China-based CARsgen Therapeutics Holdings Limited (HKG: 2171), a leading developer of innovative CAR T-cell therapies, and Huadong Medicine (Hangzhou) Co., Ltd., a wholly-owned subsidiary of Huadong Medicine Co., Ltd (SHE: 000963), have announced a strategic collaboration to commercialize CARsgen’s BCMA CAR-T product, CT053, in mainland China. Under the agreement, CARsgen will receive an upfront payment of RMB 200 million (USD 29.5 million) and is eligible for additional regulatory and commercial milestone payments of up to RMB 1,025 billion (USD 151.4 million). CARsgen will continue to oversee the development, regulatory approval, and manufacturing of CT053 in mainland China.
CT053: A Promising BCMA CAR-T Therapy
CT053 (zevorcabtagene autoleucel; zevor-cel) is a fully human, autologous BCMA CAR T-cell therapy designed to treat relapsed/refractory multiple myeloma (r/r MM). The New Drug Application (NDA) for CT053 is based on positive Phase I/II data from the LUMMICAR STUDY 1 in China and is currently under review by the National Medical Products Administration (NMPA). CARsgen is also conducting the Phase Ib/II LUMMICAR STUDY 2 in North America to evaluate the safety and efficacy of CT053 in treating r/r MM in that region, with plans to expand into earlier-line multiple myeloma treatments.
Regulatory Milestones and Global Recognition
CT053 has garnered significant regulatory attention, receiving Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the US FDA in 2019. The therapy also secured Priority Medicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively. In China, CT053 was awarded a Breakthrough Therapy designation by the NMPA in 2020 and received priority review status for the r/r MM indication in October of last year. These designations highlight the potential of CT053 to address significant unmet needs in the treatment of multiple myeloma.-Fineline Info & Tech
