Sanofi SA · EURONEXT:SAN
Sanofi SA — Investor Day (July 2022)
48 slides from Sanofi SA's investor day presentation, Jul 13, 2022
Slide 1: Sanofi
sanofi
Slide 2: Hemophilia Investor Event
Hemophilia Investor Event London, UK July 13, 2022
Slide 3: Forward-looking statements
Forward-looking statements This document contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward- looking statements are statements that are not historical facts. These statements include projections and estimates and their underlyi
Slide 4: Agenda
Agenda Hemophilia Investor Event 01 Sanofi’s ambition in Rare 03 Clinical experience with Blood Disorders efanesoctocog alfa Dietmar Berger Angela Weyand, MD Global Head of Development, CMO Assistant Professor at Michigan Medicine 02 Clinical experience with 04 Advancing hemophilia treatment fitusir
Slide 5: Sanofi's ambition in Rare Blood Disorders
Sanofi’s ambition in Rare Blood Disorders Dietmar Berger Global Head of Development, CMO 5
Slide 6: Transforming the lives of people with rare blood disorders
Transforming the lives of people with rare blood disorders Sanofi RBD portfolio Marketed products Molecules under investigation Drug Indication Ph I Ph II Ph III Hem A efanesoctocog alfa (w/o inhibitors) Hemophilia Hem A and B fitusiran (w/ w/o inhibitors) rilzabrutinib ITP SAR4450881 CAD CAD ∂ rilz
Slide 7: Broad set of innovative pipeline assets addressing unmet needs
Broad set of innovative pipeline assets addressing unmet needs Target Drug Indication Description Modality Unmet need Hem A Factor VIII efanesoctocog alfa (w/o inhibitors) replacement Biologic • Breakthrough bleeds and joint deterioration • Treatment burden Hem A and B • Access to treatment fitusira
Slide 8: Rilzabrutinib is a potential first in class oral, reversible BTK inhibitor in ITP
Rilzabrutinib is a potential first in class oral, reversible BTK inhibitor in ITP Rilzabrutinib has the potential to target the underlying cause of the disease High unmet need • Patients needs extend beyond platelet counts to fatigue and quality of life • Inflammatory process plays an important role
Slide 9: Positive Phase I/II published in NEJM, strong effect across all analyzed subgroups
Positive Phase I/II published in NEJM, strong effect across all analyzed subgroups The New England Journal of Medicine, April 14, 2022 Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. David J. Kuter, M.D, et al. N Engl J Med 2022; 386:1421-1431 DOI: 10.1056/NEJMoa2110297 Copyright ©
Slide 10: Sanofi is well positioned to define new standards in hemophilia with two novel molecules
Sanofi is well positioned to define new standards in hemophilia with these two novel molecules Hemophilia A without inhibitors: Hemophilia A or B, with or without inhibitors: efanesoctocog alfa fitusiran First-in-class siRNA targeting anti-thrombin rFVIIIFc-VWF-XTEN® XTEN® polypeptide insertion to i
Slide 11: Efanesoctocog alfa has the potential to revolutionize hemophilia A treatment
Efanesoctocog alfa has the potential to revolutionize hemophilia A treatment by providing close to normal factor activity levels Single Dose Study: PK Comparison with EHL and SHL Factors efanesoctocog alfa • Positive Phase 3 data : primary and key secondary endpoints met • FDA BLA submitted; Breakth
Slide 12: Fitusiran has a robust clinical program for hemophilia A or B
Fitusiran has a robust clinical program designed for all patients with hemophilia A or B, with or without inhibitors Study Indication 2019 2020 2021 2022 2023 2024+ ALN-AT3SC- • Completed three Phase 3 studies in Hem A or B 003 (N=60) (w/ inhibitors) adults and adolescents (>12years) with 80 mg mont
Slide 13: Clinical experience with fitusiran
Clinical experience with fitusiran Guy Young, MD Director, Hemostasis and Thrombosis Center at Children's Hospital Los Angeles 13
Slide 14: What are the unmet needs?
