Biolinerx Ltd. · TASE:BLRX
Biolinerx — Investor Presentation (April 2013)
53 slides from Biolinerx's investor presentation, Apr 30, 2013
Slide 1: Company Presentation April/May 2013
Company Presentation April/May 2013
Slide 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
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
Slide 5: Our Business Model
Our Business Model • Strong scientific basis • High chance of regulatory approval • Competitive advantage • Strong IP • Validate results of inventors • Resolve main concerns regarding toxicity, CMC, etc. • Accelerated development program • FDA/EMA standards • Continued clinical development • Regulat
Slide 6: Our Project Infrastructure Support
BD In-licensing and Out-licensing Expert Project Managers Medical and Clinical Team Pre-Clinical Team Multiple Projects Our Project Infrastructure Support
Slide 7: Pipeline of programs and indications
ca —_»_~_~_ Ventricular remodeling post-AM! ‘Out-licensed to kara, nc. following completion of Phase Ul ‘Skin lesions Inflammatory bowel disease (BD) ANIL & other hematological cancers Neuropathic pain Hepatitis C (HCV) Celiac disease Cachexa Inflammatory diseases Hepatitis C (HCV) ‘Severe allergies
Slide 8: Selected Programs
SELECTED PROGRAMS
Slide 9: BL-1020: First-in-class GABA-enhanced anti-psychotic for schizophrenia
BL-1020: FIRST-IN-CLASS GABA-ENHANCED ANTI-PSYCHOTIC FOR SCHIZOPHRENIA
Slide 10: BL-1020 - Status Update and Next Steps
BL-1020 - Status Update and Next Steps • Phase II/III CLARITY study interim analysis results were announced on March 20 – Primary and secondary cognition endpoints would not be reached with any reasonable number of patients in CLARITY study – Several statistical parameters specified in the statistic
Slide 11: 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)
Slide 12: 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 stand
Slide 13: Current Treatment Methodologies and Unmet Medical Need
Current Treatment Methodologies and Unmet Medical Need Vessel occlusion Tissue damage
Slide 14: 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 15: BL-1040 Prevents Structural Changes Following AMI
Normal size left ventricle Normal LV wall Dilated left ventricle Thin LV wall BL-1040 Prevents Structural Changes Following AMI Untreated BL-1040 Porcine AMI model, day 60
Slide 16: 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 – 27 patients, safety and preliminary efficacy in patients with primary MI at high risk for LV remodeling • 9 si
Slide 17: 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 18: 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 19: 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
Slide 20: 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
Slide 21: 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 –
Slide 22: Treatment Profile Comparison
BL-5010 Cryotherapy Laser Electro- Desiccation Surgery PDT Speed of treatment Quick Very Quick Medium Slow Slow Slow Efficacy Very high High High Very high Very High High Pain Minimal Moderately Painful Mod. Painful Mod. Painful Painful Yes Scarring Minimal Hypopigmentation None Some Significant Non
Slide 23: 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 24: 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 25: Representative Before and After Pictures
Representative Before and After Pictures
Slide 26: 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 27: 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 28: BL-7040: Orally available TLR-9 agonist for IBD
BL-7040: ORALLY AVAILABLE TLR-9 AGONIST FOR TREATMENT OF INFLAMMATORY BOWEL DISEASE
Slide 29: 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 (2 nd generation) – Oligo sequence was c
Slide 30: 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 31: BL-7040 Pre-Clinical Efficacy Data
BL-7040 Pre-Clinical Efficacy Data • BL-7040 significantly reduced colitis severity in levels comparable to Dexamethasone in -vivo • BL-7040 reduced LPS induced cytokine secretion in intestinal explants BL-7040 BL-7040 + LPS Study design: 1. Colitis induced in Balb/c mice using TNBS 2. Mice were adm
Slide 32: Response Rates in Representative Studies of Moderate to Severe UC
Response Rates in Representative Studies of Moderate to Severe UC Budesonide MMX (corticosteroid)/Mesalamine (5-ASA) - oral • Clinical response (8-week treatment): 25% (placebo) versus ~33%% study drug • Mucosal Healing (8-week treatment): 33% (placebo) versus ~40% study drug Adalimumab (Humira)* -
Slide 33: 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 34: Main Endpoints of Study
Main Endpoints of Study Met Primary - at least a 3-point decrease and 30% reduction from baseline in Mayo score, plus a ≥ 1-point decrease in rectal bleeding sub-score or an absolute rectal bleeding sub-score of ≤ 1 2-point reduction - at least a 2-point decrease from baseline in Mayo score 1-point
Slide 35: 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 36: BL-8040: Best-in-class CXCR4 antagonist for hematological cancers
BL-8040: BEST-IN-CLASS CXCR4 ANTAGONIST FOR TREATMENT OF HEMATOLOGICAL CANCERS
Slide 37: 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 38: 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 Time post BL-8040/Mozobil injection BL-8040 12 mg/kg Mozobil 3.2 mg/kg Time post BL-8040/Mozobil injection BL-8
Slide 39: 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 BL-8040 Anti-Cancer Activity Compared with Mozobil
Slide 40: BL-8040 Inhibits Tumor Growth in Mouse Xenograft Leukemia Model
BL-8040 Inhibits Tumor Growth in Mouse Xenograft Leukemia (NB4) Model
Slide 41: 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 42: BL-8040 AML Study Design
BL-8040 AML Study Design • Study expected to initiate in Q2 2013 − Regulatory approval in US already received • Partial safety, tolerability and efficacy results expected by end of 2013 • Full results expected in 2014 Screening BL-8040 BL-8040 + Chemotherapy Evaluation of response 1 2 3 4 5 6 7 ----
Slide 43: BL-8020: Oral, synergistic treatment for HCV
BL-8020: ORAL, SYNERGISTIC TREATMENT FOR HCV
Slide 44: 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 45: 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 46: Ex-Vivo Results
Ex-Vivo Results HCQ RBV The antiviral activity of HCQ showed time and dose-dependent inhibitory effects on HCV replication
Slide 47: HCQ and Ribavirin - Synergetic Effect
HCQ and Ribavirin - Synergetic Effect HCQ+RBV 1µM HCQ+RBV 10µM HCQ+RBV 20µM HCQ+RBV 50µM
Slide 48: BL-8020: Upcoming Phase I/II Clinical Trial
Period 1 Ribavirin (weight based dosage, BID) Period 2 Ribavirin (Weight based, BID) + 575mg HCQ (QD) • 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
Slide 49: Corporate
CORPORATE
Slide 50: 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 51: Analyst Coverage
Analyst Firm Raghuram Selvaraju Aegis Capital Corp. Steven Tepper Harel Finance Robert Hazlett Roth Capital Partners, LLC Analyst Coverage BioLineRx Ltd. is followed by the analysts listed below: Please note that any opinions, estimates or forecasts regarding BioLineRx Ltd.'s performance made by the
Slide 52: 2013-14 Anticipated Clinical Milestones
2013-14 Anticipated Clinical Milestones
Slide 53: BIOLINEM ><
BIOLINEM ><