US-based Cabaletta Bio, Inc. (NASDAQ: CABA) has announced that the US Food and Drug Administration (FDA) has cleared the company’s second Investigational New Drug (IND) application for CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy. The clearance is for a Phase I/II study in patients with active idiopathic inflammatory myopathy (IIM), also known as myositis.
CABA-201’s Clinical Trial Approval and Global Rights Agreement
CABA-201 obtained clinical trial approval for use in Systemic lupus erythematosus (SLE) in April of this year. In October 2022, China-based IASO Biotherapeutics granted Cabaletta global development, manufacturing, and commercialization rights related to its CD19 sequence, which is applied in the development of autoimmune diseases treatments by Cabaletta.
Understanding Myositis and Its Subtypes
Myositis refers to a group of autoimmune diseases characterized by inflammation and muscle weakness. In some cases, myositis may also affect other organs and systems in the body, such as the lungs, heart, or skin. The condition is classified into several subtypes based on the underlying immune mechanisms and clinical characteristics. Although the pathogenesis of myositis is not well understood, there are several subtypes thought to be driven by B cells, including dermatomyositis (DM), anti-synthetase syndrome (ASyS), and immune-mediated necrotizing myopathy (IMNM). These three subtypes impact approximately 66,000 patients in the US alone and typically affect middle-aged individuals, particularly women. All three subtypes can lead to severe functional impairment and may be life-threatening.
Current Treatment Landscape and Unmet Needs
Current treatment for myositis typically involves medications to suppress the immune system and/or chronic intensive therapies such as intravenous immunoglobulin (IVIg). Despite these therapies, a significant portion of myositis patients have disease that remains refractory to existing medications, highlighting the need for new treatment options like CABA-201.-Fineline Info & Tech
