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 Diagnos(c and therapeu(c products for neurological condi(ons OTC/QB: AMBS Aegis Capital Healthcare & Technology Conference September 2014 1 Forward-­‐Looking Statements This presentaCon contains “forward-­‐looking statements” within the meaning of the “safe-­‐harbor” provisions of the Private SecuriCes LiCgaCon Reform Act of 1995. Such statements involve known and unknown risks, uncertainCes and other factors that could cause the actual results of the Company to differ materially from the results expressed or implied by such statements, including changes from anCcipated levels of sales, future internaConal, naConal or regional economic and compeCCve condiCons, changes in relaConships with customers, access to capital, difficulCes in developing and markeCng new products and services, markeCng exisCng products and services, customer acceptance of exisCng and new products and services and other factors. Accordingly, although the Company believes that the expectaCons reflected in such forward-­‐looking statements are reasonable, there can be no assurance that such expectaCons will prove to be correct. The Company has no obligaCon to update the forward-­‐looking informaCon contained in this presentaCon. 2 Amarantus Bioscience Holdings: AMBS Amarantus is a biotechnology holding company focused on the transla(onal development of breakthrough science that addresses key unmet medical needs in the diagnos(c and therapeu(c sectors of the neurology space 3 Management Team Gerald E. Commissiong, President & CEO Led acquisiCon of diagnosCc assets and MANF strategic development Stanford University: Management Science & Engineering Robert Farrell, JD, Chief Financial Officer Former CEO and CFO at Titan PharmaceuCcals Former CFO, Fresenius David A. Lowe, PhD, Board of Directors President & CEO at NeuroAssets, Sarl (overseeing MANF development) Former Head of CNS R&D at Roche, NovarCs & Sandoz (now Bayer) CharloDe Keywood, MD, CMO TherapeuKcs Division Former CMO at Addex TherapeuCcs Former Medical Director at Vernalis Colin Bier, PhD, Corporate Advisor President & CEO at ABA Research (overseeing LymPro commercial development) Former Director at Nymox CorporaCon 4 Holdings Structure Amarantus DiagnosCcs Division TherapeuCcs Division PhenoGuard Division 5 Pipeline Asset Pre-­‐
Clinical Phase 1 Phase 2 Phase 3 Commercial LymPro Test®: Alzheimer’s disease (CLIA) Eltoprazine: Parkinson’s disease (Phase 2b) MANF: ReKniKs Pigmentosa Pre-­‐Clinical 6 LymPro Test® for Alzheimer’s: Overview Ø  LymPro Test®: Stage-­‐independent diagnosCc blood test for Alzheimer’s disease Ø  Science: cell cycle dysregulaCon present in Peripheral Blood Lymphocytes (PBLs) ü  Immune dysfuncCon common in brain and blood Ø  2 peer-­‐reviewed studies published to date (2001 and 2012); Ø  C L I A C o m m e r c i a l i z a C o n a s a L a b o r a t o r y D e v e l o p e d T e s t Icon Central Laboratories: 4Q/14 Ø  Worldwide potenCal market opportunity esCmated at $3B Ø  IniCal Target Market: Alzheimer’s TherapeuCc Clinical Trials -­‐ $150M Ø  Secondary potenCal applicaCons in TBI and CTE 7 Clinical Results for LymPro Test® ü  CD69 cell surface marker measures response in different lymphocyte sub-­‐populaCons v  CD19+ v  CD4+ AD=45 HC=27 SKmulaKon Index (= CD69-­‐sCm/CD69-­‐unsCm) Ø  Alzheimer’s subjects have impaired immune response to mitogenic sCmulaCon CD4+ CD19+ ü  CD69 expression downregulated in AD subjects ü  2 independent peer-­‐reviewed publicaCons v  SCeler, JT et, Neuroreport 2001; 12(18):3969-­‐3972 v  Steiler J et al, Neurobio Aging 2012 33: 234-­‐341 Amarantus-­‐NIO Sept. 2013 8 LP-­‐002 Study Design Primary Study ObjecKve: Replicate published work that shows separaCon of Alzheimer’s disease from Healthy Controls with LymPro (Version 1) and evaluate new condiCons that may improve separaCon (Version 2) Ø Enrollment: 72 paCent – 36 Alzhimer’s and 36 healthy controls Ø Entry criteria: ü Alzheimer’s = MMSE ≤ 22 (moderate to severe) ü Healthy Control = MMSE ≥ 29 Ø Exclusion criteria: Ø Autoimmune disorders Ø Immune medicaCons 9 Interim Results: Single Marker ROC Plot
