Kura Oncology, Inc. · NASDAQ:KURA
Kura Oncology — Investor Presentation (September 2017)
39 slides from Kura Oncology's investor presentation, Sep 7, 2017
Slide 1: Corporate Presentation September 2017
Corporate Presentation September 2017 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements. Such statements include, but are not limited to, statements regarding our research, pre-clinical and clinical development activities, plans and projected timelines for tipifarnib, KO-947 and KO-539, plans regarding reg
Slide 3: Kura Oncology Investment Highlights
Kura Oncology Investment Highlights Developing precision medicines for cancer in diseases with significant commercial potential Lead program tipifarnib in multiple Phase 2 trials Positive Phase 2 trial in head and neck squamous cell carcinomas (HNSCC), reported in September 2017 Additional data read
Slide 4: Leadership Team
Leadership Team Chief Executive Officer Troy Wilson, Ph.D., J.D. Chief Scientific Officer Yi Liu, Ph.D. SVP, Chemistry and Pharmaceutical Sciences Pingda Ren, Ph.D. Chief Medical Officer Antonio Gualberto, M.D., Ph.D. Chief Financial Officer Heidi Henson, CPA SVP and General Counsel Annette North, L
Slide 5: Biomarkers Have Potential to Unlock the Value of Precision Medicines
Biomarkers Have Potential to Unlock the Value of Precision Medicines + Biomarkers Protein expression Histology Genetics Enhanced Clinical Benefit for Patients Precision Medicines (BRAF V600E) (HER2) (BCR-ABL) (EGFR T790M) (BCR-ABL) (PD-1)
Slide 6: Product Candidate Pipeline
Product Candidate Pipeline PROGRAM PRECLINICAL PHASE 1 PHASE 2 Tipifarnib (Farnesyl Transferase Inhibitor) KO-947 (ERK Inhibitor) KO-539 (Menin-MLL Inhibitor) HRAS Mutant Solid Tumors Peripheral T-cell Lymphomas Myelodysplastic Syndromes Chronic Myelomonocytic Leukemia Solid Tumors Liquid Tumors
Slide 7: Pipeline Targeting Large Market Opportunity
Pipeline Targeting Large Market Opportunity Product Candidate Tumor Type Target Population Tipifarnib (Phase 2) HRASmut HNSCC 2,800-3,400 HRASmut Sq-NSCLC 1,000-1,700 PTCL 1,500-2,500* MDS 15,000-18,000** CMML 750* KO-947 (Phase 1) HNSCC 10,000-20,000* KRASmut NSCLC 23,000 BRAFmut NSCLC 5,000 KO-539
Slide 8: Multiple Near-Term Milestones
Multiple Near-Term Milestones 2015 2016 2017* 2018* Initiated P2 HRAS trial for tipifarnib Listed on NASDAQ Initiated P2 PTCL trial Initiated P2 lower-risk MDS trial Reported positive preliminary data from P2 HRAS trial IND accepted for KO-947 KO-539 selected as development candidate Initiated P2 CM
Slide 9: Tipifarnib in HRAS Mutant Solid Tumors
Tipifarnib in HRAS Mutant Solid Tumors
Slide 10: Tipifarnib: Unlocking Value in a Lower Risk Asset
Tipifarnib: Unlocking Value in a Lower Risk Asset SUBSTANTIAL CLINICAL EXPERIENCE Extremely potent and selective inhibitor of protein farnesylation Well characterized > 5,000 patients with activity in subpopulations Developed before advent of personalized medicine approaches Licensed from Janssen KU
Slide 11: Tipifarnib Inhibits Cancer Cell Growth Signaling Pathways
Tipifarnib Inhibits Cancer Cell Growth Signaling Pathways Normal FTase Tipifarnib Inhibits FTase = Blocking farnesylation prevents HRAS membrane localization Tipifarnib can block farnesylation and membrane localization of both wild-type and mutant HRAS proteins Farnesyl transferase (FTase) attaches
Slide 12: Tipifarnib Inhibits HRAS Mutant Tumors
Tipifarnib Inhibits HRAS Mutant Tumors Normal HRAS Mutant With Tipifarnib Growth and survival of normal cells is driven by growth factor interaction with cell receptors and intracellular signaling Mutation of HRAS protein can switch signaling into a permanently “on” state, making tumor growth and pr
Slide 13: Tipifarnib is Active in Preclinical PDX Models of HRAS Mutant HNSCC
Tipifarnib is Active in Preclinical PDX Models of HRAS Mutant HNSCC (Preliminary data)
Slide 14: HNSCC Represents Significant Unmet Medical Need
