Attralus Receives U.S. FDA Orphan Drug Designation for AT-01 (Iodine (I-124) Evuzamitide), an Investigational Diagnostic for the Management of Transthyretin Amyloidosis
SAN FRANCISCO, Aug. 08, 2022 (GLOBE NEWSWIRE) --Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for 124I-AT-01 (iodine (I-124) evuzamitide) as a diagnostic for the management of transthyretin amyloidosis (ATTR). 124I-AT-01 utilizes the Company’s pan-amyloid binding peptide as an amyloid-specific imaging agent to detect and quantify amyloid in multiple types of systemic amyloidosis and key organs involved by PET/CT imaging.
“124I-AT-01 has the potential to be the first amyloid-specific imaging agent designed to detect amyloid across key organs, including the heart,” said Gregory Bell, MD, Chief Medical Officer at Attralus. “AT-01 has the potential to become an essential tool not only to streamline diagnosis, but also to provide a comprehensive assessment of disease burden and a means to monitor disease progression in patients with ATTR amyloidosis."
The Phase 1/2 trial, conducted by the University of Tennessee Medical Center, evaluated the ability of 124I-AT-01 to detect amyloid deposits by PET/CT imaging in patients with diverse forms of systemic amyloidosis, including ATTR. The trial enrolled a total of 57 subjects including 20 patients with ATTR amyloidosis. Additional data on AT-01 will be presented at the 2022 International Symposium on Amyloidosis scheduled for September 4-8.
FDA Orphan Drug Designation program is granted to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides benefits to sponsors designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include tax credits for clinical costs, exemptions from certain FDA fees and potential seven years of marketing exclusivity.
“Today, the diagnosis of ATTR is a challenging process for patients, with many going years without an accurate diagnosis, and losing critical time in the process,” said Isabelle Lousada, Founder and CEO of the Amyloidosis Research Consortium. “There is a significant unmet need, and the development of an amyloidosis specific diagnostic has the potential to transform the diagnosis process for ATTR amyloidosis patients and allow patients to get onto treatment sooner.”
About AT-01 Pan-Amyloid Diagnostic
124IAT-01 (iodine (I-124) evuzamitide) utilizes the company’s pan-amyloid binding peptide as an amyloid-specific imaging agent to detect and quantify amyloid in multiple types of systemic amyloidosis by PET/CT imaging. In initial clinical trials, AT-01 has been shown to detect multiple types of amyloid deposits, including AL and ATTR, in major organs such as the heart, kidney, liver and spleen. Attralus obtained exclusive rights to commercialize AT-01 under a commercial license agreement with the University of Tennessee Research Foundation. The similar PAR-peptide technology is utilized in AT-02 and AT-04, two of the company’s therapeutic candidates.
About Transthyretin Amyloidosis (ATTR)
Systemic amyloidosis encompasses a diverse group of rare diseases that occur due to accumulation of toxic amyloid deposits in tissues and organs, a consequence of aberrant protein misfolding events. Transthyretin amyloidosis (ATTR) is caused by a protein called transthyretin, or TTR, produced in the liver that misfolds and accumulates, as amyloid fibrils, in the heart, nerves, kidneys and other organs. ATTR is progressive, debilitating and often fatal. There is a significant unmet need for new therapies and diagnostics in systemic amyloidosis, especially ATTR.
Attralus is a clinical stage biopharmaceutical company focused on creating transformative medicines to improve the lives of patients with systemic amyloidosis. The company’s proprietary pan-amyloid removal (PAR) therapeutics are designed to directly bind to and remove toxic amyloid in organs and tissues. By targeting the universal disease-causing pathology in systemic amyloidosis diseases, PAR therapeutics have the potential to treat and reverse disease in patients with all types and stages of systemic amyloidosis. Attralus was founded by scientific experts in the field of amyloidosis and the company is headquartered in San Francisco.
This press release contains forward-looking statements, including statements related to the efficacy, continued development, and potential of AT-01. Words such as “developing,” “potential,” “shown” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Attralus' current expectations. Forward-looking statements involve risks and uncertainties. Attralus' actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties. Attralus expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Attralus' expectations with regard thereto or any change in events, conditions, or circumstances on which any such statements are based.