Precision Therapies. for. Women s Cancers

Transcription

1 Precision Therapies for Women s Cancers 1

2 Company Snapshot Founded Q3 2016, a Delaware Incorporation Spin-out of Oncology Venture Aps, Denmark Seed of $3.5 million completed December, 2016 Focus: advancing novel anti-cancer drug portfolio addressing significant women s cancer markets Addressable women s health therapeutics: market opportunity $15 billion Pipeline: 3 clinical stage cancer drugs, completed phase 1/2 trials and have shown patient response Unique proprietary biomarker technology, DRP : generating drug-specific RNA based biomarkers for selecting optimal indication & highly responsive patients, and serving as companion diagnostic Experienced drug development team Seeking $25 million series A to fund clinical development plan 2

3 New Therapies for Hard-to-Treat Women s Cancers 1 in 3 women will develop Cancer - 1 in 5 women will die from Cancer Breast Cancer The most common Cancer among women Affects 1 out of 8 women: 232,000 new cases/year (USA) 30% of metastatic Breast Cancer patients develop hard-to-treat brain metastases Ovarian Cancer The most lethal genital tract Cancer in women Affects 1 out of 75 women: 23,000 new cases/year (USA) Endometrial Cancer The 4:th most common Cancer among women Affects 1 out of 39 women: 60,000 new cases/year (USA) Primary, Malignant Brain Cancer 36,000 new patients annually worldwide 3

4 $ 15 Billion Market Opportunity Brain Metastases of Breast Cancer (BCBM) Over 50 % of HER2 + BCBM patients die from brain lesions - 160,000 new patients annually worldwide No existing therapy option for this patient group % of patients with mbc will develop brain metastases - 34,000 new patients annually worldwide Annual worldwide sales = 2.9 billion target $50,000 per course of therapy & 30 % DRP cut-off Cancers with HR-DDR Deficiency (incl. BRCA) 20% of Ovarian Cancer patients have BRCA mutations - 50,000 new patients annually worldwide Up to 10% Breast Cancer patients have BRCA mutations - 170,000 new patients annually worldwide Annual worldwide sales = 9.9 billion target $150,000 per course of therapy & 30% DRP cut-off Advanced Endometrial Cancer 80,000 new patients annually worldwide No second-line treatment options after chemotherapy Annual worldwide sales = 1.2 billion target $50,000 per course of therapy & 30% DRP cut-off Primary, Malignant Brain Cancer (Glioblastoma Multiforme, GBM) 36,000 new patients annually worldwide Highly lethal - No new front-line therapies in 20 years - Existing second-line therapies largely ineffective Annual worldwide sales = 702 million target $65,000 per course of therapy & 30% DRP cut-off 4

5 2X Oncology Business Strategy Acquire promising and novel clinical stage oncology assets that have shown efficacy in women (PoC) for further clinical development with a high probability of success Accelerate & De-risk Development with DRP (Drug Response Predictor) platform generating DRP biomarkers to select highly likely responder patients and the best indication for each drug Jump Start Targeted Phase 2a Trials in Denmark that can lead to rapid value inflection at a reduced cost Leverage existing, screened Breast Cancer patients (>1,000) Advance One Asset to Phase 2/3 Pivotal (randomized) Trial to further increase acquisition valuation Partner or Commercialize for Phase 3 clinical development to maximize return on investment 5

6 Pipeline Products Existing PoC and Safety Data 2X-111 Glutathione Enhanced PEGylated liposomal Doxorubicin Crosses blood brain barrier, BBB Phase 1 trial as single agent in 37 patients: well tolerated Phase 2a trial as single agent completed in Breast Cancer 14 patients = 41% had reduction in overall tumors 3 month PFS rate of 59% - 6 month PFS 18% Phase 2a trial as single agent completed in malignant, primary Brain Cancer (GBM) 14 patients: 40% had disease control, 14% had tumor reductions of 20% 2X-121 Small Molecule, PARP 1&2 Inhibitor and Tankyrase Inhibitor (oral) Crosses blood brain barrier, BBB Phase 1/2 trial as single agent completed in cancer patients 28 patients: 46% disease control rate and 7.1% partial response (PR) 2X-131 Small Molecule, Topoisomerase 1 Inhibitor (oral) Crosses blood brain barrier, BBB Phase 2 trials as single agent completed in Breast and Ovarian Cancer 119 patients: 24.6 % partial response (PR) in Ovarian Cancer, 42.8% PR and stable disease (SD) in Breast Cancer Less toxic than competitive drugs Topotecan and Irinotecan 6

7 Competition 2X-111 Glutathione Enhanced PEGylated liposomal Doxorubicin No current competing therapies for HER2+ or triple negative Breast Cancer patients who develop brain metastases Herceptin (trastuzumab) does not cross the brain blood barrier (BBB) 2X-121 Small Molecule, PARP 1&2 Inhibitor and Tankyrase Inhibitor (oral) Olaparib (Astra Zeneca: Lynparza ) approved for BRCA1/2 mutated Ovarian Cancer in EU/US (not approved for breast cancer in US/EU) Rucaparib (Clovis) approved in December 2016 Several other drugs (Velaparib, Niraparib, talazoparib and others) under development Olaparib does not cross the BBB and does not have synergistic Tankyrase inhibitory function 2X-131 Small Molecule, Topoisomerase 1Inhibitor (oral) Irinotecan approved for Colorectal Cancer. Topotecan approved for Cervical, Ovarian, and Lung Cancer Existing drugs have substantial myelotoxicity and/or GI toxicity and are disfavored by oncologists 2X-131 has improved safety profile compared to Irinotecan and Topotecan 7

