Biolinerx Ltd. · TASE:BLRX
Biolinerx — Investor Presentation (June 2013)
51 slides from Biolinerx's investor presentation, June 2013
Slide 1: Company Presentation
Company Presentation June 2013
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: BioLineRx Highlights
BioLineRx Highlights • 7 clinical stage assets in variety of indications, 2 in advanced clinical stages • Broad pre-clinical pipeline – providing multiple opportunities for next generation clinical projects • Special strategic relationships and access to Israeli technology • Strong balance sheet - $
Slide 4: Overview and Business Model
OVERVIEW AND BUSINESS MODEL 4
Slide 5: Our Business Model
Our Business Model Identify promising Conduct Feasibility Develop through Partner projects Testing` human POC • Strong scientific • Validate results of • Accelerated basis • Continued clinical inventors development development • High chance of program • Resolve main • Regulatory approval regulatory
Slide 6: Our Project Infrastructure Support
Our Project Infrastructure Support Expert Medical and Project Clinical Team Managers BD In-licensing and Pre-Clinical Out-licensing Team Multiple Projects Chemistry Team (Organic, Analytical, Intellectual Bioanalytical, Property Experts Formulation) Regulatory Affairs Team 6
Slide 7: Current Pipeline
Current Pipeline a | BL-1040 Ventricular remodeling post-AMI Out-licensed to Ikaria, Inc. following completion of Phase I/II Skin lesions Inflammatory bowel disease (IBD) AML & other hematological cancers Neuropathic pain Hepatitis C (HCV) BL-1020 Schizophrenia Phase IV/Ill study discontinued follow
Slide 8: Selected Programs
SELECTED PROGRAMS 8
Slide 9: BL-1040: First-in-Class Myocardial Implant
BL-1040: FIRST-IN-CLASS MYOCARDIAL IMPLANT FOR PREVENTION OF VENTRICULAR REMODELING FOLLOWING AMI Out-licensed to Ikaria Inc. and being developed as Bioabsorbable Cardiac Matrix (BCM) 9
Slide 10: 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 - conducted by Ikaria Inc. • Product highlights: • Resorbable polymer solution administered via intracoronary injection during standa
Slide 11: Current Treatment Methodologies and Unmet Medical Need
Current Treatment Methodologies and Unmet Medical Need Vessel occlusion Tissue damage Successfully treated with PCI and stents No sufficiently effective treatment for myocardial damage 11
Slide 12: How Does BL-1040 Work?
How Does BL-1040 Work? Infarct related artery (intra-vascular) injection Deposition into infarcted tissue Liquid to gel phase transition upon contact with infarcted tissue Phase transition at infarct area creates resorbable bioprosthetic scaffold providing mechanical support to damaged cardiac muscl
Slide 13: BL-1040 Prevents Structural Changes Following AMI
BL-1040 Prevents Structural Changes Following AMI Porcine AMI model, day 60 Dilated left Normal size ventricle left ventricle Normal LV Thin LV wall wall Untreated BL-1040 13
Slide 14: BL-1040 Safe and Well-Tolerated in Phase I/II
BL-1040 Safe and Well-Tolerated in Phase I/II Clinical Trial Results • Clinical program based on extensive interaction with FDA (CDRH division) • Pilot study in Europe – completed January 2010 – 27 patients, safety and preliminary efficacy in patients with primary MI at high risk for LV remodeling •
Slide 15: Ikaria BL-1040 Out-Licensing Transaction
Ikaria BL-1040 Out-Licensing Transaction • Ikaria - proven expertise in drug and medical device development and singular focus on acute care • Terms: √ $7 million upfront √ $10 million first milestone – ~$115 million in additional regulatory and developmental milestones – ~$150 million in commercial
Slide 16: BL-1040 Clinical Development Program
BL-1040 Clinical Development Program • Current development program includes two pivotal trials – CE Mark Registration trial for European approval – Second pivotal trial for US approval • Pivotal CE Mark Registration trial currently progressing at full steam – Multiple sites currently active in six c
Slide 17: BL-1040 Clinical Development Program (cont.)
