CASI (China) Pharmaceuticals Technology Co., Ltd.

~ NMPA approved CB-5339 CTA for Multiple Myeloma indication ~ ~ Expected enrollment to begin in 2023 ~ ROCKVILLE, Md. and BEIJING, Jan. 6, 2023 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, announces that the China National Medical Products Administration (NMPA) has approved the Company's Clinical Trial Application (CTA) for CB-5339, a first-in-class VCP/p97 inhibitor from Cleave Therapeutics. CASI is planning a Phase 1 development program in China of CB-5339 as a single agent to evaluate the PK/safety profile, select the Recommended Phase 2 Dose, and assess early signs of clinical efficacy. The Phase 1 development program is expected to start in 2023. Wei-Wu He, Ph.D., CASI's Chairman, and Chief Executive Officer, commented, "This is exciting news for CASI, as we are now a step closer to make CB-5339 available to patients and healthcare providers across Greater China. CB-5339 represents a promising new agent for selectively targeting VCP/p97 in cancers and is a complementary addition to our growing portfolio of approved and investigational therapies for hematology oncology indications. CASI is responsible for the conduct of the clinical trials in China and will initiate the studies as soon as possible." About CB-5339 (VCP/p97 inhibitor) CB-5339 is an oral second-generation, small molecule VCP/p97 inhibitor, and is currently being evaluated by Cleave Therapeutics in a Phase 1 clinical trial in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and myeloproliferative neoplasms (NCT04402541). VCP/p97 inhibitors exploit molecular features that define cancer cell growth and metabolism. VCP/p97 plays a critical role in protein homeostasis processes such as endoplasmic reticulum associated degradation (ERAD) and chromatin-associated degradation (CAD), as well as the DNA damage response (DDR). These key cellular stress pathways are known to represent sensitivities critical to cancer cell survival. Cellular stress provides an attractive means of targeting non-oncogene addiction as a way to combat tumor heterogeneity and emerging resistance to targeted therapies. Such an approach has the added benefit of broad applicability, not dependent on a single driver mutation and potential efficacy across cancer types. About Cleave Therapeutics Cleave Therapeutics is a clinical-stage biopharmaceutical company focused on VCP/p97 as a novel target in protein homeostasis and cellular stress pathways for therapeutic use in cancer. The privately held company, based in San Francisco, is studying CB-5339, its second-generation, small molecule VCP/p97 inhibitor, in a Phase 1 clinical trial in patients with acute myeloid leukemia, myelodysplastic syndrome and myeloproliferative neoplasms. Cleave investors include 5AM Ventures, Bristol-Myers Squibb, Orbimed, U.S. Venture Partners (USVP), Arcus Ventures, Astellas Venture Management, and Osage University Partners. For additional information, visit . 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 . 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: [email protected] SOURCE CASI Pharmaceuticals, Inc.

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 . 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: [email protected] SOURCE CASI Pharmaceuticals, Inc.

