Biolinerx Ltd. · TASE:BLRX
Biolinerx — Investor Presentation (March 2015)
38 slides from Biolinerx's investor presentation, Mar 5, 2015
Slide 1: Company Presentation
Exhibit 99.1 Company Presentation February 2015
Slide 2: Forward Looking Statements
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 u
Slide 3: Offering Summary
Offering Summary Issuer: BioLineRx Ltd. (NasdaqCM:BLRX / TASE:BLRX) Offering Size: ~$30 million (100% primary; excluding overallotment) Overallotment Option: 15.0% (100% primary) Offering Type: Confidentially-Marketed Follow-On American Depositary Shares Securities Offered: (1 ADS represents 10 Ordi
Slide 4: BioLineRx Snapshot
BioLineRx Snapshot • Drug development company focused on oncology & immunology – Founded in 2003 by Teva and other key players in Israeli Life Sciences industry • Bridge “development gap” for Israeli assets – Leverage carefully selected early-stage technology, primarily at academia level, following
Slide 5: Long Term Relationships with Israeli Institutions
Long Term Relationships with Israeli Institutions Technion Rambam Medical Center Haifa Tel Aviv University Sheba Medical Tel- Aviv Center Hebrew Rehovot Jerusalem University Be’er Hadassah Medical Sheva Center Weizmann Institute Ben-Gurion Sourasky Medical University Center Extensive in-licensing tr
Slide 6: Main Pipeline Assets
INDICATION LEAD DEVELOPMENT PROGRAMS AML ‘Stem cell mobilization BL-7010 Celiac disease ADDITIONAL DEVELOPMENT PROGRAMS BL-7040 Inflammatory bowel disease BL-9010 ‘Severe asthma/allergies BL-1110 Neuropathic pain/scleroderma — PROGRAMS PARTNERED OR IN CO-DEVELOPMENT BL-1040 Ventricular remodeling po
Slide 7: Main 2014 Achievements
Main 2014 Achievements • R&D Achievements in 2014 – All three lead development programs met their objectives in 2014 • BL-8040 (r/r AML) - phase 2 study partial results – Clinical evidence for MOA (mobilization and apoptosis) • BL-7010 (celiac program) - phase 1/2 completion • BL-1040 (AMI) - comple
Slide 8: Main 2015 Value Drivers
Main 2015 Value Drivers • BL-8040 stem cell mobilization study – Top line results by end of Q1 2015 • BL-8040 r/r AML study – Top line results H2 2015 • BL-1040 CE Mark registration study – Topline results mid-year 2015 • BL-7010 project – Final approval of device regulatory path in EU from Notified
Slide 9: Lead Development Programs
LEAD DEVELOPMENT PROGRAMS 9
Slide 10: BL-8040: Best-in-Class CXCR4 Antagonist for Treatment of Hematological Cancers
BL-8040: BEST-IN-CLASS CXCR4 ANTAGONIST FOR TREATMENT OF HEMATOLOGICAL CANCERS 10
Slide 11: BL-8040 Highlights
BL-8040 Highlights • Platform Molecule: Can Address Multiple Cancer Indications – Acute myeloid leukemia (AML) & other blood cancers – Received Orphan designation from FDA for lead indications (accelerates development) • Mode of Action: Inhibits CXCR4 (a cell surface protein) – Present in high quant
Slide 12: AML - Treatment and Unmet Medical Need
AML - Treatment and Unmet Medical Need • AML is most common acute leukemia in adults – Over 60,000 new cases recorded worldwide in 2010, growing to 130,000 by 2020 – 14,000 cases of AML diagnosed in the US in 2012 – Majority of AML patients relapse and require repeated treatment cycles • AML has poo
