BL-8040: A Novel CXCR4 Antagonist

Transcription

1 BL-8040: A Novel CXCR4 Antagonist December 2012

2 Forward Looking Statements This presentation contains "forward-looking statements." These statements include words like "may," "expects," "believes," plans, scheduled," and "intends," and describe opinions about future events. These forward-looking statements involve known and unknown risks and uncertainties that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. 2

3 Proposed BL-8040 AML study A Phase 2, Multicenter, Open-label Study Designed to Evaluate Safety and Efficacy Profile of Repeated Escalating Doses of BL-8040 in Adult Subjects with Relapsed or Refractory Acute Myeloid Leukemia Study design Relapsed or Refractory AML, aged Up to 50 patients Dose escalation 3+3 design, up to 5 escalating doses ( mg/kg) Expand safe, efficacious dose group Treatment Prophase 2 doses of BL-8040 mono-therapy on consecutive days 5 days of BL Cytarabine 1.5-3g/m 2 (ARA-C) Endpoints Safety and tolerability Efficacy Rate and magnitude of response Proportion of AML blasts in Peripheral Blood and Bone Marrow Leukemic cell apoptosis in Peripheral Blood and Bone Marrow 3

4 BL-8040 AML study design Relapse Treatment Follow Up BM biopsy: Day: Screening BL-8040 BL Chemotherapy Evaluation of response BM biopsy Study is expected to initiate Q Preliminary safety, tolerability and efficacy results are anticipated by the end of

5 BL-8040 AML Study Medical Centers and PI s Dr. Hagop Kantarjian Dr. Jorge Cortes Dr. Gautam Borthakur Dr. Arnon Nagler Dr. Ofer Shpilberg Dr. Dina Ben-Yehuda Dr. Itai Levi Dr. Ella Naparstek Dr. Irit Avivi 5

6 BL-8040 Product Positioning Outline BL-8040 will also be tested for treatment of ALL Accounts for 10%-15% of leukemia cases mainly pediatric Adult patients account for less than 40%, with a very poor prognosis BL-8040 can be further developed to target additional oncological and oncology-related conditions Can be expanded to treat non-hematological solid tumor types In-vivo results demonstrate tumor regression in NSCLC, pancreatic cancer and melanoma Can be combined with anti-cancer agents, including anti CD-20 targeted therapy and Velcade 6

7 Percentage distribution Acute Leukemia Drug Development Leukemia drug development is focused on underserved and treatment resistant patient populations Majority of leukemia pipeline treatments are targeted treatments Leukemia pipeline was projected to reach ~$8 billion in 2019 Up to 50% of all leukemias are acute with poorer prognoses than chronic disease 100% 90% 13% 9% 7% 18% 80% 70% 60% 33% 35% 33% 50% 40% 30% 12% 13% 15% 8% 10% 20% 10% 29% 34% 31% 0% US Japan 5EU AML ALL CML CLL Other 7

8 Summary BL-8040 is a phase II ready novel CXCR4 antagonist to be developed for treatment of AML and ALL BL-8040 may be additionally developed to target a wide range of hematological and non-hematological tumors. BL-8040 binds and antagonizes CXCR4 with high affinity BL-8040 has shown good safety profile in clinical trial in Multiple Myeloma patients BL-8040 has significant inhibitory effect on tumor growth in numerous cancer types BL-8040 has synergic effect in-vitro when combined with chemoand bio-based anti cancer therapies 8

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