Abivax Sa · EURONEXT:ABVX
Abivax Sa — Investor Presentation (December 2021)
41 slides from Abivax Sa's investor presentation, December 2021
Slide 1: Modulating the immune system to fight inflammatory and viral diseases, as well as cancer
Modulating the immune system to fight inflammatory and viral diseases, as well as cancer Abivax, a late-stage clinical biotech company December 2021
Slide 2: Disclaimer
Disclaimer This presentation contains information pertaining to Abivax S.A. (“Abivax” or the “Company”). Neither Abivax, nor its management, shareholders, directors, advisors, employees or representatives make any representation or warranty, express or implied, as to the fairness, the accuracy, comp
Slide 3: Abivax and ABX464: Main take-home messages
Abivax and ABX464 for chronic inflammatory diseases: Main take-home messages 1 ABX464 is a potent anti-inflammatory drug candidate with a novel first-in-class mechanism of action: Upregulation of the anti-inflammatory microRNA, miR-124 2 Safety and efficacy established in phase 2a and 2b studies in
Slide 4: Abivax in a nutshell: A phase 3 biotech
Abivax in a nutshell: A phase 3 biotech Milestones Shareholder structure1 and market cap2 IPO (ABVX) on Euronext Paris in Founded in 2013 June 2015, Truffle Capital by Truffle Capital raising € 57.7m 35% 48% € 454M Sofinnova Partners Sept. 2018: Focus Board & management ABX464 on chronic inflammatio
Slide 5: ABX464: A promising candidate addressing large unmet medical needs
ABX464: A promising candidate addressing large unmet medical needs Coming from the proprietary Abivax library of compounds, Total market size1 designed to modulate RNA biogenesis (>2,200 molecules); in inflammatory Collaboration with EVOTEC diseases Small molecule, administered as an oral capsule (o
Slide 6: ABX464: Mechanism of Action
ABX464: Mechanism of Action Modulating the immune system to fight inflammatory and viral diseases, as well as cancer 6
Slide 7: ABX464 and ABX464-N-Glu bind to the Cap Binding Complex
ABX464 and its main metabolite ABX464-N-Glu bind to the Cap Binding Complex (CBC), a key factor for RNA processing CBC binds to the 5’ end of all RNAs generated in the nucleus and ensures their processing Binding of ABX464 and ABX464-N-Glu to CBC was demonstrated by CryoEM CBC 20 ABX464-N-Glu ABX 46
Slide 8: ABX464 novel mechanism of action: upregulation of miR-124
ABX464 novel mechanism of action: Potent and specific upregulation of miR-124, activating a “physiological brake” of inflammation • First-in-class mechanism of action related to intracellular upregulation of miR-124. • microRNAs work by down-regulating the translation of their respective target gene
Slide 9: ABX464 is the only small molecule that can specifically induce miR-124
ABX464 is the only small molecule in clinical development that can specifically induce miR-124 expression • ABX464 main metabolite, ABX464-N-Glu also induces miR-124 expression miR-124 modulation • miR-124 is transcribed from 3 genomic loci, but ABX464 induces in PBMCs miR-124 expression only from L
Slide 10: Pronounced anti-inflammatory effect in vivo: DSS mouse model
Pronounced anti-inflammatory effect in vivo: ABX464 showed efficacy in the DSS mouse model* by preventing tissue damage and inflammation Colorectal tissue of mouse exposed Colorectal tissue of mouse exposed ABX464 protects mice from weight to DSS without ABX464 leads to to DSS with ABX464: Protectio
Slide 11: ABX464: Clinical Development in IBD
ABX464: Clinical Development in IBD Modulating the immune system to fight inflammatory and viral diseases, as well as cancer 11