Slide 13: BL-8040 Mechanism Of Action
BL-8040 Mechanism Of Action • Binds CXCR4 with high affinity (1-2 nM) • Maintains extended inhibition of CXCR4 through long receptor occupancy (>24 hours) • Works as inverse agonist of CXCR4 • Induces apoptosis of tumor cells dependent on CXCR4 for survival • Increases sensitivity to anti-cancer age
Slide 14: BL-8040 Shows Superiority to Current Therapy
BL-8040 Shows Superiority to Current Therapy BL-8040 12 mg/kg Mozobil 3.2 mg/kg BL-8040 Stimulates Greater Mobilization of Cells from the Bone Marrow Compared to Mozobil Time post BL-8040/Mozobil injection Abraham M., Stem Cells 2007;25:2158 -2166 Mozobil BL-8040 Causes Significant Cancer Cell Death
Slide 15: BL-8040 is Best-in-Class vs. Competitors
BL-8040 is Best-in-Class vs. Competitors BMS-936564 BL-8040 Mozobil (MDX1338) Affinity for CXCR4 1-2 nM 84 nM 5nM Inhibition Inverse agonist Antagonist (partial agonist) Antagonist Extracellular domains in the Trans-membrane regions in the Extracellular domains in the CXCR4 Binding site CXCR4 recept
Slide 16: Phase 2a - Treatment of r/r AML patients
Phase 2a - Treatment of r/r AML patients Open-label study to evaluate safety and efficacy profile of repeated escalating doses of BL -8040 in up to 70 adult subjects with relapsed or refractory AML Study design: – Dose escalation phase - 3+3 design (last update given re 5th cohort of 1.5 mg/kg) – Ex
Slide 17: Partial Results in AML Phase 2 Study
Partial Results in AML Phase 2 Study • No BL-8040 related SAEs and no AEs were considered DLTs • Robust leukemic blast mobilization was observed (median of 6-fold increase) • BL-8040 monotherapy decreased amount of leukemic cells in BM by median of ~70% • BL-8040 monotherapy achieved 3.5-fold increa
Slide 18: 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 19: Three New Studies to be Initiated in 2015
Three New Studies to be Initiated in 2015 Results Study Collaborator Description ΠAML German Study Double-blind, placebo-controlled, Consolidation Topline results Alliance repeated-administration, multi Phase 2b Q4 2017 Leukemia Group treatment cycles (200 patients) Open-label, 2 parts: Partial res
Slide 20: BL-8040 Summary
BL-8040 Summary • CXCR4 is a validated target • BL-8040 has robust mobilization activity and apoptosis – Validated in preliminary data from Phase II study in AML, and Phase 1/2 study in multiple myeloma – BL-8040 has very favorable profile in comparison with leading CXCR4 antagonists • BL-8040 is an
Slide 21: BL-1040: First-in-Class Myocardial Implant for Prevention of Ventricular Remodeling
BL-1040: FIRST-IN-CLASS MYOCARDIAL IMPLANT FOR PREVENTION OF VENTRICULAR REMODELING FOLLOWING AMI Out-licensed to Bellerophon (f/k/a Ikaria) and being developed as Bioabsorbable Cardiac Matrix (BCM) 21
Slide 22: BL-1040 Highlights
BL-1040 Highlights • Indication: Cardiac remodeling post-AMI • Mode of Action: Provides support to ischemic tissue during healing • Status: CE Mark registration trial - enrollment completed; top-line results in mid-2015 • Device designation (including FDA) • Partnered with Bellerophon BCM (f/k/a Ika
Slide 23: Unmet Medical Need
Unmet Medical Need Vessel occlusion Tissue damage 23
Slide 24: How Does BL-1040 Work?
