Slide 1: Meet AZN Management: ASBMR 2026
Meet AZN Management: ASBMR 2026 Conference call and webcast for investors and analysts 12 October 2026 1
Slide 2: Cautionary statements regarding forward-looking statements
Cautionary statements regarding 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 certai
Slide 3: Meet AZN Management @ ASBMR – agenda
Meet AZN Management @ ASBMR – agenda Marc Dunoyer, CEO, Alexion, AstraZeneca Furthering our ambition in Rare Disease Rare Disease efzimfotase alfa: developed to address Gianluca Pirozzi, SVP, Head of Development, broad HPP population Regulatory & Safety Dr. Raja Padidela, Consultant Pediatric efzimf
Slide 4: Furthering our ambition in Rare Disease
Furthering our ambition in Rare Disease Marc Dunoyer CEO, ALEXION, ASTRAZENECA RARE DISEASE 4
Slide 5: Ambition – $80bn Total Revenue by 2030 & sustained 2030+ growth
Ambition – $80bn Total Revenue by 2030 & sustained 2030+ growth Beyond 2030 Working on “today, tomorrow and the day after" ADCs and Radioconjugates Illustrative only, not to scale Cell therapy and T-cell engagers Launching key NMEs $80bn Gene therapy AZD0120 saruparib Etcamah surovatamig Next-genera
Slide 6: Rare Disease – significant unmet need globally
Rare Disease – significant unmet need globally 10,000+ known rare diseases1 7 in 10 (<10% have approved treatment2) rare diseases begin in childhood1 80% 6+ years of rare diseases are genetic1 average time to diagnosis1 Our vision is to advance the next wave of rare disease innovation to revolutioni
Slide 7: Rare Disease – our disease areas of focus
Rare Disease – our disease areas of focus Haematology, Bone & Cardiology & Nephrology & Neurology Endocrine Rare Tumours Amyloidosis Transplant Disorders Targeting areas of high unmet need with best- or first-in-class potential 7 Appendix: Glossary.
Slide 8: efzimfotase alfa: developed to address broad HPP population
efzimfotase alfa: developed to address broad HPP population Gianluca Pirozzi SVP, HEAD OF DEVELOPMENT, REGULATORY & SAFETY 8
Slide 9: HPP is an inherited, heterogeneous disease affecting patients of all ages
Bone & Endocrine Disorders HPP is an inherited, heterogeneous disease affecting patients of all ages Hypophosphatasia (HPP) is caused by deficient ALP activity affecting patients of all ages. HPP is heterogeneous and multisystemic, with a range of skeletal and functional manifestations significantly
Slide 10: efzimfotase alfa is a second-generation ERT for broad HPP
Bone & Endocrine Disorders efzimfotase alfa is a second-generation ERT designed to address broad spectrum of patients with HPP Improved molecule with key efzimfotase alfa differences vs Strensiq Paed onset + All HPP patients Target patients bone symptoms >2yrs Enhanced Lower therapeutic dose enzyme
Slide 11: HPP – efzimfotase alfa Phase III programme
Bone & Endocrine Disorders HPP – efzimfotase alfa Phase III programme 2–<12 yr 2–<12 yr ≥12 yr Treatment-naive Switch from Strensiq (asfotase alfa) Treatment-naive Controla Placebo Active (asfotase alfa) Placebo Blinding Double-blind Open-label Double-blind Treatment allocation 2:1 (N=29) 1:1 (N=43)
Slide 12: Key takeaways from MULBERRY and CHESTNUT trials
Bone & Endocrine Disorders HPP: efzimfotase alfa – key takeaways from MULBERRY and CHESTNUT trials Primary endpoint | Bone Health by RGI-Ca,1 2–<12 yr, open-label 2–<12 yr, treatment-naive switch from asfotase alfa • Met primary objective: similar TEAE incidences with efzimfotase alfa (90.5%d) and a