BL-1040 Clinical Development Program (cont.) • Second pivotal trial for US approval in planning stages − Final discussions with FDA − Overall design: • Placebo controlled • >1,000 patients • 12 month follow-up 17
Slide 18: BL-5010: Novel Formulation for Non-Surgical Removal of Skin Lesions
BL-5010: A NOVEL FORMULATION FOR NON-SURGICAL REMOVAL OF SKIN LESIONS, EMBEDDED WITHIN NEW APPLICATOR PEN (BL-5010P) - FOR TOPICAL USE 18
Slide 19: BL-5010 Highlights
BL-5010 Highlights • Positioning: Novel formulation for non surgical removal of variety of skin lesions • Status: Pilot study completed in benign lesions (60 pts) • Product Highlights: – Single application office-based treatment – State-of-the-art applicator developed for streamlined application – P
Slide 20: Treatment Profile Comparison
Treatment Profile Comparison Electro- Surgery BL-5010 Cryotherapy Laser PDT Desiccation Speed of Slow Quick Very Quick Medium Slow Slow treatment Efficacy Very high High High Very high Very High High Pain Minimal Moderately Painful Mod. Painful Mod. Painful Painful Yes Scarring Minimal Hypopigmentat
Slide 21: BL-5010 Phase I/II Clinical Trial Overview
BL-5010 Phase I/II Clinical Trial Overview • Open-label, single-arm, safety and efficacy study • 60 patients with seborrheic keratosis investigated for BL-5010 single application safety and efficacy • Patients treated and followed-up for 6 months • Conducted in Germany and Holland • Endpoints: – Saf
Slide 22: BL-5010 Phase I/II Clinical Trial Results
BL-5010 Phase I/II Clinical Trial Results • Safety – BL-5010 has a good safety profile - no persistent irreversible adverse effects were observed at treated site • Efficacy – Lesion removal - lesions fell off by Day 30 in >96% of patients – Cosmetic outcome – 94.6% of investigator-assessed cosmetic
Slide 23: Representative Before and After Pictures
Representative Before and After Pictures 23
Slide 24: BL-5010P - A Novel Applicator Pen for Topical Use
BL-5010P - A Novel Applicator Pen for Topical Use • Distribution accuracy on target tissue • Volume consistency • Single patient use with potential to treat multiple lesions • Allows application of pressure • Ready for use • Easy to use • Hand-held device • Aesthetic and ergonomic design • Straightf
Slide 25: BL-5010: Summary and Current Status
BL-5010: Summary and Current Status • BL-5010 designated as a medical device in Europe • Pilot safety and efficacy study successfully completed in seborrheic keratosis patients • Pivotal CE Mark Registration study to commence in H213 – Bridging study of BL-5010 pen-like applicator in seborrheic kera
Slide 26: BL-7040: Oral TLR-9 Agonist for IBD
BL-7040: ORALLY AVAILABLE TLR-9 AGONIST FOR TREATMENT OF INFLAMMATORY BOWEL DISEASE 26
Slide 27: BL-7040 Highlights
BL-7040 Highlights • Indication: Inflammatory bowel disease (IBD) • Mode of Action: Toll-Like Receptor 9 (TLR-9) modulator/agonist • Status: Phase IIa proof-of-concept study completed • Product Highlights: – Orally available synthetic oligodeoxynucleotide (2nd generation) – Oligo sequence was chemic
Slide 28: IBD Overview
IBD Overview • A group of inflammatory conditions of the colon and small intestine • Two major types: – Ulcerative colitis (UC): limited to the colon – Crohn’s disease (CD): involves multiple segments of the gastrointestinal tract • Common end pathway for both types consists of inflammation of the i
Slide 29: BL-7040 Pre-Clinical Efficacy Data
BL-7040 Pre-Clinical Efficacy Data BL-7040 is as Effective as Dexamethasone in Treating Established IBD BL-7040 BL-7040 + LPS Study design: • BL-7040 significantly reduced colitis severity in levels comparable to 1. Colitis induced in Balb/c mice using TNBS Dexamethasone in -vivo 2. Mice were admini
Slide 30: Response Rates in Representative Studies of Moderate to Severe UC
Response Rates in Representative Studies of Moderate to Severe UC Adalimumab (Humira) Mesalamine (5-ASA) Open-label Phase IIa, 20 UC patients Double blind Phase III, 509 UC patients showed clinical response rate of 25% showed clinical response rate of 33% 30
Slide 31: BL-7040 Phase IIa Study Design
BL-7040 Phase IIa Study Design • General – Open-label study to evaluate efficacy, pharmacodynamics, safety and tolerability – Up to 30 patients with moderately active ulcerative colitis • Recruitment and Treatment – 22 patients enrolled in the study – 6 patients discontinued and were not included in
Slide 32: Main Endpoints of Study
Main Endpoints of Study Primary Endpoint – Mayo Score Change in Rectal Bleeding (end of treatment) (RB) from Baseline 12 * 12 9 Number of patients 10 8 8 6 75% 56% 4 50% 2 0 Met Primary 2 points 1 point Met Primary - at least a 3-point decrease and 30% * Note - 6 of these 9 patients (67%) demonstrat
Slide 33: Summary of BL-7040 Phase IIa Results
