CORPORATE PRESENTATION

Transcription

1 CORPORATE PRESENTATION May 2015 NASDAQ: APTO TSX: APS

2 FORWARD-LOOKING STATEMENTS Except for historical information, this presentation contains forward-looking statements, which reflect APTOSE Biosciences, Inc. s (the Company ) current expectations regarding future events. These forward-looking statements involve risks and uncertainties, which may cause actual results to differ materially from those statements. Those risks and uncertainties include, but are not limited to, our ability to raise the funds necessary to continue our operations, changing market conditions, the successful and timely completion of clinical studies, the establishment and maintenance of corporate alliances, the market potential of our product candidates, the impact of competitive products and pricing, new product development, uncertainties related to the regulatory approval process and other risks detailed from time to time in the Company s ongoing quarterly filings and annual reports. Forward-looking statements contained in this document represent views only as of the date hereof and are presented for the purpose of assisting potential investors in understanding the Company s business, and may not be appropriate for other purposes. The Company does not undertake to update any forward-looking statements, whether written or oral, that may be made from time to time by or on its behalf, except as required under applicable securities legislation. Investors should read the Company s continuous disclosure documents available at and EDGAR at especially the risk factors detailed therein. 2

3 APTOSE : BIOTECH COMPANY Mission to Create Personalized Agents and Companion Diagnostics for Patients with Life-Threatening Cancers APTO-253 Lead Agent at Phase Ib/II Stage of Dev t For the treatment of AML, MDS and other hematologic malignancies APTOSE was Formerly Lorus Therapeutics, Inc. November 2013 Instituted Complete Transformation of Company New Management and Clinical Development Teams Introduced Changed Name in September 2014 to APTOSE Biosciences Personnel in San Diego, San Francisco, Toronto Built Strong Financial Foundation (>$45 Million) Listed on NASDAQ Capital Market Oct. 23,

4 APTOSE : BIOTECH COMPANY 4

5 APTOSE : BIOTECH COMPANY 5

6 NEW VISION NEW COMPANY Small Publicly-Traded Oncology Company in Toronto : Lorus APTO-253 induces KLF4, tumor suppressor that regulates cell fate APTO-253 phase I solid tumor trial, safety & antitumor activity Recent Scientific Findings Set in Motion a New Vision Silencing KLF4 is material driver in >90% AML Patients APTO-253 only clinical stage agent developed as KLF4 Inducer APTO-253 Potential as a Targeted Therapy for AML Potential for diagnostics to select and monitor most sensitive patients Build a New Company with a New Strategic Plan U.S. Based Management Team New Targeted Therapeutic Approach APTO-253 Elite Clinical Development Team Financings with Blue Chip Investors 6

7 NEW U.S. BASED MANAGEMENT TEAM Dr. William G. Rice, PhD (Chairman and CEO) Achillion Pharmaceuticals: Founder, CEO, President, CSO, Director National Cancer Institute-FCRDC: Sr. Scientist, Drug Mechanism Lab Emory School of Medicine: Faculty, Pediatric Hematology/Oncology Cylene Pharmaceuticals: Chairman, CEO, President, CSO Avanish Vellanki (Chief Business Officer) Citigroup Global Markets: Biopharma Investment Banking Wedbush Securities: Biopharma Investment Banking Bear, Stearns & Co: Equity Research Publishing Analyst Proteolix, Inc.: Sr. Director of Corporate Development Gregory Chow (Chief Financial Officer) RBC Capital Markets: Director, Head of Life Sciences Private Placements Wedbush Securities: Managing Director & Director of Private Placements Wells Fargo: Led Private Capital Group BDO Seidman, LLP: Senior Auditor, CPA (inactive), State of California 7

