CARsgen Announces NDA Acceptance of BCMA CAR T Zevor-cel (CT053) by China NMPA

SHANGHAI,Oct. 18,2022 -- CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK),a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors,announces that the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for zevorcabtagene autoleucel ("zevor-cel," R&D code: CT053),a fully human,autologous BCMA CAR T-cell therapy for the treatment of relapsed and/or refractory multiple myeloma (R/R MM).

The acceptance of the NDA is based on data from an open-label,single arm Phase I/II clinical trial (LUMMICAR STUDY 1 [Protocol number CT053-MM-01]) in China. Study results showed that zevor-cel has excellent safety and efficacy profiles.Zevor-celalso represents a promising treatment option for patients with high-risk disease.

Multiple myeloma is a fatal blood cancer in which plasma cells found in bone marrow grow out of control and create abnormal proteins that can damage vital organs,including heart and kidneys.[1] According to the World Health Organization,there were more than 21,000 new cases and nearly 16,200 deaths caused by multiple myeloma estimated in China in 2020. [2] With patient survival averaging longer than five years,there were an estimated 113,000 prevalent patients with multiple myeloma including those with newly diagnosed and refractory/relapsed disease in China during the same period. Frost & Sullivan have forecasted that through the 2020s,this prevalence will continue to increase 8-10% each year. [3] Although patients may achieve remission with traditional therapies,most of them experience repeated disease progression. [4] Patients who relapse after traditional therapies,including protease inhibitors,immunomodulatory agents and/or anti-CD38 monoclonal antibodies,have poorer prognoses and few treatment options. [5-6] Therefore,these patients have a substantial clinical unmet need for an efficacious,safe,and convenient treatment.

Prof. Wenming Chen,the principal investigator of the CT053-MM-01 study,Director of Hematology Department,Beijing Chao-Yang Hospital,Capital Medical University,said: "Results from the LUMMICAR-1 study show that the fully human autologous BCMA CAR T-cell product,zevor-cel,demonstrated strong and durable efficacy in patients with relapsed/refractory multiple myeloma,and was generally well tolerated. We are delighted to see that the NDA of this CAR T-cell product with independent intellectual property rights has been officially accepted by the NMPA,and we are looking forward to its early launch and bringing clinical benefits to Chinese patients with relapsed/refractory multiple myeloma."

Prof. Chengcheng Fu,the First Affiliated Hospital of Soochow University,said: "In the China confirmatory clinical trial,zevor-cel has shown favorable safety and deep and durable responses in patients with relapsed/refractory multiple myeloma. Excellent patient experience and sufficient scientific evidence have laid a strong foundation for the NDA acceptance. I hope zevor-cel can be approved and launched in China as soon as possible to allow patients early access to this efficacious and safe product."

Dr. Zonghai Li,Founder,Chairman of the Board,Chief Executive Officer,and Chief Scientific Officer of CARsgen Therapeutics,said: "We are delighted that the New Drug Application (NDA) for zevor-cel,a BCMA CAR T-cell fully developed in house by CARsgen,has been accepted by National Medical Products Administration. This milestone cannot be achieved without the joint efforts from CARsgen team,clinical physicians,the strong support from patients and their families,or other partners of CARsgen. I would like to express my cordial thanks to all of them. We look forward to the early approval of zevor-cel to benefit more patients with multiple myeloma. Driven by our vision of 'Making Cancer Curable',we will continue to develop more innovative cell therapies for cancer patients."

AboutZevor-cel

Zevor-cel (CT053) is a fully human,autologous BCMA CAR T-cell product candidate for the treatment of R/R MM. CARsgen is conducting a Phase 1b/2 clinical trial (LUMMICAR STUDY 2) in North America to evaluate the safety and efficacy of zevor-cel for R/R MM. The Company also plans to conduct additional clinical trials to develop zevor-cel as an earlier line of treatment for multiple myeloma.

Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) in 2019,as well as the PRIority MEdicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020,respectively. Zevor-cel also received Breakthrough Therapy designation from the NMPA in 2020.

The Company believes that zevor-cel is well positioned to potentially reshape the treatment paradigm for multiple myeloma and become a foundational treatment for multiple myeloma patients.

About CARsgen Therapeutics Holdings Limited

CARsgen is a biopharmaceutical company with operations in China and the U.S. and is focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors. The Company has built an integrated cell therapy platform with in-house capabilities that span target discovery,antibody development,clinical trials,and commercial-scale manufacturing. CARsgen has internally developed novel technologies and a product pipeline with global rights to address major challenges of CAR T-cell therapies,such as improving the safety profile,enhancing the efficacy in treating solid tumors and reducing treatment costs. The Company's vision is to become a global biopharmaceutical leader that brings innovative and differentiated cell therapies to cancer patients worldwide and makes cancer curable.

References

American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction. Accessed December 2021.

Ferlay J,Ervik M,Lam F,Colombet M,Mery L,Piñeros M,Znaor A,Soerjomataram I,Bray F (2020). Global Cancer Observatory: Cancer Today. Lyon,France: International Agency for Research on Cancer. Available from: https://gco.iarc.fr/today/data/factsheets/populations/160-china-fact-sheets.pdf,accessed 10 August 2022.

Frost and Sullivan. Cellular Immunotherapy Market. Independent Market Research version March,2021. Data on file.

Abdi J,Chen G,Chang H,et al.Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186–2207.

Kumar SK,Lee JH,Lahuerta JJ,et al.Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012;26:149-57.

Gandhi UH,Cornell RF,Lakshman A,Gahvari ZJ,McGehee E,Jagosky MH,Gupta R,Varnado W,Fiala MA,Chhabra S,Malek E,Mansour J,Paul B,Barnstead A,Kodali S,Neppalli A,Liedtke M,Narayana S,Godby KN,Kang Y,Kansagra A,Umyarova E,Scott EC,Hari P,Vij R,Usmani SZ,Callander NS,Kumar SK,Costa LJ. Outcomes of patients with multiple myeloma refractory to CD38-targeted monoclonal antibody therapy. Leukemia. 2019 Sep;33(9):2266-2275. doi: 10.1038/s41375-019-0435-7. Epub 2019 Mar 11. PMID: 30858549; PMCID: PMC6820050.

For more information,please visit https://www.carsgen.com/

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