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, 9 to 12 October 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 |
|---|---|---|
Lead Author HPP | ||
Lead Author Peroutka, C. | Abstract Title Genetic Characterization and Clinical Manifestations in Adults with Hypophosphatasia in the United States | Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-465 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-465 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Moss, K. | Abstract Title Understanding Disease Burden and Characterizing Diagnosis, Assessment, and Management of Adults with Hypophosphatasia in the UK: A Delphi Study | Presentation Details Late Breaking Poster Session I Abstract #SAT-551 10 October 2026 |
Lead Author Dahir, K. | Abstract Title Assessing the compliance, usability, health insights, and perceived value from an at-home digital assessment toolkit for adults with hypophosphatasia: a multicenter observational study | Presentation Details Poster Session I Abstract #SAT-489 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Lynch, L. | Abstract Title Treatment Experience in Adult Patients with Hypophosphatasia (HPP): Results of a US Survey | Presentation Details Poster Session I Abstract #SAT-482 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Ohata, Y. | Abstract Title Clinical Course and Candidate Biomarkers in Perinatal Hypophosphatasia | Presentation Details Poster Session I Abstract #SAT-483 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Padidela, R. | Abstract Title Diagnostic Delay and Clinical Burden in Hypophosphatasia: Results from a Cross-Sectional, Multinational, Mixed-Methods Study | Presentation Details Poster Session I Abstract #SAT-481 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Dhaliwal, R. | Abstract Title A Real-World US Survey Study Characterizing the Holistic Disease Burden in Adults with Hypophosphatasia | Presentation Details Poster Session II Abstract #SUN-480 11 October 2026 02:00 – 03:30 PM ET |
Lead Author Tanfous, M. | Abstract Title Cascade Diagnosis of the Rare Metabolic Disease Hypophosphatasia (HPP) Within Families: A Case Series | Presentation Details Poster Session II Abstract #SUN-492 11 October 2026 02:00 – 03:30 PM ET |
Lead Author Dahir, K. | Abstract Title 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 | Presentation Details Oral Presentation Abstract #1116 11 October 2026 05:00 – 05:12 PM ET |
Lead Author HypoPT | ||
Lead Author Vizcaya, D. | Abstract Title Chronic Hypoparathyroidism in the US: Prevalence and Incidence From 2018 to 2024, With Baseline Clinical Characteristics | Presentation Details Welcome Reception and Plenary Poster Session Abstract #FRI-005 9 October 2026 05:30 – 07:15 PM ET Poster Session I Abstract #SAT-005 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Siggelkow, H. | Abstract Title Leveraging Structured and Unstructured EHR Data to Improve Treatment and Clinical Context Capture in Chronic Hypoparathyroidism | Presentation Details Poster Session I Abstract #SAT-015 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Siggelkow, H. | Abstract Title Bone Manifestations in Chronic Hypoparathyroidism: A Retrospective Cohort Study | Presentation Details Poster Session I Abstract #SAT-019 10 October 2026 02:00 – 03:30 PM ET |
Lead Author Khan, A. | Abstract Title 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) | Presentation Details Oral Presentation Abstract #1089 11 October 2026 12:06 – 12:18 PM ET |
Lead Author Erdmann, M. | Abstract Title Methodological Differences Limit the Feasibility of an Indirect Treatment Comparison Between the Phase III CALYPSO (Eneboparatide) and PaTHway (Palopegteriparatide) Trials in Chronic Hypoparathyroidism | Presentation Details Late Breaking Poster Session II Abstract #SUN-519 11 October 2026 02:00 – 03:30 PM ET |
Lead Author Vizcaya, D. | Abstract Title Epidemiology of Low Bone Density and Osteoporosis in Chronic Hypoparathyroidism: Baseline Prevalence and Medication Usage from National US Claims | Presentation Details Poster Session II Abstract #SUN-014 11 October 2026 02:00 – 03:30 PM ET |
Lead Author Early Research | ||
Lead Author Celen, I. | Abstract Title Plasma Proteomics Distinguishes Clinical Expression in ADO and Highlights Differential Inflammatory and Matrix Remodeling Pathways | Presentation Details Oral Presentation Abstract #1143 12 October 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.
AstraZeneca
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation 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.
