The board of directors of the CARsgen Therapeutics Holdings Limited announced that the National Medical Products Administration of China has approved the New Drug Application for zevorcabtagene autoleucel for the treatment of adult patients with relapsed or refractory multiple myeloma who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and immunomodulatory agent). Zevorcabtagene autoleucel is an autologous BCMA-targeted CAR T-cell product generated by transducing T cells with a lentivirus encoding a CAR comprising a fully human BCMA-specific single chain variable fragment ("scFv"), the human CD8 hinge domain, CD8 transmembrane domain, 4-1 BB co-stimulatory domain and CD3 activation domain. The proprietary novel fully-human scFv has high binding affinity and stability.

The approval of zevorcabtagene autoleucel is based on an open-label, single arm, multi-center Phase II clinical trial (LUMMICAR STUDY 1, NCT03975907) conducted in China. The trial results were released at the 2022 Annual Meeting of the American Society of Hematology and zevorcabtagene autoleucel demonstrated encouraging efficacy and a favorable safety profile. Multiple myeloma is an incurable malignant plasma cell disorder that accounts for approximately 10% of all hematological cancers.

With China's ageing population coupled with an increase in life expectancy, the number of patients with multiple myeloma is expected to expand. Frost & Sullivan forecasts that the prevalence of multiple myeloma in China in 2023 is approximately 153,000 per annum, and the number of new cases would be 23,200 per annum. It is estimated that the prevalence of multiple myeloma in China is expected to grow to 266,300 by 2030.

Zevorcabtagene autoleucel is a fully human, autologous BCMA CAR T-cell product for the treatment of R/R MM. As informed by the NMPA on March 1, 2024, zevorcabtagene autoleucel was approved on February 23, 2024 for the treatment of adult patients with relapsed or refractory multiple myeloma who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and immunomodulatory agent). CARsgen is conducting a separate Phase 1b/2 LUMMICAR STUDY 2 clinical trial in North America to evaluate the safety and efficacy of zevorcabtagene autoleucel in R/R MM.

Zevorcabtagene autoleucel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019, as well as Priority Medicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively. Zevorcabtagene autoleucel also received Breakthrough Therapy designation from the NMPA in 2020.