it 0.7
r 0.5
r 0.3
No discrimination
AUC: 80%
%CD19+69+ (as a % of CD19+)
%CD19+69+ (as a % of
False positive rate (1-Specificity)
95% CI
0.628 to 0.966
10 Interim Data: 2 Marker Score Scatterplot 11 LymPro: SigniKicant Opportunity Huge Unmet Need Ø  1 in 9 Americans over 65 has AD Ø  5.2 million Americans have AD Ø  500,000 new diagnoses per year Ø  Severely misdiagnosed Ø  $400B costs in the U.S. health system ü  10% of healthcare budget Ø  Growing rapidly with aging populaCon Ø  Key Unmet need: effecCve early diagnosCc 12 Patient Selection for AD Trials Ø  Pre-­‐screening of paCents to reduce pharma paCent acquisiCon cost 30-­‐40% Ø  Data generated will add validity to test while generaCng revenue Ø  Mechanism is fundamental disease biology: Emerging TherapeuCc Target “The scien(fic community and the FDA believe that it is criKcal to idenKfy and study paKents with very early Alzheimer's disease before there is too much irreversible injury to the brain…It is in this popula(on that most researchers believe that new drugs have the best chance of providing meaningful benefit to pa(ents.” Ø  Russell Katz, MD, Director of the Division of Neurology Products in the FDA's Center for Drug EvaluaCon and Research 13 LymPro Takeaways Key Takeaways Ø  LymPro is most advanced diagnosCc blood test for cell cycle dysregulaCon Ø  Clinical data published in 2 peer-­‐reviewed journals Ø  CLIA distribuCon channel to support product launch at Icon Central Labs Ø  IniCal target market: $150M Research Use Only market for pharma trials in AD Ø  Total Market opportunity esCmated at $3B Upcoming Milestones Ø  LP-­‐002 Data Ø  Assay ValidaCon at Icon Ø  CLIA Submission Ø  Commercial Launch 14 Eltoprazine for Parkinson’s Overview Ø Originally developed by Solvay (now Abbvie) Ø Parkinson’s key unmet need: Levadopa Induced Dyskinesia (“LID”) ü  Side effect of L-­‐Dopa treatment 5-­‐7 years post-­‐diagnosis ü  60-­‐80% of PD paCents Ø Eltoprazine TherapeuCc IndicaCons: ü  Parkinson’s disease Levadopa induced dyskinesia (PD LID) ü  Adult ADHD ü  Schizophrenia 15 Eltoprazine: Positive Phase 2a LID Data Efficacy Data Ø  22 paCents (3 dosing regimens, 5mg most effecCve) Ø  Primary endpoint was 25% reducCon of LID as measured by Clinical Dyskinesia RaCng Scale (CDRS): p=0.0007 Ø  No interference observed with L-­‐Dopa effect as measured by UPDRS Ø  8-­‐week study (baseline, 1x/week for 6 weeks, final visit) Ø  0 SAEs (30% Treatment-­‐Emergent AEs) Confidential
16 Eltoprazine: Strong Safety ProKile 700 human paKents treated in Phase 1 and Phase 2 trials ü  Dosed at up to 6x therapeuCc dosing levels ü  6 month repeat toxicity in rat and dog ü  Genotoxicity, reproducCve and development toxicity studies ü  No significant safety pharmacology or toxicology findings in human ü  No effect on hERG, QT or cardiovascular acCvity in animals or humans ü  Plasma half-­‐life: ~8 hrs – good oral bioavailability ü  No CYP inhibiCon / liyle CYP metabolism ü  Low binding to plasma proteins (<15%) 17 Eltoprazine: PD LID Market Opportunity Ø  1.5M Americans have PD; 60-­‐80% get LID Ø  60,000 new diagnosis annually 3,500
Ø  3M million by 2032 3,000
Ø  Total cost to U.S.: $25B 2,500