HNSCC Represents Significant Unmet Medical Need HNSCC comprises different malignant tumors that develop in or around the throat, larynx, nose, sinuses and mouth Low response rates and limited duration of clinical benefit with existing therapeutic options for HNSCC Estimated incidence of HNSCC in the
Slide 15: Tipifarnib: Phase 2 in HRAS Mutant Solid Tumors
Tipifarnib: Phase 2 in HRAS Mutant Solid Tumors Design of Phase 2 Trial Originally designed to enroll patients into two single-arm study cohorts, each with a 2-stage design (11+7 evaluable patients) Cohort 1: HRAS mutant thyroid cancers Cohort 2: Other (non-thyroid) HRAS mutant solid tumors Primary
Slide 16: Phase 2 HRAS Mutant HNSCC – Key Takeaways
Phase 2 HRAS Mutant HNSCC – Key Takeaways Positive Phase 2 results obtained in September 2017 Partial responses observed in four of the first six evaluable patients and disease stabilization observed in two remaining patients with HRAS mutant HNSCC Primary endpoint has been met; patient enrollment w
Slide 17: Tipifarnib: Foundation Medicine Collaboration
Tipifarnib: Foundation Medicine Collaboration Leader in tissue-based next-generation sequencing (NGS) using a comprehensive genomic profiling assay Focused on outreach to patients with HRAS mutant HNSCC, particularly those in the community setting SmartTrials Precision Enrollment program will contac
Slide 18: Tipifarnib: Patent Protects Use in HRAS Mutant HNSCC Until August 2036
Tipifarnib: Patent Protects Use in HRAS Mutant HNSCC Until August 2036 U.S. patent 9,707,221 provides exclusivity in U.S. for tipifarnib in HRAS mutant HNSCC indication Kura is pursuing U.S. and foreign patent protection in this and other indications This patent illustrates potential of broader stra
Slide 19: Potential to Initiate 1st Pivotal Trial for Tipifarnib in 2018
Potential to Initiate 1st Pivotal Trial for Tipifarnib in 2018 FOUR ONGOING KURA PHASE 2 TRIALS CRITERIA FOR ADVANCEMENT TO PIVOTAL DEVELOPMENT Biomarker validation Evidence of durable, clinical benefit Sufficient ORR Potential for rapid clinical development Opportunity to move into earlier lines of
Slide 20: Tipifarnib in Peripheral T-Cell Lymphoma (PTCL)
Tipifarnib in Peripheral T-Cell Lymphoma (PTCL)
Slide 21: Tipifarnib: Phase 2 Trial in PTCL
Tipifarnib: Phase 2 Trial in PTCL RATIONALE: Diverse group of aggressive non-Hodgkin lymphomas characterized by presence of malignant T-cells or natural killer (NK) cells Effective options for relapsed patients are limited with objective responses Approved therapies show 25-30% response rate and a m
Slide 22: Preliminary Phase 2 Data Identify CXCL12 as a Potential Biomarker in PTCL
Preliminary Phase 2 Data Identify CXCL12 as a Potential Biomarker in PTCL 7/18 patients (39%) experienced PR/SD with a median of 4 prior therapies1 Patients with SD had meaningful tumors size reductions2 Gene expression data available for 13 patients Patients with tumors with high expression of CXCL
Slide 23: Preliminary Phase 2 Data Identify CXCL12 as a Potential Biomarker in PTCL
Preliminary Phase 2 Data Identify CXCL12 as a Potential Biomarker in PTCL* * Preliminary data as of July 15, 2017
Slide 24: Rationale for CXCL12 Biomarker
Rationale for CXCL12 Biomarker Tumor T Cell Mesenchymal Stroma Cell VEGF, CXCL13, ANGPT1 CXCL12 T-cell tumors secrete growth factors for stromal cells, which in turn produce growth factors for T-cells, including CXCL12 (SDF-1)1-5 Lab Invest. 2004; 84:1512-9 Cancer Cell. 2015 ;27:755-68 Stem Cells In
Slide 25: Tipifarnib in MDS and CMML
Tipifarnib in MDS and CMML
Slide 26: Tipifarnib: Phase 2 Trial in MDS
Tipifarnib: Phase 2 Trial in MDS RATIONALE AND UNMET NEED: MDS are a group of blood and bone marrow disorders with both proliferative and dysplastic phenotypes characterized by ineffective hematopoiesis leading to cytopenias Estimated annual U.S. incidence of 13,000; estimated U.S. prevalence of 50,
