Ab Science Sa · EURONEXT:AB
Ab Science Sa — Investor Presentation (July 2024)
46 slides from Ab Science Sa's investor presentation, July 2024
Slide 1: Corporate Presentation
Corporate Presentation July 2024
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: Overview
Overview Management Team Stock Information ALAIN MOUSSY OLIVIER HERMINE, Co-founder and MD, PhD ▪ Listed on Euronext Compartment B CEO Co-founder and Chairman of ▪ ISIN : FR0010557264 Former strategic consultant at Booz, Scientific Allen & Hamilton and former Head of Committee ▪ Tickers : AB.PA (Reu
Slide 4: Core Pipeline
Core Pipeline AB Science has two platforms, with masitinib in phase 3, primarily centered around neuro-degenerative diseases, and with microtubule destabilizer agents (MDAs) in haemato-oncology Therapeutic Phase Phase Confirmatory Platform Drug Indication Preclinical Phase 2B area 1 2 Phase 3 Amyotr
Slide 5: Next expected Milestones
Next expected Milestones Partnership of masitinib platform with a pharmaceutical company is expected in 2024 Scope: ▪ Indications : from single indication to all indications All options are considered ▪ Geography : from single country to global Process: ▪ Top advisors (bankers, consultants) hired to
Slide 6: Masitinib Platform
MASITINIB PLATEFORM
Slide 7: Masitinib Platform – Drug Profile
Masitinib Platform – Drug Profile 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 critical role
Slide 8: Masitinib Platform – Validated MoA based on Modulation of the Neuroimmune System
Masitinib Platform – Validated MoA based on Modulation of the Neuroimmune System Innmune cells (microglia and mast cells) are at the cutting edge of research regarding the pathophysiological mechanisms of NDDs, with a consensus that drugs aimed at these targets will have strong therapeutic potential
Slide 9: Masitinib Platform – Validated MoA based on Modulation of the Neuroimmune System
Masitinib Platform – Validated MoA based on Modulation of the Neuroimmune System Masitinib has demonstrated neuroprotective benefits in three challenging NDDs, showing that targeting microglia and mast cells is a valid strategy across a broad range of indications, and that masitinib’s dual-targeting
Slide 10: Masitinib in Amyotrophic Lateral Sclerosis
Masitinib in Amyotrophic Lateral Sclerosis
Slide 11: ALS - Status & Positioning
ALS - Status & Positioning In ALS, no drug except riluzole has generated a consensus based on definitive evidence of efficacy, there is a need for combination of treatments due to the multifactorial nature of the disease MA Holder / Heath Canada Current clinical Drug Mechanism of action FDA Status E
Slide 12: Multiple Sclerosis - Pharmacology
Multiple Sclerosis - Pharmacology Masitinib has demonstrated ability to lower blood levels of neurofilament light (NfL) in a neurodegenerative disease model (EAE model) Day 8 Day 15 Relative quantification of NFL in serum pools NFL quantification in serum pool from EAE induced mice at Day 15 from EA
Slide 13: Masitinib clinical development plan in ALS
Masitinib clinical development plan in ALS The development program in ALS consists of AB10015 phase 2B/3 study (published) and AB19001 phase 3 confirmatory study Primary Patient Related Phase Study code Design Dose Tested endpoint target publications Prospective, double-blind, ▪ Masitinib 3.0 mg/kg/
Slide 14: Phase 2B/3 – Design
Phase 2B/3 – Design Study AB10015 had broad inclusion criteria and evaluated masitinib after 48 weeks of treatment Design Statistical analysis Design: Two distinct populations were differentiated: Double blind, placebo controlled, 2-parallel groups ▪ ‘Normal Progressors’: rate <1.1 points/month 84%
Slide 15: Phase 2B/3 – Primary analysis
Phase 2B/3 – Primary analysis Masitinib (4.5) demonstrated a significant benefit in patients defined as Normal Progressors (ALSFRS-R progression of <1.1 point/ month), which was the population for primary analysis Normal Progressor (Primary Efficacy Population): 27% slowing of functional deteriorati
Slide 16: Phase 2B/3 – Secondary endpoint
