FORM 6-K
SECURITIES
AND EXCHANGE COMMISSION
Washington,
D.C. 20549
Report
of Foreign Issuer
Pursuant
to Rule 13a-16 or 15d-16 of
the
Securities Exchange Act of 1934
For the
month of March 2025
Commission
File Number: 001-11960
AstraZeneca PLC
1
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AstraZeneca PLC
INDEX
TO EXHIBITS
1.
Eneboparatide Phase III trial met primary endpoint
17 March 2025
Eneboparatide met primary endpoint of normalising serum calcium in
adults with hypoparathyroidism at 24 weeks in CALYPSO Phase III
trial
Trial
continues as planned to 52 weeks to further characterise the
risk-benefit profile
High-level results from the CALYPSO Phase III trial showed that
eneboparatide (AZP-3601), an investigational parathyroid
hormone (PTH) receptor 1 agonist, met its primary endpoint with
statistical significance in adults with chronic hypoparathyroidism
(HypoPT) at 24 weeks, compared to placebo. The primary endpoint is
a composite of normalisation of albumin-adjusted serum calcium
levels and independence from active vitamin D and oral calcium
therapy.
HypoPT is a rare endocrine disease caused by a deficiency of PTH
and characterised by impaired regulation of calcium and phosphate
levels in the blood. This dysregulation of the physiological action
of PTH can lead to clinical manifestations, including negative
impact on the kidney and bone.1 HypoPT
is one of the largest known rare diseases, affecting over 200,000
people in the United States and the European Union, approximately
80% of whom are women.2-4
Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare
Disease, said: "People living with HypoPT, a rare endocrine
disease, are often at increased risk of hypercalciuria, osteopenia
and osteoporosis, and these results from the CALYPSO trial
underscore eneboparatide's potential to be another option for these
patients. We look forward to reviewing clinical results at 52
weeks to fully characterise the risk-benefit profile."
Eneboparatide was well tolerated. After the 24-week randomised
treatment period, all patients receive eneboparatide in the ongoing
long-term extension period until 52 weeks. Full efficacy and safety
data will be analysed at 52 weeks. Alexion plans to share
these data with global health authorities and present
them at forthcoming medical meetings.
Notes
Hypoparathyroidism
Hypoparathyroidism (HypoPT) is a rare endocrine disease caused by a
deficiency of parathyroid hormone (PTH) and characterised by
decreased calcium and elevated phosphate levels in the blood, which
can lead to a variety of neuromuscular, renal and skeletal
manifestations.1,5,6 The
primary cause in approximately 75% of people with HypoPT is injury
to or removal of the parathyroid glands during neck
surgery.5,6 There
are over 200,000 people in the United States and the European Union
living with HypoPT, approximately 80% of whom are
women.2-4
Despite available
treatments, people living with HypoPT continue to experience a
significant unmet need.1,8
CALYPSO
CALYPSO is a global Phase III, randomised, double-blind,
placebo-controlled, multicentre trial designed to evaluate the
efficacy and safety of eneboparatide in adults with chronic
hypoparathyroidism. A total of 202 patients treated with standard
of care (active vitamin D and oral calcium supplementation) were
randomised in a 2:1 ratio to receive eneboparatide or
placebo.9
The primary efficacy endpoint is a composite of the proportion of
patients that achieve albumin-adjusted serum calcium within the
normal range and independence from standard of care after 24 weeks
of treatment. The key secondary efficacy endpoints include
normalisation of 24-hour urinary calcium in patients with
hypercalciuria at baseline and assessment of patient-reported
outcomes that reflect physical symptoms and impact on quality of
life.9
After the 24-week randomised main treatment period, all patients
receive eneboparatide treatment in the ongoing long-term extension
period.9
Eneboparatide (AZP-3601)
Eneboparatide (AZP-3601) is an investigational parathyroid
hormone (PTH) receptor 1 agonist for the treatment of chronic
hypoparathyroidism (HypoPT). It is designed to bind with high
affinity to a specific conformation of the PTH receptor 1 to
restore PTH function to manage the symptoms of HypoPT, while
preserving kidney function and bone health.6 Eneboparatide
has been granted fast track designation and orphan drug designation
by the US Food and Drug Administration and orphan designation by
the European Medicines Agency for the treatment of
HypoPT.
Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients
and families affected by rare diseases and devastating conditions
through the discovery, development and delivery of life-changing
medicines. A pioneering leader in rare disease for more than three
decades, Alexion was the first to translate the complex biology of
the complement system into transformative medicines, and today it
continues to build a diversified pipeline across disease areas with
significant unmet need, using an array of innovative modalities. As
part of AstraZeneca, Alexion is continually expanding its global
geographic footprint to serve more rare disease patients around the
world. It is headquartered in Boston, US.
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's innovative
medicines are sold in more than 125 countries and used by millions
of patients worldwide. Please visit astrazeneca.com and
follow the Company on social media @AstraZeneca.
Contacts
For details on how to contact the Investor Relations Team,
please click here. For
Media contacts, click here.
References
1. Bilezikian JP.
Hypoparathyroidism. J Clin Endocrinol
Metab. 2020;105(6):1722-1736.
doi:10.1210/clinem/dgaa113.
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. Deering KL, et al. Economic burden
of patients with post-surgical chronic and transient
hypoparathyroidism in the United States examined using insurance
claims data. Orphanet J Rare
Dis.
2024;19(1):164.
5. Gafni RI, Collins MT.
Hypoparathyroidism. N Engl J
Med. 2019;380(18):1738-1747.
doi:10.1056/NEJMcp1800213
6. Shoback
DM, Bilezikian JP, Costa AG, et al. Presentation of
hypoparathyroidism: etiologies and clinical
features. J Clin
Endocrinol Metab. 2016;101(6):2300-2312.
doi:10.1210/jc.2015-3909
7. Clarke
BL, et al. Epidemiology and diagnosis of
hypoparathyroidism. J Clin
Endocrinol Metab.
2016;101(6):2284-99.
8. Abate
EG, et al. Review of hypoparathyroidism. Front
Endocrinol (Lausanne). 2017;7:172.
9. ClinicalTrials.gov.
Evaluation of the safety and efficacy of eneboparatide (AZP-3601)
in patients with chronic hypoparathyroidism (CALYPSO). NCT
Identifier: NCT05778071. Available here.
Accessed March 2025.
Adrian Kemp
Company Secretary
AstraZeneca PLC
SIGNATURES
Pursuant
to the requirements of the Securities Exchange Act of 1934, the
Registrant has duly caused this report to be signed on its behalf
by the undersigned, thereunto duly authorized.
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AstraZeneca
PLC
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Date:
17 March 2025
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By: /s/
Adrian Kemp
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Name:
Adrian Kemp
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Title:
Company Secretary
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