Summary of BL-7040 Phase IIa Results • Efficacy – Primary clinical endpoint in study - reduction in Mayo score between baseline and completion of treatment – was achieved. • 50% of patients (8 patients) met primary endpoint; remaining 8 patients demonstrated stable clinical condition or minor improv
Slide 34: BL-8040: Best-in-Class CXCR4 Antagonist for Hematological Cancers
BL-8040: BEST-IN-CLASS CXCR4 ANTAGONIST FOR TREATMENT OF HEMATOLOGICAL CANCERS 34
Slide 35: BL-8040 Highlights
BL-8040 Highlights • Indications: AML & other hematological cancers • Mode of Action: CXCR4 antagonism – CXCR4 over-expressed in >70% of tumors, and correlates with disease severity. • Status: Phase II development • Product Highlights: – Peptide – Activity against various cancer types has been demon
Slide 36: BL-8040 Mobilization Compared with Mozobil
BL-8040 Mobilization Compared with Mozobil • BL-8040 affords superior mobilization of neutrophils and progenitor cells • Mobilization superiority correlates with CXCL12 levels in the blood BL-8040 12 mg/kg Number of Neutrophils Mozobil 3.2 mg/kg Time post BL-8040/Mozobil injection CXCL12 serum level
Slide 37: BL-8040 Anti-Cancer Activity Compared with Mozobil
BL-8040 Anti-Cancer Activity Compared with Mozobil • BL-8040, in contrast to Mozobil, exerts potent cytotoxicity against human leukemia and myeloma cells in-vitro in a dose-dependent manner 120 NB4 HL60 120 NB4 HL60 (% of control) 100 (% of control) 100 Viable cells Viable cells 80 80 60 60 40 40 20
Slide 38: BL-8040 Inhibits Tumor Growth in Mouse Xenograft Leukemia Model
BL-8040 Inhibits Tumor Growth in Mouse Xenograft Leukemia (NB4) Model I II Tumor size (cm2) 2.4 Ctrl 2.4 2 2 weight (g) BL-8040 200 1.6 1.6 1.2 1.2 ** 0.8 0.8 0.4 0.4 0 0 0 4 8 12 16 20 24 28 ctrl BL-8040 Time (days) DAPI TUNEL III x200 x200 Ctrl x200 x200 BL-8040 38
Slide 39: Upcoming BL-8040 Phase II Study in AML
Upcoming BL-8040 Phase II Study in AML • Study summary – Multicenter, open-label study, carried out at multiple centers in US and Israel – Adult subjects (aged 18-70) with relapsed or refractory AML • Study design – Up to 50 patients – Dose escalation phase - up to 5 escalating doses (0.5-1.5 mg/kg)
Slide 40: BL-8040 AML Study Design
BL-8040 AML Study Design Relapse Treatment Follow Up BM biopsy: Day: 1 2 3 4 5 6 7 ------------------------------------------------------------ 30 Screening BL-8040 BL-8040 + Chemotherapy Evaluation of response BM biopsy • Study expected to initiate in Q2 2013 − Regulatory approval in US already rec
Slide 41: BL-8020: Oral, Synergistic Treatment for HCV
BL-8020: ORAL, SYNERGISTIC TREATMENT FOR HCV 41
Slide 42: BL-8020 Highlights
BL-8020 Highlights • Mode of Action: Inhibition of HCV-induced autophagy • Status: Phase I/II • Product highlights: – Unique mechanism of action - inhibition of HCV-induced autophagy, a process essential for viral replication – Has demonstrated synergy with various other anti-HCV agents of different
Slide 43: Pre-Clinical Data
Pre-Clinical Data • In-vitro studies demonstrated synergistic effect with a number of HCV therapies • Ex-vivo analysis – Uninfected human liver slices were infected with HCV and cultured up to 10 days – At day 4 post-infection, liver slices were treated for 6 days with HCQ at different concentration
Slide 44: Ex-Vivo Results
Ex-Vivo Results The antiviral activity of HCQ showed time and dose-dependent inhibitory effects on HCV replication HCQ RBV 44
Slide 45: HCQ and Ribavirin - Synergetic Effect
HCQ and Ribavirin - Synergetic Effect HCQ+RBV 1µM HCQ+RBV 10µM HCQ+RBV 20µM HCQ+RBV 50µM 45
Slide 46: BL-8020: Upcoming Phase I/II Clinical Trial
BL-8020: Upcoming Phase I/II Clinical Trial • Open-label study to evaluate efficacy, safety and tolerability of ribavirin mono- therapy, followed by a combined treatment with Ribavirin and HCQ • Study conducted at two leading sites in France – Regulatory approval received in March 2013; FPI announce
Slide 47: Corporate
CORPORATE 47
Slide 48: Financial Summary
Financial Summary • Available cash as of March 31, 2013 - ~$28 million − Annual burn rate between $12-14 million − Cash expected to last into 2015 • Number of employees - 43 • Capital structure (on basis of ADSs) − 22.3 million outstanding – basic capital − 27.8 million outstanding – fully diluted c
Slide 49: Analyst Coverage
Analyst Coverage BioLineRx Ltd. is followed by the analysts listed below: Analyst Firm Raghuram Selvaraju Aegis Capital Corp. Steven Tepper Harel Finance Robert Hazlett Roth Capital Partners, LLC Please note that any opinions, estimates or forecasts regarding BioLineRx Ltd.'s performance made by the
Slide 50: 2013-14 Anticipated Clinical Milestones
2013-14 Anticipated Clinical Milestones BL-7040 (IBD) phase IIa POC study results √ BL-8020 (HCV) phase I/II initiation √ 2013 BL-8040 (AML) phase II initiation BL-5010 (Skin Lesions) pivotal EU study initiation BL-8020 (HCV) partial results BL-7010 (Celiac Disease) pilot study initiation BL-8040 (A
Slide 51: Bench1o Bedoideto Patner
Bench1o Bedoideto Patner BIOLINEM ><