CARsgen Therapeutics, a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announces that today the National Medical Products Administration ("NMPA") of China has approved the New Drug Application ("NDA") for zevorcabtagene autoleucel (R&D code: CT053, an autologous CAR-T product candidate against BCMA), for the treatment of adult patients with relapsed or refractory multiple myeloma who have previously progressed after at least 3 lines of therapy (including a proteasome inhibitor and immunomodulator 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 CD8α hinge domain, CD8α transmembrane domain, 4-1 BB co-stimulatory domain and CD3ζ 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 ("ASH"), 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.^[1] 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.^[2]

Prof. Wenming Chen, the principal investigator of the LUMMICAR STUDY 1, Director of Hematology Department, Beijing Chao-Yang Hospital, Capital Medical University, said, "In the realm of traditional treatments, the prognosis for patients dealing with relapsed or refractory multiple myeloma remains notably grim, given the limitations of available therapeutic alternatives. These individuals confront substantial unmet clinical needs, necessitating an expeditious adoption of an effective, safe, and convenient treatment modalities. The approval of zevorcabtagene autoleucel not only expands the array of choices available to clinical practitioners but also brings new hope to patients."

Prof. Chengcheng Fu, the principal investigator of the LUMMICAR STUDY 1, Director of Hematology Department, the First Affiliated Hospital of Soochow University, said, "Based on the published results of the LUMMICAR-1 study, zevorcabtagene autoleucel has demonstrated profound and enduring therapeutic efficacy in patients with relapsed or refractory multiple myeloma, exhibiting overall favorable tolerability. We are pleased to witness the regulatory approval and market launch of zevorcabtagene autoleucel. We look forward to its potential to benefit a larger number of individuals, aiding them in achieving high-quality, long-term survival."

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "This year marks the tenth anniversary of CARsgen, and I am delighted to announce the NDA approval of zevorcabtagene autoleucel. This achievement stands as a significant milestone in the company's development and serves as a testament to the unwavering dedication of our team. I would like to express heartfelt gratitude to our team members, investigators, patients, and the broader community for their support and trust. We look forward to zevorcabtagene autoleucel bringing renewed hope to adult patients with relapsed or refractory multiple myeloma, thereby improving their survival. Guided by the vision of 'Making Cancer Curable,' we remain committed to exploring new technologies, expanding our product pipeline with global rights to address the major challenges of CAR T-cell therapies, and bringing innovative and differentiated cell therapies to cancer patients worldwide."