Ab Science Sa · EURONEXT:AB

Ab Science Sa — Investor Presentation (January 2020)

39 slides from Ab Science Sa's investor presentation, January 2020

  1. Slide 1: AB Science Corporate Presentation January 2020

    AB SCIENCE CORPORATE PRESENTATION January 2020

  2. 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

  3. Slide 3: Overview of AB Science

    Overview of AB Science AB Science has two lead compounds. Both compounds are 100% owned by AB Science.  Selective kinase inhibitor  Targeting mast cells and macrophages/microglia MASITINIB  Developed in neurology, oncology and inflammatory diseases  Three positive Phase 3, in amyotrophic lateral

  4. Slide 4: Management team and scientific committee

    Overview of AB Science Management team and scientific committee Alain MOUSSY, MBA Co-founder and Chief Executive Officer. Laurent Guy, MBA Chief Financial Officer Christian FASSOTTE, MD Global Chief Medical Officer Shailja DIXIT, MD US Head of Medical & Regulatory Affairs Oliver HERMINE, MD, PhD Pro

  5. Slide 5: Overview of 2019

    Overview of 2019 AB Science reported important positive news event in 2019 that have contributed in the significant appreciating in share price and trading volumes. Newsflew, share price and liquidity since 2019 January 8: FDA DAILY TRADING VALUE (€) May 28: ANSM August 19: 10M€ authorization for SH

  6. Slide 6: Upcoming milestones

    Upcoming milestones Important events are expected each quarter, including 4 key milestones in Q1 2020. CURRENT STATUS NEXT NEWS EVENTS Compound Program Program/Process Enrolment Q1 2020 Q2 2020 Q3 2020 Q4 2020 Amyotrophic Lateral 1) Initiation 394 Sclerosis (ALS) confirmatory study Completed Severe

  7. Slide 7: Interim analyses

    Interim analyses On-going Phase 3 programs are de-risked based on interim analyses that passed the conditional power ≥ 80% test based on 50% of patients or events (75% of patients in Alzheimer’s disease).  Interim analysis features : Tests futility and conditional power ≥80% (probability of success

  8. Slide 8: Masitinib

    MASITINIB

  9. Slide 9: Masitinib profile

    Masitinib profile Masitinib is an orally-administered selective kinase inhibitor that targets mast cells and macrophages/microglia.  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

  10. Slide 10: Indications with positive phase 2/3 results

    Indications with positive phase 2/3 results  Amyotrophic Lateral Sclerosis (ALS)  Indolent Systemic Mastocytosis (ISM)  Severe Asthma Uncontrolled by Oral Corticosteroids

  11. Slide 11: ALS – Phase 2/3 design

    ALS – Phase 2/3 design The first Phase 2/3 study (AB10015) has been completed. ❖ Double blind, placebo controlled, randomized 1:1:1, oral BID, 48w  Masitinib 4.5 mg/kg/day + riluzole  Masitinib 3.0 mg/kg/day + riluzole  Control : Placebo + riluzole ❖ Main Inclusion Criteria  Disease duration  3

  12. Slide 12: ALS – Phase 2/3 main efficacy results

    ALS – Phase 2/3 main efficacy results Masitinib (4.5 mg/kg/day) demonstrated a significant benefit in ALS patients with a baseline ALSFRS-R progression of <1.1 point/month, which was the population for primary analysis. 27% slowing of functional deterioration; P=0.0158 25% delay in disease progressi

  13. Slide 13: ALS – Analysis of subgroup selected for Phase 3 confirmatory study

    ALS – Analysis of subgroup selected for Phase 3 confirmatory study Subgroup analysis of Phase 2/3 AB10015 confirmed that patients with less severe disease at the start of treatment are those likely to benefit most from masitinib. Primary Analysis: Change from baseline to week 48 in ALSFRS-R AB10015

  14. Slide 14: ALS – Optimization of confirmatory study design

    ALS – Optimization of confirmatory study design The design of the Phase 3 confirmatory study has been optimized to increase the probability of success and has been validated by EMA through protocol assistance. DESIGN OF CONFIRMATORY PHASE 3 OPTIMIZATIONS FROM PREVIOUS PHASE 2/3 ❖ Double blind, place

