Ab Science Sa · EURONEXT:AB
Ab Science Sa — Investor Presentation (September 2020)
35 slides from Ab Science Sa's investor presentation, September 2020
Slide 1: Corporate Presentation
Corporate Presentation September 2020
Slide 2: Disclaimer
Disclaimer This presentation, together with the material set forth herein, does not constitute an offer of securities for sale nor the solicitation of an offer to purchase securities in any jurisdiction. Distribution of such presentation in certain jurisdiction may constitute a breach of applicable
Slide 3: Investment Highlights
Investment Highlights Portfolio based on Unique Mechanism of Action with Multiple Late-Stage Programs • Four positive Phase 2B/3 read outs, in amyotrophic lateral sclerosis (ALS), progressive forms of Late Stage Pipeline multiple sclerosis, mastocytosis (ISM), and in Severe Asthma uncontrolled by or
Slide 4: Investment Highlights
Investment Highlights Diversified portfolio with four positive and four pending read-outs from phase 2B/3 trials Positive Results Read-outs to come Reported Masitinib Masitinib (Multiple Sclerosis PPMS (Alzheimer) & non-active SPMS) Masitinib Masitinib (Undisclosed Kinase) (Severe Asthma (Severe Ast
Slide 5: Market Potential
Market Potential Blockbuster potential but also addressing orphan diseases Estimated Number of patients (USA and EU) in targeted indications with masitinib Alzheimer's disease 5 000 000 5 000 000 Severe Asthma 825 000 Blockbuster potential Multiple Sclerosis (Primary & non-active secondary 500 000 p
Slide 6: Critical Role of Mast Cells & Macrophages/Microglia
Critical Role of Mast Cells & Macrophages/Microglia Mast cells and glia contribute to neuro-inflammation, which is strongly influenced by their potential for mutual interaction and exacerbation of pathology Activation of mast cells leads to degranulation and secretion of numerous mediators that are
Slide 7: Masitinib Profile and Mechanism of Action
Masitinib Profile and Mechanism of Action Orally-administered kinase inhibitor selectively targeting mast cells and macrophages Masitinib targets mast cells Kinase inhibition profile of masitinib • Masitinib is a potent and selective inhibitor of c-Kit, Lyn, and Fyn kinases. These kinases play criti
Slide 8: Masitinib Safety Database Across Indications
Masitinib Safety Database Across Indications Well established safety profile with long-term exposure Safety Patients exposed for at least population All ≥ 6 months ≥ 12 months ≥ 2 years ≥ 5 years Healthy Volunteers subjects 114 0 0 0 0 Non Oncology subjects 3,317 2,120 1,515 662 50 Oncology subjects
Slide 9: Experienced Management Team
Experienced Management Team ALAIN MOUSSY CHRISTIAN FASSOTTE OLIVIER HERMINE, MD, PHD LAURENT GUY Co-founder and CEO Global Chief Medical Officer Chief Financial Officer Chairman of Scientific Committee Former strategic consultant at Booz, Allen Former positions in the banking industry Medical Doctor
Slide 10: Neurology
Multiple Sclerosis (PPMS and nSPMS) Neurology Amyotrophic Lateral Sclerosis (ALS) Alzheimer's Disease (AD)
Slide 11: Multiple Sclerosis
Multiple Sclerosis Tremendous unmet need, with no approved drugs for non-active SPMS and only one for PPMS Masitinib Positioning Label Manufacturer First approved Non-active Primary Active Secondary Relapsing Secondary Progressive MS Progressive MS Remitting MS Progressive MS* Distribution of patien
Slide 12: Multiple Sclerosis
Multiple Sclerosis Phase 2B/3 demonstrated significant benefit on disability progression Significant reduction in progression on EDSS (Primary Endpoint) 42% risk reduction of time to disability progression LSM of EDSS Change from Baseline - LOCF/OC by Visit (M4.5 vs P) (mITT) 03FEB2020 0.2 P=0.0256
Slide 13: ALS
ALS Phase 2B/3 demonstrated significant delay in disease progression (Database locked on 16/03/2017) (Database locked on 16/03/2017) AB Science - AB10015 ALS PHASE 2/3 AB Science - AB10015 ALS PHASE 2/3 27% slowing of functional deterioration (Primary Endpoint) -- Edited on 03APR17 25% delay in dise
Slide 14: ALS
ALS Clinical benefit further enhanced when treatment initiated early 51% slowing of functional deterioration (vs 27%) Significant improvement on Combined Assessment of Function & Survival % reduction in change in ALSFRS (primary endpoint) At least 1 subgroup Diff. of means p-value Primary Analysis S
Slide 15: ALS
ALS Next Step Confirmatory phase 3 study, with design optimized based on first phase 3 study Confirmatory phase 3 design Optimizations from previous phase 3 Design: Double blind, placebo controlled, randomized 1:1:1:1, Enriched inclusion criteria comparing masitinib titration up to 6.0 mg/kg/day wit
Slide 16: Alzheimer's disease
Alzheimer’s disease On-going phase 2B/3 with top-line results expected in Q3 2020 Activity on Cognition established in proof of concept study On-going phase 2B/3 design Change in ADAS-Cog -6 ADAS-Cog Masitinib Placebo Design: Double blind, placebo controlled, comparing masitinib 4.5 mg/kg/day vs. pl
Slide 17: Neurology
Neurology Blockbuster Opportunity across three indications Annual cost of Market Market Phase of No No Indication registered Drugs Penetration Opportunity US Opportunity EU Development US Patients EU Patients (USD) (M€) (M€) Positive phase 2/3 study 800 1,200 ALS Launch of confirmatory • Radicava (1
