TIDMAZN
RNS Number : 8023U
AstraZeneca PLC
07 December 2021
7 December 2021 07:00 GMT
AstraZeneca and Ionis sign deal to develop and commercialise
eplontersen
Liver-targeted antisense therapy in Phase III development for
the treatment of transthyretin amyloidosis, a systemic, progressive
and fatal condition
AstraZeneca has entered into a new global development and
commercialisation agreement with Ionis Pharmaceuticals, Inc.
(Ionis) for eplontersen, formerly known as IONIS-TTR-L(RX.)
Eplontersen is a ligand-conjugated antisense investigational
medicine currently in Phase III clinical trials for amyloid t
ransthyretin cardiomyopathy (ATTR-CM) and amyloid t ransthyretin
polyneuropathy (ATTR-PN). It is designed to reduce the production
of transthyretin (TTR protein) to treat both hereditary and
non-hereditary forms of TTR amyloidosis (ATTR).
The companies will jointly develop and commercialise eplontersen
in the US, while AstraZeneca will develop and commercialise it in
the rest of the world, except in Latin America.
ATTR-CM is a systemic, progressive and fatal condition that
leads to progressive heart failure and death 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) Hereditary ATTR-PN is a
debilitating disease that leads to peripheral nerve damage with
motor disability within five years of diagnosis and, without
treatment, is generally fatal within a decade.(3)
Mene Pangalos, Executive Vice President, BioPharmaceuticals
R&D, AstraZeneca, said: "Eplontersen has the potential to halt
the progression of TTR-mediated amyloidosis, irrespective of
whether it's caused by genetic mutations or aging. Thanks to its
precise liver-targeting properties, it also has the potential to be
a best-in-class treatment for patients suffering from this
devastating disease and who currently have limited options."
Hereditary ATTR-PN is expected to be the first indication for
which the companies will seek regulatory approval for eplontersen,
with the potential to file a new drug application with the US Food
and Drug Administration by the end of 2022.
Financial considerations
AstraZeneca will pay Ionis an upfront payment of $200m and
additional conditional payments of up to $485m following regulatory
approvals. It will also pay up to $2.9bn of sales-related
milestones based on sales thresholds between $500m and $6bn, plus
royalties in the range of low double-digit to mid-twenties
percentage depending on the region. The collaboration includes
territory-specific development, commercial and medical affairs
cost-sharing provisions.
The transaction will be funded with cash and is expected to be
neutral to Core earnings in 2021. It will be accounted for as an
intangible asset acquisition, recognised initially at the upfront
amount, with any potential future milestone payments capitalised
into the intangible asset as they are recognised.
Ionis will continue to manufacture and supply eplontersen for
the existing clinical studies and process qualification.
AstraZeneca will be responsible for commercial supply, with
transition timing to be agreed by both parties. AstraZeneca will
book all sales generated under the agreement.
The transaction is expected to close in the fourth quarter of
2021, subject to customary closing conditions and regulatory
clearances. The transaction does not impact the AstraZeneca's
financial guidance for 2021.
Notes
Eplontersen
Eplontersen is a ligand-conjugated antisense (LICA)
investigational medicine designed to reduce the production of
transthyretin, or TTR protein, to treat all types of ATTR, a
systemic, progressive and fatal disease.
TTR Amyloidosis (ATTR)
Cardiomyopathy and polyneuropathy due to ATTR are caused by
aging or genetic mutations resulting in misfolded TTR protein and
accumulation as amyloid fibrils in the cardiac myocardium and
peripheral nerves, respectively. In patients with ATTR, both the
mutant and wild type TTR protein builds up as fibrils in tissues,
such as the peripheral nerves, heart, gastrointestinal system,
eyes, kidneys, central nervous system, thyroid and bone marrow. 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(4,5) and 10,000 - 40,000
patients with ATTR-PN(2) .
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 and follow the
Company on Twitter @AstraZeneca .
Contacts
For details on how to contact the Investor Relations Team,
please click here . For Media contacts, click here .
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. Cortese A, et al. Diagnostic challenges in hereditary
transthyretin amyloidosis with polyneuropathy: avoiding
misdiagnosis of a treatable hereditary neuropathy. J Neurol
Neurosurg Psychiatry. 2017 May;88(5):457-458. doi:
10.1136/jnnp-2016-315262.
4. 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.
5. 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.
Adrian Kemp
Company Secretary
AstraZeneca PLC
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