Cardiff Oncology, Inc. · NASDAQ:CRDF
Cardiff Oncology — Trovagene — Investor Presentation (October 2013)
31 slides from Cardiff Oncology's investor presentation, Oct 22, 2013
Slide 1: A better clinical pathway for cancer monitoring
A better clinical pathway for cancer monitoring Antonius Schuh, Ph.D. | Chief Executive Officer October 2013
Slide 2: Forward-Looking Statements
Forward-Looking Statements Statements in this presentation about the Company's expectations, applications of its technology, markets, launch of tests and other statements that are not historical facts are "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 an
Slide 3: Our goal: Towards better treatment for cancer
Our goal: Towards better treatment for cancer INTRODUCTION A better clinical pathway for cancer monitoring Novel clinical utility: non-invasive, near real-time detection of oncogene mutations for any tumor type
Slide 4: Introduction: Welcome to Trovagene, Inc.
Introduction Welcome to Trovagene, Inc. Addresses a high unmet need in cancer by enabling easier, more frequent monitoring of emergence of oncogene mutation status, disease progression and recurrence Unique IP around cell-free DNA in urine creates a powerful core diagnostic platform for the non-inva
Slide 5: Experienced Senior Management Team
Experienced Senior Management Team TROVAGENE CORE TEAM Thomas Adams, PhD (Chair) John P. Brancaccio, CPA Gary S. Jacob, PhD Antonius Schuh PhD CEO CEO Sorrento Therapeutics, AviaraDx, Arcturus Biosciences, Sequenom PhD Pharm. Chem Stephen Zaniboni CFO Mark Erlander PhD CSO Keith McCormick VP, Sales
Slide 6: Understanding cell-free DNA for cancer monitoring
Understanding “cell-free” DNA for cancer monitoring THE CLINICAL UTILITY OF CELL-FREE DNA FROM URINE Cell-free DNA is released by dying cells in the human body Cells in the body die continuously in a process known as “apoptosis” , releasing DNA/RNA into the bloodstream This “cell-free” DNA, detectab
Slide 7: Why not simply use a blood specimen?
Why not simply use a blood specimen? THE CLINICAL UTILITY OF CELL-FREE DNA FROM URINE Blood Draw Sample quantity is limited Sample frequency is limited Inconvenient for patient – requires an appointment Painful and perceived as threatening Requires a medical professional Specimen is a biohazard Requ
Slide 8: Kidneys filter cell-free DNA from blood into the urine
Kidneys filter cell-free DNA from blood into the urine THE CLINICAL UTILITY OF CELL-FREE DNA FROM URINE By isolating cell-free DNA from urine, Trovagene eliminates the need for the cost, complexity and inconvenience of a blood sample Truly non-invasive Patient can self-sample at home or clinic No me
Slide 9: Enabled by a confluence of emerging technologies
Enabled by a confluence of emerging technologies THE CLINICAL UTILITY OF CELL-FREE DNA FROM URINE Ability to detect single DNA molecules in a urine sample Trovagene IP Next Gen sequencing Digital PCR RainDance Droplet Digital PCR up to 5,000,000 target sequences Oncogene mutations are rare events Tu
Slide 10: Why Cell-free DNA?
Why Cell-free DNA? Cell-free DNA gives a global view of tumor heterogeneity for metastatic cancer patients THE CLINICAL UTILITY OF CELL-FREE DNA FROM URINE Detection of specific tumor mutations Monitoring of tumor load/response to therapy Detection of emerging tumor resistant mutations/new directed
Slide 11: Detecting and monitoring cancer mutations key to effective therapy
Healthy Subject Oncogenic Mutation DRUG A CYCLE 1 CYCLE 2 CYCLE 3 CYCLE 4 SURVIVAL TUMOR MUTATION#1 Selective pressure of targeted drugs enrich tumor tissue with new mutant cells that are drug resistant Treatment SURVIVAL DRUG B DRUG C DRUG D SURVIVAL CELL DEATH CELL DEATH CELL DEATH MUTATION#2 MUTA
Slide 12: Opportunity to implement monitoring of tumor dynamics, demonstrated in plasma
Opportunity to implement monitoring of tumor dynamics, demonstrated in plasma CLINICAL DEVELOPMENT PROGRAM Disease progression 1Dawson et al., NEJM 2013 Online, DOI: 10.1056 2Su et al., Ann N Y Acad Sci 2008, 1137:197-206 Cell-free DNA was the only marker that predicted response CELL-FREE DNA / PIK3
Slide 13: Improved Patient Outcomes: Building value through clinical evidence
Improved Patient Outcomes Building value through clinical evidence CLINICAL DEVELOPMENT PROGRAM PHASE 1 Demonstrate concordance of oncogene mutation status between urine and tumor tissue. Clinical Utility: Determine mutational status of actionable biomarkers in urine when a biopsy is not an option.
