Janssen and Legend Biotech Complete Rolling BLA Submission to FDA for BCMA-Targeted CAR-T Therapy Cilta-cel in Relapsed/Refractory Multiple Myeloma

Legend Biotech stated in a regulatory filing submitted to the U.S. Securities and Exchange Commission (SEC) that its partner, Janssen Pharmaceuticals, Inc., has completed the rolling submission of the Biologics License Application (BLA) for ciltacabtagene autoleucel (cilta-cel), a CAR-T therapy targeting B-cell maturation antigen (BCMA), to the U.S. Food and Drug Administration (FDA). The therapy is indicated for the treatment of adult patients with relapsed/refractory multiple myeloma. Given that this innovative CAR-T therapy received Breakthrough Therapy Designation from the FDA in 2019, its BLA is expected to qualify for priority review, with potential FDA approval by the end of this year.

Multiple Myeloma (MM) is an incurable blood cancer caused by the malignant transformation of white blood cells known as plasma cells. These cancerous plasma cells proliferate rapidly in the bone marrow, displacing normal hematopoietic cells. Although some patients with multiple myeloma are asymptomatic, most diagnosed individuals present with symptoms such as fractures or bone pain, anemia, fatigue, hypercalcemia, renal impairment, or infections. Despite the availability of numerous therapeutic options for MM, the disease relapses in the majority of patients following remission.

Cilta-cel is a CAR-T cell therapy with a differentiated structure, comprising a 4-1BB co-stimulatory domain and two BCMA-targeting antibody domains, which facilitates the expansion of CD8-positive T cells. BCMA is a protein highly expressed on myeloma cells. In December 2017, Janssen Pharmaceuticals, Inc. entered into an exclusive global license and collaboration agreement with Legend Biotech to jointly develop and commercialize cilta-cel.

The latest clinical data presented at last year’s American Society of Hematology (ASH) Annual Meeting demonstrated that cilta-cel continued to exhibit a very high overall response rate (ORR) in the Phase 1b/2 CARTITUDE-1 trial. With a median follow-up of 12.4 months, the ORR reached 97%, and responses deepened over time, with 67% of patients achieving a stringent complete response. At a median follow-up of 12.4 months, the median duration of response and progression-free survival had not yet been reached.

Recently, Abecma (idecabtagene vicleucel), jointly developed by Bristol Myers Squibb (BMS) and bluebird bio, received FDA approval, becoming the first BCMA-targeted cell therapy. Janssen/Legend Biotech’s cilta-cel is expected to become the second approved BCMA-targeted cell therapy this year, providing more treatment options for patients with multiple myeloma.

References:

[1] Form 20-F, Legend Biotech Corporation, Retrieved April 5, 2021, from https://www.sec.gov/Archives/edgar/data/0001801198/000156459021017439/legn-20f_20201231.htm

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