Ab Science Sa · EURONEXT:AB
Ab Science Sa — Investor Presentation (April 2024)
37 slides from Ab Science Sa's investor presentation, April 2024
Slide 1: Corporate Presentation
Corporate Presentation April 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: Masitinib Platform - Partnership
Masitinib Platform - Partnership 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)
Slide 6: 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 7: 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 8: Masitinib in Amyotrophic Lateral Sclerosis
Masitinib in Amyotrophic Lateral Sclerosis
Slide 9: ALS - Status & Positioning
ALS - Status & Positioning In ALS, no drug has generated a consensus based on definitive evidence of efficacy Confirmatory Phase 3 Status Masitinib Positioning ▪ Preceded by first phase 2B/3 positive and published1 ▪ Like masitinib, the final registration of the most advanced compounds are dependent
Slide 10: ALS – Positioning
ALS – Positioning Market research from top advisory firm hired for partnership indicates that if masitinib is approved, it can become the standard of care in ALS and can generate substantial revenue in the USA and the EU Market size While ALS market is dynamic, we forecast masitinib could capture $3
Slide 11: ALS – Conditional Marketing Authorization
ALS – Conditional Marketing Authorization Decision from EMA on conditional marketing authorization for masitinib in ALS is expected in Q2 2024 and decision from Health Canada on NOC/c reconsideration is expected in Q3 2024 EMA ❖ Application filed in August 2022 ❖ Whereas an Oral Explanation was plan
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: ALS - Phase 2B/3
ALS - Phase 2B/3 Phase 2B/3 demonstrated with masitinib 4.5 mg/kg/day a significant delay of disease progression over a 48- week period in the primary analysis population and significant OS benefit in subgroup of patients with moderate ALS Normal Progressors (Primary Endpoint) : 27% slowing of + 25
Slide 14: ALS - Phase 2B/3
ALS - Phase 2B/3 Benefit on functional score (ALSFRS-R) was supported by improvement in secondary endpoints in (time to disease progression or death (PFS), quality of life, and respiratory function) (Database locked on 16/03/2017) B Science - AB10015 ALS PHASE 25% delay 2/3 in disease progression (P
Slide 15: ALS - Phase 2B/3 Consistent Pattern
ALS - Phase 2B/3 There is a consistent pattern across key efficacy endpoints of increasing treatment effect with decreasing baseline severity Increasing benefit Moderate / Severe / Very Moderate / Severe ALS * Moderate ALS * Population (ΔFS<1.1, not including fast progressors) Severe ALS * (step 3)
Slide 16: ALS - Phase 2B/3
ALS - Phase 2B/3 Results from phase 2B/3 and long-term survival were published in two peer-reviewed journals 16
Slide 17: Masitinib in Multiple Sclerosis
Masitinib in Multiple Sclerosis
Slide 18: 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 19: 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 20: 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 21: 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 22: Multiple Sclerosis – Phase 2B/3
Multiple Sclerosis – Phase 2B/3 Results from phase 2B/3 were published in peer-reviewed journal 22
Slide 23: Masitinib in Alzheimer’s Disease
Masitinib in Alzheimer’s Disease
Slide 24: 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 25: 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 26: AD – Phase 2B/3
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 mg (N=176) 3 2 LS Mean (+
Slide 27: AD – Phase 2B/3
AD – Phase 2B/3 Results from phase 2B/3 were published in peer-reviewed journal 27
Slide 28: Masitinib in other indications
Masitinib in other indications
Slide 29: 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 30: 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 31: 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 32: AB8939
AB8939
Slide 33: 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 34: 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 35: Patent protection
Patent protection
Slide 36: 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 37: 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