What are the unmet needs? Non-inhibitor Inhibitor Curing patients patients hemophilia We need equally safe and We need (much) better agents efficacious treatments which to prevent bleeding in inhibitor the ultimate unmet need reduce treatment burden patients Necessary to reduce mortality, morbid
Slide 15: Fitusiran has completed three phase 3 studies in adults and adolescents
Fitusiran has completed three phase 3 studies in adults and adolescents ≥ 12 years ALN-AT3SC-003 (N=54) ALN-AT3SC-004 (N=120) ALN-AT3SC-009 (N=80) • Patients with Hem A or B aged ≥12 years • Patients with Hem A or B aged ≥12 years • Patients with Hem A or B aged ≥12 years • With inhibitors • Without
Slide 16: Phase 3 studies demonstrate significant reduction in bleeding, median ABR of zero
Phase 3 studies demonstrate statistically significant reduction in bleeding and a median ABR of zero across on-demand patient populations Estimated ABR reduction: 89.2% Estimated ABR reduction: 89.9% (95%CI; 79.3; 94.4) (p<0.0001) (95% CI; 84.1; 93.6) (p<0.0001) 21.8 (Q1, 8.4; Q3, 41.0) Median ABR (
Slide 17: Median ABR of zero when compared with prior prophylaxis
Phase 3 studies demonstrate statistically significant reduction in bleeding and a median ABR of zero when compared with prior prophylaxis Estimated ABR reduction: 61.1% (95% CI; 32; 77.6) (p=0.0008) Median ABR (IQR) 4.4 (Q1, 2.2; Q3, 10.9) 0.0 (Q1, 0.0; Q3, 0.0) Factor/BPA Fitusiran 80 mg prophylaxi
Slide 18: Majority of people with hemophilia experienced zero treated bleeds
Phase 3 studies consistently demonstrated majority of people with hemophilia experienced zero treated bleeds Percentage of participants with zero bleeds in the efficacy period 65.8% 50.6% 63.1% 5.3% 5.0% 16.9% Fitusiran On-demand Fitusiran On-demand Fitusiran Factor/BPA prophylaxis 80mg BPA (n=19) p
Slide 19: Fitusiran improved quality of life: Haem-A-QoL physical health
Fitusiran prophylaxis improved health-related quality of life as measured by Haem-A-QoL physical health domain Measured by Reduction from Baseline to End of Study BPA on-demand Fitusiran 80mg Factor on-demand Fitusiran 80mg Factor/BPA Fitusiran 80 mg (N=18) prophylaxis (N=32) (N=38) prophylaxis (N=7
Slide 20: Fitusiran improved quality of life: Haem-A-QoL total score
Fitusiran prophylaxis improved health-related quality of life as in Haem- A-QoL total score Measured by Reduction from Baseline to End of Study BPA on-demand Fitusiran 80mg Factor on-demand Fitusiran 80mg Factor/BPA Fitusiran 80 mg (N=18) prophylaxis (N=32) (N=38) prophylaxis (N=72) prophylaxis (n=6
Slide 21: ATLAS-INH: Safety and Tolerability
ATLAS-INH: Fitusiran vs on-demand bypassing agents, hem A or B with inhibitors: Safety and Tolerability1 Overview of Treatment Emergent Adverse Events (TEAEs) BPA On-demand Fitusiran 80 mg Category, n (%) (N=19) Prophylaxis (N=41) Any TEAE 11 (57.9%) 38 (92.7%) Any TESAE (Treatment-Emergent Serious
Slide 22: ATLAS-A/B: Safety and Tolerability
ATLAS-A/B: Fitusiran vs on demand factor, hem A or B without inhibitors: Safety and Tolerability1 On-demand Factor Fitusiran 80 mg TEAE Category, n (%) Concentrates (N=40) Prophlyaxis (N=79) Any TEAE 18 (45.0) 62 (78.5) Any TESAE 5 (12.5) 5 (6.3) Any TEAE leading to treatment discontinuationa - 2 (2
Slide 23: ATLAS-PPX: Safety and Tolerability
ATLAS-PPX: Fitusiran vs prior factor/BPA prophylaxis, hem A or B, with or without inhibitors: Safety and Tolerability1 Factor/BPA prophylaxis Fitusiran 80 mg prophylaxis Event, n (%) (N=65a) (N=67a,b) Participants with any AE (Adverse Event) 22 (33.8) 48 (71.6) Participants with any SAE (Serious Adv
Slide 24: Rationale for revised dose and dosing regimen
Rationale for revised dose and dosing regimen Vascular Patient characteristics AT thrombotic Age range Hem subtype and Medical history/comments category • Data suggest that the risk of vascular eventb (years) inhibitor status thrombotic events may be greater with Cerebrovascular 30–40 Hem A patient
