CASI PHARMACEUTICALS ANNOUNCES CNCT19 (CD19 CAR-T) GRANTED ORPHAN DRUG DESIGNATION BY THE U.S. FDA

ROCKVILLE, Md. and BEIJING, Jan. 18, 2022 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, announced today, that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its partner Juventas Cell Therapy Ltd (Juventas), for CNCT19, an investigational CD-19 directed CAR-T therapy, for the treatment of patients with Acute Lymphoblastic Leukemia (ALL).

CNCT19 is currently being developed independently by Juventas to meet the urgent clinical needs of patients with hematologic malignancies globally. The National Medical Products Administration (NMPA) has granted CTA approval for CNCT19 in two indications (relapsed/refractory B-All and B-NHL) in Nov. 2019. Currently, the Phase II clinical trials of CNCT19 (for both B-ALL and B-NHL) are in progress. Positive clinical data for adult and pediatric patients with relapsed/refractory B-ALL has been presented at the December 2021 ASH annual meeting, which further demonstrated its safety and efficacy profile. CNCT 19 is expected to be the first domestic CD19 directed CAR-T product in China with independent intellectual property rights.

Dr. Wei-Wu He, CASI's Chairman, and CEO commented, "Our partner Juventas continues to make encouraging progress in developing their CD19 CAR-T therapy. The Orphan Drug Designation, from the FDA, and the Breakthrough Designation status, granted by the China Center of Drug Evaluation (CDE) in December 2020, represent significant milestones that demonstrate our belief that CNCT19's commercialization will not only be successful in China, but potentially on a global scale. CASI has worldwide co-commercial rights of CNCT19, and will start the global commercialization process according to CNCT19's regulatory progress outside China."

About Juventas

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.

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., which is located in Beijing, China. The Company has built a commercial team of more than 100 hematology oncology sales and marketing specialists based in China. More information on CASI is available at www.casipharmaceuticals.com.

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.

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SOURCE CASI Pharmaceuticals, Inc.