On December 15, 2022 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported that China National Medical Products Administration (NMPA) has accepted the new drug application (NDA) from Juventas Cell Therapy, Ltd., (Juventas) for CNCT19 (Inaticabtagene Autoleucel), an investigational CD-19 directed CAR-T therapy, for the treatment of adult patients with relapsed/refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-ALL) (Press release, CASI Pharmaceuticals, DEC 15, 2022, View Source [SID1234625336]).
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CNCT19 is the first CAR-T cell therapy for the treatment of B-ALL submitted for NDA and has the potential to be the first approved domestic developed CD19 directed CAR-T therapy in China. Owing to the unique CD19 scFv（HI19a）structure and the leading CMC manufacturing techniques, CNCT19 indicated an 100% success rate of drug production from r/r B-ALL patients in the registrational trial. It has been granted the Orphan Drug Designation from the FDA and the Breakthrough Designation status by the China Center of Drug Evaluation (CDE).
The r/r B-ALL NDA submission for CNCT19 contains the initial data from study NCT04684147, a Phase II, single-arm, open-label, single-dose, multicenter clinical trial. The study results reveal that CNCT19 has demonstrated a high level of efficacy, durable remissions, and substantially reduced CAR-T related toxicity for the treatment of adults with r/r B-ALL. Professor Wang Jianxiang, the Principal Investigator of CNCT19 from the Institute of Hematology and Blood Diseases Hospital, and his team presented the study results at the 64th Annual Meeting of American Hematology (2022 ASH (Free ASH Whitepaper)) on December 12th, 2022.
CNCT19 CAR-T cell therapy achieved outstanding ORRs with high rates of MRD-negative complete remission in adult patients with r/r B-ALL within three months and by the end of the third month. Of the 39 evaluable patients, 32 (82.1%) patients reached CR/CRi within three months and 25 (64.1%) patients remained CR/CRi by the end of the third months. The CR rates were 66.7% (26/39) and 51.3% (20/39) respectively. The MRD-negative rate was 100% (32/32) within three months and persisted at 92% (23/25) by the end of the third month. Median duration of response (DOR) has not been reached.
The most common CNCT19 related adverse events (AEs) were cytokine release syndrome (CRS) and neuro toxicity (NT). There were 4 cases of Grade ≥3 CRS (n=4, 10.3%) and 3 cases of Grade ≥ 3 NT (n=3, 7.7%). Following CNCT19 infusion, all the patients recovered without any unexpected AEs reported.
Dr. Wei-Wu He, CASI’s Chairman and CEO commented, "CNCT19 NDA acceptance by NMPA represents a significant milestone. We are in preparation for the anticipated CNCT-19 commercial launch. This product is truly exciting as it proved to have significant clinical benefits for unmet medical needs. CASI and Juventas will work side by side to deliver CNCT19 to patients in China, and eventually worldwide."