On March 31, 2025 CASI Pharmaceuticals, Inc. (NASDAQ:CASI), ("CASI" or the "Company"), a Cayman incorporated biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported business and financial results for the fourth quarter ended December 31, 2024, and provided an update on key highlights for 2024 (Press release, CASI Pharmaceuticals, MAR 31, 2025, https://feeds.issuerdirect.com/news-release.html?newsid=5642877492672387&symbol=CASI [SID1234652235]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "2024 was a transformative year for CASI as we strategically pivoted our company toward the development of CID-103 for organ transplant rejection and autoimmune disease. CID-103 is an anti-CD38 antibody with the potential to be a best-in-class treatment for a myriad of autoimmune diseases as well as antibody mediated rejection (AMR). We are encouraged by our clinical progress, achieving a milestone with the first Immune Thrombocytopenia (ITP) patient dosed in January 2025. Simultaneously, we are diligently working towards resolution of the FDA renal allograft AMR clinical hold."

Dr. He continued, "As we advance into 2025, we remain firmly committed to progressing CID-103 at an accelerated pace. We expect to reach multiple important milestones this year. Our focused approach and prudent capital allocation position CASI to deliver sustainable long-term value creation for both patients and shareholders."

Key Business Highlights

CASI reported the fourth quarter revenue of $13.4 million and full-year revenue of $28.5 million.

Received a proposal with respect to the acquisition of CASI’s China business from Dr. Wei-Wu He on June 21, 2024.

Completed a $15.0 million private placement financing with leading healthcare investors including Venrock Healthcare Capital Partners, Foresite Capital, Panacea Venture and Dr. Wei-Wu He, on July 15, 2024.

Pipeline and Program Updates

Received FDA IND clearance of ITP on May 15, 2024.

Received CTA approval from China’s NMPA on ITP on October 24, 2024.

First patient dosed in Phase 1/2 clinical trial in ITP on January 3, 2025. Patient enrollment and treatment continues.

Upcoming Milestones

Anticipate FDA feedback on clinical hold for AMR in Q2, 2025.

Target IND submission for Aplastic Anemia in Q2, 2025.

Report the interim data from the ongoing ITP phase 1 study in mid-2025.

Anticipate finalizing the equity transfer agreement regarding Precision Autoimmune Therapeutics ("PAT") in Q2 2025. Upon completion of this transaction, CASI will have the sole global ownership of all indications in CID-103.

Product and Pipeline Highlights

CID-103 (Anti-CD38 antibody)

CID 103 is a full 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, and which we have exclusive global rights. CID 103 is being developed for the treatment of patients with chronic Immune Thrombocytopenia (ITP), renal allograft antibody mediated rejection (AMR). In May 2024, we announced the clearance of IND application with the US FDA for the initiation of a phase 1/2 study of CID-103 in adults with ITP. In October 2024, the Center for Drug Evaluation (CDE) approved the Clinical Trial Application (CTA) for a phase 1/2 study of CID-103 in patients with chronic ITP in China. The Chinese ITP study is part of the global development program which has been approved by the US FDA in May 2024. In January 2025, CASI announced the first patient was enrolled and dosed in the ITP clinical study. Furthermore, we are making steady progress towards resolution of the FDA clinical hold.

EVOMELA (melphalan for injection)

On December 3, 2018, CASI received the NMPA approval for importation, marketing and sales in China for EVOMELA , and on August 12, 2019, CASI announced the commercial launch of EVOMELA in China. Prior to EVOMELA’s entry into the Chinese market, an average of 800 stem cell transplants per year were conducted in the multiple myeloma (MM) treatment setting. Following EVOMELA’s launch in August of 2019, CASI worked closely with KOLs to improve market awareness and expedite adoption in the Chinese market. In 2023, nearly 10,000 patients were treated with EVOMELA. In 2024, the launch of an undifferentiated generic formulation of melphalan for injection product by a Chinese domestic manufacture has presented challenges, resulting in a decline of EVOMELA sales.

FOLOTYN (Pralatrexate)

On July 31, 2023, CASI entered into a tripartite assignment agreement with Mundipharma International Corporation Limited ("MICL"), Mundipharma Medical Company (MMCo), and Acrotech Biopharma Inc. (Acrotech) for the commercialization of FOLOTYN (Pralatrexate) in China. FOLOTYN (Pralatrexate) is a dihydrofolate reductase inhibitor indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This product was approved by both the FDA and China’s NMPA for PTCL. CASI announced the first patient was dosed with FOLOTYN in China on February 15, 2024.

Fourth Quarter and Full-Year 2024 Financial Results

Revenue for the fourth quarter of 2024 was $13.4 million, representing a 94% increase compared to $6.9 million in the same period last year. The quarterly growth reflects a successful execution of commercial strategy implemented in the second half of the year. Full-year revenue was $28.5 million, compared to $33.9 million in 2023, a decrease of 16%. The annual performance was impacted by intensified competition following the market entry of domestically produced injectable melphalan, which created pricing pressure throughout the year.

R&D expenses for the fourth quarter was $3.7 million, a 61% increase from $2.3 million in the same period last year. The increase is primarily for the development of CID-103, supporting the company’s focused pivot toward opportunities in organ transplant rejection and autoimmune indications. Full-year R&D expenses was $8.9 million, compared to $9.9 million in 2023, representing a decrease of 10%. The reduction primarily resulted from lower amortization expenses following the decision to write off generic portfolio at the end of 2023.

G&A expenses for the fourth quarter of 2024 was $7.1 million, compared to $6.4 million in the same period in 2023, a 11% increase. This quarter-over-quarter increase is primarily attributable to legal expenses associated with ongoing arbitration proceedings with Juventas. Full-year G&A expenses was $23.6 million, compared to $25.4 million in 2023, representing a decrease of 7%. This year-over-year improvement demonstrates the effectiveness of our operational efficiency initiatives and cost control measures, reinforcing our commitment to disciplined financial management.

Net loss for the year ended December 31, 2024 was $39.3 million compared to $26.3 million for the year ended December 31, 2023.

As of December 31, 2024, CASI had cash and cash equivalents of $13.5 million compared to cash and cash equivalents plus short term investments of $29.1 million as of December 31, 2023.

Further information regarding the Company, including its Annual Report on Form 20-F for the year ended December 31, 2024, can be found at www.casipharmaceuticals.com. The Company will provide a hard copy of its annual report containing the audited consolidated financial statements, free of charge, to its shareholders upon request. Requests should be directed to Investor Relations Department, CASI Pharmaceuticals, Inc., Rm 1701-1702, China Central Office Tower 1, No.81 Jianguo Road Chaoyang District, Beijing, 100025, China.