CASI Pharmaceuticals, Inc.

CASI Pharmaceuticals, Inc. operates as a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products primarily in China, the United States, and globally, with a strategic emphasis on hematology oncology, autoimmune, and organ transplant rejection therapeutic areas ¹. The company's business model involves generating revenue primarily from product sales, which are supported by in-licensing agreements for product candidates and distribution agreements with third parties ². A significant portion of its operations is conducted through its Chinese subsidiaries, CASI China and CASI Wuxi, which oversee sales, marketing, medical affairs, regulatory and clinical development, and manufacturing ³.

The company's product portfolio includes several key assets. EVOMELA® (Melphalan for Injection) is approved in China for conditioning treatment prior to stem cell transplantation and as a palliative treatment for multiple myeloma ⁴. FOLOTYN® (Pralatrexate), a dihydrofolate reductase inhibitor, was indicated for relapsed or refractory peripheral T-cell lymphoma (PTCL) and was approved by both the US FDA and China's NMPA ⁵. However, the Import Drug Registration License for FOLOTYN® expired on August 25, 2025, leading to the cessation of its sale in China ⁶. MAFALAN® (generic melphalan) received NMPA marketing approval in May 2025 as the company's first self-manufactured commercial product in China, approved for high-dose conditioning treatment prior to hematopoietic stem cell transplantation in multiple myeloma patients and for palliative treatment of multiple myeloma ⁷. Thiotepa, a chemotherapeutic agent, received NMPA approval in February 2026 for which the company holds exclusive China license and distribution rights ⁸. Additionally, the company distributes YuTuo® (Zimberelimab), a PD-1 inhibitor, in China under a three-year non-exclusive distribution agreement with Guangzhou Gloria Biosciences Co., Ltd., entered into in January 2025 ⁹.

In terms of pipeline candidates, CID-103, a full human IgG1 anti-CD38 monoclonal antibody, is being developed for multiple myeloma, organ transplant rejection, and autoimmune diseases like immune thrombocytopenia (ITP), with exclusive global rights ¹⁰. An IND for CID-103 in ITP was cleared by the US FDA in May 2024, and the first patient was dosed in January 2025 ¹¹. An IND for CID-103 in active and chronic active renal allograft antibody-mediated rejection (AMR) was cleared by the FDA in August 2025, and a CTA for a Phase 1/2 clinical trial in chronic active renal allograft AMR was approved by China NMPA in January 2026 ¹². BI-1206, a novel anti-FcγRIIB antibody, is under an exclusive licensing agreement with BioInvent International AB for development and commercialization in mainland China, Taiwan, Hong Kong, and Macau ¹³. It is being investigated in Phase 1/2 trials for solid tumors and relapsed/refractory non-Hodgkin lymphoma (NHL), with Phase 1 enrollment completed in December 2025 ¹⁴. CNCT19 (Inaticabtagene Autoleucel), an autologous CD19 CAR-T investigational product, received NMPA market approval in November 2023 for relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) in China, for which the company has exclusive worldwide co-commercial and profit-sharing rights with Juventas ¹⁵. CB-5339, an oral second-generation VCP/p97 inhibitor, is being developed for various malignancies, and the company obtained global intellectual property rights in July 2023 ¹⁶.

For the fiscal year ended December 31, 2025, the company reported total revenues of US$20.7 million ¹⁷, a gross profit of US$10.1 million ¹⁸, and a gross margin of 48.6% ¹⁹. Operating expenses totaled US$55.1 million ²⁰, leading to a loss from operations of US$45.0 million ²¹. The net loss for the year was US$48.1 million ²², resulting in a diluted EPS of -$2.34 ²³. Cash and cash equivalents stood at US$5.6 million ²⁴, with net current liabilities of US$36.2 million ²⁵. Net cash used in operating activities was US$20.8 million ²⁶. Total debt includes a long-term borrowing of US$23.8 million ²⁷ and bank borrowings of US$1.0 million ²⁸, along with a convertible note of US$5.0 million ²⁹.

Year-over-year, revenues decreased by 27.3% from US$28.5 million in 2024 to US$20.7 million in 2025 ³⁰. Gross profit decreased from US$11.1 million in 2024 to US$10.1 million in 2025 ³¹, while gross margin improved from 39.1% in 2024 to 48.6% in 2025 ³². Costs of revenues decreased by 39.1% from US$17.4 million in 2024 to US$10.6 million in 2025, primarily due to a US$2.4 million decrease in royalty costs for EVOMELA® and FOLOTYN®, and a US$3.6 million decrease in inventory write-downs ³³. Research and development expenses decreased from US$8.9 million in 2024 to US$6.3 million in 2025 ³⁴, mainly due to decreased expenses for CID-103 (US$0.8 million), six ANDAs sold in 2024 (US$0.7 million), and BI-1206 (US$0.4 million) ³⁵. General and administrative expenses increased from US$23.6 million in 2024 to US$28.3 million in 2025, driven by a US$7.6 million increase in legal fees related to ongoing arbitrations, partially offset by decreases in personnel cost (US$1.8 million) and depreciation cost (US$0.6 million) ³⁶. Selling and marketing expenses slightly increased from US$17.9 million in 2024 to US$18.3 million in 2025 ³⁷. The share of net loss in an equity investee increased from US$1.2 million in 2024 to US$2.2 million in 2025 ³⁸.

