Attralus, Inc., in collaboration with Brigham and Women's Hospital, has commenced a Phase 3 clinical trial (REVEAL) to assess the efficacy of 124I-evuzamitide (AT-01) in diagnosing cardiac amyloidosis. The trial, which enrolled its first patient, seeks to determine the sensitivity and specificity of this novel imaging agent in detecting amyloid deposits in the heart. Cardiac amyloidosis, often underdiagnosed, presents a significant unmet need for more accurate and earlier diagnostic tools.
REVEAL Study Design and Objectives
The REVEAL (Research with 124I-EVuzamitide to Elucidate Cardiac AmyLoidosis) study plans to enroll up to 200 participants with suspected cardiac amyloidosis across the United States. The study's primary objective is to evaluate the diagnostic efficacy of 124I-evuzamitide using PET/CT imaging. Co-primary endpoints include determining the sensitivity and specificity of the imaging agent based on visual scan interpretation by independent PET readers. A secondary objective focuses on evaluating the safety of a single intravenous administration of 124I-evuzamitide. Exploratory objectives include assessing the agent's ability to detect extracardiac organ involvement and differentiate between AL and ATTR amyloidosis.
124I-evuzamitide: A Pan-Amyloid Diagnostic
124I-evuzamitide represents a significant advancement as the first non-invasive pan-amyloid PET imaging agent specifically designed for systemic amyloidosis. It utilizes a pan-amyloid binding peptide labeled with iodine-124, functioning as an amyloid-specific radiotracer to detect all types of systemic amyloidosis via PET/CT imaging. Clinical trials have demonstrated its ability to detect various amyloid deposits, including ATTR and AL, in major organs such as the heart, kidney, liver, and spleen.
Regulatory Designations and Clinical Significance
The U.S. Food and Drug Administration (FDA) has granted 124I-evuzamitide Breakthrough Therapy Designation for PET imaging in patients with suspected or known cardiac amyloidosis. This designation was based on clinical data from Attralus-sponsored and investigator-initiated studies involving over 200 trial participants. Furthermore, both the FDA and the European Commission have granted Orphan Drug Designations to 124I-evuzamitide as a diagnostic for managing ATTR and AL amyloidosis.
Dr. Sharmila Dorbala, Principal Investigator and Sponsor of the REVEAL study, stated, "124I-evuzamitide PET/CT imaging offers an exciting opportunity to noninvasively detect various types of amyloid deposition in the heart and other organs... With effective FDA-approved treatments now available, a scan that can definitively detect amyloidosis or exclude it would be a major step forward to reduce diagnostic delays and improve patient outcomes."
Addressing Unmet Needs in Cardiac Amyloidosis
Systemic amyloidosis comprises a group of rare diseases characterized by the accumulation of toxic amyloid deposits in tissues and organs, leading to progressive and often fatal outcomes. Cardiac involvement is prevalent, with approximately 95% of ATTR and 75% of AL amyloidosis patients affected. The underdiagnosis of cardiac amyloidosis is a critical issue due to low awareness, non-specific symptoms, and a lack of disease-specific diagnostics. Spencer Guthrie, Chief Operating Officer of Attralus, emphasized that "There are no FDA-approved diagnostic imaging agents for cardiac amyloidosis... Early diagnosis in this disease leads to better patient outcomes."
Enrollment in the REVEAL study is expected to be completed by the end of 2025, with additional trial details available on clinicaltrials.gov.
