Biolinerx Ltd. · TASE:BLRX
Biolinerx — Investor Day (December 2014)
33 slides from Biolinerx's investor day presentation, Dec 12, 2014
Slide 1: BL-8040: A Novel Platform for the Treatment of Hematological Malignancies
BL-8040 A NOVEL PLATFORM FOR THE TREATMENT OF HEMATOLOGICAL MALIGNANCIES INVESTOR AND ANALYST BREAKFAST DECEMBER 12, 2014
Slide 2: BL-8040 Overview
Indication AML & other hematological indications (Orphan designation for AML and SC mobilization) Mode of Action CXCR4 antagonism (CXCR4 over-expressed in >70% of tumors, correlates with disease severity) Status Phase II ongoing (under IND) Product Highlights • Induces apoptosis in cancer cells • Mo
Slide 3: BL-8040's Unique Mechanism Presents an Opportunity Across Many Hematological Indications
BL-8040 BL-8040 + SOC BL-8040’s Unique Mechanism Presents An Opportunity Across Many Hematological Indications Results And MOA • Binds CXCR4 with high affinity (1-2 nM) and works as inverse agonist • Maintains extended inhibition through long receptor occupancy (>24 hours) • Induces apoptosis of tum
Slide 4: Clinical Program
Clinical Program December 2014
Slide 5: Projected Clinical Program Targets Several Hematological Indications With High Unmet Need
INDICATION PROTOCOL Ph2a - Ongoing - Topline results Q1/2015 Ph1/2 - Planned for Q2/2015-Q2/2017 Ph1/2 - Completed Ph1 - Ongoing - Q1/2015 Ph2b - Planned for Q1/2015-Q3/2017 Ph1/2 - Planned for Q1/2015-Q3/2016 Pre-Clinical Phase I Phase II Phase III R/R AML BL-8040.01 hMDS and Aplastic Anemia BL-804
Slide 6: rrAML
rrAML BL-8040.01 study Ongoing Ph2a study
Slide 7: Results From Ongoing Study
Results From Ongoing Study • First four cohorts were completed (0.5, 0.75, 1 and 1.25 mg/kg) • There were no BL-8040 related SAEs and none of the AEs were considered DLTs • Robust leukemic blast mobilization was observed (median of 6-fold increase) • Two days of BL-8040 monotherapy decreased amount
Slide 8: BL-8040.01 Currently Taking Place at Multiple Leading Medical Centers in the US and Israel
BL-8040.01 Currently Taking Place at Multiple Leading Medical Centers in the US and Israel Dr. Arnon Nagler Dr. Yishai Ofran Dr. Nadav Sarid Dr. James Foran Dr. Martin Tallman Dr. Jessica Altman Dr. Gautam Borthakur Dr. Dina Ben-Yehuda Dr. Jacob Rowe
Slide 9: AML Consolidation
AML Consolidation BL-8040.01 - the BLAST study In Collaboration with German Study Alliance Leukemia Group
Slide 10: Phase 2b - Consolidation Treatment for AML
Phase 2b - Consolidation Treatment for AML Patients Rationale • BL-8040 will deepen response by eliminating minimal residual disease left in BM after induction therapy • Safety of combination with high dose Ara-C is well established by data from r/r AML study encouraging use of BL-8040 as part of th
Slide 11: Phase 2b - Consolidation Treatment for AML
Phase 2b - Consolidation Treatment for AML Patients A Phase 2, double-blind, placebo-controlled, randomized, multicenter study to assess efficacy of BL-8040 on top of Ara-C for AML patients in first complete remission, compared to placebo on top of Ara-C Study Design • Double-blind, placebo-controll
Slide 12: AML FLT3-ITD
AML FLT3-ITD In Collaboration with MD Anderson Cancer Center
Slide 13: FLT3 Mutations in AML
FLT3 Mutations in AML • FLT3 is a member of class III receptor tyrosine kinase family, mainly expressed by early myeloid and lymphoid progenitor cells • FLT3 has important role in proliferation, survival, and differentiation of progenitor cells • FLT3 mutations present in 30-35% of AML patients & ar
Slide 14: FLT3 and CXCR4 in AML
FLT3 and CXCR4 in AML • Emerging data highlight the importance of the bone marrow (BM) niche for the survival of FLT3-mutated AML • Through CXCL12/CXCR4 interaction, leukemia cells retained in microscopic niches within the BM leading to increased proliferation and survival • CXCR4 expression is sign
Slide 15: BL-8040 Affects AML Survival In-Vivo (Pre-Clinical Data)
BL-8040 Affects AML Survival /n-Vivo (Pre-Clinical Data) In-vivo FLT3-ITD AML model in NSG mice. Mice are treated with BL-8040 for 7 daysafter which they are scarified and blood, BMand spleen are collected and analyzed Multiple BL-8040 injections reduces the number of AML cells in blood, BM and sple
Slide 16: Phase 1/2 - BL-8040 in Combination with Sorafenib for FLT3-ITD AML Patients
Phase 1/2 - BL-8040 in Combination with Sorafenib for the Treatment of FLT3-ITD AML Patients Rationale • CXCR4 expression is significantly higher in FLT-ITD AML than in FLT3 wild-type AML • CXCR4 inhibition enhances the sensitivity of FLT3-mutated leukemic cells to the apoptogenic effects of Sorafen
