Astrazeneca Plc · LSE:AZN
Astrazeneca — Investor Day (2024)
31 slides from Astrazeneca's investor day presentation, 2024
Slide 1: Rare Disease
Rare Disease Marc Dunoyer, CEO, Alexion and Chief Strategy Officer, AstraZeneca Gianluca Pirozzi, SVP, Head of Development and Safety, Alexion Investor Day • 2024
Slide 2: Forward looking statements
Forward looking statements In order, among other things, to utilise the 'safe harbour' provisions of the US Private Securities Litigation Reform Act of 1995, AstraZeneca (hereafter ‘the Group’) provides the following cautionary statement: This document contains certain forward-looking statements wit
Slide 3: Rare Disease – next wave of growth to 2030 and beyond
Rare Disease – next wave of growth to 2030 and beyond Illustrative only, not to scale Beyond 2030 Complement continued leadership Launching key NMEs ALXN2220 Genomic medicines Existing anselamimab eneboparatide1 portfolio efzimfotase alfa gefurulimab Beyond Complement $7.8bn focused on first- and/or
Slide 4: Alexion has delivered sustainable and robust growth since acquisition
Alexion has delivered sustainable and robust growth since acquisition Rare Disease Total Revenue growth (%) Rare Disease patient growth (%) 25% 21% 18% 16% 12% 10% 2022 2023 Q1 2024 2022 2023 Q1 2024 FY 2022 growth rates on medicines acquired with Alexion have been calculated on a pro forma basis co
Slide 5: Emerging Markets growth leveraging AstraZeneca global footprint
Emerging Markets growth leveraging AstraZeneca global footprint Emerging Markets Total Revenue growth (%) Strategic opportunity in China 73% 62% Leveraging AstraZeneca’s presence to address 6% underserved rare populations in China 2022 2023 Q1 2024 Launching in new countries globally 2023 2030 Natio
Slide 6: Complement
Complement 6 Investor Day • 2024
Slide 7: Sustainable growth of C5 Franchise
$5bn+* Sustainable growth of C5 Franchise C5 Total Revenue growth (%) 10% Rapid conversion from 8% Soliris to Ultomiris 7% Superior efficacy with rapid, sustained 2022 2023 Q1 2024 complement inhibition and steroid sparing data C5 patient growth by indication (%) Geographic reach leveraging 17% 20%
Slide 8: Neurology driving C5 growth in the short- to mid-term
Neurology driving C5 growth in the short- to mid-term Neurology Total Revenue growth (%)1 Sustained long-term improvements 23% 88% of Ultomiris patients saw clinically 15% meaningful improvements in activities of daily 11% living to Week 1642 2022 2023 Q1 2024 Steroid sparing data >75% of Soliris pa
Slide 9: gMG – expanding reach with next-generation gefurulimab
gMG – expanding reach with next-generation gefurulimab Opportunity to treat earlier and broader patient population1 Soliris US diagnosed patients: 72,000 AChR+ across gMG MGFA Classes II to IV SoC for more severe, Q2W refractory patients 66,000 36,000 9,000 3rd immuno-suppressant or IVIg/PP/PLEX Sol
Slide 10: gMG – forecasted market evolution across Top 7 countries
gMG – forecasted market evolution across Top 7 countries gMG market evolution across Top 7 countries1 80% of patients to move to 100% branded medicines by 20342 90% 80% 70% 60% gMG launched in 31 countries 50% with additional 25 countries 40% by 2025 30% 20% 10% We anticipate patients 0% 2022 2023 2
Slide 11: Ultomiris indication expansion, maximising complement-mediated and adjacent diseases
Ultomiris indication expansion, maximising $5bn+* complement-mediated and adjacent diseases Phase III near-term catalysts Patients diagnosed1 Haematopoietic stem cell transplantation-associated 4k thrombotic microangiopathy (HSCT-TMA) TM-313/4 2025 HSCT-TMA data readout IgA nephropathy (IgAN) 400k2
Slide 12: HSCT-TMA – potential for Ultomiris in rare, life-threatening complication of bone marrow transplant
HSCT-TMA – potential for Ultomiris in rare, life-threatening complication of bone marrow transplant Phase III trials ongoing 26 weeks Factors associated with HSCT induce dysregulation Adult R 1:1 Ultomiris + best SoC of the complement system TM-313 Placebo + best SoC N = 94 26 weeks Adult and paedia
Slide 13: IgAN – potential to transform course of disease with complement inhibition
IgAN – potential to transform course of disease with complement inhibition IgAN patient progression First-line intervention Complement inhibition 120k diagnosed reduces renal functional decline has potential to modify disease with IgAN in US ACE inhibitor Poor IgAN outcomes, or ARB few patients avoi
Slide 14: IgAN – rapid and sustained proteinuria reduction in Phase II supports Phase III trial
IgAN – rapid and sustained proteinuria reduction in Phase II supports Phase III trial Ultomiris Phase II SANCTUARY1 Ultomiris Phase III ICAN Mean spot UPCR over 26 weeks >40% reduction in Ultomiris +SoC | Q8W i.v. R Ultomiris placebo proteinuria 1:1 Placebo +SoC | Q8W i.v. N = 450 UPCR Stable eGFR r
Slide 15: Future plans to improve success rates of kidney transplant