Slide 25: Fitusiran revised dose regimen to enhance benefit risk balance
Fitusiran revised dose and dose regimen is designed to enhance the benefit risk balance In all ongoing Phase 3 trials, all patients start on 50mg, 6 injections per year ~90% of all patients require zero or one dose change Individualized dosing based on AT levels 90% of patients 50mg QM • Target rang
Slide 26: Conclusions
Conclusions Based on three completed Phase 3 studies with 80 mg dose, monthly fitusiran prophylaxis resulted in a sustained lowering of antithrombin and increased thrombin generation1 The present findings and low ABR in the 80 mg monthly dose suggest that fitusiran rebalances hemostasis, provides co
Slide 27: Clinical experience with efanesoctocog alfa
Clinical experience with efanesoctocog alfa Angela Weyand, MD Assistant Professor at Michigan Medicine 27
Slide 28: Efanesoctocog alfa has a comprehensive clinical development program
Efanesoctocog alfa has a comprehensive clinical development program Phase 1/2 PK Studies Phase 3 Studies Complete Complete Adult PTPs (18–65 years) Adult and adolescent PTPs (≥12 years) • Phase 1 Single-Dose Study1 • XTEND-1 Phase 3 Pivotal Study4 • Phase 1 Repeat Dose Study2 • Sequential PK study3
Slide 29: XTEND-1 was an open-label, multicenter, phase 3 study
XTEND-1 was an open-label, multicenter, phase 3 study of efa in previously treated patients1 Key eligibility criteria Primary endpoint • Adult and adolescent patients (≥12 years) with severe hemophilia A ABR in the prophylaxis treatment arm (ARM A) • Previous treatment with any recombinant and/or pl
Slide 30: Efanesoctocog alfa prophylaxis provided highly effective protection against bleeds
Efanesoctocog alfa prophylaxis provided highly effective protection against bleeds, superior to Prior FVIII Therapy Primary Endpoint Met Key Secondary Endpoint Met Clinically meaningful bleed control Superior bleed control versus prior FVIII prophylaxis Intra-patient ABR comparison (n=78)b,c Arm A (
Slide 31: The majority of patients had zero bleeds
The majority of patients receiving efanesoctocog alfa prophylaxis had zero bleeds Percentage of Patients with Zero Bleeds Key Secondary Endpoint On-demand vs Prophylaxis Superior bleed control versus prior FVIII on-demand (Arm B) Switched to efanesoctocog Switched to efanesoctocog 25 prophylaxis fro
Slide 32: Efanesoctocog alfa sustained FVIII levels in normal to near-normal range
Efanesoctocog alfa sustained FVIII levels in the normal to near-normal range for most of the week Sequential PK studya,b Week 1 (n=17) 123.1% Week 26 (n=17) At Week 26, patients had a geometric mean FVIII activity (IU/dL) 68.0% 100 100% (95% CI) half-life of 47.0 (42.3–52.3) hoursa Mean (SD) plasma
Slide 33: Meaningful improvements in physical health and pain
Efanesoctocog alfa prophylaxis led to clinically meaningful improvements in physical health and pain Haem-A-QoL Physical Health Scorea,b PROMIS Pain Intensitya,c (Arm A) (Arm A) 40 37.02 2.8 In participants who Mean Haem-A-Qol 35 2.6 Physical Health completed exit interviews, 2.47 Mean PROMIS Pain I
Slide 34: Significant improvement in joint health
Significant improvement in joint health with efanesoctocog alfa prophylaxis 96% 72% All 45 target jointsa resolved reduction in target joint bleeds Improved joint of patients in Arm A did in the 14 patients that had was observed in Arm B outcomes as measured not experience any joint at least 12 mont
Slide 35: Highly effective in treatment of bleeding episodes and perioperative management
Efanesoctocog Alfa was highly effective in treatment of bleeding episodes and perioperative management of bleeding episodes were resolved with a single 50 IU/kg injection 96.7% of efanesoctocog alfa (350/362 bleeds)a All patients’ hemostatic response (100%) to efanesoctocog alfa use during the perio
Slide 36: Efanesoctocog alfa was well tolerated