ROCKVILLE, Md. and BEIJIN, Nov. 14, 2022 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, today reported financial results for the third quarter of 2022. Wei-Wu He, Ph.D., CASI's Chairman and Chief Executive Officer, commented, "We are pleased to report $10.2 million in EVOMELA® sales revenue for the third quarter of 2022. This is a 26% of increase compared to the same period last year. Our teams in both China and US delivered strong performance despite the continued COVID lockdown in several major cities in China. Since the third quarter, the COVID-19 pandemic has gradually spread nationwide in China, with many small to medium-sized cities upgrading their control measures. The implementation of these enhanced pandemic control measures is expected to adversely impact our sales activities for the remainder of the year." Dr. He continued, "Advancement, development, and commercialization of the portfolio remains our strategic focus. Through our partner Juventas, the CNCT-19 New Drug Application (NDA) submission to the National Medical Products Administration (NMPA) is on track, and we are in preparation for the anticipated CNCT-19 launch in China. As we continue the development of the regulatory framework for BI-1206 in China, we were pleased with the recent dosing of the first patient in the BI-1206 phase I trial in China. We expect CB-5339 to receive Clinical Trial Application approval from the NMPA in early 2023; Meanwhile, our CID-103's Phase I study continues. We plan to build on the momentum to drive our portfolio forward by executing on several milestones in the quarters ahead." Third Quarter 2022 Financial Highlights Revenues consist primarily of product sales of EVOMELA. Revenue was $10.2 million for the three months ended September 30, 2022, compared to $8.1 million for the three months ended September 30, 2021. Costs of revenues consist primarily of the cost of inventories of EVOMELA and sales-based royalties related to the sale of EVOMELA. Costs of revenues were $4.2 million for the three months ended September 30, 2022, compared to $3.4 million for the three months ended September 30, 2021. Research and development expenses for the three months ended September 30, 2022, were $3.9 million, compared with $2.9 million for the three months ended September 30, 2021. The increase of research and development expenses for the three months periods was mainly due to expense incurred for BI-1206 and CID-103. General and administrative expenses for the three months ended September 30, 2022, were $4.8 million, compared with $5.3 million for the three months ended September 30, 2021. Selling and marketing expenses for the three months ended September 30, 2022, were $3.6 million, compared with $3.4 million for the three months ended September 30, 2021. Acquired in-process R&D expenses for the three months ended September 30, 2022 and 2021, were both nil. As of September 30, 2022, CASI had cash and cash equivalents of $29.2 million. Further information regarding the Company, including its Quarterly Report on Form 10-Q for the quarter ended September 30, 2022, can be found at . 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 majority of the Company's operations are now located in China. 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 is executing its plan to become a biopharmaceutical leader by launching medicines in the greater China market, leveraging its China-based regulatory, clinical 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 . 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, 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 no duty to update forward-looking statements is assumed. Actual results could differ materially from those currently anticipated due to a number of factors, including: the risk that we may be unable to continue as a going concern as a result of our inability to raise sufficient capital for our operational needs; the possibility that we may be delisted from trading on The Nasdaq Capital Market; the volatility in the market price of our common stock; the outbreak of the COVID-19 pandemic and its effects on global markets and supply chains; the risk of substantial dilution of existing stockholders in future stock issuances; the difficulty of executing our business strategy in China; our inability to enter into strategic partnerships for the development, commercialization, manufacturing and distribution of our proposed product candidates or future candidates, including with respect to BI-1206, CB-5339 and CID-103; our lack of experience in manufacturing products and uncertainty about our resources and capabilities to do so on a clinical or commercial scale; risks relating to the commercialization, if any, of our products and proposed products (such as marketing, safety, regulatory, patent, product liability, supply, competition and other risks); our inability to predict when or if our product candidates will be approved for marketing by the U.S. Food and Drug Administration (FDA), National Medical Products Administration (NMPA), or other regulatory authorities; our inability to enter into strategic partnerships for the development, commercialization, manufacturing and distribution of our proposed product candidates or future candidates, including with respect to our partnerships with Juventas and BioInvent; the risks relating to the need for additional capital and the uncertainty of securing additional funding on favorable terms; the risks associated with our product candidates, and the risks associated with our other early-stage products under development; the risk that result in preclinical and clinical models are not necessarily indicative of clinical results; uncertainties relating to preclinical and clinical trials, including delays to the commencement of such trials; our ability to protect our intellectual property rights; our ability to design and implement a development plan for our ANDAs held by CASI Wuxi; the lack of success in the clinical development of any of our products; and our dependence on third parties; the risks related to our dependence on Juventas to conduct the clinical development of CNCT19 and to partner with us to co-market CNCT19; risks related to our dependence on Juventas to ensure the patent protection and prosecution for CNCT19; risks relating to the commercialization, if any, of our proposed products (such as marketing, safety, regulatory, patent, product liability, supply, competition and other risks); risks relating to interests of our largest stockholders and our Chairman and CEO that differ from our other stockholders; and risks related to the development of a new manufacturing facility by CASI Wuxi. Such factors, among others, could have a material adverse effect upon our business, results of operations, and financial condition. We caution readers not to place undue reliance on any forward-looking statements, which only speak as of the date made. Additional information about the factors and risks that could affect our business, financial condition, and results of operations, are contained in our filings with the U.S. Securities and Exchange Commission, which are available at . EVOMELA® is proprietary to Acrotech Biopharma Inc. and its affiliates. COMPANY CONTACT: Rui Zhang CASI Pharmaceuticals, Inc. Phone: 240.864.2643 Email: [email protected] (Financial Table Follows) SOURCE CASI Pharmaceuticals, Inc.