Alexion to showcase Phase III data in hypophosphatasia and hypoparathyroidism at ASBMR 2026 Annual Meeting

Results from HICKORY trial will add to evidence supporting the potential of
efzimfotase alfa to improve outcomes in broad HPP patient population

Alexion, AstraZeneca Rare Disease, will deliver 16 presentations, including three oral presentations, across hypophosphatasia (HPP), chronic hypoparathyroidism (HypoPT) and early research at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Boston, Massachusetts, October 9 to 12, 2026. 

Highlights include:

  • HICKORY: results from Phase III trial of efzimfotase alfa (ALXN1850) in treatment-naïve adolescents and adults with HPP
  • CALYPSO: data on skeletal parameters from Phase III trial evaluating eneboparatide, an investigational parathyroid hormone (PTH) 1 receptor agonist, in adults with HypoPT

Gianluca Pirozzi, Senior Vice President, Head of Development, Regulatory and Safety, Alexion, said: “At this year’s ASBMR Annual Meeting, findings across 16 presentations will notably demonstrate the breadth of our research in HPP and HypoPT, two rare, multisystemic diseases that can have far-reaching impacts on patients’ daily lives. In HPP, results from HICKORY, the first Phase III clinical trial to include patients with adult-onset disease, will further reinforce the potential of efzimfotase alfa to reduce treatment burden and redefine care expectations, while real-world evidence will provide important insights into the impact of HPP. Together with new bone data from the CALYPSO Phase III trial of eneboparatide in HypoPT, our presence reflects our commitment to developing meaningful innovations that address the needs of patients and their families.”

Alexion presentations during the 2026 ASBMR Annual Meeting

Lead Author Abstract Title Presentation Details
HPP

Peroutka, C.

Genetic Characterization and Clinical Manifestations in Adults with Hypophosphatasia in the United States

Welcome Reception and Plenary Poster Session
Abstract #FRI-465

 

October 9, 2026
05:30 – 07:15 PM ET

 

Poster Session I
Abstract #SAT-465

 

October 10, 2026
02:00 – 03:30 PM ET

Moss, K.

Understanding Disease Burden and Characterizing Diagnosis, Assessment, and Management of Adults with Hypophosphatasia in the UK: A Delphi Study

Late Breaking Poster Session I
Abstract #SAT-551

 

October 10, 2026
02:00 – 03:30 PM ET

Dahir, K.

 

 

Assessing the compliance, usability, health insights, and perceived value from an at-home digital assessment toolkit for adults with hypophosphatasia: a multicenter observational study

 

 

Poster Session I
Abstract #SAT-489

 

October 10, 2026
02:00 – 03:30 PM ET

Lynch, L.

 

 

Treatment Experience in Adult Patients with Hypophosphatasia (HPP): Results of a US Survey

 

 

Poster Session I
Abstract #SAT-482

October 10, 2026
02:00 – 03:30 PM ET

Ohata, Y.

 

 

Clinical Course and Candidate Biomarkers in Perinatal Hypophosphatasia

 

 

Poster Session I
Abstract #SAT-483

 

October 10, 2026
02:00 – 03:30 PM ET

Padidela, R.

 

Diagnostic Delay and Clinical Burden in Hypophosphatasia: Results from a Cross-Sectional, Multinational, Mixed-Methods Study

 

 

Poster Session I
Abstract #SAT-481

 

October 10, 2026
02:00 – 03:30 PM ET

Dhaliwal, R.

 

 

A Real-World US Survey Study Characterizing the Holistic Disease Burden in Adults with Hypophosphatasia

 

 

Poster Session II
Abstract #SUN-480

 

October 11, 2026
02:00 – 03:30 PM ET

Tanfous, M.

 

 

Cascade Diagnosis of the Rare Metabolic Disease Hypophosphatasia (HPP) Within Families: A Case Series

 

 

Poster Session II
Abstract #SUN-492

 

October 11, 2026
02:00 – 03:30 PM ET

Dahir, K.

 

 

Efficacy and Safety of Alkaline Phosphatase (ALP) Enzyme Replacement Therapy (ERT) Efzimfotase Alfa in Adolescents and Adults with Hypophosphatasia (HPP): Results of HICKORY as Part of a Three-Trial Phase 3 Clinical Program

 

 

Oral Presentation
Abstract #1116

 

October 11, 2026
05:00 – 05:12 PM ET

HypoPT

Vizcaya, D.

 

 

Chronic Hypoparathyroidism in the US: Prevalence and Incidence From 2018 to 2024, With Baseline Clinical Characteristics

 

 

Welcome Reception and Plenary Poster Session
Abstract #FRI-005

 

October 9, 2026
05:30 – 07:15 PM ET

 

Poster Session I
Abstract #SAT-005

 

October 10, 2026
2:00 – 03:30 PM ET

Siggelkow, H.

 

 

Leveraging Structured and Unstructured EHR Data to Improve Treatment and Clinical Context Capture in Chronic Hypoparathyroidism

 

 

Poster Session I
Abstract #SAT-015

 

October 10, 2026
02:00 – 03:30 PM ET

Siggelkow, H.

 

 

Bone Manifestations in Chronic Hypoparathyroidism: A Retrospective Cohort Study

 

 

Poster Session I
Abstract #SAT-019

 

October 10, 2026
02:00 – 03:30 PM ET

Khan, A.

 

 

Balanced Effects of Eneboparatide on Skeletal Parameters: Results From a Phase 3, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Eneboparatide in Adults With Chronic Hypoparathyroidism (CALYPSO)

 

 

Oral Presentation
Abstract #1089

 

October 11, 2026
12:06 – 12:18 PM ET

Erdmann, M.

 

 

Methodological Differences Limit the Feasibility of an Indirect Treatment Comparison Between the Phase III CALYPSO (Eneboparatide) and PaTHway (Palopegteriparatide) Trials in Chronic Hypoparathyroidism

 

 

Late Breaking Poster Session II
Abstract #SUN-519

 

October 11, 2026
02:00 – 03:30 PM ET

Vizcaya, D.

 

 

Epidemiology of Low Bone Density and Osteoporosis in Chronic Hypoparathyroidism: Baseline Prevalence and Medication Usage from National US Claims

 

 

Poster Session II
Abstract #SUN-014

 

October 11, 2026
02:00 – 03:30 PM ET

Early Research 

Celen, I.

 

 

Plasma Proteomics Distinguishes Clinical Expression in ADO and Highlights Differential Inflammatory and Matrix Remodeling Pathways

 

 

Oral Presentation
Abstract #1143

 

October 12, 2026
12:30 – 12:42 PM ET

Notes

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. For more information, please visit www.alexion.us.

AstraZeneca 
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialization of prescription medicines in Oncology, Rare Disease, 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-us.com and follow the Company on social media @AstraZeneca.

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