Efanesoctocog alfa was well tolerated Safety Results from XTEND-1 Overall n (%)a (N=159) Patients with ≥1 TEAE 123 (77%) Inhibitor development to FVIII was not Patients with ≥1 TESAE 15 (9%)b detected TEAEs leading to death 1 (1%)c No reports of serious allergic reactions, TEAEs leading to treatment
Slide 37: Once-weekly prophylaxis provided superior bleed protection
Once-weekly efanesoctocog alfa prophylaxis provided superior bleed protection with clinically meaningful improvements in patient outcomes High sustained Superior bleed Clinically meaningful Well tolerated factor activity protection improvements within normal to near- to prior SOC FVIII prophylaxis i
Slide 38: Raising the bar: creating new possibilities for people with hemophilia
Raising the bar: creating new possibilities for people with hemophilia "I think there has been a switch in my mind, of can do, rather than can’t do. I used to shy away from doing stuff before, in case I had a problem, to a certain extent. Whereas now, I think you know, I’ll…I feel like I can push my
Slide 39: Advancing hemophilia treatment standards
Advancing hemophilia treatment standards Bill Sibold GBU Head Specialty Care 39
Slide 40: Hemophilia is the key driver of the >€1.2B Sanofi RBD franchise
Hemophilia is the key driver of the >€1.2B Sanofi RBD franchise Global revenue by product Enjaymo® (Q2 2021 – Q1 2022) First treatment for CAD, recently launched € 1.2bn Cablivi® First treatment for aTTP Alprolix® Hemophilia represents First EHL launched for hem B ~€1bn1 Eloctate® First EHL launched
Slide 41: Factor remains a cornerstone of hemophilia A treatment
Factor remains a cornerstone of hemophilia A treatment Hemophilia A non-inhibitor: global revenues 2021 WW Market sales • Of the €7.4bn hemophilia A non- €7.4bn inhibitor market today, 70% is factor 2021 WW factor product sales €2.4 €5bn • The €5bn factor market today is Other EHL Adynovate® highly
Slide 42: Patient preference will determine how the growing hemophilia market evolves
Patient preference will determine how the growing hemophilia market evolves Hemophilia A non-inhibitor global revenues Treatment Attitudes1 2027 WW Market Sales €2.9bn 2021 WW €10.3bn Market Sales €7.4bn Neutral 40% 16% 44% I’d rather have the I’d rather have efficacy with highest efficacy levels th
Slide 43: Sanofi is advancing hemophilia A treatment by setting new standards
Sanofi is advancing hemophilia A treatment by setting new standards High (bleed protection, hemostasis, disease control) efa High efficacy class fitusiran efanesoctocog alfa Q1W EHLs Q4W potential gold-standard in protection with weekly Efficacy dosing emicizumab SHLs Extended efficacy class fitusir
Slide 44: Close
Close Dietmar Berger Global Head of Development, CMO 44
Slide 45: Key takeaways
Key takeaways Sanofi is committed to bringing The growing hemophilia A Sanofi is well positioned to set innovative first-in-class market will evolve based on new standards and transform therapies in Rare Blood patient preference for high the lives of people with Disorders efficacy or extended effica
Slide 46: Q&A session
Q&A session Dietmar Berger, MD PhD Bill Sibold Global Head of Development, GBU Head Specialty Care CMO Guy Young, MD Angela Weyand, MD Director, Hemostasis and Thrombosis Assistant Professor at Michigan Center at Children's Hospital Los Angeles Medicine 46 Hemophilia Investor Event
Slide 47: Abbreviations (1/2)
Abbreviations (1/2) AE Adverse Event HIV Human Immunodeficiency Virus AESI Adverse Event of Special Interest HJHS Hemophilia Joint Health Score ALT Alanine Aminotransferase IQR Interquartile Range AST Aspartate Aminotransferase Ig Immunoglobulin AT Antithrombin ITP Immune Thrombocytopenia aTTP acqui
Slide 48: Abbreviations (2/2)
Abbreviations (2/2) ROA Route Of Administration SAE Serious Adverse Event siRNA small interfering RNA SD Standard Deviation SHL Standard Half-Life SOC Standard Of Care SS Steady State SYK Spleen Tyrosine Kinase TEAE Treatment-Emergent Adverse Event TEAESI Treatment Emergent Adverse Events of Special