Slide 13: efzimfotase alfa – Phase III HICKORY
efzimfotase alfa – Phase III HICKORY Dr. Raja Padidela, CONSULTANT PAEDIATRIC ENDOCRINOLOGIST & METABOLIC BONE DISEASES, ROYAL MANCHESTER CHILDREN’S HOSPITAL 13
Slide 14: HICKORY – Trial Design
Bone & Endocrine Disorders HICKORY – Trial Design ≥12 yr, treatment-naive Primary endpoint: N=84 • 6-minute walk test efzimfotase alfa (s.c. Q2W) 24wks Key secondary endpoints: Study population: • 30-second • Treatment-Naïve patients R 2:1 Sit-to-Stand, Lower Extremity with HPP Functional Scale •
Slide 15: HICKORY – Baseline characteristics
Bone & Endocrine Disorders HICKORY – Baseline characteristics ≥12 yr, treatment-naive Placebo efzimfotase alfa (n=84) (n=40) Sex, n (%) Female 61 (72.6) 31 (77.5) Age, median (min, max), years 45.5 (12, 75) 43.5 (12, 78) Race, n (%)a,b White 59 (70.2) 30 (75.0) Asian 17 (20.2) 8 (20.0
Slide 16: HICKORY – improvement in 6MWT but not statistical significance
Bone & Endocrine Disorders HICKORY – efzimfotase alfa showed ≥12 yr, treatment-naive improvement in 6MWT but not statistical significance 6MWT efzimfotase alfa change from baseline Placebo (n=40) (n=84) at week 25a LS mean difference 17.05 (12.223) vs PBO (SE), 0.1675 P value a. Graph shows descrip
Slide 17: HICKORY – 6MWT results during randomised period and OLE
Bone & Endocrine Disorders HICKORY – efzimfotase alfa 6MWT results ≥12 yr, treatment-naive during randomised period and OLEa Mean change, Week 0–49: Mean change in OLE 61.9 metresb (Wk 25–49): 43.0 metresc Number of participants efzimfotase alfa 84 81 77 75 72 Placebo 40 39 39 38 36 • Patients origi
Slide 18: HICKORY – key secondary endpoints numerically favoured efzimfotase alfa
Bone & Endocrine Disorders HICKORY – key secondary endpoints ≥12 yr, treatment-naive numerically favoured efzimfotase alfa MCID: 31 (adults); 43 meters (adolescent) b MWPC: 1 to 4 reps MWPC: 2 to 13 30-second Lower Extremity MWPC: –2.25BPI-SF to –0.50 MWPC: 6 to 14 Key secondary FACIT-Fatigue S
Slide 19: HICKORY – improvement in physical function in the OLE
Bone & Endocrine Disorders HICKORY – efzimfotase alfa continued ≥12 yr, treatment-naive improvement in physical function in the OLE 30-second Sit-to-Stand Lower Extremity Functional Scale Improvement Improvement 19 Patients switching to efzimfotase alfa in the OLE had clinically meaningful improveme
Slide 20: HICKORY – improvement in pain and fatigue in the OLE
Bone & Endocrine Disorders HICKORY – efzimfotase alfa continued ≥12 yr, treatment-naive improvement in pain and fatigue in the OLE BPI-SF pain severity FACIT-Fatigue Improvement Improvement 20 Patients switching to efzimfotase alfa in the OLE had clinically meaningful improvements in BPI-SF pain sev
Slide 21: HICKORY – combined analysis of paediatric-onset subgroups
Bone & Endocrine Disorders HICKORY – combined analysis of ≥12 yr, treatment-naive prespecified paediatric-onset subgroups 30-second Lower Extremity BPI-SF Pain LS mean difference 6MWT FACIT-Fatigue Sit-to-Stand Functional Scale Severity vs placebo (SE), MCID: 31 meters Nominal P value MWPC:
Slide 22: Favourable safety across Phase III programme
