Item 7.01 |
Regulation FD Disclosure. |
On February 1, 2024, Cabaletta Bio, Inc. (the “Company”) issued a Press Release announcing that the U.S. Food and Drug Administration (the “FDA”) granted Orphan Drug Designation to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of idiopathic inflammatory myopathies (IIM, or myositis) (the “Press Release”). A copy of the Press Release is attached hereto as Exhibit 99.1 to this Current Report on Form 8-K.
The information contained in Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1 attached hereto, is being furnished and shall not be deemed to be “filed” for the purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section and shall not be incorporated by reference in any filing under the Securities Act or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.
On February 1, 2024, the Company issued the Press Release announcing that the FDA has granted Orphan Drug Designation to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of idiopathic inflammatory myopathies (IIM, or myositis). CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells. Four RESET™ (REstoring SElf-Tolerance) Phase 1/2 trials are advancing for the evaluation of CABA-201 across multiple autoimmune conditions, including the Phase 1/2 RESET-Myositis™ trial.
The RESET-Myositis™ trial is a Phase 1/2 open-label study of CABA-201 in subjects with active idiopathic inflammatory myopathy (IIM, or myositis), including the subtypes of dermatomyositis (DM), anti-synthetase syndrome (ASyS) and immune-mediated necrotizing myopathy (IMNM). Subjects will receive a one-time infusion of CABA-201 at a dose of 1 x 106 cells/kg, preceded by a standard preconditioning regimen of fludarabine and cyclophosphamide. Key inclusion criteria include patients between ages 18 to 65 (inclusive), evidence of active disease and disease activity despite prior or current treatment with standard of care treatments. Key exclusion criteria include cancer-associated myositis, significant lung or cardiac impairment, treatment with a B cell depleting agent within the prior approximately six months or treatment with a biologic agent within the prior approximately three months. As part of Cabaletta’s CARTA (Chimeric Antigen Receptor T cells for Autoimmunity) strategy, this trial is intended to evaluate the potential ability of CABA-201 to transiently, but fully, eliminate B cells, potentially enabling durable remissions via a “reset” of the immune system.
CABA-201 is designed to deeply and transiently deplete CD19-positive B cells following a one-time infusion, which may enable an “immune system reset” with the potential for durable remission off therapy in patients with autoimmune diseases. To date, Cabaletta has received clearance from the FDA for Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions including systemic lupus erythematosus (SLE), myositis, systemic sclerosis (SSc) and generalized myasthenia gravis (gMG). Cabaletta is conducting four Phase 1/2 clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement.
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. Myositis 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 typically involves medications to suppress the immune system and/or chronic intensive therapies such as intravenous immunoglobulin, or IVIg. Despite these therapies, a significant portion of myositis patients have disease that remains refractory to existing medications.