Significant operational developments during the period include the NMPA marketing approval for MAFALAN® in May 2025, marking the company's first self-manufactured commercial product ³⁹. The company entered into a three-year non-exclusive distribution agreement for YuTuo® in January 2025 ⁴⁰. The Import Drug Registration License for FOLOTYN® expired on August 25, 2025, leading to the cessation of its sale in China ⁴¹. In May 2025, the company entered into a definitive agreement to sell its two wholly-owned subsidiaries in China and transfer certain regional rights to BI-1206, CID-103 (in Asia excluding Japan), and Thiotepa for an aggregate purchase price of $20.0 million, which includes assumption of up to $20.0 million of indebtedness ⁴². This transaction, subject to certain conditions including the resolution of a judicial freeze on the Target Equity Interests, represents a strategic pivot towards developing CID-103 for organ transplant rejection and autoimmune diseases ⁴³. The company also resolved its dispute with Acrotech regarding the EVOMELA® license through an amicable settlement in April 2026 ⁴⁴. In December 2025, the company issued a convertible note with a principal amount of US$5.0 million to ETP Global III Fund LP, a partnership controlled by Dr. Wei-Wu He ⁴⁵.

The company's strategic outlook is significantly shaped by its recent divestiture of China-based operations and a pivot towards global development of CID-103. Following the definitive equity and assets transfer agreement with Kaixin Pharmaceuticals Inc. in May 2025, the company plans to sell its two wholly-owned subsidiaries in China and transfer certain regional rights to BI-1206 (in China), CID-103 (in Asia excluding Japan), and Thiotepa (in China excluding Hong Kong, Macau and Taiwan) for an aggregate purchase price of $20.0 million, including the assumption of up to $20.0 million of indebtedness ⁴⁶. After this transaction, the company expects to retain rights related to CID-103 (in Japan and non-Asian regions), EVOMELA®, FOLOTYN®, CNCT19, and CB-5339, with a firm commitment to accelerating CID-103 development for organ transplant rejection and autoimmune diseases ⁴⁷.

A major growth area for the company is the accelerated global development of CID-103. The US FDA cleared an IND application for CID-103 in adults with active and chronic active renal allograft antibody-mediated rejection (AMR) in August 2025 ⁴⁸. Subsequently, in January 2026, China NMPA approved a CTA to conduct a Phase 1/2 clinical trial for CID-103 in adults with chronic active renal allograft AMR ⁴⁹. This Phase 1/2 trial is designed as a dose-ranging and safety study to evaluate tolerability and efficacy in this patient population ⁵⁰. The company's investment in this program is intended to provide a runway to fund a Phase 1 study in China for renal allograft AMR, as well as development towards a stable, high-concentration protein solution for subcutaneous formulation ⁵¹.

Another growth area is the commercialization of MAFALAN® (generic melphalan), which received NMPA marketing approval in May 2025 as the company's first self-manufactured commercial product in China ⁵². The company intends to invest time, resources, and efforts in its commercialization in China and, over time, in global markets, focusing on hospital formulary inclusions, physician engagement, and reimbursement opportunities ⁵³. The company also received NMPA approval for Thiotepa in February 2026 and intends to advance and commercialize this product in China ⁵⁴.

Operationally, the company faces a challenging external environment with new generic formulations of melphalan for injection entering the China market ⁵⁵. The company's strategy will shift to generic melphalan products in the future ⁵⁶. The renewal application for FOLOTYN® was not granted, leading to the cessation of its sale in China ⁵⁷. The company's manufacturing facility in Wuxi, which produces MAFALAN®, has a productive capacity of 100,000 vials per year, with current utilization at approximately 30% ⁵⁸. The company expects its operations in China to continue to grow, with a staff of 205 full-time employees, including 128 hematology and oncology sales and marketing specialists ⁵⁹.

Regarding capital allocation, the company had capital expenditures of US$29,000 in 2025 ⁶⁰. In December 2025, the company entered into a convertible note purchase agreement with ETP Global III Fund LP, a partnership controlled by Dr. Wei-Wu He, to issue and sell convertible notes in an aggregate principal amount of US$20 million ⁶¹. This investment is planned to fund the company through a Phase 1 study in China for renal allograft AMR and development of a subcutaneous formulation for CID-103 ⁶². As of the date of the annual report, notes with an aggregate principal amount of US$20 million in four tranches have been issued ⁶³. The convertible notes mature in 36 months and bear interest of 12% per annum ⁶⁴. The company also sold 4,053,836 ordinary shares through an Open Market Sale Agreement for net proceeds of $5.7 million ⁶⁵.

