REG - AstraZeneca PLC - Alexion enters gene therapy agreement with Pfizer

AstraZenecaAnnouncement

28/07/2023 07:15

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RNS Number : 4880H  AstraZeneca PLC  28 July 2023

28 July 2023

 

Alexion, AstraZeneca Rare Disease, enters agreement with Pfizer to acquire a
portfolio of preclinical rare disease gene therapies

 

Agreement furthers Alexion's ambition to transform patient outcomes with
genomic medicine and provides synergistic opportunities across AstraZeneca

 

Portfolio includes preclinical gene therapy programmes and enabling
technologies with potential across several therapeutic areas

 

Alexion, AstraZeneca Rare Disease, today announced that it has entered a
definitive purchase and licence agreement for a portfolio of preclinical gene
therapy programmes and enabling technologies from Pfizer Inc. (Pfizer). The
agreement furthers Alexion and AstraZeneca's commitment to advancing
next-generation genomic medicines with the addition of complementary pipeline
assets and innovative technologies.

 

As part of the agreement, the transaction will bring to Alexion a number of
novel adeno-associated virus (AAV) capsids. AAV capsids have been shown to be
an effective mechanism for delivering therapeutic gene cargos for gene therapy
and gene editing.(1) These new resources build on Alexion and AstraZeneca's
combined capabilities in genomic medicine, recently strengthened with the
acquisition of LogicBio, with the objective to develop new genetic therapies
with improved safety and efficacy profiles. Additionally, Alexion will seek to
welcome talent from Pfizer associated with the portfolio.

 

Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease,
said: "Today's announcement represents another major step forward in Alexion
and AstraZeneca's ambition to be an industry leader in genomic medicine, which
has potential to be transformative and even curative for patients with
devastating diseases. We look forward to continuing our work to develop
enhanced platforms and technologies with broad therapeutic application while
integrating best-in-class expertise to accelerate promising therapeutics into
the clinic."

 

There are more than 7,000 known rare diseases, and around 80% of rare diseases
are believed to be caused by a genetic mutation.(2,3) Genomic medicines are
designed to treat or cure these diseases by addressing the malfunctioning
gene. This can be done through addition, alteration or inactivation of the
gene to help the body fight the disease.(4)

 

Financial considerations

Under the agreement, Alexion will purchase and licence the assets of Pfizer's
early-stage rare disease gene therapy portfolio for a total consideration of
up to $1bn, plus tiered royalties on sales.

 

Alexion plans to close the transaction in Q3 2023, subject to the satisfaction
of closing conditions.

Notes

Alexion

Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on
rare diseases, created following the 2021 acquisition of Alexion
Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years,
Alexion is focused on serving patients and families affected by rare diseases
and devastating conditions through the discovery, development and
commercialisation of life-changing medicines. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade and its
development efforts on haematology, nephrology, neurology, metabolic
disorders, cardiology and ophthalmology. Headquartered in Boston,
Massachusetts, Alexion has offices around the globe and serves patients in
more than 50 countries.

 

AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical
company that focuses on the discovery, development, and commercialisation of
prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals,
including Cardiovascular, Renal & Metabolism, and Respiratory &
Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries
and its innovative medicines are used by millions of patients worldwide.
Please visit astrazeneca.com (http://www.astrazeneca.com/) and follow the
Company on Twitter @AstraZeneca (https://twitter.com/AstraZeneca) .

 

Contacts

For details on how to contact the Investor Relations Team, please click here.
For Media contacts, click here
(https://www.astrazeneca.com/media-centre/contacts.html) .

 

References

1.   Wang D, et al. Adeno-associated virus vector as a platform for gene
therapy delivery. Nat Rev Drug Discov. 2019 May;18(5):358-378.

2.   FDA. Rare Diseases at FDA. Available at:
https://www.fda.gov/patients/rare-diseases-fda. Accessed July 2023.

3.   European Commission. Expert Group on Public Health. Rare diseases.
Available at:
https://health.ec.europa.eu/non-communicable-diseases/expert-group-public-health/rare-diseases_en.
Accessed July 2023.

4.   FDA. How Gene Therapy Can Cure or Treat Diseases. Available at:
https://www.fda.gov/consumers/consumer-updates/how-gene-therapy-can-cure-or-treat-diseases.
Accessed July 2023.

 

Adrian Kemp

Company Secretary

AstraZeneca PLC

 

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