8 Intellectual Property Portfolio Composition-of-matter patents / method-of-use patents coverage in major countries planned for commercialization of 2X Oncology portfolio compounds 2X-111 GST based delivery system patented through at least 2025 (US, JP, CN) and applications pending in EP, additional applications pending (filed 2010) Liposome drug conjugates for BBB delivery patented through at least 2028 in EP, additional application pending in US 2X-121 Compound and method-of-use patented through at least 2028 (US, EP, JP, CN), additional applications allowed for and/or pending elsewhere 2X-131 Compound patented through March 2020 (US, JP, CN, AU, NZ and elsewhere) Used to treat Uterine Cancers, patented through 2025 JP, CN, AU, NZ and elsewhere) Crystalline forms & formulations, patented through 2025/2026 (JP, CN, AU, NZ and elsewhere) DRP Biomarkers New patent applications are filed on a rolling basis as each new drug specific biomarker is developed US Pat. No (Issued on May 21, 2013) and others FDA Regulatory Strategies: Label will include companion diagnostic (CDx) - DRP biomarker. Orphan drug exclusivity (potential) = 7 years + listing of DRP patents on Orange Book. 8

9 Key Advantages of cdx Technology Improves outcomes Reduces Risk and Cost Highly Validated Proven in nearly 40 clinical retrospective validation studies for many different approved Cancer drugs. 80%+ success rate in generating robust DRP biomarkers. Overcomes Limitations of Other Current Knowledge-Driven Approaches NGS-based somatic mutation analysis. Single biomarker (e.g. receptor TKI expression) reliance. Enables Selection of Highest Likely Responder Patients for Enrollment in Clinical Trials 9

10 Management Team & Board of Directors Executive Management Board of Directors Marie Foegh CEO/CMO Jarne Elleholm Chief Financial Officer Clinical Development Support Team Ulla H. Buhl James G. Cullem Peter Buhl Jensen, M.D., Ph.D. Chairman CEO, Oncology Venture CEO, Medical Prognosis Institute Ulla Hald Buhl COO, Oncology Venture COO, Medical Prognosis Institute Clinical Liaison Steen Knudsen Ph.D. Biomarker Founder J.D. BD Liaison Bruce Pratt, Ph.D. Drug Manufacturing Thomas Jensen CTO, Oncology Venture CTO, Medical Prognosis Institute 10

11 Advisory Board Joyce A. O Shaughnessy, M.D. Baylor University US Oncology Mansoor Raza Mirza, M.D. Medical Director NSGO-CTU Vice-Chairman DGCG, Rigshospitalet University of Copenhagen Henry S. Friedman, M.D. Duke University Tisch Brain Tumor Center Daniel D. Von Hoff, M.D. University of Arizona Mayo Clinic US Oncology Mary Lake Polan, M.D. Yale University, Dept. Of OB/Gyn, 11

12 Development Timeline Jump Start Clinical Trials Q Q Q Q Q Q Q Manufacturing Manufacturing Approval of Screening protocols 2X-111 Metastatic Breast Cancer trial 15 patients Early Value Inflection Point 2X-111 Glioblastoma trial 15 patients 2X-121 Metastatic Breast Cancer trial 15 patients 2X-131 Ovarian Cancer trial 15 patients Selection 2X-1?1 Pivotal trial ~ patients IPO/ Trade Sale Trial Protocol Approval First patient response Interim data ~ 8 patients 12

13 EXIT Opportunities Acquisition of Entire Business US $10.2B (2016) AbbVie acquisition of Stemcentryx $550M (2016) Roche acquisition of Tensha Tx $780M (2016) Somitomo Dainippon acquisition of Tolero Pharma $14B (2016) Pfizer acquisition of Medivation - primarily for Talazoparib, a PARP inhibitor in Phase 3 development for treatment of BRCA mutated Breast Cancer Asset Sale $570 (2015) Medivation purchase of Talazoparib from BioMarin $182.5M (2016) Tracon pharma and J&J for two early stage oncology assets IPO $50M (2016) Kura Oncology Tipifaranib $186M (2016) BeiGene BTK Inhibitor $53M (2016) Syndax entinostat $53.5M (2016) Merus bi-specific antibody therapeutics 13

14 Why Invest In 2X Oncology? Focused Women s Health Company Three clinically active, novel therapeutics that have shown efficacy in women De-risked Approach Use of DRP biomarker to identify and enrol high likelihood responders Thanks to DRP selection, trials are small relative to other cancer compounds Database of existing Cancer patients improves development program and shortens time Experienced Team Strong management team and support from Oncology Venture required experience Capital Efficient Model & Short Term Exit Small trials equals low cost of development Short time to value inflection points Superior Return on Investment 14

15 Precision Therapies for Women s Cancers Marie Foegh, MD, DrSc President & CEO/CMO marie.foegh@2xoncology.com