The company explicitly flags several structural headwinds and execution risks. The closing of the transaction to sell its China subsidiaries is subject to certain conditions, including the resolution of a judicial freeze on the Target Equity Interests due to an ongoing arbitration with Juventas, which presents a significant obstacle ⁶⁶. If this transaction cannot be completed, the operation of the current business may be affected ⁶⁷. The company is also involved in arbitration proceedings against Juventas regarding the purported termination of CNCT19 agreements, with a final award expected by June 30, 2026 ⁶⁸. An unfavorable outcome in this arbitration could delay or adversely impact the commercialization of CNCT19 ⁶⁹. Furthermore, the company's recurring operating losses, accumulated deficit of $748.1 million as of December 31, 2025, and net current liabilities of $36.2 million raise substantial doubt about its ability to continue as a going concern ⁷⁰. The company will require additional liquidity to fund operations and cannot assure success in raising additional equity or debt capital ⁷¹. The provision allowing Wuxi LP to request immediate repayment of the long-term borrowing if CASI Wuxi fails to generate revenue for a certain threshold represents a significant liquidity risk, as CASI Wuxi failed to satisfy this threshold in 2024 and 2025 ⁷².

The company faces material risks including recurring operating losses and an accumulated deficit of $748.1 million ⁷³ as of December 31, 2025, raising substantial doubt about its ability to continue as a going concern, exacerbated by net current liabilities of $36.2 million ⁷⁴. The ongoing transaction to sell its China subsidiaries, including the transfer of rights to BI-1206 (in China), CID-103 (in Asia excluding Japan), and Thiotepa (in China excluding Hong Kong, Macau and Taiwan) for an aggregate purchase price of $20.0 million ⁷⁵, is subject to the resolution of a judicial freeze on the Target Equity Interests, posing a significant obstacle to completion ⁷⁶. The company is also engaged in arbitration with Juventas regarding the purported termination of CNCT19 agreements, with a final award expected by June 30, 2026 ⁷⁷, which could adversely impact CNCT19 commercialization. Competition in the melphalan market in China is intense, with one new generic injectable melphalan product entering in 2025 and three additional generic applications under CDE review ⁷⁸, eroding market share for EVOMELA® and MAFALAN®. The Import Drug Registration License for FOLOTYN® expired on August 25, 2025, leading to the cessation of its sale in China ⁷⁹. The company's manufacturing facility in Wuxi, while operational, has limited experience at commercial scale and faces risks related to cGMP compliance, raw material supply chain stability, production yields, costs, and regulatory inspections ⁸⁰. The provision allowing Wuxi LP to request immediate repayment of the long-term borrowing if CASI Wuxi fails to meet revenue thresholds, which it did in 2024 and 2025 ⁸¹, presents a significant liquidity risk. Furthermore, the company's ordinary shares were delisted from Nasdaq on February 23, 2026, and now trade on the OTCQB market under "CASIF" ⁸², which could impair the ability to sell or purchase shares and significantly affect capital raising efforts.

Management's message to shareholders reflects a strategic pivot, emphasizing the divestiture of certain China-based operations to focus on the global development of CID-103 for organ transplant rejection and autoimmune diseases. The company entered into a definitive agreement in May 2025 to sell its two wholly-owned subsidiaries in China and transfer specific regional rights for BI-1206, CID-103 (in Asia excluding Japan), and Thiotepa (in China excluding Hong Kong, Macau and Taiwan) for an aggregate purchase price of $20.0 million, which includes the assumption of up to $20.0 million of indebtedness ⁸³. This initiative is explicitly stated to align with the strategic pivot toward developing CID-103 ⁸⁴. Management remains committed to progressing CID-103 at an accelerated pace, retaining global rights for CID-103 (in Japan and non-Asian regions), EVOMELA®, FOLOTYN®, CNCT19, and CB-5339 ⁸⁵. A key strategic priority is the accelerated global development of CID-103, evidenced by the FDA clearance of an IND for CID-103 in renal allograft AMR in August 2025 and China NMPA's approval of a CTA for a Phase 1/2 clinical trial in the same indication in January 2026 ⁸⁶. Another priority is the commercialization of MAFALAN®, the company's first self-manufactured product in China, and the newly approved Thiotepa ⁸⁷. Management also acknowledges the challenging external environment, including intensified competition from generic melphalan products and the expiration of FOLOTYN®'s Import Drug Registration License, leading to a strategic shift towards generic melphalan products ⁸⁸.