Biolinerx Ltd. · TASE:BLRX
Biolinerx — Investor Presentation (April 2010)
45 slides from Biolinerx's investor presentation, April 2010
Slide 1: Company Presentation
Company Presentation April 2010
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: Table of Contents
Table of Contents • Background • Lead Clinical Programs – BL-1020 – BL-1040 • “Next in Line” • Management & Financial • Summary 3
Slide 4: Background
Background 4
Slide 5: BioLineRx at a Glance
BioLineRx at a Glance • Founded in 2003. Raised ~ $80m in three public offerings since 2007 • Market cap ~$150 million- symbol “BLRX” • Key Shareholders: • Public – 65% • Pan Atlantic Investments - 13% • Teva Pharmaceuticals - 10% • Clal Holdings - 9% • Directors & management – 4% • 49 employees • C
Slide 6: Company Highlights
Company Highlights • Clinical stage company with three compounds in clinical trials for large market indications • BL-1020 meets primary efficacy endpoints with uniquely differentiated target product profile in phase 2b study • Potential for out-licensing event in mid 2010 for BL-1020 • Validated bu
Slide 7: Founding Assumptions
Founding Assumptions • Drug development is risky and difficult and requires expertise, time and money • Successful innovative drugs can generate high value and high returns • Innovative candidates can be monetized at high values after showing clinical POC (proof of safety and efficacy, i.e. phase 2
Slide 8: The Business Model
The Business Model • Proactive screening from universities and research labs to identify promising programs • Use rigorous proprietary screening methodology to select compounds for in-licensing – >1000 compounds screened (approx 70% Israel / 30% ROW) – 29 compounds entered development – 11 active pr
Slide 9: The Business Model (continued)
The Business Model (continued) • BioLineRx acquires exclusive license to early stage compounds from research facilities, small companies, universities, etc. in exchange for approximately 25% of future payments received • BioLineRx continues development through Phase 2 safety and efficacy studies • B
Slide 10: Lead Clinical Programs
Lead Clinical Programs 10
Slide 11: BL-1020: First in Class GABA-Enhanced Antipsychotic for Schizophrenia
BL-1020: First in Class GABA-Enhanced Antipsychotic for Schizophrenia 11
Slide 12: BL-1020: First-In-Class GABA Enhanced Antipsychotic for Schizophrenia
BL-1020: First-In-Class GABA Enhanced Antipsychotic for the Treatment of Schizophrenia • Indication: Schizophrenia • Efficacy: – Highly efficacious, similar to best in class SGA and FGA • Safety: – Overall: clean safety profile; better than FGA & SGA – Metabolic: no metabolic side effects (BL-1020 >
Slide 13: BL-1020 Mode Of Action
BL-1020 Mode Of Action • Schizophrenia involves an imbalance in two key neurotransmitters – Dopamine hyperactivity (Current antipsychotics block dopamine) – GABA hypoactivity (free GABA cannot cross the BBB) • BL-1020 MOA: – Dopamine antagonism – GABAergic activity 13
Slide 14: Phase 2b EAGLE Trial Overview
Phase 2b EAGLE Trial Overview • Double blind, multi-center, 6-week study • Designed to determine the efficacy, safety and tolerability of BL-1020 in hospitalized patients with acute exacerbation of schizophrenia • Full GCP trial conducted under U.S. FDA IND at 40 sites in the USA, Europe and India •
Slide 15: EAGLE Trial Summary
EAGLE Trial Summary • Statistically significant improvement in PANSS as compared to placebo and equivalent to Risperidone • Statistically significant improvement in secondary efficacy endpoints CGI-S and CGI-C • No significant safety issues vs. placebo • No metabolic side effects • Minimal EPS • Cli
Slide 16: EAGLE Study Efficacy Results
EAGLE Study Efficacy Results • Patients treated with 20-30mg BL-1020 showed statistically significant improvements vs. placebo • Efficacy similar to Risperidone standard of care Endpoint BL-1020 Placebo PANSS -23.6 -14.4 P=0.002 NS vs. Risperidone CGI-S -1.27 -0.68 P<0.001 NS vs. Risperidone Strauss
Slide 17: EAGLE Trial Safety Results
EAGLE Trial Safety Results Parameter BL-1020 Placebo Risperidone (20-30mg) Severe Adverse Events (SAE , 0 6.5 3.3 % patients) Discontinuation due to Adverse 4.5 4.3 8.8 Events (AE , %) ESRS 10.8 1.6 10.8 Metabolic- weight gain ( % 4.9 3.6 7.3 notable gain) Metabolic- cholesterol NC NC NC 17
