FDA clears way for CAR T-cell therapy trial in patients with myositis
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Key takeaways:
- The therapy is cleared for a phase 1/2 trial in patients with idiopathic inflammatory myopathy. The first trial will include patients with several disease subtypes.
The FDA has granted an investigational new drug application for CABA-201, a 4-1BB-containing fully human CD19-CAR T-cell therapy, to be studied in patients with myositis, according to a press release from the manufacturer.
The therapy is now cleared to begin a phase 1/2 trial in patients with idiopathic inflammatory myopathy, read the release from Cabaletta Bio. The investigational new drug (IND) application approval is the second such decision CABA-201 has received in 2 months, following a prior FDA clearance allowing a similar phase 1/2 trial design in patients with systemic lupus erythematosus.
“The clearance of our second IND application for CABA-201, within two months of the first IND clearance in SLE, allows us to initiate a clinical trial in patients with myositis and underscores the efficiency of our organization, along with the value of our experience in the development of cellular therapies for patients with autoimmune diseases,” Steven Nichtberger, MD, CEO and co-founder of Cabaletta, said in the release.
The open-label phase 1/2 trial will investigate the therapy in patients with myositis and several subtypes, including dermatomyositis, immune-mediated necrotizing myopathy and anti-synthetase syndrome, according to the company. The initial trial will include six patients with dermatomyositis, six with anti-synthetase syndrome and six with immune-mediated necrotizing myopathy. Patients will receive a dose of 1 x 106 cells per kg.
The trial will include adults aged 18 to 65 years who demonstrate active disease. Patients will be excluded if they if they have cancer-associated myositis, significant lung or cardiac impairment, or have undergone therapy with a B-cell depleting therapy within 6 months or therapy with a biologic agent within 3 months.
“Similar to our phase 1/2 trial design in SLE, this clinical trial will include patients with several different subtypes of myositis where B cells may be involved in disease pathology,” Nichtberger said. “With an experienced team well-versed in conducting autoimmune-focused cell therapy trials, and a product candidate specifically engineered for patients with autoimmune diseases, we look forward to evaluating the potential for CABA-201 to change the treatment paradigm for patients with autoimmune diseases.”
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