FDA Grants Orphan Drug Status to Attralus AT-02 for ATTR Amyloidosis Treatment

NAPLES, Fla., Nov. 12, 2024 – Attralus, Inc., a clinical stage biopharmaceutical company dedicated to advancing medicines and diagnostics for systemic amyloidosis, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for their lead therapeutic candidate, AT-02, aimed at treating transthyretin-associated amyloidosis (ATTR). ATTR is a rare, progressive disorder that can lead to severe organ damage and is often fatal.

AT-02, which is designed to remove harmful amyloid deposits, is currently being tested in a three-part Phase 1 clinical trial as well as a Phase 2 open label extension trial involving patients with systemic amyloidosis. Gregory Bell, M.D., Chief Medical Officer at Attralus, expressed satisfaction with the FDA's decision, noting that existing therapies focus on reducing amyloid formation but do not address the need to eliminate existing amyloid deposits that contribute to organ damage and increased mortality.

The FDA's Orphan Drug Designation program offers support for the development of treatments for rare diseases affecting fewer than 200,000 people in the United States. Benefits under this program include tax credits for clinical trial expenses, exemption from certain FDA fees, and potential seven-year market exclusivity.

Earlier this year, the European Medicines Agency (EMA) also provided a positive opinion on AT-02 for ATTR and light chain (AL) amyloidosis, which was subsequently adopted by the European Commission in August 2024.

AT-02, Attralus' lead pan-amyloid removal (PAR) therapeutic candidate for systemic amyloidosis, is a fusion protein consisting of humanized immunoglobulin G1 (IgG1) with a pan amyloid-reactive peptide (p5R) integrated into the light chain's C-terminus. This design enables the antibody to bind to various types of amyloid deposits. The Fc region of the antibody then activates the immune system to clear the amyloid deposits.

AT-02 employs a pan-amyloid peptide similar to 124I-evuzamitide, Attralus' diagnostic agent that has demonstrated selective binding to amyloid deposits in multiple organs, including the heart, liver, and kidneys, across various types of amyloidosis. This suggests that AT-02 could potentially target amyloid deposits in systemic amyloidosis patients effectively. Preclinical data supports AT-02’s capability to bind to multiple amyloid types in major organs and promote amyloid removal through macrophage-mediated phagocytosis. Current evaluations of AT-02 are ongoing in the aforementioned clinical trials involving ATTR and AL amyloidosis patients.

Transthyretin amyloidosis (ATTR) is a rare, severe, progressive disease often underdiagnosed. It is characterized by amyloid deposits accumulating in different body parts such as the heart, nerves, connective tissues, kidneys, and gastrointestinal tract. Cardiac involvement in ATTR, known as ATTR-CM, leads to high mortality with a median survival of 3-5 years. ATTR has two main forms: hereditary ATTR (hATTR), caused by a variant in the TTR gene, and wild-type ATTR (wtATTR), which occurs with aging. Both forms significantly impact the heart and affect 300,000-400,000 individuals in developed countries.

Attralus, based in Naples, FL, is a biopharmaceutical company focused on developing transformative medicines and diagnostics for systemic amyloidosis. Their proprietary pan-amyloid removal therapeutics are designed to target and eliminate toxic amyloid deposits in organs and tissues, addressing the underlying pathology of systemic amyloidosis. This approach holds the potential to treat and possibly reverse the disease in patients across all types and stages of systemic amyloidosis.