Slide 18: Immunology
Indolent Systemic Mastocytosis (ISM) Immunology Severe Asthma
Slide 19: Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis Inhibition of mast cells, regardless of c-Kit mutation status, through c-Kit, Lyn and Fyn kinases The c-Kit receptor is primarily responsible for mast cell growth, differentiation and survival with mast cell mediator release being initiated through the integration of d
Slide 20: Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis Effectiveness regardless of c-Kit mutation status confirmed in phase 2 and sustainable Improvement in disease symptoms Reduction in urticaria pigmentosa Sustained efficacy % Change from baseline at week 12 % of patients still under treatment Flush (per day) Pruritus De
Slide 21: Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis Phase 3 demonstrates significant reduction in symptoms 3.6 fold improvement in most prevalent symptoms Improvement in objective markers of the disease Masitinib Placebo p-value Odds ratio Masitinib Placebo p-value 4H75% Primary Tryptase - Patients with baseline tryptas
Slide 22: Indolent Systemic Mastocytosis
Indolent Systemic Mastocytosis Next Step Confirmatory phase 3 study, with design optimized based on first phase 3 study Confirmatory phase 3 design Optimizations from previous phase 3 Design: Double blind, placebo controlled, randomized 1:1, Dose Titration comparing masitinib titration up to 6.0 mg/
Slide 23: Severe Asthma Uncontrolled with Oral Corticosteroids
Severe Asthma Uncontrolled with Oral Corticosteroïds Significant decreases in asthma exacerbations regardless of eosinophil level Annualized severe asthma exacerbation rate Greater benefit in patients with higher cumulative OCS Primary Analysis Sequential Analysis Sensitivity Analysis Sensitivity An
Slide 24: Immunology
Immunology Strong market potential across two indications Market Market Opportunity Phase of Annual cost of registed No No Indication Penetration Opportunity US EU Development Drugs (USD) US Patients EU Patients (M€) (M€) Launch of 500 800 ISM confirmatory No registered drug 25,0004 40,0004 50% (bas
Slide 25: Oncology
First Line Metastatic Castrate Resistant Prostate Cancer (mCRPC) Oncology First Line Non Resectable Locally Advanced or Metastatic Pancreatic Cancer Acute Myeloid Leukemia (relapsed/refractory or unfit for intensive chemotherapy)
Slide 26: Pancreatic cancer and Prostate Cancer
Pancreatic cancer and Prostate Cancer Masitinib has no direct “tumor killer” general activity but has shown efficacy on tumor proliferation in vivo, mediated through the tumor micro-environment No direct effect on tumor cells Two on-going phase 3 combining masitinib …but decreases tumor volume growt
Slide 27: Acute Myeloid Leukemia
Acute Myeloid Leukemia AB8939, Novel Microtubule-destabilizing Agent for Acute Myeloid Leukemia Key Differentiating factors Activity in AMKL26 PDX model Activity in Ara-C resistant/refractory AML • Overcomes P-glycoprotein (Pgp) and myeloperoxidase (MPO) mediated Control resistance • Active in Ara-C
Slide 28: Oncology
Oncology Strong market potential across three indications Annual cost of Market Market Opportunity Phase of No No Indication registered Drugs Penetration Opportunity US EU Development US Patients EU Patients (USD) (M€) (M€) • Xtandi (90,000) Positive phase 2/3 • Jevtana (48,000) 1,000 1,500 Prostate
Slide 29: Next Steps & Financial Information
Next Steps & Financial Information
Slide 30: Upcoming newsflow
Upcoming newsflow Important catalysts are expected through H2 2020 • Report top-line results from phase 2B/3 with masitinib in Alzheimer Disease (n=720) • Report top-line results from phase 3 with • Report top-line results from phase 3 with masitinib + docetaxel in metastatic prostate masitinib in H
Slide 31: Financial Information
Financial Information • Euronext Paris: AB.PA • Market Cap (€): ≈ 550M • Shares Outstanding: 44.1M • Debt (€): None* • Cash position (€): 5.7M (as of December 31, 2019) • Raised EUR 12.3M in March 2020 * Non-current liabilities amount to €25,043,000 as of 31 December 2019 and comprise the following
Slide 32: Back-up
Back-up
Slide 33: Masitinib - Intellectual Property
Masitinib - Intellectual Property Masitinib IP rights are secured until 2037 in ALS and between 2031 and 2036 in other indications Protection Item Duration of protection Status Patent on composition of matter Patent on composition of matter has been filed and delivered. It will be further extended
Slide 34: Masitinib - Publications
Masitinib - Publications Neurology Program Data Publications • Trias et al, 2020 : Schwann Cells Orchestrate Peripheral Nerve Inflammation Through the Expression of CSF1, IL-34, and SCF in Amyotrophic Lateral Sclerosis • Trias et al, 2018 : Mast cells and neutrophils mediate peripheral motor pathway
Slide 35: Masitinib - Publications
Masitinib - Publications Immunology & Oncology Program Data Publications • Humber et al, 2010 : Masitinib combined with standard gemcitabine chemotherapy: in vitro and in vivo studies in human pancreatic Preclinical tumour cell lines and ectopic mouse model Pancreatic Cancer Phase 2 • Mitry et al, 2