REG - AstraZeneca PLC - Eneboparatide Phase III trial met primary endpoint

AstraZenecaAnnouncement

17/03/2025 07:15

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RNS Number : 9031A  AstraZeneca PLC  17 March 2025

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 (http://www.astrazeneca.com/)  and follow the
Company on social media @AstraZeneca
(https://www.linkedin.com/company/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
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.

 

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
(https://clinicaltrials.gov/study/NCT05778071) . Accessed March 2025.

 

Adrian Kemp

Company Secretary

AstraZeneca PLC

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