Growth Opportunities in Rare Diseases

Growth Opportunities in Rare Diseases

@financepresentations
@financepresentations
7 Followers
1 week ago 41

This session highlights AstraZeneca's strategic focus on rare diseases, addressing growth opportunities through innovative therapies. Key insights from leaders demonstrate how emerging markets and new medicines are paving the way for sustained revenue growth. Anticipated product launches and a robust pipeline are crucial for long-term success, particularly amid evolving patient needs and market dynamics.

Growth Opportunities in Rare Diseases

@financepresentations1 week ago

Investor Day • 2024
Rare Disease
Marc Dunoyer, CEO, Alexion and Chief Strategy Officer, AstraZene…
1/31
Investor Day • 2024
Forward looking statements
2
In order, among other things, to utilise the 's…
2/31
Investor Day • 2024
FY 2022 growth rates on medicines acquired with Alexion have been calculated o…
3/31
Investor Day • 2024
Rare Disease patient growth (%)
FY 2022 growth rates on medicines acquired wi…
4/31
Investor Day • 2024
Emerging Markets growth leveraging AstraZeneca global 
footprint
5
FY 2022 …
5/31
6 Investor Day • 2024
Complement
6/31
Investor Day • 2024
C5 patient growth by indication (%)
Sustainable growth of C5 Franchise
7
*P…
7/31
Investor Day • 2024
Neurology driving C5 growth in the short- to mid-term
8
1. Internal estimate…
8/31
Investor Day • 2024
gMG – expanding reach with next-generation gefurulimab
9 Epidemiology refers …
9/31
Investor Day • 2024
gMG – forecasted market evolution across Top 7 countries
10
1. Top 7 = US, E…
10/31
Investor Day • 2024
Ultomiris indication expansion, maximising 
complement-mediated and adjacent …
11/31
Investor Day • 2024
HSCT-TMA – potential for Ultomiris in rare, life-threatening 
complication of…
12/31
Investor Day • 2024
IgAN – potential to transform course of disease with 
complement inhibition 
…
13/31
Investor Day • 2024
IgAN – rapid and sustained proteinuria reduction 
in Phase II supports Phase …
14/31
Investor Day • 2024
Future plans to improve success rates of kidney transplant
15
1. Carrie A Sc…
15/31
Investor Day • 2024
Future innovation planned in complement
16 Acronym definitions can be found i…
16/31
17 Investor Day • 2024
Beyond Complement:
bone and endocrine disease
17/31
Investor Day • 2024
Hypophosphatasia – building on Strensiq, 
efzimfotase alfa drives innovation …
18/31
Investor Day • 2024
>250K patients in Top 8 countries1
>50% are peri- or postmenopausal women 
…
19/31
20 Investor Day • 2024
Beyond Complement:
amyloidosis
20/31
Investor Day • 2024
Novel anti-fibril depleter mechanisms with potential 
to restore normal organ…
21/31
Investor Day • 2024
a
Leveraging CVRM and Rare Disease expertise in ATTR-CM 
22 1. Alexion, Astr…
22/31
Investor Day • 2024
a
Amyloidosis (ATTR-CM) – depleter mechanism 
with the potential to reverse …
23/31
Investor Day • 2024
Amyloidosis (AL) – transforming patient outcomes 
with potential first-in-cla…
24/31
26 Investor Day • 2024
Pipeline and new 
modalities
25/31
Investor Day • 2024
Genomic medicines – unlocking transformative and 
potentially curative therap…
26/31
Investor Day • 2024
Several major assets with blockbuster potential support 
our growth ambition
…
27/31
Investor Day • 2024
Q&A session
Marc Dunoyer
CHIEF EXECUTIVE OFFICER, 
ALEXION
Gianluca Pirozz…
28/31
30 Investor Day • 2024
Appendix
29/31
31 Investor Day • 2024
Glossary – 1 of 2
CLL chronic lymphocytic leukaemia
cm centimetre
CM car…
30/31
32 Investor Day • 2024
Glossary – 2 of 2
NST neoadjuvant systemic treatment
NT-proBNP N-terminal…
31/31


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