Growth Opportunities in Rare Diseases

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1 month ago 107

Category: Business | Tags: #HealthcareInnovation #PharmaceuticalGrowth #AstraZeneca #InvestorDay2024 #RareDiseases

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AstraZeneca's rare disease strategy focuses on complement-mediated diseases, bone and endocrine disorders, and amyloidosis.
Key therapeutic areas of focus include neurology, renal indications, and transplantology, with expansion plans for Ultomiris.
The company is leveraging its global footprint, particularly in emerging markets like China, to address underserved rare disease populations.
Innovation in genomic medicines and new modalities are crucial for transformative and potentially curative therapies.
AstraZeneca aims to advance up to 2 INDs (Investigational New Drug applications) per year to 2030 in the genomic medicine space.

#HealthcareInnovation #PharmaceuticalGrowth #AstraZeneca #InvestorDay2024 #RareDiseases

Growth Opportunities in Rare Diseases

@financepresentations1 month 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

6/31

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…

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Investor Day • 2024
Future innovation planned in complement
16 Acronym definitions can be found i…

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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

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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Growth Opportunities in Rare Diseases

@financepresentations

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Growth Opportunities in Rare Diseases

Please summarize the main topics of these documents for me.

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