REG - AstraZeneca PLC - AstraZeneca and Neurimmune sign deal for NI006

AstraZenecaAnnouncement

07/01/2022 07:00

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RNS Number : 7555X  AstraZeneca PLC  07 January 2022

07 January 2022 07:00 GMT

 

AstraZeneca and Neurimmune sign exclusive global collaboration and licence
agreement to develop and commercialise NI006

 

Investigational human monoclonal antibody in Phase Ib development for the
treatment of transthyretin amyloid cardiomyopathy, a systemic, progressive and
fatal condition

 

Alexion, AstraZeneca's Rare Disease group, has entered into an exclusive
global collaboration and licence agreement with Neurimmune AG for NI006, an
investigational human monoclonal antibody currently in Phase Ib development
for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). NI006
specifically targets misfolded transthyretin and is designed to directly
address the pathology of ATTR-CM by enabling removal of amyloid fibril
deposits in the heart, with the potential to treat patients with advanced
ATTR-CM.

 

Under the agreement, Alexion will be granted an exclusive worldwide licence to
develop, manufacture and commercialise NI006.

 

ATTR-CM is a systemic, progressive and fatal condition that leads to
progressive heart failure and high rate of fatality within four years from
diagnosis.(1) It remains underdiagnosed and its prevalence is thought to be
underestimated due to a lack of disease awareness and the heterogeneity of
symptoms.(2)

 

Marc Dunoyer, Chief Executive Officer, Alexion, said: "With 30 years of
experience in developing medicines for people with rare diseases, Alexion is
uniquely positioned to advance innovative science for small patient
populations who are frequently underdiagnosed. We look forward to applying
this expertise to the development of NI006, which is designed to clear cardiac
amyloid fibril deposits with the potential to improve cardiac function for
patients living with advanced ATTR-CM, who are currently underserved by
existing treatment options."

 

There is a significant unmet medical need for patients with various types and
levels of severity of amyloidosis that may require multiple mechanisms of
action to address those needs. NI006, an ATTR depleter, adds a novel and
complementary approach to AstraZeneca and Alexion's pipeline of
investigational therapies focused on amyloidosis and strengthens our broader
commitment to addressing cardiomyopathies that can lead to heart failure.

 

Financial considerations

Alexion will pay Neurimmune an upfront payment of $30m with the potential for
additional contingent milestone payments of up to $730m upon achievement of
certain development, regulatory and commercial milestones, as well as
low-to-mid teen royalties on net sales of any approved medicine resulting from
the collaboration.

 

Neurimmune will continue to be responsible for completion of the current Phase
Ib clinical trial on behalf of Alexion, and Alexion will pay certain trial
costs. Alexion will be responsible for further clinical development,
manufacturing and commercialisation.

 

The transaction is expected to close following satisfaction of customary
closing conditions and regulatory clearances.

 

Notes

 

Heart failure

Heart failure (HF) is a life-threatening chronic disease that prevents the
heart from pumping sufficient levels of blood around the body. HF affects
approximately 64 million people worldwide. HF remains as fatal as some of the
most common cancers in both men (prostate and bladder cancers) and women
(breast cancer).(5) Chronic HF is the leading cause of hospitalisation for
those over the age of 65 and represents a significant clinical and economic
burden.(6)

( )

AstraZeneca's ambition is to be the leading company in HF, expanding from
Forxiga today in heart failure with reduced ejection fraction (HFrEF), to the
full HF spectrum including cardiomyopathies. AstraZeneca is investing in
multiple investigational therapies with diverse mechanisms of action to
address the spectrum of patient need in this area.

 

ATTR-CM

Cardiomyopathy due to ATTR is caused by aging or genetic mutations resulting
in misfolded TTR protein and accumulation as amyloid fibrils in the cardiac
myocardium. In patients with ATTR-CM, both the mutant and wild type TTR
protein builds up as fibrils in tissues, including the heart. The presence of
TTR fibrils interferes with the normal functions of these tissues. As the TTR
protein fibrils enlarge, more tissue damage occurs and the disease worsens,
resulting in poor quality of life and eventually death.

 

Worldwide, there are an estimated 300,000-500,000 patients with ATTR-CM(3,4);
however, many of those patients remain undiagnosed.

 

NI006

NI006 is an investigational human monoclonal antibody that specifically
targets misfolded transthyretin and is designed to directly address the
pathology of ATTR-CM by enabling removal of amyloid fibril deposits in the
heart.

 

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
(https://www.astrazeneca.com/investor-relations.html#Contacts) . For Media
contacts, click here (https://www.astrazeneca.com/media-centre/contacts.html)
.

References

1.   Lauppe RE, et al. Nationwide prevalence and characteristics of
transthyretin amyloid cardiomyopathy in Sweden. Open Heart. 2021
Oct;8(2):e001755. doi: 10.1136/openhrt-2021-001755.

2.   González-Duarte A, et al. Impact of non-cardiac clinicopathologic
characteristics on survival in transthyretin amyloid polyneuropathy. Neurol
Ther. 2020;9(1):135-149. doi:10.1007/s40120-020-00183-7.

3.   Mohamed-Salem L, et al. Prevalence of wild type ATTR assessed as
myocardial uptake in bone scan in the elderly population. Int J Cardiol. 2018
Nov 1;270:192-196. doi: 10.1016/j.ijcard.2018.06.006.

4.   Cuscaden C, et al. Estimation of prevalence of transthyretin (ATTR)
cardiac amyloidosis in an Australian subpopulation using bone scans with
echocardiography and clinical correlation. J Nucl Cardiol. 2020 May 8. doi:
10.1007/s12350-020-02152-x.

5.   Mamas MA et al. Do Patients Have Worse Outcomes in Heart Failure than
in Cancer? A Primary Care-Based Cohort Study with 10-year Follow-up in
Scotland. Eur J Heart Fail 2017; 19(9):1095-104.

6.   Azad N, Lemay G. Management of Chronic Heart Failure in the Older
Population. J Geriatr Cardiol 2014; 11(4):329-37.

Adrian Kemp

Company Secretary

AstraZeneca PLC

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