 What Is Next for ADI-PEG 20? The clinical development strategy of ADI-PEG 20 should resonate with the corporate vision and business development There are two main focus for the clinical development and business development of ADI-PEG 20 in : 1. Obtain worldwide approval 2. Maximize shareholder s value

17 ADI-PEG 20 Clinical Development Strategy 1. Obtain market approval via Biologics License Application (BLA) The most significant and immediate value generator in the pharmaceutical industry is to get a drug approved, for any indication 2. Expand market by testing more ASS-deficient tumors in trials The value of a drug can be further increased with expanded market 3. Enhance efficacy by combination therapies Demonstrate superior efficacy and safety to the current treatment can significantly increase the market penetration within each cancer market

18 ADI-PEG 20 Competitive Edge Late stage project with multiple ongoing clinical trials Significant de-risking has already occurred as project already in Phase II, III studies ASS deficiency common across multiple cancer types An expanded market scope with multiple potential indications Unique mechanism of action perfect for combination therapy Complementary with other anti-cancer treatments Well tolerated by patients as normal cells can make arginine ASS companion diagnostic personalized medicine Identify patients most likely to benefit from ADI-PEG 20 In-house cgmp production ensures supply and quality of drug Strong collaborations with world-class cancer centers Complements the oncology pipeline of all global pharmaceutical companies and is primed for strategic partnership

19 Polaris Group Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers Late Stage Clinical Development Already in multiple global Phase II, III clinical studies Fully Integrated Pharmaceutical Company Early stage R&D, worldwide clinical trials, cgmp production

20 Polaris Group Corporate Structure CONFIDENTIAL Polaris Group (Cayman) TDW Group (Cayman) Polaris Pharmaceuticals (California) TDW HK (Hong Kong) DRX-USA (California) DRX-CD (China) DRX-SH (China) TDW Pharmaceuticals (Taiwan)

21 Polaris Group Subsidiaries Polaris Group Founded in 2008 as the holding company in Cayman. Owns the worldwide rights of ADI-PEG 20, a novel biologics being developed for a variety of cancers in 22 completed and ongoing clinical studies globally. Polaris Pharmaceutical, Inc. Founded in 2008, based in San Diego. Has a R&D team that conducts early stage discovery research, translational research, pre-clinical research and global clinical studies. Collaborates with over 20 world-class cancer institutes and academia centers for the development of ADI-PEG 20. TDW Pharmaceuticals (TDW) Founded in 2003, responsible for clinical studies in Taiwan and Korea. DesigneRx Pharmaceuticals -USA (DRX-USA) Founded in Built a cgmp facility for biologics in 2005 and has been providing ADI-PEG 20 for all clinical studies for over 10 years. DesigneRx Pharmaceuticals -Chengdu (DRX-CD) Founded in Acquired 68 acres in Chengdu Hightech Industrial Park in Currently building a worldclass large scale cgmp facility for biologics; projected to be completed and validated in DesigneRx Pharmaceuticals -Shanghai (DRX-SH) Founded in 2007 in Shanghai, China. Responsible for conducting in vitro and in vivo studies to identify additional cancer types for ADI-PEG 20. It is also in charge of clinical development in China.

22 Experienced Management Team Name Title Previous Experiences Bor-Wen Wu, Ph.D. John Bomalaski, M.D. Shaw Chen, M.D., Ph.D. Wei He, Ph.D. James Thomson, Ph.D. Steve Wu Amanda Johnston, Ph.D. Jenny Kung, Ph.D. Archie Prestayko, Ph.D. Irene Kuo Chairman and CEO EVP Medical Affairs EVP Regulatory Affairs VP Translational Research President DRX-SH, CSO DRX-CD VP R&D, President DRX-USA VP TWD Pharma, COO DRX-CD VP Clinical Affairs VP Product Compliance VP Corporate Affairs VP Financial Affairs

23 DesigneRx - Vacaville

24 DesigneRx - Chengdu

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26 Polaris Group by Numbers

27 The Company No. of subsidiaries: 9 No. of years since the first Polaris company was founded: 14 No. of current oncology projects: 6 No. of global patents issued: 6 No. of new global patent applications: 5 No. of employees: 135 No. of Ph.D., M.D. and J.D.: 32

28 ADI-PEG 20: R&D Numbers No. of years since the first ADI experiment: 26 No. of years since ADI-PEG 20 was first made: 20 No. of published ADI-PEG 20 research articles: 95 No. of collaborators participated in ADI-PEG 20 research: >30 No. of different ADI species purified and characterized: 40 No. of ADI mutants made for characterization: >230 No. of ADI crystal structures solved in-house: 9 No. of second generation ADI patent applications: 3 No. of second generation ADI publications: 0

29 ADI-PEG 20: Clinical Numbers No. of years since the first clinical study: 15 No. of completed clinical trials: 11 No. of ongoing clinical trials: 11 No. of patients treated worldwide: >1000 No. of cancer types treated in clinical trials: 15 No. of additional cancer types treated in compassionate use: 4 No. of countries that have participated in ADI studies: 10 No. of hospitals that have participated in ADI studies : 88

30 ADI-PEG 20: Regulatory Numbers No. of IND (Investigation of New Drug) for ADI: 4 No. of years since the first IND was filed: 15 No. of orphan drug designations granted in US and EU: 5 No. of communications sent to the FDA at the end of 2015: 304 No. of Special Protocol Assessment granted: 1 No. of clinical study reports submitted to the FDA: 11

31 ADI-PEG 20: cgmp Production Numbers No. of years since the first cgmp facility was built: 12 No. of cgmp production lines: 2 (current); 1 (coming) No. of doses to be manufactured per year: 6,000 (previously) 250,000 (now) 3,000,000 (in 3 years) No. of years since the first US government audit: 11 No. of audits by US government or EU Qualified Person: 9 No. of ADI-PEG 20 formulations: 3 No. of years that ADI-PEG 20 are stable in storage conditions: >3 No. of current QC test methods: 25

32 Polaris Group Main Asset Has Great Market Potential A novel biologic for a wide variety of cancers Late Stage Clinical Development Already in multiple global Phase II, III clinical studies Fully Integrated Pharmaceutical Company Early stage R&D, worldwide clinical trials, cgmp production

33 核心價值 創新 : 以創新的思維切入新藥研發, 建立新的技術平台 誠信 : 誠信公開, 以完整確實的試驗數據訂定研發方向及經營策略 務實 : 實事求是, 以最快的速度將最好的新藥帶入市場 關懷 : 以 同理心 了解病人需求, 關懷病人家屬

34 THANK YOU