Phase 2B/3 – Secondary endpoint The primary endpoint analyses were supported by multiple key secondary endpoints Masitinib 4.5 mg/kg/day – Normal Progressors Significant ALSAQ40 benefit based on the most Analysis Difference, [95% CI] p-value conservative imputation methods ALSAQ-40 Multiple Imputati
Slide 17: Phase 2B/3 – Secondary endpoint – Overall Survival
Phase 2B/3 – Secondary endpoint – Overall Survival There was a trend of + 6 month increase in median overall survival Analysis of Overall survival (placebo switch vs no switch to masitinib) OS analysis was performed, censoring placebo patients at the time they switched to masitinib treatment. Median
Slide 18: Phase 2B/3 – Subgroup Analysis prior to complete loss of function
Phase 2B/3 – Subgroup Analysis “ALS prior to complete loss of function in any individual component of the ALSFRS-R There was a disbalance in a subset of the study population (defined as patients with a complete loss of function in at least one individual component of the ALSFRS-R (i.e., a score of z
Slide 19: Phase 2B/3 – Subgroup Analysis prior to complete loss of function
Phase 2B/3 – Subgroup Analysis “ALS prior to complete loss of function in any individual component of the ALSFRS-R The treatment effect is clearly increased in patient prior to any complete loss of function, even when considering both normal and fast progressors Primary analysis Population Subgroup
Slide 20: The CHMP confirmed that the safety of masitinib is deemed acceptable
AB Science - Confidential The CHMP confirmed that the safety of masitinib is deemed acceptable CHMP Latest Assessment Report “Having considered the data from masitinib studies, the safety Conclusion on clinical safety profile of mentioned medicinal product is considered acceptable”; 20
Slide 21: Phase 2B/3 – Publication
Phase 2B/3 – Publication Results from phase 2B/3 and long-term survival were published in two peer-reviewed journals 21
Slide 22: Status of Conditional Marketing Authorization
Status of Conditional Marketing Authorization At this stage, negative opinion received on the application for conditional marketing authorization of masitinib in the treatment of ALS Status with EMA ▪ EMA adopted a trending toward a negative opinion ▪ AB Science intends to request a re-examination S
Slide 23: Masitinib in Multiple Sclerosis
Masitinib in Multiple Sclerosis
Slide 24: Multiple Sclerosis – Status & Positioning
Multiple Sclerosis – Status & Positioning There is no approved drugs for non-active SPMS and only one for PPMS, and masitinib stands-out as the only non BTKi into phase 3 Confirmatory Phase 3 Status Masitinib Positioning ▪ Preceded by first phase 2B/3 positive and published1 ▪ Primary Progressive MS
Slide 25: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Phase 2B/3 enrolled 656 patients Sub-study I Sub-study II Dosing Dosing Dosing Masitinib 4.5 mg/kg/day vs Masitinib 4.5 to 6.0 mg/kg/day 6.0 mg/kg/day matching placebo titration vs matching placebo ITT population ITT population N=301 N=45 N=310 (M=200 ; P=101) (M=203
Slide 26: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Phase 2B/3 demonstrated a significant benefit on disability progression with masitinib 4.5 mg/kg/day in patients with advanced stage of the disease Significant reduction in progression on EDSS (Primary Endpoint*) Patients were enrolled at advanced disease stage LSM of
Slide 27: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Phase 2B/3 demonstrated a significant reduction of risk of first disability progression by 42% and a reduction of risk of confirmed (12-week) disability progression by 37% 42% risk reduction of time to disability progression 37% risk reduction of time to confirmed dis
Slide 28: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Masitinib has the potential to become a best-in-class drug for PPMS and nSPMS Time to confirmed disability progression Reduction in confirmed Study Size Drug Type of Progressive MS Hazard Ratio (3 months) disability (patients) progression Masitinib 4.5 mg/kg/day 300 P
Slide 29: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Results from phase 2B/3 were published in peer-reviewed journal 29