Future plans to improve success rates of kidney transplant Current pipeline Expansion into transplantology 12% incidence of cAMR in adult kidney transplants1 Antibody- mediated Chronic kidney rejection diseases: IgAN, 75% of kidney transplant LN, other GN AMR Acute AMR Chronic patients diagnosed wit
Slide 16: Future innovation planned in complement
Future innovation planned in complement Soliris Soliris Soliris danicopan Continued PNH aHUS gMG, NMOSD GA innovation in complement Ultomiris Ultomiris Ultomiris PNH aHUS gMG, NMOSD Voydeya Ultomiris gefurulimab HSCT-TMA, CSA-AKI, PNH-EVH gMG LN, IgAN vemircopan LN, IgAN ALXN2030 Marketed cAMR Devel
Slide 17: Beyond Complement: bone and endocrine disease
Beyond Complement: bone and endocrine disease 17 Investor Day • 2024
Slide 18: Hypophosphatasia – building on Strensiq, efzimfotase alfa drives innovation and expanded access
Hypophosphatasia – building on Strensiq, $3–5bn* efzimfotase alfa drives innovation and expanded access Strensiq Total Revenue growth (%) efzimfotase alfa (ALXN1850) 21% 21% Phase III trials ongoing Q2W Convenient dosing with data anticipated >2025 Q2W vs. 6–12 injections 18% s.c. per two weeks with
Slide 19: Hypoparathyroidism – Amolyt Pharma acquisition yields potential best-in-class therapy with eneboparatide
Hypoparathyroidism – Amolyt Pharma acquisition $1–3bn* yields potential best-in-class therapy with eneboparatide Clinical priorities eneboparatide Phase IIa2 Normalising serum calcium levels Serum calcium (mean ADsCa, mg/dL) Strengthening Alexion preventing muscular cramps and 11.5 cardiac effects 1
Slide 20: Beyond Complement: amyloidosis
Beyond Complement: amyloidosis 20 Investor Day • 2024
Slide 21: Novel anti-fibril depleter mechanisms with potential to restore normal organ function
Novel anti-fibril depleter mechanisms with potential to restore normal organ function ALXN2220 | transthyretin (ATTR) amyloidosis anselamimab | light-chain (AL) amyloidosis ADCs and Selectively binds to RCs Cell therapy Selectively binds ATTR amyloid fibrils κ and λ light chain fibrils ADCs and RCs
Slide 22: Leveraging CVRM and Rare Disease expertise in ATTR-CM
Leveraging CVRM and Rare Disease expertise in ATTR-CM Complementary mechanisms % NYHA I NYHA II NYHA III NYHA IV population 9% 49% 36% 6% Pathophysiology Medicine and modality less severe more severe 1 TTR Silencer production Wainua – Cardio-TTRansform with data 2025+ Wainua blocks TTR in the liver
Slide 23: Amyloidosis (ATTR-CM) – depleter mechanism with the potential to reverse course of disease
Amyloidosis (ATTR-CM) – depleter mechanism $3–5bn* with the potential to reverse course of disease ALXN2220 (NI006) Phase Ib1 Phase III DepleTTR-CM Patient 1, 75yo male, ATTRwt, 60 mg/kg, on tafamidis ALXN2220 Q4W i.v. R 2:1 a placebo N = 1000 24 to 48 months Primary endpoints include composite even
Slide 24: Amyloidosis (AL) – transforming patient outcomes with potential first-in-class depleter
Amyloidosis (AL) – transforming patient outcomes $1–3bn* with potential first-in-class depleter anselamimab addressing key clinical outcomes Cause progressive accumulation of amyloid fibrils in tissues Novel depleter mechanism Two trials ongoing in and organs including to eliminate deposited Mayo St
Slide 25: Pipeline and new modalities
Pipeline and new modalities 26 Investor Day • 2024
Slide 26: Genomic medicines – unlocking transformative and potentially curative therapies for rare diseases
Genomic medicines – unlocking transformative and potentially curative therapies for rare diseases Today Tomorrow Future building capabilities through engineering portfolio with addressing diseases strategic investments curative potential in areas of high unmet need 80% of rare diseases Advancing up
Slide 27: Several major assets with blockbuster potential support our growth ambition
Several major assets with blockbuster potential support our growth ambition Growth drivers to 2030 and beyond 2024 2025 Peak Year Revenue potential acoramidis2 Ultomiris TM-313/4 ATTR-CM HSCT-TMA $1–3bn $3–5bn $5bn+ anselamimab efzimfotase alfa Koselugo KOMET eneboparatide1 CALYPSO AL amyloidosis hy
Slide 28: Q&A session
Q&A session Marc Dunoyer Gianluca Pirozzi C H IEF EX EC UT IVE O F F IC ER, S VP, H EAD O F DEVEL O PMEN T , AL E X IO N REGUL AT O RY & S AF ET Y Nicola Heffron Seng Cheng S VP, H EAD O F GL O B AL S VP, H EAD O F RES EARC H MARK E T IN G & MARK E T AC C E S S & PRO DUC T DEVEL O PMEN T Investor Da
Slide 29: Appendix
Appendix 30 Investor Day • 2024
Slide 30: Glossary – 1 of 2
Glossary – 1 of 2 1L, 2L, 3L first-, second-, third-line CLL chronic lymphocytic leukaemia GLP-1/glu glucagon-like peptide 1 receptor/glucagon dual peptide agonist 6MWT 6-minute walk test cm centimetre GLP-1RA glucagon-like peptide 1 receptor agonist AAV adeno-associated virus CM cardiomyopathy gMG
Slide 31: Glossary – 2 of 2
Glossary – 2 of 2 K+ potassium NST neoadjuvant systemic treatment RSV respiratory syncytial virus KCCQ Kansas City Cardiomyopathy Questionnaire NT-proBNP N-terminal pro-B-type natriuretic peptide s. asthma severe asthma LA amylin long-acting amylin NYHA New York Heart Association s.c. subcutaneous L