Slide 17: Phase 1/2 - BL-8040 in Combination with Sorafenib
Phase 1/2 - BL-8040 in Combination with Sorafenib for the Treatment of Patients with FLT3-ITD AML An Open-label Phase 1/2 Study of BL-8040 in Combination with Sorafenib for Treatment of Patients with FLT3- ITD Mutated AML Study Design • Open label, two parts, repeated administrations, multiple treat
Slide 18: hMDS and Aplastic Anemia
hMDS and Aplastic Anemia In Collaboration with MD Anderson Cancer Center
Slide 19: Hypoplastic Myelodysplastic Syndrome (hMDS)
Hypoplastic Myelodysplastic Syndrome (hMDS) • MDS is an heterogeneous group of malignant clonal disorders characterized by bone marrow dysplasia, ineffective hematopoiesis, cytopenias, and the potential to transform into acute myelogenous leukemia (AML) • Hypoplastic MDS is a subtype of MDS where th
Slide 20: Aplastic Anemia (AA)
Aplastic Anemia (AA) • Deficiency of red cells, neutrophils, monocytes, and platelets without morphological evidence of another marrow disorder. Examination of bone marrow reveals a near absence of hematopoietic precursors and fatty replacement. • Anemia leads to fatigue, dyspnea, and cardiac sympto
Slide 21: CXCR4 Involvement in Hypoplastic MDS and Aplastic Anemia
December, 2014 | Slide 21 CXCR4 Involvement in Hypoplastic MDS (hMDS) and Aplastic Anemia (AA) • hMDS and AA are hematological disorders characterized by bone marrow dysplasia, ineffective hematopoiesis and cytopenias • In AA and hMDS, an autoimmune microenvironment within the bone marrow niche has
Slide 22: Effect of BL-8040 on BM Regeneration (Preclinical Data)
December, 2014 | Slide 22 Effect of BL-8040 on BM Regeneration (Preclinical Data) • Repeated doses of BL-8040 led to marked increase in the number of hematopoietic progenitor cells (HPCs) and hematopoietic stem cells (HSCs) in the bone marrow and peripheral blood of mice • BL-8040 also promoted incr
Slide 23: Effect of BL-8040 on BM Regeneration - Granulocytes (Clinical Data)
Effect of BL-8040 on BM Regeneration - Granulocytes (Clinical Data) Baseline BM Day 3 BM • Preliminary data from BL-8040.01 trial suggest that BL-8040 induces the differentiation of BM leukemic cells into mature cells
Slide 24: Effect of BL-8040 on RBC Production (Clinical Data)
December, 2014 | Slide 24 Effect of BL-8040 on RBC Production (Clinical Data) • Preliminary data from BL-8040.01 trial suggest that BL-8040 induces the production of mature red blood cells • The effect was more dramatic in patients where the AML was secondary to MDS RBC Hemoglobin
Slide 25: Phase 1/2 - Combination of BL-8040 with Immunosuppressive Therapy in AA or hMDS
Phase 1/2 - Combination of BL-8040 with Immunosuppressive Therapy in Patients with AA or hMDS Rationale * Dual targeted strategy to improve response rates and outcomes * hATG and cyclosporine: Target autoimmune attack on hematopoietic precursors with standard immunosuppressive therapy * BL-8040: Add
Slide 26: Phase 1/2 Study of BL-8040 with Immunosuppressive Therapy in AA or hMDS
Phase 1/2 Study of the Combination of BL-8040 with Immunosuppressive Therapy in Patients with Aplastic Anemia (AA) or Hypoplastic Myelodysplastic Syndrome (hMDS) Study Design • Open label, repeated administrations, single treatment cycle Endpoints • Safety, tolerability, and toxicities of the combin
Slide 27: Stem Cell Mobilization
Stem Cell Mobilization BL-8040.02 study Ongoing Ph1 study
Slide 28: Phase 1 - Single Agent Stem Cell Mobilization
Phase 1 - Single Agent Stem Cell Mobilization A Phase 1, Two-Part Study Exploring the Safety, Tolerability, Pharmacodynamic and Pharmacokinetic Effect of Ascending Doses of BL-8040 in Healthy Subjects Study design: • Part 1 - Dose escalation, randomized, placebo controlled - up to 4 escalating doses
Slide 29: BIOLINERM X<
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Slide 30: Closing Remarks
Closing Remarks Investor/Analyst Breakfast December 12, 2014 Dr. Kinneret Savitsky
Slide 31: Major BL-8040 Development Milestones (Next Years)
Major BL-8040 Development Milestones (Next 3 Years) BL-8040 (hMDS) study completion BL-8040 (AML) phase 2 completion BL-8040 (AML) phase 2 partial results BL-8040 (SC Mobilization) phase 1 completion BL-8040 (hMDS & AA) FPI BL-8040 (FLT-3) study completion BL-8040 (Consolidation) FPI BL-8040 (FLT-3)
Slide 32: Lead Program Update
Lead Program Update BL-7010: • Announced Phase 1/2 topline results • Dose was selected • Pivotal study for EU to start in H2 2015 BL-1040: • Enrolled over 280 patients out of ~300 • Enrollment to be completed for year-end • Study completion in mid-2015
Slide 33: Bencho Bedside Xo Patiner
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