Slide 27: Tipifarnib: Role of CXCL12 and Isolated Neutropenia in MDS
Tipifarnib: Role of CXCL12 and Isolated Neutropenia in MDS HYPOTHESIS: Neutropenia at study entry may enrich for response to tipifarnib in patients with MDS RATIONALE FOR TESTING: CXCL12 implicated in the homing of myeloid cells in the bone marrow. Activating mutations in the CXCL12 pathway known to
Slide 28: Tipifarnib: Phase 2 Trial in MDS
Tipifarnib: Phase 2 Trial in MDS DESIGN OF PHASE 2 CLINICAL TRIAL: Two cohorts of 18 patients, each with a Simon 2-stage design The trial will enroll patients who have failed up to two prior therapies; inclusion to be opened to all MDS patients independent of AML risk Trial inclusion to be limited t
Slide 29: Tipifarnib: Phase 2 Trial in CMML
Tipifarnib: Phase 2 Trial in CMML RATIONALE: CMML is a disorder of bone marrow stem cells with characteristics of both myoproliferative and myelodysplastic diseases Prognosis of CMML is very poor; 5 year survival is ~18-20% Limited therapeutic options Previous Phase 2 study from Janssen supports con
Slide 30: KO-947 (ERK Inhibitor)
KO-947 (ERK Inhibitor)
Slide 31: KO-947: Potent ERK1/2 Inhibitor
KO-947: Potent ERK1/2 Inhibitor Aberrant signaling caused by mutations or dysregulation of MAPK pathway associated with numerous tumor types Inhibitors of RAF and MEK have validated MAPK pathway in cancer Competitors have demonstrated limited clinical activity in selected patients, but it has been c
Slide 32: KO-947: Translational Research Identified Potential Lead Clinical Indications
KO-947: Translational Research Identified Potential Lead Clinical Indications EXTENSIVE PRECLINICAL EVALUATION OF KO-947 ANTI-TUMOR ACTIVITY IN MAPK DYSREGULATED TUMORS KO-947 evaluated in ~200 PDX models across 20 potential indications Identified broad tumor classes sensitive to ERK inhibition (> 5
Slide 33: KO-947 is Highly Active in Preclinical Models in Biomarker Subsets of HNSCC
KO-947 is Highly Active in Preclinical Models in Biomarker Subsets of HNSCC Final dose 3/3 CRs on QW dosing Final dose Restart dosing 2/3 CRs on Q2D or QW dosing Regressions of Large HNSCC Tumors Complete Responses in Smaller HNSCC Tumors All animals dosed orally Vehicle 120mg/kg Q2D 300mg/kg QW Tum
Slide 34: KO-539 (Menin-MLL Inhibitor)
KO-539 (Menin-MLL Inhibitor)
Slide 35: KO-539: Potential First-in-Class Inhibitor of the Menin-MLL Interaction
KO-539: Potential First-in-Class Inhibitor of the Menin-MLL Interaction Chromosomal translocations of the MLL gene play a causative role in the onset, development and progression of a subset of acute leukemias MLL-r fusion proteins and a similar mutation, MLL partial tandem duplication, drive overex
Slide 36: KO-539 Displays Prolonged Efficacy in Xenograft Model
KO-539 Displays Prolonged Efficacy in Xenograft Model KO-539 is a potent and selective inhibitor of the menin-MLL interaction KO-539 demonstrated robust efficacy in in vivo models of MLL-r AML Tumor regressions sustained at 30 days following end of dosing period Dosing Period In Vitro Potency KO-539
Slide 37: Kura Is Building a Robust Patent Portfolio
Kura Is Building a Robust Patent Portfolio Tipifarnib (Farnesyl Transferase Inhibitor) U.S. patent 9,707,221 provides exclusivity in U.S. for tipifarnib in HRAS mutant HNSCC indication until August 2036 Additional patent applications pending in the U.S. and foreign countries for HRAS mutant HNSCC as
Slide 38: Anticipated Milestones
Anticipated Milestones PROGRAM MILESTONES ESTIMATED Tipifarnib (Farnesyl Transferase Inhibitor) Initiate Phase 2 clinical trial in CMML ü Additional data from Phase 2 clinical trial in HRAS mutant HNSCC ü Data from Phase 2 clinical trial in PTCL ü Additional data from Phase 2 clinical trial in HRAS
Slide 39: Kura: Developing Precision Medicines to Treat Cancer
eQKURA KURA DEVELOPING PRECISION MEDICINES TO TREAT CANCER
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