ASTRAZENECA PLC Rare Disease
Marc Dunoyer, CEO, Alexion and Chief Strategy Officer, AstraZeneca Gianluca Pirozzi, SVP, Head of Development and Safety, Alexion
Investor Day • 2024
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 with respect to the operations, performance and financial condition of the Group, including, among other things, statements about expected or targeted revenues, margins, earnings per share or other financial or other measures (including the Financial Ambition Statements described in this presentation). Although the Group believes its expectations and targets are based on reasonable assumptions and has used customary forecasting methodologies used in the pharmaceutical industry and risk-adjusted projections for individual medicines (which take into account the probability of success of individual clinical trials, based on industry-wide data for relevant clinical trials at a similar stage of development), any forward-looking statements, by their very nature, involve risks and uncertainties and may be influenced by factors that could cause actual outcomes and results to be materially different from those predicted. The forward-looking statements reflect knowledge and information available at the date of preparation of this document and the Group undertakes no obligation to update these forward-looking statements. The Group identifies the forward-looking statements by using the words 'anticipates', 'believes', 'expects', 'intends' and similar expressions in such statements. Important factors that could cause actual results to differ materially from those contained in forward-looking statements, certain of which are beyond the Group's control, include, among other things: the risk of failure or delay in delivery of pipeline or launch of new medicines; the risk of failure to meet regulatory or ethical requirements for medicine development or approval; the risk of failures or delays in the quality or execution of the Group's commercial strategies; the risk of pricing, affordability, access and competitive pressures; the risk of failure to maintain supply of compliant, quality medicines; the risk of illegal trade in the Group's medicines; the impact of reliance on third-party goods and services; the risk of failure in information technology or cybersecurity; the risk of failure of critical processes; the risk of failure to collect and manage data in line with legal and regulatory requirements and strategic objectives; the risk of failure to attract, develop, engage and retain a diverse, talented and capable workforce; the risk of failure to meet regulatory or ethical expectations on environmental impact, including climate change; the risk of the safety and efficacy of marketed medicines being questioned; the risk of adverse outcome of litigation and/or governmental investigations; intellectual property-related risks to the Group's products; the risk of failure to achieve strategic plans or meet targets or expectations; the risk of failure in financial control or the occurrence of fraud; the risk of unexpected deterioration in the Group's financial position; the impact that global and/or geopolitical events may have, or continue to have, on these risks, on the Group's ability to continue to mitigate these risks, and on the Group's operations, financial results or financial condition There can be no guarantees that the conditions to the closing of the proposed transaction with Fusion will be satisfied on the expected timetable, or at all, or that "FPI-2265"(Ac225-PSMA I&T) or any combination product will receive the necessary regulatory approvals or prove to be commercially successful if approved. There can be no guarantees that the conditions to the closing of the proposed transaction with Amolyt Pharma will be satisfied on the expected timetable, or at all, or that eneboparatide ('AZP-3601') will receive the necessary regulatory approvals or prove to be commercially successful if approved.
This presentation includes references to new molecular entities and life-cycle management programmes that are being investigated in current or future clinical trials, and as such have not been approved by any regulatory agency. For a list of new molecular entities and indications in development, see pages 7-11 of the Clinical Trials Appendix that accompanied AstraZeneca's Q1 2024 results.