Slide 12: Phase 2a POC induction study in ulcerative colitis
ABX464 phase 2a POC induction study in ulcerative colitis: Impressive efficacy achieved for all endpoints at the end of induction Study Design: • PI: Prof. Severine Vermeire, Leuven, BE • 32 patients with moderate to severe UC: randomized (2:1) 50mg ABX464 vs placebo, double blind, placebo controlle
Slide 13: Phase 2a POC maintenance study in ulcerative colitis
ABX464 phase 2a POC maintenance study in ulcerative colitis: Impressive efficacy results achieved after 1, 2 and 3 years of treatment 22/23 4/6 Eligible patients 16/19 15/16 29/32 Countries granted enrolled in the 16 out of 19 patients 15 out of 16 patients Patients completed the regulatory approval
Slide 14: ABX464 phase 2b study design in ulcerative colitis
Study design: ABX464 phase 2b clinical study in patients with moderate-to-severe ulcerative colitis • 254 patients in 15 countries in Europe, US and Canada (130 study sites) • Moderate-to-severe active UC (Modified Mayo Score 5-9) • Central reading of endoscopies Modulating the immune system to figh
Slide 15: Baseline characteristics well-balanced among the treatment groups
Baseline characteristics well-balanced among the treatment groups, indicating a moderate to severe UC population, very similar to data published recently 100mg 50mg 25mg Placebo (N=64) (N=63) (N=61) (N=64) Age (years) Mean (SD) 42.2 (12.34) 40.2 (13.94) 41.5 (14.16) 41.1 (14.43) Male n (%) 41 ( 64.1
Slide 16: Week 8 Efficacy Results (ITT): Primary Endpoint met
Week 8 Efficacy Results (ITT): Primary Endpoint met - Efficacy confirmed for all patients and also for subset of Bio-refractory patients Week 8 Results Placebo 25mg 50mg 100mg (ITT1 population / n=252) Primary Endpoint All -1.9 -3.1 ** -3.2 ** -2.9 * Modified Mayo Score patients Mean change from bas
Slide 17: Week 8 Efficacy Results (ITT): Secondary endpoints
Week 8 Efficacy Results (ITT): Secondary endpoints - Efficacy confirmed Week 8 Results Placebo 25mg 50mg 100mg (ITT population / n=252) Key Secondary Endpoints (not powered for statistical significance) All patients 8 (13.6%) 20 (34.5%) * 21 (39.6%) * 24 (44.4%) * Endoscopic Improvement a† Bio-refra
Slide 18: Secondary endpoints: Fecal calprotectin and Robarts Histopathology Index
Secondary endpoints: Fecal calprotectin and Robarts Histopathology Index Mean Change from baseline in Fecal calprotectin (µg/g) Change from baseline in Robarts Histopathology Index 0 0 -1 -500 -2 -3 -1000 -1027.7 -3.40 -4 -1500 -5 -6 -2000 -5.90 -7 -2192.8 -7.30 -7.30 -2500 -2316.8 -2280.9 -8 Placeb
Slide 19: Week 8 Efficacy Results (ITT): Fast onset of action
Week 8 Efficacy Results (ITT): Fast onset of action Change from Baseline pMMS (least square mean) 0 -0.4 -0.7 -0.9 -0.8 P<0.001 Placebo -1,6 P<0.001 -1.8 100mg 50mg -2.2 -2.3 25mg Modulating the immune system to fight inflammatory and viral diseases, as well as cancer 19
Slide 20: One-year efficacy results: phase 2b maintenance study
One-year efficacy results: ABX464 phase 2b maintenance study – excellent long-term efficacy confirmed 217/222 eligible patients enrolled into maintenance The first 101 patients* of the 217 were to complete 1st year of maintenance by Sept. 15, 2021: (12 patients were drop-outs, and no data available
Slide 21: ABX464 well positioned in the competitive landscape
ABX464 well positioned in the competitive landscape for both induction and maintenance Clinical Remission Rates Drug Study Active Placebo Delta Drug Active Placebo Delta Results of Induction studies (ITT)* Results of Maintenance studies (ITT)* (Patients with clinical response at the end of the induc
