14 March, 2024
AstraZeneca to
acquire Amolyt
Pharma,
expanding late-stage rare
disease pipeline
Amolyt
development portfolio includes Phase III asset with potential to
address significant unmet need in patients with chronic
hypoparathyroidism
Acquisition to further expand the Alexion, AstraZeneca Rare
Disease pipeline beyond complement inhibition, building on
company's success in bone metabolism and opportunity in rare
endocrinology
AstraZeneca announced that it has entered into
a definitive agreement to acquire Amolyt
Pharma, a clinical-stage biotechnology company focused
on developing novel treatments for rare endocrine
diseases.
The proposed acquisition will bolster the Alexion,
AstraZeneca Rare Disease late-stage pipeline and expand on its bone
metabolism franchise with the notable addition of eneboparatide
(AZP-3601), a Phase III investigational therapeutic peptide
with a novel mechanism of action designed to meet key therapeutic
goals for hypoparathyroidism. Additionally, Alexion is looking
forward to welcoming talent from Amolyt Pharma.
In patients with
hypoparathyroidism, a deficiency in parathyroid hormone (PTH)
production results in significant dysregulation of calcium and
phosphate, which can lead to life-altering symptoms and
complications, including chronic kidney disease.1 It is
one of the largest known rare diseases, affecting an estimated
115,000 people in the United States and 107,000 people
in the European Union, approximately 80% of whom are
women.2,3
Marc Dunoyer, Chief Executive Officer, Alexion,
AstraZeneca Rare Disease, said: "Chronic
hypoparathyroid patients face a significant need for an alternative
to current supportive therapies, which do not address
the underlying hormone deficiency. As leaders in rare
disease, Alexion is uniquely positioned to drive the late-stage
development and global commercialisation of
eneboparatide, which has the potential to lessen the often debilitating impact of low parathyroid
hormone and avoid the risks of high-dose calcium
supplementation. We believe this programme, together
with Amolyt's talented team, expertise
and earlier pipeline, will enable our expansion into rare
endocrinology."
Thierry Abribat, Chief
Executive Officer, Amolyt Pharma, said: "We enthusiastically
welcome the proposed acquisition of Amolyt by AstraZeneca, an
organisation that shares our dedication to delivering life-changing
treatments to people living with rare diseases. This agreement
offers the opportunity to meaningfully advance our pipeline
therapies. Strong Phase II data suggest eneboparatide has the
potential to improve outcomes for patients and to shift the
treatment paradigm for hypoparathyroidism, and we look forward to
seeing the continued advancement of the Phase III
trial."
Eneboparatide is a PTH receptor 1 (PTHR1) agonist
with a novel mechanism of action rationally designed to meet the
therapeutic goals of hypoparathyroidism.4 Phase II data
showed that eneboparatide achieved normalisation of serum calcium
levels as well as the potential to eliminate dependence on daily
calcium and vitamin D supplementation. In adults with chronic
hypoparathyroidism and hypercalciuria, results showed that
eneboparatide normalised calcium in urine. In addition, for
patients with hypoparathyroidism, eneboparatide preserved bone
mineral density, an important potential benefit in patients
with an increased risk of osteopenia or
osteoporosis.5
Financial
considerations
Under the terms of the agreement,
AstraZeneca will acquire all of Amolyt Pharma's
outstanding shares for a total consideration of up to $1.05
billion, on a cash and debt free basis. This includes $800 million
upfront at deal closing, plus the right for Amolyt
Pharma's shareholders to receive an additional
contingent payment of $250 million payable upon achievement of a
specified regulatory milestone.
Subject to the satisfaction of customary
closing conditions in the acquisition agreement, including
regulatory clearances, the transaction is expected to close by the
end of the third quarter of 2024.
Notes
Eneboparatide
(AZP-3601)
Eneboparatide (AZP-3601) is an
investigational therapeutic peptide designed to bind with high
affinity to a specific conformation of the parathyroid hormone
(PTH) receptor 1. Therapeutic goals include: regulating and
maintaining serum calcium levels in the normal range, and thereby
managing the symptoms of hypoparathyroidism; limiting urine calcium
excretion by restoring calcium reabsorption by the kidney; and
potentially preventing progressive decline in kidney function and
the development of chronic kidney disease. Eneboparatide is also
designed to have a short plasma half-life to potentially restore
bone turnover to a more physiologic state and to help preserve bone
integrity.
