Legend Biotech and Janssen Submit New Drug Application in Japan for Cilta-cel CAR-T Therapy for Relapsed/Refractory Multiple Myeloma

On December 6, 2021, local time, Legend Biotech (NASDAQ: LEGN) announced in Somerset, New Jersey, that its partner, Janssen Pharmaceuticals, Inc. (Janssen), submitted a New Drug Application (NDA) for Cilta-cel to Japan's Ministry of Health, Labour and Welfare (MHLW) today.

Cilta-cel is an investigational chimeric antigen receptor T-cell (CAR-T) therapy targeting B-cell maturation antigen (BCMA), intended for the treatment of adult patients with relapsed or refractory multiple myeloma who have previously received at least three prior lines of therapy, including proteasome inhibitors (PIs), immunomodulatory agents (IMiDs), and anti-CD38 monoclonal antibodies.

This application is based on the results of the Phase 1b/2 CARTITUDE-1 study conducted in the United States and Japan, which evaluated the efficacy and safety of ciltacabtagene autoleucel in patients with relapsed or refractory multiple myeloma. Ciltacabtagene autoleucel is currently undergoing regulatory review by multiple health authorities worldwide, including in the United States and Europe.

Legend Biotech CEO & CFO, Dr. Ying Huang, stated: "The submission of today's application marks an encouraging step in our mission to provide multiple myeloma patients with a potentially revolutionary cell therapy. We look forward to collaborating closely with our partner Janssen Pharmaceuticals and the Japanese Ministry of Health, Labour and Welfare to bring cilta-cel to patients with relapsed or refractory multiple myeloma who have exhausted several standard therapies and face poor prognoses as soon as possible."

About Cilta-cel

Cilta-cel is a chimeric antigen receptor T-cell (CAR-T) therapy, previously known as JNJ4528 in the United States and Europe, and as LCAR-B38M cell therapy in China. Cilta-cel is currently undergoing multiple clinical studies for the treatment of patients with relapsed or refractory multiple myeloma. It is a CAR-T therapy with a differentiated structure composed of two single-domain antibodies targeting BCMA. In December 2017, Legend Biotech entered into an exclusive global license and collaboration agreement with Janssen Pharmaceuticals, Inc. for the development and commercialization of cilta-cel. Cilta-cel not only received Breakthrough Therapy Designation from the U.S. FDA in December 2019, but also obtained PRIME designation from the European Commission in April 2019, and was granted Breakthrough Therapy Designation in China in August 2020. Additionally, cilta-cel was granted Orphan Drug Designation by the U.S. FDA in February 2019 and by the European Commission in February 2020. The U.S. FDA has accepted the Biologics License Application for cilta-cel, and the European Medicines Agency has accepted the Marketing Authorization Application for cilta-cel.

About CARTITUDE-1

CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multi-center study designed to evaluate the safety and efficacy of ciltacabtagene autoleucel in treating adult patients with relapsed or refractory multiple myeloma who have previously received at least three prior lines of therapy or are double-refractory to both proteasome inhibitors (PI) and immunomodulatory drugs (IMiD). Prior treatments include receiving one PI, one IMiD, and an anti-CD38 monoclonal antibody, with documented disease progression during or within 12 months after the last therapy.

About Multiple Myeloma

Multiple Myeloma: An Incurable Blood CancerMultiple myeloma is an incurable blood cancer originating from the bone marrow, characterized by the excessive proliferation of plasma cells. In Japan, there are over 7,000 new cases of multiple myeloma and nearly 5,000 deaths annually. While current treatments can provide relief, most patients eventually relapse. Relapsed multiple myeloma refers to the recurrence of the disease after initial treatment-induced remission, without meeting the definition of refractory. Refractory multiple myeloma refers to cases where patients show no response to the latest treatment or experience progression within 60 days post-treatment. Although some patients with multiple myeloma may be asymptomatic, the majority are diagnosed due to symptoms such as bone-related events, low blood cell counts, elevated blood calcium levels, kidney problems, or infections. For patients who relapse after receiving standard treatments (including proteasome inhibitors and immunomodulatory agents), the prognosis is poor, with very limited treatment options available.

Editor: Liu Li

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