AstraZeneca Shows Dedication to Amyloidosis Patients at 2024 ISA

AstraZeneca and its Rare Disease division, Alexion, are set to present a comprehensive array of 14 studies on amyloidosis therapies at the upcoming International Symposium on Amyloidosis (ISA). The symposium, scheduled for May 26-30, 2024, in Rochester, MN, will showcase both clinical and real-world evidence to deepen the understanding of amyloidosis and its treatments.

One of the spotlight presentations will be on WAINUA™ (eplontersen), a medication approved by the US FDA in December 2023 for treating polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (hATTR-PN). New subgroup analyses from the Phase 3 NEURO-TTRansform study of WAINUA will be discussed. Additionally, clinical data on ALXN2220 and anselamimab, both currently in Phase III trials for ATTR and light chain (AL) amyloidosis respectively, will be presented.

Sarah Walters, Vice President of US Cardiovascular, Renal & Metabolic Diseases at AstraZeneca, emphasized the company's dedication to amyloidosis research, highlighting that the data presented at the ISA exemplifies their commitment to developing superior treatment options for amyloidosis patients. Christophe Hotermans, Senior Vice President and Head of Global Medical Affairs at Alexion, echoed this sentiment, stressing the importance of their diverse pipeline in addressing the complex needs of amyloidosis patients.

The presentations will include live-cell imaging and Phase I clinical data on ALXN2220’s ability to remove amyloid from cardiac tissue, bolstering its potential as a treatment for advanced ATTR cardiomyopathy. Moreover, findings on epidemiology and renal outcomes in patients will underline the necessity for better diagnosis and treatment options for AL amyloidosis.

The symposium will also feature data from the OverTTuRe study on the prevalence and characteristics of ATTR phenotypes in the US and Japan, as well as details from the MaesTTRo study, which aims to bridge gaps in understanding the effectiveness of treatments for ATTR. The CARDIO-TTRansform study, the largest study of its kind with over 1,400 participants, will also be discussed, focusing on WAINUA’s application for cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM).

As part of a global agreement, AstraZeneca and Ionis are jointly commercializing WAINUA for hATTR-PN treatment in the US and pursuing regulatory approvals globally.

High-resolution live-cell imaging will provide insights into the mechanisms behind ALXN2220’s ability to mediate amyloid removal. Data will illustrate how the addition of ALXN2220 to cardiac tissue from ATTR patients can trigger phagocytosis, recruiting macrophages to target and degrade amyloid deposits. Furthermore, an initial human study of ALXN2220 indicated it was well tolerated, with observed immune activation suggesting its effective role in amyloid clearance.

A significant study on anselamimab will also be presented, detailing the demographics of the 406 participants in the Alexion CARES clinical program, which is the largest prospective study on advanced stage AL amyloidosis to date.

Additional retrospective data from the US EMR TriNetX database will highlight renal outcomes in AL amyloidosis patients treated with bortezomib or daratumumab, demonstrating a pressing need for improved therapeutic options.

The symposium will include several poster sessions featuring AstraZeneca’s abstracts that cover various aspects of amyloidosis research, from treatment patterns and patient outcomes to novel therapeutic mechanisms.

Overall, AstraZeneca and Alexion’s extensive research portfolio reflects their leadership in the field of amyloidosis, showcasing innovative strategies to address the significant unmet needs of patients suffering from this devastating group of diseases.

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