Ø  Key Unmet need: treaCng dyskinesia 2,000
Ø  FDA approval Cmeline 1,500
v 2015: Phase 2b 1,000
v 2016-­‐2018: 2 parallel Phase 3s 500
v 2019: Submission/Approval 0
Parkinson's disease-specific sales ($m)
Ø  PotenCal sales worldwide: $1B+ Ø  Patent protecCon through 2031 Mexico
Rest of the world
All other EU countries
18 Eltoprazine: Positive Phase 2a ADHD Data Non-­‐sKmulant, non-­‐scheduled Ø  Primary endpoint as measured by change from baseline vs. placebo in ADHD-­‐RS-­‐IV score in 5mg (p=0.003) ü  For the InayenCon subscale, both 5mg staCsCcally significant benefit over placebo (0.003) ü  For the HyperacCvity subscale, the 5mg dose showed a staCsCcally significant benefit (p=0.008); ü  CGI-­‐I scores (p=0.023) Ø  Secondary endpoints also met ü  Profile of Mood States (POMS) (p=0.006) ü  Anger-­‐hosClity score (p<0.001) ü  Tension anxiety score (p=0.046) ü  Barnes Akathisia Scale (BAS) (p=0.029 ) ü  Awareness of Restlessness subscore (p=0.003 ) Ø  Data Compares favorably with currently approved sCmulants Confidential
19 MANF Overview Development Programs Ø ReCniCs Pigmentosa (Orphan) Ø Parkinson’s disease AddiKonal ApplicaKons Ø Orphan Ocular Diseases Ø Diabetes Ø Ischemic Heart Disease Mechanism of AcKon Ø Protein Misfolding / ER Stress Ø Calcium Channel regulaCon Strong IP PosiKon Ø Comp of Mayer / Method IP Ø Licenses from UniversiCes Lindholm and Saarma,, DevNeurobiol (2010)
20 Retinitis Pigmentosa (RP) About RP Ø  GeneCc disease: starts late teens -­‐ blind by age 40 Ø  ER Stress mechanism Ø  100k paCents in the US: Orphan Disease Ø  No treatments currently approved; NTFs in dev. Ø  $1B+ market opportunity MANF Data Ø  Significantly protected rods and cones in s334ter Line 3 model Ø  PosiCve Toxicology Data Upcoming Milestones Ø  Orphan Drug DesignaCon ApplicaCon Ø  IniCaCon of GMP Manufacturing 21 Parkinson’s Disease Disease biology Ø Neurotrophic factors are believed to be among the most promising potenCal disease modifying therapeuCc targets. Ø  MANF has unique properCes that could reverse clinical course of Parkinson’s MANF Data Ø Dopaminergic re-­‐innervaCon of striatum •  Increased dopamine concentraCons •  Reduced behavioral deficits Upcoming milestone Ø Drug delivery data w/Renishaw 22 MANF Key Takeaways KEY TAKEAWAYS Ø 
Disease-­‐modifying treatment for ReCniCs Pigmentosa (orphan) Disease-­‐modifying treatment for Parkinson’s disease PotenCal in other indicaCons (orphan ocular, diabetes, heart disease Unique Biology with significant untapped potenCal $1B+ product potenCal in RP, $1B+ Parkinson’s; $5B-­‐$10B Total Upcoming Milestones Ø  Orphan Drug DesignaCon ApplicaCon Ø  Pharmacology Data in Orphan Ocular Disease Ø  Drug Delivery Data in Parkinson’s disease 23 Financial & Trading Overview Trading on the Over the Counter (OTCQB) Ø  $2.4M on hand (as of 8/25/14) Ø  $18M equity line w/ Lincoln Park Capital + $5M in callable warrants Ø  751million common shares outstanding (as of 8/15/2014) Ø  0.11/share trading on the OTCQB Ø  $80 million market cap (8/15/2014) Ø  Seeking to uplist to NASDAQ in Q4/2014 24 Investment Summary LymPro Test ® Alzheimer’s disease blood test Ø  2 Peer reviewed publicaCons Ø  Ongoing LP-­‐002 clinical study Ø  CLIA launch at Icon Central Laboratories in process Eltoprazine: Phase 2b-­‐ready small molecule for PD LID Ø  PosiCve Data in Phase 2a trial in PD LID Ø  Over 700 paCents dosed to date, with strong safety profile Ø  Phase 2b iniCaCon in process MANF unique biology for muKple indicaKons Ø  PosiCve Data in ReCniCs Pigmentosa (Orphan) Ø  Orphan Drug DesignaCon applicaCon in process 25 
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