Slide 22: Complete resolution of UC lesions in an ABX464 treated patient
Complete resolution of UC lesions in an ABX464 treated patient (vedolizumab, infliximab and adalimumab resistant) during 3 years of open-label maintenance treatment Endoscopy before Endoscopy after Endoscopy after Endoscopy after ABX464 1st year of ABX464 2nd year of ABX464 3rd year of ABX464 ➢ 52-y
Slide 23: Favorable safety results
Favorable safety results ✓ No new safety signal ✓ Low overall drop-out rate (8.7%) at week 8 despite Covid-19 ✓ Serious adverse events (SAEs) (related and non-related) • 6.2% (placebo), 1.6% (25mg), 6.3% (50mg), 6.2% (100mg) ✓ Severe (grade 3 or 4) treatment emergent adverse events (TEAEs) (related
Slide 24: Favorable ABX464 safety profile
Favorable ABX464 safety profile Most common (> 5%) Adverse Events (AE): Drug-related or non-drug-related Placebo ABX464 25mg ABX464 50mg ABX464 100mg (N=64) (N=63) (N=63) (N=64) System Organ Class Adverse effect n (%) of Pts. n (%) of Pts. n (%) of Pts. n (%) of Pts. Number Number Number Number with
Slide 25: Following phase 2b results, ABX464 is moving to phase 3
Following phase 2b results, ABX464 is moving to phase 3 Primary endpoint (statistically significant reduction of Modified Mayo Score ) was met with once-daily ABX464 (25mg, 50mg, 100mg) at week 8 in these 254 patients randomized, double- blind and placebo-controlled clinical trial (p<0.01, intent-to
Slide 26: Regulatory feedback for ABX464 phase 3 program in UC
Regulatory feedback for ABX464 phase 3 program in UC Response from the US regulator FDA Response from European regulator EMA • Guidance and a path forward to advance ABX464 into a phase 3 clinical program in UC and a subsequent potential marketing authorization submission and commercialization • The
Slide 27: ABX464 clinical differentiation factors and positioning
ABX464 clinical differentiation factors and positioning First-in-class small molecule with unique mechanism of action Easy, oral once-daily administration ABX464 has all necessary ...2nd and 3rd line clinical therapy in moderate to Fast onset of action differentiation severe ulcerative colitis facto
Slide 28: ABX464: A potential blockbuster in IBD
ABX464: A potential blockbuster in IBD... Size of target market doubling in UC and increasing by nearly 25% in CD (2020 - 2026) 2020 In million cases UC Epidemiology Total Cases WW 12.8 Total Cases G7 3.5 Total moderate to severe cases G7 1.9 Moderate to severe cases G7 treated patients 1.5 Moderate
Slide 29: ABX464: Clinical Development in rheumatoid arthritis
ABX464: Clinical Development in rheumatoid arthritis Modulating the immune system to fight inflammatory and viral diseases, as well as cancer 29
Slide 30: ABX464 prevents joint swelling in the Collagen Induced Arthritis model
ABX464 prevents joint swelling in the Collagen Induced Arthritis model ABX464 joint swelling monitoring Age (week) 9 12 14 injection of collagen injection of collagen joint swelling (Averages of 4 legs) 2.6 P value 0,0015 * * 2.4 2.2 mm 2.0 1.8 1.6 64 C 2O M X4 H 2- 2- AB BA BA CIA CIA +ABX464 2- D
Slide 31: ABX464 study design for phase 2a in RA
ABX464 study design for phase 2a in RA following positive results in Collagen Induced Arthritis Model Phase 2a Randomized, double-blind, placebo controlled Primary objective Evaluate the safety profile of ABX464 given at two different doses (100mg and 50mg) vs placebo in combination with methotrexat