Slide 14: Phase 1: Demonstrate concordance of urine-based DNA testing with tumor biopsy
Phase 1: Demonstrate concordance of urine-based DNA mutational testing with tumor tissue biopsy Principal Investigator: Filip Janku MD PhD; Investigational Cancer Therapeutics (Phase I Clinical Trials Program) MD Anderson Cancer Center Objective 1: Measure the degree of concordance between results o
Slide 15: Two-Step Assay Design for 28-30 bp footprint
A STEP ONE Pre-amplification with wild-type suppression DNA template with mutation (“M”; Patient DNA) B STEP TWO Amplification with A,B primers, digital droplet PCR (Raindance) DNA template with no mutation (Wild Type (“WT”; Patient DNA) WT Blocker of A,B primers M WT A B M M TAQMAN Probe Amplificat
Slide 16: BRAF V600E mutation detection by droplet digital PCR
BRAF V600E mutation detection by droplet digital PCR CLINICAL DEVELOPMENT PROGRAM BRAF mutation Empty droplets No mutation (wild type) Healthy (negative) Control Positive Control Empty droplets No mutation (wild type)
Slide 17: Concordance of BRAF V600E mutations
Concordance of BRAF V600E mutations1 CLINICAL DEVELOPMENT PROGRAM Thresholds for mutation detection were determined by assessing data from 50 healthy controls and 39 patient samples using a classification tree. Minimizing the percentage of false negatives was given a higher importance than minimizin
Slide 18: Longitudinal Urinary cf-DNA BRAF V600E Mutation Correlates with Therapy Response
Longitudinal Urinary cf-DNA BRAF V600E Mutation Correlates with Therapy Response CLINICAL DEVELOPMENT PROGRAM Patient 5: Melanoma Progressive Disease Vemurafenib only
Slide 19: Longitudinal Urinary cf-DNA BRAF V600E Mutation Correlates with Therapy Response
Longitudinal Urinary cf-DNA BRAF V600E Mutation Correlates with Therapy Response CLINICAL DEVELOPMENT PROGRAM Progressing on Chemotherapy Patient 16 Colorectal Cancer Patient 44 Appendiceal Cancer Progressing on chemotherapy
Slide 20: Summary
Summary A cell free DNA (cf-DNA) BRAF V600E mutation test from urine has high concordance with patient-matched known BRAF mutational status from tumor tissue biopsy BRAF mutations were detected in a range of tumor types including melanoma, colorectal, appendiceal, lung, glioblastoma, papillary thyro
Slide 21: Next Gen Sequencing (NGS) of Urinary cf-DNA: Content Rich
Content Rich Ability to detect multiple mutations in a single run and any nucleotide at each position 9 KRAS mutations in a single assay: G12A, G12C, G12D, G12F, G12R, G12S, G12V, G13C, G13D Target Multiplexing Multiple Genes and multiple sites per gene can be interrogated The potential to monitor d
Slide 22: NGS provides generic assay design for all mutations
NGS provides generic assay design for all mutations CLINICAL DEVELOPMENT PROGRAM Enables unsupervised query for any mutation, eliminating the need for the creation of individual assays for specific mutations. Anatomy of a KRAS Next Gen Sequencing Assay Specifications: High sensitivity (0.01-0.05% mu
Slide 23: cf-DNA NGS KRAS Assay
cf-DNA NGS KRAS Assay Knowing KRAS mutational status in metastatic colorectal cancer patients is currently standard of care for anti-EGFR therapy To assess the performance of our assay we utilized mutant containing cell line DNA with known mutational status to optimize the performance with each muta
Slide 24: NGS cf-DNA KRAS has high specificity
NGS cf-DNA KRAS has high specificity CLINICAL DEVELOPMENT PROGRAM NGS assay of cell lines with 7 different known KRAS mutations indicates high specificity
Slide 25: Fold Enrichment of KRAS G12D Mutation
Fold Enrichment of KRAS G12D Mutation Actual Mutation Frequency Observed Frequency 50.00% 91.14% 12.50% 86.74% 3.13% 72.26% 0.78% 41.32% 0.20% 16.59% 0.05% 5.72% 0.01% 2.24% 0% 0.30% NGS assay has required detection sensitivity CLINICAL DEVELOPMENT PROGRAM
Slide 26: NGS assay detects KRAS mutations in Urinary DNA from Metastatic Cancer Patients
NGS assay detects KRAS mutations in Urinary DNA from Metastatic Cancer Patients CLINICAL DEVELOPMENT PROGRAM Initial Study indicates high concordance 8 out of 9 KRAS mutations detected by NGS assay NGS assay enables quantitation of amount of mutation (versus normal) present in urine
Slide 27: Core IP extends into assay detection and development
Core IP extends into assay detection and development CORE INTELLECTUAL PROPERTY
Slide 28: Reimbursement Strategy
Reimbursement Strategy Our tests must be positioned as different from other mutation assays: New Technology Code “81479” being considered Allows for billing at list price Single Mutation - $400-500 Multiple Mutation Profile - $1500-2000 Appeal cases of low-pay or no-pay Medicare providership will be
Slide 29: 2012 achievements
2012 achievements Acquired CLIA laboratory CLIA-certified, cap-accredited high complexity molecular diagnostic lab Strengthened patent portfolio Issued U.S. Patent for NPM mutants to diagnose and monitor acute myeloid leukemia Issued European patent for detection of pathogenic infections Improved li
Slide 30: 2013 achievements
2013 achievements Demonstrated proof of concept for tumor mutation detection from urine samples of metastatic cancer patients Validated the presence of BRAF, KRAS in multiple cancer types Initiated R&D partnership with PerkinElmer Collaborate to develop test to determine liver cancer risk Initiated
Slide 31: For further information
For further information Please contact: Antonius Schuh, CEO aschuh@trovagene.com Stephen Zaniboni, CFO szaniboni@trovagene.com