Isatuximab-IRFC

BEIJING, June 26, 2024 /PRNewswire/ -- CASI Pharmaceuticals, Inc. (Nasdaq: CASI) ("CASI" or the "Company"), a biopharmaceutical company specializing in the development and commercialization of innovative therapeutic and pharmaceutical products, announced today that the Company is planning to submit an Investigational New Drug ("IND") application to the U.S. Food and Drug Administration ("FDA") for CID-103 for the treatment of antibody-mediated rejection ("AMR") in kidney transplant recipients by the end of 2024. CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody recognizing a unique epitope that has demonstrated an encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies. The Company believes that with the proceeds from the previously announced private placement financing, together with its existing cash and cash equivalents, it will have sufficient capital to complete the Phase II clinical trial for AMR. CASI also announced that its board of directors (the "Board") received a preliminary non-binding proposal letter (the "Proposal Letter") dated June 21, 2024, from Dr. Wei-Wu He, Chairman of the Board and CEO of the Company, to acquire the entire business operations of the Company in China and all license-in, distribution and related rights in Asia (excluding Japan) related to all of the Company's pipeline products, including but not limited to EVOMELA®, FOLOTYN®, CNCT19, BI-1206, CB-5339,CID-103 and Thiotepa, for an aggregate purchase price of $40.0 million, which shall include assumption of up to $20.0 million of indebtedness of the Company (the "Proposed Transaction"). On June 25, the Board has formed a special committee comprised solely of incumbent independent directors (the "Special Committee") to evaluate the transaction contemplated under the Proposal Letter and such other strategic and business alternatives available to the Company in respect of the Company's business operations in China. The Board and the Special Committee caution the Company's shareholders and others considering trading the Company's securities that no decisions have been made with respect to the Proposed Transaction or any alternative strategic option that the Company may pursue. There can be no assurance that any definitive offer will be received, that any definitive agreement will be executed relating to the Proposed Transaction or that any other transaction will be approved or consummated. The Company does not undertake any obligation to provide any updates with respect to any transaction, except as required under applicable law. About CASI Pharmaceuticals CASI Pharmaceuticals, Inc. is a 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. Forward-Looking Statements This announcement contains forward-looking statements. These statements are made under the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements can be identified by terminology such as "will," "expects," "anticipates," "future," "intends," "plans," "believes," "estimates," "confident" and similar statements. Among other things, the business outlook and quotations from management in this announcement, as well as the Company's strategic and operational plans, contain forward-looking statements. The Company may also make written or oral forward-looking statements in its periodic reports to the U.S. Securities and Exchange Commission (the "SEC"), in in its annual report to shareholders, in press releases and other written materials and in oral statements made by its officers, directors or employees to third parties. Statements that are not historical facts, including statements about the Company's beliefs and expectations, are forward-looking statements. Forward-looking statements involve inherent risks and uncertainties. A number of factors could cause actual results to differ materially from those contained in any forward-looking statement, including but not limited to the following: 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 if we fail to satisfy applicable continued listing standards; the volatility in the market price of our ordinary shares; the risk of substantial dilution of existing shareholders in future share issuances; the difficulty of executing our business strategy on a global basis including China; our inability to enter into strategic partnerships for the development, commercialization, manufacturing and distribution of our proposed product candidates or future candidates; legal or regulatory developments in China that adversely affect our ability to operate in China, 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 FDA, European Medicines Agency, PRC National Medical Products Administration, 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; 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; 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 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 shareholder and our Chairman and CEO that differ from our other shareholders; and risks related to the development of a new manufacturing facility by CASI Pharmaceuticals (Wuxi) Co., Ltd. Further information regarding these and other risks is included in the Company's filings with the SEC. All information provided herein is as of the date of this announcement, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law. COMPANY CONTACT: Rui Zhang CASI Pharmaceuticals, Inc. 240.864.2643 [email protected] SOURCE CASI Pharmaceuticals