Basis of AstraZeneca ambitions, forecasts and targets
AstraZeneca ambitions, forecasts and targets in this presentation (the "Financial Ambition Statements") are derived from AstraZeneca's most recent risk-adjusted mid- and long-term plans, adjusted for developments in the business since those plans were finalised. Financial Ambition Statements presented are based on management's risk-adjusted projections for individual medicines and individual clinical trials. Estimates for these probabilities are based on industry-wide data for relevant clinical trials in the pharmaceutical industry at a similar stage of development adjusted for management's view on the risk profile of the specific asset. The peak year revenue (PYR) potential for individual medicines referred to in this presentation are the maximum estimated Total Revenue to be recognised by AstraZeneca in a single calendar year, during the lifecycle of the medicine, and are based on management's latest non-risk adjusted forecast estimates. Estimates are based on customary forecasting methodologies used in the pharmaceutical industry. Peak year revenue may occur in different years for each NME depending on trial outcomes, approval label, competition, launch dates and exclusivity periods, amongst other variables. The peak year revenue figures are derived from net sales at nominal values and are not risk-adjusted or time-value discounted. The development of pharmaceutical products has inherent risks given scientific experimentation and there are a range of possible outcomes in clinical results, safety, efficacy and product labelling. Clinical results may not achieve the desired product profile and competitive environment, pricing and reimbursement may have material impact on commercial revenue forecasts. By their nature, forecasts are based on a multiplicity of assumptions and actual performance in future years may vary, significantly and materially, from these assumptions. The Financial Ambition Statements in this presentation are based on Q1 2024 exchange rates; AZ undertakes no obligation to update those statements based on future currency movements
2 | Investor Day • 2024 |
Rare Disease - next wave of growth to 2030 and beyond
Illustrative only, not to scale
Beyond 2030 | |
Launching key NMEs | ALXN2220 |
Genomic medicines |
Existing | anselamimab | |
portfolio | eneboparatide1 | |
efzimfotase alfa | ||
$7.1bn | $7.8bn | gefurulimab |
12% | ||
10% |
Lost of Exclusivity (Soliris)
2022 | 2023 | 2030 |
Complement
continued leadership
Beyond Complement focused on first- and/or best-in-classmedicines
Technologies
investing in new, potentially curative, modalities
FY 2022 growth rates on medicines acquired with Alexion have been calculated on a pro forma basis comparing to the corresponding period in the prior year. In FY 2022 Total Revenue from Koselugo is included in Rare Disease (FY 2021: Oncology) and Total Revenue from Andexxa is included in BioPharmaceuticals: CVRM (FY 2021: Rare Disease). The growth rate shown has been calculated as though these changes had been implemented in FY 2021.
- 1. Amolyt acquisition remains subject to customary external clearances; all clinical development plans mentioned herein subject to deal closure. All growth rates at CER. Acronym definitions can be found in Glossary.
Investor Day • 2024
Alexion has delivered sustainable and robust growth since acquisition
Rare Disease Total Revenue growth (%) | Rare Disease patient growth (%) |
25%
21%
18%
16%
10% 12%
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 comparing to the corresponding period in the prior year. In FY 2022 Total Revenue from Koselugo is included in Rare Disease (FY 2021: Oncology) and Total Revenue from Andexxa is included in BioPharmaceuticals: CVRM (FY 2021: Rare Disease). The growth rate shown has been calculated as though these changes had been implemented in FY 2021. All growth rates at CER.
- Acronym definitions can be found in Glossary.
Investor Day • 2024
Emerging Markets growth leveraging AstraZeneca global footprint
Emerging Markets Total Revenue growth (%)
62%73%
6%
2022 | 2023 | Q1 2024 |
Launching in new countries globally
Strategic opportunity in China
Leveraging AstraZeneca's presence to address underserved rare populations in China
2021
2023
2030
National | |||
Reimbursement | PNH | aHUS | NF1-PN |
Drug List | |||
20 70 100
countries countries countries
gMG |
FY 2022 growth rates on medicines acquired with Alexion have been calculated on a pro forma basis comparing to the corresponding period in the prior year. In FY 2022 Total Revenue from Koselugo is included in Rare Disease (FY 2021: Oncology) and Total Revenue from Andexxa is included in BioPharmaceuticals: CVRM (FY 2021: Rare Disease). The growth rate shown has been calculated as though these changes had been implemented in FY 2021. All growth rates at CER.
- Acronym definitions can be found in Glossary.
