casi

Company Profile: CASI Pharmaceuticals, Inc.

Background

CASI Pharmaceuticals, Inc., established in 1991 as EntreMed, Inc., is a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products. In 2014, the company rebranded to CASI Pharmaceuticals to emphasize its commitment to combating cancer through advanced science and innovation, particularly in the Greater China market. The company's mission is to address unmet medical needs in oncology, hematology, and autoimmune diseases by leveraging its global drug development expertise and China-based regulatory and commercial competencies.

Key Strategic Focus

CASI Pharmaceuticals' strategic focus centers on:

• Advancing Clinical Development: The company is dedicated to the development of CID-103, a fully human anti-CD38 monoclonal antibody, targeting conditions such as immune thrombocytopenia (ITP) and renal allograft antibody-mediated rejection (AMR). In May 2024, CASI received FDA Investigational New Drug (IND) clearance for CID-103 in ITP, with the first patient dosed in a Phase 1/2 study in January 2025. The company plans to initiate a Phase 1 study for CID-103 in renal AMR in the first quarter of 2026.

• Commercializing Existing Products: CASI continues to commercialize EVOMELA® (melphalan for injection) and FOLOTYN® (pralatrexate) in regions outside of China, including Japan and non-Asian markets. The company also retains rights to CNCT19, an autologous CD19 CAR-T cell therapy, and CB-5339, a novel VCP/p97 inhibitor, in certain territories.

• Strategic Divestiture: In May 2025, CASI announced the divestiture of its China-based operations and assets, including commercial products like EVOMELA and FOLOTYN, to Kaixin Pharmaceuticals Inc. This strategic move aims to streamline operations and focus resources on the development of CID-103.

Financials and Funding

As of September 30, 2025, CASI Pharmaceuticals reported cash and cash equivalents of $4.7 million, with a net loss of $10.9 million for the third quarter of 2025. The company is actively seeking additional capital to support its operations and strategic initiatives. In December 2025, CASI announced an agreement to receive $20 million in funding from Emerging Technology Partners, LLC, which is expected to bolster its financial position and support the advancement of its pipeline programs.

Pipeline Development

CASI Pharmaceuticals' pipeline includes:

• CID-103: A fully human anti-CD38 monoclonal antibody in Phase 1/2 development for ITP, with plans to initiate a Phase 1 study in renal AMR in early 2026.

• CNCT19: An autologous CD19 CAR-T cell therapy for B-cell acute lymphoblastic leukemia (B-ALL) and B-cell non-Hodgkin lymphoma (B-NHL), with a New Drug Application accepted by China's National Medical Products Administration (NMPA) in December 2022.

• BI-1206: A novel anti-FcγRIIB antibody in Phase I trials in combination with rituximab for relapsed/refractory non-Hodgkin lymphoma.

• CB-5339: A VCP/p97 inhibitor in Phase I trials for acute myeloid leukemia and myelodysplastic syndrome.

Technological Platform and Innovation

CASI Pharmaceuticals leverages several proprietary technologies and scientific methodologies:

• CID-103: A fully human anti-CD38 monoclonal antibody designed to deplete persistent plasma cells, offering a differentiated profile for treating organ rejection and autoimmune diseases.

• CAR-T Cell Therapy: CNCT19 represents the company's commitment to innovative immuno-oncology treatments, utilizing autologous T cells engineered to target CD19-positive malignancies.

• VCP/p97 Inhibition: CB-5339 targets the VCP/p97 protein, involved in protein homeostasis and DNA damage response, aiming to disrupt cellular stress pathways in cancer cells.

Leadership Team

• Wei-Wu He, Ph.D.: Chairman and Chief Executive Officer. Dr. He has extensive experience in the pharmaceutical industry, leading CASI Pharmaceuticals since its rebranding in 2014.

• David Cory: Chief Executive Officer. Appointed in July 2025, Mr. Cory brings over 30 years of operational management experience in the biopharmaceutical industry, having held executive positions in both private and public companies.

• Deanna Qian: Chief Financial Officer. Ms. Qian assumed the role in May 2025, continuing her responsibilities as Global Controller.

Competitor Profile

CASI Pharmaceuticals operates in a competitive landscape, facing challenges from both large pharmaceutical companies and emerging biotech firms. Key competitors include:

• Pfizer, Inc.: A global pharmaceutical leader with a broad oncology portfolio, competing in similar therapeutic areas.

• Novartis AG: A multinational company with a strong focus on oncology and hematology, presenting competition in related markets.

• Amgen Inc.: A biotechnology firm specializing in novel therapeutics, including oncology treatments, competing in overlapping therapeutic areas.

• Gilead Sciences, Inc.: Known for antiviral drugs, Gilead also invests in oncology and hematology, posing competition in these sectors.

• BeiGene, Ltd.: A rapidly emerging biotech company specializing in oncology, particularly in the Chinese market, representing significant competition.

Strategic Collaborations and Partnerships

CASI Pharmaceuticals has engaged in strategic collaborations to enhance its pipeline and market presence:

• Juventas Biotechnology (Tianjin) Co., Ltd.: Partnered for the development of CNCT19, with a New Drug Application accepted by NMPA in December 2022.

• Precision Autoimmune Therapeutics Co., Ltd. (PAT): Entered into a sub-license agreement for CID-103, with CASI holding a 15% equity stake in PAT.

Operational Insights

CASI Pharmaceuticals' strategic divestiture of its China-based operations aims to streamline its focus on high-potential pipeline assets, particularly CID-103. This move is intended to reduce operational complexity and allocate resources more effectively toward the development of its lead asset.

Strategic Opportunities and Future Directions

The company's strategic roadmap includes:

• Advancing CID-103: Focusing on the development of CID-103 for ITP and renal AMR, with plans to initiate a Phase 1 study in renal AMR in early 2026.

• Expanding Global Presence: Leveraging its partnerships and retained product rights to expand its footprint in non-Asian markets.

• Enhancing Financial Position: Utilizing proceeds from strategic divestitures and partnerships to strengthen its financial position and support ongoing and future development programs.