The China Center for Drug Evaluation, National Medical Products Administration Has Recommended Breakthrough Therapy Designation for ciltacabtagene autoleucel (cilta-cel, LCAR-B38M CAR-T Cells), an Investigational BCMA CAR-T Cell Therapy

Legend Biotech Corporation (NASDAQ:LEGN) announced today that the China Center for Drug Evaluation, National Medical Products Administration (CDE, NMPA) has recommended Breakthrough Therapy Designation (BTD) for ciltacabtagene autoleucel (cilta-cel; LCAR-B38M CAR-T cells), an investigational B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy being studied for the treatment of adults with relapsed or refractory multiple myeloma (RRMM).

The BTD for cilta-cel (LCAR-B38M CAR-T cells) is based on the ongoing Phase 2 CARTIFAN-1 study being conducted in China (MMY2002, NCT03758417, CTR20181007), the ongoing Phase 1b/2 CARTITUDE-1 study of cilta-cel (JNJ-4528) being conducted in the US (MMY2001, NCT03548207) and Japan and the Phase 1, first-in-human LEGEND-2 study conducted in China (NCT03090659). Ciltacabtagene autoleucel (cilta-cel) refers to both LCAR-B38M CAR-T cells and JNJ-4528. LCAR-B38M CAR-T cell identifies the investigational product being studied in China and JNJ-4528 identifies the investigational product being studied outside of China, both of which are representative of the same CAR-T cell therapy.

The BTD procedure is part of the recently revised Drug Registration Regulation which went into effect on July 1, 2020. The BTD process is designed to expedite the development and review of therapies that are intended for treatment of serious diseases for which there is no existing treatment and where preliminary evidence indicates advantages of the therapy over available treatment options.¹ Cilta-cel is the first product that has been recommended for BTD in China.² As per the working procedure for BTD (2020 No.82) issued by NMPA on July 8, 2020, CDE had completed the review and recommended to grant the BTD on August 4^th, and BTD will be granted after 5 working days of publicity period (August 5 to 12) on the CDE website.

In December 2017, Legend Biotech entered into a worldwide collaboration and license agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to jointly develop and commercialize cilta-cel in patients with multiple myeloma. Cilta-cel is a structurally differentiated CAR-T cell therapy containing a 4-1BB co-stimulatory domain and two BCMA-targeting single domain antibodies designed to confer avidity.