How Does BL-1040 Work? Porcine AMI model, day 60 Arterial injection deposits Untreated BL- material into infarcted tissue 1040 L L Turns from liquid to gel on V V contact with infarcted tissue Gel-like scaffold provides mechanical support to damaged tissue • Dilated • Normal size • Thin LV wall • No
Slide 25: BL-1040 Clinical studies
BL-1040 Clinical studies Pivotal CE Mark Registration trial progressing at full steam •Placebo controlled, enrollment of 303 patients P • 6-month follow-up •Includes ~90 sites in nine countries (including 16 sites in US) •Endpoints: end diastolic volume, QLQ, six-minute walk test •Top-line results e
Slide 26: BL-1040 Summary
BL-1040 Summary • Huge unmet medical need – >$1 billion market • Designated as device in both US and Europe • Pilot study successfully completed – No safety or tolerability issues after six months of follow-up – Promising efficacy in comparison to historical data • Top-line results of CE mark regist
Slide 27: BL-7010: Novel Gliadin Binding Polymer for Celiac Disease
BL-7010: NOVEL GLIADIN BINDING POLYMER FOR CELIAC DISEASE 27
Slide 28: BL-7010: Polymeric Binder for Celiac Disease
BL-7010: Polymeric Binder for Celiac Disease • Indication: Celiac disease • Mode of Action: Non-absorbable polymer with high affinity to gliadins (immunogenic peptides contained in gluten) • Status: Phase 1/2 completed • Product Highlights – Prevents pathological damage to small intestine – Non-abso
Slide 29: Celiac Disease - Large Unmet Medical Need
Celiac Disease - Large Unmet Medical Need • 1% of world’s population suffers from celiac disease – Number underestimated due to lack of awareness/diagnostic tools • Market projected to reach $8 billion by 2019 • No current pharmacological agents approved for celiac – Only treatment option is life-lo
Slide 30: Gluten Gluten BL-7010
Gluten Gluten BL-7010 Copolymer of sodium styrene sulfonate (SS) and 2-hydroxyethyl methacrylate (HEMA) Gliadin Enterocytes BL-7010 demonstrates distinguished specificity towards gliadin Small intestinal damage with loss of absorptive villi and hyperplasia of the crypts, typically leading to malabso
Slide 31: BL-7010 Maintains Normal Structure of GI
BL-7010 Maintains Normal Structure of GI Model: HLA-DQ8/HCD4 transgenic male mice sensitized to gliadin non-sensitized Gluten-sensitized Gluten-sensitized mice mice mice + BL-7010 Villus-to-crypt ratio 5.96 ± 1.23 2.58 ± 0.43 4.89 ± 1.51 31
Slide 32: BL-7010 Clinical Program Overview
BL-7010 Clinical Program Overview • Phase 1/2 study in celiac patients completed – Single ascending dose – 14 days repeated administration – Safety endpoints – 3 times per day – No efficacy endpoints – Safety w/o efficacy endpoints – Assessment of systemic exposure – Assessment of systemic exposure
Slide 33: BL-7010 Summary
BL-7010 Summary • Celiac disease is huge unmet medical need – There are only a handful of clinical-stage programs in development • BL-7010 has unique and simple MOA • Successful Phase 1/2 pilot study – Well tolerated, no systemic exposure – Will likely be classified as medical device in Europe • Piv
Slide 34: Corporate
CORPORATE 34
Slide 35: Transformative Collaboration with Novartis
Transformative Collaboration with Novartis • Novartis selected BioLineRx as its leading partner for identification and early development of Israeli-sourced drug candidates – Exclusive first look at all Israeli-based projects scouted by BioLineRx – Co-develop selected projects through clinical proof-
Slide 36: Financial and Corporate Summary
Financial and Corporate Summary Cash position • $29.6 million as of September 30, 2014 – Does not include $10 million received from Novartis in December • Funds operational capital into 2016 Capital structure • Traded on NASDAQ and TASE (Symbol: BLRX) • 39 million shares outstanding; 45 million full
Slide 37: Major Development Milestones - 2015 and 2016
Major Development Milestones - 2015 and 2016 BL-1040 (AMI) complete CE mark study enrollment √ BL-8040 (SC Mobilization) phase 1 completion √ BL-8040 (AML) phase 2 partial results* BL-8040 (FLT-3) phase 1/2 initiation BL-8040 (SC Mobilization) phase 1 apheresis data BL-8040 (hMDS & AA) phase 1/2 ini
Slide 38: Benchto Bedsideo Pads ner
Benchto Bedsideo Pads ner R 4 4 = mm Ne : , ~ oe . A “a i™~ j X > J BIOUINE™>*<