8 NEW CLINICAL DEVELOPMENT TEAM WITH ONCOLOGY PEDIGREE Dr. Stephen Howell, MD Serves as Chief Medical Officer Distinguished Professor of Medicine, UCSD Moores Cancer Center Physician scientist conducting research to address drug resistance Expertise in pharmacology and design and conduct of clinical trials Dr. Daniel Von Hoff, MD, FACP Serves as SVP of Medical Affairs Key Advisor Winner of 2010 Karnofsky Memorial Award Prior President of AACR and Board Member of ASCO Appointed to President s National Cancer Advisory Board Dr. Brian Druker, MD Collaborator & Chair of Scientific Advisory Board Key Role in Development of Gleevec and Member, National Academy of Sciences Winner of Karnofsky Award and Lasker America s Nobel Award Leader of Inter-institutional Beat AML Initiative Dr. Michael Andreeff, MD, PhD Collaborator & Member of Scientific Advisory Board Professor of Medicine, Department of Leukemia and Department of Stem Cell Transplantation, Paul and Mary Haas Chair in Genetics, MD Anderson Cancer Center Physician Scientist, expert in AML and drug resistance, expert in drug mechanisms, published over 450 peer-reviewed papers, 5 books and 75 book chapters 8

9 FINANCINGS BY BLUE CHIP INVESTORS PROVIDE CASH, RUNWAY, FINANCIAL STABILITY 9

10 PIPELINE AND NEW THERAPEUTIC FOCUS FOR APTO-253 Drug Indication Partners Discovery Pre-Clinical Phase I Phase II APTO-253 (KLF4 Inducer) Solid Tumors -- Hematologic Malignancies (AML & MDS) -- IL-17E (IL-17E Receptor Agonist) Oncology Genentech (1) APTO-500 (MELK Inhibitor) Oncology -- Small Molecule Program Various Eli Lilly / Elanco (2) Early Discovery Program Various -- (1) APTOSE owns global rights in oncology (2) Exclusive rights to license for veterinary applications Completed Ongoing 10

11 ACUTE MYELOID LEUKEMIA: MOST COMMON FORM OF ADULT ACUTE LEUKEMIA AGGRESSIVE BLOOD CANCER Transforming Role of KLF4 in AML

12 AML INCIDENCE INCREASES WITH AGE AND PROGNOSIS WORSENS Incidence (per 100,000) US Population Statistics Prevalence 35,726 Incidence 18,860 Annual Deaths 10,460 Treatment Statistics 5 Year Survival (Overall) ~20% 5 Year Survival (over age 65) ~5% Elderly Pts w/ Residual Disease / Induction Fail. 50% AML Incidence by Age AML most prevalent in elderly patients >65 years of age Therapy Strategies Clinical Trial 51% Chemotherapy 28% Supportive Care 21% Source: Datamonitor Age (in years) Source: American Cancer Society, Leukemia & Lymphoma Society Source: SEER (Surveillance, Epidemiology and End Results) Cancer Statistics Review, National Cancer Institute; Source: Cancer Research UK 2013, Datamonitor 12

13 AML MEDICAL NEED, MARKET AND CHALLENGES TO CREATE NEW DRUGS Dismal Survival Among Elderly Population Function of Standard of Care ( 7+3 ) Elderly exhibit poor response and significant toxicity No broadly active treatment approval since 1970 s Tremendous Need for Less Toxic, Targeted Agents Need for multiple agents for combination therapy Challenge of Creating Targeted Drugs to Treat AML Extreme Heterogeneity of Disease Conventional Wisdom There is No Unifying Mechanism that Drives AML 13

14 UNDERLYING CAUSE OF AML LINKED TO ALTERATIONS IN CDX2 AND KLF4 GENES 14

15 INAPPROPRIATE EXPRESSION OF CDX2 GENE IS ASSOCIATED WITH AML CDX2 Inappropriately Expressed in AML (1) Embryonic Gene that Constitutes Bone Marrow & Other Organs Should Not be Expressed in Adult Hematopoietic Cells Fröhling & Scholl (JCI) (1) Real-Time Quantitative PCR Analysis of CDX2 mrna Levels CDX2 expressed in: 0% Normal Hematopoietic Cells 90% AML (All Subtypes) 40% MDS (Pre-AML) 20% CML (advanced blasts) NORMAL Figure 1 CDX2 expression in AML. CDX2 mrna levels were measured by RQ-PCR in 170 AML patients from different cytogenetic subgroups, as well as in BMMCs (n = 10), CD34 + cells (n = 3), HSCs (n = 3), CMPs (n = 3), GMPs (n = 3), and MEPs (n = 3) from normal individuals. Circles indicate patients with genomic amplification of the CDX2 locus, as assessed by acgh and FISH (25). Bars indicate median values (1) J. Clin. Invest. 2007; 117 (4) ;

