CASI PHARMACEUTICALS' PARTNER JUVENTAS ANNOUNCES NEW DRUG APPLICATION FOR CNCT19 ACCEPTED BY CHINA NATIONAL MEDICAL PRODUCTS ADMINISTRATION

Rhea-AI Summary

CASI Pharmaceuticals announced that the China National Medical Products Administration has accepted the new drug application for CNCT19, an investigational CAR-T therapy targeting relapsed/refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-ALL). This marks CNCT19 as the first CAR-T therapy for B-ALL submitted for NDA and the potential first domestically developed CD19-directed CAR-T therapy in China. The registrational trial showed a 100% drug production success rate and promising efficacy results with an 82.1% overall response rate.

Positive

• Acceptance of CNCT19 NDA by NMPA is a significant milestone.
• CNCT19 shows a 100% success rate of drug production in trials.
• 82.1% overall response rate achieved in the registrational trial.
• Potential to be the first approved domestic CD19 CAR-T therapy in China.

Negative

• Potential risks associated with CAR-T therapy, including cytokine release syndrome and neurotoxicity.

ROCKVILLE, Md. and BEIJING, Dec. 15, 2022 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, today announces 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).

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) 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."

About CASI Pharmaceuticals

CASI Pharmaceuticals, Inc. is a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The Company is focused on acquiring, developing, and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The Company intends to execute its plan to become a leader by launching medicines in the Greater China market, leveraging the Company's China-based regulatory and commercial competencies and its global drug development expertise. The Company's operations in China are conducted through its wholly-owned subsidiary, CASI Pharmaceuticals (China) Co., Ltd., located in Beijing, China. The Company has built a commercial team of more than 100 hematology and oncology sales and marketing specialists based in China. More information on CASI is available at www.casipharmaceuticals.com.

About Juventas Cell Therapy Ltd.

Juventas is a biopharmaceutical company headquartered in China dedicated to the development and commercialization of cell therapies globally. Utilizing innovative and integrated technology platforms, the company has developed a diverse pipeline of cellular immunotherapies for treatment of hematological malignancies, solid tumors, and other non-oncological conditions both in China and globally. At present, the company is conducting two pivotal clinical trials of CNCT19 for treating adult r/r-B-ALL and r/r-B-NHL in China. CNCT19 has the potential to become the first launched domestically developed CD19 CAR-T therapy in China and the first CAR-T product for the treatment of adult R/R B-ALL in China.

Forward-Looking Statements

This news release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act with respect to the outlook for expectations for future financial or business performance, revenue growth, strategies, expectations, and goals. Forward-looking statements are subject to numerous assumptions, risks, and uncertainties, which change over time. Forward-looking statements speak only as of the date they are made, and we assume no duty to update forward-looking statements. New factors emerge from time to time, and it is not possible for us to predict which factors will arise. In addition, we cannot assess the impact of each factor on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Actual results could differ materially from those currently anticipated due to a number of factors.

COMPANY CONTACT:

Rui Zhang
CASI Pharmaceuticals, Inc.
Phone: 240.864.2643
Email: ir@casipharmaceuticals.com

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SOURCE CASI Pharmaceuticals, Inc.

FAQ

What is CASI Pharmaceuticals' new drug application for CNCT19?

CASI Pharmaceuticals' new drug application for CNCT19 targets relapsed/refractory B-Cell Acute Lymphoblastic Leukemia and has been accepted by the NMPA.

What are the efficacy results of CNCT19?

In the registrational trial, CNCT19 achieved an 82.1% overall response rate with a 100% drug production success rate.

When was the NDA for CNCT19 submitted?

The NDA for CNCT19 was accepted by the NMPA on December 15, 2022.

What are the common adverse events related to CNCT19?

Common adverse events for CNCT19 include cytokine release syndrome and neurotoxicity.

What significance does the NDA acceptance of CNCT19 hold?

The NDA acceptance represents a major milestone for CASI Pharmaceuticals and indicates the potential for commercial launch of CNCT19 in China.