OSAKA, Japan & CAMBRIDGE, MA, USA I June 22, 2024 I Takeda ( TSE:4502/NYSE:TAK ) today presented positive results from its Phase 2b, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP), a rare immune-mediated bleeding disorder. ITP is characterized by the accelerated destruction of platelets in blood, resulting in a decreased platelet count and an increase of bleeding that can be debilitating. These data (Abstract #LB 01.1) were presented at the oral Late-Breakthrough Session at the 32 nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Bangkok, Thailand. Takeda plans to initiate a global Phase 3 trial of mezagitamab in patients with ITP in the second half of FY2024. The TAK-079-1004 trial ( NCT04278924) evaluated three different doses of subcutaneous mezagitamab (100mg, 300mg and 600mg) versus placebo, given once weekly for eight weeks in patients with chronic or persistent primary ITP, followed by >8 weeks of safety follow-up. The primary endpoint is the percentage of patients with at least one Grade 3 or higher treatment emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events (AEs) leading to mezagitamab discontinuation. 2 Secondary endpoints included platelet response, complete platelet response, clinically meaningful platelet response, and hemostatic platelet response. 1,2 The Phase 2b trial results demonstrated that mezagitamab treatment improved platelet response compared to placebo, across all three dose levels of mezagitamab tested. Patients treated with mezagitamab showed rapid and sustained increases in platelet counts (above the 50,000/μL therapeutic threshold) 4 , that persisted eight weeks after the last dose through to Week 16, illustrating the rapid and post-therapy effects of mezagitamab on platelet response. 1 “Despite treatment with currently available therapies, there is still a significant disease burden and need for a disease modifying treatment that people living with ITP can tolerate,” said David Kuter, M.D., D.Phil., a leading expert in ITP and study presenter at the ISTH oral Late-Breakthrough Session. “These Phase 2b trial results are especially encouraging because they show mezagitamab’s favorable efficacy and safety profile – setting the stage for the generation of additional clinical evidence for this anti-CD38 monoclonal antibody with best-in-class potential for efficacy in ITP.” In this study, mezagitamab had a favorable safety profile in patients with ITP, with no new safety signals and consistent with prior studies of mezagitamab. 1 The rates of TEAEs leading to discontinuation, Grade >3 TEAEs, and SAEs, between the mezagitamab dose groups combined versus placebo were 14.3% versus 0%, 17.9% versus 23.1%, and 14.3% versus 7.7% respectively. 1 “It is a privilege to have these Phase 2b mezagitamab results selected for presentation as a late-breaking abstract at the ISTH Congress,” said Obi Umeh, M.D., M.Sc., Vice President, Franchise Global Program Leader at Takeda. “Based on these results, we plan to initiate a Phase 3 study of mezagitamab in ITP in the second half of FY2024, further underscoring our goal to develop transformative treatments in therapeutic areas with high unmet patient needs.” About Mezagitamab Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb), with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells), resulting in their depletion. Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and to restore platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP and Fast Track Designation for treatment of chronic/persistent ITP from the U.S. Food and Drug Administration. Mezagitamab is an investigational compound that has not been approved for use by any regulatory authority. About the Mezagitamab Phase 2b Trial in ITP The data presented at the ISTH oral Late-Breakthrough Session are from a pre-specified interim analysis of the Phase 2b trial, a randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, and efficacy of mezagitamab in patients with persistent or chronic primary ITP. The trial had two parts: 25 participants were randomized (1:1:1) to mezagitamab 100mg or 300mg, or placebo in Part A while 16 participants were randomized (2:1) to mezagitamab 600mg or placebo in Part B. Participants received once weekly subcutaneous mezagitamab or placebo for 8 doses, followed by ≥8 weeks of safety follow-up. 1 The primary endpoint is the percentage of patients with TEAEs including Grade 3 or higher events, SAEs and AEs leading to mezagitamab discontinuation. Secondary efficacy endpoints include and are defined as: platelet response (a platelet count ≥50,000/μL and ≥20,000/μL above baseline); complete platelet response (a platelet count ≥100,000/μL); clinically meaningful platelet response (a platelet count ≥20,000/μL above baseline); and hemostatic platelet response (participants with a baseline platelet count of <15,000/μL who achieve a platelet count of ≥30,000/μL and ≥20,000/μL above baseline). 2 For all secondary efficacy endpoints, platelet counts must be achieved on at least two visits without a dosing period-permitted rescue treatment in the previous four weeks and without any other previous rescue therapy. 2 About ITP ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood components responsible for preventing or stopping bleeding. It is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening. The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable. 3,4 About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com . References: SOURCE: Takeda Pharmaceutical Co