Bone & Endocrine Disorders Favourable safety across Phase III programme 2–<12 yr, treatment-naive 2–<12 yr, open-label ≥12 yr, treatment-naive switch from asfotase alfa ―Efzimfotase alfa had a favourable safety and tolerability profile across all 3 trials, including patients switching from asfotase
Slide 23: Conclusions from HICKORY and broader programme
Bone & Endocrine Disorders Conclusions from HICKORY and broader programme ― In the HICKORY overall population, clinical benefit was observed in physical function, pain and fatigue, despite not meeting the primary endpoint of 6MWT ― Paediatric-onset subgroup: 6MWT showed nominally significant, clinic
Slide 24: efzimfotase alfa – opportunity beyond Strensiq
efzimfotase alfa – opportunity beyond Strensiq Marc Dunoyer CEO, ALEXION, ASTRAZENECA RARE DISEASE 24
Slide 25: Broader clinical evidence supporting patient reach across HPP spectrum
Bone & Endocrine Disorders HPP – efzimfotase alfa broader clinical evidence supporting patient reach across the spectrum of HPP ~14K addressable HPP patients in Top 8 1-4 Strensiq label | % approved to treat HPP patients Age of symptom onset5 Paediatric and adults with paediatric 20% ~80% onset 20%
Slide 26: Potential to expand the treatment opportunity beyond Strensiq
Bone & Endocrine Disorders HPP – efzimfotase alfa has the potential to expand the treatment opportunity beyond Strensiq Lower treatment Better tolerability Broader label Global expansion burden days per year free Enhanced manufacturing fewer injections Evidence generated across 12x per year 361 from
Slide 27: Establishing the pipeline for today and tomorrow
Establishing the pipeline for today and tomorrow Marc Dunoyer CEO, ALEXION, ASTRAZENECA RARE DISEASE 27
Slide 28: Multiple launch opportunities across high-value rare disease indications
Multiple launch opportunities across high-value and underserved rare disease indications efzimfotase alfa HICKORY/ I CAN TMA-313/4 PREVAIL CAEL-301/2 IgAN HSCT-TMA gMG MULBERRY/CHESTNUT AL-A HPP C5 inhibitor, dual binding Enzyme replacement C5 inhibitor, mAb Novel anti-fibril, mAb nanobody Fc fusion
Slide 29: Expanding Rare Disease growth opportunity through novel targets and modalities
Expanding Rare Disease growth opportunity through novel targets and modalities Haematology, Bone & Cardiology & Nephrology & Neurology Endocrine Rare Tumours Amyloidosis Transplant Disorders Ultomiris ALXN2230 eneboparatide cliramitug elrelesertib (multifunctional fusion (PTH1R agonist peptide) (AZD
Slide 30: Closing Remarks and Q&A Session
Closing Remarks and Q&A Session Key External Expert Dr. Raja Padidela CONSULTANT PAEDIATRIC ENDOCRINOLOGIST & METABOLIC BONE DISEASES, ROYAL MANCHESTER CHILDREN’S HOSPITAL AstraZeneca Leadership Marc Dunoyer CEO, ALEXION, ASTRAZENECA RARE DISEASE Gianluca Pirozzi SVP, HEAD OF DEVELOPMENT, REGULATORY
Slide 31: Appendix
Appendix 31
Slide 32: Glossary
Glossary 6MWT 6-Minute Walk Test IRA Inflation Reduction Act AAV Adeno-associated virus ISR Injection site reaction ADC Antibody-drug conjugate LEFS Lower Extremity Functional Scale AL-A Light-chain amyloidosis LS Least squares ALP Alkaline phosphatase mAb Monoclonal antibody ANCA Antineutrophil cyt
Slide 33: Confidentiality Notice
Confidentiality Notice This file is private and may contain confidential and proprietary information. If you have received this file in error, please notify us and remove it from your system and note that you must not copy, distribute or take any action in reliance on it. Any unauthorized use or dis