Slide 18: Brief Assessment of Cognition in Schizophrenia (BACS)
Brief Assessment of Cognition in Schizophrenia (BACS) • The Brief Assessment of Cognition in Schizophrenia (BACS) assesses aspects of cognition found to be most impaired and most strongly correlated with outcome in patients with schizophrenia • Tests include: – List Learning (verbal memory) – Digit
Slide 19: EAGLE Trial Cognition Results
EAGLE Trial Cognition Results BL-1020 treatment leads to clinically relevant and statistically significant improvement in cognitive function Placebo BL-1020 Risperidone 20-30mg BACS (LS mean, 6.01 9.27 6.2 LOCF) P value vs. placebo P=0.027 NS P value vs. P=0.027 Risperidone 19
Slide 20: BL-1020 GABA Enhanced Profile Results in Cognitive Improvements
BL-1020 GABA Enhanced Profile Results in Cognitive Improvements • Cognition is an unmet medical need in schizophrenia, as well as other psychiatric and neurological conditions • In vivo studies show BL-1020 is effective in reversing cognitive impairment induced by PCP by increasing cortical and hipp
Slide 21: BL-1020 Phase 2a Trial Summary
BL-1020 Phase 2a Trial Summary Trial Design Main Findings Phase 2a Open label, 6 week MTD study Safe, efficacious and well 36 chronically ill schizophrenia patients tolerated Escalating doses: Efficacy 20mg on days 1, 2 and 3 Statistically significant 30mg on days 4, 5 and 6 improvement in PANSS and
Slide 22: BL-1020 Phase 1b Trial Summary
BL-1020 Phase 1b Trial Summary Trial Design Result Summary Phase 1b 20 healthy volunteers Direct evidence of Dopamine blocking activity BL-1020 administered at a single No adverse events dose (10-20mg) Receptor occupancy data indicates 15-20mg dose will Equimolar doses of be efficacious
Slide 23: BL-1020 Phase 1a Trial Summary
BL-1020 Phase 1a Trial Summary Trial Design Result Summary Phase 1a 48 healthy adult males Safe and well tolerated Single dose 2-23mg No psychiatric, neurological or ECG Placebo controlled, abnormalities double-blind study BL-1020-tri-maleate salt was used in Phase I studies Doses are expr
Slide 24: Acceptable TPPs Can Be Envisioned: Schizophrenia Pipeline Overview
Based On BLRX Data/Claims To-Date, A Number Of Potentially Acceptable TPP‟s Can Be Envisioned Schizophrenia Pipeline Overview Compounds By Phase Compounds By MOA Dopamine/S Phase III Others erotonin N = 58 16% Antagonist Loxapine (Alexza) 24% Lurasidone GABA (Dainippon Sumitomo) 4% Preregistration D
Slide 25: A Truly Differentiated Profile Can Generate Significant Revenues
A Truly Differentiated Profile Can Generate Significant Revenues, Depending On The Profiles Of Interim Competitive Launches Product Profile Scenarios 1 2 3 4 Adverse Comparable to best in Comparable to best in Comparable to best in Comparable to best in Events class atypical; superior to class atypi
Slide 26: A Truly Differentiated Profile Can Generate Significant Revenues
A Truly Differentiated Profile Can Generate Significant Revenues, Depending On The Profiles Of Interim Competitive Launches Model Output – Peak Year TRx Share Revenues (US-2017) e.g. New products launched prior Cursory Projections to 1020 having enhanced efficacy profiles would indicate peak US sale
Slide 27: BL-1020 Summary
BL-1020 Summary • Positive Phase 2b EAGLE study results • Clinical and pre-clinical studies confirm positioning of highly effective antipsychotic with minimal motor and no metabolic side effects • Pre-clinical studies confirm GABA mechanism of action with potential for improved cognition • Potential
Slide 28: BL-1040: First-In-Class Myocardial Implant
BL-1040: First-In-Class Myocardial Implant for The Treatment of Tissue Damaged by AMI Out-licensed to Ikaria Holdings Inc. 28
Slide 29: BL-1040: First-In-Class Myocardial Implant for Acute MI
BL-1040: First-In-Class Myocardial Implant (Medical Device) for The Treatment of Acute MI • Indication: – Treatment of Acute Myocardial Infarction (AMI) • Efficacy: – Prevention of ventricular remodeling following AMI • Safety – Safe for use without therapeutic side effects • Mode of delivery – Intr
Slide 30: Current Treatment Methodologies and Unmet Medical Need