Hypoparathyroidism
Hypoparathyroidism is a rare condition defined by a
deficiency of parathyroid hormone that results in decreased calcium
and elevated phosphorus levels in the blood.1
Approximately 80% of the estimated 115,000 people in the United
States and 107,000 in the European Union with hypoparathyroidism
are women.2,3 Despite available treatments, patients
experience persistent, life-altering symptoms and often develop
complications and comorbidities that diminish quality of life and
create segments of the patient population with specific clinical
needs. Clinical manifestations of hypoparathyroidism impact many
tissues and organ systems, in particular, the kidneys and
bone.6,7
Forward-looking
statements
This announcement may include statements that are not
statements of historical fact, or "forward-looking statements,"
including with respect to AstraZeneca's proposed acquisition of
Amolyt Pharma. Such forward-looking statements
include, but are not limited to, the ability of AstraZeneca and
Amolyt Pharma to complete the transactions
contemplated by the acquisition agreement, including the parties'
ability to satisfy the conditions set forth in the acquisition
agreement, statements about the expected timetable for completing
the transaction, AstraZeneca's and Amolyt Pharma's beliefs and
expectations and statements about the benefits sought to be
achieved in AstraZeneca's proposed acquisition of Amolyt Pharma,
the potential effects of the acquisition on both AstraZeneca and
Amolyt Pharma, the possibility of any termination of the
acquisition agreement, as well as the expected benefits and success
of eneboparatide (AZP-3601). These statements are based upon the
current beliefs and expectations of AstraZeneca's and Amolyt
Pharma's management and are subject to significant risks and
uncertainties. There can be no guarantees that the conditions to
the closing of the proposed transaction will be satisfied on the
expected timetable or at all or that eneboparatide (AZP-3601) will
receive the necessary regulatory approvals or prove to be
commercially successful if approved. If underlying assumptions
prove inaccurate or risks or uncertainties materialise, actual
results may differ materially from those set forth in the
forward-looking statements. Risks and uncertainties include, but
are not limited to, uncertainties as to the timing of the
acquisition; the possibility that various conditions to the
consummation of the acquisition contemplated by the acquisition
agreement may not be satisfied or waived; the ability to obtain
necessary regulatory approvals or to obtain them on acceptable
terms or within expected timing; the effects of disruption from the
transactions contemplated by the acquisition agreement and the
impact of the announcement and pendency of the transactions on
Amolyt Pharma's business; the risk that shareholder litigation in
connection with the offer or the acquisition may result in
significant costs of defence, indemnification and liability; the
possibility that the achievement of the specified milestone
described in the acquisition agreement may take longer to achieve
than expected or may never be achieved and the resulting contingent
milestone payment may never be realised; general industry
conditions and competition; general economic factors, including
interest rate and currency exchange rate fluctuations; the impact
of COVID-19; the impact of pharmaceutical industry regulation and
health care legislation in the United States and internationally;
competition from other products; and challenges inherent in new
product development, including obtaining regulatory approval.
Neither AstraZeneca nor Amolyt Pharma undertakes any
obligation to publicly update any forward-looking statement,
whether as a result of new information, future events or otherwise,
except to the extent required by law. Additional factors that could
cause results to differ materially from those described in the
forward-looking statements can be found in AstraZeneca's Annual
Report on Form 20-F for the year ended 31 December 2023, as amended
by any subsequent filings made with the SEC. These and other
filings made by AstraZeneca with the SEC are available at
www.sec.gov.
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 more than 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 research efforts on
novel molecules and targets that include the complement cascade,
and development efforts in haematology, nephrology, neurology,
metabolic disorders, cardiology and ophthalmology. Headquartered in
Boston, Massachusetts, Alexion has offices around the globe and
serves patients in more than 70 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 and
follow the Company on social media @AstraZeneca.
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References
1. Clarke BL, et al.
Epidemiology and diagnosis of hypoparathyroidism. J Clin Endocrinol Metab.
2016;101(6):2284-99.
2. Vadiveloo, T, et al.
A Population-based study of the Epidemiology of Chronic
Hypoparathyroidism. J Bone Miner
Res. 2018;33(3):478-485.
3. Villarroya-Marquina I, et al. Influence of gender and women's age on the prevalence of
parathyroid failure after total thyroidectomy for multinodular
goiter. Gland Surg.
2020;9(2):245-251.
4. Khan A, et al.
Evaluation and management of hypoparathyroidism summary statement
and guidelines from the second international workshop.
J Bone Miner
Res. 2022;37(12):2568-2585.
5. Kamenicky P, et al.
OR23-04 Treatment of chronic hypoparathyroidism with eneboparatide
(AZP-3601), a novel PTH 1 receptor agonist: results from a phase 2
trial. Journal of the Endocrine Society.
2023;7(Supplement_1): bvad114.562.
6. Bilezikian JP.
Hypoparathyroidism. J Clin
Endocrinol Metab. 2020;105(6):1722-36.
7. Abate EG, et al.
Review of Hypoparathyroidism. Front Endocrinol (Lausanne).
2017;7:172.
Adrian Kemp
Company Secretary
AstraZeneca PLC