Slide 30: Masitinib in Alzheimer's Disease
Masitinib in Alzheimer’s Disease
Slide 31: AD - Status & Positioning
AD - Status & Positioning Masitinib is the only drug into confirmatory phase 3 developed in mild and moderate Alzheimer Confirmatory Phase 3 Status Masitinib Positioning ▪ Preceded by first phase 2B/3 positive and ▪ Theoretical market for drugs in mild and moderate AD (masitinib positioning) publish
Slide 32: AD – Phase 2B/3
AD – Phase 2B/3 Phase 2B/3 enrolled 718 patients from 118 sites in 21 countries Sub-study I Sub-study II Dosing Dosing Dosing Dosing Masitinib 4.5 mg/kg/day vs Masitinib 4.5 to 6.0 mg/kg/day 3.0 mg/kg/day 6.0 mg/kg/day matching placebo titration vs matching placebo ITT population ITT population N=58
Slide 33: AD – Phase 2B/3
AD – Phase 2B/3 Phase 2B/3 study demonstrated a significant reduction in cognitive impairment based on ADAS-COG (p=0.0003) and improvement on daily activity based on ADCS-ADL (p=0.0381) with masitinib 4.5 mg/kg/day Significant effect on cognitive function after 24 weeks of treatment Change in ADAS-C
Slide 34: AD – Phase 2B/3
AB Science - Confidential AD – Phase 2B/3 The treatment effect on COG was sustained at week 48 Significant effect on cognitive function after 48 weeks of treatment LSM of ADAS-Cog Change from Baseline - Masitinib 4.5 versus Placebo (Full Analysis Set Population) Mastinib 4.5 mg (N=182) Placebo 3/4.5
Slide 35: AD – Phase 2B/3
AD – Phase 2B/3 Results from phase 2B/3 were published in peer-reviewed journal 35
Slide 36: Masitinib in other indications
Masitinib in other indications
Slide 37: Masitinib Platform – Sickle Cell Disease
Masitinib Platform – Sickle Cell Disease SCD is the largest monogenic disease worldwide, with a disproportionate burden on Black communities and masitinib will address severe form of SCD Growing Market, driven by the high cost of novel drugs Major Health Problem and rising awareness SCD is a group o
Slide 38: Masitinib Platform – Sickle Cell Disease
Masitinib Platform – Sickle Cell Disease Phase 2 of masitinib as a new treatment of SCD for patients harboring a specific biomarker is funded through public collaborative program ▪ Mast cells appear to play a critical role for the severe forms of SCD Scientific Rationale ▪ Masitinib has demonstrated
Slide 39: Masitinib Platform – Other programs
Masitinib Platform – Other programs Masitinib pipeline in oncology and inflammatory diseases can add value to the neurology platform Therapeutic Indication Results Development Status area ▪ First phase 3 completed (135 patients) ▪ Confirmatory phase 3 ongoing Indolent systemic mastocytosis ▪ Signifi
Slide 40: Patent protection
Patent protection
Slide 41: Masitinib - Patents
Masitinib - Patents Masitinib intellectual property rights are secured until 2037 in ALS, potentially up to 2041 in MS and AD Method of use patents Title Reference Duration Status Granted Use of masitinib for treatment of an USA, Europe, China, Japan, South Korea, amyotrophic lateral sclerosis patie
Slide 42: AB8939 Platform
AB8939 PLATEFORM
Slide 43: MDA Platform – AB8939
MDA Platform – AB8939 AB8939 has the potential to improve AML treatment based on its original mechanism of action ▪ Microtubule is a validated target and is a gold standard in many cancers (paclitaxel, vincristine, vinblastine…) Scientific ▪ Unlike other MDAs, AB8939 is not a transported by PgP/BRCP
Slide 44: MDA Platform – AB8939 Phase I
MDA Platform – AB8939 Phase I MTD has been reached in Step 1 of Phase 1 and key agencies have authorized to proceed with Step 2 Step 1 Step 2 Step 3 MTD* 3 DAYS MTD 14 DAYS MTD 14 DAYS AB8939 alone AB8939 alone AB8939 + Azacitidine • Dose 1: 0.9 mg/m2 • Dose 1: MTD3d- 1 • Dose 1: MTD14d- 1 ▪ Phase 1
Slide 45: Patent protection
Patent protection
Slide 46: AB8939 - Patents
AB8939 - Patents AB8939 intellectual property rights in AML are secured until 2036 through a ‘composition of matter’ patent and potentially until 2044 in AML with chromosome abnormality through a ‘second medical use’ patent Protection Item Duration of protection Status Patent on composition of Paten