CASI Announces First Patient Dosed in Phase 1/2 Clinical Trial of CID-103 in Immune Thrombocytopenia

Rhea-AI Summary

CASI Pharmaceuticals (Nasdaq: CASI) has announced the dosing of the first patient in its Phase 1/2 clinical trial evaluating CID-103 for chronic Immune Thrombocytopenia (ITP) in China. CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody that targets a unique epitope and has shown promising preclinical efficacy and safety compared to other anti-CD38 antibodies.

The trial aims to assess the safety and tolerability of CID-103 in adult ITP patients. According to Dr. Wei-Wu He, CASI's Chairman and CEO, this milestone represents significant progress for both the CID-103 program and patients with chronic ITP who currently have treatment options. The study is designed to provide important safety and dose-response data, which could potentially guide CID-103's development for additional autoimmune indications with unmet medical needs.

Positive

• Initiation of Phase 1/2 clinical trial for CID-103
• Potential expansion into multiple autoimmune indications
• Promising preclinical efficacy and safety profile

Negative

• Early-stage clinical development with uncertain outcomes
• Competition from existing anti-CD38 antibodies in the market

Insights

Clinical Research Analyst

The initiation of CID-103's Phase 1/2 trial represents a strategic advancement in CASI's clinical pipeline. The study targeting Immune Thrombocytopenia (ITP) addresses a significant market opportunity in autoimmune disorders. The CD38-targeting approach has proven commercially successful, as evidenced by Darzalex (daratumumab), which generates over $6 billion in annual sales for multiple myeloma.

The trial's design focusing on both safety and dose-response optimization suggests a well-planned development strategy. CID-103's unique epitope binding characteristic could potentially differentiate it from existing anti-CD38 antibodies, offering improved efficacy or safety profiles. The expansion potential into other autoimmune indications significantly enhances the drug's commercial prospects, though early-stage trial results will be important for validating this approach.

Given CASI's market cap of $44.7 million, successful progression through clinical phases could trigger substantial value inflection points. However, investors should note that Phase 1/2 trials typically take 12-18 months to complete, with additional time needed for data analysis and regulatory discussions.

Biotech Investment Specialist

The advancement of CID-103 into clinical trials marks a pivotal development for CASI's pipeline valuation. The ITP market, valued at approximately $2 billion globally, presents a substantial commercial opportunity. CID-103's potential competitive advantages through its novel epitope targeting mechanism could position it favorably against existing treatments.

For a micro-cap biotech company, initiating a Phase 1/2 trial represents a critical value-creation milestone. The study's dual-phase design is cost-efficient, potentially accelerating the development timeline. The company's focus on the Chinese market, where regulatory pathways can be more streamlined, may enable faster development compared to US/EU markets.

While this news is fundamentally positive, CASI's current market capitalization suggests the market is not fully pricing in CID-103's potential. Early clinical success could trigger partnership interests from larger pharmaceutical companies, particularly those looking to expand their autoimmune disease portfolios in the Asian market.

BEIJING, CHINA / ACCESSWIRE / January 6, 2025 / CASI Pharmaceuticals, Inc. (Nasdaq:CASI), a biopharmaceutical company specializing in the development and commercialization of innovative therapeutic and pharmaceutical products announced that the first patient has been dosed in the Phase 1/2 trial to evaluate the safety and tolerability of CID-103 in adult patients with chronic Immune Thrombocytopenia (ITP) in China.

"Dosing the first patient in our phase 1/2 study marks a significant milestone for both CID-103 program and chronic ITP patients who have limited treatment options," said Dr. Wei-Wu He, Chairman and CEO of CASI, "The rapid execution underscores our commitment to accelerating clinical development. This phase 1/2 study is designed to deliver important safety and dose-response data, providing valuable insights to guide the future development of CID-103 for additional autoimmune indications with significant unmet medical needs."

CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody recognizing a unique epitope that has demonstrated encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies.

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 focus on hematology oncology therapeutics and therapeutics for organ transplant rejection and autoimmune disease, 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. More information on CASI is available at www.casipharmaceuticals.com.

CASI 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 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: uncertainties related to the non-binding proposal to acquire the Company's certain business operations in China; 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 U.S. Food and Drug Administration, European Medicines Agency, PRC National Medical Products Administration, or other regulatory authorities; our inability to receive approval for renewal of license of our existing products; 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 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; the risk related to the Company's ongoing development of and regulatory application for CID-103 with respect to the treatment of antibody-mediated rejection for organ transplant and the license arrangements of CID-103; 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. We caution readers not to place undue reliance on any forward-looking statements contained herein.

EVOMELA^® is proprietary to Acrotech Biopharma Inc. and its affiliates. FOLOTYN^® is proprietary to Acrotech Biopharma Inc and its affiliates. Please refer to the Company's earlier SEC filing with respect to Evomela® for further information.

Contact Information

Rui Zhang
ir@casipharmaceuticals.com
240.864.2643

SOURCE: CASI Pharmaceuticals

View the original press release on accesswire.com

FAQ

What is the purpose of CASI's Phase 1/2 trial for CID-103?

The trial aims to evaluate the safety and tolerability of CID-103 in adult patients with chronic Immune Thrombocytopenia (ITP) in China.

What makes CID-103 different from other anti-CD38 antibodies?

CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody that recognizes a unique epitope and has demonstrated encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies.

When did CASI begin dosing patients in the CID-103 Phase 1/2 trial?

CASI announced the first patient dosing on January 6, 2025.

What are the potential therapeutic applications for CASI's CID-103?

Beyond ITP, CID-103 could potentially be developed for additional autoimmune indications with significant unmet medical needs.

What stage of development is CASI's CID-103 currently in?

CID-103 is currently in Phase 1/2 clinical trials, with the first patient recently dosed in China.