CARsgen Presents Data from China Pivotal Phase II Trial of BCMA CAR T Zevor-cel (CT053) at 2022 ASH Annual Meeting

SHANGHAI,Dec. 12,2022 -- 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 at the 2022 American Society of Hematology (the "ASH") Annual Meeting,the Company presented a poster with the results of the phase II LUMMICAR STUDY 1 clinical trial of zevorcabtagene autoleucel ("zevor-cel",R&D code: CT053,an autologous CAR T-cell product candidate against BCMA) in Chinese patients with relapsed/refractory multiple myeloma (R/R MM). This poster reports for the first time the pivotal phase II safety and efficacy data of LUMMICAR STUDY 1.

Poster: Phase Ⅱ Study of Fully Human BCMA-Targeted CAR T Cells (Zevorcabtagene Autoleucel) in Patients with Relapsed/Refractory Multiple Myeloma

Zevor-cel,a fully human autologous CAR T-cell product targeting B-cell maturation antigen (BCMA),is being evaluated for patients with R/R MM in LUMMICAR STUDY 1 (NCT03975907),an ongoing phase I/II clinical trial in China. Results for the 14 subjects treated in phase I of LUMMICAR STUDY 1 showed a well-tolerated safety profile,plus deep and durable responses with an objective response rate (ORR) of 100% and a complete response/stringent complete response (CR/sCR) rate of 78.6%.

A total of 102patients with R/R MM were treated with zevor-cel at the dose of 150×106 CAR+ T cells. The patients had a median age of 59.5 years,the median time from diagnosis was 3.6 years,and the median prior lines of therapywas 4 (range,3–15). Of the treated patients,39(38.2%) hadInternational Staging System stage III disease; 46(45.1%) patients had high-risk cytogenetic abnormalities,defined as del(17p),t(4; 14),t(14; 16),t(14; 20); and 24(23.5%) patients had received autologous stem cell transplantation. The lymphodepletion regimen comprised cyclophosphamide at 300mg/m2 and fludarabine at 25mg/m2 daily for 3days before zevor-cel treatment.

Safety

Zevor-cel was well tolerated with manageable safety profile. The treatment-related adverse events (AEs) were reported for 102 patients,and the AEs mainly comprised the lymphodepletion-related hematologic toxicities. A total of 92(90.2%) patients experienced cytokine release syndrome (CRS) with 7(6.9%) at grade 3 or 4,and all patients recovered from CRS. No grade 3 or higher neurotoxicity was identified. Two immune effector cell-associated neurotoxicity syndrome (ICANS) events (both grade 1) occurred,and the patients recovered without the use of tocilizumab or steroids. Treatment-related serious adverse events (SAEs) occurred in 38 (37.3%) patients,mainly comprising hematologic toxicities and infections. No AE led to discontinuation of zevor-cel infusion. One treatment-related death occurred 5 months post CAR T-cell infusion from severe pneumonia in a patient who had a history of chronic pneumonia.

Efficacy

Atthe update cut-off date of August 16,2022,102 patients had at least 3months follow-up or early withdrawal,among whom 60 patients had at least 6months follow-up or early withdrawal. For all 102 patients,the median follow-up was 9months (range,0.4–17.8months),the ORR was 92.2% (95% CI,85.13,96.55),the rate of very good partial response (VGPR) or better was 85.3%,and the CR/sCR rate was 45.1%. Further,among the first 60 patients treated in the current study with a median follow-up of 12.1months (0.4–17.8months),the CR/sCR rate was 56.7%. The median DOR and median PFS were not reached. At 9months median follow-up,the DOR rate was 86.1% and the PFS rate was 84.6%. Minimal residual disease (MRD) status was assessed in patients achieving VGPR or better. The MRD negativity rate (sensitivity of <10−5 nucleated cells) was 100% in patients with CR/sCR and 96.3% in patients with VGPR or better.

Conclusion

Preliminaryresults of the pivotal phase II LUMMICAR STUDY 1 showed that zevor-cel delivered deep and durable responses in patients with heavily pre-treated R/R MM.

Dr. Raffaele Baffa,Chief Medical Officer of CARsgen Therapeutics,said: "We are delighted to present the pivotal phase II data from the LUMMICAR STUDY 1 of zevor-cel at the ASH Annual Meeting this year. The data highlighted the promising safety and efficacy of zevor-cel in patients with heavily pre-treated relapsed/refractory multiple myeloma. The New Drug Application for zevor-cel has been accepted by NMPA and is currently under priority review. We look forward to the approval of zevor-cel in China and will continue to accelerate the global development to benefit patients worldwide."

About Zevor-cel

Zevor-cel (CT053) is a fully human,autologous BCMA CAR T-cell product candidate for the treatment of R/R MM. The New Drug Application (NDA) for zevor-cel based on the phase I/II data from LUMMICAR STUDY 1 in China has been accepted by NMPA. CARsgen is conducting the phase 1b/2 LUMMICAR STUDY 2 clinical trial in North America to evaluate the safety and efficacy of zevor-cel for R/R MM in that population. 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. 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 thatzevor-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.

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