Current Treatment Methodologies and Unmet Medical Need Vessel occlusion Tissue damage Successfully treated with No current treatment for PCI and stents myocardial damage ? 30
Slide 31: BL-1040: Mechanism and Pathway
BL-1040: Mechanism and Pathway 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 cardi
Slide 32: BL-1040 Deposits in the Damaged Tissue Following Intra-Coronary Injection
BL-1040 Deposits in the Damaged Tissue Following Intra-Coronary Injection Targeting mechanism: 1. Injection occurs in the infarct related artery 2. Capillary bed compromised secondary to ischemia allows inflow of BL-1040 3. Physiological condition of ischemic region enables liquid to gel transition
Slide 33: BL-1040 is a Medical Device
BL-1040 is a Medical Device • BL-1040 is a medical device – FDA review by CDRH – European regulatory bodies concur • Pilot study – Recruitment complete – Final data January 2010 • Single Pivotal/Approval trial to begin in 2010 – Interim analysis as basis for European reimbursement approval – Final d
Slide 34: Pilot Clinical Trial
Pilot Clinical Trial • 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 • 9 sites: 6 in Germany, 3 in Belgium Prelimina
Slide 35: Next In Line
“Next In Line” 35
Slide 36: BL-5010: Novel Formulation for Non-Surgical Removal of Benign Skin Lesions
BL-5010: Novel Formulation for the Non- Surgical Removal of Benign Skin Lesions • Indication: – Seborrheic keratosis; other skin lesions • Efficacy: – Non surgical removal of lesions • Safety – Safe for use without therapeutic side effects – Possibility to attain histological examination • Mode of d
Slide 37: BL-5010 Clinical Trial Update
BL-5010 Clinical Trial Update • Interim results from the first 25 patients show: – All treated skin lesion were completely removed within 30 days of treatment following a single application – BL-5010 is safe and is not associated with any Adverse Events including irritation and inflammation – A pati
Slide 38: BL-5010 Clinical Trial Update
BL-5010 Clinical Trial Update before Day 7 • Patient 2 data: – Lesion was completely removed with no AE‟s (no edema, no swelling or blistering, moderate erythema) – Histological examination shows structure preservation Day 30 38
Slide 39: BL-1021: Orally Available NCE for Neuropathic Pain
BL-1021: Orally Available NCE For the Treatment of Neuropathic Pain • Indication: – Neuropathic pain • Efficacy: – Greater efficacy than current therapies (anticonvulsants, antidepressants) • Safety – Enhanced Safety over current therapies • Developmental status – Advanced pre-clinical – Clinical tr
Slide 40: BL-1021 Efficacy in the Chung Model of Neuropathic Pain
BL-1021 Efficacy in the Chung Model of Neuropathic Pain • An established model of neuropathic pain, in which long lasting thermal hyperalgesia and mechanical allodynia are induced by spinal nerve ligation (SNL) (Kim & Chung, 1992) • 6 treatment arms (n=10): BL-1021, 13.2 mg/kg, po Nortriptyline,
Slide 41: BL-1021 Reduces Mechanical Allodynia in the Chung Model
BL-1021 Reduces Mechanical Allodynia in the Chung Model of Neuropathic Pain 40 Vehicle BL-1021 Fumarate 13.2mg/kg 35 ** ** 30 Threshold (gr) 25 20 15 10 0 1 2 3 4 5 6 7 Time (Hours) **p<0.01 vs. Vehicle
Slide 42: BL-1021 Reduces Thermal Hyperalgesia in the Chung Model
BL-1021 Reduces Thermal Hyperalgesia in the Chung Model of Neuropathic Pain Efficacy Vs. Currently Used Drugs * # 1h post administration *p<0.05 vs. Gabapentin, #p<0.05 vs. vehicle control group 42
Slide 43: RotaRod Test Confirms that BL-1021 is Not Sedative
RotaRod Test Confirms that BL-1021 is Not Sedative • The RotaRod test was performed to test possible sedative effects • SD rats were administered with drugs 4h prior to testing 350 • Experimental groups: – BL-1021 (79.2mg/kg, p.o, higher than the 300 effective level used in the Chung) – Nortriptylin
Slide 44: Company Summary
Company Summary • Clinical stage company with three clinical compounds • Professional drug development company with validated business model – “bench to bedside to partner” • Positive Phase 2b results for BL-1020 with unique differentiating properties • Potential for significant out-licensing event
Slide 45: Thank you for your time.
Thank you for your time. For more information please visit www.BioLineRx.com