  15. Slide 15: ALS – Phase 3 confirmatory study timelines

    ALS – Phase 3 confirmatory study timelines The confirmatory study is expected to be completed in Q1 2022.  Status Europe  Design reviewed through scientific advice plus input from registration dossier  Protocol harmonization on-going across European countries  Status USA  Design reviewed throug

  16. Slide 16: ISM – Clinical Development

    ISM – Clinical Development The planned indication for masitinib is indolent or smouldering systemic mastocytosis, which are the most prevalent forms of mastocytosis. Disease according to WHO % Masitinib Category Characteristics Symptoms classification patients scope Cutaneous mastocytosis (CM) Prima

  17. Slide 17: ISM – Phase 3 design

    ISM – Phase 3 design The first Phase 3 study (AB06006) evaluated masitinib versus placebo in 135 patients with ISM and severe symptoms at baseline.  Study Design :  Blinded, placebo controlled, masitinib versus placebo,  Randomization 1:1  Masitinib 6 mg/kg/day  135 patients with ISM  To be ev

  18. Slide 18: ISM – Phase 3 main efficacy results

    ISM – Phase 3 main efficacy results The Phase 3 study in ISM met its primary analysis and was published in The Lancet.  The primary analysis measuring 75% reduction in symptoms between baseline and 6 months time point was positive. Odds ratio Masitinib Placebo Diff. p-value (CI95%) 4H75% Primary Cu

  19. Slide 19: ISM – Optimization of confirmatory study design

    ISM – Optimization of confirmatory study design The design of the phase 3 confirmatory study benefits from the two scientific advice from EMA on the first phase 3 and from the assessment by EMA of the results of the first phase 3. DESIGN OF CONFIRMATORY PHASE 3 OPTIMIZATIONS FROM PREVIOUS PHASE 2/3

  20. Slide 20: ISM – Phase 3 confirmatory study timelines

    ISM – Phase 3 confirmatory study timelines The confirmatory study is expected to be completed in early 2022.  Status Europe  Design reviewed through 2 scientific discussions plus input from registration dossier  Protocol approved in France (ANSM) and other European countries  Status USA  Design

  21. Slide 21: ISM – Masitinib positioning

    ISM – Masitinib positioning Masitinib is the most advanced compound in ISM and its safety profile is suitable for lifelong treatment.  Mariana Castells (Brigham and Women’s Hospital Mastocytosis Center in Boston) “Masitinib has a benefit and toxicity profile which sets it apart from other current t

  22. Slide 22: Asthma - Phase 2/3 study design

    Asthma - Phase 2/3 study design Study AB07015 evaluated masitinib 6.0 mg/kg/day in patients with severe asthma uncontrolled by oral corticosteroids. ❖ Double blind, placebo controlled, randomized 2:1 ❖ Main Inclusion Criteria  Oral corticosteroids (OCS) dose ≥ 7.5 mg daily for at least 3 months pri

  23. Slide 23: Asthma - Phase 2/3 study disposition of patients

    Asthma - Phase 2/3 study disposition of patients The Primary analysis was pre-specified in the claim 1 (severe asthma population). A sequential analysis was pre-specified for the claim 2 (severe asthma and high eosinophils). ITT and FAS populations were analyzed for sensitivity. ITT population N=419

  24. Slide 24: Asthma - Phase 2/3 study efficacy

    Asthma - Phase 2/3 study efficacy The pre-specified primary analysis in the severe asthma population demonstrated a statistically significant reduction in severe asthma exacerbations. The sensitivity analyses were also statistically significant, indicating that the results are consistent and robust.

  25. Slide 25: Asthma - Phase 2/3 study efficacy

    Asthma - Phase 2/3 study efficacy There was a center effect, with greater efficacy in the EU countries (-51% reduction in severe asthma exacerbations, p=0.0038). AB07015 - Severe Exacerbation Rate – Sensitivity analyses in EU countries - Claim 1 (Severe Asthma) Average Exposure Yearly Exacerbation (

  26. Slide 26: Asthma - Phase 2/3 study efficacy

    Asthma - Phase 2/3 study efficacy The pre-specified analysis in the severe asthma with high eosinophils (≥150K/uL) population (claim 2) also demonstrated a statistically significant reduction in severe asthma exacerbations. AB07015 - Severe Exacerbation Rate – Claim 2 (Severe Asthma with high eosino