Slide 32: Phase 2a clinical study in RA – Study results (ACR)
Phase 2a clinical study in RA – Study results (ACR) Strong efficacy signal observed with 50mg o.d. → Patients’ characteristics well-balanced among the treatment arms → 70% (n=42) had inadequate response to methotrexate → 30% (n=18) had inadequate response/intolerance to TNFα inhibitors → Although no
Slide 33: Phase 2a RA study: other efficacy endpoints and biomarkers
Phase 2a clinical study in RA – Study results Other efficacy endpoints and biomarkers Placebo 50mg 100mg PP ITT PP ITT PP ITT (n=19) (n=20) (n=16) (n=21) (n=7) (n=19) DAS28-CRP -0.63 -0.60 -1,79* -1,41* -1,94* -0,72 change from baseline Low Disease Activity 2 (11%) 2 (10%) 4 (25%) 4 (19%) 3 (43%) 3
Slide 34: Phase 2a RA study: miR-124 induction and IL-6
Phase 2a clinical study in RA – Study results Other efficacy endpoints and biomarkers miR-124 induction at week 8 IL-6 compared to baseline 300 *** Placebo 100000 ** Fold increase (with median) 50mg QD 10000 200 Percentage 100mg QD 1000 * 100 100 10 1 0 ** ** 0.1 0.01 ** p<0.01 -100 ** p<0.01 0 28 8
Slide 35: Phase 2a RA study – Topline safety results summary
Phase 2a clinical study in RA – Topline safety results summary No new safety signal reported with ABX464 + MTX ✓ Serious Adverse Events: 1 (5%) placebo, 0 (0%) 50mg, 1 (5%) with 100mg ✓ No new safety signal reported. An increased incidence of AE was reported in the 100mg treatment group (mainly GI),
Slide 36: ABX464 favourable safety profile in RA
ABX464 favourable safety profile Most frequent adverse events reported in the phase 2a clinical study in RA Placebo ABX464 50mg ABX464 100mg (N=20) (N=21) (N=19) System Organ Class Adverse effect Number n (%) of Pts. Number n (%) of Pts. Number n (%) of Pts. of with AE of with AE of with AE reports
Slide 37: Phase 2a clinical study in RA: top-line results
Phase 2a clinical study in RA Top-line results allowing to move into phase 2b Baseline characteristics well balanced across all groups Primary endpoint met with ABX464 demonstrating good safety and tolerability profile with 50mg once daily oral administration A statistically significant difference (
Slide 38: How to bring ABX464 to the finish line in chronic inflammation
How to bring ABX464 to the finish line in chronic inflammation Ulcerative colitis phase 3 preparation on track • FDA end of phase 2 meeting feedback received with guidance and a path forward • EMA scientific advice scheduled for early Q1 2022 • IQVIA involved in study preparation • ∼ 2 x 700 patient
Slide 39: Portfolio newsflow through Q2 2022
Portfolio newsflow through Q2 2022 Q3 2021 Q4 2021 Q1 2022 Q2 2022 EMA feedback UC - Phase 2b (ABX464) FDA feedback ✓ Top-line results FPI phase 3 study (One-year maintenance data) Top-line results RA - Phase 2a (ABX464) (One-year maintenance data) Poster presentation of top-line results at HCC - Ph
Slide 40: Abivax financing strategy
Abivax financing strategy Current cash runway until the end of Q3 2022 Core strategic plan Partnering (ranging from licensing to M&A) Abivax to decide between two possible options: Option 1 Option 2 Partnering in 2022 before Partnering after UC phase 3 start, UC phase 3 start but before registration
Slide 41: Highly experienced Executive Committee
Highly experienced Executive Committee • Prof. Hartmut • Didier Blondel • Sophie Biguenet, • Bob Clay, Ehrlich, M.D. M.D. MSc, MBA • EVP, Chief Financial • Chief Executive Officer Officer & Board • VP, Chief Medical • Regulatory Strategy Secretary Officer Adviser • Pierre Courteille • Paul Gineste •