16 APTO-253: ONLY DRUG TO TARGET KLF4 MECHANISM IN AML NORMAL KLF4 ON CDX2 OFF 100% CDX2 Protein Genetic and Epigenetic Alterations Turn On the CDX2 Gene in 90% AML AML (1) KLF4 Klf4 ON CDX2 ON (1) Genetically Induce KLF4 APTO-253 o KLF4 o p21 o G1 Arrest o Caspase 3 APOPTOSIS AML Death Personalized Medicine Companion Diagnostics CDX2-KLF4 Signature Select Sensitive Patients Seek Early Clinical POC 16 (1) Source: J. Clin. Invest. 2013; 123(1);

17 APTO-253: ACTS THROUGH INTENDED MECHANISM IN AML PI + APTO-253 KLF4 p21 Cell Cycle Arrest (Pause) CASPASE 3 Apoptosis (Delete) KLF p21 G1 Arrest - DMSO DMSO Fold Increase p21 DMSO um APTO Annexin V 0.5 um APTO DMSO 0.5 um APTO DMSO 0.5 um APTO-253 Fold Increase KLF4 DMSO um APTO Fold Increase p21 DMSO um APTO

18 APTO-253: AML CELL LINES HIGHLY SENSITIVE IN VITRO AML Cells Highly Sensitive (red bars) nm IC 50 in anti-proliferative assays AML cells more sensitive than solid tumor cell lines (gray bars) Sensitivity to APTO-253 Correlates with the Level of KLF4 Induction Lower IC 50 with higher KLF4 induction Source: APTOSE Biosciences, Inc AACR Poster 18

19 THP-1 AML Tumor size (mm3) Mouse Weight (g) APTO-253 PRECLINICAL IN VIVO EFFICACY AND TOLERABILITY IN MURINE XENOGRAFTS Schedule Optimization as a Single Agent Xenografts H229 in Nude Mice APTO-253 HCl at 15 mg/kg Dosed Days 1,2 Every Week Dosed Days 1,2 Every 2 Weeks Weekly Dosing is Superior Drug Combination Mean Body Weight Profile Xenografts THP-1 in Nude Mice APTO-253 HCL 15 mg/kg, 2x/wk Vehicle Control APTO-253, 15mg/kg bid, 2x/week Azacitidine, 10 mg/kg, q4d APTO-253, 15 mg/kg bid, 2x/week + Azacitidine, q4d Azacitidine 10 mg/kg, Q4d 200 Enhanced combo activity; no overt additional toxicity Observation Period (Day) 19

20 APTO-253: PRECLINICAL FINDINGS SUPPORT DEVELOPMENT IN AML & MDS APTO-253 Small Molecule, HCL Salt CoM & Use Patent Coverage Through 2028, Plus Typical Patent Extension Developing Companion CDX2 & KLF4 Diagnostics: IP Filed Planned for Patient Selection and Therapeutic Signals Developing as Targeted Agent for AML and HR-MDS Expect Single Agent Efficacy in the Clinic Could be Foundation of Combination Therapy for AML/MDS Synergistic with Marketed AML Drugs (cytarabine, daunorubicin) Synergistic with Marketed MDS Drugs (azacitidine, decitabine) Safe in Humans: Prior Phase 1 in Solid Tumor Patients APTO-253 Does NOT Suppress Bone Marrow (1) (1) Cercek et al, ECC ESMO

