Attralus Gets EMA COMP Approval for AT-02 to Treat ATTR and AL Amyloidosis

BURLINGAME, Calif., July 29, 2024 — Attralus, Inc., a clinical stage biopharmaceutical company focused on developing transformative treatments for systemic amyloidosis, has announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has given positive opinions on granting orphan medicinal product designations to AT-02 for treating transthyretin-associated amyloidosis (ATTR) and immunoglobulin light-chain-associated (AL) amyloidosis, the two most common forms of systemic amyloidosis.

AT-02, Attralus' leading candidate for pan-amyloid removal therapy, is currently under evaluation in a three-part Phase 1 clinical study and a Phase 2 open-label extension trial. These trials are being conducted with patients suffering from ATTR and AL amyloidosis to support AT-02's potential use as an immunotherapy for systemic amyloidosis. The designation from COMP highlights the unique capabilities of AT-02 to treat not only ATTR and AL amyloidosis but also other forms of systemic amyloidosis.

Dr. Gregory Bell, Chief Medical Officer at Attralus, expressed pleasure at receiving the positive opinions from the COMP. He emphasized the urgent need for new therapies that can remove existing toxic amyloid fibrils responsible for organ damage and increased mortality, as current treatments mainly focus on slowing disease progression by targeting precursor protein production.

In the EU, orphan medicinal product designation is awarded to drugs and biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For a condition to qualify, its prevalence in the EU must be rare, affecting no more than five in 10,000 people. Orphan designation is granted when the drug or biologic offers a significant benefit over existing treatments. Following a positive opinion from the COMP, a legally binding final decision is made by the European Commission. This designation provides companies with various benefits, including specialized scientific advice, reduced regulatory fees, research grants, and up to ten years of market exclusivity in the EU if the product is approved.

Earlier this year, Attralus was also granted Small or Medium-Sized Enterprise (SME) status in the EU. Combining this status with orphan designation allows Attralus to receive additional administrative and procedural assistance from the Agency's SME office, along with further fee reductions.

AT-02 is the company's leading pan-amyloid removal therapeutic for systemic amyloidosis. It is a humanized IgG1 monoclonal antibody fused with Attralus' proprietary pan-amyloid binding peptide, allowing it to bind to various types of amyloid deposits. The antibody's Fc region activates the immune system to clear amyloid deposits bound by AT-02. Similar to the company's diagnostic agent, 124I-evuzamitide, AT-02 has demonstrated selective binding to amyloid in major organs such as the heart, liver, and kidneys. Preclinical data show that AT-02 can bind to several amyloid types in major organs and induce macrophage-mediated amyloid phagocytosis and removal.

Systemic amyloidosis refers to a group of rare diseases caused by the accumulation of toxic amyloid deposits in tissues and organs due to protein misfolding. These diseases are progressive, debilitating, and often fatal. Systemic amyloidosis is significantly underdiagnosed because of low awareness, lack of specific symptoms, and the absence of disease-specific diagnostics. There are approximately 17 types of systemic amyloidosis, with ATTR and AL being the most common. While current treatments focus on slowing the disease by targeting precursor proteins, there is a considerable need for therapies that can remove toxic amyloid deposits, improving organ function and patient quality of life.

Attralus is a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for systemic amyloidosis. The company's proprietary pan-amyloid removal therapeutics are designed to target and eliminate toxic amyloid deposits in organs and tissues. By addressing the underlying pathology of systemic amyloidosis, these therapeutics have the potential to treat and potentially reverse the disease across all types and stages of systemic amyloidosis. Founded by experts in amyloidosis, Attralus is headquartered in Burlingame, CA.