  27. Slide 27: Asthma - Phase 2/3 study safety

    Asthma - Phase 2/3 study safety The study demonstrated an acceptable safety profile for the targeted population. AB07015 - Overall Summary of Adverse Events (AEs) - Safety Population Masitinib Placebo (N = 271) (N = 133) n (%) n (%) At least one AE 226 ( 83.4) 109 ( 82.0) At least one serious AE (no

  28. Slide 28: Asthma - Positioning

    Asthma - Positioning Masitinib demonstrated efficacy in a difficult-to-treat population and can be complementary to biologics strategies.  Lavinia Davidescu (University of Oradea) “Masitinib generated positive results with a favorable safety profile”  Pascal Chanez (Marseille University ) “The cli

  29. Slide 29: Asthma - Positioning

    Asthma - Positioning Masitinib has several strategic positioning opportunities in severe asthma that are complementary to biologics strategies. Eosinophil Options Masitinib Positioning Count (cells/μL) In single agent >300 After biologics Severe asthma insufficiently controlled by biologics in combi

  30. Slide 30: Indications with phase 2/3 results in Q1 2020

    Indications with phase 2/3 results in Q1 2020  Multiple sclerosis (primary and secondary progressive)  Alzheimer’s disease

  31. Slide 31: PP&SP MS

    PP&SP MS In multiple sclerosis, the high medical need is in primary progressive (PPMS) and non- active secondary progressive forms (non-active SPMS), with one drug registered in PPMS and no drug registered in non-active SPMS. Label First Manufacturer Non-active Active PPMS RRMS approved SPMS* SPMS**

  32. Slide 32: PP&SP MS – Phase 2/3 design

    PP&SP MS – Phase 2/3 design The Phase 2/3 study (AB07002) evaluation masitinib in PPMS and non-active SPMS, and read out is expected early 2020. ❖ Double blind, placebo controlled  Masitinib 4.5 mg/kg/day vs. placebo (randomization 2:1)  Masitinib titration up to 6.0 mg/kg/day vs. placebo (randomi

  33. Slide 33: AD – Phase 2a main efficacy results

    AD – Phase 2a main efficacy results In a proof-of-concept study, masitinib activity on cognitive functions (ADAS-Cog) was established. Efficacy results in mild-to-moderate Alzheimer's disease (Phase II ; n=34 patients) ADAS-Cog Masitinib Placebo ADAS-ADL Masitinib Placebo -6 12 worsening - improveme

  34. Slide 34: AD – Phase 2/3 design

    AD – Phase 2/3 design The Phase 2/3 study (AB09004) read-out is expected in Q1 2020. ❖ Double blind, placebo controlled  Masitinib 4.5 mg/kg/day vs. placebo (randomization 1:1)  Masitinib titration up to 6.0 mg/kg/day vs. placebo (randomization 2:1) ❖ Main Inclusion Criteria  Dementia of Alzheime

  35. Slide 35: IP status, Potential and competitive landscape

    IP status Potential and competitive landscape

  36. Slide 36: Masitinib - Intellectual Property

    Masitinib - Intellectual Property Masitinib IP rights are secured until 2037 in ALS and and between 2031 and 2036 in other indications. Protection Item Duration of protection Status  Patent on composition of matter has been filed and Patent on composition of matter delivered. It will be further ext

  37. Slide 37: Masitinib potential and competitive landscape

    Masitinib potential and competitive landscape Masitinib is targeting large, relatively uncompetitive markets, except in asthma. Number of patients targeted by masitinib Registered masitinib competitors Indication (US treatment price in $) Europe USA • No approved drug as an add-on to Alzheimer Disea

  38. Slide 38: List of masitinib publications and key press releases (1/2)

    List of masitinib publications and key press releases (1/2) Masitinib clinical program resulted in many publications. Program Data Publications / Press release • Publication : Trias et al, 2018 : Mast cells and neutrophils mediate peripheral motor pathway degeneration in ALS • Publication : Trias et

  39. Slide 39: List of masitinib publications and key press releases (2/2)

    List of masitinib publications and key press releases (2/2) Masitinib clinical program resulted in many publications. Program Data Publications & Press release Prostate • Press release, June 2018 : IDMC recommendation to continue the masitinib phase 3 study based on interim Phase 3 Cancer analysis •

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