21 APTO-253 Clinical Development in AML & MDS

22 ONGOING PHASE 1(B) TRIAL Patients with Relapsed or Refractory Hematologic Malignancies AML (other acute leukemias) MDS (>10% bone marrow blasts) Lymphomas (Hodgkins & Non-Hodgkins) Multiple Myeloma Primary Objectives Identify MTD and DLT on a Twice Weekly Schedule (28 day cycle) Establish RP2D for Patients with Specific Hematologic Malignancies Secondary Objectives KLF4 Reported Operative in Subpopulations of All Indications Understand PK & Safety Profile on a Twice Weekly Schedule Demonstrate Ability to Move Biomarkers, Including CDX2, KLF4 and p21 Seek Evidence of Antitumor Activity Specific to Each Type of Malignancy Radiographic, Hematologic, Bone Marrow Improvement, Physical Exam, PET Scan Premier Hematologic Malignancy Clinical Sites for Multicenter Study 22

23 CLINICAL DEVELOPMENT PLAN Phase 1b Patient Selection: CDX KLF4 Phase 1b Dose Escalation: Arm A AML and High Risk MDS 1 o Endpt: MTD, DLT & RP2D - Twice Weekly Schedule 2 o Endpts: PK, Biomarkers, Efficacy, Transfusions Phase 1b Dose Escalation: Arm B Lymphomas and Multiple Myeloma 1 o Endpt: MTD, DLT & RP2D - Twice Weekly Schedule 2 o Endpts: PK, Biomarkers, Efficacy Phase 1b Single Agent Expansion AML ORR, Efficacy, Biomarkers, Safety Phase 1b Single Agent Expansion MDS ORR, Efficacy, Transfusions, Biomarkers, Safety Phase 2 Phase 2 AML Drug Combination Trial Approved Drug + APTO o : Biomarkers (p21, CDX2, KLF4) 2 o : Efficacy Phase 2 MDS Drug Combination Trial Approved Drug + APTO o : Biomarkers (p21, CDX2, KLF4) 2 o : Efficacy/Transfusions Note: Phase 1b expansion cohorts and Phase 2 trials contingent on Phase 1b outcomes 23

24 CORPORATE TIMELINES Drug / Category APTO-253 Event Preclinical: Single Agent & Combo Efficacy in AML Xenografts Clinical: Commence Phase 1b Dose Escalation Trial Timeline Achieved ASH Dec 2014 Achieved Preclinical: Response of Primary Heme Cancer Patient Isolates Late-2015 (ASH) Clinical: Potential Phase 1b Dose Escalation Interim Data (ASH) Late-2015 (ASH) Clinical: Complete Dosing in Phase 1b Dose Escalation Late-2015 / 1H 2016 Clinical: Phase 1b Dose Escalation Study Results 2H 2016 Clinical: Commence Phase 1b Single Agent Expansion Studies 2016 Clinical: Commence Phase 2 Drug Combination Studies

25 SUMMARY FINANCIAL DATA $ in CAD At December 31, 2014 Exchanges: Cash, Cash Equivalents and Investments: Debt - Promissory and Convertible Notes: NASDAQ: APTO TSX: APS $30.5M $0.44M Basic Shares Outstanding: 11.7M Fully Diluted Shares Outstanding 1 : 13.4M Institutional Ownership: ~55% Notes: 1) Comprised of A) 11,699,873 shares outstanding B) Warrants to purchase 208,969 shares, C) options to purchase 1,374,013 shares at a weighted average price of $5.95 per share and D) convertible notes that convert into 121,528 shares 25

26 EXECUTIVE SUMMARY: BUILDING FOR SUCCESS Built Experienced Executive Team Built Clinical Team with Oncology Pedigree Focused APTO-253 as Targeted Agent for AML Targeted Approach: AML, MDS and Hematologic Malignancies Personalized Drug: Opportunity for Companion Diagnostic Built Strong Financial and Operational Foundation Raised >$45 Million from Premier Fundamental Healthcare Investors Listed on NASDAQ (APTO) Looking Forward Seek Clinical Efficacy with APTO-253 in Patients with AML/MDS Seek to Build a Staged Pipeline of Targeted Cancer Drugs 26

27 Thank You!