Investor Day • 2024
Complement
6 | Investor Day • 2024 |
Sustainable growth of C5 Franchise
C5 Total Revenue growth (%)
$5bn+*
7%8%
10%
Rapid conversion from
Soliris to Ultomiris
2022 | 2023 | Q1 2024 |
Superior efficacy with rapid, sustained complement inhibition and steroid sparing data
C5 patient growth by indication (%)
8%
2022
PNH
20% | Geographic reach leveraging | ||||
17% | well-established AstraZeneca network | ||||
Indication expansion to maximise | |||||
2023 | Q1 2024 | complement-mediated and adjacent | |||
aHUS | Neurology | diseases |
*Peak Year Revenues, non-risk adjusted. FY 2022 growth rates on medicines acquired with Alexion have been calculated on a pro forma basis comparing to the corresponding period in the prior year. Neurology includes gMG and NMOSD. In | |
FY 2022 Total Revenue from Koselugo is included in Rare Disease (FY 2021: Oncology) and Total Revenue from Andexxa is included in BioPharmaceuticals: CVRM (FY 2021: Rare Disease). The growth rate shown has been calculated as though | Investor Day • 2024 |
7 these changes had been implemented in FY 2021. All growth rates at CER. Acronym definitions can be found in Glossary. |
Neurology driving C5 growth in the short- to mid-term
Neurology Total Revenue growth (%)1
23%
15%
11%
2022 | 2023 | Q1 2024 |
Neurology patient growth (%)
40%42%
15%
2022 | 2023 | Q1 2024 |
Sustained long-term improvements
88% of Ultomiris patients saw clinically meaningful improvements in activities of daily living to Week 1642
Steroid sparing data >75% of Soliris
patients were on low dose steroids after 2 years3
Vs competition (FcRN) switch dynamic
has consistently favoured Ultomiris since Q3 20234
Long-term evidence of superior efficacy in gMG
1. Internal estimated growth rates. 2. Vu et al. AAN 13-18 April 2024 Long term efficacy and safety of ravulizumab, a long-acting terminal complement inhibitor, in adults with AChR Ab+ generalized myasthenia gravis: Final results from the Phase 3 CHAMPION MG open-label extension. 3. Pulley M. et al., AANEM 2023 Annual Meeting, November 1-4, 2023, Phoenix, AZ, USA. 4. Based on raw claims data from Komodo Prism 17 April 2024. Neurology includes gMG and NMOSD.
- Acronym definitions can be found in Glossary.
Investor Day • 2024
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 | Soliris | |||||
immuno-suppressant | ||||||
Ultomiris | corticosteroids | 1st immuno- | 2nd immuno- | orIVIg/PP/PLEX | ||
or | ||||||
biologic of choice for | suppressant | suppressant | ||||
a | Soliris | |||||
Q8W | population | |||||
broader a | ||||||
gefurulimab | Ultomiris | |||||
QW | (ALXN1720) | |||||
potential best-in-class | gefurulimab (ALXN1720) | |||||
self-administrative s.c. option |
9 Epidemiology refers to diagnosed US AChR+ gMG patients. Acronym definitions can be found in Glossary. | Investor Day • 2024 |
gMG - forecasted market evolution across Top 7 countries
gMG market evolution across Top 7 countries1
100%
90%
80%
70%
60%
50%
40%
30%
20%
10%
0%
2022 | 2023 | 2024 | 2025 | 2026 | 2027 | 2028 | 2029 | 2030 | 2031 | 2032 | 2033 | 2034 |
Majority of market | New branded | Continued growth | unbranded | |||||||||
and off-label | ||||||||||||
unbranded | market entrants | of branded market | branded |
80% of patients to move to
branded medicines by 20342
gMG launched in 31 countries
with additional 25 countries
by 2025
We anticipate patients
treated with Ultomiris, Soliris
and gefurulimab to grow significantly over the next 10 years
1. Top 7 = US, EU4 (Germany, France, Italy, Spain), Japan and Canada; based on market research and internal forecasts. 2. Branded products include non-steroid and non-immunosuppressants. 3. Excluding China, from 2024-2034. Acronym 10 definitions can be found in Glossary.
Investor Day • 2024
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AstraZeneca plc published this content on 21 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 21 May 2024 12:39:04 UTC.
